Registry for Patients With X-Linked Hypophosphatemia
An International, Multicentre, Prospective, Non-interventional Observational Registry for Patients With X-Linked Hypophosphatemia (XLH)
1 other identifier
observational
1,343
19 countries
117
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2017
Longer than P75 for all trials
117 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 13, 2017
CompletedFirst Posted
Study publicly available on registry
June 20, 2017
CompletedStudy Start
First participant enrolled
September 12, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 1, 2029
September 19, 2024
September 1, 2024
11.4 years
June 13, 2017
September 4, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
10 years
Eligibility Criteria
Patients with XLH independent of treatment regimen
You may qualify if:
- 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
- A patient who meets any of the following criteria at the enrolment visit (baseline) will be excluded from this XLH Registry
You may not qualify if:
- Patient or their legally designated representative does not have the cognitive capacity to provide informed consent.
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Kyowa Kirin Pharmaceutical Development Ltdlead
- Iqvia Pty Ltdcollaborator
Study Sites (118)
Hopital Universitaire des Enfants Reine Fabiola
Brussels, 1020, Belgium
Universitair Ziekenhuis Brussel
Brussels, 1090, Belgium
Cliniques Universitaires Saint-Luc
Brussels, 1200, Belgium
UZA
Edegem, 2650, Belgium
Universitair Ziekenhuis Gent
Ghent, 9000, Belgium
UZ Leuven
Leuven, 3000, Belgium
CHU de Liège
Liège, 4000, Belgium
SHATPD "Prof Ivan Mitev" EAD
Sofia, 1606, Bulgaria
UMHAT "SofiaMed", OOD
Sofia, 1797, Bulgaria
Children Hospital
Brno, 625 00, Czechia
Fakultni nemocnice v Motole
Prague, 150 06, Czechia
Aalborg University Hospital
Aalborg, 9100, Denmark
Århus Universitetshospital - Aarhus sygehus
Aarhus, 8200, Denmark
Århus Universitetshospital
Aarhus, 8200, Denmark
Hospital South West Jutland
Esbjerg, 6700, Denmark
Rigshospitalet - Paediatric
København Ø, 2100, Denmark
Rigshospitalet
København Ø, 2100, Denmark
OUH
Odense, 5000, Denmark
Groupe Hospitalier Pellegrin - Hôpital des Enfants
Bordeaux, 33000, France
CHU de Lyon - Hôpital Femme Mere Enfant
Bron, 69677, France
CHRU Lille
Lille, 59037, France
Hopital Roger Salengro - CHU Lille
Lille, 59037, France
Hopital Arnaud de Villeneuve
Montpellier, 34295, France
CHU de Nantes
Nantes, 44000, France
Hôpital Cochin
Paris, 75679, France
CHU Paris Sud - Hopital Bicêtre
Paris, 94270, France
Hôpital Bicêtre
Paris, 94275, France
"Hopital Pierre-Paul Riquet Centre de Rhumatologie"
Toulouse, 31059, France
Hopital Purpan
Toulouse, 31059, France
Klinikum Ernst-von-Bergmann Potsdam und Bad Belzig
Bad Belzig, Germany
Uniklinikum Bochum
Bochum, 44791, Germany
Uniklinikum Koeln
Cologne, 50937, Germany
MVZ Dr. Eberhard & Partner
Dortmund, 44137, Germany
Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
Medizinische Hochschule Hannover
Hanover, 30625, Germany
Universitaetsklinikum
Leipzig, 04103, Germany
Hormon and Stoffwechselzentrum
Mainz, 55122, Germany
Semmelweis Egyetem
Budapest, 1083, Hungary
Semmelweis Egyetem
Budapest, 1085, Hungary
The Children's University Hospital
Dublin, DO1 XD99, Ireland
Rambam Health Care Center
Haifa, 3109601, Israel
Schneider Medical Center
Petah Tikva, 4920235, Israel
Sheba Medical Center
Ramat Gan, 5265601, Israel
Dana-Dwek Children's Hospital
Tel Aviv, 64239, Israel
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
Bari, 70124, Italy
AO S. Pio G. Rumno
Benevento, 82100, Italy
Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi
Bologna, 40138, Italy
Presidio Ospedaliero Di Summa Antonio Perrino
Brindisi, 72100, Italy
Azienda Ospedaliera Brotzu
Cagliari, 09121, Italy
Azienda Ospedaliera Universitaria Careggi
Florence, 50134, Italy
Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
Genova, 16147, Italy
ICOT-Istituto Chirurgico Ortopedico Traumatologico
Latina, 04100, Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, 98125, Italy
Ospedale San Raffaele - Adult
Milan, 20132, Italy
Ospedale San Raffaele
Milan, 20132, Italy
Azienda Ospedaliera di Padova
Padua, 35100, Italy
Azienda Ospedaliera di Padova
Padua, 35128, Italy
Istituto Auxologico Italiano -I.R.C.C.S.
