Ravulizumab in TMA after HSCT

• Research type

  Research Study

• Full title

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

• IRAS ID

  1003339

• Contact name

  Cristina Guzman

• Contact email

  Cristina.Guzman@alexion.com

• Sponsor organisation

  Alexion Pharmaceuticals, Inc.

• Eudract number

  2020-000144-61

• Clinicaltrials.gov Identifier

  NCT04543591

• Research summary

  This study will look at an investigational drug called ravulizumab which is being developed to treat Thrombotic Microangiopathy (TMA) after Haematopoietic Stem Cell Transplant (HSCT)[HSCT-TMA] when received in addition to the standard treatment.
  This is a 2-stage, Phase 3 study of ravulizumab in participants 12 years of age and older with HSCT-TMA. In Stage 1, the dosing regimen for patients with HSCT-TMA has been confirmed in an open-label, single-arm period. The UK hasn’t taken part in Stage 1 and will only participate in the next stage. In Stage 2, participants will be randomly assigned to receive either ravulizumab plus best supportive care (BSC) or matching placebo (looks like the study drug but it doesn’t contain any active ingredient) plus BSC. Eligible participants include adults and adolescents who received HSCT within 6 months prior to Screening and subsequently developed TMA that does not resolve within 72 hours after withdrawal or adjustment of any TMA-associated medication and/or treatment of any associated underlying triggering condition.

  For all participants enrolled, the study will consist of 3 periods:
  - a Screening Period of up to 7 days;
  - a 26-week Treatment Period;
  - and a 26-week Follow-up Period

  Participants in either treatment group who meet defined criteria for clinical worsening at any time during the blinded Treatment Period or no improvement after 21 days of treatment initiation will be eligible for rescue treatment and may be unblinded for the Investigator to determine appropriate rescue treatment.

  Approximately 184 participants will be enrolled to the study. This will include 10 participants in Stage 1 and 174 participants in Stage 2.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  21/LO/0346

• Date of REC Opinion

  25 Jun 2021

• REC opinion

  Further Information Favourable Opinion