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 Safety Study of KPT-330 (Selinexor) in Patients With Advanced or Metastatic Solid Tumor Cancer (NCT01607905), a non-randomized phase 1 study. The primary outcome is Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01607905 |
| Phase | Phase 1 |
| Condition | Solid Tumor |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 192 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Karyopharm Therapeutics Inc |
| Results posted | Yes |
2. Clinical Question
Population
Participants with advanced or metastatic solid tumors, represented in seven cancer-specific groups: colorectal cancer, gynecological cancer, squamous cell cancer, castrate-resistant prostate cancer, glioblastoma multiforme, melanoma, and other solid tumors. The safety population included participants who received at least one dose of selinexor. The registry record reports 192 enrolled participants, but the adverse-event results are organized by cancer group rather than by dose level.
Intervention
Oral selinexor, administered as a single agent in the cancer-specific groups and across different schedules. The study also lists acetaminophen as an intervention, but its role is not reported in the registry record. The complete schedule descriptions are not reported in the registry record.
Comparator
There was no randomized control group. The study used a non-randomized, parallel design with no masking. The cancer groups are not like-for-like treatment comparisons: they represent different cancer populations, and the registry record does not present them as randomized treatment arms.
Primary question
The study’s primary aim was to evaluate selinexor safety and tolerability and determine a Recommended Phase 2 Dose for advanced or metastatic solid tumors. The primary outcome counted participants with treatment-emergent adverse events and treatment-emergent serious adverse events. These counts describe reported safety events in the treated population; they do not establish a comparative effect against an untreated or active control group.
The primary outcome concerns events that began after study drug was received, with serious events identified using criteria including fatality, life-threatening events, inpatient hospitalization or its prolongation, persistent or significant disability, congenital anomaly or birth defect, and other important medical events. The registry record specifies the observation period as from the start of study drug administration to 30 days after the last dose, with a maximum duration of 45 months.
Pharmacokinetic measures and best overall response were also listed as secondary outcomes. Their result values are not reported in the registry record. The primary safety question can therefore be described from the posted adverse-event counts, but the listed secondary outcomes cannot be interpreted from these results.
3. Trial Design
This was a Phase I, completed study sponsored by Karyopharm Therapeutics Inc. The registry record lists an enrollment of 192 and a non-randomized, parallel design with no masking. It describes the study objective as evaluating safety and tolerability and determining a Recommended Phase 2 Dose of selinexor for advanced or metastatic solid tumor malignancies.
Participants were divided into seven cancer-specific groups: colorectal cancer, gynecological cancer, squamous cell cancer, castrate-resistant prostate cancer, glioblastoma multiforme, melanoma, and other solid tumors. The arm descriptions identify oral selinexor as a single agent and refer to multiple dosing schedules. They also indicate schedules based on dosing during weeks of a 28-day cycle. However, the complete schedule descriptions and the details needed to map every participant to a dose level are not reported in the registry record.
The study did not use random assignment to compare selinexor with a control treatment. The named arms are organized by cancer type, so differences between their reported safety counts could reflect differences in the groups as well as treatment exposure. The registry record explicitly says data were not collected per dose level and presents the available safety information by cancer stratification. This limits what can be learned about dose-specific safety from the reported counts.
The stated safety population includes all participants who received at least one dose of selinexor. The registry record does not provide a full account of how many enrolled participants received treatment, nor does it report dose-specific populations. It lists acetaminophen as an intervention, but the role of that intervention is not reported in the registry record.
The study began on 2012-06-18, reached primary completion on 2016-03-15, and is listed as completed. The primary outcome counted participants with treatment-emergent adverse events and serious treatment-emergent adverse events during the specified safety observation period. Secondary outcomes included pharmacokinetic measures and the number of participants with best overall response; their results are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: A total of 192 participants were enrolled, of which 191 participants started study (1 participant reported missing malignancy), 189 participants received at least 1 dose of study drug.
Only the first 6 of 7 groups are shown.
