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Clinical Trial 20871

Cancer Type: Malignant Hematology
Study Type: Treatment
NCT#: NCT04543591

Phase: Phase III
Prinicipal Investigator: Nishihori, Taiga

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or 1-800-679-0775 Learn More
Overview

Study Title

A Phase 3, Open-label, Randomized, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

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.

Objective

Primary: To assess the efficacy of ravulizumab plus BSC versus BSC-only in the treatment of adult and adolescent participants with HSCT-TMA Secondary: To characterize TMA response after treatment with ravulizumab. To assess improvement in organ dysfunction. To assess TMA relapse and duration of response. To assess overall survival. To assess non-relapse mortality. To assess platelet response.

Treatments

Therapies

Therapy (NOS)

Medications

Placebo (); Ravulizumab ()

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 > 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

  • If you are interested in learning more about clinical trials, our clinical trial navigators can discuss your options and recommend opportunities that may be suitable for you. Call 813-745-6100 or 1-800-679-0775 (toll-free) or submit a clinical trials inquiry form.