The Children's Hospital of Philadelphia (CHOP)
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States7 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study of SGT-212 Gene Therapy in Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Dyskinesias, Friedreich ataxia, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Signs and Symptoms, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 1 | 2026-09-29 | NCT07180355 | |
| A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction disease | Recruiting | Not stated | 2026-09-25 | NCT06078553 | |
| 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 | |
| NS-089/NCNP-02-201 in Boys 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 | 2026-09-15 | NCT05996003 | |
| Long-term Follow-Up for RGX-121 | 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 | Not stated | 2026-08-19 | NCT04597385 | |
| A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study | Acid-Base Imbalance, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, acidosis disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, propionic acidemia | Recruiting | Phase 1 / Phase 2 | 2026-03-23 | NCT05130437 | |
| Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism | endocrine system disorder, hypoparathyroidism, parathyroid gland disorder | Active, not recruiting | Phase 3 | 2025-09-29 | NCT05778071 |