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 Evaluation of the Impact of Nurse-led Telephone on Treatment Compliance (NCT02483416), a registered phase not stated study. The primary outcome is The Cumulated Dose Milligram (mg) of Oral Targeted Therapy During the 3-month Follow-up..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02483416 |
| Phase | Phase not stated |
| Condition | Carcinoma, Non-Small-Cell Lung |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Boehringer Ingelheim |
| Results posted | Yes |
2. Clinical Question
Population
Patients treated for locally advanced or metastatic non-small-cell lung cancer with activating EGFR mutation(s). The record describes treatment with oral targeted therapy and an intended follow-up period of 3 months.
Intervention
Remote additional personalised nurse-led telephone follow-up alongside routine care from the medical team. The brief summary describes nurse calls during follow-up, while stating that the nurse would not intervene in medical care or answer questions about the disease or treatment.
Comparator
Routine healthcare from the medical team without the additional remote nurse-led follow-up. The registry describes these two care approaches, but allocation for the actual comparison is not stated.
Primary question
What was the cumulated dose, in milligrams, of oral targeted therapy taken during the 3-month follow-up? The primary endpoint concerns the amount taken, not a direct measure of clinical benefit. The record does not report a comparison of this endpoint between the care groups.
The clinical question is whether adding personalised telephone contact from a nurse is associated with a different amount of oral targeted therapy taken over follow-up, compared with routine care alone. The planned intervention was an addition to usual medical-team care, not a replacement for it. The record states that the medical team remained the patient's main contact for questions about the disease and treatment.
The primary analysis population is described as randomised patients meeting essential inclusion and exclusion criteria and having information available for the major evaluation criteria, including the primary criterion, using observed cases. However, the registry does not state how patients were allocated in the actual study. It therefore does not establish that the reported groups form a randomized, like-for-like comparison. Most importantly, no primary endpoint values or analysis results are reported in the registry record, so the clinical question cannot be answered from this record.
3. Trial Design
The study is titled “Evaluation of the Impact of Nurse-led Telephone on Treatment Compliance,” and is also identified as the PARTAGE Study. Boehringer Ingelheim is listed as the sponsor. The condition is non-small-cell lung carcinoma. The study start date is 2015-11-19, and the primary completion date is 2017-02-27. Its status is TERMINATED.
The enrollment listed in the record is 30. The brief summary describes a planned study lasting 27 months, with recruitment intended to last approximately 24 months and each patient's participation approximately 3 months. It describes routine care alone as one group and routine care plus additional nurse calls as the other. It also describes a 3:1 allocation plan and planned group sizes of 100 and 300 patients. Those planned figures do not match the enrollment listed in the record. The actual allocation method is not stated, so the planned ratio should not be treated as evidence of the allocation used or of a valid randomized comparison.
For the group intended to receive calls, the brief summary lists contacts at D1, D7, D14, D21, D28, D44, D59 and D89. It says all patients would continue to be seen according to normal practice by the study medical team. The primary endpoint is the cumulated dose, in milligrams, of oral targeted therapy taken during the 3-month follow-up, summarized as a median with a full range.
The record lists 25 outcome measures and zero analyses. It does not report primary endpoint values, a between-group estimate, or an uncertainty interval. The study's termination status and listed enrollment do not, by themselves, explain why it ended or establish the completeness of follow-up. A reason for termination 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: Patients for whom a decision of treatment with afatinib monotherapy has been taken in the frame of its marketing authorization were included in this trial.
