[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"cytopenia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:cytopenia":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,39,64,87,120,150,184,207,229],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":28,"startDateStruct":31,"completionDateStruct":33,"leadSponsor":35,"locationsCount":38},"100538053","phase-2-a-pilot-study-of-emapalumab-for-the-treatment-of-car-t-cell-therapy-associated-prolonged-cytopenia-100538053",false,"NCT06285825","A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia","Inclusion Criteria:\n\nEligible participants will be considered for inclusion in this study if they meet the following criteria based on tests obtained less than 4 weeks from first dose:\n\n1. Relapsed or refractory FL (grade 1, 2, 3a and 3b), MCL, DLBCL, PMBCL, tFL, or HGBCL\n2. Received standard of care therapy with axi-cel, tisa-cel or liso-cel\n3. Age ≥ 18 years\n4. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2\n5. Either in remission after CART and\u002For with refractory\u002Fprogressive disease not requiring urgent treatment in the opinion of the treating physician\n6. Grade 3-4 cytopenia defined as either absolute neutrophil count of \\\u003C 1.0x109\u002FL, or platelet count \\\u003C 50x109\u002FL or haemoglobin\\\u003C 8 g\u002FdL starting or persisting between day 30 and day 90 after CART\n7. Creatinine clearance (as estimated by Cockcroft Gault) ≥ 30 mL\u002Fmin\n8. Serum alanine transaminase (ALT) \u002F aspartate transaminase (AST) ≤ 2.5 upper limit of normal (ULN), independently from etiology\n9. Total bilirubin ≤1.5 mg\u002FdL, or ≤ 3 UNLin participants with Gilbert's syndrome.\n10. Baseline oxygen saturation \\> 92% on room air 11 Females of childbearing potential must have a negative serum or urine pregnancy test (females who have undergone surgical sterilization or who have been postmenopausal for at least 2 years are not considered to be of childbearing potential)\n\n12\\. Participant will be willing to undergo a bone marrow biopsy before and at the end of each cycle, and at time of severe cytopenia recurrence; archived samples can be accepted during the screening period in absence of intercurrent treatment 13. The effects of emapalumab on the developing human fetus are unknown. For this reason, 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 up to 6 months after the administration of emapalumab (whichever is administered last). (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the participants presents with an applicable exclusionary factor which may be one of the following:\n\n* Postmenopausal (no menses in greater than or equal to 12 consecutive months).\n* History of hysterectomy or bilateral salpingo-oophorectomy.\n* Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).\n* History of bilateral tubal ligation or another surgical sterilization procedure.\n* Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject\u002FPartner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. 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.\n* Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 6 months after completion of emapalumab administration.\n\n  14\\. Ability to understand and the willingness to sign a written informed consent document.\n\n  15\\. Participant will need to test for latent tuberculosis infections using the purified protein derivative (PPD) or IFNƒÁ release assay and will be evaluated for tuberculosis risk factors within 3 months from initiation of treatment. We will administer tuberculosis prophylaxis to participants at risk for tuberculosis, or known to have a positive PPD test result, or positive IFNƒÁ release assay.\n\nExclusion Criteria:\n\nParticipants will be ineligible for this study if they meet the following criteria:\n\n1. History of malignancy other than nonmelanoma skin cancer or localized carcinoma (e.g., cervix, bladder, breast, prostate) unless disease free for at least 3 years.\n2. History of Richter's transformation of chronic lymphocytic leukemia (CLL)\n3. Presence of grade 3-4 cytopenia before initiation of lymphodepleting chemotherapy for CART\n4. Grade 3-4 cytopenia due to use of experimental non-commercially available CART product\n5. Bone marrow evidence of LBCL, hypersplenism, HLH, or a second malignancy (e.g., MDS) that could be contributing to cytopenia, with no alternative, identifiable potential cause for the cytopenia.\n6. Relapsed or progressive disease requiring immediate initiation of anti-lymphoma treatment\n7. Known presence of fungal, bacterial, viral, or other infection that is uncontrolled or requiring IV antimicrobials for management, including leishmania infection. Simple UTI and uncomplicated bacterial pharyngitis are permitted if responding to active treatment and after consultation with the Principal investigator.\n8. Known history of infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive). A history of HIV, hepatitis B or hepatitis C is permitted if the viral load is undetectable per quantitative PCR and\u002For nucleic acid testing. For participants with a history of Hepatitis B or C, standard of care monitoring for viral reactivation will be conducted. Subjects with a history of Hepatitis B will be required to undergo hepatitis B reactivation prophylaxis unless contraindicated.\n9. History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 6 months of enrolment\n10. History of primary immunodeficiency\n11. History of symptomatic deep vein thrombosis or symptomatic pulmonary embolism within 1 month of enrolment considered significant in the opinion of the principal investigator\n12. Any medical condition likely to interfere with assessment of safety or efficacy of study treatment in the investigator's opinion\n13. History of severe immediate hypersensitivity reaction to any of the agents used in this study, including E coli-derived proteins.\n14. Receipt of BCG vaccine within 12 weeks prior screening or receipt of a live or attenuated (other than BCG) vaccine within 4 weeks prior to screening\n15. Women of child-bearing potential who are pregnant or breastfeeding.\n16. Women of child-bearing potential and men with partners who are of child-bearing potential who are not willing to practice birth control as noted in section 4.1 from the time of consent through 6 months after the completion of emapalumab.