Gene therapy for gaucher disease: 5-year safety check begins
NCT ID NCT06545136
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.
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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Locations
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Hospital Quironsalud Zaragoza
Zaragoza, Spain
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Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil
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Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066, United States
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Salford Royal Hospital
Salford, United Kingdom