The Dynamic interplay between bleeding phenotype and baseline factor level in moderate and mild hemophilia A and B

Completed

• Conditions: bleeding disorder; Hemophilia A and B; 10064477

• Registration Number: NL-OMON55705
• Lead Sponsor: Academisch Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 190

Inclusion Criteria

- Moderate or mild hemophilia A (FVIII:C 0.02*0.35 IU/mL) or hemophilia B
(FIX:0.02*0.35 IU/ml)
- 12-55 years old
Extra for substudy:
- 24 years of age or older

Exclusion Criteria

- Other clotting disorder
- Participation in another trial with an investigational product
- Comorbidity affecting the musculoskeletal status
- Clinically relevant inhibitor status at present or in the past
- Hemophilia B Leyden
- Use of anticoagulants

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Part 1 Cohort study<br /><br>Data will be collected on demographics, physical activity level,<br /><br>musculoskeletal status, genotype, measured factor levels (both one stage and<br /><br>chromogenic), the nature and duration of all bleeding episodes, type of<br /><br>treatment and treatment response. Central laboratory assays for FVIII or FIX<br /><br>levels will be performed.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Part 2: Detailed substudy<br /><br>A subset of 50 patients aged 24 years or older (50 with moderate/mild<br /><br>hemophilia A) will be investigated in more detail by data collection including<br /><br>analysis of a) physical joint status and function; b) 3Tesla MRI imaging and c)<br /><br>biomarkers for joint damage. </p><br>