Von Willebrand Disease (VWD)NCT06610201
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
An observational study of Von Willebrand Disease (VWD) and Von Willebrand Disease (VWD), Type 1, run by Hemab ApS at 17 sites in 3 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2
- What is being tested?
- Clinical outcomes of patients with VWD, Type 1 and Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
- How many people
- 200 participants
- Where
- 17 sitesin 3 countries
- Study dates
- 30 Aug 2024to Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants ≥16 years of age with VWD.
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 12Show fewer
- 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.
Trial locations and contacts
17 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 3 countries
United States13 sites, 13 recruiting
- Recruiting
University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, 48109
Angela Weyand, 734-764-9336, acweyand@med.umich.edu
- Recruiting
Emory Children's Center
Atlanta, Georgia, 30329
Gabrille Dean, 404-785-3153, gabrielle.dean@choa.org
- Recruiting
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Yu-Min Shen, 214-648-1929, yu-min.shen@utsouthwestern.edu
- Recruiting
Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
Sanjay Ahuja, 317-871-0000, sahuja@inheme.org
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202-3591
Maggie McDuffie, 501-364-5436, mcduffiemd@archildrens.org
- Recruiting
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Sibgha Zaheer, 323-361-4908, szaheer@chla.usc.edu
- Recruiting
University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
Fernando Corrales-Medina, 305-243-8652, ffc5@med.miami.edu
- Recruiting
Tulane University School of Medicine
New Orleans, Louisiana, 70112-2699
Melody S. Benton, 504-988-3596, mbenton@tulane.edu
- Recruiting
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Taylor Janiec, 602-933-0091, tjaniec@phoenixchildrens.com
- Recruiting
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
Nicoletta Manchin, 412-209-7280, machinnc2@upmc.edu
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239-3098
Kristina Haley, 503-494-8716, hemeresearch@ohsu.edu
- Recruiting
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
Rajiv Pruthi, 507-266-1944, domresearchhub@mayo.edu
- Recruiting
Washington Institute For Coagulation (WIC)
Seattle, Washington, 98101
Abigail deLa Cruz, 206-821-2594, abigail.delacruz@wacbd.org
Australia3 sites, 3 recruiting
- Recruiting
Royal Prince Alfred Hospital
Camperdown, Sydney, NSW 2050
Lindsey Kirby, +61 (02) 9515 7682, lindsey.kirby@health.nsw.gov.au
- Recruiting
The Alfred Hospital
Melbourne, Victoria, VIC 3004
Christine Mazis, +61 (03) 9076 0890, c.mazis@alfred.org.au
- Recruiting
Fiona Stanley Hospital
Murdoch, Perth, WA 6150
Marina Goruppi, +61 (08) 6152 3420, marina.goruppi@health.wa.gov.au
United Kingdom1 site, 1 recruiting
- Recruiting
Richmond Pharmacology
London, SE1 1YR
Volunteer Recruitment Team, +44 (0) 20 7042 5800, volunteer@richmondpharmacology.com
About this study
Official title: Velora Discover: A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease
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)
Read the 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.
- 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
- Being tested
- Clinical outcomes of patients with VWD, Type 1, Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
- Keywords
- Von Willebrand Disease (VWD), Prospective Study, Type 1 VWD, Type 2 VWD, Type 3 VWD, Prophylaxis, Von Willebrand Factor (VWF)
- Medical subject headings
- von Willebrand Diseases, von Willebrand Disease, Type 2, von Willebrand Disease, Type 3
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinical outcomes of patients with VWD, Type 1Other
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 3Other
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.
| Study group | Role | What they receive |
|---|---|---|
| VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL) | Not described Receives: Other: Clinical outcomes of patients with VWD, Type 1 | |
| VWD Type 2A, Type 2M, Type 2N, or Type 3 | Not described Receives: Other: Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized bleeding event rate
Measured: 4.5 to 12.5 months
Annualized treated bleeding rate
Measured: 4.5 to 12.5 months
Annualized heavy menstrual bleed rate
Measured: 4.5 to 12.5 months
Number of overnight admissions
Hospitalization monitoring of bleeding events.
Measured: 4.5 to 12.5 months
Secondary outcomes (6)
Prophylactic and on demand treatment
Details of treatment used for bleeding events
Measured: 4.5 to 12.5 months
Iron status
Blood samples will be collected for the determination of ferritin and hemoglobin levels.
Measured: 4.5 to 12.5 months
Patient-Reported Outcomes Measurement Information System (PROMIS)-29
An assessment which evaluates four items from each of the seven PROMIS domains. Each question is rated on a scale of 1 to 5. In addition, the PROMIS-29 includes one pain intensity question which is rated on a scale of 1 to 10. Higher scores on the PROMIS-29 indicate worse symptoms.
Measured: 4.5 to 12.5 months
Menstrual Bleeding Questionnaire (MBQ)
Measure the effect of heavy menstrual bleeding on a self-assessment of menstrual blood loss, limitations in social and leisure activities, physical activities, and work activities.
Measured: 4.5 to 12.5 months
Epistaxis Severity Score (ESS)
Assessment of nose bleed severity
Measured: 4.5 to 12.5 months
Oral Bleeding Experience
Questionnaire to evaluate bleeding symptoms
Measured: 4.5 to 12.5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2024
- Study start30 Aug 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hemab ApS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- VP of Clinical Research, Study director, Hemab ApS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06610201
- Sponsor's protocol ID
- HMB-002-101_SCR
- Status last verified
- Apr 2026
- First submitted
- 13 Sep 2024
- Last updated
- 21 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Velora Discover: A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT06610201
- Acronym / sponsor ID
- HMB-002-101_SCR
- 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
- Keywords
- Von Willebrand Disease (VWD), Prospective Study, Type 1 VWD, Type 2 VWD, Type 3 VWD, Prophylaxis, Von Willebrand Factor (VWF)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Hemab ApSIndustry (pharma, biotech, devices)
- Study start
- 30 Aug 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 24 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants ≥16 years of age with VWD.
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Clinical outcomes of patients with VWD, Type 1Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1 | Other | VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL) |
| Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3Accumulate 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. | Other | VWD Type 2A, Type 2M, Type 2N, or Type 3 |
Primary outcome
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
Hospitalization monitoring of bleeding events.
6 secondary outcomes
Prophylactic and on demand treatment
Time frame: 4.5 to 12.5 months
Iron status
Time frame: 4.5 to 12.5 months
Patient-Reported Outcomes Measurement Information System (PROMIS)-29
Time frame: 4.5 to 12.5 months
Menstrual Bleeding Questionnaire (MBQ)
Time frame: 4.5 to 12.5 months
Epistaxis Severity Score (ESS)
Time frame: 4.5 to 12.5 months
Oral Bleeding Experience
Time frame: 4.5 to 12.5 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
VP of Clinical Research study director
Hemab ApS
17 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease (VWD) and Von Willebrand Disease (VWD), Type 1, run by Hemab ApS at 17 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Australia, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials (USA; UK) (+1 888 493 8148; 080 8304 6409, Clinicaltrials@hemab.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized bleeding event rate (time frame: 4.5 to 12.5 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2026; AzTrials last refreshed its copy on 26 Oct 2026.