[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemoglobinopathy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemoglobinopathy":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,45,70],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100540173","early-phase-1-determination-of-red-cell-survival-in-sickle-cell-disease-and-other-hemoglobinopathies-using-biotin-labeling-100540173",false,"NCT06313398","Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling","* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Provision of signed and dated informed consent form\n2. Stated willingness to comply with all study procedures and availability for the duration of the study\n3. Male or female, aged 18 years or greater with confirmed diagnosis of SCD (all genotypes), thalassemia (beta and\u002For alpha), or other inherited hemoglobinopathy not otherwise specified.\n4. Be at steady state for their underlying disease (e.g. SCD or thalassemia) or post-bone marrow transplantation status, as evidenced by medical history.\n5. Ability to have blood samples drawn.\n6. For female participants of child-bearing potential, agree to use birth control during study participation. Female subjects of child-bearing potential must agree to use a medically acceptable method of birth control such as an oral contraceptive, intrauterine device, barrier and spermicide, or contraceptive implant\u002Finjection from start of screening through 4 months after infusion.\n7. Agreement to adhere to Lifestyle Considerations throughout study duration\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n1. Consumption of biotin supplements or raw eggs within the last 30 days.\n2. Blood loss within the previous 8 weeks (\\>540 mL).\n3. Red cell transfusion for their underlying SCD and\u002For thalassemia within the last 3 months.\n\n   a. Participants may be eligible after three months following their last transfusion.\n4. Patients on hemodialysis, due to possibility of early removal of biotinylated RBCs.\n5. Pregnancy, lactation or absence of adequate contraception for fertile female subjects.\n6. Pediatric subjects will not participate in this study.\n7. Known allergic reactions to biotin, due to risk of possible life-threatening allergic reaction.\n8. Current diagnosis of malignancy (liquid and\u002For solid).","ALL","18 Years","100 Years",{"count":19,"type":20},100,"ESTIMATED","INTERVENTIONAL",[23],"EARLY_PHASE1","Background:\n\nSickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells. SCD and other blood disorders can be managed with drugs or cured with a bone marrow transplant. Researchers want to know how long RBCs survive in people with SCD and other blood disorders before and after treatment compared to those who had a bone marrow transplant.\n\nObjective:\n\nTo learn how long RBCs survive in the body in people with SCD and other blood disorders compared to those whose disease was cured with a bone marrow transplant.\n\nEligibility:\n\nPeople aged 18 years or older with SCD or another inherited blood disorder. People whose SCD or blood disorder was cured with a bone marrow transplant are also needed.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood and urine tests.\n\nParticipants will have about 7 tablespoons of blood drawn. In the lab, this blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs. This process is called \"biotin labeling of RBCs.\" The next day, the participant s own biotin-labeled RBCs will be returned to their bloodstream.\n\nParticipants will return regularly to have smaller blood samples (about 2 teaspoons) drawn. These samples will be tested to detect the percentage of cells that have biotin labels. These visits may be every 2 weeks, 4 weeks, or some other interval. Participants will continue this schedule for up to 20 weeks or until biotin can no longer be detected....",[26,27,28],"Sickle Cell Disease","Thalassemia","Hemoglobinopathy",[30,28,27,26,31],"Red Cell","Biotin","RECRUITING","2026-02-21",{"date":35,"type":36},"2026-02-24","ACTUAL",{"date":38,"type":36},"2024-05-17",{"date":40,"type":20},"2029-06-15",{"name":42,"class":43},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":15,"minAge":52,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":56,"phases":4,"briefSummary":57,"conditions":58,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":59,"lastUpdatePostDateStruct":60,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":69},"100621670","a-long-term-follow-up-study-in-patients-who-received-beam-101-100621670","NCT07373639","A Long-term Follow-up Study in Patients Who Received BEAM-101","A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells","Inclusion Criteria:\n\n1. Have received BEAM-101 in Study BTX-AUT-001 and are in the process of completing that study's end-of-study (EOS) visit.\n2. Provide signed, written informed consent according to local institutional review board (IRB)\u002Findependent ethics committee (IEC) and institutional requirements.\n\nExclusion Criteria:\n\n* There are no exclusion criteria for this study.","14 Years","37 Years",{"count":55,"type":20},50,"OBSERVATIONAL","This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed for 13 years (a total of 15 years after receiving BEAM-101).",[28,26],"2026-01-23",{"date":61,"type":36},"2026-01-28",{"date":63,"type":36},"2025-12-15",{"date":65,"type":20},"2043-02-12",{"name":67,"class":68},"Beam Therapeutics Inc.","INDUSTRY",17,{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":4,"eligibilityCriteria":76,"healthyVolunteers":77,"sex":15,"minAge":16,"maxAge":78,"enrollmentInfo":79,"targetDuration":4,"studyType":21,"phases":81,"briefSummary":83,"conditions":84,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":85,"lastUpdatePostDateStruct":86,"startDateStruct":88,"completionDateStruct":90,"leadSponsor":92,"locationsCount":44},"100599651","phase-1-a-phase-i-study-of-snh-119014-in-healthy-volunteers-100599651","NCT07087262","A Phase I Study of SNH-119014 in Healthy Volunteers","A Study to Evaluate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic of SNH-119014 in Healthy Adult Volunteers","Inclusion Criteria:\n\n* Healthy male or female subjects;\n* 18-45 years of age, inclusive;\n* Male subjects with body weight ≥50 kg, female subjects with body weight ≥45 kg; BMI ≥18.5 and ≤28.0 kg\u002Fm2.\n* Must provide written informed consent.\n\nExclusion Criteria:\n\n* History of or current clinically significant circulatory system, endocrine system, nervous system, digestive system, respiratory system, urinary system, ophthalmology, hematology, immunology, psychiatry disorder, as judged by the investigator；\n* Subjects who have undergone major surgery 6 months prior to screening or who plan to undergo surgery during the study period;\n* Clinically significant abnormal physical examination, vital signs, chest X-ray, laboratory tests during the screening period as judged by the investigator;\n* Subjects with QTcF\\>450 ms, or with other ECG clinically significant abnormalities during the screening period as judged by the investigator;\n* Subject has a positive test result for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCVAb), treponema pallidum antibody, or human immunodeficiency virus (HIV) antibody at screening;\n* Subjects who have a known chronic liver disease, or who have clinically significant abnormal liver function test results during the screening period;\n* History of urinary system diseases.\n* History of dysphagia or any gastrointestinal disorder affecting drug absorption.\n* History of malignancy.\n* History of severe allergies or allergic to the study drug or any of its components. Serious adverse reaction or hypersensitivity to any drug or the formulation excipients.\n* Failure to follow a consistent diet.\n* Over 5 cups (200 ml per cup) of tea, coffee, or caffeinated beverages were consumed daily during the 3 months prior to screening.\n* Consumption of a specific diet (e.g. grapefruit and grapefruit-containing products, chocolate, any food containing caffeine), or vigorous exercise, or other factors affecting drug absorption, distribution, metabolism, excretion within 48 hours before the dose of study drug.\n* Subjects who have taken any drugs that alter the activity of liver enzymes and\u002For transporters within 28 days prior to screening.\n* Females who are pregnant or lactating and subjects who are unable to use one or more nonpharmacological contraceptives.\n* Subjects who have taken any prescribed or over-the-counter drug or herbal remedies or nutraceutical product within 14 days before the dose of study drug.\n* Subjects who smoked more than 5 cigarettes per day during the 3 months prior to screening, or who cannot accept a smoking ban throughout the study.\n* Alcohol intake \\>14 units per week within 6 months prior to screening (1 unit is equal to 14g of alcohol such as 360mL of beer, 45mL of 40% spirit, 150mL of wine, or a confirmed positive alcohol breath test, or alcohol prohibition is not acceptable throughout the study.\n* History of drug abuse in the past 1 year prior to screening, or a confirmed positive drugs of abuse test result before randomization.\n* Subjects who plan to conceive or donate sperm or eggs during the study or within 3 months after completion of study.\n* Subjects has received study drug in another clinical study within 3 months before the dose of study drug.\n* Subjects who have donated blood or loss ≥400 mL of blood within 3 months before the dose of study drug, or who plan to donate blood or blood components during the study or within 3 months after completion of study.\n* Subjects who have received a live attenuated vaccine within 28 days before the dose of study drug.\n* Subjects who cannot tolerate venipuncture, or those with a history of needle fainting or blood fainting.\n* In the opinion of the investigator, the subject is not suitable for entry into the study.",true,"45 Years",{"count":80,"type":20},96,[82],"PHASE1","SNH-119014 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias. This initial study will characterize the safety, tolerability and the pharmacokinetics\u002Fpharmacodynamics (PK\u002FPD) of a single ascending dose and multiple ascending doses of SNH-119014 in the context of Phase 1 studies in healthy volunteers. The effects of food on the absorption of SNH-119014 will also be evaluated in healthy volunteers.",[28],"2025-07-23",{"date":87,"type":36},"2025-07-25",{"date":89,"type":36},"2025-06-27",{"date":91,"type":20},"2026-03-28",{"name":93,"class":68},"ScinnoHub Pharmaceutical Co., Ltd."]