Clinical trial NCT04618237: Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
Official title: Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Hemophilia
Arthropathy
Lead sponsor: Taipei Medical University Hospital
Sponsor class: OTHER
Collaborators: name: Sanofi
First posted: 2020-11-05
Enrollment: 35
Sex: ALL
Minimum age (years): 20
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 35 severe Hemophilia A patients

Brief 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 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:

1. Investigate the association among thermography findings and functional status in hemophilia arthroplasty
2. 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.

Eligibility criteria: Inclusion Criteria:

1. Willing to sign inform consent form and complied with study procedure
2. Patient received confirmative diagnosis of Hemophilia A Disease
3. 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)

Exclusion Criteria:

1. Unwilling to sign inform consent form.
2. History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
3. History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
4. Significantly impaired vision / hearing
5. Cannot communicate in Mandarin Chinese
6. Recent joint bleeding and trauma 3 months prior to study

Primary outcomes:
measure: Hemophilia Joint Health Score (HJHS)
timeFrame: Baseline to 24 months follow up
measure: Musculoskeletal ultrasound/HEAD-US scoring system (Haemophilia Early Arthropathy Detection by UltraSound)
timeFrame: Baseline to 24 months follow up
measure: X-rays/Pettersson score (PS)
timeFrame: Baseline to 24 months follow up
measure: Thermography
timeFrame: Baseline to 24 months follow up
Secondary outcomes:
measure: Hemophilia activities list (HAL)
timeFrame: Baseline to 24 months follow up
measure: Hemophilia Quality of Life Questionnaire for Adults (HAEM-A-QOL)
timeFrame: Baseline to 24 months follow up
measure: Quality of life assessment (EQ-5D) - Descriptive System
timeFrame: Baseline to 24 months follow up
measure: Quality of life assessment (EQ-5D) - Visual Analogue Scale (VAS)
timeFrame: Baseline to 24 months follow up
measure: Serum markers: cartilage oligomeric matrix protein (COMP)
timeFrame: Baseline to 24 months follow up
measure: Serum markers: chondroitin-sulphate aggrecan turnover 846 epitope (CS846)
timeFrame: Baseline to 24 months follow up
measure: 6 mins walk test
timeFrame: Baseline to 24 months follow up
Other outcomes:
Locations:
city: Taipei
country: Taiwan
facility: Taipei Medical University
Study officials:
References: