Clinical Studies Sponsored by PTC Therapeutics

Clinical Studies where

Trial Sponsor

Name	NCT Number	Health Conditions in Trial	Clinical Trial Start Date	Study Completion Date	Clinical Trial Study Type
‌ PTC124 for Cystic Fibrosis	NCT00234663	Cystic fibrosis	2005	2006	Interventional
‌ Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy	NCT00264888	‌ Duchenne muscular dystrophy	2005	2007	Interventional
‌ A Study of Sepiapterin in Participants With Phenylketonuria (PKU)	NCT06302348	Phenylketonuria	February 29, 2024	February 28, 2031	Interventional
‌ Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy	NCT02592941	‌ Duchenne muscular dystrophy
‌ An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)	NCT06254482		August 25, 2023	July 20, 2027	Interventional
‌ Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome	NCT01721733		October 31, 2012	May 31, 2015	Interventional
‌ An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy	NCT02090959		March 20, 2014	June 12, 2018	Interventional
‌ Ataluren for Nonsense Mutation Methylmalonic Acidemia	NCT01141075		July 19, 2010	November 3, 2011	Interventional
‌ A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	NCT04577352		December 17, 2020	October 2, 2023	Interventional
‌ Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)	NCT02460679	Amyotrophic lateral sclerosis	January 14, 2016	February 23, 2018	Interventional
‌ Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers	NCT02485431		2015	2015	Interventional
‌ A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy	NCT02251600	‌ Duchenne muscular dystrophy	2014	2015	Interventional
‌ A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics	NCT02286609		2014	2015	Interventional
‌ Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada	NCT01557400	‌ Duchenne muscular dystrophy	May 20, 2012	January 19, 2018	Interventional
‌ Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)	NCT02139306	Cystic fibrosis	2014	2016	Interventional
‌ Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)	NCT00592553	‌ Duchenne muscular dystrophy	February 29, 2008	December 31, 2009	Interventional
‌ Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	NCT01826487		March 26, 2013	August 20, 2015	Interventional
‌ Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)	NCT00759876	‌ Duchenne muscular dystrophy	August 13, 2008	May 17, 2010	Interventional
‌ Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis	NCT00237380	Cystic fibrosis	November 30, 2005	May 31, 2006	Interventional
‌ A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)	NCT04439071	Pneumonia COVID-19	July 9, 2020	July 20, 2022	Interventional
‌ A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	NCT05485987		October 13, 2022	October 5, 2024	Interventional
‌ Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort	NCT02286635		2014	2014	Interventional
‌ An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort	NCT02295748	‌ Duchenne muscular dystrophy	2014	August 24, 2017	Interventional
‌ PTC596 in Patients With Advanced Solid Tumors	NCT02404480	Cancer	2016	February 6, 2017	Interventional
‌ PTC299 for Treatment of Neurofibromatosis Type 2	NCT00911248		July 31, 2009	March 31, 2012	Interventional

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