Phase 1 Muscular Dystrophy Trial, Recruiting

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

Who might be able to join this trial:

• The child must have been born male, and be between 3 and just under 5 years old (Part 1) or between 2 and just under 3 years old (Part 2) when the parent or guardian signs the consent form.
• The child must be able to walk at least 10 meters on their own, without help from another person or any walking aids.
• The child must have a confirmed diagnosis of Duchenne muscular dystrophy (DMD) based on genetic testing, with a specific type of genetic change located in a particular region of the DMD gene (exons 18 to 58) — the exact type of genetic change will be checked by the trial team.
• The child must be able to cooperate with physical movement and motor skill tests.
• The child must have received all age-appropriate vaccinations recommended by health authorities (with some flexibility around flu and COVID-19 vaccines, which can be discussed with the child's doctor).

Who may not be able to join:

• Children who have previously received any gene therapy or cell-based therapy.
• Children who have been treated with certain DMD-specific medications (exon skipping therapies or stop codon readthrough drugs) within the 6 months before joining.
• Children whose heart is not pumping well enough (less than 50% efficiency on a heart scan), or who show signs of heart muscle disease.
• Children with an irregular heartbeat or significant abnormalities shown on a heart tracing (ECG).
• Children who have had major surgery within 3 months before the study starts, or who have planned surgery that might interfere with the study.
• Children with any other serious health condition — including heart, lung, liver, kidney, blood, immune, or behavioural problems — that the doctor feels would make the trial unsafe or affect the results.
• Children with evidence of an active or ongoing HIV, hepatitis B, or hepatitis C infection.
• Children who have had a significant infection (such as pneumonia or a serious respiratory illness) within 4 weeks before the study begins.
• Children who have certain physical conditions that would make a spinal injection (into the lower back) unsafe, such as spina bifida, bleeding disorders, or significant clotting problems.
• Children whose cognitive or developmental delays might make it difficult to accurately assess their motor development, in the opinion of the study doctor.
• Children whose blood test shows high levels of antibodies against the virus used to deliver the therapy (anti-AAV9 antibodies above a certain level) (confirm with trial site).

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