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 A Phase 2B Open-Label, Single-Arm, Repeat-Dose Study to Evaluate the Reliability of an Autoinjector (NCT02124798), a na phase 2 study. The primary outcome is Number of Participants Successfully Able to Self-administer Their Observed First and Second Doses in Weeks 1 and 2 (Inside Clinic).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02124798 |
| Phase | Phase 2 |
| Condition | Systemic Lupus Erythematosus |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 95 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GlaxoSmithKline |
| Results posted | Yes |
2. Clinical Question
Population
Participants with systemic lupus erythematosus who were enrolled and treated with at least 1 dose of belimumab. The primary outcome population is described as the intention-to-treat population, with analyses limited to participants with data available at the indicated time points.
Intervention
Self-administration of subcutaneous belimumab using an autoinjector, injected into the thigh or abdomen. The study planned 8 weekly doses, with clinic-observed and outside-clinic administrations.
Comparator
There was no comparison group. The study is recorded as single-arm, open-label, and non-randomized.
Primary question
How many participants successfully self-administered their observed first and second doses in weeks 1 and 2 inside the clinic, as an assessment of the device’s suitability for self-administration?
The question is about device use in the setting of belimumab administration, rather than a comparison of treatments. The registry describes success as a Yes/No assessment of whether drug was successfully injected. The wider usability assessment considered successful self-administered injections relative to attempted injections, and included participants who were able to administer injections inside and outside the clinic without assistance.
The assessment distinguishes clinic-observed use from use outside the clinic. The primary outcome concerns the first two observed doses; secondary outcomes concern observed doses in weeks 4 and 8 and participants’ reports about doses administered outside the clinic in weeks 3, 5, 6, and 7. These are different observations and should not be treated as interchangeable measures.
The registry record does not report the numerical primary outcome result. It therefore does not establish from the posted results how many participants met the success definition at the primary time points, or how precisely that proportion or count was estimated.
3. Trial Design
NCT02124798 is recorded as a phase 2 study of an autoinjector that administers belimumab subcutaneously to participants with systemic lupus erythematosus. Its title describes a phase 2B, open-label, single-arm, repeat-dose study. The study has no randomized allocation and no masking. The registry status is completed.
The registry lists enrollment of 95. The brief summary describes a plan to enroll 118 participants treated with at least one dose. Those statements refer to recorded enrollment and planned enrollment, respectively; the record does not explain the difference. The registry lists the start date as 2014-05-20 and primary completion date as 2015-04-13.
Participants were to self-administer subcutaneous belimumab into the thigh or abdomen using the device for 8 weekly doses. Four doses were to be administered under observation in the clinic, and four outside the clinic without observation. The brief summary also describes a follow-up visit 4 weeks after the last dose. Injections were to be assessed by investigators using direct observation and/or the participant diary.
The single-arm design supports a descriptive account of use and safety in the enrolled group, but it does not provide a concurrent control group. There is no like-for-like randomized comparison from which to estimate a relative treatment effect. Open-label conduct means participants and investigators were not masked to the device being used. The registry record does not report further details of allocation methods, a control condition, or masking procedures.
The stated primary objective was to assess the suitability of the autoinjector for self-administration under real-life conditions, both inside and outside the clinic. Safety and tolerability of subcutaneous belimumab administered through the device were also to be assessed. The available record describes these objectives and procedures, but does not report a numerical primary endpoint result.
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 119 participants were screened out of which only 95 were enrolled to the study treatment. 25 were screen failures the reasons for which were did not meet inclusion/exclusion criteria (23), lost to follow up (1), withdrew consent (1).
Overall Study
| Stage | Total |
|---|---|
| STARTED | 95 |
| COMPLETED | 91 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Total |
|---|---|
| Adverse Event | 3 |
| Lost to Follow-up | 1 |
Analysis population for the primary outcome, as defined in the registry: The intention-to-treat (ITT) population was defined as all participants who were enrolled and treated with at least 1 dose of belimumab. Only those participants with data available at the indicated time points were analyzed.
