[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Jazz Pharmaceuticals\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":505},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,17,0,[8,47,76,102,127,161,193,217,247,273,298,335,360,385,415,459,484],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100600814","phase-2-a-phase-2-neoadjuvant-study-of-zanidatamab-in-combination-with-chemotherapy-in-participants-with-her2-positive-breast-cancer-100600814",false,"NCT07102381","A Phase 2 Neoadjuvant Study of Zanidatamab in Combination With Chemotherapy in Participants With HER2-positive Breast Cancer","A Phase 2, Randomized, Multicenter, Open-label Neoadjuvant Study Evaluating Zanidatamab in Combination With Chemotherapy in Participants With HER2-positive Breast Cancer","EmpowHER 208","Inclusion Criteria:\n\n1. Has newly diagnosed Stage II or III histologically confirmed invasive breast carcinoma.\n2. Has histologically confirmed HER2-positive breast cancer\n3. Has a known hormone receptor (HR) status of the primary tumor\n4. Participants with multifocal or multicentric disease are eligible if the largest tumor (which must be larger than or equal to 2 cm in diameter) is HER2-positive, and the treating physician has determined the participant should be treated as HER2-positive.\n5. Agrees to undergo a mastectomy or breast conserving surgery (BCS) after neoadjuvant therapy.\n6. Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.\n7. Adequate organ function\n8. Has an LVEF ≥ 50% as determined by either ECHO or MUGA obtained within 6 weeks prior to randomization.\n9. Adequate contraceptive precautions\n10. Participants with known HIV are eligible unless:\n\n    1. CD4+ T Cell count is less than or equal to 350 microliters (uL)\n    2. Detectable viral load, or\n    3. Receiving protease inhibitors or cobicistat\n\nExclusion Criteria:\n\n1. Has Stage IV (metastatic) breast cancer.\n2. Has bilateral breast cancer.\n3. Has a history of any severe and\u002For uncontrolled medical conditions or other conditions that, in the opinion of the investigator, could affect the participant's involvement in the study.\n4. Has uncontrolled hypertension\n5. Has significant symptoms from peripheral neuropathy\n6. Has an active uncontrolled infection\n7. Has a history of life-threatening hypersensitivity to monoclonal antibodies or to recombinant proteins or excipients in the drug formulation of zanidatamab or other study interventions.\n8. Known active hepatitis B or C infection.\n9. Has another malignancy diagnosed within the last 5 years. Exceptions include previously treated non melanomatous skin cancers, carcinoma in-situ, and melanoma in-situ. Participants with prior ipsilateral ductal carcinoma in situ (DCIS) or invasive breast cancer are not eligible.\n10. Was treated with surgery, chemotherapy, anti-HER2 therapy, radiation therapy, endocrine therapy, or experimental therapy for invasive breast cancer\n11. Is planning to receive concurrent therapy with any other investigational agent or anti-cancer therapy not specified in the protocol\n12. Receipt of a live vaccine within 4 weeks prior to enrollment\n13. Has a known hypersensitivity to any components of the study interventions, including chemotherapy","ALL","18 Years",{"count":20,"type":21},125,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","The purpose of this study is to see if zanidatamab is safe and effective, when combined with chemotherapy, in treating people who has Human Epidermal Growth Factor Receptor 2 (HER2)-positive, early-stage breast cancer",[27,28],"HER2-positive Breast Cancer","Breast Cancer",[30,31,32,33],"HER2-positive early breast cancer","invasive breast carcinoma","zanidatamab","breast neoplasm","RECRUITING","2026-06-30",{"date":37,"type":38},"2026-07-02","ACTUAL",{"date":40,"type":38},"2025-09-24",{"date":42,"type":21},"2030-08-01",{"name":44,"class":45},"Jazz Pharmaceuticals","INDUSTRY",30,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":54,"minAge":4,"maxAge":4,"enrollmentInfo":55,"targetDuration":4,"studyType":57,"phases":4,"briefSummary":58,"conditions":59,"keywords":64,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":75},"100524784","pregnancy-surveillance-program-of-patients-exposed-to-epidiolexepidyolex-during-pregnancy-100524784","NCT06113237","Pregnancy Surveillance Program of Patients Exposed to Epidiolex\u002FEpidyolex During Pregnancy","Observational Pregnancy Surveillance Program of Patients Exposed to Epidiolex\u002FEpidyolex During Pregnancy to Assess the Risk of Pregnancy and Maternal Complications and Other Events of Interest on the Developing Fetus, Neonate, and Infant","Inclusion Criteria:\n\n1. Patients with exposure to at least 1 dose of Epidiolex\u002FEpidyolex during the 13 days prior to their LMP or at any time during pregnancy.\n2. Verbal or written informed consent to participate\n\nExclusion criteria:\n\n1\\) Patients exposed to a non-regulatory approved product containing Cannabidiol (CBD) during pregnancy who do not also have exposure to Epidiolex during the 13 days prior to their LMP or during the pregnancy.","FEMALE",{"count":56,"type":21},50,"OBSERVATIONAL","The purpose of this study is to evaluate pregnancy-related health outcomes in participants who are exposed to Epidiolex\u002FEpidyolex during pregnancy and their infant up to 12 months of life.",[60,61,62,63],"Maternal Complications","Pregnancy Complication","Birth Outcomes, Adverse","Pregnancy",[65,66],"Epidiolex","Epidyolex","2026-06-26",{"date":69,"type":38},"2026-06-29",{"date":71,"type":38},"2025-08-05",{"date":73,"type":21},"2033-08-31",{"name":44,"class":45},2,{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":4,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":83,"targetDuration":4,"studyType":22,"phases":85,"briefSummary":86,"conditions":87,"keywords":90,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":101},"100614668","phase-2-study-of-onc206-jzp3507-in-advanced-pheochromocytoma-and-paraganglioma-100614668","NCT07282587","Study of ONC206 (JZP3507) in Advanced Pheochromocytoma and Paraganglioma","A Phase 2 Study of ONC206 in Advanced Pheochromocytoma and Paraganglioma","Inclusion criteria:\n\n1. Has histologically confirmed pheochromocytoma or paraganglioma that is unresectable as determined by the Investigator.\n2. Has failed, is not a candidate for, or has declined standard of care treatment for PCPG. There is no limit on the number of prior systemic therapies.\n3. Must have measurable disease per RECIST v1.1, as assessed by the Investigator.\n4. Has adequately controlled blood pressure defined as blood pressure ≤150\u002F90 mmHg and with no change in antihypertensive medications (for participants with concomitant hypertension) for at least 14 days before the first dose of study treatment.\n5. Is ≥18 years of age.\n6. Is able to swallow oral tablets.\n7. Has an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2, assessed within 7 days before the first dose of study treatment.\n8. Has laboratory test results meeting the following parameters within 14 days before the first dose of study treatment\n9. Has an expected survival of at least 12 weeks, as predicted by the physician.\n10. Has pharmacologic control of catecholamine-associated symptoms if participant has functional disease.\n\nExclusion criteria:\n\n1. Has known hypersensitivity to ONC206 or any excipient used in the ONC206 study treatment formulation.\n2. Has active cardiac disease\u002Fcondition including any of the following:\n\n   1. Corrected QT interval (QTc) \\>480 msec (based on the mean from triplicate electrocardiogram \\[ECGs\\] performed during Screening).\n   2. History of documented congestive heart failure (New York Heart Association function classification III-IV).\n   3. Unstable angina, acute myocardial infarction, or arterial bypass or percutaneous transluminal coronary angioplasty within 6 months before the first dose of study treatment.\n3. Has previous exposure to ONC206 or dordaviprone (ONC201) from any source.\n4. Has a known additional malignancy that is progressing or has required active treatment within the past 2 years. Exceptions include participants with basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer, or Von Hippel-Lindau disease-associated tumors that do not require immediate surgery or intervention.\n5. Has received any of the following interventions within the specified time periods before the first dose of study treatment or plans to receive any of the following interventions during study participation:\n\n   * a. Any prior anticancer therapy or investigational agents within 4 weeks or 5 half-lives, whichever is shorter. Note: Denosumab and zoledronic acid are permissible.\n\n     \\*\\*i. Any treatment with somatostatin analog or lanreotide within 21 days before the baseline Positron Emission Tomography (PET) scan.\n   * b. Strong cytochrome P450 (CYP) inhibitors within 14 days. c. Strong CYP inducers within 14 days. d. Any radiotherapy within 14 days. e. Any major surgery, open biopsy or significant traumatic injury within 1 month (30 days).\n6. Is pregnant, breastfeeding, or planning to become pregnant while receiving study treatment or within 3 months after the last dose.\n7. Has uncontrolled intercurrent illness or any other medical, psychiatric, or social condition that, in the opinion of the Investigator, may interfere with participant safety or the ability to comply with study requirements.\n8. Has unresolved toxicities from previous locoregional, systemic, or any other therapies, defined as toxicities (other than Grade ≤2 neuropathy or alopecia) not yet resolved to the National Cancer Institute Common Terminology Criteria for Adverse Events Grade ≤1, or baseline and considered clinically significant; consult with Medical Monitor.\n9. Has an active infection that requires systemic therapy.",{"count":84,"type":21},90,[24],"This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG).",[88,89],"Advanced Pheochromocytoma and Paraganglioma","PCPG",[89,91,92],"ONC206","JZP3507","2026-06-09",{"date":95,"type":38},"2026-06-10",{"date":97,"type":38},"2026-01-02",{"date":99,"type":21},"2028-12-15",{"name":44,"class":45},9,{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":4,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":17,"minAge":109,"maxAge":110,"enrollmentInfo":111,"targetDuration":4,"studyType":22,"phases":113,"briefSummary":115,"conditions":116,"keywords":118,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":121,"completionDateStruct":123,"leadSponsor":125,"locationsCount":126},"100610873","phase-1-a-study-to-evaluate-the-efficacy-and-safety-of-cannabidiol-oral-solution-cbd-os-gwp42003-p-jzp926-for-the-treatment-of-focal-onset-seizures-100610873","NCT07233239","A Study to Evaluate the Efficacy and Safety of Cannabidiol Oral Solution (CBD-OS [GWP42003-P, JZP926]) for the Treatment of Focal-Onset Seizures","A Phase 1b, Multicenter, Open-Label, Single-arm Study to Evaluate the Efficacy and Safety of Cannabidiol Oral Solution (CBD-OS [GWP42003-P, JZP926]) in Participants Aged 12 to 75 Years for the Treatment of Focal-Onset Seizures","Participants are eligible to be included in the main study only if all of the following criteria apply:\n\n1. Participant has a documented diagnosis of focal epilepsy according to the ILAE Classification of Epilepsy, 2017, characterized by focal seizure types with typical interictal\u002Fictal EEG findings (eg, history of an EEG with focal sharp waves or slowing). Participants with a documented diagnosis of focal epilepsy and a normal EEG are eligible for inclusion.