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Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints.
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This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.
The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ataluren | Experimental | Participants will be administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ataluren | Drug | Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section. | Baseline up to Week 100 |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter | Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section. | Baseline up to Week 100 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24 | Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEV1 was calculated as follows: (percent-predicted FEV1 - Baseline percent-predicted FEV1/Baseline percent-predicted FEV1)*100. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Joseph McIntosh, MD | PTC Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Miller Children's Hospital Long Beach |
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All eligible participants, including those who received placebo in the double-blind study (PTC124-GD-021-CF; NCT02139306), were enrolled to this open-label extension study. To avoid interruption in treatment, when possible, Screening/Baseline for this extension study was to occur on the same day as the End-of-Study visit for the double-blind study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ataluren | Participants were administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 27, 2015 | Apr 1, 2020 |
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| Baseline, Week 24 |
| Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24 | Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FVC was calculated as follows: (percent-predicted FVC - Baseline percent-predicted FVC/Baseline percent-predicted FVC)*100. | Baseline, Week 24 |
| Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24 | Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: (percent-predicted FEF25-75 - Baseline percent-predicted FEF25-75/Baseline percent-predicted FEF25-75)*100. | Baseline, Week 24 |
| Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | A modified Fuchs' exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. The 48-week rate = (the total number of events/ treatment duration by week)*48. | Baseline up to Week 48 |
| Long Beach |
| California |
| 90806 |
| United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Children's Hospital and Research Center at Oakland | Oakland | California | 94609 | United States |
| Stanford University-Children's Hospital | Palo Alto | California | 94304 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Nemours Children's Clinic | Jacksonville | Florida | 32207 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| Miami Children's Hospital | Miami | Florida | 33155 | United States |
| All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Morristown Medical Center | Morristown | New Jersey | 07960 | United States |
| Beth Israel Medical Center | New York | New York | 10011 | United States |
| New York University Langone Medical Center | New York | New York | 10016 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| University of Cincinnati | Cincinnati | Ohio | 45221 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Drexel University College of Medicine | Philadelphia | Pennsylvania | 19129 | United States |
| Texas Children's Hospital | Houston | Texas | 77094 | United States |
| University of Texas Health Science Center | Tyler | Texas | 75708 | United States |
| Childrens Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Hospital de Ninos Ricardo Gutierrez | Buenos Aires | 1330 | Argentina |
| Hospital Universitario Austral | Buenos Aires | Argentina |
| Royal Adelaide Hospital | Adelaide | 5000 | Australia |
| Prince Charles Hospital | Chermside | 4032 | Australia |
| Princess Margaret Hospital | Perth | 6840 | Australia |
| University Hospital Brussels | Brussels | 1090 | Belgium |
| Hopital Universitaire des Enfants Reine Fabiola | Brussels | 15 | Belgium |
| University Hospital Leuven | Leuven | 3000 | Belgium |
| Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul | Porto Alegre | Brazil |
| University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD | Plovdiv | Bulgaria |
| University Multiprofile Hospital for Active Treatment Aleksandrovska EAD | Sofia | Bulgaria |
| Clinical Research Institute of Montreal | Montreal | H2W 1R7 | Canada |
| University of Toronto Hospital for Sick Children | Toronto | M5G 1X8 | Canada |
| British Columbia Children's Hospital | Vancouver | V6H 3V4 | Canada |
| Hoptial Arnaud de Villeneuve | Montpellier | 34295 | France |
| Hopital Necker - Enfants Malades | Paris | 75015 | France |
| Centre de Perharidy | Roscoff | 29684 | France |
| Centre Hospitalier Regional Sud Reunion | Saint-Pierre | 97448 | France |
| Charite-Universitatsmedizin Berlin | Berlin | 10117 | Germany |
| St. Josef Hospital GmbH | Bochum | 44791 | Germany |
| University of Cologne Children's Hospital | Cologne | 50937 | Germany |
| Christiane Herzog CF-Zentrum | Frankfurt am Main | 60590 | Germany |
| Universitatsklinikum Jena | Jena | 1 | Germany |
| LMU Klinikum der Universitat Muchen | Munich | 80539 | Germany |
| Dr. Von Haunersches Kinderspital | München | 80337 | Germany |
| Ippokratio General Hospital Of Thessaloniki | Thessaloniki | 541 | Greece |
| Meyer Children's Hospital | Haifa | 31096 | Israel |
| Hadassah University Hospital | Jerusalem | 91240 | Israel |
| Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi | Ancona | 71 | Italy |
| Azienda Ospedaliera A Meyer | Florence | 50139 | Italy |
| Lombardia Cystic Fibrosis Center | Milan | 20122 | Italy |
| Azienda Policlinico Umberto I | Rome | 00161 | Italy |
| Ospedale Pediatrico Bambino Gesu | Rome | 4 | Italy |
| Azienda Ospedaliera di Verona | Verona | Italy |
| Hagaziekenhuis | s-Gravenweg | South Holland | 2545 CH | Netherlands |
| Radboud University | Nijmegen | 6525 GA | Netherlands |
| Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku | Gdansk | Poland |
| Institute of Mother and Child | Warsaw | 01-211 | Poland |
| Hospital Universitario Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital University | Barcelona | 08035 | Spain |
| Hospital San Juan | Esplugues de Llobregat | 08950 | Spain |
| Hospital Regional Universitario de Malaga | Málaga | 29001 | Spain |
| Hospital de Sabadell, Consorci Sanitari Parc Tauli | Sabadell | 08208 | Spain |
| Hospital Universitario Virgen del Rocio | Seville | 41013 | Spain |
| St James University Hospital | Leeds | LS9 7TF | United Kingdom |
| Royal Brompton Hospital | London | SW3 6NP | United Kingdom |
| As-Treated Population | Participants who received at least 1 dose of ataluren. |
|
| Intent-to-treat (ITT) Population | Participants in the As-Treated Population with at least 1 postbaseline efficacy assessment. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Participants who received at least 1 dose of ataluren (As-Treated Population).
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Ataluren | Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks. |
| 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 | Number | 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) | TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section. | Participants who received at least 1 dose of ataluren (As-Treated Population). | Posted | Count of Participants | Participants | Baseline up to Week 100 |
|
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter | Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section. | Participants who received at least 1 dose of ataluren (As-Treated Population). | Posted | Count of Participants | Participants | Baseline up to Week 100 |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24 | Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEV1 was calculated as follows: (percent-predicted FEV1 - Baseline percent-predicted FEV1/Baseline percent-predicted FEV1)*100. | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEV1 data. | Posted | Mean | Standard Deviation | percentage of predicted FEV1 | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24 | Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FVC was calculated as follows: (percent-predicted FVC - Baseline percent-predicted FVC/Baseline percent-predicted FVC)*100. | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FVC data. | Posted | Mean | Standard Deviation | percentage of predicted FVC | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24 | Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: (percent-predicted FEF25-75 - Baseline percent-predicted FEF25-75/Baseline percent-predicted FEF25-75)*100. | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEF25-75 data. | Posted | Mean | Standard Deviation | percentage of FEF25-75 | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | A modified Fuchs' exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. The 48-week rate = (the total number of events/ treatment duration by week)*48. | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population). | Posted | Mean | Standard Deviation | exacerbations | Baseline up to Week 48 |
|
|
Baseline up to Week 100
Adverse events collected from participants who received at least 1 dose of ataluren (As-Treated Population).
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 | Ataluren | Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks. | 1 | 245 | 89 | 245 | 191 | 245 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchopulmonary aspergillosis allergic | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Mycobacterium abscessus infection | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Viral pericarditis | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Duodenal ulcer | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | 17.0 | Systematic Assessment |
| |
| Protein urine present | Investigations | 17.0 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | 17.0 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | 17.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | 17.0 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | 17.0 | Systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | 17.0 | Systematic Assessment |
| |
| Cystic fibrosis lung | Congenital, familial and genetic disorders | 17.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | 17.0 | Systematic Assessment |
| |
| Squamous cell carcinoma of the cervix | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 17.0 | Systematic Assessment | This is a sex-specific adverse event that only affects female participants. |
|
| Depressed level of consciousness | Nervous system disorders | 17.0 | Systematic Assessment |
| |
| Pregnancy | Pregnancy, puerperium and perinatal conditions | 17.0 | Systematic Assessment | This is a sex-specific adverse event that only affects female participants. |
|
| Appendicitis | Infections and infestations | 17.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | 17.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 17.0 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | 17.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | 17.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | 17.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | 17.0 | Systematic Assessment |
|
This study was terminated early because the CF data from the double-blind CF Study PTC124-GD-021-CF (NCT02139306) did not meet endpoints.
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 | Jul 14, 2017 | Apr 1, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C515878 | ataluren |
Not provided
Not provided
Not provided
| White-Arabic/North African Heritage |
|
| Non-White |
|
| Hispanic or Latino |
|
| Not Hispanic or Latino |
|
| Title | Measurements |
|---|---|
|
| Severe TEAE |
|
| Life-Threatening TEAE |
|
| Fatal TEAE |
|
| Serious TEAE |
|
| TEAE Unrelated to Study Drug |
|
| TEAE Unlikely Related to Study Drug |
|
| TEAE Possibly Related to Study Drug |
|
| TEAE Probably Related to Study Drug |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
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|
|
|
|