Recruiting

Observational Study

Sponsor:

Hemab ApS

Code:

NCT06610201

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

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

Clinical outcomes of patients with VWD, Type 1

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

Study Details

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

Study ID

NCT06610201

Start date

Aug 30, 2024

Status verified date

Apr, 2026

Completion date

Dec, 2026

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Arms

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

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

Interventions

Clinical outcomes of patients with VWD, Type 1

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

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

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.

Primary outcome measure

  • Annualized bleeding event rate [ Time Frame: 4.5 to 12.5 months ]
  • Annualized treated bleeding rate [ Time Frame: 4.5 to 12.5 months ]
  • Annualized heavy menstrual bleed rate [ Time Frame: 4.5 to 12.5 months ]
  • Number of overnight admissions [ Time Frame: 4.5 to 12.5 months ]

Central Contacts and Locations

Central contacts

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Contacts

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202-3591

Contacts

Children's Hospital of Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center

Recruiting

Miami, Florida, United States, 33136

Contacts

Fernando Corrales-Medina

305-243-8652ffc5@med.miami.edu

Emory Children's Center

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center

Recruiting

Indianapolis, Indiana, United States, 46260

Contacts

Tulane University School of Medicine

Recruiting

New Orleans, Louisiana, United States, 70112-2699

Contacts

University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Mayo Clinic - Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Oregon Health & Science University

Recruiting

Portland, Oregon, United States, 97239-3098

Contacts

Hemophilia Center of Western Pennsylvania

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

Contacts

The University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

Contacts

Washington Institute For Coagulation (WIC)

Recruiting

Seattle, Washington, United States, 98101

Contacts

More Information

Sponsor

Hemab ApS

Last update posted

Apr 21, 2026

Last verified

Apr, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Hemab ApS on 2026-04-21.