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NCT04849741 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
A Phase 3 study of Alexander Disease, sponsored by Ionis Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 54
- Enrollment target
- 13
- Study locations
NCT04849741 is a Phase 3 study of Alexander Disease that is active but no longer recruiting, run by Ionis Pharmaceuticals. The registered enrollment target is 54 participants. The trial reports 13 study locations across 9 states.
The verdict
NCT04849741, a Phase 3 study of Alexander Disease, is active but no longer recruiting, sponsored by Ionis Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 54 participants
- Enrollment target
- 13
- Study locations
Study Summary
The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG zilganersen
Study Locations (13)
Other
- Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center - Tel Aviv
- Ospedale dei Bambini Vittore Buzzi - Milan
- Ospedale Pediatrico Bambino Gesù - Roma
- University College London Hospitals NHS Foundation Trust - London
- Great Ormond Street Hospital for Children NHS Foundation Trust - London
California
- Lucile Packard Children's Hospital Stanford - Palo Alto
Georgia
- Children's Hospital of Atlanta - Atlanta
Massachusetts
- Massachusetts General Hospital - Boston
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Victoria
- Murdoch Children's Research Institute - Parkville
Quebec
- McGill University Health Centre - Montreal
Kodaira-shi
- National Center of Neurology and Psychiatry - Tokyo
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2021-06-01 |
| Est. Completion | 2029-09 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04849741
The ClinicalTrials.gov registry entry for NCT04849741 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ionis Pharmaceuticals, which has 76 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Alexander Disease appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04849741 reports 13 study locations spanning 9 distinct geographic areas - top geographies include Other, California, Georgia.
Frequently Asked Questions
What is clinical trial NCT04849741 about?
NCT04849741 is a clinical study titled "A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)". The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
What is the current status of trial NCT04849741?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 54 participants. The study started on 2021-06-01. Estimated completion is 2029-09.
What conditions does trial NCT04849741 study?
This clinical trial studies the following conditions: Alexander Disease.
What interventions are being tested in trial NCT04849741?
The interventions under investigation include: Placebo (DRUG), zilganersen (DRUG).
Who is sponsoring clinical trial NCT04849741?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04849741 being conducted?
This trial has 13 study locations across California, Georgia, Massachusetts, Pennsylvania, Victoria. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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