[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Sumitomo Pharma America, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":172},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,41,63,84,118,141],{"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":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100376029","phase-1-a-study-of-oral-nuvisertib-tp-3654-in-patients-with-myelofibrosis-100376029",false,"NCT04176198","A Study of Oral Nuvisertib (TP-3654) in Patients With Myelofibrosis","A Phase 1\u002F2, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Oral Nuvisertib (TP-3654) in Patients With Intermediate or High-Risk Primary or Secondary Myelofibrosis","Patients must meet all of the following inclusion criteria to be eligible:\n\nNuvisertib (TP-3654) Monotherapy Arm:\n\n* Confirmed pathological diagnosis of primary myelofibrosis (PMF) or post-PV-MF\u002Fpost-ET- MF and intermediate or high-risk primary or secondary MF\n* Previously treated with JAK inhibitor(s) and is intolerant, resistant, refractory or has lost response to the JAK inhibitor(s) or is ineligible to be treated with JAK inhibitor\n* Fulfill the following clinical laboratory parameters:\n* Platelet count ≥ 25 x 10\\^9 \u002FL, without assistance of growth factors or platelet transfusions\n* ANC ≥ 1 x 10\\^9\u002FL without assistance of granulocyte growth factors\n* Peripheral blood blast count \\\u003C 5%\n* ECOG performance status ≤ 1\n* Life expectancy ≥ 6 months\n* Adequate renal function\n* Adequate hepatic function\n* Adequate coagulation function\n* Splenomegaly (spleen volume of ≥ 450 cm3 by MRI or CT scan) within 2 weeks prior to Cycle 1 Day 1.\n* Dose escalation: At least 2 symptoms measurable (score ≥ 1) using the MF-SAF\n* Dose expansion: At least 2 symptoms measurable with each score of ≥ 3 or a total average score of ≥ 10 per MFSAF\n\nNuvisertib (TP-3654) + Ruxolitinib Arm:\n\n* Confirmed pathological diagnosis of PMF or post-PV-MF\u002Fpost ET- MF and intermediate or high-risk primary or secondary MF\n* On ruxolitinib treatment for ≥ 6 months, and on a stable dose of ruxolitinib (5 to 25 mg BID) for ≥ 8 weeks prior to the first dose of nuvisertib, but has either lost response or had a suboptimal or plateau in response\n* Fulfills the following clinical laboratory parameters:\n* Platelet count ≥ 50 × 10\\^9\u002FL (without assistance of growth factors or platelet transfusions)\n* ANC ≥ 1 × 109\u002FL without assistance of granulocyte growth factors\n* Peripheral blood blast count \\\u003C 5% at screening\n* Adequate renal function\n* Adequate hepatic function\n* Adequate coagulation function\n* Splenomegaly (spleen volume of ≥ 450 cm3 by MRI\u002FCT scan) within 2 weeks prior to Cycle 1 Day 1\n* At least 2 symptoms measurable with each score ≥ 3 or a total average score of ≥ 10 per MFSAF v4.0\n* ECOG performance status ≤ 1\n* Life expectancy ≥ 6 months\n\nNuvisertib (TP-3654) + Momelotinib Arm\n\n* Confirmed pathological diagnosis of PMF or post-PV-MF\u002Fpost ET-MF and intermediate or high-risk primary or secondary MF\n* Previously treated with an approved JAK inhibitor (except momelotinib) for PMF or Post-PV\u002FET MF for ≥ 12 weeks, or ≥ 4 weeks if JAK inhibitor therapy was complicated by a transfusion requirement of ≥ 4 units of red blood cells in 8 weeks, or Grade 3\u002F4 AEs of thrombocytopenia, anemia, or hematoma\n* Fulfills the following clinical laboratory parameters:\n* Anemic, defined as Hb \\\u003C10 g\u002FdL or requiring RBC transfusion at baseline\n* Platelet count ≥ 50 × 109\u002FL (without assistance of growth factors or platelet transfusions)\n* ANC ≥ 1 × 109\u002FL without assistance of granulocyte growth factors\n* Peripheral blood blast count \\\u003C 5% at screening\n* Adequate renal function\n* Adequate hepatic function\n* Adequate coagulation function\n* Splenomegaly (spleen volume of ≥ 450 cm3 by MRI\u002FCT scan) within 2 weeks prior to Cycle 1 Day 1\n* At least 2 symptoms measurable with each score of ≥ 3 or a total average score of ≥ 10 per MFSAF v4.0\n* ECOG performance status ≤ 1\n* Life expectancy ≥ 6 months\n\nPatients meeting any one of these exclusion criteria will be prohibited from participating in this study:\n\nNuvisertib (TP-3654) Monotherapy Arm:\n\n* Received previous systemic antineoplastic therapy or any experimental therapy within 2 weeks or 5 half-lives, whichever is longer, prior to Cycle 1 Day 1. Hydroxyurea or anagrelide are allowed up to 24 hours prior to Cycle 1 Day 1).\n* Major surgery within 4 weeks prior to Cycle 1 Day 1 and\u002For not recovered adequately from from surgery prior to first dose.\n* Splenic irradiation within 6 months prior to Screening or prior splenectomy.\n* Prior allogeneic stem cell transplant within the last 6 months.\n* Eligible for allogeneic bone marrow or stem cell transplantation.\n* Unresolved Grade ≥ 2 non-hematological toxicity related to prior treatment\n* History of symptomatic congestive heart failure, or myocardial infarction, or uncontrolled arrhythmia within 6 months prior to Cycle 1 Day 1; left ventricular ejection fraction (LVEF) \\\u003C 45% by echocardiogram within 4 weeks prior to Cycle 1 Day 1.\n* Corrected QT interval \\> 480msec.\n* Prior or concurrent malignancy that could interfere with the investigational regime.\n* Known history of chronic liver disease, e.g. portal hypertension or any of its complications, cirrhosis, Child-Pugh C, auto-immune hepatitis, alpha-1 anti-trypsin deficiency, Wilson's disease, etc.\n* Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic antimicrobial within 1 week prior to Cycle 1 Day 1.\n* Chronic active or acute viral hepatitis A, B, or C infection (testing for hepatitis B and C are required)\n* Exhibited allergic reactions or sensitivity to nuvisertib, or similar compound.\n* Medical condition or GI tract surgery that could impair absorption or result in short bowel syndrome with diarrhea.\n* Systemic steroid therapy (\\>10 mg daily prednisone or equivalent) within 1 week prior to the first dose of study treatment (note: topical, inhaled, nasal, and ophthalmic steroids are not prohibited).\n* Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or thalassemia, or severe GI bleeding.\n* Pregnant or breastfeeding\n* Currently receiving any other investigational agent.\n\nNuvisertib (TP-3654) + Ruxolitinib Arm:\n\n* Received previous systemic antineoplastic therapy (other than ruxolitinib) or any other experimental therapy within 2 weeks or 5 half-lives, whichever is longer, prior to Cycle 1 Day 1 (Note: Prior treatment with nuvisertib is not allowed. Hydroxyurea or anagrelide are allowed up to 24 hours prior to Cycle 1 Day 1).\n* Received systemic steroid therapy (\\>10 mg daily prednisone or equivalent) within 1 week prior to Cycle 1 Day 1 (Note: Topical, inhaled, nasal, and ophthalmic steroids are not prohibited)\n* Known allergic reactions or sensitivity to nuvisertib, or similar compound.\n* Splenic irradiation within 6 months prior to Screening or prior splenectomy\n* Prior allogeneic stem cell transplant within the last 6 months (Note: Patients who have relapsed after 6 months post-transplant and do not have active GVHD are eligible).\n* Eligible for allogeneic bone marrow or stem cell transplantation (Note: Patients who are not willing to undergo transplantation or for whom a suitable donor is not available are considered as transplant ineligible.)\n* Major surgery within 4 weeks prior to Cycle 1 Day 1 and\u002For have not recovered adequately prior to first dose.\n* Active, uncontrolled bacterial, viral, or fungal infections, requiring parenteral antimicrobial within 1 week prior to Cycle 1 Day 1\n* Chronic active or acute viral hepatitis A, B, or C infection (testing for hepatitis B and C are required)\n* Known history of chronic liver disease (eg, portal hypertension or any of its complications, cirrhosis, Child-Pugh C, auto-immune hepatitis, alpha-1 anti-trypsin deficiency, Wilson's disease, etc) (Note: Abnormal liver morphology at baseline imaging may require additional testing, as needed).\n* Unresolved Grade ≥ 2 non-hematological adverse events related to prior treatment (stable Grade 2 conditions may be permitted in consultation with the Sponsor)\n* History of myocardial infarction or symptomatic congestive heart failure or uncontrolled arrhythmia within 6 months prior to Cycle 1 Day 1; LVEF \\\u003C45% by echocardiogram within 4 weeks prior to Cycle 1 Day 1\n* Corrected QTcF of \\> 480 msec\n* Prior or concurrent malignancy that could interfere with the safety or efficacy assessment of the study intervention\n* History of a medical condition or GI tract surgery that could impair absorption or could result in short bowel syndrome with diarrhea\n* Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or thalassemia, or severe GI bleeding\n* Pregnant or breastfeeding\n\nNuvisertib (TP-3654) + Momelotinib Arm:\n\n* Received previous systemic antineoplastic therapy or any experimental therapy within 2 weeks or 5 half-lives, whichever is longer, prior to Cycle 1 Day 1 (Notes: Prior treatment with momelotinib or nuvisertib is not allowed; in patients with ongoing JAK inhibitor therapy, ie, ruxolitinib, at screening, JAK inhibitor therapy must be tapered over a period of at least 1 week. Patients on a low dose of ruxolitinib (eg, 5 mg QD) may have a reduced taper period or no taper; hydroxyurea or anagrelide are allowed up to 24 hours prior to Cycle 1 Day 1).\n* Received systemic steroid therapy (\\>10 mg daily prednisone or equivalent) within 1 week prior to Cycle 1 Day 1 (Note: Topical, inhaled, nasal, and ophthalmic steroids are not prohibited).\n* Known allergic reactions or sensitivity to nuvisertib, momelotinib, or any structurally similar drug, or to any component of the formulations of either study intervention\n* Splenic irradiation within 6 months prior to screening or prior splenectomy\n* Prior allogenic stem cell transplant within the last 6 months (Note: Patients who have relapsed after 6 months post-transplant and do not have active GVHD are eligible).\n* Eligible for allogeneic bone marrow or stem cell transplantation (Note: Patients who are not willing to undergo transplantation or for whom a suitable donor is not available are considered as transplant ineligible).\n* Major surgery within 4 weeks prior to Cycle 1 Day 1 and\u002For have not recovered adequately from surgery prior to first dose.\n* Active, uncontrolled bacterial, viral, or fungal infections, requiring parenteral antimicrobial within 1 week prior to Cycle 1 Day 1\n* Chronic active or acute viral hepatitis A, B, or C infection (testing for hepatitis B and C are required)\n* Known history of chronic liver disease (eg, portal hypertension or any of its complications, cirrhosis, Child-Pugh C, auto-immune hepatitis, alpha-1 anti-trypsin deficiency, Wilson's disease, etc) (Note: Abnormal liver morphology at baseline imaging may require additional testing, as needed)\n* Unresolved Grade ≥ 2 non-hematological adverse events related to prior treatment (stable Grade 2 conditions may be permitted in consultation with the Sponsor)\n* Presence of Grade ≥ 2 peripheral neuropathy\n* History of myocardial infarction or symptomatic congestive heart failure or uncontrolled arrhythmia within 6 months prior to Cycle 1 Day 1; LVEF \\\u003C 45% by echocardiogram within 4 weeks prior to Cycle 1 Day 1\n* Corrected QTcF of \\> 480 msec\n* Prior or concurrent malignancy that could interfere with the safety or efficacy assessment of the study intervention\n* History of a medical condition or GI tract surgery that could impair absorption or could result in short bowel syndrome with diarrhea\n* Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or thalassemia, or severe GI bleeding\n* Pregnant or breastfeeding","ALL","18 Years",{"count":19,"type":20},240,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","This study is a Phase 1\u002F2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics and pharmacodynamics of nuvisertib (TP-3654) in patients with intermediate or high-risk primary or secondary MF.",[27],"Myelofibrosis","RECRUITING","2026-04-30",{"date":31,"type":32},"2026-05-01","ACTUAL",{"date":34,"type":32},"2019-12-16",{"date":36,"type":20},"2030-04-30",{"name":38,"class":39},"Sumitomo Pharma America, Inc.","INDUSTRY",84,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":48,"targetDuration":4,"studyType":21,"phases":50,"briefSummary":51,"conditions":52,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":54,"lastUpdatePostDateStruct":55,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":62},"100584633","phase-1-a-study-to-investigate-the-safety-of-dsp-3077-after-a-unilateral-eye-injection-in-male-and-female-participants-18-years-of-age-or-older-with-retinitis-pigmentosa-100584633","NCT06891885","A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa","A Prospective, Open-label, Single-arm, Dose-escalation Study of the Safety and Tolerability of a Single Subretinal Uniocular Injection of Allogeneic Induced Pluripotent Stem Cell (iPSC)-Derived Retinal Sheets (DSP-3077) in Adults With Retinitis Pigmentosa (RP)","Inclusion Criteria:\n\n* Participant is \\>= 18 years of age at the time of signing the informed consent.\n* Participant has a clinical diagnosis of nonsyndromic retinitis pigmentosa.\n* Participant is willing to consent to genetic testing, if not already done.\n* Cohorts 1 and 2: Participant will have BCVA in the study eye between hand motion and 20 ETDRS letter score (approximately \\\u003C= 20\u002F400 Snellen equivalent), inclusive at Screening and Baseline.\n* Cohort 3: Participant will have BCVA in the study eye between 20 ETDRS letter score (approximately \\>= 20\u002F400 Snellen equivalent) and 35 ETDRS letter score (approximately \\\u003C= 20\u002F200 Snellen equivalent), inclusive at Screening.\n* Participant is in good physical health, based on medical history, physical examination, vital signs, electrocardiogram (ECG), and clinical laboratory tests at Screening.\n\nExclusion Criteria:\n\n* Participant has an eye disease or visual disorder other than RP that impairs visual function (eg, retinal vascular disease, glaucoma).\n* Participant has any other eye condition (eg, ocular media opacity, nystagmus), which in the opinion of the investigator, would preclude an accurate evaluation at any time during the study and\u002For make surgical delivery more challenging.\n* Participant has any clinically significant unstable medical condition or any clinically significant chronic disease that in the opinion of the investigator, would limit the participant's ability to complete and\u002For participate in the study.\n* Female participant who is pregnant or lactating or planning to become pregnant.\n* Participant has received treatment with any nonapproved, experimental, or investigational therapy in either eye (systemic, topical, intravitreal) and\u002For received treatment in an interventional clinical trial for an eye disease or disorder within 90 days or 5 half-lives, whichever is longer, prior to Screening.\n* Participant has previously received cell therapy, gene augmentation therapy, genome editing therapy, or any subretinal administered therapy for an eye disease or disorder.",{"count":49,"type":20},12,[23,24],"The Goal of this study is to evaluate the safety, tolerability, and clinical responses following single dose of DSP-3077. Study enrolls both male and female patients in 3 cohorts with each cohort defined by visual acuity (VA) criteria and dose level of DSP-3077. Each cohort will include 4 participants.",[53],"Retinitis Pigmentosa","2026-04-14",{"date":56,"type":32},"2026-04-17",{"date":58,"type":20},"2026-04-16",{"date":60,"type":20},"2032-10-31",{"name":38,"class":39},2,{"id":64,"slug":65,"hasResults":11,"nctId":66,"briefTitle":67,"officialTitle":68,"acronym":4,"eligibilityCriteria":69,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":70,"targetDuration":4,"studyType":21,"phases":72,"briefSummary":73,"conditions":74,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":76,"lastUpdatePostDateStruct":77,"startDateStruct":79,"completionDateStruct":81,"leadSponsor":83,"locationsCount":62},"100627102","phase-1-how-14c-dsp-5336-is-absorbed-broken-down-and-removed-from-the-body-after-a-single-oral-dose-in-patients-with-advanced-blood-cancers-100627102","NCT07444268","How [14C]-DSP-5336 is Absorbed, Broken Down, and Removed From the Body After a Single Oral Dose in Patients With Advanced Blood Cancers","A Phase 1, Open-label Study of the Absorption, Metabolism, and Excretion of DSP-5336 Following a Single Oral Dose in Patients With Refractory or Relapsed Acute Leukemia, Myelodysplastic Syndrome, Multiple Myeloma, or Myeloproliferative Neoplasms","Inclusion Criteria:\n\n* Male or female, of any race, ≥ 18 years of age. Female patients must be surgically sterile or postmenopausal. Male patients must be permanently sterile or agree to use contraception.\n* Have an advanced hematological malignancy that is relapsed, refractory, or has progressed following receipt of standard and available treatments.\n* Any prior pre-treatment toxicities resolved to ≤Grade 1 prior to enrolment, with exception of ≤Grade 2 alopecia or neuropathy.\n* Adequate kidney and liver function\n* ECOG performance status of ≤ 2.\n* Able to attend the required study visits, including the confinement period for monitoring and collection of bowel movements and micturition.\n* Able to comprehend and are willing to sign the ICF and abide by the study restrictions.\n\nExclusion Criteria:\n\n* Histologic diagnosis of acute promyelocytic leukemia.\n* Abnormal ECG that is clinically significant, such as QTcF \\> 480 msec. QT interval correction can be performed in the case of bundle branch block.\n* History of torsades de pointes.\n* Left ventricular ejection fraction ≤ 45%, as determined by echocardiogram.\n* Have any concurrent conditions that could pose an undue risk or interfere with interpretation of the study results, including, but not limited to clinically significant non-healing or healing wounds, concurrent congestive heart failure, unstable angina, cardiac arrhythmia requiring treatment (excluding asymptomatic atrial fibrillation), myocardial infarction within 6 months, acute coronary syndrome within 6 months, significant pulmonary disease (shortness of breath at rest or on mild exertion; eg, due to concurrent severe obstructive pulmonary disease, hypertension not controlled with concomitant medication, or diabetes mellitus with \\> 2 episodes of ketoacidosis in the prior 6 months).\n* History of stomach or intestinal surgery or resection that would potentially alter absorption and\u002For excretion of orally administered drugs (uncomplicated appendectomy and hernia repair are allowed).\n* History or evidence of severe dysphagia, short-gut syndrome, gastroparesis, gastrointestinal tract disease, malabsorption syndrome, the requirement for intravenous alimentation, gastric\u002Fjejunal feeds, any uncontrolled gastrointestinal disease, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally, including the inability to swallow oral medication\n* Have cognitive, psychological, or psychosocial impediment that would impair their ability to receive therapy according to the protocol or would adversely affect their ability to comply with the informed consent process, protocol, or protocol-required visits and procedures.\n* History of bowel obstruction, abdominal fistula, gastrointestinal perforation, or intra-abdominal abscess within 6 months of check-in, unless approved by the investigator and medical monitor.\n* Active and uncontrolled bacterial, viral, or fungal infection requiring parenteral therapy.\n* Positive hepatitis panel and\u002For positive human immunodeficiency virus test indicative of active infection. Patients whose results are compatible with prior immunization may be included.\n* Undergone HSCT, chimeric antigen receptor cell therapy, or other modified T-cell therapy within 60 days prior to dosing.\n* Received donor lymphocyte infusion within 28 days prior to dosing, receiving immunosuppressive therapy post-HSCT, or have clinically active GVHD or GVHD requiring active medical intervention other than the use of topical steroids for ongoing cutaneous GVHD.\n* Received systemic calcineurin inhibitors within 2 weeks prior to dosing.\n* Received other anticancer drugs or other investigational treatment within 14 days or 5 half-lives, whichever is shorter, prior to dosing.\n* Major surgery within 28 days prior to dosing.\n* Any known intolerance or hypersensitivity to components of the study intervention.\n* Patients who have previously been dosed in \\> 2 radiolabeled drug studies in the last 12 months. For patients who have previously been dosed in ≤ 2 radiolabeled drug studies within the last 12 months, the previous radiolabeled dose must be at least 4 months prior to check-in to the study site where exposures are known to the investigator or 6 months prior to check-in to the study site for a radiolabeled drug study where exposures are not known to the investigator.\n* Poor peripheral venous access.\n* Patients with exposure to significant diagnostic or therapeutic radiation or current employment in a job requiring radiation exposure monitoring within 12 months prior to check-in.\n* Patients who, in the opinion of the investigator or designee, should not participate in this study.",{"count":71,"type":20},8,[23],"The purpose of this study is to evaluate the absorption, metabolism, and excretion of DSP-5336 following a single oral administration of the study drug in patients with hematologic malignancies whose disease has progressed after available standard therapies.",[75],"Advanced Hematologic Malignancies","2026-04-07",{"date":78,"type":32},"2026-04-08",{"date":80,"type":32},"2026-03-26",{"date":82,"type":20},"2026-08",{"name":38,"class":39},{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":89,"acronym":4,"eligibilityCriteria":90,"healthyVolunteers":11,"sex":16,"minAge":91,"maxAge":4,"enrollmentInfo":92,"targetDuration":4,"studyType":21,"phases":94,"briefSummary":95,"conditions":96,"keywords":101,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":117},"100438382","phase-1-a-phase-12-study-of-enzomenib-dsp-5336-in-patients-with-acute-leukemia-horizen-1-100438382","NCT04988555","A Phase 1\u002F2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)","A Phase 1\u002F2, Open-Label, Dose-Escalation, Dose-Expansion Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia and Other Selected Hematologic Malignancies, With and Without Mixed Lineage Leukemia (MLL) Rearrangement or Nucleophosmin 1 (NPM1) Mutation (Horizen-1)","Inclusion Criteria:\n\nFor patients in Phase I:\n\n1. Have a diagnosis of relapsed or refractory AML, ALL or acute leukemia of ambiguous lineage according to World Health Organization (WHO) 2022 classification, or, in selected sites and regions, a diagnosis of MDS or MM as determined by pathology review at the treating institution, and whose disease has progressed after available standard therapies known to be active for their AML, ALL, or acute leukemia of ambiguous lineage or, in selected sites and regions, for MM or MDS. If acute leukemia patients are transformation from MDS or other hematologic malignancies, patients need to receive available standard therapies as acute leukemia after AML transformation and before enrolling this trial. In regions or countries where required by regulatory authorities, participants must have a documented KMT2A (MLL) fusion or NPM1 mutation, including those with coexisting FLT3 genomic alterations and\u002For IDH1\u002F2 mutation. Participants who are candidates for stem cell transplantation must have been offered this therapeutic option.