Actively Recruiting
A Phase 1, Open-label Study of a Single Intrathecal Injection of INS1201 Gene Therapy in Ambulatory Males With Duchenne Muscular Dystrophy (The ASCEND Study)
Led by Insmed Gene Therapy LLC · Updated on 2026-05-18
12
Participants Needed
10
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This trial focuses on ambulatory male children aged 2 to under 5 years who have Duchenne Muscular Dystrophy (DMD). Researchers are studying the safety and how the gene therapy INS1201 spreads in the body when given as a single injection into the spinal fluid. The study is a Phase 1, open-label trial designed to evaluate safety and tolerability in this young population. Participants are divided into groups based on their age and receive one of two dose levels of INS1201 administered intrathecally on Day 1. Children aged 3 to under 5 years take part in Part 1 of the study receiving either dose level 1 or 2, and those aged 2 to under 3 years participate in Part 2 with similar dosing options. This single-dose approach helps researchers understand the proper dosing and effects of the treatment. Throughout the study, participants are monitored for treatment-emergent adverse events up to 96 weeks. Researchers also assess the recommended phase 2 dose by Week 16 and measure changes in micro-dystrophin DNA and protein expression at Weeks 16 and 48. Participants will undergo motor assessments and safety evaluations, with the total study duration extending across nearly two years to track both safety and biological responses.
CONDITIONS
Brief 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 participants aged 3 to under 5 years (Part 1) or 2 to under 3 years (Part 2) at consent signing.
- Able to walk at least 10 meters unassisted without devices.
- Definitive diagnosis of Duchenne Muscular Dystrophy based on genetic testing showing specific mutations between exons 18 to 58.
- Able to cooperate with motor assessments.
- Received recommended vaccinations for age and DMD per CDC, WHO, or local guidelines, with shared decision-making allowed for flu and COVID-19 vaccines.
You will not qualify if you...
- Prior treatment with gene or cell-based therapy.
- Use of exon skipping or stop codon readthrough therapies within 6 months before enrollment.
- Left ventricular ejection fraction below 50% or signs/symptoms of cardiomyopathy.
- Cardiac arrhythmia or significant ECG abnormalities.
- Major surgery within 3 months before Day 1 or planned surgery interfering with the study.
- Other significant illnesses or conditions that pose risks or interfere with study participation.
- Current or chronic infection with HIV, hepatitis B, or hepatitis C.
- Symptomatic infection within 4 weeks prior to Day 1.
- Contraindications to intrathecal injection or lumbar puncture.
- Cognitive or developmental delays affecting motor assessment.
- High anti-AAV9 antibody levels above 1:50 within 14 days before Day 1.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day
Participants receive a single intrathecal injection of INS1201 gene therapy.
1 treatment visit (in-person) on Day 1
Duration - Up to 96 weeks
Participants are monitored for safety and gene therapy effects after treatment.
Multiple follow-up visits up to Week 96
Trial Site Locations
Total: 10 locations
1
USA012
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
USA010
Davis, California, United States, 95616
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3
USA009
Los Angeles, California, United States, 90095
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4
USA002
Palo Alto, California, United States, 94070
Actively Recruiting
5
USA005
San Diego, California, United States, 93123
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6
Rare Disease Research (USA004)
Atlanta, Georgia, United States, 30329
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7
USA008
Rochester, New York, United States, 14642
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8
USA006
Columbus, Ohio, United States, 43205
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9
USA001
Memphis, Tennessee, United States, 38105
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10
USA015
Norfolk, Virginia, United States, 23507
Actively Recruiting
Research Team
I
Insmed Medical Information
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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