[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"anemia-sickle-cell\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:anemia-sickle-cell":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,43,74,108,143],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100553070","phase-1-a-study-to-evaluate-bms-986470-in-healthy-volunteers-and-participants-with-sickle-cell-disease-100553070",false,"NCT06481306","A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease","A Phase 1\u002F2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470","Inclusion Criteria\n\n\\- Cohort A.\n\ni) Healthy male and female (who are not of childbearing potential) participants, as determined by the investigator based on medical history and other determinations. Females not of childbearing potential must have been amenorrhoeic for at least 12 months without an alternative medical cause and have follicle-stimulating hormone (FSH) levels of at least 40 IU\u002FL or have undergone a hysterectomy, bilateral oophorectomy, or bilateral salpingectomy.\n\nii) Body mass index (BMI) of 18.0 to 32.0 kg\u002Fm\\^2, inclusive. BMI = weight (kg)\u002F (height \\[m\\])\\^2 as measured at screening.\n\niii) No evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, ECG, or clinical laboratory assessments beyond what is consistent with the target population.\n\n\\- Cohort B.\n\ni) Participants with a documented diagnosis of Sickle Cell Disease (SCD) with genotype HbSS, HbSβ0-thal, or HbSβ+-thal.\n\nii) Participants with ≥ 4 vaso-occlusive crises (VOCs) within the previous 12 months or ≥ 2 VOCs within the previous 6 months.\n\niii) Participant has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.\n\niv) Must have the following laboratory values:.\n\nA. Hemoglobin ≥ 5.5 and ≤ 12 g\u002FdL (males) or ≥ 5.5 and ≤ 10.6 g\u002FdL (females).\n\nB. Absolute neutrophil count ≥ 1500\u002FμL.\n\nC. Platelet count ≥ 100 × 10\\^3\u002FμL.\n\nD. Absolute reticulocyte count \\> 100 × 10\\^3\u002FμL or \\> 50 × 10\\^3\u002FμL if taking hydroxyurea.\n\nExclusion Criteria\n\n\\- Cohort A.\n\ni) Any significant medical condition or any condition that confounds the ability to interpret data from the study.\n\nii) Participant has any condition, including the presence of laboratory abnormalities, that places the participant at unacceptable risk if the participant was to participate in the study.\n\niii) Any major surgery or planned surgery (except GI surgery) within 12 weeks of the first study intervention administration.\n\n\\- Cohort B.\n\ni) Participants with any condition, including significant acute or chronic medical illness, active or uncontrolled infection, or the presence of laboratory abnormalities, that places participants at unacceptable risk if participating in this study.\n\nii) Participants with more than 6 severe VOCs defined as VOCs requiring ≥ 24 hours of hospital admission within 12 months prior to the first dose of study intervention or any VOC requiring ≥ 24 hours of hospital admission within 30 days prior to the first dose of study intervention.\n\niii) Participants with any episode of acute chest syndrome within the last 6 months prior to the first dose of study intervention.\n\niv) Creatinine clearance (CrCl) \\\u003C 60 mL\u002Fmin\u002F1.72m2 using Chronic Kidney Disease Epidemiology (CKD-EPI) equation\n\n* Cohort A and B.\n\n  i) Participant is receiving regularly scheduled RBC or platelet transfusions or has received a RBC transfusion within 28 days and a platelet transfusion within 14 days prior to starting treatment with BMS-986470.\n* Other protocol-defined Inclusion\u002FExclusion criteria apply.",true,"ALL","18 Years",{"count":20,"type":21},184,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.",[28,29],"Anemia, Sickle Cell","Healthy Volunteers","RECRUITING","2026-06-15",{"date":33,"type":34},"2026-06-16","ACTUAL",{"date":36,"type":34},"2024-07-17",{"date":38,"type":21},"2027-11-16",{"name":40,"class":41},"Bristol-Myers Squibb","INDUSTRY",34,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":17,"minAge":50,"maxAge":51,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":56,"conditions":57,"keywords":58,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":73},"100538405","a-socio-ecological-approach-for-improving-self-management-in-adolescents-with-scd-100538405","NCT06290401","A Socio-ecological Approach for Improving Self-management in Adolescents With SCD","SC-Thrive","Inclusion Criteria:\n\n* Patient of a participating SCD Clinic\n* Confirmed diagnosis of SCD\n* 13-21 years of age\n\nExclusion Criteria:\n\n* Another chronic disease (which would complicate measurement of patient activation)\n* Non-English-speaking\n* Cognitive or psychiatric disorder that the physician or study therapists believe would impair study participation.","13 Years","21 Years",{"count":53,"type":21},310,[55],"NA","The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents and young adults with sickle cell disease (SCD) ages 13-21 receiving care at 1 of 4 pediatric SCD clinics.\n\nThe main question\\[s\\]it aims to answer are:\n\n* Does SCThrive improve patient activation?\n* Does SCThrive improve self-management behaviors, daily functioning, and decrease emergency room visits?\n* Are any improvements maintained 3 months after treatment?\n\nParticipants will complete self-management related surveys before, after, and 3 months following their participation in an 8- week, virtual group intervention with an accompanying mobile app (SCThrive).\n\nResearchers will compare outcomes for participants who receive SCThrive and participants who receive uniform standard care (SCHealthED which = standard of care plus SCD educational text messages) to see if there are differences in patient activation, self-management behaviors, daily functioning, and emergency room visits.",[28],[59,60,61,62],"adolescent and young adult (AYA)","self-management","behavioral intervention","sickle cell disease","2026-05-06",{"date":65,"type":34},"2026-05-11",{"date":67,"type":34},"2025-02-03",{"date":69,"type":21},"2028-06-30",{"name":71,"class":72},"Children's Hospital Medical Center, Cincinnati","OTHER",4,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":81,"enrollmentInfo":82,"targetDuration":4,"studyType":22,"phases":84,"briefSummary":85,"conditions":86,"keywords":87,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":107},"100527206","phase-1-a-safety-efficacy-and-pharmacokinetic-pk-study-of-hbi-002-an-oral-carbon-monoxide-co-therapeutic-in-subjects-with-sickle-cell-disease-scd-100527206","NCT06144749","A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)","An Open Label Phase 2a Ascending Multiple Dose Safety, Efficacy, and Pharmacokinetic Study of HBI-002 Carbon Monoxide Oral Liquid Drug Product in Adolescents and Adults With Sickle Cell Disease.","Inclusion Criteria:\n\n1. Signed informed consent.\n2. Male or female 14-55 years of age inclusive.\n3. Negative Hepatitis B surface antigen (HBsAg), anti-Hepatitis C (aHCV), anti-Human Immunodeficiency Virus (aHIV), and SARS-CoV-2 test. Subjects that test SARS-CoV-2 positive at screen or baseline can be re-screened at least four weeks after the positive test so long as they are asymptomatic and test negative.\n4. Non-smoker or vaper (no use of tobacco or marijuana products within 3 months of screening).\n5. Body weight. For 18 years of age and above, between 45 kg and 110 kg (inclusive) and with body mass index (BMI) less than 30 kg\u002Fm2. For 14 to 18 years of age, above 35 kg and with BMI less than 30 kg\u002Fm2.\n6. Established Hb-SS or Sβ0 SCD with:\n\n   1. ≤10 vaso-occlusive crises (VOC) per year over the prior two years; and\n   2. a history of one or more episodes of one of the following: acute chest syndrome, stroke, priapism, bone avascular necrosis, or splenic sequestration.\n7. Subjects may or may not be receiving hydroxyurea (HU) for SCD, but if receiving HU, they must be taking this medication according to physician instructions and without dose adjustment for a minimum of one month prior to the inclusion to this study.\n8. Normal cardiac function as evidenced by clinical, ECG and laboratory findings.\n9. Carboxyhemoglobin level by co-oximetry ≤ 3.5% (prior to first dose).\n10. The absence of current clinically relevant abnormalities identified by a detailed medical history, full physical examination including blood pressure and pulse rate measurement, 12-lead ECG, and clinical chemistries including liver and kidney function, as determined by the Investigator.\n11. Hematology including hemoglobin \\>6g\u002FdL, baseline white blood cell count \\>7,000\u002FuL and absolute neutrophil count (ANC) \\>3,500\u002FuL, platelet count \\>80,000\u002FuL and including coagulation labs (PT, PTT), with all levels documented as stable or not clinically significant changes over the period between screening and baseline.\n12. Negative pregnancy test for females.\n13. Subjects must be willing to use a highly effective method of contraception for the duration of the study and for 30 days thereafter, if applicable.\n\n    1. Male subjects, without a vasectomy, must use a condom and be instructed that their female partner should use another form of contraception such as an IUD, diaphragm with spermicide, oral contraceptive, injectable progesterone, subdermal implant if the female partner could become pregnant.\n    2. Female subjects of childbearing potential (not surgically sterilized and less than one year post-menopausal) should use a form of contraception such as an intrauterine device (IUD), diaphragm with spermicide, oral contraceptive, injectable progesterone, subdermal implant, and be instructed that their male partners should use a condom, if not vasectomized.\n\nExclusion Criteria:\n\n1. Hemoglobinopathy other than SCD or S\u002Fbeta 0 thalassemia.\n2. Clinical, ECG or laboratory evidence of cardiac dysfunction as determined by the PI.\n3. Acute chest syndrome or VOC within 21 days or signs or symptoms of an impending vaso-occlusive crisis as determined by both the subject and PI immediately prior to first dosing with HBI-002\u002Fplacebo.\n4. Current smoker.\n5. Clinically significant illness or surgery other than that associated with SCD within 3 months prior to dosing.\n6. Blood transfusion within six weeks prior to the first administration of study drug.\n7. Exposure to any live vaccine within 28 days prior to study drug administration.\n8. History of febrile or infective illness within 14 days prior to dosing.\n9. Positive pregnancy test or breast feeding for females.\n10. Weight loss or gain of more than 5 kg within 3 months prior to dosing.\n11. History of alcohol abuse or dependence or regular use of alcohol within six months prior to dosing (defined as more than 14 units of alcohol per week; 1 Unit= 150 mL wine, 360 mL beer or 45 mL of 40% alcohol)\n12. History of renal dysfunction with glomerular filtration rate \\\u003C30 mL\u002Fmin\u002F1.73m2.\n13. History of pulmonary infiltrate or pneumonia within 6 months prior to dosing or pulmonary\u002Fbronchial infection within 2 weeks prior to dosing.\n14. Subject on domiciliary oxygen\n15. History of cancer, with the exception of adequately treated basal cell or squamous cell carcinoma of the skin more than 1 year prior.\n16. History of cardiac disease.\n17. History of drug abuse, including administration of opioids for reasons other than pain control. Use of opioids for control of pain is permitted.\n18. Use of voxelotor or crizanlizumab or prescription drugs other than prescribed for SCD, within 7 days or 5 half-lives (whichever is longer) prior to dosing. Herbal and vitamin supplements (excluding L-glutamine) must be discontinued 14 days prior to dosing. Prescribed analgesics for regular self-administration are permitted provided that dosing of such has not been increased or new drugs added over the past 7 days.\n19. Unwilling or unable to comply with the requirements of the protocol.\n20. Treatment with an investigational drug within the longer of 30 days or five half-lives.\n21. Clinically significant ECG abnormalities or vital sign abnormalities (systolic blood pressure lower than 90 or above 140 mmHg, diastolic blood pressure lower than 50 or above 90 mmHg, or heart rate less than 45 or above 100 bpm or arrhythmia) at screening and\u002For baseline, as determined by the Investigator. Heart rate corrected QT interval-Frederica's method (QTcF) \\>450 msec male, \\>470 msec female.\n22. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into the study.\n23. Any coincident disease or condition that in the opinion of the investigator will make the subject inappropriate for entry into the study or will confound the assessment of safety.\n24. History of allergic reactions to any of the drug product excipients.","55 Years",{"count":83,"type":21},9,[24,25],"This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.",[28],[28,88,89,90,91,92,93,94,95,96,97],"Anemia, Hemolytic","Congenital Anemia","Hemolytic Anemia","Hematologic Diseases","Hemoglobinopathies","Genetic Diseases, Inborn","Carbon Monoxide","CO","Heme Oxygenase","HO-1","2026-01-07",{"date":100,"type":34},"2026-01-08",{"date":102,"type":34},"2025-02-15",{"date":104,"type":21},"2026-06-30",{"name":106,"class":41},"Hillhurst Biopharmaceuticals, Inc.",2,{"id":109,"slug":110,"hasResults":11,"nctId":111,"briefTitle":112,"officialTitle":113,"acronym":4,"eligibilityCriteria":114,"healthyVolunteers":16,"sex":17,"minAge":115,"maxAge":51,"enrollmentInfo":116,"targetDuration":4,"studyType":22,"phases":118,"briefSummary":119,"conditions":120,"keywords":124,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":142},"100393728","metabolic-and-hemodynamic-reserve-in-pediatric-sca-100393728","NCT04406818","Metabolic and Hemodynamic Reserve in Pediatric SCA","The Role of Metabolic and Hemodynamic Reserve in Age-Related Brain Vulnerability in Pediatric Sickle Cell Anemia","Healthy Controls:\n\n* Healthy controls ages 4-21 years of age\n* Able to participate in MRI scan without sedation\n* Not currently pregnant\n* No significant psychiatric history, defined as having a severe psychiatric diagnosis, per PI discretion\n* No history of epilepsy\n* No history of stroke or cerebrovascular disease\n* May have occasional headaches if not taking a daily preventative medication for headaches\n* Not on vasodilatory medication, such as sildenafil or verapamil\n\nSickle Cell Anemia Participants:\n\n* Ages 4-21 years of age\n* Hb SS or SBeta-thal\n* Able to participate in MRI scan without sedation\n* Not currently pregnant\n* Not on vasodilatory medication, such as sildenafil or verapamil\n* No known vasculopathy","4 Years",{"count":117,"type":21},120,[55],"The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life. SCA participants and healthy controls are age and sex-matched for comparison. Within the SCA cohort, children with infarcts may have thinner cortices than those without, reflecting a greater loss.