A Phase 1/2 interventional study of Pelabresib and Ruxolitinib in Myelofibrosis, Leukemia, Myelocytic, Acute and Myelodysplastic/Myeloproliferative Neoplasm, sponsored by Constellation Pharmaceuticals. Completed at 48 sites in 9 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-06-04.
Sponsored by Constellation Pharmaceuticals · Phase 1/2, Interventional, and Treatment
Phase 1 Part: This was an open-label, sequential dose escalation study of pelabresib (CPI-0610) in patients who had previously been treated for Acute Leukemia, Myelodysplastic/Myeloproliferative Neoplasms.
Phase 2 Part: This was an open-label study of pelabresib (CPI-0610), administered with and without Ruxolitinib, in patients diagnosed with Myeloproliferative Neoplasms (Myelofibrosis and Essential Thrombocythemia).
Pelabresib (CPI-0610) was a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
625 studies on the registry are indexed under Primary Myelofibrosis; 124 are open to participants now.
This study's enrollment of 336 is above the median of 43 across 489 interventional studies indexed under Primary Myelofibrosis.
Browse Primary Myelofibrosis studies →Constellation Pharmaceuticals is the lead sponsor of 7 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Phase I (Dose Escalation) - Inclusion and Exclusion Criteria:
Inclusion Criteria (Phase I):
Diagnosis: or cytologically confirmed diagnosis of one of the following hematologic :
Organ Function:
Hematology (MF only):
Other:
Exclusion Criteria (Phase I):
Phase II (Expansion) - Inclusion \& Exclusion Criteria:
Inclusion Criteria (Phase II):
MF Arms (Prior JAKi, Add-on JAKi, JAKi Naïve)
Platelets:
Treatment History:
ET Arm (High-Risk ET)
High-Risk: At least one of:
Exclusion Criteria (Phase II)
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients were enrolled in sequential cohorts (acute leukemia, including acute myelogenous leukemia (AML), acute lymphocytic leukemia (ALL), and acute undifferentiated or biphenotypic leukemia; chronic myelogenous leukemia (CML) in blast crisis; myelodysplastic syndrome (MDS); myelodysplastic/myeloproliferative neoplasms (MDS/MPN); or myelofibrosis (MF)) and received escalating doses of pelabresib (CPI-0610).
Drug: Pelabresib
* Cohort 1A: Was open to patients with MF who were Transfusion Dependent (TD) and who had previously been treated with a JAKi and were intolerant, resistant, refractory, or had lost response to the JAKi, or were ineligible to be treated with a JAKi (pelabresib (CPI-0610) alone). * Cohort 1B: Was open to patients with MF who were not TD and who had previously been treated with a JAKi and were intolerant, resistant, refractory, or had lost response to the JAKi, or were ineligible to be treated with a JAKi (pelabresib (CPI-0610) alone).
Drug: Pelabresib
* Cohort 2A: Was open to patients with MF who were Transfusion Dependent (TD) and were taking ruxolitinib but had disease that was not adequately controlled by ruxolitinib (pelabresib (CPI-0610) + Ruxolitinib). * Cohort 2B: Was open to patients with MF who were not TD and were taking ruxolitinib but had disease that was not adequately controlled by ruxolitinib (pelabresib (CPI-0610) + Ruxolitinib).
Drug: Pelabresib · Drug: Ruxolitinib
Was open to patients with MF who had not previously received a JAKi (pelabresib (CPI-0610) + Ruxolitinib).
Drug: Pelabresib · Drug: Ruxolitinib
Was open to high-risk patients with ET who were resistant or intolerant to hydroxyurea (HU) (pelabresib (CPI-0610) alone).
Drug: Pelabresib
CPI-0610 was administered orally once daily for 14 consecutive days, followed by a 7-day break (1 cycle = 21 days)
Also known as: CPI-0610, DAK539
Ruxolitinib was given orally, twice daily (BID), on a continuous basis for 21 consecutive days of each 21-day cycle.
Phase 1: Frequency of Dose-limiting Toxicities (DLTs)
A dose-limiting toxicity (DLT) was defined as an adverse event or abnormal laboratory value assessed by the Investigator as unrelated to disease progression, intercurrent illness, or concomitant medications that occurred within the first cycle of treatment (21-day cycle) with pelabresib (CPI-0610), and that met any of the criteria specified in the protocol.
Time frame: Up to 21 days
Phase 2 (Cohorts 1B, 2B, and Arm 3): Number of Participants With Splenic Response Rate (SVR35) at Week 24
Splenic Response Rate (SVR35) at Week 24 was defined as the proportion of participants who demonstrated a reduction of at least 35% in spleen size from baseline, as measured by imaging techniques (MRI or CT), following 24 weeks of treatment.
Time frame: Week 24 (Cycle 9 Day 1)
Phase 2 (Cohorts 1A and 2A): Number of Participants Enrolled as Transfusion Dependent (TD) With Conversion Rate From Red Blood Cell (RBC) Transfusion Dependence (TD) to Transfusion Independence (TI)
Conversion rate was defined as the proportion of participants who converted from transfusion dependence (TD) to transfusion independence (TI). TD was characterized by receiving an average of at least 2 units of red blood cell (RBC) transfusions per month-amounting to a minimum of 6 units over the 12 weeks prior to enrollment-while TI was defined as the absence of RBC transfusions during any consecutive 12-week period.
Time frame: Any 12 consecutive weeks (rolling window) during active treatment with pelabresib (CPI-0610), from first dose through treatment discontinuation (up to approximately 8 years for Phase II)
Phase 2 (Arm 4): Number of Participants With Complete Hematological Response (CHR) Rate
Complete Hematological Response (CHR) Rate was defined as the proportion of participants who fulfilled the criteria for CHR, based on the modified European LeukemiaNet (ELN) guidelines (Barosi et al 2009): platelet count ≤400 × 10⁹/L, white blood cell (WBC) count ≤10 × 10⁹/L, confirmation of laboratory values after one treatment cycle, and normal spleen size determined by palpation or imaging.
