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Clinical trials
catalyst calendar across sponsors →Studies where PTCT is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
61 interventional · 1 observational · 2 expanded access · 33 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| A Study of Sepiapterin in Participants With Phenylketonuria (PKU)NCT06302348Mean Change From Baseline in Full-scale Intelligence Quotient (FSIQ) Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition (WPPSI-IV) ScoreOpen-label · Treatment | Phase 3 | Recruiting | Drug: Sepiapterin | Phenylketonuria | 56 | 2031-02-28 | in 1,654 d | 2026-07-23 |
| Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)NCT07681713Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24Open-label · Treatment | Phase 3 | Not yet recruiting | Drug: Vatiquinone | Friedreich's Ataxia | 120 | 2029-03-15 | in 939 d | 2026-08-14 |
| A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich AtaxiaNCT05515536Number of Participants With Adverse Events (AEs)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: Vatiquinone | Friedreich Ataxia | 130 | 2027-12-31 | in 499 d | 2025-10-30 |
| A Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaNCT05166161Number of Treatment-Emergent Adverse Events (TEAEs)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: PTC923 | Phenylketonuria | 200 | 2026-06-30 | — | 2026-06-23 |
| A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric ParticipantsNCT04903288results2025-01-01Change From Baseline in HVA Metabolite Level at the End of the Trial PhaseOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Active, not recruiting | Genetic: Eladocagene Exuparvovec | AADC Deficiency | 13 | 2023-05-22actual | — | 2026-04-24 |
| Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)NCT01247207Number of Participants With Adverse EventsOpen-label · Treatment | Phase 3 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 270 | 2026-02-10actual | — | 2026-03-09 |
| A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseNCT05218655results2025-12-22Number of Participants With Treatment-emergent Adverse Events (TEAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Vatiquinone | Inherited Mitochondrial Disease | 101 | 2025-04-15actual | — | 2025-12-22 |
| Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT02369731observationalPercentage of Participants With Adverse Events | — | Completed | — | Muscular Dystrophy, Duchenne | 316 | 2025-03-31actual | — | 2026-02-24 |
| Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALSNCT05349721results2025-08-01Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Company decision | Drug: PTC857, Placebo | Amyotrophic Lateral Sclerosis | 336 | 2024-09-26actual | — | 2025-08-01 |
| A Study of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaNCT05485987Plasma Concentration of VatiquinoneOpen-label · Treatment | Phase 2 | Completed | Drug: Vatiquinone | Friedreich Ataxia | 5 | 2024-08-29actual | — | 2025-08-22 |
| A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)NCT05269355results2025-06-13Progression Free Survival (PFS) Per Independent Central Review Using Response Evaluation Criteria in Solid Tumors (RECIST) V1.1Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Terminatedsponsor's stated reason: Business decision | Drug: Unesbulin, Dacarbazine · Other: Placebo | Leiomyosarcoma | 359 | 2024-06-17actual | — | 2025-06-13 |
| A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)NCT03761095MTD and RP2D of Unesbulin in Combination With DacarbazineOpen-label · Treatment | Phase 1 | Completed | Drug: Unesbulin, Dacarbazine | Leiomyosarcoma | 41 | 2024-02-05actual | — | 2024-03-15 |
| Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh SyndromeNCT02352896Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 ScoreOpen-label · Treatment | Phase 2 | Completed | Drug: EPI-743 | Leigh Syndrome | 30 | 2023-10-30actual | — | 2024-10-23 |
| A 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)NCT04336826results2024-04-01Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Ataluren | Nonsene Mutation Duchenne Muscular Dystrophy | 6 | 2023-08-07actual | — | 2024-04-01 |
| A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaNCT04577352results2026-04-08Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis SetRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Completed | Drug: Vatiquinone, Placebo | Friedreich Ataxia | 146 | 2023-04-04actual | — | 2026-04-13 |
| A Study of PTC923 in Participants With PhenylketonuriaNCT05099640results2024-01-10Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: PTC923, Placebo | Phenylketonuria | 157 | 2023-04-03actual | — | 2024-01-10 |
| A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyNCT04378075results2026-03-25Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind PeriodRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Terminatedsponsor's stated reason: Sponsor decision. | Drug: Vatiquinone · Other: Placebo | Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome | 68 | 2023-03-18actual | — | 2026-03-31 |
| A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)NCT04439071results2023-06-26Time From Randomization to Respiratory ImprovementRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Terminatedsponsor's stated reason: Sponsor decision | Drug: PTC299, Placebo · Other: SOC | Pneumonia, COVID-19, Coronavirus | 189 | 2022-07-20actual | — | 2023-06-26 |
| Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyNCT03179631results2026-03-10DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) PopulationRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Ataluren, PLACEBO | Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn | 360 | 2022-03-05actual | — | 2026-03-10 |
| Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute LeukemiasNCT03761069Number of Participants Who Discontinued Study Drug Due to Adverse Event (AE)Open-label · Treatment | Phase 1 | Terminatedsponsor's stated reason: Sponsor terminated the study. | Drug: PTC299 | Leukemia, Myeloid, Acute, AML | 33 | 2021-12-28actual | — | 2022-02-02 |
| A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)NCT03642145Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs)Randomized · Open-label · Treatment | Phase 3 | Withdrawnsponsor's stated reason: Study is no longer necessary given the safety and efficacy of emflaza in this age range has already been established after review of already available data. | Drug: Deflazacort | Duchenne Muscular Dystrophy | 0 | 2021-07-31 | — | 2019-06-21 |
| A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation AniridiaNCT04117880Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Open-label · Treatment | Phase 2 | Withdrawnsponsor's stated reason: Study cancelled before protocol was implemented. | Drug: Ataluren | Aniridia | 0 | 2021-01-31 | — | 2019-10-07 |
| Study of Ataluren in Participants With Nonsense Mutation AniridiaNCT02647359results2022-05-27Percent Change From Baseline in Maximum Reading Speed of Oculus Unitas (OU) (Both Eyes) at Week 48, as Measured Using the Minnesota Low Vision Reading Test (MNREAD) Acuity ChartsRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Ataluren, Placebo | Aniridia | 39 | 2021-01-22actual | — | 2022-05-27 |
| A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)NCT03783923results2022-06-27Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With DeflazacortOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19. | Drug: Deflazacort | Limb-Girdle Muscular Dystrophy | 11 | 2021-01-01actual | — | 2022-06-27 |
| A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT03648827results2022-04-05Percent Change From Baseline in Dystrophin Level at Week 40, as Measured by ECLOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 20 | 2020-10-23actual | — | 2022-04-05 |
| A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaNCT03519711results2023-11-14Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: PTC923 | BH4 Deficiency, Hyperphenylalaninemia | 8 | 2020-10-02actual | — | 2023-11-14 |
| A Study of CNSA-001 in Women With Diabetic GastroparesisNCT03712124results2022-01-05Change From Baseline (Day 1) in Maximal Tolerated Volume Consumed During the Nutrient Satiety Test at Day 14Randomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: CNSA-001, Placebo | Gastroparesis | 21 | 2019-09-08actual | — | 2022-01-05 |
| A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenNCT03796637results2022-04-05Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL)Open-label · OtherStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 6 | 2019-06-03actual | — | 2022-04-05 |
| An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyNCT02090959results2020-08-11Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: Sponsor decision due to commercial availability of ataluren. | Drug: Ataluren | Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn | 219 | 2018-06-12actual | — | 2020-08-11 |
| Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)NCT02460679results2020-09-23Number of Participants With Drug-Related Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: EPI-589 | Amyotrophic Lateral Sclerosis | 19 | 2018-02-23actual | — | 2020-10-14 |
| Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyNCT02819557results2020-08-28Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy | 14 | 2018-02-09actual | — | 2020-08-28 |
| Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaNCT01557400results2020-11-25Number of Participants With Treatment Emergent Adverse Events (TEAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Ataluren | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy | 94 | 2018-01-19actual | — | 2020-11-25 |
