Finding the right burosumab dose for children with XLH
Official title: A Retrospective Observational Study of the Effect of Dosing Regimen of Burosumab on Biochemical Control of Serum Phosphate Levels in Patients With X-linked Hypophosphataemia (XLH)
Run by University of Nottingham · NCT07183579 · Data updated 2026-11-12
This project tests whether children with X-linked hypophosphataemia can achieve good phosphate control on starting burosumab doses below 0.8 mg/kg.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 2 Years to 18 Years |
| Sex | All |
| Enrollment | 120 (estimated) |
| Sponsor | University of Nottingham Academic / other |
| Drugs / interventions | Burosumab |
| Locations | 1 site (Nottingham) |
| Status | Recruiting |
| Started | November 3, 2025 |
| Main results expected | July 1, 2026 |
| Study ends | July 1, 2026 |
| Registry record updated | May 11, 2026 |
| Trial ID | NCT07183579 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are paediatric patients with genetically confirmed XLH who began burosumab under paediatric criteria and have at least 12 months of continuous treatment before their 18th birthday.
✕Probably not a fit
Adults managed under adult dosing criteria, patients without genetic confirmation of XLH, very young children below paediatric initiation age, or those with severe disease requiring higher doses are unlikely to benefit from lower-dose recommendations.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a multi-centre, pragmatic retrospective observational review of paediatric metabolic bone centre records in England. The study will include children with genetically confirmed PHEX-related XLH who received at least 12 months of continuous burosumab under paediatric dosing criteria. Investigators will describe dosing variation across centres and compare biochemical control, growth and rickets-related outcomes for patients started on lower versus standard dosages. The goal is to determine how often lower starting doses maintain target phosphate levels and to inform real-world dosing practice.
Why it matters
Potential benefit: If successful, the results could support using lower burosumab starting doses for some children, reducing drug exposure, injection burden, and treatment costs while keeping phosphate under control.
How similar studies have gone: Randomised and open-label trials have shown burosumab normalises phosphate and improves rickets at recommended doses, but real-world evidence on routine success with lower starting doses is limited.
Who can join: the full rules
Show full inclusion / exclusion 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 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).
Where it's running
-
Nottingham University Hospitals NHS Trust
Nottingham, United Kingdom Recruiting
Study contacts
- Principal investigator: James Law, BMBS, PhD, MRCPCH — University of Nottingham
- Study coordinator: James M Law, BMBS, PhD, MRCPCH
- Email: james.law@nottingham.ac.uk
- Phone: 01159249924
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.