NCT04543591

Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants Who Have Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 148 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). In Stage 1, an open-label, single-arm period, the dosing regimen will be confirmed. In Stage 2, participants will be randomized to receive either blinded ravulizumab plus best supportive care or matching placebo plus best supportive care. The treatment period is 26 weeks (open-label for Stage 1, and randomized, double-blind, and placebo-controlled for Stage 2) followed by a 26-week follow-up period.

Conditions

• Thrombotic Microangiopathy

Interventions

Type	Name	Description
BIOLOGICAL	Ravulizumab	Weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.
OTHER	Placebo	Matching placebo
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: 2020-12-10
• Primary completion: 2025-09-19
• Completion: 2026-03-20
• First posted: 2020-09-10
• Last updated: 2026-04-09

Locations

66 sites across 17 countries: United States, Australia, Belgium, Brazil, Canada, China, France, Germany, Greece, Israel, Italy, Japan, Netherlands, South Korea, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study