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 man or woman between 18 and 65 years old with a diagnosis of a muscle disease called LGMD2I/R9, confirmed by a genetic test showing a change in the FKRP gene.
- You are able to walk or run 10 meters in less than 30 seconds.
- You are able to understand and follow all the steps involved in the study.
- If you are a woman who could become pregnant, or a male participant whose partner could become pregnant, you must be willing to use a barrier form of contraception (such as condoms) for the first 6 months after receiving the study treatment.
Who may not be able to join:
- You have significant heart problems, such as a weakened heart (pumping less than 40% efficiency), certain heart rhythm or electrical issues, moderate-to-severe heart failure, or scarring of the heart muscle shown on a scan.
- You cannot have an MRI scan, or you have had allergic reactions to contrast dyes, shellfish, or iodine.
- You have implanted metal devices such as spinal rods, a pacemaker, or other implants that could affect heart scan images.
- You have an active or ongoing liver disease (such as hepatitis) or abnormal liver blood test results.
- Your kidneys are not working well enough, based on a specific blood test measuring kidney function.
- You have or have had a serious or life-threatening illness, including most cancers, within the past 5 years (skin cancers of certain types may be excepted — confirm with trial site).
- In the doctor's opinion, you have an existing health condition that makes the risks of joining greater than the potential benefits.
- You need a breathing machine or ventilator during the day.
- Your steroid medication dose has been changed within the last 3 months.
- You have taken part in another drug trial in the last 3 months, or you have ever received gene therapy before.
- You are currently taking part in another treatment-based clinical trial.
- A blood test shows a high level of antibodies against a specific virus used in the study treatment (AAV9), above a level of 1:5 (confirm with trial site).
- You are pregnant, planning to become pregnant in the next 12 months, or currently breastfeeding.
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: 919-561-6210
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
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Trial details
Where this trial is recruiting
Primary endpoints
Adverse Events
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.