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 LDK378 in Adult Patients With ALK-activated NSCLC Previously Treated With Chemotherapy and Crizotinib (NCT01685060), a na phase 2 study. The primary outcome is Overall Response Rate (ORR) to LDK378 Per Investigator Assessment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01685060 |
| Phase | Phase 2 |
| Condition | Non-Small Cell Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 140 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Adults with ALK-activated non-small cell lung cancer previously treated with chemotherapy and crizotinib. The primary endpoint population is the Full Analysis Set, defined as patients who received at least one dose of ceritinib.
Intervention
LDK378, described in the record as ceritinib/LDK378, at 750 mg once daily in a fasted state. The study description says treatment could continue until specified reasons for stopping, including unacceptable toxicity, discontinuation by the investigator or patient, a new anti-cancer therapy, or death.
Comparator
There was no concurrent comparison group. The study is identified as single-arm, with allocation recorded as not applicable and masking as none. Consequently, the endpoint describes outcomes observed among treated participants rather than a contrast between treatment groups.
Primary question
What percentage of participants had a best overall confirmed complete or partial response, as assessed by the investigator using RECIST 1.1, during 6 cycles of 28 days up to 24 weeks? The registry record gives the endpoint definition and confidence-interval type, but the primary outcome value and its 95% confidence interval are not reported in the registry record.
That question is descriptive in this design. It concerns the proportion meeting a prespecified tumor-response definition in the study population; it is not a randomized estimate of how response would differ under LDK378 versus another treatment or no treatment. Other listed outcomes include response assessed by a blinded independent review committee, duration of response, disease control rate, time to response, and progression-free survival. Their presence does not supply comparative evidence, and their numeric results are not reported in the registry record.
3. Trial Design
This was a phase II, multicenter, single-arm, open-label study of LDK378 in adults with ALK-activated non-small cell lung cancer previously treated with chemotherapy and crizotinib. The registry lists enrollment as 140 and identifies the study as completed. Its recorded start date is 2012-11-26 and its primary completion date is 2016-03-29.
All enrolled participants were assigned to the LDK378 study treatment; there was no separate control arm. The intervention description specifies 750 mg once daily, fasted. Open-label means treatment masking was not used. Without random allocation or a concurrent comparator, observed response cannot by itself separate the treatment effect from other influences on outcome, such as the selected population, the course of disease, or assessment practices. Those are general considerations for interpreting a single-group study, not findings about what happened to individual participants.
The study description states that treatment continued until unacceptable toxicity precluded further treatment, the investigator or patient discontinued treatment, a new anti-cancer therapy began, or death occurred. It also says treatment could continue after investigator-assessed RECIST-defined progression when the investigator judged that there was clinical benefit. Tumor assessments were to continue on the stated assessment schedule while treatment continued; participants who stopped medication without progression were to remain under tumor assessment until investigator-assessed progression.
The record identifies a Full Analysis Set for the primary endpoint: patients who received at least one dose of ceritinib. How missing assessments were handled, whether the analysis set was applied exactly as described in the final analysis, and the detailed statistical analysis plan are not reported in the registry record. The existence of a treatment and assessment description does not establish the exact conduct of every participant’s follow-up.
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: Approximately 137 patients were planned to be enrolled. A total of 140 patients were enrolled and treated with ceritinib.
Overall Study
| Stage | LDK378 750mg |
|---|---|
| STARTED | 140 |
| Entered Post-treatment Efficacy f/u | 7 |
| Entered Survival Follow up | 98 |
| Discontinued From Study | 35 |
| COMPLETED | 0 |
| NOT COMPLETED | 140 |
Reasons for not completing this period:
| Reason | LDK378 750mg |
|---|---|
| Rollover patients | 16 |
| Adverse Event | 12 |
| Progressive disease | 69 |
| Physician Decision | 14 |
| Subject/guardian decision | 20 |
| Lost to Follow-up | 1 |
| Death | 8 |
Analysis population for the primary outcome, as defined in the registry: Full Analysis Set (FAS) consisted of all patients who received at least one dose of ceritinib.
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 (FAS) consisted of all patients who received at least one dose of ceritinib.
| Characteristic | Unit | LDK378 750mg |
|---|---|---|
| Participants analysed | 140 | |
| Age, Continuous | Years | 51.2 (SD 11.62) |
| Sex: Female, Male: Female | Participants | 70 |
| Sex: Female, Male: Male | Participants | 70 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate (ORR) to LDK378 Per Investigator Assessment, measured over 6 cycles of 28 days up to 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the overall response rate (ORR) by investigator assessment. It is defined as the percentage of participants whose best overall confirmed response was complete response or partial response, using RECIST 1.1. A complete response requires disappearance of all non-nodal target lesions and reduction of any pathological lymph nodes assigned as target lesions to a short axis < 10 mm. A partial response requires at least a 30% decrease in the sum of target-lesion diameters from the baseline sum.
ORR reduces tumor assessment to a binary classification for each participant: a confirmed complete or partial response meets the endpoint definition, and other assessed outcomes do not. The percentage is a population-level summary of that classification. The registry specifies a 95% confidence interval, which would express uncertainty around the estimated response percentage; the estimate and interval themselves are not reported in the registry record.
As a general statistical point, response rate is useful for describing the frequency of a defined tumor change, but it does not describe how long a response lasts or when progression or death occurs. The listed secondary outcomes include duration of response, time to response, and progression-free survival, which address different aspects of the clinical course. The registry gives the primary assessment timeframe as 6 cycles of 28 days up to 24 weeks. The recorded endpoint description does not provide the numeric primary result or details of its observed assessment completeness.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limitation is that the registry record names the primary endpoint and its method of assessment but does not report the observed ORR, its 95% confidence interval, or a numeric primary result. The record therefore supports an explanation of what was intended to be measured, but not a statement about the response proportion observed. No conclusion about the magnitude or precision of the primary result can be drawn from the endpoint definition alone.
The endpoint was investigator-assessed, while the listed secondary outcomes also include response assessed by a blinded independent review committee. Investigator assessment reflects the designated primary assessment in the record. The existence of an independent-review endpoint is relevant because tumor response requires interpretation of imaging and application of response criteria. However, neither the BIRC result nor a comparison between investigator and BIRC assessments is reported in the registry record. The open-label design also means treatment masking was absent; this is a design feature, not evidence that assessments were biased.
This was a single-arm study, so its ORR would describe the proportion of treated participants meeting the response criteria. It would not estimate a randomized difference from a control group. General statistical interpretation of single-arm outcomes must recognize that response may be influenced by patient selection, prior treatment, disease course, assessment schedules, and other factors. Without a concurrent comparator, those influences cannot be separated from the observed proportion through randomized comparison.
The listed secondary measures represent distinct questions. Duration of response concerns persistence of response among responders; time to response concerns when a response occurs; and progression-free survival concerns time to progression or death under the applicable definition. Disease control rate is another response-related summary. The registry lists these outcomes and identifies investigator or BIRC assessment for several of them, but does not report their numeric findings in this record. They cannot fill the gap in the missing primary result.
Any displayed planning illustration based on an observed effect should be read as a hypothetical design calculation, not as an observed property of this study. The stated assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; they do not describe this single-arm trial’s allocation. Such illustrations are not observed power and do not establish that the study was adequately powered. The registry’s own sample-size justification is not reported in the registry record.
In short, the design and endpoint are identifiable, and the record gives an enrollment figure of 140 and a completed status. But the response estimate, uncertainty interval, and detailed analysis results are not reported in the registry record. Any interpretation should distinguish this absence of a posted numeric finding from evidence of either benefit or lack of benefit.
9. Statistical Methods Explained
Several statistical ideas help clarify what this study’s endpoint would mean and what cannot be inferred from its registry entry. The endpoint is a percentage, not a comparison between randomized groups, and the recorded design is single-arm. The following explanations are general statistical principles; they do not fill in results or analysis details that are not reported in the registry record.
What does a response percentage describe?
A response percentage summarizes the fraction of an analysis population classified as having a qualifying response. Here, the classification is based on best overall confirmed complete response or partial response under RECIST 1.1, as assessed by the investigator. The registry defines the Full Analysis Set as patients who received at least one dose of ceritinib. The percentage is a summary across that population, not a statement that every participant had the same clinical course.
Because the endpoint is binary at the participant level, each participant’s assessment is reduced to whether the response definition was met. That makes ORR comparatively straightforward to describe, but it discards other information. It does not, on its own, indicate how large a tumor change was beyond the response threshold, how long a response persisted, or what happened after the assessment window. The listed duration-of-response, time-to-response, and progression-free-survival outcomes address different questions.
What does the confidence interval add?
A confidence interval is a statistical expression of uncertainty around an estimated quantity. In general, an interval for a response percentage communicates the range of values compatible with the data and the interval procedure, under its assumptions. It is not a guarantee that the true value lies inside that particular interval, nor does an interval alone resolve whether a result is clinically important. The registry specifies a 95% confidence interval for the primary endpoint but does not report its limits or the response estimate. Therefore, the precision of the primary finding cannot be evaluated from this record.
The width of an interval depends on the amount and pattern of information available and on the method used to calculate it. A precise description of the interval method is not reported in the registry record. No alternative interval method should be presumed from the stated confidence level alone.
Why does the single-arm design matter?
In a randomized comparison, allocation helps create groups that can be compared under a design intended to reduce systematic differences. This study has no concurrent control group: the record identifies it as single-arm, with allocation not applicable. Its response percentage would thus be an outcome description for treated participants, not a treatment-versus-control effect estimate.
As a general principle, an outcome observed in a single group can reflect both treatment and other influences. Prior treatment, who entered the study, disease trajectory, follow-up, and outcome assessment can all matter. A one-group percentage cannot distinguish these influences by randomization. This does not make the descriptive result meaningless; it defines what kind of inference the design can support.
How should assessment and confirmation be understood?
The record defines response as best overall confirmed response and names investigator assessment as the primary assessment. Confirmation is part of the endpoint definition, but the detailed visit schedule and operational confirmation rules are not reported in the registry record. General use of a confirmation requirement can reduce reliance on a single assessment, while also making completeness and timing of follow-up relevant to classification.
The listed BIRC-assessed ORR is a separate outcome. A blinded independent review may provide an assessment distinct from the investigator’s, but the registry entry does not report the BIRC result or explain how disagreements between assessments were handled. The study itself was open-label. These points should be kept separate: open-label describes masking in the trial, while BIRC refers to a listed assessment source.
What do planning calculations mean?
Planning calculations translate an assumed effect and design into an illustrative resource requirement under statistical assumptions. The stated planning assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those are planning assumptions, not a description of this single-arm study’s actual allocation or analysis. The registry’s own sample-size rationale and any planned target effect are not reported in the registry record.
Such calculations are not observed power. Observed power is not a substitute for estimating the result and its uncertainty; a planning illustration instead asks what a hypothetical design might require under its assumptions. For time-to-event outcomes, the stated convention is to report a total number of events; other illustrations are expressed as participants per arm. Neither convention changes the fact that the trial record identifies no control arm.
Why can over-dispersion matter?
In general, over-dispersion occurs when variation in data is greater than assumed by a statistical model. If ignored, uncertainty can be understated and inference may be too confident. It is particularly relevant to some count or clustered-data models, but the registry record does not describe an over-dispersion adjustment or even identify such a model for the primary endpoint. It should therefore be treated as a general concept, not a feature established for this analysis.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| LDK378 750mg | 140 | 57 | 40.7% | 32.5% to 49.3% | 32.9% to 49.0% | 32.9% to 49.0% |
R code and output
arm <- c("LDK378 750mg")
n <- c(140)
events <- c(57)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM LDK378 750mg 140 57 40.7 32.5 49.3 32.9 49.0 32.9 49.011. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary ORR endpoint and multiple secondary outcomes, including investigator- and BIRC-assessed measures. When several outcomes or analyses are examined, the chance of at least one apparently unusual result can increase. General statistical practice may address this through a prespecified testing hierarchy, adjustment, or a clear distinction between confirmatory and exploratory findings. The multiplicity strategy for this study is not reported in the registry record, so it is not possible to determine whether or how the listed outcomes were incorporated into a formal testing plan.
The entry identifies 12 outcome measures and zero analysis records. That registry information does not establish which outcomes were analyzed, whether any planned outcomes were omitted, or whether results are available elsewhere. Numeric primary and secondary results are not reported in the registry record. No interim analyses or detailed statistical analysis plan are reported in the registry record.
The Full Analysis Set is defined as patients who received at least one dose of ceritinib. The record does not explain the handling of participants with missing tumor assessments, incomplete follow-up, or other missing endpoint information. It also does not report whether any sensitivity analyses were performed. General methods for missing data depend on why information is missing and what assumptions can reasonably be made; no particular method should be inferred.
The described follow-up plan included continued tumor assessment for participants who stopped medication without progression, and continued assessments for those treated beyond investigator-assessed progression until permanent discontinuation. This provides context for planned observation but does not show how much follow-up was achieved or how assessments were used in the final analysis. The exact analysis population for secondary outcomes and safety summaries is not reported in the registry record.
12. 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 |
|---|---|---|---|
| LDK378 750 mg | 67 | 140 | 47.9% |
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 |
|---|---|
| LDK378 750 mg | 139 of 140 |
Most frequent serious adverse event terms
| Event term | Organ system | LDK378 750 mg |
|---|---|---|
| PYREXIA | General disorders | 8 of 140 |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | 7 of 140 |
| PNEUMONIA | Infections and infestations | 6 of 140 |
| VOMITING | Gastrointestinal disorders | 4 of 140 |
| DEHYDRATION | Metabolism and nutrition disorders | 4 of 140 |
| SEIZURE | Nervous system disorders | 4 of 140 |
| ABDOMINAL PAIN | Gastrointestinal disorders | 3 of 140 |
| NAUSEA | Gastrointestinal disorders | 3 of 140 |
| ASTHENIA | General disorders | 3 of 140 |
| MALAISE | General disorders | 3 of 140 |
Most frequent non-serious adverse event terms
| Event term | Organ system | LDK378 750 mg |
|---|---|---|
| DIARRHOEA | Gastrointestinal disorders | 115 of 140 |
| NAUSEA | Gastrointestinal disorders | 115 of 140 |
| VOMITING | Gastrointestinal disorders | 92 of 140 |
| ALANINE AMINOTRANSFERASE INCREASED | Investigations | 65 of 140 |
| DECREASED APPETITE | Metabolism and nutrition disorders | 59 of 140 |
| ASPARTATE AMINOTRANSFERASE INCREASED | Investigations | 55 of 140 |
| FATIGUE | General disorders | 54 of 140 |
| WEIGHT DECREASED | Investigations | 48 of 140 |
| ABDOMINAL PAIN | Gastrointestinal disorders | 45 of 140 |
| CONSTIPATION | Gastrointestinal disorders | 42 of 140 |
Serious events with the highest arm-level rate: pyrexia (5.7%), dyspnoea (5.0%), pneumonia (4.3%), vomiting (2.9%), dehydration (2.9%).
The adverse-event summary identifies an LDK378 750 mg group with 140 participants at risk for serious adverse events and 67 affected, and 140 at risk for other adverse events with 139 affected. These are separate registry summaries; they should not be added together, treated as mutually exclusive categories, or interpreted as numbers of unique participants. The record does not provide a death count in this summary.
The listed serious adverse event terms and percentages are pyrexia, 5.7%; dyspnoea, 5.0%; pneumonia, 4.3%; vomiting, 2.9%; dehydration, 2.9%; and seizure, 2.9%. These figures describe the listed terms in the registry adverse-event summary. They are not a comparison with another treatment group, and the registry record does not report comparative risk estimates.
As a general statistical point, adverse-event summaries depend on the definitions, observation period, coding, and population used. The record does not provide enough detail to interpret event timing, recurrence, severity beyond the serious-event categorization, or causal attribution. A listed event should not be assumed to have been caused by treatment solely because it occurred during the study. This safety summary also cannot substitute for the missing primary efficacy result or establish an overall benefit-risk conclusion.
13. What the Result Does, and Does Not, Mean
The record describes a study asking whether participants with previously treated ALK-activated non-small cell lung cancer met an investigator-assessed RECIST response definition during the stated assessment timeframe. It identifies LDK378 as the study treatment and defines the primary response population. Those details explain the intended meaning of the endpoint, but the primary response percentage and its 95% confidence interval are not reported in the registry record.
Consequently, the posted registry information cannot establish the observed frequency of response or how precisely that frequency was estimated. The listed secondary outcome names do not provide their results, and their presence does not show that the outcomes favored any particular conclusion. The safety summary records event counts and selected serious-event percentages, but it is not a comparative safety analysis and does not establish causation.
The single-arm design is also important to the scope of inference. Even if a response percentage were reported, it would describe participants treated in this study; without a concurrent randomized comparison, it would not quantify a randomized difference between treatment and control. General considerations such as selection, prior therapy, follow-up, and assessment can affect interpretation of a one-group result.
