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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02979119
Recruitment Status : Recruiting
First Posted : December 1, 2016
Last Update Posted : November 6, 2017
Lund University Hospital
Information provided by (Responsible Party):
Marijke van den Berg, PedNet Haemophilia Research Foundation

Brief Summary:


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.


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

Condition or disease Intervention/treatment
Factor VIII Deficiency Factor IX Deficiency Drug: Coagulation proteins

Detailed Description:

Design: Prospective observational cohort


Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and 1-1-2020.


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 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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 2200 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 20 Years
Official Title: The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry
Study Start Date : June 2014
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Group/Cohort Intervention/treatment
Prospective Birth cohort
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 2020 who have been or are to be treated with coagulation proteins in one of the participating centres
Drug: Coagulation proteins
Documentation of factor VIII and factor IX products from the first exposure day onwards
Other Name: Factor VIII, Factor IX

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

Secondary Outcome Measures :
  1. 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 bleedings and joint damage

  2. Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor. [ Time Frame: From date first positive inhibitor titer 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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 2020 who have been or are to be treated in one of the participating centres

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02979119

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Contact: H. Marijke van den Berg, MD, PhD +31850299993
Contact: Ella van Hardeveld, MANP +31850299993

  Hide Study Locations
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Universitäts-Klinik für Kinder- und Jugendheilkunde Completed
Graz, Austria, A-8036
Medical University of Vienna - Department of Paediatrics Recruiting
Vienna, Austria
Contact: Christoph Male, MD, PhD         
Service of Pediatric Haematology University Hospital Leuven Recruiting
Leuven, Belgium, B-3000
Contact: Christel Van Geet, MD, PhD         
Division of Hematology/Oncology Hôpital St Justine Recruiting
Montréal, Canada, H3T 1C5
Contact: George Rivard, MD         
Division of Haematology/Oncology Hospital for Sick Children Recruiting
Toronto, Canada, M5G-1X8
Contact: Manuel Carcao, MD, PhD         
Department of Pediatrics Århus Kommunehospital Skejby Sygehus Recruiting
Aarhus, Denmark, DK-8200
Contact: Niels Clausen, MD         
Children's Hospital Helsinki University Hospital Recruiting
Helsinki, Finland, FIN-00029
Contact: Anne Mäkipernaa, MD, PhD         
Service Hématologique Centre Regional Traitement d'Hemophilie Bicetre Recruiting
Le Kremlin Bicêtre, France, F-94270
Contact: Anne Rafowicz, MD         
Service d'Hématologie Pédiatrique Hôpital Universitaire La Timone Recruiting
Marseille Cedex-05, France, F-13385
Contact: Hervé Chambost, MD, PhD         
Centre de traitement des hémophiles Hôpital Universitaire Purpan Recruiting
Toulouse, France, F-31052
Contact: Ségolène Claeyssens, MD         
Institut für Experimentelle Hämatologie und Transfusionsmedizin Universitätsklinikum Bonn Recruiting
Bonn, Germany, D-53127
Contact: Johannes Oldenburg, MD, PhD         
Klinik Bremen-Mitte Prof.-Hess-Kinderklinik Recruiting
Bremen, Germany, D 28177
Contact: Martina Bührlen, MD         
University Hospital Frankfurt & Goethe University - Clinical and Molecular Hemostasis, Department of Pediatrics Recruiting
Frankfurt am Main, Germany
Contact: Christoph Königs, MD, PhD         
Dr. v. Haunersches Kinderspital University of Munich Recruiting
Munich, Germany, D-80337
Contact: Karin Kurnik, MD         
Hämophilie Zentrum Rhein Main Recruiting
Mörfelden-Walldorf, Germany, D-64546
Contact: Carmen Escuriola, MD         
Haemophilia-Haemostasis Unit St. Sophia Children's Hospital Recruiting
Athens, Greece, GR-11527
Contact: Helen Platokouki, MD         
Dept of Paediatric Haematology Our Lady's Children's Hospital for Sick Children Crumlin Recruiting
Dublin, Ireland
Contact: Beatrice Nolan, MD         
The National Hemophilia Center Sheba Medical Center, Tel Hashomer Recruiting
Ramat Gan, Israel, 52621
Contact: Gili Kenet, MD         
Gaslini Hospital Recruiting
Genova, Italy, I-16184
Contact: Angelo Claudio Molinari, MD         
A. Bianchi Bonomi Hemophilia and Thrombosis Centre IRCCS Ca' Granda Ospedale Maggiore Policlinico Recruiting
Milano, Italy, 20122
Contact: Elena Santagostino, MD, PhD         
Van Creveld Kliniek University Medical Center Utrecht Recruiting
Utrecht, Netherlands, 3508 GA
Contact: Kathelijn Fischer, MD, PhD         
Oslo University Hospital Not yet recruiting
Oslo, Norway
Contact: Heidi Glosli, MD         
Hospital Santa Maria - Serviço de Imuno-hemoterapia Not yet recruiting
Lisbon, Portugal
Contact: Cristina Oliveira, MD         
Centro Hospitalar São João, S. Imuno-hemoterapia Recruiting
Porto, Portugal
Contact: Manuela Carvalho, MD         
Unitat Hemofilia Hospital Vall d'Hebron Recruiting
Barcelona, Spain, 08035
Contact: Carmen Altisent Roca, MD         
Unidad de Coagulopatías Hospital Universitario La Paz Recruiting
Madrid, Spain, 28046
Contact: Maria Theresa Alvarez Roman, MD         
Hospital General Unidad de Hemofilia 1 Sur Hospitales Universitarios Virgen del Rocio Completed
Seville, Spain, 41013
Unidad de Coagulopatias Congenitas Hospital Universitario la Fe Recruiting
Valencia, Spain, 46009
Contact: Ana Rosa Cid, MD         
Lund University Hospital Recruiting
Malmo, Sweden, S-20502
Contact: Rolf Ljung, MD, PhD         
Department of Pediatrics, Clinic of Coag. Disorders Karolinska Hospital Recruiting
Stockholm, Sweden, S-17176
Contact: Susanna Ranta, MD         
Hämophiliezentrum Recruiting
Wabern, Switzerland, CH_3084
Contact: Rainer Kobelt, MD         
United Kingdom
Birmingham Children's Hospital NHS Trust - Department of Haematology Recruiting
Birmingham, United Kingdom
Contact: Mike Williams, MD         
Royal Hospital for Sick Children Recruiting
Edinburgh, United Kingdom, EH9 1LF
Contact: Angela Thomas, MD, PhD         
Department of Haematology Royal Hospital for Sick Children Recruiting
Glasgow, United Kingdom, G 3885
Contact: Elizabeth Chalmers, MD, PhD         
Haemophila Center Great Ormond Street Hospital for Children Recruiting
London, United Kingdom, WC1N3JH
Contact: Ri Liesner, MD         
Sponsors and Collaborators
PedNet Haemophilia Research Foundation
Lund University Hospital
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Principal Investigator: Rolf Ljung, MD PhD Lund University Hospital, Malmo, Sweden
Study Director: H. Marijke Van den Berg, MD PhD University Medical Center Utrecht, The Netherlands

Additional Information:
Publications of Results:

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Marijke van den Berg, Director, PedNet Haemophilia Research Foundation Identifier: NCT02979119     History of Changes
Other Study ID Numbers: Version 5.1 June 2014
First Posted: December 1, 2016    Key Record Dates
Last Update Posted: November 6, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Marijke van den Berg, PedNet Haemophilia Research Foundation:
Children, ,
Long-term outcome
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Factor VIII