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

NCT ID NCT06545136

Ongoing Disease control Sponsor: Spur Therapeutics Source: ClinicalTrials.gov ↗

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.

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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

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