A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation

N/AEnrolling By InvitationNCT04503213

Mayo Clinic20 enrolled

Overview

The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Mayo Clinic in Florida

Jacksonville, Florida, United States

Outcomes

Primary Outcomes

Improvement in cognitive and motor function

Number of participants to demonstrate stability/improvements in cognitive and motor function by detailed clinical assessment and radiographic markers of disease.

Time frame: Through study completion, approximately 5 years

Data from ClinicalTrials.gov

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