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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

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Other: Ideal Body Weight First Other: Actual Body Weight First Not Applicable

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
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
Official Title: Comparison of Ideal vs. Actual Weight Base Factor Dosing
Actual Study Start Date : January 2017
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Ideal Body Weight First
Randomized to receive factor product based on ideal body weight first
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)].

Experimental: Actual Body Weight First
Randomized to receive factor product based on actual body weight first
Other: Actual Body Weight First
Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.




Primary Outcome Measures :
  1. 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

  2. Underdosing [ Time Frame: Change from baseline at up to two months ]
    Determine the likelihood of underdosing when using ideal body weight

  3. Overdosing [ Time Frame: Change from baseline at up to two months ]
    Determine the likelihood of overdosing when using actual body weight


Secondary Outcome Measures :
  1. Effect of half-life [ Time Frame: Change from baseline at up to two months ]
    Determine the effect on half-life of these dosing strategies

  2. 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

  3. 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

  4. Overweight vs. obese [ Time Frame: Change from baseline at up to two months ]
    Determine the differences, if any, between overweight and obese participants



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03286153


Contacts
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Contact: Heidi Thielmann, PhD 206-689-6234 hthielmann@bloodworksnw.org
Contact: Rebecca Kruse-Jarres, MD, MPH 206-689-6593 RebeccaKr@BloodWorksNW.org

Locations
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United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239-3098
Contact: Michael Recht, MD, PhD    503-494-8311    rechtm@ohsu.edu   
United States, Washington
Washington Center for Bleeding Disorders at Bloodworks Northwest Recruiting
Seattle, Washington, United States, 98104
Contact: Rebecca Kruse-Jarres, MD, MPH    206-689-6593    RebeccaKr@BloodWorksNW.org   
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Amanda Blair, MD    206-987-2106    Amanda.Blair@seattlechildrens.org   
Providence Sacred Heart Children's Hospital Recruiting
Spokane, Washington, United States, 99220-2555
Contact: Judy Felgenhauer, MD    509-474-2777    Judy.Felgenhauer@providence.org   
Sponsors and Collaborators
Bloodworks
Seattle Children's Hospital
Oregon Health and Science University
Providence Health & Services
Investigators
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Principal Investigator: Rebecca Kruse-Jarres, MD, MPH Washington Center for Bleeding Disorders at Bloodworks Northwest
  Study Documents (Full-Text)

Documents provided by Bloodworks:
Informed Consent Form: Main  [PDF] August 7, 2017
Study Protocol  [PDF] October 12, 2016


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Responsible Party: Bloodworks
ClinicalTrials.gov Identifier: NCT03286153     History of Changes
Other Study ID Numbers: BDC Ideal Body Weight Dosing
First Posted: September 18, 2017    Key Record Dates
Last Update Posted: September 18, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Bloodworks:
Hemophilia A
FVIII
Ideal body weight
Actual body weight

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn