Comparison of Ideal vs. Actual Weight Base Factor Dosing

NCT ID: NCT03286153

Last Updated: 2017-09-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-31

Study Completion Date

2018-12-31

Brief Summary

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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

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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.

Conditions

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Hemophilia A

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

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 Study Purpose

HEALTH_SERVICES_RESEARCH

Blinding Strategy

NONE

Study Groups

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Ideal Body Weight First

Randomized to receive factor product based on ideal body weight first

Group Type EXPERIMENTAL

Ideal Body Weight First

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

Actual Body Weight First

Randomized to receive factor product based on actual body weight first

Group Type EXPERIMENTAL

Actual Body Weight First

Intervention Type OTHER

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

Interventions

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

Intervention Type OTHER

Actual Body Weight First

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

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Seattle Children's Hospital

OTHER

Sponsor Role collaborator

Oregon Health and Science University

OTHER

Sponsor Role collaborator

Providence Health & Services

OTHER

Sponsor Role collaborator

Bloodworks

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rebecca Kruse-Jarres, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Washington Center for Bleeding Disorders at Bloodworks Northwest

Locations

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Oregon Health & Science University

Portland, Oregon, United States

Site Status RECRUITING

Washington Center for Bleeding Disorders at Bloodworks Northwest

Seattle, Washington, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Providence Sacred Heart Children's Hospital

Spokane, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Heidi Thielmann, PhD

Role: CONTACT

206-689-6234

Rebecca Kruse-Jarres, MD, MPH

Role: CONTACT

206-689-6593

Facility Contacts

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Michael Recht, MD, PhD

Role: primary

503-494-8311

Rebecca Kruse-Jarres, MD, MPH

Role: primary

206-689-6593

Amanda Blair, MD

Role: primary

206-987-2106

Judy Felgenhauer, MD

Role: primary

509-474-2777

Provided Documents

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Document Type: Informed Consent Form: Main

View Document

Document Type: Informed Consent Form: Consent/Assent SC

View Document

Document Type: Informed Consent Form: Assent Age 15 to 17

View Document

Document Type: Informed Consent Form: Assent Age 7 to 14

View Document

Document Type: Study Protocol

View Document

Other Identifiers

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BDC Ideal Body Weight Dosing

Identifier Type: -

Identifier Source: org_study_id