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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by University of South Florida
Sponsor:
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida
ClinicalTrials.gov Identifier:
NCT01921868
First received: July 3, 2013
Last updated: March 18, 2014
Last verified: March 2014
  Purpose

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 Intervention
Friedreich's Ataxia
Drug: Acetyl-L-Carnitine

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Changes in patient global impression of improvement [ Time Frame: Every 6 months, up to 24 months ] [ Designated as safety issue: No ]
  • Changes in Time 25-foot Walk [ Time Frame: Every 6 months, up to 24 months ] [ Designated as safety issue: No ]
  • Changes in frequency and severity of adverse events [ Time Frame: Every 6 months, up to 24 months ] [ Designated as safety issue: Yes ]
  • Changes in patient quality of life (SF-36) [ Time Frame: Every 6 months, up to 24 months ] [ Designated as safety issue: No ]
  • Changes in clinical global impression of improvement [ Time Frame: Every 6 months, up to 24 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: August 2013
Estimated Primary Completion Date: August 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Acetyl-L-Carnitine Drug: Acetyl-L-Carnitine
Other Name: ALCAR

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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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  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01921868

Contacts
Contact: Brittany Golden, B.A. 813-974-9413 bgolden1@health.usf.edu
Contact: Jessica Shaw, M.P.H. 813-974-5633 jshaw@health.usf.edu

Locations
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
Contact: Brittany Golden, B.A.    813-974-9413    bgolden1@health.usf.edu   
Principal Investigator: Theresa Zesiewicz, M.D.         
Sponsors and Collaborators
University of South Florida
Investigators
Principal Investigator: Theresa A Zesiewicz, MD University of South Florida
  More Information

No publications provided

Responsible Party: Theresa Zesiewicz, Professor, Director of USF Ataxia Research Center, University of South Florida
ClinicalTrials.gov Identifier: NCT01921868     History of Changes
Other Study ID Numbers: ALCAR-8499
Study First Received: July 3, 2013
Last Updated: March 18, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Friedreich Ataxia
Ataxia
Brain Diseases
Central Nervous System Diseases
Cerebellar Diseases
Dyskinesias
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolic Diseases
Mitochondrial Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Signs and Symptoms
Spinal Cord Diseases
Spinocerebellar Degenerations
Acetylcarnitine
Carnitine
Central Nervous System Agents
Growth Substances
Micronutrients
Nootropic Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses
Vitamin B Complex
Vitamins

ClinicalTrials.gov processed this record on October 21, 2014