Gemcabene for the Treatment of Pediatric NAFLD: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Gemcabene for the Treatment of Pediatric NAFLD (NCT03436420), a na phase 2 study. The primary outcome is Percent Change in Alanine Aminotransferase (ALT).

2. Clinical Question

The study is described as a multicenter, prospective, open-label, Phase 2, proof-of-concept study. Its stated hypothesis concerns potential changes in ALT, hepatic steatosis, dyslipidemia, and de novo lipogenesis. Those statements describe the study's aims, not findings. The registry lists 6 participants enrolled, but the number included in the primary endpoint analysis is not reported in the registry record.

The main interpretive issue is the absence of a comparison group and of a reported endpoint estimate. A change from baseline, if reported, would describe change among the analyzed participants over the study period. Without a concurrent comparator, the design cannot separate a treatment-associated change from other explanations such as natural variation or changes over time. The registry record does not report a primary endpoint estimate, confidence interval, or p-value, so the magnitude and precision of any change cannot be assessed from the posted results.

3. Trial Design

The registry identifies this as a Phase 2a study of gemcabene for pediatric NAFLD. It describes a prospective, multicenter, open-label, single-group design. The allocation field is listed as NA, and the record explicitly identifies the study as not being a randomized comparison. Masking is listed as none. The only described arm is Gemcabene, consisting of children receiving 12 weeks of treatment.

The study description states a hypothesis that 300 mg of gemcabene once a day for 12 weeks will reduce ALT, hepatic steatosis, and dyslipidemia and down-regulate de novo lipogenesis. This is a stated hypothesis, not a reported result. The registry does not give further details about dose adjustment, adherence assessment, co-interventions, or procedures for measuring the other proposed biological outcomes. Such details are not reported in the registry record.

The study lists 6 participants enrolled. Its primary endpoint timeframe is baseline and Week 12, and the primary population is described as participants completing 12 weeks of gemcabene. The number completing treatment, the number with evaluable endpoint measurements, and the reasons for any missing measurements are not reported in the registry record. The study status is TERMINATED. The reason for termination is not reported in the registry record, so the status alone does not establish why the study ended or whether termination affected outcome collection.

Primary completion is recorded as 2019-08-27, and the start date as 2018-03-29. The record lists 38 outcome measures and 0 analyses. It also names several secondary outcomes, including ALT, hepatic steatosis, pancreatic fat, AST, fasting insulin, fasting glucose, and HOMA-IR. The presence of listed outcomes does not mean that results for them are available. A control group, the rationale for the enrollment target, an interim analysis plan, and the statistical analysis plan are not reported in the registry record.

In a single-group design, the observed before-and-after pattern is descriptive of participants who contribute measurements. General statistical interpretation requires caution because a within-person change does not by itself establish what would have happened over the same period without the intervention. The open-label design also means the record does not describe masking of participants or study personnel. These design characteristics define the context for reading any result, but they do not reveal the size or direction of the unreported endpoint finding.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: This analysis includes participants completing 12 weeks of gemcabene.

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: The baseline analysis includes all children who began the trial.

6. Primary Endpoint

The registry lists the primary outcome as Percent Change in Alanine Aminotransferase (ALT), measured over Baseline, Week 12.

The primary endpoint is percent change in alanine aminotransferase (ALT) from baseline to Week 12. ALT is an enzyme measured in blood, and the registry description says ALT elevation is the most common screening test used for detecting NAFLD. The record gives context that normal ALT ranges vary by age, sex, and assay, and describes ALT as a screening measure rather than a direct measure of every aspect of liver disease.

In general, a percent-change endpoint expresses a follow-up measurement in relation to a baseline measurement. It can help describe relative change when participants begin with different baseline values, although its interpretation depends on the exact calculation and the distribution of the measurements. The registry record identifies the endpoint as a mean with standard deviation, but does not report the mean percent change, its standard deviation, the analyzed sample size, or a confidence interval. The exact calculation details are not reported in the registry record.

