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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)

NCT04768972 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 89

Last updated 2026-02-18

No results posted yet for this study

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).

Conditions

Interventions

DRUG

ION363

ION363 will be administered by IT bolus injection.

DRUG

Placebo

Placebo will be administered by IT bolus injection.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-06-14
Primary Completion
2026-06-30
Completion
2028-03-31
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • Canada
  • Germany
  • Ireland
  • Italy
  • Japan
  • Netherlands
  • Poland
  • South Korea
  • Spain
  • Sweden
  • Switzerland
  • Taiwan
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04768972 on ClinicalTrials.gov