Von Willebrand Factor DeficiencyLow VWFNCT02933411
Low VW Activity in Adolescent HMB
An observational study of Von Willebrand Factor Deficiency, run by Baylor College of Medicine at 12 sites in United States.
Trial summary
This is a research study for patients diagnosed with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF). Menstruation, also known as a period, is the regular discharge of blood and tissues from the uterus. HMB is having a heavier amount of discharge during menstrual period. Read more about this study
- Who is it for?
- Ages Up to 21 years, women only
- Conditions the study is for
- Von Willebrand Factor Deficiency
- What is being tested?
- Genetic Analysis, Medical Record Data Abstraction and Pictorial Blood Assessment Chart (PBAC) score
- Type of treatment
- Injection or infusion
- How many people
- 120 participants
- Where
- 12 sitesUnited States
- Study dates
- 5 Jan 2017to Nov 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Up to 21 years
- Women only
- No healthy volunteers
- Child, Adult
Study population: Females under the age of 21 years old with heavy menstrual bleeding and low von willebrand factor
Who may be able to join
- Post-menarchal females less than 21 years of age
- HMB defined as PBAC score greater than 100
- VWF:Activity more than or equal to 30 and less than or equal to 50 IU/dL x 2
- VWF: Activity /VWF:Ag ratio greater than or equal to 0.6
- Normal VW multimers, if performed
Who cannot join
- Post menarchal females age greater than or equal to 21 years
- VWF: Activity less than 30 or greater than 50 IU/dL consistently, type 2 or type 3 VWD
- Presence of other bleeding disorders (thrombocytopenia, platelet function defect, coagulation factor deficiency, fibrinogen defect or deficiency)
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Children's Hospital of Atlanta
Atlanta, Georgia, 30322
Hemophilia Center of Western New York
Buffalo, New York, 14209
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwestern Medical Center- Children's Medical Center
Dallas, Texas, 75235
Michigan State University
East Lansing, Michigan, 48823
Joseph M Sanzari Children's Hospital
Hackensack, New Jersey, 07601
Texas Children's Hospital
Houston, Texas, 77030
Children's Mercy Hospital
Kansas City, Missouri, 64108
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Mary M. Gooley Hemophilia Center
Rochester, New York, 14621
About this study
Official title: Genotypic and Phenotypic Analysis of Adolescents With Heavy Menstrual Bleeding and Low Von Willebrand Activity
This is a research study for patients diagnosed with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF). Menstruation, also known as a period, is the regular discharge of blood and tissues from the uterus. HMB is having a heavier amount of discharge during menstrual period. Low Von Willebrand Factor means that the participant has lower level of a blood protein that is important for clotting of blood and so, the participant is at a higher risk for bleeding.
The purpose of this project is to study the genetic differences of adolescent females with HMB and low VWF activity and compare the genetic differences with their bleeding manifestations, response to medications and outcome.
Read the detailed description
One hundred and twenty subjects will be enrolled. Adolescent females with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF) will be recruited.
Data collection will occur from participant's medical records in regards to their low VWF activity and HMB medical history.
Participants will be asked to complete symptom questionnaires in regards to their HMB.
A blood sample will be collected to analyze how many participants have the disease causing sequence variation in the VWF gene and other genes affecting bleeding, clotting and blood vessel biology and correlated with their bleeding history.
The blood sample will be deidentified and stored indefinitely for future research.
- Conditions
- Von Willebrand Factor Deficiency
- Being tested
- Genetic Analysis, Medical Record Data Abstraction, Pictorial Blood Assessment Chart (PBAC) score, Complete Bleeding Symptom ISTH Bleeding Assessment Tool
- Keywords
- Bleeding Disorders, Heavy Menstrual Bleeding
- Medical subject headings
- Von willebrand factor, deficiency, Hemostatic Disorders, Menorrhagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genetic AnalysisOther
Blood sample will be drawn to assess the number of participants enrolled that have sequence variation in the von willebrand factor gene and other genes affecting bleeding, clotting and blood vessel biology.
