In short
Clinical trials are investigating ACETYLCARNITINE HYDROCHLORIDE in people with amyotrophic lateral sclerosis (ALS). These studies aim to measure whether it can help slow loss of function and improve self-sufficiency, while also looking at treatment effects in patient groups with this disease.
Key points
- Clinical trials of ACETYLCARNITINE HYDROCHLORIDE are studying people with amyotrophic lateral sclerosis (ALS). The main trial is a randomized study with a placebo group and planned enrollment of 246 participants. It is described as Phase 4 and also as a phase II/III trial, showing that researchers are testing clinical effects in patients. The main goal is to see whether more people stay self-sufficient after 48 weeks. The study uses the ALSFRS-R scale to measure daily function, including swallowing, cutting food, handling utensils, and walking.
Trial overview
The available clinical trial is studying ACETYLCARNITINE HYDROCHLORIDE in people with amyotrophic lateral sclerosis (ALS), a disease that affects nerve cells and muscle function. The study is titled as a randomized phase II/III trial on the biological and clinical effects of acetyl-L-carnitine in ALS, and it is listed as authorised.
This research is focused on whether the treatment can help with the progression of functional disability, which means loss of the ability to do everyday tasks. The trial also looks at self-sufficiency, or how well a person can manage daily activities on their own.
Who is being studied
The target population is people with amyotrophic lateral sclerosis. The source data does not give more detailed rules for who can join, such as age, sex, or disease stage, so those details cannot be added here.
The study plans to enroll 246 participants. This number gives the researchers a group large enough to compare outcomes between treatment and placebo groups.
Study design and phase
This is an interventional study, which means the researchers give a treatment and then measure the results. It is described as randomized, so participants are assigned to groups by chance rather than by choice.
The phase information in the source is listed as Phase 4, and the title also describes the trial as phase II/III. This shows that the study is examining clinical effects in people and is not just an early test in the lab.
What is being measured
The main outcome is the proportion of participants who remain self-sufficient after 48 weeks. In this study, self-sufficiency is defined using ALSFRS-R items for swallowing, cutting food, handling utensils, and walking, and participants must score three or higher in all three items listed in the source description.
The brief summary says the trial is assessing the effect of ACETYLCARNITINE HYDROCHLORIDE on progression of functional disability, measured by the ALSFRS-R scale. The ALSFRS-R is a standard rating tool used in ALS research to track daily function over time.
Treatment groups and doses
The study includes a placebo group and an active treatment group using Nicetile 500 mg polvere per soluzione orale, which is ACETYLCARNITINE HYDROCHLORIDE. The summary also says that two dosages are being tested: 1.5 g/day and 3 g/day.
A placebo is a comparison treatment that does not contain the active study drug. Comparing the active treatment with placebo helps researchers see whether the study drug changes outcomes more than no active treatment.
Patient-friendly terms
- Functional disability means difficulty doing normal daily activities because of illness.
- Self-sufficiency means being able to manage important daily tasks without help.
- Swallowing, cutting food, handling utensils, and walking are everyday tasks used in the ALSFRS-R scale to measure function.
- Randomized means the study assigns people to groups by chance, which helps keep the comparison fair.
- Placebo means a non-active comparison treatment used in research studies.