Tests FLT201 gene therapy in adults with Gaucher disease type 1
Tests FLT201 gene therapy in adults with Gaucher disease type 1.
Plain-language title generated from the registry record
Registry title: A Gaucher Disease Gene Therapy Trial With FLT201
Key facts
- Where
- Barcelona and Zaragoza, SpainSee the 2 sites
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with Gaucher disease type 1 who have taken enzyme replacement or substrate reduction treatment without a break for at least 2 years.
- Age
- 18 years or older
- Sex
- All sexes can join
- Disease stage
- Clinical diagnosis of Gaucher disease type 1, with stable hemoglobin and stable platelet count at the start
- Earlier treatment
- Must have received ERT or SRT without interruption for at least 2 years. Must not have had any gene therapy or cell therapy.
- Biomarker or test result
- Must not be positive for AAVS3 neutralizing antibodies
Main reasons for not taking part
- Diagnosed or suspected Gaucher disease type 2 or type 3
- Positive for AAVS3 neutralizing antibodies
- Positive pregnancy test or breastfeeding
- Past stem cell, bone marrow or solid organ transplant
- Past total splenectomy
Read by machine on 12 November 2026.
Where in Spain
- Hospital Universitari Vall d'HebronBarcelona, SpainNot yet recruiting
- Hospital QuirónSalud ZaragozaZaragoza, SpainNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Spur Therapeutics
- Trial code
- NCT07223944EU 2025-520765-50-00
- Record updated
- 14 October 2026
- Original description
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.Read full original descriptionShow less
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
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.
Inclusion: 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: Positive for AAVS3 neutralizing antibodies; History of receiving any gene therapy or cell therapy; Diagnosed or suspected Gaucher disease type 2 or type 3; History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant; History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply; Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study; Positive pregnancy test or lactating.