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 Rollover Study of VX-770 in Cystic Fibrosis Subjects (NCT01117012), a na phase 3 study. The primary outcome is Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01117012 |
| Phase | Phase 3 |
| Condition | Cystic Fibrosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 192 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Vertex Pharmaceuticals Incorporated |
| Results posted | Yes |
2. Clinical Question
Population
Participants with cystic fibrosis in the Study 105 safety set. The registry defines this set as participants who received at least 1 dose of study drug; the reported safety denominator is 192.
Intervention
VX-770, also named ivacaftor, given as a 150 milligram (mg) tablet orally twice daily (q12h).
Comparator
No concurrent comparator group is reported. The study is listed as single-group, with allocation recorded as NA and masking as NONE.
Primary question
How many participants had non-serious adverse events and how many had serious adverse events during the stated Study 105 timeframe?
The primary objective was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis. The secondary objective was to evaluate efficacy. The registry lists secondary measures covering change and rate of decline in percent predicted forced expiratory volume in 1 second (FEV1), respiratory-domain scores on the Cystic Fibrosis Questionnaire-Revised (CFQ-R), pulmonary exacerbation events and their duration, and weight.
This is a descriptive safety question rather than a randomized comparison of treatment strategies. The registry record reports 70 participants with serious adverse events and 190 with non-serious adverse events among 192 in the VX-770 group. Those counts describe the recorded experience in this group; without a separate control group, they do not establish how the counts compare with what would have occurred under another treatment or no treatment.
The registry identifies the study as a rollover study, but eligibility criteria and details of participants’ prior study participation are not reported in the registry record. The results therefore address the recorded safety experience in the defined safety set, not a broader population or a comparison between treatment choices.
3. Trial Design
NCT01117012 is a Phase 3, open-label, single-group rollover study titled Rollover Study of VX-770 in Cystic Fibrosis Subjects. Its official title describes an open-label rollover study evaluating long-term safety and efficacy of VX-770 in subjects with cystic fibrosis. The sponsor is Vertex Pharmaceuticals Incorporated. The record lists the study as completed, with a start date of 2010-07 and primary completion in 2014-05.
There is a single VX-770 group. Participants received ivacaftor as a 150 milligram (mg) tablet orally twice daily (q12h). Masking is recorded as NONE, and allocation is recorded as NA. These design features mean the registry record does not describe randomized assignment or blinded treatment assessment. It also does not report a concurrent control group, so the study’s primary safety counts are not a between-group contrast.
The recorded enrollment is 192. For the primary safety outcome, the registry defines the Safety Set as all participants who received at least 1 dose of study drug during Study 105. The timeframe for the primary outcome is Study 105: Day 1 up to Week 168. This stated outcome timeframe should be distinguished from the secondary outcome measures whose descriptions specify assessment through Week 96.
The primary objective concerns long-term safety; the secondary objective concerns efficacy. The registry names secondary measures for FEV1, CFQ-R respiratory-domain score, pulmonary exacerbation event rate and duration, and weight. Detailed eligibility criteria, visit schedules, prior-study requirements, and the protocol’s rationale for the design are not reported in the registry record. The record also does not report a sample-size justification for the trial itself.
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: A total of 192 subjects were enrolled. Efficacy results are presented as per the treatment received in previous study (Placebo/Ivacaftor \[VX-770\]).
Overall Study
| Stage | Placebo/VX-770 | VX-770/VX-770 |
|---|---|---|
| STARTED | 89 | 103 |
| COMPLETED | 81 | 98 |
| NOT COMPLETED | 8 | 5 |
Reasons for not completing this period:
| Reason | Placebo/VX-770 | VX-770/VX-770 |
|---|---|---|
| Adverse Event | 0 | 1 |
| Death | 1 | 1 |
| Withdrawal by Subject | 3 | 0 |
| Undefined | 4 | 3 |
Analysis population for the primary outcome, as defined in the registry: Safety Set included all participants who received at least 1 dose of study drug during Study 105.
