Observational Study of Long-Term Joint Health Outcome in Hemophilia

Status Enrolling by invitation

Clinical 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 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04618237
Study type	Observational
Source	Taipei Medical University Hospital
Contact
Status	Enrolling by invitation
Phase
Start date	July 20, 2020
Completion date	December 31, 2024

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05617209` - In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
Completed	`NCT05039008` - Restricting Blood Flow in Improving Muscle Strength in Patients With Hemophilic Arthropathy	N/A
Recruiting	`NCT04398628` - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed	`NCT02546622` - ATHN 2: Factor Switching Study
Terminated	`NCT02586012` - Weight-based Dosing in Hemophilia A	Phase 2
Completed	`NCT02165462` - Bilateral Deficit Phenomenon in Patients With Haemophilic Arthropathy	N/A
Unknown status	`NCT02165592` - Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia	N/A
Unknown status	`NCT02433782` - Myofascial Therapy in Patients With Hemophilic Arthropathy	N/A
Completed	`NCT01232634` - Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles	Phase 2
Completed	`NCT05104164` - Self-myofascial Release in Hemophilic Ankle Arthropathy	N/A
Terminated	`NCT01191372` - First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients	Phase 1
Completed	`NCT05173129` - Posture Analysis for Patients With Haemophilia	N/A
Completed	`NCT03818529` - ATHN 8: Previously Untreated Patients (PUPs) Matter Study
Withdrawn	`NCT03996486` - Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia	Phase 1
Completed	`NCT01708564` - A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B	Phase 1
Completed	`NCT03842605` - Efficacy of Strength Training in Improving Elbow Range of Motion and Function in Adults With Hemophilia	N/A
Completed	`NCT05549843` - Manual Therapy in the Treatment of Hemophilic Arthropathy of the Ankle	N/A
Recruiting	`NCT06010953` - SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment	Phase 1/Phase 2
Completed	`NCT05027230` - A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor	Phase 1/Phase 2
Not yet recruiting	`NCT06014320` - Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms