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A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Testing a new treatment for boys with Duchenne Muscular Dystrophy.

Recruiting
2-4 years
Male
Phase 1

Researchers are studying a treatment called **INS1201** to see if it's safe for boys who can walk and have a condition called **Duchenne Muscular Dystrophy (DMD)**. This condition makes muscles weaker over time. The study involves a single injection of INS1201 directly into the spine, which is known as an **intrathecal (IT) injection**. Boys who join the study need to be 2 to 5 years old and able to walk at least 10 meters without help. They also need to have a specific change in their **DMD gene** that affects a protein called **dystrophin**. Boys cannot join if they have had certain treatments before or have other health issues like heart problems or infections. This study does not involve gene or cell-based treatments.

  • The study involves a single injection of the medication.
  • Participants must be 2-5 years old and able to walk.
  • Previous treatments or certain health conditions may exclude participation.
Study details
    Duchenne Muscular Dystrophy

NCT06817382

Insmed Gene Therapy LLC

26 April 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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