[{"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":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,48,74,106],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":16,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":29,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100587709","growth-evaluation-health-promotion-and-clinical-management-in-children-and-adolescents-with-thalassemia-100587709",false,"NCT06931912","Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia","Inclusion Criteria:\n\n* Subjects diagnosed with transfusion-dependent thalassemia (TDT)\n* Male or female age ≤18 years\n* Subjects who are willing and able to provide written informed consent\n\nExclusion Criteria:\n\n* Not applicable","ALL","18 Years",{"count":18,"type":19},369,"ESTIMATED","INTERVENTIONAL",[22],"NA","There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a substantial economic burden on their families and a serious social strain on the labor force. Investigating the growth and development of these children and adolescents, and establishing targeted intervention plans, holds significant social value for public health practice.\n\n1. To screen and identify pediatric patients with growth problems by conducting growth and development assessments in high-incidence areas of China, including physical development, endocrine function, nutritional status, brain function and lifestyle behaviors.\n2. Implement the MENBS clinical interventions for pediatric patients with growth problems, concentrating on the following areas:\n\n   * Monitor: Continuously monitor health-related indicators through regular follow-up.\n   * Education: Provide health education to improve the cognition of patients and their families.\n   * Nutrition: Assess patients' nutritional risks and develop personalized diet plans.\n   * Behavior: Recommend appropriate exercise plans to promote physical development.\n   * Support: Conduct home visits, offer free clinics and establish a support network.\n3. Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions.\n4. Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.",[25,26,27,28],"Thalassemia Intermedia","Thalassemia Major","Growth Delay","Puberty, Delayed",[30,31,32,33,34],"Thalassemia","Children and adolescents","Growth development","Health Promotion","Clinical Management","RECRUITING","2026-02-09",{"date":38,"type":39},"2026-02-11","ACTUAL",{"date":41,"type":39},"2024-12-01",{"date":43,"type":19},"2026-11-30",{"name":45,"class":46},"Institute of Hematology & Blood Diseases Hospital, China","OTHER",1,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":15,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":20,"phases":58,"briefSummary":59,"conditions":60,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":47},"100601608","effect-of-incentive-respiratory-training-on-pulmonary-functions-and-functional-capacity-in-children-with-b-thalassemia-major-100601608","NCT07112703","Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major","Inclusion Criteria:\n\n1. Diagnosed as thalassemia \\& subjected to blood transfusion\n2. Their age range from 6 to 10 years old\n3. Both sexes, male and female, will participate.\n4. Subjects are able to follow simple verbal commands or instructions included in the procedures.\n\nExclusion Criteria:\n\n1. Children with mental retardation or any other disability .\n2. Children with visual or hearing defects.\n3. Children with cardiovascular, chest pulmonary, and heart diseases.\n4. Children with any postoperative cardiovascular, chest pulmonary, and heart conditions.","6 Years","10 Years",{"count":57,"type":19},50,[22],"The study was done to:\n\n1. Investigate the effect of respiratory training on functional lung capacity and\n2. To detect the effect of respiratory training on pulmonary functions in children with β-thalassemia major.\n3. To detect the level of oxygen saturation and heart rate during and after blood transfusion in children with β-thalassemia major.",[61,62,63,64,26],"Incentive Spirometer","Pulmonary Function","Functional Capacity","Child, Only","2025-08-02",{"date":67,"type":39},"2025-08-08",{"date":69,"type":39},"2025-04-07",{"date":71,"type":19},"2025-10-08",{"name":73,"class":46},"Rawan Mohammed Khairy Mostafa Elsawy",{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":78,"acronym":79,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":15,"minAge":81,"maxAge":82,"enrollmentInfo":83,"targetDuration":4,"studyType":20,"phases":85,"briefSummary":87,"conditions":88,"keywords":91,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":96,"lastUpdatePostDateStruct":97,"startDateStruct":99,"completionDateStruct":101,"leadSponsor":103,"locationsCount":47},"100553787","phase-2-unraveling-the-impact-of-thalidomide-at-diverse-doses-in-transfusion-dependent-beta-thalassemia-100553787","NCT06490627","Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia","BTM","Inclusion Criteria:\n\n* Know case of beta thalassemia major\u002F intermediate ( transfusion dependent)\n* willing to give informed consent\n\nExclusion Criteria:\n\n* Patients with comorbidities such as liver dysfunction\n* Married patients\n* Lactating mother\n* H\u002FO thrombosis and fits","8 Years","35 Years",{"count":84,"type":19},54,[86],"PHASE2","The project \"Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia\" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects.\n\nData will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.",[89,26,90],"Fetal Hemoglobin","Genetic Disease",[92,93,94,95],"thalidomide","safety and efficacy","thalassemia","fetal hemoglobin inducer","2025-05-26",{"date":98,"type":39},"2025-05-30",{"date":100,"type":39},"2024-04-22",{"date":102,"type":19},"2026-04-22",{"name":104,"class":105},"National Institute of Blood and Marrow Transplant (NIBMT), Pakistan","OTHER_GOV",{"id":107,"slug":108,"hasResults":11,"nctId":109,"briefTitle":110,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":11,"sex":15,"minAge":113,"maxAge":82,"enrollmentInfo":114,"targetDuration":4,"studyType":20,"phases":116,"briefSummary":117,"conditions":118,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":120,"lastUpdatePostDateStruct":121,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":47},"100519290","safety-and-efficacy-evaluation-of-autologous-crispr-cas12b-edited-hematopoietic-stem-cells-100519290","NCT06041620","Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells","A Study to Evaluate the Efficacy and Safety of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells in Transfusion-dependent β Thalassemia Patients","Inclusion Criteria:\n\n* Age 3-35 years old (inclusive), male or female;\n* The subject and\u002For his\u002Fher legally recognized representative\u002Fparent\u002Fguardian fully understands the study and all information related to the study and has signed the informed consent form;\n* Clinical diagnosis of transfusion-dependent β-thalassemia (TDT) with a blood transfusion record within 2 years (inclusive) prior to screening showing a history of ≥ 10 units (U)\u002Fkg\u002Fyear (or ≥ 100 mL\u002Fkg\u002Fyear) or ≥ 8 times\u002Fyear of suspended RBC transfusions in at least 1 consecutive 12-month period;\n* Karnofsky score (for subjects aged ≥ 16 years) or Lansky score (for subjects aged \\\u003C 16 years) of ≥ 80;\n* Subjects in stable disease state who are eligible for hematopoietic stem cell transplantation as per investigator's judgment;\n* Access to diagnosis and treatment records issued by medical professional institutions within 2 years prior to screening, including the records of blood transfusions, hematology, serum chemistry, and other examinations;\n* Willing and able to comply with study procedures, with good compliance, and willing to receive and complete the follow-up study with a duration of at least 2 years;\n* Subjects of childbearing potential (including female subjects of childbearing potential and male subjects whose partners are of childbearing potential) must use effective contraception within 12 months of treatment.\n\nExclusion Criteria:\n\n* Diagnosis of associated α-thalassemia: \\> 1 alpha chain deletion or alpha gene functional defect;\n* Have available HLA-fully matched donors and acceptable for allogeneic hematopoietic stem cell transplantation;\n* Irregular antibody or platelet antibody positive;\n* Prior allogeneic bone marrow transplantation or gene therapy;\n* Subjects with clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator at screening, including but not limited to those with positive etiology of human immunodeficiency virus (HIV-1\u002F2), human cytomegalovirus (HCMV-DNA), Epstein-Barr virus (EBV-DNA), or Treponema pallidum antibody (TP-Ab), or with previous hepatitis B or C infection;\n* Subjects with an injury of major organs\n* Contraindications for hematopoietic stem cell collection and poor collection efficiency judged by the investigator;\n* Contraindications to the clinical investigational product and its excipients, G-CSF (hematopoietic stem cell mobilization), plerixafor (hematopoietic stem cell mobilization), busulfan (myeloablation), and other drugs;\n* Participation within 3 months prior to screening or current participation in another interventional clinical study;\n* History or family history of malignancy or myeloproliferative disorder;\n* History of uncontrollable epilepsy, mental disorder, or other psychiatric disorders;\n* Abuse of psychoactive substance, drug, or alcohol within 6 months prior to enrollment;\n* Pregnant or breastfeeding females;\n* Other diseases or reasons that interfere with study procedures;\n* Any other conditions that the investigator deems unsuitable for the subject's participation in the study.","3 Years",{"count":115,"type":19},2,[22],"This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.",[119,26],"Thalassemia, Beta","2023-10-16",{"date":122,"type":39},"2023-10-17",{"date":124,"type":39},"2023-08-31",{"date":126,"type":19},"2026-06-30",{"name":45,"class":46}]