[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"all\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:all":16},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,42,88,118,155,195,221,242,266,296,322,350],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100329599","phase-1-ruxolitinib-and-chemotherapy-in-adolescents-and-young-adults-with-ph-like-acute-lymphoblastic-leukemia-100329599",false,"NCT03571321","Ruxolitinib and Chemotherapy in Adolescents and Young Adults With Ph-like Acute Lymphoblastic Leukemia","Phase I Trial of Ruxolitinib in Combination With a Pediatric Based-regimen for Adolescents and Young Adults (AYAs) With Ph-like Acute Lymphoblastic Leukemia (ALL)","Inclusion Criteria:\n\n* Newly diagnosed de novo B-precursor acute lymphoblastic leukemia (ALL) as determined by World Health Organization (WHO) criteria. Patients must have unequivocal diagnosis of precursor B ALL. This includes an institutional immunophenotyping report that is to assign B-lineage or T-lineage.\n* \"Ph-like\" signature, as determined by low density micro-array (LDA) card\n* Jak-targetable genetic signature as defined by any of the following:\n\n  * Cytokine receptor-like factor 2 (CRLF2) rearranged (JAK2 mutant or wild-type)\n  * JAK2 or erythropoietin receptor (EPOR) fusions.\n  * Other JAK pathway alterations at the discretion of the principle investigator including, but not limited to:\n* SH2B adaptor protein 3 (SH2B3) deletions\n* Interleukin-7 receptor subunit alpha (IL7RA) mutations\n* Prior therapy\n\n  * Prior to starting ruxolitinib, patients must have completed a 4-drug induction regimen with intrathecal chemotherapy (modified aBFM regimen or equivalent) as per the institutional standard of care. Recommended induction treatment is outlined in Section 5.1.2.\n  * No additional prior therapy for acute leukemia except emergency therapy (corticosteroids or hydroxyurea) for blast cell crisis, superior vena cava syndrome, or renal failure due to leukemic infiltration of the kidneys. When indicated, leukapheresis or exchange transfusion is recommended to reduce the white blood cell count (WBC).\n  * Screening may occur at any point prior to or during induction therapy\n* Age ≥ 18 years and \\\u003C 40 years. Because this is specifically a study of the adolescent and young adult population and no adverse event data are currently available on the use of this pediatric-based chemotherapy regimen in patients ≥ 40 years of age, older adults are excluded from this study, but may be eligible for future trials.\n* Eastern Cooperative Oncology Group (ECOG) performance status ≤2 (Karnofsky ≥ 60%)\n* Platelet count \\> 25,000\u002FuL.\n* Patients must have normal organ function as defined below:\n\n  * total bilirubin ≤ 2 mg\u002FdL\n  * aspartate aminotransferase (AST) \u002F alanine aminotransferase (ALT) ≤ 2.5 × institutional upper limit of normal\n  * creatinine within normal institutional limits OR creatinine clearance ≥ 60 mL\u002Fmin\u002F1.73 m2 for patients with creatinine levels above institutional normal.\n* Because the therapeutic agents used in this study are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation.\n* Ability to understand and the willingness to sign a written informed consent document.\n\nExclusion Criteria:\n\n* Patients who are receiving any other investigational agent.\n* Patients with a \"currently active\" second malignancy other than non-melanoma skin cancers. Patients are not considered to have a \"currently active\" malignancy if they have completed therapy and are free of disease for ≥ 3 years.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to ruxolitinib or other agents used in study.\n* Use of any potent cytochrome P450 (CYP) 3A4 inhibitor or inducer within 5 half-lives before the first dose of the study drug. Potent inhibitors of CYP3A4 include systemic ketoconazole, posaconazole, voriconazole, clarithromycin, itraconazole, nefazodone, and telithromycin. At the fluconazole dose of 200mg daily used this regimen, there is minimal inhibition of CYP3A4 \\[36\\] and therefore fluconazole is not prohibited on this trial and no dose modifications should be made in the presence of fluconazole.\n\nBecause the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated list such as http:\u002F\u002Fmedicine.iupui.edu\u002Fclinpharm\u002Fddis\u002Ftable.aspx; medical reference texts such as the Physicians' Desk Reference may also provide this information. As part of the enrollment\u002Finformed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product.\n\n* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n* Pregnant women are excluded from this study because ruxolitinib is a class C agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ruxolitinib breastfeeding should be discontinued if the mother is treated with ruxolitinib. These potential risks may also apply to other agents used in this study.\n* Down Syndrome due to the likelihood of excessive toxicity resulting. These patients should be treated in consultation with a pediatric oncologist.\n* Burkitt type leukemia\n* Ph+ ALL at time of diagnosis","ALL","18 Years","39 Years",{"count":20,"type":21},15,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","This study will test if adding ruxolitinib to standard multi-drug chemotherapy regimen will be safe and tolerated in adolescents and young adults with newly diagnosed Ph-like acute lymphoblastic leukemia (ALL).",[27,28,16],"Acute Lymphoblastic Leukemia","ALL, Childhood","RECRUITING","2026-06-24",{"date":32,"type":33},"2026-06-29","ACTUAL",{"date":35,"type":33},"2019-05-28",{"date":37,"type":21},"2027-09-05",{"name":39,"class":40},"University of Chicago","OTHER",2,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":54,"conditions":55,"keywords":74,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":78,"lastUpdatePostDateStruct":79,"startDateStruct":81,"completionDateStruct":83,"leadSponsor":85,"locationsCount":87},"100526212","pharmacokinetic-study-of-venetoclax-tablets-crushed-and-dissolved-into-a-solution-100526212","NCT06131801","Pharmacokinetic Study of Venetoclax Tablets Crushed and Dissolved Into a Solution","A Pharmacokinetic Study of Venetoclax Tablets Crushed and Dissolved Into a Solution in Children and Young Adults With Hematologic Malignancies","Inclusion Criteria:\n\n* Age: Patients must be \\\u003C39 years of age at time of study enrollment\n* Diagnosis: Patients may have a diagnosis of any hematologic malignancy\n* Central access: Patients must have an existing venous or arterial access line for PK blood draws\n* Weight requirement: Patients must weigh at least 5.5 kg at the time of enrollment\n* Venetoclax: Patients must be receiving any dose of venetoclax given as a solution made from crushed tablets by mouth (PO) or via nasogastric (NG), or G-tube as prescribed by their treating oncologist.