Spinal gene injection aims to slow duchenne in toddlers

NCT ID NCT06817382

Recruiting now Disease control Sponsor: Insmed Gene Therapy LLC Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 2 times

Summary

This early-stage study tests a single injection of a gene therapy called INS1201, given into the spinal fluid of young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to check if the treatment is safe and to see how it spreads in the body. Researchers will also measure if the therapy helps produce a shortened version of the missing muscle protein.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Rare Disease Research (USA004)
RECRUITING

Atlanta, Georgia, 30329, United States
USA001
RECRUITING

Memphis, Tennessee, 38105, United States
USA002
RECRUITING

Palo Alto, California, 94070, United States
USA005
RECRUITING

San Diego, California, 93123, United States
USA006
RECRUITING

Columbus, Ohio, 43205, United States
USA008
RECRUITING

Rochester, New York, 14642, United States
USA009
RECRUITING

Los Angeles, California, 90095, United States
USA010
RECRUITING

Davis, California, 95616, United States
USA012
RECRUITING

Little Rock, Arkansas, 72202, United States
USA015
RECRUITING

Norfolk, Virginia, 23507, United States

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