PTC Therapeutics Clinical Trials: 64 Led Studies

Archive historyOct 2022 to Apr 2026

69 recorded changes across PTC Therapeutics trials

Every status move, completion date shift and enrollment change ComplyRx has recorded across 18 of these trials. One is shown below.

Status changeStatus moved to completed from enrolling by invitationNCT01247207 · recorded 1 Apr 2026

Track to read them

+65 more recorded changes · Track a drug to see every change the night it lands

Start tracking drugs

Most active programmes

The drugs appearing most often across PTC Therapeutics's recently active trials. Comparator arms are excluded.

Ataluren24 trials
Deflazacort8 trials
Vatiquinone6 trials
PTC2995 trials
PTC9233 trials
PTC1243 trials

Upcoming readouts

Next primary completion dates among PTC Therapeutics's active phase 2 and phase 3 trials. Dates are registry estimates and move often, which is exactly what the tracker watches.

Feb 2031EPIPHENY: A Study of Sepiapterin in Participants With Phenylketonuria (PKU)Phase 3

Top therapeutic areas

Conditions studied most often across PTC Therapeutics's recently active trials.

Duchenne Muscular Dystrophy14 trials
Cystic Fibrosis9 trials
Muscular Dystrophy, Duchenne4 trials
Becker Muscular Dystrophy4 trials
Phenylketonuria3 trials
Friedreich Ataxia3 trials

PTC Therapeutics trials

The 50 most significant of PTC Therapeutics's recently active trials, soonest estimated readout first.
Select any row for the full record; the change timeline unlocks with tracking.

