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

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ClinicalTrials.gov Identifier: NCT02979119
Recruitment Status : Recruiting
First Posted : December 1, 2016
Last Update Posted : November 6, 2017
Sponsor:
Collaborator:
Lund University Hospital
Information provided by (Responsible Party):
Marijke van den Berg, PedNet Haemophilia Research Foundation

Brief Summary:

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.


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

Detailed Description:

Design: Prospective observational cohort

Population:

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

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 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.



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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
Criteria

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 ClinicalTrials.gov identifier (NCT number): NCT02979119


Contacts
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Contact: H. Marijke van den Berg, MD, PhD +31850299993 h.marijke.vandenberg@pednet.eu
Contact: Ella van Hardeveld, MANP +31850299993 e.m.vanhardeveld@pednet.eu

  Show 35 Study Locations
Sponsors and Collaborators
PedNet Haemophilia Research Foundation
Lund University Hospital
Investigators
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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 ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Marijke van den Berg, Director, PedNet Haemophilia Research Foundation
ClinicalTrials.gov 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:
Hemophilia
Children, ,
Inhibitors,
Prophylaxis
Long-term outcome
Congenital

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
Coagulants