Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Sponsor

Bicetre Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT04419363

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

1.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Rare Diseases X-linked Hypophosphatemia	Drug: Burosumab Injection	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

57 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

12-months of Treatment With Burosumab in Children and Adolescents With X-linked Hypophosphatemia: a Prospective Longitudinal Cohort Study

Actual Study Start Date :

Mar 18, 2018

Actual Primary Completion Date :

Mar 20, 2019

Anticipated Study Completion Date :

Sep 16, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: The whole cohort Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab	Drug: Burosumab Injection Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab

Arm

Intervention/Treatment

Experimental: The whole cohort

Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab

Drug: Burosumab Injection

Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab

Outcome Measures

Primary Outcome Measures

Radiological changes in rachitic lesions evaluated with knee MRI [12 months]
maximum width of the physis and transverse extent of widening

Secondary Outcome Measures

serum phosphate [12 months]
mmol/l
renal phosphate reabsorption [12 months]
mmol/l
alkaline phosphatase [12 months]
U/l
1,25(OH)vitaminD [12 months]
pg/ml
parathyroid hormone [12 months]
ng/l
height [12 months]
standard deviation score
functional capacity [12 months]
6-minute walk test, standard deviation score
incidence of dental abcesses [12 months]
dental examination
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
incidence of hyperparathyroidism [12 months]
blood levels of parathyroid hormone
incidence of any side effects [12 months]
registration of any side effects during the treatment by telephone call

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 20 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.