Clinical trial NCT02306694: Prospective Biomarkers of Bone Metabolism in Hemophilia A
Official title: Prospective Biomarkers of Bone Metabolism in Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia
Bone Disease
Keywords: hemophilia
bone disease
clinical study
biomarkers
Lead sponsor: Oregon Health and Science University
Sponsor class: OTHER
Collaborators: name: Baxter Healthcare Corporation
First posted: 2014-12-03
Enrollment: 16
Sex: MALE
Minimum age (years): 16
Maximum age (years): 85
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Bone Biomarker Density (BMD)
timeFrame: 5 days
measure: Joint Health
timeFrame: 5 days
measure: Quality of Life Using the VAS and EQ-5D-3L
timeFrame: 5 days
measure: Plasma Cytokine Concentration Differences From 0-hour to 24-hour
timeFrame: 24 hours
Secondary outcomes:
Other outcomes:
measure: Medication Adherence
timeFrame: 5 days
measure: Hemophilia Activities List (HAL) and the International Society of Thrombosis and Hemostasis- Bleeding Assessment Tool (ISTH-BAT)
timeFrame: 5 days
measure: Participant Quality of Life
timeFrame: 5 days
Locations:
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
Study officials:
affiliation: Oregon Health and Science
name: Jason Taylor, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
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