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

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ClinicalTrials.gov Identifier: NCT01921868
Recruitment Status : Unknown
Verified April 2016 by Theresa Zesiewicz, University of South Florida.
Recruitment status was:  Recruiting
First Posted : August 13, 2013
Last Update Posted : April 12, 2016
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida

Brief 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.

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: Acetyl-L-Carnitine Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
Study Start Date : August 2013
Estimated Primary Completion Date : June 2017
Estimated Study Completion Date : June 2017

Arm Intervention/treatment
Experimental: Acetyl-L-Carnitine
Open-label administration of Acetyl-L-Carnitine, up to 2 g/day for 24 months.
Drug: Acetyl-L-Carnitine
Acetyl-L-Carnitine, 2 g/day, up to 24 months.
Other Name: ALCAR

Primary Outcome Measures :
  1. To observe the changes in cardiac functioning in patients with Friedreich's Ataxia between study endpoint and baseline. [ Time Frame: Every 12 months, up to 24 months ]
  2. To assess the changes in Friedreich's Ataxia symptoms and severity (as measured by clinical rating scales) compared to baseline. [ Time Frame: Every 6 months, up to 24 months ]

Secondary Outcome Measures :
  1. Changes in patient global impression of improvement [ Time Frame: Every 6 months, up to 24 months ]
  2. Changes in Time 25-foot Walk [ Time Frame: Every 6 months, up to 24 months ]
  3. Changes in frequency and severity of adverse events [ Time Frame: Every 6 months, up to 24 months ]
  4. Changes in patient quality of life (SF-36) [ Time Frame: Every 6 months, up to 24 months ]
  5. Changes in clinical global impression of improvement [ Time Frame: Every 6 months, up to 24 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  11. Presence of severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (AST or Alanine transaminase(ALT)>2x times normal) (as evidenced by labs reported within the past 6 months).
  12. Clinically significantly abnormal white blood cell, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
  13. Subjects with blood work showing carnitine deficiency (<60nmol/mg total carnitine in the urine or <35umol/L total carnitine in the plasma).


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01921868

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Contact: Tanya Aranca, BS 813-974-5909 taranca@health.usf.edu
Contact: Jessica Shaw, MPH 813-974-5909 jshaw@health.usf.edu

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United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
Contact: Tanya Aranca, B.S.    813-974-5909    taranca@health.usf.edu   
Contact: Jessica Shaw, MPH    813-974-5909    jshaw@health.usf.edu   
Principal Investigator: Theresa Zesiewicz, M.D.         
Sponsors and Collaborators
University of South Florida
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Principal Investigator: Theresa A Zesiewicz, MD University of South Florida
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Responsible Party: Theresa Zesiewicz, Professor, Director of USF Ataxia Research Center, University of South Florida
ClinicalTrials.gov Identifier: NCT01921868    
Other Study ID Numbers: ALCAR-8499
First Posted: August 13, 2013    Key Record Dates
Last Update Posted: April 12, 2016
Last Verified: April 2016
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Vitamin B Complex
Physiological Effects of Drugs
Nootropic Agents