The stated timeframe is baseline and Week 12. The primary analysis population is described as participants completing 12 weeks of gemcabene. This focus on completers means the endpoint description does not establish how participants without Week 12 measurements were handled. ALT is used because it is relevant to the study's stated question and is described as a common screening test; an ALT change alone should not be read as a complete measure of liver health or as evidence about every listed secondary outcome.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes the primary measure as a mean of a continuous endpoint, with standard deviation as the stated measure of dispersion. A continuous measure can take values across a scale rather than belonging to a set of categories. General statistical explanation: the mean describes the central level of observed measurements, while the standard deviation describes how spread out individual measurements are around that mean. They answer different questions: the mean concerns the average observed change, and the standard deviation concerns participant-to-participant variability.

What does a mean percent change describe?

The endpoint is percent change in ALT from baseline to Week 12. In general, a percent change expresses change relative to a starting value, rather than reporting only the raw difference in measurement units. Its interpretation depends on the precise calculation, including how baseline and follow-up values are combined across participants. The registry record does not provide that calculation detail or a numerical estimate. It therefore is not possible to recover the endpoint result from the description alone.

A mean is a summary of the values included in an analysis, not a description of every participant. General statistical explanation: the same mean can arise from different participant-level patterns, such as values clustered near the average or values spread widely around it. A standard deviation helps describe this spread, but neither the mean nor the standard deviation alone communicates the precision of the estimated mean. A confidence interval is commonly used for that purpose. No confidence interval is reported in the registry record.

How does the design affect interpretation?

This is a single-group study, not a randomized comparison. General statistical explanation: a within-group change compares follow-up with baseline among the observed participants, while a comparative treatment effect contrasts outcomes between treatment groups. Those are distinct quantities. A before-and-after change can describe the study participants' course, but without a concurrent comparator it does not show what their course would have been under an alternative condition. The registry record contains no randomized or otherwise described control group.

Open-label means masking is listed as none. General statistical explanation: masking can reduce the influence of knowledge about treatment assignment on some aspects of study conduct or assessment. Its absence does not prove that an outcome was affected, and the registry record does not report whether any safeguards were used. The endpoint is a laboratory measure, but details of laboratory assessment and site procedures are not reported in the registry record.

What does the analysis population mean?

The primary population is described as participants completing 12 weeks of gemcabene. General statistical explanation: an analysis limited to completers summarizes outcomes among those with the specified follow-up, and may not represent everyone enrolled if some participants do not complete follow-up. The registry lists 6 participants enrolled, but it does not report the number completing treatment or the number with an evaluable primary endpoint. It also does not report how missing baseline or follow-up values were handled. Therefore, the contribution of incomplete follow-up to the analysis cannot be assessed.

Missing data methods matter because an analysis may depend on whether missing measurements are excluded, imputed, or handled through another approach. These methods rely on assumptions, and different assumptions can lead to different estimates. General statistical explanation: reporting the amount and pattern of missingness, the analytic population, and the method used helps readers judge the robustness of results. Those details are not reported in the registry record.

What can be inferred from the reported dispersion?

The endpoint is specified as a mean with standard deviation, but no numerical values are posted. General statistical explanation: standard deviation describes variability among individual observations; it is not interchangeable with the standard error or a confidence interval. The standard error concerns uncertainty in an estimated mean, while a confidence interval presents an interval for that estimate under the statistical method used. Since the record does not report the mean, standard deviation, sample size analyzed, or confidence interval, neither variability nor precision can be evaluated.

For continuous outcomes, distributions can matter. General statistical explanation: a mean and standard deviation may not fully characterize a strongly skewed distribution or a dataset influenced by unusually high or low values. The registry does not report participant-level results, a distribution plot, or diagnostic checks for the primary endpoint. No conclusion about the shape of the observed ALT changes is possible from the endpoint specification.

What about multiple outcomes and sample size?

The registry lists 38 outcome measures and 0 analyses. General statistical explanation: when investigators examine many outcomes or comparisons, the chance of at least one apparently unusual result can increase unless the analysis plan accounts for multiplicity. The record does not report whether outcomes were prioritized beyond naming a primary endpoint, how multiple testing was handled, or which analyses were performed. The count of registered outcome measures should not be mistaken for a count of completed statistical tests.

The record does not report a sample-size justification or an inferential analysis plan. It includes planning assumptions for an illustrative sample-size calculation based on an observed effect, but no numerical planning results or observed effect estimate are reported in the registry entries. General statistical explanation: planning calculations depend on assumptions about the effect of interest and variability, and are not observed evidence about an intervention. A planning illustration is not observed power and does not substitute for an estimate with uncertainty from the actual study.

