A Phase 3 interventional study of Crizanlizumab (SEG101) and Placebo in Sickle Cell Disease (SCD), sponsored by Novartis Pharmaceuticals. Active, not recruiting at 58 sites in 21 countries. Open to participants aged 12 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.
Study CSEG101A2301 (STAND) is an ongoing Phase III, multicenter, randomized, double-blind study to assess efficacy and safety of two doses of crizanlizumab (5 mg/kg and 7.5 mg/kg) versus placebo, with or without hydroxyurea/ hydroxycarbamide therapy (HU/HC), in adolescent and adult patients with SCD and history of VOC leading to healthcare visit.
This is a multicenter clinical trial comparing 2 doses of crizanlizumab (5 mg/kg and 7.5 mg/kg) versus placebo in addition to standard of care participants might be taking at the time of study start, in adolescent and adult participants with confirmed diagnosis of sickle cell disease (SCD) and history of vaso-occlusive crisis (VOC) leading to a healthcare visit.
240 participants (including 48 adolescents) were planned to be randomized in a 1:1:1 ratio to either 5 mg/kg, 7.5 mg/kg of crizanlizumab or placebo. Randomized participants were stratified by concomitant HU/HC usage (yes/no) and baseline rate of VOCs leading to a healthcare visit in 12 months prior to screening visit (2-4 vs. ≥ 5 VOCs) at the time of enrollment. In November 2020, a capping of 90 adult participants per strata was implemented to ensure adequate opportunity for enrollment into each of the 4 strata.
104 studies on the registry are indexed under Vaso-Occlusive Crises; 31 are open to participants now.
This study's enrollment of 253 is above the median of 81 across 86 interventional studies indexed under Vaso-Occlusive Crises.
Browse Vaso-Occlusive Crises studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Compared within Vaso-Occlusive Crises, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Anemia, Sickle Cell. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria:
Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history. Prior VOC leading to healthcare visit must resolve at least 7 days prior to Week 1 Day 1 and must include:
Patients must meet the following central laboratory values prior to Week 1 Day 1:
Key Exclusion Criteria:
History or current diagnosis of abnormalities indicating significant risk of safety such as:
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.
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.
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).
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.
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.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
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.
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 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
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.
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.
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.
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.
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.
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.
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.
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.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
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.
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.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
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.
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.
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.
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.
Participants received Crizanlizumab (SEG101) at 5.0 mg/kg
Drug: Crizanlizumab (SEG101)
Participants received Crizanlizumab (SEG101) at 7.5 mg/kg
Drug: Crizanlizumab (SEG101)
Participants received the placebo drug.
Drug: Placebo
Crizanlizumab was supplied in single use 10 mL glass vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab. This is a concentrate for solution for infusion. IV.
Also known as: SEG101
Placebo was supplied in single use 10 mL glass vials at a concentration of 0 mg/mL. This is a concentrate for solution for infusion IV.
Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit
VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit is defined as any visit to a medical facility such as emergency room (ER), hospital and/or office visit, which includes pain management of VOC in situ. Annualized rate of corresponding VOC events = (Number of corresponding VOC events \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
Time frame: 1 year
Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary)
VOCs are based on documentation by provider following contact with participant. VOC:pain crisis requiring therapy with oral/parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome, priapism and hepatic or splenic sequestration. Healthcare visit:a visit to a medical facility (ER, hospital \&/or office visit resulting in pain management of VOC. Managed at home: no visit to any medical facility \&/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice is allowed. Annualized rate of corresponding VOC events = (# of corresponding VOC events \* 365)/(# of days in observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor \& erythropoietin therapies to treat SCD \&/or to prevent/reduce VOCs), date of randomization + 365 days)
Time frame: 1 year
Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over 5 Years Post Randomization (Key Secondary)
To compare the efficacy of 5.0 mg/kg vs placebo \& 7.5 mg/kg vs placebo on the annualized rate of all VOCs based on documentation by provider following contact with participant. VOC is defined as pain crisis (an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) requiring therapy with oral/parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome, priapism and hepatic or splenic sequestration. Healthcare visit is defined as a visit to a medical facility (emergency room, hospital and/or office visit resulting in pain management of VOC. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice is allowed. The annualized rate of VOC leading to healthcare visit is the number of VOC leading to healthcare visit multiplied by 365 \& divided by the number of days in observation period.
Time frame: 5 years
Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
To assess the duration of VOCs leading to healthcare visit in each group. Mean duration of VOC per participant is defined as the average duration of all individual episodes of VOCs leading to healthcare visits of a given participant (a VOC duration is defined as end date of the VOC - start date of the VOC + 1). Participants with no VOC leading to healthcare visits have been excluded.
