Brief Title
Prospective Biomarkers of Bone Metabolism in Hemophilia A
Official Title
Prospective Biomarkers of Bone Metabolism in Hemophilia A
Brief Summary
One of the major shortcomings in studying bone disease in hemophilia is the lack of fracture outcome data demonstrating the clinical significance of decreased BMD and altered bone biomarkers in the hemophilia population. This study demonstrates that PwH have an increased risk of fracture compared to the general population and that the issue of bone health will increase in importance as the PwH population ages.
Detailed Description
This is a pilot study to determine the impact of factor replacement on bone biomarkers in up to 20 hemophilia A subjects. Subjects will be recruited over 1 year for the 5-day protocol. Following a 72-hour washout period, factor levels and bone biomarkers will be followed before and after 50 units/kg replacement on Day 1 and 20 units/kg replacement on Day 3. Each subject can serve as their Figure 4. Fracture rates in PwH compared to historic controls.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Bone Biomarker Density (BMD)
Condition
Hemophilia
Intervention
Advate
Study Arms / Comparison Groups
Open label
Description: Everyone receives Advate (antihemophilic factor) on Day 1 and 3.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
16
Start Date
December 2014
Completion Date
April 16, 2018
Primary Completion Date
April 16, 2018
Eligibility Criteria
Inclusion Criteria: 1. Males with a diagnosis of hemophilia A with a historic baseline FVIII level ≤ 2%. 2. Age > 16 years old 3. Currently using ADVATE as FVIII replacement therapy Exclusion Criteria: 1. Subject or guardian is unwilling or unable to give written informed consent and/or assent 2. Joint or muscle bleeding within 2 weeks of Study Day 1 3. Presence of a current factor inhibitor (>0.6 BU/mL via Nijmegan-modified Bethesda assay) 4. Known collagen vascular bone disease.
Gender
Male
Ages
16 Years - 85 Years
Accepts Healthy Volunteers
No
Contacts
Jason Taylor, MD, PhD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02306694
Organization ID
e11104
Responsible Party
Sponsor
Study Sponsor
Oregon Health and Science University
Collaborators
Baxter Healthcare Corporation
Study Sponsor
Jason Taylor, MD, PhD, Principal Investigator, Oregon Health and Science
Verification Date
March 2020