\n\n   For patients with MDS (selected sites and regions):\n   1. Patients with MDS must have bone marrow blasts ≥ 5%\n   2. Patients with MDS must have relapsed or refractory disease and have exhausted available standard therapies including at least 2 cycles of treatment with HMA\n\n      For patients with MM (selected sites and regions):\n   3. Have a confirmed diagnosis of multiple myeloma according to International Myeloma Working Group (IMWG) 2016 classification (Kumar, 2016) and whose disease has progressed after treatment with a minimum of 3 prior anti-myeloma regimens including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 monoclonal antibody (mAb); patients must not be candidates for available therapies with established clinical benefit\n   4. Have measurable disease as defined in the protocol\n   5. Meet the laboratory parameters set in the protocol\n\n      For patients with relapsed\u002Frefractory AML in the venetoclax and azacitidine combination cohort (in countries and sites where permitted):\n   6. Have MLLr or NPM1m.\n\n      For patients with relapsed\u002Frefractory AML in the gilteritinib combination cohort (in countries and sites where permitted):\n   7. Have MLLr or NPM1m AND any of the following FLT3 mutations: FLT3-ITD, FLT3-TKD\u002FD835 or FLT3-TKD\u002FI836.\n\n      For patients with relapsed\u002Frefractory AML with NPM1 enrolled in the RP2D confirmation cohort:\n   8. Must have ≥5% blasts in bone marrow by morphologic assessment\n   9. Must not have received prior treatment with a menin inhibitor\n\n      For patients with newly diagnosed AML:\n   10. Must have AML as defined by WHO 2022 criteria with a documented MLLr or NPM1m (patients with AML characterized by MLL partial tandem duplications, MLL deletions, or trisomy 11 are not eligible)\n   11. Must not have received treatment for AML with the exception of hydroxyurea for control of white blood cell counts.\n\n   For patients in Phase 2:\n2. Have a confirmed diagnosis of relapsed AML or ALL according to WHO 2022 classification, as determined by pathology review at the treating institution, and who have ≥5% blasts by morphologic assessment in the bone marrow. Patients with extramedullary disease or peripheral blasts as the only manifestation of relapse are not eligible. Patients must have received clinically applicable standard therapies with confirmed survival benefit. Patients must not have had prior exposure to a menin inhibitor.\n3. Have a documented KMT2A (MLL)-fusion assessed at relapse or immediately prior to the determination of refractory status. KMT2A genetic alterations other than fusions (eg, KMT2A-PTD, amplification, point mutation) are not permitted.\n\n   For all patients:\n4. Be \\> 18 years of age. For countries and sites where approved, for DSP-5336 monotherapy, acute leukemia patients ≥12 years of age who weigh ≥40 kg may be enrolled.\n5. Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤2.\n6. For monotherapy, WBC below 30,000\u002Fμ at enrollment. For the combination arms, WBC count must be below 25,000\u002FuL at enrollment and prior to starting treatment. (Hydroxyurea and steroids for cytoreduction purposes are allowed prior to enrollment and during study treatment)\n7. Clearance of creatinine level ≥ 50 ml\u002Fmin, assessed by the CPK-EPI formula (2021 version and Cystatin C not required)\n8. Total bilirubin ≤1.5 the upper limit of normal (ULN) (or ≤2.0 ULN for patients with known Gilbert's syndrome)\n9. Aspartate aminotransferase (AST) ≤3.0 times ULN\n10. Alanine aminotransferase (ALT) ≤3.0 times ULN\n11. Any prior treatment-related toxicities resolved to Grade ≤1 prior to enrollment, with the exception of Grade ≤2 alopecia or neuropathy\n12. Be willing to attend study visits as required by the protocol\n13. Have an estimated life expectancy ≥3 months, based on the investigator's assessment\n14. Females of childbearing potential must have a negative serum pregnancy test. Females of childbearing potential are defined as women who have (1) experienced menarche and have not undergone sterilization procedures (hysterectomy, or bilateral oophorectomy), or have (2) not experienced menopause as defined in the protocol.\n15. All men and all women of childbearing potential and male patients' partners who are women of childbearing potential are required to use a highly effective method of contraception during the study and for 6 months (for females and males alike) after the last dose of study drug. Further guidelines noted in protocol.\n16. Have AML\u002FALL\u002FMDS\u002FMM bone marrow material suitable for genomic analysis of AML,ALL, MDS, or MM genetic alterations. Note: If a bone marrow material is insufficient, an alternative suitable tissue (ex: peripheral blood) must be provided.\n\nExclusion Criteria:\n\n1. Has a left ventricular ejection fraction (LVEF) \\\u003C50%, as determined by ECHO\n2. Histological diagnosis of acute promyelocytic leukemia\n3. Received systemic calcineurin inhibitors within 2 weeks prior to the first dose of DSP 5336\n4. Have abnormal ECGs at screening that are clinically significant, such as (QTc \\>480 msec, with QTc corrected according to Fridericia's formula (QTcF). For clinical sites in the UK, have abnormal ECGs at screening that are clinically significant, such as QTc ≥470 msec and ≥450 msec with QTc corrected according to Fridericia's formula (QTcF), for females and males, respectively. In addition, patients with a history of prolonged QT syndrome or who are required to take therapies associated with QT-interval prolongation are excluded.\n\n   Note: In case of bundle branch block, QT interval correction can be performed.\n5. Has an active and uncontrolled, bacterial, viral, or fungal infection requiring parenteral therapy. Note: Patients must be afebrile with negative blood cultures at least 72 hours prior to Cycle 1 Day 1.\n6. Receives concurrent sensitive substrates with a narrow safety window or strong inhibitors or inducers of CYP3A4\u002F5, including specifically: ketoconazole, isavuconazole and itraconazole. Other antifungals that are used as standard of care to prevent or treat infections are permitted. If a patient is on one of the excluded azole class antifungals, he\u002Fshe can be taken off or switched to a permitted azole 7 or more days prior to first dose, then the patient could be allowed on study (Arm B) with approval of the medical monitor.\n7. Had major surgery within 28 days prior to the first dose of DSP-5336\n8. Has active central nervous system leukemia (prophylactic intrathecal chemotherapy is allowed).\n9. Underwent HSCT or chimeric antigen receptor cell (CAR-T) therapy or other modified T-cell therapy within 60 days prior to the first dose of DSP-5336. For clinical sites in the UK, underwent CAR-T therapy or other modified T-cell therapy within 6 months prior to the first dose of DSP-5336.\n10. Received a donor lymphocyte infusion within 28 days prior to the first dose of DSP-5336, or receiving immunosuppressive therapy post-HSCT at the time of screening, or with clinically active GVHD or GVHD requiring active medical intervention other than the use of topical steroids for ongoing cutaneous GVHD\n11. Received antineoplastic agents (except hormonal therapies as adjuvant maintenance for breast or prostate cancers if a patient is taking before starting study treatment, and hydroxyurea given for controlling blast cells) or other investigational treatment within 7 days or 5 half-lives, whichever is shortest, prior to the first dose of DSP-5336\n12. In the opinion of the treating investigator, have any concurrent conditions that could pose an undue medical hazard or interfere with interpretation of study results; these conditions include, but are not limited to: clinically significant non-healing or healing wounds; concurrent congestive heart failure (New York Heart Association Functional Classification Class III or IV; see Section 21.2); concurrent unstable angina; concurrent cardiac arrhythmia requiring treatment (excluding asymptomatic atrial fibrillation); recent (within the prior 6 months) myocardial infarction; acute coronary syndrome within the previous 6 months; significant pulmonary disease (shortness of breath at rest or on mild exertion), eg, due to concurrent severe obstructive pulmonary disease, concurrent hypertension not controlled with concomitant medication, or diabetes mellitus with more than 2 episodes of ketoacidosis in the prior 6 months\n13. Have a known detectable viral load for human immunodeficiency virus or hepatitis C, or evidence of hepatitis B surface antigen, all being indicative of active infection.\n\n    For sites in Japan, Taiwan, and Korea only: Hepatitis B core (HBc) antibody or hepatitis B surface (HBs) antibody test should be performed if HBsAg is negative. If HBc antibody or HBs antibody test is positive, HBV DNA quantification test should be performed to confirm that HBV DNA is negative.\n14. Have severe dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally, including the inability to swallow oral medication\n15. Have cognitive, psychological, or psychosocial impediment that would impair the ability of the patient to receive therapy according to the protocol, or adversely affect the ability of the patient to comply with the informed consent process, protocol, or protocol-required visits and procedures\n16. Are pregnant or breastfeeding or planning to become pregnant. Note: Patients who are breastfeeding may be enrolled if they interrupt breastfeeding prior to the first dose of any study drugs and do not feed the baby with breast milk expressed after receiving the first dose of any study drugs. Breastfeeding should not be resumed for at least 6 months after the last dose of study drug\n17. Have any history or complication of interstitial lung disease (for sites in Japan in Phase 1 dose escalation).\n\n    For clinical sites in the EU, have a history of Grade ≥ 2 drug-induced interstitial lung disease or Grade ≥ 2 non-infectious pneumonitis within 6 months of starting study treatment.\n18. Have a history of Torsades de Pointes\n19. Received systemic calcineurin inhibitors within 4 weeks prior to the first dose of DSP-5336\n20. Have plasma cell leukemia (\\>2.0 x 109 \u002FL plasma cells in blood by standard differential) (for patients with MM)\n21. For patients intending to enroll into the combination cohort with gilteritinib: Patients must be gilteritinib-naïve or sensitive and have not received a FLT3 inhibitor in the relapsed refractory setting (prior FLT3 inhibitor in front line therapy is allowed)\n22. Have a known intolerance of hypersensitivity reaction to components of the investigational medicinal product\n23. For clinical sites in the UK: In Arm E (DSP-5336 + venetoclax\u002Fazacitidine), have received a live vaccine within 30 days prior to the first dose of DSP-5336","12 Years",{"count":93,"type":20},606,[23,24],"A phase 1\u002F2 dose escalation \u002F dose expansion study of Enzomenib (DSP-5336) in patients with acute leukemia.",[97,98,99,100],"Leukemia, Myeloid, Acute","Leukemia, Lymphocytic, Acute","Multiple Myeloma","Myelodysplastic Syndromes",[102,103,104,105,106,107,108],"Relapsed or refractory AML","MLLr","Menin","NPM1m","KMT2A","MDS","MM","2026-03-20",{"date":111,"type":32},"2026-03-24",{"date":113,"type":32},"2022-02-28",{"date":115,"type":20},"2027-12-31",{"name":38,"class":39},104,{"id":119,"slug":120,"hasResults":11,"nctId":121,"briefTitle":122,"officialTitle":123,"acronym":4,"eligibilityCriteria":124,"healthyVolunteers":11,"sex":16,"minAge":125,"maxAge":126,"enrollmentInfo":127,"targetDuration":4,"studyType":21,"phases":129,"briefSummary":130,"conditions":131,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":62},"100573980","phase-1-a-multicenter-study-to-evaluate-safety-tolerability-and-clinical-responses-of-dsp-1083-into-subjects-with-parkinsons-disease-100573980","NCT06753331","A Multicenter Study to Evaluate Safety, Tolerability, and Clinical Responses of DSP-1083 Into Subjects With Parkinson's Disease","A Multicenter, Sham-controlled, Randomized Study to Evaluate the Safety, Tolerability, and Clinical Responses Following Stereotactic Intracranial Implantation of DSP-1083 Into Subjects With Parkinson's Disease","Inclusion Criteria:\n\n1. Men or women aged ≥ 40 and ≤ 72 years at the time of informed consent with a clinically established diagnosis of Parkinson's disease in accordance with the Movement Disorder Society (MDS) Clinical Diagnostic Criteria for Parkinson's Disease.