Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

  • Duchenne Muscular Dystrophy (DMD)
Trial Status:


This trial runs in
  • Atlanta
  • Baltimore
  • Buenos Aires
  • Chicago
  • Cincinnati
  • Columbus
  • Esplugues de Llobregat
  • Essen
  • Gainesville
  • Gent
  • Göteborg
  • Iowa City
  • Kansas City
  • Kodaira
  • Las Vegas
  • Leiden
  • Liverpool
  • London
  • Lyon
  • Matsumoto
  • Messina
  • Milano
  • Nantes
  • New Haven
  • Nijmegen
  • Nishinomiya
  • Orlando
  • Ottawa
  • Paris
  • Parkville
  • Phoenix
  • Roma
  • Sacramento
  • Seattle
  • Sendai
  • South Brisbane
  • St. Louis
  • Stanford
  • Strasbourg
  • Toyonaka
  • València
  • Westmead
  • Worcester
Trial Identifier:

NCT03039686 2016-001654-18 CN001-016

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      The source of the below information is the publicly available website It has been summarised and edited into simpler language.

      The below information is taken directly from the publicly available website within a week of any updates, and has not been edited.

      Results Disclaimer

      Trial Summary

      This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

      Hoffmann-La Roche Sponsor
      Phase 2/Phase 3 Phase
      NCT03039686 , CN001-016 , 2016-001654-18 Trial Identifier
      RO7239361, Placebo for RO7239361 Treatments
      Duchenne Muscular Dystrophy Condition
      Official Title

      A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

      Eligibility Criteria

      Male Gender
      ≥6 Years & ≤ 11 Years Age
      No Healthy Volunteers
      Inclusion Criteria
      • Diagnosed with DMD by confirmed medical history and genetic testing
      • Able to walk without assistance
      • Minimum North Star Ambulatory Assessment score of 15 at screening
      • Able to walk up 4 stairs in 8 seconds or less
      • Weigh at least 15 kg (33 lbs)
      • Taking corticosteroids for DMD
      Exclusion Criteria
      • Any behavior or mental issue that will affect the ability to complete the required study procedures
      • Previously or currently taking medications like androgens or human growth hormone
      • Use of a ventilator during the day
      • Unable to have blood samples collected or receive an injection under the skin
      • Concomitant or previous participation at any time in a gene therapy study

      Other protocol defined Inclusion/Exclusion Criteria could apply.

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