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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT04503213
Other study ID # 20-006124
Secondary ID
Status Enrolling by invitation
Phase
First received
Last updated
Start date July 21, 2020
Est. completion date December 2025

Study information

Verified date September 2023
Source Mayo Clinic
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


Recruitment information / eligibility

Status Enrolling by invitation
Enrollment 20
Est. completion date December 2025
Est. primary completion date December 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United States Mayo Clinic in Florida Jacksonville Florida

Sponsors (1)

Lead Sponsor Collaborator
Mayo Clinic

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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. Through study completion, approximately 5 years
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