\n2. Participant is currently treated with at least 1, but no more than 4, antiseizure medications on a stable regimen.\n3. Participant is aged 12 to 75 years old, inclusive, at Screening.\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n1. Has a concurrent, confirmed diagnosis of non-epileptic seizures or events that can confound the assessment of the efficacy measures, in the opinion of the investigator.\n2. Has clinically significant unstable medical condition(s), other than epilepsy.\n3. History of suicidal behavior, current suicidal risk as determined from history, or presence of active suicidal ideation as indicated by a positive response to Item 4 or Item 5 on the C-SSRS or is considered at risk of suicide or self-harm based on the clinical judgement of the investigator following interview with the participant and\u002For caregiver.\n4. Has known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention, such as sesame oil.\n5. Is currently treated with Epidiolex or received treatment with Epidiolex within 28 days prior to Screening (Visit 1).\n6. Is currently using or has used recreational or medicinal cannabis, cannabinoid\u002FCBD based medications, products, or supplements (botanical or synthetic) within 28 days prior to Screening (Visit 1) and\u002For is unwilling to abstain for the duration of the study.\n7. Presence of only nonmotor focal aware seizures or primary generalized epilepsies.","12 Years","75 Years",{"count":112,"type":21},100,[114],"PHASE1","Cannabidiol oral solution (CBD-OS) is approved in the US for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or Tuberous sclerosis complex (TSC) in patients 1 year of age and older. This study will assess the efficacy and safety of CBD-OS in participants aged 12 to 75 years for the treatment of focal-onset seizures (FOS).",[117],"Focal Seizures",[117],"2026-06-05",{"date":93,"type":38},{"date":122,"type":38},"2025-11-26",{"date":124,"type":21},"2027-11-29",{"name":44,"class":45},24,{"id":128,"slug":129,"hasResults":11,"nctId":130,"briefTitle":131,"officialTitle":132,"acronym":4,"eligibilityCriteria":133,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":134,"targetDuration":4,"studyType":22,"phases":136,"briefSummary":137,"conditions":138,"keywords":150,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":154,"startDateStruct":155,"completionDateStruct":157,"leadSponsor":159,"locationsCount":160},"100569561","phase-2-a-phase-2-study-of-zanidatamab-in-patients-with-her2-expressing-tumors-100569561","NCT06695845","A Phase 2 Study of Zanidatamab in Patients With HER2-expressing Tumors","A Phase 2, Open-label, Multicenter Study to Evaluate Efficacy and Safety of Zanidatamab for the Treatment of Participants With Previously Treated HER2-expressing Solid Tumors (DiscovHER PAN-206)","Inclusion Criteria:\n\n1. Is at least 18 years of age inclusive at the time of signing the informed consent\n2. Participants with locally advanced, unresectable, or metastatic solid tumors (except Biliary Tract Cancer (BTC), defined as gallbladder cancer or cholangiocarcinoma) who have progressed following at least 1 prior systemic treatment for metastatic or advanced disease and have no available treatment options that have confirmed benefit. Prior treatment with HER2-targeted therapy is not permitted (Cohort 1 only). For participants with breast cancer (Cohort 2) or GEA (Cohort 3), prior HER2-targeted therapy is permitted and prior therapy with trastuzumab deruxtecan (T-DXd) is required.\n3. HER2 overexpression (IHC 3+) must be determined by a sponsor designated central laboratory.\n4. All participants must have adequate tumor sample for submission to allow central HER2 testing.\n5. Presence of at least 1 measurable lesion as assessed by Independent Central Review (ICR) based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1)\n6. Has Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.\n7. Has a life expectancy of at least 3 months, in the opinion of the investigator.\n8. Participants with history of treated and stable CNS metastases are eligible, provided the following criteria are met:\n\n   1. Participants also have measurable metastatic disease with HER2 overexpression (IHC 3+) outside the CNS.\n   2. Participants with treated CNS metastases that are no longer symptomatic may be included in the study if they recovered to \\\u003C Grade 1 (CTCAE Version 5.0 or higher) or baseline from the acute toxic effect associated with the treatment \\> 7 days prior to Cycle 1 Day 1.\n   3. Prior stereotactic radiosurgery or stereotactic radiotherapy should be completed at least 7 days (≥ 7 days) before the first dose of study intervention.\n9. Adequate organ functions.\n10. Females of childbearing potential must have a negative pregnancy test result.\n11. Females of childbearing potential and males with a partner of childbearing potential must be willing to use 2 methods of birth control.\n\nExclusion Criteria:\n\n1. Has known or suspected leptomeningeal disease and\u002For untreated brain metastasis.\n2. Has uncontrolled or significant cardiovascular disease\n3. Has ongoing toxicity related to prior cancer therapy\n4. Has uncontrolled infection or requiring IV antibiotics, antivirals, or antifungals.\n5. Has known Human Immunodeficiency Virus (HIV) infection.\n6. Has active hepatitis B or C infection.\n7. Has an active SARS-CoV-2 infection.\n8. Has a history of life-threatening hypersensitivity to monoclonal antibody (mAbs) or to recombinant proteins or excipients in the drug formulation of zanidatamab.\n9. Has any serious underlying medical or psychiatric condition that would impair the ability of the participant to receive or tolerate the planned treatment at the investigational site.\n10. Has any issue or condition that, in the opinion of the investigator, would contraindicate the participant's participation in the study or confound the results of the study.\n11. Prior treatment with HER2-targeted therapy (Cohort 1 only).\n12. Has a history of trauma or major surgery\n13. Was treated with systemic antineoplastic therapy, including hormonal therapies for breast cancer, or any investigational therapy within 4 weeks or 5 half-lives (whichever is longer) prior to Cycle 1 Day 1.\n14. Received zanidatamab at any time prior to the current study.\n15. Colorectal Cancer (CRC) participants with known KRAS\u002FNRAS and BRAF mutations.\n16. Non-Small Cell Lung Cancer (NSCLC) participants with known ALK, EGFR mutations and ROS1 fusion.\n17. Female participants who are breastfeeding or pregnant, and female and male participants planning a pregnancy.\n18. Prior or concurrent invasive malignancy other than the disease under study, whose natural history or treatment has, in the opinion of the investigator or medical monitor, the potential to interfere with the safety or efficacy assessment of the investigational regimen.",{"count":135,"type":21},200,[24],"The purpose of this study is to evaluate the efficacy and safety of zanidatamab for the treatment of participants with previously treated solid tumors that have Human Epidermal Growth Factor Receptor 2 (HER2) Immunohistochemistry (IHC) 3+ overexpression.",[28,139,140,141,142,143,144,145,146,147,148,149],"Gastric Cancer","Esophageal Cancer","Gastroesophageal Cancer","Colorectal Cancer","Endometrial Cancer","Non-small Cell Lung Cancer","Ovarian Cancer","Urothelial Carcinoma","Salivary Gland Cancer","Pancreatic Cancer","HER-2 Protein Overexpression",[151,152,153],"JZP598","ZW25","HER2 IHC 3+ Overexpression Solid Tumors",{"date":93,"type":38},{"date":156,"type":38},"2025-01-14",{"date":158,"type":21},"2027-12-31",{"name":44,"class":45},28,{"id":162,"slug":163,"hasResults":11,"nctId":164,"briefTitle":165,"officialTitle":166,"acronym":167,"eligibilityCriteria":168,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":110,"enrollmentInfo":169,"targetDuration":4,"studyType":22,"phases":171,"briefSummary":173,"conditions":174,"keywords":177,"overallStatus":184,"whyStopped":4,"lastUpdateSubmitDate":185,"lastUpdatePostDateStruct":186,"startDateStruct":188,"completionDateStruct":189,"leadSponsor":191,"locationsCount":192},"100640164","phase-3-a-study-evaluating-the-efficacy-and-safety-of-xywav-expanded-dosing-vs-placebo-in-participants-with-narcolepsy-or-ih-100640164","NCT07625280","A Study Evaluating the Efficacy and Safety of Xywav Expanded Dosing vs Placebo in Participants With Narcolepsy or IH","A Phase 3, Multicenter, Double-blind, Placebo-controlled, Randomized-withdrawal Study to Evaluate the Efficacy and Safety of Expanded Dosing Regimens for Xywav in Adult Participants With Narcolepsy or Idiopathic Hypersomnia","XYRISE","Inclusion Criteria:\n\n1. Has a primary diagnosis of IH or narcolepsy Type 1 or Type 2 (NT1 or NT2)\n2. If not currently treated with oxybate, has clinically significant symptoms of excessive daytime sleepiness (EDS) with an Epworth Sleepiness Scale (ESS) score \\> 11 at screening.\n3. If currently treated with oxybate, must have documented improvement of EDS with oxybate treatment per the investigator's clinical judgement.\n4. If currently treated with oxybate, has been taking the same stable dosing regimen at a total nightly dosage of 3 g to 9 g (inclusive) for at least 2 months at screening.\n5. If previously treated with (and not currently taking) oxybate, must have been off oxybate treatment for at least 2 weeks prior to screening. Must not have previously discontinued oxybate due to reasons related to intolerability, safety, or lack of efficacy.\n6. If currently treated with anticataplectics (NT1 only) and\u002For alerting agents, has been taking the same dosage for at least 1 month prior to screening and has no current plans to adjust the dosage during the study period.\n7. If currently treated with nicotine replacement therapy, has been taking the same dosage for at least 1 month prior to screening and has no current plans to adjust the dosage during the study period.\n8. Adequate contraceptive precautions\n\nExclusion criteria:\n\n1. Shows evidence of a previous untreated or inadequately treated sleep disorder considered by the investigator to negatively impact the conduct of the study, including sleep-disordered breathing, parasomnias, circadian rhythm sleep disorders, or restless legs syndrome determined by a previous sleep-laboratory diagnosis or interview utilizing modules of the Diagnostic Interview for Sleep Patterns and Disorders.\n2. Has succinic semi-aldehyde dehydrogenase deficiency by medical history.\n3. Has uncontrolled hypothyroidism as determined by central clinical laboratory test results.\n4. Has a current seizure disorder.