Study of Ravulizumab in Pediatric Participants With HSCT-TMA (NCT04557735) | Clinical Trial Compass
CompletedPhase 3
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
United States41 participantsStarted 2020-12-07
Plain-language 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.
Who can participate
Age range17 Years
SexALL
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Inclusion criteria
✓. ≥ 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.
Exclusion criteria
✕. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.
✕. Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
What they're measuring
1
Participants With Thrombotic Microangiopathy (TMA) Response