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July 1, 2014
09:12 EDTRAREUltragenyx initiates Phase 2 study of KRN23
Ultragenyx Pharmaceutical announced the first patient screened and enrolled in the Phase 2 study of the human monoclonal anti-FGF23 antibody KRN23 in pediatric patients with X-linked hypophosphatemia, or XLH. XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness. The multi-center, randomized, open-label, dose-finding Phase 2 clinical study will evaluate safety and efficacy in approximately 30 prepubertal pediatric XLH patients. The primary objectives of the study are to identify a dose and dosing regimen and to establish the safety profile of treatment with KRN23 in pediatric XLH patients. Preliminary clinical effects of KRN23 treatment on bone health and deformity as measured by radiographic assessments, growth, muscle strength, and motor function will also be assessed, as well as markers of bone health and patient-reported outcomes of pain, disability, and quality of life. The study has been evaluated and accepted for conduct by the FDA, the United Kingdom Medicinal and Health Regulatory Authority, or MHRA, and the Dutch Medicines Evaluation Board, or CBG-MEB.
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