\n5. Has a history of head trauma associated with loss of consciousness in the past 5 years\n6. Has a history or presence of bipolar disorder, bipolar-related disorders, schizophrenia, schizophrenia spectrum disorders, or other psychotic disorders\n7. Has a history or presence of any unstable or clinically significant medical condition, behavioral or psychiatric disorder, or history or presence of another neurologic disorder or surgical history that might affect the participant's safety and\u002For interfere with the conduct of the study, in the opinion of the investigator.\n8. Has any other significant disease or disorder that, in the opinion of the investigator, may either put the participant, other participants, or study staff at risk because of participation in the study, may influence the result of the study, or may affect the participant's safety or ability to take part in the study.\n9. Any past or current medical conditions or experience that, in the investigator's clinical judgment, would preclude treatment with a once-nightly dose \\> 6 g up to 7.5 g dose or twice-nightly regimen with a total nightly dosage \\> 9 g up to 12 g (divided into 2 doses).\n10. Has any severe drug allergy or a history of allergic or severe adverse reactions or intolerance to Xyrem, Xywav, Gamma-hydroxybutyrate (GHB), or any components of the dosage forms.\n11. Has recently taken, is taking, or plans to take any of the following:\n\n    1. A substance or medication contraindicated with Xywav use\n    2. A medication with a known drug-drug interaction with Xywav\n    3. Medications known to have clinically significant CNS sedating effects:\n    4. Other medications, natural health products, or substances from which the participant experiences clinically significant sedation\n12. Has recently taken, is taking, or plans to take an Orexin 2 receptor (OX2R) agonist during the study.\n13. Has tobacco-use disorder or uses vaping products that impact sleep\n14. Has excessive caffeine consumption that may impact sleep\n15. Has clinically significant abnormal laboratory values\n16. Has an occupation that requires nighttime or variable shift work\n17. Has plans for travel across more than 3 time zones during the study",{"count":170,"type":21},108,[172],"PHASE3","The purpose of this study is to evaluate the efficacy and safety of expanded Xywav dosing regimens in adult participants with narcolepsy or idiopathic hypersomnia (IH).",[175,176],"Narcolepsy","Idiopathic Hypersomnia",[178,179,180,181,182,183],"Xywav","Narcolepsy Type 1","NT1","Narcolepsy Type 2","NT2","IH","NOT_YET_RECRUITING","2026-05-27",{"date":187,"type":38},"2026-06-04",{"date":35,"type":21},{"date":190,"type":21},"2028-01-06",{"name":44,"class":45},1,{"id":194,"slug":195,"hasResults":11,"nctId":196,"briefTitle":197,"officialTitle":198,"acronym":4,"eligibilityCriteria":199,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":200,"targetDuration":4,"studyType":22,"phases":201,"briefSummary":202,"conditions":203,"keywords":205,"overallStatus":184,"whyStopped":4,"lastUpdateSubmitDate":210,"lastUpdatePostDateStruct":211,"startDateStruct":212,"completionDateStruct":214,"leadSponsor":216,"locationsCount":192},"100628284","phase-2-a-study-of-lurbinectedin-in-combination-with-durvalumab-for-the-treatment-of-participants-with-es-sclc-100628284","NCT07459634","A Study of Lurbinectedin in Combination With Durvalumab for the Treatment of Participants With ES-SCLC","A Phase 2, Multicenter, Open-Label, Single-Arm Study of Lurbinectedin in Combination With Durvalumab as First-line Maintenance Therapy in Participants With Extensive-Stage Small-Cell Lung Cancer Following Induction Treatment With Platinum Based Chemotherapy and Durvalumab","Inclusion Criteria:\n\n1. Participant has histologically or cytologically confirmed ES-SCLC (per Veterans Administration Lung Study Group staging system).\n2. Participant has received 4 cycles of definitive platinum-based chemotherapy with durvalumab\n3. Has Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 or 1.\n4. Must have a life expectancy of ≥ 12 weeks as assessed by the treating physician.\n5. Adequate hematologic and end-organ function for at least 7 days prior to dosing.\n6. Has a body weight \\> 30 kg.\n7. Adequate contraceptive precautions.\n\nExclusion Criteria:\n\n1. History of leptomeningeal carcinomatosis.\n2. Uncontrolled pleural effusion, pericardial effusion, or ascites requiring recurrent drainage procedures\n3. History of another primary malignancy\n4. Presence or history of Central Nervous System (CNS) metastases\n5. History of allogeneic organ transplantation.\n6. History of idiopathic pulmonary fibrosis, organizing pneumonia, drug-induced pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis on the screening chest CT scan.\n7. Any concurrent chemotherapy, study intervention, biologic, or hormonal therapy for cancer treatment.\n8. Receipt of live attenuated vaccine within 30 days prior to the first dose of study intervention.\n9. Concurrent enrollment in another clinical study\n10. Prior enrollment or treatment in a previous durvalumab clinical study regardless of treatment arm assignment.\n11. Pregnant or breastfeeding or intending to become pregnant during the study or within 6 months after the last dose of study intervention.",{"count":56,"type":21},[24],"The purpose of this study is to evaluate the efficacy and safety of lurbinectedin in combination with durvalumab for the treatment of participants with extensive stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first line (1L) induction therapy.",[204],"Extensive-stage Small-cell Lung Cancer",[206,207,208,209],"Zepzelca","Lurbinectedin","Durvalumab","Imfinzi","2026-05-22",{"date":185,"type":38},{"date":213,"type":21},"2026-06-12",{"date":215,"type":21},"2028-04-30",{"name":44,"class":45},{"id":218,"slug":219,"hasResults":11,"nctId":220,"briefTitle":221,"officialTitle":222,"acronym":4,"eligibilityCriteria":223,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":224,"targetDuration":4,"studyType":22,"phases":226,"briefSummary":227,"conditions":228,"keywords":230,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":238,"lastUpdatePostDateStruct":239,"startDateStruct":241,"completionDateStruct":243,"leadSponsor":245,"locationsCount":246},"100537803","phase-3-efficacy-and-safety-of-zanidatamab-with-standard-of-care-therapy-against-standard-of-care-therapy-for-advanced-her2-positive-biliary-tract-cancer-100537803","NCT06282575","Efficacy and Safety of Zanidatamab With Standard-of-care Therapy Against Standard-of-care Therapy for Advanced HER2-positive Biliary Tract Cancer","An Open-label Randomized Trial of the Efficacy and Safety of Zanidatamab With Standard-of-care Therapy Against Standard-of-care Therapy Alone for Advanced HER2-positive Biliary Tract Cancer","Inclusion Criteria\n\n1. Histologically- or cytologically-confirmed Biliary Tract Cancer (BTC), including Gallbladder Cancer (GBC), Intrahepatic Cholangiocarcinoma (ICC), or Extrahepatic Cholangiocarcinoma (ECC).\n2. Locally advanced unresectable or metastatic BTC and not eligible for curative resection, transplantation, or ablative therapies.\n3. Received no more than 2 cycles of systemic therapy which is limited to Cisplatin and Gemcitabine (CisGem) with or without a PD-1\u002FL1 inhibitor (physician's choice of durvalumab or pembrolizumab, where approved under local regulations) for advanced unresectable or metastatic disease.\n4. HER2-positive disease (defined as IHC 3+; or IHC 2+\u002F ISH+) by IHC and in situ Hybridization (ISH) assay (in participants with IHC 2+ tumors) at a central laboratory on new biopsy tissue or archival tissue from the most recent biopsy.\n5. Assessable (measurable or non-measurable) disease as defined by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1), per investigator assessment.\n6. Male or female ≥ 18 years or age (or the legal age of adulthood per country-specific regulations).\n7. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.\n8. Adequate organ function\n9. Females of childbearing potential must have a negative pregnancy test result.\n10. Females of childbearing potential and males with a partner of childbearing potential must be willing to use 2 methods of birth control.\n\nExclusion Criteria\n\n1. Prior treatment with a HER2-targeted agent\n2. Prior treatment with checkpoint inhibitors, other than durvalumab or pembrolizumab\n3. The following BTC histologic subtypes are excluded: small cell cancer, neuroendocrine tumors, lymphoma, sarcoma, mixed tumor histology, and mucinous cystic neoplasms detected in the biliary tract region.\n4. Use of systemic corticosteroids.\n5. Brain metastases\n6. Severe chronic or active infections\n7. History of allogeneic organ transplantation.\n8. Active or prior autoimmune inflammatory conditions\n9. History of interstitial lung disease or non-infectious pneumonitis.\n10. Participation in another clinical trial with an investigational medicinal product within the last 3 months.\n11. Females who are breastfeeding\n12. Any other medical, social, or psychosocial factors that, in the opinion of the investigator, could impact safety or compliance with study procedures.\n13. Use of phenytoin",{"count":225,"type":21},286,[172],"The purpose of this study is to evaluate the efficacy and safety of Zanidatamab plus CisGem (Cisplatin and Gemcitabine) with or without the addition of a programmed death protein 1\u002Fligand-1 (PD-1\u002FL1) inhibitor (physician's choice of either Durvalumab or Pembrolizumab, where approved under local regulations) as first line of treatment for participants with human epidermal growth factor receptor 2 (HER2)-positive biliary tract cancer.",[229],"Biliary Tract Cancer",[151,152,231,232,233,234,235,236,237],"HER-2 positive BIliary Tract cancer","Gallbladder Cancer","Intrahepatic Cholangiocarcinoma (ICC)","Extrahepatic Cholangiocarcinoma (ECC)","Zanidatamab","HER-2 overexpression","HER-2 amplification","2026-05-07",{"date":240,"type":38},"2026-05-11",{"date":242,"type":38},"2024-07-19",{"date":244,"type":21},"2030-04-01",{"name":44,"class":45},185,{"id":248,"slug":249,"hasResults":11,"nctId":250,"briefTitle":251,"officialTitle":252,"acronym":4,"eligibilityCriteria":253,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":254,"targetDuration":4,"studyType":22,"phases":256,"briefSummary":257,"conditions":258,"keywords":262,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":264,"lastUpdatePostDateStruct":265,"startDateStruct":267,"completionDateStruct":269,"leadSponsor":271,"locationsCount":272},"100482055","phase-1-a-study-of-jzp815-oral-capsules-in-adult-participants-with-advanced-or-metastatic-solid-tumors-harboring-mitogen-activated-protein-kinase-mapk-pathway-alterations-to-investigate-the-safety-dosing-and-antitumor-activity-of-jzp815-100482055","NCT05557045","A Study of JZP815 Oral Capsules in Adult Participants With Advanced or Metastatic Solid Tumors Harboring Mitogen Activated Protein Kinase (MAPK) Pathway Alterations to Investigate the Safety, Dosing, and Antitumor Activity of JZP815","Phase 1, FIH, Open-label, Nonrandomized, Multicenter Study of JZP815 in Participants With Advanced or Metastatic Solid Tumors Harboring Alterations in the MAPK Pathway","Inclusion Criteria:\n\n* Participant must be ≥ 18 years of age, at the time of signing the informed consent\n* Participants who have histological or cytological diagnosis of an advanced or metastatic solid tumor carrying a documented, clinically significant, MAPK pathway alteration\n* Participants must have exhausted all available standard of care therapies, or in the opinion of the investigator would be unlikely to tolerate or derive clinically meaningful benefit from available standard of care therapy\n* Performance status (ECOG) of 0 or 1, measured within 72 hours before start of treatment. For Arm 7 (NRAS Q61 mutated anaplastic thyroid cancer) in Part B (Expansion), ECOG of 0 to 2, measured within 72 hours before the start of treatment.