Stem cell hope for rare brain disease: 20 patients tracked
NCT ID NCT04503213
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Mayo Clinic in Florida
Jacksonville, Florida, 32224, United States