[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06363760":3,"trial-entities:NCT06363760":252,"trial-summary:NCT06363760":35},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":6,"overall_status":7,"completion_date":8,"status_verified_date":9,"last_update_date":10,"start_date":11,"sponsor_name":12,"lead_sponsor_class":13,"has_dmc":14,"brief_summary":15,"detailed_description":16,"conditions":17,"keywords":21,"study_type":34,"primary_purpose":35,"phases":36,"enrollment_info":37,"interventions":40,"primary_outcomes":47,"secondary_outcomes":59,"sex":97,"minimum_age":98,"maximum_age":99,"healthy_volunteers":14,"eligibility_criteria":100,"std_ages":107,"locations":110,"central_contacts":248,"overall_officials":249,"references":250,"see_also_links":251},"NCT06363760","EDIT-301-LTFU-001","A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301","ENROLLING_BY_INVITATION","2040-08","2025-03","2025-04-02","2024-06-17","Editas Medicine, Inc.","INDUSTRY",false,"The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.","This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.",[18,19,20],"Sickle Cell Disease","Transfusion-dependent Beta-Thalassemia","Hemoglobinopathies",[18,22,23,24,25,26,20,27,28,29,30,31,32,33],"Sickle Cell Anemia","Sickling Disorder Due to Hemoglobin S","Beta-Thalassemia","Thalassemia Major","Thalassemia Intermedia","CRISPR-Cas12a","Autologous CD34+","Genetic Diseases, Inborn","Hematologic Diseases","Anemia","Anemia, Hemolytic, Congenital","Anemia, Hemolytic","OBSERVATIONAL",null,[],{"count":38,"type":39},54,"ESTIMATED",[41],{"type":42,"name":43,"description":44,"armGroupLabels":45},"OTHER","Safety and efficacy assessments","Assessed throughout the duration of the study.",[46],"All participants",[48,51,53,55,57],{"measure":49,"timeFrame":50},"Adverse events of special interest (AESI)","up to 15 years post EDIT-301 infusion",{"measure":52,"timeFrame":50},"Adverse events of interest (AEI)",{"measure":54,"timeFrame":50},"All-cause mortality",{"measure":56,"timeFrame":50},"Adverse events (AEs) related to EDIT-301",{"measure":58,"timeFrame":50},"Serious adverse events (SAEs)",[60,62,64,66,68,70,72,74,76,78,80,82,84,86,88,90,92,95],{"measure":61,"timeFrame":50},"Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion",{"measure":63,"timeFrame":50},"Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion",{"measure":65,"timeFrame":50},"Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants",{"measure":67,"timeFrame":50},"Proportion of TDT participants with sustained transfusion independence (TI) over time",{"measure":69,"timeFrame":50},"Proportion of TDT participants with sustained transfusion reduction (TR) over time",{"measure":71,"timeFrame":50},"Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants",{"measure":73,"timeFrame":50},"SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time",{"measure":75,"timeFrame":50},"SCD and TDT: Total hemoglobin (Hb) concentration (g\u002FdL) over time",{"measure":77,"timeFrame":50},"SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time",{"measure":79,"timeFrame":50},"Average HbF (pg) per RBC over time for SCD participants",{"measure":81,"timeFrame":50},"Complete Blood Count (CBC) red cell indices assay over time for SCD participants",{"measure":83,"timeFrame":50},"Average HbF (pg) per F-cell over time for SCD participants",{"measure":85,"timeFrame":50},"HbF and HbS concentration (g\u002FdL) over time for SCD participants",{"measure":87,"timeFrame":50},"The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.",{"measure":89,"timeFrame":50},"Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g\u002FdL of all Hb variants, excluding HbA) over time for TDT participants.",{"measure":91,"timeFrame":50},"HbF concentration (g\u002FdL) over time for TDT participants",{"measure":93,"description":94,"timeFrame":50},"Iron overload of TDT participants","As measured by a) Liver iron concentration (LIC) as assessed by R2\\* magnetic resonance imaging (MRI) for the first 3 years in this study; b) Cardiac iron concentration (CIC) as assessed by T2\\* MRI for the first 3 years of this study; c) Serum ferritin levels over time.",{"measure":96,"timeFrame":50},"Proportion of TDT participants receiving iron chelation therapy over time.","ALL","12 Years","50 Years",{"inclusion":101,"exclusion":104,"raw_text":106},[102,103],"Participant must have received an EDIT-301 infusion as part of a clinical study.","Participant or legal representative\u002Fguardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.",[105],"Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.","Inclusion Criteria:\n\n* Participant must have received an EDIT-301 infusion as part of a clinical study.