Can stem cells stop a rare brain disease?

NCT ID NCT04503213

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

First seen Oct 31, 2025 · Last updated May 14, 2026 · Updated 30 times

Summary

This study looks at whether a stem cell transplant can help people with CSF1R-related leukoencephalopathy, a rare genetic brain disease. Researchers will track 20 adults who are already scheduled for the transplant to see if their thinking and movement skills improve or stay stable. The goal is to understand if this treatment can control the disease long-term.

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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.

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Contacts and locations

Locations

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224, United States

Conditions

Explore the condition pages connected to this study.