Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06512571
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-16
Brief Title:
CVI Alterations in FD a Prospective Multicenter Observational Cohort Study
Sponsor:
None
Organization:
None
Study Overview
Official Title:
The Characteristics of Cardiovascular Imaging Alterations in Patients With Fabry Cardiomyopathy a Prospective Multicenter Observational Cohort Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Fabry disease FD is a rare X-linked lysosomal storage disorder caused by α-galactosidase A GLA gene mutations leading to reduced or undetectable α galactosidase A α-Gal A enzyme activity resulting in progressive accumulation of globotriaosylceramide GL3 and its deacylated form globotriaosylsphingosine Lyso-GL-3 in multiple organs causing neural renal cardiac dermatological gastrointestinal and ophthalmic manifestations even leading to life-threatening complications Cardiovascular and cerebrovascular complications ie heart failure stroke etc or end-stage renal disease even premature death can be seen in severe cases The life expectancy of male patients is reduced by 1520 years while that of female patients is reduced by 610 years
The exact prevalence of FD is currently unknown Based on an estimated prevalence of 160000 there are approximately 23000 affected FD patients in China The clinical manifestations of FD are diverse and non-specific which may lead to misdiagnosis in patients with non-typical clinical manifestations in the absence of a family history of FD Cardiac involvement can be recognized in up to 68 patients with FD significantly higher than in other organs and the positive screening rate for FD in adults with unexplained left ventricular hypertrophy LVHhypertrophic cardiomyopathy was 09
Cardiovascular disease is the leading cause of death in patients with FD cardiomyopathy 402 The 2020 Expert Consensus Document on the Management of Cardiovascular Manifestations of Fabry Disease recommends early screening in patients with suspected LVH for early diagnosis Therefore strengthened screening strategy in high-risk patients with LVH will improve the diagnosis and treatment of FD in China
The exact prevalence of FD is currently unknown Based on an estimated prevalence of 160000 there are approximately 23000 affected FD patients in China The clinical manifestations of FD are diverse and non-specific which may lead to misdiagnosis in patients with non-typical clinical manifestations in the absence of a family history of FD Cardiac involvement can be recognized in up to 68 patients with FD significantly higher than in other organs and the positive screening rate for FD in adults with unexplained left ventricular hypertrophy LVHhypertrophic cardiomyopathy was 09
Cardiovascular disease is the leading cause of death in patients with FD cardiomyopathy 402 The 2020 Expert Consensus Document on the Management of Cardiovascular Manifestations of Fabry Disease recommends early screening in patients with suspected LVH for early diagnosis Therefore strengthened screening strategy in high-risk patients with LVH will improve the diagnosis and treatment of FD in China
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None