[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"thalassemia-beta\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:thalassemia-beta":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,47,73,101,120],{"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":30,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":46},"100495839","phase-2-a-study-of-immune-suppression-treatment-for-people-with-sickle-cell-disease-or--thalassemia-who-are-going-to-receive-an-allogeneic-hematopoietic-cell-transplantation-hct-100495839",false,"NCT05736419","A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)","Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)","Inclusion Criteria:\n\n* Age ≥ 2 and ≤ 50 years\n* Suitable haploidentical donor.\n* Performance score ≥ 70% by Karnofsky Performance Scale or 0 to 1 by ECOG (age \\> 16 years), or Lansky Play-Performance Scale ≥ 70% (age ≤ 16 years).\n* Adequate major organ system function as demonstrated by:\n\n  * For patients ≥ 18 years of age:\n  * eGFR ≥ 50 mL\u002Fmin by Cockcroft-Gault formula Formula: ((140 - Age) x Weight (kg)) \u002F (72 x Serum Creatinine (mg\u002FdL) Female Adjustment: Multiply result by 0.85\n  * For patients \\\u003C 18 years of age:\n  * Serum creatinine clearance: glomerular filtration rate \\[GFR\\]) must be \\>50 mL\u002Fmin\u002F1.73 m2 as calculated by the Schwartz formula\n* Conjugated (direct) bilirubin less than 3x upper limit of normal.\n* ALT or AST ≤ 3 times institutional upper limit of normal.\n* Left ventricular ejection fraction ≥ 50%.\n* Diffusing capacity for carbon monoxide (DLCO) ≥ 50% predicted, corrected for hemoglobin. For children \\\u003C 7 years of age who are unable to perform PFT, oxygen saturation \\> 92% on room air by pulse oximetry.\n* For SCD patients: HbSS, HbSC, HbS\u002Fβ° with one or more of the following complications:\n\n  * Acute chest syndrome: 2 or more episodes in the 2 years preceding enrollment\n  * Vaso-occlusive episodes: 3 or more episodes in the 2 years preceding enrollment\n  * Recurrent priapism: 2 or more episodes in the 2 years preceding enrollment\n  * History of osteomyelitis or osteonecrosis\n  * Cerebrovascular disease:\n* Imaging evidence of prior overt or silent stroke\n* History of a neurologic event resulting in focal neurologic deficits lasting \\> 24 hours\n* Abnormal transcranial Doppler: Timed average maximum mean velocity ≥ 200 cm\u002Fsec in terminal portion of the carotid or proximal portion of the middle cerebral artery or \\> 185 cm\u002Fsec plus evidence of intracranial vasculopathy if imaging TCD is used\n\n  * Pulmonary hypertension: Confirmed by right heart catheterization with mean pulmonary arterial pressure ≥ 25 mmHg or mean pulmonary vascular resistance \\> 2 Wood units\n  * Red blood cell alloimmunization (\\> 3 alloantibodies)\n* For thalassemia patients: Any genotype, with all of the following:\n\n  * Onset of red blood cell transfusion dependence during the first 3 years of life\n  * RBC transfusion history \\> 225 mL\u002Fkg\u002Fyear or \\> 15 lifetime RBC transfusions\n  * Pre-transfusion hemoglobin ≤ 7 g\u002FdL\n  * Hepatosplenomegaly\n* Patient or the patient's legal representative, parent(s) or guardian should be able to provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.\n* For sexually active men and women of childbearing potential, must agree to use a form of contraception considered effective and medically acceptable by the Investigator.\n\nExclusion Criteria:\n\n* Prior myeloablative allogeneic HCT.\n* Overt stroke or CNS instrumentation (e.g. for Moyamoya disease) within 6 months of enrollment.\n* Liver cirrhosis. Mild fibrosis will be permitted, i.e. fine reticulin or grade 1 of 4, with bridging fibrosis.\n* Hepatic iron content ≥ 3 mg Fe\u002Fg liver dry weight, if applicable\n* Active hepatitis B or C.\n* Other uncontrolled infections.\n* Other malignancy\u002Fcancer diagnosis unless in remission after definitive therapy for a minimum of 2 years. Exceptions: Ductal carcinoma in situ, basal cell carcinoma, cervical intraepithelial neoplasia.\n* Positive pregnancy test in a woman with child-bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization.\n* Inability to comply with medical therapy or follow-up.\n* Known history of allergic reactions to any constituents of the stem cell product, including a known history of allergic reactions to DMSO.","ALL","2 Years","50 Years",{"count":20,"type":21},24,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","Hematopoietic Cell Transplantation\u002FHCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.",[27,28,29],"Sickle Cell Disease","Thalassemia, Beta","Thalassemia",[31,27,32,29,33,34],"allogeneic hematopoietic cell transplantation","Beta Thalassemia","23-009","Memorial Sloan Kettering Cancer Center","RECRUITING","2026-06-02",{"date":38,"type":39},"2026-06-03","ACTUAL",{"date":41,"type":39},"2023-02-09",{"date":43,"type":21},"2027-02-09",{"name":34,"class":45},"OTHER",6,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":59,"conditions":60,"keywords":4,"overallStatus":61,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":72},"100539037","long-term-follow-up-study-of-brl-101-for-tdt-100539037","NCT06298630","Long-term Follow-up Study of BRL-101 for TDT","Long-term Follow-up Study of BRL-101 in the Treatment of Transfusion-dependent β-thalassemia","Inclusion Criteria:\n\n1. Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)\u002Flegal guardian(s)\n2. Treated with BRL-101 for therapy of transfusion-dependent β-thalassemia.\n\nExclusion Criteria:\n\n\\- There are no exclusion criteria for this study","3 Years","35 Years",{"count":57,"type":21},45,"OBSERVATIONAL","Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.",[28],"NOT_YET_RECRUITING","2024-03-21",{"date":64,"type":39},"2024-03-22",{"date":66,"type":21},"2024-12-08",{"date":68,"type":21},"2038-10-15",{"name":70,"class":71},"Bioray Laboratories","INDUSTRY",7,{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":77,"acronym":78,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":80,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":82,"conditions":83,"keywords":86,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":100},"100500658","study-of-the-role-of-genetic-modifiers-in-hemoglobinopathies-100500658","NCT05799118","Study of the Role of Genetic Modifiers in Hemoglobinopathies","INHERENT","Inclusion Criteria:\n\n* Clinical diagnosis of an inherited hemoglobinopathy, including sickle cell disease (SCD), β-thalassemia, and α-thalassemia; all genotypes will be considered.\n* Age ≥ 2 years old at the time of the collection of the phenotypic data.\n* There will be no limits on study participants in terms of gender, ethnicity, morbidities.\n\nExclusion Criteria:\n\n* Patients treated with stem cell transplantation or genetic therapy.\n* Age \\\u003C 2 years old at the time of the collection of the phenotypic data.\n* Patient or legal representative for minors unwilling or unable to give consent.",{"count":81,"type":21},30000,"This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).",[27,28,84,85],"Thalassemia Alpha","Hemoglobinopathies",[87,88,89,90],"GWAS","thalassemia","sickle cell disease","genetic modifiers","2024-03-19",{"date":93,"type":39},"2024-03-20",{"date":95,"type":39},"2022-10-01",{"date":97,"type":21},"2027-09-30",{"name":99,"class":45},"Cyprus Institute of Neurology and Genetics",26,{"id":102,"slug":103,"hasResults":11,"nctId":104,"briefTitle":105,"officialTitle":106,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":107,"maxAge":55,"enrollmentInfo":108,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":109,"conditions":110,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":119},"100540260","long-term-follow-up-study-of-bhc001-for-tdt-100540260","NCT06314529","Long-term Follow-up Study of BHC001 for TDT","Long-term Follow-up Study of BHC001 in the Treatment of Transfusion-dependent β-thalassemia","5 Years",{"count":46,"type":21},"Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.",[28],"2024-03-11",{"date":113,"type":39},"2024-03-18",{"date":115,"type":39},"2022-12-14",{"date":117,"type":21},"2037-05-10",{"name":70,"class":71},2,{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":125,"acronym":4,"eligibilityCriteria":126,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":55,"enrollmentInfo":127,"targetDuration":4,"studyType":22,"phases":128,"briefSummary":130,"conditions":131,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":142},"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.",{"count":119,"type":21},[129],"NA","This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.",[28,132],"Thalassemia Major","2023-10-16",{"date":135,"type":39},"2023-10-17",{"date":137,"type":39},"2023-08-31",{"date":139,"type":21},"2026-06-30",{"name":141,"class":45},"Institute of Hematology & Blood Diseases Hospital, China",1]