Phase 3 study for people with Muscular Dystrophy
Recruiting Phase 3
This trial at a glance
Where this trial is running
| Site | City | State or country | Status at this site |
|---|---|---|---|
| The Children's Hospital at Westmead | Westmead | NSW | Recruiting |
voxsanity.com.au · trial sites from public registries · registry record synced 15 October 2026 · not medical advice
Who can join
The official eligibility criteria
Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.
Key Inclusion Criteria:
* Ambulatory males with clinical and genetic diagnosis of DMD
* Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping
* 7 to 16 years of age at time of consent
* TTR and NSAA assessment completed within the protocol specified parameters at Screening
* On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.
Key Exclusion Criteria:
* Previous treatment cell or gene therapy.
* Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
* Lab values outside of the protocol specified range at Screening
* If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:
* Less than 1 month, for growth hormone and/or testosterone
* Less than 6 months for givinostat
Dates on the record
Results, publications and the official record
- No results are posted on the official record yet.
- No publications are listed on the official record yet.
- Official record and contacts on ClinicalTrials.gov – contact details for each site are kept there, and are not copied onto this page.
Sources and dates
- ClinicalTrials.gov – the record this page reformats
- ClinicalTrials.gov processing date: 13 October 2026.
- This page last checked by Voxsanity: 15 October 2026.
Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.
Not medical advice.
Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.
Letter for your doctor
A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.