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Last hope drug trial for kids with fatal brain disease

NCT ID NCT07300397

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 child with Cree Leukoencephalopathy (CLE) access to an experimental drug called fosigotifator. CLE is a rare, inherited brain disease that destroys white matter and leads to early death. The drug aims to slow or stop the damage, possibly easing symptoms and improving quality of life. The study will monitor the child closely to see if the benefits outweigh the risks.

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

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 1 person

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

Started

Dec 2025

Expected to finish

Jan 2029

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

1 month 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: * • Molecularly confirmed diagnosis of CLE * Pre-symptomatic or early symptomatic patient * Signed informed consent from the Legal Guardians/caregivers (parents) * Must not be eligible for any actively enrolling trial of fosigotifator in CLE/VWM * Must be at least 1 month of age at the Baseline visit * Must weigh at least 5kg at the Baseline visit 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

Contacts and locations

Locations

  • McGill University Health Centre

    Montreal, Quebec, H4A3J1, Canada

More trials for these conditions

Other studies related to the condition(s) this trial covers.