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

Recruiting Phase 1 Phase 2 Interventional Study
Fabry Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
18 – 50
Sex
Male
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
12 (estimated)
Sponsor
UniQure Biopharma B.V. · Industry
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About This Trial

The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.

Trial Locations
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Eligibility Criteria
Key Inclusion Criteria: * Male of age ≥ 18 years and ≤50 years * Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: 1. Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR 2. α-galactosidase A (GLA) pathogenic or …
Contacts

uniQure

1-866-520-1257

medinfo@uniqure.com

CONTACT

Christy Quintana

734-680-7773

medinfo@uniqure.com

CONTACT