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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

Tracking Information
First Submitted Date August 4, 2020
First Posted Date August 7, 2020
Last Update Posted Date December 8, 2021
Actual Study Start Date July 21, 2020
Estimated Primary Completion Date December 30, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 4, 2020)
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.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation
Official Title Longitudinal Assessment of CSF1R-Related Leukoencephalopathy Following Stem Cell Transplantation
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 Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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).
Condition
  • CSF1R-related Leukoencephalopathy
  • ALSP
  • POLD
  • Hematopoietic Stem Cell Transplantation
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Enrolling by invitation
Estimated Enrollment
 (submitted: August 4, 2020)
20
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 30, 2026
Estimated Primary Completion Date December 30, 2025   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04503213
Other Study ID Numbers 20-006124
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Current Responsible Party Zbigniew K. Wszolek, Mayo Clinic
Original Responsible Party Same as current
Current Study Sponsor Mayo Clinic
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators
Principal Investigator: Zbigniew K Wszolek, MD Mayo Clinic
PRS Account Mayo Clinic
Verification Date December 2021