Gene Therapy Trial for Gaucher Disease Type 1 with FLT201
This study is testing a gene therapy called FLT201 for adults with Gaucher disease Type 1. FLT201 is a special type of virus that delivers genetic material. The goal is to see how well FLT201 works and if it's safe, especially after people stop their current enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). You might be able to join if you are at least 18 years old, have Gaucher disease Type 1, and have been on stable ERT or SRT for at least two years. We will know if FLT201 is effective after one year. The study plans to enroll 45 participants, but the current recruitment status is unclear.
- Study design
- This is a Phase 3 study, meaning it's in a later stage of testing, and it's not randomized. It will involve multiple centers and plans to enroll 45 adult participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary goal is to evaluate the efficacy of FLT201 at 1 year, suggesting follow-up for at least this duration.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Gaucher Disease Gene Therapy Trial With FLT201
At a glance
Conditions
NCT07223944
Where you'd take part
This study runs at 33 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvaniastudy coordinator listed
Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohiostudy coordinator listed
Not yet recruiting
Columbia University
New York, New Yorkstudy coordinator listed
Not yet recruiting
Duke University Medical Center
Durham, North Carolinastudy coordinator listed
Recruiting
Emory Healthcare
Atlanta, Georgiastudy coordinator listed
Recruiting
Hackensack University Medical Center
Hackensack, New Jerseystudy coordinator listed
Not yet recruiting
Hospital Quironsalud de Zaragoza
Zaragoza, Spainstudy coordinator listed
Not yet recruiting
Kaiser Permanente - CA
Los Angeles, Californiastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
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Inclusion
Exclusion
What this trial measures
- To evaluate the efficacy of FLT2011 Year
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52