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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: 2026-05-20
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 (5)
USA004Recruiting
La Jolla, California, United States
USA002Recruiting
Palo Alto, California, United States
USA001Recruiting
Columbia, Missouri, United States
USA007Recruiting
Columbus, Ohio, United States
USA006Recruiting
Philadelphia, Pennsylvania, United States