[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100547786":3},{"organization":4,"armGroups":7,"interventions":26,"overallOfficials":32,"centralContacts":75,"locations":81,"responsibleParty":98,"collaborators":32,"id":100,"slug":32,"hasResults":101,"nctId":102,"briefTitle":103,"officialTitle":103,"acronym":32,"eligibilityCriteria":104,"healthyVolunteers":101,"sex":105,"minAge":106,"maxAge":107,"enrollmentInfo":108,"targetDuration":32,"studyType":111,"phases":112,"briefSummary":114,"conditions":115,"keywords":120,"overallStatus":84,"whyStopped":32,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":131,"completionDateStruct":133,"leadSponsor":135,"locationsCount":136},{"fullName":5,"class":6},"Masonic Cancer Center, University of Minnesota","OTHER",[8,22],{"label":9,"type":10,"description":11,"interventionNames":12},"Arm A: No clonal hematopoiesis","EXPERIMENTAL","Participants 25 years of age and younger with no clonal hematopoiesis. Active study treatment includes the conditioning regimen followed by the stem cell infusion and GvHD prophylaxis through day +180. Supportive care and follow up activities continue through two years post HCT.",[13,14,15,16,17,18,19,20,21],"Drug: Rituximab","Drug: Rabbit ATG","Drug: Cyclophosphamide","Drug: Fludarabine","Radiation: Total Body Irradiation","Biological: Cell Infusion","Drug: Post-Transplant G-CSF","Drug: Tacrolimus","Drug: Mycophenolate Mofetil",{"label":23,"type":10,"description":24,"interventionNames":25},"Arm B: Clonal hematopoiesis","Participants 25-75 years old and\u002For with clonal hematopoiesis. Active study treatment includes the conditioning regimen followed by the stem cell infusion and GvHD prophylaxis through day +180. Supportive care and follow up activities continue through two years post HCT.",[13,14,15,16,17,18,19,20,21],[27,33,39,43,47,54,59,65,69],{"type":28,"name":29,"description":30,"armGroupLabels":31,"otherNames":32},"DRUG","Rituximab","For patients with EBV IgG seropositivity or EBV PCR positivity on pre-transplant evaluations, Rituximab 375 mg\u002Fm2 is given IV once on day -14 (+\u002F-2 day) in the outpatient setting. Pre-medicate 30 minutes prior to rituximab with methylprednisolone (1 mg\u002Fkg) IV, acetaminophen 15 mg\u002Fkg (maximum 650mg) IV or PO and diphenhydramine 1 mg\u002Fkg (maximum 50mg) IV or PO.",[9,23],null,{"type":28,"name":34,"description":35,"armGroupLabels":36,"otherNames":37},"Rabbit ATG","Rabbit ATG will be administered at doses and days indicated above, infused through a 0.22 micrometer filter over 4-6 hours. Pre-medicate 30 minutes prior to ATG infusion with methylprednisolone 1 mg\u002Fkg IV, (max dose = 125 mg), acetaminophen 15 mg\u002Fkg dose (max dose = 650 mg) enterally and diphenhydramine 1 mg\u002Fkg\u002Fdose (max dose = 50 mg) enterally or IV.",[9,23],[38],"Thymoglobulin",{"type":28,"name":40,"description":41,"armGroupLabels":42,"otherNames":32},"Cyclophosphamide","Cyclophosphamide 14.5 mg\u002Fkg is be given as a 2-hour infusion on day -6. If the patient is obese (actual body weight (ABW) \\>\u002F= 125% of the ideal body weight (IBW)), cyclophosphamide should be dosed using the adjusted body weight (AdjBW): 0.5(ABW-IBW) + IBW. Uroprotection with MESNA (14.5 mg\u002Fkg\u002Fday) in IV continuous infusion will be provided per institutional guidelines. Hyperhydration is not required for 14.5 mg\u002Fkg cyclophosphamide doses.\n\nCyclophosphamide will be administered at 50 mg\u002Fkg using ABW over 2 hours on days +3 and +4. If the patient is obese (ABW \\>\u002F= 125% of the ideal body weight (IBW)), cyclophosphamide should be dosed using the adjusted body weight (AdjBW): 0.5(ABW-IBW) + IBW. Uroprotection with MESNA (50 mg\u002Fkg\u002Fday) in IV continuous infusion as well as hyperhydration will be provided per institutional guidelines.",[9,23],{"type":28,"name":44,"description":45,"armGroupLabels":46,"otherNames":32},"Fludarabine","For all patients, fludarabine dosing will be model-based using Bayesian methodology IV every 24 hours on days -6 to -3 with a cumulative area under the curve (cAUC) of 20 mg\\*hr\u002FL.",[9,23],{"type":48,"name":49,"description":50,"armGroupLabels":51,"otherNames":52},"RADIATION","Total Body Irradiation","For patients age \\>\u002F= 25 years, with myelodysplasia, or clonal hematopoiesis, total body irradiation will be 4 Gy, provided in two fractions on day -1. For all other patients, total body irradiation will be 2 Gy provided in a single fraction on day -1.\n\nEach dose of 2 Gy will be given at a dose rate between 1 and 1.9 Gy\u002Fminute prescribed to the midplane of the patient at the level of the umbilicus.",[9,23],[53],"TBI",{"type":55,"name":56,"description":57,"armGroupLabels":58,"otherNames":32},"BIOLOGICAL","Cell Infusion","On day 0 the cells will be infused per cell source specific institutional guidelines.",[9,23],{"type":28,"name":60,"description":61,"armGroupLabels":62,"otherNames":63},"Post-Transplant G-CSF","Beginning on day +5, patients will receive G-CSF SQ or IV 5 micrograms\u002Fkg once daily until post-nadir ANC \\> 1500\u002FμL for 3 consecutive days or \\>3000\u002FμL for 1 day.",[9,23],[64],"Filgrastim",{"type":28,"name":66,"description":67,"armGroupLabels":68,"otherNames":32},"Tacrolimus","Tacrolimus will begin on day +5 at an initial dose of 0.03 mg\u002Fkg\u002Fday IV via continuous infusion. Goal trough levels will be 10-15 ug\u002FmL until day +14 posttransplant, then decreased to a goal of 5-10 ng\u002FmL thereafter. In the absence of GvHD, tacrolimus will discontinue at day +180 without a taper.",[9,23],{"type":28,"name":70,"description":71,"armGroupLabels":72,"otherNames":73},"Mycophenolate Mofetil","Mycophenolate mofetil (MMF) therapy will begin on day +5. For pediatric service patients dosing of MMF will be 15 mg\u002Fkg\u002Fdose (max = 1000 mg) three times daily. For adult service patients dosing of MMF will be 15 mg\u002Fkg\u002Fdose (max = 1500 mg) twice daily. The same dosage is used orally or intravenously. Consider dose modification and\u002For pharmacokinetic measurements if renal and\u002For hepatic impairment (GFR\\\u003C25 mL\u002Fminute corrected). Stop MMF at Day +35 or 7 days after engraftment achieved (ANC\\>500 x 106 neutrophils\u002FL x 3 days) if later than day +35. If sufficient acute GvHD is observed to require systemic therapy, MMF should be continued for 7 days after initiation of systemic therapy. Afterward, use of MMF is at the discretion of the treating physician.",[9,23],[74],"MMF",[76],{"name":77,"role":78,"phone":79,"phoneExt":32,"email":80},"Meera Srikanthan, MD","CONTACT","(612) 626-2961","srika038@umn.edu",[82],{"facility":83,"status":84,"city":85,"state":86,"zip":87,"country":88,"countryCode":89,"cosmosGeoPoint":90,"geoPoint":95,"contacts":96},"University of Minnesota Masonic Cancer Center","RECRUITING","Minneapolis","Minnesota","55455","United States","US",{"type":91,"coordinates":92},"Point",[93,94],-93.26384,44.97997,{"lat":94,"lon":93},[97],{"name":77,"role":78,"phone":32,"phoneExt":32,"email":80},{"type":99,"investigatorFullName":32,"investigatorTitle":32,"investigatorAffiliation":32,"oldNameTitle":32,"oldOrganization":32},"SPONSOR","100547786",false,"NCT06412497","MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.","Inclusion Criteria:\n\n* Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL AND bone marrow cellularity \\\u003C50% (with \\\u003C 30% residual hematopoietic cells)\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%), without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic SAA with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Paroxysmal Nocturnal Hemoglobinuria (PNH), including AA-PNH overlap syndrome, acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone \\>\u002F= 10%\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%) without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic PNH, aPRCA, or aAT with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Adequate organ function within 30 days of conditioning regimen\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment\n* Uncontrolled infection\n* Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy\n* Known allergy to any of the study components\n* Prior radiation therapy deemed excessive by radiation therapist for proposed low dose TBI exposure on this protocol\n* Diagnosis of an inherited bone marrow failure disorder such as Fanconi anemia, Telomere biology disorder, or Schwachman-Diamond syndrome, unless reviewed by the principal investigator and deemed appropriate for this approach (e.g. GATA2 deficiency)\n* Advanced myelodysplastic syndrome (MDS; BM blasts \\>5%) or acute myeloid leukemia\n* Psychiatric illness\u002Fsocial situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements\n* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study","ALL","0 Years","75 Years",{"count":109,"type":110},60,"ESTIMATED","INTERVENTIONAL",[113],"PHASE2","A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and\u002For clonal hematopoiesis.",[116,117,118,119],"Severe Aplastic Anemia","Acquired Amegakaryocytic Thrombocytopenia","Acquired Pure Red Cell Aplasia","Paroxysmal Nocturnal Hemoglobinuria",[121,122,123,124,125,126],"HCT","RIC","SAA","PTCy","aAT","aPRCA","2026-06-02",{"date":129,"type":130},"2026-06-03","ACTUAL",{"date":132,"type":130},"2024-06-05",{"date":134,"type":110},"2036-05-01",{"name":5,"class":6},1]