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

study id #: NCT03039686

condition: Duchenne Muscular Dystrophy

status: recruiting

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).

intervention: RO7239361, Placebo for RO7239361

mechanism of action: Anti-myostatin adnectin to promote muscle growth

results: https://clinicaltrials.gov/ct2/show/results/NCT03039686

last updated: April 27, 2019

start date: July 6, 2017

estimated completion: December 30, 2020

phase of development: Phase 2/Phase 3

size / enrollment: 159

primary outcomes:

• 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 outcomes:

• 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 ]

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

sponsor: Hoffmann-La Roche

contacts:
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

investigators: Clinical Trials, Hoffmann-La Roche

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