University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States4 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder | Recruiting | Phase 3 | 2026-10-08 | NCT06247085 | |
| Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, laminopathy, restrictive cardiomyopathy, subvalvular aortic stenosis | Active, not recruiting | Not stated | 2026-09-02 | NCT05112237 | |
| A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder | Enrolling by invitation | Phase 3 | 2026-05-28 | NCT06431893 | |
| Investigation of Remimazolam in Children Undergoing Sedation for Medical Procedures | Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, childhood acute lymphoblastic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasm | Active, not recruiting | Phase 2 / Phase 3 | 2025-07-01 | NCT04851717 |