PTC Therapeutics
PTC Therapeutics
Explore linked clinical trials.8 results ยท Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study of Sepiapterin in Participants With Phenylketonuria (PKU) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Phase 3 | 2026-08-28 | NCT06302348 | |
| Study of PTC844 in Participants With Active Rheumatoid Arthritis | Skin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, musculoskeletal system disorder, rheumatic disorder, rheumatoid arthritis | Not yet recruiting | Phase 2 | 2026-08-21 | NCT07779616 | |
| Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) | 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 | Not yet recruiting | Phase 3 | 2026-08-14 | NCT07681713 | |
| Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene) | CTNNB1; L-DOPA | Recruiting | Not applicable | 2026-05-29 | NCT07614126 | |
| A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants | aromatic L-amino acid decarboxylase deficiency | Active, not recruiting | Phase 2 | 2026-04-24 | NCT04903288 | |
| A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma | Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, malignant glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumor | Active, not recruiting | Phase 1 | 2026-03-27 | NCT03605550 | |
| A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich 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 | Active, not recruiting | Phase 3 | 2025-10-30 | NCT05515536 | |
| Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study | Digestive System Diseases, Digestive System Neoplasms, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Neoplasm Metastasis, Neoplasms by Site, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, Urogenital Neoplasms, colonic disorder, colorectal cancer, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endometrium neoplasm, female reproductive system disorder, female reproductive system neoplasm, intestinal disorder, neoplasm, rectal disorder, uterine disorder | Recruiting | Phase 1 / Phase 2 | 2023-02-01 | NCT04014530 |