Clinical Trial: Study of KRN23, a Recombinant Fully Human Monoclonal Antibody Against FGF23, in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Randomized, Open-Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the Anti-FGF23 Antibody, KRN23, in Pediatric Patients With X-linked Hypophospha
Brief Summary: UX023-CL201 is a randomized, multicenter, open-label, dose finding, Phase 2 study. The study will be conducted in prepubescent children aged 5-12 years with XLH to assess the pharmacodynamics and safety of KRN23 administered via subcutaneous injections monthly (every 4 weeks) or biweekly (every 2 weeks) for a total of 64 weeks. The study consists of a 16-week individual dose Titration Period, followed by a 48-week Treatment Period. The study will enroll approximately 50 pediatric patients with XLH and radiographic evidence of bone disease. Subjects will need to discontinue oral phosphate and vitamin D metabolite therapy prior to randomization and throughout the duration of the study.
Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc
Current Primary Outcome:
- Serum Phosphorus levels [ Time Frame: 64 weeks of treatment ]
- Incidence, frequency, and severity of adverse events and serious adverse events [ Time Frame: 64 weeks of treatment ]
Original Primary Outcome: Serum Phosphorus levels [ Time Frame: 64 weeks of treatment ]
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Ultragenyx Pharmaceutical Inc
Dates:
Date Received: June 9, 2014
Date Started: June 30, 2014
Date Completion: December 2018
Last Updated: May 2, 2017
Last Verified: May 2017
