An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
- Status
- COMPLETED
- Phase
- PHASE1
Clinical Trials Related to PTC Therapeutics
Related studies linked through entity extraction aliases.
Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
- Status
- COMPLETED
- Phase
- PHASE1
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
- Status
- COMPLETED
- Phase
- PHASE1
A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
- Status
- COMPLETED
- Phase
- PHASE1
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
- Status
- COMPLETED
- Phase
- PHASE1
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
- Status
- COMPLETED
- Phase
- PHASE3
Study of Ataluren (PTC124) in Cystic Fibrosis
- Status
- TERMINATED
- Phase
- PHASE3
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
- Status
- TERMINATED
- Phase
- PHASE3
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
- Status
- COMPLETED
- Phase
- N/A
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Status
- COMPLETED
- Phase
- PHASE3
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
- Status
- COMPLETED
- Phase
- PHASE2
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
- Status
- COMPLETED
- Phase
- PHASE2
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
- Status
- COMPLETED
- Phase
- PHASE3
A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
EPI-743 for Mitochondrial Respiratory Chain Diseases
- Status
- NO_LONGER_AVAILABLE
- Phase
- N/A
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
- Status
- COMPLETED
- Phase
- PHASE3
PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
- Status
- COMPLETED
- Phase
- PHASE1
Ataluren for Nonsense Mutation Methylmalonic Acidemia
- Status
- TERMINATED
- Phase
- PHASE2
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
- Status
- COMPLETED
- Phase
- PHASE3
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
- Status
- TERMINATED
- Phase
- PHASE2