Medical Record Data AbstractionOther
The subject's response to intranasal or intravenous desmopressin (DDAVP) challenge performed as part of standard of care will be recorded. The desmopressin is a drug that increases the clotting factor in blood to prevent bleeding.
Medical and family history including and not limited to age, diagnoses, race/ethnicity, lab values, HMB and low VWF activity diagnoses, and treatment history and outcome will also be recorded.
Pictorial Blood Assessment Chart (PBAC) scoreOther
Also called PBAC Score
PBAC is a pictorial tool to assess menstrual blood loss. Study team will complete the PBAC assessment with each study participant evaluating and measure response to different treatments for menstrual blood loss in the clinic setting between those with VWD versus other bleeding disorders.
Complete Bleeding Symptom ISTH Bleeding Assessment ToolOther
Also called ISTH BAT Assessment
Study participants will complete the ISTH BAT assessment. The ISTH BAT is a questionnaire to aid in the standardized evaluation of the presence and severity of bleeding symptoms.
| Study group | Role | What they receive |
|---|---|---|
| Group A | Adolescent women with heavy menstrual bleeding and low von willebrand factor activity. Receives: Other: Genetic Analysis; Other: Medical Record Data Abstraction; Other: Pictorial Blood Assessment Chart (PBAC) score; Other: Complete Bleeding Symptom ISTH Bleeding Assessment Tool |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adolescents with Low VWF and HMB with genetic variations in VWF gene and other genes affecting bleeding, clotting and blood vessel biology
The genetic variations of adolescent females with heavy menstrual bleeding and low von Willebrand factor activity in VWF gene and other genes affecting bleeding, clotting and blood vessel biology
Measured: 3 years
Secondary outcomes (1)
Number of adolescents with Low VWF and HMB with genetic variations and bleeding phenotype (including PBAC score and ISTH-BAT score, response to DDAVP challenge, HMB therapy)
The correlation of subjects with and without genetic variations with bleeding phenotype (including PBAC score, ISTH BAT score, response to DDAVP challenge and HMB therapy)
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2016 (estimated)
- Study start5 Jan 2017
- Primary completionJun 2020
- Study completionNov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Factor DeficiencyStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Shire
- Responsible party
- Principal investigator, Sarah Sartain, Baylor College of Medicine
- Investigators
- Sarah Sartain, MD, Principal investigator, Texas Children's Hospital, an affiliate of Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02933411
- Sponsor's protocol ID
- H-39295 LOW VWF
- Acronym
- Low VWF
- Status last verified
- Apr 2022
- First submitted
- 10 Oct 2016
- Last updated
- 11 Apr 2022
- 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 11 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Genotypic and Phenotypic Analysis of Adolescents With Heavy Menstrual Bleeding and Low Von Willebrand Activity
- ClinicalTrials.gov ID
- NCT02933411
- Acronym / sponsor ID
- Low VWF / H-39295 LOW VWF
- Conditions
- Von Willebrand Factor Deficiency
- Keywords
- Bleeding Disorders, Heavy Menstrual Bleeding
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Shire
- Study start
- 5 Jan 2017 (actual)
- Primary completion
- Jun 2020 (actual)
- Study completion
- Nov 2025 (estimated)
- First posted
- 14 Oct 2016 (estimated)
- 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.
This is a research study for patients diagnosed with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF). Menstruation, also known as a period, is the regular discharge of blood and tissues from the uterus. HMB is having a heavier amount of discharge during menstrual period. Low Von Willebrand Factor means that the participant has lower level of a blood protein that is important for clotting of blood and so, the participant is at a higher risk for bleeding.
The purpose of this project is to study the genetic differences of adolescent females with HMB and low VWF activity and compare the genetic differences with their bleeding manifestations, response to medications and outcome.
