[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"amlmds\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:amlmds":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,44,69],{"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":43},"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",2,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":51,"sex":15,"minAge":52,"maxAge":4,"enrollmentInfo":53,"targetDuration":4,"studyType":20,"phases":55,"briefSummary":56,"conditions":57,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":43},"100515033","phase-1-danvatirsen-monotherapy-followed-by-combination-with-venetoclax-in-relapsedrefractory-mds--aml-100515033","NCT05986240","Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed\u002FRefractory MDS & AML","A Phase I Study Investigating the Safety & Efficacy of Danvatirsen as Monotherapy Followed by Combination With Venetoclax in Patients With Relapsed\u002FRefractory MDS & AML","Inclusion Criteria:\n\n* Subjects must be at least 18 years of age at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and be able to meet all study requirements\n* Morphologically confirmed diagnosis of AML or MDS in accordance with World Health Organization (WHO) diagnostic criteria\n* Subjects with relapsed\u002Frefractory AML who are refractory or relapsed to all conventional therapy and do not have any FDA approved or standard therapeutic options \\& subjects with intermediated\u002Fhigh\u002Fvery high IPSS-R MDS who are refractory or relapsed to at least 2 cycles of hypomethylating agent based therapy (azacitidine \u002F decitabine based) OR patients with rapid progression of disease regardless of number of cycles of therapy\n* At least 3 months from Allogenic stem cell transplantation and no clinical sign of active graft vs host disease (GVHD)\n* WBC must be \\\u003C25,000 cells\u002FuL and may be reduced with hydroxyurea to reach this goal prior to study start. Hydroxyurea can be administered on trial with an increase in WBC counts at the discretion of the PI\n* A bone marrow biopsy must be performed within the screening window (day-28-day-1) and tissue collected for correlative analysis for entrance to this trial. Correlative sample collection is essential on this study\n* Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2\n* Recovery to ≤ Grade 1 or baseline for any toxicities considered to be due to prior systemic treatments, excluding alopecia\n* Must have adequate hepatic and renal function as follows:\n\nALT (SGPT) and\u002For AST (SGOT) ≤ 3x upper limit of normal (ULN) or ≤ 5x ULN if considered to be leukemia related; Direct bilirubin ≤ 1.5 x ULN or ≤ 3x ULN (in patients with known Gilberts syndrome or if considered to be leukemia related)\n\n* Serum creatinine clearance ≥ 45 mL\u002Fmin\u002F1.73 m2 either measured or calculated using standard Cockroft-Gault formula\n* Subjects enrolled within childbearing ages of 18-50 years should use 2 forms of contraception while on study\n\nExclusion Criteria:\n\n* Acute Promyelocytic Leukemia\n* Low or very low risk MDS by IPSS-R after failure\u002Fprogression of first line therapy with hypomethylating agents\n* Active, uncontrolled infection. Patients with infection under active treatment and controlled with antibiotics are eligible. Use of prophylactic anti-microbials per institutional standards is allowed\n* Active documented central nervous system (CNS) leukemia. Patients with a known history of CNS leukemia will be eligible if they have at least two most recent consecutive LPs showing clearance of CNS disease and no active\u002Fprogressive symptoms thought to be related to the CNS disease\n* Concurrent treatment with a non-permitted concomitant medication (as noted in protocol appendix)\n* Concurrent anticancer treatment, major surgery, or the use of any investigational drug within 14 days before the start of trial treatment\n* Other malignancy currently being treated or likely to need treatment in next 6 months with the exception of basal or squamous cell carcinoma of the skin or cervical carcinoma in situ, surgically removed malignancies or malignancies definitively treated with chemotherapy, XRT and\u002For surgery with no evidence of active malignancy or not anticipated to need treatment in next 6 months or malignancies on maintenance therapy (e.g. tamoxifen for breast cancer) will be allowed after discussion and approval by both MPIs\n* Pregnant or breastfeeding females\n* Known current alcohol or drug abuse\n* Clinically significant cardiovascular disease within the past 6 months (e.g. percutaneous intervention, coronary artery bypass graft, documented NYHA class III\u002FIV cardiac heart failure, unstable angina or MI, poorly controlled atrial or ventricular arrhythmia) as determined by the investigator\n* Any psychiatric condition that would prohibit the understanding or rendering of informed consent\n* Legal incapacity or limited legal capacity to sign consent and\u002For participate in the trial\n* Any condition deemed by the investigator to make the patient a poor candidate for clinical trial and\u002For treatment with investigational agents\n* Previous exposure to the investigational agent (danvatirsen)",true,"18 Years",{"count":54,"type":19},38,[22],"This is a Phase 1 study investigating the safety and efficacy of Danvatirsen as a monotherapy followed by combination with Venetoclax in patients with relapsed\u002Frefractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML).\n\nFunding Source: FDA OOPD",[25,58,59],"Acute Myeloid Leukemia","Myelodysplastic Syndromes","2025-11-06",{"date":62,"type":35},"2025-11-10",{"date":64,"type":35},"2024-05-08",{"date":66,"type":19},"2030-03",{"name":68,"class":42},"Montefiore Medical Center",{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":75,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":15,"minAge":52,"maxAge":77,"enrollmentInfo":78,"targetDuration":4,"studyType":20,"phases":80,"briefSummary":83,"conditions":84,"keywords":85,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":99},"100492007","phase-2-innate-donor-effector-allogeneic-lymphocyte-infusion-after-stem-cell-transplantation-the-ideal-trial-100492007","NCT05686538","Innate Donor Effector Allogeneic Lymphocyte Infusion After Stem Cell Transplantation: the IDEAL Trial","Innate Donor Effector Allogeneic Lymphocyte Infusion After Stem Cell Transplantation: The IDEAL Trial","IDEAL","Inclusion Criteria:\n\n* Diagnoses: AML, MDS\n* Age: ≥18 years\n* Graft type: PBSC\n* Donor: ≥18 years\n* Informed consent from both donor and recipient\n\nExclusion Criteria:\n\n• Donors with need for central venous access for the leukapheresis procedure","80 Years",{"count":79,"type":19},80,[81,82],"PHASE2","PHASE3","The curative principle behind allogeneic hematopoietic stem cell transplantation (HSCT) is eradication of the malignant cells of the patient (recipient) by donor graft cells, a process termed graft-versus-leukemia (GVL) effect. GVL is traditionally mediated by donor αβ T cells in an immunological process driven by genetical differences between individuals, i.e. an allogeneic response. For this reason, αβ T cells also cause an unwanted and dangerous complication of HSCT called graft-versus-host disease (GVHD) in which healthy recipient cells are targeted by donor cells with great risk of morbidity and mortality to the patient. In addition to αβ T cells, other cells from the donor stem cell graft, termed innate effector lymphocytes, can contribute to the GVL effect. These are termed natural killer (NK) cells and T-cell receptor (TCR) γδ cells, the latter being a subset of T cells. NK and TCR γδ cells can recognize and eliminate leukemic cells in a direct tumor response independent of conventional allogeneicity. Therefore, opposite αβ T cells, innate effector lymphocytes cells can mediate GVL but are not likely to cause GVHD. The main indications for HSCT in adults are acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Approximately 50% of AML\u002FMDS transplant patients experience significant acute GVHD and 30% experience relapse of the malignant disease. Prospective clinical studies from the research group of the investigators have shown that patients with high doses of innate lymphocytes in stem cell grafts and during early immune reconstitution after HSCT have a reduced risk of both GVHD and relapse. The aim of this clinical trial is therefore to administer innate donor lymphocyte infusion (iDLI) enriched in NK and TCR γδ cells and depleted of αβ T cells in patients early after HSCT. By improving the HSCT procedure with iDLI cell therapy the scope is less GVHD and less relapse of the malignant disease and thereby improved survival and life quality in AML\u002FMDS patients.",[25],[86,87,88,89],"allogeneic stem cell transplantation","NK cells","TCR gamma delta cells","DLI","2024-04-09",{"date":92,"type":35},"2024-04-10",{"date":94,"type":35},"2022-01-01",{"date":96,"type":19},"2030-01",{"name":98,"class":42},"Rigshospitalet, Denmark",1]