Ignite Creation Date:
2025-12-25 @ 2:36 AM
Ignite Modification Date:
2026-01-08 @ 4:24 PM
Study NCT ID:
NCT02497534
Status:
RECRUITING
Last Update Posted:
2025-09-23
First Post:
2015-07-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Biomarkers in Friedreich's Ataxia
Sponsor:
University of Florida
Organization:
Study Overview
Official Title:
Biomarkers in Friedreich's Ataxia
Status:
RECRUITING
Status Verified Date:
2025-09
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:
The purpose of this project is to characterize measures of cardiac performance and neuromuscular physiology in FA patients using novel techniques, including echocardiography and magnetic resonance imaging (MRI), metabolic exercise testing, and neurophysiological outcomes.
Detailed Description:
Friedreich's ataxia (FA) is an autosomal recessive disease caused by a mutation in the frataxin gene (FXN). Although rare, FA is the most common form of hereditary ataxia, affecting 1 in every 50,000 people in the United States. Currently, palliative therapies are the only treatment for FA patients. However, current gene therapy efforts in other neuromuscular diseases have positioned the investigator's research program to extend these discoveries and techniques to FA. As new therapies become available for clinical application, it is crucial to identify non-invasive outcomes measures of cardiac and neuromuscular performance with adequate sensitivity to detect the impact of treatments.
Study Oversight
Has Oversight DMC:
False
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| UL1TR000064 | NIH | None | https://reporter.nih.gov/quic… | View |