Clinical Trial: Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and Safety of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

Brief Summary: 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.

Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc

Current Primary Outcome: Proportion of subjects achieving mean serum phosphorus levels above the lower limit of normal [ Time Frame: 24 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Brief Pain Inventory (BPI) Q3 pain [ Time Frame: 24 weeks ]
  • Serum 1,25-dihydroxyvitamin D (1,25(OH)2D) [ Time Frame: 24 weeks ]
  • Proportion of subjects achieving mean serum phosphorus levels above the Lower Limit of Normal (LLN) at the end of the dosing cycle, mid-point of dosing cycle,end of dosing cycle and cumulative exposure [ Time Frame: 24 weeks ]
  • Procollagen type 1 N-propeptide (P1NP) [ Time Frame: 24 weeks ]
  • BPI Pain Severity [ Time Frame: 24 weeks ]
  • Stiffness and Physical Function domains of the Western Ontario and McMaster University Osteoarthritis Index (WOMAC®) [ Time Frame: 24 weeks ]
  • Urinary phosphorus [ Time Frame: 24 weeks ]
  • Tubular reabsorption of phosphate (TRP) [ Time Frame: 24 weeks ]
  • Ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR) [ Time Frame: 24 weeks ]
  • Carboxy-terminal cross-linked telopeptide of type I collagen (CTx) [ Time Frame: 24 weeks ]
  • Bone-specific alkaline phosphatase (BALP) [ Time Frame: 24 weeks ]
  • Brief Fatigue Inventory (BFI) Severity [ Time Frame: 24 weeks ]


Original Secondary Outcome: Same as current

Information By: Ultragenyx Pharmaceutical Inc

Dates:
Date Received: July 17, 2015
Date Started: October 2015
Date Completion: March 6, 2018
Last Updated: May 2, 2017
Last Verified: May 2017