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

Insmed Gene Therapy LLC

Status and phase: Enrolling; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Genetic: INS1202

Study type: Interventional

Funder types: Industry

Identifiers

NCT07290062

INS1202-101

Details and patient eligibility

About

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.

Enrollment: 23 estimated patients

Sex: All

Ages: 18 to 79 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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23 participants in 3 patient groups

Cohort 1

Experimental group

Description:

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

Treatment:

Genetic: INS1202

Cohort 2

Experimental group

Description:

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

Treatment:

Genetic: INS1202

Cohort 3

Experimental group

Description:

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

Treatment:

Genetic: INS1202