[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"low-risk-myelodysplastic-syndromes\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:low-risk-myelodysplastic-syndromes":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,47,73],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100462944","phase-1-study-of-r289-in-patients-with-lower-risk-myelodysplastic-syndromes-lr-mds-100462944",false,"NCT05308264","Study of R289 in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS)","An Open-label, Phase 1b Study of R289, an IRAK1\u002F4 Inhibitor, in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS) Who Are Relapsed\u002FRefractory\u002FResistant to Prior Therapies","Inclusion Criteria:\n\n* Patient must be ≥ 18 years of age at the time of signing the informed consent.\n* Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5% bone marrow myeloblasts.\n* Must be relapsed, refractory\u002Fresistant, intolerant, or have inadequate response to therapies with known clinical benefits for MDS, such as EPOs, luspatercept, and HMAs(i.e., azacytidine or decitabine). Patients with del (5q) must have failed prior lenalidomide therapy.\n* DOSE ESCALATION PHASE:\n\n  a. Must meet at least one of the following criteria prior to initial administration of study treatment: 1) Symptomatic anemia with hemoglobin \\\u003C 9.0 g\u002FdL and no RBC transfusion within 16 of registration or 2) RBC transfusion dependent defined as receiving ≥ 2 units of packed red blood cells (PRBCs) within 8 weeks in the preceding 16 weeks for a hemoglobin \\\u003C9.0 g\u002FdL.\n* DOSE EXPANSION PHASE:\n\n  1. Relapsed, refractory to or ineligible for ESAs and has previously received one or more approved therapies for LR-MDS\n  2. Must be RBC transfusion dependent defined as receiving ≥ 2 units of packed red blood cells (PRBCs) within 8 weeks in the preceding 16 weeks for a hemoglobin \\\u003C9.0 g\u002FdL.\n* EXPLORATORY PHASE 1b COHORT:\n\n  1. Transfusion-dependent LR-MDS who are refractory or intolerant to, or are ineligible for ESAs.\n  2. No prior therapy with any approved or investigational therapies for MDS\n  3. No del 5q cytogenetic abnormality\n  4. RBC transfusion dependent defined as receiving ≥ 2 units of PRBCs within 8 weeks in the preceding 16 weeks for a hemoglobin \\\u003C9.0 g\u002FdL\n* All patients must have documented marrow iron stores. If marrow iron stain is not available, the transferrin saturation must be \\>20% or a serum ferritin \\> 100ng\u002F100mL\n* Must have Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 at screening.\n* Must have adequate organ function, defined as:\n\n  1. Hepatic function:\n\n     * aspartate amino transferase (AST) and alanine aminotransferase (ALT) ≤ 1.5 × upper limit of normal (ULN)\n     * total bilirubin ≤ 1.5 × ULN\n  2. Renal function defined as creatinine clearance \\> 60 mL\u002Fmin (using Cockcroft-Gault), or blood creatine \\\u003C 1.5 mg\u002FdL\n\nExclusion Criteria:\n\n* Prior treatment for MDS (i.e., TPOs, EPOs, luspatercept, HMAs) concluded \\\u003C 4 weeks prior to study treatment\n* Clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or GI bleeding.\n* MDS secondary to treatment with radiotherapy, chemotherapy, and\u002For immunotherapy for malignant or autoimmune diseases.\n* Diagnosis of chronic myelomonocytic leukemia.\n* History of uncontrolled seizures.\n* Uncontrolled bacterial or viral infection (i.e., documented HIV, hepatitis B or hepatitis C).\n* History of other malignancy that could affect compliance or interpretation of results. Patients with an malignancy other than leukemia appropriately treated with curative intent and the malignancy has been in remission without treatment for ≥ 2 years prior to study entry are eligible as are:\n\n  1. Adequately treated in situ carcinoma of the cervix uteri\n  2. Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin, or\n  3. Low grade, early-stage prostate cancer (Gleason score 6 or below, stage 1 or 2) with no requirement for therapy at any time prior to the study, or previously resected.\n* History of or active, clinically significant, cardiovascular, respiratory, GI, renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorder that, in the Investigator's opinion (or following review by the Sponsor), could affect the conduct of the study or the absorption, metabolism or excretion of the study treatment.\n* Prior history of autologous or allogeneic stem cell transplantation\n* Marked baseline prolongation of QT\u002FQTc interval (e.g., repeated demonstration of a QTc interval \\> 480 milliseconds \\[msec\\]) (Common Terminology Criteria for Adverse Events \\[CTCAE\\] Grade 1) using Fridericia's QT correction formula.\n* History of additional risk factors for TdP (e.g., symptomatic heart failure with left ventricular ejection fraction \\[LVEF\\] \\\u003C40%, hypokalemia, family history of Long QT Syndrome).\n* Receiving any other concurrent chemotherapy, radiotherapy, or immunotherapy (within 2 weeks of initiating study treatment), or the toxicity of the relevant prior treatment has not been resolved yet. For any long-acting systemic agent such as a monoclonal antibody, study treatment should not begin within two half-lives of the agent.