Trial Profile

A Phase 1/2, Single Dose, Dose Escalation Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 27 Oct 2025

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At a glance

Drugs AMT-191 (Primary)
Indications Fabry's disease
Focus Adverse reactions; First in man; Therapeutic Use
Sponsors uniQure

Most Recent Events

23 Oct 2025 Planned End Date changed from 1 Dec 2027 to 30 Apr 2031.
05 Sep 2025 According to a uniQure media release, safety and exploratory efficacy data from the 1st cohort of this study were presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan.
05 Sep 2025 According to a uniQure media release, Enrollment was completed in a second, lower dose cohort, Cohort B (2x1013 gc/kg), consisting of three patients.

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