Genetic Influence on the distribution of Factor VIII in the patient`s blood with bleeding disorder

Conditions: Haemophilia A under prophylactic Factor VIII Substitution
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2012-001669-34-AT

Lead Sponsor: Med. Univ. Wien, Univ. Klinik f. Kinder- u. Jugendheilkunde, Klin. Abt.f. Pädiatrische Kardiologie

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 100

Inclusion Criteria

This analysis will be performed on a study cohort of approximately 100 severe hemophilia A boys.The boys will be aged between 6 and 18 years and will have severe FVIII deficiency (baseline levels <1%). Only patients using recombinant FVIII products will be included. Following the acquisition of written informed consent the following investigations will be performed on the study participants
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The analysis of factor VIII clearance must be performed at a time when there is no bleeding (bleeding will use up the factor VIII and therefore shorten its circulation time). In addition, there should be no factor VIII antibodies (factor VIII inhibitors) present at the time of testing.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The theme of this study is to characterize the genetic factors that influence the clearence of F VIII in patients with severe hemophilia A.;Secondary Objective: ;Primary end point(s): The major issue that we may face is an influence of age on FVIII PK. This effect is well documented, but is largely present in very young children (<5 years) and is minimized by employing an optimal blood sampling schedule and inclusion of patients between 6 and 18 years.;Timepoint(s) of evaluation of this end point: 1 year.

Secondary Outcome Measures