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: Full Analysis Set included all participants who were enrolled and received at least 1 dose of study drug during Study 105.
| Characteristic | Unit | Placebo/VX-770 | VX-770/VX-770 | Total |
|---|---|---|---|---|
| Participants analysed | 89 | 103 | 192 | |
| Age, Continuous | years | 22.0 (SD 10.94) | 23.2 (SD 11.55) | 22.6 (SD 11.26) |
| Sex: Female, Male: Female | Participants | 44 | 58 | 102 |
| Sex: Female, Male: Male | Participants | 45 | 45 | 90 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs), measured over Study 105: Day 1 up to Week 168.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with non-serious adverse events (AEs) and serious adverse events (SAEs). The registry defines an AE as an untoward medical occurrence during the study, including an unfavorable or unintended sign, symptom, or disease, whether or not considered related to study drug. It includes a newly occurring event or a previous condition that increased in severity or frequency after informed consent and assent, where applicable.
An SAE is defined by specified consequences, regardless of the event’s relationship to study drug. These include death, a life-threatening adverse experience, inpatient hospitalization or prolonged hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event. Non-serious AEs are defined as AEs other than SAEs.
Counting participants with events is a direct way to describe how many people in the safety set experienced each category during the stated observation period. It is useful for summarizing the occurrence of safety events, but a participant count is not an event rate: it does not by itself express events per unit of time or account for differences in individual exposure. The registry does not report an exposure-adjusted primary safety rate.
The reported categories can overlap at the participant level: a person could have had both a serious and a non-serious event. The counts should therefore be read separately, not added to produce a total number of participants. The primary endpoint is a safety description; it does not, by itself, establish whether an event was caused by treatment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports 70 participants with SAEs and 190 with non-serious AEs in the VX-770 group, with 192 participants at risk for each category. These are participant counts, not a comparison of event rates between treatment groups. The SAE and non-serious AE categories are reported separately, and the same participant may be represented in both. Adding the counts would not provide a valid total number of affected participants.
The SAE count is accompanied by listed serious-event terms. Infective pulmonary exacerbation of cystic fibrosis is listed at 21.9%, haemoptysis at 3.6%, influenza at 2.1%, distal intestinal obstruction syndrome at 2.1%, bronchopulmonary aspergillosis allergic at 1.0%, and abdominal pain at 1.0%. These figures describe the registry’s listed terms; they do not give a treatment-versus-control contrast. The record does not provide a comparable set of event counts for an untreated or alternative-treatment group.
The outcome timeframe is Study 105: Day 1 up to Week 168. The count is therefore tied to the study’s stated observation window, rather than being a measure of events in a common fixed period for every participant. Individual follow-up duration and exposure time are not reported in the registry record. A participant count cannot show whether event occurrence was concentrated early or late in follow-up, nor does it distinguish event frequency from event duration.
There is no reported estimate of uncertainty, such as a confidence interval, around these counts in the registry record. General statistical interpretation: a confidence interval, when calculated, describes the range of values compatible with the data under the method’s assumptions. Without an interval or a stated inferential analysis, the reported counts should be read as descriptive observations rather than as precise estimates of an underlying risk.
The study’s open-label, single-group design constrains interpretation. The registry record does not describe a randomized comparator, and the counts cannot isolate the contribution of treatment from the underlying condition, other care, or factors affecting event reporting. The AE definition explicitly includes events whether or not judged related to study drug. The counts consequently summarize what was recorded during the study, not a causal treatment effect.
The secondary objective was efficacy, and the registry lists secondary measures concerning lung function, respiratory symptoms, exacerbations, and weight. Numerical results for those measures are not reported in the registry record. Their presence as named outcomes does not establish the direction, size, or precision of any change. The primary safety counts likewise do not answer those efficacy questions.
The registry reports the study as completed, but the completion status does not change the design limitations or supply missing statistical details. The record does not report a formal analysis plan, confidence intervals, adjustment for multiple outcomes, or a missing-data approach. In consequence, interpretation should remain focused on the stated participant counts and the limitations of a descriptive, single-group record.
9. Statistical Methods Explained
The reported primary result is a count of participants in event categories. The record gives the number at risk and the number affected for serious and non-serious AEs in the VX-770 group. These counts are straightforward to read descriptively: they tell how many participants were recorded as having events in each category during the stated timeframe. The count does not communicate event severity beyond the SAE/non-serious classification, the number of repeated events per person, or the amount of time each participant was observed.
What does a participant count represent?
