[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"xCures\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":116},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,50,91],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":17,"phases":4,"briefSummary":18,"conditions":19,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":46,"locationsCount":49},"100405976","expanded-access-to-ulixertinib-bvd-523-in-patients-with-advanced-mapk-pathway-altered-malignancies-100405976",false,"NCT04566393","Expanded Access to Ulixertinib (BVD-523) in Patients With Advanced MAPK Pathway-Altered Malignancies","Inclusion Criteria:\n\n* Main Inclusion Criterion:\n\n  1\\. Patient has a MAPK pathway-altered solid tumor(s), including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations.\n* Other Inclusion Criteria:\n\n  1. In the opinion of the treating physician, the patient has exhausted or has inadequate response to available anti-cancer treatments.\n  2. In the opinion of the treating physician, the patient has adequate organ function to tolerate ulixertinib as defined in section 6.1\n  3. Male or female patients aged ≥ 12 years.\n  4. Patient must be able to swallow and retain orally administered medication.\n\n     Note: Ulixertinib is primarily absorbed in the duodenum and therefore patients with any prior stomach or duodenal resection should be evaluated with that understanding.\n  5. For females, evidence of post-menopausal status or negative urinary or serum pregnancy test for pre-menopausal patients.\n  6. Highly effective contraception for both male and female patients throughout the treatment and for at least 4 months after last treatment administration. In patients under the age of 18, who are not sexually active, abstinence is an acceptable form.\n  7. Toxicities related to any prior treatments are either stable, stable on supportive therapy, resolved, or in the opinion of the treating physician, clinically non-significant\n  8. Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the patient's age.\n\nExclusion Criteria:\n\n1. Patient is already participating in or qualifies for and is able to enroll in a clinical trial of ulixertinib (BVD-523).\n2. Patient has received systemic therapy with an investigational agent within 5 half-lives or 14 days prior to starting ulixertinib treatment, whichever is shorter.\n3. Patient has received radiotherapy within 14 days prior to the first dose of ulixertinib treatment other than for the allowable treatment of symptomatic bone metastasis.\n4. A history of current evidence\u002Frisk of retinal vein occlusion (RVO) or central serous retinopathy (CSR)\n5. Current evidence of uncontrolled, significant intercurrent illness that would, in the treating physician's judgment, contraindicate the patient's treatment with ulixertinib due to safety concerns.\n6. Patients who, in the opinion of the treating physician, have not fully recovered from recent major surgery to a sufficient extent to tolerate treatment with ulixertinib.\n7. Known hypersensitivity to ulixertinib or any component in its formulation.\n8. Patients taking prohibited medications as described in current Investigator's Brochure.\n\n   Note: Patients who require treatment with Drugs that are strong inhibitors or inducers of CYP1A2, CYP2D6, and CYP3A4 (see Appendix 3) were excluded from the FIH study of ulixertinib and should be discussed with xCures to review if any potential benefits outweigh the potential risks.\n9. Patient is actively breastfeeding.\n10. Prior stomach or duodenal resection that in the opinion of the treating physician would affect the breakdown and absorption of ulixertinib.","ALL","12 Years","EXPANDED_ACCESS","The objective of this expanded access program is to provide ulixertinib (BVD-523) for compassionate use in advanced cancer patients with MAPK pathway-altered solid tumor(s), including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations who have incomplete response to or have exhausted available therapies.\n\nUlixertinib is available for treatment as monotherapy or in combination with other clinically tolerable agent(s), conditionally approved by the drug manufacturer.",[20,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40],"Pancreatic Cancer","Small Bowel Cancer","Colorectal Cancer","Melanoma","Non Small Cell Lung Cancer","Thyroid Cancer","Bladder Cancer","Head and Neck Cancer","Gastric Cancer","Esophageal Cancer","Cholangiocarcinoma","Ovarian Cancer","Hepatocellular Carcinoma","Glioblastoma","MAPK Gene Mutation","KRAS Activating Mutation","BRAF Gene Mutation","NRAS Gene Mutation","HRAS Gene Mutation","MEK Mutation","ERK Mutation","AVAILABLE","2026-06-02",{"date":44,"type":45},"2026-06-04","ACTUAL",{"name":47,"class":48},"xCures","INDUSTRY",26,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":56,"targetDuration":4,"studyType":59,"phases":4,"briefSummary":60,"conditions":61,"keywords":79,"overallStatus":81,"whyStopped":4,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":85,"completionDateStruct":87,"leadSponsor":89,"locationsCount":90},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":57,"type":58},1000,"ESTIMATED","OBSERVATIONAL","FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78],"Alpha-Thalassemia","Beta-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Early-Onset Alzheimer Disease","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","GM1 Gangliosidosis","Myasthenia Gravis","Pompe Disease","Sickle Cell Disease","Transthyretin Amyloid Cardiomyopathy","Rare Diseases",[80],"rare diseases","RECRUITING","2024-11-12",{"date":84,"type":45},"2024-11-14",{"date":86,"type":45},"2024-06-10",{"date":88,"type":58},"2026-06-10",{"name":47,"class":48},1,{"id":92,"slug":93,"hasResults":11,"nctId":94,"briefTitle":95,"officialTitle":96,"acronym":4,"eligibilityCriteria":97,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":98,"targetDuration":4,"studyType":59,"phases":4,"briefSummary":100,"conditions":101,"keywords":105,"overallStatus":81,"whyStopped":4,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":111,"completionDateStruct":113,"leadSponsor":115,"locationsCount":90},"100526897","xpedite-a-study-to-expedite-dipg-and-dmg-research-100526897","NCT06140719","xPedite: A Study to Expedite DIPG and DMG Research","A Retrospective and Prospective Observational Sub-Study for Diffuse Midline Glioma and Diffuse Intrinsic Pontine Glioma","Inclusion Criteria:\n\n1. Diagnosis of diffuse midline glioma according to the WHO 2021 Classification of Tumors of the Central Nervous System diagnostic criteria including diffuse intrinsic pontine glioma (DIPG). In the absence of a pathologically confirmed diagnosis, a grade IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord, or with a pontine epicenter and diffuse involvement of the pons.\n2. Patients with any performance status, comorbidity or disease severity are eligible\n3. Patients or their legally-authorized representative must be willing and able to provide electronic, informed consent (and assent, if applicable)\n4. Informed consent obtained for the XCELSIOR longitudinal outcomes registry (NCT03793088).\n5. Patients must be a resident of or receiving care within the United States or US territories.\n\nExclusion Criteria:\n\n1. Patient or legally-authorized representative is unable to provide informed consent.\n2. Patient or caregiver is unable to complete the PRO and ClinRO by an electronic platform.",{"count":99,"type":58},400,"This study will gather data from new and existing patients with patient medical records, and patient\u002Ffamily\u002Fcaregiver reported information to establish a clear natural history of disease suitable to serve as an external, contemporary or historical control arm for future therapeutic development programs of drugs, devices, or biologic interventions in DMG or DIPG.",[102,103,104],"Oncology","DIPG","DMG",[103,104,106,107],"Diffuse Midline Glioma","Diffuse Intrinsic Pontine Glioma","2024-04-29",{"date":110,"type":45},"2024-04-30",{"date":112,"type":45},"2023-11-01",{"date":114,"type":58},"2027-01",{"name":47,"class":48},""]