Time frame: 1 year
Number of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
To assess the number of participants free from VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. A participant is free from VOC if they do not have a VOC crisis.
Time frame: 1 year
Percentage of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
To assess the percentage of participants free from VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. A participant is free from VOC if they do not have a VOC crisis.
Time frame: 1 year
Time to First and Second VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
To assess the time to first and second VOC leading to healthcare visit in each group. Time to first occurrence of VOC leading to a healthcare visit is defined as the time from the date of randomization to the date of the first occurrence of the VOC. Time to second occurrence of VOC leading to a healthcare visit is defined as the time from date of randomization to the date of the second occurrence of VOC.
Time frame: 1 year
Annualized Rate of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization
To assess Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) both overall and VOC-related in each group. Annualized rate of corresponding healthcare visits =(Number of corresponding healthcare visits \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days)
Time frame: 1 year
Annualized Days of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization
To assess Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) both overall and VOC-related in each group. Annualized days of corresponding healthcare visits =(Number of days =(Number of days of corresponding healthcare visits \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
Time frame: 1 year
Evolution of Albumin Creatinine Ratio (ACR) Over the First-year Post-randomization (Change From Baseline)
Laboratory values for parameters related to renal function (creatinine, estimated glomerular filtration rate, urine microalbumin, and urine albumin/creatinine ratio) were measured at 6-month intervals over time from baseline.
Time frame: Over first year post-randomization (Baseline, Week 27 Day 1, Week 51 Day 1)
Evolution of Albuminuria (Urine Microalbumin) Over the First-year Post-randomization (Change From Baseline)
Laboratory values for parameters related to renal function (creatinine, estimated glomerular filtration rate, urine microalbumin, and urine albumin/creatinine ratio) were measured at 6-month intervals over time from baseline.
Time frame: Over first year post-randomization (Baseline, Week 27 Day 1, Week 51 Day 1)
Pharmacokinetic (PK) Profile of Crizanlizumab: AUCd15, AUCtau
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Area under the (concentration-time profile) curve.
Time frame: AUCd15 (first-dose) was assessed at W1D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose) to W3D1; AUCtau (steady-state) was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PK Profile of Crizanlizumab: Cmax
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Maximum concentration.
Time frame: first-dose was assessed at W1D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose) through to W3D1; steady-state was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PK Profile of Crizanlizumab: Tmax
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Time to maximum concentration.
Time frame: first-dose was assessed at W1D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose) through to W3D1; steady-state was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PK Profile of Crizanlizumab: Half-life
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: half life.
Time frame: steady-state was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PD Parameter (P-selectin Inhibition)
To characterize the pharmacodynamic (PD) of crizanlizumab at 5.0 and 7.5 mg/kg: P-selectin inhibition (% inhibition multipled by hr)
Time frame: AUCd15 (first dose): W1D1, W1D2, W1D4, W2D1 and W3D1; steady state: W15D1, W15D2, W15D4, W16D1, W17D1 W18D1 and W19D1
Annualized Rate of Various Subtypes of VOCs Leading to a Healthcare Visit at 1 Year
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOCs leading to healthcare visit. VOC is defined as pain crisis which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit: any visit to a medical facility such as ER, hospital and/or office visit, which includes pain management of VOC in situ. Annualized rate of corresponding VOC events = (Number of corresponding VOC events \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
Time frame: 1 year
Annualized Rate of Various Subtypes of VOCs Leading to a Healthcare Visit at 5 Years
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit is defined as any visit to a medical facility such as emergency room, hospital and/or office visit, which includes pain management of VOC in situ. The annualized rate of VOC leading to healthcare visit is the number of VOC leading to healthcare visit multiplied by 365 and divided by the number of days in the observation period.
Time frame: 5 years
Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home at 5 Years
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the rates of all VOCs (managed at home + leading to healthcare visit).
Time frame: 5 years
Number of VOCs Managed at Home at Year 1
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the number of VOC events that were managed at home. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice was allowed.
Time frame: 1 year
Number of VOCs Managed at Home at 5 Years
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the number of VOC events that were managed at home. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice was allowed.
Time frame: 5 years
Absolute Change From Baseline in Hemoglobin
To assess safety of crizanlizumab over the study period.
Time frame: 5 years
Growth and Sexual Maturity Assessment in Adolescents (Tanner Stage)
To assess safety of crizanlizumab over the study period.
