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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03193476
Other study ID # XLH Registry Version 3.0
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date September 12, 2017
Est. completion date July 1, 2029

Study information

Verified date April 2023
Source Kyowa Kirin Pharmaceutical Development Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 1236
Est. completion date July 1, 2029
Est. primary completion date February 1, 2029
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility 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 Design


Related Conditions & MeSH terms


Locations

Country Name City State
Belgium Universitair Ziekenhuis Brussel Brussels
Belgium Cliniques Universitaires Saint-Luc Bruxelles
Belgium Hopital Universitaire des Enfants Reine Fabiola Bruxelles
Belgium UZA Edegem
Belgium UZ Leuven Leuven
Bulgaria SHATPD "Prof Ivan Mitev" EAD Sofia
Bulgaria UMHAT "SofiaMed", OOD Sofia
Czechia Children Hospital Brno
Czechia Fakultni nemocnice v Motole Praha
Denmark Aalborg University Hospital Aalborg
Denmark Århus Universitetshospital Aarhus
Denmark Århus Universitetshospital - Aarhus sygehus Aarhus
Denmark Hospital South West Jutland Esbjerg
Denmark Rigshospitalet København Ø
Denmark Rigshospitalet - Paediatric København Ø
Denmark OUH Odense
France Groupe Hospitalier Pellegrin - Hôpital des Enfants Bordeaux
France CHU de Lyon - Hôpital Femme Mere Enfant Bron Cedex
France CHRU Lille Lille
France Hopital Roger Salengro - CHU Lille Lille
France Hopital Arnaud de Villeneuve Montpellier
France CHU Paris Sud - Hopital Bicêtre Paris
France Hôpital Bicêtre Paris
France Hôpital Cochin Paris
France "Hopital Pierre-Paul Riquet Centre de Rhumatologie" Toulouse
France Hopital Purpan Toulouse
Germany Klinikum Ernst-von-Bergmann Potsdam und Bad Belzig Bad Belzig
Germany Uniklinikum Bochum Bochum
Germany MVZ Dr. Eberhard & Partner Dortmund
Germany Universitaetsklinikum Freiburg Freiburg
Germany Medizinische Hochschule Hannover Hannover
Germany Uniklinikum Koeln Koeln
Germany Universitaetsklinikum Leipzig
Germany Hormon and Stoffwechselzentrum Mainz
Hungary Semmelweis Egyetem Budapest
Hungary Semmelweis Egyetem Budapest
Ireland The Children's University Hospital Dublin
Israel 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
Italy 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 Firenze
Italy Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS Genova
Italy Azienda Ospedaliera Universitaria Policlinico G. Martino Messina
Italy Ospedale San Raffaele Milano
Italy Ospedale San Raffaele - Adult Milano
Italy Azienda Ospedaliera di Padova Padova
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
Latvia Children's Clinical University Hospital Riga
Netherlands Universitair Medisch Centrum Groningen Groningen
Norway Oslo Universitetssykehus HF, Ulleval Oslo
Norway Oslo University Hospital Oslo
Norway St. Olav's University Hospital Trondheim
Portugal Hospital Pediatrico de Coimbra Coimbra
Portugal Centro Hospitalar de Lisboa Central, E.P.E. - Hospital Dona Estefânia Lisboa
Portugal Centro Hospitalar de Lisboa Norte, E.P.E. - Hospital de Santa Maria Lisboa
Portugal Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António Porto
Portugal Hospital de Sao Joao Porto
Slovakia National Institute of Childrens Health NUDCH Bratislava
Slovenia University Clinical Centre Ljubljana Ljubljana
Spain Hospital de Cruces Barakaldo
Spain Hospital Clinic de Barcelona Barcelona
Spain Hospital Universitari Vall d'Hebron 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 Central de Asturias Oviedo
Spain Hospital Universitario Central de Asturias Oviedo
Spain Hospital Universitario Nuestra Señora de la Candelaria Santa Cruz de Tenerife
Sweden Queen Silvia Children's Hospital Goteborg
Sweden Lund University Lund
Sweden Karolinska Institute Stockholm
Sweden Karolinska Institute (Pediatric) Stockholm
Switzerland Inselspital Bern Bern
Switzerland Hopitaux Universitaires de Geneve (HUG) Genève
Switzerland Kantonsspital St. Gallen St. Gallen
Switzerland Klinik Hirslanden Zuerich Zuerich
United Kingdom 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 Royal Hospital for Sick Children and Young People Edinburgh
United Kingdom Western General Hospital 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 Evelina Childrens Hospital London
United Kingdom Great Ormond Street Hospital for Children London
United Kingdom National Hospital for Neurology and Neurosurgery 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

Sponsors (1)

Lead Sponsor Collaborator
Kyowa Kirin Pharmaceutical Development Ltd

Countries where clinical trial is conducted

Belgium,  Bulgaria,  Czechia,  Denmark,  France,  Germany,  Hungary,  Ireland,  Israel,  Italy,  Latvia,  Netherlands,  Norway,  Portugal,  Slovakia,  Slovenia,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
See also
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Completed NCT03596554 - X-linked Hypophosphatemia and FGF21
Recruiting NCT03820518 - Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia Phase 4
Completed NCT01571596 - An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia Phase 1/Phase 2
Completed NCT04273490 - Characterising Pain, QoL, Body Composition, Arterial Stiffness, Muscles and Bones in Adult Persons With XLH and Healthy Controls
Completed NCT03489993 - FGF23 and Angiotensin-(1-7) in Hypophosphatemia (GAP)