Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
1343 participants
OBSERVATIONAL
2017-09-12
2029-07-01
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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OTHER
OTHER
Eligibility Criteria
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Inclusion Criteria
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
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
ALL
No
Sponsors
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Iqvia Pty Ltd
INDUSTRY
Kyowa Kirin Pharmaceutical Development Ltd
INDUSTRY
Responsible Party
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Locations
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Hopital Universitaire des Enfants Reine Fabiola
Brussels, , Belgium
Universitair Ziekenhuis Brussel
Brussels, , Belgium
Cliniques Universitaires Saint-Luc
Brussels, , Belgium
UZA
Edegem, , Belgium
Universitair Ziekenhuis Gent
Ghent, , Belgium
UZ Leuven
Leuven, , Belgium
CHU de Liège
Liège, , Belgium
SHATPD "Prof Ivan Mitev" EAD
Sofia, , Bulgaria
UMHAT "SofiaMed", OOD
Sofia, , Bulgaria
Children Hospital
Brno, , Czechia
Fakultni nemocnice v Motole
Prague, , Czechia
Aalborg University Hospital
Aalborg, , Denmark
Århus Universitetshospital - Aarhus sygehus
Aarhus, , Denmark
Århus Universitetshospital
Aarhus, , Denmark
Hospital South West Jutland
Esbjerg, , Denmark
Rigshospitalet - Paediatric
København Ø, , Denmark
Rigshospitalet
København Ø, , Denmark
OUH
Odense, , Denmark
Groupe Hospitalier Pellegrin - Hôpital des Enfants
Bordeaux, , France
CHU de Lyon - Hôpital Femme Mere Enfant
Bron, , France
CHRU Lille
Lille, , France
Hopital Roger Salengro - CHU Lille
Lille, , France
Hopital Arnaud de Villeneuve
Montpellier, , France
CHU de Nantes
Nantes, , France
Hôpital Cochin
Paris, , France
CHU Paris Sud - Hopital Bicêtre
Paris, , France
Hôpital Bicêtre
Paris, , France
"Hopital Pierre-Paul Riquet Centre de Rhumatologie"
Toulouse, , France
Hopital Purpan
Toulouse, , France
Klinikum Ernst-von-Bergmann Potsdam und Bad Belzig
Bad Belzig, , Germany
Uniklinikum Bochum
Bochum, , Germany
Uniklinikum Koeln
Cologne, , Germany
MVZ Dr. Eberhard & Partner
Dortmund, , Germany
Universitaetsklinikum Freiburg
Freiburg im Breisgau, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Universitaetsklinikum
Leipzig, , Germany
Hormon and Stoffwechselzentrum
Mainz, , Germany
Semmelweis Egyetem
Budapest, , Hungary
Semmelweis Egyetem
Budapest, , Hungary
The Children's University Hospital
Dublin, , Ireland
Rambam Health Care Center
Haifa, , Israel
Schneider Medical Center
Petah Tikva, , Israel
Sheba Medical Center
Ramat Gan, , Israel
Dana-Dwek Children's Hospital
Tel Aviv, , Israel
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
Bari, , Italy
AO S. Pio G. Rumno
Benevento, , Italy
Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi
Bologna, , Italy
Presidio Ospedaliero Di Summa Antonio Perrino
Brindisi, , Italy
Azienda Ospedaliera Brotzu
Cagliari, , Italy
Azienda Ospedaliera Universitaria Careggi
Florence, , Italy
Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
Genova, , Italy
ICOT-Istituto Chirurgico Ortopedico Traumatologico
Latina, , Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, , Italy
Ospedale San Raffaele - Adult
Milan, , Italy
Ospedale San Raffaele
Milan, , Italy
Azienda Ospedaliera di Padova
Padua, , Italy
Azienda Ospedaliera di Padova
Padua, , Italy
Istituto Auxologico Italiano -I.R.C.C.S.
