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

What Was Tested

The trial (not phased) is built around L-Carnitine.

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.

It had 1 arm (groups): Acetyl-L-Carnitine (experimental).

The registry classes the interventions as drug.

More on the Plan

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.

Who Took Part

Enrollment: 20 planned participants were the target.

It was open to people of any sex aged from 18 years up to 80 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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.

The full record lists 7 inclusion and 13 exclusion criteria; among the exclusions: pregnancy or lactation; any use of the investigational product within the past 30 days; legal incapacity or limited legal capacity.

How the Study Was Run

It was run as a single-group and open-label study.

Everyone knew when L-Carnitine was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2013-08 with completion listed for 2017-06.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: 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).

It also tracked 5 secondary outcomes, for example Changes in Time 25-foot Walk, Changes in frequency and severity of adverse events and Changes in patient quality of life (SF-36).

What This Means in Plain Language

Put simply, this is a not phased trial of L-Carnitine in 20 people.

Its size is small, so on its own it can only give an early look.

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the L-Carnitine compound page, all human trials.