Study of 4-Demethyl-4-cholesteryloxycarbonylpenclome (DM-CHOC-PEN) in Patients With Brain Tumors: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of 4-Demethyl-4-cholesteryloxycarbonylpenclome (DM-CHOC-PEN) in Patients With Brain Tumors (NCT02038218), a na phase 2 study. The primary outcome is Tumor Diameter.

2. Clinical Question

The registry describes a Phase II study of intravenous DM-CHOC-PEN in patients with primary brain tumors or cancer that had spread to the brain. Its primary outcome was mean tumor diameter measured from patient scans after treatment, with follow-up described as continuing until remission or stopping treatment because of progression.

The statistical question is whether the posted results quantify tumor diameter after treatment, and how precisely they estimate it. The record names the measure and its unit, centimeters, but does not report a mean, standard deviation, confidence interval, or other numerical tumor-diameter result. It therefore does not permit a quantitative assessment of the size or precision of the reported tumor-diameter outcome.

The study is listed as a single-group trial with no masking and no randomized allocation. The record also describes two treatment cohorts, differentiated by liver involvement or history of disease, and states that different doses were planned for the cohorts. That structure does not establish a randomized comparison between the cohorts. Their results, even if reported, would not by themselves provide a like-for-like estimate of treatment effect.

3. Trial Design

NCT02038218 is listed as a Phase II trial sponsored by DEKK-TEC, Inc. The record gives the study title as a study of DM-CHOC-PEN in patients with brain tumors and describes its official purpose as evaluating safety and tolerance in patients with malignancies involving the central nervous system. The listed conditions include primary brain tumors and metastatic malignant neoplasm to the brain.

The study model is single group, allocation is listed as not applicable, and masking is listed as none. Enrollment is recorded as 29. The record lists a start date of 2013-09, primary completion in 2016-05, and status as completed. These are registry characteristics; they do not establish how many enrolled participants contributed data to any particular outcome.

The intervention is a drug named DM-CHOC-PEN. The arm description says that patients were to receive an outpatient infusion every 21 days and be divided into two cohorts. Cohort 1 is described as including patients with liver involvement or a history of disease and receiving a dose of 85.8 mg/m2. The description of Cohort 2 is truncated in the registry record, so its complete eligibility and dosing details are not available. The record says that the cohorts were to receive different doses, but it does not report a randomized allocation between them.

The stated primary outcome is mean tumor diameter from patient scans, measured in centimeters. Its timeframe is described as continuing until remission or treatment discontinuation due to progression. The registry names the mean and standard deviation as the outcome summary, but numerical outcome values and an analysis count are not reported. The record therefore supports a description of the intended design and measure, not an estimate of treatment effect.

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: Patients with cancer involving the CNS were treated with intravenous DM-CHOC-PEN. The drug is metabolized by the liver. Abnormal neurological and hepatic values excluded the patients from treatments. If the patients were on steroids, this also excluded treatments. Steroids initiate the break down of DM-CHOC-PEN.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Mean Tumor size after Treatment

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: Patients with liver complications lower dose

6. Primary Endpoint

The registry lists the primary outcome as Tumor Diameter, measured over Until remission or off treatment due to progression.

Tumor diameter is a continuous measure of lesion size. The registry defines the primary outcome as mean tumor diameter after treatment, in centimeters, based on patient scans. It identifies the standard deviation as the measure of dispersion and gives the timeframe as until remission or stopping treatment because of progression.

As a general statistical rationale, a size measure can describe the extent of disease at an assessment and can be compared across assessments when measurement timing and methods are clearly defined. A mean gives a summary of the observed measurements, while the standard deviation describes how spread out individual measurements are around that mean. Neither summary, alone, describes every participant’s course or establishes that a change was caused by treatment.

For interpretation, the measurement schedule, rules for selecting and measuring lesions, and handling of participants without a scan at the relevant time are important. Those details are not reported in the registry record. The registry also does not report the numerical mean or standard deviation, a change from baseline, or an uncertainty interval for tumor diameter. Without those results, the outcome’s magnitude, variability, and precision cannot be evaluated from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The record names the primary measure as mean tumor diameter and the dispersion measure as standard deviation. It does not report an inferential method, a confidence interval, a p-value, or a numerical tumor-diameter result. The following concepts explain how such summaries are generally interpreted; they are not a description of an analysis confirmed for this study.

What does a mean describe?

As a general statistical concept, a mean is an arithmetic summary of measured values. For a continuous outcome such as diameter, it can give a compact account of the group’s measured tumor sizes at a defined assessment. The mean is sensitive to extreme values, so its interpretation benefits from information about the distribution of observations and the measurement process. A mean alone does not show whether most observations are close to it or whether some are much larger or smaller.

