Fabry DiseasePhase 1/2MARVEL1NCT04040049
A Fabry Disease Gene Therapy Study
A Phase 1/2 study testing FLT190 in people with Fabry Disease and Lysosomal Storage Diseases, run by Spur Therapeutics at 13 sites in 7 countries.
Trial summary
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Fabry Disease, Lysosomal Storage Diseases
- What is being tested?
- FLT190
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 13 sitesin 7 countries
- Study dates
- 8 Jul 2019to 2 May 2023
- Phase
- Phase 1/2
- 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
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult males, ≥ 18 years of age with classic Fabry disease.
- Confirmed diagnosis of classic Fabry Disease
- Decreased plasma alpha galactosidase (αGLA) activity at screening.
- One or more of the characteristic features of classic Fabry disease.
- Estimated glomerular filtration rate (eGFR) ≥60mL/min/1.73m2 at screening.
- <500 mg/g Urine Protein to Creatinine Ratio (UPCR) in a spot urine sample OR < 1g/24 hours of urinary protein (24hour urine analysis), at
Show all 11Show fewer
- Able to give full informed consent and able to comply with all requirements of the trial including the 5-year long term follow-up.
- Willingness to practice barrier contraception whilst vector shedding via semen is present.
- Lack of AAV neutralizing antibodies within 6 weeks prior to dosing.
- For inclusion in Part 1, subjects must have received either a licensed ERT or PCT for at least 12 months prior to dosing. For inclusion in Part 2, subjects must never have been previously dosed with Enzyme Replacement Therapy (ER) or Pharmacological Chaperone Therapy (PCT).
- Willingness to avoid strenuous exercise during first 3 months after dosing.
Who cannot join
- Non-classical Fabry disease.
- Prior hypersensitivity or intolerance to ERT
- Prior lack of response to ERT.
- Subjects with a history of chronic kidney disease for a minimum of 3 months.
- Subjects with severe myocardial fibrosis.
- Use of investigational therapy for Fabry disease within 60 days before enrolment. In addition, participation in any other clinical trial of an investigational medicinal product (IMP), and/or receiving any other IMP during the course of the study
Show all 17Show fewer
- Evidence of liver dysfunction as demonstrated by elevated blood levels during screening.
- Platelet count < 100 xE9L.
- Subjects receiving warfarin or other anticoagulants or subjects with a clinically significant bleeding disorder.
- 10 - 12. Either history of, or a positive serology test at screening for hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), hepatitis C antibody (HCAb) and human immunodeficiency virus (HIV) or a negative test at screening for anti-varicella zoster virus (VZV) IgG or hepatitis surface antibody (HBsAb).
- 13. Subjects with a history of or a positive screening test for tuberculosis. 14. Subjects who have received a live attenuated vaccination within 12 weeks prior to screening or intend to receive such a vaccine within the course of the study.
- 15. Uncontrolled glaucoma, diabetes mellitus, or hypertension. 16. History of any malignancy requiring treatment. 17. History or detection of significant arrhythmia during screening. 18. Subjects with uncontrolled cardiac failure, unstable chest pain, or heart attack deemed significant in the past 6 months.
- 19. History of acute myocarditis or presence of acute myocarditis during screening.
- 20. Prior treatment with any gene therapy medicinal product. 21. Known or suspected intolerance to gadolinium, tacrolimus and other macrolides, steroids, local anesthetics used for skin or renal biopsies, or any non-investigational medicinal products (NIMPs) or their excipients.
- 22. Subjects with contraindications to MRI. Including subjects with ferromagnetic metallic implants, including pacing and defibrillator devices, nerve stimulators and cochlear implants.
- 23. Subjects who have had a renal transplant. 24. Cytomegalovirus immunoglobulin positive subjects who are CMV polymerase chain reaction (PCR) positive at screening.
- 25-26.History of physical or psychiatric illness that could affect the subject's ability to participate or a history of substance abuse including alcohol abuse.
Trial locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
United States4 sites
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030
Kaiser Permanente
Los Angeles, California, 90027
Columbia University
New York, New York, 10032
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Germany3 sites
Charité - Universitätsmedizin Berlin
Berlin
UKEA University Hospital Hamburg
Hamburg
University of Würzburg
Würzburg
United Kingdom2 sites
Royal Free Hospital
London
Salford Royal NHS Foundation Trust
Salford
Canada1 site
Metabolics and Genetics in Calgary (MAGIC Clinic)
Calgary, Toronto, T2E 7Z4
About this study
Official title: A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.
