Minimize Menorrhagia in Women With Von Willebrand Disease

• Status: Recruiting
• Trial end date: 2022-08-15
• Target enrollment: 0
• Participant gender: Female

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Margaret Ragni; University of Pittsburgh
• Collaborators: Carnegie Mellon University; Duke University; University of North Carolina
• Treatments: Tranexamic Acid

Criteria

Inclusion Criteria:

1. Adult females 13-45 years of age.

2. Mild or moderate von Willebrand disease (VWF:RCo <0.50 IU/ml, normal multimers, past bleeding)

3. Menorrhagia defined as PBAC >100 in at least one of the last two menstrual cycles.

4. Regular menses, at least every 21-35 days.

5. Willingness to have blood drawn

6. No prior history of an allergic reaction or anaphylaxis to rVWF or TA.

7. Willingness to avoid aspirin (ASA) and nonsteroidal anti-inflammatory agents (NSAIDS) during the study.

8. Willingness to comply with randomization to rVWF or TA study arms.

9. 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.

10. 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.

11. Willingness to use "double-barrier" method of contraception during the study.

Exclusion Criteria:

1. Any bleeding disorder other than von Willebrand disease; or past thrombotic disease

2. Pregnant or lactating, or use of hormones (other than progesterone-only), or combined oral contraceptives, and contraceptive implants in past 3 months.

3. Platelet count < 100,000/ul.

4. Use of immunomodulatory or experimental drugs.

5. Surgery within the past 8 weeks.

6. Concomitant use of antiplatelet drugs, anticoagulants, dextran, aspirin or NSAIDs.

7. Treatment with DDAVP, cryoprecipitate, whole blood, plasma and plasma derivatives containing VWF within 5 days of study.

8. Inability to comply with study requirements.

9. Hypothyroidism as defined by elevated TSH.

10. Iron deficiency as defined by low serum ferritin, unless iron replacement has been initiated.

11. History of renal disease