\n* Concurrent chemotherapy medications: Patients may receive venetoclax as a single agent or in combination with any other chemotherapeutic agents.\n\nExclusion Criteria:\n\n* Pregnant women are excluded from this study because venetoclax has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with venetoclax, breastfeeding should be discontinued if the mother is treated with venetoclax.\n* Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method while on study treatment and for six months following completion.","0 Years","38 Years",{"count":52,"type":21},30,"OBSERVATIONAL","The use of venetoclax-based therapies for pediatric patients with relapsed or refractory malignancies is increasingly common outside of the clinical trial setting. For patients who cannot swallow tablets, it is common to crush the tablets and dissolve them in liquid to create a solution. However, no PK data exists in adults or children using crushed tablets dissolved in liquid in this manner, and as a result, the venetoclax exposure with this solution is unknown.\n\nPrimary Objectives\n\n• To determine the pharmacokinetics of venetoclax when commercially available tablets are crushed and dissolved into a solution\n\nSecondary Objectives\n\n* To evaluate the safety of crushed venetoclax tablets administered as an oral solution\n* To determine the pharmacokinetics of venetoclax solution in patients receiving concomitant strong and moderate CYP3A inhibitors\n* To determine potential pharmacokinetic differences based on route of venetoclax solution administration (ie. PO vs NG tube vs G-tube)\n* To determine the concentration of venetoclax in cerebral spinal fluid when administered as an oral solution",[56,57,58,59,16,60,61,62,63,64,65,66,67,68,69,70,71,72,73],"Hematologic Malignancy","Leukemia","Lymphoma","Acute Lymphocytic Leukemia","Acute Myelogenous Leukemia","AML","Chronic Myelogenous Leukemia","CML","Myeloproliferative Neoplasm","Non Hodgkin Lymphoma","Hodgkin Lymphoma","Diffuse Large B Cell Lymphoma","Follicular Lymphoma","Burkitt Lymphoma","T-cell Lymphoma","B Cell Lymphoma","Peripheral T Cell Lymphoma","Cutaneous B-Cell Lymphoma",[75,76,77],"Venetoclax","Pediatric AML","Pediatric Relapsed\u002FRefractory AML","2026-06-02",{"date":80,"type":33},"2026-06-04",{"date":82,"type":33},"2023-11-15",{"date":84,"type":21},"2027-12-01",{"name":86,"class":40},"Children's Hospital Medical Center, Cincinnati",5,{"id":89,"slug":90,"hasResults":11,"nctId":91,"briefTitle":92,"officialTitle":93,"acronym":4,"eligibilityCriteria":94,"healthyVolunteers":11,"sex":16,"minAge":95,"maxAge":96,"enrollmentInfo":97,"targetDuration":4,"studyType":22,"phases":99,"briefSummary":100,"conditions":101,"keywords":105,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":111,"completionDateStruct":113,"leadSponsor":115,"locationsCount":117},"100519771","phase-1-uab-2419-cd34-selection-using-the-automated-clinimacs-prodigy-100519771","NCT06047886","UAB 2419-CD34 Selection Using the Automated CliniMACS Prodigy","Feasibility Study of CD34 Selection for GVHD Prophylaxis Using the Automated CliniMACS","Inclusion Criteria:\n\n1. AML in morphologic remission with intermediate\u002Fhigh-risk features or relapsed disease 1 or 2\n2. ALL in morphologic remission with high-risk features or relapsed disease 1 or 2\n3. Lymphoid malignancies in CR or PR (e.g. non-Hodgkin's lymphoma, prolymphocytic leukemia, CLL)\n4. Myelodysplastic syndromes with \\\u003C=10% blasts\n5. CML in morphologic remission after blast phase or accelerated phase\n6. Primary myelofibrosis with \\\u003C=10% blasts \\^morphologic remission is defined as \\\u003C5% blasts on the bone marrow biopsy. Negative test for donor-specific antibody within 28 days of starting conditioning regimen, or adequate for standard desensitization protocol.\n\nExclusion Criteria:\n\n1. Non-compliant patients.\n2. No appropriate caregivers identified.\n3. Uncontrolled medical or psychiatric disorders which may preclude patients to undergo clinical studies (Discretion of the attending physician).\n4. Patients with known allergy to DMSO.\n5. Pregnant or breastfeeding women","4 Weeks","75 Years",{"count":98,"type":21},50,[24],"Patients with graft failure or delayed engraftment may benefit from a hematopoietic stem cell boost or an additional hematopoietic stem cell transplantation procedure. In such settings standard immune suppression strategies are avoided due to their myelosuppressive nature. Therefore those patients are at increased risk of graft versus host disease, and the infusion of a CD34 selected graft would reduce such a risk. The infusion of CD34 selected graft using CliniMACS plus is currently FDA FDA-approved indication for acute myeloid leukemia. However, the use of the Prodigy would streamline the processing, in terms of hands-off procedure, allowing to provision of this product to the patients without strains on the cell therapy lab team. This procedure has been demonstrated safe and effective in several single-center studies and is currently in advanced phase investigation in several studies for malignant and non-malignant conditions.",[61,16,102,103,63,104],"Lymphoid Malignancies","Myelodysplastic Syndromes","Primary Myelofibrosis",[106,107],"hematologic malignancies","graft failure","2026-04-08",{"date":110,"type":33},"2026-04-13",{"date":112,"type":33},"2025-04-22",{"date":114,"type":21},"2029-12",{"name":116,"class":40},"University of Alabama at Birmingham",1,{"id":119,"slug":120,"hasResults":11,"nctId":121,"briefTitle":122,"officialTitle":122,"acronym":4,"eligibilityCriteria":123,"healthyVolunteers":11,"sex":16,"minAge":124,"maxAge":125,"enrollmentInfo":126,"targetDuration":4,"studyType":22,"phases":128,"briefSummary":130,"conditions":131,"keywords":140,"overallStatus":146,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":117},"100632643","mynavigate-a-guide-to-after-treatment-effects-for-adolescents-and-young-adults-100632643","NCT07516353","my.naviGATE: A Guide to After-Treatment Effects for Adolescents and Young Adults","Inclusion Criteria:\n\n* Patients aged 15-24 who are currently undergoing cancer treatment at one of the three participating sites: DFCI, CNH, or RPOCH.\n* Patients must have initiated and be actively receiving cancer directed therapy for a newly diagnosed cancer.\n* Patients must be actively receiving cancer directed therapy, between the time of diagnosis and end of therapy for the following diagnoses: 1) Sarcomas: including osteosarcoma, Ewing sarcoma, and rhabdomyosarcoma; 2) Acute Myeloid Leukemia (AML); 3) Acute Lymphoblastic Leukemia\u002FLymphoma (ALL); 4) Hodgkin and mature B-cell Lymphomas; and 5) Medulloblastoma.\n* Patients whose treatment includes alkylators, anthracyclines, and\u002For radiation.