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 participant must be a boy between 4 and 8 years old (including those who are exactly 4 or 8) at the start of screening
• The participant must have been diagnosed with Duchenne Muscular Dystrophy (DMD), a muscle-weakening condition, confirmed by genetic testing that shows their specific gene change can be targeted by a treatment approach called "exon 51 skipping" (confirm with trial site)
• The participant and/or their parent or guardian must be willing to take part in the trial and able to understand and sign a consent form

Who may not be able to join:

• Boys who show signs of heart failure, specifically where the heart is pumping less than 40% of its normal capacity
• Boys whose lungs are working at less than 40% of the expected breathing capacity for someone their age and size
• Boys whose heart electrical activity test (a standard heart tracing, called an ECG) shows a certain measurement called the QTc interval that is longer than 0.45 seconds, which can indicate a heart rhythm concern

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