Comparison of Ideal vs. Actual Weight Base Factor Dosing

Overview

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.

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Crossover Assignment
    • Primary Purpose: Health Services Research
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2018

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.

Interventions

  • Other: Ideal Body Weight First
    • Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on ideal body weight. For participants age 12-19, ideal weight is calculated using the McLaren method. For participants age 20 and over, ideal weight is calculated using the following equation: [50kg + (2.3kg*every inch over 5 feet)].
  • Other: Actual Body Weight First
    • Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.

Arms, Groups and Cohorts

  • Experimental: Ideal Body Weight First
    • Randomized to receive factor product based on ideal body weight first
  • Experimental: Actual Body Weight First
    • Randomized to receive factor product based on actual body weight first

Clinical Trial Outcome Measures

Primary Measures

  • Recovery
    • Time Frame: 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
    • Time Frame: Change from baseline at up to two months
    • Determine the likelihood of underdosing when using ideal body weight
  • Overdosing
    • Time Frame: Change from baseline at up to two months
    • Determine the likelihood of overdosing when using actual body weight

Secondary Measures

  • Effect of half-life
    • Time Frame: Change from baseline at up to two months
    • Determine the effect on half-life of these dosing strategies
  • Effect on hemophilia severity
    • Time Frame: 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
  • Regular half-life vs. extended half-life Regular half-life vs. extended half-life
    • Time Frame: Change from baseline at up to two months
    • Determine differences in participants receiving regular half-life versus extended half-life products
  • Overweight vs. obese
    • Time Frame: Change from baseline at up to two months
    • Determine the differences, if any, between overweight and obese participants

Participating in This Clinical Trial

Inclusion Criteria

  • Hemophilia A – Able and willing to comply with pharmacokinetic testing schedule – Either overweight or obese BMI using CDC definitions by age Exclusion Criteria:

  • 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

Gender Eligibility: Male

Minimum Age: 12 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Bloodworks
  • Collaborator
    • Seattle Children’s Hospital
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Rebecca Kruse-Jarres, MD, MPH, Principal Investigator, Washington Center for Bleeding Disorders at Bloodworks Northwest
  • Overall Contact(s)
    • Heidi Thielmann, PhD, 206-689-6234, hthielmann@bloodworksnw.org

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