Trials / Completed
CompletedNCT02840669
A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich's Ataxia (CARFA Study)
- Status
- Completed
- Phase
- N/A
- Study type
- Interventional
- Enrollment
- 40 (actual)
- Sponsor
- Adverum Biotechnologies, Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Accepted
Summary
Friedreich's ataxia (FA) is an autosomal recessive disease with an incidence of 1/50,000 in the Caucasian population. The main manifestations of FA are progressive sensory and cerebellar ataxia and cardiomyopathy (CM). It is the most common form of inherited ataxia. A severe CM affects \~60% of FA patients, mostly young adults, and leads to cardiac failure then death. Currently, no therapy can change the course of this severe cardiomyopathy. This study is designed to characterize the cardiac manifestations of FA using cardiac magnetic resonance (CMR), echocardiography, serum cardiac biomarkers and evaluation of fatigue severity, in the context of the neurological disease.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| PROCEDURE | Cardiac magnetic resonance imaging (CMR) | |
| PROCEDURE | Exercise-stress test | |
| PROCEDURE | Echocardiography (ECHO) | |
| PROCEDURE | Cardiac-related blood studies |
Timeline
- Start date
- 2016-07-01
- Primary completion
- 2018-06-01
- Completion
- 2018-09-01
- First posted
- 2016-07-21
- Last updated
- 2018-12-10
Locations
1 site across 1 country: France
Source: ClinicalTrials.gov record NCT02840669. Inclusion in this directory is not an endorsement.