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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 : August 10, 2018
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: RO7239361 Drug: Placebo for RO7239361 Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 159 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : July 6, 2017
Estimated Primary Completion Date : December 30, 2020
Estimated Study Completion Date : December 17, 2021


Arm Intervention/treatment
Experimental: RO7239361, dose 1
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period

Experimental: RO7239361, dose 2
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period

Placebo Comparator: Placebo
Placebo solution taken 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




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in North Star Ambulatory Assessment [ Time Frame: 48 weeks ]
  2. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Stand from supine velocity [ Time Frame: 48 weeks ]
  3. 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 ]
  4. 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 ]
  5. 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 ]
  6. 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 ]
  7. Change from baseline at Week 48 in Clinical Global Impression of Change (CGI-C) [ Time Frame: 48 weeks ]


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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03039686


Contacts
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

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Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche

Additional Information:
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03039686     History of Changes
Other Study ID Numbers: CN001-016
2016-001654-18 ( EudraCT Number )
WN40227 ( Other Identifier: Hoffman-La Roche )
First Posted: February 1, 2017    Key Record Dates
Last Update Posted: August 10, 2018
Last Verified: August 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Hoffmann-La Roche:
muscular dystrophy
Duchenne's Muscular Dystrophy
DMD

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked