The European Paediatric Network for Haemophilia Management ( PedNet Registry)

  • STATUS
    Recruiting
  • End date
    Dec 26, 2029
  • participants needed
    4000
  • sponsor
    PedNet Haemophilia Research Foundation
Updated on 26 January 2021
hemophilia
antihemophilic factor

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.

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 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.

Details
Condition Hemophilia A, Hemophilia, Factor IX Deficiency, Hemophilia, christmas disease, hema
Treatment Coagulation proteins
Clinical Study IdentifierNCT02979119
SponsorPedNet Haemophilia Research Foundation
Last Modified on26 January 2021

Eligibility

Yes No Not Sure

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
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How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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