Overall Study
| Stage | Group With Nurse-led Telephone Follow-up | Group Without Nurse-led Telephone Follow-up |
|---|---|---|
| STARTED | 21 | 7 |
| COMPLETED | 18 | 6 |
| NOT COMPLETED | 3 | 1 |
Reasons for not completing this period:
| Reason | Group With Nurse-led Telephone Follow-up | Group Without Nurse-led Telephone Follow-up |
|---|---|---|
| Other reason | 0 | 1 |
| Treatment stop | 3 | 0 |
Analysis population for the primary outcome, as defined in the registry: The primary analysis population will be the population of randomised patients who satisfy the essential inclusion and exclusion criteria, and for whom information is available for the major evaluation criteria (in particular the primary criterion)- Observed cases
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: Randomised patients who satisfy the essential inclusion and exclusion criteria, and for whom information is available for the major evaluation criteria
| Characteristic | Unit | Group With Nurse-led Telephone Follow-up | Group Without Nurse-led Telephone Follow-up | Total |
|---|---|---|---|---|
| Participants analysed | 21 | 7 | 28 | |
| Age, Continuous | years | 63.6 (SD 10.9) | 69.9 (SD 12.6) | 65.2 (SD 11.4) |
| Sex: Female, Male: Female | Participants | 16 | 3 | 19 |
| Sex: Female, Male: Male | Participants | 5 | 4 | 9 |
| Race and Ethnicity Not Collected | Participants | — | — | 0 |
6. Primary Endpoint
The registry lists the primary outcome as The Cumulated Dose Milligram (mg) of Oral Targeted Therapy During the 3-month Follow-up., measured over 3 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | milligram (mg) |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the cumulated dose, in milligrams, of oral targeted therapy taken during the 3-month follow-up. The registry defines this as the sum of the doses of tablets taken by the patient between visits, accumulated over the follow-up period. The planned summary measure is the median, with the full range as the measure of dispersion.
This endpoint describes medication-taking in terms of dose amount. It is distinct from a clinical outcome such as disease control, survival, or quality of life. A dose total can capture differences in the amount taken, including changes during follow-up, but the endpoint alone does not explain why a dose was or was not taken. The registry lists other outcomes concerning doses not taken following a medical-team decision or a patient decision, as well as a Girerd Questionnaire score and patient satisfaction. These outcomes offer related perspectives, but they are not substitutes for the stated primary endpoint.
The record does not report the median dose, its full range, or an analysis comparing the groups. The endpoint's values and the precision of any group contrast are therefore not reported in the registry record. Without those results, the endpoint definition clarifies what was intended to be measured but cannot establish what the study observed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive constraint is that the registry record contains no outcome analyses. It lists 25 outcome measures but zero analyses, and it gives no primary endpoint values, group contrast, confidence interval, or p-value. Consequently, there is no reported estimate of how much oral targeted therapy participants took, either overall or by group, and no statistical basis in this record for assessing the size or precision of a difference.
The enrollment listed is 30, whereas the brief summary describes planned group sizes of 100 and 300. These are different types of information: the listed enrollment is the record's enrollment figure, while the larger figures appear in the description of the intended study groups. They should not be combined or treated as evidence that the intended sample was achieved. The actual group sizes for a primary endpoint analysis are not reported in the registry record. The adverse-event information uses a denominator of 28 participants in total, which is a safety reporting count and should not be assumed to be the primary analysis population.
The record's primary analysis population is described as randomised patients who meet essential eligibility criteria and have information available for the major evaluation criteria, analyzed as observed cases. Yet the actual allocation method is not stated, and the registry record does not provide the number meeting that analysis definition. The planned group descriptions therefore do not establish that a valid randomized comparison was carried out. The record itself identifies the arms as not a like-for-like control comparison.
The registry lists the primary endpoint as a median and full range. These summaries would describe the distribution of dose totals, but neither a median nor a range is reported. No measure of uncertainty is posted. In general, a confidence interval describes a range of values compatible with the data and statistical model; without a reported estimate and interval, precision cannot be judged. Similarly, a p-value cannot be interpreted when no analysis result is reported.
The registry includes adverse-event counts, but they do not replace efficacy or adherence outcome analyses. Safety counts describe reported events among the participants included in those summaries. They do not provide the missing primary endpoint comparison, nor do they establish a causal effect of the follow-up approach. The appropriate conclusion is limited: the record documents a study plan, a listed enrollment, and safety summaries, but it does not report analyzed results for the primary clinical question.
9. Statistical Methods Explained
The registry describes a primary endpoint based on the cumulated dose of oral targeted therapy, summarized using a median and full range. It also describes an analysis population of eligible randomised patients with available information for the major evaluation criteria, using observed cases. However, the record does not report the statistical analysis actually performed or any primary endpoint results. The methodological concepts below explain how such measures are generally read; they do not establish that a particular analysis was conducted in this study.
What does a median represent?
In general, the median is the middle value after observations are ordered. It describes the center of a distribution and is less influenced by extreme values than a mean. Reporting a full range adds the lowest and highest observed values, but the range alone does not show how values are distributed between those endpoints. A median and range can describe each group, yet a comparison also needs an appropriate estimate of the difference or another prespecified contrast and information about its uncertainty. Such results are not reported in the registry record.
Why might dose totals vary?
In general, dose totals can differ because patients take different amounts of medication over time. A dose may also change after a decision by the medical team, or because of a patient's decision. The registry lists secondary outcomes that distinguish these reasons, including dose reductions and temporary or definitive interruptions. These categories clarify the intended distinctions, but no values for those outcomes are reported. Without the observed data and analysis, it is not possible to determine how much each reason contributed to dose totals.
What does an observed-cases population imply?
In general, an observed-cases analysis uses participants for whom the relevant outcome information is available, rather than assigning unobserved outcomes automatically. This approach can leave the analysis smaller than enrollment and can be affected if missingness is related to participants' outcomes or group assignment. The registry's description specifies observed cases and available information for the major evaluation criteria. It does not report how many participants contributed to the primary analysis, how much information was missing, why data were missing, or whether any sensitivity analyses were conducted. The actual consequences of missing data therefore cannot be assessed.
How should the study groups be understood?
The brief summary describes routine care alone and routine care plus nurse calls, and gives a planned 3:1 allocation description. But the allocation method for the study as conducted is not stated. The registry's own classification says this is not a randomized comparison and that the arms are not a like-for-like control comparison. In general, when groups are not established as comparable, observed differences may reflect differences between the patients or their care pathways as well as the intervention. A statistical adjustment could sometimes address measured differences, but no adjustment plan or adjusted result is reported in the registry record. The group descriptions alone cannot establish causal attribution.
What can a p-value or confidence interval add?
In general, a p-value evaluates how compatible observed data are with a specified statistical model and null hypothesis; it is not the probability that a treatment works or that a result occurred by chance. A confidence interval communicates uncertainty around an estimate and helps readers assess which effect sizes remain compatible with the data and method. Neither statistic can be interpreted in isolation from the endpoint definition, analysis population, and comparison design. No p-value, confidence interval, or primary endpoint estimate is reported in the registry record.
How should the sample-size illustrations be read?
The planning illustrations are described as based on the observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation. Their effect scenarios are expressed as fractions of the observed effect. For non-time-to-event outcomes, the illustrations are expressed as participants per arm; time-to-event illustrations use total events. These are planning illustrations, not observed power and not evidence that the study enrolled the illustrated number of participants. Because no primary endpoint analysis is reported, the observed effect underlying such illustrations is not reported in the registry record. The trial's own sample-size justification is also not reported in the registry record.
General statistical practice also distinguishes a planned analysis from one actually carried out. A protocol may specify an endpoint, model, adjustment strategy, and missing-data method in advance, but those details cannot be inferred from a registry outcome description. The statistical analysis plan, any interim analyses, and the methods used for the posted safety summaries are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 25 outcome measures, including the primary cumulated-dose endpoint, a Girerd Questionnaire score, several categories of doses not taken, and patient satisfaction. Having multiple outcomes creates a general multiplicity issue: if many hypotheses are tested, the chance of at least one apparently notable result can increase. In general, a prespecified hierarchy or adjustment method can help address this, but the registry record does not report whether any multiplicity strategy was planned or used. With zero analyses listed, there are no reported outcome tests to interpret.
The record defines the primary analysis population as eligible randomised patients with available information for major evaluation criteria, using observed cases. This identifies an intended restriction to participants with observed outcome data, but does not report how many participants were eligible for that analysis or how many had usable primary endpoint values. The number and pattern of missing primary endpoint observations, reasons for missingness, and any imputation or sensitivity analyses are not reported in the registry record.
The planned sample-size description includes groups of 100 and 300, whereas listed enrollment is 30. The record does not provide the study's own sample-size justification, explain whether the planned enrollment changed, or report the analysis population's size. The sample-size illustrations are planning aids based on an observed effect and stated assumptions; they are not observed power and cannot substitute for a reported estimate with uncertainty. The registry record does not report whether interim analyses occurred or how any stopping decisions were made. The TERMINATED status alone does not identify a reason or establish whether outcome data collection was complete.
