RecruitingPhase 1NCT06817382

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

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 (The ASCEND Study)

Sponsor

Insmed Gene Therapy LLC

Enrollment

12 participants

Start Date

Jul 22, 2025

Study Type

INTERVENTIONAL

Conditions

Duchenne Muscular Dystrophy

Summary

The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.

Eligibility

Sex: MALEMin Age: 2 YearsMax Age: 4 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying INS1201 for people with duchenne muscular dystrophy. The study is currently recruiting participants at 10 locations. People eligible for this study include men aged 2 Years to 4 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICINS1201

Suspension for IT injection.

Locations(10)

USA012

Little Rock, Arkansas, United States

USA010

Davis, California, United States

USA009

Los Angeles, California, United States

USA002

Palo Alto, California, United States

USA005

San Diego, California, United States

Rare Disease Research (USA004)

Atlanta, Georgia, United States

USA008

Rochester, New York, United States

USA006

Columbus, Ohio, United States

USA001

Memphis, Tennessee, United States

USA015

Norfolk, Virginia, United States

View Full Details on ClinicalTrials.gov

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NCT06817382

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