Investigating the safety of INS1201 injection in boys with Duchenne Muscular Dystrophy

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)

This study is testing whether a new injection is safe for young boys with Duchenne Muscular Dystrophy to see if it could help them in the future.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 2 Years to 4 Years
• Sex: Male
• Sponsor: Insmed Incorporated (Industry-sponsored)
• Locations: 10 sites (Little Rock, Arkansas and 9 other locations)
• Trial ID: NCT06817382 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and tolerability of a single intrathecal injection of INS1201 in ambulatory male participants diagnosed with Duchenne Muscular Dystrophy (DMD). The trial focuses on young boys aged 2 to 5 years, assessing their ability to walk unassisted and confirming their genetic diagnosis of DMD. Participants will receive the treatment and undergo monitoring to determine the biodistribution and safety profile of the drug. The study aims to gather critical data that could inform future treatments for DMD.

Who should consider this trial

Good fit: Ideal candidates are ambulatory males aged 2 to 5 years with a confirmed genetic diagnosis of Duchenne Muscular Dystrophy.

Not a fit: Patients who are non-ambulatory or do not have a confirmed genetic diagnosis of DMD will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new therapeutic option for managing Duchenne Muscular Dystrophy in young patients.

How similar studies have performed: While this approach is novel, similar studies targeting genetic therapies for DMD have shown promising results in other contexts.

Eligibility criteria

Inclusion Criteria

* 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.

Exclusion Criteria

* 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:

  1. Creates unnecessary risks for undergoing gene transfer;
  2. Might compromise the participant's ability to comply with the protocol-required testing or procedures; or
  3. 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.

Where this trial is running

Little Rock, Arkansas and 9 other locations

• Usa012 — Little Rock, Arkansas, United States (Recruiting)
• Usa010 — Davis, California, United States (Recruiting)
• Usa009 — Los Angeles, California, United States (Recruiting)
• Usa002 — Palo Alto, California, United States (Recruiting)
• Usa005 — San Diego, California, United States (Recruiting)
• Rare Disease Research (USA004) — Atlanta, Georgia, United States (Recruiting)
• Usa008 — Rochester, New York, United States (Recruiting)
• Usa006 — Columbus, Ohio, United States (Recruiting)
• Usa001 — Memphis, Tennessee, United States (Recruiting)
• Usa015 — Norfolk, Virginia, United States (Recruiting)

Study contacts

• Study coordinator: Insmed Medical Information

Conditions Duchenne Muscular Dystrophy