Overall Study
| Stage | Arm A (Colorectal Cancer) | Arm B (Gynecological Cancer) | Arm C (Squamous Cell Cancer) | Arm D (Castrate-resistant Prostate Cancer) | Arm E (Glioblastoma Multiforme) | Arm F (Melanoma) |
|---|---|---|---|---|---|---|
| STARTED | 60 | 20 | 21 | 21 | 7 | 15 |
| Received Study Drug | 59 | 20 | 21 | 21 | 6 | 15 |
| Low Dose (<30 mg/m^2) | 16 | 5 | 3 | 0 | 0 | 2 |
| Medium Dose (>=30 mg/m^2 and <55 mg/m^2) | 27 | 11 | 15 | 8 | 6 | 3 |
| High Dose (>= 55 mg/m^2) | 16 | 4 | 3 | 13 | 0 | 10 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 60 | 20 | 21 | 21 | 7 | 15 |
Reasons for not completing this period:
| Reason | Arm A (Colorectal Cancer) | Arm B (Gynecological Cancer) | Arm C (Squamous Cell Cancer) | Arm D (Castrate-resistant Prostate Cancer) | Arm E (Glioblastoma Multiforme) | Arm F (Melanoma) |
|---|---|---|---|---|---|---|
| Withdrawal by participant | 7 | 2 | 4 | 6 | 0 | 4 |
| Incidence or severity of AEs | 5 | 1 | 1 | 3 | 0 | 2 |
| Disease progression | 44 | 16 | 15 | 11 | 6 | 7 |
| Non-compliance with study procedures | 0 | 0 | 0 | 0 | 0 | 0 |
| Death | 1 | 1 | 1 | 1 | 0 | 0 |
| Other treatments become available | 1 | 0 | 0 | 0 | 0 | 0 |
| Investigator discretion | 0 | 0 | 0 | 0 | 0 | 1 |
| Other | 1 | 0 | 0 | 0 | 0 | 0 |
| Randomized but not treated | 1 | 0 | 0 | 0 | 1 | 1 |
Analysis population for the primary outcome, as defined in the registry: Safety population included all participants who received at least one dose of selinexor. As pre-specified in the study Protocol data were not collected per dose level. The limited data available based on cancer stratification as pre-specified in the protocol is presented.
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: Safety Population included all participants who received at least one dose of selinexor. As pre-specified in the study Protocol data were not collected per dose level. The limited data available based on cancer stratification as pre-specified in the protocol is presented.
Only the first 6 of 8 groups are shown.
| Characteristic | Unit | Arm A (Colorectal Cancer) | Arm B (Gynecological Cancer) | Arm C (Squamous Cell Cancer) | Arm D (Castrate-resistant Prostate Cancer) | Arm E (Glioblastoma Multiforme) | Arm F (Melanoma) |
|---|---|---|---|---|---|---|---|
| Participants analysed | 59 | 20 | 21 | 21 | 6 | 15 | |
| Age, Continuous | years | 59.8 (SD 11.35) | 55.0 (SD 12.54) | 59.2 (SD 7.39) | 68.7 (SD 7.69) | 56.0 (SD 14.94) | 67.7 (SD 11.04) |
| Sex: Female, Male: Female | Participants | 25 | 20 | 9 | 0 | 3 | 4 |
| Sex: Female, Male: Male | Participants | 34 | 0 | 12 | 21 | 3 | 11 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 1 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 3 | 2 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 10 | 0 | 4 | 2 | 0 | 1 |
| Race (NIH/OMB): White | Participants | 45 | 18 | 17 | 16 | 6 | 14 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 0 | 2 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs), measured over From start of study drug administration to 30 days after last dose of study treatment (maximum duration of 45 months).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with treatment-emergent adverse events and treatment-emergent serious adverse events. A treatment-emergent adverse event was defined as an adverse event whose onset occurred after the participant received study drug. The registry record emphasizes that an adverse event is an untoward medical occurrence and does not necessarily have a causal relationship with treatment. Thus, the count concerns events observed after treatment began, not events proven to have been caused by selinexor.
Seriousness was defined using specified clinical and regulatory criteria: an event was serious if it was fatal, life-threatening, required inpatient hospitalization or prolonged existing hospitalization, caused persistent or significant disability or incapacity, involved a congenital anomaly or birth defect, or qualified as an important medical event. Seriousness is a classification of the event’s consequences or significance, not a measure of how likely the event was to be caused by the drug.
Counting participants with events is a useful descriptive safety measure in an early-phase study. It communicates how many treated participants had at least one event within the reporting category, rather than the total number of event episodes. The observation period ran from the start of study drug administration to 30 days after the last dose, up to a maximum duration of 45 months.
The endpoint supports a description of reported safety experience in the treated population. It does not, by itself, establish causation, quantify risk relative to a control group, or identify a dose-response pattern. Dose-level event data are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record reports serious treatment-emergent adverse-event counts separately for each cancer group. The reported numbers of participants with serious events were 30 of 59 in colorectal cancer, 13 of 20 in gynecological cancer, 9 of 21 in squamous cell cancer, 8 of 21 in castrate-resistant prostate cancer, 2 of 6 in glioblastoma multiforme, 5 of 15 in melanoma, and 24 of 47 in other solid tumors. These are descriptive counts within the respective groups, not treatment-versus-control estimates.
For other treatment-emergent adverse events, the reported counts were 59 of 59 in colorectal cancer, 20 of 20 in gynecological cancer, 21 of 21 in squamous cell cancer, 21 of 21 in castrate-resistant prostate cancer, 6 of 6 in glioblastoma multiforme, 15 of 15 in melanoma, and 46 of 47 in other solid tumors. The registry record does not explain the distinction between the serious and other categories beyond reporting them separately. The counts should not be interpreted as mutually exclusive participant groups: a participant may have had both serious and other events.
These results describe the safety population by cancer stratification, as specified in the registry record. Because the study was non-randomized and did not include a like-for-like control comparison, differences in counts between cancer groups are not estimates of comparative treatment effects. The groups differ by cancer type, and dose-level results were not collected for these reported data. The registry record does not provide an adjustment for those differences or a dose-specific analysis.
The registry record lists thrombocytopenia, anaemia, lung infection, hyperglycaemia, lethargy, and embolism among the top serious-event terms, with each listed at 16.7%. These terms identify reported serious-event categories; the registry record does not provide enough detail alongside these values to interpret them as participant-level risks across the entire safety population or as causal effects of selinexor.
No inferential analysis is listed for these outcomes. The record reports zero analyses, and it provides no confidence intervals or hypothesis-test results for the event counts. General statistical interpretation: an observed count is subject to sampling variability, especially in small groups. A confidence interval, when reported, expresses uncertainty around an estimate under a specified statistical model; no such intervals are reported for these counts.
The enrollment figure and the safety counts answer different questions. Enrollment is listed as 192, whereas the adverse-event results are presented for the cancer-specific safety groups. The registry record does not explain the full flow from enrollment to the safety population in the outcome results. The counts therefore should be read as the posted group-specific safety results, not as a complete participant-flow account.
Overall, the posted results document that treatment-emergent adverse events were recorded in the cancer-specific groups and report serious-event counts for each group. They do not provide a randomized comparison, dose-level safety estimates, or the inferential detail needed to quantify precision or attribute events to treatment. The listed pharmacokinetic and best-overall-response outcomes do not have result values reported in the registry record.
9. Statistical Methods Explained
What does a participant count measure?
The primary outcome is a count of participants who experienced treatment-emergent adverse events and serious treatment-emergent adverse events. A participant count represents people meeting the event definition, rather than the number of separate event episodes. If one participant experienced more than one event, a participant-based measure would still count that person within the relevant category. The registry record does not provide event-level totals or specify whether participants could appear in both reported categories, so the two categories should not be treated as mutually exclusive.
General statistical explanation: a count is easiest to interpret alongside the number of participants at risk in the same group. The posted results give both values for the cancer-stratified groups. The count and denominator describe the observed group experience; they do not alone tell whether an event was caused by treatment or how the group would have fared under another treatment.
Why is a safety population used?
