[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100475948":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":22,"centralContacts":23,"locations":29,"responsibleParty":88,"collaborators":22,"id":90,"slug":91,"hasResults":92,"nctId":93,"briefTitle":94,"officialTitle":95,"acronym":22,"eligibilityCriteria":96,"healthyVolunteers":92,"sex":97,"minAge":98,"maxAge":99,"enrollmentInfo":100,"targetDuration":22,"studyType":103,"phases":104,"briefSummary":106,"conditions":107,"keywords":22,"overallStatus":32,"whyStopped":22,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":116,"startDateStruct":119,"completionDateStruct":121,"leadSponsor":123,"locationsCount":124},{"fullName":5,"class":6},"Vertex Pharmaceuticals Incorporated","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"CTX001","EXPERIMENTAL","CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter.",[13],"Biological: CTX001",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"BIOLOGICAL","Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan",[9],[20,21],"Exagamglogene autotemcel","Exa-cel",null,[24],{"name":25,"role":26,"phone":27,"phoneExt":22,"email":28},"Medical Information","CONTACT","6173416777","medicalinfo@vrtx.com",[30,43,53,63,73,83],{"facility":31,"status":32,"city":33,"state":33,"zip":34,"country":35,"countryCode":36,"cosmosGeoPoint":37,"geoPoint":42,"contacts":22},"New York Presbyterian Hospital - Morgan Stanley Children's Hospital","RECRUITING","New York","10032","United States","US",{"type":38,"coordinates":39},"Point",[40,41],-74.00597,40.71427,{"lat":41,"lon":40},{"facility":44,"status":32,"city":45,"state":46,"zip":47,"country":35,"countryCode":36,"cosmosGeoPoint":48,"geoPoint":52,"contacts":22},"Levine Children's Hospital - Hematology","Charlotte","North Carolina","28203",{"type":38,"coordinates":49},[50,51],-80.84313,35.22709,{"lat":51,"lon":50},{"facility":54,"status":32,"city":55,"state":56,"zip":57,"country":35,"countryCode":36,"cosmosGeoPoint":58,"geoPoint":62,"contacts":22},"TriStar Medical Group Children's Specialists - Pediatric Oncology","Nashville","Tennessee","37203",{"type":38,"coordinates":59},[60,61],-86.78444,36.16589,{"lat":61,"lon":60},{"facility":64,"status":32,"city":65,"state":22,"zip":22,"country":66,"countryCode":67,"cosmosGeoPoint":68,"geoPoint":72,"contacts":22},"University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology","Düsseldorf","Germany","DE",{"type":38,"coordinates":69},[70,71],6.77927,51.22319,{"lat":71,"lon":70},{"facility":74,"status":32,"city":75,"state":22,"zip":22,"country":76,"countryCode":77,"cosmosGeoPoint":78,"geoPoint":82,"contacts":22},"IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica","Rome","Italy","IT",{"type":38,"coordinates":79},[80,81],12.51133,41.89193,{"lat":81,"lon":80},{"facility":84,"status":32,"city":85,"state":22,"zip":22,"country":86,"countryCode":87,"cosmosGeoPoint":22,"geoPoint":22,"contacts":22},"King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology","Al Mathar Ash Shamali","Saudi Arabia","SA",{"type":89,"investigatorFullName":22,"investigatorTitle":22,"investigatorAffiliation":22,"oldNameTitle":22,"oldOrganization":22},"SPONSOR","100475948","phase-3-evaluation-of-efficacy-and-safety-of-a-single-dose-of-ctx001-in-participants-with-transfusion-dependent--thalassemia-and-severe-sickle-cell-disease-100475948",false,"NCT05477563","Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease","A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease","Key Inclusion Criteria:\n\n* Participants with TDT and SCD:\n* Eligible for autologous stem cell transplant as per investigator's judgment.\n* Participants with TDT:\n* Diagnosis of TDT as defined by:\n* Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia\u002Fhemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning\n* History of at least 100 milliliter (mL)\u002Fkilograms (kg)\u002Fyear or 10 units\u002Fyear of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening\n* Participants with SCD:\n* Diagnosis of severe SCD as defined by:\n* Documented SCD genotypes\n* History of at least two severe VOCs events per year for the previous two years prior to enrollment\n\nKey Exclusion Criteria:\n\n* Participants with TDT and SCD:\n* A willing and healthy 10\u002F10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement\n* Prior hematopoietic stem cell transplant (HSCT)\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator\n* Participants with TDT:\n* Participants with associated α-thalassemia and \\>1 alpha deletion, or alpha multiplications\n* Participants with sickle cell β-thalassemia variant\n* Participants with SCD:\n* History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.","ALL","12 Years","35 Years",{"count":101,"type":102},26,"ESTIMATED","INTERVENTIONAL",[105],"PHASE3","This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.",[108,109,110,111,112,113,114],"Beta-Thalassemia","Thalassemia","Hematologic Diseases","Genetic Diseases, Inborn","Hemoglobinopathies","Sickle Cell Disease","Sickle Cell Anemia","2026-03-18",{"date":117,"type":118},"2026-03-23","ACTUAL",{"date":120,"type":118},"2022-08-02",{"date":122,"type":102},"2027-06-09",{"name":5,"class":6},6]