Time frame: 5 years
Immunogenicity: Measurement of Anti-drug Antibodies (ADA) to Crizanlizumab
To assess immunogenicity of crizanlizumab over the study period.
Time frame: 5 years
240 participants (including 48 adolescents) were planned to be randomized in a 1:1:1 ratio to either 5 mg/kg, 7.5 mg/kg of crizanlizumab or placebo. This study was conducted in 21 countries and 63 centers.
| Milestone | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Started | 84 | 83 | 85 |
| Completed | 66 | 68 | 58 |
| Not completed | 18 | 15 | 27 |
| Withdrew: Participant decision | 9 | 6 | 15 |
| Withdrew: Physician decision | 2 | 2 | 4 |
| Withdrew: Death | 2 | 2 | 2 |
| Withdrew: Adverse event | 2 | 2 | 1 |
| Withdrew: Lost to follow-up | 2 | 1 | 2 |
| Withdrew: Unsatisfactory therapeutic effect | 0 | 1 | 2 |
| Withdrew: Pregnancy | 0 | 1 | 1 |
| Withdrew: Protocol violation | 1 | 0 | 0 |
VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit is defined as any visit to a medical facility such as emergency room (ER), hospital and/or office visit, which includes pain management of VOC in situ. Annualized rate of corresponding VOC events = (Number of corresponding VOC events \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
| number of events per year | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit | 2.5 ± 2.98 | 1.9 ± 2.30 | 2.1 ± 2.81 |
VOCs are based on documentation by provider following contact with participant. VOC:pain crisis requiring therapy with oral/parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome, priapism and hepatic or splenic sequestration. Healthcare visit:a visit to a medical facility (ER, hospital \&/or office visit resulting in pain management of VOC. Managed at home: no visit to any medical facility \&/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice is allowed. Annualized rate of corresponding VOC events = (# of corresponding VOC events \* 365)/(# of days in observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor \& erythropoietin therapies to treat SCD \&/or to prevent/reduce VOCs), date of randomization + 365 days)
| number of events per year | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary) | 4.5 ± 6.49 | 3.1 ± 2.89 | 3.7 ± 3.78 |
To compare the efficacy of 5.0 mg/kg vs placebo \& 7.5 mg/kg vs placebo on the annualized rate of all VOCs based on documentation by provider following contact with participant. VOC is defined as pain crisis (an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) requiring therapy with oral/parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome, priapism and hepatic or splenic sequestration. Healthcare visit is defined as a visit to a medical facility (emergency room, hospital and/or office visit resulting in pain management of VOC. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice is allowed. The annualized rate of VOC leading to healthcare visit is the number of VOC leading to healthcare visit multiplied by 365 \& divided by the number of days in observation period.
Results for this outcome have not been posted.
To assess the duration of VOCs leading to healthcare visit in each group. Mean duration of VOC per participant is defined as the average duration of all individual episodes of VOCs leading to healthcare visits of a given participant (a VOC duration is defined as end date of the VOC - start date of the VOC + 1). Participants with no VOC leading to healthcare visits have been excluded.
| days | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 7.7 ± 6.93 | 6.0 ± 4.54 | 6.6 ± 5.55 |
To assess the number of participants free from VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. A participant is free from VOC if they do not have a VOC crisis.
| Participants | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Number of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 25 | 31 | 34 |
To assess the percentage of participants free from VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. A participant is free from VOC if they do not have a VOC crisis.
| Percentage of participants | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Percentage of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 29.8 | 37.3 | 40.0 |
To assess the time to first and second VOC leading to healthcare visit in each group. Time to first occurrence of VOC leading to a healthcare visit is defined as the time from the date of randomization to the date of the first occurrence of the VOC. Time to second occurrence of VOC leading to a healthcare visit is defined as the time from date of randomization to the date of the second occurrence of VOC.