The registry does not clarify whether the reported endpoint was intended to be a measurement at a particular scheduled visit, a value selected from multiple scans, or a summary over follow-up. Its timeframe refers to remission or stopping treatment due to progression, but detailed timing and rules for selecting measurements are not reported in the registry record. Those details would affect what the mean represents.

What does the standard deviation add?

In general, the standard deviation describes the spread of observed values around their mean. It is a measure of variation among observations, not a measure of how precisely the mean has been estimated. A small or large standard deviation should be interpreted in the context of the measurement scale, population, and assessment procedures.

Precision of a mean is more directly described by a confidence interval or another uncertainty measure. In general, a confidence interval represents a range of estimates compatible with the data and the method’s assumptions. Its width depends on the amount of information and the variability in the observations, among other factors. No confidence interval for tumor diameter is reported in the registry record, and none can be recovered from the enrollment figure alone.

What would a comparison require?

As a general principle, estimating a treatment effect requires a clearly specified contrast. A randomized comparator can help separate treatment differences from differences in prognosis, eligibility, follow-up, and other factors. Here the registry lists a single-group model and no randomized allocation. It describes two cohorts associated with liver involvement or history and different dosing, but does not report a randomized comparison between them. A difference between non-randomized cohorts would not automatically represent a treatment effect.

For a single-group outcome, a post-treatment mean can describe the measurements in the observed group. To interpret change, the analysis would need a defined baseline, a defined follow-up assessment, and rules for linking measurements within participants. To interpret a causal effect, a suitable comparison would be needed. The registry does not report a baseline tumor-diameter value, a change estimate, or a comparator analysis.

What is not known about the analysis?

The registry record lists no analyses for the outcome. The statistical plan, model specification, confidence-interval method, assumptions, and handling of repeated or incomplete measurements are not reported in the registry record. It is also not reported whether assessments were reviewed without knowledge of cohort or treatment status. Masking is listed as none for the study, but that does not specify how scans were measured or reviewed.

General statistical practice would distinguish the enrolled group from the participants included in an endpoint analysis, and would describe missing observations and the assumptions used to handle them. It would also identify whether the endpoint was analyzed as an absolute measurement, a change, or another summary. Those analysis details are unavailable. The listed mean and standard deviation identify the planned type of summary, but do not establish the exact analysis performed.

The registry’s sample-size assumptions describe a planning illustration based on an observed effect, with two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. The record characterizes this as an illustration rather than the trial’s own design. Such planning calculations are not observed power and do not establish the statistical plan used for this single-group trial. No calculated planning values are reported in the information available for this analysis.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("DM-CHOC-PEN - Arm 1", "DM-CHOC-PEN - Arm 2")
n   <- c(22, 7)
m   <- c(2.7, 4.4)
sp  <- c(1.1, 0.8)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  DM-CHOC-PEN - Arm 1  22  2.700  1.100  2.212  3.188
CARM  DM-CHOC-PEN - Arm 2  7  4.400  0.800  3.660  5.140

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures, but detailed results and analysis information for the primary endpoint are not reported in the registry record. The record does not describe a prespecified hierarchy of outcomes, adjustment for multiple comparisons, or a plan for interpreting multiple tests. Multiplicity procedures are general methods for controlling the risk of misleading findings when several hypotheses are tested; whether they were relevant to the investigators’ analysis cannot be determined.

Missing-data handling is also not reported in the registry record. The description gives a follow-up timeframe ending at remission or stopping treatment because of progression, but does not explain how tumor-diameter measurements were handled when scans were absent, measurements could not be made, or participants stopped treatment. The registry does not state how many participants contributed data to the primary outcome. Enrollment is recorded as 29, but that figure does not identify the analysis population for tumor diameter.

The analysis population is not specified. The record does not say whether the primary outcome was analyzed among all enrolled participants, only those who received treatment, or those with evaluable scans. It also does not report sensitivity analyses for missing measurements or assumptions about why data might be unavailable. As general statistical principles, the analysis population and missing-data approach can affect both the estimate and its uncertainty; their absence limits appraisal of the endpoint result.

12. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry lists serious adverse events for ARM 1 among 22 participants at risk, with 0 affected. For ARM 2, it lists 0 serious adverse events among 7 participants at risk. It also records other adverse events in 3 of 22 participants at risk in ARM 1 and 1 of 7 in ARM 2. These are registry counts, not estimates of comparative risk.

The death counts are listed as 19 in ARM 1 and 7 in ARM 2. The record does not report whether deaths were attributed to treatment, the underlying disease, or another cause. It also does not provide timing, event descriptions, or a narrative explaining the relationship between deaths, adverse events, and treatment. A recorded death count should therefore be read separately from the serious-adverse-event counts, not assumed to be a count of treatment-related events.

The arm denominators are the numbers listed as at risk for these safety categories; they should not be treated as the number enrolled in the study or the number analyzed for tumor diameter. The registry lists no top serious adverse-event terms. These figures describe what was entered in the safety record, while leaving event attribution and context unreported.

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

The registry record establishes that NCT02038218 was listed as a completed Phase II study with a stated primary outcome of mean tumor diameter after treatment. It identifies the measurement source as patient scans, the unit as centimeters, and the stated follow-up endpoint as remission or stopping treatment because of progression. It also reports safety counts for ARM 1 and ARM 2.

It does not report numerical tumor-diameter results, a standard deviation, an uncertainty interval, or an inferential analysis of the primary outcome. Therefore the record cannot quantify the observed tumor-size outcome or its precision. It also cannot establish how much tumor diameter changed from baseline, because a baseline value and a change estimate are not reported.

The design information does not establish a randomized treatment comparison. The model is listed as single group, and allocation as not applicable. The two described cohorts are associated with liver involvement or history and different dosing, rather than a reported randomized control condition. A reader should not interpret any potential difference between those cohorts as a causal treatment effect based on the information in the registry record.

The safety counts likewise do not establish that DM-CHOC-PEN caused or prevented an event. The record does not report event attribution, detailed event narratives, or a comparison designed to estimate causal safety differences. The listed deaths require particular caution: the counts are reported, but their causes and relationship to treatment are not.

In general, a registry record can document a trial’s design and selected results, but the strength of an interpretation depends on the completeness of outcome reporting and analysis details. In this case, the record supports a description of the trial and its posted safety counts. It does not support a numerical conclusion about the primary tumor-diameter outcome or a causal conclusion about treatment benefit or harm.

14. Why This Trial Matters Statistically

This study is statistically informative as an example of the difference between a named endpoint and a reported endpoint result. The registry specifies a continuous primary measure, its unit, and summary statistics intended for it. Yet the numerical mean, standard deviation, and uncertainty around the estimate are not reported in the registry record. Without those values, readers cannot assess the observed tumor-diameter distribution or the precision of its summary.

The design also matters for interpretation. A single-group study can describe outcomes in the treated population, but it does not by itself provide a randomized counterfactual. This record describes two cohorts with different liver-involvement characteristics and intended doses; it does not identify a randomized comparison. Recognizing that distinction prevents a descriptive cohort contrast from being mistaken for a treatment effect.

Safety reporting raises a separate interpretive issue. The registry records no serious adverse events among participants listed at risk in either arm, other adverse-event counts, and deaths. These categories have different meanings, and the record does not attribute deaths or events to treatment. Clear separation of counts, denominators, and causal attribution is essential when reading safety summaries.

More complete reporting of endpoint values, analysis populations, missing-data handling, measurement timing, and analysis methods would allow readers to assess both magnitude and uncertainty. Those details are not reported in the registry record. The study therefore illustrates why statistical interpretation depends not only on the planned outcome, but also on transparent reporting of the data and methods used to analyze it.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The primary limitation for interpreting this trial from the registry record is that its numerical tumor-diameter results are not reported. Although the outcome is identified as a mean with standard deviation, neither statistic is available, and no confidence interval or test result is listed. The record consequently does not show the magnitude or precision of the primary outcome.

The analysis population for tumor diameter is not reported. Enrollment is listed as 29, but the record does not state how many participants had evaluable scans or contributed to the endpoint analysis. It also does not report the number or timing of scans, the rules used to select or measure lesions, or how missing assessments were handled. These omissions prevent assessment of possible selection and measurement effects.

The cohort description is incomplete: the description of Cohort 2 is truncated. Although the record says the cohorts were to receive different doses, it does not report the full dosing information or a randomized comparison. The trial is listed as single group, so the record does not support a controlled estimate of treatment effect.

Safety interpretation is limited by the lack of event details and attribution. The record lists adverse-event counts and deaths but does not report their causes, timing, or relationship to treatment. It also does not provide a narrative of serious events. These limitations apply to conclusions from the registry record and do not establish what may have been available in other study materials.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study of 4-Demethyl-4-cholesteryloxycarbonylpenclome (DM-CHOC-PEN) in Patients With Brain Tumors — NCT02038218.
• Registry record last updated 2020-09-16; status last verified 2020-09.
• Results first posted 2020-09-16.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

NCT02038218 is listed as a completed Phase II, single-group study of intravenous DM-CHOC-PEN in patients with malignancies involving the central nervous system. Enrollment is recorded as 29. The registry lists tumor diameter, measured from patient scans in centimeters, as the primary outcome, with mean and standard deviation as the summaries and follow-up until remission or treatment discontinuation due to progression.

Numerical tumor-diameter results and an analysis of that outcome are not reported in the registry record. The safety record lists 0 serious adverse events among 22 participants at risk in ARM 1 and among 7 in ARM 2, other adverse events in 3 and 1, respectively, and deaths of 19 and 7, respectively. The record does not report attribution for the events or deaths.