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)

Status: Recruiting
Location: See all (8) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1
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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 2
Maximum Age: 4
Healthy Volunteers: f
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• 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.

Locations
United States
Arkansas
USA012
RECRUITING
Little Rock
California
USA010
RECRUITING
Davis
USA002
RECRUITING
Palo Alto
Georgia
Rare Disease Research (USA004)
RECRUITING
Atlanta
New York
USA008
RECRUITING
Rochester
Ohio
USA006
RECRUITING
Columbus
Tennessee
USA001
RECRUITING
Memphis
Virginia
USA015
RECRUITING
Norfolk
Contact Information
Primary
Insmed Medical Information
medicalinformation@insmed.com
18444467633
Time Frame
Start Date: 2025-07-22
Estimated Completion Date: 2028-03-31
Participants
Target number of participants: 12
Treatments
Experimental: Part 1: Cohort 1
Participants aged 3 to \<5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.
Experimental: Part 1: Cohort 2
Participants aged 3 to \<5 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.
Experimental: Part 2: Cohort 3
Participants aged 2 to \<3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.
Experimental: Part 2: Cohort 4
Participants aged 2 to \<3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.
Sponsors
Leads: Insmed Gene Therapy LLC

This content was sourced from clinicaltrials.gov