NCT07290062

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 23 (estimated)
• Sponsor: Insmed Gene Therapy LLC · Industry
• Sex: All
• Age: 18 Years – 79 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
GENETIC	INS1202	Suspension for injection.

Timeline

• Start date: 2026-01-09
• Primary completion: 2030-03-31
• Completion: 2030-03-31
• First posted: 2025-12-17
• Last updated: 2026-03-23

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study