A Phase 2/3 interventional study of Imetelstat Sodium and Placebo in Myelodysplastic Syndromes, sponsored by Geron Corporation. Active, not recruiting at 126 sites in 17 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-07-17.
Sponsored by Geron Corporation · Phase 2/3, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy and safety of imetelstat sodium in transfusion-dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Phase 2 study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat sodium to placebo in transfusion-dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Phase 3 study.
A separate Ventricular Repolarization Substudy (QTc Substudy) will evaluate the effect of imetelstat sodium on ventricular repolarization.
An Extension Phase has been included to allow continued treatment for those participants who are benefitting from imetelstat sodium and to continue to evaluate the long-term safety, overall survival (OS), and disease progression, including progression to acute myeloid leukemia (AML) in transfusion-dependent participants with low or immediate-1 risk MDS that is relapsed/refractory to ESA treatment.
This is a Phase 2/3, multicenter study of imetelstat sodium in which 289 participants were enrolled.
The Extension Phase was initiated at the end of the Phase 3 study (24 months after the last participant was randomized in the Phase 3) and will continue until participants who entered Phase 3 study participated in the study for up to 5 years from the first dose of imetelstat sodium (including treatment and follow-up), or 3 years of post-treatment follow-up from the last dose of study treatment, whichever occurs later, or until death, withdrawal of consent, study termination, or until a participant is lost to follow-up. Participants ongoing on imetelstat sodium and considered to be benefiting from treatment per Investigator in the Phase 3 Study or Ventricular Repolarization Substudy, have the option to continue receiving imetelstat sodium in the Extension Phase. Participants in the follow-up phase for the Phase 3 study or Ventricular Repolarization Substudy have the option to continue the follow-up in the Extension Phase.
The Phase 2, Phase 3, and VR Substudy all consist of 3 phases: a Screening phase (up to 28 days); a treatment phase; and a post-treatment follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first). The Extension Phase of the study will consist of an extended treatment phase and an extended follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first).
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
This study's enrollment of 289 is above the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Geron Corporation is the lead sponsor of 16 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Additional Exclusion Criteria for the Ventricular Repolarization Substudy:
Cardiac function abnormalities on screening as follows:
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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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 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.
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.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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.
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.
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.
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.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
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.
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.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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.
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.
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.
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.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Imetelstat sodium administered intravenously (IV), at a starting dose of 7.5 milligrams per kilogram (mg/kg), every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Drug: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Drug: Imetelstat Sodium
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Drug: Placebo
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Drug: Imetelstat Sodium
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
Drug: Placebo
Participants randomized to the imetelstat sodium arm in the Phase 3 and the VR QTc Substudy, based on the response will continue to receive imetelstat sodium IV, at the dose they were receiving in the Phase 3 or VR QTc Substudy, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, study termination, or up to 3 years whichever occurs first.
Drug: Imetelstat Sodium
Participants randomized to the placebo arm in the Phase 3 study will enter the Extended Follow-up part of the Extension Phase and continue in follow up until death, lost to follow-up, withdrawal of consent, study termination, or whichever occurs first up to approximately 3 years.
Drug: Placebo
Imetelstat sodium IV infusion.
Also known as: GRN163L
Imetelstat sodium-matching placebo IV infusion.
Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants)
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 5 years in Phase 2
Phase 2: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period in Target Population
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 5 years in Phase 2
Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 3.7 years in Phase 3
Phase 2 and Phase 3: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Deaths
TEAEs were defined as those events that 1) occurred after the first dose of study drug, through the treatment phase, and for 30 days following the last dose of study drug or until subsequent anti-cancer therapy if earlier; 2) any event that was considered study drug-related regardless of the start date of the event; or 3) any event that was presented at baseline but worsened in severity or was subsequently considered drug-related by the investigator. Serious TEAEs are any TEAEs that result in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product, is medically important. Any clinically significant vital sign measurements, clinical laboratory values, and electrocardiogram (ECG) findings were reported as TEAEs. TEAEs included both serious and non-serious TEAEs.
Time frame: Adverse events: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3; Deaths: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 24-Weeks Period
Percentage of participants without any RBC transfusion during any consecutive 24 weeks (168 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2 and the day of randomization for participants enrolled in Phase 3. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Time to the 8-Weeks RBC Transfusion Independence (TI)
Time to 8-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 8-week RBC TI period.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Time to the 24-Weeks RBC TI
Time to 24-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 24-weeks RBC TI period.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Duration of RBC TI
Duration of RBC TI was defined as the first day of the longest RBC TI period to the date of the first RBC transfusion after the TI period started. The 95% CI was based on Kaplan-Meier product limit estimates.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Percentage of Participants With Hematologic Improvement Including Erythroid Response (HI-E) as Per International Working Group (IWG) Response Criteria 2006
As per IWG Response Criteria 2006: HI-E was defined as a hemoglobin (Hb) increase by greater than or equal to (≥)1.5 grams per deciliter (g/dL) relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusion units/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of less than or equal to (≤)9 g/dL pretreatment were counted in the RBC transfusion response evaluation. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2: Percentage of Participants With Complete Remission (CR), Partial Remission (PR), or Marrow Complete Remission (mCR) as Per International Working Group (IWG) Response Criteria 2006 as Assessed by the Investigator
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood (PB): Hb ≥11 g/dL; platelets ≥100 x 10\^9/dL; neutrophils ≥1.0 x 10\^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. Percentages were rounded off to the nearest single decimal place. All participants in Phase 2 were evaluated by the Investigator for CR/PR/mCR regardless of bone marrow blasts at baseline.
Time frame: Up to 5 years in Phase 2
Phase 3: Percentage of Participants With CR, PR, or mCR as Per IWG Response Criteria 2006 as Assessed by the Independent Review Committee (IRC)
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; PB: Hb ≥11 g/dL; platelets ≥100 x 10\^9/dL; neutrophils ≥1.0 x 10\^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. CR, PR and mCR were assessed by IRC in Phase 3 and participants were required to fit at least one of the following criteria (participants with \>5% baseline blasts per central pathology reviewer assessment; participants with CR, PR, mCR, or cytogenetic response as assessed by the investigator) and have at least one post-baseline assessment.
Time frame: Up to 3.7 years in Phase 3
Phase 2 and Phase 3: Overall Survival (OS)
OS was defined as the interval from Study Day 1 to death from any cause. Survival time of living participants was censored on the last date a participant is known to be alive or lost to follow-up. The Kaplan-Meier method was used to estimate overall survival.
Time frame: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Progression Free Survival (PFS)
Progression free survival was defined as the time interval from study Day 1 to the first date of disease progression or death from any cause, whichever occurs first. Disease progression as per IWG criteria was defined as: at least one of the following: at least 50 % decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by ≥1.5 g/dL; transfusion dependence.
Time frame: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Time to Progression to Acute Myeloid Leukemia (AML)
Time to progression to AML was defined as the interval from Study Day 1 to the date of AML diagnosis. Participants who did not progress to AML and were still alive at the cutoff date for the analysis or who withdrew from the study (withdrawal of consent or lost to follow-up), data was censored at the date of the last disease evaluation.
Time frame: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Amount of RBC Transfusions in the Best 8-week Interval
Amount of RBC transfusions for 8-week interval was defined as the total number of RBC transfusion units in a given 8-week interval during study. The best 8-week interval is a post-baseline 8-week interval where the participant had the fewest post-Study Day 1 RBC transfusion units. A valid 8-week period must start before the date of last dose of study drug + 30 days or end of treatment (EOT) visit whichever occurs first and ends before the first transfusion in post-treatment follow-up or the first day of subsequent anti-cancer therapy whichever occurs first.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Percent Change in RBC Transfusions Relative to Prior Transfusion Burden
Relative percent change in RBC transfusions per 8-week = (amount of RBC transfusions per 8-week - prior RBC transfusion burden) / prior RBC transfusion burden multiplied by 100%. Prior RBC transfusion burden was defined as the maximum number of RBC units transfused over any consecutive 8 weeks prior to study entry.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Percentage of Participants Who Received Any Myeloid Growth Factors
Percentage of participants who received any myeloid growth factors starting from Study Day 1 were reported. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Duration of Myeloid Growth Factors Administration
Duration of myeloid growth factor administered starting from Study Day 1 was reported.
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Maximum Observed Plasma Concentration (Cmax)
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for pharmacokinetic (PK) data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
Time frame: Pre-dose on Cycle 1 Day 1 and pre-dose on Day 1 of every 3 cycles from Cycle 4 up to Cycle 66 in Phase 2 and Cycle 34 in Phase 3 (each cycle length= 28 days)
Phase 2 and Phase 3: Area Under the Drug Concentration-Plasma Time Curve From Time Zero to Time 28 Days (AUC0-28d)
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for PK data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
Time frame: Cycle 1 (Days 1 to 28) (Cycle duration= 28 days)
Phase 2 and Phase 3: Percentage of Participants With Anti-drug Antibodies (ADA) to Imetelstat Sodium
As pre-specified in the SAP, participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for immunogenicity data collection and analyses in this outcome measure. Percentages were rounded off to the nearest single decimal place.
Time frame: Pre-dose on Cycle 1 Day 1 and pre-dose on Day 1 of every 3 cycles from Cycle 4 up to Cycle 66 in Phase 2 and Cycle 34 in Phase 3 (each cycle length= 28 days)
Phase 3: Medical Resource Utilization Assessed Based on Percentage of Participants With Outpatient Medical Encounters
Outpatient medical encounters included various sites of care: a) emergency room (ER) visits, b) hospital outpatient visits, c) home care visit, d) visit to lab, e) visit to doctor's office, f) other visits. Percentages were rounded off to the nearest single decimal place.
Time frame: Up to 3.7 years in Phase 3
Phase 3: Medical Resource Utilization Assessed Based on Duration of Hospitalization
Hospitalization included any medical encounter defined as hospice, hospital inpatient department, and intensive care unit (ICU). Hospitalizations without end dates were not counted in the calculation of length of stay. If any participant had multiple readmissions, duration was calculated as the sum of all hospital stays.
Time frame: Up to 3.7 years in Phase 3
QTc Substudy: Change From Baseline in QT Interval by Fridericia's Correction Method (ΔQTcF)
Baseline was defined as the mean of the measured ECG intervals collected at 3 time points (-1 hour, -0.5 hour, and 0 hour) prior to treatment administration on Cycle 1 Day 1 (Cycle length= 28 days)
Time frame: Baseline, Cycle 1, Day 1: 0.5, 1, 2, 4, 6, and 8 hours post-dose (cycle length= 28 days)
Extension Phase: Number of Participants With AEs
An adverse event (AE) was any untoward medical occurrence in a clinical study participant administered a medicinal product. An AE does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to that medicinal product. AEs included clinically significant vital signs measurements, clinical laboratory values and electrocardiograms (ECGs) changes.
Time frame: Up to approximately 3 years in the extension phase
Extension Phase: Overall Survival
Time frame: Up to approximately 3 years in the extension phase
Extension Phase: Progression Free Survival (PFS) Survival
Progression free survival will be assessed as the time interval from the end of the Phase 3 study until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. As per IWG criteria disease progression is defined as: at least one of the following: at least 50 percent (%) decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by greater than or equal to ≥1.5 g/dL; transfusion dependence.
Time frame: Up to approximately 3 years in the extension phase
Participants were enrolled in Phase 2 at 48 study sites and in the Phase 3 Study at 77 study sites. Data is reported for the Phase 2, Phase 3 and QTc substudy up to the primary completion date (PCD) of 13 October 2023. Enrolment in Extension phase started after October 2023 and analyses for the participants in extension phase is still ongoing, hence data for extension phase will be reported at study completion date in 2026.
