HemophiliaNCT04618237
Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
An observational study of Hemophilia and Arthropathy, run by Taipei Medical University Hospital at 1 site in Taiwan.
Trial summary
Patients with severe hemophilia A can experience repeated bleeding into the same joint (ie, a target joint; most frequently in the ankle, knee, and elbow), which can contribute to hemophilic arthropathy and, over time, ultimately result in chronic pain, functional limitations, deformities, reduced joint of motion and… Read more about this study
- Who is it for?
- Ages 20 to 75 years
- Conditions the study is for
- Hemophilia, Arthropathy
- How many people
- 35 participants
- Where
- 1 siteTaiwan
- Study dates
- 20 Jul 2020to 31 Dec 2024
- 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 20 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 35 severe Hemophilia A patients
Who may be able to join
- Willing to sign inform consent form and complied with study procedure
- Patient received confirmative diagnosis of Hemophilia A Disease
- Severe hemophilia A patient currently who are on treatment with Eloctate prior to enrolment visit (Note: Prescription based on physician's clinical judgement and discussion with patients)
Who cannot join
- Unwilling to sign inform consent form.
- History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
- History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
- Significantly impaired vision / hearing
- Cannot communicate in Mandarin Chinese
- Recent joint bleeding and trauma 3 months prior to study
Trial locations and contacts
1 site in 1 country.
About this study
Patients with severe hemophilia A can experience repeated bleeding into the same joint (ie, a target joint; most frequently in the ankle, knee, and elbow), which can contribute to hemophilic arthropathy and, over time, ultimately result in chronic pain, functional limitations, deformities, reduced joint of motion and decreased quality of life.
Early use of prophylaxis is recommended following diagnosis of severe hemophilia A to maintain joint health and prevent joint destruction. Eloctate is produced using a human cell line and an addition of an Fc fusion protein to recombinant FVIII (rFVIIIFc) with prolonged half-life and was launched in Taiwan from Nov. 2018. The pivotal studies of rFVIIIFc show that patients maintained a low bleeding rate, with most experiencing a median annualized bleeding rate (ABR) of 0 and 97% of target joints were resolved across adult, adolescent, and pediatric subjects during 4 years of prophylaxis rFVIIIFc treatment. However, in Taiwan we are still lacking the real world treatment outcome data on rFVIIIFc, especially for the joint health evidence in Asian Hemophilia A patients.
Therefore the objective of this study is to evaluate the effectiveness of rFVIIIFc treatment on joint health over a long observational period of 36 month focused on physical and functional changes in hemophilia A patients.
Read the detailed description
Thermographic assessment has been proposed to be a potential tool in evaluating the inflammatory arthritis patients. Thermography has advantages of simple, time-saving, low skill demanding, no radiation exposure and non-invasiveness. It is possible to conduct a quantitative analysis with thermography. However, it is still unknown whether thermography evaluation can provide clinical information regarding the joint health in hemophilia arthropathy patients. The project will focus on:
- Investigate the association among thermography findings and functional status in hemophilia arthroplasty
- Investigate the novel biomarkers in predicting function in hemophilia arthropathy including: soft tissue ultrasound, body composition, thermographic findings of joints, muscle composition and serum biomarkers of joint degeneration.
- Conditions
- Hemophilia, Arthropathy
- Medical subject headings
- Hemophilia A, Joint Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemophilia Joint Health Score (HJHS)
The HJHS measures joint health, in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in hemophilia: the knees, ankles, and elbows. The HJHS provides a total score (higher score is worse; max=124), joint specific scores, and a global gait score.
Measured: Baseline to 24 months follow up
Musculoskeletal ultrasound/HEAD-US scoring system (Haemophilia Early Arthropathy Detection by UltraSound)
Musculoskeletal ultrasound could detect soft tissue alterations and be used as early detection of hemophilic arthropathy. Ultrasound assessment of bilateral knees and ankles, and bilateral gastrocnemius and quadriceps would be taken out in this study and scored with HEAD-US(Haemophilia Early Arthropathy Detection by UltraSound) scoring system. The scoring was based on three markers: synovitis (score 0-2), cartilage (score 0-4) and subchondral bone (score of 0-2) with a maximum score of eight points per joint, higher score is worse.
