Royal Manchester Children's Hospital
Royal Manchester Children's Hospital
Manchester, United Kingdom32 results · Page 1 of 2
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day | Short Stature Children Born Small for Gestational Age (SGA) | Active, not recruiting | Phase 2 | 2026-10-06 | NCT03878446 | |
| 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 | |
| The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, arterial calcification of infancy, autosomal recessive hypophosphatemic rickets, autosomal recessive inherited pseudoxanthoma elasticum, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hypophosphatemic rickets, autosomal recessive, 1, skin disorder, vascular disorder, vascular hemostatic disease | Active, not recruiting | Phase 1 | 2026-09-29 | NCT05734196 | |
| A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 3 | 2026-09-28 | NCT07082725 | |
| 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 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 3 | 2026-09-24 | NCT07054515 | |
| A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Phase 3 | 2026-09-23 | NCT05611801 | |
| Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study | Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Urogenital Diseases, glomerulonephritis, kidney disorder, nephritis, urinary system disorder | Recruiting | Phase 4 | 2026-09-09 | NCT07746895 | |
| Phase 1 Study of INBRX-109 in Subjects With Locally Advanced or Metastatic Solid Tumors Including Sarcomas | Ewing sarcoma, Neoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, colorectal adenocarcinoma, connective tissue neoplasm, neoplasm, osteosarcoma, sarcoma | Recruiting | Phase 1 | 2026-09-08 | NCT03715933 | |
| 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 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 | |
| A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults With Cancer | neoplasm | Recruiting | Phase 2 | 2026-08-07 | NCT05999994 | |
| 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 | |
| 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 | |
| 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 | |
| A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Musculoskeletal Abnormalities, Noonan syndrome, Sex Chromosome Disorders, Skin and Connective Tissue Diseases, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, connective tissue disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, musculoskeletal system disorder, sex chromosome disorder of sex development | Active, not recruiting | Phase 3 | 2026-06-12 | NCT05330325 | |
| A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Enrolling by invitation | Not stated | 2026-06-12 | NCT03745924 | |
| 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 | |
| ADH1 and ADH2 Disease Monitoring Study (DMS) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypercalciuria, Hypercalciuric Hypocalcemia, Familial, Hypocalcemia, Male Urogenital Diseases, Musculoskeletal Abnormalities, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urogenital Diseases, Urological Manifestations, Water-Electrolyte Imbalance, autosomal dominant hypocalcemia, calcinosis, calcium metabolic disease, endocrine system disorder, hypoparathyroidism, kidney disorder, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nephrocalcinosis, nephrolithiasis, nervous system disorder, neuromuscular disease, parathyroid gland disorder, urinary system disorder, urolithiasis | Active, not recruiting | Not stated | 2026-04-30 | NCT05227287 | |
| Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1) | Autosomal Dominant Hypocalcemia Type 1 (ADH1) | Recruiting | Phase 2 / Phase 3 | 2026-04-22 | NCT07080385 | |
| A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 3 | 2026-04-03 | NCT05163288 | |
| International Study for Treatment of High Risk Childhood Relapsed ALL 2010 | Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute lymphoblastic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasm | Recruiting | Phase 2 | 2026-03-17 | NCT03590171 | |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) | familial chylomicronemia syndrome, familial lipoprotein lipase deficiency | Active, not recruiting | Phase 3 | 2025-12-12 | NCT05130450 |