A Study of INS1201 for Duchenne Muscular Dystrophy
This study is testing INS1201, a genetic treatment given as a single injection into the spinal fluid (intrathecal (IT) injection), for young boys with Duchenne Muscular Dystrophy (DMD). Researchers want to see if INS1201 is safe and how well your body tolerates it. This study is for males aged 2 to under 5 years old who can walk on their own. The main goal is to track any side effects or problems that happen up to 96 weeks after the injection. The study plans to enroll 12 participants, but its current recruitment status is unclear.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 12 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 96 weeks to monitor for side effects.
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NCT06817382
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Recruiting
PHASE1Ages 2–4InterventionalTreatmentInsmed Gene Therapy LLC
~12 participants
Updated 2026-07-28 on ClinicalTrials.gov
What's tested:INS1201
At a glance
Recruiting sites
10 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Parts 1 and 2: Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Measured over Up to Week 96
Conditions
Where it's being run
10 sites across 7 statesCalifornia4
Arkansas1
Georgia1
New York1
Ohio1
Tennessee1
Virginia1
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Participant must be male at birth, 3 to \<5 years of age, inclusive (Part 1) and 2 to \<3 years of age (Part 2), at the time of legally authorized representative (LAR) signing and dating the informed consent form.
Ambulatory -as defined as the ability to walk at least 10 meters unassisted (ie, without personal assistance or use of any assistive devices) Note: children who have not yet developed the ability to walk by the time of screening (for whatever reason) will not be eligible for the study.
Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).
Able to cooperate with motor assessment testing.
Has received vaccinations recommended for the participant's age and DMD disease according to Centers for Disease Control and Prevention (CDC) Child and Adolescent Immunization Schedule by Age, World Health Organization, or local recommendation incorporating the Advisory Committee on Immunization Practices (ACIP) Vaccine Recommendations and Guidelines for Patients with Altered Immunocompetence.
Exclusion
Prior treatment with gene or cell-based therapy at any time.
Oligonucleotide-based exon skipping or small molecule stop codon readthrough-promoting therapies for at least 6 months prior to enrolment.
Has left ventricular ejection fraction \< 50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy.
Has cardiac arrhythmia or significant electrocardiogram (ECG) interval abnormalities.
Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study at any time during this study.
The presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic/allergic, behavioural disease, infection, unhealed injury, malignancy, concomitant illness, extenuating circumstance, or requirement for chronic drug treatment that, in the opinion of the Investigator:
Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
Has signs of clinically significant symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
Has contraindications for IT administration of the product or for lumbar puncture, such as anatomical abnormalities, bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or significant clotting abnormalities).
Demonstrates cognitive or developmental delay or impairment that could confound assessment of motor development in the opinion of the Investigator.
Total serum anti-AAV9 antibody titers of \> 1:50 as determined by ELISA within 14 days of Day 1.
What this trial measures
- Parts 1 and 2: Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)Up to Week 96