Comparison of Ideal vs. Actual Weight Base Factor Dosing
- Conditions
- Hemophilia A
- Interventions
- Other: Ideal Body Weight FirstOther: Actual Body Weight First
- Registration Number
- NCT03286153
- Lead Sponsor
- Bloodworks
- Brief Summary
This is a randomized, prospective, multicenter study to examine whether or not the current recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired result of bleeding prevention and cessation. This study also examines ways to prevent delivering excessive factor by using a patient's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in protective factor levels.
- Detailed Description
This is a randomized, prospective, multicenter, open-label, crossover study to examine whether or not the current recommended factor dosing strategy, i.e., dosing by actual body weight in overweight and obese patients, may deliver more clotting factor than necessary to cause bleeding to stop in participants with Hemophilia A who use Factor VIII (FVIII). This study also examines ways to prevent delivering too much factor by using a participant's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in hemostatic factor levels.
The study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at Bloodworks Northwest, Oregon Health \& Science University (OHSU), Seattle Children's Hospital (SCH), and Providence Sacred Heart Children's Hospital (SH). Cumulatively across the four sites, up to 20 participants will be enrolled. Randomization will be performed centrally at WCBD.
Participants will provide their own factor. Prior to the first study-related dose, participants will stop taking any FVIII products for either 48 hours if currently using a short-acting FVIII product or 72 hours for a long acting FVIII product. Factor levels will be measured immediately before and at multiple points after two different factor doses. Subjects will be randomized to start their dosage based either on actual body weight or ideal body weight first and then crossover to receive dosage based on the other category.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- Male
- Target Recruitment
- 20
- Hemophilia A
- Able and willing to comply with pharmacokinetic testing schedule
- Either overweight or obese BMI using CDC definitions by age
- Inhibitor of > 0.6 BU twice in the past, or documented abnormal recovery of less than 66% (of expected) in the past
- Known other bleeding disorder
- Known other prolongation in aPTT (lupus anticoagulant, FXII deficiency)
- Female
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Ideal Body Weight First Ideal Body Weight First Randomized to receive factor product based on ideal body weight first Actual Body Weight First Actual Body Weight First Randomized to receive factor product based on actual body weight first
- Primary Outcome Measures
Name Time Method Overdosing Change from baseline at up to two months Determine the likelihood of overdosing when using actual body weight
Recovery Change from baseline at up to two months Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A
Underdosing Change from baseline at up to two months Determine the likelihood of underdosing when using ideal body weight
- Secondary Outcome Measures
Name Time Method Regular half-life vs. extended half-life Regular half-life vs. extended half-life Change from baseline at up to two months Determine differences in participants receiving regular half-life versus extended half-life products
Effect of half-life Change from baseline at up to two months Determine the effect on half-life of these dosing strategies
Effect on hemophilia severity Change from baseline at 20-40 minutes, 5-7 hours, 20-26 hours, and 44-50 hours for both half-life and extended half-life and also at 69-75 hours, and 93-99 hours for extended half-life Determine the effect of pharmacokinetic differences on hemophilia severity
Overweight vs. obese Change from baseline at up to two months Determine the differences, if any, between overweight and obese participants
Trial Locations
- Locations (4)
Washington Center for Bleeding Disorders at Bloodworks Northwest
🇺🇸Seattle, Washington, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Providence Sacred Heart Children's Hospital
🇺🇸Spokane, Washington, United States