Brief Title
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Official Title
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Brief Summary
There are large inter-individual differences in the bleeding pattern of patients with
moderate or mild hemophilia. The major determinant of bleeding phenotype is the level of
coagulant factor VIII or IX. In hemophilia A, studies addressing the association between
factor VIII level and the clinical bleeding pattern yield conflicting results. In hemophilia
B such studies have not yet been performed.
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 500 patients with
moderate or mild hemophilia A (FVIII 0.02-0.35 IU/mL) and 500 patients with moderate or mild
hemophilia B (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 200 patients aged 24 years or older (100 with moderate or mild hemophilia A and
100 with moderate or mild hemophilia B) 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.
Study Type
Observational
Primary Outcome
Bleeding phenotype
Condition
Hemophilia
Intervention
Blood sample
Study Arms / Comparison Groups
Cohort study population
Description: For the main cohort study, we will include 500 patients with moderate or mild hemophilia A and 500 patients with moderate or mild hemophilia B.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
1000
Start Date
January 1, 2018
Completion Date
April 1, 2022
Primary Completion Date
April 1, 2020
Eligibility Criteria
Inclusion Criteria:
- Moderate or mild hemophilia A (FVIII:C 0.02-0.35 IU/mL) or hemophilia B (FIX:C
0.02-0.35 IU/mL)
- Age from 12 up to and including 55 years
Exclusion Criteria:
- Other clotting disorder
- 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
Gender
Male
Ages
12 Years - 55 Years
Accepts Healthy Volunteers
No
Contacts
Karin Fijnvandraat, +31205668668, [email protected]
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03623295
Organization ID
NL61564.018.17
Responsible Party
Principal Investigator
Study Sponsor
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Study Sponsor
Karin Fijnvandraat, Principal Investigator, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Verification Date
August 2018