[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"transfusion-dependent-beta-thalassemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:transfusion-dependent-beta-thalassemia":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,41,68,98,125,147,184,208],{"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},"100645364","phase-2-gene-modified-stem-cell-therapy-for-subjects-with-transfusion-dependent-beta-thalassemia-100645364",false,"NCT07680803","Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia","A Single Arm, Open Label, Multicenter, Single-dose, Phase 2b Clinical Study Evaluating Efficacy and Safety of Gene Therapy Using Autologous CD34+ Hematopoietic Stem Cells Transduced With the GLOBE Lentiviral Vector Using an Improved Transduction Protocol in Subjects With Transfusion-dependent Beta-thalassemia","Inclusion Criteria:\n\n1. Must be willing to adhere to the protocol as evidenced by written informed consent for adults or parental informed consent and subject assent for adolescents and children.\n2. Male and female adults\u002Fadolescents\u002Fchildren diagnosed with transfusion-dependent β-thalassemia (homozygous or compound heterozygous). At least 2 out of the 9 patients must have B0\u002FB0 or B0\u002FB0-like genotype. In case the genetic diagnosis available at screening wasn't performed in a certified laboratory (check under PI's or delegated investigator's responsibility), the genetic diagnosis will be repeated at clinical sites during the screening phase.\n3. Documented history of at least 100 ml\u002Fkg\u002Fyear or 10 U\u002Fyear of packed red blood cell transfusions in each of the 2 years prior to signing informed consent.\n4. Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group 2.\n5. Karnofsky Index or Lansky ≥ 80%.\n6. Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT as evidenced by:\n\n   1. Left ventricular ejection fraction (LVEF) greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease. Absence of severe pulmonary hypertension.\n   2. Diffusing capacity of the lung for carbon monoxide (DLCO) \\> 50% and forced expiratory volume in 1 sec (FEV1) and forced expiratory vital capacity (FVC) \\> 60% predicted (if non cooperative: pulse oximetry \\> 95 % in room air).\n   3. Serum creatinine \\\u003C 2 x upper limit of normal and estimated GFR \\> 60 ml\u002Fmin\u002F1.73m2, calculated using the CKD-EPI formula (Levey 2009) for adults and the modified Schwartz formula (Schwartz 2009) for pediatric patients.\n   4. Absent-mild-moderate liver iron overload on T2\\*MRI (i.e. LIC \\\u003C 15 mg Fe\u002Fgr dry weight assessed at screening. T2\\*MRI at screening can be avoided if performed less than 6 months before enrolment at treatment centers).\n   5. Absent-mild-moderate cardiac iron overload T2\\*MRI (i.e. \\> 20 msec assessed at screening. T2\\*MRI at screening can be avoided if performed less than 6 months before enrolment at treatment centers).\n   6. Absence of severe liver fibrosis or cirrhosis on Shear Wave (less than 6 months before enrolment) or of other advanced liver disorder.\n7. For all patients in reproductive age, agreement to use highly effective and adequate method of contraception for at least 12 months following DP administration (including both females of childbearing potential and males with partners of childbearing potential).\n8. Good adherence to transfusion and chelation programme, as indirect evidence of good adherence to treatment and follow-up evaluations for the current trial.\n9. Availability of an adequate and well documented transfusion history (at least previous 24 months) or availability to follow a regular transfusion regimen according to guidelines and provide a detailed transfusion record of the 24 months prior to the DP administration.\n\nExclusion Criteria:\n\n1. Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents).\n2. Severe, active viral, bacterial or fungal infection at eligibility evaluation.\n3. Current or prior malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes.\n4. Current or prior immunodeficiency disorder.\n5. History of uncontrolled seizures.\n6. Aspartate transaminase (AST), alanine transaminase (ALT) \\>3 × the upper limit of normal (ULN), or direct bilirubin value \\>2.5 × ULN.\n7. Baseline prothrombin time (International Normalized Ratio; INR) \\>1.5 × ULN.\n8. Other clinical conditions judged non compatible with the procedure and\u002For the treatment.\n9. Positivity for HIV (serology or RNA), and\u002For HbsAg and\u002For HBV DNA and\u002For HCV RNA (patients who have completed antiviral treatment for HCV may only be enrolled if they have achieved a sustained virologic response, defined as undetectable HCV RNA at least 12 weeks after completion of therapy) and\u002For evidence of active infection of Treponema Pallidum or Mycoplasma species.\n10. Positive history of significant previous thrombotic events. In case of a positive thrombophilic screening, patient's inclusion will be evaluated according to national guidelines.\n11. Active alcohol or substance abuse within 6 months of the study.\n12. Pregnancy or lactation.\n13. Previous allogeneic hematopoietic stem cell transplantation.\n14. Previous gene therapy treatment (gene addition or gene editing).\n15. For patients until the age of 14 years only: availability of an HLA-matched family donor.\n16. Patients with associated α-thalassemia and \\>1 alpha deletion, or alpha multiplications.\n17. Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.","ALL","3 Years","35 Years",{"count":20,"type":21},9,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an improved transduction protocol in 9 patients affected by transfusion dependent Beta-Thalassemia.\n\nFour study phases are foreseen:\n\n1. Screening phase, during which the conditions required by the clinical protocol for patients' inclusion\u002Fexclusion will be assessed after the signature of the informed consents\u002Fassents. Patients will be recruited from the two participating sites: IRCCS Ospedale San Raffaele (OSR), Department of Pediatric Immunohematology and adult Hematology (OSR Stem Cells Programme) (Milan) and IRCCS Ospedale Pediatrico Bambino Gesù (OPBG), Department of Haematology, Oncology and Gene and Cell Therapy (Rome).\n2. Baseline phase, carried from the end of the screening phase to the day before the start of the conditioning regimen.\n3. Treatment phase, from the first day of conditioning regimen until DP administration.\n4. Follow-up phase: from DP administration until 2 years follow-up.",[27],"Transfusion-dependent Beta-Thalassemia","NOT_YET_RECRUITING","2026-07-01",{"date":31,"type":32},"2026-07-02","ACTUAL",{"date":34,"type":21},"2026-07",{"date":36,"type":21},"2030-10",{"name":38,"class":39},"Fondazione Telethon","OTHER",2,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":22,"phases":53,"briefSummary":55,"conditions":56,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":4},"100631913","phase-3-a-study-to-investigate-the-efficacy-pharmacokinetics-and-safety-of-mitapivat-in-pediatric-participants-with-transfusion-dependent-alpha--or-beta-thalassemia---or--tdt-100631913","NCT07506863","A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)","A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia","ENERGIZEKids-T","Inclusion Criteria:\n\n* Written informed consent\u002Fassent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the duration of the study.\n* Aged 1 to \\\u003C18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent\u002Fassent.\n* Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)\u002Fβ-thalassemia, or α-thalassemia\u002Fhemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb high-performance liquid chromatography, and\u002For DNA analysis from the participant's medical record. If this information is not available from the participant's medical record, the test(s) can be performed by a local laboratory during the Screening Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.\n* Transfusion dependent, defined as 6 to 20 transfusion episodes (also referred to as \"transfusion events\") and a ≤6-week transfusion-free period during the 24-week period before randomization.\n* If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization.\n* Female participants who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent\u002Fassent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can include an acceptable barrier method.\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding.\n* Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).\n* Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.\n* Any conditions other than thalassemia expected to affect sexual maturation.\n* Currently receiving treatment with luspatercept; the last dose must have been administered ≥36 weeks before randomization.\n* Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥36 weeks before randomization.