Gaucher Disease, Type 1Phase 1GALILEO-1NCT05324943
A Gene Therapy Study in Patients With Gaucher Disease Type 1
A Phase 1 study testing FLT201 in people with Gaucher Disease, Type 1, run by Spur Therapeutics at 10 sites in 6 countries.
Trial summary
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase)… 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
- 10 participants
- Where
- 10 sitesin 6 countries
- Study dates
- 15 Apr 2022to 4 Dec 2024
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Adult ≥ 18 years of age.
- Diagnosis of Gaucher disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis.
- All female patients of childbearing potential must not be lactating and must have a negative serum pregnancy test at screening and confirmed negative by urine testing prior to dosing on Day 1. Female patients of childbearing potential and male patients must be willing to follow protocol guidelines for barrier protection/contraception.
- Able to give full informed consent for the trial.
- Treatment status at screening (screening period is 16 weeks):
- Treated with either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) and started this treatment at least 2 years prior to dosing with no change in regimen for the prior 3 months. ERT dose ≥15 U/kg and ≤60 U/kg every other week.
Who cannot join
- Diagnosed or suspected Type 2 or Type 3 Gaucher disease (including any patient with eye movement abnormality on clinical examination).
- Positive for neutralising antibodies to AAVS3 at screening.
- Evidence of significant and persistent liver dysfunction at Screening defined as >1.5 x upper limit of normal (ULN) in alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin.
- Evidence of any of the following at screening:
- Hb <8 g/dL.
- Platelets <45,000/mm3.
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- Pulmonary hypertension.
- New osteonecrosis within 12 months of screening.
- Fragility fracture or bone crisis within 12 months of screening.
- Hepatitis B surface antigen (HBsAg) positive at screening.
- Hepatitis C antibody (Hep C Ab) positive and hepatitis C RNA polymerase chain reaction (PCR) (as follow-up test if Hep C Ab-positive)-positive at screening.
- Cytomegalovirus (CMV) immunoglobulin G (IgG) and CMV DNA PCR-positive at screening.
- Human immunodeficiency virus (HIV)-1 or -2 antibody positive at screening.
- Patient has received live attenuated vaccination within 12 weeks prior to screening or intends to receive such vaccination during the study.
- History of clinically-advanced liver disease e.g. cirrhosis, portal hypertension.
- History of bone marrow transplant.
- History of splenectomy (partial or total).
- History of splenic infarct within 12 months of screening.
- History of receiving any gene transfer medicinal product.
- History of receiving any investigational therapy for Gaucher disease within 60 days of screening.
- Participation in any other clinical study of an investigational medicinal product (IMP), and/or receiving any other IMP during the study.
- History of idiopathic thrombocytopaenic purpura, thrombotic thrombocytopaenic purpura, thrombocytopaenia, anaemia, hepatomegaly, splenomegaly, and/or osteoporosis, unrelated to Gaucher disease.
- History of, or active neoplastic disease within 5 years of screening (except for basal or squamous cell carcinoma of the skin or carcinoma in situ which has been definitively treated).
- Subjects with uncontrolled cardiac failure, unstable angina, myocardial infarction, pulmonary hypertension or cardiac presentations including cardiac instability deemed significant by the investigator in the past 6 months
- History of acute myocarditis or presence of acute myocarditis during screening.
- History of substance abuse, including alcohol abuse or alcohol dependence.
- Known or suspected intolerance, hypersensitivity or contraindication to the investigational medicinal product (IMP) and non-investigational medicinal products (NIMPs) or their excipients.
- History of anaphylaxis or infusion related reactions to ERT.
- Contraindication(s) to MRI. (e.g. ferromagnetic metallic implants, some types of pacing and defibrillator devices, nerve stimulators).
- Any clinical condition (medical or psychiatric) that, in the opinion of the investigator, could jeopardise safety or compromise ability of the patient to participate in this study.
Trial locations and contacts
10 sites in 6 countries.
