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Clinical trial updates
Drug and device trials that companies register on ClinicalTrials.gov: newly completed phase 3 studies, changes of status and the dates a study is due to finish.
About this data
- What it is
- ClinicalTrials.gov is the US registry of clinical studies. Drug and device makers register their trials there and update the record as each study moves on. We list the studies that companies sponsor, and match the sponsor to a public company when the name lines up. The page opens on Phase 3 studies marked completed, the late-stage trials that come before a request for approval. Change View to see the changes of status we have noticed, or every recent update.
- Who files it
- The sponsor of the study, in its record on ClinicalTrials.gov. The registry is run by the US National Library of Medicine and does not check what sponsors report.
- How current
- We check the registry every day. Sponsors update records on their own schedule, from the same week to months later, so a change can appear in the registry well after it happened.
- Terms
- ClinicalTrials.govClinicalTrials.gov is the US government's registry of clinical studies, run by the National Library of Medicine. Sponsors register a study before it starts and update the record as it goes. The registry does not check what a sponsor reports.Phase 3The late stage of testing, usually with hundreds or thousands of people, to confirm that a treatment works and to find side effects. A study marked Phase 2/3 covers both stages.statusThe registry's own word for where a study stands: not yet recruiting, recruiting, active but not recruiting, completed, terminated (stopped early), withdrawn (never started), suspended, or unknown (the sponsor has not confirmed the record for a long time).completedThe sponsor has marked the study as finished. It says nothing about whether the treatment worked: results, if any, are reported separately and often later.status changesOur history of status changes starts the day we first stored each study, so a change made before that is not shown. The date is when we noticed the change, which can be later than when the sponsor made it.primary completionThe date the last participant is examined for the main question the study asks. The study can run on after it, for follow-up. Sponsors enter the date and revise it, and an estimated date can pass without an update.company matchWe match the sponsor's name to a public company automatically, and by hand for the large drugmakers. A subsidiary or a differently spelled name can be missed, so a study with no ticker does not mean the sponsor is private.
- Worth knowing
- A completed study is not a result: the registry says the study ended, not whether the treatment worked. Status changes start on the day we first stored each study, so earlier changes are not shown. Dates are the sponsor's own, and an estimated date can pass without an update. We match sponsors to companies automatically, with a hand-checked list for the large drugmakers, so a study with no ticker may still belong to a public company. This is information about studies, not a forecast of what they will show or of a stock's price.
- Not to be confused with
- FDA approvals and clearances: what the FDA decided, not what a sponsor reports about a study.
All of this comes from ClinicalTrials.gov, the US registry of clinical studies. We are not affiliated with it.
Source and freshness · Information, not investment advice (disclaimer).
Studies updated per day, last 30 days
All industry-sponsored studies Sponsor matched to a public company
By the day the sponsor last updated the study's record. A study updated twice counts once, on its latest day.
Latest clinical trial updates
Showing: ticker PTCT · All recent updates
Studies by the date their record was last updated.
| Updated | Study | SponsorWe match the sponsor's name to a public company automatically, and by hand for the large drugmakers. A subsidiary or a differently spelled name can be missed, so a study with no ticker does not mean the sponsor is private. | PhaseThe late stage of testing, usually with hundreds or thousands of people, to confirm that a treatment works and to find side effects. A study marked Phase 2/3 covers both stages. | StatusThe registry's own word for where a study stands: not yet recruiting, recruiting, active but not recruiting, completed, terminated (stopped early), withdrawn (never started), suspended, or unknown (the sponsor has not confirmed the record for a long time). | Enrolled | Primary completionThe date the last participant is examined for the main question the study asks. The study can run on after it, for follow-up. Sponsors enter the date and revise it, and an estimated date can pass without an update. | Source |
|---|---|---|---|---|---|---|---|
| Nov 6, 2026 | Study of PTC844 in Participants With Active Rheumatoid Arthritis NCT07779616 · Rheumatoid Arthritis PTC844, Placebo | PTCT PTC Therapeutics | Phase 2 | Recruiting was not yet recruiting | 75 | Sep 30, 2027 | Registry ↗ |
| Oct 23, 2026 | A Study of Sepiapterin in Participants With Phenylketonuria (PKU) NCT06302348 · Phenylketonuria Sepiapterin | PTCT PTC Therapeutics | Phase 3 | Recruiting | 56 | Feb 28, 2031 | Registry ↗ |
| Oct 8, 2026 | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBM… NCT01247207 · Duchenne Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 3 | Terminated | 265 | Feb 10, 2026 | Registry ↗ |
| Sep 9, 2026 | A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria NCT05166161 · Phenylketonuria PTC923 | PTCT PTC Therapeutics | Phase 3 | Completed | 247 | Jul 21, 2026 | Registry ↗ |
| Aug 14, 2026 | Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) NCT07681713 · Friedreich's Ataxia Vatiquinone | PTCT PTC Therapeutics | Phase 3 | Not yet recruiting | 120 | Mar 15, 2029 | Registry ↗ |
| Apr 24, 2026 | A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Elado… NCT04903288 · AADC Deficiency Eladocagene Exuparvovec | PTCT PTC Therapeutics | Phase 2 | Active, not recruiting | 13 | May 22, 2023 | Registry ↗ |
| Apr 13, 2026 | A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Fri… NCT04577352 · Friedreich Ataxia Vatiquinone, Placebo | PTCT PTC Therapeutics | Phase 2/3 | Completed | 146 | Apr 4, 2023 | Registry ↗ |
| Mar 31, 2026 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Partic… NCT04378075 · Mitochondrial Diseases, Drug Resistant Epilepsy Vatiquinone, Placebo | PTCT PTC Therapeutics | Phase 2/3 | Terminated | 68 | Mar 18, 2023 | Registry ↗ |
| Mar 10, 2026 | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy NCT03179631 · Muscular Dystrophy, Duchenne, Muscular Dystrophies Ataluren, PLACEBO | PTCT PTC Therapeutics | Phase 3 | Completed | 360 | Mar 5, 2022 | Registry ↗ |
| Feb 24, 2026 | Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT02369731 · Muscular Dystrophy, Duchenne | PTCT PTC Therapeutics | — | Completed | 316 | Mar 31, 2025 | Registry ↗ |
| Dec 22, 2025 | A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondria… NCT05218655 · Inherited Mitochondrial Disease Vatiquinone | PTCT PTC Therapeutics | Phase 3 | Completed | 101 | Apr 15, 2025 | Registry ↗ |
| Oct 30, 2025 | A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia NCT05515536 · Friedreich Ataxia Vatiquinone | PTCT PTC Therapeutics | Phase 3 | Active, not recruiting | 130 | Dec 31, 2027 | Registry ↗ |
| Aug 22, 2025 | A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia NCT05485987 · Friedreich Ataxia Vatiquinone | PTCT PTC Therapeutics | Phase 2 | Completed | 5 | Aug 29, 2024 | Registry ↗ |
