Vitamin D Supplementation in Women With DCIS and/or LCIS: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Vitamin D Supplementation in Women With DCIS and/or LCIS (NCT02936999), a na early/phase 1 study. The primary outcome is Ki 67 Measured.

2. Clinical Question

This is an exploratory pilot question, not a randomized comparison of treatment against an untreated or alternative-treatment group. A before-and-after comparison can describe whether measurements differ over time among participants with measurements at both visits. By itself, it cannot separate a treatment-associated change from other changes that occur over the same period. In this record, even that within-person description is unavailable because no primary outcome data were collected.

3. Trial Design

The registry describes an exploratory pilot study of vitamin D supplementation in women with DCIS and/or LCIS. It lists the phase as EARLY PHASE1, the model as single group, allocation as not applicable, and masking as none. These design features indicate that the registry does not describe a randomized, masked comparison with a separate control arm. The listed intervention is cholecalciferol, and the arm is named Vitamin D3.

The arm description specifies that participants would be dispensed a bottle containing 32 capsules, with 1 capsule containing 4000 IU to be taken by mouth once daily. The instructions specify taking the capsule with water for 29 days. These are the planned directions in the registry; whether any participant began or completed the regimen is not reported in the registry record.

The planned primary measurement was Ki67 at baseline and after treatment, with the post-treatment time point scheduled for 28 days +/- 3 days from Day 1. The registry describes the post-treatment measurement as surgical. It lists secondary outcomes concerning LC3B and ATG7 proteins in the autophagy pathway, PMCA2 calcium transport proteins, and HER2 localization. Results for these outcomes are not reported in the registry record.

The record lists enrollment as 8, while also stating that the study was terminated because of low enrollment and lack of funding and that no primary outcome data were collected. The enrollment entry should not be read as evidence that 8 participants contributed outcome measurements. The registry status is TERMINATED. The start is listed as 2016-08 and primary completion as 2019-01; further detail about the timeline or reasons for termination beyond low enrollment and lack of funding is not reported in the registry record.

The registry does not report a separate comparator, allocation procedure, masking procedure, or detailed recruitment and follow-up procedures. The planned analysis description names a paired t-test or, when appropriate, the Wilcoxon signed-rank test. It does not establish that either analysis was conducted; the record states that no data were collected.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Analysis population for the primary outcome, as defined in the registry: The study was terminated due to low enrollment and lack of funding; therefore no data were collected.

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 Ki 67 Measured, measured over 28 days +/- 3 days from Day 1 of treatment.

The primary endpoint is Ki67 measured at baseline and after vitamin D supplementation. Ki67 is described in the registry as a proliferation index. In general, a proliferation measure characterizes cell activity related to growth; measuring it at two time points can describe whether the measure changes within the same participants over the study interval. The record does not provide a definition of the assay, specimen processing, scoring method, or threshold for interpreting Ki67.

The registry planned a baseline measurement before supplementation and a surgical measurement after treatment. The stated post-treatment timeframe is 28 days +/- 3 days from Day 1. The plan was to describe the baseline and post-treatment values and compare participants’ outcomes between those assessments. A paired analysis is suited to repeated measurements because it evaluates within-person differences rather than treating the two measurements as unrelated groups.

Ki67 was selected as the primary endpoint in a study whose stated purpose included examining the effect of vitamin D supplementation on the behavior of breast cancer cells. The registry also describes interest in the development of invasive breast cancer disease, but it does not identify that as the primary endpoint. No Ki67 values, participant-level paired observations, summary estimates, or test results are reported in the registry record. The registry states that the study was terminated because of low enrollment and lack of funding and that no data were collected. Consequently, the endpoint has no reported result to interpret.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry’s primary analysis description refers to two measurements from the same participants: Ki67 at baseline and after supplementation. General statistical explanation: measurements from one person tend to be related, so a paired analysis uses the within-person relationship rather than treating each time point as an independent sample. The analysis would ordinarily require participants with measurements at both time points. The registry does not report how many participants, if any, had paired measurements; it states that no data were collected.

What a paired t-test would assess

General statistical explanation: a paired t-test evaluates the average within-person difference between two measurements. It works on the differences for each participant, not on two unrelated groups. Its usual model-based inference relies on the differences being reasonably compatible with the test’s distributional assumptions. The registry names this test as one option but does not report a test statistic, estimate, confidence interval, p-value, or assumption checks. Because the record says no data were collected, the named test should be read as planned analysis language, not a completed analysis.

What the Wilcoxon signed-rank test would assess

General statistical explanation: the Wilcoxon signed-rank test is a nonparametric approach to paired measurements. It uses the ranks and signs of within-person differences and can be considered when a paired t-test is not appropriate. It does not remove the need for paired observations, and it does not create information when measurements are missing. The registry says this test would be used when appropriate but does not specify the decision criteria for choosing between the two methods. A more detailed test-selection rule is not reported in the registry record.

