[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"prolymphocytic-leukemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:prolymphocytic-leukemia":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,41,99,134],{"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":22,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100394053","observatory-of-prolymphocytic-leukemia-t-100394053",false,"NCT04411043","Observatory of Prolymphocytic Leukemia T","Prospective and Retrospective Study Evaluating Epidemiological, Clinical, Molecular and Therapeutic Data of Prolymphocytic Leukemia T. A FILO Study.","T-PLL","Inclusion Criteria:\n\n* Man or woman aged 18 or over\n* Patient with prolymphocytic T leukemia\n\nExclusion Criteria:\n\n* Absence of signature of informed consent","ALL","18 Years",{"count":20,"type":21},50,"ESTIMATED","3 Years","OBSERVATIONAL","Prolymphocytic leukemia T is a rare disease representing approximately 2% of mature lymphoid leukemias and 20% of prolymphocytic leukemias. It mainly affects the elderly with an aggressive clinical course. It is a hemopathy exhibiting a post thymic T phenotype (Tdt-, CD1a-, CD5 +, CD2 + and CD7 +), generally CD4 + \u002F CD8-, but also CD4 + \u002F CD8 + or CD8 + \u002F CD4-.\n\nThe main feature of T-PLL is the rearrangement of chromosome 14 involving genes encoding the T cell receptor complex (TCR) subunits, leading to overexpression of the proto-oncogene TCL1.\n\nOn the molecular level, the study of Prolymphocytic leukemia T shows a substantial mutational activation of the IL2RG-JAK1-JAK3-STAT5B axis.\n\nPatients with Prolymphocytic leukemia T have a poor prognosis, due to a poor response to conventional chemotherapy. Treatment with the anti-CD52 monoclonal antibody: alemtuzumab has considerably improved the results, but the responses to treatment are transient; therefore, patients who obtain a response to alemtuzumab treatment are candidates for stem cell allograft (TSS) if they are eligible for this procedure. This combined approach extended the median survival to four years or more. However, new approaches using well-tolerated therapies that target signaling and survival pathways are necessary for most patients who are unable to receive intensive chemotherapy, such as JAK STAT axis inhibitors, anti-AKT, or anti BCL2 .\n\nMain objective: Better manage prolymphocytic T leukemias.\n\nSecondary objectives:\n\n* Molecular characterization of prolymphocytic leukemia T.\n* Study of the response to treatment, disease-free survival, overall survival.\n* Impact of prognostic factors on response to treatment, and survival.",[26,27],"Prolymphocytic Leukemia","T-cell Leukemia","RECRUITING","2026-06-29",{"date":31,"type":32},"2026-07-01","ACTUAL",{"date":34,"type":32},"2020-07-01",{"date":36,"type":21},"2028-07-30",{"name":38,"class":39},"French Innovative Leukemia Organisation","OTHER",1,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":48,"enrollmentInfo":49,"targetDuration":4,"studyType":51,"phases":52,"briefSummary":54,"conditions":55,"keywords":83,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":40},"100309946","phase-2-myeloablative-allo-hsct-with-related-or-unrelated-donor-for-heme-disorders-100309946","NCT03314974","Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders","Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Unrelated Donor for the Treatment of Hematological Diseases","-Inclusion Criteria:\n\n* Age: ≤ 60 years of age\n* Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70\n* Consent: Voluntary written consent (adult or legally authorized representative; or parental\u002Fguardian)\n* Adequate Organ Function:\n\n  * Renal: Creatinine \\\u003C2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml\u002Fmin\u002F1.73m2 as determined by an age-appropriate method, such as cystatin C GFR.\n  * Hepatic: Bilirubin, AST, alkaline phosphatase \\\u003C4 times the upper limit of institutional normal\n  * Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, \\> 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of \\>95% on room air\n  * Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \\> 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation\n  * HIV Status: HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation\n\nOther Inclusion Criteria:\n\n* Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.\n* Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program\n* Eligible Diseases and Status: Patients are eligible unless their treatment is to be guided by a higher priority protocol.\n* Acute Leukemias: Must be in remission by morphology (≤5% blasts). Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse.\n* Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients \\> 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.\n* Favorable risk AML is defined as having one of the following:\n\n  * t(8,21) without cKIT mutation\n  * inv(16) or t(16;16) without cKIT mutation\n  * Normal karyotype with mutated NPM1 and wild type FLT-ITD\n  * Normal karyotype with double mutated CEBPA\n  * Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation\n* Very high risk pediatric patients with AML: Patients \\\u003C21 years, however, are eligible with (M2 marrow) with \\\u003C 25% blasts in marrow after having failed one or more cycles of chemotherapy.\n* Acute lymphoblastic leukemia (ALL)\u002Flymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.\n* High risk ALL is defined as having one of the following:\n\n  * Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1\n  * 30 years of age or older at diagnosis\n  * White blood cell counts of greater than 30,000\u002FmcL (B-ALL) or greater than 100,000\u002FmcL (T-ALL) at diagnosis\n  * CNS leukemia involvement during the course of disease\n  * Slow cytologic response (\\>10% lymphoblasts in bone marrow on Day 14 of induction therapy)\n  * Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy\n* Very high risk pediatric patients with ALL: patients \\\u003C21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieve a complete remission.\n* Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to one or more tyrosine kinase inhibitors.\n* Plasma Cell Leukemia after initial therapy, in patients who have achieved at least a partial remission\n* Myeloproliferative Neoplasms\u002FMyelofibrosis, either primary as a result of polycythemia vera or essential thrombocythemia, with disease risk of intermediate or high-risk according to DIPSS criteria. Blasts must be \\\u003C10% by bone marrow aspirate morphology.\n* Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features. Blasts must be \\\u003C 10% by a representative bone marrow aspirate morphology.\n* Chronic Lymphocytic Leukemia\u002FSmall Lymphocytic Lymphoma (CLL\u002FSLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression\u002Frelapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting \\> 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant.\n* Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.\n* Diffuse large Cell NHL \\> CR\u002F\\> PR: Patients in CR\u002FPR with initial short remission (\\\u003C6 months) are eligible, or those who have failed\u002For are not eligible for autologous transplant.\n* Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III\u002FIV in CR1\u002FPR1 or after progression if stage I\u002FII \\\u003C 1 year.\n* Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin \\> 3 mg\u002FL, may be considered for this protocol after initial therapy.\n* Juvenile myelomonocytic leukemia\n* Biphenotypic\u002FUndifferentiated\u002FProlymphocytic Leukemias in first or subsequent CR.\n* MRD positive leukemia (AML, ALL or accelerated\u002Fblast phase CML). Selected patients in morphologic CR, but with positive immunophenotypic (flow cytometry) or molecular evidence of MRD may be eligible if recent chemotherapy has not resulted in MRD negative status.\n* Natural Killer Cell Malignancies\n* Acquired Bone Marrow Failure Syndromes except for Fanconi Anemia or Dyskeratosis Congenita\n* Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.\n\nExclusion Criteria:\n\n* Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after \\> 2 salvage regimens)\n* CML in blast crisis\n* Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.\n* Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.\n* Active central nervous system malignancy\n* if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If \\>18 years old prior myeloablative allotransplant or autologous transplant\n* Active HIV infection or known HIV positive serology\n* active uncontrolled infection\n* Pregnant or breastfeeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.","60 Years",{"count":50,"type":21},300,"INTERVENTIONAL",[53],"PHASE2","This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine\u002Fbusulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF).",[56,57,58,59,60,61,62,63,64,65,66,67,68,69,70,71,72,26,73,74,75,76,77,78,79,80,81,82],"Acute Leukemia","Acute Myeloid Leukemia","Acute Lymphoblastic Leukemia","Lymphoma","Chronic Myelogenous Leukemia","Plasma Cell Leukemia","Myeloproliferative Neoplasms","Myelofibrosis","Myelodysplasia","Refractory Anemia","High Risk Anemia","Chronic Lymphocytic Leukemia","Small Lymphocytic Lymphoma","Marginal Zone B-Cell Lymphoma","Follicular Lymphoma","Lymphoplasmacytic Lymphoma","Mantle-Cell Lymphoma","Diffuse Large Cell Non Hodgkins Lymphoma","Lymphoblastic Lymphoma","Burkitt Lymphoma","High Grade Non-Hodgkin's Lymphoma, Adult","Multiple Myeloma","Juvenile Myelomonocytic Leukemia","Biphenotypic\u002FUndifferentiated\u002FProlymphocytic Leukemias","MRD Positive Leukemia","Natural Killer Cell Malignancies","Acquired Bone Marrow Failure Syndromes",[84,17,85,86,87,88,89],"AML","MDS","NHL","CLL","CML","SLL","2026-06-23",{"date":92,"type":32},"2026-06-25",{"date":94,"type":32},"2018-03-30",{"date":96,"type":21},"2028-06-10",{"name":98,"class":39},"Masonic Cancer Center, University of Minnesota",{"id":100,"slug":101,"hasResults":11,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":4,"eligibilityCriteria":105,"healthyVolunteers":106,"sex":17,"minAge":107,"maxAge":4,"enrollmentInfo":108,"targetDuration":4,"studyType":51,"phases":110,"briefSummary":111,"conditions":112,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":40},"100377523","phase-2-donor-stem-cell-transplant-with-treosulfan-fludarabine-and-total-body-irradiation-for-the-treatment-of-hematological-malignancies-100377523","NCT04195633","Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies","Hematopoietic Stem Cell Transplantation From Haploidentical Donors in Patients With Hematological Malignancies Using a Treosulfan-Based Preparative Regimen","Inclusion Criteria:\n\n* Acute leukemia (AL) that includes acute myeloid leukemia (AML) \u002F acute lymphoblastic leukemia (ALL) \u002F mixed phenotype leukemia (MPAL) in complete morphological remission (CR) with or without detectable minimal residual disease (MRD); complete morphological remission is defined by the presence of less than 5% of detectable blasts in bone marrow specimen, evaluated per standard of care. Patients with documented CR but without hematologic recovery since last chemotherapy are considered eligible to the study\n* Chronic myelogenous leukemia (CML), except refractory blast crisis. To be eligible in first chronic phase, patients must have failed or be intolerant to at least one tyrosine-kinase inhibitor\n* Chronic myelomonocytic leukemia (CMML)\n* Myelodysplastic syndromes (MDS)\n* Lymphoblastic, Burkitt's and other high-grade lymphoma in any complete (CR) or partial (PR) response\n\n  * CR and PR are defined according to Lugano classification: Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification\n* Low grade lymphoma (chronic lymphocytic leukemia \\[CLL\\]\u002Fsmall lymphocytic lymphoma \\[SLL\\], marginal zone lymphoma, follicular lymphoma) progressed after two treatment regimens, in CR\u002FPR\n\n  * For CLL\u002FSLL, CR and PR are defined according to: International Workshop on CLL (iwCLL) guidelines for diagnosis, indications for treatment, response assessment, and supportive management of CLL\n  * CR and PR are defined according to Lugano classification: Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification\n* Large cell lymphoma in \\> second CR (CR2)\u002F \\>= PR2\n\n  * CR and PR are defined according to Lugano classification: Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification\n* Mantle cell lymphoma, lymphoplasmacytic lymphoma and prolymphocytic leukemia may be eligible after initial therapy if in CR\u002FPR\n\n  * CR and PR are defined according to Lugano classification: Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification\n  * For prolymphocytic leukemia (PLL), CR is defined as a normalization of lymphadenopathies (long-axis diameter \\\u003C 1 cm) and splenomegaly (\\\u003C 13 cm), absence of constitutional symptoms, PLL cells \\\u003C 5% in bone marrow and circulating lymphocytes count \\\u003C 4 x 10\\^9\u002FL. Patients without hematopoietic recovery are considered eligible to the study. PR is defined as a decrease of \\>= 30% of the sum of lymphadenopathies' long-axis diameters, a decrease of \\>= 50% in spleen vertical length beyond normal from baseline, peripheral blood (PB) lymphocytes =\\\u003C 30 x 10\\^9\u002FL (and a decrease of \\>= 50% from baseline)\n* Hodgkin Lymphoma in \\> CR2\u002FPR2\n\n  * CR and PR are defined according to Lugano classification: Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification\n* Subjects must be \\>= 6 months old\n* Karnofsky \\>= 70 or Eastern Cooperative Oncology Group (ECOG) 0-1 (for adults)\n* Lansky score \\>= 50 (for children)\n* Adequate cardiac function defined as absence of decompensated congestive heart failure or uncontrolled arrhythmia AND left ventricular ejection fraction \\>= 40% or shortening fraction \\> 22%\n* Adequate pulmonary function defined as absence of oxygen (O2) requirements and one of the following:\n\n  * Diffusion capacity of the lung for carbon monoxide (DLCO) corrected \\>= 70% mm Hg\n  * DLCO corrected between 60% - 69% mm Hg and partial pressure of oxygen (pO2) \\>= 70 mm Hg\n  * DLCO corrected between 50% - 59% mm Hg and pO2 \\>= 80 mm Hg Pediatric patients unable to perform pulmonary function tests must have O2 saturation \\>= 92% on room air. May not be on supplemental oxygen\n* Total bilirubin \\\u003C 2 x upper limit of normal (ULN) unless felt to be related to Gilbert's disease or hemolysis\n* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\\\u003C 2.5 x ULN\n* Alkaline phosphatase =\\\u003C 5 x ULN\n* Creatinine \\\u003C 2.0 mg\u002Fdl (adults) or estimated creatinine clearance \\> 40 ml\u002Fmin (pediatrics)\n\n  * All adults with a creatinine \\> 1.2 or a history of renal dysfunction must have estimated creatinine clearance \\> 40 ml\u002Fmin\n* If recent mold infection, e.g., aspergillus, must be cleared by infectious disease to proceed\n* Patients who have undergone prior allogeneic hematopoietic cell transplant are eligible, but the prior transplant must have been performed at least 3 months prior to enrollment, unless in case of graft failure from the prior transplant\n* Written and signed informed consent\n* DONOR: Donors must be haploidentical relatives of the patients. Donor-recipient compatibility will be tested through HLA typing at high resolution for the HLA loci (-A, -B, -C, -DRB1, -DQB1). Donor and recipient should share at least 5\u002F10 HLA loci\n* DONOR: Age \\>= 12 years\n* DONOR: Weight \\>= 40 Kg\n* DONOR: Ability of donors younger than 18 years of age to undergo apheresis without use of a vascular access device. Vein check must be performed and verified by an apheresis nurse prior to arrival.\n* DONOR: Donor must meet selection criteria as defined by the Foundation of the Accreditation of Cell Therapy (FACT) and will be screened per the American Association of Blood Banks (AABB) guidelines\n* DONOR: In case of more available haploidentical donors, selection criteria should include, in this order:\n\n  * For cytomegalovirus (CMV) seronegative recipients, a CMV seronegative donor\n  * Red blood cell compatibility\n\n    * Red blood cell (RBC) cross match compatible\n    * Minor ABO incompatibility\n    * Major ABO incompatibility\n\nExclusion Criteria:\n\n* Active, uncontrolled, life-threatening viral, bacterial or fungal infection requiring treatment at time of conditioning regiment administration and transplantation\n* Presence of a malignancy other than the one for which the transplant is being performed, with an expected survival less than 75% at 5 years\n* Pregnant or breastfeeding\n* Known hypersensitivity to treosulfan, fludarabine or cyclophosphamide\n* Dosing with another investigational agent within 30 days prior to entry in the study\n* Central nervous system (CNS) leukemic involvement not clearing with intrathecal chemotherapy and\u002For cranial radiation prior to initiation of conditioning (day -6)\n* DONOR: Since detection of anti-donor-specific-antigen antibodies (anti-DSA) is associated with higher graft rejection rate, patients will be screened for anti-DSA pre-transplant. Patients with DSA mean fluorescent intensity (MFI) \\\u003C 5000 after desensitization treatment, will be considered eligible to participate in the study. The first 10 subjects enrolled in the trial will be DSA-negative.",true,"6 Months",{"count":109,"type":21},60,[53],"This phase II trial studies how well a donor stem cell transplant, treosulfan, fludarabine, and total-body irradiation work in treating patients with blood cancers (hematological malignancies). Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.",[56,58,57,113,114,75,115,116,117,74,71,118,119,120,26,121,122,123,124],"Adult Diffuse Large Cell Lymphoma","Anaplastic Large Cell Lymphoma","Chronic Myeloid Leukemia, BCR-ABL1 Positive","Chronic Myelomonocytic Leukemia","Hodgkin Lymphoma","Mantle Cell Lymphoma","Mixed Phenotype Acute Leukemia","Myelodysplastic Syndrome","Refractory Chronic Lymphocytic Leukemia","Refractory Follicular Lymphoma","Refractory Marginal Zone Lymphoma","Refractory Small Lymphocytic Lymphoma","2026-05-27",{"date":127,"type":32},"2026-05-29",{"date":129,"type":32},"2021-01-25",{"date":131,"type":21},"2029-03-12",{"name":133,"class":39},"Fred Hutchinson Cancer Center",{"id":135,"slug":136,"hasResults":11,"nctId":137,"briefTitle":138,"officialTitle":139,"acronym":4,"eligibilityCriteria":140,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":141,"enrollmentInfo":142,"targetDuration":4,"studyType":51,"phases":144,"briefSummary":145,"conditions":146,"keywords":158,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":168,"completionDateStruct":170,"leadSponsor":172,"locationsCount":40},"100501157","phase-2-allo-hsct-using-ric-and-ptcy-for-hematological-diseases-100501157","NCT05805605","Allo HSCT Using RIC and PTCy for Hematological Diseases","Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases","Inclusion Criteria:\n\n* Age 0 to 75 years of age with Karnofsky score ≥ 70% (≥ 16 years) or Lansky score ≥ 50 (\\\u003C 16 years).\n* 5\u002F6 or 6\u002F6 related donor, OR a 7-8\u002F8 HLA-A, B, C, DRB1 allele match, OR a haplotype (at least 5\u002F10) matched related donor. Donors will be requested to provide PBSCs although bone marrow is acceptable according to donor preference.\n\nEligible Diseases Acute Leukemias: Must be in remission by morphology (≤5% blasts) AND without evidence of MRD by flow cytometry, FISH, or conventional cytogenetics. PCR based MRD detection is not an exclusion to proceed.\n\nAcute Myeloid Leukemia (AML) and related precursor neoplasms:\n\n2nd or greater complete remission (CR); first complete remission (CR1) in patients \\> 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.\n\nFavorable risk AML is defined as having one of the following:\n\n* t(8,21) without cKIT mutation\n* inv(16) or t(16;16) without cKIT mutation\n* Normal karyotype with mutated NPM1 and wild type FLT-ITD (unless persistently NPM1 positive by PCR following two cycles of chemotherapy)\n* Normal karyotype with double mutated CEBPA\n* Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation\n\nAcute lymphoblastic Leukemia (ALL) \u002Flymphoma:\n\nCR2 or greater, CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.\n\nHigh risk ALL is defined as having one of the following:\n\n* Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1\n* 30 years of age or older at diagnosis\n* White blood cell counts of greater than 30,000\u002FmcL (B-ALL) or greater than 100,000\u002FmcL (T-ALL) at diagnosis\n* CNS leukemia involvement during the course of disease\n* Slow cytologic response (\\>10% lymphoblasts in bone marrow on Day 14 of induction therapy)\n* Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy.\n\nVery high risk pediatric patients with ALL:\n\npatients \\\u003C21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.\n\nBiphenotypic\u002FUndifferentiated\u002FProlymphocytic Leukemias in first or subsequent CR.\n\nChronic Myelogenous Leukemia in chronic or accelerated phase, or CML blast crisis in morphological remission (\\\u003C5% blasts) and with negative MRD by flow cytometry (a positive PCR for BCRABL is acceptable for BMT): Chronic phase patients must have failed at least two different TKIs, been intolerant to all available TKIs or have T315I mutation. Patients with CML blast crisis in CR are only eligible if there is an feasible TKI maintenance plan following BMT.\n\nPlasma Cell Leukemia after initial therapy, who achieved at least a partial remission; or relapsed and achieved subsequent remission (CR\u002FPR) Myelodysplastic Syndrome: IPSS INT-2 or High Risk; R-IPSS High or Very High; WHO classification: RAEB-1, RAEB-2; Severe Cytopenias: ANC \\\u003C 0.8, Anemia or thrombocytopenia requiring transfusion; Poor or very poor risk cytogenetics based on IPSS or R-IPSS definitions; therapy-related MDS. Blasts must be \\\u003C 5% by bone marrow aspirate morphology. If ≥5% blasts, patient requires chemotherapy for cytoreduction to \\\u003C5% blasts prior to transplantation Leukemia or MDS in aplasia. These patients may be taken to transplant if after induction therapy they remain with aplastic bone marrow and no morphological or flow-cytometry evidence of disease ≥ 28 days post-therapy. These high risk patients will be analyzed separately.\n\nBurkitt's Lymphoma in CR2 or subsequent CR. Relapsed T-Cell Lymphoma that is chemotherapy sensitive in CR\u002FPR that has failed or ineligible for an autologous transplantNatural Killer Cell Malignancies. Relapsed Chronic Lymphocytic Leukemia\u002FSmall Lymphocytic Lymphoma (CLL\u002FSLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma which have progressed within 12 months of achieving a partial or complete remission. Patients who had remissions lasting \\> 12 months, are eligible after at least two prior therapies. Patients with bulky disease should be considered for de-bulking chemotherapy before transplant. Patients with refractory disease may be eligible, unless bulky disease and an estimated tumor doubling time of less than one month.\n\nLymphoplasmacytic Lymphoma, Mantle-Cell Lymphomais eligible after initial therapy if chemotherapy sensitive.\n\nLarge Cell and other high risk NHL \\> CR2\u002F\\> PR2: Patients in CR2\u002FPR2 with initial short remission (\\\u003C6 months) are eligible.\n\nRelapsed Multiple Myeloma: that is chemotherapy sensitive and has failed or ineligible for an autologous transplant.\n\nMyeloproliferative Neoplasms\u002FMyelofibrosis - with transfusion dependence or expected survival under 5 years by DIPSS, DIPSS-plus, or MPSS70 calculator.\n\nAcquired Bone Marrow Failure Syndromes except for Fanconi anemia Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.\n\nAdditional Criteria for Bulky Disease (lymphomas) if stable disease is best response, the largest residual nodal mass must \\\u003C 5 cm (approximately) If response to previous therapy, the largest residual mass must represent a 50% reduction and be \\\u003C 7.5 cm (approximately)\n\nOrgan Function Criteria\n\nAdequate organ function is defined as:\n\nLiver: Transaminases ≤ 5 x upper limit of normal (ULN) and total bilirubin ≤ 2.5 mg\u002FdL except for patients with Gilbert's syndrome or hemolysis.\n\nRenal: A normal creatinine (adults) or creatinine clearance ≥ 40 mL\u002Fmin (pediatrics). Adults with a creatinine \\> 1.2 mg\u002Fdl or a history of renal dysfunction must have estimated GFR ≥ 40 ml\u002Fmin\u002F1.73m2.\n\nCardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \\> 40%. For children that are not able to cooperate with MUGA and echocardiography, such should be clearly stated in the physician's note.\n\nPulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.\n\nIf recent confirmed mold infection (e.g. aspergillus) must have minimum of 30 days of therapy and responsive disease and be cleared by Infectious Disease HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation Sexually active females of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control during study treatment Voluntary written consent (adult or parent\u002Fguardian with presentation of the minor information sheet, if appropriate)\n\nRelated donors will be evaluated and collected according to UMN BMT program standard processes. Unrelated donors will be identified and collected through the National Marrow and Donor Program (NMDP) per usual steps.\n\nExclusion Criteria:\n\n* Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.\n* Untreated active infection\n* Active central nervous system malignancy\n* CML in blast crisis\n* Intermediate or high grade NHL, mantle cell NHL, and Hodgkin disease that is progressive on salvage therapy. Stable disease is acceptable to move forward provided it is non-bulky.\n* Less than 3 months since prior myeloablative transplant\n* Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.","75 Years",{"count":143,"type":21},56,[53],"This is a Phase II study following subjects proceeding with our Institutional non-myeloablative cyclophosphamide\u002F fludarabine\u002Ftotal body irradiation (TBI) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion using post-transplant cyclophosphamide (PTCy), sirolimus and MMF GVHD prophylaxis.",[147,148,149,150,26,60,61,151,152,153,75,154,155,68,156,70,157,63],"Acute Myelogenous Leukemia","Acute Lymphocytic Leukemia","Biphenotypic Acute Leukemia","Undifferentiated Leukemia","Myelodysplastic Syndromes","Leukemia, Myeloid","Myelodysplastic Syndrome With Excess Blasts-1","Relapsed T-Cell Lymphoma","Relapsed Chronic Lymphocytic Leukemia","Marginal Zone Lymphoma","Myeloproliferative Neoplasm",[85,87,89,84,88,159,160,161,162,163,164,17],"PFS","TRM","GVHD","MMF","TBI","PTCy","2025-06-27",{"date":167,"type":32},"2025-07-01",{"date":169,"type":32},"2023-05-01",{"date":171,"type":21},"2028-10-22",{"name":98,"class":39}]