Detailed description
One hundred and twenty subjects will be enrolled. Adolescent females with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF) will be recruited.
Data collection will occur from participant's medical records in regards to their low VWF activity and HMB medical history.
Participants will be asked to complete symptom questionnaires in regards to their HMB.
A blood sample will be collected to analyze how many participants have the disease causing sequence variation in the VWF gene and other genes affecting bleeding, clotting and blood vessel biology and correlated with their bleeding history.
The blood sample will be deidentified and stored indefinitely for future research.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group A | – | Other: Genetic Analysis, Other: Medical Record Data Abstraction, Other: Pictorial Blood Assessment Chart (PBAC) score, Other: Complete Bleeding Symptom ISTH Bleeding Assessment ToolAdolescent women with heavy menstrual bleeding and low von willebrand factor activity. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Females under the age of 21 years old with heavy menstrual bleeding and low von willebrand factor
Key inclusion criteria (5 listed)
- Post-menarchal females less than 21 years of age
- HMB defined as PBAC score greater than 100
- VWF:Activity more than or equal to 30 and less than or equal to 50 IU/dL x 2
- VWF: Activity /VWF:Ag ratio greater than or equal to 0.6
- Normal VW multimers, if performed
Key exclusion criteria (3 listed)
- Post menarchal females age greater than or equal to 21 years
- VWF: Activity less than 30 or greater than 50 IU/dL consistently, type 2 or type 3 VWD
- Presence of other bleeding disorders (thrombocytopenia, platelet function defect, coagulation factor deficiency, fibrinogen defect or deficiency)
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 |
|---|---|---|
| Genetic AnalysisBlood sample will be drawn to assess the number of participants enrolled that have sequence variation in the von willebrand factor gene and other genes affecting bleeding, clotting and blood vessel biology. | Other | Group A |
| Medical Record Data AbstractionThe subject's response to intranasal or intravenous desmopressin (DDAVP) challenge performed as part of standard of care will be recorded. The desmopressin is a drug that increases the clotting factor in blood to prevent bleeding. Medical and family history including and not limited to age, diagnoses, race/ethnicity, lab values, HMB and low VWF activity diagnoses, and treatment history and outcome will also be recorded. | Other | Group A |
| Pictorial Blood Assessment Chart (PBAC) scoreAlso: PBAC ScorePBAC is a pictorial tool to assess menstrual blood loss. Study team will complete the PBAC assessment with each study participant evaluating and measure response to different treatments for menstrual blood loss in the clinic setting between those with VWD versus other bleeding disorders. | Other | Group A |
| Complete Bleeding Symptom ISTH Bleeding Assessment ToolAlso: ISTH BAT AssessmentStudy participants will complete the ISTH BAT assessment. The ISTH BAT is a questionnaire to aid in the standardized evaluation of the presence and severity of bleeding symptoms. | Other | Group A |
Primary outcome
Number of adolescents with Low VWF and HMB with genetic variations in VWF gene and other genes affecting bleeding, clotting and blood vessel biology
Time frame: 3 years
The genetic variations of adolescent females with heavy menstrual bleeding and low von Willebrand factor activity in VWF gene and other genes affecting bleeding, clotting and blood vessel biology
1 secondary outcome
Number of adolescents with Low VWF and HMB with genetic variations and bleeding phenotype (including PBAC score and ISTH-BAT score, response to DDAVP challenge, HMB therapy)
Time frame: 3 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2022.
- 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. This study is not recruiting at the moment.
Sarah Sartain, MD principal investigator
Texas Children's Hospital, an affiliate of Baylor College of Medicine
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Factor Deficiency, run by Baylor College of Medicine at 12 sites in United States.
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 5 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 11 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adolescents with Low VWF and HMB with genetic variations in VWF gene and other genes affecting bleeding, clotting and blood vessel biology (time frame: 3 years). 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 11 Apr 2022; AzTrials last refreshed its copy on 30 Oct 2026.