NCT04768972

FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

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)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 89 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 10 Years
• Healthy volunteers: Not accepted

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).

Detailed description

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	ION363	ION363 will be administered by IT bolus injection.
DRUG	Placebo	Placebo will be administered by IT bolus injection.

Timeline

• Start date: 2021-06-14
• Primary completion: 2026-06-01
• Completion: 2028-03-01
• First posted: 2021-02-24
• Last updated: 2026-02-18

Locations

25 sites across 16 countries: United States, Belgium, Brazil, Canada, Germany, Ireland, Italy, Japan, Netherlands, Poland, South Korea, Spain, Sweden, Switzerland, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study