Not recruitingRare disease

Study on the Safety and Effects of AMT-162 Gene Therapy for Adults with SOD1 ALS

InvestigationalNo 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 a disease called Amyotrophic Lateral Sclerosis, commonly known as ALS. ALS is a condition that affects the nerve cells in the brain and spinal cord, leading to a loss of muscle control. The study specifically targets a form of ALS linked to a genetic mutation known as SOD1. The treatment being tested is a gene therapy called AMT-162, which is delivered as a suspension for injection. This therapy uses a special type of viral vector, known as adeno-associated viral vector serotype RH10, to deliver genetic material that may help reduce the effects of the SOD1 mutation.

The purpose of this study is to evaluate the safety and tolerability of AMT-162 in adults with SOD1-ALS. Participants will receive a single dose of the treatment administered directly into the spinal fluid, a method known as intrathecal use. The study will monitor participants for any side effects and changes in their health over time. Researchers will also explore how the body responds to the treatment, including any immune reactions and changes in specific proteins related to ALS.

The study will take place over several years, allowing researchers to gather comprehensive data on the long-term effects of the treatment. Participants will undergo regular health assessments, including laboratory tests and heart monitoring, to ensure their safety throughout the trial. The ultimate goal is to determine if AMT-162 can be a safe and effective treatment option for individuals with SOD1-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
Initial assessment
The study begins with an initial assessment to confirm eligibility. This includes reviewing previous participation in the EPISOD1 study and ensuring at least 6 months have passed since receiving AMT-162.
Informed consent is required to proceed, confirming understanding and willingness to participate.
Step 2
Administration of AMT-162
The treatment involves a single dose of AMT-162, a gene therapy administered through an injection into the spinal canal, known as intrathecal use.
This is a suspension for injection, specifically targeting the SOD1 gene associated with amyotrophic lateral sclerosis (ALS).
Step 3
Monitoring for safety and tolerability
Following administration, monitoring focuses on safety and tolerability. This includes tracking any adverse events that may occur.
Regular clinical safety laboratory tests are conducted to observe any changes from baseline measurements.
Step 4
Vital signs and ECG monitoring
Vital signs, such as blood pressure and heart rate, are monitored for any changes from baseline.
Electrocardiogram (ECG) parameters are also checked to ensure heart function remains stable.
Step 5
Secondary assessments
Additional assessments include monitoring AMT-162 vector shedding and the body's immune response, including B-cell and T-cell activity.
Changes in lung function, measured by slow vital capacity (SVC), and muscle strength, assessed through hand-held dynamometry (HHD), are evaluated.
Step 6
Long-term follow-up
Long-term follow-up involves measuring neurofilament light chain (NfL) protein levels in the blood for up to 5 years post-treatment.
This extended monitoring helps assess the ongoing impact of the treatment on the condition.

Who can join the trial?

4 criteria

The patient must have previously received the investigational drug called AMT-162 in a previous study known as EPISOD1 (AMT-162-001) at least 6 months before agreeing to participate in this study.
The patient must be willing and able to provide informed consent. This means the patient understands the details of the study and agrees to participate.
The study is open to both male and female patients.
The study includes patients within certain age ranges, which are typically adults.

Who cannot join the trial?

7 criteria

Participants who do not have a confirmed diagnosis of Amyotrophic Lateral Sclerosis (ALS) cannot participate. ALS is a disease that affects the nerve cells in the brain and spinal cord.
Individuals who are not within the specified age range for the study are excluded. The study is open to certain age groups only.
Participants who are not able to safely receive the study treatment due to other health conditions or medications they are taking will be excluded.
Individuals who are pregnant or breastfeeding cannot participate in the study.
Participants who have participated in another clinical trial recently may be excluded to ensure safety and accuracy of the study results.
Individuals who have a history of allergic reactions to similar treatments or components of the study medication will be excluded.
Participants who are unable to comply with the study procedures or follow-up visits will not be able to join the study.

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Investigated drugs

AMT-162 is a gene therapy being studied for its potential to treat SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS). This therapy is administered directly into the spinal canal, a method known as intrathecal administration. The goal of this study is to assess how safe and tolerable AMT-162 is for patients, as well as to explore its potential effectiveness in treating the symptoms of SOD1-ALS.

ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 ENCODING MIRNA AGAINST SOD1 MRNA

What is already known about the treatment

AMT-162 – This medication is administered intrathecally, meaning it is delivered directly into the spinal canal. Currently, AMT-162 is in the clinical trial phase, specifically a Phase 1/2 study, to assess its safety and tolerability in patients with SOD1 Amyotrophic Lateral Sclerosis (ALS). The main therapeutic indication for AMT-162 is to potentially treat ALS, a progressive neurodegenerative disease. At the molecular level, AMT-162 is a gene therapy designed to target and modify the SOD1 gene, which is implicated in some forms of ALS. It falls under the pharmacological classification of gene therapies, which aim to correct or replace faulty genes responsible for disease development.

Investigated diseases

Amyotrophic Lateral Sclerosis (ALS) – ALS 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 these neurons deteriorate, individuals experience muscle weakness and atrophy, often starting in the limbs. Over time, the weakness spreads to other parts of the body, affecting speech, swallowing, and breathing. The progression of muscle weakness varies among individuals, but it typically leads to increasing difficulty in performing daily activities. ALS does not affect the senses or the ability to think, allowing individuals to remain aware of their condition.