Phase 2 study for people with Muscular Dystrophy
By invitation Phase 2
This trial at a glance
Where this trial is running
| Site | City | State or country | Status at this site |
|---|---|---|---|
| Liverpool Hospital | Liverpool | NSW | Status not stated |
| Wesley Research Institute | Auchenflower | QLD | Status not stated |
| Sunshine Coast University Hospital | Birtinya | QLD | Status not stated |
| Alfred Health | Melbourne | VIC | Status not stated |
voxsanity.com.au · trial sites from public registries · registry record synced 12 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:
* Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record
* Clinical severity score between 3 and 8 (scale, 0 to 10)
* Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fraction ≥10% and less than approximately 40%
* Males or nonpregnant, nonlactating females ≥18 years of age who do not plan to become pregnant during the study, with an upper age limit of ≤70 years
* Able and willing to provide written informed consent prior to the performance of any study specific procedures
* Participants with a body mass index (BMI) between 18.0 and 35.0 kilograms/square meter, inclusive. A participant with FSHD1 and a BMI outside this range may be allowed into the study at the discretion of the principal investigator.
* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
* A 12-lead electrocardiogram at screening with no abnormalities that may compromise participant's safety in the study
* Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 9 months following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 9 months following the end of study or last dose of study medication, whichever is later.
Key Exclusion Criteria:
* Human immunodeficiency virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at screening
* Seropositive for hepatitis B or hepatitis C at screening
* Uncontrolled hypertension
* Severe cardiovascular disease
* History of thrombolic events
* Platelet count less that the lower limit of normal at screening
* History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
* Any contraindication to muscle biopsy or MRI
Note: additional inclusion/exclusion criteria may apply per protocol
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: 5 October 2026.
- This page last checked by Voxsanity: 12 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.