Children's Hospital of Philadelphia
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States434 results · Page 1 of 18
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 2 | 2026-10-09 | NCT06447025 | |
| Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia | Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence | Recruiting | Phase 1 | 2026-10-09 | NCT06376162 | |
| A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL) | B-cell neoplasm, Burkitt lymphoma, DNA Virus Infections, Disease Attributes, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence, Tumor Virus Infections, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, neoplasm, viral infectious disease | Recruiting | Phase 1 | 2026-10-09 | NCT06173518 | |
| A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab | Lugano Classification Limited Stage Hodgkin Lymphoma AJCC v8 | Recruiting | Phase 3 | 2026-10-09 | NCT05675410 | |
| A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorder | Active, not recruiting | Phase 3 | 2026-10-09 | NCT05444257 | |
| An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorder, visual epilepsy | Recruiting | Phase 3 | 2026-10-09 | NCT05066217 | |
| Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, 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, ependymoma, epithelial neoplasm, glioma, hepatoblastoma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, kidney cancer, kidney disorder, lung disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, medulloblastoma, mixed neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, respiratory system disorder, rhabdoid tumor, rhabdomyosarcoma, sarcoma, urinary system disorder | Active, not recruiting | Phase 2 | 2026-10-09 | NCT04320888 | |
| A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients | High-Risk Neuroblastoma | Recruiting | Phase 2 / Phase 3 | 2026-10-08 | NCT07549321 | |
| Physical Activity in CKD | Chronic Disease, Chronic Kidney Diseases, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, chronic renal failure syndrome, kidney disorder, kidney failure, urinary system disorder | Recruiting | Not applicable | 2026-10-08 | NCT06624358 | |
| ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Rett syndrome, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06430385 | |
| Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia | B-cell neoplasm, Burkitt lymphoma, DNA Virus Infections, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Tumor Virus Infections, acute biphenotypic leukemia, acute leukemia of ambiguous lineage, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, neoplasm, viral infectious disease | Recruiting | Phase 2 | 2026-10-08 | NCT06317662 | |
| Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder | Recruiting | Phase 3 | 2026-10-08 | NCT06247085 | |
| Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US) | Abnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder, progressive familial intrahepatic cholestasis type 1 | Recruiting | Not stated | 2026-10-08 | NCT06193928 | |
| Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma | Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, ganglioneuroblastoma, neoplasm, neuroblastoma, neuroepithelial neoplasm, nodular ganglioneuroblastoma, primitive neuroectodermal tumor | Recruiting | Phase 3 | 2026-10-08 | NCT06172296 | |
| A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL) | B-cell neoplasm, Burkitt lymphoma, DNA Virus Infections, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Tumor Virus Infections, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, viral infectious disease | Recruiting | Phase 2 | 2026-10-08 | NCT06124157 | |
| A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC) | Head and Neck Neoplasms, Neoplasms by Histologic Type, Neoplasms by Site, Otorhinolaryngologic Neoplasms, Pharyngeal Diseases, Stomatognathic Diseases, carcinoma, epithelial neoplasm, nasopharyngeal carcinoma, nasopharyngeal disorder, nasopharyngeal neoplasm, neoplasm, otorhinolaryngologic disease | Recruiting | Phase 2 | 2026-10-08 | NCT06064097 | |
| A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis | Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorder | Recruiting | Phase 2 | 2026-10-08 | NCT05828069 | |
| A Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG) | Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, epithelial neoplasm, glioma, malignant glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT05099003 | |
| AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, 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 neurofibroma | Active, not recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT01362803 | |
| Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Juvenile Macular Degeneration, Stargardt Disease 1, Stargardt disease, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-07 | NCT06467344 | |
| Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Undiagnosed Diseases, hereditary disease | Recruiting | Not stated | 2026-10-07 | NCT02450851 | |
| PEEL-224, Vincristine and Temozolomide in Pediatric Solid Tumors | Disease Attributes, Myosarcoma, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, epithelial neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumor, rhabdomyosarcoma, sarcoma | Recruiting | Phase 1 / Phase 2 | 2026-10-06 | NCT06721689 | |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Danon disease, Hypertrophy, Hypertrophy, Left Ventricular, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, X-linked disease, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, disorder of glycogen metabolism, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, inborn carbohydrate metabolic disorder, inborn errors of metabolism, intellectual disability, lysosomal storage disease, metabolic disease, nervous system disorder, subvalvular aortic stenosis | Recruiting | Phase 2 | 2026-10-06 | NCT06092034 | |
| A Study of the Drug Letermovir as Prevention of Cytomegalovirus Infection After Stem Cell Transplant in Pediatric Patients | Hemic and Lymphatic Diseases, Neoplasms by Site, hematologic disorder, hematopoietic and lymphoid cell neoplasm, hematopoietic and lymphoid system neoplasm, neoplasm | Recruiting | Phase 3 | 2026-10-06 | NCT05711667 | |
| Veliparib, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600 Mutations | Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, anaplastic astrocytoma, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioblastoma, glioma, malignant glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor | Active, not recruiting | Phase 2 | 2026-10-06 | NCT03581292 |