[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"sickle-cell-anemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:sickle-cell-anemia":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,11,0,[8,40,68,102,133,156,181,215,241,278,301],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":25,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100369718","hydroxyurea-exposure-limiting-pregnancy-and-follow-up-lactation-100369718",false,"NCT04093986","Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation","HELPFUL","Inclusion Criteria:\n\n* Medical records or data available from previous clinical care prior to June 20, 2019 of pregnant females with SCD, including women who miscarried, had a still birth, or completed labor at any gestational stage, with any hydroxyurea exposure during either pregnancy and\u002For while breastfeeding.\n* Medical records or data available from previous clinical care prior to June 20, 2019 about pregnancy and breastfeeding outcomes, both for babies with hydroxyurea exposure and other babies by these same women.\n\nExclusion Criteria:\n\n* Unavailable medical records or lack of information about hydroxyurea exposure.","ALL",{"count":18,"type":19},200,"ESTIMATED","OBSERVATIONAL","The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.",[23,24],"Sickle Cell Disease","Sickle Cell Anemia",[26],"Hydroxyurea","RECRUITING","2026-06-16",{"date":30,"type":31},"2026-06-18","ACTUAL",{"date":33,"type":31},"2019-12-22",{"date":35,"type":19},"2030-12-31",{"name":37,"class":38},"Children's Hospital Medical Center, Cincinnati","OTHER",1,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":47,"sex":16,"minAge":48,"maxAge":49,"enrollmentInfo":50,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":52,"conditions":53,"keywords":55,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":4,"leadSponsor":65,"locationsCount":39},"100098389","blood-sampling-for-research-related-to-sickle-cell-disease-100098389","NCT00542230","Blood Sampling for Research Related to Sickle Cell Disease","High Sensitivity Screening of Compound Libraries to Discover a Drug for the Treatment of Sickle Cell Disease","* INCLUSION CRITERIA:\n* Patients with sickle cell trait\n* Patients with known hemoglobinopathies involving one or two genes for sickle hemoglobin\n* Healthy volunteers for control experiments\n* Age range: adults greater than or equal to 18 years of age\n\nEXCLUSION CRITERIA:\n\n* Subjects who are unable to comprehend the investigational nature of the laboratory research are ineligible to enroll in this protocol.\n* As a safety precaution in handling the blood samples, patients with HIV, Hepatitis B or Hepatitis C will be excluded from the study. HIV, Hepatitis B or Hepatitits C testing will not be done under this study. Participants must be co-enrolled under another NIH protocol where the screening evaluation has been performed.",true,"18 Years","100 Years",{"count":51,"type":19},250,"This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body. The purpose of the study is to collect a variety of blood samples that may then be used to investigate advances and potential new drug treatments for sickle cell disease.\n\nVolunteers must be at least 18 years of old. Samples will be taken both from healthy volunteers and from volunteers who have unique red blood cell features that are related to sickle cell disease. Candidates will be screened with a medical history.\n\nDuring the study, participants will undergo a one- to two-hour outpatient procedure at the National Institutes of Health Clinical Center. Once researchers have explained the study and obtained the participant s consent, participants will donate 8 cc (approximately 2 teaspoons) of blood.\n\nBecause repeat testing helps researchers validate study findings, participants who have the unique red blood cell features mentioned above may also be asked if they are willing to return and donate another 2 cc to 8 cc of blood for additional studies. The amount of blood drawn will not exceed 50 ml with any eight-week period for adults or 7 cc within any six-week period for children.",[54,23,24],"Sickle Cell Trait",[56,57,54,58,23,59],"Erythrocytes","Drug Screen","Sickle Hemoglobin","Natural History","2026-06-05",{"date":62,"type":31},"2026-06-08",{"date":64,"type":31},"2007-11-07",{"name":66,"class":67},"National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)","NIH",{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":4,"eligibilityCriteria":74,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":75,"enrollmentInfo":76,"targetDuration":4,"studyType":78,"phases":79,"briefSummary":81,"conditions":82,"keywords":86,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":39},"100517368","phase-2-alendronate-for-osteonecrosis-in-adults-with-sickle-cell-disease-100517368","NCT06016634","Alendronate for Osteonecrosis in Adults With Sickle Cell Disease","A Feasibility Study of Alendronate as Treatment for Osteonecrosis in Adults With Sickle Cell Disease","Inclusion Criteria:\n\n* Age 18-80 years with SCD (any genotype, confirmed by hemoglobin electrophoresis or high performance liquid chromatography)\n* Ability to provide written informed consent\n* Ability to lay on a dual-energy X-ray absorptiometry (DXA) scanner\n* Negative urine pregnancy test for anyone of childbearing potential at study entry\n\nExclusion Criteria:\n\n* Pregnant women\n* Adults unable to consent\n* Individuals who are not yet adults (infants, children, teenagers)\n* Prisoners\n* Hospitalizations (for any cause) within 2 weeks of study entry","80 Years",{"count":77,"type":19},30,"INTERVENTIONAL",[80],"PHASE2","A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis",[23,24,83,84,85],"Osteonecrosis","Ischemic Necrosis","Avascular Necrosis",[87,88,89,90,91,92],"sickle cell disease","sickle cell anemia","osteonecrosis of the femoral head","hip osteonecrosis","ischemic necrosis of the femur","avascular necrosis of the femur","2026-04-08",{"date":95,"type":31},"2026-04-13",{"date":97,"type":31},"2026-03-09",{"date":99,"type":19},"2027-12-31",{"name":101,"class":38},"University of California, Davis",{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":4,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":16,"minAge":109,"maxAge":110,"enrollmentInfo":111,"targetDuration":4,"studyType":78,"phases":113,"briefSummary":115,"conditions":116,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":132},"100475948","phase-3-evaluation-of-efficacy-and-safety-of-a-single-dose-of-ctx001-in-participants-with-transfusion-dependent--thalassemia-and-severe-sickle-cell-disease-100475948","NCT05477563","Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease","A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease","Key Inclusion Criteria:\n\n* Participants with TDT and SCD:\n* Eligible for autologous stem cell transplant as per investigator's judgment.\n* Participants with TDT:\n* Diagnosis of TDT as defined by:\n* Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia\u002Fhemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning\n* History of at least 100 milliliter (mL)\u002Fkilograms (kg)\u002Fyear or 10 units\u002Fyear of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening\n* Participants with SCD:\n* Diagnosis of severe SCD as defined by:\n* Documented SCD genotypes\n* History of at least two severe VOCs events per year for the previous two years prior to enrollment\n\nKey Exclusion Criteria:\n\n* Participants with TDT and SCD:\n* A willing and healthy 10\u002F10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement\n* Prior hematopoietic stem cell transplant (HSCT)\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator\n* Participants with TDT:\n* Participants with associated α-thalassemia and \\>1 alpha deletion, or alpha multiplications\n* Participants with sickle cell β-thalassemia variant\n* Participants with SCD:\n* History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.","12 Years","35 Years",{"count":112,"type":19},26,[114],"PHASE3","This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.",[117,118,119,120,121,23,24],"Beta-Thalassemia","Thalassemia","Hematologic Diseases","Genetic Diseases, Inborn","Hemoglobinopathies","2026-03-18",{"date":124,"type":31},"2026-03-23",{"date":126,"type":31},"2022-08-02",{"date":128,"type":19},"2027-06-09",{"name":130,"class":131},"Vertex Pharmaceuticals Incorporated","INDUSTRY",6,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":139,"eligibilityCriteria":140,"healthyVolunteers":11,"sex":16,"minAge":141,"maxAge":4,"enrollmentInfo":142,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":143,"conditions":144,"keywords":145,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":150,"completionDateStruct":152,"leadSponsor":154,"locationsCount":155},"100601920","serial-assessment-of-fertility-experiences-100601920","NCT07116772","Serial Assessment of Fertility Experiences","A Prospective Longitudinal Study of Growth, Development, Fertility and Reproductive Outcomes Among Adolescents and Young Adults With Sickle Cell Anemia With Hydroxyurea","SAFE","Inclusion Criteria:\n\n* Patients with documented sickle cell anemia (SCA).\n* At least 8 years of age at the time of enrollment.\n* Enrolled on the EXTEND or SACRED study.\n* Provide informed consent.\n* Able to take part in all parts of the study, including treatments, check-ups, and follow-up visits.\n\nExclusion Criteria:\n\n* Currently taking part in another treatment study (not EXTEND or SACRED).\n* Has received other treatments for sickle cell disease in the past 6 months.","8 Years",{"count":51,"type":19},"The SAFE study is a long-term research project that watches people with sickle cell anemia (SCA) over time. The main goal is to see how a medicine called hydroxyurea affects their growth, puberty, and ability to have children. A second goal is to see how hydroxyurea affects pregnancy outcomes, by comparing people who take the medicine to those who don't.",[24],[26,146,23],"Fertility","2026-02-03",{"date":149,"type":31},"2026-02-04",{"date":151,"type":31},"2023-07-10",{"date":153,"type":19},"2035-12-31",{"name":37,"class":38},3,{"id":157,"slug":158,"hasResults":11,"nctId":159,"briefTitle":160,"officialTitle":161,"acronym":162,"eligibilityCriteria":163,"healthyVolunteers":11,"sex":16,"minAge":164,"maxAge":165,"enrollmentInfo":166,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":168,"conditions":169,"keywords":171,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":173,"lastUpdatePostDateStruct":174,"startDateStruct":176,"completionDateStruct":178,"leadSponsor":180,"locationsCount":155},"100620317","verifying-antibodies-after-live-immunization-delivery-valid-a-study-of-measles-vaccine-immunogenicity-in-children-with-sickle-cell-disease-100620317","NCT07356050","Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease","Verifying Antibodies After Live Immunization Delivery (VALID): a Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease","VALID","Inclusion Criteria:\n\n1. Participants with confirmed Sickle Cell Disease.\n2. Participants 6 months and 6 years of age and due for measles vaccination within 3 months per national guidelines.\n3. Willing and able to provide informed consent\n4. Ability to comply with study related evaluations and follow-up visits.\n\nExclusion Criteria:\n\n1\\. Known primary immunodeficiency syndrome, cancer, or acquired immunodeficiency syndrome (AIDS) that would preclude vaccination with live virus vaccines.","6 Months","6 Years",{"count":167,"type":19},130,"The goal of this study is to learn if infants with sickle cell disease (SCD) develop adequate protection after measles vaccines. (not looking at any prolonged duration)",[23,170,24],"Measles Vaccination",[172],"Measles Vaccine Immunogenicity","2026-01-20",{"date":175,"type":31},"2026-01-21",{"date":177,"type":31},"2025-03-13",{"date":179,"type":19},"2028-12-31",{"name":37,"class":38},{"id":182,"slug":183,"hasResults":11,"nctId":184,"briefTitle":185,"officialTitle":186,"acronym":187,"eligibilityCriteria":188,"healthyVolunteers":11,"sex":16,"minAge":189,"maxAge":110,"enrollmentInfo":190,"targetDuration":4,"studyType":78,"phases":192,"briefSummary":193,"conditions":194,"keywords":196,"overallStatus":205,"whyStopped":4,"lastUpdateSubmitDate":206,"lastUpdatePostDateStruct":207,"startDateStruct":209,"completionDateStruct":211,"leadSponsor":213,"locationsCount":4},"100614639","phase-2-efficacy-and-safety-of-sil-8301-for-control-of-hemolysis-in-a-uniform-sickle-cell-disease-endotype-100614639","NCT07282210","Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype","A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Determine Efficacy and Safety of SIL-8301 in Sickle Cell Disease (SCD) Patients With a Predominantly Hemolytic Phenotype","RESCUE","Inclusion Criteria:\n\n* Documented diagnosis of sickle cell disease\n* 16-35 years of age\n* Hb ≤ 9.0 g\u002FdL\n* History of no more than 1 acute SCD-related painful crises requiring a visit to a medical facility per year within the preceding 2 years\n* History of at least one hemolytic complication\n* Current treatment with hydroxyurea\n\nExclusion Criteria:\n\n* Receipt of senicapoc in a previous investigational study\n* Current Red Blood Cell (RBC) transfusion or exchange transfusion program\n* History of pulmonary hypertension\n* Active cardiovascular, neurologic, endocrine, hepatic, or renal disorders\n* Diagnosis of cancer (except non-melanoma skin cancer in situ, cervical cancer in situ, or breast cancer in situ) within the last 5 years\n* History of liver disease","16 Years",{"count":191,"type":19},105,[80],"SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children. The purpose of this study is to compare the effects of senicapoc to placebo in patients with sickle cell disease that have had fewer than 2 acute sickle-related painful crises per year over the preceding 2 years, and have a predominantly hemolytic phenotype, defined as presence or history of at least one hemolytic complication and a baseline Hb of 9 g\u002FdL or less, despite receiving hydroxyurea (an oral drug used for treatment of sickle cell disease) as standard of care. Participants will take senicapoc or matching placebo daily and continue on hydroxyurea as prescribed for up to 24 weeks.",[23,195,24],"Sickle Cell Anaemia",[197,198,199,200,119,201,121,120,202,203,204],"senicapoc","Anemia, Hemolytic, Congenital","Anemia, Hemolytic","Anemia","Hemic and Lymphatic Diseases","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","Anemia, Sickle Cell","Anaemia, Sickle Cell","NOT_YET_RECRUITING","2025-12-01",{"date":208,"type":31},"2025-12-15",{"date":210,"type":19},"2026-01",{"date":212,"type":19},"2029-01",{"name":214,"class":131},"Biossil Inc.",{"id":216,"slug":217,"hasResults":11,"nctId":218,"briefTitle":219,"officialTitle":220,"acronym":221,"eligibilityCriteria":222,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":4,"enrollmentInfo":223,"targetDuration":4,"studyType":78,"phases":225,"briefSummary":227,"conditions":228,"keywords":230,"overallStatus":205,"whyStopped":4,"lastUpdateSubmitDate":233,"lastUpdatePostDateStruct":234,"startDateStruct":236,"completionDateStruct":237,"leadSponsor":239,"locationsCount":39},"100569736","awake-prone-positioning-for-severe-acute-chest-syndrome-100569736","NCT06698120","Awake Prone Positioning for Severe Acute Chest Syndrome","Assessment of Efficacy and Safety of Awake Prone Positioning in Sickle Cell Anemia Patient Admitted in Intensive Care Unit for Severe Acute Chest Syndrome","PRONE-ACS","Inclusion Criteria:\n\n* Age \\>18 years\n* Major sickle cell anemia (SS, SC, Sβ)\n* Admission in intensive care unit for ACS\n* Registered in the French social insurance regime.\n* Written, informed consent\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding women\n* Immediate need for intubation\n* Impaired vigilance status (Glasgow scale score \\\u003C 12)\n* Pneumothorax\n* Haemodynamically unstable\n* Thoracic trauma admission\n* Severely obese with body-mass index higher than 40 kg\u002Fm²\n* EIT contraindication: pacemaker, automatic implantable defibrillators, skin lesions facing the EIT belt, unstable rachis fracture or medullary lesions",{"count":224,"type":19},15,[226],"NA","Acute chest syndrome (ACS) is the leading cause of admission to intensive care and the leading cause of death in patients with sickle cell disease. Irrespective of the cause of ACS, there is an heterogeneity in pulmonary ventilation\u002Fperfusion ratios, leading to worsening of the disease.\n\nEfficiency of awake prone positioning (APP) in acute respiratory failure (ARF) was particularly highlighted during the COVID-19 pandemic. Several physiological factors contribute to this benefit including an improvement in ventilatory drive and gas exchange.\n\nThe investigator hypothesize that APP could lead to clinical improvement in ACS in terms of oxygenation and ventilatory drive, by improving the heterogeneity of ventilation",[229,24],"Acute Chest Syndrome",[229,24,231,232],"Awake Prone Positioning","Electric Impedance","2025-08-25",{"date":235,"type":31},"2025-09-02",{"date":210,"type":19},{"date":238,"type":19},"2027-08",{"name":240,"class":38},"Assistance Publique - Hôpitaux de Paris",{"id":242,"slug":243,"hasResults":11,"nctId":244,"briefTitle":245,"officialTitle":246,"acronym":4,"eligibilityCriteria":247,"healthyVolunteers":11,"sex":16,"minAge":248,"maxAge":249,"enrollmentInfo":250,"targetDuration":4,"studyType":78,"phases":252,"briefSummary":254,"conditions":255,"keywords":258,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":269,"lastUpdatePostDateStruct":270,"startDateStruct":272,"completionDateStruct":274,"leadSponsor":276,"locationsCount":39},"100335893","phase-1-t-cell-depleted-alternative-donor-bone-marrow-transplant-for-sickle-cell-disease-scd-and-other-anemias-100335893","NCT03653338","T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias","T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias","Inclusion Criteria\n\n1. Patient, parent, or legal guardian must have given written informed consent and\u002For assent according to FDA guidelines.\n2. Ages 5 years to 40 years, at time of consent.\n3. Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following:\n\n   * Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years.\n   * Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.\n   * Stroke or neurologic event lasting \\> 24 hours with an accompanying infarct on MRI in any patient for all ages; Brain MRI with silent infarct without clinical event in patients ≤ 16 years.\n   * Chronic transfusion therapy defined as \\> 8 packed red blood cell transfusions per year in the year prior to enrollment and\u002For evidence of red blood cell alloimmunization.\n   * Elevated transcranial Doppler velocities - \\> 200 cm\u002Fs, via the non-imaging technique or \\> 185 cm\u002Fs by the imaging technique measured on 2 separate occasions ≥ 1-month apart\n   * Elevated TRV \\> 2.6m\u002Fs in patients ≥ 16 years old.\n   * Sickle-related renal insufficiency and\u002For sickle hepatopathy and\u002For any irreversible end-organ damage in patients ≥ 16 years old.\n\n   OR Diagnosis of beta-thalassemia or Diamond-Blackfan anemia complicated by transfusion dependence with evidence of iron overload.\n4. A minimum donor match of 4\u002F8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci in the related setting or minimum donor match of 6\u002F8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci (with the DRB1 locus as a full match requirement). An unrelated donor and cord blood search must have been completed without an eligible 8\u002F8 matched unrelated donor or 6\u002F8 cord blood unit available. Patients who may have acceptable cord blood donor options (4\u002F6 or better) but are limited by cell dose of a single cord will also be eligible for the proposed study.\n5. Adequate function of other organ systems as measured by:\n\n   * Creatinine clearance or GFR ≥ 45 ml\u002Fmin\u002F1.73m.\n   * Hepatic transaminases (ALT\u002FAST) ≤ 3 x upper limit of normal.\n   * Liver MR imaging for iron content should be performed in all patients with Ferritin \\> 500 ng\u002FmL. If hepatic iron content \\> 10mg Fe\u002Fg liver should have hepatology consultation and liver biopsy to confirm absence of cirrhosis, fibrosis or hepatitis.\n   * Adequate cardiac function as measure by echocardiogram (shortening fraction \\> 26% or ejection fraction \\> 40% or \\>80% of age-specific normal).\n   * Pulmonary evaluation testing demonstrating FEV1\u002FFVC ≥ 60% of predicted for age and\u002For resting pulse oximeter ≥ 92% on room air.\n   * Cardiology clearance to proceed with conditioning regimen and HSCT.\n   * Pulmonology clearance to proceed with conditioning regimen and HSCT.\n6. Subjects must be human immunodeficiency virus (HIV) negative by PCR.\n7. Negative pregnancy test for females ≥10 years old or who have reached menarche, unless surgically sterilized.\n8. All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.\n9. Subject and\u002For parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting (Refer to section,\n10. Hydroxyurea must have been trialed and failed in patients with sickle cell disease.\n\nPatient Exclusion Criteria\n\n1. Patients with alternate, superior donor options (matched sibling donor or matched unrelated donor).\n2. Patients who have undergone stem cell transplantation in the 6 months prior to anticipated conditioning.\n3. Patients with history of a central nervous system (CNS) event within six months prior to start of conditioning (patient will be delayed until eligible).\n4. Patients who are pregnant or lactating\n5. Patients with uncontrolled bacterial, viral or fungal infection\n6. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.","5 Years","40 Years",{"count":251,"type":19},5,[253,80],"PHASE1","The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and\u002For haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and\u002For haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.",[24,256,257],"Beta-thalassemia Major","Diamond-blackfan Anemia",[259,260,261,200,262,263,264,265,266,267,268],"Sickle Cell","Diamond-Blackfan","Beta-thalassemia","Stem cell transplantation","Unrelated Donor","haploidentical","hematopoietic stem cell transplant (HSCT)","bone marrow transplant (BMT)","mismatched","t-cell depletion","2025-08-07",{"date":271,"type":31},"2025-08-12",{"date":273,"type":31},"2018-08-02",{"date":275,"type":19},"2027-08-01",{"name":277,"class":38},"Paul Szabolcs",{"id":279,"slug":280,"hasResults":11,"nctId":281,"briefTitle":282,"officialTitle":283,"acronym":4,"eligibilityCriteria":284,"healthyVolunteers":47,"sex":16,"minAge":285,"maxAge":286,"enrollmentInfo":287,"targetDuration":4,"studyType":78,"phases":289,"briefSummary":290,"conditions":291,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":292,"lastUpdatePostDateStruct":293,"startDateStruct":295,"completionDateStruct":297,"leadSponsor":299,"locationsCount":39},"100556804","pressure-pain-tolerance-in-relation-to-balance-and-strength-in-children-100556804","NCT06529874","Pressure Pain Tolerance in Relation to Balance and Strength in Children","Pressure Pain Tolerance in Relation to Balance and Strength in Children With Sickle Cell Anemia","Inclusion Criteria:\n\n* Age range range was 9-14 years old.\n* Could follow given instructions.\n* Children with Sickle cell anemia were medically stable, and take their medications regularly.\n* Children with Sickle cell anemia had experience of chronic pain that lasts more than 6 months.\n* All children with Sickle cell anemia have average weight, height, and body mass index based on their age percentiles.\n* All normal healthy children did not participating in regular sport activities, particularly including lower limb strengthening.\n\nExclusion Criteria:\n\n* Defined cardiovascular or respiratory disorders.\n* Renal failure.\n* Myocardial infarction, mainly within last month.\n* Unstable angina.\n* Recent thoracoabdominal surgery.\n* Thoracic or abdominal aneurysm or current pneumothorax.\n* Painful vaso-occlusive crises.\n* Muscular contractures, Joints' stiffness, or deformities.\n* Neuromuscular problems i.e., myositis or peripheral neuropathy.","9 Years","14 Years",{"count":288,"type":19},80,[226],"Sickle cell disease (SCD) is the most frequent life-threatening genetic hemoglobinopathy in the world and occurs due to the synthesis of abnormal hemoglobin S (HbS).Cells with sickle cell hemoglobin are stiff and sticky. When they lose their oxygen, they form into the shape of a sickle or crescent. This can cause pain and tissue damage. Significant decrease in exercise capacity was seen in sickle cell anemic children. In the absence of a guidelines that can guide the prescription of exercise in SCD children. This study is a step for determine the forms of prescription of pain on the balance and muscle strength to build up in future studies a safety of physical exercises for children with sickle cell anemia and improve their functional abilities.",[24],"2025-03-26",{"date":294,"type":31},"2025-04-01",{"date":296,"type":31},"2024-02-01",{"date":298,"type":19},"2025-08-30",{"name":300,"class":38},"Adly A Adam",{"id":302,"slug":303,"hasResults":11,"nctId":304,"briefTitle":305,"officialTitle":306,"acronym":4,"eligibilityCriteria":307,"healthyVolunteers":11,"sex":16,"minAge":308,"maxAge":110,"enrollmentInfo":309,"targetDuration":4,"studyType":78,"phases":311,"briefSummary":312,"conditions":313,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":318,"lastUpdatePostDateStruct":319,"startDateStruct":321,"completionDateStruct":323,"leadSponsor":325,"locationsCount":327},"100377950","phase-2-a-trial-to-assess-haploidentical-t-depleted-stem-cell-transplantation-in-patients-with-scd-100377950","NCT04201210","A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD","A Phase II Stratified Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With Sickle Cell Disease With no Available Sibling Donor","Inclusion Criteria:\n\n* Age 2yrs to 35yrs\n* Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0\u002F+\n* Study specific