The aim of this study is to characterize the early immune response against F VIII concentrates in patients with severe hemophilia A before and during inhibitor development.
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
- FVIII-specific memory B-cells.
- T-cell characteristics.
- Cytokine/interleukin secretion.
Secondary outcome
- F VIII genotype
- Variation in immune response of certain polymorphisms in IL10, TNFA, CTLA-4
and MHC class II genes
- Copy number variations of FCGR genes
Background summary
The development of inhibiting antibodies (inhibitors) represents a major
challenge in the management of hemophilia A. However, a number of important
immunological questions remain unanswered. Insight in the early immune response
against exogenous F VIII may ultimately help reduce the risk by preventive
measures.
Study objective
The aim of this study is to characterize the early immune response against F
VIII concentrates in patients with severe hemophilia A before and during
inhibitor development.
Study design
We will perform a prospective study of 20 previously untreated severe
hemophilia A patients.
During follow-up of the first 50 exposure days we will obtain clinical relevant
data. Blood will be collected prior to each F VIII administration to obtain
PBMc for analysis of DNA, T-cell epitopes and F VIII specific B-cell
investigation.
Study burden and risks
The only burden of this study may be the drawing of twenty blood samples
combined to the first twenty intravenous administrations of F VIII
concentrates. Separate venipuncture for the study will be avoided. The total
volume of blood collected will not exceed 2*% of the total blood volume
expected for a person*s age and weight at any visit.
This burden is in our opinion in proportion to the potential value of the
study.
The results of the study may be relevant and beneficial to the patients and
their families as well as their treating physicians.
Meibergdreef 9
1105 AZ Amsterdam
NL
Meibergdreef 9
1105 AZ Amsterdam
NL
Listed location countries
Age
Inclusion criteria
Severe hemophilia A (factor VIII:C <2%)
Previously untreated Patient
Minimally treated patient (< 5 exposures to factor VIII concentrate)
Exclusion criteria
Mild/moderate hemophilia A (factor VIII:C > 2%)
Previously treated patient (>5 exposures to factor VIII concetrate)
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 | NL28069.018.09 |