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 Proof-of-Concept Study With BT063 in Subjects With Systemic Lupus Erythematosus (NCT02554019), a randomized phase 2 study. The primary outcome is Number of Participants With Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02554019 |
| Phase | Phase 2 |
| Condition | Systemic Lupus Erythematosus |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 36 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Biotest |
| Results posted | Yes |
2. Clinical Question
Population
People with systemic lupus erythematosus (SLE) were eligible for this study. The registry record lists an enrollment of 36. Further eligibility criteria and details of the analysis population are not reported in the registry record.
Intervention
The study evaluated repeated intravenous infusions of BT063. The arm description specifies 50 mg administered by intravenous infusion 8 times. The safety results also list a BT063 100 mg group, but its regimen is not reported in the registry record.
Comparator
The comparator was placebo administered by intravenous infusion 8 times. The study was randomized, placebo-controlled, and parallel-group.
Primary question
The primary outcome was the number of participants with adverse events, including serious adverse events and adverse events leading to discontinuation, from baseline through the end-of-trial visit at Week 14. The central question was whether adverse events were recorded among participants assigned to BT063 or placebo during that period. The registry record does not report a statistical comparison of these groups for the primary outcome.
3. Trial Design
This was a prospective, randomized, double-blind, placebo-controlled, repeated-dose, multicentre Phase IIa proof-of-concept study in systemic lupus erythematosus. Its design was parallel-group: participants were assigned to a study group rather than crossing between treatments. The registry record lists quadruple masking. It does not identify which roles were masked, so the masking label should not be expanded into assumptions about specific groups of people.
The registry record gives an enrollment of 36 and describes the study as completed. The listed start date is 2015-09-28, and the primary completion date is 2017-10-25. The stated purpose was to evaluate the safety and efficacy of repeated intravenous BT063 compared with placebo. The arm descriptions specify 50 mg BT063 or placebo, each administered by intravenous infusion 8 times. However, the safety results also include a BT063 100 mg group. The regimen and allocation details for that group are not reported in the registry record.
The primary outcome was a participant count for adverse events over the period from baseline through the Week 14 end-of-trial visit. Secondary outcomes included improvement in joints, improvement in skin, and percent changes in the Systemic Lupus Erythematosus Disease Activity Index 2000. The registry record lists 5 outcome measures but no outcome analyses. It does not report the allocation ratio, detailed procedures, or the rationale for the enrollment target.
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
| Stage | BT063 50 mg | BT063 100 mg | Placebo |
|---|---|---|---|
| STARTED | 12 | 12 | 12 |
| COMPLETED | 10 | 12 | 10 |
| NOT COMPLETED | 2 | 0 | 2 |
Reasons for not completing this period:
| Reason | BT063 50 mg | BT063 100 mg | Placebo |
|---|---|---|---|
| Withdrawal by Subject | 1 | 0 | 2 |
| Adverse Event | 1 | 0 | 0 |
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 | BT063 50 mg | BT063 100 mg | Placebo | Total |
|---|---|---|---|---|---|
| Participants analysed | 12 | 12 | 12 | 36 | |
| Age, Continuous | years | 37.4 (SD 12.77) | 50.7 (SD 7.18) | 48.6 (SD 13.5) | 45.6 (SD 12.63) |
| Sex: Female, Male: Female | Participants | 10 | 11 | 12 | 33 |
| Sex: Female, Male: Male | Participants | 2 | 1 | 0 | 3 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 12 | 12 | 12 | 36 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events, measured over Baseline through End of Trial Visit (Week 14).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events from baseline through the end-of-trial visit at Week 14. Its definition includes serious adverse events and adverse events leading to discontinuation. It is a participant-level count: it concerns how many participants experienced an event, not how many separate event episodes occurred. A participant may experience more than one event, so participant counts should not be treated as event counts.
Safety outcomes are used to describe harms observed during a study and to compare the recorded experience across assigned groups. The time window matters: this endpoint covers the interval beginning at baseline and ending at the Week 14 visit, not an unspecified longer-term period. Interpretation also depends on the group denominators, outcome definitions, and whether all participants were followed and included consistently.
The registry record lists the endpoint and reports serious-event and other-event participant counts by group, but it does not report an analysis of the primary endpoint. The posted counts therefore describe events recorded in the listed groups; they do not provide an inferential comparison or establish how precise any difference between groups would be.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results list 12 participants at risk in each of three groups: BT063 50 mg, BT063 100 mg, and placebo. Serious adverse events affected 1 participant in the BT063 50 mg group, 1 in the BT063 100 mg group, and 0 in the placebo group. The separate other-adverse-event counts affected 2 participants, 6 participants, and 6 participants, respectively. These are participant counts, not counts of every event episode. The categories should not be added together to create a total because the record does not explain whether participants could appear in both categories.
These counts are descriptive. With a small number of participants listed in each group, a count can change noticeably when the experience of one participant differs. Such counts alone do not establish whether a difference is related to treatment, reflects chance variation, or has another explanation. The registry record does not report confidence intervals, a statistical test, an adjusted estimate, or a primary-outcome analysis. The precision of a treatment contrast therefore cannot be assessed from the posted results.
The serious-event terms listed include Synovitis, Bronchopneumonia, and Overdose, each reported as 8.3%. The record does not attach these terms to a specific treatment group in the information available, and the percentages should not be read as group-specific risks. It also lists zero deaths in each of the three safety groups. The posted safety data do not, by themselves, answer the secondary questions about joint improvement, skin improvement, or disease-activity changes.
The study is described as a proof-of-concept trial, but that label does not substitute for outcome estimates or an analysis plan. In particular, the record's zero analysis entries mean that no statistical outcome analysis is reported in the registry record. The findings can be read as a limited account of observed adverse-event counts, not as a definitive estimate of comparative safety or efficacy.
9. Statistical Methods Explained
What does a participant count represent?
For a binary outcome such as whether a participant experienced an adverse event during a defined period, each participant is classified according to whether the event occurred. A group count records how many participants meet that outcome definition. It differs from an event count, which could count repeated episodes in one participant separately. The primary endpoint is explicitly a count of participants with adverse events. The posted serious-event and other-event figures also describe participants affected.
A count needs a denominator to give context. The safety results list 12 participants at risk in each group, alongside the affected-participant counts. Those data allow the reader to see the group sizes as recorded, but the registry record does not report a formal comparison, uncertainty interval, or adjusted effect estimate. No additional rate or difference is calculated.
Why does randomization matter?
In general, random assignment is intended to make treatment groups comparable, on average, with respect to both measured and unmeasured characteristics at the point of assignment. This supports a fairer comparison than assignment based on participant or investigator choice. Randomization does not guarantee that small groups will be balanced on every characteristic, and it does not remove uncertainty from a study with limited observations. The allocation ratio and the method used to generate or conceal assignments are not reported in the registry record.
In general, masking can reduce the influence of expectations on care, outcome assessment, and reporting. The registry record describes the study as quadruple-masked, but does not say which roles were masked or how masking was maintained. Those details should not be inferred from the label alone.
What does the record say about statistical analysis?
The registry record lists 5 outcome measures and zero analyses. It therefore does not report a statistical test or model for the primary outcome, nor does it give an estimate with a confidence interval. It also does not report an adjusted analysis, a prespecified covariate set, an interim analysis, or a statistical analysis plan. Whether those procedures were conducted outside the registry record is not reported in the registry record.
In general, a confidence interval describes the uncertainty around an estimated effect under the assumptions of the method used. A narrow interval indicates greater precision than a wide interval, while an interval that includes values compatible with no difference does not prove that groups are identical. No confidence interval is reported for this primary outcome, so its precision cannot be evaluated using that approach.
How should planning calculations be understood?
The planning illustration is described as using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It considers an effect to detect that is a stated fraction of the observed effect. The record explicitly distinguishes these calculations from the trial's own design. They are planning illustrations, not observed power and not evidence that the original trial was planned using those assumptions.
In general, a sample-size calculation estimates the amount of information needed to detect a target effect under stated assumptions. It is not a measure of whether a completed study has established an effect. Planning results depend on the selected target effect, variability or event frequency, analysis method, allocation, and desired error properties. If the observed effect is uncertain, basing a planning illustration on it also carries that uncertainty forward. The registry record does not report the trial's sample-size justification or enough outcome analysis information to evaluate such calculations against the original design.
