[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myelodysplastic-syndrome-mds\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myelodysplastic-syndrome-mds":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,47,70,94,116,152],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":31,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100299122","phase-1-unrelated-umbilical-cord-blood-transplantation-for-severe-aplastic-anemia-and-hypo-plastic-mds-using-cordintm-umbilical-cord-blood-derived-ex-vivo-expanded-stem-and-progenitor-cells-to-expedite-engraftment-and-improve-transplant-outcome-100299122",false,"NCT03173937","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count \\\u003C=1000 cells\u002F uL or for patients receiving granulocyte transfusions, absolute neutrophil count \\\u003C=1000 cells\u002F uL before beginning granulocyte transfusions).\n\nOR\n\nHistory of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C 5% myeloblasts and \\\u003C 30% of cellularity in the bone marrow on screening morphologic analysis.\n\n-Intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n\nIdentification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>=5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>=9\u002F10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn(TM) unit has been rejected or b) umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the CordIn(TM) unit has been rejected.\n\n-Availability of at least one \\>=4\u002F8 HLA matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).\n\nThe cord blood unit must contain a minimum TNC of at least 1.8 x 10\\^9 and at least 1.5x10\\^7\u002Fkg TNC and at least 8 x 10\\^6 CD34+ cells (all doses prior to thawing).\n\nException: Cord units containing at least 8 x 10\\^6 CD34+ cells but less than 1.8 x 10\\^9 TNC may be eligible for use on this trial if\n\n1. the pre-expansion CD34\u002Fkg recipient cell number is at least 1.5 x 10\\^5 \u002Fkg\n\n   AND\n2. approval for use of this cord unit for expansion is granted by Gamida Cell.\n3. the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.\n\n   * The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.\n   * Ages 4-60 years inclusive.\n   * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C 40% (evaluated by ECHO).\n* Transaminases \\> 5x upper limit of normal.\n* Serum bilirubin \\>4 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n* Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.\n* Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.","ALL","4 Years","75 Years",{"count":20,"type":21},37,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","Background:\n\nSevere aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective.\n\nObjective:\n\nTo test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.\n\nEligibility:\n\nRecipients ages 4-60 with SAA or MDS\n\nDonors ages 4-75\n\nDesign:\n\nRecipients will be screened with:\n\n* Blood, lung, and heart tests\n* Bone marrow biopsy\n* CT scan\n\nRecipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose.\n\nRecipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits:\n\n* First 3-4 months: 1-2 times weekly\n* Then every 6 months for 5 years\n\nDonors will be screened with:\n\n* Physical exam\n* Medical history\n* Blood tests\n\nDonors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein.\n\nDonors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.",[28,29,30],"Severe Aplastic Anemia","Hypo-Plastic MDS","Myelodysplastic Syndrome (MDS)",[32,33],"Haploidentical","Nonmyeloablative","RECRUITING","2026-07-01",{"date":37,"type":38},"2026-07-02","ACTUAL",{"date":40,"type":38},"2017-06-13",{"date":42,"type":21},"2032-03-01",{"name":44,"class":45},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":51,"acronym":4,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":56,"briefSummary":57,"conditions":58,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":4},"100281551","phase-2-a-study-of-blex-404-oral-liquid-in-patients-with-international-prognostic-scoring-system-ipss-intermediate-1-intermediate-2-or-high-risk-myelodysplastic-syndrome-mds-and-chronic-myelomonocytic-leukemia-cmml-100281551","NCT02944955","A Study of BLEX 404 Oral Liquid in Patients With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-Risk Myelodysplastic Syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML)","Inclusion Criteria:\n\n* Age 20 or older, able to sign informed consent, understand and comply with protocol requirements and instructions.\n* Body weight between 30 to 120 kg\n* International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) by World Health Organization (WHO) or Chronic Myelomonocytic Leukemia (CMML) patients.\n* Not previously treated with MDS and CMML therapy and require treatment with azacitidine\\* for the first time at 75 mg\u002Fm2 subcutaneously or intravenously, daily for 7 days (7 nonconsecutive day of 6-1-1 or 5-2-2 treatment schedule is acceptable in which the drug is administered Monday-Saturday and then Monday of the following week for a 6-1-1 schedule, and in which the drug is administered Monday-Friday and then Monday-Tuesday of the following week for 5-2-2 schedule) per cycle. Treatment of the study drug, BLEX 404 Oral Liquid will begin at the same time receiving the first dose of azacitidine in this study.\n* Eastern Cooperative Oncology Group (ECOG) Performance Status 0-3.\n* Adequate baseline organ function defined by the criteria below: total bilirubin ≤ 1.5x the upper limit of normal (ULN) except for Gilbert's syndrome or indirect hyperbilirubinemia due to hemolysis; ALT ≤ 2.5x ULN; AST ≤ 2.5x ULN; creatinine clearance ≥ 60 mL\u002Fmin\u002F1.73 m2.\n* Women must be either of non-child bearing potential, or women with child-bearing potential and men with reproductive potential and a female partner of childbearing potential must either have had a prior vasectomy or agree to use effective contraception from time of Screening Visit until the Follow-Up Visit.\n\nExclusion Criteria:\n\n* Historical allergic events caused by mushroom.\n* Previous treatment with hypomethylating agent or known immediate or delayed hypersensitivity reaction to drugs chemically related to azacitidine that contraindicates the subjects' participation.\n* Patients with active infections or require treatments with immunosuppressive drugs at screening visit. However, patients require ongoing treatments with corticosteroids may be recruited.\n* Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of BLEX 404 Oral Liquid.\n* Any serious and\u002For unstable pre-existing medical condition (including any advanced malignancy other than the disease under study), psychiatric disorder, or other conditions that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures.\n* Any clinically significant pulmonary, cardiovascular, endocrine, neurologic, gastrointestinal or genitourinary disease unrelated to underlying hematologic disorder.\n* Active and uncontrolled infections of the following: hepatitis B (HBV), hepatitis C (HCV), and human immunodeficiency virus (HIV) infection. However, HBV and HCV carriers with viral load test of \"not detected\" or \"negative\" results may be recruited in this study.\n* Lactating female and women with a positive serum or urine pregnancy test at screening assessments.\n* (For Part II Interventional Study subjects only) No indication, in the opinion of the treating physician, for antibiotic prophylaxis within 2 weeks prior to study treatment.","20 Years",{"count":55,"type":21},52,[25],"This is a clinical trial to determine the safety, recommended dose level (RDL), and infection control of BLEX 404 Oral Liquid in combination with azacitidine in patients with International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML).",[30],"NOT_YET_RECRUITING","2026-04-08",{"date":62,"type":38},"2026-04-13",{"date":64,"type":21},"2027-01-01",{"date":66,"type":21},"2028-12-31",{"name":68,"class":69},"BioLite, Inc.","INDUSTRY",{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":4,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":16,"minAge":77,"maxAge":4,"enrollmentInfo":78,"targetDuration":4,"studyType":22,"phases":80,"briefSummary":81,"conditions":82,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":84,"lastUpdatePostDateStruct":85,"startDateStruct":87,"completionDateStruct":89,"leadSponsor":91,"locationsCount":93},"100450323","phase-1-cln-049-in-patients-with-relapsedrefractory-acute-myeloid-leukemia-aml-or-myelodysplastic-syndrome-mds-100450323","NCT05143996","CLN-049 in Patients With Relapsed\u002FRefractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)","A Phase 1, Open-label, Preliminary Pharmacokinetics (PK) and Safety Study of CLN-049 (An Fms-like Tyrosine Kinase 3 [FLT3] x Cluster of Differentiation 3 [CD3] Bispecific T Cell Engager) in Patients With Relapsed\u002FRefractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)","Inclusion Criteria:\n\n1. Aged ≥ 18 years of age.\n2. Willing and able to give written informed consent and adhere to protocol requirements; written informed consent and any locally required authorization must be obtained from the patient prior to performing any protocol-related procedures, including screening evaluations, and serial samples of bone marrow and peripheral blood.\n3. Patient has a confirmed diagnosis of recurrent or refractory AML or MDS.\n4. Patient has received, and has progressed, recurred, or is intolerant of approved therapeutic options that are available, or declines treatment with these therapies.\n5. White blood cell (WBC) count at the time of the first dose is \\\u003C 20,000\u002FuL (hydroxyurea is permitted according to standard institutional practice). Following first dose, WBC should be checked prior to subsequent CLN-049 administration and if WBC \\> 20,000\u002FμL, CLN-049 treatment should be postponed (see Section 6.1 for further guidance).\n6. Eastern Cooperative Oncology Group (ECOG) performance status is 0 to 2.\n7. Toxicities related to prior study therapy should have resolved to Grade 1 or less according to criteria of NCI CTCAE v5.0, except for alopecia, lymphopenia, neutropenia, leukopenia, anemia, thrombocytopenia. Patients with chronic but stable toxicities may be allowed to enroll after agreement between the Investigator and Sponsor.\n8. The patient's laboratory values meet the following criteria:\n\n   1. Creatinine clearance (CrCl) as calculated by the Cockcroft-Gault formula (Appendix 1) must be ≥ 60 mL\u002Fmin;\n   2. Total bilirubin ≤ 1.5 × ULN. This does not apply for patients with confirmed Gilbert's Syndrome, hemolysis, or chronic blood transfusions, for whom total bilirubin must be less than 3.0 mg\u002FdL with a conjugated bilirubin less than 0.5 mg\u002FdL;\n   3. AST and ALT ≤ 3.0 × ULN (unless attributed to leukemic involvement).\n\nExclusion Criteria:\n\n1. Diagnosis of acute promyelocytic leukemia (APL)\n2. Active central nervous system (CNS) leukemia. For patients with a history of CNS leukemia, a lumbar puncture should be performed during screening to exclude the presence of active CNS involvement.\n3. Isolated extramedullary relapse\n4. Prior organ allograft\n5. Allogeneic hematopoietic transplantation within six months of treatment, or with clinical or laboratory evidence of GVHD, or requiring ongoing treatment with immune suppression within 2 months of the first dose of CLN-049.\n6. Treatment with any of the following:\n\n   1. Radiation therapy (XRT) within 28 days of the first dose of CLN049, or craniospinal XRT within 8 weeks of the first dose of CLN-049, or history of total body irradiation (TBI).\n   2. Prior immunotherapy with checkpoint inhibitors ≤ 42 days prior to the first dose of CLN-049.\n   3. Prior history of chimeric antigen receptor (CAR-T) cell therapy or other modified T cell therapy.\n   4. Anti-leukemic therapy except hydroxyurea for cytoreduction, and intrathecal chemotherapy ≤ 14 days or 5 half-lives, whichever is shorter, prior to the first dose of CLN-049.\n   5. Short-acting hematopoietic growth factors ≤ 7 days prior to the first dose of CLN-049\n   6. Long-acting growth factors ≤ 14 days prior to the first dose of CLN-049.\n   7. Systemic glucocorticoid therapy (except equivalent of \\\u003C 10 mg prednisone daily) or other immune-suppressive drugs ≤ 14 days prior to the first dose of CLN-049 (see separate guidelines for patients who are post allogeneic hematopoietic transplantation). The transient use of corticosteroids for transfusion premedication or the treatment of infusion or transfusion reactions will not be considered for this criterion. Topical corticosteroids and steroid eye drops are allowed, and will not exclude the patient from eligibility.\n   8. Prior treatment with a FLT3-directed bispecific molecule, or a FLT3-targeted antibody.\n7. Currently participating\u002Fpreviously participated in an interventional study and received an investigational drug within 14 days (or five half-lives, whichever is longer) prior to the first dose of CLN-049.\n8. Patients with concomitant second malignancies requiring active treatment in the past 12 months, or if additional therapy is required or anticipated during study participation.\n9. Patients with any active autoimmune disease or a history of known or suspected autoimmune disease, or history of a syndrome that requires systemic corticosteroids or immunosuppressive medications, except for patients with vitiligo, psoriasis (in consultation with the Sponsor), resolved childhood asthma\u002Fatopy or autoimmune thyroid disorders on stable thyroid hormone supplementation.\n10. A serious uncontrolled medical disorder that would impair the ability of the patient to receive protocol therapy or whose control may be jeopardized by the complications of this therapy.\n11. Any concurrent condition, therapy, or laboratory abnormality that, in the opinion of the investigator, might compromise patient safety or interfere with the evaluation of the safety of the drug.\n12. Active uncontrolled infection within seven days of first dose of CLN-049. Prophylactic use of systemic antiviral, antibacterial, or antifungal agents for treatment of chronic, controlled infection or as prophylaxis is permitted.\n13. Has a history of, or a positive test for Human Immunodeficiency Virus (HIV) 1\u002F2 or primary immunodeficiency disease such as HIV.\n14. Known history of hepatitis B (with positive testing for either hepatitis B surface antigen \\[HBsAg\\] or hepatitis B core Ab), hepatitis C (HCV) infection (with positive testing for HCV antibody and\u002For HCV ribonucleic acid \\[RNA\\] in serum), or acute hepatitis A (with positive testing for hepatitis A IgM). Note: patients with chronic HCV with undetectable viral load defined by sustained virologic response 24 weeks (SVR24) after completion of anti-hepatitis C treatment will be eligible. Patients with hepatitis B surface antigen \\[HBsAg\\] or hepatitis B core Ab with negative viral load will be eligible.\n15. Active severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection including history of positive SARS-CoV-2 testing without subsequent documentation of negative test results, patients with results that are pending but not yet known, or patients with suspected active infection based on clinical features\n16. History of the following events in conjunction with prior treatment with immunotherapy: Grade 3 or greater neurotoxicity, ocular toxicity, pneumonitis, myocarditis, or colitis; liver dysfunction meeting the laboratory criteria for Hy's Law.\n17. Live virus vaccines within 28 days of the first dose of CLN-049, during treatment, and until the end of last dose of CLN-049.\n18. Woman of child-bearing potential (WOCBP) who is pregnant or breast-feeding, plans to become pregnant within 120 days of last study drug administration, or declines to use an acceptable method to prevent pregnancy during study treatment and for 120 days after the last dose of study drug administration.\n19. Male patient who plans to father a child or donate sperm within 120 days of last study drug administration, or who has a partner who is a WOCBP, and declines to use acceptable method to prevent pregnancy during study treatment and for 120 days after the last dose of study drug administration.\n20. QT interval corrected for heart rate using Fridericia's formula (QTcF) of ≥ 480 milliseconds.\n21. Patient has history of drug-related anaphylactic reactions to any components of CLN-049. History of Grade 4 anaphylactic reaction to any bispecific molecule or monoclonal antibody therapy.\n22. Known history of prior human anti-human antibody response. Patients will not be screened for human anti-human antibody prior to study participation.\n23. Known active alcohol or drug abuse.\n24. Patients who are incapacitated or involuntarily incarcerated.","18 Years",{"count":79,"type":21},60,[24],"CLN-049-001 is a Phase 1, open-label, multicenter, first-in-human trial of CLN-049 in patients with Relapsed\u002FRefractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)",[83,30],"Relapsed\u002FRefractory Acute Myeloid Leukemia (AML)","2026-01-14",{"date":86,"type":38},"2026-01-16",{"date":88,"type":38},"2021-11-18",{"date":90,"type":21},"2027-06",{"name":92,"class":69},"Cullinan Therapeutics Inc.",11,{"id":95,"slug":96,"hasResults":11,"nctId":97,"briefTitle":98,"officialTitle":99,"acronym":4,"eligibilityCriteria":100,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":4,"enrollmentInfo":101,"targetDuration":4,"studyType":22,"phases":103,"briefSummary":104,"conditions":105,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":106,"lastUpdatePostDateStruct":107,"startDateStruct":109,"completionDateStruct":111,"leadSponsor":113,"locationsCount":115},"100409178","phase-1-a-phase-1-trial-of-astx030-in-patients-with-myelodysplastic-syndrome-100409178","NCT04608110","A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome","A Phase 1, Multi-center, Open-label, Uncontrolled, Dose-escalation Study to Evaluate the Pharmacokinetics of ASTX030 in Patients With Myelodysplastic Syndrome (MDS)","Inclusion Criteria:\n\n* Patients aged 20 years or older\n* Patients with a diagnosis of MDS (refractory anemia \\[RA\\], refractory anemia with ringed sideroblasts \\[RARS\\], refractory anemia with excess blasts \\[RAEB\\], refractory anemia with excess blasts in transformation \\[RAEB-T\\], or chronic myelomonocytic leukemia \\[CMML\\]) according to the French-American-British (FAB) classification Low-risk patients who fall under the risk category of low or intermediate-1 (Int-1) based on the International Prognostic Scoring System (IPSS) can be enrolled only if they are unlikely to respond to any other treatment or if they are currently being treated with azacytidine (AZA) injection\n* Patients with an ECOG PS score of 0 or 1 or with an ECOG PS score of 2 due to primary disease-associated conditions\n* Patients with adequate organ function as indicated below\n\n  1. Hepatic function: All of the following criteria must be satisfied.