Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
Completed · Phase 2
Conditions studied: X-linked Hypophosphatemia
In brief
The objectives of the study are to: * Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants * Establish the safety profile of burosumab for the treatment of children with XLH including ectopic mineralization risk, cardiovascular effects, and immunogenicity profile * Characterize the pharmacokinetic (PK)/PD profile of the KRN23 doses tested in the monthly (Q4) and biweekly (Q2) dose regimens in pediatric XLH patients * Determine the PD effects of burosumab treatment on markers of bone health in pediatric XLH patients * Obtain a preliminary assessment of the clinical effects of burosumab on bone health and deformity, muscle strength, and motor function * Obtain a preliminary assessment of the effects of burosumab on participant-reported outcomes, including pain, disability, and quality of life in pediatric XLH patients * Evaluate the long-term safety and efficacy of burosumab
Key facts
- Study ID
- NCT02163577
- Run by
- Kyowa Kirin, Inc.
- People needed
- 52
- Starts
- 2014-07-02
- Expected to finish
- 2018-10-30
- Last updated by the study team
- 2024-05-06
Who can join
Age: 5 and older, up to 12. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California San Francisco — San Francisco, California, United States
- Yale University School of Medicine — New Haven, Connecticut, United States
- Indiana University Hospital — Indianapolis, Indiana, United States
- Shriners Hospital for Children — St Louis, Missouri, United States
- Bicetre Hospital — Le Kremlin-Bicêtre, France
- University Medical Center Groningen — Groningen, Netherlands
- Royal Manchester Hospital — Manchester, Lancashire, United Kingdom
- Birmingham Children's University — Birmingham, United Kingdom
- Great Ormond Street Hospital — London, United Kingdom
Full record on ClinicalTrials.gov
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