This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Extension Study of Ataluren (PTC124) in Cystic Fibrosis (NCT01140451), a non-randomized phase 3 study. The primary outcome is Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01140451 |
| Phase | Phase 3 |
| Condition | Cystic Fibrosis |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 191 |
| Arms in the primary outcome | n/a |
| Lead sponsor | PTC Therapeutics |
| Results posted | Yes |
2. Clinical Question
Population
Participants with nonsense-mutation-mediated cystic fibrosis who entered the extension after receiving study drug in Study 009. The registry describes the population as adult and pediatric participants and defines the primary endpoint population as participants who received at least one dose of study drug.
Intervention
Open-label ataluren, given three times per day. The regimen is listed as 10 mg/kg with breakfast, 10 mg/kg with lunch, and 20 mg/kg with dinner, for a total dose of 40 mg/kg/day, for up to 96 weeks.
Comparator
There was no concurrent untreated or placebo group in the extension. One group had received double-blind ataluren in Study 009 and continued with open-label ataluren; the other had received double-blind placebo and then began open-label ataluren. The registry labels the allocation non-randomized.
Primary question
What proportion of participants experienced treatment-emergent adverse events during the specified study period? The record defines these events as untoward medical occurrences that began or worsened after the first study-drug dose, without requiring a judged causal connection to the drug. It also records whether an event was considered related by the investigator.
This is an extension safety question, rather than a randomized test of ataluren against placebo. By the extension, participants in both treatment-history groups received open-label ataluren. Consequently, differences between those groups cannot be interpreted as a clean estimate of the effect of ataluren versus no ataluren. Earlier treatment history distinguishes the groups, but the registry does not describe random allocation to extension treatment.
The study also planned to assess pulmonary function, pulmonary exacerbations, quality of life, antibiotic use, daily-life disruptions, body weight, and CF pathophysiology. The listed secondary outcomes include measures of FEV1, FVC, FEF25-75, and pulmonary exacerbations. The registry record does not report numerical results for these outcomes in the information, so the primary discussion is limited to the safety counts and definitions that are reported.
3. Trial Design
This was a Phase 3 extension study with a parallel-group structure, non-randomized allocation, and no masking. It enrolled 191 participants and is recorded as completed. The registry lists the study start as 2010-08-12 and primary completion as 2013-12-02. PTC Therapeutics is named as sponsor, and the study summary identifies FDA OOPD as the funding source.
The study followed participants who had received double-blind ataluren or placebo in Study 009. In the extension, both groups received open-label ataluren. The group names therefore describe prior treatment followed by extension treatment: Ataluren/Ataluren and Placebo/Ataluren. These are treatment-history groups, not a concurrent active-treatment versus placebo comparison. Because extension allocation was non-randomized and treatment was unmasked, comparisons between the groups are vulnerable to differences that existed before the extension and to differences in how events were reported or assessed.
The listed regimen was oral ataluren three times daily: 10 mg/kg with breakfast, 10 mg/kg with lunch, and 20 mg/kg with dinner, totaling 40 mg/kg/day, for up to 96 weeks. The record states that participants in the placebo/ataluren group would be followed for 4 weeks after treatment. The primary endpoint timeframe is described as extending from Baseline (Week 1 [Total Study Week 48]) up to 4 Weeks Post-Treatment (Week 100 [Total Study Week 148]) or premature discontinuation, whichever occurred first.
The endpoint definition separately says that a treatment-emergent adverse event occurred or worsened from the first dose through 6 weeks after the last dose. These are both statements in the registry record; it does not explain the difference between the endpoint timeframe and the event definition. More detail on eligibility criteria, the extension's sample-size justification, and the reasons for non-randomized allocation is not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: Participants began the open-label extension study immediately after completing end-of-study visit (Week 48) in Study 009 to avoid interruption in treatment. Most assessments performed at Study 009's final visit were used as Baseline assessments in Study PTC124-GD-009e-CF (009e). Investigators and participants remained blinded to Study 009 dosing.
Overall Study
| Stage | Ataluren/Ataluren | Placebo/Ataluren |
|---|---|---|
| STARTED | 95 | 96 |
| Completed 24 Weeks | 89 | 86 |
| Completed 48 Weeks | 78 | 70 |
| Completed 72 Weeks | 63 | 62 |
| Completed 96 Weeks | 57 | 54 |
| As-Treated Population | 95 | 96 |
| 96-Week Completer Population | 78 | 70 |
| 144-Week Completer Population | 57 | 54 |
| COMPLETED | 57 | 54 |
| NOT COMPLETED | 38 | 42 |
Reasons for not completing this period:
| Reason | Ataluren/Ataluren | Placebo/Ataluren |
|---|---|---|
| Withdrawal by Subject | 26 | 33 |
| Adverse Event | 2 | 6 |
| Physician Decision | 5 | 3 |
| Lost to Follow-up | 2 | 0 |
| Participants Planning Pregnancy | 3 | 0 |
Analysis population for the primary outcome, as defined in the registry: The Study 009e As-Treated Population: participants who received at least 1 dose of study drug.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: The Study 009e As-Treated Population: participants who received at least 1 dose of study drug
| Characteristic | Unit | Ataluren/Ataluren | Placebo/Ataluren | Total |
|---|---|---|---|---|
| Participants analysed | 95 | 96 | 191 | |
| Age, Continuous | years | 22.9 (SD 10.04) | 24.9 (SD 9.29) | 23.9 (SD 9.70) |
| Sex: Female, Male: Female | Participants | 48 | 49 | 97 |
| Sex: Female, Male: Male | Participants | 47 | 47 | 94 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs), measured over 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).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percent of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with treatment-emergent adverse events. It is a binary participant-level outcome: a participant is counted according to whether at least one qualifying event was recorded during the defined observation period. The endpoint is intended to describe how common such events were in the treated population; it does not, by itself, establish that study drug caused an event.
The registry defines a treatment-emergent adverse event as any untoward medical occurrence in a participant receiving study drug, regardless of whether a causal relationship was considered possible. Events counted as treatment-emergent began or worsened during the period extending from the first dose through 6 weeks after the last dose. The record states that an event was considered related when the investigator judged it possibly or probably caused by study drug. That relatedness judgment is distinct from the broader treatment-emergent definition.
Serious adverse events are defined by outcomes or significance, including death, initial or prolonged inpatient hospitalization, a life-threatening experience, persistent or significant disability or incapacity, or congenital anomaly. The registry notes that adverse events include both serious and non-serious events and directs readers to a separate adverse-events module for other non-serious events and all serious events, regardless of causality.
A participant-level percentage is useful for summarizing the proportion with at least one event, but it does not convey the number of episodes, their duration, severity, or clinical consequences. Those details require separate event-level information. The registry’s primary endpoint timeframe and its 6-week event-definition window are not identical descriptions, and the record does not reconcile them.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety counts show that serious adverse events were recorded for 48 of 95 participants in the Ataluren/Ataluren group and 57 of 96 participants in the Placebo/Ataluren group. For the overall population, the record lists 105 of 191 participants with serious adverse events. These are descriptive counts within treatment-history groups during the study’s reported safety observation period. They do not estimate a randomized treatment contrast.
The record also lists other adverse events for 95 of 95 participants in the Ataluren/Ataluren group, 94 of 96 in the Placebo/Ataluren group, and 189 of 191 in the overall population. These categories should not be treated as a causal comparison or as a substitute for the primary endpoint analysis. The registry’s primary endpoint is the percentage with treatment-emergent adverse events, while the adverse-event summary separates serious events from other events. The information described in the record does not provide an adjusted comparison, confidence intervals, or a hypothesis test for these group figures.
The most frequently listed serious-event term is “Cystic fibrosis lung” at 46.9%. Other listed serious-event terms include “Haemoptysis” and “Distal intestinal obstruction syndrome” at 2.1% each, “Nephrolithiasis” at 2.1%, and “Cystic fibrosis related diabetes” and “Abdominal pain” at 1.1% each. The registry record does not give the term-specific numerators and denominators in the information. These terms describe reported events; they do not identify whether an event was caused by ataluren.
Interpretation is constrained by the design. Both groups received ataluren during the extension, and the registry identifies the study as non-randomized and unmasked. Differences in counts could reflect prior treatment, participant characteristics, follow-up, underlying cystic fibrosis, event ascertainment, or other factors. The record does not permit those possibilities to be separated. Nor does the absence of a reported event in a category establish that no such event occurred: the registry reports counts for particular categories, and death counts are not reported in the record.
Numerical results for the other outcomes named in the registry are not reported in the information. The record lists 27 outcome measures, but lists no analyses in the analysis summary. Without further outcome estimates and their uncertainty, the safety counts should be read as descriptive reporting rather than evidence of a treatment effect. They characterize what was recorded during this extension; they do not establish whether ataluren changed event risk relative to an untreated or placebo group.
9. Statistical Methods Explained
Statistical explanations in this discussion are general unless they refer directly to the registry record. The posted primary outcome is a binary participant-level measure: each participant either had at least one treatment-emergent adverse event during the endpoint period or did not. A percentage expresses the proportion of the analyzed population meeting that definition. For a complete interpretation, readers need the numerator, denominator, event definition, and time window. The registry describes the treated population and provides counts for serious and other adverse-event categories, but it does not provide inferential estimates for the primary endpoint in the information.
What does a percentage with an event mean?
In general, a participant-level event percentage describes how often the event occurred across participants in the stated population. It does not count repeated episodes as separate participants, so it can differ from an event rate that includes multiple episodes per participant. Nor does it capture timing, duration, severity, or whether an event recurred. Those features require additional event-level summaries. For safety reporting, the definition determines what counts: this registry’s treatment-emergent definition includes an untoward medical occurrence without requiring a causal relationship to study drug.
The endpoint is not the same as an investigator’s causality judgment. The registry says an event was considered related if, in the investigator’s opinion, it was possibly or probably caused by study drug. A treatment-emergent event may therefore be counted even when it is not considered related. This distinction is important because events can occur during treatment for reasons related to the underlying condition, other care, or factors not established in the record. Statistical description alone cannot identify the cause.
Why does the design affect comparisons?
In general, random allocation helps make comparison groups similar on average with respect to known and unknown characteristics at the start of a trial. Here, the record identifies the extension as non-randomized and unmasked. Its groups are defined by the treatment received in Study 009, after which both groups received open-label ataluren. A difference in later event percentages would therefore be a descriptive difference between treatment histories, not an unbiased randomized estimate of ataluren versus placebo during the extension.
Unmasked treatment can also affect reporting, clinical decisions, or event attribution. That possibility does not show that reporting was biased; it means the design cannot rule out such influences on its own. In addition, participants who continued from an earlier study may differ from people who did not enter the extension. The registry does not report enough information to quantify these potential selection effects or adjust for them.
How should uncertainty be assessed?
In general, a confidence interval describes a range of values compatible with the observed data under a statistical model and sampling assumptions. It conveys precision as well as the point estimate. A narrow interval indicates greater precision than a wide interval, but neither interval width nor a p-value resolves design limitations or proves causality. The information described in the registry record does not report confidence intervals or a hypothesis test for the primary endpoint. Consequently, the event counts support descriptive reading, not a claim about statistical significance.
Small or uncommon event categories can have substantial uncertainty because their observed counts may be sensitive to a small change in the number of events. General methods for rare outcomes may use exact or other approaches suited to sparse data. The record does not state which statistical methods, if any, were used to analyze the primary safety endpoint. It would be inappropriate to infer a method from the outcome format alone.
What do planning illustrations represent?
General sample-size planning asks how many participants might be needed to detect a specified effect under stated assumptions. The registry’s planning description says its illustrations are based on the observed effect, use a two-sided significance level of 0.05, equal allocation, and a normal approximation, and target stated fractions of that effect. It also says that time-to-event illustrations give a total number of events, while other illustrations give participants per arm. These are planning illustrations, not observed trial results and not evidence that the study had a particular probability of detecting an effect. No numerical planning results are reported in the record.
