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Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients

NCT03474965 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 117

Last updated 2025-10-16

Study results available
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Summary

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.

Conditions

  • Sickle Cell Disease (SCD)

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-10-01
Primary Completion
2024-11-06
Completion
2024-11-06
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • Canada
  • Colombia
  • France
  • Germany
  • India
  • Italy
  • Lebanon
  • Oman
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03474965 on ClinicalTrials.gov