Trial · NCT02139306
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT02139306, Phase 3, Completed, PTC Therapeutics; results first posted 2020-05-14
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF). Trials, record NCT02139306. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02139306
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
- NCT number
- NCT02139306
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 279 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren (PTC124®), Placebo
- First posted
- 2014-05-15
- Last updated
- 2020-05-14
- Other registries' IDs
- eudract:2013-004581-34
- Results first posted
- 2020-05-14
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48 | From Baseline to Week 48 | 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. |
| Secondary | 48-week Rate of Pulmonary Exacerbations | Week 48 | 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. |
| Secondary | Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48 | Baseline (Day 1) and Week 48 | 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. |
| Secondary | Change From Baseline in Body Mass Index (BMI) at Week 48 | Baseline (Day 1) and Week 48 | Malnutrition is common in participants with cystic fibrosis. The BMI is an important clinical measure of nutritional status. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | 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. |
| Secondary | Number of Participants With TEAEs by Severity and Relationship to Study Drugs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | 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). |
| Secondary | Number of Participants With SAEs by Severity and Relationship to Study Drugs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | 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). |
| Secondary | Number of Participants With Abnormal Vital Signs Reported as TEAEs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | 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. |
| Secondary | Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | 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. |
| Secondary | Number of Participants With Abnormal Electrocardiogram Reported as TEAEs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | 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. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48 | From Baseline to Week 48 | The intent-to-treat (ITT) population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit. | Percentage of predicted FEV1 | LEAST_SQUARES_MEAN |
| SECONDARY | 48-week Rate of Pulmonary Exacerbations | Week 48 | The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit. | number of exacerbations per 48 weeks | MEAN |
| SECONDARY | Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48 | Baseline (Day 1) and Week 48 | The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Body Mass Index (BMI) at Week 48 | Baseline (Day 1) and Week 48 | The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit. | kilogram per meter square (kg/m^2) | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | The as-treated population included all randomized participants who actually received any study treatment. | participants | NUMBER |
| SECONDARY | Number of Participants With TEAEs by Severity and Relationship to Study Drugs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | The as-treated population included all randomized participants who actually received any study treatment. | participants | NUMBER |
| SECONDARY | Number of Participants With SAEs by Severity and Relationship to Study Drugs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | The as-treated population included all randomized participants who actually received any study treatment. | participants | NUMBER |
| SECONDARY | Number of Participants With Abnormal Vital Signs Reported as TEAEs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | The as-treated population included all randomized participants who actually received any study treatment. | participants | NUMBER |
| SECONDARY | Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | The as-treated population included all randomized participants who actually received any study treatment. | participants | NUMBER |
| SECONDARY | Number of Participants With Abnormal Electrocardiogram Reported as TEAEs | From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks) | The as-treated population included all randomized participants who actually received any study treatment. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48 | Ataluren | -1.396 | 95% Confidence Interval -2.7735 to -0.0180 | Percentage of predicted FEV1 | |
| Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48 | Placebo | -1.992 | 95% Confidence Interval -3.3271 to -0.6576 | Percentage of predicted FEV1 | |
| 48-week Rate of Pulmonary Exacerbations | Ataluren | 0.950 | Standard Deviation 1.4038 | number of exacerbations per 48 weeks | |
| 48-week Rate of Pulmonary Exacerbations | Placebo | 1.127 | Standard Deviation 2.5241 | number of exacerbations per 48 weeks | |
| Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48 | Ataluren | -0.760 | 95% Confidence Interval -3.4566 to 1.9364 | units on a scale | |
| Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48 | Placebo | -1.032 | 95% Confidence Interval -3.7368 to 1.6728 | units on a scale | |
| Change From Baseline in Body Mass Index (BMI) at Week 48 | Ataluren | 0.296 | 95% Confidence Interval 0.1126 to 0.4789 | kilogram per meter square (kg/m^2) | |
| Change From Baseline in Body Mass Index (BMI) at Week 48 | Placebo | 0.361 | 95% Confidence Interval 0.1759 to 0.5455 | kilogram per meter square (kg/m^2) | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Ataluren | TEAEs | 133 | participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Placebo | TEAEs | 135 | participants |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 140 | |
| Overall Study | STARTED | Placebo | 139 | |
| Overall Study | COMPLETED | Ataluren | 127 | |
| Overall Study | COMPLETED | Placebo | 125 | |
| Overall Study | NOT COMPLETED | Ataluren | 13 | |
| Overall Study | NOT COMPLETED | Placebo | 14 |
Source ClinicalTrials.gov
Adverse events112
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 | 13 | 140 | 15 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 12 | 139 | 20 |
| other | Nausea | Gastrointestinal disorders | Ataluren | 11 | 140 | 14 |
| other | Nausea | Gastrointestinal disorders | Placebo | 10 | 139 | 12 |
| other | Abdominal pain | Gastrointestinal disorders | Ataluren | 11 | 140 | 15 |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 5 | 139 | 6 |
| other | Vomiting | Gastrointestinal disorders | Ataluren | 8 | 140 | 8 |
| other | Vomiting | Gastrointestinal disorders | Placebo | 4 | 139 | 7 |
| other | Pyrexia | General disorders | Ataluren | 9 | 140 | 17 |
| other | Pyrexia | General disorders | Placebo | 10 | 139 | 12 |
1 to 10 of 112Source ClinicalTrials.gov
See all 112 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 0 | 140 |
| Total, all-cause mortality | Placebo | 0 | 139 |
| Total, other adverse events | Ataluren | 123 | 140 |
| Total, other adverse events | Placebo | 120 | 139 |
| Total, serious adverse events | Ataluren | 40 | 140 |
| Total, serious adverse events | Placebo | 46 | 139 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Ataluren vs Placebo: Number of Participants With Abnormal Vital Signs Reported as TEAEs | Trial comparisons |
| Ataluren vs Placebo: Number of Participants With Abnormal Electrocardiogram Reported as TEAEs | Trial comparisons |
| Ataluren vs Placebo: Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs | Trial comparisons |
| Ataluren vs Placebo: deaths from any cause | Trial comparisons |
| Ataluren vs Placebo: serious adverse events | Trial comparisons |
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.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone 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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02139306
GET https://conductscience.org/api/trials/v1/NCT02139306/results?page=2