A 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 44 skipping to evaluate the safety and efficacy of ENTR-601-44 (ELEVATE-44)

This study is for boys and young men aged 4 to 20 years old who have Duchenne muscular dystrophy (DMD). To be eligible, you must have been diagnosed with DMD and have a particular change in your dystrophin gene that the study medicine, ENTR-601-44, is designed to target. You should also be able to walk by yourself.

Before joining, you'll have some checks to make sure the study is right for you. This will involve giving samples like blood and urine, and having some other medical tests. You won't be able to join if you have certain other health problems, have recently had other experimental treatments for DMD, or are taking certain other medications that could interfere with the study medicine.

If you join this study, it will involve regular visits for health checks over several years. During these visits, you'll have physical exams, your heart rate, temperature, and blood pressure will be checked. You'll also have heart tests called an ECG and echocardiogram, and tests to see how your muscles are working. You will need to provide blood and urine samples.

For the first 25 weeks, you will either receive the study medicine, ENTR-601-44, or a placebo (a dummy medicine with no active ingredients). Neither you nor the study team will know which you are receiving during this time. After these initial 25 weeks, everyone in the study will then receive the active medicine, ENTR-601-44. You will also have two muscle biopsies during the study. This is where a tiny piece of muscle is taken to see if the medicine is making changes inside your muscles.