Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

  • End date
    Feb 2, 2024
  • participants needed
  • sponsor
    Alexion Pharmaceuticals
Updated on 2 August 2021
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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.

Condition Thrombotic microangiopathy
Treatment Placebo, Best supportive care, Ravulizumab
Clinical Study IdentifierNCT04543591
SponsorAlexion Pharmaceuticals
Last Modified on2 August 2021


Yes No Not Sure

Inclusion Criteria

12 years of age or older
Received HSCT within the past 6 months
Diagnosis of TMA that persists despite initial management of any triggering condition
Body weight 30 kilograms
Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of study intervention
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 re-vaccinated against Haemophilus influenzae type b 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 post-transplant infection prophylaxis guidances, including coverage against Neisseria meningitidis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis coverage against Neisseria meningitidis the entire Treatment Period and for 8 months following the final dose of study intervention

Exclusion Criteria

Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' deficiency (activity < 5%)
Known Shiga toxin-related hemolytic uremic syndrome
Positive direct Coombs test
Diagnosis or suspicion of disseminated intravascular coagulation
Known bone marrow/graft failure
Diagnosis of veno-occlusive disease, regardless of severity
Known human immunodeficiency virus (HIV) infection evidenced by positive HIV-1 or HIV-2 antibody titer
Unresolved meningococcal disease
Presence or suspicion of sepsis (treated or untreated) within 7 days prior to Screening
Pregnancy or breastfeeding
Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab
Previously or currently treated with a complement inhibitor
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