[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"sickle-cell-disease-scd\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:sickle-cell-disease-scd":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,15,0,[8,44,71,98,125,155,183,211,240,266,291,317,341,367,389],{"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":28,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":4},"100633767","omega-3-fatty-acids-in-children-with-sickle-cell-disease-100633767",false,"NCT07530965","Omega-3-Fatty Acids in Children With Sickle Cell Disease","A Single Arm\u002F Open Label Feasibility Trial of Omega-3- Fatty Acids in Children With Sickle Cell Disease","Inclusion Criteria:\n\n\\- Children 5-18 years with SCD at steady state. Steady state will be defined as not requiring an acute care visit for pain in the last 28 days.\n\nExclusion Criteria:\n\n* current use of antibiotics except prophylactic penicillin\n* current use of pre-or probiotic supplements\n* current use of PPI therapy\n* pregnant or lactating females\n* individuals with known allergy to Flaxseed","ALL","5 Years","18 Years",{"count":20,"type":21},20,"ESTIMATED","INTERVENTIONAL",[24],"NA","The purpose of this feasibility study is to investigate the role of a dietary supplement in modulating the gut microbiota and improving pain outcomes in children with sickle cell disease (SCD).",[27],"Sickle Cell Disease (SCD)",[29,30,31],"Nutritional trial","sickle cell disease","children","NOT_YET_RECRUITING","2026-08-19",{"date":35,"type":36},"2026-08-21","ACTUAL",{"date":38,"type":21},"2026-12-01",{"date":40,"type":21},"2029-06-30",{"name":42,"class":43},"University of Alabama at Birmingham","OTHER",{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":48,"acronym":49,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":16,"minAge":51,"maxAge":18,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":57,"conditions":58,"keywords":59,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":63,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":68,"locationsCount":70},"100574613","phase-2-optimizing-hydroxyurea-dosage-with-pharmakokinetic-in-patients-suffering-of-moderate-to-severe-sickle-cell-anemia-100574613","NCT06761560","Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia","OPTIMA","Inclusion Criteria (Group A and B) :\n\n* Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.\n* Patients with SS, SBThal0.\n* Agree to take hydroxyurea for a period of 12 months\n* Be between age of 6months old and 18 years old.\n* Have consented for participation in the study.\n\nInclusion Criteria (Group C) :\n\n* Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.\n* Patients with SS, SBThal0.\n* Have taken hydroxyurea for a period of at least 12 months, and have received HU at a stable dose and at MTD for at least 6 months.\n* Be between age of 6months old and 18 years old.\n* Have consented for participation in the study.\n\nExclusion Criteria:\n\n* Patients with sickle cell genotype other than SS or SBThal0 (SC, SBThal+, SE or SD)\n* Patients on chronic transfusion program\n* Patients have received a blood transfusion in the last 4 weeks of study enrollment.\n* Have received a hematopoietic stem-cell transplantation\n* Creatinine \\>2x normal for age\n* ALT\\>2x normal for age\n* Sexually active females unwilling to comply with reliable method of birth control\n* Pregnancy\n* Conditions which in the opinion of the investigator, would compromise participation in the study will be excluded.","6 Months",{"count":53,"type":21},29,[55,56],"PHASE2","PHASE3","The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Pharmacokinetic test is used to evaluate the process by which drugs are absorbed, distributed in the body, localized in the tissues, and is excreted.\n\nPatient will be a randomized (coin toss method) into 2 groups. Group A will have an increase of their HU dosage with pharmacokinetic results and Group B will have an increase of their HU dosage following the standard of care bloodwork follow-up.\n\nGroup C will include patient with sickle cell disease that has been taking HU for at least 12 months and will undergo a pharmacokinetic dosage to check the level of HU only one time.",[27],[60,61,30],"hydroxyurea","pharmacokinetic","RECRUITING",{"date":35,"type":36},{"date":65,"type":36},"2026-02-03",{"date":67,"type":21},"2028-11-25",{"name":69,"class":43},"Yves Pastore",1,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":75,"acronym":76,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":16,"minAge":78,"maxAge":79,"enrollmentInfo":80,"targetDuration":4,"studyType":22,"phases":82,"briefSummary":83,"conditions":84,"keywords":86,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":70},"100650846","standard-of-care-comparative-arm-of-phase-12-gene-therapy-trial-drepamir-in-severe-sickle-cell-disease-patients-100650846","NCT07752043","Standard of Care Comparative Arm of Phase 1\u002F2 Gene Therapy Trial DREPAMIR\" in Severe Sickle Cell Disease Patients","Drepamir-soc","Inclusion Criteria:\n\n* Age 12 - 35 years\n* Diagnosis of HbSS by Hb electrophoresis and genetic analysis to analyse the alpha locus\n* Clinical history or ongoing evidence of severe sickle cell anemia with one OR more of the following clinical complications demonstrating disease severity:\n\nAt least 3 vaso-occlusive crises requiring hospitalization, under hydroxyurea or transfusion, within 2 years prior to enrollment One severe acute chest syndrome (ACS) hospitalized in the intensive care unit At least 2 episodes of ACS, including one under HU. Acute priapism (at least 2 episodes \\>3h in the preceding year or in the year prior to the start of a regular transfusion program), OR stuttering priapism ≥ 1 by week under sickle cell treatment (HU, transfusion or phlebotomy).\n\nTricuspid regurgitation velocity \\>2.8m\u002Fs on cardiac echocardiograph without pulmonary hypertension confirmed by right heart catheterization (mPAP\\>\\\u003C25mmHg)\n\n* Failed hydroxyurea (HU) therapy, were unable to tolerate HU therapy, OR inadequate clinical response to HU, defined as any one of the following outcomes, while on HU for at least 3 months: 2 or more acute sickle pain crisis requiring hospitalization, requirement of transfusion to maintain Hb \\>6.0g\u002FdL, an episode of ACS despite adequate supportive care measures\n* Karnovsky\u002FLansky performance score ≥ 60%\n* Sexually active patients must be willing to use an acceptable method of double-barrier contraception for at least 12 months post-infusion (beyond 12 months at the discretion of the investigator)\n* Procedure for obtaining consent (adults, dependent minors, to give their consent)\n* Affiliation to social security\n\nExclusion Criteria :\n\n* Existence of a matched sibling donor\n* Based on myelogram, the presence of chromosomal (detected by karyotyping) or molecular abnormalities (detected by NGS) and retained dangerous by the Hemato-Oncology referent and validated during a specific multidisciplinary concerted meeting\n* Patients who have already been treated with gene therapy or BMT\n* Hematologic evaluation: Leukopenia (WBC \\\u003C3,000\u002FµL) or neutropenia (ANC \\\u003C1,000\u002FµL) or thrombocytopenia (platelet count \\\u003C100,000\u002FµL) within 90 days prior to mobilization or harvest (not due to an erytrapheresis procedure or possible acute viral infection)\n* PT\u002FINR or PTT \\>1.5 times the upper limit of normal (ULN) or clinically significant bleeding disorder\n* Two alpha deletions (risk of alpha-thalassemia after gene therapy)\n\nEvaluations within 6 months prior to screening visit:\n\n* ALT or AST \\>3 times ULN\n* Severe liver iron overload evaluated by MRI (\\>15mg Fe\u002Fg dry weight or \\>270umol Fe\u002Fg dry weight) or liver cirrhosis suspicion on echography or elastometry or CT scan or MRI AND confirmed by histology\n* Measured GFR \\\u003C60ml\u002Fmin\u002F1.73 m²\n* Cardiac evaluation: LVEF \\\u003C40% by cardiac echocardiogram or by MUGA scan or clinically significant ECG abnormalities\n* Stroke with significant CNS sequelae i.e., Rankin \\>2\n* Specific sickle cell disease cerebral vasculopathy confirmed by MRA (magnetic resonance angiography) OR transcranial doppler ultrasound with or without Moya-moya WITH an indication of chronic transfusion program (target HbS\\\u003C30%)\n* Lung interstitial infiltrate AND Forced Vital Capacity less than 70% AND DLCO less than 60% at steady state\n* Confirmed pulmonary hypertension defined by a right heart catheterization (PAPm \\>25 mmHg). Right heart catheterization is required if tricuspid regurgitation velocity \\>2.8m\u002Fs on cardiac echocardiograph OR \\>2.5m\u002Fs with an abnormal Brain Natriuretic Peptide dosage or an important decrease in transcutaneous Hb O2 saturation during the 6 minutes' walk test.\n* Seropositivity for HIV (Human Immunodeficiency Virus), HCV (Hepatitis C Virus), HTLV-1 (Human T-Lymphotropic Virus), or active Hepatitis B Virus, or active infection by CMV or parvovirus B19, based on positive blood PCR.\n* Pregnancy or breastfeeding in a postpartum female\n* Any current cancer or prior history of a malignant disease, with the exception of curatively treated non-melanoma skin cancer\n* Immediate family member with an established or suspected Familial Cancer Syndrome\n* Diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study\n* Patients who failed previous HSCT\n* Any clinically significant active infection\n* Participation in another clinical study with an investigational drug within 30 days of screening\n* Any condition, based on perspective of the medical monitor and treating investigator, which may lead to increased safety risk or inability to comply with the protocol.","12 Years","35 Years",{"count":81,"type":21},30,[24],"The purpose of this study is to compare the efficacy and safety of transplantation of gene modified autologous CD34+ cells in SCD patients within a therapeutic strategy that may include anti-inflammatory treatment as a pre-transplant treatment in case of severe inflammation detected at the inclusion analysis; the autologous CD34+ cell will be transduced by the bifunctional βAS3m\u002FmiR7m lentiviral vector expressing the therapeutical beta-globin, βAS3m, and the miRNA anti-HbS vs Standard Of Care (SOC).",[27,85],"Vaso-occlusive Events",[87,88],"Sickle cell disease","Vaso-occlusive events","2026-08-03",{"date":91,"type":36},"2026-08-07",{"date":93,"type":21},"2026-09",{"date":95,"type":21},"2030-03",{"name":97,"class":43},"Assistance Publique - Hôpitaux de Paris",{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":104,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":16,"minAge":18,"maxAge":4,"enrollmentInfo":106,"targetDuration":4,"studyType":22,"phases":108,"briefSummary":109,"conditions":110,"keywords":112,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":4},"100645640","cpap-for-hypoxemic-acute-chest-syndrome-in-sickle-cell-disease-100645640","NCT07703566","CPAP for Hypoxemic Acute Chest Syndrome in Sickle Cell Disease","Continuous Positive Airway Pressure for Hypoxemic Acute Chest Syndrome in Patients With Sickle Cell Disease","SIPAP","Inclusion Criteria:\n\n* SCD patient of all genotypes (SS, SC, S\u002Fβ0 and S\u002Fβ+)\n* Age ≥ 18 years old\n* Hospitalised for ACS (defined as the association of fever and\u002For acute respiratory symptoms with a new pulmonary infiltrate on chest imaging)\n* Requiring supplemental O2 ≥ 2 L\u002Fmin for SpO2 ≥ 95%\n* Informed consent from the patient\n* Affiliated to a social security regime\n\nExclusion Criteria:\n\n* Patient having both ACS criteria and need for supplemental O2 ≥ 2 L\u002Fmin for SpO2 ≥ 95% since more than 48 hours\n* Requirement for home supplemental O2 or home CPAP \u002F NIV.