RecruitingPHASE1INTERVENTIONAL

Intravenous Immunoglobulin (IVIG) and Blood-Brain Barrier Disruption in Amyotrophic Lateral Sclerosis (ALS)

This important study is exploring a new approach for people living with Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. Researchers are investigating if a treatment called Intravenous Immunoglobulin (IVIG) can work more effectively when delivered to the brain using a special 'Next Generation Dome Helmet' that uses focused sound waves. This helmet temporarily opens a protective barrier around the brain, potentially allowing more of the IVIG drug to get to where it's needed. The main goal of this early-stage study is to check how safe this combined treatment is and if it's possible to do, rather than looking at how well it treats the disease itself. They are also trying different doses of IVIG.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Sunnybrook Health Sciences Centre

Enrolment target

Start

15 Apr 2025

Estimated completion

30 May 2027

Amyotrophic Lateral Sclerosis ALS

What is this study about?

This study is looking into a new way to treat Amyotrophic Lateral Sclerosis (ALS), sometimes called Motor Neurone Disease. ALS is a condition that affects nerves in the brain and spinal cord, leading to muscle weakness and problems with movement. Currently, there's no cure, but treatments can help manage symptoms and slow its progression.

Researchers want to see if a medicine called Intravenous Immunoglobulin (IVIG) can be made to work better by helping more of it get into the brain. IVIG is a treatment made from human blood plasma that contains important antibodies, which are like the body's natural defenders. The brain has a shield called the 'blood-brain barrier' that rightly protects it from harmful substances, but it also makes it hard for medicines like IVIG to reach brain cells effectively. This study uses a special 'Next Generation Dome Helmet' with focused sound waves to temporarily and safely open this barrier. They also use tiny bubbles, like those used in ultrasound scans, to help this process.

This is a very early-stage study, called a Phase 1 trial. This means the main focus is on carefully checking if this new method is safe for people with ALS and if it's possible to do. It's not designed to find out how well it works as a treatment for ALS, though they will be observing participants closely. The results from this study will be crucial for deciding whether to move forward with larger studies in the future.

Key takeaways

This is an early-stage safety study for ALS treatment.
It combines an existing drug (IVIG) with a new brain delivery method (focused sound waves).
The 'Dome Helmet' aims to temporarily open the blood-brain barrier to improve drug delivery.
The main goal is to check treatment safety rather than effectiveness for ALS.
Participation involves several visits over about 6 months.
You can withdraw from the study at any time.

Who may be eligible?

To be considered for this study, you would need to be an adult (18 or older) who has received an official diagnosis of ALS based on specific criteria. You also need to be able to understand the study information and agree to take part, as well as follow the study's instructions. If you are already taking certain ALS medications like Riluzole or Edaravone, your dose must have been stable for a certain period before joining.

Your breathing capacity is also important; you should be able to lie down flat without needing a special breathing machine or feeling uncomfortable. Alternatively, your lung function test results should show a certain level of capacity. It's also vital that you can communicate clearly during the treatment sessions with the special helmet.

There are also reasons why you might not be able to join. For example, if MRI scans of your brain show certain issues like infections, specific types of bleeding, or tumours. Other reasons include having too many scars or skin conditions on your scalp where the helmet would be placed, or having certain metal implants in your head. Significant heart problems or uncontrolled high blood pressure would also prevent participation, as would having a pacemaker or certain blood vessel issues in your heart.

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.

Do you have a confirmed diagnosis of ALS?
Are you 18 years old or older?
Can you understand study information and communicate feelings during the treatment?
Is your breathing stable enough, without needing a special machine when lying flat?
Do you have any significant heart problems or uncontrolled high blood pressure?
Do you have certain metal implants in your head or skin/scars on your scalp?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be one of six participants in total. You would attend several visits over about 24 weeks (around 6 months). Initially, you'll receive the first dose of IVIG. About two weeks later, you'll receive a second dose of IVIG, and at the same time, you'll undergo the special procedure using the Next Generation Dome Helmet with focused sound waves and tiny bubbles to help the IVIG reach your brain. This helmet treatment targets specific areas in both halves of your brain involved in movement.

Throughout the study, you would have regular check-ups to monitor your health, how you're feeling, and to look for any side effects. Researchers will also be checking how the treatment affects your body and how the drug is handled by your system. There are two groups in the study, with each group of three participants receiving a different dose of IVIG – either 0.4g/kg or 0.8g/kg, split into two doses. All follow-up visits will continue for 24 weeks from your first treatment.

Potential risks and benefits

Participating in this study might offer a potential benefit by helping us learn more about treating ALS, which could help future patients. However, as this is an early-stage study focused on safety, there's no guarantee that you will personally benefit from the treatment itself. Potential risks include side effects from the IVIG medication, or from the focused sound wave helmet procedure, such as discomfort during the procedure, or mild bleeding or inflammation in the brain. Researchers will carefully explain all known and possible risks before you agree to take part. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Sunnybrook Health Sciences Centre
Verified postcode
Toronto, Canada· Recruiting

Common questions

What is ALS?

ALS, or Amyotrophic Lateral Sclerosis, is a serious condition that affects the nerves in your brain and spinal cord, leading to muscle weakness and problems with movement over time.

What is IVIG?

IVIG stands for Intravenous Immunoglobulin. It's a medicine made from human blood plasma that contains special antibodies, which are proteins that help your body fight off infections and can help with certain immune problems.

What does the 'Dome Helmet' do?

The 'Dome Helmet' uses focused sound waves to temporarily open the natural protection around your brain (the blood-brain barrier). This might help medicines like IVIG get into the brain more easily.

Is this a new treatment for ALS?

This is an early-stage study testing a new way to deliver an existing medication (IVIG) to the brain for ALS. It's mainly to check safety and feasibility, not yet to cure ALS.

How long does the study last?

If you participate, the study involves visits and follow-up over approximately 24 weeks, which is about 6 months, from your first treatment dose.

How to find out more

Nir Lipsman, MD, PhD, FRCPC

alsresearch@sunnybrook.ca (416) 480-6100 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.