Weight-based Dosing in Hemophilia A

• Status: Terminated
• Trial end date: 2020-01-10
• Target enrollment: 0
• Participant gender: Male

Summary

Hemophilia A is an inherited (genetic) disease where a protein, factor VIII (FVIII), which promotes blood clotting is missing or does not work properly. Individuals with hemophilia A are at risk for bleeding. Bleeding is prevented and/or treated with recombinant factor VIII (rFVIII), which is an FDA-approved treatment for Hemophilia A. Obesity is common among patients with hemophilia. Some studies have shown that obese hemophilia patients may be able to prevent bleeding with a lower dose of clotting factor than the dose they are currently receiving. The lower dose is calculated based on what a patient should weigh rather than what he does weigh. This is a clinical research study to test whether calculating rFVIII dosing based on lean body mass and ideal body weight (what a person should weigh based on his height) in overweight and obese patients with hemophilia is more accurate than calculating rFVIII dosing based on what a person actually weighs.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Craig Seaman; University of Pittsburgh
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

1. Adult males age 18 or older.

2. Hemophilia A (FVIII activity 40% or less).

3. Overweight or obesity defined as a BMI of 25.0-29.9 and ≥ 30 mg/m2, respectively.

Exclusion Criteria:

1. Prior history of, or currently detectable, FVIII inhibitor defined as greater than or equal to 0.6 Bethesda Units (BU); however, a subject with a past low-level non-responding inhibitor defined as less than 5 BU, with no increase in titer following FVIII exposure, and not detectable within 12 months of the study, despite FVIII exposure during that period, will be allowed to enroll on study.

2. Allergy to FVIII products.

3. Current rFVIII requirements do not include at least a 72-hour period without rFVIII administration.