A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Phase 1
Interventional Study
Amyotrophic Lateral Sclerosis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 18 – 79
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 23 (estimated)
- Sponsor
- Insmed Gene Therapy LLC · Industry
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About This Trial
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.
Trial Locations
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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…
Contacts