NCT07290062 A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
| NCT ID | NCT07290062 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Insmed Gene Therapy LLC |
| Condition | Amyotrophic Lateral Sclerosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 23 participants |
| Start Date | 2026-01-09 |
| Primary Completion | 2030-03-31 |
Trial Parameters
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Brief 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.
Eligibility Criteria
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.
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