A Study to Assess Disease Activity of Intravenously (IV) Infused Telisotuzumab Vedotin in Adult Participants With Advanced/Metastatic Non-Squamous Non-Small Cell Lung Cancer (NSCLC): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study to Assess Disease Activity of Intravenously (IV) Infused Telisotuzumab Vedotin in Adult Participants With Advanced/Metastatic Non-Squamous Non-Small Cell Lung Cancer (NSCLC) (NCT05513703), a na phase 2 study. The primary outcome is Objective Response Rate (ORR) as Assessed by an Independent Central Review (ICR).

2. Clinical Question

This question focuses on tumor response during treatment rather than on a comparison with another treatment or a control group. An observed response percentage, if available, would describe the proportion meeting the endpoint definition in the efficacy evaluable analysis set. It would not by itself establish what would have happened to similar participants under another treatment, because the study did not include a comparator arm.

The registry describes a population with previously untreated MET amplified locally advanced or metastatic non-squamous NSCLC. That description sets the clinical context for interpreting any response estimate: the estimate would pertain to that eligibility group and study setting, not automatically to other forms or stages of lung cancer, or to people with different treatment histories.

The registry record lists enrollment as 9, while its brief summary describes an intended enrollment of approximately 70 participants. Those are different record details: the former is the listed enrollment and the latter is a plan described in the summary. The reason for the difference is not reported in the registry record. The study is labeled terminated, but the reason for termination is also not reported.

3. Trial Design

This was a Phase 2, open-label study with a single-group model. Masking was listed as none, allocation as NA, and there was no control group. Participants received telisotuzumab vedotin by intravenous infusion every 2 weeks until meeting study drug discontinuation criteria. The registry describes the treatment as investigational.

The population was adults with previously untreated MET amplified locally advanced or metastatic non-squamous NSCLC. The primary efficacy analysis set was defined as all participants who received at least 1 dose of study drug. The registry lists enrollment as 9. Its brief summary separately describes an intended enrollment of approximately 70 adults and approximately 110 sites worldwide. The record does not explain the difference between the stated plan and listed enrollment, or how many sites participated.

The study start date is 2022-11-21 and the primary completion date is 2024-10-28. Its status is terminated. The reason for termination and the timing of that decision are not reported in the registry record. The study's primary outcome was objective response rate assessed by independent central review, with a timeframe of up to approximately 1.5 years. Secondary outcomes include duration of response, disease control rate, progression-free survival per independent central review, overall survival, and changes in specified symptom and quality-of-life measures.

Because there was no randomized or concurrent comparison group, the design can describe outcomes observed among participants receiving the study drug but cannot directly estimate a treatment contrast. Open-label status means masking was not used. Independent central review offers an assessment of the primary response endpoint by reviewers independent of the treating site, but the registry does not describe the review process in further detail. The allocation and masking fields do not support an inference about a planned comparison; the record explicitly describes a single group.

The registry record does not report a protocol-level rationale for the final enrollment, a sample-size justification, or whether recruitment stopped early. It also does not report the reasons for termination, whether participants completed follow-up, or how the planned site count related to actual study conduct. These gaps matter when interpreting the limited results information: listed enrollment alone does not establish the size of the primary efficacy analysis population.

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 9 participants were enrolled and received at least 1 dose of study drug.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The efficacy evaluable analysis set included all participants who received at least 1 dose of study drug.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: The efficacy evaluable analysis set included all participants who received at least 1 dose of study drug.

6. Primary Endpoint

The registry lists the primary outcome as Objective Response Rate (ORR) as Assessed by an Independent Central Review (ICR), measured over Up to approximately 1.5 years.

The primary endpoint is objective response rate, defined as the percentage of participants with a confirmed complete response or confirmed partial response. Response was to be assessed using Response Evaluation Criteria in Solid Tumors, version 1.1, by independent central review. The stated timeframe is up to approximately 1.5 years. The efficacy evaluable analysis set includes participants who received at least 1 dose of study drug.

Response rate is a binary endpoint at the participant level: a participant either meets the defined response criterion or does not. The group percentage describes how common a qualifying response was in the analysis population. Requiring confirmation means that a response must meet the confirmation rule in the endpoint definition; the registry does not give further details about assessment timing or confirmation procedures.

Response rate is used in trials to characterize tumor shrinkage reaching specified criteria. It provides a direct measure of the frequency of confirmed responses, while not capturing every aspect of disease course, duration, symptoms, or quality of life. The listed secondary outcomes address some of those other dimensions, including duration of response, disease control, survival, cough, chest pain, dyspnea, and quality of life.

The registry record states the endpoint definition but does not report an observed response percentage, the number of participants with confirmed responses, or the 95% confidence interval. Thus, it is not possible from this record to quantify the observed response rate or its precision.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry specifies a binary primary outcome expressed as a percentage of participants. In general, a binary endpoint classifies each participant according to whether a defined event occurred. For this trial, the event was a confirmed complete response or confirmed partial response under RECIST version 1.1, as determined by independent central review. A response percentage is interpretable only together with its analysis population and the number meeting the endpoint. The registry defines the efficacy evaluable analysis set as participants receiving at least 1 dose, but does not report its size or the number with a response.

A confidence interval communicates the uncertainty around an estimated quantity. For a response percentage, it gives a range of values compatible with the observed data under the chosen statistical method and its assumptions. The primary endpoint specifies a 95% confidence interval, but the interval itself and the method used to calculate it are not reported in the registry record. Without the estimate, denominator, and interval, readers cannot assess either the observed response frequency or its statistical precision. A confidence interval is not a guarantee that the true value lies within its limits, nor does it replace consideration of the study design and analysis population.

Small analysis populations often yield imprecise estimates because each participant represents a substantial part of the observed data. This is a general statistical consideration, not an estimate of precision for this study: the record does not state the primary analysis denominator. The listed enrollment is 9, but participants may not all have received a dose or met other analysis requirements; the registry's definition makes receipt of at least 1 dose the inclusion condition for the efficacy evaluable set. The actual set size is not reported, so the primary endpoint's precision cannot be inferred from enrollment alone.

The single-group design provides no randomized treatment contrast. In a comparative study, an effect estimate might compare outcomes between assigned groups. Here, no second group is listed, so the primary endpoint would be a within-group proportion. The record's allocation field is NA and the model is single group. Any interpretation of a response percentage would therefore be descriptive; it could not answer how response compares with another treatment or with an untreated counterfactual group.

Independent central review is part of the endpoint definition, while the study itself was open-label and unmasked. In general, central review can apply a consistent assessment process separate from local treating teams. The registry does not describe reviewer blinding, adjudication, image assessment procedures, or the process for missing or unevaluable scans. Those details should not be assumed. The response criteria named in the record are RECIST version 1.1.

What can be inferred from the endpoint definition?

The definition identifies the event that would count as a response and the population intended for the efficacy assessment. It does not give the response result. The outcome timeframe, up to approximately 1.5 years, describes the stated observation period for the primary endpoint; it does not report how long any individual participant was followed or whether all assessments were completed. The record does not specify the assessment schedule or the handling of treatment discontinuation before response confirmation.

What statistical plan is reported?

The registry record does not report a statistical analysis plan, a hypothesis test for the primary endpoint, a null response threshold, or a method for constructing the stated 95% confidence interval. It lists no analysis entries. Accordingly, no p-value, adjusted estimate, or sponsor analysis can be described. The absence of these details in the registry record does not show that no analysis was conducted; it means the methods and results are not reported in the record used for this account.

The record includes planning assumptions for a sample-size illustration: a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and a target effect stated as a fraction of an observed effect. The illustration is explicitly described as planning rather than the trial's own design. It is not an observed-power analysis and does not change the fact that the registered study had a single-group model. No sample-size illustration values are reported in the registry record, and the listed assumptions should not be mistaken for a protocol-specific analysis plan.

General statistical practice distinguishes planning calculations from analysis of collected outcomes. Planning calculations use assumptions to explore how a hypothetical study might be sized. Observed power is not a substitute for the outcome estimate and its uncertainty. In this study, the primary response result and its interval are not reported, so the registry record does not permit a numerical assessment of endpoint magnitude, precision, or statistical evidence.

The secondary endpoints span different data types. Duration of response and survival outcomes are time-to-event measures, whereas disease control rate and symptom or quality-of-life changes have their own outcome definitions and measurement scales. Each requires appropriate analysis choices, such as a definition of the event, censoring rules for time-to-event outcomes, and a strategy for incomplete questionnaire data. The registry lists the outcomes but does not report these analytic details. No methods for handling over-dispersion, missing observations, or repeated measurements are reported; these methods should not be inferred simply from the outcome names.

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.

R code and output

arm    <- c("Telisotuzumab Vedotin")
n      <- c(9)
events <- c(3)
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  Telisotuzumab Vedotin  9  3  33.3  7.5  70.1  12.1  64.6  12.2  65.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists multiple secondary outcomes alongside the primary objective response rate. They include duration of response, disease control rate, progression-free survival per independent central review, overall survival, and changes in cough, chest pain, dyspnea, and quality of life. Testing or interpreting multiple outcomes raises a multiplicity question: when many outcomes are examined, the overall chance of a chance-positive finding can differ from that for a single prespecified test. The registry record does not report a multiplicity adjustment, a hierarchy among outcomes, or a strategy for distinguishing confirmatory from exploratory analyses.

The primary endpoint definition includes an efficacy evaluable analysis set consisting of participants who received at least 1 dose of study drug. The record does not report the size of this set, the number analyzed for the primary endpoint, or the count of confirmed responses. It does not describe how missing tumor assessments, early discontinuation, or unevaluable assessments were handled. Missing-data handling is particularly relevant when participants stop treatment or do not complete follow-up, because the analysis rule can affect which observations contribute to an endpoint. No such rule is reported.

The registry record also does not state the analysis populations used for the secondary or safety outcomes, or whether those populations differed from the efficacy evaluable set. The listed enrollment of 9 is not a substitute for endpoint-specific denominators. Nor does the record state whether analyses were interim or final, or how follow-up data were managed after treatment discontinuation.

No statistical analysis plan or analysis entries are reported. This leaves the handling of multiplicity, missing data, censoring, and endpoint-specific populations unclear. These omissions limit the ability to evaluate the planned analysis and to reproduce or verify any results. The absence of these details in the registry record should be read as unreported information, not proof that the study team did not use an analysis procedure.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: disease progression (22.2%), diarrhoea (11.1%), intestinal obstruction (11.1%), pneumonia (11.1%), dyspnoea (11.1%).

The registry lists one telisotuzumab vedotin group, with 9 participants at risk for the adverse-event summaries. It records 3 participants with serious adverse events, 9 with other adverse events, and 4 deaths. These are separate reported summaries; the record does not explain whether the participant groups overlap, so the counts should not be added or treated as mutually exclusive.

The serious adverse-event terms with reported percentages are disease progression, 22.2%; diarrhoea, 11.1%; intestinal obstruction, 11.1%; pneumonia, 11.1%; dyspnoea, 11.1%; and pneumonitis, 11.1%. These terms describe events listed in the registry. The record does not provide further event-level details, timing, duration, severity grades, or an attribution of these events to study drug. The percentages should not be interpreted as treatment-caused risks.

Serious adverse events, other adverse events, and deaths are different categories of safety information. A serious event is not necessarily an event judged related to treatment, and a death count does not by itself indicate cause. The registry record does not state whether deaths were attributed to cancer, treatment, another cause, or a combination. It also does not provide a comparator group, so these observations cannot estimate how safety outcomes differed from another treatment or background risk.

With the limited information reported, the figures describe the listed group and the registry's event categories. They do not establish causation, comparative safety, or the full clinical context of each event.

13. What the Result Does, and Does Not, Mean

The study asks a focused descriptive question: what percentage of the efficacy evaluable participants had a confirmed complete or partial response by independent central review? The registry record defines that endpoint and identifies the population used for it, but it does not report the observed response percentage, response count, denominator, or 95% confidence interval. The available record therefore does not quantify the primary result or its precision.

The single-group design is important to what any result could mean. A response estimate could describe the experience of participants treated in this study, but the study did not compare telisotuzumab vedotin with another intervention. It cannot, on its own, establish a relative treatment effect. The open-label status and absence of masking are also part of the design context; independent central review was specified for the primary response assessment, but details of that review process are not reported.

The study is labeled terminated, and its listed enrollment is 9. Its brief summary describes an intended enrollment of approximately 70. The reason for the gap and the reason for termination are not reported in the registry record. Neither the status nor the enrollment information explains whether the study ended for efficacy, safety, recruitment, operational, or other reasons. Such explanations should not be inferred.