\n* Use of concomitant medications that prolong the QT\u002FQTc interval during study treatment\n* Use of concomitant medications that are strong CYP3A or CYP2B6 inhibitors or inducers during study treatment","ALL","18 Years",{"count":19,"type":20},86,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","Phase 1b Study of R289 in Patients with Lower-risk Myelodysplastic Syndromes (LR MDS)",[27],"Low Risk Myelodysplastic Syndromes",[29,30,31,32,33],"MDS","LR MDS","Myelodysplastic Syndromes","Hematology Oncology","Hem\u002F Onc","RECRUITING","2025-10-17",{"date":37,"type":38},"2025-10-21","ACTUAL",{"date":40,"type":38},"2022-09-12",{"date":42,"type":20},"2026-12",{"name":44,"class":45},"Rigel Pharmaceuticals","INDUSTRY",15,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":21,"phases":56,"briefSummary":57,"conditions":58,"keywords":60,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":5},"100567594","phase-1-oral-arsenic-ato-in-low-risk-myelodysplastic-syndromes-mds-100567594","NCT06670222","Oral Arsenic (ATO) in Low-risk Myelodysplastic Syndromes (MDS)","Phase I Study With Dose-escalation and Expansion Evaluating the Safety and Efficacy of Oral Arsenic (ATO) in Low-risk Myelodysplastic Syndromes Failing Erythropoiesis Stimulating Agents and Luspatercept (or Ineligible for the Latter)","Inclusion criteria:\n\nPatients must meet all the following criteria to participate in the study:\n\n1. Myelodysplastic syndrome according to WHO (World Health Organization) 2022 classification\n2. Age ≥ 18 years\n3. Patient with low-risk Myelodysplastic Syndromes according to Revised International Prognostic Scoring System (IPSS-R) classification (very low, low, intermediate):\n\n   * non-sideroblastic who failed to achieved a response or who subsequently relapse after Erythropoiesis Stimulating Agents (ESA) (at Epoetin alfa 60000UI or equivalent over at least 12 weeks) without disease progression or ineligible to ESA (defined by Erythopoietine (EPO) \\> 500UI\u002FL)\n   * sideroblastic who failed to achieved a response or who subsequently relapse after ESA (at Epoetin alfa 60000UI or equivalent over at least 12 weeks) or ineligible for ESA (defined by EPO \\>500UI\u002FL) and who failed to achieved a response or who subsequently relapse after Luspatercept\n   * del (5q) who failed to achieved a response or who subsequently relapse after ESA (at Epoetin alfa 60000IU or equivalent over at least 12 weeks) and who failed to achieved a response or who subsequently relapse after Lenalidomide\n4. Transfusion dependence (at least 3 RBC (Red Blood Cell) within a 16-week period and at least 2 transfusion episodes during this period)\n5. Patient not eligible for another clinical trial\n6. Adequate renal function defined by creatinine level less than 1.5 times the upper limit of normal and creatinine clearance ≥ 40mL\u002Fmin (according to MDRD (Modification of Diet in Renal Disease) formula)\n7. Adequate liver function defined by total bilirubin and transaminases less than 1.5 times the upper limit of normal\n8. Patient not refractory to platelet transfusions\n9. Written consent\n10. Patient must understand and voluntarily sign informed consent form\n11. Patient must be able to adhere to the visit schedule as outlined in the study and follow protocol requirements\n12. Performance status 0-2 at the time of screening\n13. A FCBP (female of childbearing potential) for this study was defined as a sexually mature woman who: (1) had not undergone a hysterectomy or bilateral oophorectomy; or (2) had not been naturally postmenopausal (amenorrhea following cancer therapy did not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months).\n\n    A FCBP participating in the study must:\n    * Have had 2 negative pregnancy tests as verified by the investigator prior to starting IP (unless the screening pregnancy test was done within 72 hours of Cycle 1 Day 1). She must have had agreed to ongoing a monthly pregnancy testing during the course of the study and after end of treatment.\n    * If sexually active, agreed to have used, and been able to comply with, highly effective contraception\\*\\* without interruption, 5 weeks prior to starting treatment, during treatment (including dose interruptions), and for 24 weeks after discontinuation of treatment.\n\n      * Highly effective contraception was defined in this protocol as the following (information also appeared in the Informed Consent Form): Hormonal contraception (eg, birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device, tubal ligation (tying your tubes), or a partner with a vasectomy.\n14. Male subjects must: Have agreed to use a condom, defined as a male latex condom or nonlatex condom NOT made out of natural (animal) membrane (eg, polyurethane), during sexual contact with a pregnant female or a FCBP while participating in the study, during dose interruptions, and for at least 24 weeks following treatment discontinuation, even if he had undergone a successful vasectomy.