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

NCT06610201 | Recruiting

• 1 other identifier: HMB-002-101_SCR
• Study Type: observational
• Target: 200
• Locations: 3 countries
• Sites: 17

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)

Conditions

Von Willebrand Disease (VWD); Von Willebrand Disease (VWD), Type 1; Von Willebrand Disease (VWD), Type 2; Von Willebrand Disease (VWD), Type 3; Von Willebrand Disease, Type 2A; Von Willebrand Disease, Type 2M; Von Willebrand Disease, Type 2N

Keywords

Von Willebrand Disease (VWD); Prospective Study; Type 1 VWD; Type 2 VWD; Type 3 VWD; Prophylaxis; Von Willebrand Factor (VWF)

Outcome Measures

Primary Outcomes (4)

• Annualized bleeding event rate

  4.5 to 12.5 months

• Annualized treated bleeding rate

  4.5 to 12.5 months

• Annualized heavy menstrual bleed rate

  4.5 to 12.5 months

• Number of overnight admissions

  Hospitalization monitoring of bleeding events.

  4.5 to 12.5 months

Secondary Outcomes (6)

• Prophylactic and on demand treatment

  4.5 to 12.5 months

• Iron status

  4.5 to 12.5 months

• Patient-Reported Outcomes Measurement Information System (PROMIS)-29

  4.5 to 12.5 months

• Menstrual Bleeding Questionnaire (MBQ)

  4.5 to 12.5 months

• Epistaxis Severity Score (ESS)

  4.5 to 12.5 months

Study Arms (2)

VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL)

Other: Clinical outcomes of patients with VWD, Type 1

VWD Type 2A, Type 2M, Type 2N, or Type 3

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

Interventions

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

Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2A, Type 2M, Type 2N and Type 3.

VWD Type 2A, Type 2M, Type 2N, or Type 3

Clinical outcomes of patients with VWD, Type 1 OTHER

Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1

VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL)

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants ≥16 years of age with VWD.

You may qualify if:

• Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
• Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
• Is 16 years and \< 70 years at the time of screening.
• Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) \<38.5 kg/m\*2.
• 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.
• 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.

You may not qualify if:

• Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies.
• Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
• 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%.
• Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding
• Has other known severe bleeding disorder(s) other than VWD.
• Planned major surgery during the study period.
• 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.
• Is pregnant or plans to become pregnant within the next 6 months following informed consent sign off.
• 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.
• 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.
• 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.
• 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.

Sponsors & Collaborators

• Hemab ApS: lead

Study Sites (17)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

Arkansas Children's Hospital

Little Rock, Arkansas, 72202-3591, United States

RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

RECRUITING

Emory Children's Center

Atlanta, Georgia, 30329, United States

RECRUITING

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, 46260, United States

RECRUITING

Tulane University School of Medicine

New Orleans, Louisiana, 70112-2699, United States

RECRUITING

University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders

Ann Arbor, Michigan, 48109, United States

RECRUITING

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239-3098, United States

RECRUITING

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

The University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Washington Institute For Coagulation (WIC)

Seattle, Washington, 98101, United States

RECRUITING

Fiona Stanley Hospital

Murdoch, Perth, WA 6150, Australia

RECRUITING

Royal Prince Alfred Hospital

Camperdown, Sydney, NSW 2050, Australia

RECRUITING

The Alfred Hospital

Melbourne, Victoria, VIC 3004, Australia

RECRUITING

Richmond Pharmacology

London, SE1 1YR, United Kingdom

RECRUITING

MeSH Terms

Conditions

von Willebrand Diseases; von Willebrand Disease, Type 2; von Willebrand Disease, Type 3

Interventions

Sarcoglycans

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Dystrophin-Associated Proteins; Muscle Proteins; Contractile Proteins; Proteins; Amino Acids, Peptides, and Proteins; Membrane Proteins; Membrane Glycoproteins

Study Officials

• VP of Clinical Research

  Hemab ApS

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials (USA; UK)

CONTACT

+1 888 493 8148; 080 8304 6409; Clinicaltrials@hemab.com

Clinical Trials (Australia)

CONTACT

+611800875216; Clinicaltrials@hemab.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2024
• First Posted: September 24, 2024
• Study Start: August 30, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (13); AU (3); GB (1)