Measured: Baseline to 24 months follow up
X-rays/Pettersson score (PS)
X-rays are used to evaluate structural changes in joints of patients with hemophilic arthropathy. X-rays images of bilateral knees and ankles will be taken in this study and scored with Pettersson scoring system. The maximum possible score for a given joint is 13 points and higher score is worse.
Measured: Baseline to 24 months follow up
Thermography
Infrared thermography could be a simple tool to detect early inflammation of joints and joint associated structures. FLIR ONE imaging camera is used to take thermal images.
Measured: Baseline to 24 months follow up
Secondary outcomes (7)
Hemophilia activities list (HAL)
The HAL measures the impact of hemophilia on self-perceived functional abilities in 7 domains, including Lying/sitting/kneeling/standing, Functions of the legs , Functions of the arms, Use of transportation, Self-care, Household tasks, Leisure activities and sports.
Measured: Baseline to 24 months follow up
Hemophilia Quality of Life Questionnaire for Adults (HAEM-A-QOL)
The HAEM-A-QOL assess health-related quality of life in haemophilia patients with 10 domains, including physical health, feelings, view, sport\& leisure, work\& school, dealing, treatment, future, family planning, and partnership.
Measured: Baseline to 24 months follow up
Quality of life assessment (EQ-5D) - Descriptive System
The EQ-5D measures generic health status with 2 components: descriptive system and visual analogue scale. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression.
Measured: Baseline to 24 months follow up
Quality of life assessment (EQ-5D) - Visual Analogue Scale (VAS)
The EQ-5D measures generic health status with 2 components: descriptive system and visual analogue scale. The visual analogue scale (VAS) records the patient's self-rated health on a vertical visual analogue scale
Measured: Baseline to 24 months follow up
Serum markers: cartilage oligomeric matrix protein (COMP)
COMP is considered a marker of cartilage breakdown, and is diagnostic of arthritis and to correlate with the disease severity. Serum COMP level is indicative of the amount of joint damage in patients with hemophilic arthropathy.
Measured: Baseline to 24 months follow up
Serum markers: chondroitin-sulphate aggrecan turnover 846 epitope (CS846)
CS846 is a sensitive biomarker reflecting degradation of cartilage and synovial tissues. Serum CS-846 levels is indicative of the amount of joint damage in patients with hemophilic arthropathy.
Measured: Baseline to 24 months follow up
6 mins walk test
The 6 mins walk test (MWT) measures the distance an individual could walk over six minutes on a hard, flat surface. The test could be used as a performance-based measure of functional exercise capacity.
Gait Analysis (RehaWatch): RehaWatch is based on inertial sensors that allow the quantitative measurement of the kinemetic variables
Measured: Baseline to 24 months follow up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2020
- Study start20 Jul 2020
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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
- Other
- Time perspective
- Other
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Taipei Medical University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04618237
- Sponsor's protocol ID
- N202001021
- Status last verified
- Sep 2020
- First submitted
- 23 Sep 2020
- Last updated
- 5 Nov 2020
- Results posted
- 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 5 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
- ClinicalTrials.gov ID
- NCT04618237
- Acronym / sponsor ID
- N202001021
- Conditions
- Hemophilia, Arthropathy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Taipei Medical University HospitalUniversity, hospital or other
- Collaborators
- Sanofi
- Study start
- 20 Jul 2020 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 5 Nov 2020 (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.
Patients with severe hemophilia A can experience repeated bleeding into the same joint (ie, a target joint; most frequently in the ankle, knee, and elbow), which can contribute to hemophilic arthropathy and, over time, ultimately result in chronic pain, functional limitations, deformities, reduced joint of motion and decreased quality of life.
Early use of prophylaxis is recommended following diagnosis of severe hemophilia A to maintain joint health and prevent joint destruction. Eloctate is produced using a human cell line and an addition of an Fc fusion protein to recombinant FVIII (rFVIIIFc) with prolonged half-life and was launched in Taiwan from Nov. 2018. The pivotal studies of rFVIIIFc show that patients maintained a low bleeding rate, with most experiencing a median annualized bleeding rate (ABR) of 0 and 97% of target joints were resolved across adult, adolescent, and pediatric subjects during 4 years of prophylaxis rFVIIIFc treatment. However, in Taiwan we are still lacking the real world treatment outcome data on rFVIIIFc, especially for the joint health evidence in Asian Hemophilia A patients.
