ClinicalTrials.gov record NCT07223944

A Gaucher Disease Gene Therapy Trial With FLT201

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age18 years old and older
SexAll
LocationArgentina, Australia, Brazil, Canada, Israel, Saudi Arabia, Spain, Turkey, United Kingdom, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3, non-randomized, single-group study gives a single intravenous dose of FLT201 to adults with Gaucher disease type 1. Participants have been on stable 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 after discontinuing ERT/SRT, with key outcomes assessed at one year.

What it is studying. The study tests whether a single IV infusion of FLT201 can maintain clinical stability (for example, stable hemoglobin) and to evaluate safety after participants stop their prior ERT or SRT.

What the researchers hope to learn. To confirm the efficacy and safety profile of FLT201 gene therapy in adults with Gaucher disease type 1 who have been on stable ERT or SRT. The primary scientific measure is the proportion of participants with stable hemoglobin at Week 52.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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 being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Genetic
  • FLT201

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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
  • Key

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will undergo screening tests (including AAVS3 neutralizing antibody testing, blood counts, and a pregnancy test). Eligible participants receive a single intravenous infusion of FLT201 and will be monitored after discontinuing ERT/SRT as part of the study plan. They will attend regular follow-up visits with blood tests to check hemoglobin, platelets, and other labs; the main outcome is assessed at about 52 weeks. The registry record does not list further procedural details.

How the study is structured. Interventional Phase 3, non-randomized, single-group study. Each participant receives one intravenous infusion of FLT201 and is followed with clinical assessments, including blood tests, through at least one year. There is no masking (open-label).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Argentina, Australia, Brazil, Canada, Israel, Saudi Arabia, Spain, Turkey, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Gaucher disease

Clinical trials64 trials listed for Gaucher diseaseEvery trial the registry lists for Gaucher disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Gaucher disease researchLatest monthly update: September 2026. Includes: Platelet release. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 7, 2026
Primary completion
Jul 1, 2028 (estimated)
Study completion
Oct 1, 2032 (estimated)
Target enrollment
45 participants (estimated)
Last updated on registry
Sep 3, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Spur Therapeutics

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (33)

Terms used on this page · explanations by MedicalUpdates.ai

randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
hematopoietic stem cell transplant
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
Exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Key Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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