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.
| Characteristic | Unit | Belimumab 200mg Auto Injector |
|---|---|---|
| Participants analysed | 95 | |
| Age, Continuous | Years | 44.8 (SD 12.50) |
| Sex: Female, Male: Female | Participants | 88 |
| Sex: Female, Male: Male | Participants | 7 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 23 |
| Race (NIH/OMB): White | Participants | 69 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants Successfully Able to Self-administer Their Observed First and Second Doses in Weeks 1 and 2 (Inside Clinic), measured over Weeks 1 and 2 (Inside clinic).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants successfully able to self-administer their observed first and second doses in weeks 1 and 2 inside the clinic. Its unit is participants, and its timeframe is weeks 1 and 2. The registry describes the assessment of whether drug was successfully injected as a Yes/No response.
This endpoint is used to assess whether participants can operate the autoinjector and complete an injection under observation. In general, a count of participants meeting a defined success criterion can communicate how often the intended task was completed in the assessed group. It is not the same as a measure of symptom change, disease control, or comparative treatment efficacy.
The registry’s description also refers to successful injections relative to attempted injections and to participants able to administer injections without assistance. That wording connects device usability to both injection-level attempts and participant-level ability. The primary outcome title, however, specifies a number of participants, so interpretation depends on the exact operational definition and handling of participants with missing assessments. Those details are not fully reported in the registry record.
No numerical result for the primary endpoint is reported in the registry record. Without that result, the number meeting the success criterion and the precision of the estimate cannot be assessed from the posted information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The study addresses a practical question: whether participants with systemic lupus erythematosus can self-administer subcutaneous belimumab with an autoinjector. Its single-arm, open-label structure makes the central evidence descriptive. The registry identifies the intended usability assessments, the dosing schedule, and safety counts, but does not report the numerical result for the primary endpoint.
As a result, the posted record does not show the number of participants who successfully self-administered the observed first and second doses in weeks 1 and 2. Nor does it give an estimate with a confidence interval, a p-value, or another measure of precision for that primary outcome. A confidence interval, in general, describes the range of values reasonably compatible with the observed data under the method used; it cannot be interpreted when the outcome estimate and interval are not reported.
The record lists two secondary usability outcomes: successful self-administration of observed doses in weeks 4 and 8, and participant reports of successful doses outside the clinic in weeks 3, 5, 6, and 7. Their titles do not supply outcome counts. The number of outcome measures is listed, but the registry does not report numerical results for these usability outcomes in the registry record for this analysis.
Because no comparison group was used, the study cannot distinguish device-related usability from other influences through a concurrent between-group comparison. Participants’ experience, clinic observation, and diary reporting are relevant to the intended use conditions, but the registry record does not give detailed results by setting or describe the degree of agreement between observation and diary reports. It also does not report a statistical comparison between clinic and outside-clinic use.
The safety counts are descriptive. The registry lists 4 participants with a serious adverse event among 95 at risk, 28 participants with other adverse events among 95 at risk, and 0 deaths. These counts describe events recorded during the study; they do not by themselves establish whether an event was caused by belimumab, the device, the underlying condition, or another factor. The registry lists six serious-event terms with percentages of 1.1% each, but does not provide further details in the record about timing, severity grades, or attribution.
The planned enrollment in the brief summary was 118 treated participants, while registry enrollment is listed as 95. The record does not explain that difference, and it does not report a sample-size justification. Although sample-size planning illustrations may be presented with the statistical material, such illustrations are not observed power and do not replace an account of the trial’s own design rationale. The registry record does not report that rationale.
Overall, the available information describes a completed device-usability study and records adverse-event counts, but it is insufficient to quantify the primary usability result or its uncertainty. The conclusions that can be drawn are bounded by those missing outcome results and by the absence of a concurrent comparator.
9. Statistical Methods Explained
The design determines what statistical questions can be answered. This study is single-arm, open-label, and non-randomized. In general, a single-arm study can describe outcomes among participants who receive the intervention, but it cannot make a randomized comparison against an alternative. There is therefore no trial-based between-group estimate of a treatment or device effect in this record.
What does the primary measure count?
The primary endpoint is a participant count: participants successfully able to self-administer the observed first and second doses in weeks 1 and 2 inside the clinic. The registry also describes success as a Yes/No assessment of whether drug was successfully injected, and says the overall usability and reliability assessment considered successful completed injections relative to attempted ones. These descriptions involve related but distinct units: an injection attempt and a participant meeting a success criterion. Clear reporting of the operational definition matters because a participant-level count does not convey how many injection attempts were successful unless attempt-level results are reported as well.
For a binary outcome, each assessed person or attempt is classified into one of two categories under the stated definition. A count is a straightforward description, while a proportion would relate the count to the number assessed. The registry record does not report the primary count or its denominator at the relevant time points, so neither an observed proportion nor its precision can be stated.
How should uncertainty be understood?
In general, a confidence interval expresses uncertainty around an estimated quantity under a specified statistical method and assumptions. A narrow interval indicates greater precision than a wide interval, but an interval is not a guarantee about an individual participant’s outcome. It also does not resolve design limitations such as the absence of a comparator. The registry record does not report a confidence interval for the primary endpoint.
For a binary endpoint, uncertainty depends on the number of observations and the observed pattern of outcomes, among other factors. The record states that only participants with data available at the indicated time points were analyzed. It does not report how unavailable observations were handled beyond that statement, or give timepoint-specific analysis denominators. The effect of missing observations on the estimate therefore cannot be assessed from the registry record.
What can the secondary measures add?
The secondary outcomes separate observed clinic use at weeks 4 and 8 from participants’ reports about outside-clinic use in weeks 3, 5, 6, and 7. In general, repeated observations can show whether performance is described at several stages of use, but these observations are not necessarily independent: the same participant may contribute information at multiple times. A comparison across time would need an analysis method that accounts for the repeated structure and an explicit definition of the comparison. Such an analysis is not reported in the registry record.
Outside-clinic success was based on participant reports according to the outcome title, whereas the study summary says injections were assessed through investigator observation and/or diaries. Differences in how outcomes are captured can affect interpretation. The registry record does not provide detailed results by ascertainment method or an assessment of agreement between sources.
What do the planning assumptions mean?
General sample-size planning asks how much information a future study might need to detect a specified effect under stated assumptions. The planning illustration described for the statistical material uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; its effect is expressed as a stated fraction of the observed effect. Those assumptions describe an illustration, not the design of this single-arm device study. The illustration is not observed power, and its sample sizes should not be read as the number enrolled or analyzed in this trial. The registry record does not report the trial’s own sample-size justification or its original design assumptions.
For general interpretation, power is a design-stage probability conditional on an assumed effect, variability, sample size, and analysis method. It is not a measure of the probability that a result is true, and it cannot be recovered simply by looking at whether a study is statistically significant. A planning exercise based on an observed effect is especially dependent on that estimate and should not be treated as independent confirmation of the effect.
What is not reported about the analysis?
The record does not report an inferential statistical plan for the primary endpoint, an adjustment for covariates, a method for confidence intervals, or a procedure for handling multiple outcome tests. It also does not report a statistical comparison between clinic and outside-clinic assessments. Without those details and without numerical endpoint results, readers cannot independently assess the analysis choices, reproduce estimates, or determine how much precision the observed data support.
In general, methods should match the outcome unit, repeated-measure structure, and missing-data pattern. For usability counts, a clear account would identify the analysis population, the number assessed at each time point, the rule defining success, and the treatment of incomplete assessments. The registry record supplies some population and endpoint descriptions, but not all of these details.
10. Multiplicity, Missing Data and Analysis Populations
The primary analysis population is described as the intention-to-treat population: participants enrolled and treated with at least 1 dose of belimumab. The registry further states that only participants with data available at the indicated time points were analyzed. This means the stated population definition does not imply that every enrolled and treated participant contributed to every timepoint analysis.
The registry record does not report timepoint-specific denominators, the number of missing observations, reasons for missingness, or a method for imputing or otherwise handling missing outcomes. It also does not describe sensitivity analyses to examine how missing data might affect the usability findings. In general, missing outcomes can limit interpretation when the availability of an assessment is related to the chance of success; the direction and size of any effect cannot be determined without information about the missingness pattern and analysis.
