Duchenne Muscular DystrophyPhase 1ASCENDNCT06817382
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 study testing INS1201 in people with Duchenne Muscular Dystrophy, run by Insmed Gene Therapy LLC at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 4 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- INS1201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 10 sitesUnited States
- Study dates
- 22 Jul 2025to 31 Mar 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 4 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
- Exception is made for seasonal influenza and coronavirus disease 2019 (COVID-19) vaccines, for which shared decision-making with the participant's physician is encouraged.
Who cannot join
- 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:
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- Creates unnecessary risks for undergoing gene transfer;
- Might compromise the participant's ability to comply with the protocol-required testing or procedures; or
- Might compromise the participant's well-being, safety, or clinical interpretability.
- 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.
- Note: Other inclusion/exclusion criteria may apply.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Rare Disease Research (USA004)
Atlanta, Georgia, 30329
USA006
Columbus, Ohio, 43205
USA010
Davis, California, 95616
USA012
Little Rock, Arkansas, 72202
USA009
Los Angeles, California, 90095
USA001
Memphis, Tennessee, 38105
USA015
Norfolk, Virginia, 23507
USA002
Palo Alto, California, 94070
USA008
Rochester, New York, 14642
USA005
San Diego, California, 93123
About this study
Official title: 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)
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
- Duchenne Muscular Dystrophy
- Being tested
- INS1201
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INS1201Genetic
Suspension for IT injection.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Cohort 1 | Experimental | Participants aged 3 to <5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1. Receives: Genetic: INS1201 |
| Part 1: Cohort 2 | Experimental | Participants aged 3 to <5 years will receive a single dose level 2 of INS1201 by IT injection on Day 1. Receives: Genetic: INS1201 |
| Part 2: Cohort 3 | Experimental | Participants aged 2 to <3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1. Receives: Genetic: INS1201 |
| Part 2: Cohort 4 | Experimental | Participants aged 2 to <3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1. Receives: Genetic: INS1201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parts 1 and 2: Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Measured: Up to Week 96
Secondary outcomes (3)
Parts 1 and 2: Recommended Phase 2 Dose (RP2D) of INS1201
Measured: Week 16
Parts 1 and 2: Change From Baseline in Quantity of Micro-Dystrophin Deoxyribonucleic Acid (DNA) as Measured by Droplet Digital Polymerase Chain Reaction (ddPCR) at Weeks 16 and 48
Measured: Baseline, Weeks 16 and 48
Parts 1 and 2: Change From Baseline in Micro-Dystrophin Protein Expression as Measured by Quantitative Protein Analysis at Weeks 16 and 48
Measured: Baseline, Weeks 16 and 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2025
- Study start22 Jul 2025
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Insmed Gene Therapy LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06817382
- Sponsor's protocol ID
- INS1201-101
- Acronym
- ASCEND
- Status last verified
- Oct 2026
- First submitted
- 4 Feb 2025
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06817382
- Acronym / sponsor ID
- ASCEND / INS1201-101
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Insmed Gene Therapy LLCIndustry (pharma, biotech, devices)
- Study start
- 22 Jul 2025 (actual)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 10 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part 1: Cohort 1 | Experimental | Genetic: INS1201Participants aged 3 to <5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1. |
| Part 1: Cohort 2 | Experimental | Genetic: INS1201Participants aged 3 to <5 years will receive a single dose level 2 of INS1201 by IT injection on Day 1. |
| Part 2: Cohort 3 | Experimental | Genetic: INS1201Participants aged 2 to <3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1. |
| Part 2: Cohort 4 | Experimental | Genetic: INS1201Participants aged 2 to <3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1. |
Who may qualify
- Ages
- 2 to 4 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| INS1201Suspension for IT injection. | Genetic | Part 1: Cohort 1, Part 1: Cohort 2, Part 2: Cohort 3, Part 2: Cohort 4 |
Primary outcome
Parts 1 and 2: Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to Week 96
3 secondary outcomes
Parts 1 and 2: Recommended Phase 2 Dose (RP2D) of INS1201
Time frame: Week 16
Parts 1 and 2: Change From Baseline in Quantity of Micro-Dystrophin Deoxyribonucleic Acid (DNA) as Measured by Droplet Digital Polymerase Chain Reaction (ddPCR) at Weeks 16 and 48
Time frame: Baseline, Weeks 16 and 48
Parts 1 and 2: Change From Baseline in Micro-Dystrophin Protein Expression as Measured by Quantitative Protein Analysis at Weeks 16 and 48
Time frame: Baseline, Weeks 16 and 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing INS1201 in people with Duchenne Muscular Dystrophy, run by Insmed Gene Therapy LLC at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Parts 1 and 2: Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) (time frame: Up to Week 96). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2026; AzTrials last refreshed its copy on 8 Nov 2026.