This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Belimumab Phase I Study in Chinese Subjects With Systemic Lupus Erythematosus (NCT02880852), a non-randomized phase 1 study. The primary outcome is Maximum Observed Concentration (Cmax) of Belimumab.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02880852 |
| Phase | Phase 1 |
| Condition | Systemic Lupus Erythematosus |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 20 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GlaxoSmithKline |
| Results posted | Yes |
2. Clinical Question
Population
Chinese subjects with systemic lupus erythematosus (SLE) who received belimumab and could contribute pharmacokinetic parameters were the primary-outcome population. The registry records enrollment of 20 participants and describes follow-up after dosing through Day 84.
Intervention
A single intravenous infusion of belimumab, also identified as GSK1550188, at 10 mg/kg on Day 0. The study was open label.
Comparator
There was no comparator group. The allocation is recorded as non-randomized, and the design as single group. The study therefore describes outcomes after dosing rather than a contrast between belimumab and another treatment or a control.
Primary question
What was the maximum observed concentration (Cmax) of belimumab in blood after the single dose? The primary outcome was defined as a geometric mean, with geometric coefficient of variation as its dispersion measure. The registry record describes the sampling schedule but does not report a Cmax result.
3. Trial Design
This was a Phase I, non-randomized, open-label, single-group study of belimumab in Chinese subjects with SLE. The registry lists GlaxoSmithKline as the sponsor and records the study as completed. Its stated purpose was to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) after a single dose. The intervention was one intravenous infusion of belimumab at 10 mg/kg on Day 0. Participants were followed for 84 days after administration.
The study began on 2017-01-23, and its primary completion date is recorded as 2017-09-08. The registry records enrollment of 20. Because there was no comparator arm and treatment allocation was not randomized, the design does not support a like-for-like treatment comparison. Its intended contribution is descriptive: characterizing observations after dosing in the enrolled study population.
The primary outcome concerns the maximum observed concentration of belimumab. Blood samples were scheduled at Day 0 before dosing and at 5 minutes, 1 hour, 6 hours, and 24 hours, followed by Day 1, Day 7, Day 14, Day 21, Day 28, Day 42, Day 56, and Day 84 post-dose. The registry also lists safety and clinical laboratory outcomes, including vital signs, ECG findings, and several chemistry measures. It does not report the results for those outcomes in the record, nor does it give a detailed statistical analysis plan.
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.
Before assignment: A total of 25 participants were screened, of which 5 participants were screen failures. The reason for screen failures were: did not meet inclusion/exclusion criteria (4 participants) and investigator discretion (1 participant).
Overall Study
| Stage | Belimumab 10 mg/kg |
|---|---|
| STARTED | 20 |
| COMPLETED | 20 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic (PK) Parameter Population, which comprised of all those participants who received a dose of belimumab and for whom PK parameters could be calculated.
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: Safety Population which comprised of all the participants who received at least one dose of study medication.
| Characteristic | Unit | Belimumab 10 mg/kg |
|---|---|---|
| Participants analysed | 20 | |
| Age, Continuous | Years | 37.4 (SD 14.07) |
| Sex: Female, Male: Female | Participants | 16 |
| Sex: Female, Male: Male | Participants | 4 |
| Race/Ethnicity, Customized: Asian - East Asian Heritage | Participants | 20 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Observed Concentration (Cmax) of Belimumab, measured over Day 0 (pre-dose, 5 minutes, 1 hour, 6 hours, 24 hours), and on Days 1, 7, 14, 21, 28, 42, 56, and 84 post-dose.
| Element | Registry entry |
|---|---|
| Result type | Geometric Mean |
| Measure of spread | Geometric Coefficient of Variation |
| Unit | Micrograms per milliliter |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is maximum observed concentration (Cmax) of belimumab in blood. Cmax is the highest measured concentration over the scheduled sampling period. It provides a direct description of peak drug exposure after administration, which is one component of pharmacokinetic characterization. It does not, by itself, describe the full concentration-time profile or establish a clinical effect.
Samples were scheduled before dosing and at multiple post-dose timepoints: Day 0 at 5 minutes, 1 hour, 6 hours, and 24 hours, then Days 1, 7, 14, 21, 28, 42, 56, and 84. The primary outcome was specified as a geometric mean, with geometric coefficient of variation as the measure of dispersion. In general, geometric summaries are useful for positive measurements whose distributions may be asymmetric, as can occur with concentration data. The dispersion measure conveys variability on a multiplicative scale rather than expressing variation as an ordinary arithmetic standard deviation.