The record's safety summaries describe reported events in the treatment group, including serious adverse events and deaths. They do not show whether events were caused by treatment, and there is no comparator group for assessing relative risk. The secondary outcome list indicates intended attention to survival, disease control, symptoms, and quality of life, but the listed outcomes are not numerical results.

Thus, the registry record supports an account of the study question, design, outcome definitions, and selected safety counts. It does not provide enough efficacy information to determine the observed response rate, and it does not support a comparative treatment conclusion. These limits are about the information reported in the registry record, not a claim about results that may or may not be available elsewhere.

14. Why This Trial Matters Statistically

This trial is statistically notable because it combines a clearly defined response endpoint with a very limited reported result record. The endpoint specifies confirmed complete or partial response by RECIST version 1.1 and independent central review. Those details make clear what would count as an event. However, without the response count, efficacy analysis denominator, estimate, and confidence interval, readers cannot evaluate how often the endpoint occurred or how uncertain its estimate would be.

The single-group design also makes the distinction between description and comparison central. A one-group response percentage can characterize the observed study population, but it cannot provide a randomized comparison with another treatment. This matters when interpreting response outcomes in a disease setting where the registry lists no control arm. The design determines the claims that the data could support, even if a numerical estimate were available.

The difference between listed enrollment of 9 and intended enrollment of approximately 70 in the brief summary highlights why registry status and analysis population should be read separately. Enrollment is not necessarily the denominator for every endpoint, and the primary efficacy set specifically depends on receiving at least 1 dose. The record does not state the actual size of that set. Termination is recorded, but the reason and its relationship to recruitment or outcomes are unreported.

Finally, the registry lists outcomes extending beyond response, including survival and patient-reported symptoms and quality of life. Such breadth can support a broader view of outcomes when results and methods are reported. Here, those endpoint listings do not come with analysis details or numerical results in the record. Statistically, the key lesson is to distinguish planned measures and definitions from analyzed results, and descriptive safety counts from causal or comparative evidence.

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 central limitation is that the registry record does not report the primary outcome result. Although it defines objective response rate and specifies a 95% confidence interval, it gives no response percentage, response count, analysis denominator, or interval. This prevents assessment of effect magnitude and precision. Listed enrollment of 9 cannot substitute for the efficacy evaluable set, which includes participants who received at least 1 dose and whose size is not separately reported.

The design was single group, open label, and unmasked, with no comparator. A response proportion, if reported, would describe observed outcomes in the treated group rather than a treatment contrast. The record does not report a statistical analysis plan, a primary hypothesis test, confidence-interval method, multiplicity strategy, or missing-data rules. It also does not report the handling of censoring for survival outcomes or the analysis populations for secondary outcomes.

The registry status is terminated, and the listed enrollment is 9, whereas the brief summary describes an intended enrollment of approximately 70. The reason for termination, the reason for the difference, actual recruitment history, and any effect on planned analyses are not reported. The record also does not explain whether follow-up was complete or how many participants contributed data to each endpoint.

For safety, the record lists group counts, deaths, and selected serious-event terms, but it does not provide event-level context, attribution, timing, or severity detail. No comparison group is available to place these counts against another treatment experience. For all outcomes, registry listings establish what was named or defined, not that results were collected, analyzed, or reported.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study to Assess Disease Activity of Intravenously (IV) Infused Telisotuzumab Vedotin in Adult Participants With Advanced/Metastatic Non-Squamous Non-Small Cell Lung Cancer (NSCLC) — NCT05513703.
• Registry record last updated 2025-10-24; status last verified 2025-10.
• Results first posted 2025-10-24.
• 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.

19. Record Summary

NCT05513703 is a Phase 2, open-label, single-group study of intravenous telisotuzumab vedotin in adults with previously untreated MET amplified locally advanced or metastatic non-squamous NSCLC. The record lists enrollment as 9, status as terminated, a study start date of 2022-11-21, and a primary completion date of 2024-10-28. Its brief summary describes an intended enrollment of approximately 70 participants and dosing every 2 weeks until study drug discontinuation criteria were met.

The primary endpoint is objective response rate by independent central review using RECIST version 1.1, expressed as the percentage with confirmed complete or partial response. The efficacy evaluable set includes participants who received at least 1 dose. The record does not report the primary endpoint estimate or its 95% confidence interval, and the reason for termination is not reported. Safety summaries list 3 participants with serious adverse events, 9 with other adverse events, and 4 deaths; these figures do not establish causation.