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 |
|---|---|---|---|
| Group With Nurse-led Telephone Follow-up | 2 | 21 | 9.5% |
| Group Without Nurse-led Telephone Follow-up | 1 | 7 | 14.3% |
| Total | 3 | 28 | 10.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group With Nurse-led Telephone Follow-up | 0 of 21 |
| Group Without Nurse-led Telephone Follow-up | 0 of 7 |
| Total | 0 of 28 |
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 |
|---|---|
| Group With Nurse-led Telephone Follow-up | 21 of 21 |
| Group Without Nurse-led Telephone Follow-up | 6 of 7 |
| Total | 27 of 28 |
Most frequent serious adverse event terms
| Event term | Organ system | Group With Nurse-led Telephone Follow-up | Group Without Nurse-led Telephone Follow-up | Total |
|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 1 of 21 | 1 of 7 | 2 of 28 |
| Drug-induced liver injury | Hepatobiliary disorders | 1 of 21 | 0 of 7 | 1 of 28 |
| Sinusitis | Infections and infestations | 0 of 21 | 1 of 7 | 1 of 28 |
| Transaminases increased | Investigations | 1 of 21 | 0 of 7 | 1 of 28 |
| Dehydration | Metabolism and nutrition disorders | 1 of 21 | 0 of 7 | 1 of 28 |
| Renal failure | Renal and urinary disorders | 1 of 21 | 0 of 7 | 1 of 28 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group With Nurse-led Telephone Follow-up | Group Without Nurse-led Telephone Follow-up | Total |
|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 17 of 21 | 4 of 7 | 21 of 28 |
| Dry skin | Skin and subcutaneous tissue disorders | 8 of 21 | 3 of 7 | 11 of 28 |
| Rash | Skin and subcutaneous tissue disorders | 8 of 21 | 3 of 7 | 11 of 28 |
| Fatigue | General disorders | 5 of 21 | 2 of 7 | 7 of 28 |
| Decreased appetite | Metabolism and nutrition disorders | 5 of 21 | 2 of 7 | 7 of 28 |
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | 5 of 21 | 0 of 7 | 5 of 28 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 4 of 21 | 1 of 7 | 5 of 28 |
| Dry eye | Eye disorders | 4 of 21 | 1 of 7 | 5 of 28 |
| Nausea | Gastrointestinal disorders | 4 of 21 | 0 of 7 | 4 of 28 |
| Asthenia | General disorders | 2 of 21 | 2 of 7 | 4 of 28 |
Serious events with the highest arm-level rate: diarrhoea (14.3%), sinusitis (14.3%), drug-induced liver injury (4.8%), transaminases increased (4.8%), dehydration (4.8%).
For the group with nurse-led telephone follow-up, the record lists 2 serious adverse-event-affected participants among 21 at risk, 21 participants with other adverse events among 21 at risk, and zero deaths. For the group without nurse-led telephone follow-up, it lists 1 serious adverse-event-affected participant among 7 at risk, 6 participants with other adverse events among 7 at risk, and zero deaths. The total row lists 3 serious adverse-event-affected participants and 27 participants with other adverse events among 28 at risk, with zero deaths.
The serious-event terms listed include Diarrhoea and Sinusitis, each at 14.3%, and Drug-induced liver injury, Transaminases increased, Dehydration, and Renal failure, each at 4.8%. These are registry-listed percentages and terms; they should not be interpreted as comparative risks or attributed to the telephone follow-up based on this record. The safety denominators differ from the enrollment listed for the study, and the record does not explain the difference. Counts of affected participants do not by themselves describe event timing, duration, severity beyond the serious classification, or causality. No comparative statistical analysis of safety is reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record establishes that a study was registered to examine nurse-led telephone follow-up alongside routine care, with the cumulated dose of oral targeted therapy over 3 months designated as the primary endpoint. It also records a TERMINATED status, enrollment of 30, 25 outcome measures, and zero analyses. These details describe the study's status and reporting record, not an estimate of the intervention's effect.
Because the primary endpoint values and analysis are not reported, the record does not show whether the groups differed in dose taken, the size or direction of any difference, or how precisely an effect was estimated. It also does not report an analysis linking the follow-up approach to adherence, clinical outcomes, or patient satisfaction. No conclusion that the calls increased or decreased medication-taking can be drawn from the registered results.
