ION363

DRUG2 trials

Sponsors

Ionis Pharmaceuticals Inc., Ionis Pharmaceuticals, Inc.

Conditions

Amyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis with Fused in Sarcoma mutations

Phase 2

ION363-CS1: 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)

Active, not recruitingCTIS2024-512163-31-00

Ionis Pharmaceuticals Inc.Amyotrophic Lateral Sclerosis with Fused in Sarcoma mutations

Start: 2022-01-26Target: 57Updated: 2025-10-27

Phase 3

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)

Active, not recruitingNCT04768972

Ionis Pharmaceuticals, Inc.Amyotrophic Lateral Sclerosis

Start: 2021-06-14End: 2028-03-01Updated: 2026-07-21