Therefore the objective of this study is to evaluate the effectiveness of rFVIIIFc treatment on joint health over a long observational period of 36 month focused on physical and functional changes in hemophilia A patients.
Detailed description
Thermographic assessment has been proposed to be a potential tool in evaluating the inflammatory arthritis patients. Thermography has advantages of simple, time-saving, low skill demanding, no radiation exposure and non-invasiveness. It is possible to conduct a quantitative analysis with thermography. However, it is still unknown whether thermography evaluation can provide clinical information regarding the joint health in hemophilia arthropathy patients. The project will focus on:
- Investigate the association among thermography findings and functional status in hemophilia arthroplasty
- Investigate the novel biomarkers in predicting function in hemophilia arthropathy including: soft tissue ultrasound, body composition, thermographic findings of joints, muscle composition and serum biomarkers of joint degeneration.
Study design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Ages
- 20 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 35 severe Hemophilia A patients
Key inclusion criteria (3 listed)
- Willing to sign inform consent form and complied with study procedure
- Patient received confirmative diagnosis of Hemophilia A Disease
- Severe hemophilia A patient currently who are on treatment with Eloctate prior to enrolment visit (Note: Prescription based on physician's clinical judgement and discussion with patients)
Key exclusion criteria (6 listed)
- Unwilling to sign inform consent form.
- History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
- History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
- Significantly impaired vision / hearing
- Cannot communicate in Mandarin Chinese
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
Hemophilia Joint Health Score (HJHS)
Time frame: Baseline to 24 months follow up
The HJHS measures joint health, in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in hemophilia: the knees, ankles, and elbows. The HJHS provides a total score (higher score is worse; max=124), joint specific scores, and a global gait score.
Musculoskeletal ultrasound/HEAD-US scoring system (Haemophilia Early Arthropathy Detection by UltraSound)
Time frame: Baseline to 24 months follow up
Musculoskeletal ultrasound could detect soft tissue alterations and be used as early detection of hemophilic arthropathy. Ultrasound assessment of bilateral knees and ankles, and bilateral gastrocnemius and quadriceps would be taken out in this study and scored with HEAD-US(Haemophilia Early Arthropathy Detection by UltraSound) scoring system. The scoring was based on three markers: synovitis (score 0-2), cartilage (score 0-4) and subchondral bone (score of 0-2) with a maximum score of eight points per joint, higher score is worse.
X-rays/Pettersson score (PS)
Time frame: Baseline to 24 months follow up
X-rays are used to evaluate structural changes in joints of patients with hemophilic arthropathy. X-rays images of bilateral knees and ankles will be taken in this study and scored with Pettersson scoring system. The maximum possible score for a given joint is 13 points and higher score is worse.
Thermography
Time frame: Baseline to 24 months follow up
Infrared thermography could be a simple tool to detect early inflammation of joints and joint associated structures. FLIR ONE imaging camera is used to take thermal images.
7 secondary outcomes
Hemophilia activities list (HAL)
Time frame: Baseline to 24 months follow up
Hemophilia Quality of Life Questionnaire for Adults (HAEM-A-QOL)
Time frame: Baseline to 24 months follow up
Quality of life assessment (EQ-5D) - Descriptive System
Time frame: Baseline to 24 months follow up
Quality of life assessment (EQ-5D) - Visual Analogue Scale (VAS)
Time frame: Baseline to 24 months follow up
Serum markers: cartilage oligomeric matrix protein (COMP)
Time frame: Baseline to 24 months follow up
Serum markers: chondroitin-sulphate aggrecan turnover 846 epitope (CS846)
Time frame: Baseline to 24 months follow up
6 mins walk test
Time frame: Baseline to 24 months follow up
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2020.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia and Arthropathy, run by Taipei Medical University Hospital at 1 site in Taiwan.
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 6 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: status not confirmed, as of the sponsor's last update on 5 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. 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?
Results are not available in the source record.
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?
Hemophilia Joint Health Score (HJHS) (time frame: Baseline to 24 months follow up). The study lists 4 primary outcomes in all. 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 5 Nov 2020.