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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
45
FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.
To evaluate the efficacy of FLT201
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
Time frame: 1 Year
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Kaiser Permanente - CA
Los Angeles, California, United States
RECRUITINGUCLA Health
Los Angeles, California, United States
RECRUITINGUniversity of California, San Francisco (UCSF)
San Francisco, California, United States
RECRUITINGUniversity of Colorado Anschutz
Aurora, Colorado, United States
RECRUITINGEmory Healthcare
Atlanta, Georgia, United States
RECRUITINGUniversity of Iowa Health Care
Iowa City, Iowa, United States
NOT_YET_RECRUITINGUniversity of Minnesota
Minneapolis, Minnesota, United States
RECRUITINGHackensack University Medical Center
Hackensack, New Jersey, United States
NOT_YET_RECRUITINGNYU Langone Health
New York, New York, United States
RECRUITINGColumbia University
New York, New York, United States
NOT_YET_RECRUITING...and 23 more locations