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 (NCT04060199) enrolled 77 participants in total — 38 received viltolarsen and 39 received a placebo (an inactive treatment used for comparison). Most participants completed the trial: 36 in the viltolarsen group and 38 in the placebo group. The trial was measuring a physical ability test called the "Time to Stand" (TTSTAND) — specifically, how quickly a participant could get up from lying flat on the floor to a standing position. This was converted into a speed score (rises per second), and the trial tracked how that score changed from the start of the study. The reported data shows a series of measurements of change in that standing-up speed score across different time points for both groups. For the viltolarsen group, the reported changes from baseline were 0.026, 0.027, 0.027, and 0.009 rises per second across the measured time points. For the placebo group, the corresponding reported changes were 0.013, 0.014, 0.027, and 0.013 rises per second. In plain terms, a positive number means participants were getting up from the floor slightly faster compared to when they started the trial. The data as submitted does not include labels identifying which specific time points each measurement corresponds to, so a full time-by-time comparison cannot be described here. 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 ↗
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 are a boy who is at least 4 years old but younger than 8 years old
- You have been confirmed to have a specific type of Duchenne Muscular Dystrophy (DMD) where a particular gene repair strategy (targeting a section called "exon 53") could help
- You are able to walk on your own without any walking aids or assistive devices
- You are able to get up from the floor in less than 10 seconds (confirm with trial site)
- You have been on a stable dose of steroid medication (such as prednisone) for at least 3 months before joining, and are expected to stay on the same dose throughout the whole study
- There may be additional requirements not listed here (confirm with trial site)
Who may not be able to join:
- You currently have, or have a history of, a long-term fungal or viral infection
- You have had an illness in the 4 weeks before the first dose of the study drug
- You are showing symptoms of a heart muscle condition (note: a heart abnormality found on a test but causing no symptoms would not automatically exclude you)
- You have a known allergy or bad reaction to the study drug or any of its ingredients
- You have significant behavioral or thinking difficulties that would make it hard to take part, in the doctor's opinion
- You have a past or current medical condition, medical history, physical findings, or abnormal test results that could affect your safety or interfere with the study, in the doctor's opinion
- You have had surgery in the 3 months before the first dose, or have surgery planned at any point during the study
- You have tested positive for Hepatitis B, Hepatitis C, or HIV
- You are currently taking, or have taken within the last 3 months, another experimental drug as part of a different study
- You have previously taken part in an interventional study involving the drug viltolarsen
- You are currently taking, or have recently taken, any other medication that works by "exon skipping" (a specific type of genetic treatment)
- You have previously received any form of gene therapy
- There may be additional reasons that could affect eligibility not listed here (confirm with trial site)
Important: Always verify eligibility with the trial site directly before applying.
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
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
Change From Baseline in Time to Stand (TTSTAND) Velocity
Can't join this trial?
Data last synced from ClinicalTrials.gov: 26 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.