Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT03039686) looked at a medicine called RO7239361 in boys with Duchenne muscular dystrophy (DMD), a condition that causes progressive muscle weakness. A total of 166 boys took part — 56 received a placebo (a dummy treatment with no active medicine), 55 received a lower dose of RO7239361, and 55 received a higher dose. The trial had two phases: a 48-week blinded period (where no one knew who was getting which treatment) and an open-label period (where participants knew they were receiving the active medicine). The main thing the trial was measuring was change in a movement and function score called the North Star Ambulatory Assessment (NSAA), which rates 17 physical tasks on a scale of 0 to 34, with higher scores meaning better physical ability. The reported data shows that at the start of the trial, average NSAA scores were similar across all three groups — around 23.1 for the placebo group, 24.5 for the low-dose group, and 22.7 for the high-dose group. After 48 weeks, all three groups showed a decline in their scores: the placebo group dropped by approximately 3.0 points, the low-dose group by approximately 3.4 points, and the high-dose group by approximately 2.4 points. For the stair-climbing speed test, all groups also showed a small decline over 48 weeks — the placebo and low-dose groups both declined by 0.15 stairs per second, while the high-dose group declined by 0.07 stairs per second. For the time taken to stand up from lying down, small declines in speed were also reported across all three groups, ranging from 0.02 to 0.05 units per second. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 38190001) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Muscular Dystrophy Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with Duchenne Muscular Dystrophy (DMD), confirmed by medical records and a genetic test
- You are able to walk without any help or assistive devices
- You scored at least 15 points on a specific walking ability test called the North Star Ambulatory Assessment (confirm with trial site)
- You are able to climb 4 stairs in 8 seconds or less
- You weigh at least 15 kg (about 33 pounds)
- You are currently taking steroid medication (such as prednisone or deflazacort) to help manage DMD
Who may not be able to join:
- You have a behavioral or mental health condition that might make it difficult to complete all parts of the study
- You are currently taking, or have previously taken, certain hormonal medications such as androgens or human growth hormone
- You need to use a breathing machine (ventilator) during the daytime
- You are unable to have blood drawn or receive an injection under the skin
- You have taken part in a gene therapy study at any time in the past, or are currently doing so
Important: Always verify eligibility with the trial site directly before applying.
Note: Additional eligibility requirements not listed here may also apply, so it is important to speak with the trial team for the full details.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Clinical Trials, Hoffmann-La Roche
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score; Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Can't join this trial?
Data last synced from ClinicalTrials.gov: 22 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.