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

April 6, 2023 updated by: Kyowa Kirin Pharmaceutical Development Ltd

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Detailed Description

Methodology

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:

  • Informed consent will be obtained from adult patients.
  • Parental informed consent for the inclusion of a child will be obtained from the child's legally designated representative in line with national guidance.
  • Assent will also be sought from children of applicable age in line with national guidance. In all cases the health professional responsible for enrolling the patient into the XLH Registry will assess the appropriateness of gaining assent from an individual at their discretion.

After the patient or legally designated representative has signed the informed consent, the patient data will be recorded in the XLH Registry, including baseline, retrospective and prospective data. Data will be collected using a web-based Electronic Data Capture (EDC) system. A patient identification number will be automatically generated by the system upon enrolment.

No pre-determined follow-up requirements will apply. However, physicians will be prompted to update patient data in the XLH Registry in relation to the patients' visits to the physician. Physicians will be reminded to update the XLH Registry at 12 months after the previous visit information was entered for a patient.

For those patients who attend clinic at more frequent intervals as part of their standard care, data for these visits will be entered at the 12-month intervals with an option to add multiple dates.

This is a prospective observational Registry and no additional interventions other than standard clinical practice are required by the protocol.

Study Type

Observational

Enrollment (Actual)

1236

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Belgium
      • Brussels, Belgium, 1090
        • Universitair Ziekenhuis Brussel
      • Bruxelles, Belgium, 1200
        • Cliniques Universitaires Saint-Luc
      • Bruxelles, Belgium, 1020
        • Hôpital Universitaire des Enfants Reine Fabiola
      • Edegem, Belgium, 2650
        • UZA
      • Leuven, Belgium, 3000
        • UZ Leuven
  • Bulgaria
      • Sofia, Bulgaria, 1797
        • UMHAT "SofiaMed", OOD
      • Sofia, Bulgaria, 1606
        • SHATPD "Prof Ivan Mitev" EAD
  • Czechia
      • Brno, Czechia, 625 00
        • Children Hospital
      • Praha, Czechia, 150 06
        • Fakultni nemocnice v Motole
  • Denmark
      • Aalborg, Denmark, 9100
        • Aalborg University Hospital
      • Aarhus, Denmark, 8200
        • Århus Universitetshospital - Aarhus sygehus
      • Aarhus, Denmark, 8200
        • Århus Universitetshospital
      • Esbjerg, Denmark, 6700
        • Hospital South West Jutland
      • København Ø, Denmark, 2100
        • Rigshospitalet
      • København Ø, Denmark, 2100
        • Rigshospitalet - Paediatric
      • Odense, Denmark, 5000
        • OUH
  • France
      • Bordeaux, France, 33000
        • Groupe Hospitalier Pellegrin - Hôpital des Enfants
      • Bron Cedex, France, 69677
        • CHU de Lyon - Hopital Femme Mère Enfant
      • Lille, France, 59037
        • CHRU Lille
      • Lille, France, 59037
        • Hopital Roger Salengro - CHU Lille
      • Montpellier, France, 34295
        • Hôpital Arnaud de Villeneuve
      • Paris, France, 75679
        • Hopital Cochin
      • Paris, France, 94270
        • CHU Paris Sud - Hopital Bicêtre
      • Paris, France, 94275
        • Hôpital Bicêtre
      • Toulouse, France, 31059
        • Hôpital Purpan
      • Toulouse, France, 31059
        • "Hopital Pierre-Paul Riquet Centre de Rhumatologie"
  • Germany
      • Bad Belzig, Germany
        • Klinikum Ernst-von-Bergmann Potsdam und Bad Belzig
      • Bochum, Germany, 44791
        • Uniklinikum Bochum
      • Dortmund, Germany, 44137
        • MVZ Dr. Eberhard & Partner
      • Freiburg, Germany, 79106
        • Universitaetsklinikum Freiburg
      • Hannover, Germany, 30625
        • Medizinische Hochschule Hannover
      • Koeln, Germany, 50937
        • Uniklinikum Koeln
      • Leipzig, Germany, 04103
        • Universitaetsklinikum
      • Mainz, Germany, 55122
        • Hormon and Stoffwechselzentrum
  • Hungary
      • Budapest, Hungary, 1083
        • Semmelweis Egyetem
      • Budapest, Hungary, 1085
        • Semmelweis Egyetem
  • Ireland
      • Dublin, Ireland, DO1 XD99
        • The Children's University Hospital
  • Israel
      • Haifa, Israel, 3109601
        • Rambam Health Care Center
      • Petah Tikva, Israel, 4920235
        • Schneider Medical Center
      • Ramat Gan, Israel, 5265601
        • Sheba Medical Center
      • Tel Aviv, Israel, 64239
        • Dana-Dwek Children's Hospital
  • Italy
      • Bari, Italy, 70124
        • Azienda Ospedaliero Universitaria Consorziale Policlinico Di Bari
      • Benevento, Italy, 82100
        • AO S. Pio G. Rumno
      • Bologna, Italy, 40138
        • Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi
      • Brindisi, Italy, 72100
        • Presidio Ospedaliero Di Summa Antonio Perrino
      • Cagliari, Italy, 09121
        • Azienda Ospedaliera Brotzu
      • Firenze, Italy, 50134
        • Azienda Ospedaliera Universitaria Careggi
      • Genova, Italy, 16147
        • Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
      • Messina, Italy, 98125
        • Azienda Ospedaliera Universitaria Policlinico G. Martino
      • Milano, Italy, 20132
        • Ospedale San Raffaele
      • Milano, Italy, 20132
        • Ospedale San Raffaele - Adult
      • Padova, Italy, 35128
        • Azienda Ospedaliera di Padova
      • Padua, Italy, 35100
        • Azienda Ospedaliera di Padova
      • Piancavallo, Italy, 20145
        • Istituto Auxologico Italiano -I.R.C.C.S.
      • Pisa, Italy, 56100
        • Stabilimento Ospedaliero Santa Chiara
      • Roma, Italy, 00165
        • Ospedale Pediatrico Bambino Gesù
      • Torino, Italy, 10126
        • Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino
      • Torino, Italy, 10126
        • Azienda Ospedaliera Città della Salute e della Scienza di Torino (Presidio Sant'Anna)
      • Trieste, Italy, 34137
        • I.R.C.C.S. Burlo Garofolo
  • Latvia
      • Riga, Latvia, LV-1002
        • Children's Clinical University Hospital
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • Universitair Medisch Centrum Groningen
  • Norway
      • Oslo, Norway, 0424
        • Oslo University Hospital
      • Oslo, Norway, 0450
        • Oslo Universitetssykehus HF, Ullevål
      • Trondheim, Norway, 7030
        • St. Olav's University Hospital
  • Portugal
      • Coimbra, Portugal, 3030
        • Hospital Pediatrico de Coimbra
      • Lisboa, Portugal, 1649-035
        • Centro Hospitalar de Lisboa Norte, E.P.E. - Hospital de Santa Maria
      • Lisboa, Portugal, 1169-045
        • Centro Hospitalar de Lisboa Central, E.P.E. - Hospital Dona Estefânia
      • Porto, Portugal, 4200-319
        • Hospital de Sao Joao
      • Porto, Portugal, 4099-001
        • Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António
  • Slovakia
      • Bratislava, Slovakia, 83340
        • National Institute of Childrenś Health NUDCH
  • Slovenia
      • Ljubljana, Slovenia
        • University Clinical Centre Ljubljana
  • Spain
      • Barakaldo, Spain, 48903
        • Hospital de Cruces
      • Barcelona, Spain, 08036
        • Hospital Clinic de Barcelona
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d'Hebron
      • Cartagena, Spain, 30202
        • Hospital General Universitario Santa Lucia
      • El Palmar, Spain, 30120
        • Hospital Universitario Virgen de la Arrixaca
      • Esplugues De Llobregat, Spain, 08950
        • Hospital Sant Joan de Déu
      • Las Palmas De Gran Canaria, Spain, 35016
        • Complejo Hospitalario Universitario Insular Materno-Infantil
      • Madrid, Spain, 28009
        • Hospital Infantil Universitario Nino Jesus
      • Oviedo, Spain, 33006
        • Hospital Universitario Central de Asturias
      • Oviedo, Spain, 33011
        • Hospital Central de Asturias
      • Santa Cruz de Tenerife, Spain, 38010
        • Hospital Universitario Nuestra Señora de la Candelaria
  • Sweden
      • Goteborg, Sweden, 416 85
        • Queen Silvia Children's Hospital
      • Lund, Sweden, 22185
        • Lund University
      • Stockholm, Sweden, 112 81
        • Karolinska Institute
      • Stockholm, Sweden, 171 77
        • Karolinska Institute (Pediatric)
  • Switzerland
      • Bern, Switzerland, 3010
        • Inselspital Bern
      • Genève, Switzerland, 1205
        • Hôpitaux universitaires de Genève (HUG)
      • St. Gallen, Switzerland, 9007
        • Kantonsspital St. Gallen
      • Zuerich, Switzerland, 8032
        • Klinik Hirslanden Zuerich
  • United Kingdom
      • Belfast, United Kingdom, BT12 6BE
        • Royal Belfast Hospital for Sick Children
      • Birmingham, United Kingdom, B4 6NH
        • Birmingham Children's Hospital
      • Bristol, United Kingdom, BS2 8AE
        • University Hospitals Bristol and Weston
      • Cambridge, United Kingdom, CB2 2QQ
        • Addenbrooke's Hospital
      • Cardiff, United Kingdom, CF14 4XW
        • University Hospital of Wales
      • Edinburgh, United Kingdom, EH4 2XU
        • Western General Hospital
      • Edinburgh, United Kingdom, EH9 1LF
        • Royal Hospital for Sick Children and Young People
      • Glasgow, United Kingdom, G12 0XH
        • Gartnavel General Hospital
      • Glasgow, United Kingdom, G12 0YN
        • Queen Elizabeth University Hospital
      • Inverness, United Kingdom, IV2 3UJ
        • Raigmore Hospital
      • Leeds, United Kingdom, LS1 3EX
        • Leeds General Infirmary
      • Liverpool, United Kingdom, L12 2AP
        • Alder Hey Children's Hospital
      • Liverpool, United Kingdom, L7 8XP
        • Royal Liverpool University Hospital
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital for Children
      • London, United Kingdom, SE1 7EH
        • Evelina Childrens Hospital
      • London, United Kingdom, NW1 2PG
        • National Hospital For Neurology and Neurosurgery
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Children's Hospital
      • Oxford, United Kingdom, OX3 7LD
        • Oxford University Hospital NHS Trust
      • Sheffield, United Kingdom, S5 7AU
        • Sheffield Teaching Hospitals
      • Sheffield, United Kingdom, S10 2RX
        • Sheffield Childrens Hospital
      • Southampton, United Kingdom, SO16 6YD
        • Southampton General Hospital
      • Stanmore, United Kingdom, HA7 4LP
        • Stanmore Royal Orthopaedic Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with XLH independent of treatment regimen

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame: 10 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Kyowa Kirin Pharmaceutical Development Ltd

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 12, 2017

Primary Completion (Anticipated)

February 1, 2029

Study Completion (Anticipated)

July 1, 2029

Study Registration Dates

First Submitted

June 13, 2017

First Submitted That Met QC Criteria

June 17, 2017

First Posted (Actual)

June 20, 2017

Study Record Updates

Last Update Posted (Actual)

April 10, 2023

Last Update Submitted That Met QC Criteria

April 6, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • XLH Registry Version 3.0

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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