\n\nInclusion of Children:\n\n• This study is designed to keep AYAs aged 15-24 engaged in cancer survivorship care and therefore a subset of participants will be \\\u003C18 years of age. The rationale for inclusion of children in this study is that AYA cancer survivors are particularly vulnerable to loss to follow-up and consequently lack of risk-based survivorship care, resulting in increased risk for preventable toxicity. Many existing interventions to improve engagement in survivorship screening and care are introduced after treatment completion and in long-term survivorship. Yet, for patients who are unaware of their late effect risks, and\u002For those who move frequently and are lost to follow-up, this may be too late. This study is designed to improve awareness of and engagement in risk-informed survivorship care for AYAs. This study presents no more than minimal risk to participants.\n\nExclusion Criteria:\n\n* Patients who are unwilling to give informed consent or assent to participate will be excluded. For patients under 18, patients whose guardians do not give informed consent will be excluded.\n* Patients with no chance of cure as identified by the AYA's physician, will be excluded given that issues of survivorship are not relevant and may be distressing to this population. Similarly, patients with relapsed or refractory disease will also be excluded.\n* Patients who are non-English-speaking and -reading will be excluded as the digital tool is being developed initially in English.\n* We will seek physician permission before offering enrollment to patients. If the provider team requests that the patient not be approached to participate, the patient will be excluded.","15 Years","24 Years",{"count":127,"type":21},143,[129],"NA","This study aims to design and test a novel, personalized digital intervention-my.naviGATE-for adolescent and young adults (AYA) with cancer. my.naviGATE is a mobile app that provides personalized survivorship education, access to virtual peer navigation, and responsive participant-reported outcomes (PROs).",[132,133,134,135,136,137,16,138,139],"Sarcoma","Osteosarcoma","Ewing Sarcoma","Rhabdomyosarcoma","Acute Myeloid Leukemia","Aml","Medulloblastoma","Acute Lymphoblastic Leukemia ,Lymphomas",[141,142,143,144,145,132,133,134,135,136,137,16,138],"Cancer Survivorship","Adolescent and Young Adult Cancer","Late Effects of Cancer Treatment","Hodgkin and mature B-cell Lymphomas","Acute Lymphoblastic Leukemia\u002FLymphoma","NOT_YET_RECRUITING","2026-03-31",{"date":108,"type":33},{"date":150,"type":21},"2026-08-20",{"date":152,"type":21},"2030-12-31",{"name":154,"class":40},"Dana-Farber Cancer Institute",{"id":156,"slug":157,"hasResults":11,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":4,"eligibilityCriteria":161,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":162,"enrollmentInfo":163,"targetDuration":4,"studyType":22,"phases":165,"briefSummary":167,"conditions":168,"keywords":176,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":187,"lastUpdatePostDateStruct":188,"startDateStruct":190,"completionDateStruct":192,"leadSponsor":194,"locationsCount":117},"100411935","phase-2-cord-blood-transplant-in-children-and-young-adults-with-blood-cancers-and-non-malignant-disorders-100411935","NCT04644016","Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders","Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders","Participant Inclusion Criteria:\n\nAge and Donor Status:\n\nPatients with age ≤ 21 years at time of consent with no available and suitably matched related or unrelated donor in the required time period.\n\nDiagnoses :\n\nI. Acute myelogenous leukemia (AML) :\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).\n  * Therapy-related AML (t-AML).\n  * White cell count at presentation \\> 100,000.\n  * Presence of extramedullary leukemia at diagnosis.\n  * Any unfavorable subtype by FAB or WHO classification.\n  * High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.\n  * Requirement for 2 or more inductions to achieve CR1.\n  * Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.\n  * Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.\n  * Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed AML with less than 10% blasts by bone marrow morphology. Patients with cytogenetic, flow cytometric, or molecular abnormalities in ≤ 10% of cells are eligible.\n\nII. Acute lymphoblastic leukemia (ALL):\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * White cell count at presentation \\> 30,000 for B-cell lineage and \\> 100,000 for T-cell lineage.\n  * Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.\n  * Failure to achieve complete remission (CR) after four weeks of induction therapy.\n  * Persistence or recurrence of MRD on therapy.\n  * Any patient unable to tolerate consolidation and\u002For maintenance chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed ALL with MRD disease after antibody therapy (e.g., blinatumomab, inotuzumab, other) and\u002For CAR-T cell therapy.\n\nIII. Other acute leukemias:\n\nLeukemias of ambiguous lineage or of other types (e.g. blastic plasmacytoid dendritic cell neoplasm) with less than 5% blasts by BM morphology. Patients with persistent\u002Frelapsed disease with cytogenetic, flow cytometric or molecular aberrations in ≤ 5% of cells are eligible.\n\nIV. Myelodysplastic Syndrome (MDS) \u002F Myeloproliferative Disorders (MPD) other than myelofibrosis:\n\n* International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis.\n* Any IPSS risk category if life-threatening cytopenia(s) exists.\n* Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia.\n* MDS\u002F myeloproliferative disorder overlap syndromes without myelofibrosis.\n* MDS\u002F MPD patients must have less than 10% bone marrow myeloblasts and ANC ≥ 0.2 (growth factor supported if necessary) at transplant work-up.\n\nV. Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high-risk of relapse or progression if not in remission:\n\n* Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR.\n* Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease\u002F CR\u002F PR with no single lesion equal to or more than 5 cm.\n* Eligible patients with HL will be those without progression of disease (POD) after salvage chemotherapy with no single lesion ≥ 5 cm.\n\nVI. Inherited Metabolic Disorders \\[also see EBMT Handbook for discussion on patient eligibility for allogeneic transplant; in general, patients are considered early in the disease course, before they develop neurologic symptoms (46)\\]:\n\n* Hurler Syndrome\n* Hunter (MPS 2 - early disease)\n* Sly syndrome (MPSVIII)\n* α-Mannosidosis\n* X- ALD\n* Osteopetrosis\n* Metachromatic Leukodystrophy\n* Globoid (GLD)\n\nVII. Non-Malignant disorders (other) \\[also see EBMT Handbook for criteria for transplant (46)\\]\n\n* Hemoglobinopathies\n* Bone Marrow Failure syndromes\n* Immunodeficiencies, including HLH\n\nOrgan Function and Performance Status Criteria:\n\n* Karnofsky or Lansky score ≥ 70% (see Appendix)\n* Bilirubin ≤ 1.5 mg\u002FdL (unless benign congenital hyperbilirubinemia).\n* ALT ≤ 3 x upper limit of normal.\n* Pulmonary function (spirometry and corrected DLCO) ≥ 50% predicted (corrected for hemoglobin) .\n* Left ventricular ejection fraction ≥ 50%.\n* Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7.\n* Renal: serum creatinine ≤ 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \\> 50 mL\u002Fmin\u002F1.73m2 (calculated or estimated) or GFR (mL\u002Fmin\u002F1.72m2) \\>30% of predicted normal for age.\n\nNormal GFR in Children and Young Adults (Age) : Mean GFR +- SD (mL\u002Fmin\u002F1.73 m2)\n\n1 week: 40.6 + \u002F - 14.8 2-8 weeks: 65.8 + \u002F - 24.8 \\>8 weeks: 95.7 + \u002F - 21.7 2-12 years: 133.0 + \u002F - 27.0 13-21 years (males): 140.0 + \u002F - 30.0 13-21 years (females: 126.0 + \u002F - 22.0\n\nGFR, glomerular filtration rate; SD, standard deviation greater than 2 years old: Normal GFR is 100 mL\u002F min. Infants: GFR must be corrected for body surf ace area.\n\nFor metabolic diseases: disease status to be evaluated according to EBMT Handbook \\[45\\].\n\nGraft Criteria\n\nCB units will be selected according to the current MSKCC unit selection algorithm. High resolution 8 allele HLA typing and recipient HLA antibody profile will be performed. Cord unit selection will occur based on HLA-match, total nucleated cell (TNC) and CD34+ cell dose adjusted per patient body weight. The cord bank of origin will also be considered. Donor specific HLA antibodies, if present, will also be taken into consideration and may influence the selection of the graft. CB graft will consist of one or two CB units (CBU) based on MSKCC selection algorithm.\n\n* Each CB unit must be at least 3\u002F8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.\n* For malignant diseases follow MSKCC CBU selection algorithm\n* For non-malignant diseases, CBU will be required to have \\> 5 x 107 TNC\u002Fkg; high HLA allele level match is preferable\n\nParticipant Exclusion Criteria:\n\n* Inadequate performance status\u002F organ function.\n* Advanced metabolic disease (EBMT handbook).\n* Active CNS leukemic involvement.\n* Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.\n* Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.\n* Autologous stem cell transplant within the preceding 6 months.\n* Any prior allogeneic stem cell transplant.\n* Active and uncontrolled infection (bacterial\u002Ffungal\u002Fviral) at time of transplantation.\n* HIV infection.\n* Seropositivity for HTLV-1.\n* Pregnancy or breast feeding.\n* Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.","21 Years",{"count":164,"type":21},31,[166],"PHASE2","This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.",[61,16,169,170,171,172,173,174,175],"MDS","MPD Withou Myelofibrosis","NHL or HL","Inherited Metabolic Disorders","Hemoglobinopathies","Bone Marrow Failure","HLH",[177,61,178,169,179,180,181,182,183,184,185,186],"Acute myelogenous leukemia","Myelodysplasia","Myeloproliferative Disorder","Therapy-Related AML and MDS","Therapy-Related Acute Myeloid Leukemia","T-AML","High-risk cytogenetics","Cord Blood Transplantation","20-480","Memorial Sloan Kettering Cancer Center","2026-03-12",{"date":189,"type":33},"2026-03-13",{"date":191,"type":33},"2020-11-20",{"date":193,"type":21},"2026-12-20",{"name":186,"class":40},{"id":196,"slug":197,"hasResults":11,"nctId":198,"briefTitle":199,"officialTitle":200,"acronym":4,"eligibilityCriteria":201,"healthyVolunteers":202,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":203,"targetDuration":205,"studyType":53,"phases":4,"briefSummary":206,"conditions":207,"keywords":209,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":212,"lastUpdatePostDateStruct":213,"startDateStruct":215,"completionDateStruct":217,"leadSponsor":219,"locationsCount":117},"100285049","amishmennonite-research-contact-registry-100285049","NCT02990572","Amish\u002FMennonite Research Contact Registry","Research Contact Registry for Members of the Amish and Mennonite Communities","Inclusion Criteria:\n\n* Amish and Mennonite men and women age 18 or older\n* Amish and Mennonite children with the permission of a parent\n\nExclusion Criteria:\n\n* Amish and Mennonite men and woman age 18 or older who do not speak English\n* Those who are not Amish or Mennonite",true,{"count":204,"type":21},1000,"100 Years","The purpose of this registry is to find members of the Plain Community who are interested in participating in future medical research studies. Having a registry makes it easier to contact and offer study participation to individuals who are appropriate for a new research study based on their own and their family medical history. Studies will focus on increasing knowledge about disorders affecting those who are Amish and Mennonite.",[208],"All",[210,211],"Amish","Mennonite","2026-01-27",{"date":214,"type":33},"2026-01-29",{"date":216,"type":4},"2016-10",{"date":218,"type":21},"2030-12",{"name":220,"class":40},"University of Pittsburgh",{"id":222,"slug":223,"hasResults":11,"nctId":224,"briefTitle":225,"officialTitle":226,"acronym":4,"eligibilityCriteria":227,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":228,"targetDuration":4,"studyType":22,"phases":229,"briefSummary":226,"conditions":230,"keywords":231,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":233,"lastUpdatePostDateStruct":234,"startDateStruct":236,"completionDateStruct":238,"leadSponsor":240,"locationsCount":117},"100612342","phase-2-a-multicenter-study-of-car-t-cells-in-primary-phall-100612342","NCT07252336","A Multicenter Study of CAR-T Cells in Primary Ph+All","A Multicenter Study of CD19 CAR-T Cells in the Treatment of Adult Patients With Primary Ph Chromosome Positive Acute Lymphoblastic Leukemia","Inclusion Criteria:\n\n1. Age ≥ 18 years old, gender not restricted;\n2. Subjects diagnosed with B-cell acute lymphoblastic leukemia (B-ALL) in accordance with the 2016 WHO Classification of Acute Leukemias;\n3. Abnormal B cells positive for CD19 and CD22 by immunophenotyping;\n4. Subjects positive for Philadelphia chromosome (Ph chromosome) and BCR\u002FABL1 fusion gene by chromosomal and corresponding genetic testing;\n5. Newly diagnosed B-ALL patients who have not received treatment with standard chemotherapy regimens;\n6. Serum total bilirubin ≤ 51 μmol\u002FL, serum ALT and AST both ≤ 3 times the upper limit of the normal reference range, and serum creatinine ≤ 176.8 μmol\u002FL;\n7. Left ventricular ejection fraction (LVEF) ≥ 50% as assessed by echocardiography;\n8. Subjects with no active pulmonary infection and oxygen saturation ≥ 92% without oxygen supplementation;\n9. Estimated survival time ≥ 3 months;\n10. ECOG performance status score 0-2;\n11. Females and males of childbearing potential must agree to use appropriate contraceptive measures before enrollment, during study participation, and within 6 months after infusion;\n12. Subjects voluntarily participate in this study and sign the informed consent form.\n\nExclusion Criteria:\n\n* Subjects meeting any of the following exclusion criteria are ineligible for enrollment in this study:\n\n  1. Subjects with a history of epilepsy or other central nervous system diseases;\n  2. Subjects with a prior history of QT interval prolongation or severe cardiac diseases;\n  3. Pregnant or lactating women (the safety of this therapy for unborn children is unknown);\n  4. Subjects with untreated active infections;\n  5. Subjects with serological evidence of chronic hepatitis B virus (HBV) infection who are unable or unwilling to receive standard prophylactic antiviral treatment, or have a detectable HBV viral DNA load; subjects with serological evidence of hepatitis C virus (HCV) infection who have not completed curative treatment or have a detectable HCV viral load;\n  6. Human immunodeficiency virus (HIV) antibody positive;\n  7. Syphilis antibody positive;\n  8. Subjects who have previously received any gene therapy products;\n  9. Subjects with other uncontrolled diseases who, in the investigator's judgment, are unsuitable for enrollment;\n  10. Any other conditions that, in the investigator's judgment, may increase the risk to the subject or interfere with the study results.",{"count":98,"type":21},[166],[16],[232],"CD19 CAR-T","2025-11-26",{"date":235,"type":33},"2025-12-04",{"date":237,"type":21},"2025-11-30",{"date":239,"type":21},"2028-11-30",{"name":241,"class":40},"Zhejiang University",{"id":243,"slug":244,"hasResults":11,"nctId":245,"briefTitle":246,"officialTitle":247,"acronym":4,"eligibilityCriteria":248,"healthyVolunteers":11,"sex":16,"minAge":124,"maxAge":249,"enrollmentInfo":250,"targetDuration":4,"studyType":22,"phases":252,"briefSummary":253,"conditions":254,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":257,"lastUpdatePostDateStruct":258,"startDateStruct":260,"completionDateStruct":262,"leadSponsor":264,"locationsCount":117},"100511555","phase-2-inotuzumab-ozogamicin-in-the-treatment-of-mrd-after-hsct-of-all-100511555","NCT05940961","Inotuzumab Ozogamicin in the Treatment of MRD+ After HSCT of ALL","A Multicenter Prospective Clinical Study of Inotuzumab Ozogamicin (INO) in the Treatment of Minimal Residual Disease Recurrent After Hematopoietic Stem Cell Transplantation of Acute Lymphoblastic Leukemia (ALL)","Inclusion Criteria:\n\n1. Patients aged ≥ 15 and ≤ 65 years.\n2. Patients diagnosed with CD22+ B-ALL according to 2023 NCCN Acute Lymphoblasts Leukaemias diagnosis standard.\n3. CD22+ B-ALL patients with MRD recurrence after HSCT. Ph+ ALL patients were eligible if treatment with 1 or more second-generation BCR::ABL1 tyrosine kinase inhibitors (TKIs) had failed,\n4. ECOG performance status score less than 3.\n5. Expected survival time #3 months.\n6. Patients without serious heart, lung, liver, or kidney disease.\n7. Ability to understand and voluntarily provide informed consent.\n\nExclusion Criteria:\n\n1. Patients who are allergic to the study drug or drugs with similar chemical structures.\n2. Pregnant or lactating women, and women of childbearing age who do not want to practice effective methods of contraception.\n3. Active infection.\n4. Active bleeding.\n5. Patients with new thrombosis, embolism, cerebral hemorrhage, or other diseases or a medical history within one year before enrollment.\n6. Patients with mental disorders or other conditions whereby informed consent cannot be obtained and where the requirements of the study treatment and procedures cannot be met.\n7. Liver function abnormalities (total bilirubin \\> 1.5 times the upper limit of the normal range, ALT\u002FAST \\> 2.5 times the upper limit of the normal range or patients with liver involvement whose ALT\u002FAST \\> 1.5 times the upper limit of the normal range), or renal anomalies (serum creatinine \\> 1.5 times the upper limit of the normal value).\n8. Patients with a history of clinically significant QTc interval prolongation (male \\> 450 ms; female \\> 470 ms), ventricular heart tachycardia and atrial fibrillation, II-degree heart block, myocardial infarction attack within one year before enrollment, and congestive heart failure, and patients with coronary heart disease who have clinical symptoms and requiring drug treatment.\n9. Surgery on the main organs within the past six weeks.\n10. Drug abuse or long-term alcohol abuse that would affect the evaluation results.\n11. Patients who have received organ transplants (excepting bone marrow transplantation).\n12. Patients not suitable for the study according to the investigator's assessment.","65 Years",{"count":251,"type":21},42,[166],"As part of postremission consolidative therapy, the decision to proceed with hematopoietic stem cell transplantation is a recommendable regimen in ALL therapy. However, The recurrence rate is high after transplantation. Minimal Residual Disease (MRD) is an important factor affecting the effect of HSCT. The hematologic recurrence rate of MRD-positive patients with adult ALL is high.\n\nMRD- is associated with better prognosis. Therefore, maintaining MRD- after transplantation is necessary for long-term survival. The purpose of this study is to explore the efficacy and safety of Inotuzumab Ozogamicin in the treatment of minimal residual disease recurrence after HSCT of ALL patients.",[16,255,256],"MRD-positive","Hematopoietic Stem Cell Transplantation","2025-11-18",{"date":259,"type":33},"2025-11-21",{"date":261,"type":33},"2022-08-01",{"date":263,"type":21},"2026-08-01",{"name":265,"class":40},"Sheng-Li Xue, MD",{"id":267,"slug":268,"hasResults":11,"nctId":269,"briefTitle":270,"officialTitle":271,"acronym":272,"eligibilityCriteria":273,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":274,"enrollmentInfo":275,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":276,"conditions":277,"keywords":278,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":286,"lastUpdatePostDateStruct":287,"startDateStruct":289,"completionDateStruct":291,"leadSponsor":293,"locationsCount":41},"100591154","a-long-term-follow-up-study-of-tscan-tcr-t-products-100591154","NCT06976736","A Long Term Follow-up Study of TScan TCR-T Products","A Long-term Follow-up Study to Assess Safety in Participants Who Received an Investigational T-Cell Receptor Engineered T-Cell (TCR-T) Product","LTFU","Inclusion Criteria:\n\n* Participants who received a TCR-T cellular therapy in a clinical study sponsored by TScan Therapeutics.\n* Signed informed consent.\n\nExclusion Criteria:\n\n* None","110 Years",{"count":204,"type":21},"The purpose of this Long-Term Follow-Up (LTFU) study is to monitor participants who have previously received TSC-100 or TSC-101 TCR-T therapies in the TSCAN-001 study. Participants will be monitored for 15 years from the date of TCR-T cell therapy administration to assess long-term safety and efficacy.",[61,16,169],[279,280,281,282,283,284,285],"TSCAN","Cell Therapies","TCR-T Cell Therapy","Long Term Follow-Up (LTFU)","TSC-101","TSC-100","TSCAN-001","2025-09-02",{"date":288,"type":33},"2025-09-10",{"date":290,"type":21},"2025-09-09",{"date":292,"type":21},"2040-09",{"name":294,"class":295},"TScan Therapeutics, Inc.","INDUSTRY",{"id":297,"slug":298,"hasResults":11,"nctId":299,"briefTitle":300,"officialTitle":301,"acronym":302,"eligibilityCriteria":303,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":304,"enrollmentInfo":305,"targetDuration":4,"studyType":22,"phases":307,"briefSummary":308,"conditions":309,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":313,"lastUpdatePostDateStruct":314,"startDateStruct":316,"completionDateStruct":318,"leadSponsor":320,"locationsCount":117},"100342505","phase-2-a-randomized-phase-ii-study-of-hyperbaric-oxygen-in-improving-engraftment-in-umbilical-cord-blood-stem-cell-transplant-100342505","NCT03739502","A Randomized Phase II Study of Hyperbaric Oxygen in Improving Engraftment in Umbilical Cord Blood Stem Cell Transplant","A Randomized Phase II Study Evaluating the Efficacy of Hyperbaric Oxygen in Improving Engraftment in Umbilical Cord Blood Stem Cell Transplantation","HBO-UBC","Inclusion Criteria:\n\n* Voluntary written informed consent\n* Patients who are considered for allogeneic transplantation based on their disease risk (see below) but lack matched sibling or unrelated donors or who are unable to proceed to allogeneic transplant within 8 weeks, will be considered for UCB transplantation on this study. Only patients for whom RIC will be considered are eligible. RIC is considered in those older than 45 or younger than 45 with Hematopoietic Cell Transplant (HCT) Comorbidity Index of 3 or higher (HCT) Comorbidity Index can be calculated using the following link: http:\u002F\u002Fwww.hctci.org\u002FHome\u002FCalculator\n* Patients with acute myeloid leukemia (AML) in CR1 that is not considered favorable-risk (favorable risk is defined as patients with t(15;17)(q22;q21), t(8;21)(q22;q22), inv(16)(p13q22)\u002Ft(16;16)(p13;q22), NPM1 mutation without FLT3-ITD, and double-mutated CEBPA58,59), AML in CR2 or subsequent CR, high-risk acute lymphoblastic leukemia (ALL) in CR1, or ALL in CR2 or higher, biphenotypic leukemia defined as coexpression of B-lymphoid and myeloid markers or T-lymphoid and myeloid markers in the blast population60or undifferentiated leukemia in ≥CR1. Myelodysplatic syndrome (MDS)\u002Fmyeloproliferative neoplasm (MPN) patients with less than 10% bone marrow blasts and no peripheral blood blasts on pre-transplant bone marrow aspirate\u002Fbiopsy are considered for FluCyTTBI regimen. Chemotherapy sensitive (achievement of at least a partial response according to Lugano classification61) Hodgkin's disease (HD) that relapsed following high-dose therapy. Chemotherapy sensitive (achievement of at least a partial response according to Lugano classification) non-Hodgkin's lymphoma (NHL) patients who relapsed post-high-dose therapy and autologous transplantation. Subjects should be enrolled within 30 days of transplant.\n* For ALL, high-risk features are defined using modified Hoelzer risk criteria62, these criteria are:\n\n  1. High white blood cell count at diagnosis (ie, \\>30,000\u002FmicroL in B-ALL or \\>100,000\u002FmicroL in T-ALL).\n  2. Clonal cytogenetic abnormalities - t(4;11), t(1;19), t(9;22), or BCR-ABL gene positivity.\n  3. Progenitor-B cell immunophenotype (eg, blasts expressing membrane CD19, CD79a, and cytoplasmic CD22).\n  4. Length of time from start of induction therapy to attainment of CR greater than four weeks.\n  5. Older age - \\>60 years old is high risk, 30 to 59 years old is intermediate risk.\n  6. MRD - a post-remission bone marrow MRD level ≥10-3 by molecular tests.\n* Subjects must be ≥ 18 years old and ≤ 70 years old\n* Karnofsky performance status (KPS) of ≥ 70% (Appendix A).\n* Adequate hepatic, renal, cardiac and pulmonary function to be eligible for transplant. Minimum criteria include:\n\n  * ALT, AST: \\\u003C 4x IULN\n  * Total bilirubin: ≤ 2.0 mg\u002FdL\n  * Creatinine: ≤ 1.5 x ULN\n  * EF measured by 2D-ECHO or MUGA scan of ≥ 45%\n  * FEV1, FVC and DLCO ≥ 50% of predicted value (corrected to serum hemoglobin).\n  * EKG with no clinically significant arrhythmia.\n* Patients should have New York Heart Association (NYHA) Functional Classification, class -1 (ordinary physical activity does not cause undue fatigue, palpitation, dyspnea, or angina pain) or class II (ordinary physical activity results in fatigue, palpitation, dyspnea, or angina pain).\n* Patients should be evaluated for fitness for HBO by a hyperbaric oxygen trained medical professional who is not part of the study team prior to starting preparative regimen.\n* Women of child-bearing potential should have a negative urine pregnancy test within 4 weeks of starting preparative regimen.\n* Women of child-bearing potential and men with partners of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 30 days following completion of therapy. Should a woman or partner become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician and the investigator immediately.\n* A woman of child-bearing potential is any female (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:\n\n  1. Has not undergone a hysterectomy or bilateral oophorectomy; or\n  2. Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months)\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding\n* Severe chronic obstructive pulmonary disease requiring oxygen supplementation\n* History of spontaneous pneumothorax\n* Active ear\u002Fsinus infection. Patients with chronic sinusitis or sinus headaches are excluded unless cleared by ear, nose, throat provider.\n* Evidence of pneumothorax or significant pulmonary fibrosis on chest imaging within 60 days of transplant.\n* Prior chest surgery requiring thoracotomy or direct chest irradiation.\n* Recent of sinus or ear surgery, excluding myringotomy or ear tubes (within the last 5 years).\n* Claustrophobia\n* Patients who had intrathecal chemotherapy within 2 weeks of starting preparative regimen or cranial irradiation within 4 weeks of starting preparative regimen.