Ruxolitinib for Cancer Cachexia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Ruxolitinib for Cancer Cachexia (NCT04906746), a na early/phase 1 study. The primary outcome is Number of Non-small Cell Lung Cancer Cachexia Patients With Toxicity With the Use of Ruxolitinib.

2. Clinical Question

3. Trial Design

The registry describes a pilot study of ruxolitinib for cancer cachexia in people with stage IV non-small cell lung cancer. Its listed phase is EARLY PHASE1, its model is single group, and allocation is not applicable. Masking is none. These features mean the registry does not describe a randomized, masked comparison between treatment groups.

The listed regimen proceeds from 0 mg ruxolitinib twice daily in Month 1 to 10 mg twice daily in Month 2 and 15 mg twice daily in Month 3. The primary outcome description states that 2 participants received at least one dose of 10 mg twice daily and proceeded to receive at least one dose of 15 mg twice daily. It also says that all enrolled patients were included in the 0 mg group, which was not assessed for outcomes because of its baseline status and is not included in the primary outcome description.

The registry lists enrollment as 10. The relationship between that enrollment figure and the smaller numbers in the dose-specific safety descriptions is not fully explained in the registry record. It does not report a detailed participant flow, reasons for non-progression through the dosing regimen, or an analysis plan for handling participants who did not receive each dose.

The study began on 2022-02-23, has a listed primary completion date of 2024-09-27, and is marked active, not recruiting. The registry does not report a randomized control group, so dose groups should not be interpreted as comparable arms from a controlled trial. Changes across dose periods may also reflect the sequence and timing of exposure, rather than dose 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.

Month 1: 0mg Ruxolitinib

Month 2: 10mg Ruxolitinib

Reasons for not completing this period:

Month 3: 15mg Ruxolitinib

Analysis population for the primary outcome, as defined in the registry: 2 Participants received at least one dose of 10 mg BID Ruxolitinib and proceeded to receive at least one dose of 15 mg BID Ruxolitinib. All patients enrolled were included in the Ruxolitinib 0mg group, which was not assessed for outcomes because of its baseline status, and is not included in this table.

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 Number of Non-small Cell Lung Cancer Cachexia Patients With Toxicity With the Use of Ruxolitinib, measured over 3 months.

The primary endpoint is the number of non-small cell lung cancer cachexia participants with toxicity associated with ruxolitinib. It is expressed as a count of participants, and its timeframe is 3 months. The description says NCI CTCAE v5.0 toxicity criteria will be used to measure ruxolitinib-related side effects, with grade 3 or higher events recorded.

A count of participants with an event can communicate how many people meet the endpoint definition, rather than the number of separate event episodes. That distinction matters when a participant experiences more than one event: a participant count and an event count answer different questions. The registry’s endpoint is framed as a participant count.

General: monitoring toxicity is important in an early-phase study because safety and tolerability are central questions when a treatment is being assessed in a clinical setting. A threshold based on event grade focuses attention on events meeting a specified severity criterion. It does not, by itself, establish whether an event was caused by the study drug.

The registry record does not report a separate numerical result for this primary endpoint. The posted adverse-event counts include serious and other events by dose group, but serious events are not interchangeable with grade 3 or higher toxicity. Without a reported mapping between those categories and the primary endpoint definition, the safety counts should not be presented as the primary endpoint result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The study is listed as a single-group, unmasked, early-phase study with allocation not applicable. General: a single-group design describes outcomes among participants receiving the study regimen, without a randomized group serving as a concurrent comparison. It can be useful for describing feasibility and observed safety, but it does not provide the same basis for causal comparisons as a randomized design. In this record, the 0 mg period is explicitly treated as baseline status rather than as an outcome-assessed control group.

What does a participant count represent?

The primary endpoint is a count of participants with toxicity, not a rate of events per unit of follow-up and not a count of every event episode. If a person has multiple events, a participant-based outcome ordinarily counts that person according to whether the endpoint definition was met. The record names grade 3 or higher events under NCI CTCAE v5.0 criteria, but does not report a primary-endpoint count that can be directly matched to that definition.

The adverse-event listing separately reports numbers at risk and affected for serious and other events. These labels should be read as separate reported categories. The registry does not explain how the categories relate to the grade threshold in the primary endpoint. Nor does the record establish whether a participant could contribute to both serious and other event counts. It is therefore inappropriate to combine the categories or treat them as interchangeable.

How should the dose groups be understood?

The regimen lists successive months at 0 mg, 10 mg, and 15 mg twice daily. The primary outcome description says that 2 participants received at least one dose at 10 mg and proceeded to at least one dose at 15 mg. This sequence is not a randomized dose comparison. General: in a sequential regimen, dose and time can be entangled; participants may have different exposure histories, and changes between periods cannot automatically be attributed to the dose change. The record does not report an analysis that separates dose effects from time or other influences.

