Registry for Patients With X-Linked Hypophosphatemia

Active, not recruiting

• Conditions: X-Linked Hypophosphatemia

• Registration Number: NCT03193476
• Lead Sponsor: Kyowa Kirin Pharmaceutical Development Ltd

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.

Study Timeline

• Study First Submitted: 2017-06-13
• Study First Submitted QC: 2017-06-17
• Study First Posted: 2017-06-20
• Study Start: 2017-09-12
• Status Verified: 2023-04
• Last Update Submitted: 2023-04-06
• Last Update Posted: 2023-04-10
• Primary Completion: 2029-02-01
• Study Completion: 2029-07-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 1236

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

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	10 years	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Trial Locations

Locations (115)

"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

MVZ Dr. Eberhard & Partner; 🇩🇪 Dortmund, Germany

Universitaetsklinikum Freiburg; 🇩🇪 Freiburg, Germany

Medizinische Hochschule Hannover; 🇩🇪 Hannover, Germany

Hôpital Bicêtre; 🇫🇷 Paris, France

Uniklinikum Koeln; 🇩🇪 Koeln, Germany

Universitaetsklinikum; 🇩🇪 Leipzig, Germany

Hôpital Cochin; 🇫🇷 Paris, France

CHU Paris Sud - Hopital Bicêtre; 🇫🇷 Paris, France

Universitair Ziekenhuis Gent; 🇧🇪 Gent, Belgium

CHU de Liège; 🇧🇪 Liege, Belgium

CHU de Nantes; 🇫🇷 Nantes, France

ICOT-Istituto Chirurgico Ortopedico Traumatologico; 🇮🇹 Latina, Italy

Centre Hopitalier Universitaire Vaudois; 🇨🇭 Lausanne, Switzerland

Universitair Ziekenhuis Brussel; 🇧🇪 Brussels, Belgium

Hopital Universitaire des Enfants Reine Fabiola; 🇧🇪 Bruxelles, Belgium

Cliniques Universitaires Saint-Luc; 🇧🇪 Bruxelles, Belgium

UZA; 🇧🇪 Edegem, Belgium

UZ Leuven; 🇧🇪 Leuven, Belgium

SHATPD "Prof Ivan Mitev" EAD; 🇧🇬 Sofia, Bulgaria

UMHAT "SofiaMed", OOD; 🇧🇬 Sofia, Bulgaria

Children Hospital; 🇨🇿 Brno, Czechia

Fakultni nemocnice v Motole; 🇨🇿 Praha, 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 Cedex, France

CHRU Lille; 🇫🇷 Lille, France

Hopital Roger Salengro - CHU Lille; 🇫🇷 Lille, France

Hopital Arnaud de Villeneuve; 🇫🇷 Montpellier, France

Hormon and Stoffwechselzentrum; 🇩🇪 Mainz, Germany

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; 🇮🇹 Firenze, Italy

Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS; 🇮🇹 Genova, Italy

Azienda Ospedaliera Universitaria Policlinico G. Martino; 🇮🇹 Messina, Italy

Ospedale San Raffaele - Adult; 🇮🇹 Milano, Italy

Ospedale San Raffaele; 🇮🇹 Milano, 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; 🇵🇹 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, 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; 🇸🇪 Goteborg, Sweden

Lund University; 🇸🇪 Lund, Sweden

Karolinska Institute; 🇸🇪 Stockholm, Sweden

Karolinska Institute (Pediatric); 🇸🇪 Stockholm, Sweden

Inselspital Bern; 🇨🇭 Bern, Switzerland

Hopitaux Universitaires de Geneve (HUG); 🇨🇭 Genève, Switzerland

Kantonsspital St. Gallen; 🇨🇭 St. Gallen, Switzerland

Klinik Hirslanden Zuerich; 🇨🇭 Zuerich, 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