\n* Must have measurable disease by RECIST v1.1\n* Tumor must be safely amenable to core needle or excisional biopsy (applies only to participants enrolled in Pre-Expansion cohorts)\n* Adequate organ function\n* Expected life expectancy of at least 12 weeks\n* For each arm in Part B (Expansion), participants must be diagnosed with the tumor type(s) carrying the mutation(s) specified and meet protocol specified requirements for prior therapy\n* Male participants must agree to refrain from donating sperm plus either be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent or must agree to use contraception\n* Female participants are eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies: is a women of nonchildbearing potential (WONCBP) or is a women of childbearing potential (WOCBP) and using a contraceptive method that is highly effective during the study intervention period and for at least 3 months after the last dose of study intervention and agrees not to donate eggs\n* A WOCBP must have a negative highly sensitive pregnancy test (urine or serum) within 3 days before the first dose of study intervention\n* Capable of giving signed informed consent\n\nExclusion Criteria:\n\n* Known uncontrolled brain metastases. Stable brain metastases either treated or being treated with a stable dose of steroids\u002Fanticonvulsants, with no dose change in the previous 4 weeks, are permitted\n* Active fungal, bacterial and\u002For known viral infection including HIV or Hepatitis A, B, C\n* Concomitant malignancies or previous malignancies with less than 2 years disease-free interval at the time of enrollment, with the exception of non-metastatic, non-melanomatous skin cancers, carcinoma in-situ, melanoma in-situ, prostate cancer with undetectable PSA, indolent thyroid cancer that are adequately treated\n* Has clinically significant (ie, active) cardiovascular disease: cerebral vascular accident\u002Fstroke (\\\u003C 6 months prior to enrollment), myocardial infarction (\\\u003C 6 months prior to enrollment), unstable angina, congestive heart failure (\\> New York Heart Association Classification Class II), QTc ≥ 470 msec, or serious cardiac arrhythmia requiring medication\n* Uncontrolled or severe intercurrent medical condition\n* Gastrointestinal condition that could impair absorption of study intervention or inability to ingest study intervention\n* In the judgement of the investigator, any important medical illness or abnormal laboratory finding that would increase the risk of participating in this study\n* Received any cancer directed therapy (chemotherapy, hormonal therapy, biologic, etc.) within 28 days or 5 half-lives (whichever is shorter) of starting study intervention. For Arm 7 (NRAS Q61 mutated anaplastic thyroid cancer) in Part B (Expansion), participants who have received radio-sensitizing chemotherapy (low-dose chemotherapy) are permitted a wash-out period of 7 days or 5 half-lives, whichever is shorter (a discussion with the sponsor is required). Participants who have received radiotherapy must have recovered from acute toxicities associated with treatment.\n* Use of any products or medicines known to be strong or moderate inducers or inhibitors of CYP3A4, which cannot be discontinued at least 4 weeks or 5 half-lives (whichever is shorter) before starting study intervention, or planned use at any time during the study\n* Use of proton pump inhibitors (eg, omeprazole) and histamine-2 receptor antagonists (eg, famotidine), which cannot be discontinued at least 2 weeks before first dose, or planned use at any time during the study\n* Concurrent therapy with any other investigational agent",{"count":255,"type":21},332,[114],"This phase 1 study will investigate the safety, dosing, and initial antitumor activity of JZP815 in participants with advanced or metastatic solid tumors harboring alterations in the MAPK pathway.",[259,260,261],"Advanced Cancer","Metastatic Cancer","Solid Tumor",[259,260,261,263],"JZP815","2026-04-22",{"date":266,"type":38},"2026-04-23",{"date":268,"type":38},"2022-10-10",{"date":270,"type":21},"2028-04-01",{"name":44,"class":45},15,{"id":274,"slug":275,"hasResults":11,"nctId":276,"briefTitle":277,"officialTitle":278,"acronym":4,"eligibilityCriteria":279,"healthyVolunteers":280,"sex":17,"minAge":18,"maxAge":281,"enrollmentInfo":282,"targetDuration":4,"studyType":22,"phases":284,"briefSummary":285,"conditions":286,"keywords":288,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":290,"lastUpdatePostDateStruct":291,"startDateStruct":293,"completionDateStruct":295,"leadSponsor":297,"locationsCount":192},"100621970","phase-1-a-study-to-investigate-the-safety-tolerability-pharmacokinetics-and-food-effect-of-jzp047-in-healthy-participants-100621970","NCT07377539","A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Food Effect of JZP047 in Healthy Participants","A Double-Blind, Sponsor-Unblinded, Randomized, Placebo-Controlled Study of the Safety, Tolerability, and Pharmacokinetics of Single-Ascending Doses of JZP047 and an Open-Label Food Effect Study of JZP047 in Healthy Male and Female Participants","Participants are eligible to be included in the study only if all of the following criteria apply:\n\n1. Healthy participants 18 to 55 years of age, willing and able to comply with study requirements.\n2. Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 120 days after the last dose of study intervention:\n\n   * Refrain from donating sperm.\n   * Use contraception\u002Fbarrier as follows:\n\n     * Use a male condom with female partner and use of an additional highly effective contraceptive method with a failure rate of \\\u003C 1% per year and should also be advised of the benefit of a female partner using a highly effective method of contraception, as a condom may break or leak, when having sexual intercourse with a woman of childbearing potential (WOCBP) who is currently not pregnant.\n     * Agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person.\n3. Female participants are eligible to participate if:\n\n   * She is a woman of nonchildbearing potential (WONCBP).\n   * Pregnancy testing will be performed prior to administration of study intervention to confirm female participants are not pregnant.\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n1. History of or presence of clinically significant medical illness or disorder or have a medical issue that may interfere with absorption, distribution, metabolism, or excretion of drugs..\n2. History or presence of clinically significant allergy (other than seasonal allergies that do not require treatment during the study) or clinically significant allergy to adhesive bandages, adhesive dressing, ECG patches, or medical tape.\n3. History (within 5 past years) or presence of a diagnosis of alcohol abuse, a substance abuse disorder, known drug dependence, or seeking of treatment for an alcohol- or substance abuse-related disorder.\n4. Current diagnosis of or receiving treatment for depression; past (within 5 years) clinically significant major depressive episode.\n5. History of suicide attempt, current suicidal risk as determined from history, or presence of active suicidal ideation.\n6. Presence of any other condition that will cause a risk to participants if they participate in the study as determined by the investigator.\n7. Poor peripheral venous access or history of fainting (or passing out) during blood draws.\n8. Use or intent to use any prescription medications or nonprescription drugs (including vitamins, recreational drugs, and dietary or herbal supplements) within 14 days or for 5 half-lives, whichever is longer prior to Check-in and throughout the conduct of the study.",true,"55 Years",{"count":283,"type":21},86,[114],"This is a Phase 1, double-blind, sponsor-unblinded, randomized, placebo-controlled study designed to characterize the safety, tolerability, and PK of JZP047 in healthy participants following single-ascending doses of study intervention. Additionally, the effect of food on PK following a single dose of JZP047 will be assessed through comparison of PK between the fed and fasted states.",[287],"Healthy Participants",[287,289],"JZP047","2026-04-15",{"date":292,"type":38},"2026-04-20",{"date":294,"type":38},"2026-02-03",{"date":296,"type":21},"2026-07-05",{"name":44,"class":45},{"id":299,"slug":300,"hasResults":11,"nctId":301,"briefTitle":302,"officialTitle":303,"acronym":304,"eligibilityCriteria":305,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":306,"targetDuration":4,"studyType":22,"phases":308,"briefSummary":309,"conditions":310,"keywords":313,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":326,"lastUpdatePostDateStruct":327,"startDateStruct":329,"completionDateStruct":331,"leadSponsor":333,"locationsCount":334},"100483863","phase-3-onc201-in-h3-k27m-mutant-diffuse-glioma-following-radiotherapy-the-action-study-100483863","NCT05580562","ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)","ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study","ACTION","Inclusion Criteria:\n\n1. Able to understand the study procedures and agree to participate in the study by providing written informed consent (by participant or legally authorized representative), and assent when applicable.\n2. Body weight ≥ 10 kg at time of randomization.\n3. Histologically diagnosed H3 K27M-mutant diffuse glioma (new diagnosis). Detection of a missense K27M mutation in any histone H3-encoding gene detected by testing of tumor tissue (immunohistochemistry \\[IHC\\] or next-generation sequencing \\[NGS\\] in a Clinical Laboratory Improvement Amendments \\[CLIA\\]-certified or equivalent laboratory). \\[Site to provide (as available): ≥ 11 unstained formalin-fixed paraffin-embedded (FFPE) slides from tumor tissue.\\]\n4. At least one, high-quality, contrast-enhanced MRI of the brain obtained prior to starting radiotherapy for submission to sponsor's imaging vendor for central read. For participants who had a surgical resection, this scan must be post-resection; for participants who did not have a resection, this scan may be pre- or post-biopsy.\n5. At least one, high-quality, contrast-enhanced MRI of the brain obtained 2 to 6 weeks after completion of frontline radiotherapy. If unable to obtain contrast-enhanced imaging due to lack of venous access after multiple attempts, a patient may still be eligible after collection of a nonenhanced MRI of the brain. \\[Site to also provide all available MRIs completed prior to initiating treatment with study intervention.\\]\n6. Received frontline radiotherapy\n\n   1. Initiated radiotherapy within 12 weeks from the initial diagnosis of H3 K27M-mutant diffuse glioma.