Gene therapy may free gaucher patients from lifelong infusions

NCT ID NCT07223944

Recruiting now Disease control Sponsor: Spur Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 4 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.

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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

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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
NOT_YET_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: •••••@•••••

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