Trial	Phase	Stability	Status	Enrollment	Est. readout
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich AtaxiaNCT05515536Vatiquinone	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	130 est.	31 Dec 2027
PROVE-FALong-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)NCT07681713Vatiquinone	Phase 3	Not yet scored	OngoingNot yet recruiting	120 est.	15 Mar 2029
EPIPHENYA Study of Sepiapterin in Participants With Phenylketonuria (PKU)NCT06302348Sepiapterin	Phase 3	Locked · track to reveal	OngoingRecruiting	56 est.	28 Feb 2031
A Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaNCT05166161PTC923	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	200 est.	30 Jun 2026
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)NCT01247207Ataluren	Phase 3	Locked · track to reveal	Completed	270 est.	10 Feb 2026
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseNCT05218655Vatiquinone	Phase 3	Locked · track to reveal	Completed	101 est.	15 Apr 2025
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT02369731	—	Not yet scored	Completed	316 est.	31 Mar 2025
CARDINALSStudy to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALSNCT05349721PTC857	Phase 2	Locked · track to reveal	StoppedTerminated	336 est.	26 Sept 2024
A Study of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaNCT05485987Vatiquinone	Phase 2	Locked · track to reveal	Completed	5 est.	29 Aug 2024
SUNRISELMSA Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)NCT05269355Unesbulin	Phase 2	Locked · track to reveal	StoppedTerminated	359 est.	17 Jun 2024
A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)NCT03761095Unesbulin	Phase 1	Locked · track to reveal	Completed	41 est.	5 Feb 2024
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh SyndromeNCT02352896EPI-743	Phase 2	Locked · track to reveal	Completed	30 est.	30 Oct 2023
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)NCT04336826Ataluren	Phase 2	Locked · track to reveal	Completed	6 est.	7 Aug 2023
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric ParticipantsNCT04903288Eladocagene Exuparvovec	Phase 2	Locked · track to reveal	OngoingActive, not recruiting	13 est.	22 May 2023
MOVE-FAA Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaNCT04577352Vatiquinone	Phase 2	Locked · track to reveal	Completed	146 est.	4 Apr 2023
A Study of PTC923 in Participants With PhenylketonuriaNCT05099640PTC923	Phase 3	Locked · track to reveal	Completed	157 est.	3 Apr 2023
MIT-EA Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyNCT04378075Vatiquinone	Phase 2	Locked · track to reveal	StoppedTerminated	68 est.	18 Mar 2023
FITE19A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)NCT04439071PTC299	Phase 2	Locked · track to reveal	StoppedTerminated	189 est.	20 Jul 2022
Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyNCT03179631Ataluren	Phase 3	Locked · track to reveal	Completed	360 est.	5 Mar 2022
Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute LeukemiasNCT03761069PTC299	Phase 1	Locked · track to reveal	StoppedTerminated	33 est.	28 Dec 2021
PTCEMFA Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)NCT03642145Deflazacort	Phase 3	Locked · track to reveal	StoppedWithdrawn	0 est.	31 Jul 2021
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation AniridiaNCT04117880Ataluren	Phase 2	Locked · track to reveal	StoppedWithdrawn	0 est.	31 Jan 2021
STARStudy of Ataluren in Participants With Nonsense Mutation AniridiaNCT02647359Ataluren	Phase 2	Locked · track to reveal	Completed	39 est.	22 Jan 2021
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)NCT03783923Deflazacort	Phase 3	Locked · track to reveal	StoppedTerminated	11 est.	1 Jan 2021
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT03648827Ataluren	Phase 2	Locked · track to reveal	Completed	20 est.	23 Oct 2020
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaNCT03519711PTC923	Phase 1	Locked · track to reveal	Completed	8 est.	2 Oct 2020
A Study of CNSA-001 in Women With Diabetic GastroparesisNCT03712124CNSA-001	Phase 2	Locked · track to reveal	Completed	21 est.	8 Sept 2019
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenNCT03796637Ataluren	Phase 2	Locked · track to reveal	Completed	6 est.	3 Jun 2019
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyNCT02090959Ataluren	Phase 3	Locked · track to reveal	StoppedTerminated	219 est.	12 Jun 2018
EPI-589Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)NCT02460679EPI-589	Phase 2	Locked · track to reveal	Completed	19 est.	23 Feb 2018
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyNCT02819557Ataluren	Phase 2	Locked · track to reveal	Completed	14 est.	9 Feb 2018
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaNCT01557400Ataluren	Phase 3	Locked · track to reveal	Completed	94 est.	19 Jan 2018
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortNCT02295748Deflazacort	Phase 1	Locked · track to reveal	Completed	24 est.	24 Aug 2017
Study of Ataluren (PTC124) in Cystic FibrosisNCT02107859Ataluren	Phase 3	Locked · track to reveal	StoppedTerminated	61 est.	5 Jun 2017
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisNCT02456103Ataluren	Phase 3	Locked · track to reveal	StoppedTerminated	246 est.	2 Jun 2017
PTC596 in Patients With Advanced Solid TumorsNCT02404480PTC596	Phase 1	Locked · track to reveal	Completed	31 est.	6 Feb 2017
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)NCT02139306Ataluren (PTC124®)	Phase 3	Locked · track to reveal	Completed	279 est.	Nov 2016
Safety and Efficacy of EPI-743 in Patients With Friedreich's AtaxiaNCT01728064EPI-743 400 mg	Phase 2	Locked · track to reveal	Completed	60 est.	31 Oct 2015
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyNCT02251600Deflazacort	Phase 1	Locked · track to reveal	Completed	24 est.	Oct 2015
ACT DMDPhase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)NCT01826487Ataluren	Phase 3	Locked · track to reveal	Completed	230 est.	20 Aug 2015
Food Effect and Bioavailability of Deflazacort Formulations in Healthy VolunteersNCT02485431Deflazacort	Phase 1	Locked · track to reveal	Completed	45 est.	Aug 2015
Effects of Rifampin on the Pharmacokinetics of AtalurenNCT02409004Ataluren	Phase 1	Locked · track to reveal	Completed	15 est.	Mar 2015
Safety and Efficacy Study of EPI-743 in Children With Leigh SyndromeNCT01721733EPI-743 15 mg/kg	Phase 2	Locked · track to reveal	Completed	35 est.	28 Feb 2015
A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort PharmacokineticsNCT02286609Deflazacort	Phase 1	Locked · track to reveal	Completed	16 est.	Feb 2015
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort PharmacokineticsNCT02286622Deflazacort	Phase 1	Locked · track to reveal	Completed	16 est.	Feb 2015
Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of DeflazacortNCT02286635Deflazacort and rifampin	Phase 1	Locked · track to reveal	Completed	58 est.	Dec 2014
Extension Study of Ataluren (PTC124) in Cystic FibrosisNCT01140451Ataluren	Phase 3	Locked · track to reveal	Completed	191 est.	2 Dec 2013
PTC299 for Treatment of Neurofibromatosis Type 2NCT00911248PTC299	Phase 2	Locked · track to reveal	StoppedTerminated	11 est.	31 Mar 2012
PTC299 and Hormonal Agent for Treatment of Metastatic Breast CancerNCT00508586PTC299	Phase 1	Locked · track to reveal	Completed	33 est.	Mar 2012
PTC299 for Treatment of Advanced CancerNCT00704821PTC299	Phase 1	Locked · track to reveal	StoppedTerminated	76 est.	24 Feb 2012

View all trials64

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Phase 3CompletedNCT01247207 ↗

Primary completion: Feb 2026; actual
Enrollment: 270; estimated
Start: Nov 2010; PTC Therapeutics

Conditions

Duchenne Muscular Dystrophy

Interventions

Ataluren

Brief summary

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed…

Full record on ClinicalTrials.gov ↗

Change timeline

4 recorded · latest 1 Apr 2026

Completion date move · 3

Status change · 1

Track PTC Therapeutics drugs to see every change the night it lands.

