A Study to Evaluate the Pharmacokinetics, Efficacy and Safety of Intravenous Golimumab in Pediatric Participants With Active Polyarticular Course Juvenile Idiopathic Arthritis Despite Methotrexate Therapy: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study to Evaluate the Pharmacokinetics, Efficacy and Safety of Intravenous Golimumab in Pediatric Participants With Active Polyarticular Course Juvenile Idiopathic Arthritis Despite Methotrexate Therapy (NCT02277444), a na phase 3 study. The primary outcome is Serum Trough Concentration (C-trough) of Golimumab.

2. Clinical Question

3. Trial Design

This completed Phase 3 study is described as a multicenter, open-label trial of intravenous golimumab in pediatric participants with active polyarticular course juvenile idiopathic arthritis despite methotrexate therapy. The registry lists a single-group model, no masking, and an allocation value of NA. Enrollment was 130.

The treatment description specifies golimumab 80 mg/m² by intravenous infusion at Weeks 0 and 4, then every 8 weeks up to Week 244. Participants also received commercial methotrexate weekly through Week 28. The registry record does not provide further usable detail about the methotrexate dose in the treatment description.

The study started on 2014-12-22, had a primary completion date of 2018-07-09, and is marked completed. Its primary endpoint was serum golimumab trough concentration at Week 28. Secondary outcomes listed include serum trough concentration and Bayesian area under the curve at steady state at Week 52.

Because this is a single-group study, the design does not provide a concurrent control arm for estimating a treatment contrast. The registry record lists no analysis entries, and the numerical primary endpoint result is not reported in the registry record. The study design and endpoint can therefore be described, but the primary pharmacokinetic result and its statistical precision cannot be evaluated from the registry entry.

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.

Treatment Period (Week 0-52)

Reasons for not completing this period:

LTE Period (Week 52 to Week 252)

Reasons for not completing this period:

ETP Period (Week 252 to Week 420)

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The pharmacokinetic (PK) analysis set included all treated participants (who received at least 1 infusion) who have sufficient PK samples for analysis. Here, 'N' (number of participants analyzed) signifies number of participants evaluable for this outcome measure.

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: Full analysis set included all participants who received at least 1 dose of study agent.

6. Primary Endpoint

The registry lists the primary outcome as Serum Trough Concentration (C-trough) of Golimumab, measured over Week 28.

The primary endpoint is serum trough concentration of golimumab at Week 28, measured in micrograms per milliliter. A trough concentration is a drug concentration measured at the low point of a dosing interval, before a subsequent dose. As a general pharmacokinetic concept, trough measurements help characterize how much drug remains in the circulation between administrations. They are not, by themselves, a direct measure of clinical benefit.

The registry describes the endpoint as a mean with standard deviation. The analysis population is defined as treated participants who received at least 1 infusion and had sufficient pharmacokinetic samples for analysis; the reported N would represent participants evaluable for this outcome. This distinction matters because enrollment and the number contributing to an endpoint need not be the same.

The endpoint is suited to describing drug exposure at a specified treatment time point. The registry record does not report the Week 28 mean, standard deviation, or evaluable N. It also does not report a confidence interval or another uncertainty estimate for this endpoint. Without those results, neither the observed concentration level nor its precision can be assessed from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary measure represent?

Serum trough concentration is a continuous pharmacokinetic measure: each value lies on a numerical scale, and a mean can describe the central level across participants. The registry specifies a mean and standard deviation for the Week 28 endpoint. As a general statistical concept, the mean summarizes the average of observed values, while the standard deviation describes how spread out those values are around the mean. These summaries answer different questions: a mean alone does not show whether participant values were tightly clustered or widely dispersed.

A trough concentration is measured at the low point of a dosing interval. In general, pharmacokinetic measurements can help characterize drug exposure and persistence in the body. They do not automatically establish a clinical effect, and the registry’s primary endpoint is not a direct measure of arthritis activity.

Who contributes to the endpoint?

The registry defines the pharmacokinetic analysis set as treated participants who received at least 1 infusion and had sufficient pharmacokinetic samples for analysis. It also says that N for this outcome represents the number evaluable for it. This is important because the enrollment total of 130 does not establish how many participants had usable Week 28 samples. The evaluable number is not reported in the registry record.

In general, an analysis restricted to participants with sufficient measurements can be affected if the availability of measurements is related to participant characteristics or outcomes. The registry record does not describe the amount or pattern of missing Week 28 samples or the method used to handle missing data, so the potential influence of missingness cannot be judged.

What can a single-group design estimate?

The trial is listed as single-group and open-label, with no masking and no separate control group. In general, a single-group study can describe outcomes among participants receiving the regimen, but it cannot directly estimate a between-group contrast against a concurrent control. Changes over time, if reported, could not be attributed to treatment by a controlled comparison using this design alone. For the primary endpoint, the registry record describes a concentration at one time point rather than a treatment-versus-control effect.

Open-label means the study is not described as masked. For a laboratory concentration, the measurement is numerical, but study conduct and availability of samples can still matter when interpreting results. The registry record does not report further details about blinding of laboratory measurement or other procedures.

How should uncertainty be understood?

As a general statistical concept, a confidence interval gives a range of values compatible with the observed data under a specified statistical method. It communicates uncertainty about an estimate; it is not a guarantee that the true value lies in that range. The Week 28 mean and standard deviation, and any confidence interval or other uncertainty estimate, are not reported in the registry record. Therefore, the precision of the primary endpoint cannot be evaluated from the posted registry information.

Standard deviation and standard error are not interchangeable. In general, standard deviation describes spread among participant measurements, while standard error describes uncertainty in an estimated mean. The registry identifies standard deviation as the planned dispersion summary. No standard error is reported in the registry record.

What do the planning illustrations mean?

Any sample-size planning illustration is distinct from an observed trial result. The stated approach uses a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a fraction of the observed effect. For time-to-event outcomes, the stated illustration concerns total events; for other outcomes, it concerns participants per arm. These are planning assumptions, not observed power.

Those assumptions describe a hypothetical two-arm planning framework, while this trial is listed as single-group. A planning illustration therefore does not turn the study into a randomized comparison and should not be read as evidence that the trial had a particular probability of detecting an effect. The record does not report the trial’s sample-size justification, and the numerical planning outputs are not reported in the registry record.

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("Golimumab")
n   <- c(87)
m   <- c(0.5)
sp  <- c(0.427)
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  Golimumab  87  0.500  0.427  0.409  0.591

11. Multiplicity, Missing Data and Analysis Populations

The registry lists four outcome measures and identifies Week 28 serum golimumab trough concentration as the primary endpoint. It also lists Week 52 serum trough concentration and Bayesian area under the curve at steady state among the secondary outcomes. The numerical results for these measures are not reported in the registry record, and no analysis entries are listed.

When a study evaluates multiple outcomes or conducts multiple statistical tests, the chance of a false-positive finding can increase if no adjustment or testing hierarchy is used. This is a general statistical concern, not a claim that a particular error occurred in this study. The registry record does not report a multiplicity adjustment, a testing hierarchy, or a strategy for interpreting multiple outcomes.

The pharmacokinetic analysis population includes treated participants who received at least 1 infusion and had sufficient pharmacokinetic samples for analysis. The record does not report the number evaluable for the primary endpoint. It also does not describe how missing measurements were handled, whether any imputation was used, or how the reasons for missing samples were assessed. These details are not reported in the registry record.

Enrollment was 130, but that total alone cannot establish the endpoint analysis size or the completeness of follow-up. Without the evaluable N and missing-data methods, readers cannot assess how closely the endpoint summary represents all enrolled participants. The record also does not report a statistical analysis plan or further details about the analysis populations beyond the stated pharmacokinetic set.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: calcinosis (3.1%), ocular implant exposure (3.1%), appendicitis (3.1%), myopericarditis (0.8%), supraventricular tachycardia (0.8%).

The registry lists safety counts for three named periods. During the Treatment Period (Week 0 to 52), 9 of 127 participants were affected by serious adverse events, 74 of 127 were affected by other adverse events, and 1 death was recorded. During the LTE Period (Week 52 to 252), the corresponding counts were 18 of 127, 57 of 127, and 1 death. During the ETP (Week 252 to 420), they were 3 of 32, 8 of 32, and 0 deaths.

These are period-specific counts, not a controlled comparison. The periods cover different follow-up windows, and the number at risk differs in the ETP; their counts should not be read as directly comparable estimates of risk. The registry record does not state in this listing whether the deaths or adverse events were judged related to treatment. An event occurring during a study period does not, by itself, establish causation.

Selected serious-event terms are listed with percentages: Calcinosis, Ocular Implant Exposure, and Appendicitis each at 3.1%; Myopericarditis, Supraventricular Tachycardia, and Glaucoma each at 0.8%. The corresponding term-specific counts and further details are not reported in the registry record. These entries should be read as selected terms, not as a complete account of every safety outcome.

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

The registry record describes a study designed to evaluate pharmacokinetics, efficacy, and safety, with serum golimumab trough concentration at Week 28 as the primary endpoint. The posted information identifies the endpoint and its intended summary, but does not give the numerical result. It therefore cannot show the average Week 28 concentration, the variability of the measurements, the number analyzed, or the precision of the estimate.

The study is a completed, open-label, single-group trial. Its design can provide descriptive information about participants receiving the listed regimen, but it does not supply a concurrent control comparison. In general, this distinction limits conclusions about differences that might have occurred under another treatment or without the study regimen. The primary endpoint is a pharmacokinetic concentration, so even a reported concentration would describe drug exposure rather than directly demonstrate a clinical outcome.

The safety record reports participants affected by serious and other adverse events in separate periods, along with deaths and selected serious-event terms. These counts are useful descriptions of recorded events, but without a control group and attribution details they do not establish that treatment caused or prevented any event. Differences in the periods’ follow-up windows and numbers at risk also limit direct comparisons across those periods.

Accordingly, the registry record supports a description of the study design, endpoint definition, analysis population definition, and period-specific safety listings. It does not support a quantitative interpretation of the primary pharmacokinetic result or its uncertainty, because those numerical results are not reported in the registry record. The sample-size planning assumptions, where discussed, should be treated as hypothetical planning illustrations rather than observed evidence or observed power.

14. Why This Trial Matters Statistically

This trial raises several useful statistical distinctions for clinical researchers. First, enrollment is not the same as the number analyzed for an endpoint. The registry enrolled 130 participants but defines the pharmacokinetic analysis set using treatment receipt and availability of sufficient samples. Because the evaluable number is not reported, readers cannot determine how many participants contributed to the Week 28 summary.

Second, the endpoint measures drug concentration, not a clinical outcome. A mean and standard deviation would describe the level and spread of concentrations, while an uncertainty interval would address precision in the estimated mean. The registry record does not report these numerical results, illustrating why a clearly defined endpoint is not enough to interpret the evidence: its observed value and uncertainty are also needed.

Third, design determines the scope of inference. A single-group, open-label study can describe observations under its regimen, but it does not create a concurrent treatment contrast. Planning calculations framed around two arms do not change that fact and should not be confused with observed power or with the actual study design.

Finally, safety counts need their denominators and observation windows. The record gives period-specific counts and numbers at risk, but the periods differ in duration and one has a smaller at-risk group. Reading those figures as if they were directly comparable would overlook the structure of follow-up. Together, these features show why endpoint definitions, analysis populations, design, uncertainty, and observation time all matter when interpreting clinical trial statistics.

15. Limitations

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

The primary limitation is that the registry record does not report the numerical Week 28 trough concentration result. The mean, standard deviation, and number analyzed are absent, as are confidence intervals or other uncertainty summaries. The primary endpoint’s observed level and precision therefore cannot be assessed.

The study is listed as single-group, open-label, and without masking. There is no concurrent control group described, so the design does not support a direct controlled comparison. The registry record does not report a statistical analysis plan or the trial’s sample-size justification. It lists no analysis entries.

The defined pharmacokinetic population requires treatment and sufficient samples, but the number evaluable and the approach to missing measurements are not reported in the registry record. This prevents assessment of endpoint completeness and the potential effect of missing data. Numerical results for the secondary pharmacokinetic outcomes are also not reported in the registry record.

The safety information is organized by periods with different follow-up windows and, for the ETP, a different number at risk. The listing does not establish treatment causality, and the registry record does not provide attribution details for the events or deaths. The selected serious-event terms are accompanied by percentages, but term-specific counts and fuller event details are not reported. These limitations restrict both quantitative interpretation of efficacy-related outcomes and comparative interpretation of safety.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study to Evaluate the Pharmacokinetics, Efficacy and Safety of Intravenous Golimumab in Pediatric Participants With Active Polyarticular Course Juvenile Idiopathic Arthritis Despite Methotrexate Therapy — NCT02277444.
• Registry record last updated 2025-11-14; status last verified 2025-10.
• Results first posted 2020-11-18.
• 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

NCT02277444 is a completed Phase 3, multicenter, open-label, single-group study of intravenous golimumab in pediatric participants with active polyarticular course juvenile idiopathic arthritis despite methotrexate therapy. Enrollment was 130. The listed primary endpoint was serum golimumab trough concentration at Week 28, with mean and standard deviation specified.

The registry record defines the pharmacokinetic analysis population but does not report the Week 28 numerical result or evaluable N. It lists Week 52 pharmacokinetic secondary outcomes and period-specific adverse-event counts. The design has no concurrent control group, and the registry record lists no analysis entries. The available information supports description of the design and recorded safety counts, but not assessment of the primary endpoint’s observed value or statistical precision.