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Joint Health Study

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ClinicalTrials.gov Identifier: NCT03358836
Recruitment Status : Active, not recruiting
First Posted : December 2, 2017
Last Update Posted : January 21, 2019
Sponsor:
Collaborator:
CSL Behring
Information provided by (Responsible Party):
Bloodworks

Brief Summary:
This is a prospective, non-randomized, controlled study to examine whether or not having a higher trough during prophylactic treatment with clotting factor offers better joint protection than the standard trough of 1% Factor IX (FIX or Factor 9). This study will test the hypothesis that an extended half-life (EHL) FIX product with an intended trough of >10% could offer better protection than previous treatment concentrates. This study also examines whether or not joint damage could be diagnosed earlier using ultrasound images.

Condition or disease
Hemophilia B

Detailed Description:

This is a prospective, non-randomized, controlled study to examine whether or not having a higher trough during prophylactic treatment with clotting factor offers better joint protection than the standard trough of 1% Factor IX (FIX or Factor 9). This study will test the hypothesis that an extended half-life (EHL) FIX product with an intended trough of >10% could offer better protection than previous treatment concentrates. This study also examines whether or not joint damage could be diagnosed earlier using ultrasound images. The primary research question is whether EHL rIX with an intended trough level of >10% will improve the outcome of joint health of elbow, ankle, and knee joints as assessed with ultrasound assessments in patients with severe Hemophilia B.

Depending on their current treatment regimen, subjects will be in one of three groups: 1) on demand, 2) prophylaxis with an intended trough of 1-5%, and 3) prophylaxis with an intended trough of >10%. Subjects will have four annual study visits over three years: baseline, year 1, year 2, and year 3. At each of these visits, subjects will complete questionnaires, joint assessments, have their blood taken, and have ultrasound images of their joints. The first 10 subjects will also have x-rays and MRIs of their joints for ultrasound validation.

Subjects are encouraged to come in during a painful episode for an examination, assessment, and ultrasound of their joints and to come in again within 1-2 weeks after the painful episode for a repeat examination, assessments, and ultrasound.

This study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at Bloodworks Northwest, Oregon Health & Science University (OHSU), University of California San Diego (UCSD), University of California Los Angeles (UCLA), and Tulane University (TU). Cumulatively across the five sites, up to 50 participants will be enrolled. Additional sites may be added.


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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Joint Health Study
Actual Study Start Date : November 15, 2017
Estimated Primary Completion Date : December 31, 2027
Estimated Study Completion Date : December 31, 2027

Resource links provided by the National Library of Medicine


Group/Cohort
Group A
Episodic treatment with FIX concentrates for bleeding episodes
Group B
Prophylaxis using any FIX concentrate with an intended trough of 1-5%
Group C
Prophylaxis with an extended half-life (EHL) FIX with an intended trough of >10%



Primary Outcome Measures :
  1. Joint health status [ Time Frame: Change from baseline at up to three years ]
    Joint health status in all six major joints (elbows, knees, and ankles) in all three groups as assessed by ultrasound


Secondary Outcome Measures :
  1. Joint and overall health status [ Time Frame: Change from baseline at up to three years ]
    Observational assessment of joint and overall health status evaluated by activity level, functional assessment, pain assessment, joint examination, and adherence

  2. Joint health at year 1 [ Time Frame: Change from baseline at up to one year ]
    Observational assessment of joint health at year one in the different groups

  3. Joint health at year 2 [ Time Frame: Change from baseline at up to two years ]
    Observational assessment of joint health at year two in the different groups

  4. Acute events/bleeding [ Time Frame: Change from baseline at up to three years ]
    Observational assessment of ultrasound findings during acute events/bleeding with an opportunity to follow longitudinally to gain understanding of natural evolution of bleeding as shown by ultrasound

  5. Biomarkers [ Time Frame: Change from baseline at up to three years ]
    Exploration of potential biomarkers for joint health


Biospecimen Retention:   Samples Without DNA
We will draw up to 40 mL (3 tablespoons) of blood to test for factor level, inhibitor level, and biomarkers


Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants are male, age 16 or older, have severe hemophilia B, and are currently on either an episodic or prophylactic treatment regimen with FIX concentrates
Criteria

Inclusion Criteria:

  • Severe hemophilia B (FIX <1%)
  • Either on demand or on prophylaxis with rFIX or EHL-rIX products with the intention to stay on the current regimen for the next 3 years
  • For Group C, start of this treatment regimen up to 6 months prior is permissible

Exclusion Criteria:

  • Other known bleeding disorder
  • Other rheumatologic disorder affecting joints
  • Other known neuromotor defect (making physical exam difficult)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03358836


Locations
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United States, California
Orthopaedic Institute for Children at University of California Los Angeles
Los Angeles, California, United States, 90007
University of California San Diego
San Diego, California, United States, 92122
United States, Louisiana
Tulane University
New Orleans, Louisiana, United States, 70112
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239-3098
United States, Washington
Washington Center for Bleeding Disorders at Bloodworks Northwest
Seattle, Washington, United States, 98104
Sponsors and Collaborators
Bloodworks
CSL Behring
Investigators
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Principal Investigator: Rebecca Kruse-Jarres, MD, MPH Washington Center for Bleeding Disorders at Bloodworks Northwest
  Study Documents (Full-Text)

Documents provided by Bloodworks:
Informed Consent Form: Consent Form  [PDF] October 10, 2017


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Responsible Party: Bloodworks
ClinicalTrials.gov Identifier: NCT03358836     History of Changes
Other Study ID Numbers: Joint Health Study
First Posted: December 2, 2017    Key Record Dates
Last Update Posted: January 21, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Bloodworks:
Hemophilia B
FIX
Factor IX
Factor 9

Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked