A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
NCT Number: NCT06525636
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1|PHASE2
Conditions: X-linked Hypophosphatemia
Interventions: DRUG: KK8123
Sponsor: Kyowa Kirin, Inc.
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 24
Study Design: Allocation: NON_RANDOMIZED|Intervention Model: SEQUENTIAL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-07-29
Completion Date: 2028-05-10
Locations: University of California - San Francisco, San Francisco, California, 94115, United States|Yale School of Medicine, New Haven, Connecticut, 06510, United States|Indiana University School of Medicine University Hospital, Indianapolis, Indiana, 46202, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232, United States|CHU le Kremlin Bicetre, Paris, 94275, France|University of Wuerzburg, Würzburg, Sanderring, 97070, Germany|Institute of Osteology and Biomechanics (IOBM), Hamburg, 22529, Germany|Hospital Universitario La Paz, Madrid, Fuencarral-El Pardo, 28046, Spain

Summary:
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.

Primary Outcome Measures: Part 1: Number of participants with TEAEs, For up to 44 weeks.|Part 1: Percentage of participants with TEAEs, For up to 44 weeks.|Part 2: Number of participants with TEAEs, For up to 52 weeks.|Part 2: Percentage of participants with TEAEs, For up to 52 weeks.|Part 1: Change from baseline for haematology laboratories values, For up to 44 weeks.|Part 2: Change from baseline for haematology laboratories values, For up to 52 weeks.|Part 1: Change from baseline for clinical chemistry laboratories values, For up to 44 weeks.|Part 2: Change from baseline for clinical chemistry laboratories values, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for FSH, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for FSH, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for estradiol, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for estradiol, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for temperature, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for temperature, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for pulse rate, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for pulse rate, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for respiratory rate, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for respiratory rate, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for systolic and diastolic blood pressure, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for systolic and diastolic blood pressure, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for QT, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for QT, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for QTc, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for QTc, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for QTCF, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for QTCF, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for QRS, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for QRS, For up to 52 weeks.|Part 1: Change from baseline in continuous variables for heart rate, For up to 44 weeks.|Part 2: Change from baseline in continuous variables for heart rate, For up to 52 weeks.|Part 1: The presence or absence of abnormality on ectopic mineralization, any sign of left ventricular hypertrophy or heart failures before and after administration of KK8123 on Echocardiogram, For up to 44 weeks.|Part 2: The presence or absence of abnormality on ectopic mineralization, any sign of left ventricular hypertrophy or heart failures before and after administration of KK8123 on Echocardiogram, For up to 52 weeks.|Part 1: Before and after administration presented at each time point in categorical variables for renal ultrasound, For up to 44 weeks.|Part 2: Before and after administration presented at each time point in categorical variables for renal ultrasound, For up to 52 weeks.|Part 1: KK8123 concentrations by maximum plasma concentration (Cmax), For up to 44 weeks.|Part 2: KK8123 concentrations by maximum plasma concentration (Cmax), For up to 52 weeks.|Part 1: KK8123 concentrations by maximum serum concentration (tmax), For up to 44 weeks.|Part 2: KK8123 concentrations by maximum serum concentration (tmax), For up to 52 weeks.|Part 1: KK8123 concentrations by area under the serum concentration time curve from zero to last detectable time point (AUClast), For up to 44 weeks.|Part 2: KK8123 concentrations by area under the serum concentration time curve from zero to last detectable time point (AUClast), For up to 52 weeks.|Part 1: KK8123 concentrations by the area under the serum concentration time curve from zero to infinity (AUC00-inf), For up to 44 weeks.|Part 2: KK8123 concentrations by the area under the serum concentration time curve from zero to infinity (AUC00-inf), For up to 52 weeks.|Part 1: KK8123 concentrations by apparent volume of distribution (V/F), For up to 44 weeks..|Part 2: KK8123 concentrations by apparent volume of distribution (V/F), For up to 52 weeks.|Part 1: KK8123 concentrations by terminal half-life (t1/2), For up to 44 weeks.|Part 2: KK8123 concentrations by terminal half-life (t1/2), For up to 52 weeks.|Part 1: KK8123 concentrations by apparent clearance (CL/F), For up to 44 weeks.|Part 2: KK8123 concentrations by apparent clearance (CL/F), For up to 52 weeks.|Part 1: KK8123 concentrations by maximum plasma concentration steady state (Cmax,ss), For up to 44 weeks.|Part 2: KK8123 concentrations by maximum plasma concentration steady state (Cmax,ss), For up to 52 weeks.|Part 1: KK8123 concentrations by time to maximum serum concentration steady state (tmax,ss), For up to 44 weeks.|Part 2: KK8123 concentrations by time to maximum serum concentration steady state (tmax,ss), For up to 52 weeks.|Part 1: KK8123 concentrations over time by area under the serum concentration curve within a dosing interval at steady state (AUCtau,ss), For up to 44 weeks.|Part 2: KK8123 concentrations over time by area under the serum concentration curve within a dosing interval at steady state (AUCtau,ss), For up to 52 weeks.|Part 1: KK8123 concentrations by time to steady state, For up to 44 weeks.|Part 2: KK8123 concentrations by time to steady state, For up to 52 weeks.|Part 1: KK8123 concentrations by accumulation ratio, For up to 44 weeks.|Part 2: KK8123 concentrations by accumulation ratio, For up to 52 weeks.|Part 1: To evaluate the effect of single and multiple SC administrations of KK8123 on serum phosphorus levels, For up to 44 weeks.|Part 2: To evaluate the effect of multiple SC administrations of KK8123 on serum phosphorus levels, For up to 52 weeks.
Secondary Outcome Measures: Part 1: ADA positivity titers to be assessed by absolute number, For up to 44 weeks.|Part 1: ADA positivity titers to be assessed by percentage, For up to 44 weeks.|Part 2: ADA positivity titers to be assessed by absolute number, For up to 52 weeks.|Part 2: ADA positivity titers to be assessed by percentage, For up to 52 weeks.