Read the detailed description
Patients who provide consent to participate in this study will be screened for eligibility.
Eligible patients will attend the study site on the day prior to infusion (Day -1) for a baseline visit. On Day 0, FLT190 will be administered as a single dose, slow intravenous infusion. Following FLT190 treatment the patient will be discharged from the investigational site and will continue to be monitored at outpatient visits for a period of approximately 9 months; following which, the patient will enter a period of long-term follow-up conducted under a separate protocol.
The study will be conducted in 2 parts;
Part 1: Enrolment of previously treated patients (Dose escalation)
Part 2: Enrolment of previously untreated patients (Dose expansion).
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Being tested
- FLT190
- Keywords
- Gene Therapy
- Medical subject headings
- Fabry Disease, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FLT190Genetic
Gene Therapy product.
| Study group | Role | What they receive |
|---|---|---|
| FLT190 | Experimental | FLT190 is a recombinant adeno- associated viral (AAV) vector. Administered by a single intravenous infusion. Receives: Genetic: FLT190 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Treatment-emergent Adverse Events (AEs)
To investigate the safety of systemic administration of FLT190.
Measured: From screening to W38
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2019
- Study start8 Jul 2019
- Primary completion2 May 2023
- Study completion2 May 2023
Why it stopped: Freeline has decided to pause development of FLT190 in Fabry disease to focus its resources on advancing FLT201.
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 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
- NCT04040049
- Sponsor's protocol ID
- FLT190-01
- Acronym
- MARVEL1
- Status last verified
- Aug 2026
- First submitted
- 26 Jul 2019
- Last updated
- 3 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11.
PubMed 36631545
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT04040049
- Acronym / sponsor ID
- MARVEL1 / FLT190-01
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Keywords
- Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Freeline has decided to pause development of FLT190 in Fabry disease to focus its resources on advancing FLT201.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Spur TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2019 (actual)
- Primary completion
- 2 May 2023 (actual)
- Study completion
- 2 May 2023 (actual)
- First posted
- 31 Jul 2019 (actual)
- Results
- Posted 6 Aug 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.
Detailed description
Patients who provide consent to participate in this study will be screened for eligibility.
Eligible patients will attend the study site on the day prior to infusion (Day -1) for a baseline visit. On Day 0, FLT190 will be administered as a single dose, slow intravenous infusion. Following FLT190 treatment the patient will be discharged from the investigational site and will continue to be monitored at outpatient visits for a period of approximately 9 months; following which, the patient will enter a period of long-term follow-up conducted under a separate protocol.
The study will be conducted in 2 parts;
Part 1: Enrolment of previously treated patients (Dose escalation)
Part 2: Enrolment of previously untreated patients (Dose expansion).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FLT190 | Experimental | Genetic: FLT190FLT190 is a recombinant adeno- associated viral (AAV) vector. Administered by a single intravenous infusion. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Adult males, ≥ 18 years of age with classic Fabry disease.
- Confirmed diagnosis of classic Fabry Disease
- Decreased plasma alpha galactosidase (αGLA) activity at screening.
- One or more of the characteristic features of classic Fabry disease.
- Estimated glomerular filtration rate (eGFR) ≥60mL/min/1.73m2 at screening.
Key exclusion criteria (17 listed)
- Non-classical Fabry disease.
- Prior hypersensitivity or intolerance to ERT
- Prior lack of response to ERT.
- Subjects with a history of chronic kidney disease for a minimum of 3 months.
- Subjects with severe myocardial fibrosis.
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 |
|---|---|---|
| FLT190Gene Therapy product. | Genetic | FLT190 |
Primary outcome
Frequency of Treatment-emergent Adverse Events (AEs)
Time frame: From screening to W38
To investigate the safety of systemic administration of FLT190.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36631545Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11.
Related trials for Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Disease
RecruitingObservational study
Fabry Disease Registry & Pregnancy Sub-registry
Fabry Disease
Not yet recruiting
Therapeutic Implications of CMR Low Native T1 in Non-treated Non-hypertrophic Females With Fabry Disease (FD)
Fabry Disease
Active, not recruitingPhase 3
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
Fabry Disease
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.
13 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FLT190 in people with Fabry Disease and Lysosomal Storage Diseases, run by Spur Therapeutics at 13 sites in 7 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 11 inclusion and 17 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: stopped early, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Germany, United Kingdom, Austria, Canada and 2 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 6 Aug 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?
Frequency of Treatment-emergent Adverse Events (AEs) (time frame: From screening to W38). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04040049, which also lists 1 linked publication.
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 3 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.