[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Shanghai BDgene Co., Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":156},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,45,73,100,121,140],{"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":28,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100606031","a-clinical-study-on-the-evaluation-of-bd114-for-the-treatment-hpv-16-related-cervical-hsil-100606031",false,"NCT07170254","A Clinical Study on the Evaluation of BD114 for the Treatment HPV-16-Related Cervical HSIL","An Open-Label, Two-Arm, Non-Randomized Clinical Study On The Safety And Efficacy Of Instantaneous CRISPR\u002FCas9 Gene Editing Therapy For Treating Chinese Patients With HPV-16-Related High-Grade Squamous Intraepithelial Lesions (HSIL)","Inclusion Criteria:\n\n1. Female, aged 25 to 50 years, without childbearing demand;\n2. Confirmed histopathological evidence of cervical HSIL (CIN3) at the screening period or within 1 month prior to screening;\n3. Confirmed cervical HPV-16 positive by HPV test during the screening period or within 1 month prior to screening, without other high-risk HPV types detected;\n4. Complete visibility of both the squamocolumnar junction (SCJ Types 1\u002F2) and the upper margin of acetate-white epithelium or suspected HSIL lesions under colposcopy examine during the screening period or within 1 month prior to screening;\n5. No evidence of precancerous lesions or malignancy by endocervical curettage (ECC) ;\n6. The biopsy sampling of cervical lesions are performed;\n7. Visible residual cervical lesions after screening biopsy;\n8. Willing to maintain abstinence or use a highly effective contraceptive method (oral contraceptives, injections, implants, or barrier methods) for women of childbearing potential (WOCBP) from enrollment until Week 36, or her partner undergone surgical sterilization (e.g., vasectomy);\n9. Good compliance to protocol-specified procedure in study duration assessed by investigator;\n10. Voluntarily participating in the study and willing to provided signed informed consent.\n\nExclusion Criteria:\n\n1. Positive detection of antibodies or viral test of human immunodeficiency virus (HIV), or hepatitis B virus (HBV), or hepatitis C virus (HCV), or Treponema pallidum (TP) at screening;\n2. Confirmed histopathologically epithelial carcinoma, glandular carcinoma or precancerous lesions in the endometrium at screening;\n3. Confirmed histopathologically or macroscopically high-grade intraepithelial neoplasia or invasive carcinoma in the vulva, vagina or anus at screening;\n4. HSIL with partial location in cervical canal and incomplete colposcopic visualization ;\n5. Undergone the treatment for cervical HSIL within 4 weeks prior to screening;\n6. Vaccination history of any therapeutic HPV vaccine;\n7. Family history of malignancy, or a history\u002Fcurrent presence of any malignant tumor;\n8. Severe uncontrolled diseases of major organs, including but not limited to: acute myocardial infarction, stroke, liver cirrhosis, severe kidney disease, diabetes mellitus, chronic obstructive pulmonary disease (COPD), hematologic disorders, psychiatric disorders, etc;\n9. Pregnant (a positive urine or serum pregnancy test) or lactating women;\n10. Participating in another drug or device clinical trial at screening, or participated in one within 3 months prior to screening;\n11. The history of any form of gene and\u002For cell therapy;\n12. Drug abuse or alcohol addiction no compliance to protocol-specific procedure;\n13. Any other condition unsuitable for participating this study judged by the investigator.","FEMALE","25 Years","50 Years",{"count":20,"type":21},12,"ESTIMATED","INTERVENTIONAL",[24],"NA","This study is intented to evaluate the safety, and tolerability and preliminary efficacy of Instantaneous CRISPR\u002FCas9 Gene Editing Therapy (BD114 virus-like particle, also BD114) for the treatment of high-grade squamous intraepithelial lesions (HSIL) associated with HPV-16 infection.",[27],"High-grade Squamous Intraepithelial Lesions (HSIL)",[29,30,31],"High-grade squamous intraepithelial lesions","HPV-16 infection","Gene Editing Therapy","RECRUITING","2025-09-05",{"date":35,"type":36},"2025-09-12","ACTUAL",{"date":38,"type":21},"2025-09",{"date":40,"type":21},"2026-12",{"name":42,"class":43},"Shanghai BDgene