A 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 45 skipping to evaluate the safety and efficacy of ENTR-601-45 (ELEVATE-45)

This study is particularly looking for specific participants. It's for boys and young men who were assigned male at birth, are between 4 and 20 years old, and have a clear diagnosis of Duchenne muscular dystrophy. Crucially, their DMD must be caused by a specific genetic change that allows the new medicine, ENTR-601-45, to work by skipping a particular 'exon 45' in their genes.

There are also some reasons why someone might not be able to join. For example, if they have other serious health problems, especially with their kidneys, or if they've recently had another illness. People who have had certain other DMD treatments, like gene therapy or other 'exon skipping' therapies, or are taking certain medications, also wouldn't be able to participate. This helps make sure the study focuses on the effects of ENTR-601-45 by itself.

If you decide to take part in this study, you'll first go through a screening process to make sure you're a good fit. This will involve giving samples like blood and urine, along with other health checks. Once enrolled, you'll be randomly assigned to receive either the study medicine, ENTR-601-45, or a placebo (a dummy medicine with no active ingredients).

The study will last for 25 weeks. During this time, you'll have regular hospital visits for health checks. These check-ups will include physical exams, measuring your heart rate and blood pressure, heart scans (ECG and echocardiogram), and tests to see how your muscles are working. You will also have two small muscle biopsies, which is when a tiny piece of muscle is taken for examination. This helps doctors see if the medicine is making changes to the muscle. After the 25 weeks, all participants, including those who received the placebo, may have the option to receive ENTR-601-45 in a longer follow-up study.