\n\nThe investigators will examine brain blood flow and metabolism using magnetic resonance imaging (MRI). The brain's blood vessels expand and constrict to regulate blood flow based on the brain's needs. The amount of expanding and contracting the blood vessels may vary by age. The brain's blood flow changes in small ways during everyday activities, such exercise, deep concentration, or normal brain growth. Significant illness or psychological stress may increase the brain's metabolic demand or cause other bigger changes in blood flow. If blood vessels are not able to expand to give more blood flow when metabolic demand is high, the brain may not get all of the oxygen it needs. In extreme circumstances, if the brain is unable to get enough oxygen for a long time, a stroke may occur. Sometimes small strokes occur without other noticeable changes and are only detectable on an MRI. These are sometimes called \"silent strokes.\" In less extreme circumstances, not having a full oxygen supply may cause the brain to grow and develop more slowly than when it has a full supply.\n\nOne way to test the ability of blood vessels to expand is by measuring blood flow while breathing in carbon dioxide. Carbon dioxide causes blood vessels in the brain to dilate without increasing brain metabolism.\n\nDuring this study participants may be asked to undergo a blood draw, MRI, cognitive assessments, and brief questionnaires. The study team will use a special mask to control the amount of carbon dioxide the participants breathe in.",[121,122,123,28],"Child, Only","Brain Diseases","Sickle Cell Disease",[125,126,127,128,129,130,131,132],"sickle cell anemia","cerebrovascular reactivity","cerebral oxygen metabolism","cortical thickness","gray matter","carbon dioxide","magnetic resonance imaging","brain development","2025-09-03",{"date":135,"type":34},"2025-09-05",{"date":137,"type":34},"2021-06-30",{"date":139,"type":21},"2026-03-31",{"name":141,"class":72},"Washington University School of Medicine",1,{"id":144,"slug":145,"hasResults":11,"nctId":146,"briefTitle":147,"officialTitle":147,"acronym":148,"eligibilityCriteria":149,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":150,"enrollmentInfo":151,"targetDuration":153,"studyType":154,"phases":4,"briefSummary":155,"conditions":156,"keywords":168,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":175,"lastUpdatePostDateStruct":176,"startDateStruct":178,"completionDateStruct":180,"leadSponsor":182,"locationsCount":142},"100535343","european-rare-blood-disorders-platform-enrol-100535343","NCT06250595","European Rare Blood Disorders Platform (ENROL)","ENROL","Inclusion Criteria:\n\n* Patients must meet all of the following criteria to be included in the ENROL Registry\n* Age from 0-100, both female and male\n* Diagnosed as RHDs according to ORPHANET classification\n* Able and willing to provide written informed consent (patient or legal representative for minors) if needed according to national legislation.\n\nExclusion Criteria:\n\n* Patients diagnosed as traits or trait conditions for other recessive RHDs","100 Years",{"count":152,"type":21},37090,"15 Years","OBSERVATIONAL","ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform.\n\nENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data.\n\nAccordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when \u002F where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies.\n\nThe standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources.\n\nMoreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal.\n\nENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670",[157,158,159,160,161,162,163,164,28,165,166,167],"Anemia","Bone Marrow Failure","Bleeding Disorder","Iron Metabolism Disorders","Myeloma","Lymphoid Neoplasm","Myeloma, Malignant","Leukemia","Thalassemia","Blood Cancer","Red Cell Membrane and Enzyme Abnormalities",[157,158,169,170,171,172,173,164,174,165,123],"Bleeding disorder","Iron metabolism disorder","Myeloid","Lymphoid","Blood cancer","Red Cell membrane and Enzyme Abnormalities","2024-02-06",{"date":177,"type":34},"2024-02-09",{"date":179,"type":34},"2022-07-01",{"date":181,"type":21},"2037-07",{"name":183,"class":72},"Hospital Universitari Vall d'Hebron Research Institute"]