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 Phase 1/2 Trial of Selinexor (KPT-330) With Docetaxel for Non-small Cell Lung Cancer (NSCLC) (NCT03095612), a na phase 1, phase 2 study. The primary outcome is Number of Participants With Dose Limiting Toxicities (DLT).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03095612 |
| Phase | Phase 1, Phase 2 |
| Condition | Non-small Cell Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 41 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Texas Southwestern Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned people with previously treated, advanced KRAS mutant non-small cell lung cancer. The registry record does not report detailed eligibility criteria or describe the characteristics of the participants who received treatment.
Intervention
The study evaluated selinexor, an investigational drug, as monotherapy and in combination with docetaxel. The record describes combination dose-escalation and dose-expansion cohorts, including selinexor with docetaxel 75mg/m2. It also describes planned selinexor monotherapy dosing cohorts, but participants did not receive selinexor monotherapy before the study was halted.
Comparator
There was no randomized control group. The registry lists a single-group model and allocation as not applicable. Dose cohorts are not a like-for-like control comparison: they may reflect different dose levels or study stages, and the record does not report random assignment between them.
Primary question
The primary outcome was the number of participants with dose-limiting toxicities (DLTs), collected to determine the maximum-tolerated dose. The study's stated safety question was whether selinexor could be evaluated with docetaxel in people previously treated for advanced KRAS mutant lung cancer. The registry record does not report analyzed DLT results, so it does not establish an outcome-based answer to that question.
3. Trial Design
This was an investigator-sponsored Phase 1/2 trial of selinexor, alone and with docetaxel, for previously treated, advanced KRAS mutant non-small cell lung cancer. The registry identifies a single-group model, no masking, and allocation as not applicable. It reports enrollment of 41 and a terminated status. The study began on 2018-03-22, with primary completion dated 2022-11-08.
The record describes dose escalation and dose expansion for the combination, with listed cohorts of selinexor 60mg plus docetaxel 75mg/m2 and selinexor 80mg plus docetaxel 75mg/m2. A selinexor 100mg plus docetaxel cohort is also listed, as are selinexor monotherapy cohorts. The adverse-event entries show no participants at risk in the 100mg combination cohort or the monotherapy cohorts. The primary outcome description states that the study was halted before participants received selinexor monotherapy and that no data were collected or analyzed for that cohort.
The monotherapy dosing description includes weekly and biweekly schedules, while the combination description says selinexor would start one week before chemotherapy. The registry record does not report the full treatment schedule for every combination cohort, nor does it explain the reason for termination. Because the study was not randomized and has no control arm, comparisons between its dose groups cannot be interpreted as randomized treatment effects. The cohorts describe safety observations at particular treatment levels and study stages, not a controlled comparison of selinexor with another treatment.
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: 41 subjects consented;1 screen failed prior to receiving treatment. Participants started at dosing selinexor 60 mg + docetaxel 75 mg/m2. The second dose level was 80 mg +docetaxel 75 mg/m2 to which 4 subjects were assigned. Then 33 patients enrolled and started on Dose expansion: selinexor 60 mg weekly plus docetaxel 75 mg/m2 3 weekly. No data was collected or analyzed for the selinexor Monotherapy cohort, study was halted due to funding.
Only the first 6 of 7 groups are shown.
Dose Escalation:Selinexor 60mg+Docetaxel
| Stage | Dose Escalation Phase: Selinexor 60 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 80 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 100 mg and in Combination With Docetaxel | Dose Expansion Phase: Selinexor 60 mg and in Combination With Docetaxel | Selinexor 40 mg Monotherapy | Selinexor 60 mg Monotherapy |
|---|---|---|---|---|---|---|
| STARTED | 3 | 0 | 0 | 0 | 0 | 0 |
| COMPLETED | 3 | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
Dose Escalation:Selinexor 80mg+Docetaxel
| Stage | Dose Escalation Phase: Selinexor 60 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 80 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 100 mg and in Combination With Docetaxel | Dose Expansion Phase: Selinexor 60 mg and in Combination With Docetaxel | Selinexor 40 mg Monotherapy | Selinexor 60 mg Monotherapy |
|---|---|---|---|---|---|---|
| STARTED | 0 | 4 | 0 | 0 | 0 | 0 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 0 | 4 | 0 | 0 | 0 | 0 |
Reasons for not completing this period:
| Reason | Dose Escalation Phase: Selinexor 60 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 80 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 100 mg and in Combination With Docetaxel | Dose Expansion Phase: Selinexor 60 mg and in Combination With Docetaxel | Selinexor 40 mg Monotherapy | Selinexor 60 mg Monotherapy |
|---|---|---|---|---|---|---|
| Dose not tolerated by the subject | 0 | 4 | 0 | 0 | 0 | 0 |
Dose Escalation:Selinexor100mg+Docetaxel
| Stage | Dose Escalation Phase: Selinexor 60 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 80 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 100 mg and in Combination With Docetaxel | Dose Expansion Phase: Selinexor 60 mg and in Combination With Docetaxel | Selinexor 40 mg Monotherapy | Selinexor 60 mg Monotherapy |
|---|---|---|---|---|---|---|
| STARTED | 0 | 0 | 0 | 0 | 0 | 0 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
Dose Expansion:Selinexor 60 mg+Docetaxel
| Stage | Dose Escalation Phase: Selinexor 60 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 80 mg and in Combination With Docetaxel | Dose Escalation Phase: Selinexor 100 mg and in Combination With Docetaxel | Dose Expansion Phase: Selinexor 60 mg and in Combination With Docetaxel | Selinexor 40 mg Monotherapy | Selinexor 60 mg Monotherapy |
|---|---|---|---|---|---|---|
| STARTED | 0 | 0 | 0 | 33 | 0 | 0 |
| COMPLETED | 0 | 0 | 0 | 33 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The study was halted prior to participants receiving selinexor monotherapy. No data was collected or analyzed for the selinexor Monotherapy Cohort.
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: 41 subjects were consented, but 1 screen failed and only 40 total were assigned to combination treatment cohort. 2nd dose level of 80 mg +docetaxel 75 mg/m2 was not tolerated and hence did not proceed to next dose of 100 mg+ docetaxel 75 mg/m2 as well. The study was halted prior to participants receiving any of the selinexor monotherapy. No data was collected or analyzed for the selinexor Monotherapy cohort.
| Characteristic | Unit | Dose Escalation: Selinexor 60 mg + Docetaxel | Dose Escalation: Selinexor 80 mg + Docetaxel | Dose Expansion Phase: Selinexor 60 + Docetaxel | Total |
|---|---|---|---|---|---|
| Participants analysed | 3 | 4 | 33 | 40 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 1 | 18 | 20 |
| Age, Categorical: >=65 years | Participants | 2 | 3 | 15 | 20 |
| Sex: Female, Male: Female | Participants | 3 | 3 | 17 | 23 |
| Sex: Female, Male: Male | Participants | 0 | 1 | 16 | 17 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 4 | 32 | 39 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 0 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 | 0 | 1 | 4 |
| Race (NIH/OMB): White | Participants | 0 | 3 | 30 | 33 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 1 | 1 |
| Region of Enrollment: United States | participants | 3 | 4 | 33 | 40 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Dose Limiting Toxicities (DLT), measured over Each 21 day cycle for 2 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the number of participants with dose-limiting toxicities (DLTs). The record defines a DLT as a Grade 3 or 4 adverse event using Common Terminology Criteria for Adverse Events Version 4.0, with collection intended to determine the maximum-tolerated dose. It further describes treatment-related missed doses during cycle 1 and early discontinuation due to study-drug-related toxicity as potential DLT criteria when considered at least possibly related to study treatment.
In general, a dose-limiting-toxicity endpoint is used in early-phase research to characterize tolerability at tested dose levels and inform dose selection for further evaluation. It focuses on events meeting the protocol's severity and attribution criteria during a defined assessment period; it is not a complete summary of every possible benefit or harm.
The registry specifies the DLT timeframe as each 21 day cycle for 2 years. However, it does not report analyzed counts for the primary endpoint or the number of participants assessed for DLT in each cohort. The registry's adverse-event entries are not a substitute for those missing primary-endpoint results: serious adverse events and DLTs are related safety concepts, but the record defines DLT using its own severity, attribution, and treatment-exposure criteria.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically focused safety outcome but does not report analyzed results for that primary endpoint. It lists zero analyses, and the primary outcome information gives its definition and intended use rather than observed DLT counts. Consequently, the DLT frequency, its precision, and any dose-level conclusion based on the primary endpoint are not reported in the registry record.
The safety entries do contain serious-adverse-event counts for combination cohorts. In the dose-escalation cohort of selinexor 60mg plus docetaxel 75mg/m2, zero of 3 participants at risk are listed as having a serious adverse event. In the selinexor 80mg plus docetaxel 75mg/m2 dose-escalation cohort, 4 of 4 are listed. In the dose-expansion cohort of selinexor 60 mg plus docetaxel 75mg/m2, zero of 33 are listed. No participants are listed at risk in the selinexor 100mg combination cohort or the monotherapy cohorts. These are descriptive registry counts, not estimates from a randomized comparison.
The serious-event pattern differs across the listed combination cohorts, but it should not be read as evidence that one dose is safer or more harmful than another. The cohorts are not reported as randomly assigned, their sizes differ, and the registry record does not provide a comparative analysis or uncertainty estimates. Counts from a small cohort can be especially imprecise as representations of a broader population. General statistical principle: a count or proportion based on limited observations may be compatible with a wide range of underlying event frequencies.
The record lists the top serious-event terms with percentages, including Platelets Decreased at 75.0%, Fatigue at 50.0%, and Hyponatremia at 50.0%, alongside other terms. The record does not make clear in this listing the denominator or whether a participant could contribute to more than one term. These percentages therefore describe the posted term listing, not a basis for reconstructing participant-level risk or comparing treatment groups.
The record reports zero deaths in each listed adverse-event group. This is a description of the posted group entries, not proof that a treatment has no mortality risk. Follow-up, the number of participants actually exposed in every group, and a comparative mortality analysis are not reported in the registry record. The study's terminated status and the absence of primary-endpoint analyses further limit conclusions about dose selection and safety.
9. Statistical Methods Explained
The primary outcome is a count of participants with a defined event. For each participant, the relevant classification is whether a DLT meeting the registry definition occurred during the specified assessment period. General statistical principle: binary outcomes are often summarized as the number with the event and the number assessed, or as a proportion with an accompanying confidence interval. A confidence interval expresses uncertainty around an estimate; it does not guarantee that the true value lies within the interval. The registry record does not report a primary-endpoint estimate or confidence interval for this trial.
What does the DLT definition capture?
The endpoint is narrower than a count of all adverse events. It requires a Grade 3 or 4 adverse event according to CTCAE 4.0, and the description adds treatment-related conditions involving missed study-treatment doses or discontinuation during cycle 1. Such criteria make the endpoint relevant to dose selection because they focus on serious tolerability concerns in the treatment period specified by the study. The classification also depends on severity grading and judgments about possible relationship to study treatment. The registry record does not report participant-level adjudication details or how uncertain attribution was handled.
Why do cohort counts not establish a dose comparison?
The study is listed as single-group, with no masking and allocation not applicable. Its dose cohorts are descriptive groups, not randomized arms. General statistical principle: a difference in event counts between nonrandomized groups may reflect differences in who entered each group, treatment exposure, follow-up, or other factors, as well as dose. Without random allocation and a stated comparative analysis, those influences cannot be separated from a dose effect using these counts alone.
Group size also matters when interpreting a count. A count of events should be read with its denominator and the amount of follow-up; the same count can imply different observed proportions in groups of different sizes. Even when a proportion is available, uncertainty generally depends on the amount of information in the group. The registry lists groups at risk for the adverse-event entries, but it does not report analyzed primary-endpoint denominators or confidence intervals. No additional denominator should be inferred for the DLT endpoint from the adverse-event entries.
How should the planning illustrations be understood?
The posted planning illustrations are described as calculations based on an observed effect, not as the trial's original design justification. Their stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is expressed as a stated fraction of the observed effect. General statistical principle: a planning calculation asks how much information a hypothetical future study might require under chosen assumptions; it does not describe what happened in the trial. Such sample-size illustrations are not observed power. The registry's own sample-size justification and statistical design rationale are not reported in the registry record.
In general, power is a design property defined relative to an assumed effect, variability, analysis, and significance threshold. It is not a measure of the probability that a particular result is true after seeing the data. An illustration based on an observed effect can be especially sensitive to the size and uncertainty of that estimate. Here, no numerical planning estimates or analyzed primary endpoint results are reported in the registry record, so no numerical sample-size conclusion can be drawn from the record alone.
The record describes a binary primary endpoint, so the sample-size assumptions about time-to-event event totals do not define this trial's endpoint. General statistical principle: time-to-event planning commonly focuses on the number of events, whereas binary-outcome planning commonly expresses group size as participants per arm. The registry record does not report a randomized two-arm analysis for this trial, and a hypothetical equal-allocation calculation should not be mistaken for the study's single-group cohort structure.
What analysis details are unavailable?
The registry record does not report a statistical analysis plan, a primary-endpoint estimate, confidence intervals, hypothesis tests, adjustment for covariates, or a model for repeated observations across cycles. It also does not report interim analyses or stopping rules. These details matter because analysis choices determine which observations contribute, how uncertainty is quantified, and how multiple outcomes are interpreted. No unreported method should be assumed to have been used.
General statistical principle: over-dispersion arises when observed variability exceeds what a chosen probability model expects. When present, standard errors based on a simpler model can be too small, making uncertainty appear less than it is. The record does not report a model-based analysis of this kind, and the primary endpoint is not presented as an analyzed series of rates. This concept is relevant only as a general consideration, not as a claim about the trial's data.
10. Multiplicity, Missing Data and Analysis Populations
The registry record does not report how multiplicity was handled. The study includes a primary safety outcome and a secondary outcome describing tumor-size assessment using RECIST v1.1, as well as safety-event listings. It does not state whether formal hypotheses were tested across these outcomes, whether any adjustment was planned, or whether the analyses were intended to be descriptive. Without that information, the chance of false-positive findings across multiple tests cannot be assessed from the record.
Missing-data methods and analysis populations are also not reported in the registry record. The record does not explain how participants who stopped treatment, missed assessments, or had incomplete follow-up would be handled in the primary analysis. It does not identify an intention-to-treat, safety, or per-protocol population for the primary endpoint, nor does it describe imputation or sensitivity analyses.
These omissions are important because dose-limiting toxicity depends on both whether a participant experiences an event and whether the participant is observed during the relevant treatment period. General statistical principle: incomplete follow-up can make an event proportion difficult to interpret if the chance of missing an event is related to treatment exposure or a participant's health. The registry record does not provide enough detail to determine whether such concerns affected the posted counts.
The monotherapy cohort did not receive selinexor and had no data collected or analyzed, according to the primary outcome description. Its absence is therefore not an ordinary missing outcome to be imputed from the combination cohorts. The registry's reason for halting the study and its approach to participants with incomplete combination-cohort data are not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Dose Escalation: Selinexor 60mg + Docetaxel 75mg/m2 | 0 | 3 | 0.0% |
| Dose Escalation: Selinexor 80mg + Docetaxel 75mg/m2 | 4 | 4 | 100.0% |
| Dose Expansion: Selinexor 60 mg + Docetaxel 75mg/m2 | 0 | 33 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dose Escalation: Selinexor 60mg + Docetaxel 75mg/m2 | 0 of 3 |
| Dose Escalation: Selinexor 80mg + Docetaxel 75mg/m2 | 0 of 4 |
| Dose Escalation:Selinexor 100mg + Docetaxel 75mg/m2 | 0 of 0 |
| Dose Expansion: Selinexor 60 mg + Docetaxel 75mg/m2 | 0 of 33 |
| Selinexor 40mg Monotherapy | 0 of 0 |
| Selinexor 60mg Monotherapy | 0 of 0 |
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 |
|---|---|
| Dose Escalation: Selinexor 60mg + Docetaxel 75mg/m2 | 3 of 3 |
| Dose Escalation: Selinexor 80mg + Docetaxel 75mg/m2 | 4 of 4 |
| Dose Escalation:Selinexor 100mg + Docetaxel 75mg/m2 | 0 of 0 |
| Dose Expansion: Selinexor 60 mg + Docetaxel 75mg/m2 | 33 of 33 |
| Selinexor 40mg Monotherapy | 0 of 0 |
| Selinexor 60mg Monotherapy | 0 of 0 |
Most frequent serious adverse event terms
| Event term | Organ system | Dose Escalation: Selinexor 60mg + Docetaxel 75mg/m2 | Dose Escalation: Selinexor 80mg + Docetaxel 75mg/m2 | Dose Escalation:Selinexor 100mg + Docetaxel 75mg/m2 | Dose Expansion: Selinexor 60 mg + Docetaxel 75mg/m2 | Selinexor 40mg Monotherapy | Selinexor 60mg Monotherapy |
|---|---|---|---|---|---|---|---|
| Platelets Decreased | Blood and lymphatic system disorders | 0 of 3 | 3 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Fatigue | General disorders | 0 of 3 | 2 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Hyponatremia | Metabolism and nutrition disorders | 0 of 3 | 2 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Abdominal Pain | Gastrointestinal disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Hypotension | Blood and lymphatic system disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Nausea | Gastrointestinal disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Vomiting | Gastrointestinal disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
| Head Laceration | General disorders | 0 of 3 | 1 of 4 | 0 of 0 | 0 of 33 | 0 of 0 | 0 of 0 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dose Escalation: Selinexor 60mg + Docetaxel 75mg/m2 | Dose Escalation: Selinexor 80mg + Docetaxel 75mg/m2 | Dose Escalation:Selinexor 100mg + Docetaxel 75mg/m2 | Dose Expansion: Selinexor 60 mg + Docetaxel 75mg/m2 | Selinexor 40mg Monotherapy | Selinexor 60mg Monotherapy |
|---|---|---|---|---|---|---|---|
| Fatigue | General disorders | 1 of 3 | 2 of 4 | 0 of 0 | 23 of 33 | 0 of 0 | 0 of 0 |
| Nausea | Gastrointestinal disorders | 3 of 3 | 1 of 4 | 0 of 0 | 21 of 33 | 0 of 0 | 0 of 0 |
| Diarrhea | Gastrointestinal disorders | 2 of 3 | 0 of 4 | 0 of 0 | 19 of 33 | 0 of 0 | 0 of 0 |
| Anemia | Blood and lymphatic system disorders | 3 of 3 | 3 of 4 | 0 of 0 | 11 of 33 | 0 of 0 | 0 of 0 |
| Anorexia | Metabolism and nutrition disorders | 2 of 3 | 1 of 4 | 0 of 0 | 13 of 33 | 0 of 0 | 0 of 0 |
| Vomiting | Gastrointestinal disorders | 3 of 3 | 1 of 4 | 0 of 0 | 11 of 33 | 0 of 0 | 0 of 0 |
| Neutropenia | Blood and lymphatic system disorders | 2 of 3 | 0 of 4 | 0 of 0 | 13 of 33 | 0 of 0 | 0 of 0 |
| Lymphocytes Decreased/Lymphopenia/Leukopenia | Investigations | 3 of 3 | 3 of 4 | 0 of 0 | 7 of 33 | 0 of 0 | 0 of 0 |
| Hyponatremia | Metabolism and nutrition disorders | 1 of 3 | 2 of 4 | 0 of 0 | 8 of 33 | 0 of 0 | 0 of 0 |
| Constipation | Gastrointestinal disorders | 1 of 3 | 0 of 4 | 0 of 0 | 10 of 33 | 0 of 0 | 0 of 0 |
Serious events with the highest arm-level rate: platelets decreased (75.0%), fatigue (50.0%), hyponatremia (50.0%), abdominal pain (25.0%), respiratory failure (25.0%).
The serious-adverse-event entries are cohort-specific counts. The dose-escalation cohort receiving selinexor 60mg plus docetaxel 75mg/m2 lists 0 of 3 at risk with a serious event; the cohort receiving selinexor 80mg plus docetaxel 75mg/m2 lists 4 of 4. The dose-expansion cohort receiving selinexor 60 mg plus docetaxel 75mg/m2 lists 0 of 33. The 100mg combination and monotherapy cohorts list zero participants at risk, so their zero event counts are not evidence of observed safety in exposed participants.
The listed top serious-event terms include Platelets Decreased (75.0%), Fatigue (50.0%), Hyponatremia (50.0%), Abdominal Pain (25.0%), Respiratory failure (25.0%), and Hypotension (25.0%). The registry record does not clarify the denominator for these term percentages or whether participants could be counted under multiple terms. They should not be treated as mutually exclusive categories or used to calculate an overall event rate.
These entries do not establish comparative safety. Cohorts were not randomized comparisons, and the record does not provide uncertainty intervals, exposure-adjusted rates, or a comparative analysis. It reports zero deaths in each listed group; that descriptive information does not establish an absence of mortality risk.
12. What the Result Does, and Does Not, Mean
The registry record documents a study intended to evaluate selinexor alone and with docetaxel, with dose-limiting toxicity as the primary outcome. It also records serious-adverse-event counts in combination cohorts. These registry entries describe the trial's stated purpose and the safety entries posted to the registry; they do not provide a completed statistical assessment of the primary endpoint.
Because analyzed DLT counts and their precision are not reported in the registry record, the record does not show the observed primary-endpoint result or establish a maximum-tolerated dose. The serious-event counts can be read as descriptive observations within the listed cohorts, but not as estimates from a controlled comparison. In particular, the cohorts cannot be treated as though participants had been randomly assigned to different doses or to a control treatment.
The monotherapy question remains unanswered in the posted record: the study was halted before participants received selinexor monotherapy, and no monotherapy data were collected or analyzed. The record also does not report the reason for termination. This limits interpretation of the planned scope, but it does not justify guessing why the study ended.
In general, early-phase safety results are intended to characterize tolerability under defined treatment and observation conditions. They do not, by themselves, establish comparative effectiveness or predict outcomes for people outside the studied groups. The registry lists a secondary tumor-size assessment using RECIST v1.1, but analyzed results for that outcome are not reported in the registry record. No treatment recommendation follows from these registry entries.
The responsible interpretation is therefore limited: the record identifies the intended endpoint and posts selected serious-event information, while leaving primary-endpoint analyses, comparative estimates, and details needed to assess uncertainty unavailable.
13. Why This Trial Matters Statistically
This trial illustrates an important distinction in early-phase research: a stated safety objective is not the same as a reported analysis of that objective. The registry defines dose-limiting toxicity and explains its role in determining the maximum-tolerated dose, but it does not provide analyzed DLT counts. A clear endpoint definition helps readers understand what investigators intended to measure; it cannot replace the results needed to evaluate that measurement.
The record also makes the limits of dose-cohort comparisons visible. A single-group, nonrandomized design can describe what was observed among participants treated at particular dose levels. It cannot by itself isolate a dose effect from differences in cohort composition, timing, or exposure. Descriptive adverse-event counts are useful as observations, but interpretation should not turn them into unsupported causal comparisons.
The halted study also demonstrates why planned and completed cohorts need to be distinguished. Selinexor monotherapy appears in the trial description, yet the record states that participants did not receive it and that no data were collected or analyzed for that cohort. Treating a planned cohort as an observed one would misrepresent the evidence.
Finally, registry completeness matters for statistical interpretation. Analysis populations, missing-data procedures, multiplicity handling, and the reason for termination are not reported in the registry record. Their absence limits assessment of precision and the process by which results might have been selected or interpreted. Naming those gaps is part of a careful reading, not a substitute for evidence.
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 record identifies the trial as terminated but does not report the reason for termination. It also does not report a complete accounting of participants by treatment received, the timing or duration of individual exposure, or follow-up completeness for the safety outcomes. Enrollment of 41 is reported, but that figure should not be substituted for a cohort-specific denominator or a primary-endpoint analysis population.
The primary outcome description says that the study halted before monotherapy participants received selinexor and that no data were collected or analyzed for the monotherapy cohort. The monotherapy groups therefore do not provide observed safety evidence. The registry lists combination cohorts at different dose levels and study stages, but it does not report a randomized allocation process or a control group. Their counts should not be compared as if treatment assignment were randomized.
The registry record does not report analyzed DLT counts, confidence intervals, a statistical analysis plan, handling of incomplete observations, or details of how adverse events were attributed to treatment. It does not clarify the denominator behind the listed serious-event percentages. These omissions prevent a full assessment of the primary endpoint and of uncertainty around the safety entries. The record also does not report analyzed results for the secondary tumor-size assessment.
These limitations are specific to the information recorded for this trial. They do not establish that analyses or procedures were absent from the study itself; those details are simply not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase 1/2 Trial of Selinexor (KPT-330) With Docetaxel for Non-small Cell Lung Cancer (NSCLC) — NCT03095612.
- Registry record last updated 2024-06-13; status last verified 2024-06.
- Results first posted 2024-06-13.
- 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
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18. Record Summary
NCT03095612 is a terminated investigator-sponsored Phase 1/2 trial of selinexor monotherapy and selinexor with docetaxel for previously treated, advanced KRAS mutant non-small cell lung cancer. The registry reports enrollment of 41, a single-group model, no masking, and no applicable allocation.
The primary outcome was the number of participants with dose-limiting toxicities, intended to inform the maximum-tolerated dose. The record states that monotherapy participants did not receive selinexor and that no monotherapy data were collected or analyzed. It lists serious-adverse-event counts for combination cohorts, but does not report analyzed primary-endpoint results, a comparative analysis, or the reason for termination.
Accordingly, the posted record supports descriptive reading of its cohort-specific safety entries, not a conclusion about comparative treatment effects or a confirmed maximum-tolerated dose.