Study of Ravulizumab in Pediatric Participants With HSCT-TMA
0 - 17 Years, Male and Female
ALXN1210-TMA-314 (primary)
NCI-2021-04301
2020-000761-16
Summary
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.
Eligibility
- = 28 days of age up to < 18 years of age at the time of signing the informed consent.
- Received HSCT within the past 12 months.
- Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.
- A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or =14 days prior to the Screening Period.
- Body weight = 5 kilograms at Screening or =7 days prior to the start of the Screening Period (date of consent).
- Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
- Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants <18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.
- Participants or their legally authorized representative must be capable of giving signed informed consent or assent.
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