Single intrathecal injection of INS1202 for adults with ALS (SOD1 or no known mutation)

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

This study will try a single injection of INS1202 into the spinal fluid to see if it is safe and how it acts in adults with ALS who have SOD1 mutations or no known ALS gene mutation.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 23 (estimated)
• Ages: 18 Years to 79 Years
• Sex: All
• Sponsor: Insmed Incorporated (Industry-sponsored)
• Locations: 5 sites (La Jolla, California and 4 other locations)
• Trial ID: NCT07290062 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, dose-finding trial administering a single intrathecal dose of INS1202 to characterize safety, tolerability, and pharmacodynamics. Participants are adults aged 18 to <80 with symptomatic ALS diagnosed by Gold Coast criteria, with separate cohorts for confirmed pathogenic SOD1 mutation carriers and for sporadic ALS patients with no known monogenic mutations. Key eligibility includes BMI ≥18 kg/m2, ALSFRS-R ≥24, and disease duration ≤42 months, while prior cellular or gene therapies and other investigational treatments are excluded. The trial uses ascending single doses at three U.S. sites to define safety and biological effects in the cerebrospinal fluid and clinical measures.

Who should consider this trial

Good fit: Ideal candidates are adults 18–79 with symptomatic ALS (Gold Coast criteria), ALSFRS‑R ≥24, disease duration ≤42 months, and either a confirmed pathogenic SOD1 mutation (for SOD1 cohorts) or no known monogenic ALS mutation (for sporadic cohorts).

Not a fit: People with advanced disease outside the eligibility windows, prior gene or cell therapies, current investigational treatments, or those younger than 18 or older than or equal to 80 are unlikely to qualify or benefit from this single-dose study.

Why it matters

Potential benefit: If safe and biologically active, INS1202 could slow or modify disease processes for some people with ALS, particularly those with SOD1-related disease.

How similar studies have performed: Genetic-targeted approaches for SOD1 ALS, such as antisense therapies, have produced mixed but sometimes promising biological and clinical signals, so this approach builds on partially successful prior work.

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.

Where this trial is running

La Jolla, California and 4 other locations

• Usa004 — La Jolla, California, United States (Recruiting)
• Usa002 — Palo Alto, California, United States (Recruiting)
• Usa001 — Columbia, Missouri, United States (Recruiting)
• Usa007 — Columbus, Ohio, United States (Recruiting)
• Usa006 — Philadelphia, Pennsylvania, United States (Recruiting)

Study contacts

• Study coordinator: Insmed Medical Information

Conditions Amyotrophic Lateral Sclerosis