A Gaucher Disease Gene Therapy Trial With FLT201
Public ClinicalTrials.gov record NCT07223944. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.
Study identification
- NCT ID
- NCT07223944
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 45 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 6, 2026
- Primary completion
- Jun 30, 2028
- Completion
- Sep 30, 2032
- Last update posted
- Sep 2, 2026
2026 – 2032
United States locations
- U.S. sites
- 16
- U.S. states
- 12
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Kaiser Permanente - CA | Los Angeles | California | 90027 | Recruiting |
| UCLA Health | Los Angeles | California | 90095 | Recruiting |
| University of California, San Francisco (UCSF) | San Francisco | California | 94609 | Recruiting |
| University of Colorado Anschutz | Aurora | Colorado | 80045 | Recruiting |
| Emory Healthcare | Atlanta | Georgia | 30322 | Recruiting |
| University of Iowa Health Care | Iowa City | Iowa | 52242 | Not yet recruiting |
| University of Minnesota | Minneapolis | Minnesota | 55454 | Recruiting |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | Not yet recruiting |
| NYU Langone Health | New York | New York | 10017 | Recruiting |
| Columbia University | New York | New York | 10032 | Not yet recruiting |
| Duke University Medical Center | Durham | North Carolina | 27710 | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | Not yet recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | Recruiting |
| University of Texas Southwestern | Dallas | Texas | 75390 | Recruiting |
| Lysosomal Rare Disorders Research and Treatment Center | Fairfax | Virginia | 22030-6066 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 17 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07223944, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 2, 2026 · Synced Sep 7, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07223944 live on ClinicalTrials.gov.