\n* Signs of worsening respiratory failure mandating intubation (as defined in (Helms et al., 2024))\n* Current enrolment in another interventional research concerning a respiratory support during ACS\n* Known legal incapacity (patients under guardianship or curatorship)\n* Exacerbation of asthma, chronic obstructive pulmonary disease or another known or suspected chronic respiratory disease\n* Absolute contraindications to CPAP, including any of the following: patient not cooperating or opposing the technique, pneumothorax not drained, chest wound blowing, uncontrollable vomiting, upper gastrointestinal bleeding, craniofacial trauma, severe upper airway obstruction, traumatic tetraplegia at the initial phase, cardiac arrest, shock (need for vasopressor), or Coma Glasgow scale \\\u003C12.\n* Known pregnancy, breast feeding, women with childbearing potential will be tested for pregnancy and excluded if pregnant,",{"count":107,"type":21},140,[24],"Sickle cell disease (SCD) is a severe hemoglobinopathy, considered the first monogenic disease in the world. Acute chest syndrome (ACS), one of the most frequent and serious complications of SCD, is defined by the association of fever and\u002For acute respiratory symptoms with a new pulmonary infiltrate on chest imaging. ACS is characterized by lung consolidation, severe pulmonary vascular dysfunction, with potential role for regional alveolar hypoxia. Therefore, improving alveolar oxygenation and limiting lung consolidation are key objectives of the treatment of ACS, in addition to ensuring pain relief and giving blood transfusions and antibiotics. Bilevel non-invasive ventilation failed in improving outcomes during ACS (Fartoukh 2010). These results are in accordance with those reported in other forms of acute lung injury (Frat 2015), with conflicting results. Among other explanations, NIV may favour high tidal volume ventilation leading to patient self-inflicted lung injury (P-SILI) (Carteaux 2016). Continuous positive airway pressure (CPAP) is a simple to use and affordable technique for non-invasive ventilatory support, that theoretically exposes to a lower risk of P-SILI (Carteaux 2021). In patients with acute hypoxemic respiratory failure (AHRF), applying a positive pressure to the airway opening has been shown to mitigate the reduction in functional residual capacity and to improve respiratory mechanics and gas exchange. In a randomized controlled trial (RCT) conducted in patients with AHRF, CPAP achieved early physiologic improvement (Delclaux 2000). Recent results also suggest that CPAP reduces the composite outcome of intubation or death in adults with AHRF due to COVID-19 in a large multicentre study (RECOVERY-R) (Perkins 2022). In addition, CPAP can be safely used at early stages in the wards, with a frugal approach, using virtual valves (Carteaux 2021). In patients with SCD, CPAP has shown benefits when used at night in children with sleep apnea (Marshall 2009), or for the peri-operative management (Leff 2007). CPAP is also used in clinical practice for hypoxemic ACS (Heilbronner 2021), but it has not been formally assessed in this setting.",[27,111],"Acute Chest Syndrome (ACS)",[113,114,115,116],"CPAP","Acute chest syndrome (ACS)","Sickle cell disease (SCD)","supplemental oxygen","2026-07-21",{"date":119,"type":36},"2026-07-23",{"date":121,"type":21},"2026-09-01",{"date":123,"type":21},"2028-12-01",{"name":97,"class":43},{"id":126,"slug":127,"hasResults":11,"nctId":128,"briefTitle":129,"officialTitle":130,"acronym":4,"eligibilityCriteria":131,"healthyVolunteers":11,"sex":16,"minAge":132,"maxAge":133,"enrollmentInfo":134,"targetDuration":4,"studyType":22,"phases":136,"briefSummary":138,"conditions":139,"keywords":141,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":70},"100647007","phase-4-low-dose-bolus-ketamine-for-use-in-sickle-cell-pain-crisis-100647007","NCT07682662","Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis","Evaluation of a Standardized Low-Dose Bolus Ketamine Pathway for Management of Pediatric Sickle Cell Patients Presenting to the Emergency Department With Pain","Inclusion Criteria:\n\n* Confirmed Sickle Cell Disease (any genotype), Presenting to the Emergency Department with Vaso-occlusive crisis\u002Fpain crisis, Consent obtained\n\nExclusion Criteria:\n\n* Ketamine allergy, Severe agitation\u002Fpsychosis, Pregnancy, Hemodynamic instability (as judged by physician), Increased intracranial pressure, Severe hepatic impairment, Ketamine use within the previous 24 hours, Presentation for non-VOC-related pain (i.e. fever, acute chest syndrome, stroke, traumatic injuries etc).","2 Years","21 Years",{"count":135,"type":21},400,[137],"PHASE4","The goal of this study is to learn if Ketamine works more efficiently, as compared to Opioids, for Sickle Cell Pain The main questions it aims to answer are:\n\nDoes Ketamine lower the number of times participants need to be admitted for continued pain control during a Sickle Cell Pain Crisis.