| Months | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Time to first occurrence of VOC | 3.9 (2.4 to 6.0) | 6.2 (3.2 to 10.3) | 6.2 (2.8 to 11.9) |
| Time to second occurrence of VOC | 10.6 (7.7 to NA) | NA (10.2 to NA) | NA (8.9 to NA) |
To assess Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) both overall and VOC-related in each group. Annualized rate of corresponding healthcare visits =(Number of corresponding healthcare visits \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days)
| number of events per year | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Annualized rate of all clinic, hospitalizations and ER visits | 3.5 ± 3.65 | 2.6 ± 3.21 | 3.0 ± 3.87 |
| Annualized rate of VOC-related clinic, hospitalizations and ER visits | 3.0 ± 3.52 | 2.2 ± 3.01 | 2.5 ± 3.53 |
To assess Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) both overall and VOC-related in each group. Annualized days of corresponding healthcare visits =(Number of days =(Number of days of corresponding healthcare visits \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
| number of days per year | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Annualized days of all clinic, hospitalizations and ER visits | 17.6 ± 27.80 | 11.3 ± 14.29 | 12.9 ± 18.32 |
| Annualized days of all VOC-related clinic, hospitalizations and ER visits | 13.8 ± 18.12 | 9.3 ± 12.62 | 11.3 ± 18.35 |
Laboratory values for parameters related to renal function (creatinine, estimated glomerular filtration rate, urine microalbumin, and urine albumin/creatinine ratio) were measured at 6-month intervals over time from baseline.
| g/mol | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Change from baseline at Week 27 Day 1 (n = 51, 64, 54) | 0.1 (-30 to 96) | -0.0 (-157 to 28) | -0.1 (-20 to 40) |
| Chage from baseline at Week 51 Day 1 (n = 59, 58, 53) | 0.1 (-34 to 187) | -0.1 (-103 to 49) | -0.2 (-21 to 59) |
Laboratory values for parameters related to renal function (creatinine, estimated glomerular filtration rate, urine microalbumin, and urine albumin/creatinine ratio) were measured at 6-month intervals over time from baseline.
| g/L | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Change from baseline at Week 27 Day 1 (n = 65, 70, 64) | 0.0 (-1 to 1) | 0.0 (-1 to 0) | 0.0 (-0 to 0) |
| Change from baseline at Week 51 Day 1 (n = 68, 64, 61) | 0.0 (-1 to 1) | 0.0 (-1 to 1) | 0.0 (-0 to 0) |
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Area under the (concentration-time profile) curve.
| hr*ug/mL | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg |
|---|---|---|
| AUCd15 (first dose) (n = 64, 66) | 11300 ± 2950 | 17000 ± 4100 |
| AUCtau (steady state) (n = 44, 44) | 18800 ± 5470 | 30200 ± 8580 |
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Maximum concentration.
| ug/mL | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg |
|---|---|---|
| Cmax (first dose) (n = 74, 75) | 95.9 ± 29.4 | 143 ± 45.3 |
| Cmax (steady state) (n = 47, 52) | 108 ± 60.2 | 162 ± 57.4 |
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Time to maximum concentration.
| hr | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg |
|---|---|---|
| Tmax (first dose) (n = 74, 75) | 2.08 (0.467 to 25.5) | 2.08 (0.500 to 24.2) |
| Tmax (steady state) (n = 47, 52) | 2.00 (0.25 to 24.9) | 3.58 (0.50 to 24.9) |
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: half life.
| day | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg |
|---|---|---|
| PK Profile of Crizanlizumab: Half-life | 9.51 ± 3.67 | 11.2 ± 3.55 |
To characterize the pharmacodynamic (PD) of crizanlizumab at 5.0 and 7.5 mg/kg: P-selectin inhibition (% inhibition multipled by hr)
| h * % | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg |
|---|---|---|
| PD-AUCd15 (first dose) (n = 55, 61) | 32700 ± 3830 | 33100 ± 3530 |
| PD-AUCd29 (steady state) (n = 36, 39) | 65100 ± 7000 | 66100 ± 7190 |
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOCs leading to healthcare visit. VOC is defined as pain crisis which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit: any visit to a medical facility such as ER, hospital and/or office visit, which includes pain management of VOC in situ. Annualized rate of corresponding VOC events = (Number of corresponding VOC events \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).
| number of events per year | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Subtype of VOC: Uncomplicated sickle cell-vaso-occlusive crisis | 2.3 ± 2.74 | 1.8 ± 2.27 | 2.0 ± 2.79 |
| Subtype of VOC: Acute chest syndrome | 0.2 ± 1.44 | 0.0 ± 0.21 | 0.1 ± 0.44 |
| Subtype of VOC: Hepatic sequestration | 0.0 ± 0.00 | 0.0 ± 0.00 | 0.0 ± 0.00 |
| Subtype of VOC: Splenic sequestration | 0.0 ± 0.00 | 0.0 ± 0.11 | 0.0 ± 0.00 |
| Subtype of VOC: Priapism | 0.0 ± 0.00 | 0.0 ± 0.15 | 0.0 ± 0.11 |
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit is defined as any visit to a medical facility such as emergency room, hospital and/or office visit, which includes pain management of VOC in situ. The annualized rate of VOC leading to healthcare visit is the number of VOC leading to healthcare visit multiplied by 365 and divided by the number of days in the observation period.
