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Recruiting Phase 1 NCT07290062

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

Phase 1 – testing safety in a small group of people
Conditions: Amyotrophic Lateral Sclerosis

Sponsor: Insmed Gene Therapy LLC

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Overview
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.
Who can participate
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.
Interventions
INS1202
GENETIC
Locations 3
United States (3)
USA001
Columbia , Missouri
USA007
Columbus , Ohio
USA006
Philadelphia , Pennsylvania
Technical details
Status
Recruiting
Phase
Phase 1
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
18 Years
Maximum age
79 Years
Healthy volunteers
No
Start date
09.01.2026
Completion date
31.03.2030
Registry ID
NCT07290062
Source
clinicaltrials.gov
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