\n\nDoes Ketamine decrease the amount of time it takes to reach adequate pain control\u002Fpain score improvement, as compared to Opioids.\n\nPatients could have too low or too high blood pressure or sleepiness. Researchers will compare Ketamine to Opioids (Morphine or Dilaudid) to see if Ketamine works to treat pain enough that you do not need to be admitted to the hospital.\n\nParticipants will:\n\nOn arrival to the Children's ER for Sickle Cell Pain crisis will get Ketamine, instead of Morphine or Dilaudid, along with the typical Tylenol, Toradol, Lidocaine patch for pain control while in the ER.\n\nDuring this time we will follow your reported pain scale (0-10) to monitor your pain response to the Ketamine, as well as follow rate of hospital admission.",[140,27],"Vaso-Occlusive Pain Episode in Sickle Cell Disease",[142,143,144,145],"Pain dosed ketamine","pediatric sickle cell pain crisis","Ketamine in sickle cell pain","sickle cell pain","2026-06-26",{"date":148,"type":36},"2026-07-06",{"date":150,"type":21},"2026-08-01",{"date":152,"type":21},"2029-10-01",{"name":154,"class":43},"University of Mississippi Medical Center",{"id":156,"slug":157,"hasResults":11,"nctId":158,"briefTitle":159,"officialTitle":159,"acronym":160,"eligibilityCriteria":161,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":162,"enrollmentInfo":163,"targetDuration":4,"studyType":165,"phases":4,"briefSummary":166,"conditions":167,"keywords":169,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":174,"lastUpdatePostDateStruct":175,"startDateStruct":177,"completionDateStruct":179,"leadSponsor":181,"locationsCount":182},"100643053","epidemiology-of-invasive-bacterial-infections-in-children-with-sickle-cell-disease-in-france-between-2020-and-2025-100643053","NCT07639008","Epidemiology of Invasive Bacterial Infections in Children With Sickle Cell Disease in France Between 2020 and 2025","DREPIBAC","Inclusion Criteria\n\n* Children with sickle cell disease aged under 18 years\n* Hospitalized between January 2020 and December 2025\n* Hospitalized for an invasive bacterial infection clinically defined as meningitis, pleuropneumonia, osteoarticular infection, or primary bacteremia\n* Bacterium identified by culture or PCR\n\nExclusion Criteria\n\n* Opposition from the child or his\u002Fher parents\n* Bacterium identified by serology\n* Urinary tract infection","17 Years",{"count":164,"type":21},350,"OBSERVATIONAL","Children with sickle cell disease are at high risk of invasive bacterial infections, which may lead to serious complications. Preventive measures, including antibiotic prophylaxis and vaccination, have changed the epidemiology of these infections over time. New pneumococcal conjugate vaccines have recently become available, and updated data are needed to better understand which bacteria are currently responsible for invasive infections in this population.\n\nThe aim of this retrospective study is to describe the bacterial distribution of invasive bacterial infections in children with sickle cell disease in France between 2020 and 2025. The results may help improve knowledge of these infections and guide future prevention strategies, including antibiotic management and vaccination policies.",[27,168],"Invasive Bacterial Infections",[87,170,171,172,173],"Pneumococcal conjugate vaccines","Streptococcus pneumoniae","Invasive bacterial infection","Children","2026-06-05",{"date":176,"type":36},"2026-06-10",{"date":178,"type":21},"2026-07-15",{"date":180,"type":21},"2027-07-15",{"name":97,"class":43},31,{"id":184,"slug":185,"hasResults":11,"nctId":186,"briefTitle":187,"officialTitle":188,"acronym":189,"eligibilityCriteria":190,"healthyVolunteers":11,"sex":16,"minAge":191,"maxAge":192,"enrollmentInfo":193,"targetDuration":4,"studyType":165,"phases":4,"briefSummary":195,"conditions":196,"keywords":197,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":201,"lastUpdatePostDateStruct":202,"startDateStruct":204,"completionDateStruct":206,"leadSponsor":208,"locationsCount":210},"100588437","comparing-the-effectiveness-of-matched-related-donor-hematopoietic-stem-cell-transplantation-to-disease-modifying-therapy-in-pediatric-patients-with-sickle-cell-disease-100588437","NCT06941389","Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease","Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.","WeDecide","Inclusion Criteria:\n\n* Pediatric patients aged between 3 and 20.9 years.\n* Children diagnosed with sickle cell Anemia (HB SS or HBSB0 Thalassemia)\n* For the MRD HCT group, children who are candidates for matched related donor hematopoietic stem cell transplantation (MRD HCT).\n* For the NT-DMT group, children who are receiving non-transplant disease-modifying therapies (NT-DMT) for SCD.\n* Participants (or their guardians) must provide informed consent to be part of the study.\n* Participants must be willing to undergo the necessary assessments and follow-up visits over the 3-year study period.\n\nExclusion Criteria:\n\n* Children younger than 3 years or older than 20.9 years.\n* Children who do not have sickle cell anemia or related conditions.\n* For the MRD HCT group, children who are not eligible for the transplant or do not have a matched related donor.\n* Children who are currently enrolled in other clinical trials that might interfere with the WeDecide study.\n* Children who are unable to adhere to the study protocol or follow-up requirements.","3 Years","20 Years",{"count":194,"type":21},480,"The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options.",[27],[198,199,200],"SCD","MRD HCT","NT DMT","2026-06-02",{"date":203,"type":36},"2026-06-04",{"date":205,"type":36},"2024-06-01",{"date":207,"type":21},"2030-10-01",{"name":209,"class":43},"University of Rochester",37,{"id":212,"slug":213,"hasResults":11,"nctId":214,"briefTitle":215,"officialTitle":215,"acronym":216,"eligibilityCriteria":217,"healthyVolunteers":11,"sex":218,"minAge":18,"maxAge":219,"enrollmentInfo":220,"targetDuration":4,"studyType":22,"phases":222,"briefSummary":223,"conditions":224,"keywords":225,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":230,"lastUpdatePostDateStruct":231,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":239},"100567269","phase-4-clinical-and-biomarker-effects-of-depot-medroxyprogesterone-acetate-in-females-with-sickle-cell-disease-100567269","NCT06665997","Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease","SCD Depo","Inclusion Criteria:\n\n1. Provision of signed and dated informed consent form\n2. Female, aged 18-50 years old\n3. Diagnosis of sickle cell disease (SS, SB0,SB+,SC)\n4. Report of at least 1 vaso-occlusive pain episode per month on average in the previous 6 months\n5. At least 1 and no more than 10 medical presentations (e.g. hospitalization, emergencyroom visit, outpatient infusion visit) for vaso-occlusive pain during the past year, unless approved by study PI Andrea Roe\n6. Willing to discontinue any hormonal contraception at the time of enrollment. Washout period of 1 month since last use of all hormonal contraception, and 4 months since most recent administration of depot medroxyprogesterone, is required prior to enrollment in the study.\n7. Have regular menstrual cycles (when not on hormonal contraception) with a usual length of 21 to 35 days.\n8. Stable dose of hydroxyurea and other sickle cell-related medications for the past 6 months\n9. Access to a device with text-messaging capability\n10. Must be able to read and understand English\n11. Willing to comply with study procedures\n\nExclusion Criteria:\n\n1. Chronic inflammatory conditions, such as lupus or inflammatory bowel disease\n2. History of VTE or stroke\n3. Current use of crizanlizumab, voxelotor, or chronic transfusion therapy, including simple transfusion and red cell exchange transfusion, history of hematopoietic stem cell transplantation\n4. Current use of hormonal contraception or the copper intrauterine device\n5. Current pregnancy or pregnancy within the last 6 months\n6. Current lactation\n7. Polycystic ovary syndrome or irregular periods\n8. Blood pressure \\>= 160 systolic or \\>=100 diastolic at screening visit\n9. Has a history or current evidence of any condition, therapy, or other circumstance that in the opinion of the investigator exposes the participant to risk through participating in the trial, may confound the results of the trial, or could interfere with participation for the full duration of the trial.","FEMALE","50 Years",{"count":221,"type":21},65,[137],"This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease.\n\nParticipants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain. They will complete a 3-month \"baseline \"with no use of hormonal contraception, and then a 3-month follow-up after receiving an injection of Depo-Provera. Participants will complete 6 to 7 in-person visits with a urine pregnancy test, blood draw, and surveys, as well as complete remote weekly surveys and monthly home pregnancy tests.",[27,140],[226,227,228,229],"Sickle Cell Disease","Depot Medroxyprogesterone Acetate (Depo-Provera)","Contraception","Vaso-Occlusive Pain","2026-05-26",{"date":232,"type":36},"2026-05-28",{"date":234,"type":36},"2025-06-26",{"date":236,"type":21},"2028-07-31",{"name":238,"class":43},"University of Pennsylvania",2,{"id":241,"slug":242,"hasResults":11,"nctId":243,"briefTitle":244,"officialTitle":244,"acronym":4,"eligibilityCriteria":245,"healthyVolunteers":246,"sex":16,"minAge":18,"maxAge":4,"enrollmentInfo":247,"targetDuration":4,"studyType":22,"phases":248,"briefSummary":249,"conditions":250,"keywords":254,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":257,"lastUpdatePostDateStruct":258,"startDateStruct":260,"completionDateStruct":262,"leadSponsor":264,"locationsCount":70},"100634521","development-of-a-provider-focused-intervention-to-improve-health-outcomes-in-pediatric-sickle-cell-disease-100634521","NCT07540767","Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease","Inclusion Criteria:\n\n* Licensed health care provider (HCP) who provides care to youth with SCD\n* HCP employed by Connecticut Children's, Yale New Haven Children's Hospital, or Children's Hospital of Philadelphia and primary work area is Hematology\u002FOncology\n\nExclusion Criteria:\n\n* HCP who does not provide care to youth with SCD\n* HCP is a medical trainee, not including fellows\n* HCP not employed by CT Children's, Yale New Haven Children's Hospital, or Children's Hospital of Philadelphia\n* Not fluent in English",true,{"count":81,"type":21},[24],"The goal of this interventional study is to learn about the impact of an intervention for health care providers that teaches individuation and perspective-taking (IPT) skills to enhance patient-centered communication in pediatric sickle cell disease (SCD). The main question it aims to answer is:\n\nDoes an intervention that teaches individuation and perspective-taking (IPT) skills to pediatric sickle cell disease (SCD) health care providers (HCPs) enhance patient-centered communication?\n\nResearchers will compare the IPT intervention to a control group who will receive education about SCD pain management to see if the IPT intervention improves patient-centered communication.\n\nParticipants will complete baseline surveys and then be randomly assigned into the intervention or control group. After completing their assigned session (IPT training or education), they will be asked to complete the same surveys as completed at baseline.",[226,251,27,252,253],"Sickle Cell Anemia in Children","Sickle Cell","Sickle Cell Anemia (HbSS)",[255,256],"sickle cell","pediatrics","2026-04-24",{"date":259,"type":36},"2026-04-29",{"date":261,"type":21},"2027-09-01",{"date":263,"type":21},"2030-06-30",{"name":265,"class":43},"Connecticut Children's Medical Center",{"id":267,"slug":268,"hasResults":11,"nctId":269,"briefTitle":270,"officialTitle":271,"acronym":272,"eligibilityCriteria":273,"healthyVolunteers":246,"sex":16,"minAge":18,"maxAge":274,"enrollmentInfo":275,"targetDuration":277,"studyType":165,"phases":4,"briefSummary":278,"conditions":279,"keywords":4,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":282,"lastUpdatePostDateStruct":283,"startDateStruct":285,"completionDateStruct":287,"leadSponsor":289,"locationsCount":239},"100597875","sickle-cell-kidney-biorepository-100597875","NCT07064174","Sickle Cell Kidney Biorepository","Sickle Cell Kidney (SCeK) Biorepository","SCeK","Inclusion Criteria:\n\n1. Age over 18 years,\n2. Estimated glomerular filtration rate greater than or equal to 15 mL\u002Fmin,\n3. Presence of any hemoglobinopathy (will need to be confirmed by hemoglobin electrophoresis or genetic testing),\n4. Controls (absence of hemoglobinopathy) will be subject to review and only selected if demographics are identical to a currently enrolled participant with a hemoglobinopathy.\n\nExclusion Criteria:\n\n1. Age 66 years or older,\n2. Estimated glomerular filtration rate less than 15 mL\u002Fmin or on dialysis,\n3. Active pregnancy (may be enrolled 4 weeks or more after delivery),\n4. Active sickle cell pain episode requiring hospitalization or emergency room visit or pain infusion clinic visit (may be enrolled 2 weeks or more after resolution of severe pain),\n5. Active malignancy on induction or consolidation treatment. Maintenance chemotherapy in remission will be considered,\n6. Prisoners.","65 Years",{"count":276,"type":21},800,"10 Years","Kidney disease is a major cause of illness and death in people with sickle cell disease and sickle cell trait. Despite these concerning facts, we do not (1) have an in-depth understanding of how kidney disease starts in sickle cell disease and sickle cell trait, (2) have detailed insights into why kidney disease is worse in people with sickle cell disease and sickle cell trait, (3) have management options that are tailored to treating or preventing kidney disease in people with sickle cell disease or sickle cell trait.\n\nThe SCeK Biorepository is a specialized, secure repository designed for the collection of blood and urine samples from people with sickle cell disease and sickle cell trait. These samples are connected to detailed medical records, with the sole purpose of allowing researchers to better understand how kidney disease starts and progresses in people with the sickle cell gene. By studying these stored samples (using new tests) together with health information, researchers can find better early warning signs of kidney injury and develop better ways to protect kidney health in people with sickle cell disease and sickle cell trait.",[280,27,281],"Sickle Cell Trait","Chronic Kidney Disease","2026-04-06",{"date":284,"type":36},"2026-04-13",{"date":286,"type":36},"2024-08-22",{"date":288,"type":21},"2049-12",{"name":290,"class":43},"University of Texas Southwestern Medical Center",{"id":292,"slug":293,"hasResults":11,"nctId":294,"briefTitle":295,"officialTitle":295,"acronym":296,"eligibilityCriteria":297,"healthyVolunteers":11,"sex":16,"minAge":298,"maxAge":51,"enrollmentInfo":299,"targetDuration":4,"studyType":22,"phases":301,"briefSummary":302,"conditions":303,"keywords":304,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":308,"lastUpdatePostDateStruct":309,"startDateStruct":311,"completionDateStruct":313,"leadSponsor":315,"locationsCount":70},"100630503","phase-4-integrating-point-of-care-testing-poct-for-newborn-screening-and-early-care-for-sickle-cell-disease-in-yopougon-cte-divoire-100630503","NCT07488520","Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire","STEP","Inclusion Criteria:\n\n1. Children aged 0 - 6 months old identified at selected health facilities within the study area\n2. Babies whose age plus the gestation period reaches 37 weeks or more\n3. Children who did not receive a blood transfusion within the past 90 days\n4. Children whose parents or guardians have consented to participate in the study by signing a consent form\n\nExclusion Criteria:\n\n1. Children already diagnosed with SCD\n2. Children that are participating in another clinical research project\n3. Children that are critically ill and require emergency treatment","0 Months",{"count":300,"type":21},120,[137],"The goal of this clinical trial is to learn if a multifaceted intervention composed of Gazelle-Multispectral sickle cell disease (SCD) point of care testing (POCT) and early initiation of comprehensive SCD care in children with SCD disease aged 0 - 6 months can improve their clinical outcomes.\n\nThe main questions it aims to answer are:\n\n* Does the intervention lower the number of SCD-related illnesses?\n* Does the intervention lower the illness incidence defined as seeking health care at any health facility - with or without treatment - for any episode related to a SCD related illness?\n* Does the intervention lower all-cause death rate (at the end of 1, 2, 3 and 3.5 years of follow-up)?\n\nResearchers will compare the multifaceted intervention results with those of historical data (based on erratic SCD testing and treatment) from the region.\n\nParticipants will undergo a SCD screening test using the Gazelle Multispectral platform and if positive they will undergo confirmatory testing with HemoTypeSC™. Participants with SCD will receive early comprehensive clinical interventions i.e. standard administration of antibacterial and antimalarial prophylaxis, vaccinations for pneumococcal and Haemophilus influenzae type b (Hib), hydroxyurea, parental education about the need for regular and, if necessary, urgent medical care.",[27],[87,305,306,307],"Newborn Screening","Early Care for Sickle Cell Disease","Point of care testing","2026-03-18",{"date":310,"type":36},"2026-03-23",{"date":312,"type":21},"2026-04",{"date":314,"type":21},"2029-12",{"name":316,"class":43},"Swiss Tropical & Public Health Institute",{"id":318,"slug":319,"hasResults":11,"nctId":320,"briefTitle":321,"officialTitle":322,"acronym":323,"eligibilityCriteria":324,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":18,"enrollmentInfo":325,"targetDuration":4,"studyType":22,"phases":326,"briefSummary":327,"conditions":328,"keywords":329,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":332,"lastUpdatePostDateStruct":333,"startDateStruct":335,"completionDateStruct":337,"leadSponsor":339,"locationsCount":70},"100563671","exploratory-study-on-global-reflexology-in-sickle-cell-disease-100563671","NCT06619197","Exploratory Study on Global Reflexology in Sickle Cell Disease","Exploratory Study on Global Reflexology Proposed in the Management of Pain in Children With Sickle Cell Disease in Vaso-occlusive Crisis","REFLEXODREPANO","Inclusion Criteria:\n\n* Patient under 18 years of age\n* Patient suffering from sickle cell disease\n* Patient hospitalized in the UHCD or USC unit of the Women\\'s Mother and Child Hospital of the Hospices Civils de Lyon for the management of a CVO\n* Informed consent signed by at least one holder of parental authority\n* Collection of the patient\\'s assent as soon as his age allows it.\n\nExclusion Criteria:\n\n* Patient with PCA (Patient Controlled Analgesia)\n* Patient with NCA (Nurse Controlled Analgesia)\n* Patient not affiliated to a social security scheme or beneficiaries of a similar scheme.",{"count":81,"type":21},[24],"Sickle cell disease (or sickle cell anemia) is the most common genetic disease in France with 586 children screened in 2019. This chronic disease is characterized by the presence of abnormal Hemoglobin (Hb) S and a deformation of the red blood cells which take the elongated shape of a sickle and become more rigid and more fragile. Sickle cell disease manifests itself among other things by very painful vaso-occlusive crises (VOC) and for some chronic pain.\n\nTheir management is an emergency and often requires hospitalization. Despite analgesic treatment, some patients have persistent pain.\n\nIn 2013, a childcare assistant trained in Canadian global reflexology EMC offered reflexology sessions to 12 sickle cell patients. She observed a relief in all patients with a decrease in the pain score in 8 of them. These sessions seem to show us a double interest: the reduction of the child\\'s pain and the emergence of a technique that can be used by paramedics in the context of their own role.\n\nThe investgators hypothesize that global reflexology is an effective and acceptable complementary technique for pain management in addition to the usual analgesic management in sickle cell children under 18 years of age in CVO. In order to verify our hypothesis, the investigators propose to explore the practice of Canadian global reflexology as an innovative therapeutic option complementary to drug treatments in the hospital management of sickle cell children.",[27],[330,331,30],"Reflexology","Pain","2025-07-04",{"date":334,"type":36},"2025-07-08",{"date":336,"type":36},"2025-06-30",{"date":338,"type":21},"2027-07-30",{"name":340,"class":43},"Hospices Civils de Lyon",{"id":342,"slug":343,"hasResults":11,"nctId":344,"briefTitle":345,"officialTitle":346,"acronym":347,"eligibilityCriteria":348,"healthyVolunteers":246,"sex":16,"minAge":18,"maxAge":4,"enrollmentInfo":349,"targetDuration":4,"studyType":165,"phases":4,"briefSummary":351,"conditions":352,"keywords":353,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":358,"lastUpdatePostDateStruct":359,"startDateStruct":361,"completionDateStruct":363,"leadSponsor":365,"locationsCount":70},"100581440","megakaryocyte-heterogeneity-in-sickle-cell-disease-100581440","NCT06850337","Megakaryocyte Heterogeneity in Sickle Cell Disease","Characterization of Megakaryocytic Subpopulations and the \"Immune\" Phenotype of Platelets of the Sickle Cell Disease Patient","MegaDrep","Inclusion Criteria:\n\n* patients with SS or SC SCD\n* diagnosis of SCD performed by electrophoresis or HPLC in a reference laboratory for hemoglobinopathies\n* patients older than 18 years at inclusion\n* clinically in a steady state at inclusion (without complication in the last month and without transfusion in the three last months)\n* patient followed up for SCD at the sickle cell center of Guadeloupe (University hospital of Guadeloupe, Pointe à Pitre)\n* patients who will provide written informed consent in accordance with the Declaration of Helsinki\n* patients affiliated to national social security\n* the control group (AA subjects) will be patients older than 18 years old who come at the University hospital of Guadeloupe, (Pointe à Pitre) for hip or knee replacement and will do not suffer from chronic disease.