Phase 1 Study of a Single Intrathecal Injection of INS1202 for Safety and Pharmacodynamics in Adults with Amyotrophic Lateral Sclerosis

What this Trial Is About

Researchers are evaluating the safety, tolerability, and pharmacodynamics of a single dose of INS1202 given by intrathecal IT injection in adults aged 18 to under 80 years with amyotrophic lateral sclerosis ALS. The study focuses on participants who either have mutations in the superoxide dismutase type 1 SOD1 gene or have no known ALS-related genetic mutation. This is a Phase 1, open-label, dose-finding trial aiming to determine the appropriate dose and safety profile of INS1202. Participants will receive a single IT injection of INS1202 at one of three dose levels depending on their cohort. Cohort 1 includes participants with sporadic ALS receiving dose level 1, while Cohorts 2 and 3 include participants with either sporadic ALS or SOD1-ALS receiving dose levels 2 and 3 respectively. The treatment is administered only once on Day 1, and participants are monitored over time for safety and biological effects. During the study, participants will be followed for up to 48 weeks to assess the incidence and severity of any treatment-emergent adverse events. Researchers will also evaluate the recommended Phase 2 dose and monitor viral vector shedding at multiple time points including baseline and weeks 1, 2, 4, 16, and 32. The involvement includes clinical assessments and laboratory tests to track the treatments effects and safety over nearly a year.

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)