A participant count treats each person as affected or not affected for the specified category, regardless of whether that person experienced one event or multiple events in that category. It differs from an event count, which could count repeated occurrences, and from an incidence rate, which relates events or affected participants to time at risk. General statistical explanation: these measures answer different questions, so they should not be substituted for one another. The registry’s primary measure is explicitly a number of participants.
The SAE and non-serious AE results should be read as separate categories. The registry defines non-serious AEs as AEs other than SAEs, but a participant can experience events in both categories at different times or in connection with different occurrences. The available counts do not state how many participants experienced both categories. They cannot be combined to obtain a unique count of people with any AE.
Why is the design important?
The study is described as open-label and single-group, with no comparator group listed. An open-label study does not mask treatment assignment from participants or investigators as described in the registry. A single-group study can describe events observed in its treated group, but it does not supply a concurrent group to represent what might have happened under another condition. General statistical explanation: a treatment effect is a contrast between outcomes under different treatment conditions; a single group’s observed outcome alone does not provide that contrast.
Even an observed difference from a baseline value, where such a measure is reported, is not equivalent to a randomized comparison. Changes over time can reflect the condition’s course, concurrent care, changes in measurement, or other influences. The registry names secondary outcomes measured from Study 105 baseline, but numerical results for those outcomes are not reported in the registry record. No conclusions about their magnitude or precision can be drawn from their titles alone.
How should uncertainty be understood?
General statistical explanation: a point estimate is a single summary derived from observed data; an interval estimate conveys uncertainty around that summary under specified assumptions. For a participant proportion, a confidence interval would express uncertainty about the underlying proportion in a target population, not uncertainty about whether the recorded participants experienced the events. No confidence intervals or inferential results for the primary counts are reported in the registry record.
General statistical explanation: precision depends on the amount and structure of information, not merely on the visual size of a count. Follow-up time, loss to follow-up, outcome definitions, and the way participants were selected can all matter. The registry gives the primary timeframe and defines the Safety Set, but individual follow-up, missingness, and the handling of incomplete observations are not reported. The record therefore does not allow an assessment of whether all participants contributed comparable observation time.
What do the planning assumptions mean?
The registry’s sample-size assumptions describe a planning illustration computed from the observed effect, not the design of this single-group trial. The illustration assumes a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is a stated fraction of the observed effect. General statistical explanation: such an illustration asks what sample size a hypothetical study might require under its chosen assumptions. It is not observed power, and it does not mean that the rollover study was designed with those assumptions. The record does not report a trial-specific sample-size justification.
General statistical explanation: power is a design property under specified assumptions about an effect, variability, significance threshold, and analysis. It is not a measure of how likely the observed result is to be true. A planning calculation based on an observed effect can be especially dependent on that estimate and on the chosen assumptions. It should not be interpreted as evidence that the current study had adequate power to answer a question that its single-group design does not directly compare.
What analytical details are absent?
The registry record does not report the statistical procedures used to analyze the primary outcome, any adjusted or unadjusted effect estimate, or an interval estimate. It does not describe a prespecified analysis population beyond the Safety Set definition for the primary outcome. The registry lists secondary outcomes, but analysis methods and numerical results for them are not reported in the registry record.
General statistical explanation: outcomes expressed as rates, such as annualized exacerbation event rates, require methods that account for the relationship between event counts and time observed. If event counts vary more than a simple model expects, over-dispersion can make a model’s standard errors too small unless the method accounts for it. These are general considerations, not claims about the methods used in this study; the registry record does not report the methods used for the named rate outcomes.
Likewise, general statistical explanation: analyses of repeated measurements can depend on how within-participant observations are related, while analyses of time-to-event outcomes depend on event timing and censoring. No such details should be inferred from an outcome title. The registry record does not state the model, assumptions, or missing-data procedures used for any of the secondary measures.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety outcome and secondary efficacy outcomes covering FEV1, CFQ-R respiratory-domain score, pulmonary exacerbation events and their duration, and weight. Multiple outcomes create a multiplicity issue when several statistical tests are used to support conclusions: the chance of at least one apparently positive result can increase as the number of tests increases. General statistical explanation: prespecifying a hierarchy or applying a multiplicity adjustment can address that issue, but the appropriate approach depends on the study’s aims and analysis plan.
The registry record does not report a multiplicity strategy, test hierarchy, or adjustment method. It also does not provide inferential results for the listed secondary outcomes. No statement about whether multiplicity was controlled can be made from the outcome list alone. The record lists no analysis entries, and a detailed statistical analysis plan is not reported in the registry record.
For the primary safety outcome, the Safety Set is defined as all participants who received at least 1 dose of study drug during Study 105. The displayed at-risk count is 192. The registry record does not explain how missing safety observations, withdrawal, incomplete follow-up, or differing exposure duration were handled. It does not report a separate efficacy analysis population or explain how missing values for the secondary outcomes were treated.
General statistical explanation: missing data can affect estimates when the probability or pattern of missingness is related to participant characteristics or outcomes. Different assumptions and methods can lead to different interpretations. Because the registry does not describe the extent or handling of missingness, the impact on the reported counts and any unreported secondary analyses cannot be assessed from this record.
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 |
|---|---|---|---|
| VX-770 | 70 | 192 | 36.5% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| VX-770 | 190 of 192 |
Most frequent serious adverse event terms
| Event term | Organ system | VX-770 |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 42 of 192 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 7 of 192 |
| Influenza | Infections and infestations | 4 of 192 |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | 4 of 192 |
| Bronchopulmonary aspergillosis allergic | Infections and infestations | 2 of 192 |
| Abdominal pain | Gastrointestinal disorders | 2 of 192 |
| Constipation | Gastrointestinal disorders | 2 of 192 |
| Headache | Nervous system disorders | 2 of 192 |
| Appendicitis | Infections and infestations | 1 of 192 |
| Aspergilloma | Infections and infestations | 1 of 192 |
Most frequent non-serious adverse event terms
| Event term | Organ system | VX-770 |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 94 of 192 |
| Cough | Respiratory, thoracic and mediastinal disorders | 86 of 192 |
| Upper respiratory tract infection | Infections and infestations | 65 of 192 |
| Headache | Nervous system disorders | 45 of 192 |
| Nasopharyngitis | Infections and infestations | 39 of 192 |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 39 of 192 |
| Pyrexia | General disorders | 35 of 192 |
| Sinusitis | Infections and infestations | 29 of 192 |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | 26 of 192 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 26 of 192 |
Serious events with the highest arm-level rate: infective pulmonary exacerbation of cystic fibrosis (21.9%), haemoptysis (3.6%), influenza (2.1%), distal intestinal obstruction syndrome (2.1%), bronchopulmonary aspergillosis allergic (1.0%).
The VX-770 group has 70 participants reported with serious adverse events and 190 with non-serious adverse events, with 192 at risk in each category. These are counts of participants, not counts of all event occurrences. A participant may have had more than one event, and the same participant may appear in both categories; the counts should not be added.
The listed serious-event terms include infective pulmonary exacerbation of cystic fibrosis (21.9%), haemoptysis (3.6%), influenza (2.1%), distal intestinal obstruction syndrome (2.1%), bronchopulmonary aspergillosis allergic (1.0%), and abdominal pain (1.0%). These listed terms are not a comparison with another group. The registry record does not report deaths in the group listing.
The registry’s SAE definition includes events meeting specified seriousness criteria regardless of their relationship to study drug. The non-serious category includes all AEs except SAEs. Thus, these counts describe events recorded under the stated definitions; they do not establish that treatment caused an event or that an event was unrelated to treatment.
The timeframe is Study 105: Day 1 up to Week 168. The registry does not report participant-level follow-up duration or an exposure-adjusted event rate for this endpoint. The counts are best read as descriptive safety findings in the recorded safety set, not as a measure of comparative risk.
12. What the Result Does, and Does Not, Mean
The results describe a long-term safety study of VX-770 in a single group of participants with cystic fibrosis. The registry reports 70 participants with SAEs and 190 with non-serious AEs among 192 at risk. The event definitions, safety population, and observation timeframe provide context for those counts. The record also lists serious-event terms with percentages.
These observations do not establish a causal effect of treatment. There is no concurrent comparator group in the registry record, and the open-label, single-group design does not support a randomized treatment contrast. The AE definition includes events regardless of whether they were judged related to study drug. The counts therefore show what was recorded during the study, not what would have happened to the same participants under another treatment condition.
