A Multicenter, Open-label, Phase 1/2, Dose-escalation and Subsequent Safety Extension Study of Subcutaneous KK8123 in Adult Patients With X-linked Hypophosphatemia
Kyowa Kirin Co., Ltd.
Summary
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.
Description
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 28 days (including obtaining informed consent). Part 1 is a Dose Escalation Period consisting of a nominal (planned) Treatment Period (all cohorts) and Observation Period of 32 to 44 weeks, and Part 2 is an optional Extension Period.
Eligibility
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugKK8123
Subcutaneous administration
Locations (9)
- University of California - San FranciscoSan Francisco, California
- Yale Center for XLH/ Yale University School of MedicineNew Haven, Connecticut
- Indiana University School of Medicine University HospitalIndianapolis, Indiana
- Mayo ClinicRochester, Minnesota
- Vanderbilt University Medical CenterNashville, Tennessee
- Hoptial BictreLe Kremlin-Bicêtre, Paris