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 (NCT02760264) tested four different daily doses of a drug called vamorolone in boys aged 4 to under 7 years old who had Duchenne muscular dystrophy (DMD), a condition that causes progressive muscle weakness. A total of 48 boys took part, with 12 assigned to each of the four dose groups (0.25, 0.75, 2.0, and 6.0 mg per kilogram of body weight per day). All 48 boys completed the trial. The trial was primarily looking at unwanted side effects (called adverse events) that occurred during treatment, and also measured certain markers in the blood related to how the body handles sugar and hormones. The reported data shows that across the four dose groups, the total number of treatment-related adverse events (any unwanted health change recorded after starting the drug) was 16, 18, 13, and 11 events respectively. The number of participants in each group who experienced at least one such event was 13, 13, 11, and 9 out of 12. Regarding blood sugar levels (fasting glucose), the reported data shows small changes from the start of the study to week 2, ranging from a decrease of about 5.8 mg/dL to a very slight increase of 0.2 mg/dL across the groups. For insulin (a hormone that helps control blood sugar), changes from the start to week 2 ranged from a small decrease in the lowest dose group to an increase of 2.78 units in the highest dose group. A morning cortisol reading (a hormone that reflects how the body's stress response system is working) was also reported at the end of the study period, with values of 10.4, 9.8, 7.3, and 3.0 micrograms per decilitre across the four dose groups from lowest to highest dose — noting that percentage-change data for cortisol from the start of the study was not reported in the submitted data. 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 38521535) ↗
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 have signed a consent form before any study-related steps begin
- The child must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD), verified by a central genetic counselor, based on specific test results and a clinical picture consistent with DMD
- The child must be at least 4 years old and under 7 years old at the time of joining the study
- The child must be able to stand up from the floor without help, as tested during the screening visits
- The child's blood test results must be normal, or only slightly outside normal in a way the doctor considers unimportant, at the screening visit
- The child must show immunity to chickenpox, confirmed by a positive blood test at the screening visit
- Both the child and parent or guardian must be willing and able to attend all scheduled visits and follow the study plan
Who may not be able to join:
- Children who have, or have had, serious kidney or liver problems, diabetes, or a weakened immune system
- Children who have, or have had, long-term fungal or viral infections affecting the whole body
- Children who have had an illness in the 4 weeks before the first study dose
- Children who have taken certain blood pressure or hormone-related medications (such as spironolactone or eplerenone) within the 4 weeks before the first study dose
- Children who are showing symptoms of heart muscle disease (note: an abnormal heart test result without symptoms would not automatically exclude participation — confirm with trial site)
- Children who are currently taking, or have previously taken, steroid tablets (such as oral glucocorticoids) or other immune-suppressing medicines — though some limited past use may be reviewed case by case (confirm with trial site)
- Children who have used a medication called idebenone within 4 weeks before the first study dose
- Children who have a known allergy or sensitivity to the study medication or any of its ingredients
- Children who have severe behavioural or thinking difficulties that would prevent them from taking part, in the opinion of the study doctor
- Children who have any other medical condition, history, or test result that the study doctor believes could affect their safety or the reliability of the study results
- Children who are currently taking, or have taken within the past 3 months, any other experimental medication
- Children who have previously been enrolled 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: Paula R Clemens, MD, University of Pittsburgh
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
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.