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Registry for patients with X-Linked Hypophosphatemia

An International, Multicentre, Prospective, Non-interventional Observational Registry for Patients With X-Linked Hypophosphatemia (XLH)

Observational Kyowa Kirin Co., Ltd. · NCT03193476

This study is collecting information from people of all ages with X-Linked hypophosphatemia to better understand their treatment and health over time.

Quick facts

Study typeObservational
Enrollment1343 (estimated)
SexAll
SponsorKyowa Kirin Co., Ltd. Industry-sponsored
Drugs / interventionsburosumab
Locations118 sites (Brussels and 117 other locations)
Trial IDNCT03193476 on ClinicalTrials.gov

What this trial studies

This international, multicentre, prospective registry collects data on patients with X-Linked hypophosphatemia (XLH) to characterize their treatment, disease progression, and long-term outcomes. It includes patients of all ages and genders, regardless of their treatment status. The registry aims to gather natural history data and may also contribute to a Post-Authorisation Safety Study (PASS) to monitor safety concerns related to burosumab. Patients currently involved in interventional clinical trials are excluded until their participation concludes.

Who should consider this trial

Good fit: Ideal candidates for this registry are individuals of any age diagnosed with X-Linked hypophosphatemia.

Not a fit: Patients currently participating in interventional clinical trials will not benefit from this registry until their trial involvement ends.

Why it matters

Potential benefit: If successful, this registry could enhance understanding of XLH and improve treatment strategies for patients.

How similar studies have performed: While this registry approach is observational and may not have direct precedents, similar registries have successfully contributed to understanding rare diseases.

Eligibility criteria

Show full inclusion / exclusion criteria 
A patient must meet the following criteria at the enrolment visit (baseline) to be eligible for inclusion into this XLH Registry

Inclusion Criteria:

1. Patients aged from ≥0 years of age at baseline
2. In the opinion of the treating physician the patient has a clinical presentation, radiological, biochemical or genetic investigation results that support diagnosis of XLH
3. Patient is not currently participating in an interventional clinical trial

A patient who meets any of the following criteria at the enrolment visit (baseline) will be excluded from this XLH Registry

Exclusion Criteria:

1. Patient or their legally designated representative does not have the cognitive capacity to provide informed consent.
2. 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).
3. 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

Where this trial is running

Brussels and 117 other locations

+68 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions X-Linked HypophosphatemiaCrysvitaBurosumabX-linked HypophosphatemiaPHEX Gene MutationXLH Registry
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.