Active not recruitingEARLY_PHASE1INTERVENTIONAL

Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease

This study is for a very rare and serious brain condition called Cree Leukoencephalopathy (CLE), which mainly affects people from the Cree community in Northern Quebec. This condition, also known as Vanishing White Matter disease, causes damage to the brain and can be life-threatening, with no current effective treatments. Researchers are testing a new investigational medicine called Fosigotifator (FGT) to see if it can help slow down or stop the brain damage caused by CLE. The goal is to see if FGT can reduce symptoms and improve the patient's quality of life. This study is important because it could help find a new way to treat this condition, offering hope to those who currently have no treatment options.

At a glance

Status

Active not recruiting

Phase

EARLY_PHASE1

Sponsor

McGill University Health Centre/Research Institute of the McGill University Health Centre

Enrolment target

Start

14 Nov 2024

Estimated completion

31 Dec 2027

Cree Leukoencephalopathy Vanishing White Matter

What is this study about?

Cree Leukoencephalopathy (CLE) is a very rare and serious condition that affects the brain. It mostly affects a specific group of people in Northern Quebec. It causes the 'white matter' of the brain, which helps brain cells talk to each other, to slowly get damaged. This leads to problems with movement and other brain functions, and sadly, it can shorten a person's life. At the moment, there are no effective treatments for CLE.

This study is looking into a new medicine called Fosigotifator, often shortened to FGT. This medicine is also being studied for a similar condition called Vanishing White Matter (VWM) disease. The main goal of this particular study is to offer FGT to a patient with CLE/VWM who currently has no other treatment choices. Researchers want to see if FGT is safe and if it can help slow down or stop the damage to the brain.

By understanding how CLE affects the brain, and by testing FGT, this research hopes to find a way to manage the disease better. The results could be very important for developing new treatments and improving the lives of people with CLE. It's about finding hope where there currently isn't any.

Key takeaways

CLE is a rare, serious brain disease with no current treatment.
FGT is an experimental drug hoping to slow down brain damage.
This study offers FGT to a patient with no other options.
Participation involves taking the drug and regular health checks.
There are potential benefits and unconfirmed risks.
Parents/guardians must give consent for participation.

Who may be eligible?

To be considered for this study, a patient needs to have a confirmed diagnosis of Cree Leukoencephalopathy (CLE) that has been identified through genetic testing. They should either not have symptoms yet, or be in the very early stages of having symptoms.

Because this study might involve very young patients, a parent or legal guardian will need to understand the study and agree to their child taking part by signing a consent form. This ensures that the patient's best interests are protected.

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.

Does the patient have a confirmed diagnosis of Cree Leukoencephalopathy (CLE) from genetic testing?
Is the patient not yet showing symptoms, or only showing very early signs?
Are the patient's parents or legal guardians willing and able to understand the study and provide consent?
Is the patient aged 4 months or older?

Answer every question to see your result.

What does participation involve?

As this is a 'compassionate use' study for a single patient with a very rare disease, the specifics of participation will be carefully planned with the patient's family and medical team. It will involve regularly taking the investigational medicine, FGT. There will likely be regular visits to the hospital or clinic for check-ups, where doctors will monitor the patient's health and how the medicine is affecting them. This could include tests like blood samples or brain scans to see if the disease is changing. The aim is to understand both the safety of the medicine and if it's helping the patient. The total duration of participation would be ongoing as long as the treatment is beneficial and safe.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. The potential benefit of this study is that FGT could help slow down or stop the progression of CLE/VWM disease, which currently has no treatment. This could lead to fewer symptoms and a better quality of life for the patient. However, since FGT is a new medicine, there might be side effects or risks that are not yet fully known. The patient will be closely monitored for any unwanted effects. It's important to remember that participating is completely voluntary, and the patient's legal guardians have the right to withdraw from the study at any time without affecting their future medical care.

Locations (1)

McGill University Health Centre
Verified postcode
Montreal, Canada

Common questions

What is Cree Leukoencephalopathy (CLE)?

CLE is a very rare and serious brain condition that mainly affects a specific population, causing damage to the brain's 'white matter'.

What is FGT (Fosigotifator)?

FGT is a new medicine being tested to see if it can help slow or stop the brain damage caused by conditions like CLE and Vanishing White Matter disease.

Why is this study only for one patient?

This is a compassionate use study, often done for very rare, serious conditions when there are no other treatment options, to provide access to a promising new drug for a specific patient.

Will FGT cure the disease?

The goal is to see if FGT can slow down or halt the disease's progression, not necessarily to cure it, as this is a very complex condition.

What if we change our mind about participating?

You have the right to withdraw from the study at any time, for any reason, and it will not affect your child's medical care in the future.