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

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ClinicalTrials.gov Identifier: NCT03039686
Recruitment Status : Recruiting
First Posted : February 1, 2017
Last Update Posted : September 7, 2018
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

January 27, 2017
February 1, 2017
September 7, 2018
July 6, 2017
December 30, 2020   (Final data collection date for primary outcome measure)
  • Change from baseline in the 4 stair climb velocity in RO7239361 treated participants. [ Time Frame: 48 Week ]
    Number of seconds to complete 4 stair climb.
  • Change from baseline in the 4 stair climb velocity in placebo treated participants. [ Time Frame: 48 Week ]
    Number of seconds to complete 4 stair climb.
  • Change from baseline in the 4 stair climb velocity in BMS-986089 treated participants. [ Time Frame: 48 Week ]
  • Change from baseline in the 4 stair climb velocity in placebo treated participants. [ Time Frame: 48 Week ]
Complete list of historical versions of study NCT03039686 on ClinicalTrials.gov Archive Site
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in North Star Ambulatory Assessment [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Stand from supine velocity [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 10 M walk/run velocity [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in PODCI transfers and basic mobility subscale [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Proximal lower extremity flexor (knee extension and knee flexion) strength, measured using manual myometry [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 6 Minute Walk Distance (6MWD) [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in Clinical Global Impression of Change (CGI-C) [ Time Frame: 48 weeks ]
Not Provided
Not Provided
Not Provided
 
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
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).
Not Provided
Interventional
Phase 2
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
  • Drug: RO7239361
    Take RO7239361 subcutaneously on specified days over a 48 week blinded period
  • Drug: Placebo for RO7239361
    Take placebo subcutaneously on specified days over a 48 week blinded period
  • Experimental: RO7239361, dose 1
    Take RO7239361 subcutaneously on specified days over a 48 week blinded period
    Intervention: Drug: RO7239361
  • Experimental: RO7239361, dose 2
    Take RO7239361 subcutaneously on specified days over a 48 week blinded period
    Intervention: Drug: RO7239361
  • Placebo Comparator: Placebo
    Placebo solution taken subcutaneously on specified days over a 48 week blinded period
    Intervention: Drug: Placebo for RO7239361
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
159
Same as current
December 17, 2021
December 30, 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosed with DMD by confirmed medical history and genetic testing
  • Able to walk without assistance
  • 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.

Sexes Eligible for Study: Male
6 Years to 11 Years   (Child)
No
Contact: Reference Study ID Number: WN40227 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. and Canada) global-roche-genentech-trials@gene.com
Argentina,   Australia,   Belgium,   Canada,   France,   Germany,   Italy,   Japan,   Netherlands,   Spain,   Sweden,   United Kingdom,   United States
 
 
NCT03039686
CN001-016
2016-001654-18 ( EudraCT Number )
WN40227 ( Other Identifier: Hoffman-La Roche )
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Hoffmann-La Roche
Hoffmann-La Roche
Not Provided
Study Director: Clinical Trials Hoffmann-La Roche
Hoffmann-La Roche
September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP