Evaluate whether FVIII/VWF concentrates successfully induce immune tolerance in patients who have already experienced and failed immune tolerance induction with VWF-free FVIII concentrates.
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Blood and lymphatic system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
abolition of the inhibitor to < 0.6 BU within 33 months of ITI with a factor
VIII recovery >= 66% and half-life >= 6 hrs
Secondary outcome
Maintenance of immune tolerance
Time to achieve success
Safety
Compliance to treatment
Cost of care
Background summary
Patients with hemophilia A are have a lack of clottingfactor VIII and therefore
suffer from a bleeding disorder. These patients are treated by intravenous
administration of the missing clotting factor. Regularly antibodies are formed
in these patients as a consequence of this treatment. These antibodies are
directed against the administered FVIII and neutralise them. Hence, the
treatment is severly hampered in these patients. It is therefore of great
importance that if these so called inhibitors are formed, to eradicate them. A
possibility to do so is immune tolerance induction therapy. In this way,
patients are immunized against their medication, by exposing them extensively
to FVIII. Regularly this is done with the same product that initiated the
inhibitor development. Sometimes however, this is not successful. There are
indications the immune tolerance induction with a FVIII-product that also
contains the clottingfactor "Von Willebrand Factor (VWF)" in these patients can
be a succesful immune tolerance induction treatment. This is investigated in
this study.
This study does neither imply a new form of therapy nor a new product for it,
but is aimed to inventorise worldwide the amount of succesful immune tolerance
induction therapies and the time it takes to become succesful.
Study objective
Evaluate whether FVIII/VWF concentrates successfully induce immune tolerance in
patients who have already experienced and failed immune tolerance induction
with VWF-free FVIII concentrates.
Study design
This is an observational study in which patients that undergo an immune
tolerance induction treatment with a VVWF-contacining FVIII-product are
followed in time
Intervention
The intervention is the infusion of Haemate P (200 IU/kg daily)
Study burden and risks
The burden for the patient will be the time to complete the questionaires. This
will be done at start and stop of the study, as well as every 6 months. This
will take approximately 10 minutes every time. This does not apply any risk.
Furthermore, per visit some extra blood will be drawn. This will probably not
take much more time, or increase the risk since blood will be drawn at that
moment for his treatment as well.
Heidelberglaan 100
3584 CX Utrecht
NL
Heidelberglaan 100
3584 CX Utrecht
NL
Listed location countries
Age
Inclusion criteria
- severe hemophilia A (FVIII<1%)
- male patients, 18 years or older;
- high responders (peak inhibitor levels > 5 BU);
- any inhibitor level at study enrollment
- with ability and willingness to participate to the study;
- previous ITI course of at least 9 months with a VWF-free FVIII concentrate (recombinant FVIII and/or monoclonally purified FVIII) at any dosage (patients who initially succeeded to clear the inhibitor and then relapsed can be included)
Exclusion criteria
- Concomitant systemic treatment with drugs with immunosuppressive side effects
- Concomitant experimental treatment
- Previous history of myocardial infarction and/or cerebral stroke
- High risk of cardiovascular, cerebrovascular or other thromboembolic events as deemed by the treating clinician
Design
Recruitment
Medical products/devices used
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 |
|---|---|
| EudraCT | EUCTR2008-007019-33-NL |
| ClinicalTrials.gov | NCT01051076 |
| CCMO | NL36352.041.11 |