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:

• The patient (or their parent/guardian) must give written agreement to take part in the trial
• The patient must be a boy aged 4 to 9 years old who can walk and has been officially diagnosed with Duchenne muscular dystrophy (DMD) with symptoms of the disease
• The patient must have a specific type of genetic change (a frameshift or nonsense mutation) in the DMD gene
• The patient must have a high level of a muscle enzyme called creatine phosphokinase (above 5000 U/L) in their blood
• The patient must have a low level of antibodies against a particular virus carrier (AAV9) used in gene therapy (confirm with trial site)
• The patient must be able to communicate with the study doctor and complete physical activity tests
• The patient must reach certain scores in physical ability tests at the screening stage, including a movement test score of 22 or higher, be able to get up from lying down in under 5 seconds, and/or walk at least 350 metres in 6 minutes
• The patient must have been taking steroid tablets (glucocorticosteroids) at a steady dose for at least 12 weeks before joining, and must plan to continue taking them during the study
• If the patient is taking a steroid called deflazacort, the doctor must believe that switching to prednisolone will not seriously harm the patient's health
• The patient must have received certain meningitis vaccines no later than 4 weeks before receiving the study treatment (confirm with trial site for exact timing requirements)

Who may not be able to join:

• The patient has a known allergy or sensitivity to any ingredient in the study drug or placebo
• The patient has thinking or learning difficulties, or does not move around enough, that might affect their ability to complete physical tests — as judged by the doctor
• The patient has certain specific genetic changes (mutations) in particular sections of the DMD gene (confirm with trial site, as the affected sections depend on which group the patient would be placed in)
• The patient shows signs of heart muscle disease, including poor heart pumping function seen on an ultrasound of the heart
• The patient cannot have an MRI scan for any reason
• The patient has a history of any autoimmune disease (where the immune system attacks the body), except for a thyroid condition that is well controlled with medication
• The patient has a history of tuberculosis, or tests positive or has an unclear result for tuberculosis screening
• The patient tests positive for hepatitis B, hepatitis C, or HIV
• The patient has had a recent infection that cleared up less than 4 weeks before receiving the study treatment
• The patient has received a live vaccine in the last 3 months, or any other type of vaccine in the last 4 weeks before receiving the study treatment
• The patient has certain abnormal blood or liver test results (confirm specific values with trial site)
• The patient has previously received certain DMD treatments, including exon-skipping drugs, ataluren, gene therapy, or cell therapy
• The patient has taken immune-suppressing medications (other than steroids) within the past 12 weeks
• The patient has taken part in another clinical trial within the last 6 months
• The patient or their parent/guardian is unwilling or unable to follow the rules and procedures of the trial
• The patient has any other health condition that the doctor or trial organiser believes would make participation unsafe or unsuitable

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