A Gaucher Disease Gene Therapy Trial With FLT201

a study on Gaucher Disease

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA UCSF
Dates
study started
study ends around
Principal Investigator
by Irene Chang (ucsf)

Description

Summary

This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.

Official Title

A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1

Keywords

Gaucher Disease Type 1, Gaucher Disease, FLT201

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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

You CAN'T join if...

  • 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

Locations

  • UCLA Health accepting new patients
    Los Angeles California 90095 United States
  • University of California, San Francisco (UCSF) accepting new patients
    San Francisco California 94609 United States
  • Kaiser Permanente - CA accepting new patients
    Los Angeles California 90027 United States

Lead Scientist at University of California Health

  • Irene Chang (ucsf)
    Associate Professor, Pediatrics, School of Medicine. Authored (or co-authored) 42 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Spur Therapeutics
ID
NCT07223944
Phase
Phase 3 Gaucher Disease Research Study
Study Type
Interventional
Participants
Expecting 45 study participants
Last Updated
Contact us about this trial