consent given\n* Preexisting severe or moderate SCD related complications:\n\n  * Clinically significant neurological event (stroke) or deficit\n  * Silent crisis, neurocognitive deficit\n  * Pathological angio-MRI with TOF Sequence\n  * TCD velocity \\>200 cm\u002Fs at 2 occasions \\>1 month apart\n  * More than 5 vaso-occlusive crises (VOC) in the past 1 year or more than 20 VOC in a lifetime\n  * Two or more episodes of acute chest syndrome (ACS) in a lifetime or one episode of ACS in the past 24 months\n  * Chronic transfusion requirement or more than 8 transfusions or one exchange transfusion in a lifetime\n  * Transfusion-refractory allo-immunization\n  * More than five SCD-related hospitalizations in a lifetime\n  * Beginning pulmonary hypertension\n  * Osteonecrosis at more than 2 sites\n  * Beginning SCD Nephropathy\n  * Recurrent priapism (\\>2)\n\nExclusion Criteria:\n\n* Karnofsky or Lansky Performance Score \\\u003C 70%\n* Patients with donor-specific antibodies (DSA) against the potential stem cell donor by either\n\n  * Cell-based crossmatched assays (Complement-dependent cytotoxicity; CDC) or\n  * Flow cytometry crossmatch test or\n  * Solid-phase immunoassays (SPI) or\n  * Modified SPI such as C4d and C1q assays Whichever method the participating center is experienced in.\n* Patients with major AB0 incompatibility defined according to EBMT Handbook, Edition 2019 Tab 23.1.:\n\nABO incompatibility Recipient Donor Major O A O B O AB A AB B AB\n\n* Cardiac function:\n\n  * Ejection fraction at rest \\\u003C45.0% on echocardiography or\n  * Shortening fraction of \\\u003C27.0% by echocardiogram or radionuclide scan (MUGA)\n  * Patients with \\> grade II hypertension by Common Toxicity Criteria (CTC)\n* Renal function:\n\n  * Estimated creatinine clearance (for patients \\> 12 years) greater than 50.0 mL\u002Fminute\n  * for pediatric patients (\\> 1 year to 12 years), GFR estimated by the updated Schwartz formula ≥ 90.0 mL\u002Fmin\u002F1.73 m2. If \\\u003C 90 mL\u002Fmin\u002F1.73 m2, renal function must be measured by 24-hour creatinine clearance or nuclear GFR and must be \\> 70.0 mL\u002Fmin\u002F1.73 m2 or\n  * Creatinine clearance below threshold defined for stem cell transplantation according to local clinical standard\n* Pulmonary function:\n\n  * DLCO \\>50% (adjusted for hemoglobin), and FVC and FEV1≥50%; children unable to perform for PFTs, O2 saturation \\\u003C92% on room air.\n* Liver function:\n\n  * Total bilirubin \\> 2x the upper limit of normal (unless elevated bilirubin is attributed to Gilbert's Syndrome) and ALT\u002FAST \\> 2.5x the upper limit of normal.\n  * Chronic active viral hepatitis\n* Women who are pregnant (positive serum or urine βHCG) or breastfeeding. Note: Women of childbearing potential must have a negative serum pregnancy test at study entry.\n* Adults of reproductive potential not willing to use an effective method of birth control during study treatment and for at least 12 months thereafter,\n* History of uncontrolled autoimmune disease or on active treatment\n* Patient unable to comply with the treatment protocol\n* Prior autologous or allogeneic hematopoietic stem cell transplant\n* Vaccination with a live virus vaccine during the trial\n* HIV infection\n* Patients with a history of psychiatric illness or a condition which could interfere with their ability to understand the requirements of the study (this includes alcoholism\u002Fdrug addiction)\n* Patients unwilling or unable to comply with the protocol or unable to give informed consent.\n* Concurrent severe or uncontrolled medical disease (e.g. uncontrolled diabetes, congestive heart failure, myocardial infarction within 6 months prior to the study, unstable and uncontrolled hypertension, chronic renal disease, or active uncontrolled infection) which by assessment of the treating physician could compromise participation in the study","2 Years",{"count":310,"type":19},212,[80],"HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß\u002FCD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.",[314,315,24,316,317,23],"HbS Disease","Hemoglobin S Disease","Sickle Cell Disorders","Sickling Disorder Due to Hemoglobin S","2024-05-10",{"date":320,"type":31},"2024-05-13",{"date":322,"type":31},"2021-06-30",{"date":324,"type":19},"2030-03-31",{"name":326,"class":38},"University of Regensburg",9]