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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04503213
Recruitment Status : Enrolling by invitation
First Posted : August 7, 2020
Last Update Posted : December 8, 2021
Sponsor:
Information provided by (Responsible Party):
Zbigniew K. Wszolek, Mayo Clinic

Study Description
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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
CSF1R-related Leukoencephalopathy ALSP POLD Hematopoietic Stem Cell Transplantation

Study Design
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Study Type : Observational
Estimated 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 : July 21, 2020
Estimated Primary Completion Date : December 30, 2025
Estimated Study Completion Date : December 30, 2026
Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Improvement in cognitive and motor function [ Time Frame: 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
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects will be identified through the Mayo Clinic Florida Neurology and/or Hematology Departments. Qualifying individuals will be identified on the basis of a genetically-confirmed diagnosis of CSF1R-related leukoencephalopathy and a tentative treatment plan including haematopoietic stem cell transplantation (HSCT).
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04503213


Locations
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United States, Florida
Mayo Clinic in Florida
Jacksonville, Florida, United States, 32224
Sponsors and Collaborators
Mayo Clinic
Investigators
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Principal Investigator: Zbigniew K Wszolek, MD Mayo Clinic
More Information
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Additional Information:

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Responsible Party: Zbigniew K. Wszolek, Principal Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT04503213    
Other Study ID Numbers: 20-006124
First Posted: August 7, 2020    Key Record Dates
Last Update Posted: December 8, 2021
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Zbigniew K. Wszolek, Mayo Clinic:
HDLS, Leukoencephalopathy
Additional relevant MeSH terms:
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Leukoencephalopathies
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases