[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05477563":3,"trial-entities:NCT05477563":168,"trial-summary:NCT05477563":172},{"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":15,"conditions":17,"keywords":25,"study_type":26,"primary_purpose":27,"phases":28,"enrollment_info":30,"interventions":33,"primary_outcomes":42,"secondary_outcomes":48,"sex":89,"minimum_age":90,"maximum_age":91,"healthy_volunteers":92,"eligibility_criteria":93,"std_ages":113,"locations":116,"central_contacts":159,"overall_officials":165,"references":166,"see_also_links":167},"NCT05477563","VX21-CTX001-161","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","RECRUITING","2027-06-09","2026-03","2026-03-23","2022-08-02","Vertex Pharmaceuticals Incorporated","INDUSTRY",null,"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.",[18,19,20,21,22,23,24],"Beta-Thalassemia","Thalassemia","Hematologic Diseases","Genetic Diseases, Inborn","Hemoglobinopathies","Sickle Cell Disease","Sickle Cell Anemia",[],"INTERVENTIONAL","TREATMENT",[29],"PHASE3",{"count":31,"type":32},26,"ESTIMATED",[34],{"type":35,"name":36,"description":37,"armGroupLabels":38,"otherNames":39},"BIOLOGICAL","CTX001","Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan",[36],[40,41],"Exagamglogene autotemcel","Exa-cel",[43,46],{"measure":44,"timeFrame":45},"Fetal Hemoglobin (HbF) Concentration Over Time","Up to 12 Months After CTX001 Infusion",{"measure":47,"timeFrame":45},"Total Hemoglobin (Hb) Concentration Over Time",[49,52,55,57,60,63,65,68,70,72,74,77,79,81,83,85,87],{"measure":50,"timeFrame":51},"TDT and SCD: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)","From Signing of Informed Consent up to 12 Months After CTX001 Infusion",{"measure":53,"timeFrame":54},"TDT and SCD: Proportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days)","Within 42 Days After CTX001 Infusion",{"measure":56,"timeFrame":45},"TDT and SCD: Time to Engraftment",{"measure":58,"timeFrame":59},"TDT and SCD: Incidence of Transplant-Related Mortality (TRM) Within 100 Days After CTX001 Infusion","Within 100 Days After CTX001 Infusion",{"measure":61,"timeFrame":62},"TDT and SCD: Incidence of TRM Within 12 Months After CTX001 Infusion","Within 12 Months After CTX001 Infusion",{"measure":64,"timeFrame":51},"TDT and SCD: Incidence of All-cause Mortality",{"measure":66,"timeFrame":67},"TDT and SCD: Relative Reduction in Annualized Volume of RBC Transfusions","From Day 60 up to 12 Months After CTX001 Infusion",{"measure":69,"timeFrame":45},"TDT and SCD: Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time",{"measure":71,"timeFrame":45},"TDT and SCD: Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time",{"measure":73,"timeFrame":45},"TDT: Duration Transfusion Free in Participants",{"measure":75,"timeFrame":76},"SCD: Relative Reduction in Annualized Rate of Severe Vaso-Occlusive Crises (VOCs)","From Baseline up to 12 Months After CTX001 Infusion",{"measure":78,"timeFrame":76},"SCD: Relative Reduction in Annualized Rate of Inpatient Hospitalizations for Severe VOCs",{"measure":80,"timeFrame":76},"SCD: Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs",{"measure":82,"timeFrame":76},"SCD: Relative Reduction in Haptoglobin",{"measure":84,"timeFrame":76},"SCD: Relative Reduction in Lactate dehydrogenase",{"measure":86,"timeFrame":76},"SCD: Relative Reduction in Total Bilirubin",{"measure":88,"timeFrame":76},"SCD: Relative Reduction in Indirect Bilirubin","ALL","12 Years","35 Years",false,{"inclusion":94,"exclusion":105,"raw_text":112},[95,96,97,98,99,100,101,102,103,104],"Participants with TDT and SCD:","Eligible for autologous stem cell transplant as per investigator's judgment.","Participants with TDT:","Diagnosis of TDT as defined by:","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","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","Participants with SCD:","Diagnosis of severe SCD as defined by:","Documented SCD genotypes","History of at least two severe VOCs events per year for the previous two years prior to enrollment",[95,106,107,108,97,109,110,101,111],"A willing and healthy 10\u002F10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement","Prior hematopoietic stem cell transplant (HSCT)","Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator","Participants with associated α-thalassemia and \\>1 alpha deletion, or alpha multiplications","Participants with sickle cell β-thalassemia variant","History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening","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.",[114,115],"CHILD","ADULT",[117,125,133,141,148,155],{"facility":118,"status":8,"city":119,"state":119,"zip":120,"country":121,"geoPoint":122},"New York Presbyterian Hospital - Morgan Stanley Children's Hospital","New York","10032","United States",{"lat":123,"lon":124},40.71427,-74.00597,{"facility":126,"status":8,"city":127,"state":128,"zip":129,"country":121,"geoPoint":130},"Levine Children's Hospital - Hematology","Charlotte","North Carolina","28203",{"lat":131,"lon":132},35.22709,-80.84313,{"facility":134,"status":8,"city":135,"state":136,"zip":137,"country":121,"geoPoint":138},"TriStar Medical Group Children's Specialists - Pediatric Oncology","Nashville","Tennessee","37203",{"lat":139,"lon":140},36.16589,-86.78444,{"facility":142,"status":8,"city":143,"country":144,"geoPoint":145},"University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology","Düsseldorf","Germany",{"lat":146,"lon":147},51.22319,6.77927,{"facility":149,"status":8,"city":150,"country":151,"geoPoint":152},"IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica","Rome","Italy",{"lat":153,"lon":154},41.89193,12.51133,{"facility":156,"status":8,"city":157,"country":158},"King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology","Al Mathar Ash Shamali","Saudi Arabia",[160],{"name":161,"role":162,"phone":163,"email":164},"Medical Information","CONTACT","6173416777","medicalinfo@vrtx.com",[],[],[],{"nct_id":4,"conditions":169,"biomarkers":171},[170,23],"Beta Thalassemia",[],{"nct_id":4,"found":173,"summary":174,"prompt_version":184},true,{"design":175,"status":176,"heading":177,"summary":178,"follow_up":179,"word_count":180,"commitments":181,"compensation":182,"drugs_mentioned":183},"This is an open-label study, meaning both you and the researchers will know what treatment you are receiving. It plans to enroll 26 participants.","completed","CTX001 for Transfusion-Dependent Beta-Thalassemia and Severe Sickle Cell Disease","This study is looking at a single dose of CTX001 for people aged 12 to 35 with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD). CTX001 is a treatment that uses your own modified blood stem cells. Researchers want to see how safe and effective this treatment is. To be eligible, you must be considered suitable for a stem cell transplant by your doctor. For those with TDT, you need a confirmed diagnosis of homozygous beta-thalassemia or compound heterozygous beta-thalassemia. The study will measure changes in your fetal hemoglobin (HbF) and total hemoglobin (Hb) levels for up to 12 months after receiving CTX001 to see if it helps reduce the need for blood transfusions. The study plans to enroll 26 participants, but its current status is unclear.","You will be followed for up to 12 months after receiving the CTX001 infusion.",128,"Not specified in the trial record.","Not stated in the trial record.",[36],"v2"]