Clinical trial NCT03193476: Registry for Patients With X-Linked Hypophosphatemia
Official title: An International, Multicentre, Prospective, Non-interventional Observational Registry for Patients With X-Linked Hypophosphatemia (XLH)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: X-Linked Hypophosphatemia
Keywords: Crysvita
Burosumab
X-linked Hypophosphatemia (XLH)
PHEX Gene Mutation
XLH Registry
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Iqvia Pty Ltd
First posted: 2017-06-20
Enrollment: 1343
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with XLH independent of treatment regimen

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.

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.

Eligibility 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

Primary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: Universitair Ziekenhuis Brussel
city: Bruxelles
country: Belgium
facility: Hopital Universitaire des Enfants Reine Fabiola
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Edegem
country: Belgium
facility: UZA
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Leuven
country: Belgium
facility: UZ Leuven
city: Liege
country: Belgium
facility: CHU de Liège
city: Sofia
country: Bulgaria
facility: SHATPD "Prof Ivan Mitev" EAD
city: Sofia
country: Bulgaria
facility: UMHAT "SofiaMed", OOD
city: Brno
country: Czechia
facility: Children Hospital
city: Praha
country: Czechia
facility: Fakultni nemocnice v Motole
city: Aalborg
country: Denmark
facility: Aalborg University Hospital
city: Aarhus
country: Denmark
facility: Århus Universitetshospital - Aarhus sygehus
city: Aarhus
country: Denmark
facility: Århus Universitetshospital
city: Esbjerg
country: Denmark
facility: Hospital South West Jutland
city: København Ø
country: Denmark
facility: Rigshospitalet - Paediatric
city: København Ø
country: Denmark
facility: Rigshospitalet
city: Odense
country: Denmark
facility: OUH
city: Bordeaux
country: France
facility: Groupe Hospitalier Pellegrin - Hôpital des Enfants
city: Bron Cedex
country: France
facility: CHU de Lyon - Hôpital Femme Mere Enfant
city: Lille
country: France
facility: CHRU Lille
city: Lille
country: France
facility: Hopital Roger Salengro - CHU Lille
city: Montpellier
country: France
facility: Hopital Arnaud de Villeneuve
city: Nantes
country: France
facility: CHU de Nantes
city: Paris
country: France
facility: Hôpital Cochin
city: Paris
country: France
facility: CHU Paris Sud - Hopital Bicêtre
city: Paris
country: France
facility: Hôpital Bicêtre
city: Toulouse
country: France
facility: "Hopital Pierre-Paul Riquet Centre de Rhumatologie"
city: Toulouse
country: France
facility: Hopital Purpan
city: Bad Belzig
country: Germany
facility: Klinikum Ernst-von-Bergmann Potsdam und Bad Belzig
city: Bochum
country: Germany
facility: Uniklinikum Bochum
city: Dortmund
country: Germany
facility: MVZ Dr. Eberhard & Partner
city: Freiburg
country: Germany
facility: Universitaetsklinikum Freiburg
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Koeln
country: Germany
facility: Uniklinikum Koeln
city: Leipzig
country: Germany
facility: Universitaetsklinikum
city: Mainz
country: Germany
facility: Hormon and Stoffwechselzentrum
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Dublin
country: Ireland
facility: The Children's University Hospital
city: Haifa
country: Israel
facility: Rambam Health Care Center
city: Petah Tikva
country: Israel
facility: Schneider Medical Center
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
city: Tel Aviv
country: Israel
facility: Dana-Dwek Children's Hospital
city: Bari
country: Italy
facility: Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
city: Benevento
country: Italy
facility: AO S. Pio G. Rumno
city: Bologna
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi
city: Brindisi
country: Italy
facility: Presidio Ospedaliero Di Summa Antonio Perrino
city: Cagliari
country: Italy
facility: Azienda Ospedaliera Brotzu
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Genova
country: Italy
facility: Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
city: Latina
country: Italy
facility: ICOT-Istituto Chirurgico Ortopedico Traumatologico
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico G. Martino
city: Milano
country: Italy
facility: Ospedale San Raffaele - Adult
city: Milano
country: Italy
facility: Ospedale San Raffaele
