Phase 3
N=166
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT03039686 ↗Enrolled (actual)
166
Serious AEs
3.7%
Results posted
Dec 2020
Primary outcome: Primary: Baseline for the North Star Ambulatory Assessment (NSAA) Total Score — 23.1; 24.5; 22.7 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- RO7239361 (Drug); Placebo for RO7239361 (Drug)
- Age
- Pediatric · 6+ yrs
- Sex
- Male
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Apr 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Baseline for the North Star Ambulatory Assessment (NSAA) Total Score |
23.1; 24.5; 22.7 | — |
| PRIMARY Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48 |
-2.99; -3.44; -2.41 | — |
| SECONDARY Baseline Time for 4 Stair Climb |
3.81; 3.85; 3.92 | — |
| SECONDARY Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV) |
-0.15; -0.15; -0.07 | — |
| SECONDARY Baseline for the Time to Stand From Supine |
6.28; 6.15; 7.24 | — |
| SECONDARY Change From Baseline at Week 48 in Stand From Supine Velocity |
-0.05; -0.02; -0.02 | — |
| SECONDARY Baseline Time for 10 Meter Walk/Run |
5.38; 5.51; 5.68 | — |
| SECONDARY Change From Baseline at Week 48 in 10 M Walk/Run Velocity |
-0.23; -0.14; -0.23 | — |
| SECONDARY Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale |
85.59; 86.54; 84.47 | — |
| SECONDARY Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale |
-5.47; -7.47; -4.51 | — |
| SECONDARY Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength |
5.58; 6.25; 5.76; -1.19; -0.47; -0.88 | — |
| SECONDARY Baseline for the 6 Minute Walk Distance (6MWD) |
388.33; 399.73; 370.73 | — |
| SECONDARY Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD) |
-41.3; -39.6; -30.0 | — |
| SECONDARY Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48 |
0; 0; 0; 5.6; 2.7; 3.2 | — |
| SECONDARY Change From Baseline at Week 48 in 95th Percentile Stride Velocity |
1.69; 1.54; 1.57; -0.25; -0.22; -0.28 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) |
46; 48; 49; 57; 56 | — |
| SECONDARY Number of Participants With AEs Leading to Discontinuation |
0; 0; 0; 0; 0 | — |
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).
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT03039686). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.