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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06610201




Registration number
NCT06610201
Ethics application status
Date submitted
13/09/2024
Date registered
24/09/2024
Date last updated
11/06/2025

Titles & IDs
Public title
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Scientific title
A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Secondary ID [1] 0 0
HMB-002-101_SCR
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Von Willebrand Disease (VWD) 0 0
Von Willebrand Disease, Type 3 0 0
Von Willebrand Disease, Type 2B 0 0
Von Willebrand Disease (VWD), Type 1 0 0
Von Willebrand Disease (VWD), Type 2 and Type 3 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Clotting disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Clinical outcomes of patients with VWD, Type 1 (less than 40 IU per dL)
Other interventions - Clinical outcomes of patients with VWD, Type 1 (less than 50 IU per dL)
Other interventions - Clinical outcomes of patients with VWD, Type 2 and Type 3

VWD, Type 1 (residual VWF antigen and/or activity less than 40 IU per dL) -

VWD, Type 1 (activity less than 50 IU per dL) -

VWD, Type 2 and Type 3 -


Other interventions: Clinical outcomes of patients with VWD, Type 1 (less than 40 IU per dL)
Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1 (less than 40 IU per dL).

Other interventions: Clinical outcomes of patients with VWD, Type 1 (less than 50 IU per dL)
Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1(less than 50 IU per dL).

Other interventions: Clinical outcomes of patients with VWD, Type 2 and Type 3
Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2 and Type 3.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized bleeding event rate
Timepoint [1] 0 0
4.5 to 12.5 months
Primary outcome [2] 0 0
Annualized treated bleeding rate
Timepoint [2] 0 0
4.5 to 12.5 months
Primary outcome [3] 0 0
Annualized heavy menstrual bleed rate
Timepoint [3] 0 0
4.5 to 12.5 months
Primary outcome [4] 0 0
Number of overnight admissions
Timepoint [4] 0 0
4.5 to 12.5 months
Secondary outcome [1] 0 0
Prophylactic and on demand treatment
Timepoint [1] 0 0
4.5 to 12.5 months
Secondary outcome [2] 0 0
Iron status
Timepoint [2] 0 0
4.5 to 12.5 months
Secondary outcome [3] 0 0
Iron replacement use
Timepoint [3] 0 0
4.5 to 12.5 months
Secondary outcome [4] 0 0
Patient-Reported Outcomes Measurement Information System (PROMIS)-29
Timepoint [4] 0 0
4.5 to 12.5 months
Secondary outcome [5] 0 0
Menstrual Bleeding Questionnaire (MBQ)
Timepoint [5] 0 0
4.5 to 12.5 months

Eligibility
Key inclusion criteria
1. Has the ability to provide informed consent to participate in the study, in accordance with the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guideline E6 (R2) (2016) and applicable national and international regulations, before any protocol directed interventions are carried out.
2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
3. Is 16 years or older at the time of screening.
4. First approximately 50 participants: congenital Type 1 VWD with a residual VWF antigen and/or activity less than 40 IU/dL (40%)

Next approximately 50 participants: Type 1 VWD with residual VWF antigen and activity less than 50 IU/dL (50%)

After the first approximately 100 participants: congenital Type 1, Type 2A, Type 2M, Type 2N, or Type 3 VWD

Note: Participants may be enrolled if they have documented laboratory results for VWF antigen and activity within their medical records confirming their diagnosis (consistent with International Society on Thrombosis and Hemostasis [ISTH]/American Society of Hematology [ASH] diagnostic guidelines or British Society for Haematology [BSH]/ United Kingdom Haemophilia Centre Doctors; Organisation [UKHCDO] diagnostic guidelines). For Type 1 VWD in the first part of the study, participants also need to have a documented laboratory result within the last 2 years showing a VWF antigen and/or activity level less than 40 IU/dL. Participants meeting eligibility criteria based on documented results will proceed with having samples collected at Screening to further characterize their antigen and activity levels. These may be repeated up to 2 further times if the results are not consistent with their documented diagnosis and levels are greater than 40 IU/dL.

If participants do not have confirmed results in their medical records as described above, the screening assessment may be repeated up to 2 further times to establish baseline levels for inclusion.
5. Has symptomatic disease as defined by a history of bruising or bleeding events, and typically experiencing bleeding symptoms every month.
Minimum age
16 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
2. Has a significant family history of unprovoked thromboembolic events in first degree relatives.
3. Has a congenital or acquired bleeding disorder other than VWD.
4. Has planned major surgery within the next 6 months.
5. Is pregnant or plans to become pregnant within the next 6 months.
6. Has any concurrent disease, treatment (including ongoing anticoagulation, antiplatelet, or non-steroidal anti-inflammatory drugs), 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.
7. Has received any investigational product within 30 days prior to screening.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/08/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2026
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
PerthSydneyVIC
Recruitment hospital [1] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment hospital [2] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
WA 6150 - Murdoch
Recruitment postcode(s) [2] 0 0
NSW 2050 - Camperdown
Recruitment postcode(s) [3] 0 0
VIC 3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
United States of America
State/province [11] 0 0
Washington
Country [12] 0 0
United Kingdom
State/province [12] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Hemab ApS
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
VP of Clinical Research
Address 0 0
Hemab ApS
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trials (USA; UK)
Address 0 0
Country 0 0
Phone 0 0
+1 888 493 8148; 080 8304 6409
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06610201