An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|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
- Study Details
- Tabular View
- Results Submitted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Friedreich's Ataxia||Drug: Acetyl-L-Carnitine||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Open-label administration of Acetyl-L-Carnitine, up to 2 g/day for 24 months.
Acetyl-L-Carnitine, 2 g/day, up to 24 months.
Other Name: ALCAR
- 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 ]
- 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 ]
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, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years to 80 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
- Friedreich'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 Friedreich'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).
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
|Contact: Tanya Aranca, BSfirstname.lastname@example.org|
|Contact: Jessica Shaw, MPHemail@example.com|
|United States, Florida|
|University of South Florida||Recruiting|
|Tampa, Florida, United States, 33612|
|Contact: Tanya Aranca, B.S. 813-974-5909 firstname.lastname@example.org|
|Contact: Jessica Shaw, MPH 813-974-5909 email@example.com|
|Principal Investigator: Theresa Zesiewicz, M.D.|
|Principal Investigator:||Theresa A Zesiewicz, MD||University of South Florida|
|Responsible Party:||Theresa Zesiewicz, Professor, Director of USF Ataxia Research Center, University of South Florida|
|Other Study ID Numbers:||
|First Posted:||August 13, 2013 Key Record Dates|
|Last Update Posted:||April 12, 2016|
|Last Verified:||April 2016|
Nervous System Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Vitamin B Complex
Physiological Effects of Drugs