Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCTID	NCT06270316 (View at clinicaltrials.gov)
Description	This is an open-label, multi-center study to evaluate safety, tolerability, and exploratory efficacy of a single dose of intravenously-administered AMT-191. The plan is to investigate 2 sequential dose cohorts with 3-6 Participants per cohort. Participants will continue receiving regularly scheduled enzyme replacement therapy (ERT) until they meet the criteria for withdrawal. (Show More)
Indication	Fabry Disease
Compound Name	AMT-191
Sponsor	UniQure Biopharma B.V.
Funder Type	Industry
Status	Recruiting
Enrollment Count	12

Target Gene/Variant	GLA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type	none
Dose 1	6.0E13 gc/kg
Dose 2	3.0E14 gc/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	First patient dosed 8/15/24; IDMC recommended proceeding with enrollment of 2nd cohort