An Open Label, Dose Range, Proof-of-Concept Study to Assess the Safety and Efficacy of HM15421/GC1134A in Patients With Fabry Disease
GC Biopharma Corp
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
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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 investi…