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Last hope: experimental drug tested in single child with rare brain disease

NCT ID NCT07272525

Disease control Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study gives one patient with Cree Leukoencephalopathy, a rare and fatal brain disease, access to an experimental drug called fosigotifator. The drug aims to slow or stop the brain damage that causes severe disability and early death. Researchers will track whether the patient survives or needs a breathing machine after two years.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Fosigotifator (FGT/ABBV-CLS-7262)
What this could lead to
If it works, this could point toward a treatment that slows or halts brain damage in Cree Leukoencephalopathy, offering hope for a disease with no current options.
What could go wrong
This is a very early study in just one patient, so results may not apply to others. The drug is still investigational and its safety and effectiveness are not yet proven.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 1 person

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2024

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: The patient meets the following criteria: * Molecularly confirmed diagnosis of CLE * Pre-symptomatic or early symptomatic patient * Signed informed consent from the Legal Guardians/caregivers (parents) Exclusion Criteria: N/A

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Conditions

The condition(s) this trial relates to.

Leukoencephalopathies leukoencephalopathy with vanishing white matter leukoencephalopathy with vanishing white matter 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cree leukoencephalopathy Vanishing white matter

Contacts and locations

Locations

  • McGill University Health Centre

    Montreal, Quebec, H4A3J1, Canada

More trials for these conditions

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