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Study on the Effects of Acetylcarnitine Hydrochloride in Patients with Amyotrophic Lateral Sclerosis (ALS)

Fast replyRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a treatment for amyotrophic lateral sclerosis (ALS), a progressive disease that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. The treatment being tested is called acetyl-L-carnitine, which is a chemical compound that may help slow down the progression of ALS. The study will compare two different dosages of acetyl-L-carnitine: 1.5 grams per day and 3 grams per day. Additionally, some participants will receive a placebo to help understand the treatment's effects better.

The purpose of the study is to assess how effective acetyl-L-carnitine is in slowing down the loss of self-sufficiency in people with ALS. Participants will be randomly assigned to one of the treatment groups or the placebo group. The study will last for 48 weeks, during which participants will be monitored to see how well they maintain their ability to perform daily activities, such as swallowing, cutting food, handling utensils, and walking. The study will also look at changes in quality of life and other health markers over this period.

Throughout the study, researchers will collect information on any side effects experienced by participants and measure various health indicators to understand the treatment's impact. The goal is to determine whether acetyl-L-carnitine can help people with ALS maintain their independence and improve their quality of life. This research could provide valuable insights into new ways to manage ALS and support those living with the condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Randomization and baseline visit
Upon joining the study, participants are randomly assigned to one of the treatment groups. This process is known as randomization.
During the baseline visit, initial assessments are conducted to establish a starting point for measuring changes over time.
Step 2
Treatment administration
Participants receive either acetyl-L-carnitine or a placebo in the form of an oral solution.
Two dosages of acetyl-L-carnitine are tested: 1.5 grams per day and 3 grams per day.
The treatment is administered daily for a duration of 48 weeks.
Step 3
Ongoing assessments
Throughout the 48-week period, regular assessments are conducted to monitor the progression of functional disability using the ALSFRS-R scale.
Additional evaluations include measuring respiratory function, quality of life, and other health indicators.
Step 4
Primary endpoint evaluation
The primary endpoint is the proportion of participants who remain self-sufficient after 48 weeks, as determined by specific criteria on the ALSFRS-R scale.
Step 5
Secondary endpoint evaluation
Secondary endpoints include changes in ALSFRS-R total score, respiratory function, quality of life, and other health markers over the 48-week period.
The study also tracks the occurrence of adverse events and serious adverse events during the treatment period.
Step 6
Study completion
At the end of the 48-week treatment period, final assessments are conducted to evaluate the overall effects of the treatment.
The study is estimated to conclude by April 30, 2027.

Who can join the trial?

10 criteria

Must be **18 years or older**.
Must have **normal thinking abilities**, as determined by the study doctor.
Must have a diagnosis of **amyotrophic lateral sclerosis (ALS)** according to specific criteria known as the Gold Coast Criteria.
The disease must have started **24 months or less** ago, with symptoms like limb weakness or problems with speech and swallowing.
Must be able to take care of oneself, with **satisfactory bulbar and spinal function**. This means scoring 3 or more on a specific scale for swallowing, cutting food, handling utensils, and walking.
Must have **satisfactory breathing ability**, with a lung function test result of 80% or more of what is expected.
Must show a **documented progression of symptoms** as measured by a specific scale. The rate of disease progression must be 0.33 or higher, calculated using a formula involving the scale score and time since symptoms began.
Must be able to **understand and follow the study requirements**.
Must be able to **give written informed consent** personally or through a legally authorized representative.
Must have been treated with **riluzole**, a medication, at a dose of 50 mg twice a day for at least 4 weeks before the study starts.

Who cannot join the trial?

4 criteria

Patients who do not have amyotrophic lateral sclerosis (ALS) cannot participate. ALS is a disease that affects the nerve cells in the brain and spinal cord, leading to muscle weakness and loss of movement.
Patients who are not within the specified age range cannot participate. The age range for this study is typically adults, but the exact ages are not specified here.
Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.
Patients who do not meet the specific health and medical criteria set by the study cannot participate. These criteria are not detailed here.

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Verified sites

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Verified
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
Responsive
IRCCS Ospedale Auxologico San Luca
Milan, Italy
Responsive
Universita Degli Studi Di Roma La Sapienza
Rome, Italy

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Investigated drugs

Acetyl-L-carnitine (ALCAR) is being tested in this clinical trial to see if it can help slow down the progression of functional disability in people with ALS (Amyotrophic Lateral Sclerosis). ALS is a condition that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. The trial aims to find out if ALCAR can help maintain self-sufficiency in patients by measuring their abilities using a specific scale.

ACETYLCARNITINE HYDROCHLORIDE

What is already known about the treatment

Acetyl-L-carnitine – This medication is administered orally in the form of tablets or capsules. It is currently being studied in clinical trials for its potential benefits in treating amyotrophic lateral sclerosis (ALS). The main therapeutic indication is to slow the progression of functional disability in ALS patients. At the molecular level, acetyl-L-carnitine is believed to work by supporting energy production in cells and protecting nerve cells from damage. It is classified as a dietary supplement with potential neuroprotective properties.

Investigated diseases

Amyotrophic Lateral Sclerosis – This is a progressive neurological disease that affects nerve cells in the brain and spinal cord. It leads to the gradual degeneration and death of motor neurons, which are responsible for controlling voluntary muscles. As the disease progresses, individuals experience increasing muscle weakness and atrophy, affecting their ability to move, speak, swallow, and eventually breathe. The progression of symptoms varies among individuals, but it typically starts with muscle twitching and weakness in a limb or slurred speech. Over time, the disease spreads to other parts of the body, leading to more severe physical limitations. Despite the loss of muscle function, the disease does not usually affect the senses or cognitive abilities.