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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) EpendymomaPosterior Fossa Ependymal TumorRecruitingPhase 22026-10-06NCT07226453
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia BCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 32026-10-06NCT07080905
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseEnrolling by invitationPhase 22026-10-06NCT06867107
Study of Selinexor and Venetoclax in Combination With Chemotherapy in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid LeukemiaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute biphenotypic leukemia, acute myeloid leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, myeloid leukemia, neoplasmActive, not recruitingPhase 12026-10-06NCT04898894
Fimepinostat in Treating Brain Tumors in Children and Young AdultsNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, astrocytoma (excluding glioblastoma), brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioblastoma, glioma, medulloblastoma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingEarly phase 12026-10-05NCT03893487
Study to Learn More About the Safety and Effectiveness of the Drug VITRAKVI During Routine Use in Patients With TRK Fusion Cancer Which is Locally Advanced or Spread From the Place Where it Started to Other Places in the BodyLocally Advanced or Metastatic Solid Tumor Harboring an NTRK Gene FusionRecruitingNot stated2026-10-02NCT04142437
A Registry of Cases of Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-09-30NCT07805278
An Open-Label Intervention Trial to Reduce Senescence and Improve Frailty in Adult Survivors of Childhood CancerFrailty, Pathologic Processes, Pathological Conditions, Signs and Symptoms, childhood malignant neoplasm, neoplasmActive, not recruitingPhase 22026-09-30NCT04733534
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)Bone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderActive, not recruitingPhase 32026-09-25NCT05600426
Study of Bictegravir/Emtricitabine/Tenofovir Alafenamide Fixed Dose Combination in Adolescents and Children With Human Immunodeficiency Virus-1HIV-1 InfectionActive, not recruitingPhase 2 / Phase 32026-09-24NCT02881320
Cognitive Rehabilitation for Survivors of Childhood Medulloblastoma Using Real Time Hemodynamic NeurofeedbackMedulloblastoma, Childhood, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingNot applicable2026-09-23NCT07830134
Obesity Treatment With Phentermine-Topiramate ER in AYA Surviving Leukemia and LymphomaAcute Lymphoblastic Leukemia in Remission; Cancer Survivorship; Hodgkin Lymphoma in Remission; Non-Hodgkin Lymphoma in RemissionNot yet recruitingPhase 22026-09-23NCT07790913
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
Using Bioimpedance Analysis to Monitor Fluid Status in ChildrenEdema, Pathological Conditions, Signs and Symptoms, Signs and SymptomsNot yet recruitingNot stated2026-09-22NCT07834710
A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical ResectionNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, central nervous system cancer, central nervous system disorder, craniopharyngioma, neoplasm, nervous system disorder, primitive neuroectodermal tumorActive, not recruitingPhase 22026-09-22NCT02792582
Real World Asparaginase Therapy ToxicityChemically-Induced Disorders, Drug Toxicity, Drug-Related Side Effects and Adverse Reactions, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute biphenotypic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmNot yet recruitingNot stated2026-09-17NCT07008027
Imaging Skeletal Muscle Mitochondrial OXPHOS Activity In Acute Lymphoblastic Leukemia SurvivorsHemic and Lymphatic Diseases, Muscle Weakness, Neoplasms by Histologic Type, Neurologic Manifestations, Neuromuscular Manifestations, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Signs and Symptoms, childhood malignant neoplasm, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, muscle tissue disorder, musculoskeletal system disorder, neoplasm, nervous system disorderRecruitingNot applicable2026-09-17NCT06819475
A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid LeukemiaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 22026-09-17NCT05955261
Liver Biopsy Following Gene Therapy For HemophiliaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderNot yet recruitingNot stated2026-09-17NCT05932914
Muscle Aging Phenotypes in Childhood Cancer SurvivorsAtrophy, Muscle Weakness, Neurologic Manifestations, Neuromuscular Manifestations, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Sarcopenia, Signs and Symptoms, muscle tissue disorder, muscular atrophy, musculoskeletal system disorder, nervous system disorderRecruitingNot stated2026-09-16NCT07531498
Haploidentical HCT for Severe Aplastic AnemiaBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderRecruitingPhase 22026-09-16NCT04558736
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, graft versus host disease, hereditary disease, immune system disorder, inborn error of immunity, metabolic disease, severe combined immunodeficiencyActive, not recruitingPhase 1 / Phase 22026-09-16NCT03597594
A Prospective Trial of Dalbavancin-Based Prophylaxis in Children and Adolescents With High-Risk LeukemiaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 12026-09-15NCT06810583
Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene TherapyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, hematologic disorder, hereditary disease, inherited hemoglobinopathyRecruitingNot stated2026-09-15NCT06646640
Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed MedulloblastomaNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, brain disorder, central nervous system cancer, central nervous system disorder, epithelial neoplasm, glioma, medulloblastoma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-09-15NCT05535166