Phase 3 Study of Edasalonexent in Duchenne Muscular Dystrophy

  • Research type

    Research Study

  • Full title

    A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY

  • IRAS ID

    251874

  • Contact name

    Gary McCullagh

  • Contact email

    gary.mccullagh@mft.nhs.uk

  • Sponsor organisation

    Catabasis Pharmaceuticals, Inc.

  • Eudract number

    2018-000464-29

  • Duration of Study in the UK

    1 years, 4 months, 31 days

  • Research summary

    This is a randomized, double-blind, placebo-controlled, Phase 3, multisite study of edasalonexent in pediatric patients with DMD. Approximately 126 patients will be enrolled.
    Eligible patients will be ambulatory boys from ≥4.0 to <8.0 years of age with a confirmed diagnosis of DMD who have not used steroids within 24 weeks prior to treatment initiation (Day 1).
    The study includes a 52-week, double-blind, placebo-controlled period comparing 1 dose level of edasalonexent with placebo, followed by a 2-week follow-up.
    Following completion of the treatment period and Primary Endpoint evaluation, patients may elect to continue in the open-label extension study, pending approval by appropriate ethics committees and regulatory authorities. Patients who choose not to participate should complete the safety follow-up within 2 weeks

  • REC name

    East Midlands - Derby Research Ethics Committee

  • REC reference

    19/EM/0003

  • Date of REC Opinion

    20 Feb 2019

  • REC opinion

    Further Information Favourable Opinion