\n* History of seizures\n* No active tobacco use 72 hours prior to transplant until complete transplant recovery.","70 Years",{"count":306,"type":21},64,[166],"The UCB transplant is a type of stem cell transplant used to treat cancer of the blood or lymph glands. The UCB transplant has advantages over other types of transplants such as ease of obtaining the umbilical cord blood, absence of donor risks, reduced risks of contagious infections, and the availability for immediate use. The UCB transplant is also associated with a lower incidence of graft versus host disease, or GvHD (in GvHD, the transplanted graft attacks the recipient organs).",[61,310,311,208,312],"NHL","Hodgkin Disease","Myelodysplastic Syndrome","2025-07-02",{"date":315,"type":33},"2025-07-08",{"date":317,"type":33},"2019-02-28",{"date":319,"type":21},"2027-06",{"name":321,"class":40},"University of Rochester",{"id":323,"slug":324,"hasResults":11,"nctId":325,"briefTitle":326,"officialTitle":327,"acronym":4,"eligibilityCriteria":328,"healthyVolunteers":11,"sex":16,"minAge":329,"maxAge":162,"enrollmentInfo":330,"targetDuration":4,"studyType":22,"phases":332,"briefSummary":333,"conditions":334,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":341,"lastUpdatePostDateStruct":342,"startDateStruct":344,"completionDateStruct":346,"leadSponsor":348,"locationsCount":164},"100475887","phase-1-tagraxofusp-in-pediatric-patients-with-relapsed-or-refractory-cd123-expressing-hematologic-malignancies-100475887","NCT05476770","Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies","A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies","Inclusion Criteria:\n\nAge\n\n* Patients must be ≥ 1 and ≤21 years of age at the time of study enrollment.\n\nDiagnosis\n\n* Relapsed and\u002For refractory hematologic malignancy (including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma).\n* Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local institution.\n\nDisease Status:\n\nMonotherapy, Part 1\n\n* Second or greater relapse; or\n* Refractory after 2 or more chemotherapy cycles; or\n* First relapse after primary chemotherapy-refractory disease; or\n* BPDCN in first relapse or refractory after 1 or more chemotherapy cycles\n\nCombination therapy, Part 2\n\n* First or greater relapse; or\n* Refractory after 2 or more chemotherapy cycles; or\n* BPDCN in first relapse or refractory after 1 or more chemotherapy cycles\n\nFor relapsed\u002Frefractory leukemia, patients must have:\n\n* \\>5% blasts in the bone marrow aspirate or biopsy by morphology or flow cytometry\n* Patients with 1% - 5% blasts are eligible for Part 2, Cohort C (only), if A single bone marrow sample with flow cytometry and at least one other test (e.g. karyotype, FISH, PCR, or NGS) shows ≥ 1% leukemic blasts and\u002For flow cytometry demonstrates a stable or rising level of disease on two serial bone marrows.\n\nFor relapsed\u002Frefractory non-Hodgkin or Hodgkin lymphoma, patients must have:\n\n* Histologic verification of relapse\n* Measurable disease documented by radiographic criteria or bone marrow\n* Patients in Part 1 may have sites of non-CNS extramedullary disease, but no CNS disease. Patients in Part 2 may have CNS disease and\u002For other non-CNS extramedullary disease. No cranial irradiation is allowed during the protocol therapy.\n* Patients with Down syndrome are eligible to participate in Part 1 only.\n\nPerformance Level\n\n* Karnofsky \\> 50% for patients \\> 16 years of age and Lansky \\> 50% for patients ≤ 16 years of age (See Appendix I for Performance Scales). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n\nPrior Therapy\n\n* Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy, defined as resolution of all such toxicities to ≤ Grade 2 or lower per the inclusion\u002Fexclusion criteria.\n\nMyelosuppressive chemotherapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study. At least 14 day must have elapsed since the completion of myelosuppressive therapy. However, individuals may receive any of the following medications within 14 days without a \"wash-out period\":\n\n* Hydroxyurea: Hydroxyurea can be initiated and\u002For continued for up to 24 hours prior to the start of protocol therapy.\n* \"Maintenance-style\" therapy: therapy including vincristine (dosed a maximum of one-time weekly), oral 6-mercaptopurine, oral methotrexate (dosed a maximum of one-time weekly), intrathecal therapy (dosed a maximum of one-time weekly) and\u002For dexamethasone (dosed at ≤3 mg\u002Fm2\u002Fdose twice daily) or prednisone (dosed at ≤20 mg\u002Fm2\u002Fdose twice daily) can be continued for up to 24 hours prior to entering the study.\n* Hematopoietic stem cell transplant: Patients who have experienced their relapse after a HSCT are eligible, provided they have no evidence of acute or chronic Graft-versus-Host Disease (GVHD) and are at least 100 days post-transplant at the time of enrollment.\n* Hematopoietic growth factors: It must have been at least 7 days since the completion of therapy with granulocyte colony stimulating factor (GCSF) or other growth factors at the time of enrollment. It must have been at least 14 days since the completion of therapy with pegfilgrastim (Neulasta®).\n* Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.\n* Monoclonal antibodies: Maximum of 3 half-lives of the antibody or 21 days (whichever is shorter) must have elapsed after the last dose of monoclonal antibody.\n* Immunotherapy: At least 30 days from last infusion of chimeric antigen receptor T cell (CART) therapy or tumor vaccine.\n* Radiation Therapy (XRT):\n\n  1. ≥ 84 days must have passed, from the end of therapy, if patient received prior total body irradiation (TBI).\n  2. ≥ 42 days must have passed, from the end of therapy, if patient received craniospinal irradiation (CSI).\n  3. ≥ 14 days must have passed after whole brain radiotherapy or stereotactic radiation therapy.\n  4. No washout period is required for:\n\n  i. Extramedullary site other than CNS that is a maximum 10 x 10 cm total radiation non-CNS field. If the field is \\> 10 x 10 cm, a 14-day washout period is required. ii. Local ocular radiotherapy as long as subject has measurable\u002Fevaluable disease outside the radiation port.\n* Patients that have received other non-tagraxofusp CD123 targeting agents are eligible. Patients that have previously received tagraxofusp are not eligible.\n\nOrgan Function Requirements\n\nAdequate Bone Marrow Function Defined as:\n\n* Patients should not be known to be refractory to red blood cell or platelet transfusions.\n* Blood counts are not required to be normal prior to enrollment on trial. However, platelet count must be ≥20,000\u002Fmm3 to initiate therapy (may receive platelet transfusions).\n\nAdequate Renal Function Defined as:\n\n* Patient must have a calculated creatinine clearance or radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73m2 OR a normal serum creatinine based on age\u002Fgender in the chart below:\n\nMaximum Serum Creatinine (mg\u002FdL):\n\n* 1 to \\\u003C 2 years old - Male: 0.6, Female: 0.6\n* 2 to \\\u003C 6 years old - Male:0.8, Female: 0.8\n* 6 to \\\u003C 10 years old - Male: 1, Female: 1\n* 10 to \\\u003C 13 years old - Male: 1.2, Female: 1.2\n* 13 to \\\u003C 16 years old - Male: 1.5, Female: 1.4\n* ≥ 16 years old - Male: 1.7, Female: 1.4\n\nThe threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.