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

What this study involves

The registry says this study is recruiting. Individual locations may have different availability.

Study type

Interventional

Phase

Phase 1

Age range

18 years - 79 years

Sex eligible

All sexes

Healthy volunteers

Not accepted

Participants

23 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

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 able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

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.

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment

Not randomized

Masking

No masking

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Secondary measures

Research locations (9)

The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.

9 locations

Study team and timeline

Lead sponsor

Insmed Gene Therapy LLC

Study start

Jan 9, 2026 Actual

Primary completion

Mar 31, 2030 Estimated

Study completion

Mar 31, 2030 Estimated

First posted

Dec 17, 2025 Actual

Last updated

Nov 10, 2026 Actual

Official study contacts