[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"bpdcn\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:bpdcn":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,43],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":16,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":29,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":5},"100386972","phase-1-cd123-directed-t-cell-therapy-for-acute-myelogenous-leukemia-catchaml-100386972",false,"NCT04318678","CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)","Inclusion Criteria for Procurement and T-cell Production:\n\n* Age ≤21 years old\n* Relapsed\u002Frefractory CD123+ disease defined as follows:\n\nAML\u002FMDS\n\n* Relapsed disease: Patients developing recurrent disease after a first complete remission (CR)\n* Refractory disease: Patients not achieving a CR after 2 cycles of induction chemotherapy\n\nB-cell ALL\n\n* Relapsed disease that is CD123 positive and CD19 negative\u002Fdim or patients otherwise ineligible for CD19 directed therapies including\n\n  * Patients in 2nd or greater relapse\n  * Patients with relapse after allogeneic HSCT\n* Refractory disease that is CD123 positive and CD19 negative\u002Fdim or patients otherwise ineligible for CD19 directed therapies\n\nT-cell All • Relapsed refractory disease that is CD123 positive\n\nBPDCN\n\n• Relapsed\u002Frefractory disease that has failed front-line therapy\n\n* Estimated life expectancy of \\>12 weeks\n* Karnofsky or Lansky (age-dependent) performance score ≥50\n* Patients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to apheresis\n* Patient must have an identified, suitable HCT donor\n* For females of child-bearing age:\n* Not lactating with intent to breastfeed\n* Not pregnant with negative serum pregnancy test within 7 days prior to enrollment\n* Meets eligibility criteria to undergo autologous apheresis, or have previously undergone autologous apheresis\n\nExclusion Criteria:\n\n* Known primary immunodeficiency\n* History of HIV infection\n* Severe intercurrent uncontrolled bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection)\n* History of hypersensitivity reactions to murine protein-containing products\n* Patients with acute promyelocytic leukemia (APL, t (15;17))\n* Known contraindication to the protocol defined lymphodepleting chemotherapy regimen of fludarabine\u002Fcyclophosphamide.\n\nInclusion Criteria for Treatment:\n\n* Age≤21 years old\n* Detectable disease that is CD123+ (at least MRD+ disease)\n* Estimated life expectancy of \\>8 weeks\n* Karnofsky or Lansky (age-dependent) performance score≥50\n* Patients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to planned infusion\n* Patient must have an identified, suitable HCT donor\n* Adequate cardiac function defined as left ventricular ejection fraction \\>40%, OR shortening fraction ≥25%\n* EKG without evidence of clinically significant arrhythmia\n* Adequate renal function defined as creatinine clearance or radioisotope GFR ≥50 ml\u002Fmin\u002F1.73m2 (GFR ≥40 ml\u002Fmin\u002F1.73m2 if \\\u003C 2 years of age)\n* Adequate pulmonary function defined as forced vital capacity (FVC)≥50% of predicted value; or pulse oximetry≥92% on room air if patient is unable to perform pulmonary function testing\n* Total Bilirubin≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome\n* Alanine aminotransferase (ALT) OR aspartate aminotransferase (AST) ≤5 times the upper limit of normal for age\n* Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy\n* For females of child-bearing age\n\n  * Not lactating with intent to breastfeed\n  * Not pregnant with negative serum pregnancy test within 7 days prior to enrollment\n* If sexually active, agreement to use birth control until 3 months after T- cell infusion. Male partners should use a condom.\n* Available autologous transduced T-cell product that has met GMP release criteria\n\nExclusion Criteria:\n\n* Known primary immunodeficiency\n* History of HIV infection\n* Severe intercurrent uncontrolled bacterial, viral or fungal infection\n* History of hypersensitivity reactions to murine protein-containing products\n* History of severe hypersensitivity reactions to cornstarch or hydroxyethyl starch.\n* Receiving systemic steroids therapy exceeding the equivalent of 0.5 mg\u002Fkg\u002Fday of methylprednisolone, in the 7 days prior to CD123-CAR T- cell infusion\n* Receiving systemic therapy in the 14 days prior to CD123-CAR T-cell infusion, which will interfere with the activity of the CD123-CAR T cells in vivo (in the opinion of the study PI(s))\n* Receiving rituximab therapy in the 30 days prior to CD123-CAR T cell infusion. (This exclusion criterion is intended to prevent premature exposure of CD123-CAR T cells to rituximab, which would activate the safety switch and promote CAR T-cell apoptosis).\n* Receiving intrathecal chemotherapy in the 7 days prior to CD123-CAR T cell infusion.\n* Known contraindication to the protocol defined lymphodepleting chemotherapy regimen of fludarabine\u002Fcyclophosphamide.\n* Active CNS disease","ALL","21 Years",{"count":18,"type":19},108,"ESTIMATED","INTERVENTIONAL",[22],"PHASE1","The CD123-CAR T-cell therapy is a new treatment that is being investigated for treatment of AML\u002Fmyelodysplastic syndrome (MDS), T- or B- acute lymphoblastic leukemia (ALL) or blastic plasmacytoid dendritic cell neoplasia (BPDCN). The purpose of this study is to find the maximum (highest) dose of CD123-CAR T cells that is safe to give to these patients. This would include studying the side effects of the chemotherapy, as well as the CD123-CAR T-cell product on the recipient's body, disease and overall survival.\n\nPrimary Objective:\n\n* To determine the safety of one intravenous infusion of escalating doses of autologous, CD123-CAR T cells in patients (≤21 years) with recurrent\u002Frefractory CD123+ disease (AML\u002FMDS, B-ALL, T-ALL or BPDCN) after lymphodepleting chemotherapy.\n* To determine the safety of an intravenous infusion of escalating doses of donor derived, CD123-CAR T cells in patients (≤21 years) with recurrent\u002Frefractory CD123+ disease (AML\u002FMDS, B-ALL, T-ALL, BPDCN or MPAL) after lymphodepleting chemotherapy.\n\nSecondary Objectives\n\n\\- To evaluate the antileukemia activity of CD123-CAR T cells.\n\nExploratory Objectives\n\n* To assess the immunophenotype, clonal structure and endogenous repertoire of CD123-CAR T cells and unmodified T cells\n* To characterize the cytokine profile in the peripheral blood and CSF after treatment with CD123-CAR T cells\n* To characterize tumor cells post CD123-CAR T-cell therapy\n* To compare in vivo properties of donor-derived versus autologous CD123- CAR T cells",[25,26,27,28],"AML\u002FMDS","B-ALL","T-ALL","BPDCN",[30],"CD123+","RECRUITING","2026-05-18",{"date":34,"type":35},"2026-05-19","ACTUAL",{"date":37,"type":35},"2020-07-29",{"date":39,"type":19},"2030-07-29",{"name":41,"class":42},"St. Jude Children's Research Hospital","OTHER",{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":15,"minAge":50,"maxAge":16,"enrollmentInfo":51,"targetDuration":4,"studyType":20,"phases":53,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":74},"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":52,"type":19},54,[22],"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.",[56,57,15,28,58,59,60,61,62,63,64],"Hematologic Malignancy","AML","MDS","Lymphoblastic Lymphoma","Lymphoma, B-Cell","Lymphoma, T-Cell","Hodgkin Lymphoma","Mixed Phenotype Acute Leukemia","Acute Undifferentiated Leukemia","2024-12-04",{"date":67,"type":35},"2024-12-06",{"date":69,"type":35},"2022-11-11",{"date":71,"type":19},"2027-11-11",{"name":73,"class":42},"Therapeutic Advances in Childhood Leukemia Consortium",31]