\n* History of malignancy (active or treated) ≤5 years before providing informed consent\u002Fassent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.\n* History of active and\u002For uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent\u002Fassent.\n* Hepatobiliary disorders, including but not limited to:\n\n  * Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.\n  * Clinically symptomatic cholelithiasis or cholecystitis (prior cholecystectomy is not exclusionary).\n  * History of drug-induced cholestatic hepatitis.\n  * Aspartate Aminotransferase (AST) \\>2.5\\*Upper Limit of Normal (ULN) (unless due to hemolysis and hepatic iron deposition) and Alanine Transaminase (ALT) \\>2.5\\*ULN (unless due to hepatic iron deposition).\n* Renal dysfunction as defined by an estimated glomerular filtration rate \\\u003C60 milliliters per minute (mL\u002Fmin)\u002F1.73-meter square (m\\^2).\n* Nonfasting triglycerides \\>215 milligrams per deciliter (mg\u002FdL) \\[5 millimole per liter (mmol\u002FL)\\].\n* Active infection requiring systemic antimicrobial therapy at the time of providing informed consent\u002Fassent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed while antimicrobial therapy is being administered, and the last dose of antimicrobial therapy must be administered ≥7 days before randomization.\n* Participants with known active hepatitis B or hepatitis C virus infection.\n* Participants with known human immunodeficiency virus (HIV) infection.\n* History of major surgery (including splenectomy) ≤ 6 months before providing informed consent\u002Fassent and\u002For a major surgical procedure planned during the study.\n* Current enrollment or past participation (within ≤12 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any other clinical study involving an investigational treatment or device.\n* Receiving strong Cytochrome3A4\u002F5 (CYP3A4\u002F5) inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer), or strong CYP3A4\u002F5 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization.\n* Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥12 weeks before randomization.\n* Known allergy, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry filmcoat \\[hypromellose, titanium dioxide, lactose monohydrate triacetin, and FD\\&C Blue #2\\]).\n* Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and\u002For could confound the interpretation of the study data. Also excluded are:\n\n  * Participants who are institutionalized by regulatory or court order.\n  * Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).","1 Year","17 Years",{"count":52,"type":21},54,[54],"PHASE3","The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.",[57,27],"Transfusion-dependent Alpha-Thalassemia","2026-06-19",{"date":60,"type":32},"2026-06-23",{"date":62,"type":21},"2026-09",{"date":64,"type":21},"2032-06",{"name":66,"class":67},"Agios Pharmaceuticals, Inc.","INDUSTRY",{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":4,"eligibilityCriteria":74,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":75,"targetDuration":4,"studyType":22,"phases":77,"briefSummary":79,"conditions":80,"keywords":81,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":97},"100532932","safety-and-efficacy-of-the-lentiviral-vector-in-gene-therapy-of-beta-thalassemia-patients-100532932","NCT06219239","Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients","Evaluation of the Safety and Efficacy of KL003 Gene Therapy in Patients With Transfusion-dependent β-thalassemia With No Conditioning Regimen","Inclusion Criteria:\n\n* Male or female age between 3-35 years\n* Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL\u002Fkg\u002Fyear of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years\n* Documented baseline, or pretransfusion, Hb level≤7 g\u002FdL\n* Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects\\\u003C16 years of age\n* Eligible to undergo auto-HSCT\n* Willing and able to follow the research procedures and conditions, with good compliance\n* Willing to receive at least the 2 years follow-up and maintain detailed medical records, including transfusion history\n* Subject and\u002For legal guardians voluntarily participated in this clinical trial and signed the informed consent form, and can complete all follow-ups in accordance with the protocol requirements\n\nExclusion Criteria:\n\n* Presence of clear contraindications for hematopoietic stem cell collection\n* Diagnosis of composite α thalassemia\n* A white blood cell (WBC) count \\\u003C3×10\\^9\u002FL, and\u002For platelet count \\\u003C100×10\\^9\u002FL not related to hypersplenism\n* Subjects with severe iron overload at the time of screening: severe iron overload of the liver showed by MRI, serum ferritin ≥ 5000 ng\u002FmL, or moderate to severe iron overload of the heart\n* Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder\n* Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match\n* Prior receipt of gene therapy or allo-HSCT\n* Subjects with any severe active fungal, bacterial, viral, tuberculosis or other infection, including active hepatitis B (defined as serum HBV-DNA ≥2000 IU\u002Fml), active hepatitis C virus, HCV) infection, human immunodeficiency virus (HIV) antibody-positive or active syphilis patients, etc.\n* Immediate family member (i.e. parent or siblings) with a known Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis)\n* Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study\n* History of major organ damage including:\n\n  1. Liver function test suggest AST or ALT levels \\>3× upper limit of normal (ULN);\n  2. Total serum bilirubin value \\>2.5×ULN;if combined with Gilbert syndrome, total bilirubin \\>3×ULN and direct bilirubin value \\>2.5×ULN;\n  3. History of bridging fibrosis, cirrhosis;\n  4. Left ventricular ejection fraction \\\u003C45%;\n  5. New York Heart Association (NYHA) class III or IV congestive heart failure;\n  6. Severe arrhythmia requiring medical treatment;\n  7. Uncontrolled hypertension or unstable angina pectoris;\n  8. Myocardial infarction or bypass or stent surgery within 12 months before drug administration;\n  9. Valvular disease with clinical significance;\n  10. Baseline calculated eGFR\\\u003C60mL\u002Fmin\u002F1.73m2;\n  11. Pulmonary function: FEV1\u002FFVC\\\u003C60% and\u002For diffusion capacity of carbon monoxide (DLco) \\\u003C60% of prediction;\n  12. Evidence of clinically significant pulmonary hypertension requiring medical intervention.\n* Uncorrectable coagulation dysfunction or history of severe bleeding disorder\n* Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician\n* Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.)\n* Participated in other clinical studies within 3 months prior to screening\n* Inoculated live vaccine within 6 weeks prior to screening\n* Pregnancy or breastfeeding women; Subjects or their sexual partners were unable to take medically recognized effective contraceptive measures during the 27-month study period\n* The subjects or their parents would not comply with the study procedures outlined in the protocol\n* The subjects received hydroxyurea or thalidomide or hypomethylating drugs within 3 months before hematopoietic stem cell collection\n* Patients considered to be ineligible for the study by the investigator for reasons other than the above",{"count":76,"type":21},3,[78],"NA","This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia.",[27],[82,83,84,85,86],"Beta-Thalassemia","Hematopoietic Stem-Cell Transplantation","KL003","Gene therapy","no conditioning regimen","RECRUITING","2025-07-31",{"date":90,"type":32},"2025-08-05",{"date":92,"type":32},"2024-01-04",{"date":94,"type":21},"2026-12-31",{"name":96,"class":39},"Institute of Hematology & Blood Diseases Hospital, China",1,{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":4,"eligibilityCriteria":104,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":105,"targetDuration":4,"studyType":22,"phases":107,"briefSummary":109,"conditions":110,"keywords":111,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":116,"lastUpdatePostDateStruct":117,"startDateStruct":119,"completionDateStruct":121,"leadSponsor":123,"locationsCount":40},"100537634","phase-1--thalassemia-treatment-with-kl003-cell-injection-100537634","NCT06280378","Β-Thalassemia Treatment with KL003 Cell Injection","A Phase I\u002FII Clinical Study Evaluating the Safety and Efficacy of KL003 Cell Injection in Transfusion-dependent Β-thalassemia","Inclusion Criteria:\n\n* Male or female age between 3-35 years;\n* Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL\u002Fkg\u002Fyear of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years;\n* Karnofsky performance status ≥70 for participants≥16 years of age; Lansky performance status of ≥70 for participants\\\u003C16 years of age;\n* Eligible to undergo auto-HSCT;\n* Willing and able to follow the research procedures and conditions, with good compliance;\n* Willing to receive at least the 2 years follow-up;\n* Participant and\u002For legal guardians voluntarily participated in this clinical trial and signed the informed consent form.