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).
Sequential
HM15421/GC1134A
DrugSC
Incidences and characteristics of adverse events
Maximum serum concentration (Cmax)
PK parameter
Time to reach maximum serum concentration (Tmax)
PK parameter
Trough serum concentration (Ctrough)
PK parameter
Area under the concentration-time curve in one dosing interval (AUC0-tau)
PK parameter
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GC Biopharma Corp
Lead sponsor
Hanmi Pharmaceutical Company Limited
Collaborator
