Stem cell hope for rare brain disease: 20 patients tracked

NCT ID NCT04503213

ENROLLING_BY_INVITATION Disease control Sponsor: Mayo Clinic Source: ClinicalTrials.gov ↗

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ALSP are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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.

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.