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NCT07290062PHASE1Recruiting

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Insmed Gene Therapy LLC

Start Date

1/9/2026

Completion Date

3/31/2030

Summary

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

Age Range: 18 years to 79 years

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.

Interventions

GENETIC

INS1202

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Conditions

Amyotrophic Lateral Sclerosis

Locations

USA004

La Jolla, California 92037

United States

USA002

Palo Alto, California 94304

United States

USA001

Columbia, Missouri 65211

United States

USA007

Columbus, Ohio 43221

United States

USA006

Philadelphia, Pennsylvania 19107

United States