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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
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Recruiting Locations
More Details
- Status
- Recruiting
- Sponsor
- Insmed Gene Therapy LLC