\n17. In the investigator's judgment, the subject is unlikely to complete all protocol-required study visits or procedures, including follow-up visits, or comply with the study requirements for participationTrial Treatments","ALL","18 Years",{"count":18,"type":19},32,"ESTIMATED","INTERVENTIONAL",[22],"PHASE2","To look at the safety and effectiveness of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.",[25],"Cytopenia","RECRUITING","2026-05-14",{"date":29,"type":30},"2026-05-18","ACTUAL",{"date":32,"type":30},"2024-07-05",{"date":34,"type":19},"2028-08-22",{"name":36,"class":37},"M.D. Anderson Cancer Center","OTHER",1,{"id":40,"slug":41,"hasResults":11,"nctId":42,"briefTitle":43,"officialTitle":44,"acronym":4,"eligibilityCriteria":45,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":46,"targetDuration":4,"studyType":20,"phases":48,"briefSummary":50,"conditions":51,"keywords":53,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":55,"lastUpdatePostDateStruct":56,"startDateStruct":58,"completionDateStruct":60,"leadSponsor":62,"locationsCount":38},"100577734","early-phase-1-early-intervention-in-high-risk-ccus-100577734","NCT06802146","Early Intervention in High Risk CCUS","A Multi-Site Break Through Cancer Pilot Study Testing the Feasibility and Safety of Therapeutic Intervention for Patients With High-risk Clonal Cytopenia of Undetermined Significance (CCUS)","Inclusion Criteria:\n\n* Age ≥18 years.\n* Unexplained cytopenia(s) for at least 4 months (at least two separate labs within 4 months including at time of screening must meet this criteria). Cytopenia(s) defined as the presence of ≥ 1 of the following:\n\n  * Hemoglobin (Hgb) \\\u003C12 g\u002FdL for women and \\\u003C13g\u002FdL for men\n  * Absolute neutrophil count (ANC) \\\u003C 1.8 × 109\u002FL\\*\n  * Platelet count (Plt) \\\u003C150 × 109\u002FL \\*Patients known to have a Duffy-null genotype must have anemia (Hgb \\\u003C 12g\u002FdL for women, Hgb \\\u003C13g\u002FdL for men) and\u002For thrombocytopenia (Plt \\\u003C 150 × 109\u002FL) to be eligible for this study.\n\n    * 1 pathogenic variant detected in any myeloid driver gene with a VAF of at least 0.02 (2%) identified by local next generation sequencing (NGS) of peripheral blood or bone marrow sample within 3 months from screening bone marrow biopsy.\n* Participants must have a high risk score per the Clonal Hematopoiesis Risk Calculator (CHRS). See APPENDIX C for calculation.\n* Screening bone marrow biopsy must not be diagnostic of any overt hematologic malignancy by morphologic assessment and must be consistent with a diagnosis of clonal cytopenia of unknown significance (CCUS) as determined by multi-institutional hematopathology review.\n* ECOG performance status 0-2 (see Appendix A).\n* Participants must meet the following organ function as defined below:\n\n  * Serum aspartate aminotransferase\u002Fserum glutamic oxaloacetic transaminase (AST\u002FSGOT) and alanine aminotransferase (ALT\u002FSGPT) ≤3x upper limit of normal (ULN).\n  * Serum total bilirubin \\\u003C1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis or Gilbert's syndrome. In these cases, approval from the study Sponsor-Investigator is required.\n  * Creatinine clearance greater than 40 mL\u002Fmin based on the Cockcroft-Gault glomerular filtration rate estimation.\n* Ability to understand and the willingness to sign a written informed consent document.\n* For participants of the early pharmacologic intervention cohort: women of childbearing potential must use highly effective contraception during treatment for at least 6 months after the last dose and males with female partners of reproductive potential should use effective contraception during treatment and for 3 months after the last dose.\n\nExclusion Criteria:\n\n* Concurrent primary malignancy requiring active cytotoxic chemotherapy and\u002For ionizing radiation therapy.\n* Known inherited bone marrow failure disorder and\u002For germline predisposition to hematologic malignancy.\n* Receipt of anti-cancer therapy including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide, and targeted anti-cancer therapies including PARP inhibitors within the last 6 months. Patients with complete surgical resection of a tumor are not excluded from this study.\n* Anti-cancer therapy, including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide and targeted agents such as PARP inhibitors, planned in the next 6 months. Patients on hormonal adjuvant therapy for nonmetastatic breast and prostate cancer or other minimally-myelosuppressive maintenance therapies for non-metastatic cancer may be eligible at the discretion of the study PI.\n* Diagnosis of MDS, MPN, CMML, AML or any other hematolymphoid malignancy in the patient's lifetime. This includes individuals with MDS-defining chromosomal abnormalities identified via conventional karyotype or FISH.\n* Presence of a concurrent hematologic malignancy precursor state, such as smoldering multiple myeloma (SMM), and smoldering Waldenstrom's macroglobulinemia.\n* Presence of an early-stage hematologic precursor state-such as monoclonal gammopathy of undetermined significance (MGUS) and monoclonal B cell lymphocytosis (MBL).\n* Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs\u002Fsymptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and\u002For other treatment).\n* Recent (within 3 months) vaccination with any live attenuated vaccine or vaccination with live attenuated vaccine planned during the next 15 months. \\*Live attenuated vaccines include measles, mumps, rubella (MMR combined vaccine), rotavirus, smallpox, chickenpox, and yellow fever.\n* Laboratory evidence indicative of clinically significant red cell hemolysis.\n* Hypersplenism and\u002For evidence of portal hypertension on physical exam or imaging.\n* Pregnant or lactating.",{"count":47,"type":19},108,[49],"EARLY_PHASE1","This research is being done to find out more about the potential risks and benefits of early treatment in participants with high risk Clonal Cytopenia of Unknown Significance (CCUS). This study will give eligible CCUS participants the option of either being observed or taking an oral drug as treatment.\n\nThe names of the study drug involved in this study is:\n\n-Decitabine\u002Fcedazuridine (DEC\u002FCED) (a nucleoside metabolic inhibitor and cytidine deaminase inhibitor).",[52,25],"Clonal Cytopenia of Undetermined Significance",[52,54,25],"CCUS","2026-04-20",{"date":57,"type":30},"2026-04-23",{"date":59,"type":30},"2025-02-07",{"date":61,"type":19},"2028-12-01",{"name":63,"class":37},"Lachelle D. Weeks, MD, PhD",{"id":65,"slug":66,"hasResults":11,"nctId":67,"briefTitle":68,"officialTitle":69,"acronym":4,"eligibilityCriteria":70,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":71,"targetDuration":4,"studyType":73,"phases":4,"briefSummary":74,"conditions":75,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":86},"100599176","replacing-bone-marrow-diagnostics-with-peripheral-blood-analysis-in-cytopenia-patients-100599176","NCT07081087","Replacing Bone Marrow Diagnostics With Peripheral Blood Analysis in Cytopenia Patients","Development of a Peripheral Blood Assay to Replace BM Evaluation in Cytopenia - a Multi-Center Observational Study","Inclusion Criteria:\n\n1. Patients aged 18 and up with suspected\u002Fconfirmed MDS cases referred to BM evaluation either for diagnosis or risk assessment due to cytopenia\n2. Platelets \\\u003C 150 × 10E9\u002FL or\n3. Absolute neutrophil count \\\u003C 1.8 × 10E9\u002FL or\n4. Hemoglobin (Hgb) \\\u003C 13 g\u002FdL (males) and \\\u003C 12 g\u002FdL (female) and\n5. For both sexes, no evidence of Iron, folinic acid, or B12 deficiency\n\nExclusion Criteria:\n\n1. Women who are pregnant\n2. Previous diagnosis of leukemia; AML, MPN, ALL, CLL, MGUS\u002FMM or any other gammopathy\n3. Lymphocytes \\> 5000\u002Ful\n4. Patients who are on disease-related therapy are excluded, unless they are treated with Erythropoietin or Prednisone. See Appendix 2 for the list of excluded treatments.\n5. Patients who have undergone a bone marrow transplant.",{"count":72,"type":19},1500,"OBSERVATIONAL","This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing.",[25,76],"MDS","2026-03-22",{"date":79,"type":30},"2026-03-25",{"date":81,"type":30},"2025-06-01",{"date":83,"type":19},"2028-12",{"name":85,"class":37},"Weizmann Institute of Science",2,{"id":88,"slug":89,"hasResults":11,"nctId":90,"briefTitle":91,"officialTitle":91,"acronym":4,"eligibilityCriteria":92,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":93,"targetDuration":4,"studyType":20,"phases":95,"briefSummary":97,"conditions":98,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":119},"100282586","pre-myeloid-cancer-and-bone-marrow-failure-clinic-study-100282586","NCT02958462","Pre-myeloid Cancer and Bone Marrow Failure Clinic Study","Inclusion Criteria:\n\n* Patients with idiopathic cytopenias of unclear significance (ICUS)\n* Patients with clonal hematopoiesis of indeterminate significance (clonal hematopoiesis of indeterminate potential \\[CHIP\\]), including the recently described CHIP syndrome called VEXAS (vacuoles, E1 ubiquitin ligase, X chromosomal, autoimmune and somatic)\n* Patients with clonal cytopenias of undetermined significance (CCUS)\n* Marrow failure syndromes with myeloid malignancy predisposition - telomere dysfunction, chromosomal breakage disorders\n* Germ line inherited syndromes with risk for malignant transformation - GATA2, CEBPA, ETV-6, RUNX1, JAK2, PF6, etc.\n* Low risk MDS (idiopathic dysplasia of unclear significance)\n* Family member of a patient with one of the above conditions\n* Patient at high risk or suspected of developing one of the above conditions\n\nExclusion Criteria:\n\n* Patients under 18 years of age",{"count":94,"type":19},2000,[96],"NA","This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes. NGS is a procedure that looks at relevant cancer associated genes and what they do. Finding genetic markers for pre-malignant conditions may help identify patients who are at risk of pre-myeloid cancers and bone marrow failure syndromes and lead to earlier intervention.",[99,100,101,25,102,52,103,104,105,106,107,108,109],"Myeloid Malignancy","Inherited Bone Marrow Failure Syndrome","Clonal Expansion","Bone Marrow Failure Syndrome","Clonal Hematopoiesis of Indeterminate Potential","Hematologic Neoplasms","Hematopoietic and Lymphatic System Neoplasm","Hereditary Neoplastic Syndrome","Idiopathic Cytopenia of Undetermined Significance","Idiopathic Dysplasia of Uncertain Significance","Low Risk Myelodysplastic Syndrome","2026-02-20",{"date":112,"type":30},"2026-02-23",{"date":114,"type":30},"2017-01-16",{"date":116,"type":19},"2035-09-15",{"name":118,"class":37},"Mayo Clinic",3,{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":125,"acronym":4,"eligibilityCriteria":126,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":127,"targetDuration":4,"studyType":20,"phases":129,"briefSummary":130,"conditions":131,"keywords":133,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":139,"lastUpdatePostDateStruct":140,"startDateStruct":142,"completionDateStruct":144,"leadSponsor":146,"locationsCount":149},"100394713","phase-2-a-study-of-elritercept-to-treat-anemia-in-adults-with-very-low-low-or-intermediate-risk-myelodysplastic-syndromes-mds-100394713","NCT04419649","A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)","A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)","Inclusion Criteria:\n\n1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local study participant privacy regulations.\n2. Male or female ≥ 18 years of age, at the time of signing informed consent.\n3. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).\n4. Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception.\n5. In the opinion of the Investigator, the participant is able and willing to comply with the requirements of the protocol (e.g., all study procedures, return for follow-up visits).\n\nPart 1 Inclusion Criteria\n\nParticipants are eligible to be included in Part 1 of the study only if all the following criteria apply:\n\n1. Diagnosis of MDS according to WHO classification that meets International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.\n2. Less than (\\\u003C)5percent (%) blasts in bone marrow during the Pretreatment Period.\n3. Peripheral blood white blood cell (WBC) count \\\u003C13,000\u002Fmicroliter (μL) during the Pretreatment Period.\n4. Anemia defined as:\n\n   1. In non-transfused participants, having received no RBC transfusions within 8 weeks, Hgb concentration ≤ 10.0 g\u002FdL during the Pretreatment Period OR\n   2. In LTB participants, having received 1 to 3 units of RBCs for Hgb ≤ 9.0 g\u002FdL within 8 weeks of the Pretreatment Period.\n\n      OR\n   3. In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g\u002FdL within 8 weeks of the Pretreatment Period.\n\nPart 1 Extension - Abbreviated Inclusion Criteria\n\nParticipants from Part 1 are eligible to be included in Part 1 Extension of the study only if all the following criteria apply:\n\n1. Previously completed 4 cycles of elritercept in Part 1 with no dose-limiting toxicities (DLTs).\n2. Participant has the potential to benefit from administration of elritercept, in the opinion of the Investigator.\n3. \\\u003C 5% blasts in bone marrow.\n4. Peripheral WBC count \\\u003C 13,000\u002FμL during the 28 days prior to cycle 5 day 1 (C5D1).\n\nPart 2 Inclusion Criteria\n\nParticipants are eligible to be included in Part 2 of the study only if all the following criteria apply:\n\n1. Cohort A:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * ring sideroblast (RS)-positive as defined by WHO 2016 criteria.\n   * Requiring at least 2 units of RBC transfusions in the preceding 8 weeks before cycle 1 day 1 (C1D1).\n2. Cohort B:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Non-RS as defined by WHO 2016 criteria.\n   * Requiring at least 2 units of RBC transfusions in the 8 weeks before C1D1.\n3. Cohort C:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Has anemia, defined by Hgb ≤ 10 g\u002FdL during the Pretreatment Period, and received no RBC transfusion in the 8 weeks before C1D1.\n4. Cohort D:\n\n   * Diagnosis of CMML according to WHO classification.\n   * Has anemia, defined by Hgb ≤ 10 g\u002FdL during the Pretreatment Period, and received no RBC transfusion in the 8 weeks before C1D1.\n   * OR\n   * Received at least 2 units of RBC transfusions for anemia in the 8 weeks before C1D1.\n5. Cohort E:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Requiring ≥ 2 units of RBC transfusions in the preceding 8 weeks before C1D1.\n   * Receipt of ≥ 20 units of RBC in transfusion over the participant's lifetime.\n   * Serum ferritin \\> 1000 nanograms per milliliter (ng\u002FmL) on ≥ 2 assessments in the preceding 8 weeks before C1D1.\n   * Treated with stable dose of iron chelation therapy for ≥ 8 weeks prior to C1D1.\n6. Cohort F:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Requiring ≥ 2 units of RBC transfusions in the preceding 8 weeks before C1D1.\n   * Receipt of ≥ 20 units of RBC in transfusion over the participant's lifetime.\n   * Serum ferritin \\> 1000 ng\u002FmL on ≥ 2 assessments in the preceding 8 weeks before C1D1.\n   * Not treated with iron chelation therapy in the preceding 8 weeks before C1D1 and not eligible to initiate iron chelation therapy in the opinion of the Investigator and in accordance with local treatment guidelines for initiation of iron chelation therapy.\n7. Cohort G:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * RS-positive as defined by WHO 2016 criteria OR non-RS as defined by WHO 2016 criteria.\n   * Relapsed, refractory, or intolerant to frontline luspatercept treatment and have not received an interceding therapy (for example, erythropoiesis-stimulating agent \\[ESA\\])\n\n     * Relapsed is defined as documentation of response to luspatercept therapy and subsequent development of a need for transfusion(s).\n     * Refractory is defined as documentation of no response with luspatercept ≥ 1 mg\u002Fkg administered for ≥ 12 weeks duration.\n     * Intolerant is defined as documentation of discontinuation of luspatercept therapy due to intolerance or an AE at any time after introduction.\n   * Requiring ≥ 2 units of RBC transfusions over 8 weeks prior to C1D1.\n   * Erythropoietin (EPO) \\\u003C 500 international units per liter (U\u002FL) at Baseline.\n   * Last dose of luspatercept is ≥ 3 weeks and \\\u003C 12 months from C1D1.\n8. \\\u003C 5% blasts in bone marrow assessed by bone marrow aspirate during the Pretreatment Period.\n\nPart 1 Exclusion Criteria\n\nParticipants are excluded from Part 1 of the study if any of the following criteria apply.\n\nMedical History\n\n1. Diagnosis of MDS with deletion of chromosome 5q (Del5q).\n2. Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n3. Presence of uncontrolled heart disease or New York Heart Association (NYHA) Class III or IV heart failure.\n4. History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.\n5. History of stroke, deep venous thrombosis (DVT), or arterial embolism within 6 months prior to C1D1.\n6. Major surgery within 28 days prior to C1D1. Participants must be completely recovered from any previous surgery prior to C1D1.\n7. Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B virus (HBV), or active infectious hepatitis C virus (HCV). Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n8. Any malignancy other than MDS that has not been in remission and\u002For has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C1D1. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and\u002For radiation for other diseases).\n9. History of solid organ or hematological transplantation.\n10. Presence of uncontrolled hypertension, defined as systolic blood pressure (BP) ≥ 150 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.\n11. Body mass index (BMI) ≥ 40 kilograms per meter square (kg\u002Fm\\^2) during the Pretreatment Period.\n12. History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the investigational medicinal product (IMP).\n\nTreatment History\n\n1. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.\n2. Treatment with ESA within 56 days prior to C1D1.\n3. Prior or concurrent chronic treatment with granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF).\n4. Iron chelation therapy if initiated within 8 weeks prior to C1D1.\n5. Vitamin B12 therapy initiated within 8 weeks prior to C1D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.\n6. Treatment with another investigational drug or device or approved therapy for investigational use ≤ 28 days prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.\n\nLaboratory Exclusions (during Pretreatment Period)\n\n1. Platelet count \\> 450 ✕ 10\\^9\u002FL or \\\u003C 30 ✕ 10\\^9\u002FL.\n2. Transferrin saturation \\\u003C 15%.\n3. Ferritin \\\u003C 50 nanograms per milliliter (ng\u002FmL).\n4. Folate \\\u003C 4.5 nanomoles per liter (nmol\u002FL) (\\\u003C 2.0 ng\u002FmL).\n5. Vitamin B12 \\\u003C 148 picomoles per liter (pmol\u002FL) (\\\u003C 200 picograms per milliliter \\[pg\u002FmL\\]).\n6. Estimated glomerular filtration rate (GFR) \\\u003C 30 milliliter per minute per 1.73 meter square (mL\u002Fmin\u002F1.73 m\\^2), as determined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.\n7. Positive for HIV.\n\nMiscellaneous\n\n1. Pregnant or lactating females.\n2. Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.\n3. Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or contract research organization (CRO) employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.\n\nPart 1 Extension - Exclusion Criteria\n\nParticipants from Part 1 are excluded from Part 1 Extension of the study if any of the following criteria apply.\n\nMedical History\n\n1. Discontinuation of IMP in Part 1 for any reason.\n2. Has not completed a study visit in the past 12 months.\n3. Active infection requiring parenteral antibiotic therapy within 28 days prior to C5D1 or oral antibiotics within 14 days of C5D1. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n4. Presence of uncontrolled heart disease or NYHA Class III or IV heart failure.\n5. History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.\n6. History of stroke, DVT, or arterial embolism within 6 months prior to C5D1.\n7. Major surgery within 28 days prior to C5D1. Participants must be completely recovered from any previous surgery prior to C5D1.\n8. Known positive for HIV, active infectious HBV, or active infectious HCV. Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n9. Any malignancy other than MDS that has not been in remission and\u002For has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C5D1.\n10. History of solid organ or hematological transplantation.\n11. Presence of uncontrolled hypertension, defined as systolic BP ≥ 150 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.\n12. BMI ≥ 40 kg\u002Fm\\^2 during the 28 days prior to C5D1.\n\nTreatment History\n\n1. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.\n2. Treatment with ESA within 56 days prior to C5D1.\n3. Prior or concurrent chronic treatment with G-CSF or GM-CSF.\n4. Iron chelation therapy if initiated within 8 weeks prior to C5D1.\n5. Vitamin B12 with treatment initiated within 8 weeks prior to C5D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.\n6. Treatment with another investigational drug or device or approved therapy for investigational use ≤ 28 days prior to C5D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C5D1, whichever is longer. Previous treatment with elritercept is acceptable.\n\nLaboratory Exclusions (during Abbreviated Pretreatment Period)\n\n1. Platelet count \\> 450 × 10\\^9\u002FL or \\\u003C 30 × 10\\^9\u002FL.\n2. Transferrin saturation \\\u003C 15%.\n3. Ferritin \\\u003C 50 ng\u002FmL.\n4. Folate \\\u003C 4.5 nmol\u002FL (\\\u003C 2.0 ng\u002FmL).\n5. Vitamin B12 \\\u003C 148 pmol\u002FL (\\\u003C 200 pg\u002FmL).\n6. Estimated GFR \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2, as determined by the CKD-EPI equation.\n\nMiscellaneous\n\n1. Pregnant or lactating females.\n2. Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.\n3. Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or CRO employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.\n\nPart 2 Exclusion Criteria\n\nParticipants are excluded from Part 2 of the study if any of the following criteria apply.\n\nMedical History\n\n1. Diagnosis of MDS with Del5q.\n2. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and\u002For radiation for other diseases).\n3. Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n4. Presence of the following cardiac conditions:\n\n   1. Presence of uncontrolled heart disease or NYHA Class III or IV heart failure.\n   2. QTcF (QT interval corrected by Fridericia's formula) \\> 500 msec on the screening or C1D1 electrocardiogram (ECG; mean of 3 measurements).\n   3. Uncontrolled clinically significant arrhythmia (participants with rate-controlled atrial fibrillation are not excluded).\n   4. Acute myocardial infarction or unstable angina pectoris ≤ 6 months prior to C1D1.\n5. Presence of uncontrolled hypertension, defined as systolic BP ≥ 160 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.\n6. History of stroke, DVT, or arterial embolism within 6 months prior to C1D1.\n7. History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.\n8. Major surgery within 28 days prior to C1D1. Participants must be completely recovered from any previous surgery prior to C1D1.\n9. Any malignancy other than MDS or CMML that has not been in remission and\u002For has required major surgery or systemic therapy including radiation, chemotherapy, targeted therapy, or hormonal therapy within 1 year prior to C1D1.\n10. History of solid organ or hematological transplantation.\n11. Diagnosis of hemolytic anemia, active bleeding, hemoglobinopathies, or congenital disorders as a cause of the participant's anemia.\n12. NCI-CTCAE Grade ≥ 2 bleeding events within the 3 months prior to C1D1.\n13. Receipt of an RBC or platelet transfusion for any reason(s) or combination of reasons other than underlying MDS within the 16 weeks prior to C1D1. If a participant requires a transfusion for an unanticipated reason during the Pretreatment Period, a prolonged screening period may be considered after discussion with the Medical Monitor.\n14. Known positive for HIV, active infectious HBV with positive viral load (HBV DNA), or active infectious HCV with positive viral load (HCV RNA). Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n15. BMI ≥ 40 kg\u002Fm\\^2.\n16. History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the IMP.\n17. Diagnosis of cirrhosis, non-alcoholic steatohepatitis, alcoholic liver disease, hepatitis, or other liver disease (acute or chronic) meeting Child-Pugh C criteria for hepatic impairment. Participants with elevated liver enzymes are allowed if the liver enzyme elevation is suspected to be due to iron-overload or iron chelation, and other hepatic causes have been ruled out, in the opinion of the Investigator.\n\nTreatment History\n\n1. Prior treatment with azacitidine, decitabine, lenalidomide, or sotatercept.\n2. Prior treatment with luspatercept (Cohorts A, B, C, D, E, and F only).\n3. Treatment with ESA within 8 weeks prior to C1D1.\n4. Prior or concurrent chronic treatment with G-CSF or GM-CSF, for reasons other than for treatment of MDS.\n\n   a. Note: Previous treatment with G-CSF or GM-CSF for MDS, which has been discontinued ≥ 8 weeks prior to C1D1 is allowed.\n5. Iron chelation therapy if initiated within 8 weeks prior to C1D1. Participants on stable doses of iron chelation therapy for ≥ 8 weeks are allowed.\n6. Vitamin B12 and\u002For folate therapy initiated within 8 weeks prior to C1D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.\n7. Any need to receive a prohibited medication.\n8. Treatment with another investigational drug or device or approved therapy for investigational use within 8 weeks prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.\n\nLaboratory Exclusions (during Pretreatment Period)\n\n1. Peripheral WBC count ≥ 13,000\u002FμL.\n2. Platelet count \\> 450 × 10\\^9\u002FL or \\\u003C 25 × 10\\^9\u002FL.\n3. Transferrin saturation \\\u003C 15%.\n4. Ferritin \\\u003C 50 ng\u002FmL.\n5. Folate \\\u003C 4.5 nmol\u002FL (\\\u003C 2.0 ng\u002FmL).