Great Ormond Street Hospital
Great Ormond Street Hospital
London, United Kingdom38 results · Page 1 of 2
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3 | Recruiting | Phase 3 | 2026-10-09 | NCT06998524 | |
| Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations | Digestive System Diseases, X-linked lymphoproliferative disease due to XIAP deficiency, digestive system disorder, enterocolitis, gastroenteritis, intestinal disorder | Active, not recruiting | Phase 2 | 2026-10-09 | NCT04641442 | |
| A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Phase 3 | 2026-10-06 | NCT07416604 | |
| EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety | Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, X-linked Alport syndrome, autosomal recessive Alport syndrome, connective tissue disorder, hereditary nephritis, kidney disorder, nephritis, urinary system disorder | Recruiting | Phase 2 | 2026-09-30 | NCT07523581 | |
| A Long-term Extension Study of Ustekinumab in Pediatric Participants | Crohn disease, Digestive System Diseases, Skin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, Spondylarthritis, Spondylarthropathies, arthritic joint disease, arthropathy, bone disorder, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, musculoskeletal system disorder, psoriasis, psoriatic arthritis, skin disorder, spondylitis, ulcerative colitis, vertebral column disorder | Active, not recruiting | Phase 3 | 2026-09-28 | NCT05092269 | |
| A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease | Crohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder | Recruiting | Phase 3 | 2026-09-25 | NCT05923073 | |
| A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension | lung disorder, pulmonary arterial hypertension, pulmonary hypertension, respiratory system disorder | Active, not recruiting | Phase 2 | 2026-09-25 | NCT03492177 | |
| A Safety Study of Ustekinumab in the Treatment of Pediatric Participants Aged 6 Years and Older With Moderate to Severe Plaque Psoriasis | Skin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, psoriasis, skin disorder | Active, not recruiting | Not stated | 2026-09-25 | NCT03218488 | |
| A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids) | 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, kidney disorder, kidney failure, urinary system disorder | Recruiting | Phase 3 | 2026-09-16 | NCT07107945 | |
| Targeted Pediatric High-Grade Glioma Therapy | Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, anaplastic astrocytoma, astrocytoma (excluding glioblastoma), brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, diffuse midline glioma, H3 K27M-mutant, epithelial neoplasm, glioblastoma, glioma, grade III glioma, malignant glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumor | Recruiting | Not stated | 2026-09-08 | NCT05839379 | |
| Study of Olutasidenib and Temozolomide in HGG | Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, anaplastic astrocytoma, astrocytoma (excluding glioblastoma), brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, diffuse midline glioma, H3 K27M-mutant, epithelial neoplasm, glioblastoma, glioma, grade III glioma, malignant glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, oligodendroglioma, primitive neuroectodermal tumor, spinal cord disorder, spinal cord neoplasm | Recruiting | Phase 2 | 2026-09-01 | NCT06161974 | |
| Obe-cel in Severe, Refractory Systemic Lupus Erythematosus (SLE) With Active Lupus Nephritis (LN) | 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 | 2026-08-24 | NCT07053800 | |
| DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers. | Adnexal Diseases, Blood Protein Disorders, Cancer of Gastrointestinal Tract, Digestive System Diseases, Digestive System Neoplasms, Erdheim-Chester disease, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Non-Melanoma Skin Neoplasms, Otorhinolaryngologic Neoplasms, Thoracic Neoplasms, Thyroid Cancer, Papillary, Urogenital Diseases, Urogenital Neoplasms, adenocarcinoma, bronchial neoplasm, bronchogenic carcinoma, cancer, carcinoma, cardiovascular disorder, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endocrine system disorder, epithelial neoplasm, female reproductive system disorder, female reproductive system neoplasm, glioma, gonadal disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, hemorrhagic disease, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, intestinal disorder, laryngeal disorder, laryngeal neoplasm, lung disorder, lung neoplasm, lymphatic system disorder, lymphoproliferative syndrome, malignant endocrine neoplasm, monoclonal gammopathy, neoplasm, neuroepithelial neoplasm, non-Langerhans cell histiocytosis, non-small cell lung carcinoma, otorhinolaryngologic disease, ovarian cancer, ovarian disorder, pancreas disorder, papillary adenocarcinoma, plasma cell neoplasm, primitive neuroectodermal tumor, rectal disorder, respiratory system disorder, respiratory tract neoplasm, thyroid gland disorder, thyroid gland undifferentiated (anaplastic) carcinoma, thyroid tumor, vascular disorder, vascular hemostatic disease | Recruiting | Phase 2 / Phase 3 | 2026-08-17 | NCT07440290 | |
| A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors | Neoplasms by Site, central nervous system cancer, neoplasm, nervous system disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-14 | NCT04774718 | |
| Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | 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 1 / Phase 2 | 2026-08-11 | NCT05524883 | |
| An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3 | Active, not recruiting | Not stated | 2026-08-10 | NCT06883240 | |
| Obe-cel in Adolescent [Applicable in UK Only] and Adult Severe, Refractory Systemic Lupus Erythematosus | Skin and Connective Tissue Diseases, autoimmune disease, connective tissue disorder, immune system disorder, systemic lupus erythematosus | Active, not recruiting | Phase 1 | 2026-07-31 | NCT06333483 | |
| Adalimumab Dose Adjustment for Pediatric Uveitis Treatment Trial | anterior uveitis, eye disorder, intermediate uveitis, panuveitis, uveal disorder, uveitis | Not yet recruiting | Phase 4 | 2026-07-13 | NCT07699107 | |
| European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors | childhood malignant neoplasm, neoplasm | Recruiting | Phase 1 / Phase 2 | 2026-07-10 | NCT02813135 | |
| A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | 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 | 2026-07-08 | NCT06138639 | |
| DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial) - Master Screening Protocol | Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoproliferative syndrome, neoplasm | Recruiting | Phase 2 / Phase 3 | 2026-07-01 | NCT05722886 | |
| An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorder | Enrolling by invitation | Phase 2 / Phase 3 | 2026-06-11 | NCT06075537 | |
| Study of Ribociclib and Everolimus in HGG and DIPG or Ribociclib and Temozolomide in DHG, H3G34-mutant | Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, anaplastic astrocytoma, astrocytoma (excluding glioblastoma), brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, diffuse midline glioma, H3 K27M-mutant, epithelial neoplasm, glioblastoma, glioma, grade III glioma, malignant glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumor | Recruiting | Phase 2 | 2026-05-29 | NCT05843253 | |
| A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT) | Cytomegalovirus (CMV) | Recruiting | Phase 3 | 2026-05-22 | NCT05319353 | |
| Paediatric Hepatic International Tumour Trial | Digestive System Diseases, Digestive System Neoplasms, Liver Neoplasms, Neoplasms by Histologic Type, Neoplasms by Site, adenocarcinoma, carcinoma, epithelial neoplasm, hepatoblastoma, hepatocellular carcinoma, liver disorder, mixed neoplasm, neoplasm | Active, not recruiting | Phase 3 | 2026-05-13 | NCT03017326 |