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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Metastatic Ewing's Trial Testing Schedule Enhancement to Improve OutcomesEwing sarcoma, Neoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, connective tissue neoplasm, neoplasm, osteosarcoma, sarcomaRecruitingPhase 12026-09-25NCT07194044
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
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMITCongenital, 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 diseaseRecruitingNot stated2026-09-24NCT06564974
Targeted Immunotherapy After Myeloablative TBI-Based Conditioning & AlloHCT in CAYA With High Risk T-Cell ALL & LymphomaHemic 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 12026-09-11NCT04972942
Pediatric Precision Laboratory Advanced Neuroblastoma TherapyNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-09-04NCT02559778
Eflornithine (DFMO) and Etoposide for Relapsed/Refractory NeuroblastomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 22026-09-03NCT04301843
Neuroblastoma Maintenance Therapy TrialNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 22026-09-03NCT02679144
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabAtrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscular atrophy, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIActive, not recruitingPhase 32026-09-01NCT05626855
Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 SystemFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingNot applicable2026-08-28NCT04065438
Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in ChildrenDisease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Urogenital Diseases, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingNot applicable2026-08-28NCT02235857
Observational Study of Pediatric Rheumatic Diseases: The CARRA RegistrySkin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, Spondylarthritis, Spondylarthropathies, arthritic joint disease, arthropathy, autoimmune disease, bone disorder, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, negative rheumatoid factor polyarthritis, psoriasis, psoriatic arthritis, rheumatic disorder, skin disorder, spondylitis, vertebral column disorderRecruitingNot stated2026-08-27NCT02418442
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular AtrophySpinal Curvatures, bone disorder, central nervous system disorder, motor neuron disorder, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, scoliosis, spinal cord disorder, spinal muscular atrophy, vertebral column disorderRecruitingNot applicable2026-08-19NCT05866419
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)Congenital, 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 diseaseActive, not recruitingPhase 2 / Phase 32026-07-21NCT05693142
ARDS in Children and ECMO Initiation Strategies Impact on Neurodevelopment (ASCEND)Respiration Disorders, adult acute respiratory distress syndrome, lung disorder, respiratory system disorderActive, not recruitingNot stated2026-07-14NCT05388708
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young ChildrenCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2026-07-08NCT04509050
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)Bone Marrow Failure Disorders, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, anemia, aplastic anemia, autoimmune disease, bone marrow disorder, chronic myeloid leukemia, disease, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, inborn errors of metabolism, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphomaRecruitingNot stated2026-07-06NCT01351545
HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal TumorsNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 42026-06-03NCT02875314
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant DisordersBone Marrow Failure Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, bone marrow disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, immune system disorder, inherited hemoglobinopathy, metabolic disease, sickle cell diseaseRecruitingPhase 1 / Phase 22026-05-29NCT03128996
Safety Study of Unlicensed IND Cord Blood Units Manufactured by the National Cord Blood Program for Unrelated TransplantationInfusion ReactionsRecruitingPhase 22026-04-13NCT01656603
PV Loop & Coarctation StudyCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, aorta coarctation, cardiovascular disorder, congenital heart disease, heart disorderActive, not recruitingNot stated2026-04-08NCT05362721
Adult Congenital Heart Disease International EValuation of the Effectiveness of SGLT2i RegistryCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, congenitally corrected transposition of the great arteries, heart disorder, heart failure, tetralogy of fallot, transposition of the great arteries, univentricular heartRecruitingNot stated2026-02-13NCT06932081
Transforming Care for Individuals With Childhood-onset Systemic Lupus ErythematosusFatigue, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin and Connective Tissue Diseases, connective tissue disorder, depressive disorder, musculoskeletal system disorder, rheumatic disorderRecruitingNot applicable2025-10-30NCT06232304
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical OutcomesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingNot stated2025-10-10NCT03350828