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Anti-FGF23 (Burosumab) in Adult Patients With XLH

NCT04695860 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2023-05-03

No results posted yet for this study

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

DRUG

Burosumab

Q4w, 1mg/kg Body weight, s.c.

Sponsors & Collaborators

  • Kyowa Kirin, Inc.

    collaborator INDUSTRY

  • Wuerzburg University Hospital

    lead OTHER

Principal Investigators

  • Lothar Seefried, MD · University Hospital Wuerzburg

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-01-07
Primary Completion
2023-03-17
Completion
2023-03-17

Countries

  • Germany

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04695860 on ClinicalTrials.gov