Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Rare Diseases Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04419363
• Other study ID #: 20200528104746
• Status: Recruiting
• Phase: Phase 4
• 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
• 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.

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• Rare Diseases
• X-linked Hypophosphatemia

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