RecruitingRare disease

A study to evaluate the safety of VTx-002 in patients with amyotrophic lateral sclerosis.

Verified siteInvestigationalNo placebo

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What is this trial about?

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

This study focuses on Amyotrophic Lateral Sclerosis, also known as ALS, which is a disease that affects the nerve cells in the brain and the spinal cord. The purpose of this study is to evaluate the safety and how well the body tolerates a new experimental treatment called VTx-002. This treatment is an injection that is delivered via intracisternal use, which means it is placed into the space near the base of the brain where spinal fluid is located. The medication is prepared using sodium dihydrogen phosphate dihydrate as a diluent, which is a substance used to dissolve or dilute a medication to the correct strength.

Participants in the study will receive different doses of VTx-002 to see how the body reacts. During the course of the study, medical professionals will monitor various health indicators, including MRI scans to look at the structure of the brain and spine, and assessments of peripheral neuropathy, which refers to nerve damage that can cause numbness or weakness in the hands and feet. Other measurements may include tracking lung function through slow vital capacity, which is a way to measure how much air the lungs can hold during a normal breath.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Administration of the study medication
A single dose of vtx-002, which is a solution for injection, is administered through intracisternal use. this means the medication is injected into the space around the spinal cord.
A diluent, which is a liquid used to dissolve or dilute a substance, containing sodium dihydrogen phosphate dihydrate, is also used during this process.
Step 2
Safety and health monitoring
The safety and tolerability of the medication are monitored through various assessments. this includes checking laboratory values, which are results from blood or other bodily fluid tests, and magnetic resonance imaging (mri), which are detailed pictures of the inside of the body.
Assessments are conducted to check for peripheral neuropathy, which is a condition involving nerve damage that can cause numbness or tingling, and to monitor cellular responses to the treatment.
A scale called the columbia suicide severity rating scale (c-ssrs) is used to monitor mental health and well-being.
Step 3
Long-term follow-up
Changes in physical function are tracked over 6 and 12 months using the revised als functional rating scale (alsfrs-r), a tool used to measure the ability to perform daily tasks.
Breathing function is monitored through slow vital capacity (svc) tests over 6 and 12 months.
The study tracks overall survival and the time until the need for permanent assisted ventilation, which is a machine that helps with breathing, or death over a 12-month period.
The body's immunogenicity, or the immune system's reaction to the adeno-associated virus (aav) capsid, is also assessed.

Who can join the trial?

15 criteria

You must be at least 18 years old.
You must be able and willing to provide written informed consent, which is a signed document showing you understand and agree to participate in the study, and you must follow all study rules and visit schedules.
You must have a diagnosis of Amyotrophic Lateral Sclerosis (ALS) based on specific medical standards called the El Escorial criteria.
You must not have FUS or SOD1 types of ALS, which means genetic testing must confirm that you do not have the specific genetic mutations (changes in DNA) associated with these versions of the disease.
It must have been no more than 18 months since you first noticed symptoms like limb weakness, dysarthria (difficulty speaking), dysphagia (difficulty swallowing), or shortness of breath.
Your SVC % predicted, which is a measurement of how much air you can breathe in while sitting up, must be 80% or higher.
Your TRICALS risk score, a tool used to measure the likely speed of disease progression, must be between -2 and -6.
You must have received vaccinations for pneumococcal pneumonia and shingles within the last 10 years, or agree to get them during the study.
You must be up to date with all cancer screenings appropriate for your age and gender.
You must have a reliable caregiver, partner, or legal representative who can support you and who is willing to make decisions for you if your ability to provide consent changes due to the disease.
If you are currently taking riluzole or edaravone, you must have been on a stable dose for at least 4 weeks for riluzole or a full treatment cycle for edaravone before starting the study.
Women who can become pregnant must use highly effective contraception (birth control with a failure rate of less than 1%), must not be pregnant, must not be lactating (breastfeeding), and must have a negative serum pregnancy test, which is a blood test to check for pregnancy hormones.
Women who cannot become pregnant must have had a surgical procedure to prevent pregnancy (such as a hysterectomy or tubal ligation) at least 26 weeks ago, or be postmenopausal, meaning they have not had a period for at least 2 years and have specific levels of follicle-stimulating hormone (FSH) in their blood.
Men must agree not to donate sperm during the study and for 90 days after, and must use a condom during any activity that could result in the release of semen.
All participants must follow strict contraception rules for 30 days before the first dose, during the study, and for 90 days after the study ends.

Who cannot join the trial?

