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

Sponsor
Mayo Clinic (Other)
Overall Status
Enrolling by invitation
CT.gov ID
NCT04503213
Collaborator
(none)
20
Enrollment
1
Location
77.3
Anticipated Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    20 participants
    Observational Model:
    Case-Only
    Time Perspective:
    Prospective
    Official Title:
    Longitudinal Assessment of CSF1R-Related Leukoencephalopathy Following Stem Cell Transplantation
    Actual Study Start Date :
    Jul 21, 2020
    Anticipated Primary Completion Date :
    Dec 30, 2025
    Anticipated Study Completion Date :
    Dec 30, 2026

    Outcome Measures

    Primary Outcome Measures

    1. Improvement in cognitive and motor function [Through study completion, approximately 5 years]

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Mayo Clinic in Florida Jacksonville Florida United States 32224

    Sponsors and Collaborators

    • Mayo Clinic

    Investigators

    • Principal Investigator: Zbigniew K Wszolek, MD, Mayo Clinic

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Zbigniew K. Wszolek, Principal Investigator, Mayo Clinic
    ClinicalTrials.gov Identifier:
    NCT04503213
    Other Study ID Numbers:
    • 20-006124
    First Posted:
    Aug 7, 2020
    Last Update Posted:
    Dec 8, 2021
    Last Verified:
    Dec 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Zbigniew K. Wszolek, Principal Investigator, Mayo Clinic
    HDLS, Leukoencephalopathy
    Additional relevant MeSH terms:
    Leukoencephalopathies

    Study Results

    No Results Posted as of Dec 8, 2021