Phase 2 Muscular Dystrophy Trial, Recruiting
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:
- You are a boy aged 4 years old or older, but younger than 15 years old
- You have a confirmed genetic change in the dystrophin gene that could be helped by a technique called "exon 44 skipping" (confirm with trial site if unsure)
- You are able to walk on your own without any walking aids or devices
- You are able to complete a specific "stand up from the floor" test in under 20 seconds without help
- You have been on a stable dose of steroid medication (such as prednisone) for at least 3 months, and this dose is expected to stay the same throughout the study
- There may be other requirements — confirm all details with the trial site
Who may not be able to join:
- You weigh less than 20 kg (about 44 lbs) at the time of signing up (this applies to a specific part of the trial only — confirm with trial site)
- You have a heart muscle condition that is causing symptoms
- You have used certain muscle-building steroids (such as oxandrolone) or supplements containing resveratrol or ATP in the past 3 months
- You have taken part in another clinical drug trial or used an experimental medicine in the past 3 months (or longer, depending on the medicine)
- You have had surgery in the past 3 months, or surgery is planned at any point during the study
- You have previously taken part in a specific study called NS-089/NCNP-02
- You have received a type of treatment called "exon skipping" therapy within the past year
- There may be other reasons that could prevent joining — confirm all details with the trial site
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
Phone: 1-866-677-6276
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
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Adverse Event and Adverse Drug Reaction; Plasma pharmacokinetic (PK) parameters; Plasma pharmacokinetic (PK) parameters; Plasma pharmacokinetic (PK) parameters; Plasma pharmacokinetic (PK) parameters; Plasma pharmacokinetic (PK) parameters; Plasma pharmacokinetic (PK) parameters; Plasma pharmacokinetic (PK) parameters; Urine pharmacokinetic parameters; Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).
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.