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

Sponsored by Insmed Gene Therapy LLC

About this trial

Last updated 6 days ago

Study ID

INS1202-101

Status

Recruiting

Type

Interventional

Phase

Phase 1

Placebo

No

Accepting

18 to 79 Years
All Sexes

Trial Timing

Started 5 months ago

What is this trial about?

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.

What are the Participation Requirements?

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.

Study Documents

Plain Language Protocol Synopsis (PLPS) - INS1202-101_Lay Protocol Synopsis_Final_V1.0_English (United States)

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For more information, view the full study details:

NCT07290062