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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

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Count: 74
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NCT IDTitle
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT05358717A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT02139306Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT02251600A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT07159139Vatiquinone Expanded Access Protocol
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT04014530Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma