Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2022-000375-39 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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 available or the program is terminated.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vatiquinone | Experimental | Participants will receive vatiquinone oral solution (100 milligrams [mg]/milliliter [mL]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vatiquinone | Drug | Vatiquinone will be administered per dose and schedule specified in the arm description. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. | Baseline (Day 1) up to 30 days after last dose of study drug (956 days) |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Vinay Penematsa, MD | PTC Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, San Diego Altman Clinical and Translational Research Institute | La Jolla | California | 92037 | United States | ||
A total of 103 participants were screened, of which 101 participants were enrolled in the study and received at least 1 dose of vatiquinone.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Vatiquinone | Participants received vatiquinone oral solution (100 milligrams [mg]/milliliter [mL]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 25, 2023 | Nov 26, 2025 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Stanford University |
| Stanford |
| California |
| 94305 |
| United States |
| Yale Medicine | New Haven | Connecticut | 06511 | United States |
| Children's National | Washington D.C. | District of Columbia | 20010 | United States |
| Child Neurology Center of Northwest Florida | Gulf Breeze | Florida | 32561 | United States |
| The Johns Hopkins University | Baltimore | Maryland | 21205 | United States |
| Columbia University Medical Center - CUMC | New York | New York | 10032 | United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Children's Hospital of Philadelphia - CHOP | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina - MUSC | Charleston | South Carolina | 29425 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| UT Health The University of Texas | Houston | Texas | 77030 | United States |
| Seattle Children Hospital | Seattle | Washington | 98105 | United States |
| Chu Angers | Angers | 49933 | France |
| CHU Montpellier - Hopital Saint-Eloi | Montpellier | 34295 | France |
| Hopital Necker-Enfants Malades | Paris | 75015 | France |
| Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg | Strasbourg | 67000 | France |
| Ospedale Pediatrico Bambino Gesù | Roma | 00165 | Italy |
| PTC Clinical Site | Japanese City | Japan |
| Instytut Pomnik - Centrum Zdrowia Dziecka | Warsaw | 04-730 | Poland |
| Hospital Sant Joan de Déu | Barcelona | 08950 | Spain |
| Hospital Universitario 12 de Octubre | Madrid | 28003 | Spain |
| Hospital Ruber Internacional | Madrid | 28034 | Spain |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | WC1N 3JH | United Kingdom |
| The Newcastle Upon Tyne Hospitals NHS Foundation Trust | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Received At Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety population included all participants who received at least 1 dose of vatiquinone in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Vatiquinone | Participants received vatiquinone oral solution (100 milligrams [mg]/milliliter [mL]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. | Safety population included all participants who received at least 1 dose of vatiquinone in the study. | Posted | Count of Participants | Participants | Baseline (Day 1) up to 30 days after last dose of study drug (956 days) |
|
|
|
Baseline (Day 1) up to 30 days after last dose of study drug (956 days)
Safety population included all participants who received at least 1 dose of vatiquinone in the study.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Vatiquinone | Participants received vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube TID. | 7 | 101 | 45 | 101 | 59 | 101 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Fanconi syndrome | Congenital, familial and genetic disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Adrenal insufficiency | Endocrine disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Erosive oesophagitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumoperitoneum | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Death | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypothermia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Generalised oedema | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hepatic failure | Hepatobiliary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Streptococcal infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Postoperative ileus | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Mitochondrial cytopathy | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Autonomic nervous system imbalance | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Change in seizure presentation | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Myoclonus | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Optic neuritis | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Status epilepticus | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypercapnia | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pulmonary amyloidosis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Tracheostomy | Surgical and medical procedures | MedDRA 26.0 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
|
The study was terminated early as PTC discontinued clinical development of vatiquinone for this indication based on prior clinical study results.
The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the Sponsor for review. The Sponsor may consult with the PI to require changes to the communication or extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Patient Advocacy | PTC Therapeutics, Inc. | 1-866-562-4620 | medinfo@ptcbio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 4, 2025 | Nov 26, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D007888 | Leigh Disease |
| D002549 | Diffuse Cerebral Sclerosis of Schilder |
| D017237 | Mitochondrial Encephalomyopathies |
| D017241 | MELAS Syndrome |
| D017243 | MERRF Syndrome |
| D000069279 | Drug Resistant Epilepsy |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D015323 | Pyruvate Metabolism, Inborn Errors |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D028361 | Mitochondrial Diseases |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D017240 | Mitochondrial Myopathies |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D020191 | Myoclonic Epilepsies, Progressive |
| D004831 | Epilepsies, Myoclonic |
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D000073376 | Epileptic Syndromes |
Not provided
Not provided
| ID | Term |
|---|---|
| C571746 | alpha-tocotrienol quinone |
Not provided
Not provided
Not provided
| Black/African American |
|
| White |
|
| Other |
|
| Unknown |
|
| Missing |
|
| Not Reported |
|
| Unknown |
|
| Missing |
|