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Gene therapy may free gaucher patients from lifelong infusions

NCT ID NCT07223944

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 study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 04, 2026 · Updated 6 times

Summary

This Phase 3 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if a single dose can keep blood counts stable so patients can stop their regular enzyme replacement or substrate reduction therapy. The study will enroll 45 people who have been on stable treatment for at least 2 years.

What this could mean

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

Active substance
FLT201 gene therapy
What this could lead to
If it works, this could allow people with Gaucher disease type 1 to stop their regular enzyme or substrate therapy and maintain stable blood counts with a single treatment.
What could go wrong
This is still an early Phase 3 trial with only 45 participants. Gene therapy carries risks like immune reactions or the treatment not lasting. Patients must stop their current therapy, which could cause a relapse if FLT201 fails.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 45 people

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

Started

Apr 2026

Expected to finish

Oct 2032

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.

Key Inclusion Criteria: * Aged ≥18 years at time of screening. * Clinical diagnosis of Gaucher disease type 1 * Stable hemoglobin concentration at baseline * Stable platelet count at baseline * Receiving ERT or SRT without interruption for at least 2 years Key Exclusion Criteria: * Diagnosed or suspected Gaucher disease type 2 or type 3 * Positive for AAVS3 neutralizing antibodies. * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study * Positive pregnancy test or lactating * History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant. * History of receiving any gene therapy or cell therapy. * History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    33 sites in 10 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Addenbrooke's Hospital

    NOT_YET_RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    NOT_YET_RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Clinica Universitaria Reina Fabiola

    RECRUITING

    Córdoba, X5004ASL, Argentina

  • Columbia University

    NOT_YET_RECRUITING

    New York, New York, 10032, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Emory Healthcare

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Gazi University Medical Faculty

    NOT_YET_RECRUITING

    Ankara, Yenimahalle, 06560, Turkey (Türkiye)

  • Hackensack University Medical Center

    NOT_YET_RECRUITING

    Hackensack, New Jersey, 07601, United States

  • Hospital Italiano de Buenos Aires

    RECRUITING

    Buenos Aires, C1199ABB, Argentina

  • Hospital Militar Central

    RECRUITING

    Buenos Aires, C1426BOR, Argentina

  • Hospital Quironsalud de Zaragoza

    NOT_YET_RECRUITING

    Zaragoza, 50012, Spain

  • Hospital Universitario Vall d'Hebron

    NOT_YET_RECRUITING

    Barcelona, Barcelona, 08035, Spain

  • Hospital de Clinicas de Porto Alegre

    RECRUITING

    Porto Alegre, 90035-903, Brazil

  • Kaiser Permanente - CA

    RECRUITING

    Los Angeles, California, 90027, United States

  • King Faisal Specialist Hospital and Research Center

    NOT_YET_RECRUITING

    Riyadh, 12713, Saudi Arabia

  • Lady Davis Institute for Medical Research Jewish General Hospital

    RECRUITING

    Montreal, Quebec, T2E 7Z4, Canada

  • Lysosomal Rare Disorders Research and Treatment Center

    RECRUITING

    Fairfax, Virginia, 22030-6066, United States

  • MAGIC Clinic Ltd

    RECRUITING

    Calgary, Alberta, T2E 7H7, Canada

    Contact Email: •••••@•••••

  • Melbourne Health - Royal Melbourne Hospital

    NOT_YET_RECRUITING

    Melbourne N., Victoria, 3051, Australia

  • NYU Langone Health

    RECRUITING

    New York, New York, 10017, United States

  • Queen Elizabeth Hospital Birmingham

    NOT_YET_RECRUITING

    Birmingham, Birmingham, B15 2GW, United Kingdom

  • Rabin Medical Center, Beilinson Hospital

    RECRUITING

    Petah Tikva, Israel

  • Royal Free Hospital

    RECRUITING

    London, London, NW3 2QG, United Kingdom

  • Salford Royal Hospital

    NOT_YET_RECRUITING

    Salford, Greater Manchester, M6 8HD, United Kingdom

  • Shaare Zedek Medical Center

    RECRUITING

    Jerusalem, Israel

  • UCLA Health

    RECRUITING

    Los Angeles, California, 90095, United States

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • University of California, San Francisco (UCSF)

    RECRUITING

    San Francisco, California, 94609, United States

  • University of Colorado Anschutz

    RECRUITING

    Aurora, Colorado, 80045, United States

  • University of Iowa Health Care

    NOT_YET_RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

  • University of Texas Southwestern

    RECRUITING

    Dallas, Texas, 75390, United States

    Contact Email: •••••@•••••

More trials for these conditions

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