Have you or your loved ones been diagnosed with hemophilia?

You may be eligible to participate in a hemophilia clinical trial.

Have you or your loved ones been diagnosed with hemophilia? You may be eligible to participate in a hemophilia clinical trial.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Hemophilia Clinical Trial in Portland OR

NCT02306694 | Phase 3 | Interventional

Have you or your loved ones been diagnosed with hemophilia?

You may be eligible to participate in a hemophilia clinical trial.

Have you or your loved ones been diagnosed with hemophilia? You may be eligible to participate in a hemophilia clinical trial.

Completed

Male

16 - 85

Years old

This study has recruited 16 Participants

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.

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Details for the study

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<br /> outcome data demonstrating the clinical significance of decreased BMD and altered bone<br /> biomarkers in the hemophilia population. This study demonstrates that PwH have an increased<br /> risk of fracture compared to the general population and that the issue of bone health will<br /> 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.

Treatments and/or Procedures

Advate

Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Primary

Bone Biomarker Density (BMD)

BMD was measured as Z-scores/T-scores using Dual-Energy X-ray Absorptiometry (DEXA) scanning; specifically looking at Spine, Hip/Neck, and Hip total scores. BMD Z-scores compare what would be expected in someone your age and body size. A Z-score, is a unit of standard deviation, where above 0 would indicate the bones are more dense than expected, while a Z-score below 0 would indicate the bones were less dense.

5 days

Primary

Joint Health

Hemophilia Joint Health Score (HJHS); with a higher score representing worst outcomes, scores could range from 0 (no problems) to a max score of 120 (severe problems).

5 days

Primary

Quality of Life Using the VAS and EQ-5D-3L

Visual Analog Scale (VAS) via the EQ-5D-3L was used to report participants self-rated health. EQ-5D-3L total score ranges from 5 (no problems) to 15 (significant problems). VAS scores could range from 0 (worst health ever) to 100 (best health ever).

5 days

Primary

Plasma Cytokine Concentration Differences From 0-hour to 24-hour

cytokines were measured using ELISA/magnetic bead multiplex kits. We calculated concentration change from hour 0 to hour 24. Cytokines: FGF, C-Terminal telopeptide (CTX-1), Dickkopf WNT signaling pathway inhibitor 1(DKK1), Eotaxin, fibroblast growth factor 23(FGF23), interferon gamma, interleukin 13, interleukin 1 beta, interleukin receptor 1 antagonist, interleukin 2, interleukin 4, interleukin 6, interleukin 17, interleukin 8, interleukin 9 Insulin, interferon gamma induced protein 10 (IP10), Leptin, monocyte chemoattractant protein1, monocyte chemoattractant protein1, macrophage inflammatory protein 1a (MIP1a), osteoclasts, Osteoprotegerin, osteopontin, platelet derived growth factors, parathyroid horomone, Recepter activator of nuclear factor kappa-B ligand (RANKL), chemokine ligand 5, sclerostin, transforming growth factor-beta 1, transforming growth factor beta 2, transforming growth factor beta 3, tumor necrosis factor alpha, vascular endothelial growth factor, MIP1b.

24 hours

Other

Medication Adherence

VERITAS instrument self-reported compliance with factor replacement regimen. The VERITAS is composed of 6 subsections, where scores range from 1 (best adherence) to 5 (worst adherence). A total score was calculated with the minimum score of 24 (most adherent) and a maximum score of 120 (least adherent)

5 days

Other

Hemophilia Activities List (HAL) and the International Society of Thrombosis and Hemostasis- Bleeding Assessment Tool (ISTH-BAT)

The HAL is used to assess physical activity; scores range from 42 (severe problems) to 252 (no problems). The ISTH-BAT was used to evaluate bleeding phenotype. Each section was scored using a 0 to 4 scale for each of the 12 subsections. A total score was calculated by taking the sum of each section, resulting in a range of scores from 0 (no bleeding history) to 48 (most extensive clinical intervention for bleeding)

5 days

Other

Participant Quality of Life

Haem-A-QoL was used to assess various components indicating participants quality of life. Haem-A-QoL is composed of 10 subsections with 3-8 questions in each. Questions are rated from 1 to 5, with 5 being the most negative influence on quality of life. Some questions are worded in the inverse, such that the 1 corresponds to the greatest negative impact on quality of life. In this case, the score is inverted to match the remainder of questions for statistical analysis. A Transformed scores is calculated for each subsection and for the total of all the subjections. The scores range from 0 (best quality of life) to 225 (worst quality of life).

5 days

Study 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.