Trial · NCT00803205
Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT00803205, 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 (PTC124™) in Cystic Fibrosis. Trials, record NCT00803205. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00803205
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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
- NCT00803205
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 238 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren, Placebo
- First posted
- 2008-12-05
- Last updated
- 2020-05-14
- Other registries' IDs
- eudract:2008-003924-52
- Results first posted
- 2020-05-14
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline | Baseline (Week 1) | Spirometry was used to assess pulmonary function by measuring the percentage of predicted function, 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 assessed by using current guidelines of the American Thoracic Society (ATS) and European Respiratory Society (ERS). Baseline was the average of percent-predicted FEV1 at screening and randomization. |
| Primary | Percentage Change From Baseline in Percent-Predicted of FEV1 at Week 48 | End of Treatment (EOT) (Week 48) | Spirometry was used to assess pulmonary function by measuring the percentage of predicted function, 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 assessed 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 the average of percent-predicted FEV1 at screening and randomization. A negative change from Baseline indicates that percent-predicted of FEV1 decreased. |
| Secondary | Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Baseline to EOT (Week 48) | A Respiratory Event Form, 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 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 and dividing the sum by 48. |
| Secondary | Change From Baseline in Awake Cough Hourly Rate at Week 48 | Baseline, EOT (Week 48) | The frequency of awake cough was measured using the LifeShirt, which incorporates motion-sensing transducers, electrodes, a microphone, and a 3-axis accelerometer into a lightweight vest. The rate was determined by dividing the total number of coughs by 24 (the number of hours of the observation period). Baseline was the latest, valid assessment prior to the treatment. A negative change from Baseline indicates that coughing decreased. |
| Secondary | Change From Baseline in the Respiratory Domain Score of the Revised Cystic Fibrosis Questionnaire (CFQ-R) at Week 48 | Baseline, EOT (Week 48) | The CFQ-R consists of 44 items, including generic scales of physical functioning, role functioning, vitality, health perceptions, emotional functioning, and social functioning, and CF-specific scales of respiratory and digestive symptoms, body image, eating disturbances, and treatment burden. Each domain score ranges from 1 to 4. Scores were linearly transformed to a 0 to 100 scale, with higher scores indicating better health. Domain scores were calculated by using the following formula: 100 \* (sum of responses - minimum possible sum)/ (maximum possible sum - minimum possible sum). The minimum possible sum = number of questions \* 1; the maximum possible = the number of questions \* 4. Baseline was the latest, valid assessment prior to the treatment. A negative change from Baseline indicates that health has worsened. Participants may have switched age groups during the study. |
| Secondary | Percent-Predicted of Forced Vital Capacity (FVC) at Baseline | Baseline (Week 1) | Spirometry was used to assess pulmonary function by measuring the percentage of predicted function, 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 assessed by using current guidelines of the ATS and ERS. Baseline was the average of percent-predicted FVC at screening and randomization. |
| Secondary | Percentage Change From Baseline in Percent-Predicted of FVC at Week 48 | EOT (Week 48) | Spirometry was used to assess pulmonary function by measuring the percentage of predicted function, 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 assessed 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 the average of percent-predicted FVC at screening and randomization. A negative change from Baseline indicates that percent-predicted of FVC decreased. |
| Other | Percentage of Participants With Treatment-Emergent Adverse Events (TEAE) | Baseline up to 4 Weeks Post-Treatment (Week 52) or Premature Discontinuation (PD) | 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 first dose of study drug to 4 weeks after the last dose of study drug. A serious adverse event (SAE) was an 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. |
| Other | Rate of Study Drug Compliance by Drug Accountability | Baseline up to EOT (Week 48) | Study drug compliance was assessed by using a Pharmacy Subject Study Drug Accountability Log (completed by the investigational site personnel). The rate of compliance was defined as 100 \* (number of sachets taken/number of planned sachets) during the study. All calculations were based on the records of the first dose date to the last dose date. To differentiate dose strengths while maintaining the blind, each kit had a unique kit number and had prominent lettering "A" and "B." Each kit contained 65 packets of 1 of the dose strengths (125, 250, or 1000 mg or matching placebo). Labeling for active drug and placebo was identical. |
| Other | Rate of Study Drug Compliance by Patient-Reported Data | Baseline up to EOT (Week 48) | Patient-reported data were obtained from the participant's electronic daily diary, which was completed by the participant or the caregiver. During study treatment, the electronic daily diary was to be completed by the participant or caregiver each day for each dose. For each participant, compliance is described in terms of the percentage of study drug actually taken. All calculations were based on the records of the first dose date to the last dose date. To differentiate dose strengths while maintaining the blind, each kit had a unique kit number and had prominent lettering "A" and "B." Each kit contained 65 packets of 1 of the dose strengths (125, 250, or 1000 mg or matching placebo). Labeling for active drug and placebo was identical. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline | Baseline (Week 1) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed' signifies participants evaluable for this outcome measure. | percentage of predicted FEV1 | MEAN |
