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Study on the Safety and Effects of AP-101 for Patients with Amyotrophic Lateral Sclerosis (ALS)

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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 affects nerve cells in the brain and spinal cord, leading to muscle weakness and loss of muscle control. The study is testing a new treatment called AP-101, which is a type of medication known as a monoclonal antibody. This medication is designed to target and neutralize harmful proteins in the body that may contribute to the progression of ALS. The trial will also use a solution called 0.9% Sodium Chloride for Injection as part of the study process.

The purpose of this study is to evaluate the safety and tolerability of AP-101 in patients with ALS. The study is divided into two phases: a double-blind phase, where neither the participants nor the researchers know who is receiving the actual medication or a placebo, and an open-label extension, where all participants receive the medication. During the study, participants will receive multiple doses of AP-101 through an intravenous (IV) injection over a period of six months. The study will monitor the participants' health and any side effects that may occur.

Participants in the study will undergo regular health check-ups, including physical and neurological examinations, to ensure their well-being. The study will also measure certain markers in the blood and spinal fluid to understand how the body processes the medication. This research aims to gather important information that could lead to better treatments for ALS in the future.

The research process

The trial runs in 5 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, participation is confirmed through informed consent. This involves understanding the study's requirements and agreeing to follow the procedures outlined.
Step 2
Double-blind phase
During this phase, the medication AP-101 is administered through an intravenous (IV) injection. The treatment lasts for 6 months.
The purpose is to evaluate the safety and tolerability of AP-101 when given in multiple doses.
Participants may continue their current medications such as riluzole or edaravone, provided they have been on a stable dose for at least 30 days before starting the study. These medications cannot be initiated during this phase.
Step 3
Open-label extension
After completing the double-blind phase, participants may enter the open-label extension phase.
In this phase, AP-101 continues to be administered through IV injections. The focus remains on assessing the safety and tolerability of the medication.
Participants may start or adjust doses of riluzole or edaravone if needed, based on the investigator's opinion.
Step 4
Monitoring and assessments
Throughout the study, regular monitoring of vital signs, laboratory tests, and physical and neurological examinations is conducted.
Participants' health and any side effects are closely observed to ensure safety.
Step 5
Completion of study
Upon completing the study, participants undergo final assessments to evaluate the overall effects of the treatment.
The study aims to conclude by July 2025, with all data collected and analyzed to understand the impact of AP-101 on amyotrophic lateral sclerosis (ALS).

Who can join the trial?

16 criteria

Participants can be both men and women. If they are of childbearing potential, they must use birth control methods as per local regulations during the study and for 6 months after the last dose of the study drug.
Men must not donate sperm during the study and for 6 months after the last dose.
Women must not donate eggs during the study and for 6 months after the last dose.
Participants must be 18 years or older.
Participants must have a diagnosis of **Amyotrophic Lateral Sclerosis (ALS)**, which is a condition affecting nerve cells in the brain and spinal cord.
Participants with familial ALS must have a confirmed genetic mutation known as **SOD1 mutation**.
Participants should have experienced the onset of symptoms, like weakness, within the last 24 months before joining the study.
Participants must have a **Slow Vital Capacity (SVC)** of 50% or more of the expected value. SVC is a measure of lung function.
Participants should not use certain breathing support devices for more than 4 hours a day due to ALS symptoms.
If participants are taking medications like **riluzole** or **edaravone**, they must be on a stable dose for at least 30 days before starting the study and continue on the same dose during the study.
If participants are taking **sodium phenylbutyrate** or **taurursodiol**, they must be on a stable dose for at least 30 days before starting the study and continue on the same dose during the study.
Participants must be able to give informed consent, which means they understand the study and agree to participate.
Participants must have good **venous access**, which means their veins are suitable for taking blood samples.
Participants must have normal lab test results or results that are not considered significant by the study doctor.
Participants must be able to visit the study site for treatment.
Participants must be willing to be available for the entire duration of the study and follow the study procedures.

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Region Vaesterbotten
Umea, Sweden

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Investigated drugs

AP-101 is a medication being studied for its safety and tolerability in patients with familial and sporadic amyotrophic lateral sclerosis (ALS). It is administered through intravenous doses over a period of six months. The trial aims to understand how the body processes the medication and its effects on ALS symptoms.

Anti-(Misfolded Human Superoxide Dismutase 1) Human Igg1M3 Monoclonal Antibody

What is already known about the treatment

AP-101 – AP-101 is administered intravenously, meaning it is given directly into a vein. It is currently being studied in clinical trials, specifically in a Phase 2a trial, to assess its safety and tolerability in patients with both familial and sporadic forms of Amyotrophic Lateral Sclerosis (ALS). The main therapeutic indication for AP-101 is ALS, a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord. At the molecular level, AP-101 is believed to work by targeting specific pathways involved in the disease process, although the exact mechanism is still under investigation. It falls under the pharmacological classification of investigational drugs, as it is not yet approved for general medical use.

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, the brain loses the ability to initiate and control muscle movement. This results in muscle weakness and atrophy, often starting in the limbs and spreading to other parts of the body. Over time, individuals may experience difficulty with speaking, swallowing, and breathing. The progression of symptoms varies among individuals, but it generally leads to increasing physical disability.