The record does show a phase II, multicenter, open-label, single-arm design, a stated enrollment of 140, and completed status. It also gives treatment and follow-up descriptions. These establish the study’s broad structure, not the success or failure of the intervention. Missing numerical results should be understood as missing from the registry record, rather than interpreted as a zero response, a negative result, or evidence that no analysis was conducted.
Overall, the registry supports a careful account of the research question, response criteria, and design limitations. It does not support a conclusion about the size, precision, or comparative meaning of the primary outcome. The distinction between a clearly defined endpoint and a reported endpoint result is essential: the definition can be interpreted, but the absent estimate cannot.
14. Why This Trial Matters Statistically
This study is statistically informative as an example of how a well-defined endpoint and a design’s inferential limits must be considered together. The registry specifies an investigator-assessed ORR under RECIST 1.1, the response categories that count, a 95% confidence interval, and a Full Analysis Set definition. These details make the intended summary understandable. Yet the numeric estimate and its interval are not reported in the registry record, so readers cannot assess the observed response proportion or its precision.
The single-arm design foregrounds the difference between describing outcomes and estimating comparative effects. A response proportion can be a useful descriptive measure, but it does not itself provide a randomized contrast. This distinction is broadly important in clinical research: a statistic’s meaning depends not only on its formula but also on how participants were assigned, who entered the analysis, and how outcomes were assessed.
The study also lists both investigator-assessed and BIRC-assessed outcomes. Considering independent assessment is relevant for tumor endpoints that require interpretation, while recognizing that this record does not provide the corresponding results or the relationship between assessments. Multiple listed outcomes raise a further general issue: the prespecified treatment of multiplicity affects how confidently a collection of findings can be interpreted. That strategy is not reported in the registry record.
Finally, the safety figures illustrate why denominators, event categories, and comparison context matter. Counts and percentages can describe recorded events, but they do not alone establish causation or comparative risk. A sound reading keeps endpoint definition, analysis population, uncertainty, design, missing information, and safety reporting distinct rather than allowing one type of evidence to answer a different statistical question.
15. 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 main trial-specific limitation for interpreting the registry entry is the absence of the primary ORR estimate and its 95% confidence interval. Numeric results for the listed secondary outcomes are also not reported in the registry record. Without these results, neither effect magnitude nor statistical precision can be described.
The study was single-arm, open-label, and had no concurrent comparator. This means that its design does not support a randomized estimate of treatment effect relative to another intervention. The primary outcome was investigator-assessed, while the record also lists BIRC-assessed outcomes; the corresponding values and any reconciliation approach are not reported in the registry record.
The record defines the Full Analysis Set but does not report the number included in the primary analysis or how missing or unevaluable assessments were handled. Details of the statistical analysis plan, multiplicity control, interim analyses, and sample-size justification are not reported in the registry record. The planned assessment timeframe and follow-up descriptions do not establish the completeness of observed follow-up.
The adverse-event summary supplies selected counts and serious-event terms, but does not report a death count in that summary or provide a concurrent safety comparison. Detailed event timing, recurrence, attribution, and the analysis population for safety are not reported in the registry record. These limitations prevent a fuller evaluation of uncertainty and context, beyond the descriptive safety information listed.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: LDK378 in Adult Patients With ALK-activated NSCLC Previously Treated With Chemotherapy and Crizotinib — NCT01685060.
- Registry record last updated 2017-06-19; status last verified 2017-05.
- Results first posted 2017-05-08.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
NCT01685060 is a completed phase II, multicenter, open-label, single-arm study of LDK378 in adults with ALK-activated non-small cell lung cancer previously treated with chemotherapy and crizotinib. The registry lists enrollment of 140, a start date of 2012-11-26, and a primary completion date of 2016-03-29.
The primary endpoint is investigator-assessed ORR under RECIST 1.1, defined by confirmed complete or partial response, with a 95% confidence interval and a stated timeframe of 6 cycles of 28 days up to 24 weeks. The response estimate and interval are not reported in the registry record. Secondary outcomes include BIRC-assessed response, response duration, disease control, time to response, and progression-free survival.
The entry defines the Full Analysis Set as patients who received at least one dose of ceritinib. It also records adverse-event summaries for the LDK378 750 mg group. Detailed analysis methods, missing-data handling, and multiplicity strategy are not reported in the registry record.