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

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-79
SponsorInsmed Gene Therapy LLC

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.

Eligibility criteria

Qualifiers

Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m²).

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.

Disqualifiers

Previous treatment for ALS with cellular or gene therapies.

Any investigational medication or treatment (for ALS or other condition).

Trial design

Treatments tested in this trial

  • INS1202

Treatment groups

23 Participants
are divided into 3 treatment groups

Sponsors and collaborators