\n* Participant or legal representative\u002Fguardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.\n\nExclusion Criteria:\n\n* Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.",[108,109],"CHILD","ADULT",[111,120,128,136,144,152,160,167,170,178,186,193,201,209,217,225,232,241],{"facility":112,"city":113,"state":114,"zip":115,"country":116,"geoPoint":117},"UCSF Benioff Children's Hospital","Oakland","California","94609","United States",{"lat":118,"lon":119},37.80437,-122.2708,{"facility":121,"city":122,"state":123,"zip":124,"country":116,"geoPoint":125},"Children's Hospital Colorado","Aurora","Colorado","80045",{"lat":126,"lon":127},39.72943,-104.83192,{"facility":129,"city":130,"state":131,"zip":132,"country":116,"geoPoint":133},"Smilow Cancer Hospital","New Haven","Connecticut","06511",{"lat":134,"lon":135},41.30815,-72.92816,{"facility":137,"city":138,"state":139,"zip":140,"country":116,"geoPoint":141},"Johns Hopkins All Children's Hospital","St. Petersburg","Florida","33701",{"lat":142,"lon":143},27.77086,-82.67927,{"facility":145,"city":146,"state":147,"zip":148,"country":116,"geoPoint":149},"Children's Healthcare of Atlanta","Atlanta","Georgia","30322",{"lat":150,"lon":151},33.749,-84.38798,{"facility":153,"city":154,"state":155,"zip":156,"country":116,"geoPoint":157},"University of Minnesota","Minneapolis","Minnesota","55410",{"lat":158,"lon":159},44.97997,-93.26384,{"facility":161,"city":162,"state":162,"zip":163,"country":116,"geoPoint":164},"Columbia University Medical Center - Department of Pediatrics","New York","10032",{"lat":165,"lon":166},40.71427,-74.00597,{"facility":168,"city":162,"state":162,"zip":163,"country":116,"geoPoint":169},"Columbia University Medical Center",{"lat":165,"lon":166},{"facility":171,"city":172,"state":173,"zip":174,"country":116,"geoPoint":175},"Atrium Health","Charlotte","North Carolina","28204",{"lat":176,"lon":177},35.22709,-80.84313,{"facility":179,"city":180,"state":181,"zip":182,"country":116,"geoPoint":183},"Cleveland Clinic","Cleveland","Ohio","44195",{"lat":184,"lon":185},41.4995,-81.69541,{"facility":187,"city":188,"state":181,"zip":189,"country":116,"geoPoint":190},"Nationwide Children's Hospital","Columbus","43205",{"lat":191,"lon":192},39.96118,-82.99879,{"facility":194,"city":195,"state":196,"zip":197,"country":116,"geoPoint":198},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"lat":199,"lon":200},39.95238,-75.16362,{"facility":202,"city":203,"state":204,"zip":205,"country":116,"geoPoint":206},"Medical University of South Carolina","Charleston","South Carolina","29425",{"lat":207,"lon":208},32.77632,-79.93275,{"facility":210,"city":211,"state":212,"zip":213,"country":116,"geoPoint":214},"Tristar Medical Group Children's Specialists\u002FSarah Cannon Center for Blood Cancers","Nashville","Tennessee","37203",{"lat":215,"lon":216},36.16589,-86.78444,{"facility":218,"city":219,"state":220,"zip":221,"country":116,"geoPoint":222},"Texas Oncology - Baylor Charles A. Sammons Cancer Center","Dallas","Texas","75246",{"lat":223,"lon":224},32.78306,-96.80667,{"facility":226,"city":227,"state":220,"zip":228,"country":116,"geoPoint":229},"Cook Children's","Fort Worth","76104",{"lat":230,"lon":231},32.72541,-97.32085,{"facility":233,"city":234,"state":235,"zip":236,"country":237,"geoPoint":238},"Ottawa Hospital Research Institute","Ottawa","Ontario","K1H 8L6","Canada",{"lat":239,"lon":240},45.41117,-75.69812,{"facility":242,"city":243,"state":235,"zip":244,"country":237,"geoPoint":245},"Princess Margaret Cancer Centre-University Health Network","Toronto","M5G 2M9",{"lat":246,"lon":247},43.70643,-79.39864,[],[],[],[],{"nct_id":4,"conditions":253,"biomarkers":255},[18,254],"Transfusion Dependent Beta Thalassemia",[]]