Clinical Study of Noni Extract in Men With Very Low Risk or Low Risk Prostate Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Clinical Study of Noni Extract in Men With Very Low Risk or Low Risk Prostate Cancer (NCT02648919), a na phase 2 study. The primary outcome is Compare Genomic Prostate Score (GPS) in Prostatic Tumors.

2. Clinical Question

In statistical terms, the study’s intended question was whether GPS measurements changed during follow-up in participants receiving Noni extract. Without a comparison group, any observed within-group change would not by itself distinguish an intervention-associated change from other explanations, such as variation over time or measurement. The registry record does not report primary outcome values or an estimated change, so the planned question cannot be answered from the posted results.

3. Trial Design

This was a Phase II clinical study sponsored by the University of Hawaii. The registry lists a start date of 2015-12, a primary completion date of 2018-12, and a status of terminated. The recorded enrollment was 6. These details describe the study record; they do not establish why the study ended or whether all enrolled participants completed the planned follow-up. A reason for termination and participant-level follow-up details are not reported in the registry record.

The design is listed as single group, with allocation NA and masking none. Participants were assigned to the Noni extract regimen described as 6,000 mg/day, taken as 4 capsules with breakfast, 4 with lunch, and 4 with dinner. No control arm or randomized allocation is recorded. This means the design can describe observations within the treated group, but it cannot make a randomized comparison between Noni extract and another condition.

The primary outcome was change in GPS from screening and at 12 months or early termination. Secondary outcomes concerned serum prostate specific antigen (PSA), adverse-event frequency, and exploratory prostate-tissue molecular pathways, including cell proliferation, apoptosis, and angiogenesis. The registry record lists 6 outcome measures and 0 analyses. The absence of analyses in the record means statistical estimates and tests are not available in the posted results; it does not establish whether analyses were conducted outside the record.

The planned follow-up and the posted outcome information do not align completely: the record explains that only baseline GPS data were available. It does not give a reason for termination, a detailed analysis plan, a sample-size justification, or further information about participant flow. Those aspects 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.

Overall Study

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 Compare Genomic Prostate Score (GPS) in Prostatic Tumors, measured over Change from screening and at 12 months or early termination.

The primary endpoint was change in the Genomic Prostate Score in prostatic tumors, with measurements planned from screening to 12 months or early termination. GPS is described as a score from the Oncotype DX assay, which measures gene expression using a clinically validated 17-gene genomic assay. Its scale runs from 0-100, and a higher score means a higher risk of disease.

In general, a genomic score can summarize information about tumor gene expression in a form that can be compared across measurements. A change endpoint is intended to describe how that score varies over the specified follow-up period. It is important to distinguish the planned endpoint from the data that were available: the registry record says the assay could not be run on 12-month prostate biopsy samples in which active cancer was not identified, so only baseline data were available.

The record does not give baseline GPS values, follow-up values, or an estimated change. It therefore does not report the magnitude or direction of any change in the primary measure. It also does not report a statistical test or confidence interval for this endpoint. The rationale for using GPS is described in the record, but the available baseline-only information cannot establish a within-person change over follow-up.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the single-group design allow?

The study is recorded as a single-group study, with allocation listed as NA and masking listed as none. Participants received Noni extract at 6,000 mg/day; there was no recorded comparator arm. In general, a single-group design can describe outcomes observed among participants receiving the study intervention. It does not provide a randomized contrast with an untreated group or another intervention, so it cannot isolate the intervention’s contribution to changes observed over time.

This distinction matters even for a measure such as change from baseline. A within-group change is a comparison of measurements over time in the same study group, not a treatment-versus-control effect. Changes could reflect more than one process, and a single-group design does not supply a concurrent randomized comparison to separate those processes. The registry record does not report a primary change estimate, so no numerical within-group result can be interpreted.

How should the GPS endpoint be understood?

The primary measure was change in GPS from screening and at 12 months or early termination. The registry describes GPS as a 17-gene genomic assay score on a 0-100 scale, with a higher score indicating higher disease risk. In general, an endpoint defined as change requires measurements at the specified time points and a stated approach to comparing them. The record says only baseline data were available because the assay could not be run on 12-month biopsy samples in which active cancer was not identified. It reports no change estimate or endpoint analysis.

The intended endpoint’s timeframe includes early termination, but the record does not explain whether or how measurements at early termination were collected or analyzed. It does not report participant-level values, a summary of baseline GPS, or an analysis population for the endpoint. Those details are not reported in the registry record and should not be inferred from the planned timeframe.

What does the analysis record say?

The record lists 6 outcome measures and 0 analyses. No statistical model, test, adjustment method, or estimate of uncertainty is reported. In general, an analysis plan specifies how outcome measurements will be converted into estimates and how uncertainty will be assessed. Without those details or posted analyses, there is no basis for describing the primary result as statistically significant, nonsignificant, precise, or imprecise.

In general, a confidence interval communicates uncertainty around an estimate, while a hypothesis test assesses compatibility with a specified null hypothesis under its assumptions. Neither can be interpreted without a reported estimate and analysis. The registry record does not include a primary GPS estimate, confidence interval, or p-value. No conclusion about the size or direction of a primary effect can be drawn from the available record.

How should planning illustrations be read?

Any sample-size figures associated with this study are planning illustrations computed from an observed effect, not the trial’s own design. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. These assumptions describe hypothetical planning calculations. They do not describe the actual single-group study, which has no two-arm allocation.

In general, a sample-size calculation estimates how many participants a future study might plan for under specified assumptions about effect size, variability, and analysis. Such a calculation is not an observed result and is not observed power. The record does not report a trial-specific sample-size justification. Since the posted primary outcome contains baseline data only and no analysis, planning illustrations should not be taken as evidence that the study had adequate power to assess GPS change.

Why does outcome availability matter?

The explanation for baseline-only GPS data is specific: the assay could not be run on 12-month biopsy samples in which active cancer was not identified. In general, unavailable follow-up data limit what can be estimated. The statistical implications depend on why measurements are unavailable and how missingness relates to the outcome; those relationships cannot be determined from the registry statement alone. The record does not report a missing-data method or a sensitivity analysis, so neither should be presumed.

The lack of a reported analysis also means that no assumptions about how repeated measurements were handled can be evaluated. There is no reported analysis of PSA or the exploratory tissue outcomes in the record. These omissions limit interpretation, but they do not establish that no work occurred outside the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists 6 outcome measures, including the primary GPS outcome and secondary outcomes related to PSA, adverse events, and exploratory prostate-tissue pathways. It reports 0 analyses. No method for controlling or interpreting the multiplicity of outcomes is reported in the registry record. In general, when a study examines multiple outcomes, the chance of at least one apparently notable result can depend on how many tests are performed and how they are interpreted. Without an analysis plan or posted tests, it is not possible to assess how multiplicity was handled in this study.

The main missing-data issue described in the record concerns the GPS endpoint: the assay could not be run on 12-month biopsy samples in which active cancer was not identified, leaving only baseline data. The record does not describe a method for imputing missing follow-up scores, an alternative endpoint analysis, or a sensitivity analysis. In general, missing follow-up measurements can constrain estimation of change, and the implications depend on why measurements are unavailable. The reason stated for the assay limitation does not by itself establish a statistical missingness mechanism.

Definitions of analysis populations—such as which participants were included in each outcome analysis—are not reported in the registry record. The record also does not report how withdrawals, incomplete follow-up, or early termination were handled analytically. The planned endpoint allowed measurement at early termination, but the results record does not explain whether such data were available or used.

Because no inferential analyses or outcome estimates are posted, there is no basis for assessing multiplicity-adjusted results, missing-data sensitivity, or differences between analysis populations. These are unreported aspects, not evidence that a particular method was or was not used outside 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.

Most frequent serious adverse event terms

Serious events with the highest arm-level rate: diarrhea (16.7%), depression (16.7%).

For the Noni 6,000 mg/day group, the registry record reports 6 participants at risk for serious adverse events and 2 affected. It reports 6 at risk for other adverse events and 0 affected, with 0 deaths. Diarrhea and depression are each listed among the serious adverse event terms at 16.7%. These figures are descriptive entries in the registry record, not estimates from a controlled comparison.

In general, an at-risk count identifies the group used as the denominator for an adverse-event summary, while an affected count records participants meeting that summary’s event definition. A serious adverse event is a distinct reporting category from other adverse events; the record presents the two categories separately. The posted figures do not give the timing, duration, severity details beyond the serious classification, or attribution of the events to the intervention. They also do not establish how many events occurred within each affected participant.

The percentages attached to the listed terms should be read as the registry’s reported figures, not as reliable estimates of event frequency in a broader population. With a small recorded enrollment and no control group, the safety entries are descriptive and cannot establish a comparative risk or cause.

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

The registry record documents a Phase II, single-group study of Noni extract in men with very low risk or low risk prostate cancer. The intervention regimen was 6,000 mg/day, and the recorded enrollment was 6. The planned primary question concerned change in GPS from screening to 12 months or early termination. The record explains that only baseline GPS data were available because the assay could not be run on 12-month prostate biopsy samples in which active cancer was not identified.

As a result, the posted record does not provide an estimate of GPS change, a direction of change, or uncertainty around a change estimate. It does not report numerical findings for PSA or the exploratory tissue outcomes in the record. The lack of reported results does not mean that the outcomes showed no change; it means that the registry record does not provide results from which that question can be answered.

The single-group design also limits what could be concluded from any within-group change. In general, a randomized comparator helps distinguish intervention-related differences from changes that might occur over time or through other influences. No comparator was recorded for this study, so it cannot provide a randomized treatment contrast.

The safety entries describe serious adverse events in the study group, other adverse events, and deaths as recorded, but they do not establish causality or comparative safety. The registry reports 2 participants affected by serious adverse events, 0 affected by other adverse events, and 0 deaths. Diarrhea and depression are each listed at 16.7% among serious terms. These observations should be understood as descriptive information from this study record, not as a general estimate of risk.

The record does not report a detailed statistical analysis plan, analysis populations, or missing-data procedures. Thus, it does not support claims about statistical significance, precision, or a definitive intervention effect. Its contribution is to document the study design, planned GPS endpoint, the stated limitation on follow-up assay data, and the safety counts entered in the registry.

13. Why This Trial Matters Statistically

This study illustrates why a planned endpoint and an analyzable endpoint are not always the same. GPS change was planned from screening to 12 months or early termination, but the registry record states that the assay could not be run on 12-month biopsy samples in which active cancer was not identified. Only baseline data were available. Without follow-up scores, the planned change cannot be estimated from the posted record.

The design also makes the type of inference especially important. A single-group study can report outcomes among participants receiving an intervention, but it lacks a randomized comparator for estimating a treatment contrast. This distinction applies even when the endpoint is defined as change from baseline. A within-group change and a between-group difference answer different statistical questions.

The registry record lists 6 outcome measures and 0 analyses. That combination highlights the importance of accessible outcome estimates and analysis details: without them, readers cannot assess the primary endpoint’s magnitude, uncertainty, or handling of the secondary outcomes. The record does state why follow-up GPS assay data were unavailable, but it does not report missing-data methods or a sensitivity analysis.

The study therefore offers a useful statistical example of how study design, endpoint availability, and reporting determine what can be concluded. The registry record documents a planned question and a small single-group study, but it does not contain the results needed to quantify the primary outcome or support a comparative inference.

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 principal limitation for interpreting the primary endpoint is that the registry record reports baseline GPS data only. The intended endpoint required assessment from screening to 12 months or early termination, but the assay could not be run on 12-month prostate biopsy samples in which active cancer was not identified. No baseline score values, follow-up scores, or change estimate are reported.

The study had a single-group design, allocation listed as NA, and no masking. There was no recorded control group, so the design does not support a randomized comparison. The recorded enrollment was 6, limiting the scope of descriptive interpretation. The record does not provide enough information to assess how many participants completed each planned assessment or how early termination affected follow-up.

The study status is terminated, but the reason for termination is not reported in the registry record. The record also does not report a study-specific sample-size justification, a detailed statistical analysis plan, confidence intervals, hypothesis tests, missing-data methods, sensitivity analyses, or definitions of analysis populations. It lists 6 outcome measures and 0 analyses. These omissions prevent assessment of inferential methods and of how secondary outcomes were handled.

Safety information is also limited to the posted counts and terms. Timing, duration, event-level detail, and attribution are not reported in the registry record. The descriptive entries cannot establish cause or comparative safety. These limitations apply to interpretation of the record and should not be replaced with assumptions about unreported study procedures or results.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Clinical Study of Noni Extract in Men With Very Low Risk or Low Risk Prostate Cancer — NCT02648919.
• Registry record last updated 2021-11-26; status last verified 2021-10.
• Results first posted 2021-11-26.
• 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

This Phase II study of Noni extract in men with very low risk or low risk prostate cancer was sponsored by the University of Hawaii. The registry record lists a start date of 2015-12, a primary completion date of 2018-12, a terminated status, and enrollment of 6. It records a single-group design, allocation NA, and no masking. The intervention was Noni extract at 6,000 mg/day, taken as 4 capsules with breakfast, 4 with lunch, and 4 with dinner.

The primary outcome was change in GPS from screening and at 12 months or early termination. The record states that only baseline data were available because the assay could not be run on 12-month biopsy samples in which active cancer was not identified. It lists 6 outcome measures and 0 analyses. Safety entries include 2 participants affected by serious adverse events, 0 affected by other adverse events, and 0 deaths. The reason for termination and further statistical analysis details are not reported in the registry record.