Trials / Completed
CompletedNCT04557735
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
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
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 41 (actual)
- Sponsor
- Alexion Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 17 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Ravulizumab | Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight. |
| OTHER | Best Supportive Care | Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol). |
Timeline
- Start date
- 2020-12-07
- Primary completion
- 2024-11-26
- Completion
- 2025-05-27
- First posted
- 2020-09-22
- Last updated
- 2026-01-12
- Results posted
- 2026-01-12
Locations
26 sites across 7 countries: United States, Israel, Italy, Japan, South Korea, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04557735. Inclusion in this directory is not an endorsement.