\n\nExclusion Criteria:\n\n* Diagnosis of composite α thalassemia;\n* Prior receipt of gene therapy or allo-HSCT;\n* Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match;\n* Participants with severe iron overload at the time of screening;\n* Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody;\n* Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.);\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator;\n* Subjects positive with the following etiological tests: human immunodeficiency virus(HIV-1-2),human cytomegalovirus (HCMV-DNA),EB virus（EBV-DNA）,HBV (HBsAg\u002FHBV-DNA positive),HCV antibody (HCV-Ab), Human T-lymphotropic virus antibody (HTLV-Ab), Treponema pallidum antibody (TP-Ab);\n* Uncorrectable coagulation dysfunction or history of severe bleeding disorder;\n* History of major organ damage including:\n\n  1. Liver function test suggest AST or ALT levels \\>3× upper limit of normal（ULN）;\n  2. Total serum bilirubin value\\>2.5×ULN;if combined with Gilbert syndrome, total bilirubin\\>3×ULN and direct bilirubin value\\>2.5×ULN;\n  3. Left ventricular ejection fraction \\\u003C45%;\n  4. Baseline calculated eGFR\\\u003C60mL\u002Fmin\u002F1.73m2;\n  5. Pulmonary function:FEV1\u002FFVC\\\u003C60% and\u002For diffusion capacity of carbon monoxide (DLco) \\\u003C60% of prediction;",{"count":106,"type":21},41,[108,24],"PHASE1","This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.",[27],[112,113,114,115],"gene therapy","lentiviral vector","KL003 cell injection","transfusion-dependent β-thalassemia","2025-03-06",{"date":118,"type":32},"2025-03-10",{"date":120,"type":32},"2024-04-05",{"date":122,"type":21},"2027-05",{"name":124,"class":67},"Kanglin Biotechnology (Hangzhou) Co., Ltd.",{"id":126,"slug":127,"hasResults":11,"nctId":128,"briefTitle":129,"officialTitle":129,"acronym":4,"eligibilityCriteria":130,"healthyVolunteers":11,"sex":16,"minAge":131,"maxAge":18,"enrollmentInfo":132,"targetDuration":4,"studyType":22,"phases":133,"briefSummary":135,"conditions":136,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":138,"lastUpdatePostDateStruct":139,"startDateStruct":141,"completionDateStruct":143,"leadSponsor":145,"locationsCount":97},"100568203","early-phase-1-a-pilot-study-on-the-safety-and-efficacy-of-yolt-204-for-transfusion-dependent-beta-thalassemia-100568203","NCT06678165","A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia","Inclusion Criteria：\n\n1. Age between 18 to 35 years, gender not limited.\n2. The patient has fully understood this study and voluntarily signed an informed consent form.\n3. Clinically diagnosed as a patient with transfusion-dependent β-thalassemia, excluding the genotype: β0β0.\n4. Karnofsky Performance Status (KPS) score of at least 70.\n5. Obtain detailed medical records of red blood cell transfusions within 2 years prior to the patient signing the informed consent form, including the volume or units of transfusion and the levels of red blood cells and hemoglobin before and after transfusion.\n6. No severe hematopoietic dysfunction, with heart, lung, liver, and kidney functions essentially normal.\n7. Coagulation function: International Normalized Ratio (INR) and Activated Partial Thromboplastin Time (APTT) both ≤1.5×ULN (Upper Limit of Normal).\n8. Renal function: Creatinine ≤1.5×ULN, or when creatinine \\>1.5×ULN, the creatinine clearance is \\>50ml\u002Fmin (calculated according to the Cockcroft-Gault formula).\n9. Liver function: Alanine Aminotransferase (ALT) ≤3×ULN and Aspartate Aminotransferase (AST) ≤3×ULN; Direct Bilirubin ≤2.5×ULN.\n10. Cardiac function: Left Ventricular Ejection Fraction (LVEF) ≥50%.\n11. Good compliance, willing to adhere to visit schedules, trial plans, laboratory tests, and other trial steps.\n12. Willing to participate in long-term follow-up studies.\n\nExclusion Criteria:\n\n1. History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).\n2. Diagnosed with compound alpha-thalassemia.\n3. Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.\n4. White blood cell count (WBC) \\\u003C3×10\\^9\u002FL and\u002For platelet count \\\u003C100×10\\^9\u002FL not due to hypersplenism, as judged by the investigator.