| PRIMARY | Percentage Change From Baseline in Percent-Predicted of FEV1 at Week 48 | End of Treatment (EOT) (Week 48) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed' signifies participants evaluable for this outcome measure. | percent change | MEAN |
| SECONDARY | Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Baseline to EOT (Week 48) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed' signifies participants evaluable for this outcome measure. | exacerbations | MEAN |
| SECONDARY | Change From Baseline in Awake Cough Hourly Rate at Week 48 | Baseline, EOT (Week 48) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed' signifies participants evaluable for this outcome measure. | coughs/hour | MEAN |
| SECONDARY | Change From Baseline in the Respiratory Domain Score of the Revised Cystic Fibrosis Questionnaire (CFQ-R) at Week 48 | Baseline, EOT (Week 48) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed' signifies participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Percent-Predicted of Forced Vital Capacity (FVC) at Baseline | Baseline (Week 1) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed 'signifies participants evaluable for this outcome measure. | percentage of predicted FVC | MEAN |
| SECONDARY | Percentage Change From Baseline in Percent-Predicted of FVC at Week 48 | EOT (Week 48) | The ITT Population: all randomized participants with data for FEV1 at Baseline and a minimum of 1 post-Baseline assessment before Week 8. Here, 'Number analyzed 'signifies participants evaluable for this outcome measure. | percent change | MEAN |
| OTHER_PRE_SPECIFIED | Percentage of Participants With Treatment-Emergent Adverse Events (TEAE) | Baseline up to 4 Weeks Post-Treatment (Week 52) or Premature Discontinuation (PD) | The As-Treated Population: all randomized participants who received at least 1 dose of study drug. | percent of participants | NUMBER |
| OTHER_PRE_SPECIFIED | Rate of Study Drug Compliance by Drug Accountability | Baseline up to EOT (Week 48) | The As-Treated Population: all randomized participants who received at least 1 dose of study drug. | percent of doses taken | MEDIAN |
| OTHER_PRE_SPECIFIED | Rate of Study Drug Compliance by Patient-Reported Data | Baseline up to EOT (Week 48) | The As-Treated Population: all randomized participants who received at least 1 dose of study drug. | percent of doses taken | MEDIAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group98
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline | Ataluren | 62.092 | Standard Deviation 13.6159 | percentage of predicted FEV1 | |
| Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline | Placebo | 60.232 | Standard Deviation 15.1437 | percentage of predicted FEV1 | |
| Percentage Change From Baseline in Percent-Predicted of FEV1 at Week 48 | Ataluren | -2.534 | Standard Deviation 13.2452 | percent change | |
| Percentage Change From Baseline in Percent-Predicted of FEV1 at Week 48 | Placebo | -5.500 | Standard Deviation 12.5595 | percent change | |
| Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Ataluren | 1.42 | 95% Confidence Interval 1.05 to 1.79 | exacerbations | |
| Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks | Placebo | 1.78 | 95% Confidence Interval 1.38 to 2.17 | exacerbations | |
| Change From Baseline in Awake Cough Hourly Rate at Week 48 | Ataluren | Baseline | 28.218 | Standard Deviation 20.2726 | coughs/hour |
| Change From Baseline in Awake Cough Hourly Rate at Week 48 | Placebo | Baseline | 24.472 | Standard Deviation 16.7828 | coughs/hour |
| Change From Baseline in Awake Cough Hourly Rate at Week 48 | Ataluren | Change From Baseline | -0.595 | Standard Deviation 18.3221 | coughs/hour |
| Change From Baseline in Awake Cough Hourly Rate at Week 48 | Placebo | Change From Baseline | 0.882 | Standard Deviation 14.3936 | coughs/hour |
1 to 10 of 98Source ClinicalTrials.gov
See all 98 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 120 | |
| Overall Study | STARTED | Placebo | 118 | |
| Overall Study | Completed Week 16 | Ataluren | 107 | |
| Overall Study | Completed Week 16 | Placebo | 113 | |
| Overall Study | Completed Week 24 | Ataluren | 103 | |
| Overall Study | Completed Week 24 | Placebo | 110 | |
| Overall Study | As-Treated Population* | Ataluren | 120 | |
| Overall Study | As-Treated Population* | Placebo | 118 | |
| Overall Study | ITT Population | Ataluren | 116 | |
| Overall Study | ITT Population | Placebo | 116 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events124
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 | Abdominal pain | Gastrointestinal disorders | Ataluren | 20 | 120 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 14 | 118 | |
| other | Vomiting | Gastrointestinal disorders | Ataluren | 14 | 120 | |
| other | Vomiting | Gastrointestinal disorders | Placebo | 11 | 118 | |
| other | Diarrhoea | Gastrointestinal disorders | Ataluren | 13 | 120 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 21 | 118 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ataluren | 11 | 120 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 7 | 118 | |
| other | Nausea | Gastrointestinal disorders | Ataluren | 11 | 120 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 13 | 118 |
1 to 10 of 124Source ClinicalTrials.gov
See all 124 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Ataluren | 118 | 120 |
| Total, other adverse events | Placebo | 115 | 118 |
| Total, serious adverse events | Ataluren | 46 | 120 |
| Total, serious adverse events | Placebo | 50 | 118 |
Source ClinicalTrials.gov
Papers2
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Ataluren vs Placebo: serious adverse events |
Source 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.
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/NCT00803205
GET https://conductscience.org/api/trials/v1/NCT00803205/results?page=2