A Phase I study of LJPC-401 in healthy adult volunteers

INTERVENTION: Single dose of LJPC‐401 delivered subcutaneously. Planned ascending dose cohorts at 5mg, 18mg, 36, and 72mg. All doses will be administered on‐site by study staff. A Data Monitoring Committee (DMC) comprised of the Principal Investigator (PI), the Sponsor’s medical representatives, and others as applicable, will meet periodically to review all treatment‐emergent adverse events (TEAEs), and to make dose‐escalation decisions. CONDITION: Hemochromatosis Myelodysplasia Sickle Cell Disease Thalassemia PRIMARY OUTCOME: Changes in clinical outcomes: clinical laboratory evaluations including serum iron levels, vital signs, ECGs, physical examinations Immunogenicity testing, assessed by serum assay Treatment emergent adverse events, as reported by subjects and/or in response to an open question from study personnel or revealed by observation, physical examination or other diagnostic procedures (i.e., vital signs, clinical laboratory evaluations, ECG). SECONDARY OUTCOME: Pharmacokinetics assessed by blood sample: t1/2, Tmax, Cmax, Kel, AUClast, AUC0‐8 Serum iron parameters: total serum iron, ferritin, transferrin, TIBC, and UIBC INCLUSION CRITERIA: Normal, healthy volunteers, negative drug and alcohol tests, BMI 18‐32 inclusive