Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

Completed · Phase 3 · Has a placebo group

Conditions studied: Thrombotic Microangiopathy

In brief

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.

Key facts

Study ID: NCT04543591
Run by: Alexion Pharmaceuticals, Inc.
People needed: 148
Starts: 2020-12-10
Expected to finish: 2026-03-20
Last updated by the study team: 2026-06-18

Who can join

Age: 12 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

12 years of age or older at time of consent/assent.
Received HSCT within the past 12 months.
Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.
A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period.
Body weight ≥ 30 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).
Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants <18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.
Participants or their legally authorized representative must be capable of giving signed informed consent or assent.

You may not qualify if…

Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency
Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.
Clinical diagnosis of disseminated intravascular coagulation (DIC).
Known bone marrow/graft failure for the current HSCT.
Diagnosis of veno-occlusive disease which is unresolved at the time of Screening.
Human immunodeficiency virus (HIV) infection.
Unresolved meningococcal disease.
Presence of sepsis requiring vasopressor support.
Pregnancy or breastfeeding.
Hypersensitivity to murine proteins or to one of the excipients of ravulizumab.
Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.
Respiratory failure requiring mechanical ventilation.
Acute and/or chronic heart failure with an ejection fraction ≤ 40%.
Previously or currently treated with a complement inhibitor.
Participation in an interventional treatment study of any therapy for TMA.

Where it is running

Research Site — Grosse Pointe Farms, Michigan, United States
Research Site — Durham, North Carolina, United States
Research Site — Pittsburgh, Pennsylvania, United States
Research Site — Seattle, Washington, United States
Research Site — Parkville, Australia
Research Site — Bruges, Belgium
Research Site — Brussels, Belgium
Research Site — Chênée, Belgium
Research Site — Yvoir, Belgium
Research Site — Cerqueira César, Brazil
Research Site — Florianópolis, Brazil
Research Site — Jaú, Brazil
Research Site — Porto Alegre, Brazil
Research Site — Porto Alegre, Brazil
Research Site — Rio de Janeiro, Brazil
Research Site — São José do Rio Preto, Brazil
Research Site — São Paulo, Brazil
Research Site — Calgary, Alberta, Canada
Research Site — Suzhou, China
Research Site — Tianjin, China
Research Site — Angers, France
Research Site — La Tronche, France
Research Site — Nice, France
Research Site — Hamburg, Germany
Research Site — Tampa, Florida, United States

Full record on ClinicalTrials.gov

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