Phase 2 Muscular Dystrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• For Part A: You are between 18 and 55 years old (inclusive) at the time of signing the consent form.
• For Part B: You are between 10 and 17 years old, OR between 18 and 55 years old (inclusive) at the time of signing the consent form.
• You have myotonic dystrophy type 1 (DM1) that was not present from birth (non-congenital onset).
• You have been diagnosed by a doctor with signs of DM1, including muscle stiffness (myotonia) and muscle weakness.
• You have had a genetic test confirming DM1, showing a CTG repeat length of 50 or more on at least one copy of the relevant gene.
• You are able to walk at least 10 meters on your own (walking aids such as leg braces or ankle supports are allowed).

Who may not be able to join:

• You have antibodies in your blood that could interfere with the gene therapy vehicle (AAV.SAN011) being used in this trial (confirm with trial site).
• Your heart's pumping ability is below 50% (as measured by a specific heart test called ejection fraction).
• You have liver or bile duct problems, shown by certain blood test results being significantly above normal levels (confirm with trial site for specific values).
• You have kidney disease, shown by certain blood test results indicating reduced kidney function (confirm with trial site for specific values).
• You need breathing assistance (such as a ventilator) for 6 or more hours per day for at least 21 days in a row due to long-term breathing difficulties.
• You have a medical reason why you cannot take steroid medications (corticosteroids), or a condition that could get worse if you take them, as decided by the study doctor.
• You currently have an active hepatitis B or hepatitis C infection, or are currently taking antiviral medication for either of these.
• You have a specific hepatitis B marker in your blood (HBcAb positive) and are unable or unwilling to take preventive hepatitis B treatment during the steroid portion of the study (confirm with trial site).
• You are considered at high risk of tuberculosis becoming active again during steroid treatment, as decided by the study doctor.
• You have a known HIV infection.
• You have a serious illness that, in the study doctor's opinion, would make it unsafe for you to participate or could affect your survival.
• You have a recent history of, or currently have, drug or alcohol misuse within the past 12 months before screening.
• You have had a biopsy of a specific lower leg muscle (tibialis anterior) within the past 12 weeks, or you are planning to have one during the trial.
• You have significant difficulties with learning, intellectual disability, or serious behavioral or psychiatric symptoms, as decided by the study doctor.
• You have taken steroid medications at doses above 5 mg per day within 15 days before the first study visit.
• You have taken medication specifically to treat muscle stiffness (myotonia) within 15 days before the first study visit.
• You have been identified by the study doctor as having a serious heart risk.
• The study doctor considers you unsuitable to participate for any medical or other reason.

Important: Always verify eligibility with the trial site directly before applying.