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Desperate patients get early access to experimental brain drug

NCT ID NCT06594016

Disease control Sponsor: AbbVie 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
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 Results may not be published yet.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) This study
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This status has not been confirmed recently, so it may be out of date.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 program offers early access to fosigotifator, an oral drug, for people with Vanishing White Matter or Cree Leukoencephalopathy—two rare, serious brain diseases. A doctor must decide if the potential benefit outweighs the risks for each patient. The drug is not yet approved, so its effects are still being studied.

What this could mean

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

Active substance
fosigotifator (oral drug)
What this could lead to
If this program provides benefit, it could point toward a treatment option for Vanishing White Matter and Cree Leukoencephalopathy.
What could go wrong
This is an expanded access program, not a formal trial, so data on safety and effectiveness is limited. The drug is still investigational and may not work for all patients.

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.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

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

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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

More trials for these conditions

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