31 criteria

Having any disease of the brain or the nerves other than ALS that could explain your symptoms or make it hard to see how the study drug works.
Having any abnormal results from medical tests, such as blood or urine tests, that the doctor believes are important for your health.
Having previously received any type of gene or cell therapy, which are treatments that use genetic material or living cells to treat a disease.
Receiving live vaccines in the 4 weeks before starting the study, though certain vaccines like those for pneumonia or shingles are allowed.
Being pregnant or breastfeeding.
Using blood thinners, such as warfarin or heparin, in the 2 weeks before a spinal procedure, or needing to start them during the study.
Having a fear of enclosed spaces, known as claustrophobia, or being unable to tolerate contrast agents, which are special dyes used to make images clearer during scans like an MRI or CT scan.
Having reasons why you cannot safely undergo general anesthesia (being put completely to sleep for a procedure) or deep sedation (being very heavily relaxed and sleepy).
Testing positive for illegal drugs without a valid prescription.
Being very frail or having a medical condition that makes participating in the study unsafe or difficult.
Having a known allergy to the study drug, VTx-002, or any of its ingredients.
Having abnormalities in the brain, neck, or spine seen on imaging, such as evidence of a hemorrhage (bleeding) or an infarct (tissue death caused by lack of blood supply).
Using a tracheostomy (a tube in the windpipe to help breathing) or a feeding tube.
Having taken any experimental medicines, stem cells, or other study treatments within the last 30 days.
Having an implanted device such as a brain stimulator, a shunt (a tube used to drain fluid from the brain), or other permanent tubes in the body.
Having a history of issues that prevent the use of corticosteroids (medicines that reduce inflammation), such as uncontrolled high blood pressure, uncontrolled diabetes, or certain bone issues.
Having a liver problem or an unstable autoimmune disease, which is a condition where the body's immune system attacks its own healthy cells.
Having poorly controlled diabetes, measured by a specific blood sugar test called HbA1c.
Having a history of heart problems in the last year, such as a heart attack or unstable angina (chest pain that is unpredictable).
Having abnormalities in an ECG, which is a test that records the electrical activity of your heart.
Having uncontrolled high blood pressure (hypertension).
Having a history of cancer within the last 5 years, except for certain stable, non-dangerous types.
A history of alcohol or drug abuse within the last 2 years.
Having a psychiatric diagnosis, such as schizophrenia or severe depression, that might make it hard to follow the study rules.
Being at imminent risk of self-harm or suicide.
Having medical conditions that make a lumbar puncture (inserting a needle into the spine to collect fluid) or other injections unsafe, such as thrombocytopenia (a low number of blood cells that help clotting).
Having had a stroke or a transient ischemic attack (a "mini-stroke") within the last year.
Having a history of seizures or unexplained blackouts in the last 10 years.
Having a severe infection, such as pneumonia or meningitis (an infection around the brain and spinal cord), within the last 12 weeks.
Having abnormal blood test results, including high levels of liver enzymes, high creatinine (a sign of kidney function), or low levels of neutrophils, lymphocytes, or platelets (cells that help blood clot).
Testing positive for Hepatitis B, Hepatitis C, or HIV.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Bellvitge University Hospital
Verified
L'hospitalet De Llobregat, Spain
Katholieke Universiteit te Leuven
Responsive
Leuven, Belgium
Universitair Medisch Centrum Utrecht
Responsive
Utrecht, The Netherlands

Trial locations

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Investigated drugs

VTx-002 is an experimental medication being tested to see if it is safe and well-tolerated in people with Amyotrophic Lateral Sclerosis (ALS). It is administered as a liquid injection directly into the area near the sternum (the breastbone).

What is already known about the treatment

VTx-002
This experimental medication is administered as a solution through an injection directly into the area near the collarbone. Currently being studied in clinical trials, it is being investigated as a potential treatment for people living with Amyotrophic Lateral Sclerosis. It belongs to a class of investigational therapies designed to work at a molecular level to protect or repair nerve cells.
Sodium dihydrogen phosphate dihydrate
This substance is used as a diluent, which is a liquid used to dissolve or mix with a drug to prepare it for injection. In this study, it is administered via an injection near the collarbone to help stabilize the main experimental medication. It is a common chemical compound used in medical settings to maintain the correct balance and stability of liquid medicines.

Investigated diseases

Amyotrophic Lateral Sclerosis - This condition is a progressive disorder that affects the nerve cells in the brain and the spinal cord. It specifically targets the motor neurons, which are the cells responsible for controlling voluntary muscle movement. As these nerve cells gradually deteriorate and die, the brain loses its ability to initiate and control muscle activity. This leads to a progressive weakening of the muscles throughout the body. The loss of muscle control typically begins with small movements and spreads to larger muscle groups over time.