For time-to-event outcomes, the stated illustration uses the Schoenfeld approach and reports a total number of events; other illustrations report participants per arm. In general, time-to-event planning depends on event information and follow-up, whereas a participant-per-arm calculation describes a different planning quantity. The primary endpoint in this study is a participant count for adverse events over a specified period, not a time-to-event endpoint.
Over-dispersion is a general concern when count data vary more than a model assumes. It can make standard errors too small if the analysis treats observations as less variable than they are. The posted primary endpoint is a count of affected participants, but the registry record does not describe a count model or any other model for it. There is therefore no basis for attributing a particular modeling choice to this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 5 outcome measures. The named secondary outcomes include the number of participants with joint improvement, the number with skin improvement, and percent changes in the Systemic Lupus Erythematosus Disease Activity Index 2000. It does not report an analysis of these outcomes, or explain how they relate to any additional measures among the 5 listed.
Multiplicity arises when several outcomes, time points, groups, or statistical tests are examined. In general, conducting many tests can increase the chance of finding a seemingly notable result by chance. Researchers may address this through prespecifying a primary outcome, hierarchical testing, or other adjustment strategies. The registry record does not report a multiplicity strategy, a testing hierarchy, or an analysis plan. It is therefore not possible to determine how any multiplicity was handled.
Missing-data procedures are also not reported in the registry record. The record does not state how missing outcome observations, withdrawals, or incomplete follow-up were handled. The primary endpoint includes adverse events leading to discontinuation, but the available description does not explain how discontinuation affected later outcome ascertainment or analysis.
The analysis population is not reported in the registry record. There is no description of whether analyses used all randomized participants, participants who received treatment, or another defined population. These omissions limit interpretation of the posted counts and prevent assessment of whether the outcome definitions, follow-up, and analyses were applied consistently across groups.
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 |
|---|---|---|---|
| BT063 50 mg | 1 | 12 | 8.3% |
| BT063 100 mg | 1 | 12 | 8.3% |
| Placebo | 0 | 12 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| BT063 50 mg | 0 of 12 |
| BT063 100 mg | 0 of 12 |
| Placebo | 0 of 12 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| BT063 50 mg | 2 of 12 |
| BT063 100 mg | 6 of 12 |
| Placebo | 6 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | BT063 50 mg | BT063 100 mg | Placebo |
|---|---|---|---|---|
| Synovitis | Musculoskeletal and connective tissue disorders | 1 of 12 | 0 of 12 | 0 of 12 |
| Bronchopneumonia | Infections and infestations | 0 of 12 | 1 of 12 | 0 of 12 |
| Overdose | Injury, poisoning and procedural complications | 0 of 12 | 1 of 12 | 0 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | BT063 50 mg | BT063 100 mg | Placebo |
|---|---|---|---|---|
| PHARYNGITIS | Infections and infestations | 1 of 12 | 1 of 12 | 1 of 12 |
| HYPERTENSION | Vascular disorders | 0 of 12 | 1 of 12 | 2 of 12 |
| ASTHENIA | General disorders | 0 of 12 | 2 of 12 | 0 of 12 |
| FATIGUE | General disorders | 0 of 12 | 1 of 12 | 1 of 12 |
| BRONCHITIS | Infections and infestations | 1 of 12 | 0 of 12 | 1 of 12 |
| NASOPHARYNGITIS | Infections and infestations | 0 of 12 | 1 of 12 | 1 of 12 |
Serious events with the highest arm-level rate: synovitis (8.3%), bronchopneumonia (8.3%), overdose (8.3%).
The safety results report serious adverse events separately from other adverse events. For serious adverse events, the affected-participant counts are 1 in the BT063 50 mg group, 1 in the BT063 100 mg group, and 0 in the placebo group. For other adverse events, the respective counts are 2, 6, and 6. Each group lists 12 participants at risk. The registry record also reports zero deaths in each group.
The serious-event terms listed are Synovitis, Bronchopneumonia, and Overdose, each with a reported percentage of 8.3%. The registry record does not identify the treatment group for these terms in the information available, so those percentages should not be assigned to an individual group. A term appearing in this list is not, by itself, evidence about cause or comparative risk.
These figures describe participants affected, not the number of event episodes. The record does not say whether a participant with both a serious and another adverse event appears in both categories. The categories should therefore remain separate. The listed 100 mg safety group is not accompanied by a regimen description; the arm descriptions specify 50 mg BT063 and placebo. Further details are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record describes a randomized, placebo-controlled study of repeated intravenous BT063 in people with systemic lupus erythematosus, with a primary safety endpoint covering adverse events through the Week 14 end-of-trial visit. The posted safety results give affected-participant counts for serious and other adverse events in three listed groups, along with the number at risk in each group. These observations document what was recorded in the registry; they are not a formal comparison of treatment effects.
The record does not report a primary-outcome analysis, confidence intervals, or statistical tests. As a result, it does not quantify the precision of differences between groups. The counts cannot establish that any observed pattern is caused by treatment, nor can they demonstrate that the groups have equivalent safety profiles. Zero deaths are reported in each group, but this observation should not be treated as proof that a risk is absent.
The study's proof-of-concept designation and stated purpose include efficacy, but the available outcome information does not provide results for the listed joint, skin, or disease-activity outcomes. No conclusion about efficacy can be drawn from the safety counts alone. The registry record also does not report the analysis population, missing-data approach, or multiplicity strategy, all of which would help clarify the interpretation of outcome findings.
Overall, the posted data support a narrow descriptive reading: adverse-event participants were recorded in the specified groups and period, while the comparative analysis and its uncertainty are not reported in the registry record. Any broader conclusion would require additional outcome and methods information.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between a registered outcome and a reported analysis. The primary endpoint is clearly described as a count of participants with adverse events over a defined period, and the registry includes safety counts for three groups. Yet it lists zero analyses. Readers can identify the outcome being tracked without being able to assess a formal estimate of comparative effect or its precision.
The study also illustrates why denominators and outcome definitions matter. The same participant may experience multiple events, while the reported endpoint counts participants rather than episodes. Serious and other adverse events are presented separately, and the registry record does not explain possible overlap. Keeping those distinctions intact prevents unsupported totals and avoids treating unlike measures as interchangeable.
Randomization and masking are important design features, but they do not replace transparent reporting of analysis populations, missing-data procedures, multiplicity handling, and uncertainty. Those details are not reported in the registry record. Their absence limits what can be concluded from otherwise interpretable descriptive counts.
Finally, the safety groups include BT063 100 mg even though the arm descriptions specify 50 mg BT063 and placebo. This highlights the importance of clear alignment between treatment-group descriptions and outcome reporting. The record is useful for identifying the trial's question and the available safety observations, while also showing where further statistical reporting would be needed to support a more complete interpretation.
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 does not report a primary-outcome statistical analysis, an effect estimate, a confidence interval, or a test result. The adverse-event counts can therefore be described, but comparative precision cannot be assessed. The registry record also does not explain whether serious and other adverse-event categories overlap for individual participants.
There is a treatment-description gap: the arm descriptions specify 50 mg BT063 and placebo, each given by intravenous infusion 8 times, while the adverse-event results list BT063 50 mg, BT063 100 mg, and placebo. The regimen and assignment details for the 100 mg safety group are not reported in the registry record. This makes the relationship between the described intervention and all listed safety groups unclear.
The analysis population, missing-data handling, follow-up completeness, and multiplicity strategy are not reported in the registry record. The record also does not provide detailed eligibility criteria, allocation methods, masking roles, or the sample-size justification. The listed serious-event terms are not linked to particular groups in the information available. These limitations constrain interpretation of the counts and prevent a fuller assessment of the trial's statistical methods.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Proof-of-Concept Study With BT063 in Subjects With Systemic Lupus Erythematosus — NCT02554019.
- Registry record last updated 2020-01-27; status last verified 2020-01.
- Results first posted 2020-01-27.
- 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
The registry record describes NCT02554019, a completed, randomized, placebo-controlled, parallel-group Phase IIa proof-of-concept study of repeated intravenous BT063 in systemic lupus erythematosus. It lists an enrollment of 36 and describes quadruple masking. The arm descriptions specify 50 mg BT063 or placebo, administered by intravenous infusion 8 times.
The primary outcome was the number of participants with adverse events, including serious adverse events and events leading to discontinuation, from baseline through Week 14. Safety results list BT063 50 mg, BT063 100 mg, and placebo groups, with serious and other adverse-event participant counts. The registry record lists 5 outcome measures and zero analyses. It does not report a primary-outcome comparison, confidence intervals, analysis populations, or methods for missing data and multiplicity.