[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"severe-marrow-failure\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:severe-marrow-failure":28},{"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":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":31,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100220190","phase-2-hsct-for-patients-with-fanconi-anemia-using-risk-adjusted-chemotherapy-100220190",false,"NCT02143830","HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy","A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine","RAFA","Inclusion Criteria:\n\n* Patients must have a diagnosis of Fanconi anemia\n* Patients must have one of the following hematologic diagnoses:\n\n  1. Severe Aplastic Anemia (SAA), with bone marrow cellularity of \\\u003C25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features:\n\n     1. Platelet count \\\u003C20 x 109\u002FL or platelet transfusion dependence\\*\n     2. ANC \\\u003C1000 x 109\u002FL\n     3. Hgb \\\u003C8 gm\u002Fdl or red cell transfusion dependence\\*\n  2. Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification\n  3. Acute Myelogenous Leukemia (untreated, in remission or with refractory or relapsed disease)\n* Donors will be either human leukocyte antigen (HLA) compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor).\n* Patients and donors may be of either gender or any ethnic background.\n* Patients must have a Karnofsky adult, or Lansky pediatric performance scale status \\> 70%.\n* Patients must have adequate physical function measured by:\n\n  1. Cardiac: asymptomatic or if symptomatic then 1) left ventricular ejection fraction (LVEF) at rest must be \\> 50% and must improve with exercise or 2) Shortening Fraction \\> 29%\n  2. Hepatic: \\\u003C 5 x upper limit of normal (ULN) alanine transaminase (ALT) and \\\u003C 2.0 mg\u002Fdl total serum bilirubin.\n  3. Renal: serum creatinine \\\u003C1.5 mg\u002Fdl or if serum creatinine is outside the normal range, then CrCl \\> 50 ml\u002Fmin\u002F1.73 m2\n  4. Pulmonary: asymptomatic or if symptomatic, DLCO \\> 50% of predicted\n* Each patient must be willing to participate as a research subject and must sign an informed consent form.\n* Female patients and donors must not be pregnant or breastfeeding at the time of signing consent. Women must be willing to undergo a pregnancy test prior to transplant and avoid becoming pregnant while on study.\n\nExclusion Criteria:\n\n* Active CNS leukemia\n* Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.\n* Active uncontrolled viral, bacterial or fungal infection\n* Patient seropositive for HIV-I\u002FII; HTLV -I\u002FII","ALL","3 Months",{"count":20,"type":21},70,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.",[27,28,29,30],"Fanconi Anemia","Severe Marrow Failure","Myelodysplastic Syndrome (MDS)","Acute Myelogenous Leukemia (AML)",[32,33,34,35,36,37,38],"marrow aplasia","cytopenia","myelodysplasia","AML","bone marrow transplant","cytoreductive regimen","T-cell reduction","RECRUITING","2025-11-10",{"date":42,"type":43},"2025-11-12","ACTUAL",{"date":45,"type":4},"2014-04",{"date":47,"type":21},"2028-12",{"name":49,"class":50},"Children's Hospital Medical Center, Cincinnati","OTHER",3]