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ASCEND: Gene Therapy for Duchenne Muscular Dystrophy

About this study

Duchenne muscular dystrophy (DMD) is a serious disease caused by a missing or non-working protein called dystrophin. Without dystrophin, muscles become damaged and cannot work properly. There are very few treatments that can slow down or stop the muscle damage caused by DMD. Most current treatments do not fix what causes the problem.

The ASCEND study is testing the safety of an experimental gene therapy called INS1201. It will try to help the body make a shorter, but still helpful, version of dystrophin. This will be the first study testing INS1201 in humans.

If this treatment works, it could help many children with DMD live longer and be more active.

The treatment is given as a single injection into the spinal fluid. The goal is to see if the therapy is safe and if it can reach the muscles of the body and heart to help them work better.

By learning more about how INS1201 works and how safe it is, doctors can decide if it should be tested in older children or used in future treatments for DMD. This trial is a first step toward finding a new way to help children with DMD. It could lead to better treatments in the future.

Eligibility overview

  • Male
  • Duchenne muscular dystrophy (DMD)
  • 2–4 years old
  • Can walk at least 10 meters (about 33 feet) unassisted

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Overview

Full title:

ASCEND: A Phase 1, Multicenter, Open-label Study to Investigate the Safety and Biodistribution of a Single Intrathecal Injection of INS1201 in Ambulatory Males with Duchenne Muscular Dystrophy

Study goal:

To study INS1201 gene therapy for Duchenne muscular dystrophy (DMD)

Diagnosis:

Duchenne muscular dystrophy

Age:

2–4 years

Clinical trial 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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