Study of GT103 in Combination With Pembrolizumab in Refractory, Metastatic Non-Small Cell Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of GT103 in Combination With Pembrolizumab in Refractory, Metastatic Non-Small Cell Lung Cancer (NCT05617313), a na phase 2 study. The primary outcome is Objective Response Rate (ORR).

2. Clinical Question

Statistically, the central question is whether responses were observed among participants receiving the combination and how precisely that response proportion can be estimated. A response proportion can describe activity in the enrolled group, but without a concurrent comparator it cannot by itself establish how the response experience differs from another treatment or from what would have occurred without the combination.

The study also lists pharmacokinetic outcomes for GT103 with pembrolizumab, including half-life, time to maximum concentration, peak plasma concentration, and area under the concentration-time curve. Progression-free survival and overall survival are also named as secondary outcomes. The registry record does not report results for these outcomes. Consequently, the clinical question can be described from the design and endpoints, but the posted record does not supply an efficacy estimate for answering it.

3. Trial Design

This is a Phase II, open-label, non-randomized, single-group study of GT103 combined with pembrolizumab in refractory, metastatic NSCLC. The recorded allocation is NA, and masking is listed as none. These features mean the study is not structured as a randomized comparison between the combination and a control arm. The registry identifies 21 participants as the enrollment.

The brief study description says the design includes a safety lead-in of 10-20 patients. It then describes treating a total of 50 patients with the combination at the safest GT103 dose determined in that lead-in. It further says that if 10 additional patients are enrolled to dose level -1, the maximum of 60 subjects may be accrued. The observed enrollment listed in the registry is 21. The record does not explain how the trial’s termination relates to the planned accrual described in the summary.

The experimental group description states that pembrolizumab is given intravenously on Day 1 of a 21 day cycle. GT103 is also described as intravenous on Day 1, with the dose determined by the safety lead-in. The regimen details in the arm description are incomplete, so further dosing instructions are not reported in the registry record. The adverse-event group is labeled pembrolizumab 200 mg plus GT103 10mg/kg.

The registry lists the study start as 2023-02-17, primary completion as 2025-06-26, and status as TERMINATED. A reason for termination is not reported in the registry record. Likewise, the timing and criteria for stopping the study are not reported. The design description establishes the intended single-group framework and the planned safety-led dose selection, but it does not provide the full protocol or explain whether all planned stages were completed.

Because allocation is not randomized and there is no listed concurrent comparator, any observed outcomes would describe this treated group. They would not provide a randomized estimate of the effect of adding GT103 to pembrolizumab. The small recorded enrollment and the absence of posted endpoint results also limit what can be inferred from this registry record alone.

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.

Overall Study

Reasons for not completing this period:

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.

6. Primary Endpoint

The registry lists the primary outcome as Objective Response Rate (ORR), measured over From C1D1 until death or up to a maximum of 23 months.

The primary endpoint is objective response rate (ORR), expressed as the proportion of treated participants who have a radiographic partial or complete response according to RECIST v1.1. The registry specifies that the response assessment period begins at C1D1 and continues until death or up to a maximum of 23 months. Its description defines complete response as disappearance of all target lesions, with pathological lymph nodes, whether target or non-target, reduced in short axis to <10 mm. Partial response is defined as at least a 30% decrease in the sum of target-lesion diameters, using the baseline sum as the reference.

ORR is used in clinical studies to summarize whether measurable tumors meet prespecified radiographic response criteria during a defined assessment period. It reduces a set of lesion measurements to a participant-level response classification and then a proportion. That can make the endpoint interpretable as a frequency of response in the treated group. It does not, on its own, describe how long a response lasts, whether disease progression is delayed, or whether survival differs between treatment strategies.

The registry lists the endpoint unit as participants and its measure type as a count of participants. However, it does not report the number classified as having a complete or partial response, nor an ORR estimate or uncertainty interval. The endpoint definition is therefore available, but the result needed to evaluate the observed response proportion is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

General: A binary endpoint records whether each participant does or does not meet a defined condition. For ORR, that condition is having a complete or partial radiographic response under RECIST v1.1. The participant-level classifications can be expressed as a response count and a proportion of participants in the analysis population. The registry identifies the endpoint as a participant count, but it does not post the count of responders or the corresponding proportion.

A proportion is most useful when its denominator is clear. The enrollment recorded is 21, but enrollment alone does not prove that every participant was assessed for response or included in an endpoint analysis. Some participants may lack a post-baseline assessment or have follow-up that does not cover the intended assessment period; whether that occurred in this study is not reported in the registry record. The analysis population, evaluability rules, and missing-assessment handling are also not reported. Without those details, a reader cannot determine which participants would contribute to the ORR denominator.

General: A confidence interval describes uncertainty around an estimate under a stated statistical model. For a response proportion, it would communicate the range of values reasonably compatible with the observed count and sample, rather than only presenting a point estimate. The registry record does not report an ORR confidence interval or the method that would be used to calculate one. With no posted response count, there is no estimate whose precision can be interpreted.

The record lists no statistical analyses. The analysis plan, including any prespecified estimation method, hypothesis test, covariate adjustment, or decision threshold, is not reported in the registry record. No particular inferential method should therefore be attributed to this trial based on the available description. General statistical interpretation can explain what methods would mean, but it cannot substitute for a trial-specific analysis that is not posted.

How should the single-group design be interpreted?

General: A single-group study can estimate outcomes within the participants who received the study regimen. Without a concurrent comparison group, it does not separate treatment-associated change from other influences such as the course of disease, participant selection, assessment practices, or care received alongside the study regimen. Random allocation is commonly used to balance measured and unmeasured factors across groups; this record lists no randomization and no control group. The registry therefore does not support a randomized between-group effect estimate.

The open-label designation means masking is listed as none. In general, knowledge of the treatment can influence aspects of care, reporting, or assessment. Radiographic response criteria provide standardized definitions, but the record does not describe blinded image review or other procedures intended to reduce assessment bias. Whether such procedures were used is not reported in the registry record.

What do the planning illustrations represent?

General: A sample-size calculation based on an observed effect is a planning illustration, not a result of the trial and not observed power. It explores the sample size that might be required under stated assumptions if a future study aimed to detect an effect of a chosen size. The registry record describes planning assumptions that include a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects expressed as fractions of an observed effect. It specifies that time-to-event illustrations count total events, while other illustrations count participants per arm.

These assumptions do not describe the actual design of this study, which is listed as single-group rather than a two-arm randomized comparison. The registry does not provide an observed ORR effect to use as a basis for such a calculation. Accordingly, planning illustrations cannot establish the power of this completed or terminated study, and they should not be read as observed results. Sample-size planning and inferential analysis answer different questions: the former considers requirements under assumptions, while the latter estimates outcomes from collected participant data.

How do over-dispersion and follow-up matter in general?

General: Over-dispersion occurs when outcome variability exceeds what a statistical model assumes. In count or rate analyses, ignoring over-dispersion can make uncertainty appear smaller than it is. Whether any such model or adjustment was relevant to the trial’s analyses is not reported in the registry record, and no analysis results are posted. For an endpoint such as ORR, which is defined through participant-level response status, the most immediate requirements are a clear response classification, a defined analysis population, and a documented denominator.

Time-to-event outcomes such as progression-free survival and overall survival depend on both event information and follow-up. General methods for these outcomes account for the fact that some participants may not have experienced the event by the end of observation. The registry names PFS and OS among its secondary outcomes, but does not post their definitions, event counts, estimates, follow-up summaries, or analysis methods. No conclusion about those outcomes can be drawn from their listing alone.

10. Multiplicity, Missing Data and Analysis Populations

The registry names ORR as the primary endpoint and lists pharmacokinetic outcomes, progression-free survival, and overall survival as secondary outcomes. It records eight outcome measures in total. The full set of outcome details, testing sequence, and any plan to control the chance of false-positive findings across multiple endpoints are not reported in the registry record. No analysis is listed. It is therefore not possible to determine whether multiplicity was addressed through a hierarchy, adjustment, or another prespecified strategy.

General: When several outcomes are tested, the chance of at least one apparently notable result can increase if each is assessed independently. A prespecified primary endpoint and a clear testing plan help readers distinguish confirmatory questions from exploratory ones. Here, the primary endpoint is identified, but no result or inferential testing plan is posted. The absence of a reported multiplicity method should not be interpreted as evidence that a particular method was or was not used outside the registry information.

Missing-data methods are also not reported. The record does not state how participants with missing imaging, incomplete follow-up, or unevaluable response assessments were handled, nor does it describe an analysis population such as all treated participants or an evaluable subset. The recorded enrollment of 21 is not enough to establish the denominator for ORR or the number contributing to each secondary outcome. The degree and pattern of missing data are not reported in the registry record.

The registry does not describe an interim analysis, stopping boundary, or statistical monitoring procedure. These matters cannot be inferred from the planned safety lead-in or the TERMINATED status. The reason for termination and any associated decision process are not reported in the registry record. As a result, uncertainty remains about how endpoint selection, missing information, and multiple outcomes were handled in the study’s final analyses.

11. 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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: respiratory, thoracic and mediastinal disorders - other, specify (9.5%), bronchial obstruction (4.8%), fall (4.8%), fracture (4.8%), hypoxia (4.8%).

The adverse-event record lists one group, labeled pembrolizumab 200 mg plus GT103 10mg/kg, with 21 participants at risk for serious adverse events and 6 affected. It also lists 21 at risk for other adverse events and 21 affected. The death count is 15. These are participant counts as represented in the registry record; they should not be interpreted as evidence that treatment caused the events or deaths. The record does not report the attribution criteria or the timing of these outcomes.

The listed serious-event terms include respiratory, thoracic and mediastinal disorders - other, specify, at 9.5%; bronchial obstruction at 4.8%; fall at 4.8%; fracture at 4.8%; hypoxia at 4.8%; and lung infection at 4.8%. The registry does not explain whether these percentages use participants, events, or another denominator, nor does it provide details about overlap among terms. The percentages should be read as posted rather than converted into counts.

Serious adverse events and other adverse events are separate categories in the record, but their definitions and collection period are not reported. The listed deaths do not establish that death was an adverse event or that it was related to the study regimen. The counts describe what is recorded for the group; they do not establish comparative risk because no control group is listed.

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

The record establishes that a Phase II, open-label, non-randomized, single-group study evaluated GT103 with pembrolizumab in refractory, metastatic NSCLC. It identifies ORR as the primary endpoint and gives the response definitions and assessment window. It also records enrollment of 21, a TERMINATED status, and adverse-event counts for the listed treatment group. These registry entries describe the registered study and its reported information, not a complete account of participant outcomes.

The record does not report the ORR result. It therefore does not show how many participants had a complete or partial response, what response proportion was observed, or how uncertain such an estimate would be. It also does not report results for the named pharmacokinetic, progression-free survival, or overall survival outcomes. The listing of an endpoint is not evidence of a particular result, and the study’s status does not fill that gap.

Even with an observed response proportion, this single-group design would answer a descriptive question about outcomes in the treated group rather than provide a randomized comparison. The record does not identify a separate comparator, and treatment allocation is listed as NA. It consequently cannot estimate a between-group treatment effect from a randomized contrast. Differences against results from other studies would not resolve that limitation because the registry record does not establish that those populations, assessment methods, or follow-up conditions are comparable.

The adverse-event counts provide a descriptive safety picture as recorded, but not causal attribution. The listed 15 deaths cannot be assumed to be treatment-related, and the serious-event term percentages do not establish comparative risk. The record does not describe event timing, causes, or the percentage denominator.

In short, the available information supports a careful account of study design, endpoint definitions, enrollment, status, and selected safety counts. It does not support a conclusion about the efficacy of the combination, the precision of its response estimate, or its comparative safety. The reason for termination, statistical analysis plan, endpoint analysis populations, and missing-data procedures are not reported in the registry record.

13. Why This Trial Matters Statistically

This trial raises a useful statistical distinction between defining an outcome and reporting an estimate. ORR has a stated clinical definition, response criteria, and assessment period. Those details are essential for interpreting an endpoint, but they do not reveal the observed response count or proportion. Since the registry record does not report the ORR result, readers cannot assess its magnitude or precision.

The single-group design also illustrates how study structure shapes the question a result can answer. A response proportion in one treated group can describe that group, but without a concurrent comparator it cannot provide a randomized contrast. This distinction matters when considering whether observed outcomes can be attributed to the regimen rather than to selection, disease course, other care, or measurement conditions.

The registry combines a planned safety lead-in with a stated subsequent enrollment plan and records an actual enrollment of 21. That makes transparent reporting of dose selection, stages completed, analysis populations, and reasons for stopping especially relevant to interpretation. Those details are not reported in the registry record. The TERMINATED status alone does not identify why the study ended or what decisions were made.

The record also illustrates why safety counts need context. Serious-event counts, other-event counts, and deaths are distinct summaries, and none alone establishes causation. Clear definitions, exposure and follow-up information, attribution, and denominators are important to understand how event figures should be interpreted. Several such details are not reported.

Finally, named secondary outcomes and an identified primary endpoint do not substitute for a statistical analysis plan or posted results. Multiplicity handling, missing-data procedures, and methods for time-to-event outcomes are not reported. The statistical importance of this record is therefore less about estimating an effect from the posted data and more about recognizing which design and reporting details are necessary before an effect can be evaluated.

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 most important limitation for interpretation is that the registry record does not report the primary ORR result. It gives neither the number of participants with a complete or partial response nor an estimate of the response proportion. No confidence interval or statistical analysis is posted. Enrollment of 21 does not reveal the endpoint analysis denominator or response-evaluable count.

The study is listed as single-group, open-label, and non-randomized, with no separate comparator. This limits the study’s capacity to distinguish outcomes associated with the combination from outcomes that might occur under another treatment or in the absence of the combination. The record does not describe blinded response review or other assessment safeguards.

The registry lists a planned safety lead-in and subsequent accrual targets, but records enrollment of 21 and status TERMINATED. How much of the intended design was completed, whether dose selection proceeded as planned, and why the study was terminated are not reported in the registry record. The brief arm description is also incomplete, and the full treatment schedule is not available in the record.

Endpoint implementation is difficult to assess because the analysis populations, imaging schedule, missing-assessment rules, and follow-up completeness are not reported. The assessment window is defined, but actual participant follow-up is not. Methods for handling multiple outcomes and time-to-event endpoints are also not reported.

Safety interpretation is limited by the absence of event definitions, timing, attribution, and a stated denominator for the serious-event term percentages. The 15 deaths cannot be interpreted as treatment-related from the posted information. Together, these gaps mean the registry record permits a design-level description and a limited account of recorded safety counts, but not a full efficacy or comparative safety assessment.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Study of GT103 in Combination With Pembrolizumab in Refractory, Metastatic Non-Small Cell Lung Cancer — NCT05617313.
• Registry record last updated 2026-10-14; status last verified 2026-10.
• Results first posted 2026-10-14.
• 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.

18. Record Summary

The registry describes NCT05617313, a Phase II, open-label, non-randomized, single-group study of GT103 with pembrolizumab in refractory, metastatic NSCLC. It lists enrollment of 21, status TERMINATED, a study start of 2023-02-17, and primary completion of 2025-06-26. The reason for termination is not reported in the registry record.

ORR is the primary endpoint, defined using RECIST v1.1 from C1D1 until death or up to a maximum of 23 months. The registry does not report the response result or statistical analyses. Secondary outcomes named include pharmacokinetic measures, PFS, and OS, without posted results.

For the listed treatment group, the record reports 6 participants affected by serious adverse events, 21 affected by other adverse events, and 15 deaths. The record does not establish causality or provide all details needed to interpret these counts. The design and outcome definitions are available, but efficacy estimates, analysis methods, and key details about missing data and endpoint populations are not reported in the registry record.