BridgeMD
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)
EU CTIS ID: 2023-510107-22-00
What this study is testing
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 12 years of age or older, at the time of signing the informed consent form (ICF)
- Participants who received HSCT within the past 12 months at the time of Screening)
- A TMA diagnosis, based on meeting all of the following criteria during the Screening Period and/or ≤ 14 days prior to the Screening Period: • De novo thrombocytopenia or platelet transfusion refractoriness •Any one of the following markers of hemolysis: − LDH > ULN for age − Presence of schistocytes ≥ 2 per high power field (HPF) or ≥ 1% in peripheral blood smear • Proteinuria on spot urinalysis • De novo anemia OR the presence of hypertension
- Participants must have HSCT-TMA that persists despite initial management of any triggering condition (persists for at least 72 hours after management of triggering agent/condition)
- Body weight ≥ 30 kg at Screening or ≤ 7 days prior to the start of the Screening Period (date of consent).
- Participants must be vaccinated against meningococcal infections if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. Participants < 18 years of age must be revaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. All participants should be administered coverage with prophylactic antibiotics according to institutional posttransplant infection prophylaxis guidances including coverage against N. meningiditis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis coverage against N. meningiditis the entire Treatment Period and for 8 months following the final dose of ravulizumab
You likely can't join if
- Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%).
- Presence of sepsis requiring vasopressor support within 7 days prior to enrollment
- Hypersensitivity to murine proteins or to 1 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 by participating in the study or confound the outcome of the study. Including but not limited to, major cardiac, pulmonary, renal, endocrine, or hepatic disease
- Previously or currently treated with a complement inhibitor
- Pregnancy or breastfeeding
See the full eligibility criteria
Who can join
- 12 years of age or older, at the time of signing the informed consent form (ICF)
- Participants who received HSCT within the past 12 months at the time of Screening)
- A TMA diagnosis, based on meeting all of the following criteria during the Screening Period and/or ≤ 14 days prior to the Screening Period: • De novo thrombocytopenia or platelet transfusion refractoriness •Any one of the following markers of hemolysis: − LDH > ULN for age − Presence of schistocytes ≥ 2 per high power field (HPF) or ≥ 1% in peripheral blood smear • Proteinuria on spot urinalysis • De novo anemia OR the presence of hypertension
- Participants must have HSCT-TMA that persists despite initial management of any triggering condition (persists for at least 72 hours after management of triggering agent/condition)
- Body weight ≥ 30 kg at Screening or ≤ 7 days prior to the start of the Screening Period (date of consent).
- Participants must be vaccinated against meningococcal infections if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. Participants < 18 years of age must be revaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. All participants should be administered coverage with prophylactic antibiotics according to institutional posttransplant infection prophylaxis guidances including coverage against N. meningiditis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis coverage against N. meningiditis the entire Treatment Period and for 8 months following the final dose of ravulizumab
- Male or female Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Capable of giving signed informed consent or assent which includes compliance with the requirements and restrictions listed in the informed consent and in this protocol
What rules you out
- Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%).
- Presence of sepsis requiring vasopressor support within 7 days prior to enrollment
- Hypersensitivity to murine proteins or to 1 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 by participating in the study or confound the outcome of the study. Including but not limited to, major cardiac, pulmonary, renal, endocrine, or hepatic disease
- Previously or currently treated with a complement inhibitor
- Pregnancy or breastfeeding
- Known Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
- Positive direct Coombs test result
- Clinical diagnosis of disseminated intravascular coagulation (DIC)
- Known bone marrow/graft failure for the current HSCT
- Diagnosis of veno-occlusive disease (VOD)
- Human immunodeficiency virus (HIV) infection evidenced by HIV-1 or HIV-2 antibody titer
- Unresolved meningococcal disease
The study team makes the final eligibility decision.
Where it's taking place
- Brazil
- Australia
- United States
- Korea, Republic of
- China
- Canada
- United Kingdom
- Japan
- Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Brazil; Australia; United States; Korea, Republic of; China; Canada and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.