NCT02163577

Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)

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 Hypophosphatemia (XLH)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Kyowa Kirin, Inc. · Industry
• Sex: All
• Age: 5 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

The objectives of the study are to: * Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants * Establish the safety profile of burosumab for the treatment of children with XLH including ectopic mineralization risk, cardiovascular effects, and immunogenicity profile * Characterize the pharmacokinetic (PK)/PD profile of the KRN23 doses tested in the monthly (Q4) and biweekly (Q2) dose regimens in pediatric XLH patients * Determine the PD effects of burosumab treatment on markers of bone health in pediatric XLH patients * Obtain a preliminary assessment of the clinical effects of burosumab on bone health and deformity, muscle strength, and motor function * Obtain a preliminary assessment of the effects of burosumab on participant-reported outcomes, including pain, disability, and quality of life in pediatric XLH patients * Evaluate the long-term safety and efficacy of burosumab

Conditions

• X-linked Hypophosphatemia

Interventions

Type	Name	Description
BIOLOGICAL	burosumab	solution for SC injection

Timeline

• Start date: 2014-07-02
• Primary completion: 2018-10-30
• Completion: 2018-10-30
• First posted: 2014-06-13
• Last updated: 2024-05-06
• Results posted: 2019-05-22

Locations

9 sites across 4 countries: United States, France, Netherlands, United Kingdom