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RecruitingNCT06610201Updated Apr 21, 2026

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

An observational study in Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1 and Von Willebrand Disease (VWD), Type 2, sponsored by Hemab ApS. Recruiting at 17 sites in 3 countries. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2026-04-21.

Sponsored by Hemab ApS · Observational

From the registry’s dates

  • Started Aug 2024; still recruiting 2 years 2 months later.
Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
200
Ages
16 Years and older
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-12

Among 38 studies in von Willebrand Diseases with a site in United States — 11 open to participants now, 12 under way, 2 of them phase 3. von Willebrand Diseases · United States.

Planned enrollment
7th of 16
200 · median 127
Started
2024
with 3 others
Observational
16 of 38
6 open now
Primary completion
2026-12
later than 1 of 11 live
Registered
32nd of 38
by date
Lead sponsor
2 of 38
Hemab ApS

Among the 38 studies in von Willebrand Diseases with a site in United States, this one is the 7th largest by planned enrollment of the 16 observational ones that give a count, is one of 4 that started in 2024, is one of 16 observational ones, reaches primary completion later than 1 of the 11 other live ones, and was the 32nd registered.

von Willebrand Diseases

96 studies on the registry are indexed under von Willebrand Diseases; 25 are open to participants now.

Browse von Willebrand Diseases studies →

Open to participants now

11 of the 38 studies in von Willebrand Diseases with a site in United States are open now. Recruiting first, the newest first:

5 more — sign in →

All 11 open studies →

A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →

Who these studies take

Of the 23 von Willebrand Diseases studies recruiting now, 15 (65%) set no upper age limit and 8 (35%) stop at 75 or younger; 14 take children under 18; 3 take healthy volunteers.

This study: 16 Years and older · all sexes · no healthy volunteers.

The full table on the hub →

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Watch this landscapeDownload CSVNarrow it in search →With an account: a weekly mail of what changed among 38 studies in von Willebrand Diseases with a site in United States, or all of them as a file.
02

Study 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)

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.

03

Conditions studied

  • 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)
04

Who can participate

Ages eligible
16 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Participants ≥16 years of age with VWD.

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Has the ability to provide 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 (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

Exclusion Criteria:

  1. Has a history of 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; >30 and ≤38.5 kg/m² (moderately obese, adjusted for ethnicity and increased central adiposity); reduced mobility; active ; 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 sign off.
  9. Has clinically significant cardiovascular disease including, but not limited to: 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 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.
Terms in these criteria, in plain language(7)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

BMI (body mass index)

Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.

What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

NYHA class (heart failure)

The New York Heart Association’s four classes of how much heart failure limits physical activity.

I
No limits: ordinary activity causes no undue tiredness or breathlessness.
II
Slight limits: comfortable at rest, ordinary activity causes symptoms.
III
Marked limits: comfortable at rest, less than ordinary activity causes symptoms.
IV
Symptoms even at rest; any activity makes them worse.

Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
200 participants (estimated)
Patient registry
No

Groups and cohorts

  • 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

  • OtherClinical 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

  • OtherClinical 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.

06

What researchers measure

Primary outcomes

  1. Annualized bleeding event rate

    Time frame: 4.5 to 12.5 months

  2. Annualized treated bleeding rate

    Time frame: 4.5 to 12.5 months

  3. Annualized heavy menstrual bleed rate

    Time frame: 4.5 to 12.5 months

  4. Number of overnight admissions

    Hospitalization monitoring of bleeding events.

    Time frame: 4.5 to 12.5 months

Secondary outcomes

  1. Prophylactic and on demand treatment

    Details of treatment used for bleeding events

    Time frame: 4.5 to 12.5 months

  2. Iron status

    Blood samples will be collected for the determination of ferritin and hemoglobin levels.

    Time frame: 4.5 to 12.5 months

  3. 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.

    Time frame: 4.5 to 12.5 months

  4. 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.

    Time frame: 4.5 to 12.5 months

  5. Epistaxis Severity Score (ESS)

    Assessment of nose bleed severity

    Time frame: 4.5 to 12.5 months

  6. Oral Bleeding Experience

    Questionnaire to evaluate bleeding symptoms

    Time frame: 4.5 to 12.5 months

07

Study locations

17 of 17 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Aug 2024 (actual). Primary completion is estimated for Dec 2026, 28 months after the start.

Of 24 observational studies under von Willebrand Diseases, half reached primary completion within 71 months of starting (the median), counting those still running; a quarter within 45 and three quarters within 97. This study's planned 28 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 13, 2024
First posted
Sep 24, 2024
StartActual
Aug 30, 2024
Primary completionEst.
Dec 2026
CompletionEst.
Dec 2026
Last update
Apr 21, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Apr 21, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06610201
Lead sponsor
Hemab ApS
Responsible party
Sponsor
First posted
Sep 24, 2024
Start date
Aug 30, 2024
Primary completion
Dec 2026 (estimated)
Completion
Dec 2026 (estimated)
Last update
Apr 21, 2026

Study contacts

Clinical Trials (USA; UK)
Contact
Clinicaltrials@hemab.com
+1 888 493 8148; 080 8304 6409
Clinical Trials (Australia)
Contact
Clinicaltrials@hemab.com
+611800875216
VP of Clinical Research
study director · Hemab ApS

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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