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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: Active Phase 3 study of Alexander Disease, sponsored by Ionis Pharmaceuticals.
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, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (93% lower). The trial reports 13 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
10-Meter Walk Test (10MWT) is an assessment of gait speed.
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 |
What the registry record for NCT04849741 still lists
NCT04849741 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 54 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (93% lower).
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 lists 13 locations in 9 states (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.
Learn More About Clinical Trials
Similar trials for Alexander Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04849741's enrollment target sits among peer trials
54 1839th of 2000 higher than 162 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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