Severe hemophilia A (< 1% FVIII:C) MedDRA version: 9.1 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors
Conditions
Interventions
Pharmaceutical Form: Powder and solvent for solution for injection
Other descriptive name: HUMAN COAGULATION FACTOR VIII
Sponsors
Bayer Healthcare AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Test subjects (for MRI quality assessment) ----------------------------------------------------- • Males aged 18 - 35 years • Severe hemophilia A ( 12 bleeds/year in the last 5 years. • Written informed consent by subject and parent/legal representative, if =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Test subjects (for MRI quality assessment) ----------------------------------------------------- • Individuals for whom high-magnetic exposure is contraindicated • Individuals who, in the opinion of the physician, would not be able to meet the requirements of the MRI technique Investigation subjects only (subjects participating in the full investigation) --------------------------------------------------------------------------------------------- • Individuals with other coagulopathies (e.g., von Willebrand disease) • HIV seropositive subjects • Individuals for whom the most clinically severe joint is not one of the 4 index joints (ankle, knee) • HCV seropositive individuals who underwent interferon therapy during the last 12 months • Individuals for whom high-magnetic exposure is contraindicated (see section 7.1) • Individuals who, in the opinion of the physician or parent/guardian, would not be able to meet the requirements of the MRI technique • Synovectomy performed within the six months prior to investigation enrollment or orthopedic surgery planned to be performed within the investigation period • Joint replacement • For the on-demand treatment group, any period greater than 8 consecutive months having received = 50 IU per kg per week Factor VIII for the prevention of bleeding
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate bone and cartilage damage at different ages using MRI in four index joints of subjects with severe hemophilia A in relation to previous treatment schedule of primary or secondary prophylaxis with start at different ages, comparing to subjects who received only on-demand therapy;Secondary Objective: To compare historical bleeding frequency between treatment groups. Other objectives: evaluating bone and cartilage damage in index joints by subject age, correlating MRI findings with clinical joint function evaluation, comparing subject-reported quality-of-life between the treatment groups, and determining the feasibility of imaging four index joints during a single MRI procedure in subjects of various age groups.;Primary end point(s): Maximum ankle MRI score | — |
Countries
Germany, Greece, Italy, Spain, United Kingdom
Outcome results
None listed