NCT06270316 Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
| NCT ID | NCT06270316 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | UniQure Biopharma B.V. |
| Condition | Fabry Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2024-06-05 |
| Primary Completion | 2027-12-01 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
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
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 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 * Suboptimal response after at least 12 months of enzyme replacement therapy (ERT) treatment. Suboptimal response is defined as plasma lyso-Gb3 ≥ 2.3 nanograms per milliliter (ng/mL) at Screening and one or both of the following: * Persistent moderate or severe neuropathic pain (intermittent or continuous) over a period of at least 3 months prior to consent * Presence of gastrointestinal symptoms (abdominal cramping, constipation