Clinical Trial: Study of the Safety, Pharmacodynamics and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: An Open-Label, Phase 2 Study to Assess the Safety, Pharmacodynamics, and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
Brief Summary: UX023-CL205 is a multicenter, open-label, Phase 2 study in children from 1 to 4 years old with XLH to assess the safety, PD, and efficacy of KRN23 administered via subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 64 weeks.
Detailed Summary:
The study will enroll approximately 10 pediatric subjects between 1 and 4 years old, inclusive, with a confirmed diagnosis of XLH and radiographic evidence of rickets. Subjects will discontinue oral phosphate and active vitamin D therapy after initial Screening and for the duration of the study.
All subjects will receive treatment with KRN23. Safety, PD, pre-dose KRN23 concentrations, and efficacy measures (rickets, growth, functional outcomes) will be evaluated throughout the study. Radiographs will be taken to assess rickets periodically throughout the study duration.
Sponsor: Ultragenyx Pharmaceutical Inc
Current Primary Outcome:
- Evaluate safety by the incidence, frequency and severity of AEs and SAEs, including clinically significant changes from baseline to scheduled time points [ Time Frame: 64 weeks ]
- Determine the pharmacodynamics (PD) effects of KRN23 treatment by observing the change from Baseline over time in serum phosphorus [ Time Frame: 64 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Ultragenyx Pharmaceutical Inc
Dates:
Date Received: December 6, 2015
Date Started: May 2016
Date Completion: December 2017
Last Updated: May 2, 2017
Last Verified: May 2017
