Trial · NCT01140451
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT01140451, Phase 3, Completed, PTC Therapeutics; results first posted 2020-10-19
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Extension Study of Ataluren (PTC124) in Cystic Fibrosis. Trials, record NCT01140451. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01140451
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study of Ataluren (PTC124) in Cystic Fibrosis
- NCT number
- NCT01140451
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 191 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren
- First posted
- 2010-06-09
- Last updated
- 2020-10-19
- Other registries' IDs
- eudract:2010-019692-30
- Results first posted
- 2020-10-19
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Baseline (Week 1 [Total Study Week 48]) up to 4 Weeks Post-Treatment (Week 100 [Total Study Week 148]) or Premature Discontinuation (PD) (whichever occurred first) | A TEAE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship that occurred or worsened in the period extending from the first dose of study drug to 6 weeks after the last dose of study drug. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. AE severity was graded as follows: Grade 1: mild; Grade 2: moderate; Grade 3: severe; Grade 4: life-threatening; Grade 5: fatal. A TEAE was considered related if in the opinion of the Investigator it was possibly or probably caused by the study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the Adverse Events module. |
| Primary | Number of Participants With Any Treatment-Emergent Laboratory Abnormality (TELA) | Baseline (Week 1 [Total Study Week 48]) up to 4 Weeks Post-Treatment (Week 100 [Total Study Week 148]) or PD (whichever occurred first) | A TELA is any abnormal laboratory value that started or worsened after administration of study drug. Abnormal values were defined as values outside normal range. Values considered abnormal included -Hepatic: Serum total bilirubin ≥1.5\*upper limit of normal (ULN); serum gamma glutamyl transferase \>2.5\*ULN; serum alanine aminotransferase increase of \>150 units/liter (U/L) without increased creatine kinase; -Adrenal: plasma adrenocorticotropic hormone \>ULN (normal cortisol); -Renal: serum cystatin C \>1.33 milligrams (mg)/L; serum creatinine \>ULN-1.5\*ULN for age; serum blood urea nitrogen ≥1.5\*ULN; urine protein:creatinine \>0.40 mg/deciliter (dL):mg/dL; urine protein:osmolality \>0.30 mg/L:milliosmoles/kilogram; urine blood 2+; - Serum Electrolytes: serum sodium \>150 millimoles (mmol)/L, \<130 mmol/L; serum potassium \>5.5, \<3.0 mmol/L; serum magnesium \>1.23 mmol/L, \<0.5 mmol/L; total serum calcium \>2.9 mmol/L, \<2.0 mmol/L; serum phosphorous \<0.8 mmol/L; serum biocarbonate- \<16 mmol/L. |
| Secondary | Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in 1 Second (FEV1) at Baseline | Baseline (Week 1 [Total Study Week 48]) | Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the American Thoracic Society (ATS) and European Respiratory Society (ERS). Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e. |
| Secondary | Percentage Change From Baseline in Percent-Predicted of FEV1 at Weeks 48 and 96 | Week 48 (Total Study Week 96), End of Treatment (EOT) (Week 96 [Total Study Week 144]) | Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEV1 (the amount of air that can be exhaled in 1 second). Spirometry was validated by using current guidelines of the American Thoracic Society (ATS) and European Respiratory Society (ERS). 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. Baseline was defined as Week 1 or the most recent value of percent-predicted FEV1 prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FEV1 improved. |
| Secondary | Percent-Predicted of Forced Vital Capacity (FVC) at Baseline | Baseline (Week 1 [Total Study Week 48]) | Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FVC (the amount of air that can be exhaled after taking a deep breath). Spirometry was validated by using current guidelines of the ATS and ERS. Baseline was defined as Week 1 or the most recent value of percent-predicted FVC prior to the first dose of open-label treatment in Study 009e. |
| Secondary | Percentage Change From Baseline in Percent-Predicted of FVC at Weeks 48 and 96 | Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144]) | Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FVC (the amount of air that can be exhaled after taking a deep breath). Spirometry was validated by using current guidelines of the ATS and ERS. 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 was defined as Week 1 or the most recent value of percent-predicted FVC prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FVC improved. |
| Secondary | Percent-Predicted of Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at Baseline | Baseline (Week 1 [Total Study Week 48]) | Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEF25-75 (the rate of air flow during the middle part of an exhalation). Spirometry was validated by using current guidelines of the ATS and ERS. Baseline was defined as Week 1 or the most recent value of percent-predicted FEF25-75 prior to the first dose of open-label treatment in Study 009e. |
| Secondary | Percentage Change From Baseline in Percent-Predicted of FEF25-75 at Weeks 48 and 96 | Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144]) | Spirometry was used to assess pulmonary function by measuring the percent-predicted, which was determined on the basis of the height value obtained at the same study visit, for FEF25-75 (the rate of air flow during the middle part of an exhalation). Spirometry was validated by using current guidelines of the ATS and ERS. 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 was defined as Week 1 or the most recent value of percent-predicted FEF25-75 prior to the first dose of open-label treatment in Study 009e. A positive change from Baseline indicates that FEF25-75 improved. |
| Secondary | Number of Participants With Pulmonary Exacerbations as Defined by Modified Fuch's Criteria | Baseline (Week 1 [Total Study Week 48]) up to Week 48 and EOT (Week 96) (Total Study Weeks 96 and 144) | A Respiratory Event Form (REF), which collected data on various signs, symptoms, and effects for each event, was completed by the Investigator when informed by the participant of a respiratory event. Pulmonary exacerbations were assessed by using the modified Fuchs' criteria, which defines an exacerbation as a respiratory event requiring treatment with parenteral antibiotics for any 4 of the following 12 symptoms with or without intravenous (IV) 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; or decrease in pulmonary function by 10% or more from a previously recorded value; or radiographic changes indicative of pulmonary function. |
| Secondary | Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Baseline (Week 1 [Total Study Week 48]) up to Week 48 (Total Study Week 96) | A REF, which collected data on various signs, symptoms, and effects for each event, was completed by the Investigator when informed by the participant of a respiratory event. Pulmonary function was assessed by using the modified Fuchs' criteria, which defines an exacerbation as a respiratory event requiring treatment with parenteral antibiotics for any 4 of the following 12 symptoms with or without treatment with IV 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% or more from a previously recorded value; or radiographic changes indicative of pulmonary function. The 48-week exacerbation rate was determined by adding the weekly rates for each arm for each 48-week period and dividing the sum by 48. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes27
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Baseline (Week 1 [Total Study Week 48]) up to 4 Weeks Post-Treatment (Week 100 [Total Study Week 148]) or Premature Discontinuation (PD) (whichever occurred first) | The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. | percent of participants | NUMBER |
| PRIMARY | Number of Participants With Any Treatment-Emergent Laboratory Abnormality (TELA) | Baseline (Week 1 [Total Study Week 48]) up to 4 Weeks Post-Treatment (Week 100 [Total Study Week 148]) or PD (whichever occurred first) | The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in 1 Second (FEV1) at Baseline | Baseline (Week 1 [Total Study Week 48]) | The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percentage of predicted FEV1 | MEAN |
| SECONDARY | Percentage Change From Baseline in Percent-Predicted of FEV1 at Weeks 48 and 96 | Week 48 (Total Study Week 96), End of Treatment (EOT) (Week 96 [Total Study Week 144]) | The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percent change | MEAN |
| POST_HOC | Percent-Predicted of FEV1 in the 96-Week Completer Population at Baseline | Baseline (Week 1 [Total Study Week 48]) | Study 009/009e 96-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 48 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percentage of predicted FEV1 | MEAN |
| POST_HOC | Percentage Change From Baseline in Percent-Predicted of FEV1 in the 96-Week Completer Population Over a Total of 96 Weeks of Treatment | Week 48 (Total Study Week 96) | Study 009/009e 96-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 48 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percent change | MEAN |
