a study on X-linked Hypophosphatemia
UX023-CL304 is a Phase 3 open-label, single-arm, multicenter study to establish the effects of KRN23 on bone quality and osteomalacia associated with XLH. Approximately 14 adult subjects with a diagnosis of XLH supported by typical clinical and biochemical features and who have not received oral phosphate and vitamin D therapy in the past two years will be enrolled. Iliac crest bone biopsies will be performed at baseline and end of study. Baseline histologic and histomorphometric assessments of the bone biopsy specimens will be performed as each biopsy is completed to assess sample quality and confirm the presence of osteomalacia in at least 8 subjects.
An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
X-linked Hypophosphatemia KRN23 Fibroblast growth factor 23 (FGF23) XLH
Open to people ages 18 years to 65 years
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 based on overnight fasting (min. 8 hours):
Use of investigational product or investigational medical device within 30 days prior to Screening, 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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