Print

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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.