Co., Ltd.","INDUSTRY",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":52,"minAge":53,"maxAge":54,"enrollmentInfo":55,"targetDuration":4,"studyType":22,"phases":57,"briefSummary":59,"conditions":60,"keywords":62,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":44},"100552542","phase-2-a-phase-a-study-of-the-safety-tolerability-and-efficacy-of-bd111-in-herpes-simplex-virus-type-i-stromal-keratitis-100552542","NCT06474442","A Phase Ⅱa Study of the Safety, Tolerability and Efficacy of BD111 in Herpes Simplex Virus Type I Stromal Keratitis","A Multicenter, Single-blind, Single-dose, Randomized, Phase Ⅱa Trial to Evaluate the Safety, Tolerability and Efficacy of Intrastromal BD111 Gene Editing Therapy in Adults With HSV-1 Stromal Keratitis","Inclusion Criteria: Participants must meet all of the following inclusion criteria to be enrolled in this study.\n\n1. Aged 18 to 70 years old;\n2. Clinically diagnosed with herpes simplex virus stromal keratitis;\n3. Tear swab HSV-1 nucleic acid test (qPCR method) positive;\n4. No use of systemic antiviral drugs or corticosteroids within 48 hours before enrollment;\n5. No systemic immune eye diseases;\n6. Good eyelid structure and blinking function;\n7. Eye structure and function assessment showing potential for visual recovery;\n8. No retinal detachment, with generally normal visual function;\n9. No history of corneal trauma;\n10. Visual acuity in the fellow eye is better than 20\u002F200;\n11. Fertile males or females must use highly effective contraceptive methods (such as oral contraceptives, intrauterine devices, abstinence, or barrier contraception combined with spermicides) during the trial and continue contraception for 12 months after administration;\n12. Participants voluntarily join the study, sign an informed consent form, have good compliance, and cooperate with follow-up visits.\n\nExclusion Criteria: Patients with any of the following conditions cannot be enrolled in this study\n\n1. Active ocular infection caused by other pathogens in the target eye or the fellow eye within 30 days before enrollment, including but not limited to blepharitis, infectious conjunctivitis, keratitis, scleritis, and endophthalmitis;\n2. Patients with bilateral viral keratitis\n3. Previous corneal transplant surgery in the study eye;\n4. A history of adverse reactions or allergies to corticosteroids and sodium fluorescein, allergies to therapeutic or diagnostic protein products, allergies to ≥ two drugs or non-drug factors, or having an ongoing allergic disease;\n5. Absence of tear film and blinking function;\n6. Severe dry eye disease;\n7. Malignant ocular surface tumor;\n8. Glaucoma;\n9. Patients with systemic autoimmune diseases;\n10. Signs of systemic infection before enrollment, including fever and receiving antibiotic treatment (abnormal elevating values in white blood cells, lymphocytes, and neutrophils in routine blood tests);\n11. Abnormal major organ function or other uncontrolled clinical problems, mainly including but not limited to the following:\n\n    * Severe kidney disease history, serum creatinine ≥ 133μmol\u002FL;\n    * Liver dysfunction, transaminase level ≥ 80 IU\u002FL;\n    * Uncontrolled hypertension, systolic blood pressure ≥160 mmHg and\u002For diastolic blood pressure ≥100 mmHg;\n    * Uncontrolled diabetes, fasting blood glucose greater than or equal to 8.0 μmol\u002FL;\n    * Cardiovascular disease history, with arrhythmia, myocardial ischemia, and myocardial infarction (diagnosed by electrocardiogram examination);\n    * Platelet level ≤ 100×10\\^9\u002FμL or ≥ 450×10\\^9\u002FμL due to any cause, hemoglobin level lower than 10.0g\u002FdL (male) or 9.0g\u002FdL (female).\n12. HIV infection;\n13. Pregnant and lactating women (pregnancy in this trial is defined as a positive urine pregnancy test);\n14. Participation in other drug or medical device clinical trials;\n15. Alcohol or drug abuse;\n16. Lack of compliance with the trial or the ability to sign an informed consent form;\n17. Other situations deemed unsuitable for participation in the trial by the investigator.","ALL","18 Years","70 Years",{"count":56,"type":21},40,[58],"PHASE2","This study aims to compare the clinical efficacy and safety of BD111 injection in combination with standard therapy vs. standard therapy in herpes simplex virus type I stromal keratitis (HSK), providing preliminary confirmation of the clinical effectiveness of BD111 in combination with standard therapy.",[61],"Herpes Simplex Virus Type I Stromal Keratitis",[63,64],"Herpes Simplex Virus Type I","Stromal