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Can a One-Time gene therapy fix fabry disease for years?

NCT ID NCT04455230

Cure Sponsor: Spur Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 03, 2026 · Last updated Sep 04, 2026 · Updated 1 time

Summary

This study follows people with Fabry disease who have already received FLT190, an experimental gene therapy that delivers a working copy of the GLA gene. The goal is to see how safe the treatment is over the long term and whether its effects last. Researchers will track participants for up to 5 years after dosing, watching for side effects and checking if the body continues to produce the enzyme that Fabry disease lacks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
FLT190, a gene therapy that delivers a working copy of the GLA gene to help the body produce the missing enzyme
What this could lead to
If FLT190 proves safe and durable, it could offer a one-time treatment that reduces or eliminates the need for lifelong enzyme replacement in Fabry disease.
What could go wrong
This is an early-stage study with only 3 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions or the effect fading over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

3 people

The number who actually took part.

Started

Sep 2020

Finished

Sep 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. Exclusion Criteria: * N/A

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Conditions

The condition(s) this trial relates to.

Fabry disease lysosomal storage disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease Lysosomal storage diseases

Contacts and locations

Locations

  • Charité - Universitätsmedizin Berlin

    Berlin, Germany

  • Royal Free London

    London, United Kingdom

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