Back to Trials
NCT06610201Recruiting

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

Hemab ApS

Start Date

8/30/2024

Completion Date

12/1/2026

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)

Detailed Description

This is a prospective, screening study in participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with confirmed Type 1 VWD and associated bleeding symptoms will be enrolled. The study may also be opened to participants with Type 2 and Type 3 VWD with Sponsor approval. Up to a total of 200 participants may be enrolled in the study. The study includes screening, a baseline evaluation, and an approximately 4 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary entries and bleeding event treatments. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.

Eligibility Criteria

Age Range: 16 years to No maximum

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 Criteria: 1. Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies. 2. Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events. 3. Has a high-risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/prothrombin gene mutation, antithrombin \<50%, congenital protein C and protein S deficiency with levels \<50%. 4. Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding 5. Has other known severe bleeding disorder(s) other than VWD. 6. Planned major surgery during the study period. 7. Has other conditions that substantially increase the risk of thrombosis either individually or in combination, at the discretion of the Investigator, including but not limited to: significant family history; BMI \>30 and ≤38.5 kg/m² (moderately obese, adjusted for ethnicity and increased central adiposity); reduced mobility; active malignancy; major surgery within 6 weeks preceding Screening; or postpartum within 12 weeks preceding Screening. 8. Is pregnant or plans to become pregnant within the next 6 months following informed consent sign off. 9. Has clinically significant cardiovascular disease including, but not limited to: NYHA Class III or IV heart failure, coronary artery disease, uncontrolled arrythmia, moderate to severe valvular heart disease, peripheral vascular disease, and ischemic stroke. 10. Has other combinations of conditions that substantially increase the risk of cardiovascular events at the discretion of the Investigator including, but not limited to, smoking, uncontrolled hyperlipidemia, and uncontrolled hypertension. 11. Has any concurrent disease, treatment, medication (including but not limited to ongoing anticoagulation, antiplatelet therapy, or non-steroidal anti-inflammatory drugs or other drugs that affect hemostasis), condition, medication, or abnormality in clinical laboratory tests which may impact on the participant's bleeding symptoms or affect their ability to complete the study, in the Investigator's opinion. 12. Has received any investigational product within 30 days prior to Screening. If the participant was enrolled and dosed in Velora Pioneer (study HMB-002-102; NCT06754852), they must have completed their End of Study Visit.

Interventions

OTHER

Clinical outcomes of patients with VWD, Type 1

OTHER

Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

Von Willebrand Disease (VWD)Von Willebrand Disease (VWD), Type 1Von Willebrand Disease (VWD), Type 2Von Willebrand Disease (VWD), Type 3Von Willebrand Disease, Type 2AVon Willebrand Disease, Type 2MVon Willebrand Disease, Type 2N

Locations

Phoenix Children's Hospital

Phoenix, Arizona 85016

United States

Arkansas Children's Hospital

Little Rock, Arkansas 72202-3591

United States

Children's Hospital of Los Angeles

Los Angeles, California 90027

United States

University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center

Miami, Florida 33136

United States

Emory Children's Center

Atlanta, Georgia 30329

United States

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana 46260

United States

Tulane University School of Medicine

New Orleans, Louisiana 70112-2699

United States

University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders

Ann Arbor, Michigan 48109

United States

Mayo Clinic - Rochester

Rochester, Minnesota 55905

United States

Oregon Health & Science University

Portland, Oregon 97239-3098

United States

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania 15213

United States

The University of Texas Southwestern Medical Center

Dallas, Texas 75390

United States

Washington Institute For Coagulation (WIC)

Seattle, Washington 98101

United States

Fiona Stanley Hospital

Murdoch, Perth WA 6150

Australia

Royal Prince Alfred Hospital

Camperdown, Sydney NSW 2050

Australia

The Alfred Hospital

Melbourne, Victoria VIC 3004

Australia

Richmond Pharmacology

London, SE1 1YR

United Kingdom