Time frame: Over 2 consecutive cycles (rolling window) (1 cycle = 21 days)
Phase 1: Number of Adverse Events and Serious Adverse Events as Assessed by CTCAE Criteria
The distribution of adverse events was performed through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters. Treatment-emergent adverse events (TEAEs) in this study were defined as events that began after the first dose of study treatment and continued until 30 days after the last dose, or events that were present prior to the first dose and increased in severity based on preferred term within 30 days following the last dose of pelabresib (CPI-0610).
Time frame: Up to approximately 6 months
Phase 2 (All Arms): Number of Adverse Events and Serious Adverse Events as Assessed by CTCAE Criteria
The distribution of adverse events was performed through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters. Treatment-emergent adverse events (TEAEs) in this study were defined as events that began after the first dose of study treatment and continued until 30 days after the last dose, or events that were present prior to the first dose and increased in severity based on preferred term within 30 days following the last dose of pelabresib (CPI-0610).
Time frame: Up to approximately 387 weeks
Phase 2 (All Arms): Symptom Improvement From the Patient Global Impression of Change (PGIC) at 12 and 24 Weeks
The Patient Global Impression of Change (PGIC) was a single-question, patient-reported assessment that asked individuals to rate their overall change in myeloproliferative neoplasm (MPN) symptoms since starting study treatment. The participants selected one of seven options, ranging from 'Very much improved' to 'Very much worse'. 'No Change from Baseline' indicated stable symptoms (no improvement or worsening), negative change from Baseline indicated a reduction in symptom severity (improvement), and positive change from Baseline indicated an increase in symptom severity (worsening).
Time frame: Week 12 (Cycle 5 Day 1), Week 24 weeks (Cycle 9 Day 1)
Phase 2 (Arms 1, 2 and 3): Percent Change From Baseline in Total Symptom Score (TSS) From the Myelofibrosis Symptom Assessment Form (MFSAF v4.0) at 12 and 24 Weeks
The MFSAF (Myelofibrosis Symptom Assessment Form) was completed by participants every day for 7 days before Day 1 of each treatment cycle, including the 7 days before starting Cycle 1. It used a 24-hour recall format, asking participants to rate the worst severity of seven symptoms (fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain) during the past 24 hours. Each symptom was rated on a scale from 0 (Absent) to 10 (Worst Imaginable). The Total Symptom Score (TSS) was the sum of 7 symptoms (range: 0-70). 'No Change from Baseline' indicated stable symptoms (no improvement or worsening), negative change from Baseline indicated a reduction in symptom severity (improvement), and positive change from Baseline indicated an increase in symptom severity (worsening).
Time frame: Baseline, Week 12 (Cycle 5 Day 1), Week 24 (Cycle 9 Day 1)
Phase 2 (Arms 1, 2 and 3): Number of Participants Who Achieved a ≥ 50% Reduction in Total Symptom Score (TSS) at 12 and 24 Weeks
The proportion of study participants who experienced a reduction of at least 50% in their Total Symptom Score (TSS), as assessed using the Myelofibrosis Symptom Assessment Form (MFSAF v4.0), was evaluated at both 12 and 24 weeks relative to their baseline score.
Time frame: Week 12 (Cycle 5 Day 1), Week 24 (Cycle 9 Day 1)
Phase 2 (Arms 1, 2 and 3): Number of Participants With Overall Splenic Response Rate (Overall SVR35)
Overall Splenic Response Rate (SVR35) was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as measured by imaging (MRI or CT), at any point between Cycle 1 Day 1 and the End of Study Visit, whichever occurred first.
Time frame: Through Phase II completion, an average of 6 years
Phase 2 (Arms 1, 2 and 3): Duration of Overall Splenic Response (Overall SVR35)
Duration of Overall Splenic Response (overall SVR35) was defined as the time from the first occurrence of a at least 35% reduction in spleen volume from baseline until the earliest of the following: a reduction of less than 35% from baseline combined with an increase of more than 25% from the nadir in spleen volume (as measured by MRI or CT), or death. The nadir was defined as the lowest spleen volume recorded after baseline and up to the evaluation point at which the initial splenic response was achieved.
Time frame: From first onset of splenic response until loss of response, assessed up to approximately 6 years
Phase 2 (Cohorts 1A and 2A): Number of Participants With Splenic Response Rate (SVR35) at 12 and 24 Weeks
Splenic Response Rate (SVR35) at 12 and 24 weeks was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as determined by imaging (MRI or CT), following 12 and 24 weeks of treatment, respectively.
Time frame: Week 12 (Cycle 5 Day 1), Week 24 weeks (Cycle 9 Day 1)
Phase 2 (Cohorts 1A and 2A): Duration of Red Blood Cell (RBC) Transfusion Independence (TI) in Participants Who Enroll as Transfusion Dependent (TD)
Duration of Red Blood Cell (RBC) Transfusion Independence (TI) was defined as the longest continuous period during which participants, having achieved at least 12 weeks of transfusion independence, remained free from RBC transfusions.
Time frame: From first onset of TI to earliest onset of loss of TI, assessed up to approximately 6 years
Phase 2 (Cohorts 1A and 2A): Early Anemic Response Rate in Participants Who Enroll as Transfusion Dependent (TD)
Early anemic response rate was defined as the proportion of participants who achieved an average increase of at least 1 g/dL in hemoglobin concentration over any rolling 8-week (56-day) period following baseline. This calculation was performed after applying the 14/3 day rule, which stipulates that hemoglobin measurements must be spaced at least 14 days apart, and that at least 3 such measurements are required within the 8-week window to ensure a reliable average. Importantly, this increase had to occur without any red blood cell (RBC) transfusions during the treatment period and up to the time of the latest hemoglobin assessment for each patient.
Time frame: Through Phase II completion, an average of 6 years
Phase 2 (Cohorts 1B, 2B, and Arm 3): Number of Participants With Splenic Response Rate (SVR35) at 12 Weeks
Splenic Response Rate (SVR35) at Week 12 was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as measured by imaging (MRI or CT), following 12 weeks of treatment.
Time frame: Week 12 (Cycle 5 Day 1)
Phase 2 (Cohorts 1B, 2B, and Arm 3): Anemic Response Rate in Participants Who Enroll as Non-transfusion-dependent (Non-TD)
Anemic response was defined as a sustained average increase in hemoglobin concentration of at least 1.5 g/dL over any rolling 12-week (84-day) period following baseline. This calculation excluded periods involving red blood cell (RBC) transfusions and was performed after applying the 14/3-day rule for valid hemoglobin assessments. The response had to be maintained through to the most recent available hemoglobin measurement for each patient.
Time frame: Through Phase II completion, an average of 6 years
Phase 2 (Arm 4): Percentage of Participants Who Achieve a ≥50% Reduction From Baseline in the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Total Score
The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a patient-reported questionnaire designed to measure symptom burden in participants with myeloproliferative neoplasms (MPNs). Participants rate the severity of several symptoms (such as fatigue, night sweats, itching, abdominal discomfort, bone pain, early satiety, and others) over the past 24 hours. Each symptom is scored on a scale from 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be). The total score is the sum of all individual symptom scores, providing an overall measure of symptom burden. A 50% reduction in the MPN-SAF total score at 12 or 24 weeks means the patient's overall symptom burden has improved by half compared to their baseline (pre-treatment) score.
Time frame: Baseline, 12 weeks (Cycle 5, Day 1), 24 weeks (Cycle 9, Day 1)
Phase 2 (Arm 4): Partial Hematological Response Rate (PHR)
Partial Hematological Response Rate (PHR) was defined as the proportion of participants who met the following criteria over two consecutive cycles up to the last administration of study treatment: * Platelet count \> 400-600 x 10\^9/L * WBC count within normal range (i.e., ≤ 10 x 10\^9/L) * Laboratory results confirmed after 1 cycle (after 3weeks)
Time frame: Over 2 consecutive cycles (rolling window) (1 cycle = 21 days)
Phase 2 (Arm 4): Overall Hematological Response Rate (OHR)
Confirmed Overall Hematological Response (OHR) Rate was defined as the proportion of participants with either a confirmed complete or a partial hematological response at any time.
Time frame: Through Phase II completion, an average of 6 years
Phase 2 (Arm 4): Duration of Overall Hematological Response Rate (OHR)
Duration of Overall Hematological Response (OHR) Rate was defined as the time from when the overall hematological response was first met until the time at which the overall hematological response was lost, i.e., the criteria for an overall hematological response (CHR or PHR) were not observed or death occurred, whichever came first.
Time frame: Through Phase II completion, an average of 6 years
Phase 2 (Arm 4): Rate of Hemorrhagic and Thromboembolic (TE) Events
Rate of hemorrhagic and thromboembolic (TE) events was defined as the proportion of participants with hemorrhagic or thromboembolic events throughout the study.
Time frame: Through Phase II completion, an average of 6 years
Phase 2 (All Arms): Maximum Observed Plasma Concentration (Cmax) of Pelabresib
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (All Arms): Time to Reach Maximum Concentration (Tmax) of Pelabresib
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (All Arms): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Pelabresib
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (All Arms): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Pelabresib
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (All Arms): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Pelabresib
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (All Arms): Time of Last Measurable Concentration (Tlast) of Pelabresib
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Tlast was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2A): Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2A): Time to Reach Maximum Concentration (Tmax) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2A): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2A): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2A): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2B): Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2B): Time to Reach Maximum Concentration (Tmax) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2B): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2B): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 2 - Cohort 2B): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 3): Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 3): Time to Reach Maximum Concentration (Tmax) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 3): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 3): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
Phase 2 (Arm 3): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Ruxolitinib
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days.
This study was conducted at 54 centers across 9 countries (Belgium, Canada, France, Germany, Italy, Netherlands, Poland, United Kingdom, United States)
| Milestone | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Started | 3 | 5 | 5 | 3 | 3 | 4 | 7 | 8 | 6 | 48 | 52 | 59 | 28 | 84 | 21 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 3 | 5 | 5 | 3 | 3 | 4 | 7 | 8 | 6 | 48 | 52 | 59 | 28 | 84 | 21 |
| Withdrew: Disease progression | 1 | 3 | 3 | 2 | 3 | 2 | 3 | 1 | 0 | 5 | 9 | 13 | 7 | 12 | 1 |
| Withdrew: Adverse event | 0 | 1 | 1 | 1 | 0 | 2 | 1 | 3 | 1 | 10 | 7 | 17 | 6 | 14 | 4 |
| Withdrew: Withdrawal by subject | 1 | 1 | 1 | 0 | 0 | 0 | 1 | 3 | 2 | 5 | 8 | 6 | 3 | 10 | 4 |
| Withdrew: Death | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 | 4 | 3 | 4 | 1 | 8 | 0 |
| Withdrew: Other protocol defined stopping criteria | 1 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 2 | 1 | 0 | 2 | 3 | 0 |
| Withdrew: Physician decision | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 2 | 20 | 13 | 14 | 6 | 10 | 3 |
| Withdrew: Transitioned to pelabresib extension study | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 2 | 7 | 3 | 0 | 14 | 9 |
| Withdrew: Cell transplant | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 4 | 2 | 3 | 13 | 0 |
A dose-limiting toxicity (DLT) was defined as an adverse event or abnormal laboratory value assessed by the Investigator as unrelated to disease progression, intercurrent illness, or concomitant medications that occurred within the first cycle of treatment (21-day cycle) with pelabresib (CPI-0610), and that met any of the criteria specified in the protocol.
| Participants | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) |
|---|---|---|---|---|---|---|---|---|---|
| Phase 1: Frequency of Dose-limiting Toxicities (DLTs) | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 2 | 4 |
Splenic Response Rate (SVR35) at Week 24 was defined as the proportion of participants who demonstrated a reduction of at least 35% in spleen size from baseline, as measured by imaging techniques (MRI or CT), following 24 weeks of treatment.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|
| Phase 2 (Cohorts 1B, 2B, and Arm 3): Number of Participants With Splenic Response Rate (SVR35) at Week 24 | 19.2 (9.6 to 32.5) | 21.4 (8.3 to 41.0) | 67.9 (56.8 to 77.6) |
Conversion rate was defined as the proportion of participants who converted from transfusion dependence (TD) to transfusion independence (TI). TD was characterized by receiving an average of at least 2 units of red blood cell (RBC) transfusions per month-amounting to a minimum of 6 units over the 12 weeks prior to enrollment-while TI was defined as the absence of RBC transfusions during any consecutive 12-week period.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|---|
| Phase 2 (Cohorts 1A and 2A): Number of Participants Enrolled as Transfusion Dependent (TD) With Conversion Rate From Red Blood Cell (RBC) Transfusion Dependence (TD) to Transfusion Independence (TI) | 25.6 (13.5 to 41.2) | 26.1 (14.3 to 41.1) |
Complete Hematological Response (CHR) Rate was defined as the proportion of participants who fulfilled the criteria for CHR, based on the modified European LeukemiaNet (ELN) guidelines (Barosi et al 2009): platelet count ≤400 × 10⁹/L, white blood cell (WBC) count ≤10 × 10⁹/L, confirmation of laboratory values after one treatment cycle, and normal spleen size determined by palpation or imaging.
| Percentage of Participants | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|
| Phase 2 (Arm 4): Number of Participants With Complete Hematological Response (CHR) Rate | 57.1 (34.0 to 78.2) |
The distribution of adverse events was performed through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters. Treatment-emergent adverse events (TEAEs) in this study were defined as events that began after the first dose of study treatment and continued until 30 days after the last dose, or events that were present prior to the first dose and increased in severity based on preferred term within 30 days following the last dose of pelabresib (CPI-0610).
| Participants | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) |
|---|---|---|---|---|---|---|---|---|---|
| Participants with at least 1 TEAE | 3 | 5 | 5 | 3 | 3 | 4 | 7 | 8 | 6 |
| Participants with ≥ Grade 3 TEAEs | 2 | 4 | 3 | 3 | 2 | 4 | 7 | 8 | 6 |
| Participants with any drug-related TEAE | 3 | 3 | 5 | 3 | 3 | 3 | 6 | 7 | 5 |
| Participants with any drug-related TEAEs of ≥ CTCAE Grade 3 | 1 | 1 | 1 | 0 | 0 | 0 | 5 | 3 | 4 |
| Participants with any serious TEAEs | 1 | 4 | 3 | 1 | 2 | 4 | 6 | 6 | 6 |
| Participants with any drug-related serious TEAEs | 0 | 0 | 1 | 0 | 0 | 0 | 1 | 0 | 3 |
| Participants with any TEAEs leading to drug discontinuation | 0 | 2 | 2 | 1 | 0 | 2 | 2 | 4 | 3 |
The distribution of adverse events was performed through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters. Treatment-emergent adverse events (TEAEs) in this study were defined as events that began after the first dose of study treatment and continued until 30 days after the last dose, or events that were present prior to the first dose and increased in severity based on preferred term within 30 days following the last dose of pelabresib (CPI-0610).
| Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Participants with at least 1 TEAE | 48 | 51 | 59 | 28 | 84 | 21 |
| Participants with ≥ Grade 3 TEAEs | 38 | 40 | 49 | 21 | 66 | 10 |
| Participants with at least 1 TEAE related to pelabresib | 45 | 44 | 53 | 27 | 74 | 20 |
| Participants with ≥ Grade 3 TEAEs related to pelabresib | 27 | 21 | 33 | 14 | 41 | 6 |
| Participants with any serious TEAEs | 18 | 23 | 29 | 18 | 40 | 5 |
| Participants with any serious TEAEs related to pelabresib | 4 | 3 | 2 | 6 | 16 | 2 |
| Participants with any TEAEs leading to interruption of pelabresib | 20 | 16 | 32 | 13 | 39 | 9 |
| Participants with any TEAEs leading in reduction of pelabresib | 16 | 10 | 15 | 7 | 38 | 6 |
| Participants with any TEAEs leading to discontinuation of pelabresib | 12 | 12 | 17 | 7 | 22 | 3 |
| Participants with any TEAEs leading to study discontinuation | 11 | 11 | 18 | 9 | 22 | 5 |
The Patient Global Impression of Change (PGIC) was a single-question, patient-reported assessment that asked individuals to rate their overall change in myeloproliferative neoplasm (MPN) symptoms since starting study treatment. The participants selected one of seven options, ranging from 'Very much improved' to 'Very much worse'. 'No Change from Baseline' indicated stable symptoms (no improvement or worsening), negative change from Baseline indicated a reduction in symptom severity (improvement), and positive change from Baseline indicated an increase in symptom severity (worsening).
| Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| PGIC status at Week 12 — Improvement | 28 | 36 | 22 | 18 | 66 | 13 |
| PGIC status at Week 12 — No change | 7 | 2 | 15 | 4 | 9 | 4 |
| PGIC status at Week 12 — Worsening | 4 | 5 | 10 | 3 | 5 | 2 |
| PGIC status at Week 24 — Improvement | 20 | 29 | 27 | 16 | 58 | 8 |
| PGIC status at Week 24 — No change | 10 | 3 | 12 | 6 | 15 | 3 |
| PGIC status at Week 24 — Worsening | 2 | 5 | 4 | 0 | 6 | 4 |
The MFSAF (Myelofibrosis Symptom Assessment Form) was completed by participants every day for 7 days before Day 1 of each treatment cycle, including the 7 days before starting Cycle 1. It used a 24-hour recall format, asking participants to rate the worst severity of seven symptoms (fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain) during the past 24 hours. Each symptom was rated on a scale from 0 (Absent) to 10 (Worst Imaginable). The Total Symptom Score (TSS) was the sum of 7 symptoms (range: 0-70). 'No Change from Baseline' indicated stable symptoms (no improvement or worsening), negative change from Baseline indicated a reduction in symptom severity (improvement), and positive change from Baseline indicated an increase in symptom severity (worsening).
| Score on a scale | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|---|---|
| Percent Change from baseline to Week 12 | -38.77 ± 38.637 | -30.98 ± 35.394 | -27.18 ± 75.391 | -29.70 ± 36.106 | -39.91 ± 69.251 |
| Percent Change from baseline to Week 24 | -32.69 ± 36.474 | -39.82 ± 42.228 | -38.93 ± 61.818 | -44.73 ± 35.224 | -47.43 ± 52.460 |
The proportion of study participants who experienced a reduction of at least 50% in their Total Symptom Score (TSS), as assessed using the Myelofibrosis Symptom Assessment Form (MFSAF v4.0), was evaluated at both 12 and 24 weeks relative to their baseline score.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|---|---|
| MFSAF TSS50 response at week 12 | 33.3 (20.0 to 49.0) | 31.3 (18.7 to 46.3) | 36.2 (24.0 to 49.9) | 33.3 (16.5 to 54.0) | 52.4 (41.1 to 63.6) |
| MFSAF TSS50 response at week 24 | 15.6 (6.5 to 29.5) | 34.7 (21.7 to 49.6) | 36.2 (24.0 to 49.9) | 40.7 (22.4 to 61.2) | 56.1 (44.7 to 67.0) |
Overall Splenic Response Rate (SVR35) was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as measured by imaging (MRI or CT), at any point between Cycle 1 Day 1 and the End of Study Visit, whichever occurred first.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|---|---|
| Phase 2 (Arms 1, 2 and 3): Number of Participants With Overall Splenic Response Rate (Overall SVR35) | 16.7 (7.0 to 31.4) | 28.8 (17.1 to 43.1) | 30.8 (18.7 to 45.1) | 25.0 (10.7 to 44.9) | 79.8 (69.6 to 87.7) |
Duration of Overall Splenic Response (overall SVR35) was defined as the time from the first occurrence of a at least 35% reduction in spleen volume from baseline until the earliest of the following: a reduction of less than 35% from baseline combined with an increase of more than 25% from the nadir in spleen volume (as measured by MRI or CT), or death. The nadir was defined as the lowest spleen volume recorded after baseline and up to the evaluation point at which the initial splenic response was achieved.
| Weeks | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|---|---|
| Phase 2 (Arms 1, 2 and 3): Duration of Overall Splenic Response (Overall SVR35) | 24.1 (12.4 to NA) | 73.1 (27.1 to 106.4) | 180.9 (73.0 to NA) | 228.4 (36.1 to NA) | 197.6 (95.7 to NA) |
Splenic Response Rate (SVR35) at 12 and 24 weeks was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as determined by imaging (MRI or CT), following 12 and 24 weeks of treatment, respectively.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|---|
| SVR35 at week 12 | 9.3 (2.6 to 22.1) | 9.4 (3.1 to 20.7) |
| SVR35 at week 24 | 0 (0 to NA) | 16.4 (7.8 to 28.8) |
Duration of Red Blood Cell (RBC) Transfusion Independence (TI) was defined as the longest continuous period during which participants, having achieved at least 12 weeks of transfusion independence, remained free from RBC transfusions.
| Weeks | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|---|
| Phase 2 (Cohorts 1A and 2A): Duration of Red Blood Cell (RBC) Transfusion Independence (TI) in Participants Who Enroll as Transfusion Dependent (TD) | 35.8 (15.3 to NA) | 68.0 (34.7 to NA) |
Early anemic response rate was defined as the proportion of participants who achieved an average increase of at least 1 g/dL in hemoglobin concentration over any rolling 8-week (56-day) period following baseline. This calculation was performed after applying the 14/3 day rule, which stipulates that hemoglobin measurements must be spaced at least 14 days apart, and that at least 3 such measurements are required within the 8-week window to ensure a reliable average. Importantly, this increase had to occur without any red blood cell (RBC) transfusions during the treatment period and up to the time of the latest hemoglobin assessment for each patient.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|---|
| Phase 2 (Cohorts 1A and 2A): Early Anemic Response Rate in Participants Who Enroll as Transfusion Dependent (TD) | 18.6 (8.4 to 33.4) | 21.7 (10.9 to 36.4) |
Splenic Response Rate (SVR35) at Week 12 was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as measured by imaging (MRI or CT), following 12 weeks of treatment.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|
| Phase 2 (Cohorts 1B, 2B, and Arm 3): Number of Participants With Splenic Response Rate (SVR35) at 12 Weeks | 15.4 (6.9 to 28.1) | 10.7 (2.3 to 28.2) | 66.7 (55.5 to 76.6) |
Anemic response was defined as a sustained average increase in hemoglobin concentration of at least 1.5 g/dL over any rolling 12-week (84-day) period following baseline. This calculation excluded periods involving red blood cell (RBC) transfusions and was performed after applying the 14/3-day rule for valid hemoglobin assessments. The response had to be maintained through to the most recent available hemoglobin measurement for each patient.
| Percentage of Participants | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|---|---|
| Phase 2 (Cohorts 1B, 2B, and Arm 3): Anemic Response Rate in Participants Who Enroll as Non-transfusion-dependent (Non-TD) | 47.1 (32.9 to 61.5) | 21.4 (8.3 to 41.0) | 35.4 (25.0 to 47.0) |
The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a patient-reported questionnaire designed to measure symptom burden in participants with myeloproliferative neoplasms (MPNs). Participants rate the severity of several symptoms (such as fatigue, night sweats, itching, abdominal discomfort, bone pain, early satiety, and others) over the past 24 hours. Each symptom is scored on a scale from 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be). The total score is the sum of all individual symptom scores, providing an overall measure of symptom burden. A 50% reduction in the MPN-SAF total score at 12 or 24 weeks means the patient's overall symptom burden has improved by half compared to their baseline (pre-treatment) score.
| Percentage of Participants | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|
| MPN-SAF TSS50 response at week 12 | 25.0 (8.7 to 49.1) |
| MPN-SAF TSS50 response at week 24 | 20.0 (5.7 to 43.7) |
Partial Hematological Response Rate (PHR) was defined as the proportion of participants who met the following criteria over two consecutive cycles up to the last administration of study treatment: * Platelet count \> 400-600 x 10\^9/L * WBC count within normal range (i.e., ≤ 10 x 10\^9/L) * Laboratory results confirmed after 1 cycle (after 3weeks)
| Percentage of Participants | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|
| Phase 2 (Arm 4): Partial Hematological Response Rate (PHR) | 38.1 (18.1 to 61.6) |
Confirmed Overall Hematological Response (OHR) Rate was defined as the proportion of participants with either a confirmed complete or a partial hematological response at any time.
