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 (NCT03439670) enrolled a total of 121 participants across six treatment groups, with group sizes ranging from 15 to 30 people. The trial was measuring how a drug called vamorolone compared to a placebo (an inactive treatment), focusing on a physical ability test called the "Time to Stand" test. This test measures how quickly a person can rise from the floor, recorded in "rises per second" — essentially, a higher number means rising more quickly. The reported data shows that the primary outcome compared changes in the Time to Stand test score from the start of the trial to 24 weeks later, looking specifically at Treatment Group 1 (vamorolone at 6.0 mg/kg/day) versus Treatment Group 2 (placebo). According to the results reported on ClinicalTrials.gov, the placebo group's score changed by -0.007 rises per second (a very slight decline), while the vamorolone group's score changed by +0.054 rises per second (a slight increase). No further outcome measure results were included in the submitted data for the remaining four treatment groups, so those figures were not reported in the structured results available. It is also worth noting that not all participants who started the trial finished it — for example, 2 people did not complete it in Treatment Group 1 and 4 did not complete it in Treatment Group 2, with similar small numbers across other groups. 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 36036925) ↗
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 2 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:
- A parent or legal guardian must agree to the study in writing, and the child will also be asked for their agreement
- The child must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD), verified by a central genetics team
- The child must be at least 4 years old but not yet 7 years old when they join the study
- The child must weigh more than 13.0 kg and no more than 39.9 kg at the screening visit
- The child must be able to walk on their own without any walking aids or devices
- The child must be able to get up from the floor to a standing position without help in under 10 seconds
- Blood test results at the screening visit must be mostly normal, with certain liver-related values within the normal range
- The child must show immunity to chickenpox, either through a positive blood test or proof of having received 2 chickenpox vaccines (with the second dose given at least 14 days before starting the study)
- The child must be able to swallow tablets, confirmed by successfully swallowing a dummy (placebo) tablet during screening
- The child and their parent or guardian must be willing and able to attend all visits and follow the study schedule
Who may not be able to join:
- The child has serious kidney or liver problems, diabetes, or a weakened immune system (now or in the past)
- The child has or has had a long-term fungal or viral infection
- The child has had an illness in the 4 weeks before starting the study medication
- The child has taken certain blood pressure or hormone-related medications (such as spironolactone or eplerenone) within the past 4 weeks
- The child has a history of a condition called primary hyperaldosteronism (a hormonal disorder affecting the adrenal glands)
- The child has heart muscle problems that are causing symptoms (confirm with trial site)
- The child is currently taking or has previously taken steroid tablets (such as oral glucocorticoids) or other medicines that suppress the immune system — though some limited past use may be reviewed individually (confirm with trial site)
- The child has an allergy or sensitivity to the study drug or any of its ingredients
- The child has used a medication called idebenone within the past 4 weeks
- The child has severe behavioural or thinking/learning difficulties that would make participation too difficult, in the doctor's opinion
- The child has any other medical condition or history that could affect their safety or the reliability of the study results, in the doctor's opinion
- The child has taken herbal remedies or supplements that may affect muscle strength (such as Co-enzyme Q10 or creatine) within the past 4 weeks
- The child has taken any medication specifically for DMD (including Exondys 51 or Translarna) within the past 3 months
- The child has received a live vaccine within the 14 days before starting the study medication
- The child is currently taking or has taken another experimental drug within the past 3 months
- The child has a brother or sister who is currently enrolled in, or plans to join, another study involving the same study drug (vamorolone)
- The child has previously taken part in this same study
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
Principal Investigator: Michela Guglieri, M.D., John Walton Muscular Dystrophy Research Centre
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
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline
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.