Great Ormond Street Hospital for Children
Great Ormond Street Hospital for Children
London, United Kingdom24 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric Participants | Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Diseases, autoimmune disease, connective tissue disorder, glomerulonephritis, immune system disorder, kidney disorder, lupus nephritis, nephritis, systemic lupus erythematosus, urinary system disorder | Recruiting | Phase 2 | 2026-10-06 | NCT05039619 | |
| A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy | Duchene Muscular Dystrophy | Recruiting | Phase 2 / Phase 3 | 2026-10-02 | NCT07856329 | |
| A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 4 | 2026-10-02 | NCT05861986 | |
| A Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) | CNS demyelinating autoimmune disease, acute transverse myelitis, autoimmune disease, autoimmune disorder of the nervous system, cranial nerve neuropathy, demyelinating disease, eye disorder, immune system disorder, nervous system disorder, neuromyelitis optica, optic nerve disorder, optic neuritis | Recruiting | Phase 3 | 2026-10-01 | NCT05199688 | |
| Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients | PMM2-congenital disorder of glycosylation | Enrolling by invitation | Phase 2 | 2026-09-23 | NCT06657859 | |
| A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB) | Mucopolysaccharidosis IIIB | Recruiting | Phase 1 / Phase 2 | 2026-09-21 | NCT07640984 | |
| Efficacy and Safety of Erenumab in Pediatric Participants With Episodic Migraine | Headache, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, headache disorder, hypnic headache, migraine disorder, nervous system disorder | Active, not recruiting | Phase 3 | 2026-09-21 | NCT03836040 | |
| A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 4 | 2026-09-10 | NCT05861999 | |
| Long-term Extension of GTX-102 in Angelman Syndrome | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 3 | 2026-09-09 | NCT06415344 | |
| A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four 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 disease | Active, not recruiting | Phase 2 | 2026-09-09 | NCT06128564 | |
| A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome | Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorder | Recruiting | Phase 3 | 2026-08-24 | NCT06872125 | |
| A Phase 3 Study of Etelcalcetide in Children With Secondary Hyperparathyroidism Receiving Hemodialysis | Chronic Disease, 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, endocrine system disorder, hyperparathyroidism, kidney disorder, kidney failure, parathyroid gland disorder, secondary hyperparathyroidism, urinary system disorder | Recruiting | Phase 3 | 2026-08-21 | NCT03969329 | |
| Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Recruiting | Not stated | 2026-08-17 | NCT06605378 | |
| A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 | 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 disease | Recruiting | Phase 1 / Phase 2 | 2026-08-13 | NCT07038824 | |
| A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 | 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 disease | Recruiting | Phase 1 / Phase 2 | 2026-08-13 | NCT07037862 | |
| OLE Study With Filgotinib in JIA | Skin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, rheumatic disorder | Recruiting | Phase 3 | 2026-08-10 | NCT07553182 | |
| ARDS in Children and ECMO Initiation Strategies Impact on Neurodevelopment (ASCEND) | Respiration Disorders, adult acute respiratory distress syndrome, lung disorder, respiratory system disorder | Active, not recruiting | Not stated | 2026-07-14 | NCT05388708 | |
| Registry for Patients With X-Linked Hypophosphatemia | Bone Diseases, Metabolic, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypophosphatemia, Familial, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Rickets, X-Linked Hypophosphatemic, Urogenital Diseases, X-linked hypophosphatemic rickets, bone disorder, calcium metabolic disease, hereditary disease, hypophosphatemia, hypophosphatemic rickets, inborn errors of metabolism, inborn metal metabolism disorder, kidney disorder, metabolic disease, musculoskeletal system disorder, nutritional deficiency disease, nutritional disorder, phosphorus metabolism disease, renal tubular transport disease, rickets, urinary system disorder, vitamin D deficiency, vitamin deficiency disorder | Recruiting | Not stated | 2026-07-09 | NCT03193476 | |
| Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE) | 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 disease | Not yet recruiting | Phase 2 | 2026-07-02 | NCT07682129 | |
| A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Froelich syndrome, Male Urogenital Diseases, Urogenital Abnormalities, Urogenital Diseases, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, hypogonadism | Enrolling by invitation | Phase 2 | 2026-02-10 | NCT07156578 | |
| Thalamic Recordings in Children Undergoing SEEG | brain disorder, central nervous system disorder, epilepsy, focal epilepsy, nervous system disorder | Recruiting | Not applicable | 2026-02-02 | NCT06453759 | |
| Gene Therapy Study for Children With CLN5 Batten Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Phase 1 / Phase 2 | 2024-08-12 | NCT05228145 | |
| Micro-dosing Indocyanine Green (ICG) in Children | Digestive System Diseases, Urogenital Diseases | Recruiting | Not applicable | 2024-05-20 | NCT06421103 | |
| Simple Bone Cysts in Kids | Cysts, bone disorder, musculoskeletal system disorder, neoplasm, solitary bone cyst | Recruiting | Phase 3 | 2023-07-12 | NCT02193841 |