\n\nAdequate Liver Function Defined as:\n\n* Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x institutional upper limit of normal for age\n* SGPT (ALT) and SGOT (AST) must be less than 3x institutional upper limit of normal.\n* Serum albumin ≥3.2 g\u002FdL (albumin infusion independent).\n\nAdequate Cardiac Function Defined as:\n\n* Shortening fraction of ≥27% by echocardiogram, or\n* Ejection fraction of ≥ 50% by gated radionuclide study\u002Fechocardiogram.\n\nAdequate Pulmonary Function Defined as:\n\n* Pulse oximetry \\> 94% on room air (\\> 90% if at high altitude)\n* No evidence of dyspnea at rest and no exercise intolerance.\n\nReproductive Function\n\n* Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 2 weeks prior to enrollment.\n* Female patients with infants must agree not to breastfeed their infants while on this study.\n* Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study and for 12 weeks after the last dose of tagraxofusp.\n\nExclusion Criteria\n\nDisease Status:\n\n* Patients with CNS disease are not eligible for Part 1.\n* Patients with isolated CNS disease are not eligible for Part 1 or Part 2.\n* Patients with isolated non-CNS disease are eligible for Part 1 and Part 2.\n\nConcomitant Medications\n\n* Corticosteroids - Patients receiving corticosteroids for disease control who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.\n* Investigational Drugs - Patients who are currently receiving another investigational drug are not eligible. The definition of \"investigational\" for use in this protocol means any drug that is not licensed by the FDA, Health Canada or the Therapeutic Goods Administration to be sold in the countries they govern. (United States, Canada and Australia)\n* Anti-cancer Agents - Patients who are currently receiving or may receive while on therapy, other anti-cancer agents, radiation therapy or immunotherapy are not eligible \\[with the exceptions being laid out in the inclusion criteria under 'Prior Therapy'\\]. Intrathecal chemotherapy (at the discretion of the primary oncologist) may be given up to one week prior to the initiation of study treatment (day 1 therapy).\n* Anti-GVHD or agents to prevent organ rejection post-transplant - Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial. At least 4 weeks must have elapsed after the last dose of GVHD meds.\n\nInfection Criteria - Patients are excluded if they have:\n\n* Positive blood culture within 48 hours of study enrollment;\n* Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection. Fever that is determined to be due to tumor burden is allowed if patients have documented negative blood cultures for at least 48 hours prior to enrollment and no concurrent signs or symptoms of active infection or hemodynamic instability.\n* A positive fungal culture within 30 days of study enrollment.\n* Active fungal, viral, bacterial, or protozoal infection requiring IV treatment. Chronic prophylaxis therapy to prevent infections is allowed.\n* Patients will be excluded if they have a known allergy to any of the drugs used in the study.\n* Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures, interfere with consent, study participation, follow up, or interpretation of study results.\n* Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.","1 Year",{"count":331,"type":21},54,[24],"Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). This trial aims to examine the safety of this novel agent in pediatric patients with relapsed\u002Frefractory hematologic malignancies.\n\nThe mechanism by which tagraxofusp kills cells is distinct from that of conventional chemotherapy. Tagraxofusp directly targets CD123 that is present on tumor cells, but is expressed at lower or levels or absent on normal hematopoietic stem cells. Tagraxofusp also utilizes a payload that is not cell cycle dependent, making it effective against both highly proliferative tumor cells and also quiescent tumor cells.\n\nThe rationale for clinical development of tagraxofusp for pediatric patients with hematologic malignancies is based on the ubiquitous and high expression of CD123 on many of these diseases, as well as the highly potent preclinical activity and robust clinical responsiveness in adults observed to date.\n\nThis trial includes two parts: a monotherapy phase and a combination chemotherapy phase. This design will provide further monotherapy safety data and confirm the FDA approved pediatric dose, as well as provide safety data when combined with chemotherapy.\n\nThe goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to describe the toxicities, pharmacokinetics, and pharmacodynamic properties of tagraxofusp in pediatric patients.\n\nAbout 54 children and young adults will participate in this study. Patients with Down syndrome will be included in part 1 of the study.",[56,61,16,335,169,336,337,338,66,339,340],"BPDCN","Lymphoblastic Lymphoma","Lymphoma, B-Cell","Lymphoma, T-Cell","Mixed Phenotype Acute Leukemia","Acute Undifferentiated Leukemia","2024-12-04",{"date":343,"type":33},"2024-12-06",{"date":345,"type":33},"2022-11-11",{"date":347,"type":21},"2027-11-11",{"name":349,"class":40},"Therapeutic Advances in Childhood Leukemia Consortium",{"id":351,"slug":352,"hasResults":11,"nctId":353,"briefTitle":354,"officialTitle":354,"acronym":355,"eligibilityCriteria":356,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":357,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":359,"conditions":360,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":362,"lastUpdatePostDateStruct":363,"startDateStruct":365,"completionDateStruct":367,"leadSponsor":369,"locationsCount":371},"100439995","optical-genome-mapping-in-hematological-malignancies-100439995","NCT05009537","Optical Genome Mapping in Hematological Malignancies","CARTOGEN","Inclusion Criteria:\n\n* Minor\u002FMajor Patients\n* Patients with suspected hemopathies for whom we receive a sample for diagnosis in the chromosomal genetics laboratory or for whom a diagnosis of hemopathy has already been made.\n* No objection made or consent given\n\nExclusion Criteria:\n\n* Patients under judicial protection (guardianship, curatorship, ...),\n* Refusal to participate",{"count":358,"type":21},200,"Establish the diagnostic potential of optical genome mapping in patients with suspected hematologic cancer",[169,16,361],"Other Hematologic Malignant Neoplasms","2021-08-16",{"date":364,"type":33},"2021-08-17",{"date":366,"type":21},"2021-09",{"date":368,"type":21},"2026-09",{"name":370,"class":40},"University Hospital, Brest",4]