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Study on Neural Stem Cell and Sodium Chloride Treatment for Patients with Amyotrophic Lateral Sclerosis

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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 a disease called Amyotrophic Lateral Sclerosis (ALS), which is a condition that affects the nerve cells in the brain and spinal cord, leading to muscle weakness and loss of muscle control. The trial will explore a treatment using human neural stem cells, which are special cells that have the potential to develop into different types of cells in the nervous system. These stem cells will be administered directly into the brain, a method known as intracerebral use. The study will also involve a comparison with a placebo to evaluate the effectiveness of the treatment.

The main goal of the study is to assess the safety of the treatment by monitoring any side effects that may occur during the study period. Participants will receive the treatment and be observed for any changes in their condition. The study will also look at how the treatment affects the progression of ALS and its impact on the quality of life of the participants. This will be done by measuring various health indicators and collecting data over a period of time.

Participants in the study will be monitored for several months to see how their condition changes with the treatment. The study will collect information on how the treatment affects the symptoms of ALS and whether it can slow down the progression of the disease. The results will help researchers understand the potential benefits and risks of using human neural stem cells as a treatment for ALS.

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
Joining the study
Upon joining the study, the patient provides written informed consent, confirming understanding and acceptance of the study procedures.
A caregiver must be present to assist with follow-up visits, even if the patient's mobility declines.
Step 2
Screening and baseline assessment
The patient undergoes a screening process to confirm eligibility, including a diagnosis of amyotrophic lateral sclerosis (ALS) and specific criteria such as age and disease progression.
Baseline assessments are conducted to evaluate the patient's health status and establish initial measurements for comparison throughout the study.
Step 3
Treatment administration
The patient receives treatment through intracerebral administration of human neural stem cells.
The treatment involves a placebo-controlled approach, meaning some patients receive the active treatment while others receive a placebo for comparison.
Step 4
Monitoring and follow-up
The patient is monitored for any adverse events, including reactions at the infusion site and changes in neurological function.
Regular follow-up visits are scheduled to assess the patient's health and the treatment's impact on disease progression.
Step 5
Evaluation of treatment effects
The study evaluates the change in disease progression using specific scales and measurements, comparing the treatment and placebo groups.
Biological activity of the treatment is assessed by measuring biomarkers in the patient's cerebrospinal fluid and serum.
Step 6
Completion of the study
The study is expected to conclude by June 25, 2026, with all data collected and analyzed to determine the treatment's safety and effectiveness.
The patient may be asked to participate in additional assessments to contribute to the study's final analysis.

Who can join the trial?

10 criteria

The patient must provide written informed consent, which means they agree to participate in the study after understanding all the details.
The patient needs to have a caregiver who can help them attend all follow-up visits, even if the patient's ability to move gets worse.
The patient must have a definite or probable diagnosis of **amyotrophic lateral sclerosis (ALS)** according to specific medical guidelines called the revised El Escorial criteria.
The patient should be between 18 and 65 years old.
The patient must have a **Forced Vital Capacity (FVC)** of at least 70%. FVC is a test that measures how much air a person can exhale after taking a deep breath.
The onset of the disease should be within the last 24 months.
The patient should have an **ALS Functional Rating Scale-Revised (ALSFRS-R)** score of at least 26. This scale measures the patient's ability to perform daily activities. The patient should score at least 2 on each of the first 9 items and at least 3 on items 10 to 12.
There should be evidence that the disease is progressing quickly. This means the patient's ALSFRS-R score should decrease by at least 1 point per month during a 12-week period before the study starts.
The patient should either be on a stable dose of **Riluzole** for more than 30 days before the study or not taking Riluzole at all, and they should not plan to start taking it during the study.
The patient must be medically able to handle a temporary treatment that lowers the immune system's activity, known as transient immunosuppression.

Who cannot join the trial?

3 criteria

Patients who do not have amyotrophic lateral sclerosis (ALS). ALS is a disease that affects the nerve cells in the brain and spinal cord.
Patients who are not within the specified age range for the study. The age range is not specified here, but it is important for eligibility.
Patients who are part of a vulnerable population. This means people who might need special protection or care, like children or those unable to make decisions for themselves.

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Verified sites

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Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, Italy
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Azienda Ospedaliera di Padova
Padua, Italy
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Azienda Ospedaliero-Universitaria Maggiore Della Carita
Novara, Italy

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Investigated drugs

Neural Stem Cells are being tested in this clinical trial for their potential to treat Amyotrophic Lateral Sclerosis (ALS). These are special cells that can develop into different types of cells in the brain and spinal cord. The trial is investigating whether these cells can help repair or replace damaged nerve cells in patients with ALS, potentially slowing the progression of the disease or improving symptoms. The main focus of the trial is to ensure that the use of these cells is safe for patients.

Sodium Chloride Ph. Eur.

What is already known about the treatment

Neural Stem Cell Treatment – This treatment involves the administration of human neural stem cells directly into the brain's ventricles, a method known as intracerebroventricular administration. Currently, it is being studied in clinical trials for its potential use in treating amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease. The main therapeutic goal is to assess the safety of this treatment, focusing on any adverse effects that may arise. At the molecular level, the mechanism involves the potential for these stem cells to differentiate into various types of neural cells, possibly aiding in the repair or replacement of damaged neurons. This treatment falls under the pharmacological classification of cell-based therapies.

Investigated diseases

Amyotrophic Lateral Sclerosis – Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative 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, leading to difficulties in speaking, swallowing, and eventually breathing. The progression of ALS varies among individuals, with some experiencing a rapid decline in function, while others may have a slower progression. The exact cause of ALS is not fully understood, but it is believed to involve a combination of genetic and environmental factors. ALS is often characterized by a relentless progression, affecting the ability to perform everyday activities.