# Von Willebrand Disease Minimize Menorrhagia (VWDMin) Trial

> **NIH NIH U01** · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · 2020 · $702,245

## Abstract

ABSTRACT
 Von Willebrand disease (VWD) is the most common congenital bleeding disorder, occurring in 1% of
the population and symptomatic in 0.1%. VWD is caused by defective or deficient von Willebrand factor (VWF),
a multimeric protein encoded for on chromosome 12, which results in mucosal bleeding. In women with VWD,
menorrhagia is the most common symptom, occurring in 80-90% and associated with iron deficiency, reduced
physical and cognitive function, anxiety, depression, and poor quality of life. Current hormonal and non-
hormonal therapies are limited by ineffectiveness and intolerance, and few randomized trials are available to
guide treatment. The current non-hormonal agent of choice for menorrhagia, tranexamic acid (Lysteda®, TA) is
limited by nausea in 50%, and hormonal therapy is ineffective in 30% and poorly tolerated in 20%. Thus, the
lack of safe, effective treatment for menorrhagia in women with VWD constitutes a major public health
problem. A new recombinant von Willebrand factor (Vonvendi®, rVWF), with improved potency, purity, and
half-life, has been shown to be safe and effective in the treatment of bleeds in individuals with VWD, and is
currently under review by the FDA. However, the dose and frequency used for general bleeds do not apply to
menorrhagia. In a survey and literature review of 101 women with VWD, the use of VWF, plasma-derived
(pdVWF) or recombinant (rVWF), resulted in reduced menstrual blood loss in over 95% with no adverse
events. Yet, the optimal dose, frequency, and acceptability of VWF, which requires intravenous administration,
have not been established in women with menorrhagia. We hypothesize that intravenous recombinant von
Willebrand factor (rVWF) on day 1 of bleeding is more effective than oral tranexamic acid (TA) on day 1-5 of
bleeding, on each of two consecutive menstrual cycles, in reducing menorrhagia and improving quality of life,
despite its higher cost and intravenous route of administration.
 The proposed Collaborative R01 Investigator-Initiated Multi-Site Clinical Trial, therefore, is designed in
response to Funding Opportunity Announcement PAR-13-128 as a multi-center prospective, randomized,
crossover Phase III clinical trial, Von Willebrand Minimize (VWDMin) Trial, the purpose of which is to
evaluate if rVWF is superior to TA in reducing menorrhagia in women with VWD when given during bleeding in
two consecutive menstrual cycles each over 24 weeks. The trial, with clinical coordination by the University of
Pittsburgh and data coordination by Pitt’s Center for Research Healthcare Data Center (CRHC DC) is feasible
as 1) the trial design has been optimized for this rare population; 2) structured interviews indicate physician
and parent acceptance of the trial concept; 3) the Steering Committee indicated unanimous consensus on
study drug choice and dosing; and 4) surveys indicate there are sufficient hemophilia treatment centers (HTCs)
and eligible subjects to conduct the trial. The concept is ...

## Key facts

- **NIH application ID:** 9768531
- **Project number:** 5U01HL133815-03
- **Recipient organization:** UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- **Principal Investigator:** MARGARET VICTORIA RAGNI
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $702,245
- **Award type:** 5
- **Project period:** 2017-09-01 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9768531

## Citation

> US National Institutes of Health, RePORTER application 9768531, Von Willebrand Disease Minimize Menorrhagia (VWDMin) Trial (5U01HL133815-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9768531. Licensed CC0.

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