Burosumab in Children and Adolescents With X-linked Hypophosphatemia
- Registration Number
- NCT04419363
- Lead Sponsor
- Bicetre Hospital
- Brief Summary
In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab in real-clinical practice for \<13- and \>13-years old children affected with X-linked hypophosphatemia.
57 children with XLH were switched from conventional treatment to burosumab. After 12 months we assessed the efficacy and safety of treatment with burosumab on the whole cohort and separately on the cohort of \>13-years old adolescents.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 57
- insufficient response or refractory to conventional therapy;
- complications of conventional therapy: hypercalciuria and/or nephrocalcinosis, and/or persistent secondary hyperparathyroidism;
- need for rapid restoration of phosphate metabolism, e.g., late diagnosis (aged >8 years) and/or preparation for planned orthopaedic surgery.
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description The whole cohort Burosumab Injection Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab
- Primary Outcome Measures
Name Time Method Radiological changes in rachitic lesions evaluated with knee MRI 12 months maximum width of the physis and transverse extent of widening
- Secondary Outcome Measures
Name Time Method height 12 months standard deviation score
incidence of hearing problems 12 months ORL examination, audiogramm
incidence of neurological problems (craniosynostosis, Chiari I malformation) 12 months neurosurgical examination and brain MRI
incidence of nephrocalcinosis 12 months renal ultrasound
1,25(OH)vitaminD 12 months pg/ml
incidence of any side effects 12 months registration of any side effects during the treatment by telephone call
alkaline phosphatase 12 months U/l
functional capacity 12 months 6-minute walk test, standard deviation score
incidence of dental abcesses 12 months dental examination
incidence of hyperparathyroidism 12 months blood levels of parathyroid hormone
serum phosphate 12 months mmol/l
renal phosphate reabsorption 12 months mmol/l
parathyroid hormone 12 months ng/l
Trial Locations
- Locations (1)
Hospital Bicetre
🇫🇷Le Kremlin-Bicêtre, France