Actively Recruiting
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Led by Insmed Gene Therapy LLC · Updated on 2026-04-29
12
Participants Needed
10
Research Sites
140 weeks
Total Duration
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AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male at birth, aged 3 to under 5 years (Part 1) or 2 to under 3 years (Part 2) at consent
- Able to walk at least 10 meters unassisted
- Confirmed diagnosis of Duchenne Muscular Dystrophy by genetic testing with specific mutations in exons 18 to 58
- Able to cooperate with motor assessments
- Up to date on recommended vaccinations for age and DMD according to CDC, WHO, or local guidelines, with exceptions for influenza and COVID-19 vaccines
You will not qualify if you...
- Prior treatment with gene or cell-based therapy
- Use of oligonucleotide exon skipping or stop codon readthrough therapies within 6 months before enrollment
- Left ventricular ejection fraction less than 50% or signs of cardiomyopathy
- Cardiac arrhythmia or significant ECG abnormalities
- Major surgery within 3 months before Day 1 or planned surgery during the study
- Any other significant illness or condition that increases risk or interferes with study participation
- Current, chronic, or active HIV, hepatitis B, or hepatitis C infection
- Symptomatic infection within 4 weeks before Day 1
- Contraindications to intrathecal injection or lumbar puncture (e.g., anatomical abnormalities, bleeding disorders)
- Cognitive or developmental delays that affect motor assessment
- Anti-AAV9 antibody titers greater than 1:50 within 14 days of Day 1
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
USA012
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
USA010
Davis, California, United States, 95616
Actively Recruiting
3
USA009
Los Angeles, California, United States, 90095
Actively Recruiting
4
USA002
Palo Alto, California, United States, 94070
Actively Recruiting
5
USA005
San Diego, California, United States, 93123
Actively Recruiting
6
Rare Disease Research (USA004)
Atlanta, Georgia, United States, 30329
Actively Recruiting
7
USA008
Rochester, New York, United States, 14642
Actively Recruiting
8
USA006
Columbus, Ohio, United States, 43205
Actively Recruiting
9
USA001
Memphis, Tennessee, United States, 38105
Actively Recruiting
10
USA015
Norfolk, Virginia, United States, 23507
Actively Recruiting
Research Team
I
Insmed Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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