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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Usa005
  Phoenix, Arizona, United States Recruiting
• Usa004
  La Jolla, California, United States Recruiting
• Usa002
  Palo Alto, California, United States Recruiting
• Usa003
  Baltimore, Maryland, United States Recruiting
• Usa009
  Worcester, Massachusetts, United States Recruiting
• Usa001
  Columbia, Missouri, United States Recruiting

Study contacts

• Study coordinator: Insmed Medical Information
• Email: medicalinformation@insmed.com
• Phone: 1-844-446-7633

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.