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Ravulizumab in TMA Associated With a Trigger


Title A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants Who Have Thrombotic Microangiopathy Associated With a Trigger
Therapeutic Area Thrombotic Microangiopathy (TMA)
Principal Investigator Craig E. Gordon MD, MS
Min Age 18 Years
Gender All
Contact Jasmine Pak
617-636-2609
jpak@tuftsmedicalcenter
More Information https://clinicaltrials.gov/ct2/show/NCT04743804

Overview

This study will investigate the efficacy and safety of ravulizumab compared to placebo in adult participants with thrombotic microangiopathy (TMA) associated with a trigger. Participants will be randomized to receive either ravulizumab plus best supportive care or placebo plus best supportive care. The treatment period is 26 weeks followed by a 26-week off-treatment follow-up period.

Study Details

Inclusion Criteria

  • Diagnosis of TMA associated with a trigger
  • 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
  • Vaccinated against meningococcal infection (Neisseria meningitidis), within 3 years prior to, or at the time of, randomization

Exclusion Criteria

  • Known chronic kidney disease with estimated glomerular filtration rate ≤ 45 mL/min/1.73 m^2 due to any cause
  • Any known gene mutation that causes atypical hemolytic uremic syndrome (aHUS)
  • Postpartum aHUS

Study Requirements

One screening visit, 13 visits during the 26 week treatment period, 3 visits during the 26 week follow up period. One blood collection during the screening visit (35 to 45 mL) and during each visit (270 to 300 mL) during the treatment period. During the last follow up visit, 70 to 100 mL blood will be collected. Spot urine samples will be collected at the same visits that blood is collected. No x-rays/CT scans/MRIs are required by the study.