\n\nExclusion Criteria:\n\n* patients younger than 18 years old\n* patients with hemoglobinopathy other than SS and SC SCD\n* patients with a transfusion therapy or on bleeding therapy for less than three months\n* patients no affiliated to national social security\n* pregnant or breastfeeding patients",{"count":350,"type":21},100,"Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, painful crisis called vaso-occlusive crisis (VOC) and chronic inflammation. Activated platelets of SCD patients participated to both chronic inflammation and painful VOC. Platelets are anucleated cells from the fragmentation of megakaryocytes in bone marrow.\n\nThe main aim of this study is to characterize the distribution of the different megakaryocyte subpopulations of sickle cell disease patients SS and SC and in particular the \"immune\" megakaryocytes CD148+CD48+ and to compare it with the platelet phenotype.",[27],[30,354,355,356,357],"immune megakaryocytes","platelets","CD48","CD148","2025-05-23",{"date":360,"type":36},"2025-05-25",{"date":362,"type":21},"2025-07",{"date":364,"type":21},"2027-03",{"name":366,"class":43},"Centre Hospitalier Universitaire de la Guadeloupe",{"id":368,"slug":369,"hasResults":11,"nctId":370,"briefTitle":371,"officialTitle":371,"acronym":372,"eligibilityCriteria":373,"healthyVolunteers":11,"sex":16,"minAge":18,"maxAge":4,"enrollmentInfo":374,"targetDuration":4,"studyType":165,"phases":4,"briefSummary":376,"conditions":377,"keywords":378,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":380,"lastUpdatePostDateStruct":381,"startDateStruct":383,"completionDateStruct":385,"leadSponsor":387,"locationsCount":70},"100584328","phenotypic-and-transcriptomic-description-of-megakaryocytes-in-sickle-cell-patient-100584328","NCT06887907","Phenotypic and Transcriptomic Description of Megakaryocytes in Sickle Cell Patient","MEGADREP","Inclusion Criteria:\n\n* Sickle cell disease SS or S-béta° thalassemia\n* Patient at steady state since at least 1 year or at steady state (without crisis), or during vaso-occlusive crisis or during acute chest syndrome\n* Age \\> 18 years old\n\nExclusion Criteria:\n\n* Patient objects to take part in the study Hematologic disorder (leukemia, myeloma, myelodysplasic syndrome, myeloproliferative syndrome)\n* Immune thrombocytopenia, Immunosuppressive or anti-inflammatory (biotherapies, corticosteroids, non steroidal anti-inflammatories drugs) Page 12 sur 23\n* Anti-platelets agents\n* Red blood cell exchange or transfusion \\\u003C 3 months",{"count":375,"type":21},40,"Sickle cell disease is the most common inherited blood disorder in the world. Chronic hemolysis induces platelet activation and chronic inflammation. Platelets and megakaryocyte, as medullar platelets precursors, are known to play a role in innate immunity. Little is known about the role of megakaryocytes at basal state and during acute complication in sickle cell disease patients. The aim of this study is to evaluate the role of megakaryocytes in sickle cell disease.",[27],[379,226],"Megakaryocyte","2025-03-14",{"date":382,"type":36},"2025-03-20",{"date":384,"type":36},"2025-03-13",{"date":386,"type":21},"2028-04-01",{"name":388,"class":43},"University Hospital, Toulouse",{"id":390,"slug":391,"hasResults":11,"nctId":392,"briefTitle":393,"officialTitle":393,"acronym":394,"eligibilityCriteria":395,"healthyVolunteers":11,"sex":16,"minAge":396,"maxAge":4,"enrollmentInfo":397,"targetDuration":4,"studyType":165,"phases":4,"briefSummary":399,"conditions":400,"keywords":401,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":405,"lastUpdatePostDateStruct":406,"startDateStruct":408,"completionDateStruct":410,"leadSponsor":412,"locationsCount":239},"100563663","relationship-between-biological-phenotype-clinical-severity-of-sickle-cell-disease-and-blood-coagulation-100563663","NCT06619093","Relationship Between Biological Phenotype, Clinical Severity of Sickle Cell Disease, and Blood Coagulation","DREPA COAG","Inclusion Criteria:\n\n* Aged 8 years or older\n* Under clinical follow-up for a diagnosis of sickle cell disease, specifically genotypes S\u002FS, S\u002Fbeta0, or S\u002FC\n* Patient covered by a social security or equivalent health insurance plan\n* Collection of the non-opposition for adults\n* Information of the minor and collection of the non-opposition from both parents\n\nExclusion Criteria:\n\n* Patient who has undergone a transfusion or therapeutic phlebotomy within the 3 months prior to inclusion\n* Patient participating in another interventional research protocol that may interfere with the present protocol (at the investigator\\'s discretion)\n* Patient under guardianship, curatorship, or legal protection\n* Patient subject to a legal protection measure\n* Person admitted to a health or social care institution for purposes other than research","8 Years",{"count":398,"type":21},200,"Sickle cell disease is characterized by chronic hemolytic anemia and blood rheological alterations. In addition, blood coagulation abnormalities have been reported in patients with sickle cell disease and hemolysis-derived products could be involved. The investigators hypothesized that patients with sickle cell disease and severe hemolysis (Lactate Dehydrogenase level \\> 484 IU\u002FL) could have an increased risk of hypercoagulable state and subsequent thromboembolic complications.",[27],[402,403,404,87],"Hemolysis","Coagulation","Red blood cell","2025-01-14",{"date":407,"type":36},"2025-01-15",{"date":409,"type":21},"2025-02-01",{"date":411,"type":21},"2028-02-01",{"name":340,"class":43}]