A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH) A placebo-controlled study in adult patients with x-linked hypophosphatemia (XLH) presenting with symptoms consistent with active osteomalacia, predominantly skeletal (bone or joint) pain. The design is intended to confirm the efficacy and safety of KRN23 treatment, and establish the beneficial impact on functional and patient-reported outcomes (PROs) including skeletal pain, fatigue, stiffness, motor function, and quality of life in adults with XLH. Change in serum phosphorus was selected as the primary efficacy measure as this endpoint directly assesses the hypophosphatemia that is the hallmark of XLH and the underlying mechanism for the skeletal complications experienced by patients with XLH. Chief Investigator: Professor Stuart Ralston Number and location of participating sites (by region/ country): 30 sites globally looking for 120 patients with 2-3 in Edinburgh EudraCT number: 2014-005529-11 Funder: Ultragenyx pharmaceuticals Start and End date of grant award: February 2016- July 2017 (extended) Status: Complete Unblinding: In circumstances where unblinding is necessary please refer to the protocol and follow instructions provided. Contact details are provided below. Sponsor: Ultragenyx Pharmaceutical Inc. 60 Leveroni Court Novato, CA, USA 94949 Chief Investigator: Professor Stuart Ralston, Consultant Rheumatologist, Rheumatology Department, Western General Hospital, Crewe Road South, Edinburgh EH4 2XU Research nurses Debbie Kerr/Morag Charles ECTU involvement: Clinical support This article was published on 2024-09-24
