Ongoing, recruitingPhase II and Phase III (Integrated)Interventional

A Phase 2 Safety, Tolerability, and Proof-of-Concept Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)

This research study is looking into a new medicine, VGL101, designed to treat a rare brain condition known as Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP). ALSP is a serious condition that affects the brain. The main goals of this study are to understand how safe the medicine is and if people can tolerate it without too many problems. We'll also be looking for early signs that the medicine might be working. This type of study, called a Phase 2 trial, is crucial for learning more about new treatments. It helps scientists decide if a medicine is promising enough to test in larger studies, moving us closer to finding effective treatments for conditions like ALSP.

At a glance

Status

Ongoing, recruiting

Phase

Phase II and Phase III (Integrated)

Sponsor

Vigil Neuroscience Inc.

Enrolment target

Start

24 Jul 2023

Leukoencephalopathy

What is this study about?

This study is focused on a health problem called Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia, or ALSP for short. It's a rare and serious condition that affects the brain. We don't have many good treatments for ALSP right now, so finding new ways to help people is very important.

The main aim of this research is to test a new medicine called VGL101. Researchers want to find out if VGL101 is safe for people with ALSP and how well their bodies cope with it. They will also be looking for early signs that the medicine might be making a positive difference in the brain.

To do this, the study will involve various checks and measurements. Experts will closely watch for any side effects and use different tests to see what's happening. This includes checks like blood tests and brain scans (MRI). They will also look at certain markers in the body that can tell us about brain health. The information gathered in this study will help us understand if VGL101 could be a helpful treatment option for ALSP in the future.

Key takeaways

This study tests a new medicine called VGL101 for the rare brain condition ALSP.
The main goals are to check the medicine's safety and how well participants tolerate it.
Researchers will also look for early signs that the medicine might help.
It's a 'Phase 2' study, an important step in developing new treatments.
Participants must be 18 years or older and have an ALSP diagnosis.
Taking part involves regular visits, medical tests, and monitoring.

Who may be eligible?

To join this study, you need to be an adult, 18 years old or older. The study is open to both men and women.

Since this study is specifically for Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP), you would need to have been diagnosed with this condition.

There might be other specific health requirements or conditions that would mean you can or cannot take part, but these details are usually discussed with the study team and your doctor.

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 Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP)?
Are you able to attend regular appointments for tests, including blood tests and brain scans (MRIs)?
Are you willing to consider taking a new, experimental medicine?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be given the new medicine, VGL101, as part of the research. Your health would be carefully monitored throughout the study. This includes regular check-ups to make sure you are tolerating the medicine well.

You would have several visits to the study site for various assessments. These assessments will help the researchers understand how the medicine is affecting you. They would involve things like blood tests, urine tests, checking your heart activity with an ECG, and taking measurements like your blood pressure. You might also have brain scans like MRIs.

The study would last for a period of time, where you would receive the medicine and attend follow-up appointments. The exact duration and number of visits would be explained in detail by the study team. You'd have the opportunity to ask any questions and understand exactly what's involved before deciding.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might not directly benefit from this study, but the information gained could help others with ALSP in the future. As with any new medicine, there's a chance you could experience side effects, some of which might be unknown. The study team will closely monitor your health for any issues. You are free to withdraw from the study at any time, for any reason, without affecting your standard medical care.

Locations (3)

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

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Unverified
Germany
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Unverified
Netherlands
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Unverified
France

Common questions

What is ALSP?

ALSP stands for Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia. It's a rare brain condition that affects adults.

What is VGL101?

VGL101 is a new medicine being tested as a potential treatment for ALSP. It's in the early stages of research.

What does 'Phase 2 study' mean?

A Phase 2 study checks if a new medicine is safe, how well people tolerate it, and if it shows any early signs of working. If it does, it might then be tested in larger 'Phase 3' studies.

Will I get the actual medicine or a dummy pill?

The study description doesn't specify if this study includes a dummy pill (placebo group). This is an important question to ask the study team if you're considering joining.

How long would I need to be in the study?

The study mentions looking at changes up to Week 24 and Week 52, suggesting participation would last at least a year. The study team can give you exact dates.