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RECRUITINGPhase 1INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Key Who May Qualify: - - 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 Who Should NOT Join This Trial: - - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

GENETIC

INS1202

Suspension for injection.

Locations (3)

USA001
Columbia, Missouri, United States
USA007
Columbus, Ohio, United States
USA006
Philadelphia, Pennsylvania, United States