PTC Therapeutics Inc
PTCTHealthcare|Biotechnology|USA
$84.85
-4.70 (-5.25%)
Clinical Trials (75)
| Trial | Conditions | Phase | Status | Enrollment | Design | Drug |
|---|---|---|---|---|---|---|
| NCT05166161 A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria | Phenylketonuria | P3 | Active | 200 | Open-label | PTC923 |
| NCT06302348 A Study of Sepiapterin in Participants With Phenylketonuria (PKU) | Phenylketonuria | P3 | Recruiting | 56 | Open-label | Sepiapterin |
| NCT05515536 A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | Friedreich Ataxia | P3 | Active | 130 | Open-label | Vatiquinone |
| NCT04903288 A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants | AADC Deficiency | P2 | Active | 13 | Open-label | — |
| NCT04014530 Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study | Colorectal Cancer, Endometrium Cancer | P1P2 | Recruiting | 47 | Open-label | Ataluren + Pembrolizumab |
| NCT03605550 A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma | High Grade Glioma, Diffuse Intrinsic Pontine Glioma | P1 | Active | 64 | Open-label | PTC596 |
| NCT07614126 Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene) | CTNNB1, L-DOPA | N/A | Recruiting | 7 | Open-label | L-Dopa |
| NCT04439071 A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19) | Pneumonia, COVID-19 | P2P3 | Terminated | 189 | RCT, Double-blind | PTC299, Placebo |
| NCT02090959 An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy | Muscular Dystrophy, Duchenne, Muscular Dystrophies | P3 | Terminated | 219 | Open-label | Ataluren |
| NCT01247207 Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) | Duchenne Muscular Dystrophy | P3 | Completed | 270 | Open-label | Ataluren |
| NCT05218655 A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease | Inherited Mitochondrial Disease | P3 | Completed | 101 | Open-label | Vatiquinone |
| NCT03179631 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne, Muscular Dystrophies | P3 | Completed | 360 | RCT, Double-blind | Ataluren, PLACEBO |
| NCT03783923 A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | Limb-Girdle Muscular Dystrophy | P3 | Terminated | 11 | Open-label | Deflazacort |
| NCT01140451 Extension Study of Ataluren (PTC124) in Cystic Fibrosis | Cystic Fibrosis | P3 | Completed | 191 | Open-label | Ataluren |
| NCT07681713 Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) | Friedreich's Ataxia | P3 | Not Yet Recruiting | 120 | Open-label | Vatiquinone |
| NCT02456103 Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis | Cystic Fibrosis | P3 | Terminated | 246 | Open-label | Ataluren |
| NCT05269355 A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS) | Leiomyosarcoma | P2P3 | Terminated | 359 | RCT, Double-blind | Unesbulin, Dacarbazine |
| NCT04378075 A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Mitochondrial Diseases, Drug Resistant Epilepsy | P2P3 | Terminated | 68 | RCT, Double-blind | Vatiquinone |
| NCT02139306 Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) | Cystic Fibrosis | P3 | Completed | 279 | RCT, Double-blind | Ataluren (PTC124®), Placebo |
| NCT03642145 A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P3 | Withdrawn | — | RCT, Open-label | Deflazacort |
| NCT04577352 A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | Friedreich Ataxia | P2P3 | Completed | 146 | RCT, Double-blind | Vatiquinone, Placebo |
| NCT05099640 A Study of PTC923 in Participants With Phenylketonuria | Phenylketonuria | P3 | Completed | 157 | RCT, Double-blind | PTC923, Placebo |
| NCT01826487 Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Muscular Dystrophy, Duchenne, Muscular Dystrophies | P3 | Completed | 230 | RCT, Double-blind | Ataluren, Placebo |
| NCT01557400 Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | P3 | Completed | 94 | Open-label | Ataluren |
| NCT02107859 Study of Ataluren (PTC124) in Cystic Fibrosis | Cystic Fibrosis | P3 | Terminated | 61 | Open-label | Ataluren |
| NCT00803205 Study of Ataluren (PTC124™) in Cystic Fibrosis | Cystic Fibrosis | P3 | Completed | 238 | RCT, Double-blind | Ataluren, Placebo |
| NCT02352896 Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome | Leigh Syndrome | P2 | Completed | 30 | Open-label | EPI-743 |
| NCT02647359 Study of Ataluren in Participants With Nonsense Mutation Aniridia | Aniridia | P2 | Completed | 39 | RCT, Double-blind | Ataluren, Placebo |
| NCT04117880 A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia | Aniridia | P2 | Withdrawn | — | Open-label | Ataluren |
| NCT02926066 A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion | Aromatic Amino Acid Decarboxylase Deficiency | P2 | Completed | 12 | Open-label | AAV2-hAADC |
| NCT01009294 Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | P2 | Terminated | 6 | Open-label | Ataluren, Chronic Corticosteroid Therapy |
| NCT01141075 Ataluren for Nonsense Mutation Methylmalonic Acidemia | Amino Acid Metabolism, Inborn Errors | P2 | Terminated | 11 | Open-label | Ataluren |
| NCT00847379 Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | P2 | Terminated | 173 | Open-label | Ataluren |
| NCT02758626 Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome | Epilepsy | P2 | Completed | 15 | RCT, Double-blind | ataluren, Placebo |
| NCT01728064 Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia | Friedreich's Ataxia | P2 | Completed | 60 | RCT, Double-blind | Placebo, EPI-743 400 mg, EPI-743 200 mg |
| NCT02819557 Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | P2 | Completed | 14 | Open-label | Ataluren |
| NCT00264888 Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | P2 | Completed | 38 | Open-label | PTC124 |
| NCT00234663 PTC124 for Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 24 | Open-label | PTC124 |
| NCT01395641 A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | P1P2 | Completed | 10 | Open-label | gene therapy |
| NCT00458341 A Study of Ataluren in Pediatric Participants With Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 30 | RCT, Open-label | Ataluren |
| NCT00947193 Study of Ataluren (PTC124) in Hemophilia A and B | Hemophilia A, Hemophilia B | P2 | Terminated | 13 | Open-label | Ataluren |
| NCT03712124 A Study of CNSA-001 in Women With Diabetic Gastroparesis | Gastroparesis | P2 | Completed | 21 | RCT, Double-blind | CNSA-001, Placebo |
| NCT00759876 Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | P2 | Terminated | 36 | Open-label | Ataluren |
| NCT00237380 Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 24 | Open-label | Ataluren |
| NCT04336826 A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Nonsene Mutation Duchenne Muscular Dystrophy | P2 | Completed | 6 | Open-label | Ataluren |
| NCT03648827 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Duchenne Muscular Dystrophy | P2 | Completed | 20 | Open-label | Ataluren |
| NCT00592553 Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | P2 | Completed | 174 | RCT, Double-blind | Ataluren, Placebo |
| NCT01721733 Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome | Leigh Syndrome | P2 | Completed | 35 | RCT, Double-blind | Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg |
| NCT02460679 Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | P2 | Completed | 19 | Open-label | EPI-589 |
| NCT03519711 A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia | BH4 Deficiency, Hyperphenylalaninemia | P1P2 | Completed | 8 | RCT, Open-label | PTC923 |
| NCT00351078 PTC124 for the Treatment of Cystic Fibrosis | Cystic Fibrosis | P2 | Completed | 19 | Open-label | PTC124 |
| NCT05349721 Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS | Amyotrophic Lateral Sclerosis | P2 | Terminated | 336 | RCT, Double-blind | PTC857, Placebo |
| NCT05485987 A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | Friedreich Ataxia | P2 | Completed | 5 | Open-label | Vatiquinone |
| NCT00911248 PTC299 for Treatment of Neurofibromatosis Type 2 | Neurofibromatosis 2 | P2 | Terminated | 11 | Open-label | PTC299 |
| NCT00686842 PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma | Kaposi's Sarcoma | P1P2 | Terminated | 17 | Open-label | VEGF inhibitor PTC299 |
| NCT03796637 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren | Duchenne Muscular Dystrophy | P2 | Completed | 6 | Open-label | Ataluren |
| NCT01158300 PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors | Brain and Central Nervous System Tumors | P1 | Completed | 28 | Open-label | VEGF inhibitor PTC299 |
| NCT03206645 Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy | Ovarian Cancer | P1 | Completed | 27 | Open-label | Unesbulin |
| NCT02295748 An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort | Duchenne Muscular Dystrophy | P1 | Completed | 24 | Open-label | Deflazacort |
| NCT03761069 Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias | Leukemia, Myeloid, Acute, AML | P1 | Terminated | 33 | Open-label | PTC299 |
| NCT02251600 A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | P1 | Completed | 24 | Open-label | Deflazacort |
| NCT02286622 A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics | Renal Impairment | P1 | Completed | 16 | Open-label | Deflazacort |
| NCT02404480 PTC596 in Patients With Advanced Solid Tumors | Cancer | P1 | Completed | 31 | Open-label | PTC596 |
| NCT03761095 A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS) | Leiomyosarcoma | P1 | Completed | 41 | Open-label | Unesbulin, Dacarbazine |
| NCT02485431 Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers | Healthy Volunteers | P1 | Completed | 45 | RCT, Open-label | Deflazacort |
| NCT02286635 Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort | Healthy Volunteers | P1 | Completed | 58 | Open-label | Deflazacort and rifampin, Deflazacort and Clarithromycin |
| NCT02286609 A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics | Hepatic Impairment | P1 | Completed | 16 | Open-label | Deflazacort |
| NCT00508586 PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer | Metastatic Breast Cancer | P1 | Completed | 33 | Open-label | PTC299 |
| NCT02409004 Effects of Rifampin on the Pharmacokinetics of Ataluren | Healthy | P1 | Completed | 15 | Open-label | Ataluren, Rifampin |
| NCT00704821 PTC299 for Treatment of Advanced Cancer | Advanced Cancer | P1 | Terminated | 76 | Open-label | PTC299, Docetaxel |
| NCT02592941 Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | APPROVED_FOR_MARKETING | — | — | Deflazacort | |
| NCT01370447 EPI-743 for Mitochondrial Respiratory Chain Diseases | Mitochondrial Diseases | NO_LONGER_AVAILABLE | — | — | EPI-743 | |
| NCT01910168 A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis | Muscular Atrophy, Spinal | Completed | 36 | — | — | |
| NCT02369731 Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Muscular Dystrophy, Duchenne | Completed | 316 | — | — | |
| NCT07159139 Vatiquinone Expanded Access Protocol | Mitochondrial Disease | NO_LONGER_AVAILABLE | — | — | Vatiquinone |