Viewing Study NCT06270316



Ignite Creation Date: 2024-05-06 @ 8:09 PM
Last Modification Date: 2024-10-26 @ 3:21 PM
Study NCT ID: NCT06270316
Status: RECRUITING
Last Update Posted: 2024-05-17
First Post: 2023-12-19

Brief Title: Safety PKPD and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Sponsor: UniQure Biopharma BV
Organization: UniQure Biopharma BV

Study Overview

Official Title: A Phase 12 Single Dose Dose Escalation Study of Intravenous AAV5-GLA AMT-191 in Adult Males With Classic Fabry Disease
Status: RECRUITING
Status Verified Date: 2024-08
Last Known Status: None
Delayed Posting: No
If Stopped, Why?: Not Stopped
Has Expanded Access: False
If Expanded Access, NCT#: N/A
Has Expanded Access, NCT# Status: N/A
Acronym: None
Brief Summary: 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
Detailed Description: AMT-191 is an investigational gene therapy that encodes a recombinant serotype 5 based adeno-associated viral vector rAAV5 AMT-191 is designed to deliver a liver-specific expression of the transgene coding for human a-galactosidase A gene GLA to Fabry patients via a single one-time IV infusion Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma and leukocytes levels in patients with FD Fabry disease FD

Study Oversight

Has Oversight DMC: None
Is a FDA Regulated Drug?: True
Is a FDA Regulated Device?: False
Is an Unapproved Device?: None
Is a PPSD?: None
Is a US Export?: None
Is an FDA AA801 Violation?: None