| An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortNCT02295748Number, frequency, and severity of adverse eventsOpen-label · Treatment | Phase 1 | Completed | Drug: Deflazacort | Duchenne Muscular Dystrophy | 24 | 2017-08-24actual | — | 2017-12-08 |
| Study of Ataluren (PTC124) in Cystic FibrosisNCT02107859results2020-03-25Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Open-label · TreatmentStatistical Analysis Plan ↗ · Study Protocol ↗ | Phase 3 | Terminatedsponsor's stated reason: Based on the results of study PTC124-GD-021-CF (NCT02139306), clinical development of ataluren in cystic fibrosis was discontinued and this study was closed. | Drug: Ataluren | Cystic Fibrosis | 61 | 2017-06-05actual | — | 2020-03-25 |
| Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisNCT02456103results2020-04-15Number of Participants With Treatment Emergent Adverse Events (TEAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints. | Drug: Ataluren | Cystic Fibrosis | 246 | 2017-06-02actual | — | 2020-04-27 |
| PTC596 in Patients With Advanced Solid TumorsNCT02404480Dose-limiting toxicitiesNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: PTC596 | Cancer | 31 | 2017-02-06actual | — | 2017-12-08 |
| Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)NCT02139306results2020-05-14Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ataluren (PTC124®), Placebo | Cystic Fibrosis | 279 | Nov 2016actual | — | 2020-05-14 |
| Safety and Efficacy of EPI-743 in Patients With Friedreich's AtaxiaNCT01728064Visual FunctionRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Placebo, EPI-743 400 mg, EPI-743 200 mg | Friedreich's Ataxia | 60 | 2015-10-31actual | — | 2020-08-31 |
| A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyNCT02251600The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metaboliteOpen-label · Basic science | Phase 1 | Completed | Drug: Deflazacort | Duchenne Muscular Dystrophy | 24 | Oct 2015actual | — | 2017-08-18 |
| Food Effect and Bioavailability of Deflazacort Formulations in Healthy VolunteersNCT02485431Pharmacokinetic profileRandomized · Open-label · Other | Phase 1 | Completed | Drug: Deflazacort | Healthy Volunteers | 45 | Aug 2015actual | — | 2017-08-18 |
| Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT01826487results2020-08-04Change From Baseline in 6MWD at Week 48Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ataluren, Placebo | Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn | 230 | 2015-08-20actual | — | 2020-08-04 |
| Effects of Rifampin on the Pharmacokinetics of AtalurenNCT02409004Area under the plasma concentration versus time curve (AUC)Open-label · Treatment | Phase 1 | Completed | Drug: Ataluren, Rifampin | Healthy | 15 | Mar 2015actual | — | 2017-12-22 |
| Safety and Efficacy Study of EPI-743 in Children With Leigh SyndromeNCT01721733Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg | Leigh Syndrome | 35 | 2015-02-28actual | — | 2020-08-31 |
| A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort PharmacokineticsNCT02286609Hepatic impairment on the pharmacokinetics (PK) of deflazacort in subjects with moderate hepatic impairment including the area under the plasma concentration time curve, from time 0 to the last measurable non-zero concentration.Non-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: Deflazacort | Hepatic Impairment | 16 | Feb 2015actual | — | 2017-08-18 |
| A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort PharmacokineticsNCT02286622Renal impairment on the pharmacokinetics (PK) of deflazacort in subjects with end-stage renal disease including the area under the plasma concentration time curve, from time 0 to the last measurable non-zero concentration.Non-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: Deflazacort | Renal Impairment | 16 | Feb 2015actual | — | 2017-08-18 |
| Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of DeflazacortNCT02286635Effects of CYP3A4 inhibitors and inducers on the pharmacokinetics (PK) of deflazacort in healthy subjects including the area under the plasma concentration time curve, from time 0 to the last measurable non-zero concentration.Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Deflazacort and rifampin, Deflazacort and Clarithromycin | Healthy Volunteers | 58 | Dec 2014actual | — | 2017-08-18 |
| Extension Study of Ataluren (PTC124) in Cystic FibrosisNCT01140451results2020-10-19Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)Non-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: Ataluren | Cystic Fibrosis | 191 | 2013-12-02actual | — | 2020-10-19 |
| PTC299 and Hormonal Agent for Treatment of Metastatic Breast CancerNCT00508586Maximum Tolerated Dose (MTD) within the tested dose range.Open-label · Treatment | Phase 1 | Completed | Drug: PTC299 | Metastatic Breast Cancer | 33 | Mar 2012actual | — | 2016-03-03 |
| PTC299 for Treatment of Neurofibromatosis Type 2NCT00911248To assess the effects of PTC299 on tumor volume and/or word recognition in patients with NF2.Open-label · Treatment | Phase 2 | Terminated | Drug: PTC299 | Neurofibromatosis 2 | 11 | 2012-03-31actual | — | 2019-04-12 |
| PTC299 for Treatment of Advanced CancerNCT00704821Maximum Tolerated Dose (MTD) of PTC299 within the tested dose range.Non-randomized · Open-label · Treatment | Phase 1 | Terminated | Drug: PTC299, Docetaxel | Advanced Cancer | 76 | 2012-02-24actual | — | 2019-05-10 |
| Study of Ataluren (PTC124™) in Cystic FibrosisNCT00803205results2020-05-14Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at BaselineRandomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ataluren, Placebo | Cystic Fibrosis | 238 | 2011-11-12actual | — | 2020-05-14 |
| Ataluren for Nonsense Mutation Methylmalonic AcidemiaNCT01141075results2020-07-07Plasma Methylmalonic Acid (MMacid) LevelsOpen-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Terminated due to low enrollment and unclear pharmacologic effect in available pharmacodynamic data (not due to any safety concerns). | Drug: Ataluren | Amino Acid Metabolism, Inborn Errors | 11 | 2011-11-03actual | — | 2020-07-07 |
| Study of Ataluren (PTC124) in Hemophilia A and BNCT00947193results2020-06-16Number of Participants With a Plasma FVIII/FIX Activity Response at Day 14Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Study was terminated early due to Sponsor decision and not reflective of adverse safety findings. | Drug: Ataluren | Hemophilia A, Hemophilia B | 13 | 2011-08-30actual | — | 2020-06-16 |
| Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)NCT00847379results2020-07-15Number of Participants With Treatment-Emergent Adverse Events (AEs)Open-label · Treatment | Phase 2 | Terminated | Drug: Ataluren | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | 173 | 2010-05-24actual | — | 2020-07-15 |
| Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)NCT00759876results2020-10-29Number of Participants With Treatment Emergent Adverse Events (TEAEs)Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Terminated early because similar study with Ataluren (PTC124-GD-007-DMD; NCT00592553) exhibited lack of efficacy at the high dose (not due to safety concerns). | Drug: Ataluren | Duchenne Muscular Dystrophy | 36 | 2010-05-17actual | — | 2020-10-29 |
| Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)NCT01009294results2020-07-29Number of Participants With Treatment Emergent Adverse Events (TEAEs)Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Terminated early because a similar study with Ataluren exhibited lack of efficacy at the high dose (not due to safety concerns). | Drug: Ataluren, Chronic Corticosteroid Therapy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | 6 | 2010-03-23actual | — | 2020-07-29 |
| Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)NCT00592553results2020-04-07Change From Baseline in 6MWD at Week 48Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Ataluren, Placebo | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | 174 | 2009-12-31actual | — | 2020-04-07 |
| A Study of Ataluren in Pediatric Participants With Cystic FibrosisNCT00458341results2020-03-06Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2Randomized · Open-label · Treatment | Phase 2 | Completed | Drug: Ataluren | Cystic Fibrosis | 30 | 2008-02-29actual | — | 2020-03-06 |
| PTC124 for the Treatment of Cystic FibrosisNCT00351078CFTR activity as assessed by nasal transepithelial potential difference (TEPD)Open-label · Treatment | Phase 2 | Completed | Drug: PTC124 | Cystic Fibrosis | 19 | Jul 2007actual | — | 2017-12-13 |
| Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyNCT00264888Dystrophin expression as assessed by immunofluorescence evaluation of tissue obtained by biopsy of the extensor digitorum brevis (EDB) muscle of the foot or tibialis anterior (TA) muscle of the legNon-randomized · Open-label · Treatment | Phase 2 | Completed | Drug: PTC124 | Duchenne Muscular Dystrophy | 38 | May 2007actual | — | 2009-01-14 |
| PTC124 for Cystic FibrosisNCT00234663CFTR activity as assessed by nasal transepithelial potential difference (TEPD)Non-randomized · Open-label · Treatment | Phase 2 | Completed | Drug: PTC124 | Cystic Fibrosis | 24 | Aug 2006actual | — | 2009-01-13 |
| Safety and Efficacy of Ataluren (PTC124) for Cystic FibrosisNCT00237380Change From Baseline to End of Treatment in Total Chloride TransportOpen-label · Treatment | Phase 2 | Completed | Drug: Ataluren | Cystic Fibrosis | 24 | 2006-05-31actual | — | 2020-06-11 |
| EPI-743 for Mitochondrial Respiratory Chain DiseasesNCT01370447expanded access | — | No longer available | Drug: EPI-743 | Mitochondrial Diseases | — | — | — | 2024-01-23 |
| Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular DystrophyNCT02592941expanded access | — | Approved for marketing | Drug: Deflazacort | Duchenne Muscular Dystrophy | — | — | — | 2018-02-28 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19