\n   2. Completed radiotherapy within 2 to 6 weeks prior to randomization\n   3. Completed standard fractionated radiotherapy (eg. 54 to 60 Gy in 28 to 33 fractions given over approximately 6 weeks or hypofractionated radiotherapy (eg. 40 Gy in 15 fractions given over approximately 3 weeks).\n7. Karnofsky Performance Status or Lansky Performance Status ≥ 70 at time of randomization.\n8. Stable or decreasing dose of corticosteroids and anti-seizure medications for 7 days prior to randomization, if applicable. Stable steroid dose is defined as ≤ 2 mg\u002Fday increase (based on dexamethasone dose or equivalent dose of an alternative steroid).\n\nExclusion Criteria:\n\n1. Primary spinal tumor.\n2. Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons.\n3. Evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination.\n4. Any known concurrent malignancy.\n5. New lesion(s) outside of the radiation field.\n6. Received whole-brain radiotherapy.\n7. Received proton therapy for glioma.\n8. Use of any of the following treatments within the specified time periods prior to randomization:\n\n   1. Dordaviprone (ONC201) or ONC206 at any time.\n   2. Systemic bevacizumab (includes biosimilars) at any time since the initial diagnosis of H3 K27M-mutant diffuse glioma.\n   3. Temozolomide within past 3 weeks.\n   4. Tumor treating fields at any time.\n   5. DRD2 antagonist within past 2 weeks.\n   6. Any investigational therapy within past 4 weeks.\n   7. Strong CYP3A4 inhibitors within 3 days.\n   8. Strong CYP3A4 inducers (includes enzyme-inducing antiepileptic drugs) within 2 weeks.\n9. Laboratory test results meeting any of the following parameters within 2 weeks prior to randomization:\n\n   1. Absolute neutrophil count \\\u003C 1.0 × 109\u002FL or platelets \\\u003C 75 × 109\u002FL.\n   2. Total bilirubin \\> 1.5 × upper limit of normal (ULN) (participants with Gilbert's syndrome may be included with total bilirubin \\> 1.5 × ULN if direct bilirubin is ≤ 1.5 × ULN).\n   3. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \\> 2.5 × ULN.\n   4. Creatinine clearance ≤ 60 mL\u002Fmin as calculated by the Cockcroft Gault equation (or estimated glomerular filtration rate \\\u003C 60 mL\u002Fmin\u002F1.73 m2).\n10. QTc \\> 480 msec (based on mean from triplicate electrocardiograms) during screening.\n11. Known hypersensitivity to any excipients used in the study intervention formulation.\n12. Pregnant, breastfeeding, or planning to become pregnant while receiving study intervention or within 3 months after the last dose. Participants of childbearing potential must have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study intervention.\n13. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection requiring systemic therapy or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n14. Any other condition (eg, medical, psychiatric, or social) that, in the opinion of the investigator, may interfere with participant safety or the ability to complete the study according to the protocol.",{"count":307,"type":21},510,[172],"This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with dordaviprone (ONC201) following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.",[311,312],"H3 K27M","Glioma",[311,314,315,316,317,318,319,320,321,322,323,324,325],"H3 K28M","H3 K27-altered","histone","H3F3A","HIST1H3B","HIST1H3C","H3.1","H3.3","DMG","thalamus","thalamic","midline","2026-04-13",{"date":328,"type":38},"2026-04-16",{"date":330,"type":38},"2023-01-23",{"date":332,"type":21},"2028-06",{"name":44,"class":45},162,{"id":336,"slug":337,"hasResults":11,"nctId":338,"briefTitle":339,"officialTitle":340,"acronym":4,"eligibilityCriteria":341,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":342,"targetDuration":4,"studyType":22,"phases":343,"briefSummary":344,"conditions":345,"keywords":347,"overallStatus":184,"whyStopped":4,"lastUpdateSubmitDate":352,"lastUpdatePostDateStruct":353,"startDateStruct":354,"completionDateStruct":356,"leadSponsor":358,"locationsCount":359},"100633996","phase-2-a-study-of-jzp3507-onc206-in-recurrent-grade-2-or-3-meningioma-100633996","NCT07533942","A Study of JZP3507 (ONC206) in Recurrent Grade 2 or 3 Meningioma","A Phase 2 Study to Investigate Efficacy, Safety, Tolerability, and Pharmacokinetics of JZP3507 (ONC206) in Adults With Recurrent Grade 2 or 3 Meningioma","Key Inclusion Criteria:\n\nAge\n\n1. Is ≥ 18 years of age at the time of signing the informed consent.\n\n   Type of Participant and Disease Characteristics\n2. Has histologically confirmed Grade 2 or 3 meningioma.\n3. Has failed, is not a candidate for, or has declined standard of care treatment for meningioma. Note: There is no limit on the number of prior systemic therapies.\n4. Has measurable disease, as assessed by the investigator. Measurable disease is defined as at least one lesion measuring ≥ 10 mm on perpendicular dimensions by contrast-enhanced MRI performed within 28 days prior to study enrollment.\n5. Has progressive disease (PD) per Response Assessment in Neuro-Oncology (RANO) criteria, as assessed by the investigator using axial, contrast-enhanced T1-weighted magnetic resonance imaging (MRI). PD is defined as an increase in size of the measurable primary lesion on imaging by at least 15% in sum of product of target lesions since last treatment or between scans separated by no more than 6 months. The presence of a new lesion would also qualify as PD.\n6. Is able to submit historic disease-related imaging from at least 9 months prior to study entry to central imaging vendor (preferably all available disease-related imaging from initial diagnosis onwards).\n7. Is able to swallow oral tablets.\n8. Has a Karnofsky Performance Status (KPS) of at least 70.\n9. Has laboratory test results meeting the following parameters within 14 days before the start of study intervention:\n\n   1. Absolute neutrophil count ≥ 1.0 × 109\u002FL and platelets ≥ 75 × 109\u002FL.\n   2. Total bilirubin ≤ 1.5 × upper limit of normal (ULN) (participants with Gilbert's syndrome may be included with total bilirubin \\> 1.5 × ULN if direct bilirubin is ≤ 1.5 × ULN).\n   3. Aspartate (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN. Note: For participants with documented baseline liver metastasis, the following limits will apply: ≤ 5 × ULN for transaminase.\n   4. Creatinine clearance ≥ 50 mL\u002Fmin as calculated by the Cockcroft Gault equation (or estimated glomerular filtration rate \\[eGFR\\] \\> 60 mL\u002Fmin\u002F1.73 m2) or serum creatinine ≤ 1.5 × ULN.\n10. Has an expected survival of at least 12 weeks, as predicted by the physician.\n11. Is able to submit at least 10 (preferably ≥ 15 slides, if available) unstained formalin-fixed paraffin-embedded (FFPE) slides or a tissue block with sufficient material for \\~15 slides from participant's tumor tissue to the sponsor.\n12. Has had an MRI within 28 days before the start of study intervention, with the corticosteroid dose stable or decreasing at least 5 days prior to the scan.\n\n    Sex and Contraceptive\u002FBarrier Requirements\n13. Agrees to the following based on sex assigned at birth: is not of child-bearing potential or agrees to use appropriate contraception, as defined in protocol, for males and females.\n\nKey Exclusion Criteria:\n\nMedical Conditions\n\n1. Has known hypersensitivity to JZP3507, dordaviprone, or any excipient used in the JZP3507 study intervention formulation.\n2. Has active cardiac disease\u002Fcondition as defined in the protocol.\n3. Has a known additional malignancy that is progressing or has required active treatment within the past 2 years. Exceptions include participants with basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.\n4. Has an active infection that requires systemic therapy.\n\n   Prior\u002FConcomitant Therapy\n5. Has received any of the following interventions within the specified time periods before the first dose of study intervention or plans to receive any of the following interventions during study participation:\n\n   1. Prior anticancer therapy or investigational agents within 28 days or 5 half-lives, whichever is shorter.\n   2. Antibody-based anticancer therapy within 42 days.\n   3. Radiotherapy within 24 weeks (\\~6 months).\n   4. Strong CYP3A4 inhibitors within 14 days.\n   5. Strong CYP3A4 inducers within 14 days.\n   6. Major surgery, open biopsy, or significant traumatic injury within 30 days.\n6. Has uncontrolled intercurrent illness or any other medical, psychiatric, or social condition that, in the opinion of the investigator, may interfere with participant safety or the ability to comply with study requirements.\n\n   Prior\u002FConcurrent Clinical Study Experience\n7. Has previous exposure to JZP3507 or dordaviprone from any source.\n\n   Diagnostic Assessments\n8. Has optic nerve sheath meningioma, extracranial meningioma, or meningioma primarily localized spinal cord.\n9. Has more than 3 measurable lesions.",{"count":46,"type":21},[24],"This study will recruit participants with Grade 2 and 3 meningiomas who have failed prior therapy. Participants will receive oral doses of JZP3507. The antitumor activity and safety of JZP3507 will be evaluated.",[346],"Meningioma",[348,349,92,350,91,351],"Grade 2 Meningioma","Grade 3 Meningioma","Recurrent Meningioma","H3 K27me3 loss","2026-04-10",{"date":328,"type":38},{"date":355,"type":21},"2026-06-01",{"date":357,"type":21},"2031-10-16",{"name":44,"class":45},5,{"id":361,"slug":362,"hasResults":11,"nctId":363,"briefTitle":364,"officialTitle":365,"acronym":4,"eligibilityCriteria":366,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":367,"targetDuration":4,"studyType":22,"phases":369,"briefSummary":370,"conditions":371,"keywords":373,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":376,"lastUpdatePostDateStruct":377,"startDateStruct":379,"completionDateStruct":381,"leadSponsor":383,"locationsCount":384},"100549546","phase-3-a-study-comparing-the-efficacy-and-safety-of-zanidatamab-to-trastuzumab-each-in-combination-with-physicians-choice-chemotherapy-for-the-treatment-of-participants-with-metastatic-her2-positive-breast-cancer-100549546","NCT06435429","A Study Comparing the Efficacy and Safety of Zanidatamab to Trastuzumab, Each in Combination With Physician's Choice Chemotherapy, for the Treatment of Participants With Metastatic HER2-positive Breast Cancer","A Phase 3, Randomized, Open-label, Multicenter, Controlled Study to Evaluate the Efficacy and Safety of Zanidatamab in Combination With Physician's Choice Chemotherapy Compared to Trastuzumab in Combination With Physician's Choice Chemotherapy for the Treatment of Participants With Metastatic HER2-positive Breast Cancer Who Have Progressed on, or Are Intolerant to, Previous Trastuzumab Deruxtecan Treatment","Participants are eligible to be included in the study only if all of the following criteria apply:\n\n1. Is 18 years of age or of the legal adult age per local standard at the time of signing the informed consent.\n2. Has histologically confirmed HER2-positive breast cancer according to ASCO-CAP Guidelines as evaluated by a sponsor-designated central laboratory\n3. Participants with unresectable or metastatic HER2 positive breast cancer who have progressed on, or are intolerant to, previous T-DXd treatment.