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)
Development Status	Active
Indication	Fabry Disease
Disease Ontology Term	DOID:14499
Compound Name	AMT-191
Sponsor	UniQure Biopharma B.V.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	12
Results Posted	Not Available

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	8
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	First patient dosed 8/15/24; IDMC recommended proceeding with enrollment of 2nd cohort