a study on X-linked Hypophosphatemia
UX023-CL303 is a phase 3 multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of KRN23 in adult subjects with XLH. Approximately 120 subjects with a diagnosis of XLH supported by clinical and biochemical features consistent with XLH and/or a confirmed PHEX mutation (self or family member consistent with X-linked inheritance) and with bone or joint pain at baseline will be enrolled. Subjects not receiving supplementation therapy with oral phosphate and active vitamin D metabolites or those willing to discontinue supplementation therapy are eligible.
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and Safety of KRN23 in Adults With X-linked Hypophosphatemia (XLH)
X-linked Hypophosphatemia KRN23 Fibroblast growth factor 23 (FGF23) XLH
Open to people ages 18–65
Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs) and at least ONE of the following at Screening:
Biochemical findings consistent with XLH at Screening Visit 2 following overnight fasting (min. 8 hours):
Use of any investigational product or investigational medical device within 30 days prior to Screening Visit 1, or requirement for any investigational agent prior to completion of all scheduled study assessments.
OR, in Japan, use of any investigational product or investigational medical device within 4 months prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
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