Clinical trial NCT06610201: A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Official title: A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Von Willebrand Disease (VWD)
Von Willebrand Disease, Type 3
Von Willebrand Disease, Type 2B
Von Willebrand Disease (VWD), Type 1
Von Willebrand Disease (VWD), Type 2 and Type 3
Keywords: Von Willebrand Disease (VWD)
Prospective Study
Type 1 VWD
Type 2 and Type 3 VWD
Prophylaxis
Von Willebrand Factor (VWF)
Lead sponsor: Hemab ApS
Sponsor class: INDUSTRY
First posted: 2024-09-24
Enrollment: 200
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants ≥16 years of age with VWD.

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.

Detailed description: This is a prospective, screening study in a minimum of 100 participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with Type 1 VWD and a residual Von Willebrand Factor (VWF) antigen and/or activity of less than 40 IU per dL will be prioritized for recruitment. After approximately 50 participants with a residual VWF antigen and/or activity of less than 40 IU per dL have been included, participants with Type 1 VWD diagnosis and a residual VWF antigen and activity of less than 50 IU per dL may be screened for inclusion. Following enrollment of 100 participants with Type 1 VWD, the study may be opened to participants with Type 2 and Type 3 VWD.

The study includes screening, a Baseline evaluation, and a 4-6 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary and bleeding event treatments), and clinic visits every 12 weeks for laboratory draws. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.

Eligibility criteria: 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.

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.

Primary outcomes:
measure: Annualized bleeding event rate
timeFrame: 4.5 to 12.5 months
measure: Annualized treated bleeding rate
timeFrame: 4.5 to 12.5 months
measure: Annualized heavy menstrual bleed rate
timeFrame: 4.5 to 12.5 months
measure: Number of overnight admissions
timeFrame: 4.5 to 12.5 months
Secondary outcomes:
measure: Prophylactic and on demand treatment
timeFrame: 4.5 to 12.5 months
measure: Iron status
timeFrame: 4.5 to 12.5 months
measure: Iron replacement use
timeFrame: 4.5 to 12.5 months
measure: Patient-Reported Outcomes Measurement Information System (PROMIS)-29
timeFrame: 4.5 to 12.5 months
measure: Menstrual Bleeding Questionnaire (MBQ)
timeFrame: 4.5 to 12.5 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Miami
country: United States
facility: University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Indianapolis
country: United States
facility: Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
state: Indiana
city: New Orleans
country: United States
facility: Tulane University School of Medicine
state: Louisiana
city: Ann Arbor
country: United States
facility: University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Dallas
country: United States
facility: The University of Texas Southwestern Medical Center
state: Texas
city: Seattle
country: United States
facility: Washington Institute For Coagulation (WIC)
state: Washington
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
state: Perth
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: Sydney
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: London
country: United Kingdom
facility: Richmond Pharmacology
Study officials:
affiliation: Hemab ApS
name: VP of Clinical Research
role: STUDY_DIRECTOR
References: