[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100630555":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":18,"centralContacts":19,"locations":29,"responsibleParty":70,"collaborators":18,"id":72,"slug":73,"hasResults":74,"nctId":75,"briefTitle":76,"officialTitle":77,"acronym":18,"eligibilityCriteria":78,"healthyVolunteers":74,"sex":79,"minAge":80,"maxAge":81,"enrollmentInfo":82,"targetDuration":18,"studyType":85,"phases":86,"briefSummary":88,"conditions":89,"keywords":18,"overallStatus":91,"whyStopped":18,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":93,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":101},{"fullName":5,"class":6},"CorrectSequence Therapeutics Co., Ltd","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"CS-101 injection","EXPERIMENTAL","Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique",[13],"Genetic: CS-101 injection",[15],{"type":16,"name":9,"description":11,"armGroupLabels":17,"otherNames":18},"GENETIC",[9],null,[20,25],{"name":21,"role":22,"phone":23,"phoneExt":18,"email":24},"Xiaokai Li","CONTACT","+86 18686610731","xiaokai.li@correctsequence.com",{"name":26,"role":22,"phone":27,"phoneExt":18,"email":28},"Yaliang Li","+8618621046122","yaliang.li@correctsequence.com",[30,47,60],{"facility":31,"status":18,"city":32,"state":33,"zip":18,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":42},"The First Affiliated Hospital of Guangxi Medical University","Nanning","Guangxi","China","CN",{"type":37,"coordinates":38},"Point",[39,40],108.31667,22.81667,{"lat":40,"lon":39},[43,45],{"name":44,"role":22,"phone":18,"phoneExt":18,"email":18},"Yongrong Lai",{"name":44,"role":46,"phone":18,"phoneExt":18,"email":18},"PRINCIPAL_INVESTIGATOR",{"facility":48,"status":18,"city":49,"state":50,"zip":18,"country":34,"countryCode":35,"cosmosGeoPoint":51,"geoPoint":55,"contacts":56},"Children's Hospital of Fudan University","Shanghai","Shanghai Municipality",{"type":37,"coordinates":52},[53,54],121.45806,31.22222,{"lat":54,"lon":53},[57,59],{"name":58,"role":22,"phone":18,"phoneExt":18,"email":18},"Xiaowen Zhai",{"name":58,"role":46,"phone":18,"phoneExt":18,"email":18},{"facility":61,"status":18,"city":49,"state":50,"zip":18,"country":34,"countryCode":35,"cosmosGeoPoint":62,"geoPoint":64,"contacts":65},"Ruijin Hospital Shanghai JiaoTong University School of Medicine",{"type":37,"coordinates":63},[53,54],{"lat":54,"lon":53},[66,68],{"name":67,"role":46,"phone":18,"phoneExt":18,"email":18},"Saijuan Chen",{"name":69,"role":46,"phone":18,"phoneExt":18,"email":18},"Weili Zhao",{"type":71,"investigatorFullName":18,"investigatorTitle":18,"investigatorAffiliation":18,"oldNameTitle":18,"oldOrganization":18},"SPONSOR","100630555","phase-2-a-phase-2-safety-and-efficacy-study-evaluating-cs-101-in-participants-with--thalassemia-major-100630555",false,"NCT07489196","A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major","A Single-arm, Open-label Phase II Clinical Trial: Evaluating the Safety and Efficacy of a Single Dose of CS-101 Injection in Participants With β-thalassemia Major","Inclusion Criteria:\n\n* Voluntarily signed informed consent. Male or female participants aged 12 to 35 years (inclusive). The participant or their legally authorized representative must sign the informed consent. If the participant is under 18 years of age, their legally authorized representative must also sign the informed consent.\n* Diagnosed with β-thalassemia major (transfusion-dependent). Received at least 8 units of red blood cell transfusions within 12 months prior to screening, and documented hemoglobin level ≤ 70 g\u002FL pre-transfusion.\n* Good general condition: Karnofsky score (≥16 years of age) ≥ 60, or Lansky Play-Performance score (\\\u003C16 years of age) ≥ 60.\n* For females of childbearing potential: From the start of the screening, highly effective contraception or complete abstinence (if this is their usual lifestyle), and agree to maintain such contraception throughout the study.\n* For males of childbearing potential: Use condoms or other methods to ensure effective contraception for sexual partners continuously from mobilization through the study period.\n\nExclusion Criteria:\n\n* Received other investigational products or other experimental interventions within 30 days prior to signing informed consent or within 6 elimination half-lives of the drug (whichever is longer).\n* Received or is receiving thalidomide, hydroxyurea, and\u002For luspatercept within 3 months prior to screening.\n* Previous received allogeneic hematopoietic stem cell transplantation, gene therapy, or gene-editing therapy; or participants who can be maintained with standard therapy.\n* Participants with a matched sibling donor, or with a matched unrelated \u002F haploidentical related donor and judged by the investigator to have no high-risk factors for allogeneic hematopoietic stem cell transplantation.\n* Participants with coexisting α-thalassemia with more than 2 α-globin chain gene deletions or non-deletional mutations.\n* Known hypersensitivity to drugs used during autologous hematopoietic stem cell transplantation, excipients, or devices, judged by the investigator to be ineligible for this study.\n* Infection with HIV, cytomegalovirus, Epstein-Barr virus, or Treponema pallidum during screening; active HBV or HCV infection (participants with stable hepatitis B after treatment (HBV-DNA negative) and cured hepatitis C (HCV-RNA negative) may be included). Known active bacterial, viral, fungal, or parasitic infection.\n* Echocardiographic ejection fraction \\\u003C 50%.\n* Laboratory abnormalities: AST or ALT \\> 3 × upper limit of normal (ULN); or International normalized ratio (INR) \\> 1.5 × ULN.\n* Cardiac severe iron overload detected by MRI during screening, judged by the investigator to be unsuitable for hematopoietic stem cell transplantation.\n* Current or history of malignancy.\n* Participants with known neurological consciousness disorders, psychological problems, or psychiatric diseases judged by the investigator to be unable to comply with study procedures.\n* Participants with known history of uncontrolled seizures judged by the investigator to be ineligible for this study.\n* Uncontrolled bleeding disorders.\n* Leukocyte count \\\u003C 3 × 10⁹\u002FL and\u002For platelet count \\\u003C 100 × 10⁹\u002FL not due to hypersplenism.\n* Participants with other severe cardiovascular, pulmonary, renal, gastrointestinal, hepatic diseases, and\u002For other organ disorders judged by the investigator to be ineligible for this study.\n* Pregnant or lactating females; females of childbearing potential with a positive serum pregnancy test.\n* Received live or live-attenuated vaccine within 90 days prior to myeloablation.\n* Participants with autoimmune diseases","ALL","12 Years","35 Years",{"count":83,"type":84},20,"ESTIMATED","INTERVENTIONAL",[87],"PHASE2","The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major",[90],"β-thalassemia Major","NOT_YET_RECRUITING","2026-03-19",{"date":94,"type":95},"2026-03-24","ACTUAL",{"date":97,"type":84},"2026-04-05",{"date":99,"type":84},"2028-07-31",{"name":5,"class":6},3]