\n\n     * Total bilirubin ≤ 2.0 × upper limit of normal (ULN)\n     * Aspartate aminotransferase (AST) ≤ 2.5 × ULN\n     * Alanine aminotransferase (ALT) ≤ 2.5 × ULN\n  2. Renal function: Either of the following criteria must be satisfied.\n\n     * Serum creatinine ≤ 1.5 × ULN\n     * Creatinine clearance or glomerular filtration rate ≥ 50 mL\u002Fmin\n  3. Respiratory function: percutaneous arterial oxygen saturation (SpO2) ≥ 90%\n* Patients who are expected to survive for at least 3 months\n* Patients who give written consent to participate in the trial using the informed consent form approved by the institutional review board\n\nExclusion Criteria:\n\n* Patients who are unlikely to respond to AZA\n* Patients who have received chemotherapy, hormone therapy, antibody therapy, radiotherapy, or other exploratory anti-cancer treatments for the primary disease within 3 weeks prior to the first administration of the investigational medicinal product (IMP)\n* Patients who have used any other IMP or any privately imported medicine within 4 weeks prior to the first administration of IMP\n* Patients with heart disease of Class 3 or 4 according to the New York Heart Association classification\n* Patients with uncontrolled systemic disease or active infection\n* Patients with uncontrolled gastric or duodenal ulcer\n* Patients with prior or current interstitial lung disease\n* Patients with a history of surgical gastrectomy\n* Patients with life-threatening conditions\u002Fsymptoms, multiple organ failure, or other factors (including laboratory abnormalities) that, in the opinion of the investigator, are likely to affect their safety or the absorption and metabolism of AZA and cedazuridine (CED), or influence the trial evaluation\n* Patients with other malignancies (except appropriately treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma in situ; prostate or breast cancer stabilized by endocrine therapies; and malignancies that have not relapsed for at least 1 year since the last successful treatment)\n* Patients who are positive for HIV antibody, HBV-DNA, or HCV antibody\n* Patients with any ≥ Grade 2 AE (except alopecia) associated with prior treatment of the primary disease. However, the parameters defined in inclusion criterion above are excluded.\n* Patients who have undergone a highly invasive and extensive surgical procedure within 4 weeks prior to the first administration of IMP\n* Patients who previously underwent or plan on undergoing hematopoietic stem cell transplantation\n* Patients with a history of hypersensitivity to the active ingredient or any excipient of IMP\n* Patients who are, in the opinion of the investigator, at high risk for being unable to comply with the trial protocol because of mental disorders or other medical conditions (alcohol\u002Fsubstance abuse or addiction)\n* Pregnant or nursing female patients, or female patients with a positive pregnancy test at screening. Nursing patients cannot participate in the trial even if they discontinue breastfeeding. Female patients must undergo a pregnancy test to confirm that they are not pregnant at screening. However, a pregnancy test is not necessary for female patients without childbearing potential (ie, patients with a history of bilateral oophorectomy or hysterectomy or who have been postmenopausal for at least 12 months except for cases where menopause could be due to the effect of antineoplastic treatment).\n* Sexually active males (except those with a history of bilateral orchiectomy) or females of childbearing potential who do not agree to practice 2 different methods of birth control or remain abstinent during the trial and for 3 months (males) and 6 months (females) after the last dose of IMP. If birth control is employed, 2 of the following precautions must be used: vasectomy, tubal ligation, intrauterine device, oral contraceptive, and condom (all methods approved or certified in Japan)\n* Patients who, in the opinion of the investigator, are otherwise ineligible to participate in the trial",{"count":102,"type":21},40,[24],"The purpose of this study is to identify the doses of the oral azacitidine formulations and cedazuridine (CED) tablets which achieve a total AUC for AZA comparable to that for AZA injection at 75 mg\u002Fm2",[30],"2025-11-25",{"date":108,"type":38},"2025-12-03",{"date":110,"type":38},"2020-10-30",{"date":112,"type":21},"2026-01",{"name":114,"class":69},"Taiho Pharmaceutical Co., Ltd.",10,{"id":117,"slug":118,"hasResults":11,"nctId":119,"briefTitle":120,"officialTitle":121,"acronym":122,"eligibilityCriteria":123,"healthyVolunteers":11,"sex":16,"minAge":124,"maxAge":4,"enrollmentInfo":125,"targetDuration":4,"studyType":22,"phases":127,"briefSummary":128,"conditions":129,"keywords":133,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":141,"lastUpdatePostDateStruct":142,"startDateStruct":144,"completionDateStruct":146,"leadSponsor":148,"locationsCount":151},"100220190","phase-2-hsct-for-patients-with-fanconi-anemia-using-risk-adjusted-chemotherapy-100220190","NCT02143830","HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy","A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine","RAFA","Inclusion Criteria:\n\n* Patients must have a diagnosis of Fanconi anemia\n* Patients must have one of the following hematologic diagnoses:\n\n  1. Severe Aplastic Anemia (SAA), with bone marrow cellularity of \\\u003C25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features:\n\n     1. Platelet count \\\u003C20 x 109\u002FL or platelet transfusion dependence\\*\n     2. ANC \\\u003C1000 x 109\u002FL\n     3. Hgb \\\u003C8 gm\u002Fdl or red cell transfusion dependence\\*\n  2. Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification\n  3. Acute Myelogenous Leukemia (untreated, in remission or with refractory or relapsed disease)\n* Donors will be either human leukocyte antigen (HLA) compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor).\n* Patients and donors may be of either gender or any ethnic background.\n* Patients must have a Karnofsky adult, or Lansky pediatric performance scale status \\> 70%.\n* Patients must have adequate physical function measured by:\n\n  1. Cardiac: asymptomatic or if symptomatic then 1) left ventricular ejection fraction (LVEF) at rest must be \\> 50% and must improve with exercise or 2) Shortening Fraction \\> 29%\n  2. Hepatic: \\\u003C 5 x upper limit of normal (ULN) alanine transaminase (ALT) and \\\u003C 2.0 mg\u002Fdl total serum bilirubin.\n  3. Renal: serum creatinine \\\u003C1.5 mg\u002Fdl or if serum creatinine is outside the normal range, then CrCl \\> 50 ml\u002Fmin\u002F1.73 m2\n  4. Pulmonary: asymptomatic or if symptomatic, DLCO \\> 50% of predicted\n* Each patient must be willing to participate as a research subject and must sign an informed consent form.\n* Female patients and donors must not be pregnant or breastfeeding at the time of signing consent. Women must be willing to undergo a pregnancy test prior to transplant and avoid becoming pregnant while on study.\n\nExclusion Criteria:\n\n* Active CNS leukemia\n* Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.\n* Active uncontrolled viral, bacterial or fungal infection\n* Patient seropositive for HIV-I\u002FII; HTLV -I\u002FII","3 Months",{"count":126,"type":21},70,[25],"The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.",[130,131,30,132],"Fanconi Anemia","Severe Marrow Failure","Acute Myelogenous Leukemia (AML)",[134,135,136,137,138,139,140],"marrow aplasia","cytopenia","myelodysplasia","AML","bone marrow transplant","cytoreductive regimen","T-cell reduction","2025-11-10",{"date":143,"type":38},"2025-11-12",{"date":145,"type":4},"2014-04",{"date":147,"type":21},"2028-12",{"name":149,"class":150},"Children's Hospital Medical Center, Cincinnati","OTHER",3,{"id":153,"slug":154,"hasResults":11,"nctId":155,"briefTitle":156,"officialTitle":157,"acronym":4,"eligibilityCriteria":158,"healthyVolunteers":11,"sex":16,"minAge":77,"maxAge":4,"enrollmentInfo":159,"targetDuration":4,"studyType":22,"phases":161,"briefSummary":162,"conditions":163,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":168,"completionDateStruct":170,"leadSponsor":172,"locationsCount":174},"100558407","phase-1-a-clinical-trial-of-tqb3455-tablets-in-patients-with-hematological-malignancies-100558407","NCT06550713","A Clinical Trial of TQB3455 Tablets in Patients With Hematological Malignancies","Phase I Clinical Trial Protocol for Tolerability and Pharmacokinetics of TQB3455 Tablets in Patients With Hematological Malignancies","Inclusion