Clinical trial NCT03358836: Joint Health Study
Official title: Joint Health Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia B
Keywords: Hemophilia B
FIX
Factor IX
Factor 9
Lead sponsor: Bloodworks
Sponsor class: OTHER
Collaborators: name: CSL Behring
First posted: 2017-12-02
Enrollment: 8
Sex: MALE
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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.

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.

Eligibility 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)

Primary outcomes:
measure: Joint health status
timeFrame: Change from baseline at up to three years
Secondary outcomes:
measure: Joint and overall health status
timeFrame: Change from baseline at up to three years
measure: Joint health at year 1
timeFrame: Change from baseline at up to one year
measure: Joint health at year 2
timeFrame: Change from baseline at up to two years
measure: Acute events/bleeding
timeFrame: Change from baseline at up to three years
measure: Biomarkers
timeFrame: Change from baseline at up to three years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Orthopaedic Institute for Children at University of California Los Angeles
state: California
city: San Diego
country: United States
facility: University of California San Diego
state: California
city: New Orleans
country: United States
facility: Tulane University
state: Louisiana
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Seattle
country: United States
facility: Washington Center for Bleeding Disorders at Bloodworks Northwest
state: Washington
Study officials:
affiliation: Washington Center for Bleeding Disorders at Bloodworks Northwest
name: Rebecca Kruse-Jarres, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: