Uncommon Cures
Uncommon Cures
Chevy Chase, Maryland, United States5 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 2 | 2026-10-09 | NCT06447025 | |
| 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 | |
| A Study of TYRA-300 in Children With Achondroplasia: BEACH301 | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 | 2026-10-01 | NCT06842355 | |
| 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 | |
| A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorder | Recruiting | Phase 2 | 2025-03-17 | NCT06488313 |