Lovastatin in Treating Patients At High Risk of Melanoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Lovastatin in Treating Patients At High Risk of Melanoma (NCT00462280), a randomized phase 2 study. The primary outcome is Histopathologic Regression of Target Atypical Nevi With Treatment - Pathologist 1's Evaluation.

2. Clinical Question

3. Trial Design

This was a randomized, double-blind, placebo-controlled phase II trial with a parallel-group model. The registry records triple masking. The National Cancer Institute (NCI) was the sponsor. The recorded enrollment was 80, and the trial status is completed. The start is recorded as 2007-05, with primary completion recorded as 2011-04.

The trial included groups defined by nevus pattern: two matched nevi or one large nevus. Within each pattern, participants were assigned to lovastatin or placebo. The primary outcome analysis, however, used only the two-matched-nevi lovastatin and placebo groups. The registry states that the one-large-nevi sample data were not used or analyzed for the primary outcome because of insufficient numbers. Thus the primary estimate does not represent a comparison across all four groups.

Lovastatin and placebo were taken by mouth once daily, for up to 6 months unless disease progression or unacceptable toxicity occurred. The primary outcome timeframe is from baseline up to 24 weeks. Biopsy and laboratory biomarker analysis were listed among the interventions, consistent with assessment of tissue and molecular features as well as clinical outcomes.

The registry gives group-level primary outcome data for 24 participants in the two-matched-nevi lovastatin group and 25 in the corresponding placebo group. It also lists 27 outcome measures, with one analysis attached to the primary outcome. Details such as the original allocation procedure, the trial's intended sample-size justification, and the full prespecified statistical analysis plan are not reported in the registry record. Randomization and masking are important design features, but the registry entries alone do not establish how successfully those procedures were implemented.

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: Only the Two Matched Nevi Group - Lovastatin and Two Matched Nevi Group - Placebo data were used and analyzed for the primary outcome. One-Large Nevi Group - Lovastatin and One-Large Nevi Group - Placebo sample data were not used or analyzed due to insufficient numbers.

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 Histopathologic Regression of Target Atypical Nevi With Treatment - Pathologist 1's Evaluation, measured over From baseline up to 24 weeks.

The primary endpoint is change from baseline in the histopathologic atypia score of target atypical nevi, evaluated by Pathologist 1. The scale has seven levels, from zero for no atypia to six for melanoma. For each participant, the registry says that the change from baseline in the level of atypia was calculated. The endpoint therefore represents a change in a graded tissue assessment rather than a direct event count or a simple measure of whether a lesion was present.

A histopathologic score can capture features seen in tissue that may not be adequately represented by a visual clinical assessment alone. A change score also focuses the comparison on movement from each participant's baseline level, rather than only comparing post-treatment scores. In general, interpreting a change score requires understanding which direction on the scale represents a higher grade; on this scale, higher values indicate greater atypia and the upper end is melanoma.

The primary analysis included only the two-matched-nevi groups receiving lovastatin or placebo. The one-large-nevi groups were not used for this endpoint because their sample data were considered insufficient. Consequently, the result answers a narrower question than whether the treatment groups differed across all participants enrolled in the trial. It describes the average score change in the analyzed groups over the recorded timeframe; by itself, it does not establish a clinical outcome beyond that score.

7. Results Posted in the Registry

• Two Matched Nevi Group - Lovastatin: mean 0.50 versus -0.12 on control; difference 0.62 (95% CI -0.06 to 1.30), standardized effect d = 0.51; z-test p = 0.075.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Clinical Regression of Atypical Moles - Average of Three Reviewers' Evaluations

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Lovastatin: mean 4.03 versus 3.98 on control; difference 0.05 (95% CI -0.11 to 0.21), standardized effect d = 0.17; z-test p = 0.548.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Lovastatin and the control. The interval gives the range of effects that remain compatible with the data.

The registry lists a broad range of secondary outcomes: clinical regression assessments, total nevus counts, and serum or molecular biomarkers including HIF1alpha, (e)-Cadherin, (n)-Cadherin, VEGF, RelA, and p21 (WAF1/CIP1). It records 25 posted secondary outcomes. These measures address related but distinct aspects of atypical nevi and melanoma pathobiology; they are not interchangeable with the primary histopathologic score.

The available secondary analysis includes a clinical regression assessment based on the average of three reviewers' evaluations. Its registry-reported comparison uses a Wilcoxon (Mann-Whitney) test and gives p = 0.61. A separate unadjusted re-calculation based on summary statistics gives p = 0.548. The difference between the reported test and the re-calculation illustrates why methods and summaries should be kept distinct rather than treated as if they were the same analysis.

The secondary outcomes should be considered alongside, not as substitutes for, the primary result. The primary endpoint compares change in histopathologic atypia scores in the two-matched-nevi groups, while secondary assessments include clinical ratings and laboratory measures. Agreement across different measures could add context, but the registry record does not establish an overall pattern of concordant effects across all secondary outcomes. Numerical results for many listed measures are not reported in the registry record in the registry record for this analysis.

Multiple outcomes create opportunities for chance findings. In general, when many comparisons are examined, some may appear notable even if there is no consistent underlying effect. The registry does not report a multiplicity adjustment or a hierarchy for interpreting secondary endpoints. Therefore, individual unadjusted secondary results carry less weight than a clearly prespecified primary result, especially when the broader pattern and analysis plan are not documented.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Clinical Regression of Atypical Moles - Average of Three Reviewers' Evaluations
• Total Nevus Number on Patient's Back - Combined Three Reviewers' Evaluations
• Serum and Molecular Biomarkers - HIF1alpha: Pathologist 3's Evaluation
• Serum and Molecular Biomarkers - (e)-Cadherin: Pathologist 3's Evaluation
• Serum and Molecular Biomarkers - (n)-Cadherin: Pathologist 3's Evaluation
• Serum and Molecular Biomarkers - VEGF: Pathologist 3's Evaluation
• Serum and Molecular Biomarkers - RelA: Pathologist 3's Evaluation
• Serum and Molecular Biomarkers - p21 (WAF1/CIP1): Pathologist 3's Evaluation

9. Statistical Interpretation

10. Statistical Methods Explained

What does a mean change score represent?

The primary outcome is continuous: each participant has a change score, and the registry reports a group mean and standard deviation. A mean describes the average of the observed scores, while the standard deviation describes how dispersed those scores are around their group mean. The mean difference compares the two group averages. Here it is expressed as the lovastatin mean minus the placebo mean, so the sign tells which group had the larger average change score. In general, a group average does not show how many individual participants improved, worsened, or had no change.

The score is ordered by atypia level, with higher values corresponding to greater atypia. The registry describes calculating a change from baseline, but does not give participant-level changes or the distribution of those changes. A mean change can therefore be useful as a concise group comparison while concealing differences in individual responses. It also depends on treating the graded scale as suitable for a mean-based summary; the registry's description identifies the score and summary statistics but does not report further justification for that choice.

How should the mean difference be read?

The reported contrast is 0.62 score units, calculated as the lovastatin group mean minus the placebo group mean. A positive value means the average change score was higher in the lovastatin group in the analyzed sample. Since higher scores correspond to greater atypia, the direction of this estimate is important. The registry labels the outcome histopathologic regression, but the numerical contrast should be read together with the scale definition and the stated change-score calculation, rather than relying on the endpoint label alone.

The posted group means and standard deviations are descriptive summaries. The comparison also gives an unadjusted p-value and a confidence interval. These address different aspects of the result: the mean difference estimates the contrast, the interval expresses uncertainty around that estimate, and the p-value describes how compatible the observed data are with a specified null model under the analysis used. A p-value is not the probability that the null hypothesis is true, and it does not measure the size or importance of a difference.

What does standardization add?

The reported standardized effect, d = 0.507, places the mean difference on a scale based on pooled variability. In general, standardization can make contrasts easier to compare across measures that use different units. It does not remove uncertainty, correct bias, establish clinical relevance, or turn the estimate into a treatment effect that applies to every participant. Its interpretation depends on the variability in this particular analyzed sample.

The reported pooled standard deviation is 1.223. The group standard deviations are 1.1 and 1.33. The registry does not report an adjusted estimate for the primary comparison. The calculation is an unadjusted comparison of posted summary statistics, and the discrepancy flag is false. No sponsor analysis for the primary comparison is separately reported in the registry record.

Why might a nonparametric test also appear?

For a continuous outcome, a mean-based comparison and a rank-based comparison do not ask exactly the same statistical question. In general, a Wilcoxon (Mann-Whitney) test compares the relative ordering of observations between groups and can be useful when a distributional assumption for a mean-based model is a concern. Its interpretation is not automatically a test of the difference between arithmetic means. The registry reports this test for a secondary clinical regression outcome, not for the primary histopathologic outcome.

The secondary outcome's posted analysis reports p = 0.61 using the Wilcoxon (Mann-Whitney) method. A simple unadjusted re-calculation from its group means and standard deviations yields a different p-value, 0.548. The registry's named analysis should be distinguished from that re-calculation: each is based on a different summary or method, and the re-calculation does not supersede the reported analysis.

What is over-dispersion, and is it relevant?

In general, over-dispersion occurs when the observed variability in count data exceeds what a particular probability model assumes. It can lead to uncertainty estimates that are too narrow if the model does not account for the extra variation. The primary outcome is a continuous score summarized by means and standard deviations, not a count outcome, so over-dispersion is not the central issue for this primary analysis. The registry does not report a model for the count-based secondary outcomes or explain how their variability was handled.

What statistical-plan details are available?

The record gives the primary endpoint, its timeframe, the analyzed groups, group sizes, means, standard deviations, and a comparison with an unadjusted p-value. It also lists secondary outcomes and one named secondary analysis. The original sample-size justification, prespecified model details, covariate adjustments, missing-data procedures, interim analyses, and multiplicity strategy are not reported in the registry record. In general, these details help readers assess how fully the posted analyses correspond to the trial's planned questions. Their absence from the record does not show that a particular method was or was not used; it means the record does not establish that information.

11. Confidence Intervals and Precision

The primary estimated mean difference is 0.62, with a confidence interval that includes zero and reaches both negative and positive values. This means the interval is compatible with a range of possible underlying contrasts, including little or no difference and differences in either direction. The observed point estimate is only one value within that uncertainty range; it should not be read as a precise prediction.

A confidence interval is a statistical way to express the uncertainty associated with an estimate under the assumptions of the analysis. In general, it is not a range containing a fixed proportion of individual participant outcomes, nor does it give the probability that the true effect lies inside this particular interval. Its width reflects the information and variability in the data as well as the method used to calculate it. Here the available primary group sizes are 24 and 25, and the group standard deviations are 1.1 and 1.33.

The unadjusted p-value of 0.075 is consistent with uncertainty rather than a sharply resolved difference. It should be read alongside the estimated difference and interval, not as a standalone verdict. The interval's inclusion of zero does not demonstrate equivalence, because no equivalence margin is reported. Similarly, the point estimate does not establish the magnitude that would be expected in a broader population.

Precision is also tied to the analyzed population. The primary comparison omits the one-large-nevi groups, which were not analyzed because of insufficient numbers. The registry does not report enough detail to determine whether other missing outcome data affected the analyzed groups or how such data were handled. Those limitations restrict how confidently the estimate can be generalized beyond the posted comparison.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are participants per arm, not total event counts. They illustrate the group sizes that would be needed under stated assumptions to detect effects expressed as a fraction of the observed primary effect. The assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The effect to detect is the stated fraction of the observed effect. These are planning calculations based on the observed estimate, not the trial's original design assumptions; those original assumptions are not reported in the registry record.

The 80% power column gives the planned participants per arm for an analysis with 80% power under those assumptions. In plain terms, power is the chance that a study will yield a statistically significant result if the effect used for planning is the true effect and the assumptions hold. The 90% power column makes the corresponding calculation for 90% power. Neither column gives the chance that the treatment is effective, nor does either tell how likely the observed result is to be true.

For planning at 100% of the observed effect, the figures are 62 participants per arm for 80% power and 82 participants per arm for 90% power. For 75% of the observed effect, they are 109 and 146 participants per arm, respectively. For 50% of the observed effect, they are 245 and 328 participants per arm, respectively. The pattern reflects a general statistical principle: smaller effects are harder to distinguish from random variation, so more participants are needed to achieve the same planned power.

These calculations are illustrations, not observed power. They use the observed effect as a planning reference and cannot establish that the trial was adequately designed or that its result would replicate. The registry's original sample-size rationale is not reported.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = 0.620; pooled standard deviation σ = 1.223.
2. n = 2 × (1.96 + 0.842)² × 1.223² / 0.620² = 61.1, rounded up to 62 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- 0.62                      # observed difference in means
sigma <- 1.22286374739006                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(462280)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 62 (80% power), 82 (90% power)
 75% of observed effect: n per arm = 109 (80% power), 146 (90% power)
 50% of observed effect: n per arm = 245 (80% power), 328 (90% power)
power.t.test: 62 
Simulated power at n = 62 per arm: 0.797 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Multiplicity, Missing Data and Analysis Populations

The trial record lists 27 outcome measures, including the primary outcome and numerous secondary clinical and biomarker outcomes. Considering many outcomes raises a multiplicity issue: in general, the more statistical comparisons made, the greater the chance that at least one result will look unusual due to random variation. This does not mean that any specific result is false. It does mean that isolated findings require caution, especially when there is no reported strategy for controlling or interpreting multiple comparisons.

A multiplicity adjustment, a prespecified hierarchy of outcomes, or a clearly defined confirmatory subset can help readers understand how the results were intended to be interpreted. Such a strategy is not reported in the registry record. The record also does not report whether the primary endpoint was selected before the trial began or whether analysis choices changed during the study. The absence of these details does not establish that no plan existed; the record simply does not report them.

Missing-data handling is also not reported in the registry record. The primary endpoint description specifies that the one-large-nevi groups were not analyzed because of insufficient numbers, but it does not explain why those groups had insufficient numbers or whether missing outcome data occurred in the two-matched-nevi groups. The record does not describe imputation, complete-case analysis, or sensitivity analyses. It is therefore not possible to assess from these registry entries how missing outcomes might affect the primary comparison.

The analysis population is partly specified: the primary outcome used the two-matched-nevi lovastatin and placebo groups, with 24 and 25 participants, respectively. Further details about eligibility for analysis, exclusions after randomization, or the handling of participants who did not complete the planned assessments are not reported in the registry record. Interim analyses and any associated decision rules are also not reported. These gaps limit assessment of the full analysis process and of how closely it followed the trial's original plan.

15. Safety Results

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

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: hyperbilirubinemia (14.3%), pneumonia (3.1%), rotator cuff surgery (3.1%), elevated cpk (2.9%).

The registry reports serious adverse events by group, alongside the number at risk. In the two-matched-nevi lovastatin group, 1 of 34 participants at risk was affected; in the two-matched-nevi placebo group, 2 of 32 were affected. In the one-large-nevi lovastatin group, 0 of 7 were affected, and in the one-large-nevi placebo group, 1 of 7 was affected. These are descriptive counts from groups with different numbers at risk; they are not adjusted comparisons.

The record also reports other adverse events affecting 27 of 34 participants in the two-matched-nevi lovastatin group and 25 of 32 in the corresponding placebo group. In the one-large-nevi groups, the counts were 4 of 7 for lovastatin and 5 of 7 for placebo. These figures should be read as reported group counts, not as evidence that differences between groups were caused by treatment.

Listed serious adverse event terms include HYPERBILIRUBINEMIA at 14.3%, PNEUMONIA at 3.1%, ROTATOR CUFF SURGERY at 3.1%, and ELEVATED CPK at 2.9%. The corresponding denominators and group-specific breakdowns for these terms are not reported in the registry record. Death counts are also not reported in the registry record. The figures do not establish comparative safety, particularly given the small group sizes and lack of detail about timing, severity, and attribution.

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

The primary finding is an estimated difference in mean change scores between the two-matched-nevi lovastatin and placebo groups. The estimate is 0.62, with a confidence interval that includes zero and an unadjusted p-value of 0.075. These statistics describe the posted data and their uncertainty. They do not establish that lovastatin causes a change in atypia, and they do not establish that there is no difference.

The endpoint is a histopathologic score, with higher values representing greater atypia. The positive mean difference therefore indicates a larger average increase in the score for the lovastatin group than for placebo in this analyzed sample. The registry calls the endpoint regression, but the numerical direction should be interpreted using the score definition and change-from-baseline method. The data should not be converted into a claim about clinical benefit, disease progression, or outcomes beyond the measured score.

The result applies to the two-matched-nevi groups included in the primary analysis. The one-large-nevi groups were not used for that endpoint because of insufficient numbers. The result therefore cannot answer the same question for those groups. Nor does the overall enrollment figure describe the analyzed primary comparison: the posted group sizes for that comparison are 24 and 25.

The secondary outcomes provide additional kinds of measurement, including clinical assessments and biomarkers, but they do not form a single comprehensive conclusion about treatment effect. The registry lists many endpoints and does not report a multiplicity strategy. Individual secondary results should therefore be interpreted cautiously and in the context of the primary outcome and the incomplete reporting of the statistical plan.

In general, randomized comparisons can reduce some sources of bias when allocation and follow-up are conducted as intended. The record identifies the study as randomized and masked, but it does not provide enough information to evaluate every aspect of trial conduct or analysis. The findings are best understood as an uncertain comparison of measured outcomes in a limited analyzed population, not as definitive evidence for or against a clinical effect.

17. Why This Trial Matters Statistically

This trial is statistically informative because it combines a randomized placebo comparison with a biologically oriented endpoint. Histopathologic scoring, clinical assessment, nevus counts, and biomarker measurements capture different dimensions of the research question. The distinction between those measures matters: a change in a pathology score is not automatically equivalent to a clinical change, and a biomarker result is not itself a patient-centered outcome.

The record also illustrates why the analyzed population must be stated precisely. Although enrollment was 80, the primary comparison used the two-matched-nevi groups, with 24 participants in the lovastatin group and 25 in the placebo group. The one-large-nevi groups were excluded from the primary analysis because of insufficient numbers. Naming that restriction prevents the primary estimate from being mistaken for a result across all trial groups.

Precision is central to interpretation. The primary estimate is accompanied by an interval spanning values on both sides of zero, while the unadjusted p-value is 0.075. A careful reading considers the estimated direction, the uncertainty, the endpoint's scale, and the sample that contributed data rather than relying on a binary reading of statistical significance.

The many secondary endpoints also make transparent reporting important. Without a reported multiplicity approach or a full statistical plan, secondary signals are harder to distinguish from chance variation and should not be elevated over the primary comparison. The trial therefore offers a useful example of how design features, endpoint choice, analysis-population restrictions, and uncertainty jointly shape what a clinical trial result can support.

18. 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.

A key limitation is that the primary analysis did not include the one-large-nevi groups. The registry attributes this to insufficient numbers, but does not report more detail about why the numbers were insufficient. The primary result consequently applies only to the two-matched-nevi groups represented in the analysis.

The primary comparison has 24 participants in the lovastatin group and 25 in the placebo group. The confidence interval includes zero and extends to both negative and positive values, indicating meaningful uncertainty in the estimated contrast. The recorded enrollment of 80 should not be mistaken for the sample size contributing to this endpoint.

The registry identifies the endpoint as histopathologic regression, but the score is defined such that higher values indicate greater atypia, and the analysis uses change from baseline. The direction of the numerical estimate therefore needs to be read from the scale definition. The registry does not report additional details about scoring reliability, reader agreement, or adjudication beyond identifying Pathologist 1's evaluation for the primary outcome.

Many secondary outcomes are listed, but a multiplicity strategy is not reported in the registry record. Missing-data procedures, the original sample-size justification, interim analyses, and a full statistical analysis plan are also not reported. The record does not provide enough information to determine whether other participants had missing outcome data or how any such data were handled. These omissions limit assessment of the robustness and completeness of the reported analyses.

Finally, adverse-event figures are descriptive and the groups are small, particularly the one-large-nevi groups. The registry does not provide group-specific denominators for the listed serious-event terms, and death counts are not reported in the registry record. The available safety summaries cannot by themselves establish comparative risk or attribution.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: Lovastatin in Treating Patients At High Risk of Melanoma — NCT00462280.
• Registry record last updated 2014-10-31; status last verified 2014-02.
• Results first posted 2014-10-31.
• 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.

22. Record Summary

NCT00462280 is a completed randomized, double-blind, placebo-controlled phase II trial sponsored by the National Cancer Institute (NCI). It recorded enrollment of 80 and compared lovastatin with placebo among participants with two matched nevi or one large nevus. The primary endpoint was change from baseline in histopathologic atypia score, from baseline up to 24 weeks.

The primary analysis used only the two-matched-nevi groups: 24 participants receiving lovastatin and 25 receiving placebo. The estimated mean difference was 0.62, with an unadjusted p-value of 0.075 and a confidence interval spanning zero. The one-large-nevi groups were not analyzed for the primary outcome because of insufficient numbers.

The record lists clinical and biomarker secondary outcomes and reports adverse-event counts by group. Multiplicity procedures, missing-data handling, and the original sample-size justification are not reported in the registry record. The results are therefore best interpreted with attention to the restricted primary analysis population, endpoint definition, and uncertainty around the estimate.