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Stem cell hope for rare brain disease: 20 patients tracked

NCT ID NCT04503213

Disease control Sponsor: Mayo Clinic 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 This study
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 Results may not be published yet.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)
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This status has not been confirmed recently, so it may be out of date.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 follows 20 adults with CSF1R-related leukoencephalopathy, a rare genetic brain disease, who are scheduled for a stem cell transplant. Researchers will measure changes in thinking, movement, and brain scans over time to see if the transplant helps stabilize or improve symptoms.

What this could mean

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

Active substance
Hematopoietic stem cell transplantation (HSCT)
What this could lead to
If successful, this could show that stem cell transplants slow or improve cognitive and motor decline in people with this rare brain disease.
What could go wrong
This is a small observational study (20 people) with no control group, so results may not apply widely. Stem cell transplants carry serious risks like infection and graft-versus-host disease.

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.

Participants

About 20 people

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

Started

Jul 2020

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.

Who is studied

Subjects will be identified through the Mayo Clinic Florida Neurology and/or Hematology Departments. Qualifying individuals will be identified on the basis of a genetically-confirmed diagnosis of CSF1R-related leukoencephalopathy and a tentative treatment plan including haematopoietic stem cell transplantation (HSCT).

Ages

18 years 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: * ≥ 18 years of age. * Genetic confirmation of a mutation in the CSF1R gene. * Diagnosis of CSF1R-related leukoencephalopathy. * Anticipated to undergo haematopoietic stem cell transplantation (HSCT). Exclusion Criteria: * Concurrent diagnoses that may confound neuropsychological testing; e.g., major hearing/visual impairment. * Concurrent diagnoses that may confound ambulatory measurements; e.g., amputee. * Inability to undergo magnetic resonance imaging (MRI); e.g., MR-incompatible implant.

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Conditions

The condition(s) this trial relates to.

Leukoencephalopathies leukoencephalopathy, diffuse hereditary, with spheroids 1

As listed by the trial registrant

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

ALSP CSF1R-related leukoencephalopathy Hematopoietic stem cell transplantation POLD

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224, United States

More trials for these conditions

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