Multiplicity is relevant because the record identifies a primary outcome and secondary usability outcomes at other time points and settings. Testing or interpreting several outcomes can increase the chance of findings that appear notable by chance when no adjustment or hierarchy is used. The registry record does not report a multiplicity adjustment, a testing hierarchy, or a statistical analysis plan specifying how the outcomes were to be interpreted together. It would therefore be inappropriate to assume that any such procedure was used.
The record also does not report interim analyses, an analysis plan for repeated observations, or the exact handling of participants who attempted but did not complete an injection. These details are not reported in the registry record. The primary endpoint result itself is not numerically reported, limiting evaluation of both missing-data effects and multiplicity in practice.
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 200mg Auto Injector | 4 | 95 | 4.2% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Belimumab 200mg Auto Injector | 0 of 95 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Belimumab 200mg Auto Injector | 28 of 95 |
Most frequent serious adverse event terms
| Event term | Organ system | Belimumab 200mg Auto Injector |
|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 1 of 95 |
| Neutropenia | Blood and lymphatic system disorders | 1 of 95 |
| Pneumonia | Infections and infestations | 1 of 95 |
| Postoperative abscess | Infections and infestations | 1 of 95 |
| Pyrexia | General disorders | 1 of 95 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 1 of 95 |
| Deep vein thrombosis | Vascular disorders | 1 of 95 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Belimumab 200mg Auto Injector |
|---|---|---|
| Nausea | Gastrointestinal disorders | 6 of 95 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 4 of 95 |
| Bronchitis | Infections and infestations | 3 of 95 |
| Sinusitis | Infections and infestations | 3 of 95 |
| Diarrhoea | Gastrointestinal disorders | 3 of 95 |
| Systemic lupus erythematosus | Musculoskeletal and connective tissue disorders | 3 of 95 |
| Gastroenteritis viral | Infections and infestations | 2 of 95 |
| Herpes zoster | Infections and infestations | 2 of 95 |
| Nasopharyngitis | Infections and infestations | 2 of 95 |
| Urinary tract infection | Infections and infestations | 2 of 95 |
Serious events with the highest arm-level rate: anaemia (1.1%), neutropenia (1.1%), pneumonia (1.1%), postoperative abscess (1.1%), pyrexia (1.1%).
The registry lists 4 participants with a serious adverse event, with 95 at risk. It lists 28 participants with other adverse events, also with 95 at risk, and 0 deaths. These are participant counts, not a comparison between randomized groups; the study had a single arm.
The serious-event terms listed are Anaemia, Neutropenia, Pneumonia, Postoperative abscess, Pyrexia, and Pulmonary embolism. Each term is accompanied by 1.1% in the registry record. These term-level percentages should be read as reported, not as evidence that the events were caused by the device or by belimumab. The record does not specify further details such as event timing, severity grading, or attribution in the registry record for this analysis.
In general, adverse-event counts describe what was recorded during a study and do not establish causation. Counts also do not convey the full clinical context, duration, recurrence, or the number of events per participant. The 4 participants with serious events and the 28 with other events may include overlapping participants; the registry record does not state the overlap. These figures are descriptive safety information from this single-arm study, not estimates of excess risk relative to a control group.
12. What the Result Does, and Does Not, Mean
The study’s main question concerns whether participants could use an autoinjector to self-administer subcutaneous belimumab. It included observed clinic administrations as well as planned outside-clinic administrations, and its primary endpoint focused on the first and second observed doses. This makes the study relevant to describing device use in the particular study setting and population.
The registry record does not report the numerical primary outcome result. It therefore cannot show from the available posted information how many participants met the primary success criterion or how precise that result was. The secondary usability results are also not numerically reported in the registry record for this analysis. No conclusion about the observed rate of successful use should be inferred from endpoint titles or study objectives alone.
The design also sets important boundaries. With one open-label group and no randomized comparator, the study does not estimate a comparative effect against another device, administration method, or control condition. The record cannot establish that any reported event was caused by the device or medicine. The safety counts are descriptive and should be interpreted alongside the fact that no control-group counts are available.
In general, a usability study can inform whether a task was completed under the assessed conditions, but that is distinct from establishing clinical efficacy, comparative safety, or performance in settings and populations not studied. The registry describes planned assessments in the clinic and outside it, but does not report numerical results by setting or detailed analyses of the different ascertainment methods.
