Status:
ENROLLING_BY_INVITATION
A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation
Lead Sponsor:
Mayo Clinic
Conditions:
CSF1R-related Leukoencephalopathy
ALSP
Eligibility:
All Genders
18+ years
Brief Summary
The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.
Detailed Description
The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.
Eligibility Criteria
Inclusion
- ≥ 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
- 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.
Key Trial Info
Start Date :
July 21 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT04503213
Start Date
July 21 2020
End Date
December 1 2027
Last Update
January 8 2026
Active Locations (1)
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1
Mayo Clinic in Florida
Jacksonville, Florida, United States, 32224