Clinical trial NCT03286153: Comparison of Ideal vs. Actual Weight Base Factor Dosing
Official title: Comparison of Ideal vs. Actual Weight Base Factor Dosing
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Hemophilia A
Keywords: Hemophilia A
FVIII
Ideal body weight
Actual body weight
Lead sponsor: Bloodworks
Sponsor class: OTHER
Collaborators: name: Seattle Children's Hospital
name: Oregon Health and Science University
name: Providence Health & Services
First posted: 2017-09-18
Enrollment: 20
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
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
Primary purpose: HEALTH_SERVICES_RESEARCH

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.

Eligibility 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

Primary outcomes:
measure: Recovery
timeFrame: Change from baseline at up to two months
measure: Underdosing
timeFrame: Change from baseline at up to two months
measure: Overdosing
timeFrame: Change from baseline at up to two months
Secondary outcomes:
measure: Effect of half-life
timeFrame: Change from baseline at up to two months
measure: Effect on hemophilia severity
timeFrame: 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
measure: Regular half-life vs. extended half-life Regular half-life vs. extended half-life
timeFrame: Change from baseline at up to two months
measure: Overweight vs. obese
timeFrame: Change from baseline at up to two months
Other outcomes:
Locations:
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Seattle
country: United States
facility: Washington Center for Bleeding Disorders at Bloodworks Northwest
state: Washington
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Spokane
country: United States
facility: Providence Sacred Heart Children's Hospital
state: Washington
Study officials:
affiliation: Washington Center for Bleeding Disorders at Bloodworks Northwest
name: Rebecca Kruse-Jarres, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: