Great Ormond Street Hospital
Great Ormond Street Hospital
London, United Kingdom15 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Hyperammonemia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, liver disorder, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorder | Active, not recruiting | Phase 3 | 2026-09-30 | NCT06255782 | |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 / Phase 3 | 2026-09-24 | NCT07441876 | |
| Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Hyperammonemia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, liver disorder, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorder | Recruiting | Not stated | 2026-08-31 | NCT06805695 | |
| COMparison Between Anakinra and Tocilizumab in NORSE - "COMBAT-NORSE" | Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Status Epilepticus, febrile infection-related epilepsy syndrome, nervous system disorder, new-onset refractory status epilepticus, visual epilepsy | Not yet recruiting | Phase 3 | 2026-08-19 | NCT07281027 | |
| Development and Validation of a New Paediatric Inflammatory Bowel Disease NUTrition Risk Score (PIBD-NUTS) | Crohn disease, Digestive System Diseases, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, ulcerative colitis | Active, not recruiting | Not stated | 2026-08-11 | NCT06723418 | |
| CAR T Cells to Target GD2 for DMG | diffuse midline glioma, H3 K27M-mutant | Recruiting | Phase 1 | 2026-08-11 | NCT05544526 | |
| Multi-modular Chimeric Antigen Receptor T Cells tarGeting B7-H3 in Children, Teenage & Young Adult Sarcoma | Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, neoplasm, sarcoma | Recruiting | Phase 1 | 2026-08-07 | NCT07751380 | |
| Determining the Mechanisms of Loss of CAR T Cell Persistence | Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasm | Recruiting | Not stated | 2026-08-07 | NCT05809284 | |
| Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, GM1 gangliosidosis, Gangliosidoses, Gangliosidosis, Generalized GM1, Type 1, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, beta-Galactosidase Deficiency, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Active, not recruiting | Phase 1 / Phase 2 | 2026-06-30 | NCT04713475 | |
| CARPALL: Immunotherapy With CD19+CD22 CAR T-cells for CD19+ and CD22+ Acute Lymphoblastic Leukaemia | Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasm | Recruiting | Phase 1 | 2026-05-22 | NCT02443831 | |
| Multi-modular Chimeric Antigen Receptor Targeting GD2 in Neuroblastoma | Neoplasms 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 tumor | Active, not recruiting | Phase 1 | 2026-04-24 | NCT05990751 | |
| Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions | Pediatric SDLT CNS Disorders | Active, not recruiting | Phase 1 / Phase 2 | 2026-03-17 | NCT07410143 | |
| CiproPAL (Ciprofloxacin Prophylaxis in Acute Leukaemia) | Acute Lymphoblastic Leukaemia - Category | Active, not recruiting | Not applicable | 2026-03-05 | NCT04678869 | |
| A Multi-Arm, Platform Trial For Relapsed Neuroblastoma | Neoplasms 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 tumor | Recruiting | Phase 1 / Phase 2 | 2026-01-12 | NCT07334301 | |
| Gaucherite - A Study to Stratify Gaucher Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gaucher Disease, Type 1, Gaucher Disease, Type 3, Gaucher disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Recruiting | Not stated | 2024-08-06 | NCT03240653 |