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.
Eligibility criteria
Qualifiers
Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m²).
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.
Disqualifiers
Previous treatment for ALS with cellular or gene therapies.
Any investigational medication or treatment (for ALS or other condition).
Trial design
Treatments tested in this trial
- INS1202