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

Last updated: October 17, 2024
Sponsor: Hemab ApS
Overall Status: Active - Recruiting

Phase

N/A

Condition

Von Willebrand Disease

Treatment

Clinical outcomes of patients with VWD, Type 1 (less than 40 IU per dL)

Clinical outcomes of patients with VWD, Type 1 (less than 30 IU per dL)

Clinical outcomes of patients with VWD, Type 1 (less than 50 IU per dL)

Clinical Study ID

NCT06610201
HMB-002-101_SCR
  • Ages > 16
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Has the ability to provide informed consent to participate in the study, inaccordance with the International Council for Harmonisation (ICH) Good ClinicalPractice (GCP) Guideline E6 (R2) (2016) and applicable national and internationalregulations, before any protocol directed interventions are carried out.

  2. Has an understanding, ability, and willingness to comply with Study procedures andrestrictions.

  3. Is 16 years or older at the time of screening.

  4. First approximately 50 participants: congenital Type 1 VWD with a residual VWFantigen and/or activity less than 40 IU/dL (40%) Next approximately 50 participants: Type 1 VWD with residual VWF antigen andactivity less than 50 IU/dL (50%) After the first approximately 100 participants: congenital Type 1, Type 2A, Type 2M,Type 2N, or Type 3 VWD Note: Participants may be enrolled if they have documented laboratory results forVWF antigen and activity within their medical records confirming their diagnosis (consistent with International Society on Thrombosis and Hemostasis [ISTH]/AmericanSociety of Hematology [ASH] diagnostic guidelines or British Society for Haematology [BSH]/ United Kingdom Haemophilia Centre Doctors; Organisation [UKHCDO] diagnosticguidelines). For Type 1 VWD in the first part of the study, participants also needto have a documented laboratory result within the last 2 years showing a VWF antigenand/or activity level less than 40 IU/dL. Participants meeting eligibility criteriabased on documented results will proceed with having samples collected at Screeningto further characterize their antigen and activity levels. These may be repeated upto 2 further times if the results are not consistent with their documented diagnosisand levels are greater than 40 IU/dL. If participants do not have confirmed results in their medical records as describedabove, the screening assessment may be repeated up to 2 further times to establishbaseline levels for inclusion.

  5. Has symptomatic disease as defined by a history of bruising or bleeding events, andtypically experiencing bleeding symptoms every month.

Exclusion

Exclusion Criteria:

  1. Has a personal history of venous or arterial thrombosis or thromboembolic disease,except for catheter-associated, superficial vein thrombosis events.

  2. Has a significant family history of unprovoked thromboembolic events in first degreerelatives.

  3. Has a congenital or acquired bleeding disorder other than VWD.

  4. Has planned major surgery within the next 6 months.

  5. Is pregnant or plans to become pregnant within the next 6 months.

  6. Has any concurrent disease, treatment (including ongoing anticoagulation,antiplatelet, or non-steroidal anti-inflammatory drugs), condition, medication, orabnormality in clinical laboratory tests which may impact on the participant'sbleeding symptoms or affect their ability to complete the study, in theInvestigator's opinion.

  7. Has received any investigational product within 30 days prior to screening.

Study Design

Total Participants: 200
Treatment Group(s): 4
Primary Treatment: Clinical outcomes of patients with VWD, Type 1 (less than 40 IU per dL)
Phase:
Study Start date:
August 30, 2024
Estimated Completion Date:
February 28, 2026

Study Description

This is a prospective, screening study in a minimum of 100 participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with Type 1 VWD and a residual Von Willebrand Factor (VWF) antigen and/or activity of less than 40 IU per dL will be prioritized for recruitment. After approximately 50 participants with a residual VWF antigen and/or activity of less than 40 IU per dL have been included, participants with Type 1 VWD diagnosis and a residual VWF antigen and activity of less than 50 IU per dL may be screened for inclusion. Following enrollment of 100 participants with Type 1 VWD, the study may be opened to participants with Type 2 and Type 3 VWD.

The study includes screening, a Baseline evaluation, and a 4-6 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary and bleeding event treatments), and clinic visits every 12 weeks for laboratory draws. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.

Connect with a study center

  • Richmond Pharmacology

    London, SE1 1YR
    United Kingdom

    Active - Recruiting

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