| POST_HOC | Percent-Predicted of FEV1 in the 144-Week Completer Population at Baseline | Baseline (Week 1 [Total Study Week 48]) | Study 009/009e 144-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 96 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percentage of predicted FEV1 | MEAN |
| POST_HOC | Percentage Change From Baseline in Percent-Predicted of FEV1 in the 144-Week Completer Population Over a Total of 144 Weeks of Treatment | EOT (Week 96 [Total Study Week 144]) | Study 009/009e 144-Week Completer Population: participants who completed 48 weeks of treatment with ataluren or placebo in Study 009 and at least 96 weeks of treatment with ataluren in Study 009e. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percent change | MEAN |
| SECONDARY | Percent-Predicted of Forced Vital Capacity (FVC) at Baseline | Baseline (Week 1 [Total Study Week 48]) | The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percentage of predicted FVC | MEAN |
| SECONDARY | Percentage Change From Baseline in Percent-Predicted of FVC at Weeks 48 and 96 | Week 48 (Total Study Week 96), EOT (Week 96 [Total Study Week 144]) | The Study 009e As-Treated Population: participants who received at least 1 dose of study drug. Here, 'Number Analyzed' signifies participants evaluable for the specified week. | percent change | MEAN |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 outcomesResults by group146
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren/Ataluren | At least 1 TEAE | 100 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Placebo/Ataluren | At least 1 TEAE | 97.9 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren/Ataluren | Grade 1 TEAE | 14.7 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Placebo/Ataluren | Grade 1 TEAE | 18.8 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren/Ataluren | Grade 2 TEAE | 62.1 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Placebo/Ataluren | Grade 2 TEAE | 51.0 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren/Ataluren | Grade 3 TEAE | 23.2 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Placebo/Ataluren | Grade 3 TEAE | 26.0 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren/Ataluren | Grade 4 TEAE | 0 | percent of participants | |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Placebo/Ataluren | Grade 4 TEAE | 1.0 | percent of participants |
1 to 10 of 146Source ClinicalTrials.gov
See all 146 results by groupParticipant flow20
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren/Ataluren | 95 | |
| Overall Study | STARTED | Placebo/Ataluren | 96 | |
| Overall Study | Completed 24 Weeks | Ataluren/Ataluren | 89 | |
| Overall Study | Completed 24 Weeks | Placebo/Ataluren | 86 | |
| Overall Study | Completed 48 Weeks | Ataluren/Ataluren | 78 | |
| Overall Study | Completed 48 Weeks | Placebo/Ataluren | 70 | |
| Overall Study | Completed 72 Weeks | Ataluren/Ataluren | 63 | |
| Overall Study | Completed 72 Weeks | Placebo/Ataluren | 62 | |
| Overall Study | Completed 96 Weeks | Ataluren/Ataluren | 57 | |
| Overall Study | Completed 96 Weeks | Placebo/Ataluren | 54 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 participant flowAdverse events315
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Ataluren/Ataluren | 13 | 95 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo/Ataluren | 16 | 96 | |
| other | Diarrhoea | Gastrointestinal disorders | Overall Population | 29 | 191 | |
| other | Abdominal pain | Gastrointestinal disorders | Ataluren/Ataluren | 14 | 95 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo/Ataluren | 13 | 96 | |
| other | Abdominal pain | Gastrointestinal disorders | Overall Population | 27 | 191 | |
| other | Constipation | Gastrointestinal disorders | Ataluren/Ataluren | 12 | 95 | |
| other | Constipation | Gastrointestinal disorders | Placebo/Ataluren | 15 | 96 | |
| other | Constipation | Gastrointestinal disorders | Overall Population | 27 | 191 | |
| other | Vomiting | Gastrointestinal disorders | Ataluren/Ataluren | 9 | 95 |
1 to 10 of 315Source ClinicalTrials.gov
See all 315 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren/Ataluren | ||
| Total, all-cause mortality | Placebo/Ataluren | ||
| Total, all-cause mortality | Overall Population | ||
| Total, other adverse events | Ataluren/Ataluren | 95 | 95 |
| Total, other adverse events | Placebo/Ataluren | 94 | 96 |
| Total, other adverse events | Overall Population | 189 | 191 |
| Total, serious adverse events | Ataluren/Ataluren | 48 | 95 |
| Total, serious adverse events | Placebo/Ataluren | 57 | 96 |
| Total, serious adverse events | Overall Population | 105 | 191 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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