Start tracking drugs

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Phase 3CompletedNCT05218655 ↗

Primary completion: Apr 2025; actual
Enrollment: 101; estimated
Start: Jun 2022; PTC Therapeutics

Conditions

Inherited Mitochondrial Disease

Interventions

Vatiquinone

Brief summary

The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone becomes commercially…

Full record on ClinicalTrials.gov ↗

Change timeline

7 recorded · latest 1 Oct 2025

Status change · 3

Completion date move · 2

Enrollment change · 2

Track PTC Therapeutics drugs to see every change the night it lands.

Start tracking drugs

A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)

Phase 1CompletedNCT03761095 ↗

Primary completion: Feb 2024; actual
Enrollment: 41; estimated
Start: Mar 2019; PTC Therapeutics

Conditions

Leiomyosarcoma

Interventions

Unesbulin
Dacarbazine

Brief summary

The primary objective of this study is to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of unesbulin in combination with dacarbazine for the treatment of advanced LMS and determine the overall safety profile of unesbulin in combination with dacarbazine. This study will employ the…

Full record on ClinicalTrials.gov ↗

Change timeline

6 recorded · latest 1 Apr 2024

Completion date move · 3

Status change · 2

Enrollment change · 1

Track PTC Therapeutics drugs to see every change the night it lands.

Start tracking drugs

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)

Phase 2CompletedNCT04336826 ↗

Primary completion: Aug 2023; actual
Enrollment: 6; estimated
Start: Dec 2021; PTC Therapeutics

Conditions

Nonsene Mutation Duchenne Muscular Dystrophy

Interventions

Ataluren

Brief summary

This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to \<2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

Full record on ClinicalTrials.gov ↗

Change timeline

4 recorded · latest 1 Oct 2023

Completion date move · 2

Enrollment change · 1

Status change · 1

Track PTC Therapeutics drugs to see every change the night it lands.

Start tracking drugs

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

Phase 2Active, not recruitingNCT04903288 ↗

Primary completion: May 2023; actual
Enrollment: 13; estimated
Start: May 2021; PTC Therapeutics

Conditions

AADC Deficiency

Interventions

Eladocagene Exuparvovec

Brief summary

This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of homovanillic acid (HVA) levels and to assess the safety of the SmartFlow® magnetic resonance (MR)…

Full record on ClinicalTrials.gov ↗

Change timeline

4 recorded · latest 1 Apr 2024

Enrollment change · 2

Status change · 1

Completion date move · 1

Track PTC Therapeutics drugs to see every change the night it lands.

Start tracking drugs

MIT-E: A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Phase 2TerminatedNCT04378075 ↗

Primary completion: Mar 2023; actual
Enrollment: 68; estimated
Start: Sept 2020; PTC Therapeutics

Conditions

Mitochondrial Diseases
Drug Resistant Epilepsy
Leigh Disease
Leigh Syndrome
Mitochondrial Encephalopathy (MELAS)
Pontocerebellar Hypoplasia Type 6 (PCH6)
Alpers Disease
Alpers Syndrome

Interventions

Vatiquinone
Placebo

Brief summary

This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week,…

Full record on ClinicalTrials.gov ↗

Change timeline

6 recorded · latest 1 Oct 2024

Status change · 3

Completion date move · 2

Enrollment change · 1

Track PTC Therapeutics drugs to see every change the night it lands.

Start tracking drugs

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Phase 3TerminatedNCT02090959 ↗

Primary completion: Jun 2018; actual
Enrollment: 219; estimated
Start: Mar 2014; PTC Therapeutics

Conditions

Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked

Interventions

Ataluren

Brief summary

The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \[PTC124-GD-020-DMD {NCT01826487}\]) to augment the overall safety database. Screening and baseline…

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Phase 3CompletedNCT01557400 ↗

Primary completion: Jan 2018; actual
Enrollment: 94; estimated
Start: May 2012; PTC Therapeutics

Conditions

Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Dystrophinopathy

Interventions

Ataluren

Brief summary

Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability…

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.

ACT DMD: Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Phase 3CompletedNCT01826487 ↗

Primary completion: Aug 2015; actual
Enrollment: 230; estimated
Start: Mar 2013; PTC Therapeutics

Conditions

Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked

Interventions

Ataluren
Placebo

Brief summary

Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss…

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.