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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
Sponsor:
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida

Tracking Information
First Submitted Date  ICMJE July 3, 2013
First Posted Date  ICMJE August 13, 2013
Last Update Posted Date April 12, 2016
Study Start Date  ICMJE August 2013
Estimated Primary Completion Date June 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 18, 2014)
  • 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 ]
  • 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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: August 9, 2013)
  • To observe the changes in cardiac functioning in patients with Friedrich's Ataxia between study endpoint and baseline. [ Time Frame: Every 12 months, up to 24 months ]
  • To assess the changes in Friedrich's Ataxia symptoms and severity (as measured by clinical rating scales) compared to baseline. [ Time Frame: Every 6 months, up to 24 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 9, 2013)
  • Changes in patient global impression of improvement [ Time Frame: Every 6 months, up to 24 months ]
  • Changes in Time 25-foot Walk [ Time Frame: Every 6 months, up to 24 months ]
  • Changes in frequency and severity of adverse events [ Time Frame: Every 6 months, up to 24 months ]
  • Changes in patient quality of life (SF-36) [ Time Frame: Every 6 months, up to 24 months ]
  • Changes in clinical global impression of improvement [ Time Frame: Every 6 months, up to 24 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
Official Title  ICMJE An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE Drug: Acetyl-L-Carnitine
Acetyl-L-Carnitine, 2 g/day, up to 24 months.
Other Name: ALCAR
Study Arms  ICMJE Experimental: Acetyl-L-Carnitine
Open-label administration of Acetyl-L-Carnitine, up to 2 g/day for 24 months.
Intervention: Drug: Acetyl-L-Carnitine
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: August 9, 2013)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2017
Estimated Primary Completion Date June 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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).

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Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01921868
Other Study ID Numbers  ICMJE ALCAR-8499
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Theresa Zesiewicz, University of South Florida
Study Sponsor  ICMJE University of South Florida
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Theresa A Zesiewicz, MD University of South Florida
PRS Account University of South Florida
Verification Date April 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP