Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant (NCT04543591) | Clinical Trial Compass
CompletedPhase 3
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
United States, Australia, Belgium148 participantsStarted 2020-12-10
Plain-language 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.
Who can participate
Age range
12 Years – 100 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. 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.
Exclusion criteria
. 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.