Gene therapy may free gaucher patients from lifelong infusions
NCT ID NCT07223944
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Addenbrooke's Hospital
NOT_YET_RECRUITINGCambridge, CB2 0QQ, United Kingdom
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Children's Hospital of Philadelphia
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
NOT_YET_RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Clinica Universitaria Reina Fabiola
RECRUITINGCórdoba, X5004ASL, Argentina
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Columbia University
NOT_YET_RECRUITINGNew York, New York, 10032, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Emory Healthcare
NOT_YET_RECRUITINGAtlanta, Georgia, 30322, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Gazi University Medical Faculty
NOT_YET_RECRUITINGAnkara, Yenimahalle, 06560, Turkey (Türkiye)
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Hackensack University Medical Center
NOT_YET_RECRUITINGHackensack, New Jersey, 07601, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Italiano de Buenos Aires
NOT_YET_RECRUITINGBuenos Aires, C1199ABB, Argentina
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Hospital Militar Central
NOT_YET_RECRUITINGBuenos Aires, C1426BOR, Argentina
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Hospital Quironsalud de Zaragoza
NOT_YET_RECRUITINGZaragoza, 50012, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Universitario Vall d'Hebron
NOT_YET_RECRUITINGBarcelona, Barcelona, 08035, Spain
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Hospital de Clinicas de Porto Alegre
NOT_YET_RECRUITINGPorto Alegre, 90035-903, Brazil
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Kaiser Permanente - CA
NOT_YET_RECRUITINGLos Angeles, California, 90027, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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King Faisal Specialist Hospital and Research Center
NOT_YET_RECRUITINGRiyadh, 12713, Saudi Arabia
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Lady Davis Institute for Medical Research Jewish General Hospital
NOT_YET_RECRUITINGMontreal, Quebec, T2E 7Z4, Canada
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Lysosomal Rare Disorders Research and Treatment Center
RECRUITINGFairfax, Virginia, 22030-6066, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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MAGIC Clinic Ltd
NOT_YET_RECRUITINGCalgary, Alberta, T2E 7H7, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Melbourne Health - Royal Melbourne Hospital
NOT_YET_RECRUITINGMelbourne N., Victoria, 3051, Australia
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NYU Langone Health
NOT_YET_RECRUITINGNew York, New York, 10017, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Queen Elizabeth Hospital Birmingham
NOT_YET_RECRUITINGBirmingham, Birmingham, B15 2GW, United Kingdom
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Rabin Medical Center, Beilinson Hospital
RECRUITINGPetah Tikva, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Free Hospital
RECRUITINGLondon, London, NW3 2QG, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Salford Royal Hospital
NOT_YET_RECRUITINGSalford, Greater Manchester, M6 8HD, United Kingdom
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Shaare Zedek Medical Center
RECRUITINGJerusalem, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UCLA Health
NOT_YET_RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California, San Francisco (UCSF)
RECRUITINGSan Francisco, California, 94609, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Colorado Anschutz
NOT_YET_RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa Health Care
NOT_YET_RECRUITINGIowa City, Iowa, 52242, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55454, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Texas Southwestern
RECRUITINGDallas, Texas, 75390, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••