A planning calculation depends on the effect assumed, outcome variability, analysis method, allocation, and desired operating characteristics. If the true effect differs from the assumed effect, the number needed for a future study could also differ. An illustration based on an observed effect is especially dependent on the estimate used; observed effects can be imprecise. These general principles do not establish how this extension was designed. Its own sample-size justification and statistical plan are not reported in the registry record.
What can be concluded from the listed analyses?
The registry lists 27 outcome measures and no analyses in its analysis summary. That means the information does not supply a set of model-based results to interpret for those outcomes. It does not establish that no analyses were conducted outside the registry record. Without a reported analysis method, handling of missing observations, or uncertainty estimates, readers should avoid treating the raw safety counts as a complete statistical comparison. The clearest supported interpretation is descriptive: the registry records the stated numbers of participants with events in the stated groups and lists selected serious-event terms and percentages.
10. Multiplicity, Missing Data and Analysis Populations
The registry names a broad set of outcomes, including pulmonary function measures, pulmonary exacerbations, quality of life, antibiotic use, disruptions to daily living, body weight, and CF pathophysiology. It reports 27 outcome measures overall, while the outcome list described in the record includes secondary measures of FEV1, FVC, FEF25-75, and pulmonary exacerbations. When many outcomes are assessed, the chance of at least one apparently notable result can increase if each is tested separately. General approaches to multiplicity include prespecifying a primary comparison, adjusting significance thresholds, or treating secondary analyses as exploratory. The registry record does not report a multiplicity strategy, the hierarchy of secondary outcomes, or which tests were planned.
Missing data can affect an outcome estimate when some participants do not have measurements at a scheduled assessment or leave follow-up early. General methods may use available observations, imputation, or models that make assumptions about the missingness process. Each approach relies on assumptions, and different assumptions can produce different estimates. The registry record does not report the amount or pattern of missing outcome data, the method used to handle it, or sensitivity analyses. The primary endpoint description allows follow-up to end at premature discontinuation, but does not explain how participants with incomplete observation were handled in the analysis.
The primary endpoint population is described as the Study 009e As-Treated Population: participants who received at least one dose of study drug. This definition clarifies that the safety population is based on treatment received, but the record does not report further analysis-population definitions for secondary outcomes or explain whether all enrolled participants were included in each outcome analysis. It also does not describe exclusions from particular analyses.
The registry lists no analyses in its analysis summary. Accordingly, the statistical plan, inferential procedures, multiplicity handling, missing-data methods, and analysis populations beyond the stated safety population are not reported in the registry record. These omissions limit assessment of how complete and comparable the endpoint data were and how any multiple outcome findings should be interpreted.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Ataluren/Ataluren | 48 | 95 | 50.5% |
| Placebo/Ataluren | 57 | 96 | 59.4% |
| Overall Population | 105 | 191 | 55.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Ataluren/Ataluren | 95 of 95 |
| Placebo/Ataluren | 94 of 96 |
| Overall Population | 189 of 191 |
Most frequent serious adverse event terms
| Event term | Organ system | Ataluren/Ataluren | Placebo/Ataluren | Overall Population |
|---|---|---|---|---|
| Cystic fibrosis lung | Respiratory, thoracic and mediastinal disorders | 41 of 95 | 45 of 96 | 86 of 191 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 2 of 95 | 1 of 96 | 3 of 191 |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | 0 of 95 | 2 of 96 | 2 of 191 |
| Acute sinusitis | Infections and infestations | 1 of 95 | 1 of 96 | 2 of 191 |
| Mycobacterium abscessus infection | Infections and infestations | 1 of 95 | 1 of 96 | 2 of 191 |
