Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
Status:
Recruiting
Trial end date:
2024-02-01
Target enrollment:
Participant gender:
Summary
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.