\n2. Subject has a clinically established diagnosis of PD for ≥ 4 years.\n3. Subject has suboptimal control of PD symptoms, with optimized oral antiparkinsonian medication regimen including levodopa\u002Fcarbidopa monotherapy or levodopa\u002Fcarbidopa plus antiparkinsonian medications, with stable dosing for ≥ 2months prior to screening.\n4. Subject has a L-DOPA response of ≥ 30% without the influence of antiparkinsonian medications at Screening.\n5. Subject has a Modified Hoehn and Yahr stage 3 - 4 in the Off state.\n6. Subject has a pretreatment 18F-DOPA PET scan consistent with PD.\n7. Subject has both On and Off states as demonstrated by the MDS-UPDRS Part III\u002FIV and the Hauser patient daily diary.\n8. Subjects must meet the following race criteria: 2 of the up to 5 sentinel subjects will be of Asian race, defined as having at least 2 grandparents who are Japanese, Taiwanese, Korean, or Chinese. Subjects in Cohort 2 can be of any race.\n9. Subject is approved by the Enrollment Authorization Eligibility Committee following review of all required information collected during Screening.\n\nExclusion Criteria:\n\n1. Subject has atypical parkinsonian syndrome (eg, progressive supranuclear palsy \\[PSP\\], multiple system atrophy \\[MSA\\], dementia with Lewy bodies \\[DLB\\], corticobasal degeneration, Parkinson-plus syndrome, vascular parkinsonism, secondary parkinsonism, hereditary parkinsonism).\n2. Subject has non-PD neurological symptoms or evidence of non-PD brain disease (eg, tumor, inflammation, active or history of vascular disorder, history of cerebral hemorrhage, Alzheimer's disease, or other neurodegenerative disorder) based on neuroimaging and\u002For medical history that would preclude study participation.\n3. Subject has psychiatric symptoms, cognitive impairment, depression, dementia, or other behavioral disorder that would preclude study participation based on Investigator decision.\n4. Subject has received previous striatal or other extrapyramidal system PD treatments, including deep-brain stimulation, central nervous system (CNS) ablation (eg, pallidotomy, thalamotomy), implanted cell, or gene therapy, and\u002For focused ultrasound therapy.\n5. Subject has peak-dose dyskinesia of sufficient severity that precludes study participation, defined as any item score of ≥ 3 (moderate dyskinesia) on the UDysRS Part 1B (Patient Dyskinesia Questionnaire) AND\u002FOR any item score of ≥ 2 (moderate dyskinesia) on Part 3 (Objective Evaluation of Dyskinesia Disability) Intensity Scale: Impairment. Subject has another type (eg, diphasic dyskinesia) or an unusual pattern of dyskinesia.\n6. Subject has a history of, or concurrent abnormal immune function that may adversely affect the engraftment of the cell implants and use of adjunctive immunosuppressants.\n7. The subject has the following clinical laboratory test results at Screening:\n\n   * Neutrophil count \\\u003C 2,000\u002FμL.\n   * Platelet count \\\u003C 5.0 × 104\u002FμL.\n   * Aspartate aminotransferase (AST), alanine aminotransferase (ALT) \\> 3.0 × upper limit of normal.\n   * Total bilirubin \\> 1.5 × upper limit of normal.\n   * Persistent estimated glomerular filtration rate (eGFR) \\\u003C 60 mL\u002Fmin\u002F1.73 m2.\n   * Poorly controlled blood glucose in diabetic subjects (glycosylated hemoglobin \\> 9.0%, or fasting serum glucose ≥ 200mg\u002FdL).\n8. Subject has any disorder that would contraindicate general anesthesia, conscious sedation or stereotactic surgery.\n9. Subject has any clinically significant unstable medical condition or any clinically significant chronic disease that would pose a risk to the subject or that might confound the results of the study. In cases in which the impact of the condition upon risk to subject or study results is unclear, the Medical Monitor should be consulted.","40 Years","72 Years",{"count":128,"type":20},25,[23,24],"The Goal of this study is to evaluate the safety, tolerability, and clinical responses following implantation of DSP-1083. Study enrolls both male and female patients in 2 cohorts. This study will be held in approximately 5-8 study sites in United States",[132],"Parkinson's Disease","2026-02-26",{"date":135,"type":32},"2026-03-02",{"date":137,"type":32},"2024-12-18",{"date":139,"type":20},"2030-12-15",{"name":38,"class":39},{"id":142,"slug":143,"hasResults":11,"nctId":144,"briefTitle":145,"officialTitle":146,"acronym":4,"eligibilityCriteria":147,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":148,"targetDuration":4,"studyType":21,"phases":150,"briefSummary":151,"conditions":152,"keywords":154,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":163,"lastUpdatePostDateStruct":164,"startDateStruct":166,"completionDateStruct":168,"leadSponsor":170,"locationsCount":171},"100556570","phase-1-smp-3124lp-in-adults-with-advanced-solid-tumors-100556570","NCT06526819","SMP-3124LP in Adults With Advanced Solid Tumors","An Open-label, Phase 1 Dose Escalation and Phase 2 Dose Expansion Study to Assess Safety, Tolerability, Pharmacokinetics, and Preliminary Antitumor Activity of SMP-3124LP in Adults With Advanced Solid Tumors","Inclusion Criteria:\n\n\\- Histologically or cytologically-confirmed cancer that is advanced, recurrent, or metastatic with the following origins, and whose disease progressed on standard therapy and for whom there are no alternative therapies that may confer overall survival benefit.