Hemophilia A
Conditions
Keywords
Hemophilia A, Joint Status, Bleeds, Magnetic Resonce Imaging
Brief summary
The joint status (knees, ankles) of patients suffering from severe Hemophilia A (too little blood clotting factor VIII in blood) is evaluated in a single magnetic resonance imaging session. No study medication is given.
Interventions
PROCEDURENo Drug
Patients have received primary FVIII prophylaxis (started \< 2 years of age)
Sponsors
Bayer
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
DIAGNOSTIC
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
12 Years to 35 Years
Healthy volunteers
No
Inclusion criteria
* Males aged 12 - 35 years * Severe hemophilia A ( \< 1 % FVIII:C) * No history of Factor VIII inhibitory antibody * For prophylaxis groups, having received at least two prophylactic infusions per week for 45 weeks per year for the prevention of bleeding without relevant interruption and continuing until the present. * Complete documentation of joints bleeds and their locations prior to start of prophylaxis, * Bleeding history and/or treatments received during the last 5 years documented in the subjects medical records. * For the on-demand subjects \> 12 bleeds/year in the last 5 years. * Written informed consent by subject and parent/legal representative, if \< 18 years
Exclusion criteria
* 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) * 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 |
|---|---|
| Maximum ankle MRI score | no timeframe (single visit, snapshot of the actual joint status) |
Secondary
| Measure | Time frame |
|---|---|
| Total MRI score of the maximum index joint | no timeframe (single visit, snapshot of the actual joint status) |
| Number of bleeds in each index joint over previous 5 years | no timeframe (medical history status) |
| Number of total joint bleeds and their locations over previous 5 years | no timeframe (medical history status) |
| Physical joint score (Gilbert Score) | no timeframe (single visit, snapshot of the actual joint status) |
Countries
Germany, Greece, Italy, Spain, Sweden, United Kingdom
Outcome results
None listed