Keratitis","2025-05-14",{"date":67,"type":36},"2025-05-18",{"date":69,"type":36},"2025-04-28",{"date":71,"type":21},"2027-03",{"name":42,"class":43},{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":4,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":52,"minAge":53,"maxAge":80,"enrollmentInfo":81,"targetDuration":4,"studyType":22,"phases":83,"briefSummary":84,"conditions":85,"keywords":87,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":93,"startDateStruct":95,"completionDateStruct":97,"leadSponsor":99,"locationsCount":44},"100551857","crisprcas9-instantaneous-gene-editing-therapy-to-intraocular-hypertensive-poag-with-myoc-mutation-100551857","NCT06465537","CRISPR\u002FCas9 Instantaneous Gene Editing Therapy to Intraocular Hypertensive POAG With MYOC Mutation","A Clinical Study on CRISPR\u002FCas9 Instantaneous Gene Editing Therapy to Primary Open-angle Glaucoma With Elevated Intraocular Pressure and MYOC Gene Mutation","Inclusion Criteria:\n\n1. Signed ICF;\n2. Aged 18 to 65 years old;\n3. Primary open Angle glaucoma (POAG) with elevated intraocular pressure (IOP) was diagnosed with ≥1 year medical history record ;\n4. Good function level of organs;\n5. Good compliance and willing to comply with the visit schedule, laboratory tests and other specified test etc. per protocol;\n6. Agreeing to accept a long-term safety follow-up after 1 year of study.\n\nSpecial Inclusion Criteria for Group 1:\n\n* Target intervenning eye is no visual acuity;\n* The intraocular pressure (IOP) was ≤35 mmHg and \\> 21 mmHg after receiving a combination therapy of 2 or more drugs lowering IOP.\n\nSpecial Inclusion Criteria for Group 2:\n\n* MYOC gene mutation was detected in peripheral blood nucleated cells ;\n* The intraocular pressure (IOP) was ≤30 mmHg and \\> 21 mmHg after receiving a combination therapy of 2 or more drugs lowering IOP;\n* Both eyes have a Shaffer Angle mirror rating greater than 3.\n\nExclusion Criteria:\n\n1. Secondary glaucoma;\n2. Any active or recurrent intraocular infection or inflammation, including but not limited to uveitis;\n3. The target intervenning eye has severe xerophthalmia or clinically significant active corneal disease;\n4. Any condition no accepting the measure of IOP;\n5. Any positive of human immunodeficiency virus type 1\u002F2 (HIV-1\u002FHIV-2) antibody, treponema pallidum (TP) specific antibody, human T-lymphotropic virus type 1 or 2 (HTLV-1\u002FHTLV-2) antibody, or vesicular stomatitis virus G (VSV-G) antibody;\n6. Any of hepatitis B virus (HBV) HbsAg or HBV-DNA, hepatitis C virus (HCV) HCAb, or epstein-barr virus (EBV), or cytomegalovirus (CMV) nucleic acid test is positive;\n7. Severe active bacterial, viral, fungal, malaria or parasitic systemic infection;\n8. Any past or present malignancy, myeloproliferative or immunodeficient disease;\n9. History of major organ diseases or abnormalities in laboratory tests, including:\n\n   1. Liver cirrhosis, liver fibrosis or active hepatitis, and\u002For abnormal liver function tests (serum total bilirubin (TBIL) ≥1.5 x upper limit of normal (ULN); Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥2.5×ULN; Alkaline phosphatase ≥2.5 × ULN);\n   2. Cardiovascular and cerebrovascular diseases, including uncontrolled hypertension, myocardial infarction, myocarditis, arrhythmia, stroke, etc.;\n   3. Kidney disease, or creatinine ≥ 1.5ULN and creatinine clearance \\\u003C 30% normal level (using the Cockcroft-Gault equation);\n   4. Endocrine disorders, such as insulin-dependent diabetes mellitus, hyperthyroidism or hypothyroidism;\n   5. Severe pulmonary hypertension, chronic obstructive pulmonary disease, interstitial pneumonia;\n10. Any severe psychiatric disorders;\n11. Participating in another clinical study of a drug or device, or administrated the investigational drug within 42 days prior to the screening visit;\n12. Pregnant or lactating women;\n13. Refusing to accept any contraception measures;\n14. Allergic to clinical investigational drugs or their excipients;\n15. Other conditions assessed by the investigator as unsuitable for participation in this study.