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Taking peoplePhase 3

Tests FLT201 gene therapy in adults with Gaucher disease type 1

Tests FLT201 gene therapy in adults with Gaucher disease type 1.

Key facts

Who runs it
Spur Therapeutics
Where
Lisbon, PortugalSee the siteAlso in 10 other countries, 32 sites
Trial code
Registry title
A Gaucher Disease Gene Therapy Trial With FLT201
Status
Recruiting
Phase
Phase 3
People taking part
45 people, planned
Started
7 Apr 2026
Expected to end
1 Oct 2032
Type of study
Interventional
Treatments tested
  • FLT201
Record updated
14 October 2026

Who can take part

AI summary

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

The registry criteria, word for word

Show all 12 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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

Where in Portugal

Also in 10 other countries, 32 sites

  • Unidade Local De Saude De Santa Maria E.P.E.Lisbon, PortugalNot yet recruiting

About the trial

What the trial wants to find out

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.

The group in the trial

  • FLT201Treatment being tested

    FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Plain-language title
Tests FLT201 gene therapy in adults with Gaucher disease type 1Generated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
14 October 2026