[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100220190":3},{"organization":4,"armGroups":7,"interventions":27,"overallOfficials":68,"centralContacts":73,"locations":83,"responsibleParty":127,"collaborators":129,"id":133,"slug":134,"hasResults":135,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":139,"eligibilityCriteria":140,"healthyVolunteers":135,"sex":141,"minAge":142,"maxAge":67,"enrollmentInfo":143,"targetDuration":67,"studyType":146,"phases":147,"briefSummary":149,"conditions":150,"keywords":155,"overallStatus":99,"whyStopped":67,"lastUpdateSubmitDate":163,"lastUpdatePostDateStruct":164,"startDateStruct":167,"completionDateStruct":169,"leadSponsor":171,"locationsCount":172},{"fullName":5,"class":6},"Children's Hospital Medical Center, Cincinnati","OTHER",[8,19,23],{"label":9,"type":10,"description":11,"interventionNames":12},"Arm A: Good Risk Patients","EXPERIMENTAL","Patients 18 years old or younger with marrow aplasia or single lineage cytopenias will be receive intravenous busulfan (4 doses x 2 days), cyclophosphamide (4 doses x 4 days) and fludarabine (4 doses x 4 days), as used in our most current study that led to \\> 90% survival rates . Busulfan pharmacokinetics will not be used. All patients will receive rabbit ATG (4 doses x 4 days) prior to and granulocyte-colony stimulating factor (G-CSF) after transplant to promote engraftment. Cyclosporine will not be used for GVHD prophylaxis. The source of the stem cells for all patients will be peripheral blood stem cells mobilized by treatment of the donor with G-CSF. T-cell depletion will be performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device). Enrollment on this arm will include up to 50 patients.",[13,14,15,16,17,18],"Drug: Busulfan","Drug: Cyclophosphamide","Drug: Fludarabine","Drug: rabbit ATG","Drug: G-CSF","Biological: Peripheral blood stem cell",{"label":20,"type":10,"description":21,"interventionNames":22},"Arm B: Intermediate Risk Patients","Patients 18 years old or younger with MDS or AML will receive intravenous busulfan (4 doses x 2 days), cyclophosphamide (4 doses x 4 days) and fludarabine (4 doses x 4 days). Busulfan pharmacokinetics will be used in this arm, as the dose of busulfan is higher. All patients will receive rabbit ATG (thymoglobulin) (4 doses x 4 days) prior to and G-CSF post transplant to promote engraftment. Cyclosporine will not be used for prophylaxis against GVHD. The source of the stem cells for all patients will be peripheral blood stem cells induced and mobilized by treatment of the donor. T-cell will be performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device).\n\nThe maximum number of patients enrolled in this arm will be 10.",[13,14,15,16,17,18],{"label":24,"type":10,"description":25,"interventionNames":26},"Arm C: High Risk Patients","Patients 19 years old or older with marrow aplasia or MDS or AML will receive intravenous busulfan (4 doses x 2 days), cyclophosphamide (4 doses x 4 days) and fludarabine (4 doses x 4 days). In this study, we will test whether outcomes can be improved, yet engraftment maintained, with a slightly reduced dose of busulfan. Patients will receive rabbit ATG (4 doses x 4 days) prior to and G-CSF post transplant to promote engraftment. Cyclosporine will not be used for GVHD prophylaxis. The source of the stem cells will be peripheral blood stem cells collected from donors treated with G-CSF. T-cell depletion will be performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device). The maximum number of patients enrolled will be 10.",[13,14,15,16,17,18],[28,36,42,48,54,62],{"type":29,"name":30,"description":31,"armGroupLabels":32,"otherNames":33},"DRUG","Busulfan","A standard dose of busulfan, associated with excellent outcomes in our previous trial will be used for young patients with marrow aplasia (arm A).\n\nA higher dose of busulfan will be used in younger patients with MDS and AML (arm B) to maximize disease control.