Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)

 X-Linked Hypophosphatemia / Posted 2 months ago

Brief Summary
This is an international, multicentre, prospective, non-interventional, observational Registry of patients with X-Linked hypophosphatemia (XLH). The main objective of this XLH Registry is to collect data to characterise the treatment, progression and long-term outcomes of XLH in both adult and paediatric settings.
Detailed Description

This is an international, multicentre, prospective, non-interventional, observational Registry of patients with X-Linked hypophosphatemia (XLH). The objectives of this XLH Registry is to collect natural history data of XLH to characterise the treatment, progression and long-term outcomes of XLH in both adult and paediatric patients.

This XLH Registry will invite & include patients with XLH, of all ages & gender, irrespective of whether they are on, or not on, a treatment regimen for their XLH.

However, patients who are concurrently participating in an interventional clinical trial, including Investigator-Initiated Studies, are excluded from the XLH Registry, as per the Exclusion Criteria as set in the Protocol. These patients participating in an interventional clinical trial, when their involvement in the trial has ended, will be approached and invited to be included in the XLH Registry.

A subset of the XLH Registry data will be used to fulfil a Post-Authorisation Safety Study (PASS) as requested by the European Medicines Agency’s (EMA’s) Committee for Medical Products for Human Use (CHMP). Study centres which agree to participate in the PASS will be asked to solicit adverse events on enrolled patients. Not all centres are expected to participate in the PASS. Furthermore, as part of burosumab’s Risk Management Plan (RMP), the majority of the safety concerns are being investigated in a Category 3 Post-Authorisation Safety Study (PASS), which uses the XLH Registry. The data source to conduct the PASS is the data collected in the XLH Registry.

All eligible patients at the participating clinics will be asked to participate in the XLH Registry:

  • Inclusion Criteria :
    • Sexes Eligible for Study: All
    • Patients aged from ≥0 years of age at baseline
    • In the opinion of the treating physician the patient has a clinical presentation, radiological, biochemical or genetic investigation results that support diagnosis of XLH
    • Patient is not currently participating in an interventional clinical trial
  • Exclusion Criteria :
    • Patient or their legally designated representative does not have the cognitive capacity to provide informed consent.
    • Patient is currently participating in an interventional clinical trial. Patients will be approached for inclusion into the registry once their involvement in the trial ends (including the completion of all trial follow up assessments).
    • Participation in a Compassionate Use Program, Pre-commercial Program (i.e. Named Patient Sales, Nominative ATU) or Investigator Initiated Study does not preclude a patient from participation in this XLH Registry
  • Study end date : July 2029
  • Wales-Based Study Contact : Rebekah Pryce
  • Principal Investigator : Rebekah Pryce
Contact details

University Hospital of WalesCardiff,CF14 4XW  Show Phone Number rebekah.pryce@wales.nhs.uk cavuhb.nhs.wales

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