[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"sickle-cell-disorders\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:sickle-cell-disorders":32},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,51],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":34,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":40,"startDateStruct":43,"completionDateStruct":45,"leadSponsor":47,"locationsCount":50},"100508720","phase-1-study-to-evaluate-the-safety-and-tolerability-of-escalating-doses-of-fostamatinib-in-subjects-with-stable-sickle-cell-disease-100508720",false,"NCT05904093","Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease","A Phase I Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease","* INCLUSION CRITERIA:\n\nSubjects will enroll onto the study and undergo screening. Subjects who do not meet any of the following criteria during screening will not receive the study intervention but will be counted toward study accrual. Screen failures may be rescreened at a later time. In order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n1. Have provided signed written informed consent prior to performing any study procedure, including screening procedures.\n2. Age between 18-65 years\n3. Unequivocal diagnosis of SCA (HbSS or HbSBeta\\^0) confirmed by hemoglobin electrophoresis performed on patients at least 60 days after a blood transfusion if previously transfused.\n4. No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC)\n5. Have adequate organ function, as defined by:\n\n   1. Serum aspartate aminotransferase (AST) \\\u003C=1.5 x Upper Limit of Normal (ULN) (unless the increased AST is assessed by the Investigator as due to hemolysis) and alanine aminotransferase (ALT) \\\u003C=1.5 x ULN.\n   2. Absolute neutrophil count \\>=1.5 x 10\\^9\u002FL.\n   3. Hemoglobin \\>= 7 g\u002FdL\n   4. Platelet count \\>=100 x 10\\^9\u002FL.\n6. If on hydroxyurea, participant must have been on stable dose of hydroxyurea (defined as a stable dose for at least 3 months and inclusive of dose modifications for hematological toxicity per PI discretion) prior to signing consent.\n7. For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception).\n8. For women of reproductive potential as well as men and their partners who are women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use 2 effective forms of contraception from the time of giving informed consent, during the study, and for 28 days (both men and women) following the last dose of study treatment. An effective form of contraception is defined as hormonal oral contraceptives, injectables, patches, intrauterine or subdermal contraceptive implants, and barrier methods.\n9. Be willing to comply with all study procedures for the duration of the study.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n1. Pain crisis requiring parenteral treatment within 14 days of signing consent.\n2. Have a significant medical condition that confers an unacceptable risk to participating in the study, and\u002For that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:\n\n   1. History of neutropenia (benign ethnic neutropenia and\u002For acquired neutropenia related to drug suppression by hydroxyurea and\u002For cyclic hematopoiesis are permitted).\n   2. History of posterior reversible encephalopathy syndrome (PRES)\n   3. History of poorly controlled hypertension (defined as systolic blood pressure \\>=130 mmHg or average diastolic blood pressure \\>=90 mmHg based on an average of 3 blood pressure readings despite adequate antihypertensive therapy) unless controlled for \\>90 days prior to enrollment.\n   4. Active viral infection as evidenced by testing positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody (ab) with signs of active hepatitis B or C virus infection. If the subject is positive for HCV Ab, a reverse transcriptase-polymerase chain reaction test will be conducted. Subjects with hepatitis C may be rescreened after receiving appropriate hepatitis C treatment.\n   5. History of drug-induced cholestatic hepatitis.\n   6. History of any primary malignancy.\n   7. Testing positive for human immunodeficiency virus 1 or 2 Ab with evidence for ongoing active infection (i.e., CD 4 count \\\u003C400\u002Fmicroliter and viral load \\>100,000 copies\u002Fml) on antiretroviral therapy.\n   8. Current or recent history of psychiatric disorder that, in the opinion of the Investigator or Medical Monitor, could compromise the ability of the subject to cooperate with study visits and procedures.\n   9. Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo.\n   10. Use of newly approved SCD therapy (L-glutamine, voxelotor or crizanlizumab) is NOT permitted on this study. Subjects who have received newly approved SCD therapy in the 7 days prior to signing consent will be excluded.\n   11. Having had a prior bone marrow or stem cell transplant.\n   12. Currently pregnant or lactating.\n   13. Currently receiving strong inhibitors of CYP3A4\u002F5 that have not been stopped for \\>=5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong inducers of CYP3A4 that have not been stopped for \\>=28 days or a time frame equivalent to 5 half-lives (whichever is longer), prior to signing consent. SCD patients that are receiving treatment with CYP3A4 substrate drugs, some BCRP substrate drugs (eg. rosuvastatin), and some P-glycoprotein substrate drugs (eg. Digoxin) are excluded from the study.\n   14. Currently receiving erythropoiesis stimulating agents.",true,"ALL","18 Years","65 Years",{"count":21,"type":22},25,"ESTIMATED","INTERVENTIONAL",[25],"PHASE1","Background:\n\nSickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves. People with SCD are also at increased risk of forming blood clots in the veins and lungs, but the standard treatments for these clots can cause increased bleeding in people with SCD. Better treatments are needed.\n\nObjective:\n\nTo test a drug (fostamatinib) in people with SCD.\n\nEligibility:\n\nPeople aged 18 to 65 with SCD.\n\nDesign:\n\nParticipants will have 6 clinic visits over 12 weeks. Each visit will be 2 to 3 hours.\n\nParticipants will be screened. They will have a physical exam with blood tests. They will tell the researchers about the medications they take.\n\nFostamatinib is a tablet taken by mouth. Participants will take the drug at home, twice a day, for up to 6 weeks.\n\nParticipants will have a clinic visit every 2 weeks while they are taking the drug. At each visit they will have a physical exam with blood tests. They will talk about any side effects the drug may be causing. If they are tolerating the drug well after the first 2 weeks, they may begin taking a higher dose.\n\nParticipants will have a final visit 4 weeks after they stop taking the drug. They will have a physical exam and blood tests; they will be checked for any side effects of the drug.",[28,29,30,31,32,33],"Sickle Cell Disease","Hb-SS Disease","Hemoglobin S","Disease Sickle Cell Anemia","Sickle Cell Disorders","Hemoglobin Beta Thalassemia Disease",[35,36,37],"Sickle Cell","Tyrosine Kinase Inhibitor","Fostamatinib","RECRUITING","2026-05-27",{"date":41,"type":42},"2026-05-28","ACTUAL",{"date":44,"type":42},"2024-12-18",{"date":46,"type":22},"2027-05-14",{"name":48,"class":49},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":52,"slug":53,"hasResults":11,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":4,"eligibilityCriteria":57,"healthyVolunteers":11,"sex":17,"minAge":58,"maxAge":59,"enrollmentInfo":60,"targetDuration":4,"studyType":23,"phases":62,"briefSummary":64,"conditions":65,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":75,"leadSponsor":77,"locationsCount":80},"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","35 Years",{"count":61,"type":22},212,[63],"PHASE2","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.",[66,67,68,32,69,28],"HbS Disease","Hemoglobin S Disease","Sickle Cell Anemia","Sickling Disorder Due to Hemoglobin S","2024-05-10",{"date":72,"type":42},"2024-05-13",{"date":74,"type":42},"2021-06-30",{"date":76,"type":22},"2030-03-31",{"name":78,"class":79},"University of Regensburg","OTHER",9]