Ignite Creation Date:
2025-12-24 @ 10:16 PM
Ignite Modification Date:
2026-07-18 @ 2:52 AM
Study NCT ID:
NCT02120235
Status:
UNKNOWN
Last Update Posted:
2017-04-04
First Post:
2014-04-17
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Investigating Lysosomal Storage Diseases in Minority Groups
Sponsor:
O & O Alpan LLC
Organization:
Study Overview
Official Title:
None
Status:
UNKNOWN
Status Verified Date:
2017-04
Last Known Status:
RECRUITING
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:
Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US. Frequently, patients show progressive worsening of symptoms for several years before they get diagnosed. Since many of these diseases can be managed therapeutically, it is important to identify and treat patients in order to avoid organ damage. The investigators aim to undertake a screening study that identifies undiagnosed patients with lysosomal storage disorders and determine the prevalence of these diseases with special focus on underrepresented minority groups.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: