Study of Ravulizumab in Pediatric Participants With HSCT-TMA

  • End date
    Feb 29, 2024
  • participants needed
  • sponsor
    Alexion Pharmaceuticals
Updated on 31 July 2021
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This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

Condition Thrombotic microangiopathy
Treatment Best supportive care, Ravulizumab
Clinical Study IdentifierNCT04557735
SponsorAlexion Pharmaceuticals
Last Modified on31 July 2021


Yes No Not Sure

Inclusion Criteria

1 month of age up to < 18 years of age at the time of signing the informed consent
Received HSCT within the past 6 months
Diagnosis of TMA that persists despite initial management of any triggering condition
Body weight 5 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 ravulizumab
Participants must be vaccinated against meningococcal infections if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. Participants 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 ravulizumab

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
Human immunodeficiency virus (HIV) infection (evidenced by 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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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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