Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
- Status
- TERMINATED
- Phase
- PHASE1
Clinical Trials Related to PTC Therapeutics
Related studies linked through entity extraction aliases.
A Study of CNSA-001 in Women With Diabetic Gastroparesis
- Status
- COMPLETED
- Phase
- PHASE2
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Status
- COMPLETED
- Phase
- PHASE2
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
- Status
- WITHDRAWN
- Phase
- PHASE3
A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
- Status
- COMPLETED
- Phase
- PHASE1
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
- Status
- COMPLETED
- Phase
- PHASE3
A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
- Status
- COMPLETED
- Phase
- PHASE2
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
- Status
- COMPLETED
- Phase
- PHASE2
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
- Status
- COMPLETED
- Phase
- PHASE2
Study of Ataluren in Participants With Nonsense Mutation Aniridia
- Status
- COMPLETED
- Phase
- PHASE2
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
- Status
- APPROVED_FOR_MARKETING
- Phase
- N/A
Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
- Status
- COMPLETED
- Phase
- PHASE1
Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
- Status
- COMPLETED
- Phase
- PHASE2
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
- Status
- TERMINATED
- Phase
- PHASE3
Effects of Rifampin on the Pharmacokinetics of Ataluren
- Status
- COMPLETED
- Phase
- PHASE1
PTC596 in Patients With Advanced Solid Tumors
- Status
- COMPLETED
- Phase
- PHASE1
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Status
- COMPLETED
- Phase
- N/A
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
- Status
- COMPLETED
- Phase
- PHASE2