The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorPedNet Haemophilia Research Foundation
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
Patients referred because of an inhibitor*
Informed consent not obtained
Trial 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
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
4,000 Participants
are grouped into 4 trial groupsGroup A: Cohort I
Group B: Cohort II
Group C: Cohort III
Group D: Cohort IV
Trial outcomes
Primary outcomes
1
Number of patients with antibody development to exogenous clotting factors
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
Time frame
Until patient reaches age of 18Secondary outcomes
1
Long term outcome of haemophilia on joint status using the Hemophilia Joint Health Score (HJHS) and MRI techniques.
Effect of different prophylactic regimen on bleeding and joint damage
Time frame
From diagnose every 5 years until patient reaches age of 182
Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor.
Effect of different ITI therapies on bleeding and joint damage. Joint damage is assessed using the HJHS and MRI.
Time frame
From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov