Comparison of Ideal vs. Actual Weight Base Factor Dosing

NCT03286153 | Unknown

• 1 other identifier: BDC Ideal Body Weight Dosing
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 4

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.

Conditions

Hemophilia A

Keywords

Hemophilia A; FVIII; Ideal body weight; Actual body weight

Outcome Measures

Primary Outcomes (3)

• Recovery

  Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A

  Change from baseline at up to two months

• Underdosing

  Determine the likelihood of underdosing when using ideal body weight

  Change from baseline at up to two months

• Overdosing

  Determine the likelihood of overdosing when using actual body weight

  Change from baseline at up to two months

Secondary Outcomes (4)

• Effect of half-life

  Change from baseline at up to two months

• 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

• Regular half-life vs. extended half-life Regular half-life vs. extended half-life

  Change from baseline at up to two months

• Overweight vs. obese

  Change from baseline at up to two months

Study Arms (2)

Ideal Body Weight First

EXPERIMENTAL

Randomized to receive factor product based on ideal body weight first

Other: Ideal Body Weight First

Actual Body Weight First

EXPERIMENTAL

Randomized to receive factor product based on actual body weight first

Other: Actual Body Weight First

Interventions

Ideal Body Weight First OTHER

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: \[50kg + (2.3kg\*every inch over 5 feet)\].

Ideal Body Weight First

Actual Body Weight First OTHER

Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.

Actual Body Weight First

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bloodworks: lead
• Seattle Children's Hospital: collaborator
• Oregon Health and Science University: collaborator
• Providence Health & Services: collaborator

Study Sites (4)

Oregon Health & Science University

Portland, Oregon, 97239-3098, United States

RECRUITING

Washington Center for Bleeding Disorders at Bloodworks Northwest

Seattle, Washington, 98104, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Providence Sacred Heart Children's Hospital

Spokane, Washington, 99220-2555, United States

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Rebecca Kruse-Jarres, MD, MPH

  Washington Center for Bleeding Disorders at Bloodworks Northwest

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Heidi Thielmann, PhD

CONTACT

206-689-6234; hthielmann@bloodworksnw.org

Rebecca Kruse-Jarres, MD, MPH

CONTACT

206-689-6593; RebeccaKr@BloodWorksNW.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: August 9, 2017
• First Posted: September 18, 2017
• Study Start: January 1, 2017
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: September 18, 2017

Record last verified: 2017-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)