UCSF Benioff Children's Hospital
UCSF Benioff Children's Hospital
Oakland, California, United States10 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3A | Active, not recruiting | Phase 1 | 2026-09-08 | NCT06567769 | |
| Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Enrolling by invitation | Phase 2 | 2026-08-26 | NCT07393373 | |
| An Interventional Study of Infigratinib in Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Enrolling by invitation | Phase 2 / Phase 3 | 2026-07-16 | NCT06873035 | |
| Prospective Clinical Assessment Study in Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Not stated | 2026-07-16 | NCT06410976 | |
| Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, achondroplasia, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hereditary endocrine growth disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 | 2026-07-13 | NCT07169279 | |
| Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400. | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, beta thalassemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, lymphatic system disorder, sickle cell disease, thalassemia | Active, not recruiting | Not stated | 2025-11-12 | NCT05145062 | |
| A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome | 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 | Active, not recruiting | Phase 1 / Phase 2 | 2025-08-07 | NCT04251026 | |
| A Multicenter Multinational Observational Study of Children With Hypochondroplasia | hypochondroplasia | Recruiting | Not stated | 2025-04-03 | NCT06212947 | |
| A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, beta thalassemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease, thalassemia | Enrolling by invitation | Not stated | 2025-04-02 | NCT06363760 | |
| A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease | Active, not recruiting | Phase 1 / Phase 2 | 2025-01-31 | NCT04853576 |