ALXN-1210-TMA-314

• Research type

  Research Study

• Full title

  A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to < 18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)

• IRAS ID

  288227

• Contact name

  Sarah Lawson

• Contact email

  sarah.lawson11@nhs.net

• Sponsor organisation

  Alexion Pharmaceuticals, Inc.

• Eudract number

  2020-000761-16

• Clinicaltrials.gov Identifier

  NCT04557735

• Duration of Study in the UK

  2 years, 5 months, 24 days

• 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. TMA is a potentially fatal, post-transplant complication of HSCT in which small blood vessels inside vital organs do not function normally. Ravulizumab functions by blocking complement activity. The complement system is part of the immune system which fights against infections. In patients with HSCT-TMA, abnormal complement activity is present which may cause destruction of the red blood cells and platelets in the blood stream and can damage some organs such as the kidney, heart, lung and brain.

  This is a phase 3 open-label study with one treatment group. Ravulizumab will be given in addition to the standard treatment (also known as Best Supportive Care (BSC)) for HSCT-TMA to approximately 40 participants. The expected duration of participation for each participant will be up to 53 weeks divided into 3 different periods:
  • Screening period (up to 1 week)
  • Treatment period (26 weeks)
  • Follow up period (26 weeks)

  Key Inclusion and Exclusion criteria
  Inclusion:
  - Participants must be 1 month of age up to <18 years of age
  - Paediatric participants who received HSCT within the past 6 months at time of screening
  - A TMA diagnosis
  - Participants must have HSCT-TMA that persists for at least 72 hours after initial management of any triggering agent/condition
  Exclusion
  - Pregnancy or breastfeeding
  - Known familial or acquired ‘a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13’ (ADAMTS13) deficiency
  - Known Shiga toxin-related hemolytic uremic syndrome
  - Positive direct Coombs test
  - Known bone marrow/graft failure
  - Human immunodeficiency virus
  - Unresolved meningococcal diseas
  - Presence or suspicion of sepsis

• REC name

  West Midlands - Edgbaston Research Ethics Committee

• REC reference

  20/WM/0297

• Date of REC Opinion

  6 Jan 2021

• REC opinion

  Further Information Favourable Opinion