\n\nA lower dose of busulfan will be used in older patients (arm C) to minimize toxicity.",[9,20,24],[34,35],"Myleran","Busulfex IV",{"type":29,"name":37,"description":38,"armGroupLabels":39,"otherNames":40},"Cyclophosphamide","Arms A, B and C - Cytoxan will be given as a 1-2 hour infusion for 4 days. The dose will be adjusted according to patients ideal body weight for obese patients.",[9,20,24],[41],"Cytoxan",{"type":29,"name":43,"description":44,"armGroupLabels":45,"otherNames":46},"Fludarabine","Arms A, B and C - Fludarabine will be given IV over 30 minutes daily for 4 days. The dose will be adjusted according to renal function according to Institutional guidelines.",[9,20,24],[47],"Fludara",{"type":29,"name":49,"description":50,"armGroupLabels":51,"otherNames":52},"rabbit ATG","Arms A, B and C - 4 doses will be given prior to transplant to promote engraftment.",[9,20,24],[53],"thymoglobulin",{"type":29,"name":55,"description":56,"armGroupLabels":57,"otherNames":58},"G-CSF","All patients will also receive G-CSF post-transplant to foster engraftment.",[9,20,24],[59,60,61],"Granulocyte colony-stimulating factor","filgrastim","neupogen",{"type":63,"name":64,"description":65,"armGroupLabels":66,"otherNames":67},"BIOLOGICAL","Peripheral blood stem cell","The source of stem cells for all patients will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 4-6 days. T-cell depletion will be uniformly performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device).",[9,20,24],null,[69],{"name":70,"affiliation":71,"role":72},"Parinda Mehta, MD","CCHMC","PRINCIPAL_INVESTIGATOR",[74,79],{"name":75,"role":76,"phone":77,"phoneExt":67,"email":78},"Jamie Wilhelm","CONTACT","(513)803-1102","Jamie.Wilhelm@cchmc.org",{"name":80,"role":76,"phone":81,"phoneExt":67,"email":82},"Sara Loveless, RN","(513)803-7656","Sara.Loveless@cchmc.org",[84,97,110],{"facility":85,"status":86,"city":87,"state":87,"zip":88,"country":89,"countryCode":90,"cosmosGeoPoint":91,"geoPoint":96,"contacts":67},"Memorial Sloan Kettering Cancer Center","COMPLETED","New York","10174","United States","US",{"type":92,"coordinates":93},"Point",[94,95],-74.00597,40.71427,{"lat":95,"lon":94},{"facility":98,"status":99,"city":100,"state":101,"zip":102,"country":89,"countryCode":90,"cosmosGeoPoint":103,"geoPoint":107,"contacts":108},"Cincinnati Children's Hospital Medical Center","RECRUITING","Cincinnati","Ohio","45229",{"type":92,"coordinates":104},[105,106],-84.51439,39.12711,{"lat":106,"lon":105},[109],{"name":70,"role":72,"phone":67,"phoneExt":67,"email":67},{"facility":111,"status":99,"city":112,"state":113,"zip":114,"country":89,"countryCode":90,"cosmosGeoPoint":115,"geoPoint":119,"contacts":120},"Fred Hutchinson Cancer Research Center","Seattle","Washington","98109",{"type":92,"coordinates":116},[117,118],-122.33207,47.60621,{"lat":118,"lon":117},[121,125],{"name":122,"role":76,"phone":123,"phoneExt":67,"email":124},"Sheri Ballard","206-667-4222","sballard@fredhutch.org",{"name":126,"role":72,"phone":67,"phoneExt":67,"email":67},"K. Scott Baker, MD",{"type":128,"investigatorFullName":67,"investigatorTitle":67,"investigatorAffiliation":67,"oldNameTitle":67,"oldOrganization":67},"SPONSOR",[130,131],{"name":85,"class":6},{"name":132,"class":6},"Fred Hutchinson Cancer Center","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":144,"type":145},70,"ESTIMATED","INTERVENTIONAL",[148],"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.",[151,152,153,154],"Fanconi Anemia","Severe Marrow Failure","Myelodysplastic Syndrome (MDS)","Acute Myelogenous Leukemia (AML)",[156,157,158,159,160,161,162],"marrow aplasia","cytopenia","myelodysplasia","AML","bone marrow transplant","cytoreductive regimen","T-cell reduction","2025-11-10",{"date":165,"type":166},"2025-11-12","ACTUAL",{"date":168,"type":67},"2014-04",{"date":170,"type":145},"2028-12",{"name":5,"class":6},3]