Understanding bleeding and treatment in Von Willebrand Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to gather information on bleeding events, quality of life, and the social and clinical impacts of bleeds in individuals with Von Willebrand Disease (VWD). It will enroll at least 100 participants, primarily focusing on those with Type 1 VWD and low residual Von Willebrand Factor (VWF) levels. Participants will undergo a baseline evaluation followed by a 4-6 month observation period, including telemedicine check-ins and clinic visits for monitoring. The data collected will help establish baseline bleeding and treatment rates, serving as a comparator for future clinical studies.

Why it matters

Who can join: the full rules

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.

Where it's running

• Phoenix Children's Hospital
  Phoenix, Arizona, United States Recruiting
• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• Children's Hospital of Los Angeles
  Los Angeles, California, United States Recruiting
• University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
  Miami, Florida, United States Recruiting
• Emory Children's Center
  Atlanta, Georgia, United States Recruiting
• Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
  Indianapolis, Indiana, United States Recruiting

Study contacts

• Study coordinator: Clinical Trials (USA; UK)
• Email: Clinicaltrials@hemab.com
• Phone: +1 888 493 8148; 080 8304 6409

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Assessing HMB-002 for treating Von Willebrand Disease

  This study is testing a new treatment called HMB-002 to see if it is safe and effective for adults with Type 1 Von Willebrand Disease.

  Recruiting at 6 sites Phase 1/2 Hemab ApS · Phoenix, Arizona and 24 other locations · NCT06754852

• SR604 in Von Willebrand Disease: a Phase II study to see if it helps with bleeding

  This Phase 2 trial tests SR604 in adults with von Willebrand disease to see if it can improve bleeding control and how the drug behaves in the body.

  Recruiting at 9 sites Phase 2 Shanghai RAAS Blood Products Co., Ltd. · Changsha and 8 other locations · NCT07640893

• ALN-6400 for women with von Willebrand disease and heavy menstrual bleeding

  This study tests ALN-6400 to see if it can help reduce heavy menstrual bleeding in adult and adolescent women with von Willebrand disease.

  Recruiting Phase 2 Alnylam Pharmaceuticals · Orange, California and 20 other locations · NCT07575308

• Vonicog alfa (rVWF) use in children with von Willebrand disease

  This observational program will see how vonicog alfa (rVWF) works and whether it causes side effects in children with von Willebrand disease in Japan.

  Recruiting Takeda · Tokyo, Tokyo · NCT07404644

• Endothelial colony-forming cell behavior in people with von Willebrand disease and acquired von Willebrand syndrome

  This project will see how endothelial colony-forming cells (ECFCs) behave in people with VWD or AVWS compared with healthy volunteers.

  Recruiting Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · Milan · NCT07358013

• BT200 (Rondaptivon Pegol) for people with type 2B von Willebrand disease and low platelets

  This trial will test whether BT200 (Rondaptivon Pegol) can raise von Willebrand factor, platelet count, and factor VIII and reduce bleeding in adults with type 2B VWD who have low platelets.

  Recruiting Phase 2 Medical University of Vienna · Vienna, State of Vienna · NCT07273721

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.