\n4. Must have received at least 2 lines of HER2-directed therapy for their metastatic disease.\n\n   1. Prior HER2-targeted neo-adjuvant or adjuvant therapy that resulted in relapse within 6 months of the completion of therapy will be considered a line of treatment for metastatic disease.\n   2. Based on the physician's choice, participants' eligibility, and institutional and local guidelines, participants may also have received post-T-DXd therapy, for example, a tucatinib-based regimen and\u002For T-DM1.\n   3. Participants must not have received more than 4 lines of HER2-directed therapy in the metastatic setting.\n5. Has measurable disease per RECIST version 1.1.\n6. Is eligible to receive one of the chemotherapy options listed in the physician's choice of chemotherapy (eribulin, gemcitabine, vinorelbine, or capecitabine).\n7. Participants with history of treated or clinically inactive CNS metastases are eligible as specified in the protocol.\n8. Has a life expectancy of at least 6 months, in the opinion of the investigator.\n9. Has adequate hematologic parameters as defined in the protocol.\n10. Has adequate hepatic function as specified in the protocol.\n11. Has creatinine clearance ≥ 50 mL\u002Fminute as calculated per local institutional guidelines.\n12. Has LVEF ≥ 50% as determined by either echocardiogram or MUGA obtained within 4 weeks before the first dose of study intervention.\n13. Has ECOG performance status of 0 or 1.\n14. Participant agrees to the following based on sex assigned at birth.\n\n    1. Male participants:\n\n       Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 7 months after the last dose of study intervention or the contraception period for the combination chemotherapy of choice per local guidance\u002Fstandard practice:\n       * Refrain from donating fresh unwashed semen.\n       * Use contraception as follows as specified in the protocol\n    2. Female participants:\n\n       * A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:\n\n         * Is a women of nonchildbearing potential OR\n         * Is a woman of childbearing potential (WOCBP) and using a contraceptive method that is highly effective (with a failure rate of \\\u003C 1% per year), with low user dependency during the study intervention period and for at least 7 months after the last dose of study intervention.\n       * A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within 3 days before the first dose of study intervention.\n       * Additional requirements for pregnancy testing during and after study intervention are provided in the protocol.\n       * The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.\n15. Is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in the protocol.\n\nParticipants are excluded from the study if any of the following criteria apply:\n\nMedical Conditions\n\n1. Has clinically confirmed leptomeningeal disease, in the opinion of the investigator.\n2. Has uncontrolled or significant cardiovascular disease.\n3. Has toxicity related to prior cancer therapy that has not resolved to ≤ Grade 1, with exceptions as stated in the protocol.\n4. Has uncontrolled infection requiring IV antibiotics, antivirals, or antifungals.\n5. Has an infection with HIV-1 or HIV-2, with the exception of participants with well-controlled HIV.\n6. Has active hepatitis B or C infection.\n7. Has an active SARS-CoV-2 infection. Participants with prior infection that has resolved per local institutions' requirements and screening guidance are eligible.\n8. Has a history of life-threatening hypersensitivity to monoclonal antibodies or to recombinant proteins or excipients in the drug formulation of zanidatamab.\n9. Is unable to receive trastuzumab treatment due to medical contraindications.\n10. Has any serious underlying medical or psychiatric condition that would impair the ability of the participant to receive or tolerate the planned treatment at the investigational site.\n11. Has any condition that would prevent treatment with the physician's choice of chemotherapy.\n12. Has any issue or condition that in the opinion of the investigator would contraindicate the participant's participation in the study or confound the results of the study.\n\n    Prior\u002FConcomitant Therapy\n13. Has a history of prior allogeneic bone marrow, stem cell, or solid organ transplantation.\n14. The washout periods for prior anticancer therapies before randomization as specified in the protocol.\n15. Has a history of trauma or major surgery within 4 weeks prior to randomization.\n\n    Other Exclusions\n16. Has a known hypersensitivity to any components of the study drugs, including chemotherapy.\n17. Female participants who are breastfeeding or pregnant, and female and male participants planning a pregnancy.",{"count":368,"type":21},550,[172],"The efficacy and safety of zanidatamab in combination with physician's choice of chemotherapy compared with trastuzumab in combination with physician's choice of chemotherapy will be evaluated for the treatment of participants with metastatic HER2-positive breast cancer who have progressed on, or are intolerant to, previous T-DXd treatment.",[372],"Metastatic HER2-positive Breast Cancer",[235,374,375],"Trastuzumab","Metastatic HER2-positive breast cancer","2026-02-06",{"date":378,"type":38},"2026-02-10",{"date":380,"type":38},"2024-08-13",{"date":382,"type":21},"2030-10-18",{"name":44,"class":45},166,{"id":386,"slug":387,"hasResults":11,"nctId":388,"briefTitle":389,"officialTitle":390,"acronym":4,"eligibilityCriteria":391,"healthyVolunteers":11,"sex":17,"minAge":392,"maxAge":393,"enrollmentInfo":394,"targetDuration":4,"studyType":22,"phases":396,"briefSummary":397,"conditions":398,"keywords":402,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":408,"lastUpdatePostDateStruct":409,"startDateStruct":410,"completionDateStruct":412,"leadSponsor":414,"locationsCount":272},"100495658","phase-1-study-of-lurbinectedin-monotherapy-in-pediatric-and-young-adult-participants-with-relapsedrefractory-ewing-sarcoma-100495658","NCT05734066","Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed\u002FRefractory Ewing Sarcoma","A Phase 1\u002F2, Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), Recommended Phase 2 Dose (RP2D), and Efficacy of Lurbinectedin Monotherapy in Pediatric Participants With Previously Treated Solid Tumors Followed by Expansion to Assess Efficacy and Safety in Pediatric and Young Adult Participants With Relapsed\u002FRefractory Ewing Sarcoma.","Key Inclusion Criteria:\n\nAge\n\n* Participant must meet the following age requirements at the time the informed consent form (ICF) (and assent form, if applicable) is signed:\n\n  * Phase 1 Part 1: participants must be ≥ 2 to \\\u003C 18 years of age.\n  * Phase 1 Part 2: participants must be ≥ 2 to ≤ 30 years of age.\n  * Phase 2: participants must be ≥ 2 to ≤ 30 years of age.\n\nType of Participant and Disease Characteristics\n\n* Participant has a confirmed solid tumor\n* The participant has a Lansky\u002FKarnofsky performance status score of ≥ 50%.\n* The participant has adequate liver function, evidenced by the following laboratory values:\n\n  * Aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤ 2.5 × upper limit of normal (ULN).\n  * Total bilirubin ≤ 1.5 × institutional ULN (with the exception of participants with Gilbert's syndrome who must have bilirubin \\\u003C 3 × institutional ULN).\n* The participant has adequate bone marrow function, evidenced by the following:\n\n  * Absolute neutrophil count (ANC) ≥ 1.0 × 10\\^9\u002FL (independent of growth factor support within 1 week of screening laboratories).\n* Platelets ≥ 100 × 10\\^9\u002FL (without platelet transfusion within previous 7 days of screening laboratories).\n\n  * Hemoglobin ≥ 8 g\u002FdL (note: may have been transfused).\n* The participant has an adequate renal function:\n\n  * Calculated creatinine clearance (use Cockcroft-Gault formula for participants ≥ 18 years; Schwartz equation for participants \\\u003C 18 years) ≥ 60 mL\u002Fmin.\n* The participant has an adequate cardiac function:\n\n  * Left ventricular ejection fraction or shortening fraction per institutional norm ≥ institutional lower level of normal.\n* The participant has creatine phosphokinase ≤ 2.5 × institutional ULN.\n\nWeight\n\n* The participant has body weight ≥ 15 kg.\n\nSex and Contraceptive\u002FBarrier Requirements\n\nMale participants:\n\nMale participants are eligible to participate if they agree to the following during the study intervention period and for at least 4 months after the last dose of study intervention:\n\n* Refrain from donating sperm.\n\nPLUS, either:\n\n* Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent.\n\nOR\n\n* Must agree to use contraception\u002Fbarrier as detailed below:\n\n  * Agree to use a male condom with female partner and use of an additional highly effective contraceptive method with a failure rate of \\\u003C 1% per year when having sexual intercourse with a Woman of childbearing potential (WOCBP) who is not currently pregnant.\n  * Note: male participants who are azoospermic (vasectomized or due to a medical cause) are still required to follow the protocol-specified contraception\u002Fbarrier criteria.\n\nFemale participants:\n\nA female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:\n\n* Is a Woman of nonchildbearing potential (WONCBP). OR\n* Is a WOCBP and using an acceptable contraceptive method during the study intervention period (at least 7 months after the last dose of study intervention). The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention.\n* A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within 7 days before the first dose of study intervention.\n\n  * If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.\n* Additional requirements for pregnancy testing during and after study intervention.\n* The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.\n\nInformed Consent\n\n* Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nKey Exclusion Criteria:\n\nMedical Conditions\n\n* corrected QT interval (QTc) prolongation defined as a QTc ≥ 460 ms using the Bazett formula in age \\\u003C 18 years and QTc ≥ 470 ms using the Bazett formula in age ≥ 18 years.\n* Known symptomatic Central nervous system (CNS) metastases requiring steroids. Participants with previously diagnosed CNS metastases are eligible if they have completed their treatment and have recovered from the acute effects of radiation therapy or surgery prior to enrollment, have discontinued high dose steroid treatment for these metastases for at least 2 weeks, and are neurologically stable (physiologic doses of steroids and short courses of steroids for other indications are acceptable).\n* Persisting toxicity related to prior therapy; however, alopecia, sensory neuropathy, hypothyroidism, and rash Grade ≤ 2 are acceptable, and other Grade ≤ 2 adverse events (AEs) not constituting a safety risk based on the investigator's judgement are acceptable.