A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Hoffmann-La Roche
- Enrollment
- 166
- Locations
- 44
- Primary Endpoint
- Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Study Overview
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).
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 6 Years to 11 Years (Child)
- Sex
- Male
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
RO7239361 Low Dose
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Intervention: RO7239361 (Drug)
RO7239361 High Dose
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Intervention: RO7239361 (Drug)
Placebo
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Intervention: Placebo for RO7239361 (Drug)
Outcomes
Primary Outcomes
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Time Frame: Baseline, Week 48
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
Time Frame: Baseline
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
Secondary Outcomes
- Baseline Time for 4 Stair Climb(Baseline)
- Change From Baseline at Week 48 in Stand From Supine Velocity(Baseline, Week 48)
- Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)(Baseline, Week 48)
- Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale(Baseline)
- Baseline for the 6 Minute Walk Distance (6MWD)(Baseline)
- Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48(Baseline, Week 48)
- Baseline for the Time to Stand From Supine(Baseline)
- Change From Baseline at Week 48 in 10 M Walk/Run Velocity(Baseline, Week 48)
- Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength(Baseline, Week 48)
- Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)(Baseline, Week 48)
- Number of Participants With Adverse Events (AEs)(During DB period (48 weeks) and Whole study (up to approximately 34 months))
- Number of Participants With AEs Leading to Discontinuation(During DB period (48 weeks) and Whole study (up to approximately 34 months))
- Baseline Time for 10 Meter Walk/Run(Baseline)
- Change From Baseline at Week 48 in 95th Percentile Stride Velocity(Baseline, Week 48)
- Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale(Baseline, Week 48)