What the paired comparison could and could not show

General statistical explanation: a before-and-after analysis estimates a change over time among people with measurements at both time points. It does not, on its own, show that the intervention caused the change. Other time-related influences cannot be separated from the intervention without a suitable comparison design. Here, the registry describes a single group and no masking, with no separate comparator arm. That design context would constrain causal interpretation even if results had been available.

The primary endpoint was planned for a surgical measurement 28 days +/- 3 days from Day 1. The registry does not report whether the scheduled time point was achieved, whether measurements were obtained from the same type of specimen at both assessments, or how variability in timing would be handled. It also does not report the measurement scale, assay details, or a clinically meaningful threshold for Ki67. These omissions prevent a fuller assessment of measurement and analysis procedures.

Estimates and uncertainty

General statistical explanation: an effect estimate describes the magnitude and direction of a measured difference, while a confidence interval describes uncertainty around that estimate under the statistical model. A p-value, when reported, summarizes how compatible the observed data are with a specified null model; it does not measure clinical importance. No effect estimate, confidence interval, or p-value is reported for the primary endpoint in the registry record. Since the record states that no data were collected, there is no observed primary result whose precision or statistical compatibility can be evaluated.

The registry lists zero analyses. This is consistent with the absence of reported primary results, but the record does not give further information about analysis activity outside the posted results. A written plan to use a test does not demonstrate that the test was performed. Likewise, the registry’s enrollment entry does not establish the number with outcome data or the size of any analyzable sample.

Planning illustrations are not observed power

General statistical explanation: power calculations are planning tools that relate a target effect, a statistical threshold, a sample size, and assumptions about the data. They do not measure the chance that a completed study has produced a true finding. The planning assumptions associated with the posted illustrations describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects defined as fractions of an observed effect. The record describes this as a planning illustration, not the trial’s own design.

Those assumptions do not match the registry’s description of a single-group study with no separate comparator. Further, the registry states that no primary data were collected, so an observed effect from this trial is not available in the record. Any such illustration cannot be read as a result from this study or as evidence of observed power. The registry does not report the trial’s sample-size justification, and no such rationale should be inferred from a planning illustration.

Over-dispersion is a general statistical concern when variation in observed data exceeds what a model assumes; if present, it can affect standard errors and uncertainty estimates. The registry does not report a count model, event-rate outcome, or over-dispersion assessment for the primary Ki67 analysis. It would therefore be inappropriate to assert that over-dispersion was present or that any adjustment was used.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists one primary outcome, Ki67 measured at baseline and after treatment, and secondary outcomes involving LC3B, ATG7, PMCA2, and HER2 localization. Multiple outcomes can raise a multiplicity issue because conducting several statistical tests may increase the chance of at least one apparently notable result under a set of null hypotheses. General statistical explanation: approaches to multiplicity include defining a hierarchy of outcomes or adjusting inference across tests. The registry does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or a detailed strategy for interpreting multiple tests.

The record lists zero analyses and states that no data were collected. No primary or secondary outcome estimates or test results are reported in the registry record. Therefore, there are no posted outcome tests for which multiplicity adjustment can be assessed, and absence of reported tests should not be mistaken for a finding of no effect.

Missing-data handling is not reported in the registry record. In particular, it does not describe procedures for incomplete baseline or surgical measurements, participants who did not complete supplementation, or participants without a measurement at either time point. Since the primary analysis was planned as paired, a participant would generally need both measurements to contribute a paired difference; the registry does not report whether any such pairs existed. The statement that no data were collected means the record offers no basis to assess missingness patterns or the consequences of incomplete observations.

The analysis population is also not described in detail. The registry lists enrollment as 8 and includes 8 at risk in its safety summary, but does not report a number with primary endpoint data or define an analysis set. The listed counts should not be treated as an efficacy analysis population. Further details about intention-to-treat, per-protocol, or other analysis populations are not reported in the registry record.

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.

The registry safety summary lists 8 people at risk in the treatment group, 0 with serious adverse events, 0 with other adverse events, and 0 deaths. These are the posted counts and should be read as a description of the safety entries in the registry, not as proof that the intervention is free of risk. The record does not list serious adverse event terms.

The registry separately states that the study was terminated because of low enrollment and lack of funding and that no data were collected for the primary endpoint. The safety counts do not resolve whether all people listed as at risk received the intervention, how adverse events were collected, how long participants were monitored, or how completeness of safety follow-up was assessed. These details are not reported in the registry record.

Zero events in a registry summary means that no events are listed in that category; it does not mean that the probability of an event is known to be zero. The record supplies no uncertainty interval or detailed safety analysis. The count at risk also should not be treated as the number with an evaluable primary outcome. The safety information is therefore limited to the categories and counts posted, with important details about exposure and ascertainment not reported.

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

The central meaning of this record is that it describes a planned exploratory study but does not provide primary outcome data. The registry states that the study was terminated because of low enrollment and lack of funding, and that no data were collected. As a result, there is no reported Ki67 change to interpret and no estimate of the effect of supplementation on the primary endpoint.

The record does not show that Ki67 changed, and it does not show that Ki67 remained unchanged. Both conclusions would require outcome measurements that are not reported. The same distinction applies to the listed secondary outcomes: their presence in the registry describes planned measurements, not completed findings. The record does not report results for LC3B, ATG7, PMCA2, or HER2 localization.

The study’s single-group, unmasked design also limits what a before-and-after comparison could establish, even if paired results were available. In general, a temporal change in one group cannot by itself isolate an intervention effect from other changes over time. There was no separate comparator group in the registry’s design description. This is a design limitation, distinct from the more immediate absence of outcome data.

The safety summary lists 0 serious adverse events, 0 other adverse events, and 0 deaths among 8 at risk. These counts describe what is entered in the registry; they do not establish a complete account of exposure or follow-up, which is not reported. They also do not supply evidence about the primary endpoint.

Accordingly, the record should be read as an incomplete source of evidence about the study question, not as a positive or negative efficacy result. It documents the intended intervention, measurement plan, and reason for termination, while leaving the primary and secondary outcome questions unanswered. No clinical conclusion about benefit or lack of benefit can be drawn from the absent outcome data.

13. Why This Trial Matters Statistically

This study matters statistically because its record illustrates the difference between a planned analysis and an observed result. The registry describes a paired comparison of Ki67 before and after treatment and names a paired t-test or Wilcoxon signed-rank test. Those methods explain how data might have been analyzed; they do not establish that a comparison was carried out. The record states that no data were collected and lists zero analyses.

It also illustrates why an enrollment entry should not be confused with an analyzable outcome sample. The registry lists enrollment as 8, and its safety summary lists 8 at risk, yet no primary endpoint data are reported. A planned enrollment, an at-risk count, and a count with paired measurements answer different questions. The record does not specify how many participants had both Ki67 assessments; it states that no data were collected.

The design is statistically important as well. A single-group before-and-after comparison can describe within-person change, but it does not provide a concurrent comparator for separating treatment-associated change from other influences over time. The registry has no randomized comparison or masking. These features would matter when interpreting an estimate, if one existed.

Finally, planning calculations should be kept separate from empirical results. The posted planning assumptions describe a hypothetical two-arm, equal-allocation framework, whereas the registered study is single group. The record does not provide trial outcome data from which to interpret an observed effect. Recognizing these distinctions helps prevent planning illustrations, design descriptions, and safety counts from being mistaken for evidence about the primary clinical question.

14. Limitations

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

The most consequential limitation is the absence of primary outcome data. The registry states that no data were collected after termination for low enrollment and lack of funding. Consequently, it is not possible to estimate a Ki67 change, assess its uncertainty, or evaluate the planned paired analysis. Results for the listed secondary outcomes are also not reported in the registry record.

The record lists enrollment as 8 but does not establish that this number corresponds to participants who received treatment, completed the regimen, or had paired Ki67 measurements. It does not reconcile that entry with the statement that no data were collected. The safety summary lists 8 at risk, but the relationship between that count and treatment exposure or outcome collection is not reported.

The study was single group, unmasked, and without a separate comparator. Even with outcome data, that design would constrain causal interpretation of changes over time. The registry does not provide detailed eligibility criteria, baseline characteristics, measurement procedures, assay information, adherence data, follow-up completeness, or missing-data methods. Further detail on these matters is not reported in the registry record.

The registry names two possible paired tests but does not report how the choice between them would be made, whether assumptions would be checked, or how secondary-outcome multiplicity would be handled. It does not report a sample-size justification. The planning assumptions describe two-arm equal allocation, which does not match the registered single-group model; any planning illustration should therefore be kept separate from the study’s design and from observed evidence.

Finally, the safety counts are limited to posted event categories and totals. Details about adverse-event ascertainment, exposure, monitoring duration, and completeness are not reported. These gaps restrict interpretation of both efficacy and safety, beyond the central fact that the primary outcome data are absent.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Vitamin D Supplementation in Women With DCIS and/or LCIS — NCT02936999.
• Registry record last updated 2020-12-16; status last verified 2020-10.
• Results first posted 2020-12-16.
• 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.

18. Record Summary

The registry describes an exploratory pilot study of oral cholecalciferol in women with DCIS and/or LCIS. It lists an EARLY PHASE1 designation, a single-group model, no masking, and enrollment of 8. The intervention directions specify 1 capsule containing 4000 IU once daily for 29 days. The primary outcome was Ki67 at baseline and at a surgical assessment scheduled for 28 days +/- 3 days from Day 1.

The registry lists secondary outcomes involving LC3B, ATG7, PMCA2, and HER2 localization. It names a paired t-test or Wilcoxon signed-rank test for the primary comparison, but reports zero analyses. The study status is TERMINATED; the stated reason is low enrollment and lack of funding. The primary outcome description says no data were collected.

The safety summary lists 8 at risk, 0 serious adverse events, 0 other adverse events, and 0 deaths. The record does not report outcome estimates, test results, or detailed information on treatment receipt and follow-up. Therefore, the registry documents the intended design and reason for termination but does not answer the primary statistical question.