Summary

Eligibility
for people ages 10 years and up (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
completion around

Description

Summary

The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).

Official Title

A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS)

Details

This is a multi-center, three-part study of ION363 in up to 95 participants. Part 1 will consist of participants who will be randomized in a 2:1 ratio to receive a multi-dose regimen of ION363 or placebo for a period of 60 weeks, followed by Part 2, in which participants will receive open-label ION363 for a period of 84 weeks. Participants may continue to receive open-label ION363 in Part 3 for up to 3 additional years or until ION363 becomes commercially available in the patient's country or until the Sponsor discontinues the ION363 development program, whichever occurs earlier.

Keywords

Amyotrophic Lateral Sclerosis, Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations, Sarcoma Mutations, Sarcoma, Motor Neuron Disease, Sclerosis, ION363

Eligibility

Locations

  • University of California San Diego accepting new patients
    La Jolla California 92037 United States
  • Stanford University Medical Center accepting new patients
    Palo Alto California 94304 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT04768972
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 95 study participants
Last Updated