Ignite Creation Date:
2024-05-06 @ 3:23 PM
Last Modification Date:
2024-10-26 @ 1:48 PM
Study NCT ID:
NCT04618237
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2020-11-05
First Post:
2020-09-23
Brief Title:
Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
Sponsor:
Taipei Medical University Hospital
Organization:
Taipei Medical University Hospital
Study Overview
Official Title:
Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2020-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None