Anti-FGF23 (Burosumab) in Adult Patients With XLH (BurGER)
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|ClinicalTrials.gov Identifier: NCT04695860|
Recruitment Status : Recruiting
First Posted : January 5, 2021
Last Update Posted : January 14, 2021
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.
|Condition or disease||Intervention/treatment||Phase|
|X-linked Hypophosphatemia||Drug: Burosumab||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||34 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Investigator-sponsored Phase 3b Open-label Study of Anti- FGF23 Antibody Burosumab (KRN23) in Adult Patients With Xlinked Hypophosphatemia (XLH) in GERmany - BurGER|
|Actual Study Start Date :||January 7, 2021|
|Estimated Primary Completion Date :||June 2022|
|Estimated Study Completion Date :||December 2022|
Experimental: Open label Burosumab
Burosumab Q4W, 1mg/kg body weight s.c.
Q4w, 1mg/kg Body weight, s.c.
- Effect of Burosumab treatment on attaining serum phosphorus levels within the normal range in adults with XLH [ Time Frame: 48 weeks ]Proportion of subjects attaining a serum phosphorus concentration within the normal range
- Change in chari rise test completion time [ Time Frame: 48 weeks ]Change in chair rise test completion time from baseline to end of study visit
- Change in 6-Minute-Walk test [ Time Frame: 48 weeks ]Change in 6-Minute-Walk test distance from baseline to end of study visit
- Change in timed up-and down stairs completion time [ Time Frame: 48 weeks ]Change in timed up-and down stairs completion time from baseline to end of study visit
- Normalization of TmP/GFR [ Time Frame: 48 weeks ]Normalization of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR), proportion of subjects achieving normal values
- Activity tracking [ Time Frame: 48 weeks ]Activity tracking using 'Actibelt', to delineate total hours of physical activity per week
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04695860
|Contact: Lothar Seefried, MD||+49 931 803 ext email@example.com|
|Clinical Trial Unit, Orthopedic Department, Wuerzburg University||Recruiting|
|Würzburg, Germany, 97074|
|Contact: Jasmin Baumann +49 931 803 ext 3590 firstname.lastname@example.org|
|Contact: Hellwich Ursula +49 931 803 ext 3578 email@example.com|
|Principal Investigator: Lothar Seefried, MD|
|Principal Investigator:||Lothar Seefried, MD||University Hospital Wuerzburg|