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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 : June 5, 2019
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Tracking Information
First Submitted Date  ICMJE January 27, 2017
First Posted Date  ICMJE February 1, 2017
Last Update Posted Date June 5, 2019
Actual Study Start Date  ICMJE July 6, 2017
Estimated Primary Completion Date December 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 15, 2018)
  • Change from baseline in the North Star Ambulatory Assessment (NSAA) total score in RO7239361 treated participants. [ Time Frame: 48 Week ]
  • Change from baseline in the in the North Star Ambulatory Assessment (NSAA) total score in placebo treated participants. [ Time Frame: 48 Week ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 31, 2017)
  • 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 ]
Change History Complete list of historical versions of study NCT03039686 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 15, 2018)
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in the 4 Stair Climb Velocity (4SCV) 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 RO7239361 treated participants compared to placebo treated participants in Clinical Global Impression of Change (CGI-C) rating. [ Time Frame: 48 weeks ]
  • Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 95th percentile stride velocity [ Time Frame: 48 weeks ]
    As recorded with the ActiMyo device in a subset of the overall study population.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Official Title  ICMJE A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Brief 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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • 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
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 31, 2017)
159
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 17, 2024
Estimated Primary Completion Date December 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 6 Years to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE Argentina,   Australia,   Belgium,   Canada,   France,   Germany,   Italy,   Japan,   Netherlands,   Spain,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03039686
Other Study ID Numbers  ICMJE CN001-016
2016-001654-18 ( EudraCT Number )
WN40227 ( Other Identifier: Hoffman-La Roche )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Hoffmann-La Roche
Study Sponsor  ICMJE Hoffmann-La Roche
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Trials Hoffmann-La Roche
PRS Account Hoffmann-La Roche
Verification Date June 2019

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