NCT04695860

Anti-FGF23 (Burosumab) in Adult Patients With XLH

An Investigator-sponsored Phase 3b Open-label Study of Anti- FGF23 Antibody Burosumab (KRN23) in Adult Patients With Xlinked Hypophosphatemia (XLH) in GERmany - BurGER

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Wuerzburg University Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

X-linked hypophosphatemia (XLH) rare genetic disorder due by inactivating mutations in the PHEX gene leading to increased levels in FGF-23. Elevated FGF-23 reduces renal phosphate reabsorbtion and and limits 1-alpha hydroxylase driven Vitamin D activation, eventually leading to phosphate wasting, defective bone mineralization and additional health issues. Burosumab is a recombinant fully human IgG1 monoclonal antibody developed to treat XLH by binding FGF23, thereby restoring normal phosphate homeostasis. BUR03 is a Phase 3b open-label, single-arm, single-center study to confirm the efficacy and safety of Burosumab treatment in adult (age ≥18 years) XLH patients without upper age limit and irrespective of baseline pain level and to further evaluate the efficacy in this cohort and the assocaited effect of treatment on physical functioning, mobility and activity. The study aims at enrolling and treating 34 subjects with a confirmed diagnosis of XLH with q4w s.c. injection of Burosumab 1mg/kg body weight over 48 weeks. Primary objective is to attiain normal serum phosphorus levels, secondary objectives include key parameters of physical function and activity, mobility and mineral homeostasis.

Conditions

• X-linked Hypophosphatemia

Interventions

Type	Name	Description
DRUG	Burosumab	Q4w, 1mg/kg Body weight, s.c.

Timeline

• Start date: 2021-01-07
• Primary completion: 2023-03-17
• Completion: 2023-03-17
• First posted: 2021-01-05
• Last updated: 2023-05-03

Locations

1 site across 1 country: Germany