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.
Participants must be ≥ 18 years of age or age considered as adult in the respective country at the time of signing the informed consent.
Documented diagnosis of FD with clinical symptoms.
Females: historical genetic test results based on identification of pathogenic or likely pathogenic GLA variant of FD.
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).
Women who are pregnant, planning to become pregnant during the study, or are breast feeding.
History of dialysis or renal transplantation.
CKD stage ≥ 3.
History of acute kidney injury within 12 months prior to screening, including specific kidney diseases (eg, acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (eg, ischemia, toxic injury); as well as extrarenal pathology (eg, prerenal azotemia, and acute postrenal obstructive nephropathy).
GC Biopharma Corp
Lead sponsor
Hanmi Pharmaceutical Company Limited
Collaborator