Kyowa Kirin Co., Ltd.
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.
Study 8123-001 is a Phase 1/2, multicenter, open-label, dose-escalation study to assess the safety, tolerability, PK and PD of KK8123, with an optional safety extension period. This study is comprised of a Screening Period followed by Part 1 and Part 2. The Screening Period will last up to 35 days (including obtaining informed consent). Part 1 is a Dose Escalation Period consisting of a nominal (planned) Treatment Period (all cohorts) followed by an Observation Period, and Part 2 is an optional Extension Period.
Inclusion Criteria: * Part 1: Inclusion Criteria: 1. Male or female patients aged 18 to 65 years inclusive at the time of signing the ICF. 2. Body weight is at least 40 kg. 3. Diagnosed with XLH (as documented by the investigator). 4. Have a value of fasting serum phosphorus \< 2.5 mg/dL (0.81 mmol/L) at Screening. 5. Have a value of renal TmP/GFR \< 2.5 mg/dL (0.81 mmol/L) at Screening. 6. eGFR ≥ 60 mL/min (using the Chronic Kidney Disease Epidemiology Collaboration equation \[Inker, 2021\]) at Screening. 7. Have a corrected serum calcium level \< 10.8 mg/dL (2.7 mmol/L) at Sc…
Subcutaneous administration