Crizanlizumab (SEG101): 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.
Placebo: 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.
Study summary
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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
100 Years
Healthy volunteers
No
Key Inclusion Criteria:
1. Written informed consent must be obtained prior to any screening procedures
2. Male or female patients aged 12 years and older on the day of signing informed consent. Adolescent include patients aged 12 to 17 years old and adults ≥ 18 years
3. Confirmed diagnosis of SCD by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) \[performed locally\]. All SCD genotypes are eligible, genotyping is not required for study entry
4. 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:
1. Pain crisis defined as an acute onset of pain for which there is no other medically determined explanation other than vaso- occlusion -
2. which requires a visit to a medical facility and/or healthcare professional,
3. and receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug (NSAID) analgesia Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study
5. If receiving HU/HC or L-glutamine (local HA approved medicinal product), must have been receiving the drug for at least 6 months and at a stable dose for at least 3 months prior to Screening visit and plan to continue taking it at the same dose and schedule until the subject has reached one year of study treatment. Patients who have not been receiving such drug must not have received it for at least 6 months prior to Screening visit to be included. Patients must have evidence of insufficient control of acute pain, such as at least one VOC leading to healthcare visit while on HU/HC or L-Glutamine treatment. If receiving erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening visit and plan to continue taking the treatment to maintain stable Hb levels at least until the subject has reached one year of study treatment
6. Patients must meet the following central laboratory values prior to Week 1 Day 1:
* Absolute Neutrophil Count ≥1.0 x 109/L
* Platelet count ≥75 x 109/L
* Hemoglobin: for adults (Hb) ≥4.0 g/dL and for adolescents (Hb) ≥5.5 g/dL
* Glomerular filtration rate ≥ 45 mL/min/1.73 m2 using CKD-EPI formula in adults, and Shwartz formula in adolescents
* Direct (conjugated) bilirubin \< 2.0 x ULN
* Alanine transaminase (ALT) \< 3.0 x ULN
7. ECOG performance status ≤2.0 for adults and Karnofsky ≥ 50% for adolescents
Key Exclusion Criteria:
1. History of stem cell transplant.
2. Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted.
3. Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug or to any excipients of the study drug formulation. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
4. Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to Screening visit or plans to participate in another investigational drug trial.
5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant unless they are using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment.
6. Concurrent severe and/or uncontrolled medical conditions which, in the opinion of the Investigator, could cause unacceptable safety risks or compromise participation in the study.
7. History or current diagnosis of ECG abnormalities indicating significant risk of safety such as:
* Concomitant clinically significant cardiac arrhythmias (e.g ventricular tachycardia), and clinically significant second or third degree AV block without a pacemaker
* History of familial long QT syndrome or know family history of Torsades de Pointes
8. Not able to understand and to comply with study instructions and requirements.
9. Received prior treatment with crizanlizumab or other selectin targeting agent
Primary outcome measure(s)
Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit — 1 year 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).
Trial sites (58)
Facility
City
Region
Status
Childrens Healthcare of Atlanta
Atlanta
Georgia
Boston Medical Center
Boston
Massachusetts
Levine Cancer Insitute Carolinas Healthcare System
Charlotte
North Carolina
Univ of Tenn Health Sciences Ctr
Memphis
Tennessee
U of TX Health Science Ct
Houston
Texas
Novartis Investigative Site
Brussels
Brussels Capital
Novartis Investigative Site
Brussels
Belgium
Novartis Investigative Site
Edegem
Belgium
Novartis Investigative Site
Salvador
Estado de Bahia
Novartis Investigative Site
Belém
Pará
Novartis Investigative Site
Recife
Pernambuco
Novartis Investigative Site
Rio de Janeiro
Rio de Janeiro
Novartis Investigative Site
Porto Alegre
Rio Grande do Sul
Novartis Investigative Site
Ribeirão Preto
São Paulo
Novartis Investigative Site
São Paulo
São Paulo
Novartis Investigative Site
São Paulo
São Paulo
Novartis Investigative Site
São Paulo
São Paulo
Novartis Investigative Site
Toronto
Ontario
Novartis Investigative Site
Montreal
Quebec
Novartis Investigative Site
Barranquilla
Atlántico
Novartis Investigative Site
Valledupar
Cesar Department
Novartis Investigative Site
Montería
Colombia
Novartis Investigative Site
Helsinki
Finland
Novartis Investigative Site
Créteil
France
Novartis Investigative Site
Marseille
France
Novartis Investigative Site
Paris
France
Novartis Investigative Site
Stuttgart
Baden-Wurttemberg
Novartis Investigative Site
Cologne
North Rhine-Westphalia
Novartis Investigative Site
Berlin
Germany
Novartis Investigative Site
Essen
Germany
Novartis Investigative Site
Accra
Ghana
Novartis Investigative Site
Athens
Greece
Novartis Investigative Site
Pátrai
Greece
Novartis Investigative Site
Thessaloniki
Greece
Novartis Investigative Site
Bhubaneswar
Odisha
Novartis Investigative Site
Hyderabad
Telangana
Novartis Investigative Site
Genova
GE
Novartis Investigative Site
Milan
MI
Novartis Investigative Site
Verona
VR
Novartis Investigative Site
Naples
Italy
+ 18 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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