| Aug 1, 2025 | Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis… NCT05349721 · Amyotrophic Lateral Sclerosis PTC857, Placebo | PTCT PTC Therapeutics | Phase 2 | Terminated | 336 | Sep 26, 2024 | Registry ↗ |
| Jun 13, 2025 | A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS) NCT05269355 · Leiomyosarcoma Unesbulin, Dacarbazine | PTCT PTC Therapeutics | Phase 2/3 | Terminated | 359 | Jun 17, 2024 | Registry ↗ |
| Oct 23, 2024 | Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome NCT02352896 · Leigh Syndrome EPI-743 | PTCT PTC Therapeutics | Phase 2 | Completed | 30 | Oct 30, 2023 | Registry ↗ |
| Apr 1, 2024 | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <… NCT04336826 · Nonsene Mutation Duchenne Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 2 | Completed | 6 | Aug 7, 2023 | Registry ↗ |
| Mar 15, 2024 | A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyo… NCT03761095 · Leiomyosarcoma Unesbulin, Dacarbazine | PTCT PTC Therapeutics | Phase 1 | Completed | 41 | Feb 5, 2024 | Registry ↗ |
| Jan 23, 2024 | EPI-743 for Mitochondrial Respiratory Chain Diseases NCT01370447 · Mitochondrial Diseases EPI-743 | PTCT PTC Therapeutics | — | No longer available | — | — | Registry ↗ |
| Jan 10, 2024 | A Study of PTC923 in Participants With Phenylketonuria NCT05099640 · Phenylketonuria PTC923, Placebo | PTCT PTC Therapeutics | Phase 3 | Completed | 157 | Apr 3, 2023 | Registry ↗ |
| Nov 14, 2023 | A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyper… NCT03519711 · BH4 Deficiency, Hyperphenylalaninemia PTC923 | PTCT PTC Therapeutics | Phase 1/2 | Completed | 8 | Oct 2, 2020 | Registry ↗ |
| Jun 26, 2023 | A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With C… NCT04439071 · Pneumonia, COVID-19 PTC299, SOC | PTCT PTC Therapeutics | Phase 2/3 | Terminated | 189 | Jul 20, 2022 | Registry ↗ |
| Jun 27, 2022 | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) NCT03783923 · Limb-Girdle Muscular Dystrophy Deflazacort | PTCT PTC Therapeutics | Phase 3 | Terminated | 11 | Jan 1, 2021 | Registry ↗ |
| May 27, 2022 | Study of Ataluren in Participants With Nonsense Mutation Aniridia NCT02647359 · Aniridia Ataluren, Placebo | PTCT PTC Therapeutics | Phase 2 | Completed | 39 | Jan 22, 2021 | Registry ↗ |
| Apr 5, 2022 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystro… NCT03796637 · Duchenne Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 2 | Completed | 6 | Jun 3, 2019 | Registry ↗ |
| Apr 5, 2022 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystro… NCT03648827 · Duchenne Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 2 | Completed | 20 | Oct 23, 2020 | Registry ↗ |
| Feb 2, 2022 | Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias NCT03761069 · Leukemia, Myeloid, Acute, AML PTC299 | PTCT PTC Therapeutics | Phase 1 | Terminated | 33 | Dec 28, 2021 | Registry ↗ |
| Jan 5, 2022 | A Study of CNSA-001 in Women With Diabetic Gastroparesis NCT03712124 · Gastroparesis CNSA-001, Placebo | PTCT PTC Therapeutics | Phase 2 | Completed | 21 | Sep 8, 2019 | Registry ↗ |
| Nov 25, 2020 | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscul… NCT01557400 · Duchenne Muscular Dystrophy, Becker Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 3 | Completed | 94 | Jan 19, 2018 | Registry ↗ |
| Oct 29, 2020 | Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) NCT00759876 · Duchenne Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 2 | Terminated | 36 | May 17, 2010 | Registry ↗ |
| Oct 19, 2020 | Extension Study of Ataluren (PTC124) in Cystic Fibrosis NCT01140451 · Cystic Fibrosis Ataluren | PTCT PTC Therapeutics | Phase 3 | Completed | 191 | Dec 2, 2013 | Registry ↗ |
| Oct 14, 2020 | Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS) NCT02460679 · Amyotrophic Lateral Sclerosis EPI-589 | PTCT PTC Therapeutics | Phase 2 | Completed | 19 | Feb 23, 2018 | Registry ↗ |
