Joint Health Study

Hemophilia B Clinical Trial

Official title:

Joint Health Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03358836
• Other study ID #: Joint Health Study
• Status: Completed
• Start date: November 15, 2017
• Est. completion date: August 17, 2020

Study information

• Verified date: June 2022
• Source: Bloodworks
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: August 17, 2020
• Est. primary completion date: August 17, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 16 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Locations

Country	Name	City	State
United States	Orthopaedic Institute for Children at University of California Los Angeles	Los Angeles	California
United States	Tulane University	New Orleans	Louisiana
United States	Oregon Health & Science University	Portland	Oregon
United States	University of California San Diego	San Diego	California
United States	Washington Center for Bleeding Disorders at Bloodworks Northwest	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
Bloodworks	CSL Behring

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Joint health status	Joint health status in all six major joints (elbows, knees, and ankles) in all three groups as assessed by ultrasound	Change from baseline at up to three years
Secondary	Joint and overall health status	Observational assessment of joint and overall health status evaluated by activity level, functional assessment, pain assessment, joint examination, and adherence	Change from baseline at up to three years
Secondary	Joint health at year 1	Observational assessment of joint health at year one in the different groups	Change from baseline at up to one year
Secondary	Joint health at year 2	Observational assessment of joint health at year two in the different groups	Change from baseline at up to two years
Secondary	Acute events/bleeding	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	Change from baseline at up to three years
Secondary	Biomarkers	Exploration of potential biomarkers for joint health	Change from baseline at up to three years