Criteria:\n\nPatients meeting all of the following inclusion criteria can be included in this trial:\n\n* Age ≥ 18 years old;\n* According to the World Health Organization (WHO) classification, subjects diagnosed with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) should meet one of the following criteria:\n\n  1. Difficult to treat or recurrent (\\>5% of primitive cells reappear in the bone marrow after complete remission) AML; (Single drug group)\n  2. Newly diagnosed AML subjects recognized by researchers as unable to receive standard treatment due to age, physical condition, or risk factors; (Joint group)\n* MDS subjects belong to the following prognostic risk categories according to the revised International Prognostic Scoring System (IPSS-R):\n\n  1. Extremely high-risk (\\>6 points)\n  2. High risk (\\>4.5 points - ≤ 6 points)\n  3. Medium risk (\\>3 points - ≤ 4.5 points)\n* Clearly indicating the presence of IDH2 gene mutation;\n* Blood platelet (PLT) ≥20×10\\^9\u002FL； Or subjects with PLT\\\u003C20 × 10\\^9\u002FL, but recognized by the researchers as being caused by tumor reasons;\n* Serum total bilirubin ≤ 1.5 × ULN (for Gilbert syndrome subjects, bilirubin ≤ 3 × ULN);\n* Renal function: serum creatinine ≤ 1.5 × ULN or creatinine clearance rate ≥ 50ml\u002Fmin;\n* Recovery of toxic reactions caused by surgery, radiation therapy, or other anti-tumor treatments to ≤ Grade I;\n* Women should agree to use contraceptive measures during the study period and within 6 months after the end of the study; Male participants must agree to use contraception during the study period and within 6 months after the end of the study period;\n* The subjects voluntarily joined this study.\n\nExclusion Criteria:\n\n* Subjects who experience relapse after bone marrow transplantation;\n* Subjects who have received systemic anti-tumor therapy or radiation therapy within 3 weeks prior to the use of the investigational drug;\n* Individuals who have participated in clinical trials of other drugs within the four weeks prior to using the investigational drug;\n* Individuals with multiple factors that affect oral medication, such as inability to swallow, post gastrointestinal resection, chronic diarrhea, and intestinal obstruction;\n* Subjects who have previously used targeted isocitrate dehydrogenase 2 (IDH2) inhibitors;\n* The subject has uncontrolled systemic fungal, bacterial, or viral infections;\n* High blood pressure subjects who are still poorly controlled despite drug treatment;\n* Obvious cardiovascular diseases, such as heart failure classified as grade 2 or above by the New York Heart Association (NYHA), unstable angina in the past 3 months, myocardial ischemia or infarction, arrhythmia and grade I heart failure, or the presence of other factors at risk of prolonging the QT interval (such as arrhythmia, hypokalemia ≥ grade 3, family history of long QT interval);\n* Severe leukemia complications that endanger life, such as uncontrolled bleeding, hypoxia or shock pneumonia, disseminated intravascular coagulation;\n* Subjects known to have central nervous system leukemia or clinical symptoms of central nervous system leukemia;\n* Individuals with a history of abuse of psychotropic drugs who are unable to quit or have mental disorders;\n* Subjects with active replication of hepatitis B virus and hepatitis C virus;\n* Individuals with a history of immunodeficiency, including HIV positive or other acquired or congenital immunodeficiency diseases, or a history of organ transplantation;\n* According to the researcher's judgment, there are accompanying diseases that pose a serious threat to the safety of the subjects or affect their ability to complete the study.",{"count":160,"type":21},100,[24],"This study is a clinical trial to evaluate the tolerability and pharmacokinetics of TQB3455 tablets in patients with hematological malignancies. TQB3455 is an isocitrate dehydrogenase 2(IDH2) inhibitor . This project is divided into two stages. The first stage aims to evaluate the safety and tolerability of single or multiple oral administration of TQB3455 tablets in subjects with malignant hematological tumors. The second phase aims to evaluate the efficacy and safety of TQB3455 tablets alone or in combination with azacitidine in subjects with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).",[164,30],"Acute Myeloid Leukemia (AML)","2024-08-08",{"date":167,"type":38},"2024-08-13",{"date":169,"type":38},"2019-10-22",{"date":171,"type":21},"2026-12",{"name":173,"class":69},"Chia Tai Tianqing Pharmaceutical Group Co., Ltd.",7]