\n\nSpecial Exclusion Criteria for Group 2:\n\n* Retinal diseases: complicated with unexplained quadrant blindness, neovascularization age-related macular degeneration, retinal branch vein obstruction, central retinal vein obstruction, cystoid macular edema, macular hiatal hole and central serous retinopathy;\n* A history of anterior chamber angle stenosis, congenital glaucoma, or angle closure, clinically significant anterior peripheral adhesion, or extensive cicatricial adhesion caused by surgery\u002Flaser therapy in the anterior chamber angle;\n* The central corneal thickness is less than 480 μm or more than 620 μm.","65 Years",{"count":82,"type":21},9,[24],"This study is intented to evaluate the safety, tolerability and preliminary efficacy of CRISPR\u002FCas9 Instantaneous Gene Editing Therapy (BD113 virus-like particle, also BD113vLVP) in patients with primary open-angle glaucoma (POAG) with elevated intraocular pressure and MYOC gene mutation. The main objectives to evaluate the safety and tolerability BD113vLVP) in POAG patients with intraocular hypertension and MYOC mutation, and secondary objectives is to explore the preliminary efficacy and the metabolism characteristics of BD113vLVP in participants.",[86],"Primary Open Angle Glaucoma",[88,89,90,91],"primary open angle glaucoma","POAG","elevated intraocular pressure","MYOC gene mutation","2024-06-19",{"date":94,"type":36},"2024-06-24",{"date":96,"type":36},"2024-06-10",{"date":98,"type":21},"2025-12",{"name":42,"class":43},{"id":101,"slug":102,"hasResults":11,"nctId":103,"briefTitle":104,"officialTitle":105,"acronym":4,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":52,"minAge":107,"maxAge":108,"enrollmentInfo":109,"targetDuration":4,"studyType":22,"phases":110,"briefSummary":112,"conditions":113,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":115,"startDateStruct":116,"completionDateStruct":118,"leadSponsor":119,"locationsCount":120},"100551858","phase-1-a-phase-1-study-of-gene-modified-autologous-hematopoietic-stem-cell-bd211-treating--thalassemia-major-100551858","NCT06465550","A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major","A Phase 1 Clinical Trail of the Safety and Efficacy of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients","Inclusion Criteria:\n\n1. Participants aged 3 years (inclusive) to 18 years (exclusive), with no gender restrictions.\n2. Parents\u002Flegal guardians have fully understood and voluntarily signed a written informed consent form; and it is recommended that children aged 8 and above be involved in the decision to participate in this clinical trial and obtain a written consent form.\n3. Transfusion-dependent β-thalassemia patients. \"Transfusion-dependent\" is defined as: requiring at least 100 mL\u002Fkg of packed red blood cells annually; the genotype can be β0\u002Fβ0, β0\u002Fβ+, or β+\u002Fβ+, diagnosed through hemoglobin studies.\n4. Eligible for allogeneic hematopoietic stem cell transplantation, but without a donor or those refusing to undergo allogeneic hematopoietic stem cell transplantation.\n5. Have undergone symptomatic treatment for at least the past 2 years and have retained medical records including transfusion history.\n6. Stable condition and maintained an appropriate iron chelation regimen.\n7. Good status of organ function.\n8. Good compliance from the individual and parents\u002Flegal guardians, willing to adhere to visit schedules, trial plans, laboratory tests, and other trial procedures as stipulated in this protocol.\n9. Willing to participate in long-term follow-up research.\n\nExclusion Criteria:\n\n1. Has a fully HLA-matched hematopoietic stem cell donor and is willing to receive a fully HLA-matched hematopoietic stem cell transplant. Enrollment is otherwise only advised after review by the safety review committee.\n2. Positive for antibodies against Human Immunodeficiency Virus 1\u002F2 (HIV-1\u002FHIV-2), Treponema pallidum (TP) specific antibodies, Human T-lymphotropic Virus 1 or 2 (HTLV-1\u002FHTLV-2) antibodies, and Vesicular Stomatitis Virus G (VSV-G).\n3. Positive for Hepatitis B Virus (HBV) HbsAg or HBV-DNA; Hepatitis C Virus (HCV) HCAb positive; positive nucleic acid test for Epstein-Barr Virus (EBV) or Cytomegalovirus (CMV).\n4. Severe active bacterial, viral, fungal, malarial, or parasitic infections.\n5. Has had, or currently has, a malignant, myeloproliferative, or immunodeficiency disorder.\n6. Direct relatives with known or suspected hereditary cancer syndromes (including but not limited to breast cancer, colorectal cancer, ovarian cancer, prostate cancer, and pancreatic cancer).\n7. Autoimmune diseases that could result in transfusion difficulties.\n8. Major organ diseases or abnormal lab tests, including:\n\n   1. Liver cirrhosis, fibrosis, or active hepatitis, and\u002For abnormal liver function tests (Serum total bilirubin (TBIL) ≥ 1.5x Upper Limit of Normal (ULN); Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≥ 2.5x ULN; Alkaline phosphatase ≥ 2.5x ULN).\n   2. Heart disease, or Left Ventricular Ejection Fraction (LVEF) \\\u003C 60%.