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Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

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ClinicalTrials.gov Identifier: NCT02139306
Recruitment Status : Completed
First Posted : May 15, 2014
Results First Posted : May 14, 2020
Last Update Posted : May 14, 2020
Sponsor:
Collaborators:
Cystic Fibrosis Foundation
ECFS-Clinical Trial Network (ECFS-CTN)
Information provided by (Responsible Party):
PTC Therapeutics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: Ataluren (PTC124®)
Drug: Placebo
Enrollment 279
Recruitment Details This study was conducted from 15 August 2014 to 02 November 2016.
Pre-assignment Details  
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation. Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Period Title: Overall Study
Started 140 139
Completed 127 125
Not Completed 13 14
Reason Not Completed
Withdrawal by Subject             4             6
Adverse Event             3             4
Physician Decision             0             1
Other Unspecified             2             3
Protocol Violation             4             0
Arm/Group Title Ataluren Placebo Total
Hide Arm/Group Description Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation. Total of all reporting groups
Overall Number of Baseline Participants 140 139 279
Hide Baseline Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 140 participants 139 participants 279 participants
22.0  (11.0) 22.0  (10.44) 22.0  (10.70)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 140 participants 139 participants 279 participants
Female
59
  42.1%
74
  53.2%
133
  47.7%
Male
81
  57.9%
65
  46.8%
146
  52.3%
1.Primary Outcome
Title Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48
Hide Description The FEV1 is the volume of air forcibly exhaled in one second and is measured using forced expiratory air spirometry. Change in ppFEV1 at Week 48 was defined as the average between the change from baseline at Week 40 and that at Week 48. Baseline for ppFEV1 was defined as an average of ppFEV1 at Screening (Weeks -4 to -1) and Baseline (Day 1) visits.
Time Frame From Baseline to Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
The intent-to-treat (ITT) population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 138 136
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Percentage of predicted FEV1
-1.396
(-2.7735 to -0.0180)
-1.992
(-3.3271 to -0.6576)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ataluren, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5336
Comments [Not Specified]
Method Mixed-model, repeated-measures
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.597
Confidence Interval (2-Sided) 95%
-1.2881 to 2.4813
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.9570
Estimation Comments [Not Specified]
2.Secondary Outcome
Title 48-week Rate of Pulmonary Exacerbations
Hide Description Pulmonary exacerbations were assessed using expanded Fuchs criteria. The expanded Fuchs exacerbation is defined as the presence of at least 4 of 12 Fuchs' signs and symptoms requiring treatment with any form of antibiotic treatment (inhaled, oral, or intravenous). Fuchs' signs and symptoms included increased cough; change in sputum volume, color, or consistency; new or increased hemoptysis; increased dyspnea during moderate or mild exertion, or at rest; sinus pain or tenderness; change in sinus discharge; malaise, fatigue, or lethargy; anorexia or weight loss; temperature above 38 degrees Celsius; change in findings on chest examination; relative 10% decrease in ppFEV1, and chest radiography results consistent with pulmonary infection. The 48-week rate was calculated as: 48-week rate = total number of events /treatment duration by week*48.
Time Frame Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 138 136
Mean (Standard Deviation)
Unit of Measure: number of exacerbations per 48 weeks
0.950  (1.4038) 1.127  (2.5241)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ataluren, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4008
Comments [Not Specified]
Method Negative binomial regression
Comments [Not Specified]
Method of Estimation Estimation Parameter Rate ratio
Estimated Value 0.8567
Confidence Interval (2-Sided) 95%
0.5973 to 1.2288
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.1577
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48
Hide Description The teen/adult CFQ-R was used for this study. It was developed specifically for participants with cystic fibrosis. It is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being, and symptoms. The respiratory domain assessed respiratory symptoms like coughing, congestion, wheezing etc. Scaling of each item is done via 4-point Likert scales. Scores for each item are summed up to generate a domain score. Scores ranges from 0 to 100, with higher scores indicating better health and lower scores indicating worse health.
Time Frame Baseline (Day 1) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 138 136
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-0.760
(-3.4566 to 1.9364)
-1.032
(-3.7368 to 1.6728)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ataluren, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8881
Comments [Not Specified]
Method Mixed-model, repeated measures
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.272
Confidence Interval (2-Sided) 95%
-3.5292 to 4.0731
Parameter Dispersion
Type: Standard Error of the Mean
Value: 1.9300
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in Body Mass Index (BMI) at Week 48
Hide Description Malnutrition is common in participants with cystic fibrosis. The BMI is an important clinical measure of nutritional status.
Time Frame Baseline (Day 1) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 138 136
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kilogram per meter square (kg/m^2)
0.296
(0.1126 to 0.4789)
0.361
(0.1759 to 0.5455)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ataluren, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6208
Comments [Not Specified]
Method Mixed-model, repeated measures
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.065
Confidence Interval (2-Sided) 95%
-0.3233 to 0.1934
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.1312
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)
Hide Description An adverse event (AE) is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study treatment, whether or not considered related to the study treatment. A TEAE is defined as an AE that occurs or worsens in the period extending from first dose of study drug to 4 weeks after last dose of study drug. An SAE is an untoward medical occurrence or effect associated with the use of a study drug at any dose, regardless of whether it is considered to be related to the study drug, which results in one of the following: death; inpatient hospitalization or prolongation of existing hospitalization; life threatening adverse event; persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions; any other medically important event; or a pregnancy resulting in spontaneous abortion, stillbirth, neonatal death, or congenital anomaly.
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 140 139
Measure Type: Number
Unit of Measure: participants
TEAEs 133 135
SAEs 40 46
6.Secondary Outcome
Title Number of Participants With TEAEs by Severity and Relationship to Study Drugs
Hide Description The relationship of TEAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 140 139
Measure Type: Number
Unit of Measure: participants
Severity: Grade 1 26 18
Severity: Grade 2 88 88
Severity: Grade 3 19 29
Severity: Grade 4 0 0
Severity: Grade 5 0 0
Relationship to study drug: Unrelated 74 72
Relationship to study drug: Unlikely related 37 34
Relationship to study drug: Possible related 21 25
Relationship to study drug: Probable related 1 4
7.Secondary Outcome
Title Number of Participants With SAEs by Severity and Relationship to Study Drugs
Hide Description The relationship of SAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of SAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 140 139
Measure Type: Number
Unit of Measure: participants
Severity: Grade 1 2 1
Severity: Grade 2 23 20
Severity: Grade 3 15 25
Severity: Grade 4 0 0
Severity: Grade 5 0 0
Relationship to study drug: Unrelated 26 31
Relationship to study drug: Unlikely related 13 15
Relationship to study drug: Possible related 1 0
Relationship to study drug: Probable related 0 0
8.Secondary Outcome
Title Number of Participants With Abnormal Vital Signs Reported as TEAEs
Hide Description Vital signs included systolic and diastolic blood pressure, pulse rate, pulse oximetry, and body temperature. Participants with abnormal vital signs who required clinical intervention or further investigation (beyond ordering a repeat [confirmatory] test) unless they are associated with an already reported clinical event are reported.
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 140 139
Measure Type: Number
Unit of Measure: participants
0 1
9.Secondary Outcome
Title Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs
Hide Description Clinical laboratory tests included haematology, biochemistry, and urinalysis. Participants with abnormal laboratory parameters who required clinical intervention or further investigation (beyond ordering a repeat [confirmatory] test) unless they are associated with an already reported clinical event are reported.
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 140 139
Measure Type: Number
Unit of Measure: participants
32 30
10.Secondary Outcome
Title Number of Participants With Abnormal Electrocardiogram Reported as TEAEs
Hide Description Participants with abnormal electrocardiogram who required clinical intervention or further investigation (beyond ordering a repeat [confirmatory] test) unless they are associated with an already reported clinical event are reported.
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The as-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description:
Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.
Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
Overall Number of Participants Analyzed 140 139
Measure Type: Number
Unit of Measure: participants
1 0
Time Frame From study drug administration to 4-week post treatment follow-up visit (approximately 52 Weeks)
Adverse Event Reporting Description All randomized participants who actually received any study treatment were analysed for AEs and SAEs.
 
Arm/Group Title Ataluren Placebo
Hide Arm/Group Description Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation. Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.
