Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03474965 |
Recruitment Status :
Recruiting
First Posted : March 23, 2018
Last Update Posted : January 23, 2023
|
Tracking Information | |||||||||
---|---|---|---|---|---|---|---|---|---|
First Submitted Date ICMJE | March 16, 2018 | ||||||||
First Posted Date ICMJE | March 23, 2018 | ||||||||
Last Update Posted Date | January 23, 2023 | ||||||||
Actual Study Start Date ICMJE | October 1, 2018 | ||||||||
Estimated Primary Completion Date | May 31, 2024 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
|
||||||||
Original Primary Outcome Measures ICMJE |
|
||||||||
Change History | |||||||||
Current Secondary Outcome Measures ICMJE |
|
||||||||
Original Secondary Outcome Measures ICMJE |
|
||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients | ||||||||
Official Title ICMJE | A Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide,in Sequential,Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis | ||||||||
Brief Summary | The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study. | ||||||||
Detailed Description | Not Provided | ||||||||
Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 2 | ||||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
||||||||
Condition ICMJE | Sickle Cell Disease (SCD) | ||||||||
Intervention ICMJE | Drug: Crizanlizumab
Crizanlizumab (SEG101) is a concentrate for solution for infusion, i.v. use. Supplied in single use 10 mL vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab.
Other Name: SEG101
|
||||||||
Study Arms ICMJE | Experimental: Crizanlizumab
SEG101 (crizanlizumab) administered on Week 1 Day 1, Week3 Day 1 and Day 1 of every 4-week cycle
Intervention: Drug: Crizanlizumab
|
||||||||
Publications * | Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available. | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
|||||||||
Recruitment Information | |||||||||
Recruitment Status ICMJE | Recruiting | ||||||||
Estimated Enrollment ICMJE |
119 | ||||||||
Original Estimated Enrollment ICMJE |
100 | ||||||||
Estimated Study Completion Date ICMJE | December 17, 2025 | ||||||||
Estimated Primary Completion Date | May 31, 2024 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
6.Contraindication or hypersensitivity to any drug from similar class as study drug or to any excipients of the study drug formulation. 7.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 8.Received a monoclonal antibody or immunoglobulin-based therapy within 6 months of Screening, or has documented immunogenicity to a prior monoclonal antibody. 9.Received active treatment on another investigational trial within 30 days (or 5 half -lives of that agent, whichever is greater) prior to Screening or plans to participate in another investigational drug trial. 10.Pregnant females or females who have given birth within the past 90 days or who are breastfeeding. 11.Any documented history of a clinical stroke or intracranial hemorrhage, or an uninvestigated neurologic finding within the past 12 months. Silent infarcts (only present on imaging) are not excluding patients from study participation. 12.Any abnormal TCD within the past 12 months. 13.Use of therapeutic anticoagulation (prophylactic doses permitted) or antiplatelet therapy (other than aspirin) within the 10 days prior to Week 1 Day 1 dosing. 14.Hospitalized within 7 days prior to Week 1 Day 1 dosing. 15.Planning to undergo a major surgical procedure during the duration of the study. 16.Planning to initiate or terminate HU/HC or L-glutamine while on study (except if needed to terminate for safety reasons). 17.Patient with active human immunodeficiency virus (HIV) infection (detectable viral load). 18.Patients with known active Hepatitis B infection. 19.Patients with known Hepatitis C history. 20.Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator. 21.Malignant disease. Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 2 years prior to study treatment; any completely resected carcinoma in situ. 22.Has a serious mental or physical illness, which, in the opinion of the Investigator would compromise participation in the study. 23.Any condition which, in the opinion of the investigator, is likely to interfere with the successful collection of the measurements required for the study 24.Resting QTcF ≥450 msec at pretreatment (baseline) for patients under 12 years of age and ≥450 msec for males and ≥460 msec for female patients 12 years and older. 25.Cardiac or cardiac repolarization abnormality, including any of the following: a. History of myocardial infarction (MI), uncontrolled congestive heart failure, unstable angina, or coronary bypass graft (CABG) within 6 months prior to starting study treatment, b. Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade AV block (bifascicular block, Mobitz Type II, and third degree AV block), c. Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome ( Risk factors for Torsade de Pointes (TdP), including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure, or history of clinically significant/ symptomatic bradycardia, Inability to determine the QTcF). 26. Sexually active females who are unwilling to comply with reliable method of birth control until 15 weeks following last dose of study drug. 28.Not able to understand and to comply with study instructions and requirements. 29.Patients who are an employee of the sponsor or investigator or otherwise dependent on them. 30.Patients who are committed to an institution by virtue of an order issued either by the judicial or the administrative authorities. 31.Patients who received prior crizanlizumab treatment and/or other selectin targeting agents are not allowed 32.Patients having taken voxelotor less than 30 days prior to Screening, or planning to take voxelotor while on study are not allowed. |
||||||||
Sex/Gender ICMJE |
|
||||||||
Ages ICMJE | 6 Months to 17 Years (Child) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE |
|
||||||||
Listed Location Countries ICMJE | Belgium, Brazil, Canada, Colombia, France, Germany, India, Italy, Lebanon, Oman, Spain, Switzerland, Turkey, United Kingdom, United States | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number ICMJE | NCT03474965 | ||||||||
Other Study ID Numbers ICMJE | CSEG101B2201 2017-001747-12 ( EudraCT Number ) |
||||||||
Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product |
|
||||||||
IPD Sharing Statement ICMJE |
|
||||||||
Current Responsible Party | Novartis ( Novartis Pharmaceuticals ) | ||||||||
Original Responsible Party | Same as current | ||||||||
Current Study Sponsor ICMJE | Novartis Pharmaceuticals | ||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||
Collaborators ICMJE | Not Provided | ||||||||
Investigators ICMJE |
|
||||||||
PRS Account | Novartis | ||||||||
Verification Date | January 2023 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |