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

Purpose

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.

Condition

  • Amyotrophic Lateral Sclerosis

Eligibility

Eligible Ages
Between 18 Years and 79 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

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

Phase
Phase 1
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Cohort 1 		Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.
  • Genetic: INS1202
    Suspension for injection.
Experimental
Cohort 2 		Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.
  • Genetic: INS1202
    Suspension for injection.
Experimental
Cohort 3 		Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.
  • Genetic: INS1202
    Suspension for injection.

Recruiting Locations

USA001
Columbia 4381982, Missouri 4398678 65212

More Details

Status
Recruiting
Sponsor
Insmed Gene Therapy LLC

Study Contact

Insmed Medical Information
1-844-446-7633
medicalinformation@insmed.com