Clinical Trial
Study Details
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.
The purpose of this study was to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 2 to \<18 years with a history of Vaso-Occlusive Crisis (VOC) with or without Hydroxyurea/Hydroxycarbamide (HU/HC), receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was previously demonstrated in adults with sickle cell disease. The approach was to extrapolate from the pharmacokinetics (PK)/pharmacodynamics (PD) already established in the adult population. The study was designed as a Phase II, multicenter, open-label study.
Study Overview
This was an open-label, single-arm study of crizanlizumab in sickle-cell disease (SCD) pediatric participants.
This study consisted of 2 parts, Part A and Part B. In Part A, the dose for 3 age groups (see groups below), was first confirmed on the basis of single and multiple dose (steady state) PK data and key safety data from an initial subgroup of participants. In Part B, safety and efficacy were collected from additional participants from 6 to \<18 years (Groups 1 and 2, only).
At least 100 participants were planned to be enrolled in the trial in total, split in 3 age groups:
* Group 1 (age 12 to \<18 years): at least 26 participants (≥8 in Part A and ≥18 in Part B),
* Group 2 (age 6 to \<12 years): at least 26 participants (≥8 in Part A and ≥18 in Part B),
* Group 3 (age 2 to \<6 years): at least 8 participants (≥8 Part A). Crizanlizumab was administered every 4 weeks with a loading dose 2 weeks after the first dosing (i.e., by i.v. infusion) on Week 1 Day 1, Week 3 Day 1, and then day 1 of every fourth week) for up to 2 years. The initial dose of crizanlizumab was 5 mg/kg dose for Group 1 and Group 2 Part A. It was later adjusted to 8.5 mg/kg for Group 2 and 3 based on final dose confirmation from emerging PK data analysis and safety considerations determined in Part A of the study.
Contacts and Locations
University Of Alabama
Birmingham, Alabama 35233
United States
Childrens National Hospital
Washington D.C., District of Columbia 20010
United States
University of Florida
Gainesville, Florida 32610
United States
Joe DiMaggio Childrens Hospital
Hollywood, Florida 33021
United States
Childrens Healthcare of Atlanta
Atlanta, Georgia 30342
United States
Childrens Hosp Boston Dept of Hematology
Boston, Massachusetts 02115
United States
Childrens Hospital at Montefiore
The Bronx, New York 10467
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
East Carolina University
Greenville, North Carolina 27834
United States
Childrens Hospital Of Philadelphia
Philadelphia, Pennsylvania 19104-4399
United States
Medical Uni of South Carolina
Charleston, South Carolina 29425
United States
Cook Childrens Medical Center
Fort Worth, Texas 76104
United States
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