Hemophilia BNCT03358836
Joint Health Study
An observational study of Hemophilia B, run by Bloodworks at 5 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 16 years and older, men only
- Conditions the study is for
- Hemophilia B
- How many people
- 8 participants
- Where
- 5 sitesUnited States
- Study dates
- 15 Nov 2017to 17 Aug 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- Other known bleeding disorder
- Other rheumatologic disorder affecting joints
- Other known neuromotor defect (making physical exam difficult)
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Orthopaedic Institute for Children at University of California Los Angeles
Los Angeles, California, 90007
Tulane University
New Orleans, Louisiana, 70112
Oregon Health & Science University
Portland, Oregon, 97239-3098
University of California San Diego
San Diego, California, 92122
Washington Center for Bleeding Disorders at Bloodworks Northwest
Seattle, Washington, 98104
About this study
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.
Read the 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.
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, FIX, Factor IX, Factor 9
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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% |
What is being measured
The primary outcome is the main question the study is designed to answer.
Joint health status
Joint health status in all six major joints (elbows, knees, and ankles) in all three groups as assessed by ultrasound
Measured: Change from baseline at up to three years
Secondary outcomes (5)
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
Measured: Change from baseline at up to three years
Joint health at year 1
Observational assessment of joint health at year one in the different groups
Measured: Change from baseline at up to one year
Joint health at year 2
Observational assessment of joint health at year two in the different groups
Measured: Change from baseline at up to two years
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
Measured: Change from baseline at up to three years
Biomarkers
Exploration of potential biomarkers for joint health
Measured: Change from baseline at up to three years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2017
- Study start15 Nov 2017
- Primary completion17 Aug 2020
- Study completion17 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bloodworks
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring
- Responsible party
- Sponsor
- Investigators
- Rebecca Kruse-Jarres, MD, MPH, Principal investigator, Washington Center for Bleeding Disorders at Bloodworks Northwest
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03358836
- Sponsor's protocol ID
- Joint Health Study
- Status last verified
- Jun 2022
- First submitted
- 25 Nov 2017
- Last updated
- 22 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Joint Health Study
- ClinicalTrials.gov ID
- NCT03358836
- Acronym / sponsor ID
- Joint Health Study
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, FIX, Factor IX, Factor 9
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- BloodworksUniversity, hospital or other
- Collaborators
- CSL Behring
- Study start
- 15 Nov 2017 (actual)
- Primary completion
- 17 Aug 2020 (actual)
- Study completion
- 17 Aug 2020 (actual)
- First posted
- 2 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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% |
Who may qualify
- Ages
- 16 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- Other known bleeding disorder
- Other rheumatologic disorder affecting joints
- Other known neuromotor defect (making physical exam difficult)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
5 secondary outcomes
Joint and overall health status
Time frame: Change from baseline at up to three years
Joint health at year 1
Time frame: Change from baseline at up to one year
Joint health at year 2
Time frame: Change from baseline at up to two years
Acute events/bleeding
Time frame: Change from baseline at up to three years
Biomarkers
Time frame: Change from baseline at up to three years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rebecca Kruse-Jarres, MD, MPH principal investigator
Washington Center for Bleeding Disorders at Bloodworks Northwest
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B, run by Bloodworks at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Joint health status (time frame: Change from baseline at up to three years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2022.