[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT04503213":3,"trial-entities:NCT04503213":73,"trial-summary:NCT04503213":78},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":16,"conditions":17,"keywords":22,"study_type":24,"primary_purpose":25,"phases":26,"enrollment_info":27,"interventions":30,"primary_outcomes":31,"secondary_outcomes":36,"sex":37,"minimum_age":38,"maximum_age":25,"healthy_volunteers":15,"eligibility_criteria":39,"std_ages":50,"locations":53,"central_contacts":63,"overall_officials":64,"references":68,"see_also_links":69},"NCT04503213","20-006124","A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation","Longitudinal Assessment of CSF1R-Related Leukoencephalopathy Following Stem Cell Transplantation","ENROLLING_BY_INVITATION","2027-12","2026-01","2026-01-08","2020-07-21","Mayo Clinic","OTHER",false,"The purpose of this study is to measure the effect of Hematopoietic Stem Cell Transplantation (HSCT) on symptoms of CSF1R-related Leukoencephalopathy.",[18,19,20,21],"CSF1R-related Leukoencephalopathy","ALSP","POLD","Hematopoietic Stem Cell Transplantation",[23],"HDLS, Leukoencephalopathy","OBSERVATIONAL",null,[],{"count":28,"type":29},20,"ESTIMATED",[],[32],{"measure":33,"description":34,"timeFrame":35},"Improvement in cognitive and motor function","Number of participants to demonstrate stability\u002Fimprovements in cognitive and motor function by detailed clinical assessment and radiographic markers of disease.","Through study completion, approximately 5 years",[],"ALL","18 Years",{"inclusion":40,"exclusion":45,"raw_text":49},[41,42,43,44],"≥ 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).",[46,47,48],"Concurrent diagnoses that may confound neuropsychological testing; e.g., major hearing\u002Fvisual impairment.","Concurrent diagnoses that may confound ambulatory measurements; e.g., amputee.","Inability to undergo magnetic resonance imaging (MRI); e.g., MR-incompatible implant.","Inclusion Criteria:\n\n* ≥ 18 years of age.\n* Genetic confirmation of a mutation in the CSF1R gene.\n* Diagnosis of CSF1R-related leukoencephalopathy.\n* Anticipated to undergo haematopoietic stem cell transplantation (HSCT).\n\nExclusion Criteria:\n\n* Concurrent diagnoses that may confound neuropsychological testing; e.g., major hearing\u002Fvisual impairment.\n* Concurrent diagnoses that may confound ambulatory measurements; e.g., amputee.\n* Inability to undergo magnetic resonance imaging (MRI); e.g., MR-incompatible implant.",[51,52],"ADULT","OLDER_ADULT",[54],{"facility":55,"city":56,"state":57,"zip":58,"country":59,"geoPoint":60},"Mayo Clinic in Florida","Jacksonville","Florida","32224","United States",{"lat":61,"lon":62},30.33218,-81.65565,[],[65],{"name":66,"affiliation":13,"role":67},"Zbigniew K Wszolek, MD","PRINCIPAL_INVESTIGATOR",[],[70],{"label":71,"url":72},"Mayo Clinic Clinical Trials","https:\u002F\u002Fwww.mayo.edu\u002Fresearch\u002Fclinical-trials",{"nct_id":4,"conditions":74,"biomarkers":76},[75],"Leukoencephalopathy with Ataxia",[77],"CSF1R Gene",{"nct_id":4,"found":79,"summary":80,"prompt_version":90},true,{"design":81,"status":82,"heading":83,"summary":84,"follow_up":85,"word_count":86,"commitments":87,"compensation":88,"drugs_mentioned":89},"This is an observational study, meaning researchers will watch and collect information without giving a specific treatment. It plans to include 20 participants.","completed","Observational Study of HSCT for CSF1R-related Leukoencephalopathy","This study is looking at how hematopoietic stem cell transplantation (HSCT), a type of stem cell transplant, affects people with CSF1R-related leukoencephalopathy. This condition is a brain disorder caused by a change in the CSF1R gene. Researchers want to see if HSCT can improve symptoms like problems with thinking and movement. You might be able to join if you are 18 or older, have a confirmed CSF1R gene mutation, have CSF1R-related leukoencephalopathy, and are planning to have an HSCT. The study will consider HSCT successful if it leads to improvements in your cognitive (thinking) and motor (movement) abilities over about five years. The current enrollment status is unclear.","Participants will be followed through study completion, which is approximately 5 years.",108,"Not specified in the trial record.","Not stated in the trial record.",[],"v2"]