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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: 75
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NCT IDTitle
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
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
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT07159139Vatiquinone Expanded Access Protocol
NCT07614126Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene)
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02286622A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
NCT02139306Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT07681713Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT01009294Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT04439071A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
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
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT01910168A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT04336826A 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)