Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
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 160 weeks.
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.
Study Type: Interventional (Clinical Trial)
Actual Enrollment: 13 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date: May 2016
Estimated Primary Completion Date: October 2019
Estimated Study Completion Date: October 2019
Arms and Interventions
- Experimental: KRN23
Category | Value |
---|---|
Date last updated at source | 2017-11-21 |
Study type(s) | Interventional |
Expected enrolment | 13 |
Study start date | 2016-05-01 |
Estimated primary completion date | 2019-10-01 |