Extended Half Life Factor (EHF) Products For Heavy Menstrual Bleeding in Hemophilia Carriers

• Status: Completed
• Trial end date: 2019-12-31
• Target enrollment: 0
• Participant gender: Female

Summary

The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. These products are FDA-approved for use in males with hemophilia A and B to prevent and treat bleeding. They are not approved specifically to reduce menstrual bleeding, but may be useful for this purpose. Both products have an "extended half life" which means they circulate in the body longer than other FVIII or FIX products. The study team will gather additional information about the safety of these drugs and how well they work. The results of this feasibility study will provide information for an upcoming larger study.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Emory University
• Collaborator: Bioverativ Therapeutics Inc.
• Treatments: Factor VIII; Immunoglobulin Fc Fragments

Criteria

Inclusion Criteria:

• Females of reproductive age who experience monthly menstrual bleedings

• Female Hemophilia A or B carrier (heterozygote, lyonized or Turner's Syndrome)

• FVIII or FIX activity ≤60% at time of the study

• Baseline Pictorial Bleeding Assessment Chart >150 mean at time of recruitment

• Negative pregnancy test at time of enrollment

• Both female and her male partner have agreed to use an acceptable barrier method of birth control (e.g., diaphragm, cervical cap, male condom, female condom, and spermicidal foam, sponges, and film) throughout the duration of this study (for sexually active participants)

Exclusion Criteria:

• Has not reached menarche

• Menopause: natural or induced by surgical/medical treatment

• Pregnant or breasfeeding

• Female or her male partner refuses to use barrier method of birth control (for sexually active)

• Current use of any of the following contraceptives (copper IUD, oral combined, Progestin-only including but not limited to: Provera, Aygestin, Nexplanon, Implanon, Depo Provera, Mirena IUD)

• VWF:Ag or VWF:RCo <40%

• Diagnosis of a qualitative platelet disorder

• Personal history of thrombosis or superficial thrombosis

• First degree relative with a history of thrombosis

• Personal history of concomitant bleeding or clotting disorder

• Cigarette smoker

• Willing to avoid taking an anti-fibrinolytic agent (amicar, transexamic acid, lysteda) during the trial