Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCTID	NCT03454893 (View at clinicaltrials.gov)
Description	This was a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 15 male subjects, who were 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient alpha galactosidase A (AGA) enzyme activity who were considered treatment naïve, i.e., had not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of Screening. (Show More)
Development Status	Inactive
Indication	Fabry Disease
Disease Ontology Term	DOID:14499
Compound Name	AVR-RD-01
Sponsor	AVROBIO
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	15
Results Posted	View Results

Target Gene/Variant	GLA
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Target range: 3-20E6 CD34+ cells/kg
Dose 2	Dose range: 3.1 - 13.8E6 CD34+ cells/kg (reported in IIT trial)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,Brazil,Australia

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Fabry program was discontinued in Jan 2022