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 : September 29, 2020
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|
|Factor VIII Deficiency Factor IX Deficiency|
Design: Multicenter Prospective Observational Birth Cohort Study
Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and 1-1-2030.
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.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||4000 participants|
|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 2029|
|Estimated Study Completion Date :||December 2029|
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 2009 who have been or are to be treated with coagulation proteins in one of the participating centres
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 2009 until January 1st 2020 who have been or are to be treated with coagulation proteins in one of the participating centres
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 January 1st 2030 who have been or are to be treated with coagulation proteins in one of the participating centres
- 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
- 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
- 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.
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
|Contact: H. Marijke van den Berg, MD, PhDemail@example.com|
|Contact: Ella van Hardeveld, MANPfirstname.lastname@example.org|
|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|