The registry defines the PK parameter population as participants who received belimumab and for whom PK parameters could be calculated. The registry record does not report the Cmax estimate, its geometric coefficient of variation, or a confidence interval. Therefore, the endpoint definition and planned sampling can be described, but the observed peak concentration and its precision cannot be interpreted from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record describes a single-dose pharmacokinetic and safety study, not a comparative efficacy trial. Its primary question concerns the maximum observed blood concentration of belimumab after an intravenous infusion in Chinese subjects with SLE. The endpoint is specified as a geometric mean with a geometric coefficient of variation. However, the registry record does not report the primary outcome value or an interval describing its precision. The central quantitative result for the stated primary question is therefore unavailable in the record.
Without a comparator group, there is no randomized contrast to estimate. Observations in the treated group can characterize what was recorded after dosing, but they cannot isolate a treatment effect from other explanations, such as the underlying condition, concurrent care, or natural variation. This is especially important when interpreting safety outcomes: counts in a single treated group describe events recorded during follow-up, rather than excess risk attributable to belimumab.
The registry reports safety figures for the Belimumab 10 mg/kg group. It records 1 participant affected by a serious adverse event among 20 at risk, 11 participants affected by other adverse events among 20 at risk, and 0 deaths. Neutropenia and upper respiratory tract infection are each listed among the top serious terms at 5.0%. These figures are descriptive. The record does not establish a comparison with an untreated or alternative-treatment group, and it does not give enough detail to infer causation, event timing, duration, or severity beyond the classification recorded.
Several other outcome measures are listed, including changes in vital signs and clinical chemistry from baseline to Day 84, and clinically significant ECG findings. The record lists 23 outcome measures, but contains 0 analysis entries. This means the registry entry does not supply results for those outcomes; it does not establish that no analyses were done outside the record.
In general, estimates are more informative when accompanied by uncertainty intervals and sufficient detail about the analysis population and missing observations. Those details are not reported for the primary Cmax result in the registry record. Consequently, readers can identify the question, design, sampling schedule, and selected safety counts, but cannot assess the magnitude or precision of the primary pharmacokinetic estimate from the record.
9. Statistical Methods Explained
The study design determines which statistical conclusions are supported. This trial is recorded as non-randomized and single group, with no masking. All participants in the described study group received a single intravenous dose of belimumab. There is no control group against which to compare outcomes. In general, random assignment and a concurrent comparator help distinguish treatment-related differences from background changes and other influences. Without them, the results are best read as descriptions of outcomes after exposure in this study population, not as estimates of comparative treatment effects.
What does the primary summary measure?
Cmax is the highest observed concentration of a drug across the measured sampling times. The registry specifies a geometric mean for this outcome and a geometric coefficient of variation to describe dispersion. A geometric mean is calculated on a multiplicative scale and is commonly used for positive measurements that may have a right-skewed distribution. The geometric coefficient of variation describes relative spread on that scale. In general, this can be useful for pharmacokinetic data, where variation may be proportional to the concentration rather than constant in absolute units. The record does not include the calculated geometric mean or its dispersion estimate, so neither the peak level nor between-participant variability can be assessed from the entry.
Why does the sampling schedule matter?
A maximum observed value depends on when measurements are taken. The scheduled draws begin before dosing and include several early post-dose times, followed by measurements through Day 84. Early sampling can capture concentrations near the peak, while later observations describe concentrations over follow-up. In general, a measured maximum is the largest sampled value, not necessarily the exact concentration at the true biological peak if that peak occurs between sampling times. The registry describes the planned times but does not report individual concentration profiles, the observed timing of Cmax, or the extent of missing PK samples.
What does the analysis population mean?
The primary endpoint population is defined as participants who received a dose and for whom PK parameters could be calculated. This is narrower in principle than simply referring to everyone enrolled: a participant needs relevant dosing and usable PK data to contribute to the parameter estimate. The registry does not report how many participants met that definition, whether any participants were excluded from the PK analysis, or the reasons for any exclusions. It also does not provide the analysis methods used to handle measurements below quantification limits, incomplete sampling, or other data issues; those details are not reported in the registry record.
How should dispersion and precision be distinguished?
Dispersion describes how observations vary within a group. A confidence interval, in general, describes uncertainty about an estimated parameter under the assumptions of the analysis. These are not interchangeable: a variability measure characterizes participant-to-participant spread, while an interval around an estimate addresses estimation precision. The registry identifies a geometric coefficient of variation as the dispersion statistic for Cmax but does not report a confidence interval. Therefore the record does not show either the actual spread of concentrations or the precision of the estimated geometric mean.
What can the safety counts establish?
The adverse-event entries are counts for the single belimumab group. A count of participants affected does not necessarily equal a count of individual event episodes, because one participant may experience more than one event. The record separately reports participants at risk and participants affected, but does not give event-level timing or detail for every event. In general, a safety comparison requires a suitable comparator and consistent outcome ascertainment; a one-group count alone cannot show whether an event occurred more often because of treatment. The listed serious-event terms and deaths should therefore be read as descriptive registry entries rather than causal findings.
What analysis plan is documented?
The registry record does not report a detailed statistical analysis plan, including methods for estimating Cmax, handling missing observations, prespecified subgroup analyses, or the approach to multiple outcomes. Although the record lists 23 outcome measures, it contains 0 analysis entries. This absence of analysis entries limits review of reported estimates but does not prove that no statistical work was performed. A sample-size justification, interim-analysis plan, and rationale for the enrollment target are not reported in the registry record. No sample-size calculation or inference about power can be derived from the listed enrollment alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 23 outcome measures, including the primary Cmax outcome and secondary measures related to vital signs, ECG findings, and clinical chemistry. It records 0 analysis entries. The entry therefore does not present a set of estimates or tests across which the impact of multiple testing could be evaluated. A prespecified multiplicity strategy, such as a hierarchy of outcomes or an adjustment to control the chance of false-positive findings, is not reported in the registry record.
Missing-data handling is also not reported. The record does not describe the number of participants with missing measurements at each timepoint, reasons for missingness, or any imputation or other method used to address it. These details matter because incomplete sampling could affect a maximum observed concentration, and missing follow-up measurements could affect change-from-baseline outcomes. The primary endpoint population is defined by receipt of a dose and calculable PK parameters, but the number meeting that definition and any exclusions are not reported.
The registry identifies enrollment of 20, but a sample-size justification and analysis plan are not reported. The record does not establish whether enrollment was chosen for precision, feasibility, or another reason. The absence of analysis entries should be understood as a limitation of the registry record, not evidence that analyses were never conducted.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Belimumab 10 mg/kg | 1 | 20 | 5.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Belimumab 10 mg/kg | 0 of 20 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Belimumab 10 mg/kg | 11 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | Belimumab 10 mg/kg |
|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | 1 of 20 |
| Upper respiratory tract infection | Infections and infestations | 1 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Belimumab 10 mg/kg |
|---|---|---|
| Upper respiratory tract infection | Infections and infestations | 4 of 20 |
| Hyperglycaemia | Metabolism and nutrition disorders | 2 of 20 |
| Gingivitis | Infections and infestations | 1 of 20 |
| Pharyngitis | Infections and infestations | 1 of 20 |
| Anaemia | Blood and lymphatic system disorders | 1 of 20 |
| Hypokalaemia | Metabolism and nutrition disorders | 1 of 20 |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 1 of 20 |
| Productive cough | Respiratory, thoracic and mediastinal disorders | 1 of 20 |
| Bone infarction | Musculoskeletal and connective tissue disorders | 1 of 20 |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | 1 of 20 |
Serious events with the highest arm-level rate: neutropenia (5.0%), upper respiratory tract infection (5.0%).
For the Belimumab 10 mg/kg group, the registry records 1 participant affected by a serious adverse event among 20 at risk. It records 11 participants affected by other adverse events among 20 at risk and 0 deaths. The listed top serious-event terms are neutropenia and upper respiratory tract infection, each recorded as 5.0%.
These entries describe what the registry reports for the treated group during the study. They do not provide a comparison with a control group, and so cannot establish whether the events were caused by belimumab or occurred more often than they would otherwise have. Affected-participant counts are not necessarily counts of all event episodes; a participant may have more than one event. The record does not report event timing, duration, participant-level overlap among categories, or further detail about the listed serious events. The figures should be interpreted as descriptive safety observations, not as a comparative risk estimate.
12. What the Result Does, and Does Not, Mean
This study addresses a focused early-development question: the pharmacokinetic profile, safety, tolerability, and pharmacodynamics of a single intravenous belimumab dose in Chinese subjects with SLE. The registry specifies Cmax as the primary outcome, defines a PK population, and lays out blood-sampling times through Day 84. It also records selected adverse-event counts and lists secondary safety and laboratory outcomes. These features describe the study’s scope and the information it was designed to collect.
What the registry record does not provide is the numerical Cmax result, its dispersion, or an interval showing its precision. It also does not provide analysis results for the listed secondary outcomes. The record lists 23 outcomes and 0 analysis entries. Thus, the stated endpoint cannot be quantitatively interpreted using this entry alone.
