An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
This study is not yet open for participant recruitment.
Verified August 2013 by University of South Florida
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida
First received: July 3, 2013
Last updated: August 9, 2013
Last verified: August 2013
The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedrich's Ataxia as well as how it may affect other symptoms of Friedrich's Ataxia such as difficulties with balance, walking, or upper arm function.
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
Primary Outcome Measures:
- 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 ] [ Designated as safety issue: Yes ]
- 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 ] [ 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:
| Study Start Date:
| Estimated Primary Completion Date:
||August 2016 (Final data collection date for primary outcome measure)
Other Name: ALCAR
|Ages Eligible for Study:
||18 Years to 80 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- Outpatients with Friedrich'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.
- Age 18 years to 80 years.
- Stable medical condition for 3 months prior to screening.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- 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.
- Friedrich's Ataxia patients with systolic or diastolic dysfunction present on echocardiogram and ejection fraction between 35% - 65% at screening.
- Subjects with ejection fractions < 50% need to be stable and on optimal heart failure therapy for at least 2 weeks prior to screening.
- 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).
- Pregnancy or lactation.
- Concurrent participation in another clinical study where use of an investigational product is used. Subjects who are currently enrolled in the Friedrich's Ataxia Clinical Outcome Measures Study at any site will be allowed to enroll in this study as well.
- Any use of the investigational product within the past 30 days.
- Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 25).
- Legal incapacity or limited legal capacity.
- History of stroke.
- 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.
- Subjects with a history of seizures.
- Subjects taking warfarin or acenocoumarol.
- 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).
- Clinically significantly abnormal white blood cell, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
Subjects with blood work showing carnitine deficiency (<60nmol/mg total carnitine in the urine or <35umol/L total carnitine in the plasma).
Please refer to this study by its ClinicalTrials.gov identifier: NCT01921868
|University of South Florida
|Tampa, Florida, United States, 33612 |
|Principal Investigator: Theresa Zesiewicz, M.D. |
University of South Florida
||Theresa A Zesiewicz, MD
||University of South Florida
No publications provided
||Theresa Zesiewicz, Professor, Director of USF Ataxia Research Center, University of South Florida
History of Changes
|Other Study ID Numbers:
|Study First Received:
||July 3, 2013
||August 9, 2013
||United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on December 04, 2013
Nervous System Diseases
Signs and Symptoms
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Central Nervous System Agents
Vitamin B Complex
Physiological Effects of Drugs