| Milestone | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Placebo |
|---|---|---|---|---|---|
| Started | 57 | 118 | 60 | 35 | 19 |
| Crossover to imetelstat | 0 | 0 | 0 | 0 | 14 |
| Target population | 38 | 0 | 0 | 0 | 0 |
| Completed | 15 | 57 | 29 | 33 | 17 |
| Not completed | 42 | 61 | 31 | 2 | 2 |
| Withdrew: Death | 27 | 34 | 14 | 1 | 0 |
| Withdrew: Lost to follow-up | 2 | 3 | 2 | 0 | 0 |
| Withdrew: Withdrawal by subject | 13 | 24 | 14 | 1 | 1 |
| Withdrew: Adverse event | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Randomized, never treated | 0 | 0 | 0 | 0 | 1 |
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 2: Imetelstat Sodium |
|---|---|
| Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants) | 36.8 (24.45 to 50.66) |
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 2: Imetelstat Sodium |
|---|---|
| Phase 2: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period in Target Population | 42.1 (26.31 to 59.18) |
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|
| Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period | 39.8 (30.93 to 49.25) | 15.0 (7.10 to 26.57) |
TEAEs were defined as those events that 1) occurred after the first dose of study drug, through the treatment phase, and for 30 days following the last dose of study drug or until subsequent anti-cancer therapy if earlier; 2) any event that was considered study drug-related regardless of the start date of the event; or 3) any event that was presented at baseline but worsened in severity or was subsequently considered drug-related by the investigator. Serious TEAEs are any TEAEs that result in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product, is medically important. Any clinically significant vital sign measurements, clinical laboratory values, and electrocardiogram (ECG) findings were reported as TEAEs. TEAEs included both serious and non-serious TEAEs.
| Participants | Phase 2: Imetelstat Sodium (No Dose Escalation) | Phase 2: Imetelstat Sodium (Escalated to 9.4 mg/kg) | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|---|
| Participants with AEs | 50 | 6 | 117 | 59 |
| Participants who died | 24 | 3 | 35 | 15 |
Percentage of participants without any RBC transfusion during any consecutive 24 weeks (168 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2 and the day of randomization for participants enrolled in Phase 3. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 24-Weeks Period | 24.6 (14.13 to 37.76) | 28.0 (20.10 to 36.98) | 3.3 (0.41 to 11.53) |
Time to 8-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 8-week RBC TI period.
| weeks | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Time to the 8-Weeks RBC Transfusion Independence (TI) | 8.29 (0.1 to 100.6) | 9.29 (0.1 to 64.7) | 8.29 (0.1 to 46.9) |
Time to 24-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 24-weeks RBC TI period.
| weeks | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Time to the 24-Weeks RBC TI | 8.79 (3.3 to 100.6) | 8.43 (2.1 to 37.6) | 3.79 (0.3 to 7.3) |
Duration of RBC TI was defined as the first day of the longest RBC TI period to the date of the first RBC transfusion after the TI period started. The 95% CI was based on Kaplan-Meier product limit estimates.
| weeks | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Duration of RBC TI | 69.6 (17.00 to 92.43) | 51.6 (26.86 to 80.43) | 13.3 (8.00 to 24.86) |
As per IWG Response Criteria 2006: HI-E was defined as a hemoglobin (Hb) increase by greater than or equal to (≥)1.5 grams per deciliter (g/dL) relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusion units/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of less than or equal to (≤)9 g/dL pretreatment were counted in the RBC transfusion response evaluation. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Percentage of Participants With Hematologic Improvement Including Erythroid Response (HI-E) as Per International Working Group (IWG) Response Criteria 2006 | 61.4 (47.57 to 74.00) | 63.6 (54.20 to 72.22) | 51.7 (38.39 to 64.77) |
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood (PB): Hb ≥11 g/dL; platelets ≥100 x 10\^9/dL; neutrophils ≥1.0 x 10\^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. Percentages were rounded off to the nearest single decimal place. All participants in Phase 2 were evaluated by the Investigator for CR/PR/mCR regardless of bone marrow blasts at baseline.
| percentage of participants | Phase 2: Imetelstat Sodium |
|---|---|
| Percentage of participants with CR | 8.8 |
| Percentage of participants with PR | 0 |
| Percentage of participants with mCR | 12.3 |
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; PB: Hb ≥11 g/dL; platelets ≥100 x 10\^9/dL; neutrophils ≥1.0 x 10\^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. CR, PR and mCR were assessed by IRC in Phase 3 and participants were required to fit at least one of the following criteria (participants with \>5% baseline blasts per central pathology reviewer assessment; participants with CR, PR, mCR, or cytogenetic response as assessed by the investigator) and have at least one post-baseline assessment.
| percentage of participants | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|
| Percentage of participants with CR | 0 | 0 |
| Percentage of participants with PR | 0 | 0 |
| Percentage of participants with mCR | 0 | 0 |
OS was defined as the interval from Study Day 1 to death from any cause. Survival time of living participants was censored on the last date a participant is known to be alive or lost to follow-up. The Kaplan-Meier method was used to estimate overall survival.
| months | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Overall Survival (OS) | 55.3 (39.16 to NA) | 40.4 (37.06 to NA) | NA (32.16 to NA) |
Progression free survival was defined as the time interval from study Day 1 to the first date of disease progression or death from any cause, whichever occurs first. Disease progression as per IWG criteria was defined as: at least one of the following: at least 50 % decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by ≥1.5 g/dL; transfusion dependence.
| months | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Progression Free Survival (PFS) | 34.2 (25.10 to 39.29) | NA (29.24 to NA) | NA (16.72 to NA) |
Time to progression to AML was defined as the interval from Study Day 1 to the date of AML diagnosis. Participants who did not progress to AML and were still alive at the cutoff date for the analysis or who withdrew from the study (withdrawal of consent or lost to follow-up), data was censored at the date of the last disease evaluation.
| months | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Time to Progression to Acute Myeloid Leukemia (AML) | NA (41.36 to NA) | NA (NA to NA) | NA (NA to NA) |
Amount of RBC transfusions for 8-week interval was defined as the total number of RBC transfusion units in a given 8-week interval during study. The best 8-week interval is a post-baseline 8-week interval where the participant had the fewest post-Study Day 1 RBC transfusion units. A valid 8-week period must start before the date of last dose of study drug + 30 days or end of treatment (EOT) visit whichever occurs first and ends before the first transfusion in post-treatment follow-up or the first day of subsequent anti-cancer therapy whichever occurs first.
| number of RBC transfusions units | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Amount of RBC Transfusions in the Best 8-week Interval | 3.0 ± 3.48 | 3.1 ± 3.62 | 3.4 ± 2.13 |
Relative percent change in RBC transfusions per 8-week = (amount of RBC transfusions per 8-week - prior RBC transfusion burden) / prior RBC transfusion burden multiplied by 100%. Prior RBC transfusion burden was defined as the maximum number of RBC units transfused over any consecutive 8 weeks prior to study entry.
| percent change | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Percent Change in RBC Transfusions Relative to Prior Transfusion Burden | -62.5 ± 38.36 | -57.75 ± 50.220 | -51.01 ± 31.606 |
Percentage of participants who received any myeloid growth factors starting from Study Day 1 were reported. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Percentage of Participants Who Received Any Myeloid Growth Factors | 33 | 35.6 | 5.0 |
Duration of myeloid growth factor administered starting from Study Day 1 was reported.
| weeks | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|---|
| Phase 2 and Phase 3: Duration of Myeloid Growth Factors Administration | 1.43 (0.1 to 118.0) | 0.71 (0.1 to 12.9) | 3.14 (0.1 to 10.0) |
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for pharmacokinetic (PK) data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
| micrograms per milliliter (mcg/mL) | Phase 2 and Phase 3: Imetelstat Sodium |
|---|---|
| Phase 2 and Phase 3: Maximum Observed Plasma Concentration (Cmax) | 89.5 ± 27.3 |
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for PK data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
| hour*micrograms per milliliter(h*mcg/mL) | Phase 2 and Phase 3: Imetelstat Sodium |
|---|---|
| Phase 2 and Phase 3: Area Under the Drug Concentration-Plasma Time Curve From Time Zero to Time 28 Days (AUC0-28d) | 559 ± 43.2 |
As pre-specified in the SAP, participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for immunogenicity data collection and analyses in this outcome measure. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 2 and Phase 3: Imetelstat Sodium |
|---|---|
| Phase 2 and Phase 3: Percentage of Participants With Anti-drug Antibodies (ADA) to Imetelstat Sodium | 16.9 |
Outpatient medical encounters included various sites of care: a) emergency room (ER) visits, b) hospital outpatient visits, c) home care visit, d) visit to lab, e) visit to doctor's office, f) other visits. Percentages were rounded off to the nearest single decimal place.
| percentage of participants | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|
| Phase 3: Medical Resource Utilization Assessed Based on Percentage of Participants With Outpatient Medical Encounters | 39.0 | 38.3 |
Hospitalization included any medical encounter defined as hospice, hospital inpatient department, and intensive care unit (ICU). Hospitalizations without end dates were not counted in the calculation of length of stay. If any participant had multiple readmissions, duration was calculated as the sum of all hospital stays.
| days | Phase 3: Imetelstat Sodium | Phase 3: Placebo |
|---|---|---|
| Phase 3: Medical Resource Utilization Assessed Based on Duration of Hospitalization | 8.0 (1 to 47) | 16.5 (1 to 75) |
Baseline was defined as the mean of the measured ECG intervals collected at 3 time points (-1 hour, -0.5 hour, and 0 hour) prior to treatment administration on Cycle 1 Day 1 (Cycle length= 28 days)
| milliseconds (ms) | QTc Substudy: Imetelstat Sodium | QTc Substudy: Placebo |
|---|---|---|
| Change at 0.5 hr post-dose | 4.2 ± 1.54 | 1.9 ± 2.09 |
| Change at 1 hr post-dose | 5.4 ± 1.47 | 0.4 ± 2.00 |
| Change at 2 hr post-dose | 5.5 ± 1.56 | 3.7 ± 2.12 |
| Change at 4 hr post-dose | 1.8 ± 2.43 | 1.1 ± 3.28 |
| Change at 6 hr post-dose | 1.7 ± 2.37 | -1.1 ± 3.12 |
| Change at 8 hr post-dose | 2.3 ± 2.53 | -3.4 ± 3.36 |
An adverse event (AE) was any untoward medical occurrence in a clinical study participant administered a medicinal product. An AE does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to that medicinal product. AEs included clinically significant vital signs measurements, clinical laboratory values and electrocardiograms (ECGs) changes.
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Progression free survival will be assessed as the time interval from the end of the Phase 3 study until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. As per IWG criteria disease progression is defined as: at least one of the following: at least 50 percent (%) decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by greater than or equal to ≥1.5 g/dL; transfusion dependence.
Results for this outcome have not been posted.
Collected over Adverse events: Up to 5 years in Phase 2; up to 3.7 years in Phase 3; up to 1.3 years in QTc substudy; All-cause mortality: Up to 6.6 years in Phase 2; up to 4 years in Phase 3; up to 1.6 years in QTc substudy. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase 2: Imetelstat Sodium (No Dose Escalation) | 24/50 (48%) | 26/50 (52%) | 50/50 (100%) |
| Phase 2: Imetelstat Sodium (Escalated to 9.4 mg/kg) | 3/7 (42.9%) | 1/7 (14.3%) | 6/7 (85.7%) |
| Phase 3: Imetelstat Sodium | 35/118 (29.7%) | 41/118 (34.7%) | 117/118 (99.2%) |
| Phase 3: Placebo | 15/59 (25.4%) | 13/59 (22%) | 58/59 (98.3%) |
| QTc Substudy: Imetelstat Sodium | 1/35 (2.9%) | 8/35 (22.9%) | 33/35 (94.3%) |
| QTc Substudy: Crossover Imetelstat Sodium | 0/14 (0%) | 3/14 (21.4%) | 12/14 (85.7%) |
| QTc Substudy: Placebo | 0/18 (0%) | 0/18 (0%) | 13/18 (72.2%) |
| Event | Phase 2: Imetelstat Sodium (No Dose Escalation) | Phase 2: Imetelstat Sodium (Escalated to 9.4 mg/kg) | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Crossover Imetelstat Sodium | QTc Substudy: Placebo |
|---|---|---|---|---|---|---|---|
| Rectal haemorrhageGastrointestinal disorders | 0/50 | 1/7 | 0/118 | 0/59 | 0/35 | 0/14 | 0/18 |
| SepsisInfections and infestations | 1/50 | 0/7 | 2/118 | 0/59 | 0/35 | 1/14 | 0/18 |
| Urinary tract infectionInfections and infestations | 1/50 | 0/7 | 2/118 | 0/59 | 0/35 | 1/14 | 0/18 |
| AnaemiaBlood and lymphatic system disorders | 1/50 | 0/7 | 3/118 | 0/59 | 0/35 | 1/14 | 0/18 |
| Femur fractureInjury, poisoning and procedural complications | 3/50 | 0/7 | 2/118 | 1/59 | 0/35 | 0/14 | 0/18 |
