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

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

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON55705

Enrollment

190

Registered

2017-09-13

Start date

2018-01-16

Completion date

Unknown

Last updated

2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

bleeding disorder Hemophilia A and B

Interventions

None listed

Eligibility

Age

12 Years to 99 Years

Inclusion criteria

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

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

Design outcomes

Primary

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

Secondary

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

Countries

Netherlands

Outcome results