The design also limits the conclusions that can be drawn. With a single group and no comparator, observations after dosing cannot establish how outcomes would have differed without belimumab or under another treatment. The safety figures document events recorded in the treated group, but do not establish causality or comparative risk. Likewise, a pharmacokinetic concentration endpoint measures drug exposure; it is not itself a measure of improvement in SLE or proof of clinical benefit.
The registry describes a single dose and follow-up through Day 84. It does not, in this record, establish outcomes for other dosing regimens, longer-term use, or populations beyond the Chinese subjects with SLE studied. The enrolled group and study design offer descriptive evidence within the study’s defined context, but missing outcome estimates and analysis details limit how fully that evidence can be assessed.
Overall, the record identifies the scientific question, intervention, sampling schedule, outcome definitions, and selected safety counts. It does not report the primary result or enough analytical detail to judge its magnitude, uncertainty, or robustness. Conclusions should therefore remain confined to what the registry explicitly records, without treating the absence of posted estimates as evidence for or against a clinical effect.
13. Why This Trial Matters Statistically
Early pharmacokinetic studies answer questions that differ from those of larger comparative trials. Knowing how a drug concentration is characterized after dosing can inform development and the design of later studies. This record is statistically notable for specifying Cmax as a geometric mean and using a geometric coefficient of variation to describe dispersion, choices that reflect the positive and potentially asymmetric nature of concentration measurements. The scheduled blood draws also make clear that the endpoint depends on observations across time rather than on a single isolated measurement.
The design is important to interpretation. A single-group, non-randomized study can describe measured concentrations and observed safety events in its participants. It cannot estimate a treatment contrast against a control group. Keeping that distinction clear prevents descriptive counts from being read as causal evidence or comparative risk estimates.
The record also illustrates the difference between an outcome being defined and an outcome being reported. It identifies the primary question, defines the PK analysis population, and lists multiple secondary outcomes, yet provides no analysis entries and no Cmax estimate. Without the result and its precision, a reader cannot evaluate the magnitude or reliability of the primary estimate. Similarly, the listed enrollment does not itself explain how the study size was chosen; a justification is not reported.
For biostatistical reading, the key lesson is to distinguish protocol-level descriptions from results and to assess whether the available analysis supports the intended inference. Here, the design and outcome definitions can be described, while interpretation of the primary pharmacokinetic finding remains constrained by the absence of posted estimates and analytical details.
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 registry record gives a limited basis for evaluating the study results. It identifies the primary endpoint and sampling schedule but does not report a Cmax estimate, a geometric coefficient of variation, or a confidence interval. It also lists 23 outcome measures while recording 0 analysis entries, so numerical results for the primary and secondary outcomes are not available in the entry.
The single-group, non-randomized, open-label design has no concurrent comparator. As a result, the study can describe observations among treated participants but cannot make a like-for-like comparison or separate treatment effects from other influences. The record’s safety counts are also limited to descriptive reporting; event timing, detailed event-level data, and participant overlap among categories are not reported.
Further analysis details are not reported in the registry record. These include the number of participants in the PK analysis population, the handling of missing samples, the approach to incomplete data, and the statistical methods used for the outcome estimates. A multiplicity strategy, sample-size justification, and interim-analysis plan are likewise not reported. These omissions limit assessment of precision, potential bias, and the extent to which the analyses followed prespecified methods.
The study concerns one intravenous dose of belimumab at 10 mg/kg, with follow-up through Day 84, in Chinese subjects with SLE. The record does not establish how results would generalize to other populations, dosing regimens, or follow-up periods. In particular, absence of posted outcomes makes it impossible to judge the magnitude or uncertainty of the study’s primary pharmacokinetic finding.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Belimumab Phase I Study in Chinese Subjects With Systemic Lupus Erythematosus — NCT02880852.
- Registry record last updated 2020-03-27; status last verified 2020-03.
- Results first posted 2018-11-09.
- 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.
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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This completed Phase I study evaluated a single intravenous dose of belimumab at 10 mg/kg in Chinese subjects with SLE. The registry records a non-randomized, open-label, single-group design and enrollment of 20. The stated follow-up period was 84 days after dosing.
The primary outcome was maximum observed concentration of belimumab, summarized as a geometric mean with geometric coefficient of variation. Blood sampling was scheduled from Day 0 through Day 84. The record lists 23 outcome measures and 0 analysis entries; it does not report the primary Cmax estimate or results for the secondary outcomes.
For safety, the record reports 1 participant affected by a serious adverse event, 11 affected by other adverse events, and 0 deaths. Neutropenia and upper respiratory tract infection are each listed among the top serious terms at 5.0%. With no comparator group, these counts are descriptive and do not establish comparative risk or causation.