Active not recruitingPHASE2INTERVENTIONAL

A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)

This study is investigating a new treatment called VHB937 for people recently diagnosed with Amyotrophic Lateral Sclerosis (ALS). Researchers want to find out if VHB937 is safe and effective in helping slow down the progression of the disease. Participants will receive either VHB937 or a placebo (a dummy drug) for about 40 weeks, without knowing which one they are getting. The study will look at how the treatment affects their ability to do daily activities, their breathing, and any side effects they experience. This includes a careful review of their overall health throughout the treatment period and beyond, to ensure their safety and monitor any changes.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

251

Start

17 Oct 2024

Estimated completion

10 Jul 2028

Amyotrophic Lateral Sclerosis (ALS)

What is this study about?

This research study is trying to understand more about a new medication called VHB937 for people who have been diagnosed with Amyotrophic Lateral Sclerosis, often called ALS or motor neurone disease. ALS is a condition that affects the nerves in the brain and spinal cord, leading to muscle weakness over time. The main goal of this particular study is to see if VHB937 can help improve the lives of people with early-stage ALS, and if it's safe to use.

In this study, some people will receive VHB937, and others will receive a 'placebo'. A placebo is like a dummy treatment that looks exactly like the real medication but doesn't contain any active drug. This helps the researchers truly understand if any changes seen are due to VHB937 itself. Neither you nor your study doctor will know whether you are receiving VHB937 or the placebo, which is a standard way to get unbiased results in medical research.

Your health will be carefully watched throughout the study. The researchers will be looking at things like your ability to do everyday tasks, how your breathing is, and whether you experience any unwanted side effects. Participating in a study like this helps doctors learn more about new treatments, potentially leading to better options for people with ALS in the future. It's a way for you to contribute to medical knowledge and potentially help others down the line.

Key takeaways

This study is testing a new drug called VHB937 for early-stage ALS.
It aims to see if VHB937 is safe and can improve daily activities and breathing.
Participants will receive either VHB937 or a harmless dummy drug (placebo).
Your health will be closely monitored by a medical team.
You might help future patients with ALS by taking part.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be able to join this study, you need to be at least 18 years old. You must have a confirmed diagnosis of ALS from the study doctors, and your symptoms should have started within the last two years. The study is looking for people whose ALS symptoms are not yet severe, meaning you can still do most daily activities.

You might be suitable if you haven't started any treatment for ALS yet, or if you are already on an approved ALS treatment and your dose has been stable. You'll also need to be able to breathe out a reasonable amount of air, at least 60% of what is expected for someone your age, height, and gender.

However, some things would mean you can't join. For example, if you've recently taken part in another drug trial, have serious heart, liver, or kidney problems, or certain blood disorders, this study might not be right for you. Also, if you have other serious health conditions like certain cancers, uncontrolled diabetes, HIV, Hepatitis, or severe breathing difficulties, you might not be able to participate. The study team will carefully check all these details to make sure it's safe for you to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with ALS by a doctor?
Did your ALS symptoms start within the last two years?
Are your ALS symptoms currently mild?
Do you have stable breathing (not severe difficulties)?
Are you not currently in another drug trial for ALS?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will first go through some initial checks to see if you are suitable. If you are, you'll then be assigned to either receive VHB937 or a placebo – you won't know which one. You will take the study medication for about 40 weeks (around 10 months). During this time, you'll have regular visits to the study clinic for check-ups, where doctors will monitor your ALS symptoms, overall health, and any side effects. You'll likely fill out questionnaires about your daily activities and have breathing tests. After the main 40-week period, you may have the option to continue receiving VHB937 in an 'open-label extension' where everyone knows they are getting the active drug. The total duration of your involvement, including follow-up, will be clearly explained to you.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. You might not directly benefit from receiving VHB937, especially if you are in the placebo group. However, you would be helping researchers learn more about ALS and potentially contribute to developing new treatments for others in the future. As with any medication, VHB937 may have side effects, which the study team will monitor very closely. They will explain all known potential risks in detail before you decide to join. Remember, your participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (74)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of California San Diego
Verified postcode
La Jolla, United States
Loma Linda University Health
Verified postcode
Loma Linda, United States
Keck Medical Center USC
Verified postcode
Los Angeles, United States
UC San Francisco Medical Center
Verified postcode
San Francisco, United States
University of Miami
Verified postcode
Miami, United States
Orlando Health Clinical Trials
Verified postcode
Orlando, United States
Emory University School of Medicine
Verified postcode
Atlanta, United States
University Of Kansas Medical Center
Verified postcode
Kansas City, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
University of Nebraska Medical Center
Verified postcode
Omaha, United States
Lange Neurology PC
Verified postcode
New York, United States
University of Rochester Medical Center
Verified postcode
Rochester, United States

Common questions

What is ALS?

ALS (Amyotrophic Lateral Sclerosis) is a progressive condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and wasting over time.

What is VHB937?

VHB937 is a new medication being tested in this study to see if it can help people with ALS. We don't know yet if it will be effective or safe.

What is a placebo?

A placebo is a 'dummy' treatment that looks like the real medication but contains no active drug. It helps researchers compare the new medicine to not having it, to see if it makes a difference.

Will I know if I'm getting the drug or the placebo?

No, you and your study doctor will not know whether you are receiving VHB937 or the placebo during the main part of the study. This is called a 'blinded' study and helps make the results fair.

Can I leave the study at any time?

Yes, you can leave the study at any point, for any reason. Your decision will not affect your ongoing medical care.