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Gene therapy for gaucher disease: 5-year safety check begins

NCT ID NCT06545136

Disease control 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 This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
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This status has not been confirmed recently, so it may be out of date.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 Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 75 people with Gaucher disease type 1 who previously received FLT201 gene therapy. Researchers will monitor them for 5 years to see if the treatment remains safe and if its effects last. The goal is to understand whether a single dose can provide long-term disease control.

What this could mean

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

Active substance
FLT201 (a gene therapy using a harmless virus to deliver a working GBA gene)
What this could lead to
If successful, this could show that a single dose of FLT201 safely controls Gaucher disease for years, reducing or eliminating the need for regular enzyme infusions.
What could go wrong
This is an early-phase follow-up study, so it is small and not designed to prove effectiveness. There may be unknown long-term side effects, and some patients may still need standard treatments.

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

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2024

Expected to finish

May 2029

An estimate. End dates often move.

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

Anyone

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: * 1\. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy \[SRT\]). * 2\. Participants able to give full informed consent and able to comply with all requirements of the trial. Exclusion Criteria: * n/a

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Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type I

As listed by the trial registrant

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

Gaucher disease type 1

Contacts and locations

Locations

  • Hospital Quironsalud Zaragoza

    Zaragoza, Spain

  • Hospital de Clinicas de Porto Alegre (HCPA)

    Porto Alegre, Brazil

  • Lysosomal Rare Disorders Research and Treatment Center

    Fairfax, Virginia, 22030-6066, United States

  • Salford Royal Hospital

    Salford, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.