The primary aim of this project is to analyze the association between factor VIII and factor IX plasma levels and the bleeding phenotype.
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Part 1 Cohort study
Data will be collected on demographics, physical activity level,
musculoskeletal status, genotype, measured factor levels (both one stage and
chromogenic), the nature and duration of all bleeding episodes, type of
treatment and treatment response. Central laboratory assays for FVIII or FIX
levels will be performed.
Secondary outcome
Part 2: Detailed substudy
A subset of 50 patients aged 24 years or older (50 with moderate/mild
hemophilia A) will be investigated in more detail by data collection including
analysis of a) physical joint status and function; b) 3Tesla MRI imaging and c)
biomarkers for joint damage.
Background 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 FVIII or FIX plasma level. In hemophilia A, studies addressing
the association between FVIII plasma level and the clinical bleeding phenotype
yield conflicting results. In hemophilia B such studies have not yet been
performed.
Study objective
The primary aim of this project is to analyze the association between factor
VIII and factor IX plasma levels and the bleeding phenotype.
Study design
Observational cohort study
Study burden and risks
For part 1, Cohort study, the burden and risks of participation is the
withdrawal of one blood sample for central measurement. All data will be
collected from the medical files.
For part 2, Detailed substudy, the burden and risks of participation consist of
a physical examination addressing the joint status and function, MRI imaging of
the ankle and knee joint and withdrawal of blood samples.
Flemingweg 18
Alphen aan de Rijn 2408 AV
NL
Flemingweg 18
Alphen aan de Rijn 2408 AV
NL
Listed location countries
Age
Inclusion criteria
- Moderate or mild hemophilia A (FVIII:C 0.02*0.35 IU/mL) or hemophilia B
(FIX:0.02*0.35 IU/ml)
- 12-55 years old
Extra for substudy:
- 24 years of age or older
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
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL61564.018.17 |