[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100299122":3},{"organization":4,"armGroups":7,"interventions":15,"overallOfficials":27,"centralContacts":32,"locations":41,"responsibleParty":62,"collaborators":21,"id":64,"slug":21,"hasResults":65,"nctId":66,"briefTitle":67,"officialTitle":68,"acronym":21,"eligibilityCriteria":69,"healthyVolunteers":65,"sex":70,"minAge":71,"maxAge":72,"enrollmentInfo":73,"targetDuration":21,"studyType":76,"phases":77,"briefSummary":80,"conditions":81,"keywords":85,"overallStatus":44,"whyStopped":21,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":97},{"fullName":5,"class":6},"National Institutes of Health Clinical Center (CC)","NIH",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"1","EXPERIMENTAL","CordIn is a cryopreserved stem\u002Fprogenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.",[13,14],"Biological: Omidubicel (former CordIn)","Device: Miltenyi CliniMACS(R) CD34 Reagent System",[16,22],{"type":17,"name":18,"description":19,"armGroupLabels":20,"otherNames":21},"BIOLOGICAL","Omidubicel (former CordIn)","Stem\u002Fprogenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic umbilical\u002Funrelated cord blood (UCB) cells. CordIn(TM) comprises: 1) cord blood-derived ex vivo expanded CD133+ cells (CordIn (TM) cultured fraction (CF)); and 2) the non-cultured cell fraction (CD133-) of the same CBU (CordIn(TM) Non-cultured Fraction (NF)). Both fractions, i.e. CordIn(TM) CF and CordIn(TM) NF are kept frozen until they are infused on the day of transplantation.",[9],null,{"type":23,"name":24,"description":25,"armGroupLabels":26,"otherNames":21},"DEVICE","Miltenyi CliniMACS(R) CD34 Reagent System","The CliniMACS CD34 Reagent System is a medical device system that is designed for the in vitro enrichment of CD34+ target cells from heterogeneous hematologic cell populations. The process of CD34+ cell selection results in simultaneous passive depletion of donor lymphocytes, potentially obviating the need for immunosuppressive drugs to prevent GVHD.",[9],[28],{"name":29,"affiliation":30,"role":31},"Richard W Childs, M.D.","National Heart, Lung, and Blood Institute (NHLBI)","PRINCIPAL_INVESTIGATOR",[33,38],{"name":34,"role":35,"phone":36,"phoneExt":21,"email":37},"Melissa M Spencer, R.N.","CONTACT","(301) 402-5609","melissa.spencer@nih.gov",{"name":29,"role":35,"phone":39,"phoneExt":21,"email":40},"(301) 768-9433","childsr@nhlbi.nih.gov",[42],{"facility":43,"status":44,"city":45,"state":46,"zip":47,"country":48,"countryCode":49,"cosmosGeoPoint":50,"geoPoint":55,"contacts":56},"National Institutes of Health Clinical Center","RECRUITING","Bethesda","Maryland","20892","United States","US",{"type":51,"coordinates":52},"Point",[53,54],-77.10026,38.98067,{"lat":54,"lon":53},[57],{"name":58,"role":35,"phone":59,"phoneExt":60,"email":61},"For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)","800-411-1222","TTY8664111010","prpl@cc.nih.gov",{"type":63,"investigatorFullName":21,"investigatorTitle":21,"investigatorAffiliation":21,"oldNameTitle":21,"oldOrganization":21},"SPONSOR","100299122",false,"NCT03173937","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count \\\u003C=1000 cells\u002F uL or for patients receiving granulocyte transfusions, absolute neutrophil count \\\u003C=1000 cells\u002F uL before beginning granulocyte transfusions).\n\nOR\n\nHistory of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C 5% myeloblasts and \\\u003C 30% of cellularity in the bone marrow on screening morphologic analysis.\n\n-Intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n\nIdentification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>=5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>=9\u002F10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn(TM) unit has been rejected or b) umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the CordIn(TM) unit has been rejected.\n\n-Availability of at least one \\>=4\u002F8 HLA matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).\n\nThe cord blood unit must contain a minimum TNC of at least 1.8 x 10\\^9 and at least 1.5x10\\^7\u002Fkg TNC and at least 8 x 10\\^6 CD34+ cells (all doses prior to thawing).\n\nException: Cord units containing at least 8 x 10\\^6 CD34+ cells but less than 1.8 x 10\\^9 TNC may be eligible for use on this trial if\n\n1. the pre-expansion CD34\u002Fkg recipient cell number is at least 1.5 x 10\\^5 \u002Fkg\n\n   AND\n2. approval for use of this cord unit for expansion is granted by Gamida Cell.\n3. the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.\n\n   * The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.\n   * Ages 4-60 years inclusive.\n   * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C 40% (evaluated by ECHO).\n* Transaminases \\> 5x upper limit of normal.\n* Serum bilirubin \\>4 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n* Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.\n* Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.","ALL","4 Years","75 Years",{"count":74,"type":75},37,"ESTIMATED","INTERVENTIONAL",[78,79],"PHASE1","PHASE2","Background:\n\nSevere aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective.\n\nObjective:\n\nTo test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.\n\nEligibility:\n\nRecipients ages 4-60 with SAA or MDS\n\nDonors ages 4-75\n\nDesign:\n\nRecipients will be screened with:\n\n* Blood, lung, and heart tests\n* Bone marrow biopsy\n* CT scan\n\nRecipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose.\n\nRecipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits:\n\n* First 3-4 months: 1-2 times weekly\n* Then every 6 months for 5 years\n\nDonors will be screened with:\n\n* Physical exam\n* Medical history\n* Blood tests\n\nDonors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein.\n\nDonors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.",[82,83,84],"Severe Aplastic Anemia","Hypo-Plastic MDS","Myelodysplastic Syndrome (MDS)",[86,87],"Haploidentical","Nonmyeloablative","2026-07-01",{"date":90,"type":91},"2026-07-02","ACTUAL",{"date":93,"type":91},"2017-06-13",{"date":95,"type":75},"2032-03-01",{"name":30,"class":6},1]