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Recruiting NCT06610201

NCT06610201 A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

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Clinical Trial Summary
NCT ID NCT06610201
Status Recruiting
Phase
Sponsor Hemab ApS
Condition Von Willebrand Disease (VWD)
Study Type OBSERVATIONAL
Enrollment 200 participants
Start Date 2024-08-30
Primary Completion 2026-12

Trial Parameters

Condition Von Willebrand Disease (VWD)
Sponsor Hemab ApS
Study Type OBSERVATIONAL
Phase N/A
Enrollment 200
Sex ALL
Min Age 16 Years
Max Age N/A
Start Date 2024-08-30
Completion 2026-12
Interventions
Clinical outcomes of patients with VWD, Type 1Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3

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Brief Summary

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)

Eligibility Criteria

Inclusion Criteria: 1. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations. 2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions. 3. Is 16 years and \< 70 years at the time of screening. 4. Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) \<38.5 kg/m\*2. 5. Has Von Willebrand Disease: Type 1 VWD (including Type 1C VWD) or Type 2A VWD. All participants must have: Documented lab results confirming their diagnosis consistent with ISTH/ASH diagnostic guidelines; VWF Activity ≤30 IU/dL and FVIII activity ≤70 IU/dL during Screening. 6. Has symptomatic disease as defined by a history of bruising or bleeding events, with an expected minimum of 3 bleeding episodes (including heavy menstrual bleeding) per year that require treatment to control bleeding symptoms, and/or has recurrent and ongoing episodes of heavy menstrual bleeding at the time of enrollment. Exclusion Crit

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