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A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to <18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)
EU CTIS ID: 2023-507850-33-00
What this study is testing
To assess the efficacy of ravulizumab plus BSC in the treatment of pediatric 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
- ≥ 28 days of age up to < 18 years of age at the time of signing the informed consent
- 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: −Lactate dehydrogenase > ULN for age, − Presence of schistocytes ≥ 2 high power field (HPF) or ≥ 1% in peripheral blood smear • Proteinuria on spot urinalysis • De novo anemia OR Presence of hypertension
- Participants must have HSCT-TMA that persists for at least 72 hours after initial management of any triggering agent/condition
- Body weight ≥ 5 kg at Screening or ≤7 days prior to the start of the Screening Period (date of consent)
- 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
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%)
- Pregnancy or breastfeeding
- Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab
- Previously or currently treated with a complement inhibitor
- Known Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
- Positive direct Coombs test
See the full eligibility criteria
Who can join
- ≥ 28 days of age up to < 18 years of age at the time of signing the informed consent
- 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: −Lactate dehydrogenase > ULN for age, − Presence of schistocytes ≥ 2 high power field (HPF) or ≥ 1% in peripheral blood smear • Proteinuria on spot urinalysis • De novo anemia OR Presence of hypertension
- Participants must have HSCT-TMA that persists for at least 72 hours after initial management of any triggering agent/condition
- Body weight ≥ 5 kg at Screening or ≤7 days prior to the start of the Screening Period (date of consent)
- 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
- Vaccinated against meningococcal infections if clinically feasible, according to national guidelines and recommendationsfor immune reconstitution after HSCT. Participants must be re-vaccinated 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. meningitidis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis guidance including coverage against N. meningitidis the entire Treatment Period and for 8 months following the final dose of ravulizumab.
- Participants or their legally authorized representative must be capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent or assent form 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%)
- Pregnancy or breastfeeding
- Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab
- Previously or currently treated with a complement inhibitor
- Known Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
- Positive direct Coombs test
- Clinical diagnosis or suspicion 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
- Presence of sepsis requiring vasopressor support within 7 days prior to enrollment
The study team makes the final eligibility decision.
Where it's taking place
- Korea, Republic of
- Israel
- Japan
- United States
- United Kingdom
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. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Korea, Republic of; Israel; Japan; United States; United Kingdom. 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.