| Calculus ureteric | Renal and urinary disorders | 1 of 95 | 1 of 96 | 2 of 191 |
| Nephrolithiasis | Renal and urinary disorders | 0 of 95 | 2 of 96 | 2 of 191 |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | 1 of 95 | 1 of 96 | 2 of 191 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 95 | 1 of 96 | 1 of 191 |
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | 1 of 95 | 0 of 96 | 1 of 191 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ataluren/Ataluren | Placebo/Ataluren | Overall Population |
|---|---|---|---|---|
| Cystic fibrosis lung | Respiratory, thoracic and mediastinal disorders | 66 of 95 | 69 of 96 | 135 of 191 |
| Viral upper respiratory tract infection | Infections and infestations | 26 of 95 | 32 of 96 | 58 of 191 |
| Cough | Respiratory, thoracic and mediastinal disorders | 29 of 95 | 28 of 96 | 57 of 191 |
| Pyrexia | General disorders | 19 of 95 | 19 of 96 | 38 of 191 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 13 of 95 | 18 of 96 | 31 of 191 |
| Diarrhoea | Gastrointestinal disorders | 13 of 95 | 16 of 96 | 29 of 191 |
| Rhinitis | Infections and infestations | 13 of 95 | 15 of 96 | 28 of 191 |
| Abdominal pain | Gastrointestinal disorders | 14 of 95 | 13 of 96 | 27 of 191 |
| Constipation | Gastrointestinal disorders | 12 of 95 | 15 of 96 | 27 of 191 |
| Nasopharyngitis | Infections and infestations | 13 of 95 | 14 of 96 | 27 of 191 |
Serious events with the highest arm-level rate: cystic fibrosis lung (46.9%), haemoptysis (2.1%), distal intestinal obstruction syndrome (2.1%), nephrolithiasis (2.1%), cystic fibrosis related diabetes (1.1%).
The serious adverse-event counts are 48 of 95 participants in the Ataluren/Ataluren group and 57 of 96 in the Placebo/Ataluren group; the overall population count is 105 of 191. These figures describe participants recorded with serious adverse events, using the registry’s definition. Seriousness refers to specified outcomes or significance, such as hospitalization, a life-threatening experience, persistent or significant disability, congenital anomaly, or death. It is not a measure of how likely an event was to be caused by study drug.
The listed serious-event terms include “Cystic fibrosis lung” at 46.9%, “Haemoptysis” at 2.1%, “Distal intestinal obstruction syndrome” at 2.1%, “Nephrolithiasis” at 2.1%, “Cystic fibrosis related diabetes” at 1.1%, and “Abdominal pain” at 1.1%. These percentages should be read as registry-listed term frequencies, not as causal effects. The record does not report the numerators and denominators for each term, and does not specify a causal attribution for the listed terms.
The registry says that other non-serious adverse events and all serious adverse events, regardless of causality, are located in its adverse-events module. Death counts are not reported in the registry record. The group figures are not a randomized comparison: both groups received open-label ataluren in the extension, and allocation was non-randomized. They therefore do not establish that one treatment history caused more or fewer serious events.
12. What the Result Does, and Does Not, Mean
The results can describe the safety experience recorded during an open-label ataluren extension among participants with nonsense-mutation-mediated cystic fibrosis. The primary endpoint is framed as the percentage of participants with treatment-emergent adverse events, and the registry provides serious and other adverse-event counts for the treatment-history groups and overall population. It also lists selected serious-event terms and percentages. These registry entries are relevant to describing what was recorded during follow-up.
They do not establish a causal effect of ataluren on adverse-event risk. The extension was non-randomized and unmasked, and both groups received ataluren. Their labels reflect earlier treatment in Study 009, not an untreated comparison maintained during this extension. Potential differences between groups can reflect prior treatment or other factors as well as chance. The record does not report an adjusted comparison, uncertainty intervals, or a hypothesis test for the primary endpoint.
The findings also do not establish the absence of events that are not listed in the selected summary, nor do the reported counts show the timing, duration, recurrence, or severity of every event. The serious-event percentages are event-term summaries; they do not by themselves indicate whether events were judged related to study drug. The registry’s definition of treatment-emergent events is broader than its definition of events considered related.
The study planned assessments beyond safety, including pulmonary function, exacerbations, and other clinical and quality-of-life outcomes. Numerical results for those outcomes are not reported in the information. Thus, no conclusion about changes in those outcomes follows from the safety counts. The registry also does not report its sample-size justification, missing-data approach, multiplicity procedure, or full statistical analysis plan. Interpretation should remain within the limits of the descriptive safety information and the non-randomized extension design.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between an extension safety description and a randomized treatment comparison. The primary question focuses on treatment-emergent adverse events during longer-term exposure, while the design follows participants from an earlier study into open-label treatment. That structure can document events among participants who continued into the extension, but it does not preserve a concurrent placebo comparison.
The group labels can be easy to misread. Ataluren/Ataluren and Placebo/Ataluren identify earlier treatment followed by extension treatment; both groups received open-label ataluren during the extension. Because assignment was non-randomized and there was no masking, a numerical difference in event counts should not be interpreted as a direct estimate of treatment effect. Statistical comparisons cannot repair the absence of a randomized contemporaneous control without additional assumptions and methods, which are not reported in the registry record.
The endpoint definition also illustrates why safety statistics require careful reading. “Treatment-emergent” describes when an event occurred or worsened, not whether the treatment caused it. Seriousness is classified by outcomes or medical significance, while relatedness is an investigator judgment. These concepts answer different questions and should not be collapsed into a single label.
Finally, the record distinguishes the endpoint population from a general enrolled population and provides group-specific counts. Denominators and event definitions are essential for understanding those counts, while uncertainty estimates and missing-data methods are needed to assess precision and completeness. The lack of reported inferential analyses does not invalidate the descriptive record, but it limits the claims that can be made from it. This distinction is useful when interpreting extension studies and registry safety summaries more broadly.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The study’s extension design is a central limitation for comparative interpretation. The registry identifies allocation as non-randomized and masking as absent. Participants were grouped according to their earlier treatment in Study 009, and both groups received open-label ataluren in the extension. Differences in participant characteristics, earlier treatment experience, continuation into the extension, follow-up, or event reporting could contribute to differences in the posted counts. The registry does not report methods that would address these sources of bias.
The endpoint timing is not fully clear in the record. Its timeframe states follow-up up to 4 weeks post-treatment, while its definition says treatment-emergent events extend through 6 weeks after the last dose. The registry does not explain how these descriptions align. The group description also states that the placebo/ataluren group would be followed for 4 weeks after treatment, but the record does not clarify whether all participants had the same follow-up procedures.
The record does not report confidence intervals, hypothesis tests, a detailed statistical analysis plan, or the sample-size justification for the extension. It does not state how missing observations, premature discontinuation, or multiple outcomes were handled. Although it lists 27 outcome measures, the analysis summary lists no analyses. Therefore, the precision, completeness, and inferential status of the reported results cannot be fully evaluated from the registry record.
The serious-event term percentages are not accompanied in the information by term-specific numerators and denominators, and death counts are not reported. The registry’s event-term summaries also do not establish causality. These limitations mean that the safety figures are best treated as descriptive reporting from the extension, rather than as evidence for a treatment comparison or a complete account of all clinical outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Extension Study of Ataluren (PTC124) in Cystic Fibrosis — NCT01140451.
- Registry record last updated 2020-10-19; status last verified 2020-10.
- Results first posted 2020-10-19.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT01140451 is a completed Phase 3 extension study of ataluren in participants with nonsense-mutation-mediated cystic fibrosis. The registry records 191 enrolled participants, non-randomized allocation, no masking, and a parallel-group structure. Participants who had received double-blind ataluren or placebo in Study 009 received open-label ataluren in the extension.
The primary endpoint is the percentage of participants with treatment-emergent adverse events. The registry reports serious adverse-event counts of 48 of 95 and 57 of 96 for the treatment-history groups, and 105 of 191 overall. It also reports other adverse-event counts of 95 of 95, 94 of 96, and 189 of 191, respectively. These are descriptive safety figures, not a randomized comparison. The record lists 27 outcome measures but no analyses in its analysis summary. Statistical-plan details, missing-data methods, and numerical results for the other outcomes are not reported in the registry record.