The registry lists enrollment of 95, while the study summary states that 118 treated participants were planned. The record does not explain the difference. It also does not provide a numerical primary outcome result, timepoint-specific denominators, or enough analysis detail to assess uncertainty or the influence of missing data. These gaps limit the strength of interpretation that can be drawn from the posted information.
13. Why This Trial Matters Statistically
Device usability questions have a different statistical focus from treatment-comparison questions. Here, the central measure concerns participants’ ability to complete self-administered injections, with observed clinic use and reported outside-clinic use considered at specified weeks. Keeping those measures distinct helps readers understand what was assessed and what each type of observation can support.
The study’s single-arm structure is important. It permits description of the enrolled group’s recorded experience, but does not provide a concurrent randomized benchmark. Thus, the results would not answer whether use differed from another device or administration approach. This distinction is essential when interpreting both usability and adverse-event counts.
The endpoint also illustrates why the unit of analysis needs to be explicit. The primary outcome is expressed as a number of participants, while the study description also discusses successful injections relative to attempted injections. Participant-level success and injection-level success are related, but they are not identical summaries. Reporting the rule for classifying success, the number assessed, and the handling of incomplete attempts would help readers understand the measure.
Repeated assessments across weeks and settings raise additional questions about missing observations, dependence between observations, and differences in ascertainment. In general, repeated observations from the same participant are not automatically independent, and participant reports may not be directly equivalent to investigator observation. The registry record does not report analyses addressing these issues.
Finally, a study’s stated objectives and planned sample size are not substitutes for posted results. The registry describes planned enrollment and reports an enrollment figure, but does not give the numerical primary endpoint result or its precision. This makes transparent outcome reporting particularly important for a study designed around a practical task whose success is naturally expressed as a count or proportion.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation of the available record is that it does not report the numerical primary endpoint result. Without the count of participants meeting the success criterion and the corresponding number assessed, the primary usability finding and its precision cannot be evaluated.
The study is open-label and single-arm, with no randomized comparison. That design limits causal and comparative interpretation. Any recorded usability or safety experience describes the study group and does not establish how the experience would compare with another device or method. The registry record also does not report detailed results separately for clinic and outside-clinic assessments.
There is an unresolved difference between planned enrollment of 118 treated participants in the brief summary and registry enrollment of 95. The record does not explain whether this reflects a change in enrollment plans, completion, or another reason. The registry also does not report timepoint-specific analysis denominators, missing-data counts, reasons for missingness, or sensitivity analyses. Because the stated population analysis includes only participants with data available at each indicated time point, those omissions matter for interpreting endpoint counts.
The registry does not report the exact statistical analysis plan, any adjustment for multiple outcomes, or methods for analyzing repeated observations. It also does not detail agreement between direct observation and participant diaries. These limitations restrict independent assessment of how the usability outcome was constructed and analyzed.
For safety, event counts are descriptive, and the record does not include a control group, detailed attribution, timing, or severity information in the registry record for this analysis. The reported counts therefore should not be interpreted as evidence of causation or comparative risk.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase 2B Open-Label, Single-Arm, Repeat-Dose Study to Evaluate the Reliability of an Autoinjector — NCT02124798.
- Registry record last updated 2018-04-11; status last verified 2018-03.
- Results first posted 2017-05-30.
- 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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18. Record Summary
NCT02124798 is a completed, phase 2, open-label, single-arm study of a belimumab autoinjector in participants with systemic lupus erythematosus. The stated objective was to assess suitability for self-administration and the safety and tolerability of subcutaneous belimumab given through the device.
The study planned 8 weekly self-administered doses, with four under observation in the clinic and four outside the clinic without observation. Its primary endpoint concerned participants able to self-administer observed doses in weeks 1 and 2. The registry lists enrollment of 95, while the summary states that 118 treated participants were planned.
The registry reports safety counts of 4 participants with serious adverse events, 28 with other adverse events, and 0 deaths. It does not report a numerical primary outcome result, so the principal usability finding and its precision cannot be evaluated from the posted information. The record also does not report detailed missing-data handling or a multiplicity procedure.