Registry for Patients With X-Linked Hypophosphatemia

X-Linked Hypophosphatemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03193476
• Other study ID #: XLH Registry Version 3.0
• Status: Active, not recruiting
• 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
• 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

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• X-Linked Hypophosphatemia

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