The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
NCT ID: NCT03623295
Last Updated: 2025-04-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
304 participants
OBSERVATIONAL
2018-01-01
2021-10-01
Brief Summary
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The primary aim of this project is to analyze the association between factor VIII and factor IX levels and the bleeding phenotype. The secondary aim is to analyze potential differences in phenotype between hemophilia A and B.
The project is a multicentre observational cohort study. We will include 230 patients with moderate or mild hemophilia A or B (FVIII/FIX 0.02-0.35 IU/mL) who are 12 to 55 years old. The main cohort study consists of clinical data collection, one blood sample and an online questionnaire for patients. Data will be collected on the nature and duration of all bleeding episodes, disease and treatment characteristics, physical activity level and musculoskeletal status. One blood withdrawal will be performed for centralized laboratory assays for FVIII or FIX levels (both one-stage and chromogenic assays) and genetic analysis for the most prevalent prothrombotic mutations. The online questionnaire for patients focuses on bleeds experienced in the past.
A subset of 50 patients aged 24 years or older with mild and moderate hemophilia A will be investigated in more detail by longitudinal data collection including analysis of physical joint status, MRI imaging of joints and biomarkers for joint damage. This longitudinal observation will consist of two time points that lie two years apart, allowing us to identify any changes that occur over the observed time period with respect to joint status.
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Cohort study population
For the main cohort study, we will include 230 patients with moderate or mild hemophilia A or B.
Blood sample
Blood withdrawal.
Questionnaire
Online questionnaire about the bleeds that patients experienced in the past.
Sub study population
A subset of 50 patients of the cohort study population will be investigated in more detail by longitudinal data collection.
Blood sample
Blood withdrawal.
MRI-imaging
MRI imaging of joints.
Physical examination
Physical examination of joint status.
Interventions
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Blood sample
Blood withdrawal.
Questionnaire
Online questionnaire about the bleeds that patients experienced in the past.
MRI-imaging
MRI imaging of joints.
Physical examination
Physical examination of joint status.
Eligibility Criteria
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Inclusion Criteria
* Age from 12 up to and including 55 years
Exclusion Criteria
* Participation in another trial with an investigational product
* Comorbidity affecting the musculoskeletal status
* Clinically relevant inhibitor status at present or in the past
* Hemophilia B Leyden
* Use of anticoagulants
12 Years
55 Years
MALE
No
Sponsors
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Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
OTHER
Responsible Party
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Karin Fijnvandraat
Professor of Pediatric Hematology
Principal Investigators
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Karin Fijnvandraat
Role: PRINCIPAL_INVESTIGATOR
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Locations
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Royal Adelaide Hospital
Adelaide, , Australia
Medical University of Vienna
Vienna, , Austria
Multicentre: Leuven, Brussels
Multiple Locations, , Belgium
Multicentre: Vancouver, Toronto, Hamilton
Multiple Locations, , Canada
Helsinki University Central Hospital
Helsinki, , Finland
Multicentre: Bonn, Berlin, Frankfurt, München, Hamburg
Multiple Locations, , Germany
Multicentre: Florence, Rome, Parma, Milan, Turin
Multiple Locations, , Italy
Academic Medical Center
Amsterdam, , Netherlands
University Medical Center Groningen
Groningen, , Netherlands
Leiden University Medical Center
Leiden, , Netherlands
Maastricht University Medical Center
Maastricht, , Netherlands
Radboud University Medical Center
Nijmegen, , Netherlands
Erasmus Medical Center
Rotterdam, , Netherlands
Utrecht University Medical Center
Utrecht, , Netherlands
Máxima Medical Center
Veldhoven, , Netherlands
Multicentre: Valencia, Madrid, Barcelona
Multiple Locations, , Spain
Multicentre: Manchester, London, Liverpool, Glasgow, Cardiff, Sheffield
Multiple Locations, , United Kingdom
Countries
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Other Identifiers
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NL61564.018.17
Identifier Type: -
Identifier Source: org_study_id
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