[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hypo-plastic-myelodysplastic-syndrome-mds\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hypo-plastic-myelodysplastic-syndrome-mds":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":5},"100325717","phase-2-haplo-identical-transplantation-for-severe-aplastic-anemia-hypo-plastic-mds-and-pnh-using-peripheral-blood-stem-cells-and-post-transplant-cyclophosphamide-for-gvhd-prophylaxis-100325717",false,"NCT03520647","Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis","* 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 less than or equal to 1000 cells\u002F microL or for patients receiving granulocyte transfusions, absolute neutrophil count less than or equal to 1000 cells\u002FmicroL before beginning granulocyte transfusions).\n\nOR\n\n--History 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) \\\u003C5% myeloblasts and \\\u003C30% of cellularity in the bone marrow on screening morphologic analysis.\n\nOR\n\n* PNH that is either refractory to treatment with eculizumab\u002Fravulizumab or occurs in patients who don t have access to treatment with eculizumab associated with either a) life- threatening thrombosis and\u002For b) cytopenia associated with transfusion dependence and\u002For c) recurrent and debilitating hemolytic crisis.\n\n  * Subjects with severe aplastic anemia, hypoplastic MDS or PNH with associated bone marrow failure syndromes who have 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  * Availability of at least one HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) to serve as a stem cell donor for the allogeneic transplant.\n  * Availability of a backup stem cell source in the event of graft rejection:\n* at least one additional haploidentical related 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 haplo-transplant has been rejected.\n* umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the haplo-transplant has been rejected\n\n  * The patient does not have any HLA antibodies detectable against any of the mismatched HLA alleles expressed by the haplo-donor.\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* The patient is deemed to be a candidate for a 12\u002F12 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).\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 \\\u003C40% (evaluated by ECHO)\n* Transaminases \\> 5x upper limit of normal.\n* Direct bilirubin \\>3 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm\\^2 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\nINCLUSION CRITERIA - DONOR:\n\nDonor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA - RELATED DONOR:\n\nNone","ALL","4 Years","75 Years",{"count":19,"type":20},56,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","Background:\n\nSevere aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria\n\n(PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants.\n\nObjectives:\n\nTo see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy.\n\nEligibility:\n\nRecipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75\n\nDesign:\n\nRecipients will have:\n\n* Blood, urine, heart, and lung tests\n* Scans\n* Bone marrow sample\n\nRecipients will need a caregiver for several months. They may make fertility plans and a power of attorney.\n\nDonors will have blood and tissue tests, then injections to boost stem cells for 5-7 days.\n\nDonors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg.\n\nIn the hospital for about 1 month, recipients will have:\n\n* Central line inserted in the neck or chest\n* Medicines for side effects\n* Chemotherapy over 8 days and radiation 1 time\n* Stem cell transplant over 4 hours\n\nUp to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests.\n\nAt day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.",[26,27,28],"Severe Aplastic Anemia (SAA)","Hypo-Plastic Myelodysplastic Syndrome (MDS)","Paroxysmal Nocturnal Hemoglobinuria (PNH)",[30,31],"Haploidentical CD34+cells","Non-Myeloablative","RECRUITING","2026-07-01",{"date":35,"type":36},"2026-07-02","ACTUAL",{"date":38,"type":36},"2019-02-19",{"date":40,"type":20},"2028-06-01",{"name":42,"class":43},"National Heart, Lung, and Blood Institute (NHLBI)","NIH"]