Study Stopped
The study stopped early due to slow recruitment.
Minimize Menorrhagia in Women With Von Willebrand Disease
VWDMin
Prospective, Randomized, Crossover Trial Comparing Recombinant Von Willebrand Factor (rVWF) vs. Tranexamic Acid (TA) to Minimize Menorrhagia in Women With Von Willebrand Disease: The VWD Minimize Study
2 other identifiers
interventional
39
1 country
19
Brief Summary
This is an outpatient, 24-week Phase III prospective, randomized, crossover trial comparing recombinant von Willebrand factor (rVWF) and tranexamic acid (TA, Lysteda®) to minimize menorrhagia in women with von Willebrand disease (VWD). The purpose of this Phase III multicenter prospective, randomized, crossover arm trial is to compare recombinant von Willebrand factor (rVWF) to tranexamic acid (TA) in reducing the severity of menorrhagia in women with von Willebrand disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Feb 2019
Typical duration for phase_3
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 5, 2015
CompletedFirst Posted
Study publicly available on registry
November 17, 2015
CompletedStudy Start
First participant enrolled
February 7, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 30, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
August 30, 2022
CompletedResults Posted
Study results publicly available
July 1, 2024
CompletedJuly 1, 2024
June 1, 2024
3.2 years
November 5, 2015
March 4, 2024
June 27, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Menstrual Bleeding Severity
As measured by Pictorial Blood Assessment Chart (PBAC), 0- no theoretical limit, higher score means greater severity
4 weeks.
Secondary Outcomes (6)
Cycle Duration
4 weeks.
Ruta Menorrhagia Severity Scale
4 weeks.
Center for Epidemiology Studies Depression Scale (CES-D)
4 weeks.
Rand Short Form 36-Question Health Survey (SF-36)
4 weeks
Center for Disease Control Health-Related Quality of Life 14 Questions (CDC-HRQoL-14)
4 weeks.
- +1 more secondary outcomes
Study Arms (2)
Group I
ACTIVE COMPARATORSubjects randomized to Group I will receive Arm A recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5 of each of two menstrual cycles, Cycles 3 and 4.
Group II
ACTIVE COMPARATORGroup II will receive Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm A, recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 on each of two menstrual cycles, Cycles 3 and 4.
Interventions
Group I will receive Arm A recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm B, TA 650 mg 2 tablets orally (po) three times daily on days 1-5 of each of two menstrual cycles, Cycles 3 and 4. Group II will receive Arm B, TA 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm A, rVWF 40 IU/kg intravenously (IV) infusion on day 1 on each of two menstrual cycles, Cycles 3 and 4.
Group I will receive Arm A recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm B, TA 650 mg 2 tablets orally (po) three times daily on days 1-5 of each of two menstrual cycles, Cycles 3 and 4. Group II will receive Arm B, TA 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm A, rVWF 40 IU/kg intravenously (IV) infusion on day 1 on each of two menstrual cycles, Cycles 3 and 4.
Eligibility Criteria
You may qualify if:
- Adult females 13-45 years of age.
- Mild or moderate von Willebrand disease (VWF:RCo \<0.50 IU/ml, normal multimers, past bleeding)
- Menorrhagia defined as PBAC \>100 in at least one of the last two menstrual cycles.
- Regular menses, at least every 21-35 days.
- Willingness to have blood drawn
- No prior history of an allergic reaction or anaphylaxis to rVWF or TA.
- Willingness to avoid aspirin (ASA) and nonsteroidal anti-inflammatory agents (NSAIDS) during the study.
- Willingness to comply with randomization to rVWF or TA study arms.
- Willingness to keep a personal diary of menorrhagia bleeding frequency duration and severity by pictorial blood assessment chart, and any drugs or hemostatic agents taken.
- Willingness to make 4 visits and undergo blood sampling for coagulation studies, and accept randomization of two therapies for each of four consecutive menstrual cycles, including an end-of-study visit.
- Willingness to use "double-barrier" method of contraception during the study.
You may not qualify if:
- Any bleeding disorder other than von Willebrand disease; or past thrombotic disease
- Pregnant or lactating, or use of hormones (other than progesterone-only), or combined oral contraceptives, and contraceptive implants in past 3 months.
- Platelet count \< 100,000/ul.
- Use of immunomodulatory or experimental drugs.
- Surgery within the past 8 weeks.
- Concomitant use of antiplatelet drugs, anticoagulants, dextran, aspirin or NSAIDs.
- Treatment with DDAVP, cryoprecipitate, whole blood, plasma and plasma derivatives containing VWF within 5 days of study.
- Inability to comply with study requirements.
- Hypothyroidism as defined by elevated TSH.
- Iron deficiency as defined by low serum ferritin, unless iron replacement has been initiated.
- History of renal disease
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Margaret Ragnilead
- University of North Carolinacollaborator
- Duke Universitycollaborator
- National Heart, Lung, and Blood Institute (NHLBI)collaborator
Study Sites (19)
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234, United States
Center for Inherited Blood Disorders (CIBD)
Orange, California, 92868, United States
University of California San Francisco
San Francisco, California, 94117, United States
Emory University Afflac Blood Disorders Center
Atlanta, Georgia, 30322, United States
Henry Ford Hospital Medical Center
Detroit, Michigan, 48202, United States
Michigan State University
East Lansing, Michigan, 48824, United States
Mayo Clinic
Rochester, Minnesota, 55902, United States
Washington University St. Louis
St Louis, Missouri, 63110, United States
Cure4thekids Foundation
Las Vegas, Nevada, 89135, United States
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
State University of New York Upstate Medical Center
Syracuse, New York, 13210, United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Ohio State University Wexner Medical Center
Columbus, Ohio, 43210, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
University of Pittsburgh and Hemophilia Center Western PA
Pittsburgh, Pennsylvania, 15213, United States
Vanderbilty University
Nashville, Tennessee, 37322, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Bloodworks Northwest
Seattle, Washington, 98104, United States
Versiti Blood Center of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Publications (2)
Ragni MV, Machin N, Malec LM, James AH, Kessler CM, Konkle BA, Kouides PA, Neff AT, Philipp CS, Brambilla DJ. Von Willebrand factor for menorrhagia: a survey and literature review. Haemophilia. 2016 May;22(3):397-402. doi: 10.1111/hae.12898. Epub 2016 Feb 4.
PMID: 26843404BACKGROUNDRagni MV, Rothenberger SD, Feldman R, Nance D, Leavitt AD, Malec L, Kulkarni R, Sidonio R Jr, Kraut E, Lasky J, Pruthi R, Angelini D, Philipp C, Hwang N, Wheeler AP, Seaman C, Machin N, Xavier F, Meyer M, Bellissimo D, Humphreys G, Smith KJ, Merricks EP, Nichols TC, Ivanco D, Vehec D, Koerbel G, Althouse AD. Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trial. Lancet Haematol. 2023 Aug;10(8):e612-e623. doi: 10.1016/S2352-3026(23)00119-9. Epub 2023 Jun 26.
PMID: 37385272DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Margaret V. Ragni, MD, PHD
- Organization
- University of Pittsburgh
Study Officials
- PRINCIPAL INVESTIGATOR
Margaret V Ragni, MD, MPH
University of Pittsburgh
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Professor of Medicine
Study Record Dates
First Submitted
November 5, 2015
First Posted
November 17, 2015
Study Start
February 7, 2019
Primary Completion
April 30, 2022
Study Completion
August 30, 2022
Last Updated
July 1, 2024
Results First Posted
July 1, 2024
Record last verified: 2024-06
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
- Time Frame
- Within 12 months of trial completion.
- Access Criteria
- Qualified investigators will have access to data and bio specimens, consistent with data sharing policies and applicable laws, and upon receipt of a Research Materials Distribution Agreement, data will be transferred by secure transfers through the BioLINCC website.
The IPD to be shared include individual bleeding data (PBAC), cycle severity score (CSR), cycle length (CL), quality of life by four scales (SF-36, Ruta, CDC-HRQoL-14, CES-D), satisfaction survey, VWF assays, VWF genotype.