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)

ClinicalTrials.gov ID: NCT04768972

Public ClinicalTrials.gov record NCT04768972. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

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)

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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).

Study identification

NCT ID: NCT04768972
Recruitment status: Active, not recruiting
Study type: Interventional
Phase: Phase 3
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Enrollment: 89 participants

Conditions and interventions

Conditions

Amyotrophic Lateral Sclerosis

Interventions

ION363 Drug
Placebo Drug

Drug

Eligibility (public fields only)

Age range: 10 Years and older
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Jun 13, 2021
Primary completion: Jun 16, 2026
Completion: Feb 29, 2028
Last update posted: Jul 20, 2026

2021 – 2028

United States locations

U.S. sites: 8
U.S. states: 7
U.S. cities: 8

Facility	City	State	ZIP	Site status
University of California San Diego	La Jolla	California	92037	—
Stanford University Medical Center	Palo Alto	California	94304	—
Johns Hopkins University	Baltimore	Maryland	21205	—
Massachusetts General Hospital	Boston	Massachusetts	02114	—
Washington University School of Medicine	St Louis	Missouri	63110	—
Columbia University Medical Center	New York	New York	10032	—
The Ohio State University Wexner Medical Center	Columbus	Ohio	43210	—
University of Utah	Salt Lake City	Utah	84132	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 17 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT04768972, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Jul 20, 2026 · Synced Sep 10, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04768972 live on ClinicalTrials.gov.

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