| Percentage of Participants | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|
| Phase 2 (Arm 4): Overall Hematological Response Rate (OHR) | 66.7 (43.0 to 85.4) |
Duration of Overall Hematological Response (OHR) Rate was defined as the time from when the overall hematological response was first met until the time at which the overall hematological response was lost, i.e., the criteria for an overall hematological response (CHR or PHR) were not observed or death occurred, whichever came first.
| Weeks | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|
| Phase 2 (Arm 4): Duration of Overall Hematological Response Rate (OHR) | 21.1 (9.1 to 33.1) |
Rate of hemorrhagic and thromboembolic (TE) events was defined as the proportion of participants with hemorrhagic or thromboembolic events throughout the study.
| Percentage of Participants | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|
| Phase 2 (Arm 4): Rate of Hemorrhagic and Thromboembolic (TE) Events | 61.9 (38.4 to 81.9) |
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Phase 2 (All Arms): Maximum Observed Plasma Concentration (Cmax) of Pelabresib | 1280 ± 29.1 | 1350 ± 37.0 | 1210 ± 40.0 | 1190 ± 29.0 | 1070 ± 26.3 | 2130 ± 24.7 |
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
| Hour | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Phase 2 (All Arms): Time to Reach Maximum Concentration (Tmax) of Pelabresib | 1.94 (0.85 to 4.02) | 1.58 (0.83 to 3.63) | 1.90 (0.50 to 4.00) | 1.81 (0.92 to 3.13) | 1.71 (0.50 to 3.87) | 2.08 (1.07 to 6.00) |
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Phase 2 (All Arms): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Pelabresib | 120 ± 489.0 | 201 ± 107.0 | 244 ± 151.0 | 162 ± 84.0 | 240 ± 79.1 | 174 ± 112.0 |
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Phase 2 (All Arms): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Pelabresib | 5660 ± 36.7 | 5610 ± 35.5 | 5190 ± 40.9 | 4760 ± 26.7 | 4380 ± 26.8 | 9590 ± 28.7 |
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Phase 2 (All Arms): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Pelabresib | 6200 ± 35.3 | 6470 ± 31.7 | 5320 ± 38.1 | 4930 ± 28.8 | 4540 ± 25.3 | 10800 ± 29.0 |
Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Tlast was listed and summarized using descriptive statistics.
| Hour | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) |
|---|---|---|---|---|---|---|
| Phase 2 (All Arms): Time of Last Measurable Concentration (Tlast) of Pelabresib | 7.08 (4.50 to 9.00) | 7.07 (2.58 to 8.08) | 7.19 (6.10 to 8.05) | 7.12 (7.00 to 8.17) | 7.08 (5.07 to 8.58) | 7.08 (5.88 to 10.08) |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|
| Cohort 2A, 5 mg | 65.9 ± 56.5 |
| Cohort 2A, 7.5 mg | 142.0 ± 31.0 |
| Cohort 2A, 10 mg | 108.0 ± 29.3 |
| Cohort 2A, 15 mg | 310.0 ± 88.9 |
| Cohort 2A, 20 mg | 325.0 ± 37.7 |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
| Hour | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|
| Cohort 2A, 5 mg | 0.92 (0.50 to 2.00) |
| Cohort 2A, 7.5 mg | 1.00 (0.50 to 2.02) |
| Cohort 2A, 10 mg | 0.76 (0.50 to 1.50) |
| Cohort 2A, 15 mg | 1.55 (0.67 to 3.50) |
| Cohort 2A, 20 mg | 0.53 (0.33 to 1.58) |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|
| Cohort 2A, 5 mg | NA ± NA |
| Cohort 2A, 7.5 mg | 14.7 ± 74.9 |
| Cohort 2A, 10 mg | 13.4 ± 81.6 |
| Cohort 2A, 15 mg | 74.6 ± 94.7 |
| Cohort 2A, 20 mg | 19.8 ± 54.2 |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|
| Cohort 2A, 5 mg | 230 ± 63.3 |
| Cohort 2A, 7.5 mg | 390 ± 34.0 |
| Cohort 2A, 10 mg | 347 ± 44.7 |
| Cohort 2A, 15 mg | 1240 ± 64.7 |
| Cohort 2A, 20 mg | 829 ± 42.3 |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A |
|---|---|
| Cohort 2A, 5 mg | 239 ± 64.5 |
| Cohort 2A, 7.5 mg | 369 ± 35.9 |
| Cohort 2A, 10 mg | 375 ± 47.2 |
| Cohort 2A, 15 mg | NA ± NA |
| Cohort 2A, 20 mg | 887 ± 42.3 |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B |
|---|---|
| Cohort 2B, 5 mg | 69.5 ± 27.8 |
| Cohort 2B, 15 mg | NA ± NA |
| Cohort 2B, 20 mg | NA ± NA |
| Cohort 2B, 25 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
| Hour | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B |
|---|---|
| Cohort 2B, 5 mg | 1.57 (0.47 to 1.60) |
| Cohort 2B, 15 mg | 0.48 (NA to NA) |
| Cohort 2B, 20 mg | 0.53 (NA to NA) |
| Cohort 2B, 25 mg | 0.40 (NA to NA) |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B |
|---|---|
| Cohort 2B, 5 mg | 8.49 ± 28.4 |
| Cohort 2B, 15 mg | NA ± NA |
| Cohort 2B, 20 mg | NA ± NA |
| Cohort 2B, 25 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B |
|---|---|
| Cohort 2B, 5 mg | 207 ± 3.08 |
| Cohort 2B, 15 mg | NA ± NA |
| Cohort 2B, 20 mg | NA ± NA |
| Cohort 2B, 25 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B |
|---|---|
| Cohort 2B, 5 mg | 214 ± 3.67 |
| Cohort 2B, 15 mg | NA ± NA |
| Cohort 2B, 20 mg | NA ± NA |
| Cohort 2B, 25 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|
| Cohort 3, 5 mg | NA ± NA |
| Cohort 3, 10 mg | 190 ± 28.6 |
| Cohort 3, 15 mg | 213 ± 34.7 |
| Cohort 3, 20 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics.
