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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pyruvate Kinase Deficiency Global Longitudinal RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, congenital nonspherocytic hemolytic anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, pyruvate kinase deficiency of red cellsActive, not recruitingNot stated2026-09-28NCT03481738
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)acute leukemiaRecruitingPhase 1 / Phase 22026-09-25NCT04811560
A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 AlterationsGranuloma, Hemic and Lymphatic Diseases, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, T-cell non-Hodgkin lymphoma, anaplastic large cell lymphoma, astrocytoma (excluding glioblastoma), connective tissue neoplasm, epithelial neoplasm, glioblastoma, glioma, immune system disorder, immunoproliferative disorder, inflammatory myofibroblastic tumor, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor, sarcoma, solitary fibrous tumorRecruitingPhase 1 / Phase 22026-09-25NCT04094610
A Surveillance Study of Susceptibility to Baloxavir Marboxil in Pediatric Participants With Influenza and Transmission of Influenza to Household ContactsInfections, RNA Virus Infections, influenza, respiratory system disorder, respiratory tract infectious disorder, viral infectious diseaseRecruitingPhase 32026-09-24NCT06094010
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment OptionsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Head and Neck Neoplasms, Male Urogenital Diseases, Mouth Neoplasms, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Stomatognathic Diseases, Thyroid Cancer, Papillary, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, adenocarcinoma, benign urinary system neoplasm, brain disorder, carcinoma, central nervous system cancer, central nervous system disorder, connective tissue neoplasm, endocrine system disorder, epithelial neoplasm, glioma, hereditary disease, hereditary neoplastic syndrome, kidney cancer, kidney disorder, malignant endocrine neoplasm, medulloblastoma, mesoblastic nephroma, mixed neoplasm, mouth disorder, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, papillary adenocarcinoma, primitive neuroectodermal tumor, salivary gland cancer, salivary gland disorder, sarcoma, thyroid gland disorder, thyroid tumor, urinary system disorderActive, not recruitingPhase 1 / Phase 22026-09-23NCT02650401
Long-Term Follow-up Protocol for Participants Treated With Gene-Modified T CellsneoplasmRecruitingPhase 2 / Phase 32026-09-18NCT03435796
Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS TumorsDisease Attributes, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, ependymoma, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-09-10NCT01356290
Tumor-agnostic Precision Immuno-oncology and Somatic Targeting Rational for You (TAPISTRY) Platform StudySolid TumorsActive, not recruitingPhase 22026-09-04NCT04589845
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS TumorsNeoplasms by Site, central nervous system cancer, neoplasm, nervous system disorderActive, not recruitingPhase 1 / Phase 22026-08-14NCT04774718
SPECTA: Screening Cancer Patients for Efficient Clinical Trial AccessneoplasmRecruitingNot stated2026-06-24NCT02834884
Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the BloodDisease Attributes, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmActive, not recruitingPhase 1 / Phase 22026-04-01NCT03793478
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, methylmalonic acidemia, propionic acidemiaRecruitingNot stated2024-02-05NCT04176523