\n\nFor patients in the Dose Escalation part:\n\n1. Platinum-resistant ovarian cancer\n\n   * Histologically diagnosed ovarian, fallopian tube, or primary peritoneal cancer, with predominantly high-grade (Grade 2 or 3) epithelial features (serous and clear cell)\n   * Platinum resistant is defined as relapsed within 6 months after the last dose of platinum-based therapy\n2. Triple negative breast cancer - ER- and PR-negative with HER2 negative\n\n   * HER2 negative is defined as one of the following: 0 or 1+ by IHC, or if IHC 2+, then in situ hybridization is negative per the ASCO-CAP HER2 guidelines\n   * ER- and PR-negative is defined as \\\u003C 10% of cells expressing hormonal receptors by IHC, as per standard guidelines\n3. Squamous cell carcinoma of the anus\n\n   \\- Patient with locally advanced ineligible for surgery is allowed.\n4. Squamous cell carcinoma of the head and neck\n5. Non-small cell lung cancer (NSCLC: adenocarcinoma, large cell, and squamous cell carcinoma)\n6. Uterine serous cancer (recurrent or persistent)\n\n   For Patients in the Dose Expansion Part:\n7. Cohort A: PROC (same as above)\n8. Cohort B: TNBC (same as above)\n9. Cohort C: SCCA (same as above)\n\n   * ECOG performance \\\u003C\u002F= 2 at screening\n   * Recovered from any prior treatment related toxicities\n   * Adequate organ function as evidenced by:\n\na. Hemoglobin \\>\u002F= 9 g\u002FdL (transfusion or use of erythropoietin to obtain this are not permitted) b. Absolute neutrophil count \\>\u002F= 1500 uL (platelet transfusion not allowed to achieve this) c. Platelet count \\>\u002F= 100 x 10 (platelet transfusion not alled to achieve this) d. Bilirubin \\\u003C\u002F= 1.5 x ULN (or \\\u003C\u002F= 3.0 x if ULN if Gilbert's syndrome) e. AST and ALT \\\u003C\u002F= 3.0 x ULN (or \\\u003C\u002F= 5 x ULN if the liver has tumor involvement f. Calculated creatinine clearance \\>\u002F= 60 mL\u002Fmin using Cockcroft-Gault formula\n\n* Patient is non-fertile or agrees to use adequate methods of contraception or agrees to refrain completely from heterosexual intercourse during the study and for 6 months (for female and male patients alike) after the last dose of study intervention.\n* May be HIV positive if the following conditions are met:\n\n  1. CD4 + T-cell count \\>\u002F= 350 cells\u002FuL\n  2. HIV viral load \\\u003C 400 copies\u002Fml prior to enrollment\n  3. No history of acquired immunodefficiency syndrome (AIDS) defining opportunistic infections\n* Known hepatitis B infection mush have negative serum HbsAg. Patients with known hepatitis C virus infection must have a viral load below the limit of quantification Japan sites only: HBc antibody or HBsantibody tests should be performed if HBsAg is negative. If HBc antibody or HBs antibody tests are positive, HBV DNA quantitative tests should be performed to confirm that HBV DNA is negative.\n\nExclusion Criteria:\n\n* Patient has received prior treatment at any time with a cell cycle checkpoint inhibitor (eg, CHK1 and\u002For CHK2, WEE1, or ATR inhibition)\n* Patient has a known allergy or sensitivity to any component of SMP-3124LP, including the inactive ingredients\n* Patient has received treatment with systemic anticancer therapy, radiotherapy, or investigational therapy within 14 days prior to Study Cycle 1 Day 1. (Palliative radiotherapy with a limited field of radiation within 2 weeks will be permitted.)\n* Patient has undergone a major surgical procedure ≤ 28 days, or minor surgical procedure ≤ 7 days, prior to Cycle 1 Day 1\n* Patient has used strong CYP1A2 or 2D6 inhibitors within 14 days or 5 half-lives, whichever occurs first, prior to Cycle 1 Day 1 (examples of restricted CYP1A2 and CYP2D6, P-gp, and\u002For BCRP inducers, inhibitors, or substrates are presented in Table 16)\n* Patient has central nervous system metastasis or leptomeningeal disease\n* Prior or concurrent malignancy whose natural history or treatment would have a significant potential to interfere with the safety or efficacy assessments of the investigational regimen\n* Patient has an abnormal ECG that is clinically significant, including a corrected QT interval (corrected using Fridericia's correction formula \\[QTcF\\]) \\> 470 msec; and\u002For a history of Torsade de Pointes\n* Patient has a left ventricular ejection fraction \\\u003C 45% by echocardiogram (ECHO)\n* Patient has clinically significant cardiac disease including heart failure (eg, New York Heart Association, Class III or IV)\n* Patient has an active, uncontrolled, bacterial, viral, or fungal infection requiring parenteral antimicrobial within 1 weeks prior to Cycle 1 Day 1\n* Patient is pregnant (as evidenced by a positive serum or urine pregnancy test) or is breastfeeding. Female breastfeeding patients may be enrolled if they interrupt breastfeeding. Breastfeeding should not be resumed for at least 6 months after the last dose of study drug.\n\nFor sites in Japan only: In addition to the above, any patient deemed likely to be pregnant based on medical interview will be excluded from the study.\n\n* Patient with ovarian cancer\n\n  1. Has a history of bowel obstruction related to their underlying disease within 3 months prior to Study Day 1\n  2. Has platinum-refractory disease. Platinum refractory is defined as progression during platinum-based chemotherapy\n* Patient has any other medical or psychiatric condition that, in the opinion of the investigator, might interfere with their participation in the trial or interfere with the interpretation of trial results\n* Patient is taking a prohibited medication at baseline.",{"count":149,"type":20},120,[23,24],"An Open-label, Phase I Dose Escalation and Phase 2 Dose Expansion Study to Assess Safety, Tolerability, Preliminary Antitumor Activity of SMP 3124LP in Adults with Advanced Solid Tumors",[153],"Solid Tumor",[155,156,157,158,159,160,161,162],"first in human","open label","platinum-resistant ovarian cancer (PROC)","Triple Negative Breast Cancer (TNBC)","Metastatic squamous cell carcinoma of the anus (MSCCA)","Squamous cell carcinoma of the head and neck (SCCHN)","non-small cell lung cancer (NSCLC)","uterine serous cancer","2026-02-25",{"date":165,"type":32},"2026-02-27",{"date":167,"type":32},"2024-08-14",{"date":169,"type":20},"2029-05",{"name":38,"class":39},10,""]