| Aug 31, 2020 | Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia NCT01728064 · Friedreich's Ataxia Placebo, EPI-743 400 mg | PTCT PTC Therapeutics | Phase 2 | Completed | 60 | Oct 31, 2015 | Registry ↗ |
| Aug 31, 2020 | Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome NCT01721733 · Leigh Syndrome Placebo, EPI-743 15 mg/kg | PTCT PTC Therapeutics | Phase 2 | Completed | 35 | Feb 28, 2015 | Registry ↗ |
| Aug 28, 2020 | Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy NCT02819557 · Duchenne Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 2 | Completed | 14 | Feb 9, 2018 | Registry ↗ |
| Aug 11, 2020 | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy NCT02090959 · Muscular Dystrophy, Duchenne, Muscular Dystrophies Ataluren | PTCT PTC Therapeutics | Phase 3 | Terminated | 219 | Jun 12, 2018 | Registry ↗ |
| Aug 4, 2020 | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT01826487 · Muscular Dystrophy, Duchenne, Muscular Dystrophies Ataluren, Placebo | PTCT PTC Therapeutics | Phase 3 | Completed | 230 | Aug 20, 2015 | Registry ↗ |
| Jul 29, 2020 | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/B… NCT01009294 · Duchenne Muscular Dystrophy, Becker Muscular Dystrophy Ataluren, Chronic Corticosteroid Therapy | PTCT PTC Therapeutics | Phase 2 | Terminated | 6 | Mar 23, 2010 | Registry ↗ |
| Jul 15, 2020 | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00847379 · Duchenne Muscular Dystrophy, Becker Muscular Dystrophy Ataluren | PTCT PTC Therapeutics | Phase 2 | Terminated | 173 | May 24, 2010 | Registry ↗ |
| Jul 7, 2020 | Ataluren for Nonsense Mutation Methylmalonic Acidemia NCT01141075 · Amino Acid Metabolism, Inborn Errors Ataluren | PTCT PTC Therapeutics | Phase 2 | Terminated | 11 | Nov 3, 2011 | Registry ↗ |
| Jun 16, 2020 | Study of Ataluren (PTC124) in Hemophilia A and B NCT00947193 · Hemophilia A, Hemophilia B Ataluren | PTCT PTC Therapeutics | Phase 2 | Terminated | 13 | Aug 30, 2011 | Registry ↗ |
| Jun 11, 2020 | Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis NCT00237380 · Cystic Fibrosis Ataluren | PTCT PTC Therapeutics | Phase 2 | Completed | 24 | May 31, 2006 | Registry ↗ |
| May 14, 2020 | Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) NCT02139306 · Cystic Fibrosis Ataluren (PTC124®), Placebo | PTCT PTC Therapeutics | Phase 3 | Completed | 279 | Nov 2016 | Registry ↗ |
| May 14, 2020 | Study of Ataluren (PTC124™) in Cystic Fibrosis NCT00803205 · Cystic Fibrosis Ataluren, Placebo | PTCT PTC Therapeutics | Phase 3 | Completed | 238 | Nov 12, 2011 | Registry ↗ |
| Apr 27, 2020 | Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis NCT02456103 · Cystic Fibrosis Ataluren | PTCT PTC Therapeutics | Phase 3 | Terminated | 246 | Jun 2, 2017 | Registry ↗ |
| Apr 7, 2020 | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00592553 · Duchenne Muscular Dystrophy, Becker Muscular Dystrophy Ataluren, Placebo | PTCT PTC Therapeutics | Phase 2 | Completed | 174 | Dec 31, 2009 | Registry ↗ |
| Mar 25, 2020 | Study of Ataluren (PTC124) in Cystic Fibrosis NCT02107859 · Cystic Fibrosis Ataluren | PTCT PTC Therapeutics | Phase 3 | Terminated | 61 | Jun 5, 2017 | Registry ↗ |
| Mar 6, 2020 | A Study of Ataluren in Pediatric Participants With Cystic Fibrosis NCT00458341 · Cystic Fibrosis Ataluren | PTCT PTC Therapeutics | Phase 2 | Completed | 30 | Feb 29, 2008 | Registry ↗ |
| Oct 7, 2019 | A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia NCT04117880 · Aniridia Ataluren | PTCT PTC Therapeutics | Phase 2 | Withdrawn | 0 | Jan 31, 2021 | Registry ↗ |
| Jun 21, 2019 | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) NCT03642145 · Duchenne Muscular Dystrophy Deflazacort | PTCT PTC Therapeutics | Phase 3 | Withdrawn | 0 | Jul 31, 2021 | Registry ↗ |
The newest 50 items, most recent first. Each row links to the study's page on ClinicalTrials.gov. Updated Nov 15, 2026, 6:51 PM EST.
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