Trial Outcomes & Findings for Study of Ataluren (PTC124) in Cystic Fibrosis (NCT NCT02107859)
NCT ID: NCT02107859
Last Updated: 2020-03-25
Results Overview
AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
TERMINATED
PHASE3
61 participants
Baseline (Day 1) up to end of study (Week 196)
2020-03-25
Participant Flow
Participants with nonsense mutation cystic fibrosis (nmCF) who had completed the double-blind study PTC124-GD-009-CF (NCT00803205) were enrolled and treated in this open-label extension study.
On 2 March 2017, it was announced that the Phase 3 double-blind study PTC124-GD-021-CF (NCT02139306) did not achieve its primary or secondary endpoints. Based on these results, clinical development of ataluren in cystic fibrosis was discontinued and this ongoing open-label extension study was closed.
Participant milestones
| Measure |
Ataluren
Participants received ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
|
|---|---|
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Overall Study
STARTED
|
61
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|
Overall Study
As-treated Population
|
61
|
|
Overall Study
Intent-to-treat (ITT) Population
|
60
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
61
|
Reasons for withdrawal
| Measure |
Ataluren
Participants received ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Overall Study
Withdrawal by Subject
|
14
|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Study closure
|
41
|
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Overall Study
Other than specified
|
3
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Baseline Characteristics
Study of Ataluren (PTC124) in Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Ataluren
n=61 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Age, Continuous
|
27.5 years
STANDARD_DEVIATION 10.73 • n=5 Participants
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Sex: Female, Male
Female
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34 Participants
n=5 Participants
|
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Sex: Female, Male
Male
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
60 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
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Race/Ethnicity, Customized
White-White/Caucasian
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61 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline (Day 1) up to end of study (Week 196)Population: As-treated population included all participants who received at least 1 dose of ataluren.
AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Outcome measures
| Measure |
Ataluren
n=61 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Any TEAEs
|
61 Participants
|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Mild AEs
|
4 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Moderate AEs
|
26 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Severe AEs
|
30 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Life-threatening AEs
|
0 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Fatal AEs
|
1 Participants
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|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs unrelated to ataluren
|
35 Participants
|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs unlikely related to ataluren
|
12 Participants
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs possible related to ataluren
|
13 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs probable related to ataluren
|
1 Participants
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Serious TEAEs
|
36 Participants
|
PRIMARY outcome
Timeframe: Baseline (Day 1) up to end of study (Week 196)Population: As-treated population included all participants who received at least 1 dose of ataluren.
Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.
Outcome measures
| Measure |
Ataluren
n=61 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Number of Participants With Clinically Significant Laboratory Abnormalities
|
0 Participants
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SECONDARY outcome
Timeframe: Baseline, Week 192Population: ITT population included all participants who had at least 1 post-baseline efficacy assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable at specified timepoint.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Percent of predicted FEV1 = (observed value)/(predicted value) \* 100%. Change from baseline in percent predicted FEV1 at the end of treatment was reported.
Outcome measures
| Measure |
Ataluren
n=59 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry
Baseline
|
56.203 percentage of predicted FEV1
Standard Deviation 17.2964
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|
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry
Change at Week 192
|
-1.214 percentage of predicted FEV1
Standard Deviation 3.6384
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SECONDARY outcome
Timeframe: Baseline up to Week 192Population: ITT population included all participants who had at least 1 post-baseline efficacy assessment.
The modified Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms without the requirement for treatment with antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature greater than (\>) 38 degrees celsius (°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.
Outcome measures
| Measure |
Ataluren
n=60 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria
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68.3 percentage of participants
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SECONDARY outcome
Timeframe: Baseline up to Week 192Population: ITT population included all participants who had at least 1 post-baseline efficacy assessment.
The expanded Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring any form of antibiotic treatment (inhaled, oral, or intravenous): 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.
Outcome measures
| Measure |
Ataluren
n=60 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria
|
68.3 percentage of participants
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SECONDARY outcome
Timeframe: Baseline up to Week 192Population: ITT population included all participants who had at least 1 post-baseline efficacy assessment.
The Classic Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring treatment with parenteral antibiotics: 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.
Outcome measures
| Measure |
Ataluren
n=60 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria
|
58.3 percentage of participants
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SECONDARY outcome
Timeframe: Baseline, Week 196Population: As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration.
