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NCT04768972 · ClinicalTrials.gov registry record · 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)
A Phase 3 study of Amyotrophic Lateral Sclerosis, sponsored by Ionis Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 89
- Enrollment target
- 20
- Study locations
NCT04768972: Active Phase 3 study of Amyotrophic Lateral Sclerosis, sponsored by Ionis Pharmaceuticals.
NCT04768972 is a Phase 3 study of Amyotrophic Lateral Sclerosis that is active but no longer recruiting, run by Ionis Pharmaceuticals. The registered enrollment target is 89 participants, below the 897-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (90% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04768972, a Phase 3 study of Amyotrophic Lateral Sclerosis, is active but no longer recruiting, sponsored by Ionis Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 89 participants
- Enrollment target
- 20
- Study locations
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).
Primary Outcome
Functional impairment to be measured by joint rank analysis of the combined assessment of: In-clinic Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score, time of rescue or discontinuation from Part 1 and entering Part 2 due to a deterioration in function, and ventilation assistance-free survival (VAFS). ALSFRS-R measures functional disease severity. The scale measures four functional domains, bulbar function, gross motor skills, fine motor skills, and respiratory
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 |
What the registry record for NCT04768972 still lists
NCT04768972 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 89 participants, a relatively small participant target, below the 897-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (90% lower).
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.
NCT04768972 names 20 study sites across 10 states, led by Other, California, Maryland.
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.
What interventions are being tested in trial NCT04768972?
The interventions under investigation include: Placebo (DRUG), ION363 (DRUG).
Who is sponsoring clinical trial NCT04768972?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Where NCT04768972's enrollment target sits among peer trials
89 44th of 102 higher than 59 of 102 other Amyotrophic Lateral Sclerosis trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Amyotrophic Lateral Sclerosis trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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