\n6. Vitamin B12 \\\u003C 148 pmol\u002FL (\\\u003C 200 pg\u002FmL).\n7. Estimated GFR \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2 as determined by the CKD-EPI equation.\n\nMiscellaneous\n\n1. Pregnant or lactating females.\n2. Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.\n3. Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or CRO employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.\n\nFor Cohort G ONLY:\n\n1. Any luspatercept related AE Grade ≥ 3 that has not resolved to baseline or Grade ≤ 1.\n2. No history of allergy\u002Fanaphylaxis\u002Fhypersensitivity to luspatercept.\n3. No prior treatment with imetelstat.",{"count":128,"type":19},160,[22],"The main aim of this study is to learn how safe elritercept is and how well adults with anemia associated with lower-risk MDS tolerate treatment with different doses of elritercept. Other aims are to learn how safe elritercept is by looking at how many participants have MDS that worsens during the study and learn about the effects of elritercept on anemia linked to MDS. The study will also look to learn how elritercept affects the production of healthy RBCs.",[132,25],"Myelodysplastic Syndromes",[134,135,136,137,138],"Transfusion","Drug Therapy","Elritercept","TAK-226","KER-050","2026-02-04",{"date":141,"type":30},"2026-02-06",{"date":143,"type":30},"2020-08-19",{"date":145,"type":19},"2031-10-30",{"name":147,"class":148},"Takeda","INDUSTRY",47,{"id":151,"slug":152,"hasResults":11,"nctId":153,"briefTitle":154,"officialTitle":155,"acronym":4,"eligibilityCriteria":156,"healthyVolunteers":157,"sex":15,"minAge":158,"maxAge":159,"enrollmentInfo":160,"targetDuration":4,"studyType":20,"phases":162,"briefSummary":163,"conditions":164,"keywords":167,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":175,"lastUpdatePostDateStruct":176,"startDateStruct":178,"completionDateStruct":180,"leadSponsor":182,"locationsCount":183},"100549190","phase-2-a-study-of-emapalumab-for-pediatric-aplastic-anemia-100549190","NCT06430788","A Study of Emapalumab for Pediatric Aplastic Anemia","Phase 2a\u002F2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia","Inclusion Criteria:\n\n* Patients undergoing workup for suspected newly diagnosed sAA:\n\n  * Patients with severe cytopenias and a hypocellular marrow concerning for sAA\n  * Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:\n\nMarrow Cellularity: \\\u003C25%, or 25-50% with \\\u003C30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): \\\u003C500 x 10\\^9\u002FL Platelets: \\\u003C20 x 10\\^9\u002FL Absolute Reticulocyte Count: \\\u003C60 x 10\\^9\u002FL\n\n* Patients that do not have evidence of leukemia or MDS\n* Patients \\\u003C 25 years of age at time of diagnosis\n* Able to tolerate emapalumab and IST (with standard institutional organ function criteria)\n\nExclusion Criteria:\n\n* Uncontrolled infection at presentation.\n* Patients who have undergone previous treatment for sAA.\n* Patients with known inherited bone marrow failure\n* Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial\n* Patients with leukemia or MDS\n* Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.",true,"0 Years","25 Years",{"count":161,"type":19},35,[22],"The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.\n\nFunding Source- FDA OOPD",[165,25,166],"Aplastic Anemia","Hypocellular Marrow",[168,169,170,171,172,173,174],"pediatric aplastic anemia","aplastic anemia","cytopenia","hypocellular marrow","Emapalumab","Memorial Sloan Kettering Cancer Center","23-278","2025-12-11",{"date":177,"type":30},"2025-12-17",{"date":179,"type":30},"2024-05-21",{"date":181,"type":19},"2029-05-21",{"name":173,"class":37},6,{"id":185,"slug":186,"hasResults":11,"nctId":187,"briefTitle":188,"officialTitle":189,"acronym":190,"eligibilityCriteria":191,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":192,"targetDuration":4,"studyType":20,"phases":194,"briefSummary":195,"conditions":196,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":198,"lastUpdatePostDateStruct":199,"startDateStruct":201,"completionDateStruct":203,"leadSponsor":205,"locationsCount":86},"100575560","feasibility-and-safety-of-exercise-in-patients-with-low-risk-myeloid-cancers-and-precursor-conditions-100575560","NCT06773871","Feasibility and Safety of Exercise in Patients With Low-risk Myeloid Cancers and Precursor Conditions","Feasibility and Safety of Exercise in Patients With Low-risk (or Early-stage) Myeloid Cancers and Precursor Conditions (HemEx): a Randomized Controlled Pilot Trial","HemEx","Inclusion Criteria:\n\n* A diagnosis of either Lower-risk of Myelodysplastic Syndrome or Clonal Cytopenia of undetermined significance(WHO 2022 Classification)\n* Written informed consent prior to study procedures\n* Performance status ≤ 2\n* Age \\> 18 years old\n\nExclusion Criteria:\n\n* Physically not able to undergo exercise intervention (e.g., arthrosis, physical disabilities)\n* Exercising on a regular basis (i.e., participants must score in the category \"low\" when screening with International Physical Activity Questionnaire-Short Form; IPAQ-SF27)\n* Unwillingness to undergo exercise intervention\n* Use of metformin\n* Treatment with chemotherapy, therapeutic radiation, or immunosuppressive therapy within the last year\n* Treatment with hypomethylating agents\n* Any absolute contraindication to undergo cardiopulmonary exercise testing according to working papers from American Heart Association and Danish Society of Cardiology\n* Hemoglobin levels \\\u003C 5.5 mmol OR \\\u003C6.5 mmol and simultaneous cardiac insufficiency OR pacemaker.\n* Blood transfusion-dependent ≥ 8 units of red blood cell transfusion in 16 weeks (IWG 2018-criteria)\n* Uncontrolled co-morbidity",{"count":193,"type":19},36,[96],"Somatic mutations as seen in myeloid malignancies can also be detected in healthy, elderly individuals (clonal hematopoiesis of indeterminate potential, CHIP), in patients with unex-plained cytopenia, that do not fulfill the criteria for myeloid malignancy (clonal cytopenia of un-determined significance, CCUS) It has been shown that these conditions predispose to hema-tological cancer. For patients with CCUS, it has been reported that in a 5-year period up to 50-90 % of the patients will progress to myelodysplastic syndrome (MDS) or acute myeloid leu-kemia (AML), both devastating diseases with poor outcomes, especially for the elderly popula-tion. There is currently no treatment available for patients with CCUS besides supporting agents. Since the somatic mutations can be detected up to 10 years before a diagnosis of MDS, it opens the potential for early intervention.