| Hour | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|
| Cohort 3, 5 mg | 0.72 (NA to NA) |
| Cohort 3, 10 mg | 0.62 (0.28 to 7.00) |
| Cohort 3, 15 mg | 0.95 (0.22 to 3.87) |
| Cohort 3, 20 mg | 1.25 (NA to NA) |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics.
| ng/mL | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|
| Cohort 3, 5 mg | NA ± NA |
| Cohort 3, 10 mg | 25.3 ± 99.2 |
| Cohort 3, 15 mg | 31.4 ± 58.1 |
| Cohort 3, 20 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|
| Cohort 3, 5 mg | NA ± NA |
| Cohort 3, 10 mg | 519 ± 54.8 |
| Cohort 3, 15 mg | 677 ± 33.9 |
| Cohort 3, 20 mg | NA ± NA |
Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics.
| h*ng/mL | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) |
|---|---|
| Cohort 3, 5 mg | NA ± NA |
| Cohort 3, 10 mg | 587 ± 45.5 |
| Cohort 3, 15 mg | 696 ± 34.7 |
| Cohort 3, 20 mg | NA ± NA |
Collected over Adverse events were collected for each participant from the first dose of study treatment through 30 days after the last dose of pelabresib (CPI-0610). The duration of adverse event collection varied by participant and phase, with a maximum follow-up of approximately 2 years in Phase I and approximately 8 years in Phase II. Deaths were collected for each participant from study initiation through study completion, with an average follow-up of up to approximately 10 years.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase 1 (24 mg Capsule PO Daily) | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Phase 1 (48 mg Capsule PO Daily) | 1/5 (20%) | 4/5 (80%) | 5/5 (100%) |
| Phase 1 (120 mg Capsule PO Daily) | 2/5 (40%) | 3/5 (60%) | 5/5 (100%) |
| Phase 1 (170 mg Capsule PO Daily) | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Phase 1 (230 mg Capsule PO Daily) | 1/3 (33.3%) | 2/3 (66.7%) | 3/3 (100%) |
| Phase 1 (300 mg Capsule PO Daily) | 1/4 (25%) | 4/4 (100%) | 4/4 (100%) |
| Phase 1 (400 mg Capsule PO Daily) | 1/7 (14.3%) | 6/7 (85.7%) | 7/7 (100%) |
| Phase 1 (225 mg Tablet PO Daily) | 1/8 (12.5%) | 6/8 (75%) | 8/8 (100%) |
| Phase 1 (275 mg Tablet PO Daily) | 1/6 (16.7%) | 6/6 (100%) | 6/6 (100%) |
| Phase 1 (Overall) | 8/44 (18.2%) | 33/44 (75%) | 44/44 (100%) |
| Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | 6/48 (12.5%) | 18/48 (37.5%) | 48/48 (100%) |
| Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | 3/52 (5.8%) | 23/52 (44.2%) | 50/52 (96.2%) |
| Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | 3/59 (5.1%) | 29/59 (49.2%) | 58/59 (98.3%) |
| Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | 3/28 (10.7%) | 18/28 (64.3%) | 28/28 (100%) |
| Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | 7/84 (8.3%) | 40/84 (47.6%) | 84/84 (100%) |
| Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) | 0/21 (0%) | 5/21 (23.8%) | 21/21 (100%) |
| Phase 2 (Overall) | 22/292 (7.5%) | 133/292 (45.5%) | 289/292 (99%) |
| Event | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) | Phase 1 (Overall) | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) | Phase 2 (Overall) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| VomitingGastrointestinal disorders | 0/3 | 0/5 | 0/5 | 0/3 | 0/3 | 0/4 | 0/7 | 0/8 | 3/6 | 3/44 | 0/48 | 0/52 | 0/59 | 1/28 | 0/84 | 0/21 | 1/292 |
| Febrile neutropeniaBlood and lymphatic system disorders | 1/3 | 2/5 | 1/5 | 0/3 | 0/3 | 0/4 | 2/7 | 1/8 | 2/6 | 9/44 | 0/48 | 0/52 | 0/59 | 0/28 | 1/84 | 0/21 | 1/292 |
| FatigueGeneral disorders | 0/3 | 2/5 | 0/5 | 0/3 | 1/3 | 0/4 | 0/7 | 0/8 | 1/6 | 4/44 | 0/48 | 0/52 | 0/59 | 0/28 | 0/84 | 0/21 | 0/292 |
| AnaemiaBlood and lymphatic system disorders | 0/3 | 0/5 | 0/5 | 1/3 | 0/3 | 0/4 | 1/7 | 0/8 | 0/6 | 2/44 | 1/48 | 2/52 | 2/59 | 5/28 | 1/84 | 0/21 | 11/292 |
| Sinus tachycardiaCardiac disorders | 0/3 | 0/5 | 0/5 | 1/3 | 0/3 | 0/4 | 0/7 | 0/8 | 0/6 | 1/44 | 0/48 | 0/52 | 0/59 | 0/28 | 0/84 | 0/21 | 0/292 |
| DiarrhoeaGastrointestinal disorders | 0/3 | 0/5 | 1/5 | 1/3 | 0/3 | 0/4 | 0/7 | 0/8 | 1/6 | 3/44 | 0/48 | 1/52 | 0/59 | 0/28 | 0/84 | 0/21 | 1/292 |
| NauseaGastrointestinal disorders | 0/3 | 0/5 | 0/5 | 0/3 | 0/3 | 0/4 | 0/7 | 0/8 | 2/6 | 2/44 | 0/48 | 0/52 | 0/59 | 0/28 | 0/84 | 0/21 | 0/292 |
