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Clinical Trials in the UK / NCT03814746
Active, not recruiting Phase 3

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

NCT03814746 · tracked via the Priya Life Science UK tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2019-07-26
Last updated
2026-09-14

Condition(s) studied

Sickle Cell Disease (SCD)

Investigational drug(s) / intervention(s)

Crizanlizumab (SEG101)Placebo

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)

Trial sites (58)

FacilityCityRegionStatus
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.

More Novartis Pharmaceuticals trials in the UK

Official registry record

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.

View NCT03814746 on ClinicalTrials.gov ↗ ← All trials in the UK