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DYNEDMD: Measuring Safety and How Well DYNE-251 Works in Boys with Duchenne Muscular Dystrophy

About this study

This study tests a new drug called DYNE-251 in young males with Duchenne muscular dystrophy to see if it can improve muscle strength, function, and quality of life. The drug works by increasing the production of dystrophin in the muscles. This drug may work for those who have DMD and have a specific genetic change. This change means an area of DNA on the dystrophin gene, called exon 51, is missing. DYNE-251 is an experimental drug that has not been approved to treat DMD in people. This study is the first time DYNE-251 is being tested in humans.

Study participants will be randomly assigned to receive either the study drug or a placebo for 6 months. The drug will be given through the vein. This process is called infusion. They will receive the study drug for up to 2.5 years. After the first infusion of the study drug, participants will stay at the hospital to be monitored. Then they will have infusions every four weeks. They can go home after each of these infusions and do not need to stay in the hospital for monitoring.

Eligibility overview

  • Males aged 4 to 16 with a diagnosis of Duchenne muscular dystrophy

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Overview

Full title:

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Study goal:

The main goal of this study is to see if DYNE-251 is safe to use in young males with DMD. Researchers also want to find out how well DYNE-251 treats the disease and how it affects the body in other ways.

Diagnosis:

Duchenne muscular dystrophy

Age:

Male, age 4 to 16 years old

Clinical trials categories:

Neurological Disorders Duchenne Muscular Dystrophy

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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