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Comparison of Ideal vs. Actual Weight Base Factor Dosing

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ClinicalTrials.gov Identifier: NCT03286153
Recruitment Status : Recruiting
First Posted : September 18, 2017
Last Update Posted : September 18, 2017
Sponsor:
Collaborators:
Seattle Children's Hospital
Oregon Health and Science University
Providence Health & Services
Information provided by (Responsible Party):
Bloodworks

Tracking Information
First Submitted Date  ICMJE August 9, 2017
First Posted Date  ICMJE September 18, 2017
Last Update Posted Date September 18, 2017
Actual Study Start Date  ICMJE January 2017
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 13, 2017)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: September 13, 2017)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Comparison of Ideal vs. Actual Weight Base Factor Dosing
Official Title  ICMJE Comparison of Ideal vs. Actual Weight Base Factor Dosing
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
Subjects are randomized to receive factor based either on ideal body weight or actual body weight first followed by receiving factor based on the other category next
Masking: None (Open Label)
Primary Purpose: Health Services Research
Condition  ICMJE Hemophilia A
Intervention  ICMJE
  • 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.
Study Arms  ICMJE
  • Experimental: Ideal Body Weight First
    Randomized to receive factor product based on ideal body weight first
    Intervention: Other: Ideal Body Weight First
  • Experimental: Actual Body Weight First
    Randomized to receive factor product based on actual body weight first
    Intervention: Other: Actual Body Weight First
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 13, 2017)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2018
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Heidi Thielmann, PhD 206-689-6234 hthielmann@bloodworksnw.org
Contact: Rebecca Kruse-Jarres, MD, MPH 206-689-6593 RebeccaKr@BloodWorksNW.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03286153
Other Study ID Numbers  ICMJE BDC Ideal Body Weight Dosing
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Bloodworks
Study Sponsor  ICMJE Bloodworks
Collaborators  ICMJE
  • Seattle Children's Hospital
  • Oregon Health and Science University
  • Providence Health & Services
Investigators  ICMJE
Principal Investigator: Rebecca Kruse-Jarres, MD, MPH Washington Center for Bleeding Disorders at Bloodworks Northwest
PRS Account Bloodworks
Verification Date September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP