Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens

Study Overview
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Brief Summary
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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.

Official Title
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A Diagnostic Interventional, Controlled, Cross-sectional Evaluation of Joint Status Using Magnetic Resonance Imaging in Subjects With Severe Hemophilia A Treated With Primary Prophylaxis, Secondary Prophylaxis, or On-demand Therapy

Conditions
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Hemophilia A

Intervention / Treatment
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* Procedure: No Drug
* Procedure: No Drug
* Procedure: No Drug
* Procedure: No Drug
* Procedure: No Drug


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 35 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br> | Procedure: No Drug<br>* Patients have received primary FVIII prophylaxis (started < 2 years of age)<br>|
| Experimental: Arm 2<br> | Procedure: No Drug<br>* Patients have received secondary FVIII prophylaxis (started 2 - < 6 years of age)<br>|
| Experimental: Arm 3<br> | Procedure: No Drug<br>* Patients have received secondary FVIII prophylaxis (started 6 - < 12 years of age)<br>|
| Experimental: Arm 4<br> | Procedure: No Drug<br>* Patients have received secondary VIII prophylaxis(started 12 - 18 years of age)<br>|
| Experimental: Arm 5<br> | Procedure: No Drug<br>* Patients have received on-demand therapy<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum ankle MRI score |  | no timeframe (single visit, snapshot of the actual joint status) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia A, Joint Status, Bleeds, Magnetic Resonce Imaging