Piancavallo, 20145, Italy
Stabilimento Ospedaliero Santa Chiara
Pisa, 56100, Italy
Ospedale Pediatrico Bambino Gesù
Roma, 00165, Italy
Azienda Ospedaliera Città della Salute e della Scienza di Torino (Presidio Sant'Anna)
Torino, 10126, Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, 10126, Italy
I.R.C.C.S. Burlo Garofolo
Trieste, 34137, Italy
Children's Clinical University Hospital
Riga, LV-1002, Latvia
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
Oslo University Hospital
Oslo, 0424, Norway
Oslo Universitetssykehus HF, Ulleval
Oslo, 0450, Norway
St. Olav's University Hospital
Trondheim, 7030, Norway
Hospital Pediatrico de Coimbra
Coimbra, 3030, Portugal
Centro Hospitalar de Lisboa Central, E.P.E. - Hospital Dona Estefânia
Lisbon, 1169-045, Portugal
Centro Hospitalar de Lisboa Norte, E.P.E. - Hospital de Santa Maria
Lisbon, 1649-035, Portugal
Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António
Porto, 4099-001, Portugal
Hospital de Sao Joao
Porto, 4200-319, Portugal
National Institute of Childrenś Health NUDCH
Bratislava, 83340, Slovakia
University Clinical Centre Ljubljana
Ljubljana, Slovenia
Hospital de Cruces
Barakaldo, 48903, Spain
Hospital Universitari Vall d'Hebron
Barcelona, 08035, Spain
Hospital Clinic de Barcelona
Barcelona, 08036, Spain
Hospital General Universitario Santa Lucía
Cartagena, 30202, Spain
Hospital Universitario Virgen de la Arrixaca
El Palmar, 30120, Spain
Hospital Sant Joan de Deu
Esplugues de Llobregat, 08950, Spain
Complejo Hospitalario Universitario Insular Materno-Infantil
Las Palmas de Gran Canaria, 35016, Spain
Hospital Infantil Universitario Niño Jesus
Madrid, 28009, Spain
Hospital Universitario Central de Asturias
Oviedo, 33006, Spain
Hospital Central de Asturias
Oviedo, 33011, Spain
Hospital Universitario Nuestra Señora de la Candelaria
Santa Cruz de Tenerife, 38010, Spain
Queen Silvia Children's Hospital
Gothenburg, 416 85, Sweden
Lund University
Lund, 22185, Sweden
Karolinska Institute
Stockholm, 112 81, Sweden
Karolinska Institute (Pediatric)
Stockholm, 171 77, Sweden
Inselspital Bern
Bern, 3010, Switzerland
Hopitaux Universitaires de Geneve (HUG)
Geneva, 1205, Switzerland
Centre Hopitalier Universitaire Vaudois
Lausanne, 1011, Switzerland
Centre Hopitalier Universitaire Vaudois
Lausanne, 1011, Switzerland
Kantonsspital St. Gallen
Sankt Gallen, 9007, Switzerland
Klinik Hirslanden Zuerich
Zurich, 8032, Switzerland
Royal Belfast Hospital for Sick Children
Belfast, BT12 6BE, United Kingdom
Birmingham Children's Hospital
Birmingham, B4 6NH, United Kingdom
University Hospitals Bristol and Weston
Bristol, BS2 8AE, United Kingdom
Addenbrooke's Hospital
Cambridge, CB2 2QQ, United Kingdom
University Hospital of Wales
Cardiff, CF14 4XW, United Kingdom
Western General Hospital
Edinburgh, EH4 2XU, United Kingdom
Royal Hospital for Sick Children and Young People
Edinburgh, EH9 1LF, United Kingdom
Gartnavel General Hospital
Glasgow, G12 0XH, United Kingdom
Queen Elizabeth University Hospital
Glasgow, G12 0YN, United Kingdom
Raigmore Hospital
Inverness, IV2 3UJ, United Kingdom
Leeds General Infirmary
Leeds, LS1 3EX, United Kingdom
Alder Hey Children's Hospital
Liverpool, L12 2AP, United Kingdom
Royal Liverpool University Hospital
Liverpool, L7 8XP, United Kingdom
National Hospital for Neurology and Neurosurgery
London, NW1 2PG, United Kingdom
Evelina Childrens Hospital
London, SE1 7EH, United Kingdom
Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
Oxford University Hospital NHS Trust
Oxford, OX3 7LD, United Kingdom
Sheffield Childrens Hospital
Sheffield, S10 2RX, United Kingdom
Sheffield Teaching Hospitals
Sheffield, S5 7AU, United Kingdom
Southampton General Hospital
Southampton, SO16 6YD, United Kingdom
Stanmore Royal Orthopaedic Hospital
Stanmore, HA7 4LP, United Kingdom
Related Publications (2)
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.
PMID: 40962182DERIVEDBrandi 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.
PMID: 36082134DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- OTHER
- Time Perspective
- OTHER
- Target Duration
- 10 Years
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 13, 2017
First Posted
June 20, 2017
Study Start
September 12, 2017
Primary Completion (Estimated)
February 1, 2029
Study Completion (Estimated)
July 1, 2029
Last Updated
September 19, 2024
Record last verified: 2024-09