The registry states that everyone enrolled was included in the 0 mg group, which was not assessed for outcomes because of its baseline status. The posted adverse-event figures nevertheless list group-specific counts for 0 mg. The record does not reconcile this wording with those figures. Because the registry explicitly says the 0 mg group was not assessed as an outcome group, it should not be used as a like-for-like control in interpreting the active-dose counts.

What do the planning assumptions mean?

General: a sample-size planning illustration asks what group size might be needed under stated assumptions to detect a specified effect with a chosen statistical procedure. Such a calculation is not an observed trial result and does not show that a study had a particular probability of detecting an effect after the fact. The registry describes planning assumptions based on a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. For time-to-event outcomes, it describes total events; for other outcomes, it describes participants per arm.

Those assumptions are planning conventions, not evidence that this single-group study had equal allocation or a randomized two-arm design. The registry record does not report the trial’s own sample-size justification or a statistical analysis plan. No analysis results are listed in the record’s analysis count. Therefore, planning assumptions should not be mistaken for the study’s prespecified design or for observed evidence about efficacy or safety.

What can uncertainty tell a reader?

General: a confidence interval describes the range of effect estimates reasonably compatible with the data under a statistical model. It conveys precision as well as direction; a wide interval indicates considerable uncertainty. For small event counts, uncertainty is often substantial, and a count alone does not show how precise an estimated event frequency is. The registry record does not report confidence intervals for the posted adverse-event counts.

General: over-dispersion is a situation in which observed variation exceeds what a chosen probability model expects. It can matter when analyzing repeated counts or clustered outcomes because methods that assume less variation may understate uncertainty. The registry record does not report a count-model analysis or an over-dispersion assessment, and the primary endpoint is described as a participant count. These concepts are relevant to statistical interpretation generally, but should not be presented as methods actually used in this study.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 9 outcome measures, including the primary toxicity outcome and secondary outcomes addressing adverse events, objective response by RECIST criteria, quality of life, body weight loss, and overall survival. Considering multiple outcomes raises a general multiplicity issue: when many questions are assessed, the chance of at least one apparently notable result can increase. Statistical approaches can address multiplicity, but the registry record does not report a multiplicity adjustment or a prespecified hierarchy among the outcomes.

The registry lists 0 analyses. A statistical analysis plan, including the intended tests, estimation methods, and handling of multiple outcomes, is not reported in the registry record. Interim analyses are also not reported in the registry record. This absence does not establish what may have occurred outside the registry; it means the registry does not document those methods.

Missing-data methods and analysis populations are not reported in the registry record. Although the primary outcome description notes that 2 participants received at least one dose of 10 mg twice daily and proceeded to at least one dose of 15 mg twice daily, it does not fully describe participant disposition or explain how incomplete exposure and outcome information were handled. The relationship between the listed enrollment and the group-specific numbers at risk is not fully detailed.

Without information on missing observations, eligibility for each outcome, or analysis-population definitions, readers cannot assess whether all relevant participants contributed to each outcome or how incomplete follow-up might affect the reported counts. These gaps limit interpretation of the posted results and make it difficult to assess whether analyses followed a prespecified plan.

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: diarrhea (12.5%), death nos (12.5%), gastric perforation (12.5%), disease progression (12.5%).

The posted figures list, for each dose group, numbers at risk and numbers affected by serious events and by other events. For 0 mg, the record lists 8 at risk and 2 affected for serious events, and 8 at risk and 8 affected for other events. For 10 mg and 15 mg, it lists 2 at risk and 0 affected for serious events in each group, and 2 at risk and 2 affected for other events in each group. These are descriptive counts, not randomized comparisons.

The serious-event terms listed are Diarrhea, Death NOS, Gastric perforation, and Disease progression, each reported as 12.5%. The registry does not provide enough detail to determine how those term-level percentages relate to the dose-specific counts or whether terms overlap within participants. Do not add the term percentages or treat them as a count of unique people.

Seriousness is not the same as severity grade, and the record does not state that the listed serious events were caused by ruxolitinib. The primary endpoint concerns grade 3 or higher toxicity, while the posted safety figures use serious and other event categories. Without a reported connection between these classifications, the figures cannot be read as a direct result for the primary endpoint.

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

The registry record documents a pilot study intended to assess toxicity with ruxolitinib in non-small cell lung cancer cachexia. It describes a 3-month primary endpoint based on grade 3 or higher events measured using NCI CTCAE v5.0 criteria. It also lists group-specific counts of serious and other adverse events. These counts provide descriptive information about the participants represented in the registry’s safety data.

