Gaucher Disease Type 1Phase 3GALILEO-3NCT07223944
A Gaucher Disease Gene Therapy Trial With FLT201
A Phase 3 study testing FLT201 in people with Gaucher Disease Type 1, run by Spur Therapeutics at 32 sites in 10 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease Type 1
- What is being tested?
- FLT201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 32 sitesin 10 countries
- Study dates
- 7 Apr 2026to 1 Oct 2032
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 join
- 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
Trial locations and contacts
32 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
32 sites in 10 countries
United States15 sites, 15 recruiting
- Recruiting
Emory Healthcare
Atlanta, Georgia, 30322
Dr. William Wilcox, 404-778-8618, william.wilcox@emory.edu
- Recruiting
University of Colorado Anschutz
Aurora, Colorado, 80045
Dr. Matthew Taylor, 720-446-6680, matthew.taylor@CUAnschutz.edu
- Not yet recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Dr. Robert Hopkin, 513-636-4760, leanne.hagen@cchmc.org
- Recruiting
University of Texas Southwestern
Dallas, Texas, 75390
Dr. Markey McNutt, 214-648-1222, geneticsresearch@utsouthwestern.edu
Jordan Roberts, jordan.roberts2@UTSouthwestern.edu
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
Dr. Priya Kishnani, 919-215-0097, priya.kishnani@duke.edu
- Recruiting
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066
Lauren Noll, (703) 2616220, lnoll@ldrtc.org
Ozlem Goker-Alpan, (703) 2616220, ogoker-alpan@ldrtc.org
- Recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Dr. Helio Pedro, 551-996-8178, helio.pedro@hmhn.org
- Not yet recruiting
University of Iowa Health Care
Iowa City, Iowa, 52242
Dr. John Bernat, 319-356-2675, john-bernat@uiowa.edu
- Recruiting
Kaiser Permanente - CA
Los Angeles, California, 90027
Dr. Divya Vats, 626-646-4612, divya.vats@kp.org
- Recruiting
UCLA Health
Los Angeles, California, 90095
Dr. Nicola Longo, 310-825-5610, wsia@mednet.ucla.edu
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55454
Dr. Whitley, 612-625-1594, whitley@umn.edu
Brenda Diethelm-Okita, dieth001@umn.edu
- Recruiting
NYU Langone Health
New York, New York, 10017
Dr. Nicolas Abreu, 929-455-5629, Neurogenetics@nyulangone.org
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Dr. Can Ficicioglu, 215-590-3376, FICICIOGLU@chop.edu
- Recruiting
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Dr. Damara Ortiz, 412-692-3475, Nadene.Henderson@chp.edu
- Recruiting
University of California, San Francisco (UCSF)
San Francisco, California, 94609
Dr. Irene Chang, 510-428-3885 ext. 7217, Irene.chang@ucsf.edu
United Kingdom4 sites, 4 recruiting
- Not yet recruiting
Queen Elizabeth Hospital Birmingham
Birmingham, Birmingham, B15 2GW
- Recruiting
Addenbrooke's Hospital
Cambridge, CB2 0QQ
- Recruiting
Royal Free Hospital
London, London, NW3 2QG
Dr. Derralynn Hughes, +44-2077940500, derralynnhughes@nhs.net
- Recruiting
Salford Royal Hospital
Salford, Greater Manchester, M6 8HD
Argentina3 sites, 3 recruiting
- Recruiting
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB
- Recruiting
Hospital Militar Central
Buenos Aires, C1426BOR
- Recruiting
Clinica Universitaria Reina Fabiola
Córdoba, X5004ASL
Canada2 sites, 2 recruiting
- Recruiting
MAGIC Clinic Ltd
Calgary, Alberta, T2E 7H7
Dr. Aneal Khan, 587-885-3158, research@magiccalgary.ca
- Recruiting
Lady Davis Institute for Medical Research Jewish General Hospital
Montreal, Quebec, T2E 7Z4
Israel2 sites, 2 recruiting
- Recruiting
Shaare Zedek Medical Center
Jerusalem
Dr. Revel-Vilk, +972-2-6555143, srevelvilk@gmail.com
- Recruiting
Rabin Medical Center, Beilinson Hospital
Petah Tikva
Dr. Ruhrman Shahar, +972-3-6974905, noa.ruhrman@clalit.org.il
Spain2 sites, 2 recruiting
- Not yet recruiting
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona, 08035
- Not yet recruiting
Hospital Quironsalud de Zaragoza
Zaragoza, 50012
Dr. P. Giraldo, 0034976 72 00 00, giraldocastellano@gmail.com
Australia1 site, 1 recruiting
- Not yet recruiting
Melbourne Health - Royal Melbourne Hospital
Melbourne N., Victoria, 3051
Brazil1 site, 1 recruiting
- Recruiting
Hospital de Clinicas de Porto Alegre
Porto Alegre, 90035-903
Saudi Arabia1 site, 1 recruiting
- Not yet recruiting
King Faisal Specialist Hospital and Research Center
Riyadh, 12713
Turkey (Türkiye)1 site, 1 recruiting
- Recruiting
Gazi University Medical Faculty
Ankara, Yenimahalle, 06560
About this study
Official title: 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.
- Conditions
- Gaucher Disease Type 1
- Being tested
- FLT201
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FLT201Genetic
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.
| Study group | Role | What they receive |
|---|---|---|
| FLT201 | Experimental | FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion Receives: Genetic: FLT201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the efficacy of FLT201
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2025
- Study start7 Apr 2026
- Primary completion1 Jul 2028 (estimated)
- Study completion1 Oct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Spur Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07223944
- Sponsor's protocol ID
- FLT201-03
- Other IDs
- 2025-520765-50-00, 2025
- Acronym
- GALILEO-3
- Status last verified
- Apr 2026
- First submitted
- 30 Oct 2025
- Last updated
- 14 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
- ClinicalTrials.gov ID
- NCT07223944
- Acronym / sponsor ID
- GALILEO-3 / FLT201-03
- Conditions
- Gaucher Disease Type 1
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Spur TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 7 Apr 2026 (actual)
- Primary completion
- 1 Jul 2028 (estimated)
- Study completion
- 1 Oct 2032 (estimated)
- First posted
- 3 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FLT201 | Experimental | Genetic: FLT201FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FLT201FLT201 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. | Genetic | FLT201 |
Primary outcome
To evaluate the efficacy of FLT201
Time frame: 1 Year
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
32 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing FLT201 in people with Gaucher Disease Type 1, run by Spur Therapeutics at 32 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, United Kingdom, Argentina, Canada, Israel and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Spur Clinical Trials Contact (+44 (0)1438 906870, clinicaltrials@spurtherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the efficacy of FLT201 (time frame: 1 Year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.