The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
29
Subjects will receive escalating doses of KRN23 administered by SC injections every 28-days (up to 4 doses) based on a dosing algorithm and discretion of the investigator and sponsor.
University of California, San Francisco Medical Center
San Francisco, California, United States
Yale University School of Medicine
New Haven, Connecticut, United States
Clinical Research Center, Indiana University School of Medicine
Indianapolis, Indiana, United States
Duke Clinical Research Unit
Durham, North Carolina, United States
Safety and Efficacy of Repeated SC Injections of KRN23
Safety and efficacy of repeated SC injections of KRN23 from baseline as assessed by serum phosphorus levels,immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.
Time frame: On-Treatment: 6.5 months, 27 total visits
Evaluation of Effect of Repeated SC Injections of KRN23
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing. Summary of serum phosphorus by visit/day is captured below.
Time frame: On-Treatment: 6.5 months, 27 total visits
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University of Texas Health Science Center at Houston
Houston, Texas, United States
Shriners Hospital for Children - Canada
Montreal, Quebec, Canada