A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
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.
Key Inclusion Criteria:
Key Exclusion Criteria:
clinicaltrials@spurtherapeutics.com+44 (0)1438 906870
Los Angeles, California 90027, United States
divya.vats@kp.org626-646-4612
San Francisco, California 94609, United States
Cincinnati, Ohio 45229, United States
Pittsburgh, Pennsylvania 15224, United States
Fairfax, Virginia 22030-6066, United States
Melbourne N., Victoria 3051, Australia
Montreal, Quebec T2E 7Z4, Canada
wsia@mednet.ucla.edu310-825-5610
Irene.chang@ucsf.edu510-428-3885 ext. 7217
matthew.taylor@CUAnschutz.edu720-446-6680
william.wilcox@emory.edu404-778-8618
john-bernat@uiowa.edu319-356-2675
whitley@umn.edu612-625-1594
dieth001@umn.edu
helio.pedro@hmhn.org551-996-8178
Neurogenetics@nyulangone.org929-455-5629
gm3025@cumc.columbia.edu212-342-3647
priya.kishnani@duke.edu919-215-0097
leanne.hagen@cchmc.org513-636-4760
FICICIOGLU@chop.edu215-590-3376
Nadene.Henderson@chp.edu412-692-3475
geneticsresearch@utsouthwestern.edu214-648-1222
jordan.roberts2@UTSouthwestern.edu
lnoll@ldrtc.org(703) 2616220
ogoker-alpan@ldrtc.org(703) 2616220
research@magiccalgary.ca587-885-3158
srevelvilk@gmail.com+972-2-6555143
noa.ruhrman@clalit.org.il+972-3-6974905
giraldocastellano@gmail.com0034976 72 00 00
derralynnhughes@nhs.net+44-2077940500