In this record, the central statistical limitation is not a complicated modeling choice that can be reconstructed; the analysis results and plan are simply not reported. The registry's endpoint description tells readers what was intended to be summarized, but it does not give the estimate or enough analysis detail to judge its uncertainty, missing-data sensitivity, or relationship to the secondary outcomes.

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("Gemcabene")
n   <- c(3)
m   <- c(79.86)
sp  <- c(27.02)
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  Gemcabene  3  79.860  27.020  12.739  146.981

11. Multiplicity, Missing Data and Analysis Populations

The record lists 38 outcome measures, including a named primary endpoint and multiple secondary outcomes. It lists 0 analyses. No multiplicity strategy, outcome hierarchy beyond the primary endpoint designation, or adjustment for multiple testing is reported in the registry record. General statistical explanation: considering many outcomes can make isolated findings harder to interpret, particularly when the analysis plan is not available. The outcome count alone does not establish which measures were analyzed or whether any result was selected for emphasis.

The primary population is described as participants completing 12 weeks of gemcabene. The registry lists 6 enrolled participants, but does not report the number who completed 12 weeks, the number with baseline and Week 12 ALT measurements, or the number included in the primary analysis. The handling of missing values, withdrawals, and incomplete visits is not reported in the registry record. The reason for termination is also not reported.

General statistical explanation: missing-data methods can affect an estimate because a complete-case analysis describes those with available measurements, whereas other methods may use additional assumptions to account for missing outcomes. Readers generally need the amount and pattern of missingness, the analysis population, and the method used to evaluate whether the conclusion is sensitive to those choices. Those details are not reported in the registry record.

A sample-size justification, interim analysis plan, and prespecified statistical analysis plan are not reported in the registry record. The record's planning assumptions do not provide a reported clinical-trial power calculation or observed endpoint result. Without the analysis plan and participant flow, it is not possible to determine how the registered outcomes relate to analyses actually conducted.

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 adverse-event figures list 6 participants at risk for serious adverse events, with 0 affected. The same group lists 6 participants at risk for other adverse events, with 5 affected, and 0 deaths. No top serious adverse-event terms are listed. These figures describe the counts recorded for the Gemcabene group; they do not provide a comparison with a control group.

General statistical explanation: a count of participants affected is not necessarily the same as a count of separate events, because one participant may experience more than one event. The posted figures do not provide event-level details, severity or relatedness assessments for the non-serious events, timing, or individual outcomes. The absence of listed serious-event terms does not add information about events beyond the reported count of 0 participants affected by serious adverse events.

The group at risk includes 6 participants, but the registry does not specify the period of safety follow-up for these figures in the information available. In a small single-group study, safety counts are descriptive and cannot establish how the observed experience compares with another treatment or with no treatment. The reason the study was terminated is not reported in the registry record, so the termination status should not be interpreted as evidence of a safety-related cause.

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

The record establishes that a prospective, multicenter, open-label, single-group Phase 2a study of gemcabene in children with NAFLD was registered. It specifies percent change in ALT from baseline to Week 12 as the primary endpoint and describes the primary analysis population as participants completing 12 weeks of treatment. It also records 6 participants enrolled and a TERMINATED status.

It does not report the numerical primary endpoint result. The mean percent change, standard deviation, analyzed sample size, confidence interval, and p-value are not reported in the registry record. Consequently, the posted information does not show whether the average ALT value changed, the direction or magnitude of any change, or how precisely it was estimated. The study hypothesis should not be mistaken for an observed result.

Even if a before-and-after endpoint estimate were available, the single-group design would describe change in the participants who contributed data rather than establish a comparative effect. General statistical explanation: concurrent controls help distinguish change associated with treatment from change that might occur over time for other reasons. This record has no control group, so it cannot provide that comparison. The open-label design is another feature relevant to interpretation, although the record does not establish that it influenced any measurement.

The record names several secondary outcomes and lists 38 outcome measures, but it provides no numerical findings for them. It also does not report missing-data methods, multiplicity handling, the reason for termination, or a statistical analysis plan. The safety figures record 0 participants with serious adverse events, 5 with other adverse events, and 0 deaths among 6 at risk; they are descriptive and have no control-group context.

Accordingly, the registry information supports a description of the study's question, design, and recorded safety counts, but not a conclusion about the size, precision, or comparative meaning of the primary endpoint. It also cannot support conclusions about unreported secondary outcomes or explain why the study ended.

14. Why This Trial Matters Statistically

This study is statistically informative as an example of the distinction between a registered question and a reported result. The record specifies a primary endpoint, a measurement timeframe, and an analysis population, but does not report the endpoint estimate or its uncertainty. A clear endpoint definition is important, yet it cannot replace the numerical findings needed to understand what the data show.

The design also illustrates the limits of a single-group study for estimating comparative effects. General statistical explanation: measurements before and after treatment can describe a trajectory, but a concurrent comparison is needed to address how that trajectory differs from what might have occurred under another condition. The registry identifies no control group, so the design provides no like-for-like contrast.

The small enrollment of 6 and the completer-defined primary population make participant flow especially relevant to interpretation. The registry does not state how many participants completed 12 weeks or contributed to the endpoint analysis. Without that information, readers cannot connect enrollment to the analyzed population or assess how missing follow-up may affect the summary.

The record lists 38 outcome measures and 0 analyses. This emphasizes the value of accessible analysis plans and results: readers need to know which outcomes were analyzed, how multiple outcomes were handled, and what estimates and uncertainty accompanied each result. Those details are not reported in the registry record.

The safety record provides counts, but not a comparator or detailed event descriptions. Together, the design and reporting limitations mean the record is most useful for understanding the intended study question and the limits of the available evidence, rather than for quantifying an effect. The trial's listed status is TERMINATED, while the reason is not reported; preserving that distinction avoids turning a status label into an unsupported explanation.

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 principal trial-specific limitation is the single-group design. There is no randomized comparator, and allocation is listed as NA. Any baseline-to-Week-12 change would therefore be descriptive rather than a direct contrast with another treatment or control condition. Masking is listed as none, and the record does not describe additional safeguards or procedures that might affect outcome assessment.

The enrollment is 6, while the primary endpoint population is defined as those completing 12 weeks of gemcabene. The number completing treatment and the number analyzed are not reported in the registry record. This prevents assessment of participant flow and of how closely the analysis population corresponds to everyone enrolled. Missing-data handling and reasons for missing outcomes are also not reported.

The registry identifies the primary endpoint and its timeframe but supplies no numerical endpoint result, standard deviation, confidence interval, or p-value. It lists 38 outcome measures and 0 analyses, but does not report which outcomes were analyzed or how multiplicity was handled. A sample-size justification, interim analysis plan, and statistical analysis plan are not reported in the registry record.

The status is TERMINATED, but the reason for termination is not reported. The record also does not provide detailed laboratory procedures, adherence information, participant-level measurements, or information sufficient to assess whether outcome ascertainment differed across sites. These are reporting limitations; they should not be treated as evidence that any particular problem occurred.

Finally, the safety counts are presented for one group without a control comparison. The registry lists 0 participants with serious adverse events, 5 with other adverse events, and 0 deaths among 6 at risk, but does not provide event-level descriptions for the other adverse events. This limits how fully those counts can characterize safety experience.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Gemcabene for the Treatment of Pediatric NAFLD — NCT03436420.
• Registry record last updated 2020-12-31; status last verified 2020-12.
• Results first posted 2020-11-12.
• 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

The registry record describes NCT03436420, Gemcabene for the Treatment of Pediatric NAFLD, as a Phase 2a, prospective, multicenter, open-label, single-group study. It lists 6 participants enrolled, no masking, and a TERMINATED status. The primary endpoint is percent change in ALT from baseline to Week 12 among participants completing 12 weeks of gemcabene.

The study description states a hypothesis involving 300 mg once a day for 12 weeks. This is a stated study aim, not a finding. The record lists 38 outcome measures and 0 analyses, but does not report the primary endpoint estimate, its uncertainty, the number analyzed, or results for the listed secondary outcomes. The reason for termination and the statistical analysis plan are not reported in the registry record.

Safety figures list 0 participants with serious adverse events, 5 with other adverse events, and 0 deaths among 6 at risk. The single-group design provides no control comparison, so these counts are descriptive. Overall, the record identifies the study question and design but does not provide enough outcome information to assess the primary result or its precision.