Trial · NCT02107859
Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02107859, Phase 3, Terminated, PTC Therapeutics; results first posted 2020-03-25
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Ataluren (PTC124) in Cystic Fibrosis. Trials, record NCT02107859. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02107859
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ataluren (PTC124) in Cystic Fibrosis
- NCT number
- NCT02107859
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 61 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren
- First posted
- 2014-04-08
- Last updated
- 2020-03-25
- Other registries' IDs
- eudract:2013-005449-35
- Results first posted
- 2020-03-25
- Limitations and caveats
Based on the results of study PTC124-GD-021-CF (NCT02139306) (did not achieve its primary or secondary endpoints), clinical development of ataluren in cystic fibrosis was discontinued and this ongoing open-label extension study was closed.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Baseline (Day 1) up to end of study (Week 196) | 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. |
| Primary | Number of Participants With Clinically Significant Laboratory Abnormalities | Baseline (Day 1) up to end of study (Week 196) | 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. |
| Secondary | 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, Week 192 | 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. |
| Secondary | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria | Baseline up to Week 192 | 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. |
| Secondary | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria | Baseline up to Week 192 | 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. |
| Secondary | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria | Baseline up to Week 192 | 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. |
| Secondary | Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196) | Baseline, Week 196 | ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration. |
| Secondary | Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG | Baseline, Week 196 | Heart rate was measured using 12-lead ECG. |
| Secondary | Change From Baseline in Vital Signs at Final Visit (Week 196) | Baseline, Week 196 | Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP). |
| Other | Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry | Baseline, Week 192 | 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. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Baseline (Day 1) up to end of study (Week 196) | As-treated population included all participants who received at least 1 dose of ataluren. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Laboratory Abnormalities | Baseline (Day 1) up to end of study (Week 196) | As-treated population included all participants who received at least 1 dose of ataluren. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | 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, Week 192 | 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. | percentage of predicted FEV1 | MEAN |
| SECONDARY | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria | Baseline up to Week 192 | ITT population included all participants who had at least 1 post-baseline efficacy assessment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria | Baseline up to Week 192 | ITT population included all participants who had at least 1 post-baseline efficacy assessment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria | Baseline up to Week 192 | ITT population included all participants who had at least 1 post-baseline efficacy assessment. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196) | Baseline, Week 196 | 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. | miiliseconds | MEAN |
| SECONDARY | Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG | Baseline, Week 196 | 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. | beats/minute | MEAN |
| SECONDARY | Change From Baseline in Vital Signs at Final Visit (Week 196) | Baseline, Week 196 | As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Number analyzed' signifies participants evaluable for specified categories. | millimeters of mercury (mmHg) | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry | Baseline, Week 192 | 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. | percentage of predicted FVC | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group37
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Any TEAEs | 61 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Mild AEs | 4 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Moderate AEs | 26 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Severe AEs | 30 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Life-threatening AEs | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Fatal AEs | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | AEs unrelated to ataluren | 35 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | AEs unlikely related to ataluren | 12 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | AEs possible related to ataluren | 13 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | AEs probable related to ataluren | 1 | Participants |
1 to 10 of 37Source ClinicalTrials.gov
See all 37 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 61 | |
| Overall Study | As-treated Population | Ataluren | 61 | |
| Overall Study | Intent-to-treat (ITT) Population | Ataluren | 60 | |
| Overall Study | COMPLETED | Ataluren | 0 | |
| Overall Study | NOT COMPLETED | Ataluren | 61 |
Source ClinicalTrials.gov
Adverse events46
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 | Gastroenteritis | Infections and infestations | Ataluren | 5 | 61 | |
| other | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | Ataluren | 50 | 61 | |
| other | Influenza | Infections and infestations | Ataluren | 4 | 61 | |
| other | Nasopharyngitis | Infections and infestations | Ataluren | 5 | 61 | |
| other | Oral candidiasis | Infections and infestations | Ataluren | 4 | 61 | |
| other | Sinusitis | Infections and infestations | Ataluren | 6 | 61 | |
| other | Upper respiratory tract infection | Infections and infestations | Ataluren | 9 | 61 | |
| other | Viral upper respiratory tract infection | Infections and infestations | Ataluren | 22 | 61 | |
| other | Abdominal pain | Gastrointestinal disorders | Ataluren | 6 | 61 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ataluren | 5 | 61 |
1 to 10 of 46Source ClinicalTrials.gov
See all 46 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 1 | 61 |
| Total, other adverse events | Ataluren | 61 | 61 |
| Total, serious adverse events | Ataluren | 36 | 61 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36866921 | DERIVED |
Source PubMed
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.
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/NCT02107859
GET https://conductscience.org/api/trials/v1/NCT02107859/results?page=2