\n5. Uncorrected bleeding disorders.\n6. Received treatment with erythropoietin (EPO) within the three months prior to enrollment.\n7. Severe iron overload, with serum ferritin levels ≥5000 ng\u002Fml.\n8. Positive for hepatitis B surface antigen (HBsAg), antibodies to hepatitis C virus, antibodies to human immunodeficiency virus (HIV), or specific antibodies to Treponema pallidum (syphilis).\n9. History of hematopoietic stem cell transplantation, gene therapy, or gene editing therapy.\n10. Participation in another clinical study and use of investigational drugs within 3 months prior to starting the study drug.\n11. History or current presence of malignant tumors or myeloproliferative diseases or immunodeficiency diseases.\n12. Presence of severe mental illness that prevents cooperation with treatment; significant pulmonary arterial hypertension requiring medical intervention; recent malaria; a history of hematological tumors in immediate family members.\n13. Any past or current disease, treatment, or laboratory abnormality that may interfere with the study results, affect the patient's full participation in the study, or that the investigator deems unsuitable for participation in this clinical study.","18 Years",{"count":76,"type":21},[134],"EARLY_PHASE1","This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with transfusion-dependent β-thalassemia; to preliminarily evaluate the impact of a single -dose of YOLT-204 on the levels of fetal hemoglobin in the plasma",[137],"Transfusion Dependent Beta Thalassemia","2024-11-05",{"date":140,"type":32},"2024-11-07",{"date":142,"type":21},"2024-12-31",{"date":144,"type":21},"2026-06-01",{"name":146,"class":39},"First Affiliated Hospital of Guangxi Medical University",{"id":148,"slug":149,"hasResults":11,"nctId":150,"briefTitle":151,"officialTitle":151,"acronym":152,"eligibilityCriteria":153,"healthyVolunteers":154,"sex":16,"minAge":131,"maxAge":4,"enrollmentInfo":155,"targetDuration":4,"studyType":157,"phases":4,"briefSummary":158,"conditions":159,"keywords":165,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":175,"lastUpdatePostDateStruct":176,"startDateStruct":178,"completionDateStruct":180,"leadSponsor":182,"locationsCount":97},"100555454","plasticizer-exposure-and-its-consequences-on-health-100555454","NCT06512298","Plasticizer Exposure and Its Consequences on Health","PEACH","TDTM:\n\n* Inclusion: Adult, homozygous ß-Thalassemia Major, transfusion dependency.\n* Exclusion: Plastic implants, chronic infectious disease, pregnancy.\n\nThalassemia Intermedia\u002FMinor:\n\n* Inclusion: Adult, homozygous\u002Fheterozygous ß-Thalassemia Intermedia\u002FMinor\n* Exclusion: Plastic implants, chronic infectious disease, pregnancy, transfusion dependency\n\nHealthy adults:\n\n* Inclusion: Adult\n* Exclusion: Chronic disease, plastic implants, infectious disease, pregnancy, anemia, medication-, drug-, or alcohol-abuse\n\nGlioma patients:\n\n* Inclusion: Adult, high\u002Flow-grade glioma, tumor larger then 3cm, resection with access to the ventricular system\n* Exclusion: Large intraventricular hemorrhage, plastic implants, infectious disease\n\nICU patients:\n\n* Inclusion: Adult, brain hemorrhage, external CSF drain from ventricle\n* Exclusion: intraventricular hemorrhage, plastic implants, ECMO, hemodialysis, infectious disease\n\nPatients undergoing diagnostic lumbar-puncture:\n\n* Inclusion: Adult, diagnostic CSF sampling, outpatient setting\n* Exclusion: Ventricular hemorrhage, plastic implants, infectious disease",true,{"count":156,"type":21},120,"OBSERVATIONAL","Plasticizers are chemicals commonly found in many everyday items, from food packaging to medical equipment. Although they are pervasive in our daily lives, researchers still don't have a clear picture of their long-term effects on human health. Evidence suggests that these substances might disrupt various biological functions such as the immune system, the balance of gut bacteria, hormone regulation, and brain processes. While some studies have linked plasticizer exposure to health issues, definitive data from human studies are still lacking.\n\nThe PEACH study aims to bridge these knowledge gaps by investigating how plasticizers affect human health. The study focuses on understanding how these chemicals are absorbed, distributed, and accumulated in the body across different groups of patients. The investigators are particularly interested in how plasticizers influence gut microbiota and the functionality of immune cells, as well as their effects on neurotransmitters involved in brain function.\n\nA combination of patient data, systems biology, and laboratory models will be used to thoroughly assess the biological impacts of plasticizers. Advanced techniques such as mass spectrometry will aid in studying toxicokinetic properties, sequencing technologies will be used to examine immune effects, and radiouptake assays will be employed to explore interactions with neurotransmitter transport. This comprehensive methodology will provide new insights into the effects of both short-term and long-term exposure to plasticizers.\n\nThe PEACH study introduces innovative methods to the field, aiming to create a robust model for understanding how plasticizer compounds behave in the human body. It employs state-of-the-art techniques to assess the dynamics of these chemicals, marking a significant advancement in environmental health research.",[160,161,162,27,163,164],"Beta-thalassemia","Glioma","Healthy Controls","Intracranial Hemorrhages","Toxicity",[166,167,168,169,170,171,172,173,174],"Plasticizer","Transfusion","Systems Biology","Immunology","Microbiome","Neuropharmacology","Pharmacokinetics","Pharmacodynamics","Exposomics","2024-07-22",{"date":177,"type":32},"2024-07-24",{"date":179,"type":21},"2024-10-01",{"date":181,"type":21},"2027-09-30",{"name":183,"class":39},"Medical University of Vienna",{"id":185,"slug":186,"hasResults":11,"nctId":187,"briefTitle":188,"officialTitle":189,"acronym":4,"eligibilityCriteria":190,"healthyVolunteers":154,"sex":16,"minAge":17,"maxAge":191,"enrollmentInfo":192,"targetDuration":4,"studyType":157,"phases":4,"briefSummary":194,"conditions":195,"keywords":197,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":200,"lastUpdatePostDateStruct":201,"startDateStruct":203,"completionDateStruct":205,"leadSponsor":207,"locationsCount":97},"100515425","growth-and-development-related-outcomes-in-children-with-transfusion-dependent-beta-thalassemia-after-gene-therapy-100515425","NCT05991336","Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy","Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy","Inclusion Criteria:\n\nGene therapy group-inclusion\n\n* Male or female age of 3-14 years\n* TDT Children who have received gene therapy.\n* Subjects who are willing and able to provide written informed consent.\n\nSupportive therapy group-inclusion\n\n* Gender same as the matched case\n* Age similar to the matched case\n* Children with β-TDT\n* No history of gene therapy or allogeneic hematopoietic stem cell transplantation\n* Subjects who are willing and able to provide written informed consent.\n\nHealthy children group-inclusion\n\n* Gender same as the matched case\n* Age similar to the matched case\n* Subjects who are willing and able to provide written informed consent.\n\nExclusion Criteria:\n\n* Diagnosis of compound α-thalassemia\n* Uncontrolled systemic fungal, bacterial, or viral infection\n* History of malignant solid tumors, myeloproliferative or immunodeficiency diseases\n* Diagnosed with mental illness\n* Patients considered to be ineligible for the study by the investigator for reasons other than the above\n\nDiscontinuation of Study :\n\n* Subjects who are unwilling or unable to continue participating in the study (withdrawal of informed consent) may withdraw from the study\n* Subjects who received gene therapy or allogeneic hematopoietic stem cell transplantation during the study\n* The subject is seriously non-compliant with the study requirements, such as missing 2 consecutive visits\n* Subject lost to follow-up","14 Years",{"count":193,"type":21},100,"The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.",[27,196],"Gene Therapy",[27,112,198,199],"cohort study","case-control","2023-08-06",{"date":202,"type":32},"2023-08-14",{"date":204,"type":32},"2023-06-05",{"date":206,"type":21},"2029-12-31",{"name":96,"class":39},{"id":209,"slug":210,"hasResults":11,"nctId":211,"briefTitle":212,"officialTitle":213,"acronym":4,"eligibilityCriteria":214,"healthyVolunteers":11,"sex":16,"minAge":215,"maxAge":18,"enrollmentInfo":216,"targetDuration":4,"studyType":22,"phases":218,"briefSummary":219,"conditions":220,"keywords":222,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":226,"lastUpdatePostDateStruct":227,"startDateStruct":229,"completionDateStruct":231,"leadSponsor":233,"locationsCount":97},"100497843","early-phase-1-a-study-evaluating-the-safety-and-efficacy-of-lentired-drug-product-in-transfusion-dependent--thalassemia-tdt-100497843","NCT05762510","A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]","An Open Label Study Evaluating the Safety and Efficacy of Gene Therapy for Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a LentiRed Lentiviral Vector (GMCN-508B Drug Product, Also Called LentiRed)","Inclusion Criteria:\n\n1. The subject himself\u002Fherself or one legal guardian\u002Fagent of the subject is required to fully understand the study and voluntarily sign a written informed consent.