| PyrexiaGeneral disorders | 0/3 | 0/5 | 0/5 | 1/3 | 0/3 | 0/4 | 1/7 | 0/8 | 1/6 | 3/44 | 1/48 | 0/52 | 1/59 | 1/28 | 3/84 | 0/21 | 6/292 |
| Device related infectionInfections and infestations | 1/3 | 0/5 | 0/5 | 0/3 | 0/3 | 0/4 | 0/7 | 1/8 | 0/6 | 2/44 | 0/48 | 0/52 | 0/59 | 0/28 | 0/84 | 0/21 | 0/292 |
| SepsisInfections and infestations | 0/3 | 0/5 | 1/5 | 0/3 | 0/3 | 0/4 | 0/7 | 0/8 | 2/6 | 3/44 | 1/48 | 1/52 | 0/59 | 0/28 | 2/84 | 0/21 | 4/292 |
| Event | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) | Phase 1 (Overall) | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) | Phase 2 (Overall) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| DiarrhoeaGastrointestinal disorders | 1/3 | 0/5 | 1/5 | 1/3 | 0/3 | 4/4 | 3/7 | 5/8 | 2/6 | 17/44 | 15/48 | 26/52 | 34/59 | 18/28 | 40/84 | 10/21 | 143/292 |
| NauseaGastrointestinal disorders | 2/3 | 3/5 | 4/5 | 3/3 | 0/3 | 2/4 | 2/7 | 5/8 | 2/6 | 23/44 | 18/48 | 23/52 | 21/59 | 12/28 | 26/84 | 16/21 | 116/292 |
| FatigueGeneral disorders | 2/3 | 3/5 | 4/5 | 2/3 | 0/3 | 0/4 | 5/7 | 3/8 | 1/6 | 20/44 | 14/48 | 17/52 | 13/59 | 13/28 | 27/84 | 5/21 | 89/292 |
| VomitingGastrointestinal disorders | 0/3 | 2/5 | 1/5 | 2/3 | 0/3 | 1/4 | 3/7 | 1/8 | 3/6 | 13/44 | 7/48 | 10/52 | 10/59 | 8/28 | 15/84 | 9/21 | 59/292 |
| Platelet count decreasedInvestigations | 2/3 | 0/5 | 0/5 | 1/3 | 0/3 | 0/4 | 2/7 | 2/8 | 1/6 | 8/44 | 5/48 | 8/52 | 11/59 | 8/28 | 22/84 | 2/21 | 56/292 |
| Decreased appetiteMetabolism and nutrition disorders | 0/3 | 1/5 | 3/5 | 2/3 | 2/3 | 1/4 | 2/7 | 5/8 | 2/6 | 18/44 | 7/48 | 13/52 | 13/59 | 7/28 | 15/84 | 4/21 | 59/292 |
| AnxietyPsychiatric disorders | 2/3 | 1/5 | 0/5 | 0/3 | 0/3 | 0/4 | 0/7 | 0/8 | 0/6 | 3/44 | 0/48 | 1/52 | 3/59 | 1/28 | 4/84 | 0/21 | 9/292 |
| CoughRespiratory, thoracic and mediastinal disorders | 2/3 | 0/5 | 1/5 | 0/3 | 0/3 | 0/4 | 0/7 | 1/8 | 2/6 | 6/44 | 9/48 | 11/52 | 13/59 | 14/28 | 22/84 | 4/21 | 73/292 |
| RashSkin and subcutaneous tissue disorders | 0/3 | 1/5 | 0/5 | 2/3 | 0/3 | 0/4 | 1/7 | 0/8 | 0/6 | 4/44 | 2/48 | 7/52 | 3/59 | 4/28 | 6/84 | 4/21 | 26/292 |
| HaematomaVascular disorders | 0/3 | 0/5 | 0/5 | 0/3 | 2/3 | 0/4 | 0/7 | 0/8 | 0/6 | 2/44 | 0/48 | 3/52 | 1/59 | 3/28 | 4/84 | 1/21 | 12/292 |
| Age, Continuous(Years) | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Median | 79.0 (37 to 79) | 65.0 (55 to 76) | 69.0 (34 to 80) | 76.0 (69 to 79) | 80.0 (57 to 82) | 66.5 (27 to 76) | 63.0 (59 to 77) | 65.5 (54 to 82) | 61.0 (18 to 80) | 73.0 (60 to 88) | 69.5 (43 to 85) | 72.0 (41 to 83) | 65.5 (49 to 78) | 68.0 (37 to 85) | 64.0 (42 to 83) | 69.1 (18 to 88) |
| Sex: Female, Male(Participants) | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female | 3 | 1 | 1 | 3 | 1 | 0 | 2 | 1 | 2 | 16 | 29 | 18 | 13 | 25 | 13 | 128 |
| Male | 0 | 4 | 4 | 0 | 2 | 4 | 5 | 7 | 4 | 32 | 23 | 41 | 15 | 59 | 8 | 208 |
| Race (NIH/OMB)(Participants) | Phase 1 (24 mg Capsule PO Daily) | Phase 1 (48 mg Capsule PO Daily) | Phase 1 (120 mg Capsule PO Daily) | Phase 1 (170 mg Capsule PO Daily) | Phase 1 (230 mg Capsule PO Daily) | Phase 1 (300 mg Capsule PO Daily) | Phase 1 (400 mg Capsule PO Daily) | Phase 1 (225 mg Tablet PO Daily) | Phase 1 (275 mg Tablet PO Daily) | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1A | Phase 2 - Arm 1 (MF Participants Previously Treated With JAK Inhibitor Monotherapy) - Cohort 1B | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2A | Phase 2 - Arm 2 (Supplement to JAK Inhibitor Combination Arm for MF Participants) - Cohort 2B | Phase 2 - Arm 3 (Combination Treatment Arm for JAKi-Naïve MF Participants) | Phase 2 - Arm 4 (Monotherapy Arm for Essential Thrombocythemia (ET)) | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 | 1 | 1 | 3 | 0 | 8 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 1 | 0 | 0 | 1 | 1 | 0 | 2 | 4 | 1 | 2 | 4 | 3 | 0 | 19 |
| White | 3 | 4 | 4 | 3 | 3 | 1 | 6 | 8 | 4 | 42 | 47 | 54 | 22 | 75 | 19 | 295 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 1 | 2 | 1 | 1 | 3 | 2 | 12 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Studies of the drug tested here: CPI-0610
Constellation Pharmaceuticals