Early Phase 1 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:

• The patient (and/or their legal guardian) has read, understood, and signed a consent form agreeing to take part in the study.
• The patient is a boy aged 2 years or older but under 8 years old, and can walk at least 10 meters on his own.
• The patient has been diagnosed with Duchenne muscular dystrophy (DMD) based on medical history and confirmed by a specific genetic test.
• The patient is able to have a small muscle tissue sample (biopsy) taken while under anesthesia, with no medical reasons preventing this.
• The patient's heart is working well enough (the heart pumps at least 50% of its capacity).
• The patient's lungs are working well enough (breathing at least 50% of expected capacity, with no need for a breathing machine at night).
• The patient's kidneys are working well enough (filtering at an adequate level).

Who may not be able to join:

• The patient has another condition besides DMD that also causes muscle weakness or movement problems.
• The patient has a severe intellectual or behavioural condition (such as severe autism or severe cognitive difficulties) that the study doctor feels would affect participation.
• The patient was in hospital for breathing failure in the 8 weeks before the screening visit.
• The patient has poorly controlled asthma or another lung condition (such as bronchitis or recurring lung infections) that the study doctor feels could affect breathing.
• The patient has severe, uncontrolled heart failure, was given heart or fluid medicines through a drip, or was hospitalised due to heart problems or irregular heartbeat in the 8 weeks before screening.
• The patient has abnormal blood or liver test results that are considered medically significant (confirm with trial site for specific values).
• The patient has an irregular heartbeat that requires medication to control.
• The patient is currently taking medicines that suppress the immune system.
• The patient has previously received gene therapy, an experimental drug, or any treatment aimed at increasing a protein called dystrophin.
• The patient had major surgery in the 12 weeks before the first treatment, or is expected to have major surgery (such as spine surgery) during the study.
• The patient is allergic to the study treatment or to local anaesthetic medicines, or has a history of severe allergic reactions.
• The patient has taken part in another clinical trial or received an experimental drug in the 6 months before the first treatment.
• The patient tests positive for hepatitis B, hepatitis C, or HIV at the screening visit.
• The study doctor believes the patient has any other serious illness, medical condition, or ongoing medication that could make participation unsafe.

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