\n\nPhysical inactivity is associated with multiple solid cancers, and it has been suggested that exercise can prevent for example certain colon- or breast cancers. Studies in mice have shown that exercise can reduce tumor size and incidence of solid cancers, and different mechanisms have been suggested including increased immune cell infiltration, reduced systemic inflamma-tion, and metabolic changes. The mechanisms of disease progression of pre-leukemia and MDS are complex and probably multifactorial, but recent studies suggest that components such as natural killer cells, adipocytes, and inflammatory substances in the bone marrow mi-croenvironment play a crucial role; factors that exercise may modulate. In addition, recent stud-ies have shown that increased bone marrow adipose tissue (BMAT) may create a microenvi-ronment that supports the expansion of leukemic cells and thus may facilitate disease progres-sion, and earlier studies among healthy, younger individuals have shown that exercise can reduce the amount of BMAT significantly.\n\nTherefore, the investigators hypothesize that exercise may prevent or delay the progression from pre-leukemia to leukemia by altering the microenvironment in the bone marrow.\n\nThe purpose with this clinical, pilot trial where patients with the preleukemic condition CCUS or early stage of leukemia (i.e., lower-risk MDS) will undergo an individualized exercise interven-tion, is to investigate:\n\n1. whether an exercise intervention and the trial set-up, are feasible and safe in this cohort,\n2. potential mechanisms in leukemogenesis affected by exercise in controlling dis-ease progression,\n3. and the effect hereof on quality of life and activities of daily living. The above will inform the decision-making on designing a larger randomized, controlled trial.",[197,25],"Myelodysplastic Syndrome","2025-08-18",{"date":200,"type":30},"2025-08-19",{"date":202,"type":30},"2025-03-10",{"date":204,"type":19},"2027-01",{"name":206,"class":37},"Rigshospitalet, Denmark",{"id":208,"slug":209,"hasResults":11,"nctId":210,"briefTitle":211,"officialTitle":212,"acronym":4,"eligibilityCriteria":213,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":214,"targetDuration":4,"studyType":20,"phases":216,"briefSummary":217,"conditions":218,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":220,"lastUpdatePostDateStruct":221,"startDateStruct":223,"completionDateStruct":225,"leadSponsor":227,"locationsCount":38},"100429517","phase-2-danazol-for-treatment-of-cytopenias-in-patients-with-cirrhosis-100429517","NCT04873102","Danazol for Treatment of Cytopenias in Patients With Cirrhosis","A Phase II Single Center Single Arm Pilot Study Administering Danazol for Treatment of Cytopenias in Patients With Cirrhosis","Inclusion Criteria:\n\n* Age 18 years or older and able to provide informed consent\n* ECOG 0-2\n* Compensated Child-Pugh class A of any etiology with the exception of chronic hepatitis B with one or more of the following cytopenias\n\n  1. Leukopenia defined as white blood cell count \\\u003C2000\u002Fmm3 or absolute neutrophil count \\\u003C1000\u002Fmm3 along with thrombocytopenia \\\u003C150,000\u002Fmm3 measured on two separate occasions at least 3 months apart within 6 months of enrollment\n  2. Thrombocytopenia defined as platelet count \\\u003C50,000\u002Fmm3 along with white blood cell count \\\u003C4000\u002Fmm3 measured on two separate occasions at least 3 months apart within 6 months of enrollment\n* Compensated Child-Pugh class B cirrhosis of any etiology with the exception of chronic hepatitis B with one or more of the following cytopenias:\n\n  1\\. Leukopenia defined as white blood cell count ≤ 3500\u002Fmm3 measured on two separate occasions at least 3 months apart within 6 months of enrollment 3. Thrombocytopenia defined as platelet count ≤ 100,000\u002Fmm3 measured on two separate occasions at least 3 months apart within 6 months of enrollment\n* Enrolled patients must have one or more of the following:\n\n  * Presence of a genetic variant (defined as a known mutation, variant likely to be pathogenic or variant of undetermined significance with likely deleterious effect on transcription or translation) in at least one of the following genes: TERT, TERC, RTEL1, DKC, NOP10, NHP2, TINF2, WRAP53\n  * Shortened telomere length in peripheral blood mononuclear cells (defined as age-adjusted telomere length at or below the 5th percentile)\n  * Of note, patient's found to have telomere mutations know to confer a gain of function will be excluded\n* For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of \\\u003C 1% per year during the treatment period\n\n  * A woman is considered to be of childbearing potential if she is postmenarcheal, has not reached a postmenopausal state (\\>= 12 continuous months of amenorrhea with no identified cause other than menopause), and has not undergone surgical sterilization (removal of ovaries and\u002For uterus)\n  * Examples of contraceptive methods with a failure rate of \\\u003C 1% per year include bilateral tubal ligation, male sterilization, proper use of hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices\n  * The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient; periodic abstinence (e.g., calendar, ovulation, symptothermal, or post ovulation methods) and withdrawal are not acceptable methods of contraception\n* Women of childbearing potential (WOCBP) must have a negative serum test (minimum sensitivity 25 IU\u002FL or equivalent units of human chorionic gonadotropin \\[HCG\\]) within 72 hours prior to the start of treatment\n\nExclusion Criteria:\n\n* Cirrhosis secondary to chronic hepatitis B or any history of hepatitis B\n* Patients with telomere related mutations know to confer gain of function will be excluded\n* Patients known to be infected with HIV\n* History of any hormone sensitive malignancy, including breast cancer, prostate cancer, hepatocellular carcinoma or liver adenoma as well as any patient considered high risk for developing malignancy (i.e. history of familial cancers including a first degree relative)\n* Patients who are actively receiving anti-cancer therapy\n* Liver decompensation event within the last 6 months (i.e. variceal bleed, ascites requiring paracentesis, hepatic encephalopathy)\n* Active thrombosis or history of unprovoked thromboembolic disease, including cardiovascular events. If a patient has received and completed adequate anticoagulation for a provoked thrombosis, they can be included in the study.\n* Pregnant or planning to become pregnant\n* Females patients who are breast feeding\n* Any contraindication to danazol use\n* Uncontrolled co-morbid condition which would make the administration of danazol unsafe, including decompensated heart failure or known EF less than 40%, unstable angina pectoris, uncontrolled cardiac arrhythmia, decompensated liver failure, renal failure defined as creatinine greater than \\>1.6 or psychiatric illness that would limit compliance with study requirements\n* Alanine aminotransferase and\u002For aspartate aminotransferase \\>3x upper limit of normal\n* Alkaline phosphatase \\>2.5 x upper limit of normal\n* Total bilirubin or direct bilirubin \\>2.5 x upper limit of normal\n* Patients with known alcohol or drug abuse within the last year\n* Concomitant use of hormone stimulants or hormone blocking agents.\n* Concomitant use of other bone marrow stimulating agents that may affect white blood cell and platelet counts (i.e. G-CSF, romiplostim, eltrombopag, corticosteroids). Short term use of growth factors per standard of care in preparation for procedure or for other medical indications is acceptable. Patients taking corticosteroids above 5 mg of prednisone or the equivalent who are on a stable dose for at least 8 weeks prior to enrollment can be included.\n* Concomitant treatment with systemic immunosuppressive medications (including but not limited to cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor \\[anti-TNF\\] agents)",{"count":215,"type":19},10,[22],"This is a phase II pilot study designed to assess the safety and efficacy of danazol for treatment of cytopenias in patients with CPC A\u002FB cirrhosis. Subjects with or without telomere mutations and\u002For shortened telomeres will be treated with danazol 600 mg per day by mouth for a duration of 24 months. The goal will be to treat a total of 10 patients.",[219,25],"Cirrhosis, Liver","2025-07-09",{"date":222,"type":30},"2025-07-11",{"date":224,"type":30},"2024-08-01",{"date":226,"type":19},"2027-08-01",{"name":228,"class":37},"University of Southern California",{"id":230,"slug":231,"hasResults":11,"nctId":232,"briefTitle":233,"officialTitle":234,"acronym":4,"eligibilityCriteria":235,"healthyVolunteers":157,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":236,"targetDuration":4,"studyType":20,"phases":238,"briefSummary":239,"conditions":240,"keywords":244,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":251,"lastUpdatePostDateStruct":252,"startDateStruct":254,"completionDateStruct":256,"leadSponsor":258,"locationsCount":38},"100419455","phase-2-repurposing-metformin-as-a-leukemia-preventive-drug-in-ccus-and-lr-mds-100419455","NCT04741945","Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS","STOP-LEUKEMIA: Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS","Patients are eligible to be included in WP1 if they meet all of the following criteria:\n\nInclusion criteria:\n\n* A diagnosis of:\n\n  * LR-MDS according to the revised international prognostic scoring system (IPSS-R), i.e., very low- or low-risk disease (IPSS-R score ≤3) in addition to a bone marrow blast percentage \\\u003C5 OR\n  * CCUS defined as the presence of somatic mutation(s) or cytogenetic abnormality not diagnostic of MDS or any other malignancy in the context of persistent cytopenia (\\>6 months) with other common causes of cytopenia ruled out in the setting of bone marrow morphology that is not diagnostic of MDS or any other malignancy, and hematolytic conditions have been ruled out. Peripheral blood cytopenia is defined as hemoglobin (hgb) \\\u003C11.3 g\u002FdL (7 mmol\u002FL) in women and hgb \\\u003C12.9 g\u002FdL (8 mmol\u002FL) in men, platelet count \\\u003C150 x 109\u002FL, or neutrophil count \\\u003C1.8 x 109\u002FL\n* Menopause, if being a female, defined as females \\>45 years of age who have experienced amenorrhea for minimum 12 months, without any other obvious pathological or physiological cause\n* ≥18 years of age\n* Written informed consent\n* Willingness to comply with mandatory aspects of the protocol\n* Ability to swallow pills\n\nExclusion criteria:\n\n* Any prior treatment with metformin\n* A diagnosis of diabetes mellitus\n* Therapeutic radiation, immunosuppressive therapy (with the exception of corticosteroids), or chemotherapy within the past year\n* Treatment with granulocyte colony-stimulating factor within the past 30 days\n* Prior therapy with hypomethylating agents (i.e., azacitidine, decitabine)\n* eGFR \\\u003C45 mL\u002Fmin\n* Performance status according to the Eastern Cooperative Oncology Group \\>2\n* Other active malignancy within the past five years\n* Uncontrolled comorbidity including impaired hepatic function (total serum bilirubin \\>1.5 × upper limit of the normal range (ULN), serum alanine transaminase \\>3 × ULN), chronic hepatitis with decompensated cirrhosis, disabling psychiatric disease, severe neurologic disease, uncontrolled metabolic disease, or severe cardiac disease (NYHA class 3-4)\n\nAn eGFR calculation performed up to one month prior to inclusion may be used to assess renal function. If such an assessment is not available, it is performed at screening.\n\nHealthy volunteers are eligible to be included in WP0 if they meet all of the following criteria:\n\nInclusion criteria:\n\n* Healthy individuals matched on age, sex, and BMI, if possible, to individual patient participants in WP1\n* Written informed consent\n* Willingness to comply with mandatory aspects of the protocol\n\nExclusion criteria:\n\n* Use of metformin within the past 3 years\n* A diagnosis of diabetes mellitus, rheumatological disorders, autoimmune diseases or other inflammatory disorders, celiac disease, inflammatory bowel disease, or other gastrointestinal disorders or symptoms\n* Treatment with immunosuppressive drugs (with the exception of corticosteroids) or chemotherapy within the past year or antibiotics within the past 6 months\n* Any contraindications to MRS",{"count":237,"type":19},40,[22],"This is a single-arm pilot study of the feasibility and safety of metformin in patients with clonal cytopenia of undetermined significance (CCUS) or lower-risk myelodysplastic neoplasms (LR-MDS).",[241,242,25,243],"Preleukemia","Myelodysplastic Neoplasm","Preleukemic Anemia",[245,242,52,246,247,248,249,250],"Metformin","Safety","Feasibility","Bone Marrow Adipose Tissue","Gut Microbiota","Intestinal Permeability","2024-11-19",{"date":253,"type":30},"2024-11-22",{"date":255,"type":30},"2021-12-13",{"date":257,"type":19},"2026-02",{"name":259,"class":37},"Kirsten Grønbæk"]