A Gaucher Disease Gene Therapy Trial With FLT201
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: FLT201.
- Who it may be relevant to
- Registry conditions: Gaucher Disease Type 1. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, Australia, Brazil, Canada +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Overview
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.
Interventions
- Genetic FLT201
FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.
Primary outcome measures
- To evaluate the efficacy of FLT201 [Time frame: 1 Year]
Eligibility criteria
Inclusion criteria
- Aged ≥18 years at time of screening.
- Clinical diagnosis of Gaucher disease type 1
- Stable hemoglobin concentration at baseline
- Stable platelet count at baseline
- Receiving ERT or SRT without interruption for at least 2 years
Exclusion criteria
- Diagnosed or suspected Gaucher disease type 2 or type 3
- Positive for AAVS3 neutralizing antibodies.
- Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- Positive pregnancy test or lactating
- History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant.
- History of receiving any gene therapy or cell therapy.
- History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 16 centers
- Kaiser Permanente - CA — Los Angeles
- UCLA Health — Los Angeles
- University of California, San Francisco (UCSF) — San Francisco
- University of Colorado Anschutz — Aurora
- Emory Healthcare — Atlanta
- University of Iowa Health Care — Iowa City
- University of Minnesota — Minneapolis
- Hackensack University Medical Center — Hackensack
- … and 8 more centers
United Kingdom · 4 centers
- Queen Elizabeth Hospital Birmingham — Birmingham
- Salford Royal Hospital — Salford
- Royal Free Hospital — London
- Addenbrooke's Hospital — Cambridge
Argentina · 3 centers
- Hospital Italiano de Buenos Aires — Buenos Aires
- Hospital Militar Central — Buenos Aires
- Clinica Universitaria Reina Fabiola — Córdoba
Canada · 2 centers
- MAGIC Clinic Ltd — Calgary
- Lady Davis Institute for Medical Research Jewish General Hospital — Montreal
Israel · 2 centers
- Shaare Zedek Medical Center — Jerusalem
- Rabin Medical Center, Beilinson Hospital — Petah Tikva
Spain · 2 centers
- Hospital Universitario Vall d'Hebron — Barcelona
- Hospital Quironsalud de Zaragoza — Zaragoza
Australia · 1 center
- Melbourne Health - Royal Melbourne Hospital — Melbourne N.
Brazil · 1 center
- Hospital de Clinicas de Porto Alegre — Porto Alegre
Saudi Arabia · 1 center
- King Faisal Specialist Hospital and Research Center — Riyadh
Turkey (Türkiye) · 1 center
- Gazi University Medical Faculty — Ankara
Identifiers
NCT: NCT07223944 · FLT201-03 · 2025-520765-50-00