Ignite Creation Date:
2025-12-24 @ 10:09 PM
Ignite Modification Date:
2025-12-30 @ 2:25 AM
Study NCT ID:
NCT04557735
Status:
COMPLETED
Last Update Posted:
2025-10-02
First Post:
2020-09-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
Sponsor:
Alexion Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplantation (HSCT)
Status:
COMPLETED
Status Verified Date:
2025-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to \< 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: