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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 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Placebo
- DRUG ION363
Study Locations (20)
Other
- PSEG Centro de Pesquisa Clinica S.A. — São Paulo
- Universitaetsmedizin Rostock — Rostock
- Universitätsklinikum Ulm — Ulm
- St. James Hospital — Dublin
- Citta della Salute e della Scienza di Torino - Ospedale le Molinette — Torino
- Toho University Omori Medical Center — Tokyo
- Universitair Medisch Centrum Utrecht — Utrecht
- Linden spólka z ograniczona odpowiedzialnoscia spólka komandytow — Krakow
- Seoul National University Hospital — Seoul
- Hanyang University Seoul Hospital — Seoul
California
- University of California San Diego — La Jolla
- Stanford University Medical Center — Palo Alto
Maryland
- Johns Hopkins University — Baltimore
Massachusetts
- Massachusetts General Hospital — Boston
Missouri
- Washington University School of Medicine — St Louis
New York
- Columbia University Medical Center — New York
Ohio
- The Ohio State University Wexner Medical Center — Columbus
Utah
- University of Utah — Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 89 participants |
| Start Date | 2021-06-14 |
| Est. Completion | 2028-03 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04768972
The ClinicalTrials.gov registry entry for NCT04768972 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 89 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ionis Pharmaceuticals, which has 55 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Amyotrophic Lateral Sclerosis appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04768972 reports 20 study locations spanning 10 distinct geographic areas — top geographies include Other, California, Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04768972 about?
NCT04768972 is a clinical study titled "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)". 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).
What is the current status of trial NCT04768972?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 89 participants. The study started on 2021-06-14. Estimated completion is 2028-03.
What conditions does trial NCT04768972 study?
This clinical trial studies the following conditions: Amyotrophic Lateral Sclerosis. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04768972?
The interventions under investigation include: Placebo (DRUG), ION363 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04768972?
This trial is sponsored by Ionis Pharmaceuticals, which has 55 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04768972 being conducted?
This trial has 20 study locations across California, Maryland, Massachusetts, Missouri, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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