The record does not report a separate numerical result for the primary endpoint as defined. Serious-event counts do not automatically answer how many participants experienced grade 3 or higher toxicity, because seriousness and severity grade are distinct classifications. Nor do the posted counts establish whether events were related to ruxolitinib. The registry also does not provide an uncertainty interval for these counts.

The design further limits what can be concluded. It is a single-group, unmasked study with no randomized control group. The 0 mg group is described as baseline status and was not assessed for outcomes, so comparisons with the 10 mg or 15 mg groups cannot be treated as controlled treatment effects. The sequential dosing regimen also means that dose and time are not separated through random assignment.

The registry’s secondary outcomes include objective response, quality of life, suppression of body weight loss, and preliminary evidence relating to overall survival. Their presence as listed outcomes does not establish that results were observed or that treatment caused a change. The record lists 0 analyses, and results for those secondary questions are not reported in the registry record in the registry record for this analysis.

General: small descriptive studies can help characterize what was observed in a particular study setting, while leaving considerable uncertainty about how well observations generalize or whether an intervention caused them. The posted counts should therefore be interpreted as limited safety descriptions, not as proof that ruxolitinib is beneficial, harmful, or safe for other patients.

13. Why This Trial Matters Statistically

This study raises a clinically relevant statistical question: how to describe toxicity when an early-phase pilot evaluates a sequential regimen in a small group. The primary endpoint is framed as a participant count of grade 3 or higher toxicity over 3 months. That definition is meaningful only if the reported result can be linked clearly to the severity threshold and to the participants included in the analysis.

The record illustrates why outcome labels matter. Serious adverse events, other adverse events, and grade 3 or higher events are not interchangeable categories. Reporting each category with a clear definition and an explicit relationship to the primary endpoint helps readers understand what a count actually represents. In this record, the safety figures are descriptive, but their connection to the primary endpoint is not reported.

The study also highlights the limits of reading sequential dose groups as if they were parallel treatment arms. A single-group regimen that moves from baseline to higher doses does not create a randomized control comparison. General: design, timing, exposure, and participant progression all affect whether a contrast can be interpreted causally. The registry’s description of the 0 mg group as baseline status reinforces the need not to treat it as an outcome-assessed control.

Finally, the record lists several secondary outcome questions but no analyses. Clear reporting of analysis populations, missing-data handling, uncertainty, and multiplicity would help readers judge the precision and scope of findings. The statistical importance of this pilot is therefore not a definitive estimate, but the need to distinguish planned questions, observed counts, and conclusions that the design can 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 registry record leaves important details about participant flow unclear. It lists enrollment as 10, while the primary outcome description says 2 participants received at least one dose of 10 mg twice daily and proceeded to at least one dose of 15 mg twice daily. The record does not explain the full disposition of participants across the dosing regimen or reconcile the enrollment figure with the dose-specific numbers at risk.

The 0 mg group is described as baseline status and not assessed for outcomes, yet the safety figures include 0 mg counts. The record does not explain this apparent difference in presentation. In any case, the registry identifies no randomized control comparison, and the baseline group should not be interpreted as a concurrent untreated control.

The primary endpoint specifies grade 3 or higher toxicity, but the posted safety counts are separated into serious and other events. The registry record does not report the mapping between these classifications, a separate primary endpoint result, or whether events were judged related to ruxolitinib. It also does not report uncertainty intervals for the counts or sufficient detail to interpret the term-level serious-event percentages.

Outcome methods are limited in the registry record. A statistical analysis plan, missing-data procedures, analysis-population definitions, and multiplicity methods are not reported in the registry record. The record lists 9 outcome measures and 0 analyses, so the available information does not establish how the listed secondary questions were assessed or whether results were intended to be adjusted for multiple testing.

The sequential dosing structure and small numbers reported at active doses further limit comparisons. Dose and time are not separated by randomization, and the registry does not report an analysis addressing this. These issues constrain conclusions about treatment effects and the precision or generalizability of the safety observations.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Ruxolitinib for Cancer Cachexia — NCT04906746.
• Registry record last updated 2026-07-07; status last verified 2026-05.
• Results first posted 2026-07-07.
• 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 record describes a single-group, unmasked, early-phase pilot study of ruxolitinib for cachexia in stage IV non-small cell lung cancer. Enrollment is listed as 10. The regimen lists 0 mg twice daily in Month 1, 10 mg twice daily in Month 2, and 15 mg twice daily in Month 3. The study is marked active, not recruiting.

The primary outcome is a participant count of toxicity over 3 months, with grade 3 or higher events recorded using NCI CTCAE v5.0 criteria. The registry also lists serious and other adverse-event counts by dose group, but does not report a separate result matching the primary endpoint definition. It lists 9 outcome measures and 0 analyses. A randomized comparison, analysis plan, missing-data methods, and a direct connection between serious events and the primary toxicity endpoint are not reported in the registry record.