[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100330256":3},{"organization":4,"armGroups":7,"interventions":40,"overallOfficials":106,"centralContacts":110,"locations":115,"responsibleParty":135,"collaborators":77,"id":137,"slug":138,"hasResults":139,"nctId":140,"briefTitle":141,"officialTitle":142,"acronym":77,"eligibilityCriteria":143,"healthyVolunteers":139,"sex":144,"minAge":77,"maxAge":145,"enrollmentInfo":146,"targetDuration":77,"studyType":149,"phases":150,"briefSummary":152,"conditions":153,"keywords":77,"overallStatus":118,"whyStopped":77,"lastUpdateSubmitDate":162,"lastUpdatePostDateStruct":163,"startDateStruct":166,"completionDateStruct":168,"leadSponsor":170,"locationsCount":171},{"fullName":5,"class":6},"Masonic Cancer Center, University of Minnesota","OTHER",[8,20,25,30,35],{"label":9,"type":10,"description":11,"interventionNames":12},"Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia","EXPERIMENTAL","Given to:\n\n* Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type OR\n* Patients with an HLA- identical sibling donor recipient and myelodysplastic features, MDS, or acute leukemia",[13,14,15,16,17,18,19],"Drug: Total Body Irradiation (TBI) (Plan 1)","Drug: Cyclophosphamide (CY) (Plan 1)","Drug: Fludarabine (FLU)","Drug: Methylprednisolone (MP)","Device: Donor mobilized PBSC infusion","Drug: G-CSF","Drug: Rituximab",{"label":21,"type":10,"description":22,"interventionNames":23},"Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia","Given to:\n\n• An HLA-identical sibling donor recipients with single or multi- lineage hematopoietic failure",[15,16,17,18,24,19],"Drug: Cyclophosphamide (CY) (Plan 2)",{"label":26,"type":10,"description":27,"interventionNames":28},"Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia","Given to:\n\n* Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type who cannot tolerate TBI\n* Patients with an HLA- identical sibling donor recipient and myelodysplastic features, MDS, or acute leukemia who cannot tolerate TBI\n* Biallelic mutations in FANCD1\u002FBRCA2 who cannot receive TBI\n* Per treating physician preference",[14,15,16,19,29],"Drug: Busulfan",{"label":31,"type":10,"description":32,"interventionNames":33},"Treatment Plan 4: CY, FLU, and alemtuzumab","given to TBD patients with:\n\n* Bone marrow failure AND\n* Any donor type including haploidentical (4\u002F8) to 8\u002F8-HLA matched related donor, or 7-8\u002F8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 3 patients would be treated per year. Statistical outcomes will be descriptive.",[14,15,34],"Drug: Alemtuzumab",{"label":36,"type":10,"description":37,"interventionNames":38},"Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab.","given to TBD patients with:\n\n* Early myelodysplastic features (with or without cytogenetic abnormalities) AND\n* Any donor type including haploidentical (4\u002F8) to 8\u002F8-HLA matched related donor, or 7-8\u002F8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 2 patients would be treated per year. Statistical outcomes will be descriptive.",[14,15,34,39],"Drug: Melphalan",[41,48,54,60,66,73,78,83,87,93,97,103],{"type":42,"name":43,"description":44,"armGroupLabels":45,"otherNames":46},"DRUG","Total Body Irradiation (TBI) (Plan 1)","300 cGy with thymic shielding on day -6",[9],[47],"TBI",{"type":42,"name":49,"description":50,"armGroupLabels":51,"otherNames":52},"Cyclophosphamide (CY) (Plan 1)","10 mg\u002Fkg IV daily on days -5, -4, -3, and -2",[9,26,31,36],[53],"CY",{"type":42,"name":55,"description":56,"armGroupLabels":57,"otherNames":58},"Fludarabine (FLU)","35 mg\u002Fm2 IV daily on days -5, -4, -3, and -2",[9,21,26,31,36],[59],"FLU",{"type":42,"name":61,"description":62,"armGroupLabels":63,"otherNames":64},"Methylprednisolone (MP)","1 mg\u002Fkg IV q12h on days -5, -4, -3, -2, and -1",[9,21,26],[65],"MP",{"type":67,"name":68,"description":69,"armGroupLabels":70,"otherNames":71},"DEVICE","Donor mobilized PBSC infusion","T cell receptor alpha\u002Fbeta depletion (α\u002Fβ TCD) peripheral blood stem cell (PBSC) transplantation on day 0",[9,21],[72],"PBSC",{"type":42,"name":74,"description":75,"armGroupLabels":76,"otherNames":77},"G-CSF","Initiate G-CSF 5mcg\u002Fkg per day IV on day +1 (continue until ANC \\>2.5 x 10\\^9\u002FL for 3 consecutive days or single day ANC \\>3000 Arm 1 and Arm 3)",[9,21],null,{"type":42,"name":79,"description":80,"armGroupLabels":81,"otherNames":82},"Cyclophosphamide (CY) (Plan 2)","5 mg\u002Fkg IV daily on days -5, -4, -3, and -2",[21],[53],{"type":42,"name":84,"description":85,"armGroupLabels":86,"otherNames":77},"Rituximab","200 mg\u002Fm2 IV once on day -1",[9,21,26],{"type":42,"name":88,"description":89,"armGroupLabels":90,"otherNames":91},"Busulfan","Busulfan 0.6 mg\u002Fkg if \\> 4 years old and\u002For \\>12 kg (0.8 mg\u002Fkg IV if ≤ 4 years old and\u002For ≤ 12 kg) is given IV over 2 hours every 12 hours for 2 days.",[26],[92],"BU",{"type":42,"name":94,"description":95,"armGroupLabels":96,"otherNames":77},"Alemtuzumab","Alemtuzumab 0.2 mg\u002Fkg is given IV over 2 hours daily for 5 days (total dose 1 mg\u002Fkg)",[31,36],{"type":42,"name":98,"description":99,"armGroupLabels":100,"otherNames":101},"Melphalan","If available, MEL dosing will be model-based using Bayesian methodology. If Bayesian methodology is unavailable, MEL dosing will be weight-based: MEL 70 mg\u002Fm2 for patients ≥10 kg (2.35 mg\u002Fkg for patients \\\u003C10 kg\\^) IV for one dose over 30 minutes.",[36],[102],"MEL",{"type":42,"name":84,"description":104,"armGroupLabels":105,"otherNames":77},"Rituximab will be given once on treatment plans 1-3 on day -1.",[9,21,26],[107],{"name":108,"affiliation":5,"role":109},"Margaret MacMillan, MD, Msc, FRCPC","PRINCIPAL_INVESTIGATOR",[111],{"name":108,"role":112,"phone":113,"phoneExt":77,"email":114},"CONTACT","612-626-2961","macmi002@umn.edu",[116],{"facility":117,"status":118,"city":119,"state":120,"zip":121,"country":122,"countryCode":123,"cosmosGeoPoint":124,"geoPoint":129,"contacts":130},"Masonic Cancer Center at University of Minnesota","RECRUITING","Minneapolis","Minnesota","55455","United States","US",{"type":125,"coordinates":126},"Point",[127,128],-93.26384,44.97997,{"lat":128,"lon":127},[131],{"name":132,"role":112,"phone":133,"phoneExt":77,"email":134},"Lisa Burke, RN","612-273-8482","lburke3@Fairview.org",{"type":136,"investigatorFullName":77,"investigatorTitle":77,"investigatorAffiliation":77,"oldNameTitle":77,"oldOrganization":77},"SPONSOR","100330256","phase-2-alphabeta-tcd-hct-in-patients-with-inherited-bmf-disorders-100330256",false,"NCT03579875","Alpha\u002FBeta TCD HCT in Patients With Inherited BMF Disorders","MT2017-17:T Cell Receptor Alpha\u002FBeta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders","Patient Selection:\n\nInclusion Criteria:\n\nFor FA patients:\n\n* Diagnosis of Fanconi anemia\n\n  * Age \\\u003C65 years of age\n* Has one of the following risk factors:\n\n  * Severe aplastic anemia (SAA)\n  * Myelodysplastic features\n  * High risk genotype\n  * Immunodeficiency associated with history of recurrent infections\n* Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \\\u003C16 years of age\n\n  * Adequate pulmonary, cardiac and liver function\n  * Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care\n\nFor TBD patients:\n\n• Diagnosis of TBD\n\n* Age \\\u003C70 years of age\n* Has one of the following risk factors:\n* Severe aplastic anemia (SAA)\n* Myelodysplastic features\n* Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score\n\n  ≥ 50% for patients \\\u003C16 years of age\n* Adequate pulmonary, cardiac and liver function\n* Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration\n* Active, uncontrolled infection within 1 week prior to starting study therapy\n* Malignant solid tumor cancer within previous 2 years\n\nDonor Selection (Inclusion Criteria): meets one of the following match criteria:\n\n* an HLA-A, B, DRB1 matched sibling donor (matched sibling)\n* an HLA-A, B, DRB1 matched related donor (other than sibling)\n* a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen\n* 7-8\u002F8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus.\n* Body weight of at least 40 kilograms and at least 12 years of age\n* Willing and able to undergo mobilized peripheral blood apheresis\n* In general good health as determined by the medical provider\n* Adequate organ function defined as:\n\n  * Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin)\n  * Hepatic: ALT \\\u003C 2 x upper limit of normal\n  * Renal: serum creatinine \\\u003C 1.8 mg\u002Fdl\n* Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1\u002F2 Antibody, HTLVA 1\u002F2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B\n* Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start\n* Voluntary written consent (parent\u002Fguardian and minor assent, if \\\u003C 18 years) prior to the performance of any research related procedure","ALL","65 Years",{"count":147,"type":148},48,"ESTIMATED","INTERVENTIONAL",[151],"PHASE2","This is a phase II trial of T cell receptor alpha\u002Fbeta depletion (α\u002Fβ TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.",[154,155,156,157,158,159,160,161],"Fanconi Anemia","Severe Aplastic Anemia","Myelodysplastic Syndromes","T Cell Receptor Alpha\u002FBeta Depletion","Telomere Biology Disorder","Bone Marrow Failure","Dyskeratosis Congenita","Telomere Biology Disorders","2026-01-29",{"date":164,"type":165},"2026-01-30","ACTUAL",{"date":167,"type":165},"2018-11-13",{"date":169,"type":148},"2029-01-05",{"name":5,"class":6},1]