The registry record defines the safety population as all participants who received at least one dose of selinexor. This is a common descriptive population for safety reporting because exposure to the study drug is a prerequisite for assessing events arising after treatment begins. It differs conceptually from a population based only on enrollment, since not every enrolled participant must necessarily receive study drug. The registry record does not provide a complete participant-flow account or dose-specific safety populations, so the distinction cannot be quantified further from the posted results.
The stated observation period begins with study drug administration and continues to 30 days after the last dose, with a maximum duration of 45 months. This defines the window in which events are counted for the primary outcome. It does not mean that each participant was observed for the maximum duration; participant-specific observation times are not reported in the registry record.
How should serious-event categories be understood?
Seriousness is based on the criteria listed in the registry record: fatality, life-threatening events, inpatient hospitalization or its prolongation, persistent or significant disability or incapacity, congenital anomaly or birth defect, and important medical events. Serious adverse events are not automatically treatment-related. The record’s definition of adverse event expressly allows an event to occur without a causal relationship to treatment.
General statistical explanation: adverse-event summaries are descriptive unless the design and analysis support a causal comparison. In this study, no randomized control group is reported. The serious-event counts therefore describe observed events among treated participants and should not be interpreted as a difference attributable to selinexor.
What can be learned from the non-randomized design?
Participants were organized into parallel cancer-specific groups, not randomized treatment groups. Each group represents a different cancer population, and the posted safety results are stratified by that population. A comparison of counts across groups would not isolate a treatment effect, because the groups are not presented as comparable controls and the registry record does not report an adjustment for their differences.
The study included multiple selinexor schedules, but the registry record says safety data were not collected per dose level. As a result, the posted outcome cannot be used to identify which dose or schedule had a particular event count. The full schedule descriptions are not reported in the registry record, further limiting dose-specific interpretation.
What do the listed statistical analyses establish?
The registry record lists zero analyses for the posted results. It does not report hypothesis tests, confidence intervals, adjusted estimates, or a statistical model for the primary outcome. The primary results should therefore be read as descriptive counts, rather than evidence of a statistically tested difference.
General statistical explanation: a confidence interval is a range of values compatible with the observed data under a statistical model and its assumptions. It is useful because an estimate from a finite group is uncertain, but no confidence intervals are reported for these adverse-event counts. A p-value, when used, addresses the compatibility of data with a specified statistical hypothesis; no p-values for the primary outcome are reported.
What does the sample-size illustration mean?
The statistical material associated with the registry record includes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. The illustration is not the trial’s own sample-size justification, and the justification is not reported in the registry record.
General statistical explanation: a sample-size calculation estimates how many participants would be needed under specified assumptions to detect a defined effect with a chosen power and significance level. Such planning values are not evidence that a study had a particular probability of detecting an effect after it was completed. They are not observed power, and they do not replace the reported results. The posted illustration is especially limited for this study because its design is non-randomized and the primary safety results do not provide a like-for-like two-arm treatment comparison.
The registry record does not report the protocol’s sample-size justification or the assumptions used in its original design. No conclusion about the adequacy of the planned enrollment can be made from the planning illustration alone.
Why does over-dispersion matter in general?
General statistical explanation: for count outcomes, some models assume that variability follows a specified relationship to the mean. Over-dispersion means the observed variability is greater than that assumption allows. If a count model ignores over-dispersion, uncertainty estimates can be too narrow and inferential results can appear more precise than warranted. The posted primary outcome is a participant count, but the registry record does not report a count model, a dispersion assessment, or an inferential analysis. Over-dispersion therefore cannot be assessed from the reported results.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary safety outcome and several secondary outcomes. The secondary outcomes include selinexor pharmacokinetic measures—maximum observed plasma concentration, time to maximum observed concentration, area under the concentration-time curve through the last measured time and extrapolated to infinity, elimination half-life, apparent total body clearance, and apparent volume of distribution—as well as the number of participants with best overall response. Result values for these outcomes are not reported in the registry record.
The registry record does not report a multiplicity strategy for testing or interpreting multiple outcomes. It also does not report interim analyses or a statistical analysis plan. Without those details and without reported inferential tests, the effect of multiple testing on any statistical conclusions cannot be assessed. The primary adverse-event results are descriptive counts, and no p-values or confidence intervals are reported for them.
Missing-data handling is not reported in the registry record. The record does not explain whether outcome data were missing, how any missing observations were handled, or whether participants who discontinued treatment were followed through the full safety observation period. The listed safety population includes participants who received at least one dose, but the record does not provide enough information to reconstruct participant flow or completeness of outcome collection.
The record states that safety data were not collected per dose level and that the available results are presented by cancer stratification. Thus, missing or unavailable dose-level detail is a limitation of the reported analysis, but the registry record does not say whether dose-level data were collected elsewhere. The record also does not report a pre-specified statistical approach for comparing cancer groups. These gaps prevent assessment of any adjustment for missing data, multiplicity, or between-group differences.
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 |
|---|---|---|---|
| Arm A (Colorectal Cancer) | 30 | 59 | 50.8% |
| Arm B (Gynecological Cancer) | 13 | 20 | 65.0% |
| Arm C (Squamous Cell Cancer) | 9 | 21 | 42.9% |
| Arm D (Castrate-resistant Prostate Cancer) | 8 | 21 | 38.1% |
| Arm E (Glioblastoma Multiforme) | 2 | 6 | 33.3% |
| Arm F (Melanoma) | 5 | 15 | 33.3% |
| Arm G (Other Solid Tumors) | 24 | 47 | 51.1% |
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 |
|---|---|
| Arm A (Colorectal Cancer) | 59 of 59 |
| Arm B (Gynecological Cancer) | 20 of 20 |
| Arm C (Squamous Cell Cancer) | 21 of 21 |
| Arm D (Castrate-resistant Prostate Cancer) | 21 of 21 |
| Arm E (Glioblastoma Multiforme) | 6 of 6 |
| Arm F (Melanoma) | 15 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm A (Colorectal Cancer) | Arm B (Gynecological Cancer) | Arm C (Squamous Cell Cancer) | Arm D (Castrate-resistant Prostate Cancer) | Arm E (Glioblastoma Multiforme) | Arm F (Melanoma) |
|---|---|---|---|---|---|---|---|
| Lung infection | Infections and infestations | 2 of 59 | 2 of 20 | 1 of 21 | 0 of 21 | 1 of 6 | 0 of 15 |
| Large intestinal obstruction | Gastrointestinal disorders | 1 of 59 | 2 of 20 | 1 of 21 | 0 of 21 | 0 of 6 | 0 of 15 |
| Small intestinal obstruction | Gastrointestinal disorders | 1 of 59 | 3 of 20 | 0 of 21 | 0 of 21 | 0 of 6 | 0 of 15 |
| Fatigue | General disorders | 2 of 59 | 2 of 20 | 0 of 21 | 0 of 21 | 0 of 6 | 0 of 15 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 2 of 59 | 0 of 20 | 0 of 21 | 0 of 21 | 0 of 6 | 2 of 15 |
| Blood bilirubin increased | Investigations | 2 of 59 | 0 of 20 | 1 of 21 | 1 of 21 | 0 of 6 | 0 of 15 |
| Sepsis | Infections and infestations | 1 of 59 | 1 of 20 | 1 of 21 | 0 of 21 | 0 of 6 | 0 of 15 |
| Back pain | Musculoskeletal and connective tissue disorders | 3 of 59 | 0 of 20 | 0 of 21 | 0 of 21 | 0 of 6 | 0 of 15 |
| Syncope | Nervous system disorders | 1 of 59 | 0 of 20 | 0 of 21 | 2 of 21 | 0 of 6 | 0 of 15 |
| Embolism | Vascular disorders | 0 of 59 | 1 of 20 | 0 of 21 | 1 of 21 | 1 of 6 | 0 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm A (Colorectal Cancer) | Arm B (Gynecological Cancer) | Arm C (Squamous Cell Cancer) | Arm D (Castrate-resistant Prostate Cancer) | Arm E (Glioblastoma Multiforme) | Arm F (Melanoma) |
|---|---|---|---|---|---|---|---|
| Fatigue | General disorders | 46 of 59 | 16 of 20 | 14 of 21 | 17 of 21 | 5 of 6 | 10 of 15 |
| Nausea | Gastrointestinal disorders | 46 of 59 | 19 of 20 | 12 of 21 | 9 of 21 | 3 of 6 | 11 of 15 |
| Decreased appetite | Metabolism and nutrition disorders | 42 of 59 | 13 of 20 | 11 of 21 | 14 of 21 | 5 of 6 | 9 of 15 |
| Vomiting | Gastrointestinal disorders | 35 of 59 | 17 of 20 | 11 of 21 | 7 of 21 | 2 of 6 | 5 of 15 |
| Weight decreased | Investigations | 32 of 59 | 8 of 20 | 11 of 21 | 10 of 21 | 4 of 6 | 7 of 15 |
| Hyponatraemia | Metabolism and nutrition disorders | 36 of 59 | 8 of 20 | 7 of 21 | 6 of 21 | 1 of 6 | 9 of 15 |
| Anaemia | Blood and lymphatic system disorders | 23 of 59 | 13 of 20 | 13 of 21 | 10 of 21 | 3 of 6 | 4 of 15 |
| Thrombocytopenia | Blood and lymphatic system disorders | 26 of 59 | 6 of 20 | 9 of 21 | 12 of 21 | 3 of 6 | 8 of 15 |
| Diarrhoea | Gastrointestinal disorders | 24 of 59 | 11 of 20 | 8 of 21 | 7 of 21 | 1 of 6 | 7 of 15 |
| Constipation | Gastrointestinal disorders | 21 of 59 | 9 of 20 | 6 of 21 | 11 of 21 | 1 of 6 | 5 of 15 |
Serious events with the highest arm-level rate: thrombocytopenia (16.7%), anaemia (16.7%), lung infection (16.7%), hyperglycaemia (16.7%), lethargy (16.7%).
The serious-event figures are participant counts within each cancer-specific group. The registry record reports 30 of 59 participants with serious treatment-emergent adverse events in colorectal cancer; 13 of 20 in gynecological cancer; 9 of 21 in squamous cell cancer; 8 of 21 in castrate-resistant prostate cancer; 2 of 6 in glioblastoma multiforme; 5 of 15 in melanoma; and 24 of 47 in other solid tumors.
These figures describe the number with at least one serious event in the corresponding group, under the registry’s safety-population definition. They are not comparisons with a control group. The groups have different cancer types, and the study did not randomize participants between treatment and control conditions. The record also says results were not collected per dose level, so these counts do not identify the safety experience for an individual dose or schedule.
The six listed top serious-event terms are thrombocytopenia, anaemia, lung infection, hyperglycaemia, lethargy, and embolism; each is listed at 16.7%. The registry record does not provide sufficient detail to treat those values as overall participant-level risks or as evidence that selinexor caused the events. Seriousness indicates that an event met one or more specified seriousness criteria; it is not a causal attribution.
The counts of other treatment-emergent adverse events are reported separately. Participants could potentially be represented in both categories, and the registry record does not state that the categories are mutually exclusive.
12. What the Result Does, and Does Not, Mean
The posted results describe safety events observed among participants who received at least one dose of selinexor, organized into cancer-specific groups. They show that the registry recorded serious treatment-emergent adverse events in each group and report the corresponding participant counts. They also report counts for other treatment-emergent adverse events. These observations are useful as a record of reported events during the defined safety observation period.
The counts do not show how many events were caused by selinexor. The registry definition explicitly states that an adverse event does not necessarily have a causal relationship with treatment. The figures also do not establish how event frequency compares with another treatment or with no treatment, because the study was non-randomized and no like-for-like control group is reported.
The results do not identify a safest dose or schedule. Although the study included multiple selinexor schedules, the registry record says data were not collected per dose level. It therefore does not support dose-specific comparisons from these safety counts. Similarly, differences between cancer groups cannot be interpreted as treatment differences because the groups are defined by different cancer types and are not randomized comparators.
The primary objective included determining a Recommended Phase 2 Dose, but the registry record does not report a dose recommendation in the results. It also does not report numerical results for the listed pharmacokinetic outcomes or best overall response. Those outcomes cannot be interpreted from their names alone.
No confidence intervals, hypothesis tests, or other inferential analyses are reported for the primary safety outcome. The count data therefore provide a descriptive record, not a quantified estimate of comparative effect or statistical precision. The results should be understood within the limits of the reported population, outcome definitions, and available level of detail.
13. Why This Trial Matters Statistically
This study illustrates a common statistical challenge in early-phase research: safety information may be collected across multiple doses and disease groups, while the available results are summarized at a broader level. The registry record identifies seven cancer-specific groups and multiple selinexor schedules, but says safety data were not collected per dose level. That reporting structure limits the ability to connect observed events to a particular schedule or dose.
The distinction between a safety endpoint and a comparative endpoint is also important. Counting participants with adverse events can describe what occurred after treatment began, but without randomization and a suitable control group, it does not quantify an effect attributable to treatment. In this study, the cancer-stratified groups are not like-for-like controls, so their event counts should not be read as comparative treatment estimates.
The record also demonstrates why outcome definitions and populations matter. The safety population is defined by receipt of at least one selinexor dose, and the observation window extends from treatment initiation to 30 days after the last dose, up to a maximum duration of 45 months. The reported counts depend on those definitions and should not be generalized beyond the population and period described without further evidence.
Finally, the distinction between an event’s seriousness and its causality is central to safety interpretation. The listed serious-event criteria classify clinical significance; they do not establish that treatment caused an event. Clear separation of observed counts, causal comparisons, and dose-specific analyses helps readers avoid drawing conclusions that the reported design and data do not support.
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 main trial-specific limitation is the absence of a randomized control group. The study is non-randomized, and the cancer-specific groups are not described as comparable treatment arms. Consequently, the posted counts do not provide a controlled estimate of treatment-associated risk.
The available safety results are not organized by dose level. The registry record states that data were not collected per dose level and presents the available information by cancer stratification. Because the study included multiple schedules, the absence of dose-specific results prevents interpretation of event counts by dose or schedule. The complete schedule descriptions are not reported in the registry record.
The cancer groups are heterogeneous in diagnosis and group size. Their reported event counts should not be treated as direct comparisons, and the registry record does not report a statistical adjustment for differences between groups. The record also lists enrollment of 192 but does not provide a complete flow from enrollment to the safety population in the outcome results.
Statistical detail is limited. The registry record lists zero analyses for the posted outcomes and does not report confidence intervals, hypothesis tests, a statistical model, or the protocol’s sample-size justification. It does not report missing-data handling, interim analyses, or a multiplicity strategy. These omissions limit assessment of precision, completeness, and the planned interpretation of outcomes.
Finally, the registry record lists pharmacokinetic outcomes and best overall response without reporting their result values. The primary safety counts therefore cannot be combined with those outcomes to assess the full set of stated study aims. The record does not provide enough information to evaluate dose selection or the basis for a Recommended Phase 2 Dose.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety Study of KPT-330 (Selinexor) in Patients With Advanced or Metastatic Solid Tumor Cancer — NCT01607905.
- Registry record last updated 2023-01-26; status last verified 2023-01.
- Results first posted 2021-04-30.
- 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.
18. Record Summary
NCT01607905 is a completed Phase I, non-randomized, parallel study with no masking, sponsored by Karyopharm Therapeutics Inc. It lists 192 enrolled participants with advanced or metastatic solid tumors and evaluates selinexor across seven cancer-specific groups. Its primary outcome counts participants with treatment-emergent adverse events and serious treatment-emergent adverse events in a safety population defined as participants who received at least one dose of selinexor.
The registry reports group-specific safety counts, but says data were not collected per dose level. It does not report a randomized control comparison, inferential analyses, confidence intervals, or dose-specific safety results. Pharmacokinetic measures and best overall response are listed as secondary outcomes, but their result values are not reported in the registry record. The posted results support descriptive reading of the reported safety experience, not causal or comparative conclusions.