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 |
| Sunshine Coast University Hospital | Birtinya | QLD | Status not stated |
| Royal Brisbane And Women's Hospital | Herston | QLD | Status not stated |
| Alfred Health | Melbourne | VIC | Status not stated |
| Nucleus Network Brisbane Clinic | Herston | Australia | Status not stated |
| Mater Hospital Brisbane | South Brisbane | Australia | 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 diagnosis of DM1
* Clinician-assessed signs of DM1 including clinically apparent myotonia
* Onset of DM1 symptoms occurred after the age of 12 years
* Walk for at least 10 meters independently at screening
* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.
Key Exclusion Criteria:
* Inadequately controlled diabetes
* Confirmed diagnosis of congenital DM1
* Uncontrolled hypertension
* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period
* Clinically significant cardiac, liver or renal disease
* Human immunodeficiency virus infection (seropositive) at screening
* Seropositive for hepatitis B or hepatitis C at screening
* Untreated or poorly controlled epilepsy
* Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening.
* Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time
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.