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NCT05324943 · ClinicalTrials.gov registry record · Phase 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
A Phase 1 study, sponsored by Spur Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT05324943 is a Phase 1 study that has completed, run by Spur Therapeutics. The registered enrollment target is 10 participants.
The verdict
NCT05324943, a Phase 1 study, has completed, sponsored by Spur Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.
Interventions
- GENETIC FLT201
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2022-04-15 |
| Est. Completion | 2024-12-04 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05324943
The ClinicalTrials.gov registry entry for NCT05324943 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Spur Therapeutics, which has 6 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which FLT201 is the first listed.
NCT05324943 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT05324943 about?
NCT05324943 is a clinical study titled "A Gene Therapy Study in Patients With Gaucher Disease Type 1". This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebros...
What is the current status of trial NCT05324943?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2022-04-15. Estimated completion is 2024-12-04.
What interventions are being tested in trial NCT05324943?
The interventions under investigation include: FLT201 (GENETIC).
Who is sponsoring clinical trial NCT05324943?
This trial is sponsored by Spur Therapeutics, which has 6 total clinical trials registered on ClinicalTrials.gov.
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