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)

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 40

Inclusion Criteria

= 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

Exclusion Criteria

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