Recruiting
Phase 1

INS1202

Sponsor:

Insmed Gene Therapy LLC

Code:

NCT07290062

Conditions

Amyotrophic Lateral Sclerosis

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

INS1202

Study Details

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Study ID

NCT07290062

Start date

Jan 9, 2026

Status verified date

Sep, 2026

Completion date

Mar 31, 2030

Anticipated

Primary completion date

Mar 31, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

23 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1

Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.

experimental: Cohort 2

Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.

experimental: Cohort 3

Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.

Interventions

INS1202

Suspension for injection.

Primary outcome measure

  • Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 48 weeks ]

Central Contacts and Locations

Central contacts

Insmed Medical Information

1-844-446-7633medicalinformation@insmed.com

Locations

USA004

Recruiting

La Jolla, California, United States, 92037

Contacts

rprevite@health.ucsd.edu

USA002

Recruiting

Palo Alto, California, United States, 94304

Contacts

NEUROMUSCULARRESEARCH@STANFORD.EDU

USA003

Recruiting

Baltimore, Maryland, United States, 21205

Contacts

emosmil1@jhmi.edu

USA009

Recruiting

Worcester, Massachusetts, United States, 01655

Contacts

danielle.kokoski@umassmed.edu

USA001

Recruiting

Columbia, Missouri, United States, 65211

Contacts

heathermchatton@health.missouri.edu

USA007

Recruiting

Columbus, Ohio, United States, 43221

Contacts

ALSResearch@osumc.edu

USA006

Recruiting

Philadelphia, Pennsylvania, United States, 19107

Contacts

Alexander.McGeary@jefferson.edu

More Information

Sponsor

Insmed Gene Therapy LLC

Last update posted

Sep 23, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-02. This information was provided to ClinicalTrials.gov by Insmed Gene Therapy LLC on 2026-09-23. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.