Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
ConditionFabry Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexMale
Age18-50
SponsorUniQure Biopharma B.V.
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.
Eligibility criteria
Qualifiers
Male of age ≥ 18 years and ≤50 years
Absent or minimal αGAL A enzyme activity < 1% of mean normal measured in plasma regardless of variant status; OR
α-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with plasma αGLA A enzyme activity below lower bound of the reference range (as measured at trough enzyme replacement therapy [ERT] levels).
eGFR ≥ 40 mL/min/1.73 m2
Disqualifiers
Any allergic hypersensitivity reaction to ERT or infusion reaction in the 12 months prior to consent that was of severity grade 3 or above based on Common Terminology Criteria for Adverse Events (CTCAE v5.0) and required emergency intervention for hypertension/hypotension to stabilize blood pressure or hypoxia OR any other life-threatening complication.
Proteinuria, with random urine protein/creatinine ratio (rUPCR) ≥1 mg/mg at Screening
Current use of chaperone therapy such as migalastat (Galafold®)
Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin
Trial design
Treatments tested in this trial
- AMT-191
Treatment groups
12 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov