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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
UltraTrac Clinical Pilot StudyHemodynamic MonitoringNot yet recruitingNot stated2026-10-08NCT07864714
Blood-Brain Barrier Disruption Using Exablate Magnetic Resonance-Guided Low Intensity Focused Ultrasound With Microbubbles in Combination With T-Cell Infusion in Patients With Newly Diagnosed Diffuse Midline Glioma (LIFT)Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 12026-10-07NCT07796061
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingPhase 22026-10-06NCT03962543
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)Acid-Base Imbalance, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Cognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Ear Diseases, MELAS syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Tremor, acidosis disorder, anxiety disorder, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, cognitive disorder, dystonic disorder, eye disorder, headache disorder, hearing disorder, hearing loss disorder, hereditary disease, hypnic headache, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, lactic acidosis, metabolic disease, migraine disorder, mitochondrial encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, otorhinolaryngologic disease, psychiatric disorder, vascular disorder, vision disorder, visual epilepsyNot yet recruitingPhase 1 / Phase 22026-10-05NCT06792500
Study of Cabozantinib With Selumetinib for Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaRecruitingPhase 12026-10-02NCT06502171
Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain TumorsBCOR ITD sarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, central nervous system cancer, central nervous system disorder, central nervous system sarcoma, epithelial neoplasm, mesenchymal cell neoplasm, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorNot yet recruitingNot applicable2026-10-01NCT07851636
Study of Bictegravir/Lenacapavir in Children and Adolescents With HIV-1HIV-1-infectionActive, not recruitingPhase 2 / Phase 32026-09-29NCT06532656
Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual DiseaseMedulloblastoma, 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 tumorRecruitingPhase 1 / Phase 22026-09-28NCT07346157
A Phase 2 Study of WU-CART-007, an Anti-CD7 Allogeneic CAR-T Cell Therapy in T-Cell Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma (T-RRex)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, T-cell acute lymphoblastic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmRecruitingPhase 22026-09-28NCT06514794
A Prospective Analysis of Long-Term Clinical Outcomes and 3D Spine Growth in Anterior Vertebral Body TetheringScoliosis IdiopathicRecruitingNot applicable2026-09-28NCT04914507
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
Bowel Continence Across the Lifespan in People With Spina BifidaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fecal Incontinence, central nervous system malformation, colonic disorder, digestive system disorder, functional colonic disease, intestinal disorder, nervous system disorder, neurogenic bowel, rectal disorder, spina bifidaRecruitingNot stated2026-09-24NCT07390318
Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell TransplantationCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseActive, not recruitingPhase 22026-09-24NCT04018937
Impact of AI Care Summaries on Caregiver ComprehensionComprehension; Health Literacy; Patient SatisfactionRecruitingNot applicable2026-09-21NCT07831018
Massive Transfusion in Children-2: A Trial Examining Life Threatening Hemorrhage in ChildrenAccidental Injuries, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Shock, Shock, Hemorrhagic, injuryRecruitingPhase 32026-09-17NCT06070350
NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain TumorsNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, mixed neoplasm, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumor, rhabdoid tumorRecruitingPhase 12026-09-09NCT07664176
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 42026-09-09NCT05232929
Assessment of Worldwide Acute Kidney Injury Epidemiology in Neonates 2.0Chronic Disease, Disease Attributes, Edema, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Obstetric Labor Complications, Obstetric Labor, Premature, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Premature Birth, Signs and Symptoms, Urogenital Diseases, acute kidney injury, cardiovascular disorder, chronic renal failure syndrome, hypertensive disorder, kidney disorder, kidney failure, pregnancy disorder, urinary system disorder, vascular disorderEnrolling by invitationNot stated2026-09-04NCT06823115
Dabrafenib and Trametinib for BRAF V600 Mutant Low-Grade Gliomaslow grade gliomaRecruitingPhase 22026-09-03NCT07110246
Study of Lenacapavir as a Once-Yearly Injection for HIV Pre-exposure Prophylaxis (PrEP)HIV Pre-exposure ProphylaxisActive, not recruitingPhase 32026-08-28NCT07047716
Family Intervention for Black Teens With Type 1 DiabetesNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingNot applicable2026-08-27NCT06275412
Open-Label, Long-Term, Extension Study of Infigratinib in Children With HypochondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasiaEnrolling by invitationPhase 22026-08-26NCT07393373
Pre-pubertal Low Dose Transdermal Estradiol in Turner SyndromeAdnexal Diseases, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Male Urogenital Diseases, Sex Chromosome Disorders, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, disorder of sexual differentiation, endocrine system disorder, female reproductive system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, ovarian disorder, primary ovarian failure, sex chromosome disorder of sex developmentNot yet recruitingPhase 22026-08-25NCT07784582
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).Pediatric; Symptomatic Obstructive Hypertrophic CardiomyopathyActive, not recruitingPhase 2 / Phase 32026-08-18NCT06412666
Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Myosarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, connective tissue neoplasm, epithelial neoplasm, hereditary disease, hereditary neoplastic syndrome, kidney cancer, kidney disorder, mixed neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, rhabdomyosarcoma, sarcoma, urinary system disorderRecruitingPhase 12026-08-13NCT07172958