\n\nExclusion criteria:\n\nAny patient meeting one of the following criteria cannot be included in the study:\n\n1. Severe infection or any uncontrolled severe condition\n2. Uncontrolled hypertension\n3. Significant cardiac disease - NYHA (New York Heart Association) Class III or IV or having suffered a myocardial infarction in the last 6 months\n4. QTcF (Fridericia's corrected QT interval) \\> 460ms\n5. Use of investigational agents within 30 days or any anticancer therapy (including IMiD (Immunomodulatory treatments)) within 2 weeks before the study entry with the exception of hydroxyurea. The patient must have recovered at least a grade 1 from all acute toxicity from any previous therapy. However, patients may have received Lenalidomide, hypomethylating agent, or anti-lymphocytic serum (ALS) (but not within 4 weeks before the study entry and, for ALS, within 16 weeks before the study entry).\n6. Use of EPO within 4 weeks before the study entry\n7. Active cancer or cancer during the year prior to trial entry other than basal cell carcinoma, or carcinoma in situ of the cervix or breast\n8. Patient already enrolled in another therapeutic trial of an investigational drug\n9. Known Human Immunodeficiency Virus infection or active hepatitis B or C\n10. Women who are or could become pregnant or who are currently breastfeeding\n11. Any medical or psychiatric contraindication that would prevent the patient from understanding and signing the informed consent form\n12. Patient eligible for allogeneic stem cell transplantation\n13. No affiliation to a health insurance system",{"count":55,"type":20},24,[23],"Phase I study with dose-escalation and expansion evaluating the safety and efficacy of oral Arsenic (ATO) in low-risk Myelodysplastic Syndromes having failed to Erythropoiesis Stimulating Agents and Luspatercept (or ineligible for the latter).",[59],"Low-risk Myelodysplastic Syndromes",[61,62],"Oral Arsenic","Low-risk myelodysplastic syndromes","2025-07-29",{"date":65,"type":38},"2025-07-31",{"date":67,"type":38},"2025-07-22",{"date":69,"type":20},"2027-07",{"name":71,"class":72},"Groupe Francophone des Myelodysplasies","OTHER",{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":79,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":83,"phases":4,"briefSummary":84,"conditions":85,"keywords":86,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":98},"100563317","transfusion-in-lower-risk-mds-patients-predictors-of-adequacy-of-transfusion-and-quality-of-life-in-lower-risk-mds-100563317","NCT06614595","Transfusion in Lower Risk MDS Patients: Predictors of Adequacy of Transfusion and Quality of Life in Lower Risk MDS","Transfusion in Lower Risk MDS Patients: Predictors of Adequacy of Transfusion and Quality of Life Changes","MDS-QUAL","Subjects must meet all of the following inclusion criteria:\n\n1. They must be able to understand the study procedures, comply with them, and consent in writing prior to any specific study procedure.\n2. Must be able to understand the study procedures, comply with them, and give written informed consent prior to any study-specific procedure.\n3. Adult subjects ≥ 18 years of age with a diagnosis of myelodysplastic syndrome.\n4. Very low, low or intermediate IPSS-R risk category.\n5. Subject requires are red blood cell transfusion dependent with the following criteria: requires an average of 2 to 6 units of CH on average over an 8-week period.\n6. ECOG 0-3.\n\nExclusion criteria\n\n1. Subject undergoing any of the following treatments: azacitidine, decitabine, venetoclax, cytotoxic chemotherapy, radiotherapy, arsenic trioxide, interferon or interleukin.\n2. High or very high IPSS-R risk category.\n3. Subject diagnosed with any active neoplasm except:\n\n   * epidermoid or basal cell carcinoma,\n   * carcinoma in situ of the uterine cervix\n   * Carcinoma in situ of the breast\n4. Subjects with a score on the New York Heart Association Scale IV.\n5. Subjects with major surgery within 8 weeks prior to study inclusion. Subjects must have fully recovered from any previous surgery prior to inclusion in the study.\n6. Subject with known clinically significant anaemia due to iron, vitamin B12 or folate deficiencies or autoimmune or hereditary haemolytic anaemia or uncontrolled hypothyroidism or known clinically significant haemorrhage or sequestration or subject with drug induced anaemia.\n7. New onset or uncontrolled seizures.\n8. Subjects with uncontrolled systemic fungal, bacterial or viral infections (defined as ongoing signs or symptoms related to the infection without improvement despite appropriate antibiotics or antimicrobial therapy).\n9. Pregnant or breastfeeding women.\n10. The subject has any condition, including the presence of laboratory abnormalities that would place the subject at unacceptable risk if he\u002Fshe were to participate in the study.\n11. The subject has any condition or receives concomitant medication that confounds the ability to interpret the study data.",{"count":82,"type":20},30,"OBSERVATIONAL","Observational study to evaluate changes in quality of life (QoL) in patients diagnosed with low risk MDS who are red blood cell (abc) transfusion dependent.\n\nChanges in QoL will be correlated with patient cardiorespiratory function. Cardiac biomarkers and plasma cytokines will also be evaluated.",[27],[31,87,88],"Red blood cell transfusion","Quality of life","2024-09-25",{"date":91,"type":38},"2024-09-27",{"date":93,"type":38},"2024-09-20",{"date":95,"type":20},"2025-12-21",{"name":97,"class":72},"Fundación para la Investigación Biosanitaria del Principado de Asturias",1]