| COVID-19 pneumoniaInfections and infestations | 1/50 | 0/7 | 2/118 | 3/59 | 0/35 | 0/14 | 0/18 |
| PneumoniaInfections and infestations | 2/50 | 0/7 | 3/118 | 1/59 | 1/35 | 0/14 | 0/18 |
| ThrombocytopeniaBlood and lymphatic system disorders | 2/50 | 0/7 | 0/118 | 0/59 | 0/35 | 0/14 | 0/18 |
| Cardiac failureCardiac disorders | 2/50 | 0/7 | 3/118 | 0/59 | 0/35 | 0/14 | 0/18 |
| Abscess limbInfections and infestations | 0/50 | 0/7 | 0/118 | 2/59 | 0/35 | 0/14 | 0/18 |
| Event | Phase 2: Imetelstat Sodium (No Dose Escalation) | Phase 2: Imetelstat Sodium (Escalated to 9.4 mg/kg) | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Crossover Imetelstat Sodium | QTc Substudy: Placebo |
|---|---|---|---|---|---|---|---|
| NeutropeniaBlood and lymphatic system disorders | 36/50 | 2/7 | 89/118 | 5/59 | 26/35 | 8/14 | 2/18 |
| ThrombocytopeniaBlood and lymphatic system disorders | 33/50 | 2/7 | 89/118 | 6/59 | 25/35 | 9/14 | 3/18 |
| Alanine aminotransferase increasedInvestigations | 7/50 | 3/7 | 13/118 | 4/59 | 6/35 | 4/14 | 1/18 |
| FatigueGeneral disorders | 5/50 | 2/7 | 9/118 | 4/59 | 5/35 | 2/14 | 1/18 |
| AstheniaGeneral disorders | 4/50 | 2/7 | 21/118 | 8/59 | 2/35 | 1/14 | 1/18 |
| Influenza like illnessGeneral disorders | 2/50 | 2/7 | 0/118 | 0/59 | 2/35 | 0/14 | 0/18 |
| DiarrhoeaGastrointestinal disorders | 7/50 | 2/7 | 14/118 | 7/59 | 6/35 | 3/14 | 1/18 |
| Abdominal pain upperGastrointestinal disorders | 3/50 | 2/7 | 2/118 | 4/59 | 0/35 | 0/14 | 0/18 |
| Aspartate aminotransferase increasedInvestigations | 6/50 | 2/7 | 11/118 | 4/59 | 3/35 | 3/14 | 1/18 |
| HeadacheNervous system disorders | 11/50 | 2/7 | 15/118 | 3/59 | 3/35 | 1/14 | 1/18 |
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug. Phase 3: Intent-to-Treat (ITT) Analysis Set included all participants randomized into the phase 3 study. The QTc Substudy Analysis Set included all participants who participated in QTc Substudy and received at least one dose of study drug.
| Age, Continuous(years) | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Placebo | Total |
|---|---|---|---|---|---|---|
| Mean | 70.1 (46 to 83) | 70.4 (44 to 87) | 71.7 (39 to 85) | 69.7 (43 to 84) | 67.8 (54 to 81) | 69.9 (39 to 87) |
| Sex: Female, Male(Participants) | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Placebo | Total |
|---|---|---|---|---|---|---|
| Female | 25 | 47 | 20 | 8 | 2 | 102 |
| Male | 32 | 71 | 40 | 27 | 16 | 186 |
| Ethnicity (NIH/OMB)(Participants) | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Placebo | Total |
|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 6 | 5 | 0 | 0 | 12 |
| Not Hispanic or Latino | 48 | 100 | 48 | 29 | 15 | 240 |
| Unknown or Not Reported | 8 | 12 | 7 | 6 | 3 | 36 |
| Race (NIH/OMB)(Participants) | Phase 2: Imetelstat Sodium | Phase 3: Imetelstat Sodium | Phase 3: Placebo | QTc Substudy: Imetelstat Sodium | QTc Substudy: Placebo | Total |
|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 2 | 8 | 2 | 3 | 0 | 15 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 1 | 1 | 2 | 1 | 1 | 6 |
| White | 46 | 95 | 48 | 29 | 15 | 233 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 8 | 14 | 8 | 2 | 2 | 34 |
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Studies of the drug tested here: Imetelstat
Geron Corporation