The planned group descriptions and proposed allocation ratio do not establish how participants were actually assigned. The record states that allocation is not stated and that the arms are not a like-for-like control comparison. Thus, even if group outcome values were available, interpretation would need to account for the design and comparability of the groups. The safety summaries provide counts and listed event terms, but do not supply a causal comparison between care approaches.
The study may still be relevant as a record of a planned approach to supporting patients taking oral targeted therapy. However, relevance of the question is separate from evidence about its answer. The registry entry does not report the reason for termination, whether follow-up was complete, or why primary endpoint analyses are absent. Those omissions prevent a fuller account of the study's conduct and results. The most defensible reading is that the registry record documents the intended question and some safety information, while leaving the primary statistical question unanswered.
13. Why This Trial Matters Statistically
This study raises a useful statistical distinction between defining an outcome and reporting evidence about it. The registry gives a specific primary measure: the accumulated milligram dose of oral targeted therapy over follow-up, summarized by a median and full range. That definition tells readers what investigators intended to measure. It does not, by itself, reveal the observed values, a group contrast, or the uncertainty around that contrast.
The record also illustrates why planned enrollment and actual enrollment must be kept separate. The brief summary describes planned groups of 100 and 300, while the enrollment listed is 30. A planned allocation description cannot establish the actual assignment process, analysis sample, or adequacy of the final data. The registry does not report the sample-size justification or an analyzed sample size for the primary endpoint.
There is also a design issue: the registry says allocation is not stated and the arms are not a like-for-like control comparison. Statistical comparisons are most interpretable when the groups are comparable or when differences are handled transparently through a justified analysis. No such analysis is reported. The missing primary results therefore matter not only because effect size and precision are unavailable, but also because the record does not establish the basis for a causal interpretation.
Finally, the presence of many listed outcomes alongside zero analyses highlights the difference between outcome registration and outcome reporting. Outcomes, missing-data handling, multiplicity decisions, and analysis populations all affect interpretation. Here, the record provides some definitions but little information about how the study's statistical questions were answered. This makes careful limits on interpretation essential.
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.
A major trial-specific limitation of interpretation is the mismatch between the planned group sizes described in the brief summary and the enrollment listed as 30. The registry does not explain whether the plan changed, how many participants entered each group, or how many contributed to the primary endpoint analysis. The safety summary's total at-risk count is 28, but the record does not explain how that group relates to enrollment or the primary analysis population.
The record states that the allocation method is not stated and identifies the arms as not a like-for-like control comparison. The primary population description refers to randomised patients, but that wording does not resolve the record's explicit uncertainty about allocation in the study as conducted. The actual allocation process and group comparability are not reported in the registry record.
The study is marked TERMINATED, but the reason for termination, completeness of outcome collection, and status of planned analyses are not reported. Although the primary endpoint is clearly defined, its results are absent. The number and pattern of missing observations, details of analysis methods, adjustment for group differences, multiplicity handling, and sensitivity analyses are also not reported in the registry record. These gaps prevent evaluation of the estimate, its precision, and the robustness of any conclusions.
The registry's sample-size illustrations use stated planning assumptions and an observed-effect basis, but they are not observed power, and the registry record does not provide the trial's own sample-size justification. The listed safety counts are useful descriptively but do not resolve the absent outcome analysis or demonstrate a comparative safety effect. These limitations arise from what the record reports and should not be mistaken for proof that particular analyses or data were never collected.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Evaluation of the Impact of Nurse-led Telephone on Treatment Compliance — NCT02483416.
- Registry record last updated 2019-03-01; status last verified 2018-10.
- Results first posted 2019-03-01.
- 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
The record describes the PARTAGE Study, sponsored by Boehringer Ingelheim, on nurse-led telephone follow-up and treatment compliance in patients with locally advanced or metastatic non-small-cell lung cancer with activating EGFR mutation(s). It lists enrollment of 30, a start date of 2015-11-19, a primary completion date of 2017-02-27, and status TERMINATED.
The primary endpoint is the cumulated milligram dose of oral targeted therapy during the 3-month follow-up, summarized as a median and full range. The brief summary describes routine care with or without additional nurse calls, but the actual allocation method is not stated, and the record says the arms are not a like-for-like control comparison. It lists 25 outcome measures and zero analyses. Primary endpoint results, the reason for termination, and the statistical analysis plan are not reported in the registry record. Safety counts are reported for 28 participants in total.