Trial · NCT02456103
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT02456103, Phase 3, Terminated, PTC Therapeutics; results first posted 2020-04-15
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis. Trials, record NCT02456103. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02456103
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
- NCT number
- NCT02456103
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 246 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren
- First posted
- 2015-05-28
- Last updated
- 2020-04-27
- Results first posted
- 2020-04-15
- Limitations and caveats
- This study was terminated early because the CF data from the double-blind CF Study PTC124-GD-021-CF (NCT02139306) did not meet endpoints.
Source ClinicalTrials.gov
Registered outcomes6
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 up to Week 100 | TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section. |
| Primary | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter | Baseline up to Week 100 | Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section. |
| Secondary | Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24 | Baseline, Week 24 | Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. 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. |
| Secondary | Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24 | Baseline, Week 24 | Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. 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. |
| Secondary | Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24 | Baseline, Week 24 | Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. 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. |
| Secondary | Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Baseline up to Week 48 | A modified Fuchs' exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: 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. The 48-week rate = (the total number of events/ treatment duration by week)\*48. |
Source ClinicalTrials.gov
Outcomes6
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 up to Week 100 | Participants who received at least 1 dose of ataluren (As-Treated Population). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter | Baseline up to Week 100 | Participants who received at least 1 dose of ataluren (As-Treated Population). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24 | Baseline, Week 24 | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEV1 data. | percentage of predicted FEV1 | MEAN |
| SECONDARY | Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24 | Baseline, Week 24 | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FVC data. | percentage of predicted FVC | MEAN |
| SECONDARY | Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24 | Baseline, Week 24 | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEF25-75 data. | percentage of FEF25-75 | MEAN |
| SECONDARY | Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Baseline up to Week 48 | Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population). | exacerbations | MEAN |
Source ClinicalTrials.gov
Results by group19
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 | At least 1 TEAE | 222 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Mild TEAE | 28 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Moderate TEAE | 147 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Severe TEAE | 46 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Life-Threatening TEAE | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Fatal TEAE | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Serious TEAE | 89 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | TEAE Unrelated to Study Drug | 140 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | TEAE Unlikely Related to Study Drug | 55 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | TEAE Possibly Related to Study Drug | 23 | Participants |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 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 | 246 | |
| Overall Study | As-Treated Population | Ataluren | 245 | |
| Overall Study | Intent-to-treat (ITT) Population | Ataluren | 244 | |
| Overall Study | COMPLETED | Ataluren | 0 | |
| Overall Study | NOT COMPLETED | Ataluren | 246 |
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 | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | Ataluren | 136 | 245 | |
| other | Influenza | Infections and infestations | Ataluren | 13 | 245 | |
| other | Sinusitis | Infections and infestations | Ataluren | 25 | 245 | |
| other | Upper respiratory tract infection | Infections and infestations | Ataluren | 21 | 245 | |
| other | Viral upper respiratory tract infection | Infections and infestations | Ataluren | 30 | 245 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Ataluren | 32 | 245 | |
| other | Haemoptysis | Respiratory, thoracic and mediastinal disorders | Ataluren | 14 | 245 | |
| other | Forced expiratory volume decreased | Investigations | Ataluren | 16 | 245 | |
| other | Headache | Nervous system disorders | Ataluren | 16 | 245 | |
| other | Nausea | Gastrointestinal disorders | Ataluren | 14 | 245 |
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 | 245 |
| Total, other adverse events | Ataluren | 191 | 245 |
| Total, serious adverse events | Ataluren | 89 | 245 |
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.
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/NCT02456103
GET https://conductscience.org/api/trials/v1/NCT02456103/events?page=2