Summary

Eligibility
for people ages 18-79 (full criteria)
Location
at UCSD
Dates
study started
study ends around

Description

Summary

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.

Official Title

A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis, INS1202

Eligibility

You can join if…

Open to people ages 18-79

-

  • 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.
  • 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 CAN'T join if...

-

  • 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.

Locations

  • USA004 accepting new patients
    La Jolla California 92037 United States
  • USA002 accepting new patients
    Palo Alto California 94304 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Insmed Gene Therapy LLC
ID
NCT07290062
Phase
Phase 1 Amyotropic Lateral Sclerosis (ALS) Research Study
Study Type
Interventional
Participants
Expecting 23 study participants
Last Updated