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Effective Dosing of Burosumab in XLH

Recruitingn/aAges 2 Years - 18 YearsAllUniversity of Nottingham
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In plain words

What the trial is, and whether it could suit you

X-linked hypophosphataemia (XLH) is a rare, hereditary condition. The genetic defect leads to low blood phosphate levels and vitamin D suppression. Phosphate is required for strong bones and teeth and to store energy in cells. Low phosphate leads to soft bones (rickets). Patients experience bowed legs, short stature, bone pain and dental pain. Prior to Burosumab, conventional treatment of XLH previously consisted of two medications. On this regimen, patients take oral phosphate supplements 4-6 times a day and an active form of vitamin D daily. This treatment can leave patients with residual symptoms. They report significant disabilities and reduced quality of life. Burosumab (Crysvita, Kyowa Kirin) is now the standard paediatric treatment for XLH. It is given once a fortnight by injection under the skin. Early studies used a starting dose of 0.4mg/kg per dose. NICE recommends a startin

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๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * A diagnosis of x-linked hypophosphataemia (XLH) including genetic confirmation of a PHEX mutation. * Has received at least 12 months of continuous Burosumab treatment under paediatric criteria (given Burosumab is not started till a child is 12 months old in England, the minimum age will, therefore, be 2 years old) prior to their 18th birthday. Exclusion Criteria: * Burosumab received under adult criteria (patients who have received both Burosumab under paediatric arrangements and, subsequently, adult arrangements, can have data obtained during paediatric dosing included).
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What it tests

  • Not listed
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Where

1 site
Nottingham University Hospitals NHS Trust
Nottingham, United Kingdom
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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