Results for this outcome have not been posted.
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the rates of all VOCs (managed at home + leading to healthcare visit).
Results for this outcome have not been posted.
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the number of VOC events that were managed at home. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice was allowed.
| total number of VOC events | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo |
|---|---|---|---|
| Number of VOCs Managed at Home at Year 1 | 163 | 106 | 129 |
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the number of VOC events that were managed at home. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice was allowed.
Results for this outcome have not been posted.
To assess safety of crizanlizumab over the study period.
Results for this outcome have not been posted.
To assess safety of crizanlizumab over the study period.
Results for this outcome have not been posted.
To assess immunogenicity of crizanlizumab over the study period.
Results for this outcome have not been posted.
Collected over This primary analysis report includes data with a cut-off date 31-Aug-2022. Adverse events and deaths were collected from first dose of study treatment to105 days post-last infusion (defined as on-treatment), up to max on-treatment period of approx. 5 years.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 2/84 (2.4%) | 35/84 (41.7%) | 67/84 (79.8%) |
| Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 2/83 (2.4%) | 22/83 (26.5%) | 71/83 (85.5%) |
| Placebo (On-treatment) | 2/85 (2.4%) | 26/85 (30.6%) | 66/85 (77.6%) |
| Event | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | Placebo (On-treatment) |
|---|---|---|---|
| COVID-19Infections and infestations | 5/84 | 6/83 | 4/85 |
| PneumoniaInfections and infestations | 6/84 | 5/83 | 1/85 |
| Pulmonary embolismRespiratory, thoracic and mediastinal disorders | 4/84 | 1/83 | 1/85 |
| AnaemiaBlood and lymphatic system disorders | 1/84 | 3/83 | 1/85 |
| HaemolysisBlood and lymphatic system disorders | 2/84 | 3/83 | 0/85 |
| SepsisInfections and infestations | 3/84 | 1/83 | 2/85 |
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 0/84 | 2/83 | 0/85 |
| PyrexiaGeneral disorders | 1/84 | 2/83 | 0/85 |
| Urinary tract infectionInfections and infestations | 0/84 | 2/83 | 1/85 |
| Haemolytic anaemiaBlood and lymphatic system disorders | 2/84 | 0/83 | 0/85 |
| Event | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | Placebo (On-treatment) |
|---|---|---|---|
| PyrexiaGeneral disorders | 22/84 | 17/83 | 22/85 |
| COVID-19Infections and infestations | 16/84 | 21/83 | 14/85 |
| HeadacheNervous system disorders | 21/84 | 13/83 | 15/85 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 11/84 | 17/83 | 12/85 |
| Back painMusculoskeletal and connective tissue disorders | 10/84 | 11/83 | 16/85 |
| NauseaGastrointestinal disorders | 14/84 | 8/83 | 8/85 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 10/84 | 10/83 | 13/85 |
| FatigueGeneral disorders | 6/84 | 11/83 | 3/85 |
| Urinary tract infectionInfections and infestations | 9/84 | 8/83 | 8/85 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 9/84 | 6/83 | 8/85 |
The FAS comprises all participants to whom study treatment has been assigned by randomization.
| Age, Customized(participants) | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Adolescents, 12 - < 18 years | 16 | 18 | 20 | 54 |
| 18 - < 65 years | 68 | 65 | 63 | 196 |
| 65 - < 85 years | 0 | 0 | 2 | 2 |
| Sex: Female, Male(Participants) | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Female | 45 | 45 | 49 | 139 |
| Male | 39 | 38 | 36 | 113 |
| Ethnicity (NIH/OMB)(Participants) | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Hispanic or Latino | 22 | 20 | 18 | 60 |
| Not Hispanic or Latino | 54 | 56 | 57 | 167 |
| Unknown or Not Reported | 8 | 7 | 10 | 25 |
| Race/Ethnicity, Customized(Participants) | Crizanlizumab (SEG101) at 5.0 mg/kg | Crizanlizumab (SEG101) at 7.5 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Black or African American | 46 | 34 | 43 | 123 |
| White | 27 | 31 | 26 | 84 |
| Asian | 6 | 7 | 6 | 19 |
| American Indian or Alaska Native | 3 | 7 | 6 | 16 |
| Multiple (White and Black or African American) | 1 | 0 | 0 | 1 |
| Unknown | 1 | 4 | 4 | 9 |
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: Crizanlizumab