The European Paediatric Network for Haemophilia Management ( PedNet Registry) (PedNet)

October 26, 2023 updated by: Gili Kenet, PedNet Haemophilia Research Foundation

The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry

Rationale:

Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.

Objective:

To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.

Study Overview

Status

Recruiting

Conditions

Detailed Description

Design: Multicenter Prospective Observational Birth Cohort Study

Population:

Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and 1-1-2030.

Intervention:

No intervention; only documentation of patient characteristics and parameters of routine patient care and outcome

Main outcome parameters:

Outcome: clinically relevant inhibitor development, bleeding pattern and joint status on physical examination and imaging.

Determinants: baseline FVIII/IX levels, measurement of inhibitory antibodies, family history, FVIII/IX gene mutation, details on replacement therapy (according to each infusion for the first 50 treatment days, and annually thereafter) and surgeries.

Nature and extent of the burden and risks associated with participation, benefit and group relatedness:

  • No burden for the patients. Well-defined clinical data will be collected from the medical files. Participating in this registry will not change the number of visits to the clinic. All outcome parameters that are collected (including laboratory results) are part of routine clinical care.
  • Direct benefit is not to be expected. However, the direct interaction between centres that treat patients with rare diseases improves both clinical care and will result in better guidelines and as such may provide indirect benefit.
  • Multicentre participation: haemophilia is a very rare condition. Therefore, collecting data on a multi-centre observational cohort is the only way to study this specific population.
  • The registry concerns young boys and girls with haemophilia and cannot be performed in older patients, as >90% of inhibitors occur develop during the first 50 exposure days, and the results of prophylactic replacement therapy are highly dependent on the initiation of this treatment.

Study Type

Observational

Enrollment (Estimated)

4000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Austria
      • Graz, Austria, A-8036
        • Completed
        • Universitäts-Klinik für Kinder- und Jugendheilkunde
      • Vienna, Austria
        • Recruiting
        • Medical University of Vienna - Department of Paediatrics
        • Contact:
          • Christoph Male, MD
  • Belgium
      • Leuven, Belgium, B-3000
        • Recruiting
        • Service of Pediatric Haematology University Hospital Leuven
        • Contact:
          • Christel Van Geet, MD, PhD
  • Canada
      • Montréal, Canada, H3T 1C5
        • Recruiting
        • Division of Hematology/Oncology Hôpital St Justine
        • Contact:
          • George Rivard, MD
      • Toronto, Canada, M5G-1X8
        • Recruiting
        • Division of Haematology/Oncology Hospital for Sick Children
        • Contact:
          • Manuel Carcao, MD, PhD
  • Czechia
      • Brno, Czechia, 613 00
        • Recruiting
        • Haemophilia Comprehensive Care Centre, Centre for Thrombosis and Haemostasis Children's University Hospital Brno
        • Contact:
          • Jan Blatny, MD, PhD
      • Praha, Czechia, 150 06
        • Recruiting
        • Department of Paediatric Haematology/oncology - University Hospital Motol
        • Contact:
          • Esther Zapotocka, MD
  • Denmark
      • Aarhus, Denmark, DK-8200
        • Recruiting
        • Department of Pediatrics Århus Kommunehospital Skejby Sygehus
        • Contact:
          • Torben Stamm Mikkelsen, MD
  • Finland
      • Helsinki, Finland, FIN-00029
        • Recruiting
        • Children's Hospital Helsinki University Hospital
        • Contact:
          • Minna Koskenvuo, MD, PhD
  • France
      • Le Kremlin Bicêtre, France, F-94270
        • Recruiting
        • Service Hématologique Centre Regional Traitement d'Hemophilie Bicetre
        • Contact:
          • Roseline d'Oiron, MD
      • Marseille Cedex-05, France, F-13385
        • Recruiting
        • Service d'Hématologie Pédiatrique Hôpital Universitaire La Timone
        • Contact:
          • Hervé Chambost, MD, PhD
      • Toulouse, France, F-31052
        • Recruiting
        • Centre de traitement des hémophiles Hôpital Universitaire Purpan
        • Contact:
          • Caroline Oudot, MD
  • Germany
      • Bonn, Germany, D-53127
        • Recruiting
        • Institut für Experimentelle Hämatologie und Transfusionsmedizin Universitätsklinikum Bonn
        • Contact:
          • Johannes Oldenburg, MD, PhD
      • Bremen, Germany, D 28177
        • Recruiting
        • Klinik Bremen-Mitte Prof.-Hess-Kinderklinik
        • Contact:
          • Martina Bührlen, MD
      • Frankfurt am Main, Germany
        • Recruiting
        • University Hospital Frankfurt & Goethe University - Clinical and Molecular Hemostasis, Department of Pediatrics
        • Contact:
          • Christoph Königs, MD, PhD
      • Munich, Germany, D-80337
        • Recruiting
        • Dr. v. Haunersches Kinderspital University of Munich
        • Contact:
          • Martin Olivieri, MD
      • Mörfelden-Walldorf, Germany, D-64546
        • Recruiting
        • Hämophilie Zentrum Rhein Main
        • Contact:
          • Carmen Escuriola, MD
  • Greece
      • Athens, Greece, GR-11527
        • Recruiting
        • Haemophilia-Haemostasis Unit St. Sophia Children's Hospital
        • Contact:
          • Helen Pergantou, MD
  • Ireland
      • Dublin, Ireland
        • Recruiting
        • Dept of Paediatric Haematology Our Lady's Children's Hospital for Sick Children Crumlin
        • Contact:
          • Beatrice Nolan, MD
  • Israel
      • Ramat Gan, Israel, 52621
        • Recruiting
        • The National Hemophilia Center Sheba Medical Center, Tel Hashomer
        • Contact:
          • Gili Kenet, MD
  • Italy
      • Florence, Italy
        • Recruiting
        • Azienda Ospedaliero Universitaria Careggi
        • Contact:
          • Giancarlo Castaman, MD
      • Genova, Italy, I-16184
        • Recruiting
        • Gaslini Hospital
        • Contact:
          • Angelo Claudio Molinari, MD
      • Milano, Italy, 20122
        • Completed
        • A. Bianchi Bonomi Hemophilia and Thrombosis Centre IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Netherlands
      • Utrecht, Netherlands, 3508 GA
        • Recruiting
        • Van Creveld Kliniek University Medical Center Utrecht
        • Contact:
          • Kathelijn Fischer, MD, PhD
  • Norway
      • Oslo, Norway
        • Not yet recruiting
        • Oslo University Hospital
        • Contact:
          • Heidi Knüdsen, MD
  • Portugal
      • Porto, Portugal
        • Recruiting
        • Centro Hospitalar São João, S. Imuno-hemoterapia
        • Contact:
          • Manuela Carvalho, MD
  • Spain
      • Barcelona, Spain, 08035
        • Recruiting
        • Unitat Hemofilia Hospital Vall d'Hebron
        • Contact:
          • Olga Benítez Hidalgo, MD
      • Madrid, Spain, 28046
        • Recruiting
        • Unidad de Coagulopatías Hospital Universitario La Paz
        • Contact:
          • Maria Theresa Alvarez Roman, MD
      • Seville, Spain, 41013
        • Completed
        • Hospital General Unidad de Hemofilia 1 Sur Hospitales Universitarios Virgen del Rocio
      • Valencia, Spain, 46009
        • Recruiting
        • Unidad de Coagulopatias Congenitas Hospital Universitario la Fe
        • Contact:
          • Ana Rosa Cid, MD
  • Sweden
      • Malmo, Sweden, S-20502
        • Recruiting
        • Lund University Hospital
        • Contact:
          • Nadine Gretenkort Adersson, MD, PhD
      • Stockholm, Sweden, S-17176
        • Recruiting
        • Department of Pediatrics, Clinic of Coag. Disorders Karolinska Hospital
        • Contact:
          • Susanna Ranta, MD
  • Switzerland
      • Bern, Switzerland
        • Recruiting
        • Inselspital Bern, University Children's Hospital
        • Contact:
          • Mutlu Kartal-Kaess, MD
  • United Kingdom
      • Birmingham, United Kingdom
        • Recruiting
        • Birmingham Children's Hospital NHS Trust - Department of Haematology
        • Contact:
          • Jayashree Motwani, MD
      • Edinburgh, United Kingdom, EH9 1LF
        • Completed
        • Royal Hospital for Sick Children
      • Glasgow, United Kingdom, G 3885
        • Recruiting
        • Department of Haematology Royal Hospital for Sick Children
        • Contact:
          • Fernando Pinto, MD
      • London, United Kingdom, WC1N3JH
        • Completed
        • Haemophila Center Great Ormond Street Hospital for Children