Outcome measures
| Measure |
Ataluren
n=59 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Baseline: RR duration
|
828.17 miiliseconds
Standard Deviation 132.15
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Change at Week 196: RR duration
|
-14.27 miiliseconds
Standard Deviation 115.62
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Baseline: PR duration
|
145.02 miiliseconds
Standard Deviation 19.29
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Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Change at Week 196: PR duration
|
-2.69 miiliseconds
Standard Deviation 12.25
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Baseline: QRS duration
|
83.90 miiliseconds
Standard Deviation 8.00
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Change at Week 196: QRS duration
|
0.33 miiliseconds
Standard Deviation 6.12
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Baseline: QT duration
|
370.71 miiliseconds
Standard Deviation 28.11
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Change at Week 196: QT duration
|
-4.38 miiliseconds
Standard Deviation 25.63
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Baseline: QTCB duration
|
408.92 miiliseconds
Standard Deviation 22.05
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Change at Week 196: QTCB duration
|
-0.71 miiliseconds
Standard Deviation 17.96
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Baseline: QTCF duration
|
395.47 miiliseconds
Standard Deviation 19.23
|
|
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Change at Week 196: QTCF duration
|
-2.27 miiliseconds
Standard Deviation 17.13
|
SECONDARY outcome
Timeframe: Baseline, Week 196Population: As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories.
Heart rate was measured using 12-lead ECG.
Outcome measures
| Measure |
Ataluren
n=59 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
|
|---|---|
|
Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG
Baseline
|
74.31 beats/minute
Standard Deviation 12.06
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|
Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG
Change at Week 196
|
2.04 beats/minute
Standard Deviation 10.11
|
SECONDARY outcome
Timeframe: Baseline, Week 196Population: As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Number analyzed' signifies participants evaluable for specified categories.
Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP).
Outcome measures
| Measure |
Ataluren
n=61 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
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Change From Baseline in Vital Signs at Final Visit (Week 196)
Baseline: SBP
|
114.8 millimeters of mercury (mmHg)
Standard Deviation 9.10
|
|
Change From Baseline in Vital Signs at Final Visit (Week 196)
Change at Week 196: SBP
|
0.6 millimeters of mercury (mmHg)
Standard Deviation 12.62
|
|
Change From Baseline in Vital Signs at Final Visit (Week 196)
Baseline: DBP
|
71.2 millimeters of mercury (mmHg)
Standard Deviation 8.93
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|
Change From Baseline in Vital Signs at Final Visit (Week 196)
Change at Week 196: DBP
|
-0.3 millimeters of mercury (mmHg)
Standard Deviation 9.93
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 192Population: ITT population included all participants who had at least 1 post-baseline efficacy assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable at specified timepoint.
FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Change from baseline in percent predicted FVC at the end of treatment was reported.
Outcome measures
| Measure |
Ataluren
n=59 Participants
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
|
|---|---|
|
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry
Baseline
|
73.576 percentage of predicted FVC
Standard Deviation 14.6552
|
|
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry
Change at Week 192
|
-2.286 percentage of predicted FVC
Standard Deviation 4.5722
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 192Population: Due to change in planned analysis FEV25-75 was not calculated and summarized.
FEF25-75 is the forced expiratory flow between 25 and 75% of vital capacity.
Outcome measures
Outcome data not reported
Adverse Events
Ataluren
Serious adverse events
| Measure |
Ataluren
n=61 participants at risk
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
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|---|---|
|
Infections and infestations
Appendicitis
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Cellulitis
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Clostridium difficile infection
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Device related infection
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
44.3%
27/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
11.5%
7/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Constipation
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Distal ileal obstruction syndrome
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
4.9%
3/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Investigations
Blood creatine increased
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Investigations
Forced expiratory volume decreased
|
3.3%
2/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Investigations
Pulmonary function test decreased
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Renal and urinary disorders
Renal failure
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Renal and urinary disorders
Renal failure acute
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Congenital, familial and genetic disorders
Cystic fibrosis
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Hepatobiliary disorders
Gallbladder pain
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colon cancer
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Vascular disorders
Deep vein thrombosis
|
1.6%
1/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
Other adverse events
| Measure |
Ataluren
n=61 participants at risk
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
|
|---|---|
|
Infections and infestations
Gastroenteritis
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
82.0%
50/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Influenza
|
6.6%
4/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Nasopharyngitis
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Oral candidiasis
|
6.6%
4/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Sinusitis
|
9.8%
6/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Upper respiratory tract infection
|
14.8%
9/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
36.1%
22/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Abdominal pain
|
9.8%
6/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Constipation
|
11.5%
7/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Diarrhoea
|
13.1%
8/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Haemorrhoids
|
6.6%
4/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Nausea
|
9.8%
6/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Gastrointestinal disorders
Vomiting
|
6.6%
4/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.8%
9/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
19.7%
12/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
6.6%
4/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Immune system disorders
Drug hypersensitivity
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
|
Nervous system disorders
Headache
|
8.2%
5/61 • Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER