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An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia | Clinical Trials | Clareo Health

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An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia

NCT01921868•University of South Florida

View on ClinicalTrials.gov

Summary

The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedreich's Ataxia as well as how it may affect other symptoms of Friedreich's Ataxia such as difficulties with balance, walking, or upper arm function.

Detailed Description

This study is an open label, pilot study of ALCAR in subjects with FA. In this study 20 patients with FA will receive ALCAR every day for 24 months. At the study endpoint, subjects will be assessed for changes in cardiovascular outcomes and FA symptoms. To determine the effects of LC on changes in cardiomyopathy, echocardiography with strain rate will be calculated. This technique has been validated in clinical studies and used in other studies of FA patients for the comparison of regional deformation and myocardial wall thickness.

Eligibility

Age

18 - 80 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Outpatients with Friedreich's Ataxia diagnosed by a movement disorder specialist and confirmed by genetic testing (of the patient or in a first degree relative of the patient). Subject may be non-ambulatory.
•2. Age 18 years to 80 years.
•3. Stable medical condition for 3 months prior to screening.
•4. Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
•5. If on cardiac medications such as beta-blockers or ace inhibitors, patients must be on a stable dose for 6 months prior to study entry and for the duration of the study.
•6. Friedreich's Ataxia patients with systolic or diastolic dysfunction present on echocardiogram and ejection fraction between 35% - 65% at screening.
•7. Subjects with ejection fractions \< 50% need to be stable and on optimal heart failure therapy for at least 2 weeks prior to screening.

Exclusion Criteria

•1. Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).
•2. Pregnancy or lactation.
•3. Concurrent participation in another clinical study where use of an investigational product is used. Subjects who are currently enrolled in the Friedreich's Ataxia Clinical Outcome Measures Study at any site will be allowed to enroll in this study as well.
•4. Any use of the investigational product within the past 30 days.
•5. Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 25).
•6. Legal incapacity or limited legal capacity.
•7. History of stroke.
•8. Subjects with a history of thyroid disease (hypothyroidism). Clinical laboratory evaluations of thyroid stimulating hormone levels taken 3 months prior to the study or at screening will be used to confirm absence of current thyroid problems.
•9. Subjects with a history of seizures.
•10. Subjects taking warfarin or acenocoumarol.
+4 more criteria

Locations (1)

University of South Florida

Tampa, Florida, United States

Key Dates

Start Date

August 1, 2013

Primary Completion Date

June 1, 2017

Completion Date

June 1, 2017

Last Updated

April 12, 2016

Enrollment

ESTIMATED participants

Conditions

Friedreich's Ataxia

Interventions

Acetyl-L-Carnitine

DRUG

Contacts

Tanya Aranca, BS

CONTACT

813-974-5909 taranca@health.usf.edu

Jessica Shaw, MPH

CONTACT

813-974-5909 jshaw@health.usf.edu

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 12, 2016Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia

Inclusion Criteria

Exclusion Criteria

Biomarkers in Friedreich's Ataxia

Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)

Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)

Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia