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Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

NCT04543591 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 148

Last updated 2026-04-09

No results posted yet for this study

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

Interventions

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).

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-10
Primary Completion
2025-09-19
Completion
2026-03-20
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Canada
  • China
  • France
  • Germany
  • Greece
  • Israel
  • Italy
  • Japan
  • Netherlands
  • South Korea
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

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 NCT04543591 on ClinicalTrials.gov