An Open-Label Extension Study of Edasalonexent in DMD Patients

• Research type

  Research Study

• Full title

  An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy

• IRAS ID

  272813

• Contact name

  Gary McCullagh

• Contact email

  gary.mccullagh@mft.nhs.uk

• Sponsor organisation

  Catabasis Pharmaceuticals Inc.

• Eudract number

  2019-003563-22

• Duration of Study in the UK

  2 years, 5 months, days

• Research summary

  This is a Phase 3, open-label, treatment extension study to evaluate the safety, tolerability and durability of effect in long-term dosing of edasalonexent in male pediatric patients with a genetically confirmed diagnosis of DMD.
  Approximately 140 patients will be enrolled.
  Eligible patients include boys who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys ≥4.0 to <13.0 years of age.
  Siblings would need to meet the eligibility criteria.
  The study includes a 104-week open label treatment period with edasalonexent at 100 mg/kg/day.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  20/EM/0031

• Date of REC Opinion

  23 Apr 2020

• REC opinion

  Further Information Favourable Opinion