\n   3. Kidney diseases, or serum creatinine ≥ 1.5ULN, creatinine clearance rate \\\u003C 30% of the normal level (measured or calculated by the Cockcroft-Gault equation).\n   4. Endocrine disorders, such as insulin-dependent diabetes, hyperthyroidism, or hypothyroidism.\n   5. Severe iron overload, serum ferritin ≥ 5000 ng\u002FmL.\n   6. Cardiac T2\\* \\\u003C 20 ms, and\u002For liver iron content (LIC) ≥ 15mg\u002Fg liver weight by MRI.\n   7. Significant pulmonary hypertension diagnosed clinically according to guidelines, requiring clinical medical intervention.\n9. Uncorrected bleeding disorders.\n10. Severe psychiatric disorders.\n11. Peripheral blood white cell (WBC) count \\\u003C 3x10\\^9\u002FL or platelets count \\\u003C 120x10\\^9\u002FL.\n12. Received hydroxyurea treatment within the last 3 months before stem cell collection.\n13. Used erythropoiesis-stimulating agents within the 3 months prior to HSC collection.\n14. History of allogeneic transplantation.\n15. Previously received any type of gene and\u002For cell therapy.\n16. Participating in another clinical trial and is within a 30-day screening period.\n17. Has contraindications to anesthesia.\n18. Has contraindications to hematopoietic stem cell collection.\n19. Allergic to the investigational drug or its excipients.\n20. Any other conditions determined by the investigator as unsuitable for participation in this clinical trial.","3 Years","35 Years",{"count":82,"type":21},[111],"PHASE1","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.",[114],"β-thalassemia",{"date":94,"type":36},{"date":117,"type":36},"2024-01-05",{"date":40,"type":21},{"name":42,"class":43},3,{"id":122,"slug":123,"hasResults":11,"nctId":124,"briefTitle":125,"officialTitle":126,"acronym":4,"eligibilityCriteria":127,"healthyVolunteers":11,"sex":52,"minAge":107,"maxAge":53,"enrollmentInfo":128,"targetDuration":4,"studyType":22,"phases":130,"briefSummary":112,"conditions":131,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":132,"lastUpdatePostDateStruct":133,"startDateStruct":135,"completionDateStruct":137,"leadSponsor":139,"locationsCount":44},"100498705","safety-and-efficacy-of-gene-modified-autologous-hematopoietic-stem-cells-to-treat-transfusion-dependent-beta-thalassemia-100498705","NCT05773729","Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-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 3 to 18 years old, including:\n\n   The parents or legal guardians must be able to understand and provide ICFs. If available, it is strongly recommended that children aged ≥8 years in treatment decisions and obtain written ICFs and be clearly documented; 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. No eligiblity for allogeneic hematopoietic stem cell transplantation.\n3. The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.\n4. The subjects' parents\u002Flegal guardians must be willing and able to follow the study procedures in the study protocol.\n5. Good organs' functions.\n6. 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. Had autoimmune diseases that may make blood transfusions difficult.\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. There are bleeding diseases that have not been cured.\n15. The subject involved with another clinical study in a 30-day screening period.\n16. Allergic to the research drug and its excipients.\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.\n20. Had diseases that interfere with hematopoietic stem cells collections.\n21. Any other conditions being ineligible for HSC transplantation determined by the investigator.",{"count":129,"type":21},10,[24],[114],"2024-06-13",{"date":134,"type":36},"2024-06-14",{"date":136,"type":36},"2023-09-15",{"date":138,"type":21},"2026-10",{"name":42,"class":43},{"id":141,"slug":142,"hasResults":11,"nctId":143,"briefTitle":144,"officialTitle":126,"acronym":4,"eligibilityCriteria":145,"healthyVolunteers":11,"sex":52,"minAge":146,"maxAge":108,"enrollmentInfo":147,"targetDuration":4,"studyType":22,"phases":148,"briefSummary":112,"conditions":149,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":132,"lastUpdatePostDateStruct":151,"startDateStruct":152,"completionDateStruct":154,"leadSponsor":155,"locationsCount":44},"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","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":129,"type":21},[24],[150],"β-thalassemia Major",{"date":134,"type":36},{"date":153,"type":36},"2023-08-10",{"date":138,"type":21},{"name":42,"class":43},""]