A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
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
Sex
ALL
Min age
18 Years
Max age
79 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) — Up to 48 weeks
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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