Clinical Trial: A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia

Brief Summary: The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.

Detailed Summary:
Sponsor: Kyowa Hakko Kirin Pharma, Inc.

Current Primary Outcome: Safety and Efficacy of repeated SC injections of KRN23 [ Time Frame: On-Treatment: 6.5 months, 27 total visits ]

Safety and efficacy of repeated SC injections of KRN23 from baseline as assessed by serum phosphorus levels,immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.


Original Primary Outcome: Evaluate safety (number of AEs) and efficacy (serum Phosphorus levels), etc of repeat-doses of KRN23 [ Time Frame: On-Treatment: 4 months, 25 total visits ]

Current Secondary Outcome: Evaluation of effect of repeated SC injections of KRN23 [ Time Frame: On-Treatment: 6.5 months, 27 total visits ]

Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing.


Original Secondary Outcome:

  • Evaluate changes in phamacokinetic parameters by KRN23 dose levels [ Time Frame: On-Treatment: 4 months, 25 total visits ]
  • Evaluate changes in pharmacodynamic parameters (TmP/GFR, etc) by KRN23 dose levels [ Time Frame: On-Treatment: 4 months, 25 total visits ]
  • Evaluate changes in bone biomarkers, bone mineral density and histomorphometric measurements by KRN23 dose levels [ Time Frame: On-Treatment: 4 months, 25 total visits ]
  • Evaluate changes in immunogenicity by KRN23 dose levels [ Time Frame: On-Treatment: 4 months, 25 total visits ]
  • Evaluate changes in quality of life measures by KRN23 dose levels [ Time Frame: On-Treatment: 4 months, 25 total visits ]


Information By: Kyowa Kirin Pharmaceutical Development, Inc.

Dates:
Date Received: April 15, 2011
Date Started: April 2011
Date Completion:
Last Updated: June 10, 2016
Last Verified: January 2015