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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04419363
Other study ID # 20200528104746
Secondary ID
Status Recruiting
Phase Phase 4
First received
Last updated
Start date March 18, 2018
Est. completion date September 16, 2022

Study information

Verified date June 2020
Source Bicetre Hospital
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 57
Est. completion date September 16, 2022
Est. primary completion date March 20, 2019
Accepts healthy volunteers No
Gender All
Age group 1 Year to 20 Years
Eligibility Inclusion Criteria:

- 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Burosumab Injection
Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab

Locations

Country Name City State
France Hospital Bicetre Le Kremlin-Bicêtre

Sponsors (1)

Lead Sponsor Collaborator
Bicetre Hospital

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Primary Radiological changes in rachitic lesions evaluated with knee MRI maximum width of the physis and transverse extent of widening 12 months
Secondary serum phosphate mmol/l 12 months
Secondary renal phosphate reabsorption mmol/l 12 months
Secondary alkaline phosphatase U/l 12 months
Secondary 1,25(OH)vitaminD pg/ml 12 months
Secondary parathyroid hormone ng/l 12 months
Secondary height standard deviation score 12 months
Secondary functional capacity 6-minute walk test, standard deviation score 12 months
Secondary incidence of dental abcesses dental examination 12 months
Secondary incidence of hearing problems ORL examination, audiogramm 12 months
Secondary incidence of neurological problems (craniosynostosis, Chiari I malformation) neurosurgical examination and brain MRI 12 months
Secondary incidence of nephrocalcinosis renal ultrasound 12 months
Secondary incidence of hyperparathyroidism blood levels of parathyroid hormone 12 months
Secondary incidence of any side effects registration of any side effects during the treatment by telephone call 12 months
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