\n2. Ages 5 to 35, no gender limitation.\n3. The clinical diagnosis of TDT includes β0\u002Fβ0, β+\u002Fβ0, βE\u002Fβ0 and β+\u002Fβ+ genotypes. TDT was defined as severe anemia in patients with thalassemia (Hb persistent \\\u003C70 g\u002FL), regular RBC transfusion and standard iron removal therapy to survive for life.\n4. Karnofsky Level of Performance (KPS) score ≥70 in adult subjects and Lansky Level of Performance (LPS) score ≥70 in children subjects.\n5. Subjects were determined to undergo autologous hematopoietic stem cell transplantation by the principle investigator.\n6. Subjects must have been treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.\n\nExclusion Criteria:\n\n1. Hepatitis B virus (HBV) : HbsAg or HbcAb positive, nucleic acid test positive; Hepatitis C virus (HCV) : HCAb positive, nucleic acid test positive; Positive for Human immunodeficiency virus (HIV) antibody or Treponema pallidum (TP) specific antibody; Tuberculosis: positive interferon gamma release test.\n2. A white blood cell (WBC) count \\\u003C3×10\\^9\u002FL and\u002For platelet count \\\u003C100×10\\^9\u002FL, splenectomy was performed before.\n3. Uncured bleeding abnormalities.\n4. Any previous or current malignancy, myeloproliferative disease, or immune deficiency disease.\n5. Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndromes, hereditary non-polyposis colorectal cancer syndromes and familial adenomatous polyposis).\n6. Previous hematopoietic stem cell transplantation (HSCT).\n7. Advanced liver disease, defined as: 1) Baseline alanine aminotransferase (ALT) or direct bilirubin ≥3 normal upper limit (ULN), or 2) Liver biopsy demonstrating cirrhosis, any evidence of bridging fibrosis, or acute hepatitis.\n8. Baseline estimated glomerular filtration rate (eGFR) \\\u003C 70 mL\u002Fmin \u002F1.73 m2, as determined using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation for ≥18 years of age, and Besides Schwartz Equation calculator \\\u003C 18 years of age.\n9. Uncontrolled seizure disorder.\n10. Diffusion capacity of Carbon monoxide dispersion (DLco) \\\u003C50% of predicted (corrected for hemoglobin and or alveolar ventilation, as clinically indicated ).\n11. A cardiac T2\\* \\\u003C20 ms by magnetic resonance imaging (MRI).\n12. Severe iron overload, which in the opinion of the physician is grounds for exclusion.\n13. Clinically significant pulmonary hypertension.\n14. Participation in another clinical study with an investigational drug within 30 days of screening.\n15. Failure to obtain appropriate informed consent.\n16. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or investigator.\n17. Contraindications to the conditioning regimen.\n18. Prior receipt of genetic stem cell therapy.\n19. Diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study.\n20. Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile subjects. Females of child-bearing potential are required to use effective contraception from the screening period until at least 6 months after drug product infusion. Male subjects are also required to use effective contraception (including condoms) from the screening period until at least 6 months after drug product infusion.\n21. Live vaccines were administered within 6 weeks prior to screening.\n22. Known history of hypersensitivity to the ingredients used in the trial.\n23. An assessment by the investigator that the subject would not comply with the study procedures outlined in the protocol.","5 Years",{"count":217,"type":21},5,[134],"This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.",[221],"Transfusion Dependent Beta-Thalassemia",[223,82,224,112,225],"LentiRed","Human Hematopoietic Stem Cell","CD34+","2023-05-05",{"date":228,"type":32},"2023-05-09",{"date":230,"type":32},"2023-02-22",{"date":232,"type":21},"2030-10-31",{"name":146,"class":39}]