Clinical Trial: Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional
Official Title: A Phase 2b, Open-Label, Long-Term Extension Study to Evaluate the Safety and Pharmacodynamics of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
Brief Summary: UX023-CL203 is a Phase 2b, open-label, long-term extension study. The study will be conducted in adults to assess the safety and pharmacodynamics of KRN23 administered via subcutaneous injections monthly (every 4 weeks) for a total of 68 weeks. The study will enroll approximately 25 adults with XLH who participated in Kyowa Hakko Kirin Pharma, Inc.'s (KHK's) study KRN23-INT-001 or KRN23-INT-002. Subjects will need to discontinue oral phosphate and vitamin D metabolite therapy prior to enrollment and throughout the duration of the study.
Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc
Current Primary Outcome: Incidence, frequency, and severity of adverse events and serious adverse events [ Time Frame: 72 Weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change from Baseline in serum FGF23 [ Time Frame: 72 Weeks ]
- Change from Baseline in serum ALP [ Time Frame: 72 Weeks ]
- Change from Baseline in serum 1,25(OH)2D [ Time Frame: 72 Weeks ]
- Change from Baseline in serum iPTH [ Time Frame: 72 Weeks ]
- Change from Baseline in serum and urine phosphorus [ Time Frame: 72 Weeks ]
- Change from Baseline in serum TRP and TMP/GFR [ Time Frame: 72 Weeks ]
- Change from Baseline in serum BALP [ Time Frame: 72 Weeks ]
- Change from Baseline in serum CTx [ Time Frame: 72 Weeks ]
- Change from Baseline in serum P1NP [ Time Frame: 72 Weeks ]
Original Secondary Outcome: Same as current
Information By: Ultragenyx Pharmaceutical Inc
Dates:
Date Received: December 3, 2014
Date Started: December 2014
Date Completion: August 16, 2018
Last Updated: May 2, 2017
Last Verified: May 2017
