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
‌ A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia	NCT04117880	Aniridia	December 31, 2018	January 31, 2021	Interventional
‌ PTC299 for Treatment of Advanced Cancer	NCT00704821		July 3, 2008	February 24, 2012	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
‌ A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria	NCT05166161	Phenylketonuria	February 14, 2022	February 7, 2025	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
‌ Study of Ataluren (PTC124) in Cystic Fibrosis	NCT02107859	Cystic fibrosis	May 23, 2014	June 5, 2017	Interventional
‌ Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis	NCT02456103	Cystic fibrosis	August 31, 2015	June 2, 2017	Interventional
‌ A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	NCT03648827	‌ Duchenne muscular dystrophy	December 21, 2018	October 23, 2020	Interventional
‌ EPI-743 for Mitochondrial Respiratory Chain Diseases	NCT01370447		February 28, 2010	September 28, 2023	Interventional
‌ Extension Study of Ataluren (PTC124) in Cystic Fibrosis	NCT01140451	Cystic fibrosis	August 12, 2010	December 2, 2013	Interventional
‌ Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)	NCT00847379	‌ Duchenne muscular dystrophy	January 31, 2009	May 24, 2010	Interventional
‌ A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics	NCT02286622		2014	2015	Interventional
‌ PTC124 for the Treatment of Cystic Fibrosis	NCT00351078	Cystic fibrosis	2006	2007	Interventional
‌ Effects of Rifampin on the Pharmacokinetics of Ataluren	NCT02409004	‌ Healthy	2015	2015	Interventional
‌ PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer	NCT00508586	Metastatic breast cancer	2007	2012	Interventional
‌ Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort	NCT02286635		2014	2014	Interventional

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