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.
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