Fabry DiseasePhase 1/2NCT04455230
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
A Phase 1/2 study testing FLT190 in people with Fabry Disease and Lysosomal Storage Diseases, run by Spur Therapeutics at 2 sites in 2 countries.
Trial summary
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously expressed throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the… 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
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 8 Sep 2020to 28 Sep 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
- Subjects who have previously received FLT190
- Provision of full informed consent and able to comply with all requirements of the study including long-term follow-up for 60 months (5 years) post-treatment.
Who cannot join
- N/A
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously expressed throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the accumulation of specific glycosphingolipids that are associated with the pathophysiology of the disease. Current treatment for Fabry disease is limited to the symptomatic management of pain, conventional management of complications, and methods to increase the availability of functional αGLA.
This clinical study aims to investigate the long-term safety and durability of αGLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.
- 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
FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).
| Study group | Role | What they receive |
|---|---|---|
| Participants who have received gene therapy vector (FLT190) | Experimental | Not described Receives: Genetic: FLT190 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Measured: Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2020
- Study start8 Sep 2020
- Primary completion28 Sep 2023
- Study completion28 Sep 2023
Why it stopped: The study was suspended early due to a corporate financial decision. A final SAP was written to specify the impact on final outcome measurements.
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 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease
- 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
- NCT04455230
- Sponsor's protocol ID
- FLT190-02
- Status last verified
- Sep 2026
- First submitted
- 29 Jun 2020
- Last updated
- 3 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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 Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease
- ClinicalTrials.gov ID
- NCT04455230
- Acronym / sponsor ID
- FLT190-02
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Keywords
- Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was suspended early due to a corporate financial decision. A final SAP was written to specify the impact on final outcome measurements.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Spur TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 8 Sep 2020 (actual)
- Primary completion
- 28 Sep 2023 (actual)
- Study completion
- 28 Sep 2023 (actual)
- First posted
- 2 Jul 2020 (actual)
- Results
- Posted 3 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously expressed throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the accumulation of specific glycosphingolipids that are associated with the pathophysiology of the disease. Current treatment for Fabry disease is limited to the symptomatic management of pain, conventional management of complications, and methods to increase the availability of functional αGLA.
This clinical study aims to investigate the long-term safety and durability of αGLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease
| Arm | Type | Receives |
|---|---|---|
| Participants who have received gene therapy vector (FLT190) | Experimental | Genetic: FLT190 |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Subjects who have previously received FLT190
- Provision of full informed consent and able to comply with all requirements of the study including long-term follow-up for 60 months (5 years) post-treatment.
Key exclusion criteria (1 listed)
- N/A
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 |
|---|---|---|
| FLT190FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA). | Genetic | Participants who have received gene therapy vector (FLT190) |
Primary outcome
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Time frame: Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
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.
- Individual participant dataSharing planned: No
Related trials for Fabry Disease
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A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
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Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
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A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
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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.
2 sites in 2 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 2 sites in 2 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 2 inclusion and 1 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 2 sites in Germany, United Kingdom. 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 3 Sep 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?
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. (time frame: Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)). 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 3 Sep 2026.