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A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT07290062 · Insmed Incorporated
In plain English
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Official title
A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis
About this study
The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to \<80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.
Eligibility criteria
Key Inclusion Criteria: -
* Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m\^2).
* Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria.
* Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS.
* SOD1-ALS (Cohorts 2 and 3 only): Confirmed pathogenic SOD1 mutation, with negative testing for other genetic mutations associated with familial ALS.
* Any polymorphism or mutation in the coding region will require additional review by the Sponsor to determine compatibility with the study intervention.
* Baseline ALSFRS-R ≥ 24.
* ALS disease duration ≤ 42 months.
Key Exclusion Criteria: -
* Previous treatment for ALS with cellular or gene therapies.
* Any investigational medication or treatment (for ALS or other condition).
Note: Other protocol-defined inclusion/exclusion criteria may apply.
Study design
Enrollment target: 23 participants
Allocation: non_randomized
Masking: none
Age groups: adult, older_adult
Timeline
Starts: 2026-01-09
Estimated completion: 2030-03-31
Last updated: 2026-03-23
Interventions
Genetic: INS1202
Primary outcomes
- • Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) (Up to 48 weeks)
Sponsor
Insmed Gene Therapy LLC · industry
Contacts & investigators
ContactInsmed Medical Information · contact · medicalinformation@insmed.com · 1-844-446-7633
All locations (3)
USA001Recruiting
Columbia, Missouri, United States
USA007Recruiting
Columbus, Ohio, United States
USA006Recruiting
Philadelphia, Pennsylvania, United States