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until January 1st 2030 who have been or are to be treated in one of the participating centres

Description

Inclusion Criteria:

  • Diagnosed with Haemophilia A or B
  • Factor VIII/ IX activity of <1 to 25%
  • Complete records of Factor treatment and bleeds
  • Treated in one of the participating centres

Exclusion Criteria:

  • Patients referred because of an inhibitor*
  • Informed consent not obtained

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Cohort I
Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until December 31st 2009 who have been or are to be treated with coagulation proteins in one of the participating centres
Cohort II
Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2010 until December 31st 2019 who have been or are to be treated with coagulation proteins in one of the participating centres
Cohort III
Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2020 until December 31st 2029 who have been or are to be treated with coagulation proteins in one of the participating centres

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of patients with antibody development to exogenous clotting factors
Time Frame: Until patient reaches age of 18
Allo-antibodies against Factor VIII and IX; Blood test: measurement in Bethesda units (BU), positive according to local standards, for most labs >0.5 BU
Until patient reaches age of 18

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Long term outcome of haemophilia on joint status using the Hemophilia Joint Health Score (HJHS) and MRI techniques.
Time Frame: From diagnose every 5 years until patient reaches age of 18
Effect of different prophylactic regimen on bleeding and joint damage
From diagnose every 5 years until patient reaches age of 18
Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor.
Time Frame: From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18
Effect of different ITI therapies on bleeding and joint damage. Joint damage is assessed using the HJHS and MRI.
From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

PedNet Haemophilia Research Foundation

Collaborators

Lund University Hospital

Investigators

  • Study Director: Gili Kenet, PhD, MD, The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, Israel
  • Study Chair: Christoph Male, MD, Medical University of Vienna
  • Principal Investigator: Gili Kenet, PhD, MD, The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, Israel

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2014

Primary Completion (Estimated)

December 1, 2029

Study Completion (Estimated)

December 1, 2029

Study Registration Dates

First Submitted

October 21, 2014

First Submitted That Met QC Criteria

November 28, 2016

First Posted (Estimated)

December 1, 2016

Study Record Updates

Last Update Posted (Actual)

October 30, 2023

Last Update Submitted That Met QC Criteria

October 26, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Version 6.4 November 2022

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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