RECRUITINGPhase 1INTERVENTIONAL
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis
About This Trial
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.
Who May Be Eligible (Plain English)
Key Who May Qualify: -
- 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 Who Should NOT Join This Trial: -
- 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.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
GENETIC
INS1202
Suspension for injection.
Locations (3)
USA001
Columbia, Missouri, United States
USA007
Columbus, Ohio, United States
USA006
Philadelphia, Pennsylvania, United States