10 sites in 6 countries
Israel3 sites
Shaare Zedek Medical Center
Jerusalem
Rabin Medical Center - PPDS
Petah Tikva
Tel Aviv Sourasky Medical Center
Tel Aviv
United States2 sites
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066
Kaiser Permanente
Los Angeles, California, 90027
United Kingdom2 sites
Royal Free Hospital
London
Salford Royal Hospital
Salford
About this study
Official title: A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.
- 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 (4.5 × 10^11 vg/kg) | 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.
Number of Participants With Treatment-emergent Adverse Events Over Time
Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe.
Measured: Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Secondary outcomes (5)
Change From Baseline in Lyso-Gb1 in Plasma
Change from baseline to week 38/month 9 of lyso-Gb1 in plasma.
Measured: From baseline to week 38/9 months.
Change From Baseline in Spleen Volume Measured by MRI
Change from baseline to week 38/month 9 in spleen volume measured by MRI.
Measured: From baseline to week 38/9 months.
Change From Baseline in Liver Volume Measured by MRI
Change from baseline to week 38/month 9 in liver volume measured by MRI.
Measured: From baseline to week 38/9 months.
Change From Baseline in Hemoglobin
Change from baseline to week 38/month 9 in hemoglobin.
Measured: From baseline to week 38/9 months.
Change From Baseline in Platelet Count
Change from baseline to week 38/month 9 in platelet count.
Measured: From baseline to week 38/9 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2022
- Study start15 Apr 2022
- Primary completion4 Dec 2024
- Study completion4 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spur Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05324943
- Sponsor's protocol ID
- FLT201-01
- Acronym
- GALILEO-1
- Status last verified
- May 2026
- First submitted
- 28 Feb 2022
- Last updated
- 2 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)
- ClinicalTrials.gov ID
- NCT05324943
- Acronym / sponsor ID
- GALILEO-1 / FLT201-01
- Conditions
- Gaucher Disease, Type 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Spur TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 15 Apr 2022 (actual)
- Primary completion
- 4 Dec 2024 (actual)
- Study completion
- 4 Dec 2024 (actual)
- First posted
- 13 Apr 2022 (actual)
- Results
- Posted 2 Jun 2026
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 first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FLT201 (4.5 × 10^11 vg/kg) | 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 (6 listed)
- Adult ≥ 18 years of age.
- Diagnosis of Gaucher disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis.
- All female patients of childbearing potential must not be lactating and must have a negative serum pregnancy test at screening and confirmed negative by urine testing prior to dosing on Day 1. Female patients of childbearing potential and male patients must be willing to follow protocol guidelines for barrier protection/contraception.
- Able to give full informed consent for the trial.
- Treatment status at screening (screening period is 16 weeks):
Key exclusion criteria (30 listed)
- Diagnosed or suspected Type 2 or Type 3 Gaucher disease (including any patient with eye movement abnormality on clinical examination).
- Positive for neutralising antibodies to AAVS3 at screening.
- Evidence of significant and persistent liver dysfunction at Screening defined as >1.5 x upper limit of normal (ULN) in alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin.
- Evidence of any of the following at screening:
- Hepatitis B surface antigen (HBsAg) positive at screening.
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 (4.5 × 10^11 vg/kg) |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events Over Time
Time frame: Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe.
5 secondary outcomes
Change From Baseline in Lyso-Gb1 in Plasma
Time frame: From baseline to week 38/9 months.
Change From Baseline in Spleen Volume Measured by MRI
Time frame: From baseline to week 38/9 months.
Change From Baseline in Liver Volume Measured by MRI
Time frame: From baseline to week 38/9 months.
Change From Baseline in Hemoglobin
Time frame: From baseline to week 38/9 months.
Change From Baseline in Platelet Count
Time frame: From baseline to week 38/9 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FLT201 in people with Gaucher Disease, Type 1, run by Spur Therapeutics at 10 sites in 6 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 6 inclusion and 30 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: finished, as of the sponsor's last update on 2 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Israel, United States, United Kingdom, Brazil, Germany and 1 more country. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent Adverse Events Over Time (time frame: Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months.). 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 2 Jun 2026.