\n* An uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n* Any other major illness that, in the investigator's judgment, could substantially increase the risk associated with participation in this study.\n* Any other diseases, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the participant at high-risk for treatment complications.\n\nPrior\u002FConcomitant Therapy\n\n* Received prior treatment with lurbinectedin or trabectedin.\n* Received prior treatment with any investigational product within 4 weeks of first infusion of study intervention. Observational studies are permitted.\n* Received live or live attenuated vaccines within 4 weeks of the first dose of study treatment or plans to receive live vaccines during study participation. Administration of inactive vaccines or messenger ribonucleic acid (mRNA) vaccines (for example, inactivated influenza vaccines or COVID-19 vaccines) are allowed.\n* Had major surgery ≤ 4 weeks or radiation therapy ≤ 2 weeks prior to enrollment unless fully recovered. Prior palliative radiotherapy is permitted, provided it was completed at least 2 weeks prior to participant enrollment.\n* Received prior allogeneic bone marrow transplantation or solid organ transplant.\n* Received chemotherapy ≤ 3 weeks prior to start of study intervention.\n\nDiagnostic Assessments\n\n* Hepatitis B virus (HBV) or Hepatitis C virus (HCV) infection at screening (positive HBV surface antigen or Polymerase chain reaction (PCR) test for HCV RNA if HCV antibody test is positive).\n* Human immunodeficiency infection at screening (positive anti-HIV antibody).\n\nOther Exclusions\n\n* Has a known or suspected hypersensitivity to any of the components of the study intervention.\n* The participant or parent(s)\u002Fguardian(s) is\u002Fare unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator","2 Years","30 Years",{"count":395,"type":21},60,[114,24],"This study is conducted in two phases. The phase 1 portion of the study evaluates the safety, tolerability, pharmacokinetics (PK), recommended phase 2 dose (RP2D), and effectiveness of lurbinectedin monotherapy in pediatric participants with previously treated solid tumors. This is followed by the phase 2 portion, to further assess the effectiveness and safety in pediatric and young adult participants with recurrent\u002Frefractory Ewing sarcoma.",[399,400,401],"Refractory Ewing Sarcoma","Relapsed Ewing Sarcoma","Ewing Sarcoma",[403,404,405,406,407],"Solid Tumors","lurbinectedin","ewing's sarcoma","soft tissue sarcoma","sarcomas","2026-01-30",{"date":294,"type":38},{"date":411,"type":38},"2023-05-23",{"date":413,"type":21},"2028-04-20",{"name":44,"class":45},{"id":416,"slug":417,"hasResults":11,"nctId":418,"briefTitle":419,"officialTitle":420,"acronym":4,"eligibilityCriteria":421,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":422,"targetDuration":4,"studyType":22,"phases":424,"briefSummary":425,"conditions":426,"keywords":450,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":451,"lastUpdatePostDateStruct":452,"startDateStruct":454,"completionDateStruct":456,"leadSponsor":458,"locationsCount":192},"100404034","phase-1-phase-i-study-of-oral-onc206-in-recurrent-and-rare-primary-central-nervous-system-neoplasms-100404034","NCT04541082","Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms","A First-in-human Phase I Single-agent Dose-escalation, Food Effect and Dose Expansion Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms","Inclusion Criteria:\n\nPatients must meet all the following criteria to participate in the study:\n\n1. Patients aged ≥18 years with a recurrent, primary CNS neoplasm. For all cohorts, patients must have a histologically confirmed primary CNS neoplasm. Primary CNS neoplasms in this study include, but are not limited to, the following: glioblastoma and glioblastoma histologic subtypes, gliosarcoma, primary CNS sarcomas, anaplastic glial neoplasms including anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic mixed neuronal-glial tumors, and pilocytic astrocytoma with anaplastic features, diffuse astrocytoma, oligodendroglioma, gliomatosis cerebri, pleomorphic xanthoastrocytoma, anaplastic pleomorphic xanthoastrocytoma, diffuse midline gliomas and histone mutated gliomas (NOTE: Patients with H3 K27M-mutant diffuse gliomas are excluded unless the primary tumor is located in the pons or spinal cord, or the patient has completed front line radiation or received ONC201 therapy prior to 01 January 2023), ependymoma, anaplastic ependymoma, and all ependymoma subtypes, medulloblastoma and all medulloblastoma subtypes, atypical teratoid\u002Frhabdoid tumor, primary CNS embryonal\u002Fprimitive neuroectodermal tumors, atypical and anaplastic meningiomas, choroid plexus tumors, and pineal region tumors.\n2. Patients must have recurrent and measurable disease as defined by RANO criteria, using either the HGG and\u002For LGG RANO criteria based on tumor type, after having received established standard of care treatment for their disease and have no standard treatment options available as determined by the investigators. There is no limit on the number of total recurrences or prior therapies. However, prior therapies with known clinical benefit (including radiation) for specific tumor types are required. If patients are deemed ineligible for such therapies in the opinion of the Investigator, the Investigator must document the reason the patient is considered ineligible.\n3. Patients must have a Karnofsky Performance Score (KPS) of greater than or equal to 70. Patients with severe paraparesis\u002Fparaplegia who need minimal assistance for self-care due to their motor deficit but are otherwise functionally independent will be considered eligible.\n4. (Inclusion Criterion #4 was removed in Amendment 3.)\n5. Patients must not have received prior investigational or approved cytotoxic chemotherapy within 28 days prior to the first dose of study drug (Cycle 1, Day 1); 42 days in the case of nitrosoureas; 42 days in the case of bevacizumab; 28 days or 5 half-lives (whichever is less; but not less than 14 days) in case of investigational or approved molecularly targeted agent; 14 days in the case of radiotherapy.\n6. (Inclusion Criterion #6 was removed in Amendment 7.)\n7. Patients with AEs Grade ≥2 related to prior therapies (chemotherapy, radiotherapy, and\u002For surgery) must have all their AEs resolved prior to the first dose of study drug (Cycle 1, Day 1), except for alopecia or neuropathy; Grade 1 or 2 lymphopenia is allowed.\n8. Patients must not have undergone major surgery 4 weeks prior to the first dose of study drug (Cycle 1, Day 1) and must have completely recovered from any surgery (minor surgical procedures such as skin biopsies and port placement done on an outpatient basis do not require a waiting period).\n9. Patients must have normal organ and marrow function as defined below:\n\n   * Absolute neutrophil count (ANC) ≥1,500\u002FmcL.\n   * Platelets ≥100,000\u002FmcL.\n   * Hemoglobin ≥9.0 mg\u002FdL without transfusion in 2 prior weeks.\n   * Total bilirubin ≤1.5 × upper limit of normal (ULN) (patients with Gilbert's syndrome may be included with total bilirubin \\>1.5 × ULN if direct bilirubin is ≤1.5 × ULN).\n   * Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤2.5 × ULN.\n   * Measured or estimated creatinine clearance (CLcr) ≥40 mL\u002Fminute for patients with creatinine levels above normal. CLcr will be calculated by the Cockcroft-Gault equation for renal function.\n10. (Inclusion Criterion #10 was removed in Amendment 3)\n11. Patients must provide a tumor specimen (paraffin-embedded block and\u002For frozen tissue) from a prior resection or biopsy available that is sufficient to perform biomarker assays, ≥15 unstained slides for immunohistochemistry (IHC) analysis must be received by the NOB by the first dose of study drug (Cycle 1, Day 1). For patients with ≥10 to \\\u003C15 slides, eligibility will be reviewed on a case-by-case basis.\n12. Dependent upon dose level assignment and drug formulation (i.e., capsules versus powder in bottle \\[PIB\\]), patients must be able to either swallow oral capsules or swallow liquids.\n13. Patients must provide study-specific informed consent prior to enrollment. No Durable Power of Attorney or Next of Kin can provide initial consent.\n14. Patients must be able to tolerate a magnetic resonance imaging (MRI) study with intravenous gadolinium contrast.\n15. (Inclusion Criterion #15 was removed in Amendment 6)\n16. Patients must have a negative COVID-19 test within 72 hours of the first dose of study drug (Cycle 1, Day 1). Patients who had documented COVID-19 infection within 90 days of treatment but more than 20 days from infection do not need to be tested.\n17. (Inclusion Criterion #17 was removed in Amendment 6)\n\nExclusion Criteria:\n\nPatients who meet any of the following criteria will be excluded from the study:\n\n1. (Exclusion Criterion #1 was removed in Amendment 3)\n2. Patients with a history of allergic reactions attributed to compounds of similar chemical or biologic composition to ONC206 (e.g., ONC201) or its excipients.\n3. Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n4. Patients who are unable or unwilling to abide by the study protocol or cooperate fully with the Investigator.\n5. Patients with a known HIV-positive test on combination anti-retroviral therapy are ineligible for this initial first-in-human trial because of the potential for PK interactions with ONC206.\n6. Patients with active cardiac disease, including any of the following:\n\n   * Corrected QT interval (QTc) ≥470 msec on screening electrocardiogram (ECG; using the QTc by Fridericia's \\[QTcF\\] formula);\n   * Angina pectoris that requires the use of anti-anginal medication;\n   * Ventricular arrhythmias except for benign premature ventricular contractions;\n   * Supraventricular and nodal arrhythmias requiring a pacemaker or not controlled with medication;\n   * Conduction abnormality requiring a pacemaker;\n   * Valvular disease with documented compromise in cardiac function; and\u002For\n   * Symptomatic pericarditis.\n7. Patients with a history of cardiac dysfunction including any of the following:\n\n   * Myocardial infarction within the last 6 months, documented by persistent elevated cardiac enzymes or persistent regional wall abnormalities on assessment of left ventricular ejection fraction function;\n   * History of documented congestive heart failure (New York Heart Association functional classification III-IV); and\u002For\n   * Documented cardiomyopathy.\n8. Patients who have had an ischemic or hemorrhagic stroke in the last 3 months. If the patient has had a recent tumor resection, cerebral ischemic or hemorrhagic changes that occur peri operatively are not an exclusion.\n9. Patients with refractory epilepsy are excluded. Patients with primarily or secondarily generalized seizures in the 28 days prior to study enrollment will be excluded. Peri-operative seizures, defined as seizures occurring within the 7 days after a stereotactic biopsy, open biopsy, or surgical resection will not be an exclusion as long as the patient has had no generalized seizures starting 8 days after the surgical procedure. Patients with prior seizures must be on stable doses of 1 or 2 seizure medications for at least 14 days prior to study enrollment.