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Padua
country: Italy
facility: Azienda Ospedaliera di Padova
city: Piancavallo
country: Italy
facility: Istituto Auxologico Italiano -I.R.C.C.S.
city: Pisa
country: Italy
facility: Stabilimento Ospedaliero Santa Chiara
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Torino
country: Italy
facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino (Presidio Sant'Anna)
city: Torino
country: Italy
facility: Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
city: Trieste
country: Italy
facility: I.R.C.C.S. Burlo Garofolo
city: Riga
country: Latvia
facility: Children's Clinical University Hospital
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen
city: Oslo
country: Norway
facility: Oslo University Hospital
city: Oslo
country: Norway
facility: Oslo Universitetssykehus HF, Ulleval
city: Trondheim
country: Norway
facility: St. Olav's University Hospital
city: Coimbra
country: Portugal
facility: Hospital Pediatrico de Coimbra
city: Lisboa
country: Portugal
facility: Centro Hospitalar de Lisboa Central, E.P.E. - Hospital Dona Estefânia
city: Lisboa
country: Portugal
facility: Centro Hospitalar de Lisboa Norte, E.P.E. - Hospital de Santa Maria
city: Porto
country: Portugal
facility: Centro Hospitalar do Porto, E.P.E. - Hospital de Santo António
city: Porto
country: Portugal
facility: Hospital de Sao Joao
city: Bratislava
country: Slovakia
facility: National Institute of Childrenś Health NUDCH
city: Ljubljana
country: Slovenia
facility: University Clinical Centre Ljubljana
city: Barakaldo
country: Spain
facility: Hospital de Cruces
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Cartagena
country: Spain
facility: Hospital General Universitario Santa Lucía
city: El Palmar
country: Spain
facility: Hospital Universitario Virgen de la Arrixaca
city: Esplugues De Llobregat
country: Spain
facility: Hospital Sant Joan de Deu
city: Las Palmas De Gran Canaria
country: Spain
facility: Complejo Hospitalario Universitario Insular Materno-Infantil
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Niño Jesus
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
city: Oviedo
country: Spain
facility: Hospital Central de Asturias
city: Santa Cruz de Tenerife
country: Spain
facility: Hospital Universitario Nuestra Señora de la Candelaria
city: Goteborg
country: Sweden
facility: Queen Silvia Children's Hospital
city: Lund
country: Sweden
facility: Lund University
city: Stockholm
country: Sweden
facility: Karolinska Institute
city: Stockholm
country: Sweden
facility: Karolinska Institute (Pediatric)
city: Bern
country: Switzerland
facility: Inselspital Bern
city: Genève
country: Switzerland
facility: Hopitaux Universitaires de Geneve (HUG)
city: Lausanne
country: Switzerland
facility: Centre Hopitalier Universitaire Vaudois
city: Lausanne
country: Switzerland
facility: Centre Hopitalier Universitaire Vaudois
city: St. Gallen
country: Switzerland
facility: Kantonsspital St. Gallen
city: Zuerich
country: Switzerland
facility: Klinik Hirslanden Zuerich
city: Belfast
country: United Kingdom
facility: Royal Belfast Hospital for Sick Children
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: Edinburgh
country: United Kingdom
facility: Western General Hospital
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Sick Children and Young People
city: Glasgow
country: United Kingdom
facility: Gartnavel General Hospital
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
city: Inverness
country: United Kingdom
facility: Raigmore Hospital
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Hospital
city: Liverpool
country: United Kingdom
facility: Royal Liverpool University Hospital
city: London
country: United Kingdom
facility: National Hospital for Neurology and Neurosurgery
city: London
country: United Kingdom
facility: Evelina Childrens Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
city: Oxford
country: United Kingdom
facility: Oxford University Hospital NHS Trust
city: Sheffield
country: United Kingdom
facility: Sheffield Childrens Hospital
city: Sheffield
country: United Kingdom
facility: Sheffield Teaching Hospitals
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
city: Stanmore
country: United Kingdom
facility: Stanmore Royal Orthopaedic Hospital
Study officials:
References:
citation: 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.