All-Cause Mortality
Ataluren Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/140 (0.00%)      0/139 (0.00%)    
Hide Serious Adverse Events
Ataluren Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   40/140 (28.57%)      46/139 (33.09%)    
Congenital, familial and genetic disorders     
Pseudocholinesterase deficiency  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Gastrointestinal disorders     
Constipation  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Distal intestinal obstruction syndrome  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Small intestinal obstruction  1  1/140 (0.71%)  1 0/139 (0.00%)  0
General disorders     
Thrombosis in device  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Infections and infestations     
Infective pulmonary exacerbation of cystic fibrosis  1  25/140 (17.86%)  36 34/139 (24.46%)  50
Pseudomonas infection  1  2/140 (1.43%)  2 0/139 (0.00%)  0
Aspergillus infection  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Bronchopneumonia  1  1/140 (0.71%)  1 1/139 (0.72%)  2
Bronchopulmonary aspergillosis allergic  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Device related sepsis  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Peritonitis  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Pneumonia  1  1/140 (0.71%)  1 2/139 (1.44%)  2
Pneumonia influenzal  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Respiratory tract infection  1  1/140 (0.71%)  2 0/139 (0.00%)  0
Gastroenteritis  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Mycobacterium abscessus infection  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Pneumonia staphylococcal  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Respiratory tract infection fungal  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Respiratory tract infection viral  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Sinusitis  1  0/140 (0.00%)  0 2/139 (1.44%)  2
Investigations     
Pulmonary function test decreased  1  1/140 (0.71%)  3 0/139 (0.00%)  0
Forced expiratory volume decreased  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Metabolism and nutrition disorders     
Diabetes mellitus  1  1/140 (0.71%)  1 1/139 (0.72%)  1
Musculoskeletal and connective tissue disorders     
Costochondritis  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Nervous system disorders     
Headache  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Syncope  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Psychiatric disorders     
Suicidal ideation  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Renal and urinary disorders     
Nephrolithiasis  1  3/140 (2.14%)  3 0/139 (0.00%)  0
Renal failure acute  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Haemoptysis  1  4/140 (2.86%)  4 1/139 (0.72%)  2
Cough  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Nasal polyps  1  1/140 (0.71%)  1 1/139 (0.72%)  1
Tachypnoea  1  1/140 (0.71%)  1 0/139 (0.00%)  0
Bronchopneumopathy  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Pneumothorax spontaneous  1  0/140 (0.00%)  0 1/139 (0.72%)  1
Skin and subcutaneous tissue disorders     
Red man syndrome  1  1/140 (0.71%)  1 0/139 (0.00%)  0
1
Term from vocabulary, MedDRA (17.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ataluren Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   123/140 (87.86%)      120/139 (86.33%)    
Gastrointestinal disorders     
Diarrhoea  1  13/140 (9.29%)  15 12/139 (8.63%)  20
Nausea  1  11/140 (7.86%)  14 10/139 (7.19%)  12
Abdominal pain  1  11/140 (7.86%)  15 5/139 (3.60%)  6
Vomiting  1  8/140 (5.71%)  8 4/139 (2.88%)  7
General disorders     
Pyrexia  1  9/140 (6.43%)  17 10/139 (7.19%)  12
Infections and infestations     
Infective pulmonary exacerbation of cystic fibrosis  1  75/140 (53.57%)  142 78/139 (56.12%)  145
Viral upper respiratory tract infection  1  27/140 (19.29%)  43 21/139 (15.11%)  25
Upper respiratory tract infection  1  21/140 (15.00%)  25 21/139 (15.11%)  26
Sinusitis  1  16/140 (11.43%)  20 11/139 (7.91%)  11
Nasopharyngitis  1  10/140 (7.14%)  13 8/139 (5.76%)  10
Rhinitis  1  10/140 (7.14%)  12 8/139 (5.76%)  9
Influenza  1  7/140 (5.00%)  7 8/139 (5.76%)  9
Pharyngitis  1  7/140 (5.00%)  8 2/139 (1.44%)  3
Pseudomonas infection  1  4/140 (2.86%)  4 9/139 (6.47%)  10
Staphylococcal infection  1  4/140 (2.86%)  4 8/139 (5.76%)  9
Musculoskeletal and connective tissue disorders     
Back pain  1  7/140 (5.00%)  7 3/139 (2.16%)  7
Nervous system disorders     
Headache  1  12/140 (8.57%)  23 16/139 (11.51%)  18
Respiratory, thoracic and mediastinal disorders     
Cough  1  25/140 (17.86%)  36 25/139 (17.99%)  31
Haemoptysis  1  12/140 (8.57%)  17 10/139 (7.19%)  22
1
Term from vocabulary, MedDRA (17.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Joseph McIntosh
Organization: PTC Therapeutics, Inc
Phone: 908 912-9138
EMail: jmcintosh@ptcbio.com
Layout table for additonal information
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02139306    
Other Study ID Numbers: PTC124-GD-021-CF
2013-004581-34 ( EudraCT Number )
First Submitted: May 13, 2014
First Posted: May 15, 2014
Results First Submitted: May 30, 2017
Results First Posted: May 14, 2020
Last Update Posted: May 14, 2020