Effective Dosing of Burosumab in XLH

Recruiting Observational Study
X-linked Hypophosphatemia (XLH)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
2 – 18
Sex
Any
Study type
Observational
Participants needed
120 (estimated)
Sponsor
University of Nottingham · Other
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About This Trial
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 pre…
Trial Locations
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Eligibility Criteria
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 minimu…
Contacts

James M Law, BMBS, PhD, MRCPCH

01159249924

james.law@nottingham.ac.uk

CONTACT