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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: Completed Phase 1 study, sponsored by Spur Therapeutics.
NCT05324943 is a Phase 1 study that has completed, run by Spur Therapeutics. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe.
Interventions
- GENETIC FLT201
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2022-04-15 |
| Est. Completion | 2024-12-04 |
| Phase | Phase 1 |
What the finished NCT05324943 record still lists
NCT05324943 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 1 intervention - of which FLT201 is the first listed.
NCT05324943 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05324943's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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