[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100335893":3},{"organization":4,"armGroups":7,"interventions":20,"overallOfficials":56,"centralContacts":60,"locations":69,"responsibleParty":87,"collaborators":26,"id":91,"slug":92,"hasResults":93,"nctId":94,"briefTitle":95,"officialTitle":96,"acronym":26,"eligibilityCriteria":97,"healthyVolunteers":93,"sex":98,"minAge":99,"maxAge":100,"enrollmentInfo":101,"targetDuration":26,"studyType":104,"phases":105,"briefSummary":108,"conditions":109,"keywords":113,"overallStatus":72,"whyStopped":26,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":129,"completionDateStruct":131,"leadSponsor":133,"locationsCount":134},{"fullName":5,"class":6},"University of Pittsburgh","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Hematopoietic Stem Cell Transplantation","EXPERIMENTAL","All patients will receive a CD3+\u002FCD19+ depleted stem cell transplant. In this study, the investigators will use HLA mismatched unrelated or haploidentical related donor peripheral blood stem cells. Prior to transplantation, the marrow (90-95%) will be negatively selected for CD3\u002FCD19 using the ClinicMACs® depletion device. The remaining (5-10%) will undergo CD45+RA+ depletion and be frozen for future use as an immune boost.\n\nSubjects will undergo hematopoietic stem cell transplant utilizing CD3+\u002FCD19+ depleted cells following conditioning therapy.",[13,14,15,16,17,18,19],"Biological: CD3\u002FCD19 depleted leukocytes","Biological: CD45RA depleted leukocytes","Drug: Hydroxyurea","Drug: Rituximab","Drug: Alemtuzumab","Drug: Fludarabine","Drug: Thiotepa",[21,27,31,38,43,48,53],{"type":22,"name":23,"description":24,"armGroupLabels":25,"otherNames":26},"BIOLOGICAL","CD3\u002FCD19 depleted leukocytes","Negative selection for CD3+\u002FCD19+ cells will be performed on the CliniMACS® depletion device.",[9],null,{"type":22,"name":28,"description":29,"armGroupLabels":30,"otherNames":26},"CD45RA depleted leukocytes","Negative selection for CD45RA will be performed on the CliniMACS® depletion device.",[9],{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":36},"DRUG","Hydroxyurea","Sickle Cell Disease Conditioning",[9],[37],"HU, Hydrea",{"type":32,"name":39,"description":34,"armGroupLabels":40,"otherNames":41},"Rituximab",[9],[42],"Rituxan",{"type":32,"name":44,"description":34,"armGroupLabels":45,"otherNames":46},"Alemtuzumab",[9],[47],"Campath-1H",{"type":32,"name":49,"description":34,"armGroupLabels":50,"otherNames":51},"Fludarabine",[9],[52],"Fludara",{"type":32,"name":54,"description":34,"armGroupLabels":55,"otherNames":26},"Thiotepa",[9],[57],{"name":58,"affiliation":5,"role":59},"Paul Szabolcs, MD","PRINCIPAL_INVESTIGATOR",[61,65],{"name":58,"role":62,"phone":63,"phoneExt":26,"email":64},"CONTACT","412-692-6225","paul.szabolcs@chp.edu",{"name":66,"role":62,"phone":67,"phoneExt":26,"email":68},"Shawna McIntyre, RN","412-692-5552","mcintyresm@upmc.edu",[70],{"facility":71,"status":72,"city":73,"state":74,"zip":75,"country":76,"countryCode":77,"cosmosGeoPoint":78,"geoPoint":83,"contacts":84},"Children's Hospital of Pittsburgh of UPMC","RECRUITING","Pittsburgh","Pennsylvania","15224","United States","US",{"type":79,"coordinates":80},"Point",[81,82],-79.99589,40.44062,{"lat":82,"lon":81},[85],{"name":86,"role":59,"phone":26,"phoneExt":26,"email":26},"Paul Pszabolcs, MD",{"type":88,"investigatorFullName":89,"investigatorTitle":90,"investigatorAffiliation":5,"oldNameTitle":26,"oldOrganization":26},"SPONSOR_INVESTIGATOR","Paul Szabolcs","Chief, Division of Blood and Marrow Transplantation and Cellular Therapy","100335893","phase-1-t-cell-depleted-alternative-donor-bone-marrow-transplant-for-sickle-cell-disease-scd-and-other-anemias-100335893",false,"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.","ALL","5 Years","40 Years",{"count":102,"type":103},5,"ESTIMATED","INTERVENTIONAL",[106,107],"PHASE1","PHASE2","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.",[110,111,112],"Sickle Cell Anemia","Beta-thalassemia Major","Diamond-blackfan Anemia",[114,115,116,117,118,119,120,121,122,123,124],"Sickle Cell","Diamond-Blackfan","Beta-thalassemia","Anemia","Stem cell transplantation","Unrelated Donor","haploidentical","hematopoietic stem cell transplant (HSCT)","bone marrow transplant (BMT)","mismatched","t-cell depletion","2025-08-07",{"date":127,"type":128},"2025-08-12","ACTUAL",{"date":130,"type":128},"2018-08-02",{"date":132,"type":103},"2027-08-01",{"name":89,"class":6},1]