The figures do not represent the number of event episodes, a rate per unit of exposure, or a comparison of risk across treatments. The separate serious and non-serious counts may include the same participant and should not be summed. The record does not report uncertainty intervals for these counts, so their precision as estimates beyond the observed study group is not quantified.
The secondary outcomes indicate that the study also planned to assess lung function, respiratory symptoms, pulmonary exacerbations, and weight. Numerical secondary results and their statistical analyses are not reported in the registry record. The named outcomes alone cannot show whether those measures changed, by how much, or with what precision.
Any sample-size planning illustration based on an observed effect is a hypothetical planning exercise under stated assumptions, not observed power and not a substitute for a randomized comparison. The registry record does not report the trial’s own sample-size justification. Interpretation should therefore remain limited to the study’s descriptive safety record and the design and reporting details available.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between describing outcomes and estimating a comparative treatment effect. Its primary outcome records the number of participants with serious and non-serious AEs in a single group. Those counts can convey the observed safety experience, but they cannot answer how that experience differs from a comparator that was not part of the recorded design.
The safety definitions and timeframe matter to interpretation. An AE includes events whether or not considered related to study drug, while SAE status is based on specified consequences. The primary timeframe extends from Study 105: Day 1 up to Week 168. The observed counts should be understood in that context, and not mistaken for a rate adjusted for individual exposure time.
The record also illustrates why outcome names are not results. It lists secondary measures across lung function, respiratory-domain scores, pulmonary exacerbations, and weight, but numerical estimates and analysis details for those outcomes are not reported in the registry record. Without those results, their direction and precision cannot be evaluated.
Finally, a planning illustration and a trial’s actual design answer different questions. The stated illustration uses a hypothetical two-arm allocation and specified assumptions; the actual trial is single-group. Planning assumptions do not turn a descriptive study into a comparative study, and they are not observed power. Keeping design, estimation, and planning distinct helps prevent overinterpretation of registry findings.
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 principal trial-specific limitation is the single-group, open-label design. The registry lists no randomized allocation or concurrent control group, so the safety counts are descriptive rather than comparative. The study’s rollover designation does not supply details about eligibility, previous participation, or how the study group was assembled; those details are not reported in the registry record.
The primary endpoint is a participant count over Study 105: Day 1 up to Week 168. Individual observation time and exposure duration are not reported, and no exposure-adjusted primary safety rate is given. The registry also does not report whether follow-up was complete for all participants or how missing safety information was handled. These omissions limit assessment of how completely the stated period was observed across participants.
The record lists selected serious-event terms and their percentages, but does not provide a comparator group or a causal assessment for those figures. Deaths are not reported in the registry record. Numerical results for the secondary outcomes are also not reported, despite their inclusion in the outcome list. The record does not provide confidence intervals, the statistical analysis plan, a multiplicity strategy, or a trial-specific sample-size justification.
These limits mean the registry record supports a description of the recorded safety counts, but not a controlled estimate of treatment effect, a complete evaluation of efficacy, or a detailed assessment of statistical uncertainty. Further protocol and analysis details are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Rollover Study of VX-770 in Cystic Fibrosis Subjects — NCT01117012.
- Registry record last updated 2015-07-07; status last verified 2015-06.
- Results first posted 2015-07-07.
- 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
NCT01117012 is a completed Phase 3, open-label, single-group rollover study sponsored by Vertex Pharmaceuticals Incorporated. Its start date is 2010-07, and its primary completion date is 2014-05. The intervention is VX-770 (ivacaftor), given as a 150 milligram (mg) tablet orally twice daily (q12h); recorded enrollment is 192.
The primary objective was long-term safety. The registry reports 70 participants with serious adverse events and 190 with non-serious adverse events in the VX-770 group, with 192 at risk for each category. The primary timeframe is Study 105: Day 1 up to Week 168. The registry also lists secondary efficacy outcomes concerning FEV1, CFQ-R respiratory-domain score, pulmonary exacerbations, and weight, but their numerical results are not reported in the registry record.
Because the study is single-group and open-label, the reported safety counts are descriptive and do not provide a randomized comparison. Further statistical analysis details are not reported in the registry record.