\n10. Patients with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of ONC206 (uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).\n11. Patients who have been treated with any hematopoietic colony-stimulating growth factors (CSFs) (e.g., granulocyte-CSF, granulocyte-macrophage-CSF) ≤2 weeks prior to starting study drug. Erythropoietin or darbepoetin therapy, if initiated at least 2 weeks prior to enrollment, may be continued.\n12. Patients who are currently taking therapeutic doses of warfarin sodium or any other coumadin derivative anticoagulant.\n13. Patients who are taking strong inhibitors or inducers of cytochrome P450 (CYP) 3A4, 2D6, 1A2, 2C9, and 2C19 within at least 14 days prior to the first dose of study drug (Cycle 1, Day 1); these medications are excluded throughout the study.\n14. Women who are pregnant or breast feeding.\n15. Women of child-bearing potential with a positive serum pregnancy test ≤72 hours prior to the first dose of study drug (Cycle 1, Day 1).\n16. Patients who are receiving concomitant standard and\u002For investigational anti-cancer therapy.\n17. Patients with alcohol or substance abuse which, in the opinion of the Investigator, would interfere with compliance or safety.\n18. Patients with the presence of any other serious and\u002For unstable pre-existing medical disorder, psychiatric disorder, or other conditions that could interfere with patients' safety, obtaining informed consent or compliance to the study procedures as determined by the Investigators.\n19. Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, or men who do not agree to use highly effective contraception during treatment and for 16 additional weeks after the final dose of study drug.\n\n    Highly effective contraception is defined as either:\n    * True abstinence: When this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.\n    * Sterilization: Females must have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least 6 weeks ago. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.\n    * Male partner sterilization (with the appropriate post-vasectomy documentation of the absence of sperm in the ejaculate). For female patients on the study, the vasectomized male partner should be the sole partner for that patient.\n    * If patients are not practicing true abstinence and\u002For if the patient or sexual partner have not had a sterilization procedure as listed above, patients and their sexual partners must follow double barrier contraception in accordance with the guidelines for contraception below:\n\n      * Females of childbearing potential:\n\n        * Must use an intrauterine device or intrauterine system, during dosing of any study agent and for 16 weeks after final dose of study drug; or\n        * Must use a double barrier method of contraception: use of an occlusive cap (diaphragm or cervical\u002Fvault cap) with spermicide for women combined with use of a condom by their male partners capable of conceiving offspring.\n      * Males capable of conceiving offspring must use condoms during dosing of study agent and for an additional 16 weeks after final dose of study drug.\n\n    Note: Oral, implantable, or injectable contraceptives may be affected by CYP interactions, and are therefore not considered effective for this study.\n20. Previous receipt of ONC201, placebo, or blinded study drug from an ONC201 clinical study, or from any other source for H3 K27M-mutant diffuse glioma on or after 01 January 2023.",{"count":423,"type":21},102,[114],"The primary objective of this Phase 1, open-label, dose-escalation, and exploratory study is to evaluate the safety and tolerability profile (establish the maximum-tolerated dose) and evaluate the occurrence of dose-limiting toxicities (DLTs) following single weekly or multiple-day weekly dose regimens of single-agent, oral ONC206 in patients with recurrent, primary central nervous system (CNS) neoplasms.",[427,428,429,430,431,432,433,434,435,436,437,438,439,440,441,442,443,444,445,446,447,448,449],"Central Nervous System Neoplasms","Glioblastoma","Gliosarcoma, Adult","Anaplastic Oligodendroglioma","Anaplastic Astrocytoma","Pilocytic Astrocytoma","Oligodendroglioma","Gliomatosis Cerebri","Pleomorphic Xanthoastrocytoma","Anaplastic Pleomorphic Xanthoastrocytoma","Diffuse Midline Glioma, H3 K27M-Mutant","Ependymoma","Ependymoma, Anaplastic","Medulloblastoma","Teratoid Rhabdoid Tumor","Neuroectodermal Tumors, Primitive","Neuroectodermal Tumors","Anaplastic Meningioma","Atypical Meningioma","Choroid Plexus Neoplasms","Pineal Tumor","Diffuse Astrocytoma","Glial Tumor",[428,431,430,448,433],"2025-12-17",{"date":453,"type":38},"2025-12-18",{"date":455,"type":38},"2020-10-26",{"date":457,"type":21},"2026-12",{"name":44,"class":45},{"id":460,"slug":461,"hasResults":11,"nctId":462,"briefTitle":463,"officialTitle":464,"acronym":4,"eligibilityCriteria":465,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":466,"targetDuration":4,"studyType":22,"phases":468,"briefSummary":469,"conditions":470,"keywords":473,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":475,"lastUpdatePostDateStruct":476,"startDateStruct":478,"completionDateStruct":480,"leadSponsor":482,"locationsCount":483},"100524386","phase-1-jzp898-intravenous-infusion-as-monotherapy-and-combination-with-pembrolizumab-in-adults-with-advancedmetastatic-solid-tumors-100524386","NCT06108050","JZP898 Intravenous Infusion as Monotherapy and Combination With Pembrolizumab in Adults With Advanced\u002FMetastatic Solid Tumors","A Phase 1, First-in-human, Open-label, Multicenter Study of JZP898 as Monotherapy and in Combination With Pembrolizumab in Participants With Advanced or Metastatic Solid Tumors","Inclusion Criteria\n\n* Adult ≥ 18 years of age\n* Histological or cytological diagnosis of advanced or metastatic solid tumor.\n\n  1. Previously treated participants with solid tumors that are amenable to CPI therapy (eg. NSCLC, melanoma, HNSCC, RCC, HCC, gastroesophageal carcinomas, UC, or CRC \\[MSI-H\\]) for whom, in the opinion of the investigator, there is no SoC available to convey clinical benefit.\n  2. Parts A2 and B: previously-treated (≥ 1 line of prior anticancer therapy) participants with select tumor types (NSCLC, HNSCC, melanomas, RCC, and UC) who have progressed on\u002Fafter prior CPI therapy based on investigator assessment per RECIST version 1.1.\n* Participants in select tumor types:\n\n  1. NSCLC: eligible for platinum-based therapy and received platinum-based therapy prior to inclusion in the study.\n  2. HNSCC: eligible for platinum therapy and received platinum-based therapy prior to inclusion in this study.\n  3. Melanoma with known BRAFv600 mutation: received BRAF\u002FMEKi therapy before this study.\n* ECOG score of 0 to 1.\n* Measurable disease per RECIST version 1.1 criteria.\n* Parts A1 and A2 only: willing to consent to mandatory tumor biopsies (both pretreatment and post-treatment with JZP898) unless medically infeasible\n* Adequate organ and bone marrow function as indicated by the following laboratory values (within 4 weeks prior to starting the study interventions)\n* Men and women of reproductive potential to observe highly effective birth control for the duration of treatment and for 4 months following the last dose of study drug;\n* Additional criteria may apply\n\nExclusion Criteria\n\n* Unresolved toxicities from previous therapy that is \\> Grade 1.\n* Hypersensitivity to mAb, IFNα, or study intervention components.\n* Primary CNS tumor or symptomatic CNS metastases.\n* Have a second primary malignancy treated within the previous 2 years (exceptions: non-metastatic, non-melanomatous skin cancers, carcinoma in-situ, and melanoma in-situ).\n* Active autoimmune disease (in the last 2 years) requiring systemic steroids or immunosuppressive agents.\n* Active or history of pneumonitis (noninfectious) or interstitial lung disease that required steroids or has current pneumonitis\u002Finterstitial lung disease.\n* Any history of suicidal behavior or any suicidal ideation\n* Clinically significant ischemic\u002Fhemorrhagic cerebrovascular accident\u002Fstroke and\u002For clinically significant active cardiovascular disease\n* Received any anticancer therapy within 5 half-lives or 4 weeks (whichever is shorter) prior to the first dose of study drug\n* Received prior radiotherapy within 2 weeks of the first dose of study drug or have had a history of radiation pneumonitis\n* Major surgery within 2 weeks prior to the first dose of study intervention.\n* Participant is pregnant, breastfeeding, or expecting to conceive or father children within the projected duration of the study\n* Had a stem cell\u002Fsolid organ transplant.\n* Receipt of prior IFNα therapy",{"count":467,"type":21},177,[114],"This Phase 1 first-in-human study will investigate the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary antitumor activity of JZP898 monotherapy as well as JZP898 in combination with pembrolizumab in adult participants with advanced or metastatic solid tumors.",[471,472],"Advanced Solid Tumor","Metastatic Solid Tumor",[471,472,474],"JZP898","2025-12-08",{"date":477,"type":38},"2025-12-16",{"date":479,"type":38},"2023-11-07",{"date":481,"type":21},"2028-05-31",{"name":44,"class":45},10,{"id":485,"slug":486,"hasResults":11,"nctId":487,"briefTitle":488,"officialTitle":489,"acronym":4,"eligibilityCriteria":490,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":491,"enrollmentInfo":492,"targetDuration":494,"studyType":57,"phases":4,"briefSummary":495,"conditions":496,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":380,"lastUpdatePostDateStruct":497,"startDateStruct":499,"completionDateStruct":501,"leadSponsor":503,"locationsCount":504},"100431581","child-and-adolescent-registry-for-participants-with-narcolepsy-100431581","NCT04899947","Child and Adolescent Registry for Participants With Narcolepsy","Children, Adolescents and Their Providers: the Narcolepsy Assessment Partnership (CATNAPTM)","Inclusion Criteria:\n\n* Any child or adolescent with a physician-confirmed diagnosis of narcolepsy\n* Age less than 18 years\n* Willing to participate in the Registry and complete the informed consent form\n* Able to participate in English based registry\n\nExclusion Criteria:\n\n* Age 18 years or more\n* Fail to complete the informed consent form","17 Years",{"count":493,"type":21},500,"4 Years","CATNAP is a patient registry designed to improve the understanding of the natural history of narcolepsy in pediatric patients. Descriptive statistics on disease characteristics will be performed.\n\nThe study has 16 active clinical sites and a virtual site that widens participation to anywhere in the United States. For more information about the study or to access the Online Patient Enrollment System, visit the CATNAP website: https:\u002F\u002Fcatnap.healthie.net\u002Fwelcome or email catnap@pulseinfoframe.com. The Online Patient Enrollment System, CATNAP website, can also be found in the references section.",[175],{"date":498,"type":38},"2024-08-15",{"date":500,"type":38},"2020-10-10",{"date":502,"type":21},"2024-10-01",{"name":44,"class":45},25,""]