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NCT04519749 · ClinicalTrials.gov registry record · Phase 1
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
A Phase 1 study of Fabry Disease, sponsored by 4D Molecular Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 18
- Enrollment target
- 4
- Study locations
NCT04519749: Active Phase 1 study of Fabry Disease, sponsored by 4D Molecular Therapeutics.
NCT04519749 is a Phase 1 study of Fabry Disease that is active but no longer recruiting, run by 4D Molecular Therapeutics. The registered enrollment target is 18 participants, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (97% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04519749, a Phase 1 study of Fabry Disease, is active but no longer recruiting, sponsored by 4D Molecular Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 18 participants
- Enrollment target
- 4
- Study locations
Study Summary
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease.
Primary Outcome
Safety and tolerability of 4D-310 following a single IV dose, as assessed by incidence and severity of adverse events, serious adverse events and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters
Conditions Studied
Interventions
- BIOLOGICAL 4D-310
Study Locations (4)
California
- University of California at San Diego - La Jolla
Georgia
- Emory University - Atlanta
Pennsylvania
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
Virginia
- Lysosomal & Rare Disorders Research & Treatment Center, Inc - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 18 participants |
| Start Date | 2020-09-01 |
| Est. Completion | 2030-06 |
| Phase | Phase 1 |
What the registry record for NCT04519749 still lists
NCT04519749 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 18 participants, a relatively small participant target, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 1 intervention - of which 4D-310 is the first listed.
NCT04519749 reports a single indexed study location in California, Georgia, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT04519749 about?
NCT04519749 is a clinical study titled "An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease". This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease.
What is the current status of trial NCT04519749?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 2020-09-01. Estimated completion is 2030-06.
What conditions does trial NCT04519749 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT04519749?
The interventions under investigation include: 4D-310 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04519749?
This trial is sponsored by 4D Molecular Therapeutics, which has 9 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04519749 being conducted?
This trial has 4 study locations across California, Georgia, Pennsylvania, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04519749's enrollment target sits among peer trials
18 25th of 32 higher than 8 of 32 other Fabry Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Fabry Disease 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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