[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"-thalassemia-major\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:-thalassemia-major":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,41],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"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":20,"type":21},20,"ESTIMATED","INTERVENTIONAL",[24],"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",[27],"β-thalassemia Major","NOT_YET_RECRUITING","2026-03-19",{"date":31,"type":32},"2026-03-24","ACTUAL",{"date":34,"type":21},"2026-04-05",{"date":36,"type":21},"2028-07-31",{"name":38,"class":39},"CorrectSequence Therapeutics Co., Ltd","INDUSTRY",3,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":18,"enrollmentInfo":49,"targetDuration":4,"studyType":22,"phases":51,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":56,"lastUpdatePostDateStruct":57,"startDateStruct":59,"completionDateStruct":61,"leadSponsor":63,"locationsCount":65},"100498893","safety-and-efficacy-of-gene-modified-autologous-hematopoietic-stem-cells-to-treat-transfusion-dependent--thalassemia-100498893","NCT05776173","Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia","Safety and Efficacy of Lentiviral Vector Transduction of β-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent β-thalassemia","Inclusion Criteria:\n\n1. Ages 6 to 35 years old, including:\n\n   Subjects should be able to provide an ICF. Diagnosed as Transfusion Dependent β-thalassemia with any genotype (β0, β+, βE\u002Fβ0, βS\u002FS, βS\u002Fβ0, βS\u002Fβ+), confirmed the Hb analysis. No alfa chain genetic abnormalities. Subjects must stabilize and maintain an appropriate iron chelation regimen. Transfusion-dependent types are defined as requiring at least 100 mL\u002Fkg\u002F year of red blood cells (pRBCs).\n2. The tumor genes chip detection results about acute leukemia and myeloid tumor gene mutations (panel) showed no abnormality.\n3. There were candidates for HLA gene semi-compatible hematopoietic stem cell transplantation.\n4. No eligiblity for allogeneic hematopoietic stem cell transplantation.\n5. The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.\n6. The investigator confirmed that subject was willing to follow the research procedures.\n7. Having complete medical records including a history of blood transfusions testified subject received treatment and followed up for at least two years prior to screening.\n\nExclusion Criteria:\n\n1. Availability of voluntary, fully HLA-matched hematopoietic cell donors, unless recommended for inclusion by the Monitoring Committee.\n2. HIV-1 and HIV-2 were positive, and \u002F or HTLV-1, HTLV-2 and VSV-G antibodies were positive.\n3. An active bacterial, viral, fungal or parasitic infection.\n4. Contraindicated for the extraction of bone marrow under anesthesia.\n5. Any malignancy, myeloproliferative, or immunodeficient disease and relevant medical history.\n6. Peripheral blood white blood cell (WBC) count \\\u003C 3×10\\^9\u002FL or platelet count \\\u003C 120×10\\^9\u002FL.\n7. A history of allo-transplantation.\n8. Erythropoietin was used within 3 months prior to HSC cell collection.\n9. Immediate family members with known or suspected familial cancer syndromes (including but not limited to breast, colorectal, ovarian, prostate, and pancreatic cancers).\n10. Subjects with a diagnosis of major mental illness may had a serious disability to participate in the study.\n11. Active recurrent malaria.\n12. Pregnant or postpartum nursing or unable to use contraception.\n13. History of major organ injury including:\n\n    Liver disease, transaminase \\> 3 times the upper limit of normal. (If the liver biopsy does not reveal evidence of widespread bridging fibrosis, cirrhosis, or acute hepatitis, this indicator will not be used as a criterion for the exclusion); Widely bridging fibrosis, histopathological evidence of acute hepatitis or cirrhosis showed in liver biopsy Heart disease, left ventricular ejection fraction \\\u003C 25%; Kidney disease, creatinine clearance \\\u003C 30% normal level; Of severe iron overload, confirmed by the study doctor; An heart MRI detection of T2 \\* \\\u003C 10 ms; Significant pulmonary hypertension needing clinical medical intervention.\n14. Any other conditions being ineligible for HSC transplantation determined by the investigator.\n15. The subject involved with another clinical study in a 30-day screening period.\n16. Subjects who expected to become parents during the 27-month study period.\n17. Prior treatment with any type of gene and\u002For cell therapy.\n18. As assessed by the investigator, the subjects or their parents are unable to comply well with the study procedures per protocol.\n19. Hydroxyurea treatment within 3 months prior to hematopoietic stem cell collection.","6 Years",{"count":50,"type":21},10,[52],"NA","This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.",[27],"RECRUITING","2024-06-13",{"date":58,"type":32},"2024-06-14",{"date":60,"type":32},"2023-08-10",{"date":62,"type":21},"2026-10",{"name":64,"class":39},"Shanghai BDgene Co., Ltd.",1]