NCT04618237

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

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 35 (estimated)
• Sponsor: Taipei Medical University Hospital · Academic / Other
• Sex: All
• Age: 20 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia
• Arthropathy

Timeline

• Start date: 2020-07-20
• Primary completion: 2023-12-31
• Completion: 2024-12-31
• First posted: 2020-11-05
• Last updated: 2020-11-05

Locations

1 site across 1 country: Taiwan