Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Bicetre Hospital

Status and phase: Unknown; Phase 4

Conditions: X-linked Hypophosphatemia; Rare Diseases

Treatments: Drug: Burosumab Injection

Study type: Interventional

Funder types: Other

Identifiers

NCT04419363

20200528104746

Details and patient eligibility

About

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.

Enrollment: 57 estimated patients

Sex: All

Ages: 1 to 20 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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57 participants in 1 patient group

The whole cohort

Experimental group

Description:

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

Treatment:

Drug: Burosumab Injection