A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
Trial Parameters
Brief Summary
This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patients with FD.
Eligibility Criteria
Inclusion Criteria: 1. Participants must be ≥ 18 years of age or age considered as adult in the respective country at the time of signing the informed consent. 2. Documented diagnosis of FD with clinical symptoms. 3. Females: historical genetic test results based on identification of pathogenic or likely pathogenic GLA variant of FD. 4. Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/mL, LLN in leucocytes=32 nmol/hr/mg/protein). 5. Patients who are naive or have not received FD therapy including investigational therapy for FD within the past 6 months prior to screening and have negative ADA testing at screening. 6. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. 7. Plasma lyso-Gb3 levels greater than 1.5 times the upper limit of normal (ULN). 8. Male participants: * Male participants are eligible to participa