Piancavallo, , Italy
Stabilimento Ospedaliero Santa Chiara
Pisa, , Italy
Ospedale Pediatrico Bambino Gesù
Roma, , Italy
Azienda Ospedaliera Città della Salute e della Scienza di Torino (Presidio Sant'Anna)
Torino, , Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, , Italy
I.R.C.C.S. Burlo Garofolo
Trieste, , Italy
Children's Clinical University Hospital
Riga, , Latvia
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Oslo University Hospital
Oslo, , Norway
Oslo Universitetssykehus HF, Ulleval
Oslo, , Norway
St. Olav's University Hospital
Trondheim, , Norway
Hospital Pediatrico de Coimbra
Coimbra, , Portugal
Centro Hospitalar de Lisboa Central, E.P.E. - Hospital Dona Estefânia
Lisbon, , Portugal
Centro Hospitalar de Lisboa Norte, E.P.E. - Hospital de Santa Maria
Lisbon, , Portugal
Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António
Porto, , Portugal
Hospital de Sao Joao
Porto, , Portugal
National Institute of Childrenś Health NUDCH
Bratislava, , Slovakia
University Clinical Centre Ljubljana
Ljubljana, , Slovenia
Hospital de Cruces
Barakaldo, , Spain
Hospital Universitari Vall d'Hebron
Barcelona, , Spain
Hospital Clinic de Barcelona
Barcelona, , Spain
Hospital General Universitario Santa Lucía
Cartagena, , Spain
Hospital Universitario Virgen de la Arrixaca
El Palmar, , Spain
Hospital Sant Joan de Deu
Esplugues de Llobregat, , Spain
Complejo Hospitalario Universitario Insular Materno-Infantil
Las Palmas de Gran Canaria, , Spain
Hospital Infantil Universitario Niño Jesus
Madrid, , Spain
Hospital Universitario Central de Asturias
Oviedo, , Spain
Hospital Central de Asturias
Oviedo, , Spain
Hospital Universitario Nuestra Señora de la Candelaria
Santa Cruz de Tenerife, , Spain
Queen Silvia Children's Hospital
Gothenburg, , Sweden
Lund University
Lund, , Sweden
Karolinska Institute
Stockholm, , Sweden
Karolinska Institute (Pediatric)
Stockholm, , Sweden
Inselspital Bern
Bern, , Switzerland
Hopitaux Universitaires de Geneve (HUG)
Geneva, , Switzerland
Centre Hopitalier Universitaire Vaudois
Lausanne, , Switzerland
Centre Hopitalier Universitaire Vaudois
Lausanne, , Switzerland
Kantonsspital St. Gallen
Sankt Gallen, , Switzerland
Klinik Hirslanden Zuerich
Zurich, , Switzerland
Royal Belfast Hospital for Sick Children
Belfast, , United Kingdom
Birmingham Children's Hospital
Birmingham, , United Kingdom
University Hospitals Bristol and Weston
Bristol, , United Kingdom
Addenbrooke's Hospital
Cambridge, , United Kingdom
University Hospital of Wales
Cardiff, , United Kingdom
Western General Hospital
Edinburgh, , United Kingdom
Royal Hospital for Sick Children and Young People
Edinburgh, , United Kingdom
Gartnavel General Hospital
Glasgow, , United Kingdom
Queen Elizabeth University Hospital
Glasgow, , United Kingdom
Raigmore Hospital
Inverness, , United Kingdom
Leeds General Infirmary
Leeds, , United Kingdom
Alder Hey Children's Hospital
Liverpool, , United Kingdom
Royal Liverpool University Hospital
Liverpool, , United Kingdom
National Hospital for Neurology and Neurosurgery
London, , United Kingdom
Evelina Childrens Hospital
London, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Oxford University Hospital NHS Trust
Oxford, , United Kingdom
Sheffield Childrens Hospital
Sheffield, , United Kingdom
Sheffield Teaching Hospitals
Sheffield, , United Kingdom
Southampton General Hospital
Southampton, , United Kingdom
Stanmore Royal Orthopaedic Hospital
Stanmore, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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References
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Carpenter TO, Fukumoto S, Haffner D, Imel EA, Ozono K, Ishii H, Li Z, Sandilands K, Joos-Vandewalle P, Lee C, Kanematsu M, McCullough KP, Brandi ML. Advancing Patient Evidence in XLH (APEX): Baseline analysis of a global data unification program. Bone. 2025 Dec;201:117649. doi: 10.1016/j.bone.2025.117649. Epub 2025 Sep 15.
Brandi ML, Ariceta G, Beck-Nielsen SS, Boot AM, Briot K, de Lucas Collantes C, Emma F, Giannini S, Haffner D, Keen R, Levtchenko E, Mӓkitie O, Nilsson O, Schnabel D, Tripto-Shkolnik L, Zillikens MC, Liu J, Tudor A, Mughal MZ. Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia: rationale and description. Ther Adv Chronic Dis. 2022 Sep 5;13:20406223221117471. doi: 10.1177/20406223221117471. eCollection 2022.
Other Identifiers
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XLH Registry Version 3.0
Identifier Type: -
Identifier Source: org_study_id
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