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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03039686
• Date of registration: 27/01/2017
• Prospective Registration: Yes
• Primary sponsor: Hoffmann-La Roche
• Public title: Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
• Scientific title: A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
• Date of first enrolment: July 6, 2017
• Target sample size: 166
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03039686
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Canada, France, Germany, Italy, Japan, Netherlands, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Clinical Trials
• Affiliation: Hoffmann-La Roche

Key inclusion & exclusion criteria

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.



Age minimum: 6 Years
Age maximum: 11 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Placebo for RO7239361

Drug: RO7239361

Primary Outcome(s)

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]

Secondary Outcome(s)

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 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 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 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 Stand from supine velocity [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]

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]

Secondary ID(s)

2016-001654-18

CN001-016

WN40227

Source(s) of Monetary Support

Please refer to primary and secondary sponsors