Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient

NCT04618237 | Unknown

• 1 other identifier: N202001021
• Study Type: observational
• Target: 35
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia; Arthropathy

Outcome Measures

Primary Outcomes (4)

• 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.

  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.

  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.

  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.

  Baseline to 24 months follow up

Secondary Outcomes (7)

• Hemophilia activities list (HAL)

  Baseline to 24 months follow up

• Hemophilia Quality of Life Questionnaire for Adults (HAEM-A-QOL)

  Baseline to 24 months follow up

• Quality of life assessment (EQ-5D) - Descriptive System

  Baseline to 24 months follow up

• Quality of life assessment (EQ-5D) - Visual Analogue Scale (VAS)

  Baseline to 24 months follow up

• Serum markers: cartilage oligomeric matrix protein (COMP)

  Baseline to 24 months follow up

Eligibility Criteria

• Age: 20 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

35 severe Hemophilia A patients

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Taipei Medical University Hospital: lead
• Sanofi: collaborator

Study Sites (1)

Taipei Medical University

Taipei, Taiwan

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Sample will be kept in ultra low temp. freezer and given subject numbers as identifiers.

MeSH Terms

Conditions

Hemophilia A; Joint Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Musculoskeletal Diseases

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2020
• First Posted: November 5, 2020
• Study Start: July 20, 2020
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2024
• Last Updated: November 5, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1)