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)
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.
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)
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
Interventions
- ION363 Drug
- Placebo Drug
Drug
Eligibility (public fields only)
- Age range
- 10 Years and older
- Sex
- All
- Healthy volunteers
- Not listed
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
- May 31, 2026
- Completion
- Feb 29, 2028
- Last update posted
- Feb 17, 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 Feb 17, 2026 · Synced Apr 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04768972 live on ClinicalTrials.gov.