Actively Recruiting
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Led by Insmed Gene Therapy LLC · Updated on 2026-04-29
23
Participants Needed
5
Research Sites
220 weeks
Total Duration
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AI-Summary
What this Trial Is 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.
CONDITIONS
Official Title
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant with body mass index (BMI) 18 kilograms per square meter (kg/m�5E2).
- 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.
You will not qualify if you...
- Previous treatment for ALS with cellular or gene therapies.
- Any investigational medication or treatment (for ALS or other condition).
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 5 locations
1
USA004
La Jolla, California, United States, 92037
Actively Recruiting
2
USA002
Palo Alto, California, United States, 94304
Actively Recruiting
3
USA001
Columbia, Missouri, United States, 65211
Actively Recruiting
4
USA007
Columbus, Ohio, United States, 43221
Actively Recruiting
5
USA006
Philadelphia, Pennsylvania, United States, 19107
Actively Recruiting
Research Team
I
Insmed Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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