Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases (NCT00811174), a na phase 3 study. The primary outcome is Adverse Events.

2. Clinical Question

3. Trial Design

This was a phase 3, single-group study of Octagam 10% in primary immunodeficiency diseases. The record lists an enrollment of 5 and identifies Octapharma as the sponsor. It records no allocation to comparison arms and no masking. With no control group, the design can describe outcomes observed among participants receiving the intervention, but it cannot by itself separate an intervention effect from other explanations for those outcomes.

The recorded study start was 2009-01 and primary completion was 2010-09. The registry status is terminated. The reason for termination is not reported in the registry record; the status alone does not explain why the study ended or whether the planned follow-up was completed.

The study aimed to evaluate efficacy, safety and kinetics of Octagam 10% as replacement therapy. The primary outcome focused on adverse events around infusions. Listed secondary measures included vital signs, laboratory parameters, viral safety, pre-next-dose total IgG, IgG subclasses and specific antibodies, and therapeutic efficacy measures such as infections, missed school or work days, hospitalisation days and antibiotic use. The registry record lists 8 outcome measures and reports 0 analyses. Detailed eligibility criteria, treatment schedule, follow-up beyond the stated outcome timeframes, and analysis populations are not reported in the registry record.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Reasons for not completing this period:

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: The study was terminated prematurely. Owing to the limited data available (only 5 patients enrolled, of whom 2 completed the study and 3 ended treatment prematurely), efficacy and PK analyses were not performed. The safety analysis did not yield any unexpected results suggestive of a change in the risk-benefit evaluation of Octagam 10%.

6. Primary Endpoint

The registry lists the primary outcome as Adverse Events, measured over During infusion or within 72 hours after end of infusion.

The primary endpoint was adverse events, measured as the number of participants with an event during infusion or within 72 hours after the end of infusion. This is a safety endpoint: it records whether participants experienced adverse events within the specified period, rather than measuring a change in disease symptoms or another measure of efficacy.

Counting participants with events is a binary-outcome approach. In general, it can make the frequency of an event straightforward to describe, while not conveying how many events each affected participant experienced, how severe each event was, or how long it lasted. Those details require separate reporting. The registry record names the endpoint and its timeframe, but does not report an analysis of this primary outcome in the registry record.

The posted safety figures separately report participants affected by serious adverse events and other adverse events. Those figures should not automatically be treated as results for the primary endpoint: the registry record does not establish that the safety summaries use precisely the same timeframe or endpoint definition. The distinction matters when relating a safety summary to the primary outcome.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design estimate?

This study is classified as single-group, with allocation listed as not applicable and masking as none. In general, a single-group design describes outcomes in participants who receive the intervention. A randomized control group can help distinguish intervention-related differences from background changes, but no such comparator is recorded for this study. Consequently, the posted results cannot estimate a relative effect against a concurrent control group.

The enrollment recorded is 5. Small datasets can describe what was observed, but usually leave substantial uncertainty about how representative those observations are of a broader population. This is especially important for event counts: an event observed in a small study may not reflect a stable underlying rate. The registry record does not report confidence intervals or an inferential model for the posted safety counts.

How should participant counts be understood?

The primary outcome is defined as the number of participants with an adverse event in the specified infusion-related period. In general, a participant-level count records whether each person had at least one qualifying event. It does not necessarily count every event, and it does not describe duration, severity, or recurrence unless those are reported separately. The record also provides serious and other adverse-event counts. Those categories are useful for describing reported safety, but the registry entries do not say that these summaries share the primary endpoint’s timeframe.

General statistical explanation: event counts can be analyzed as binary outcomes when the question is whether participants experienced an event. An estimated event proportion is often accompanied by a confidence interval to show uncertainty. No interval or analysis result is reported, and none should be inferred from the counts alone. In particular, a calculated percentage or a significance test would add a result not reported by the registry.

Why does the absence of a comparator matter?

Without a control arm, there is no concurrent reference group for comparing adverse-event frequency, infection outcomes, or other measures. General explanation: even when outcomes are measured after an intervention, changes may reflect the underlying condition, time, other care, or chance as well as the intervention. A control group helps address some of these alternative explanations, though its usefulness depends on the design and analysis. This record does not provide a controlled comparison.

The registry lists therapeutic efficacy measures, including numbers of infections, missed days at school or work, hospitalisation days and antibiotic use. These are outcome definitions, not evidence that an efficacy analysis was conducted. The record lists 8 outcome measures and 0 analyses. Results for those secondary outcomes and their statistical methods are not reported in the registry record.

What can a sample-size illustration say?

General explanation: planning calculations estimate how many participants might be needed to detect a specified effect under assumptions about the significance level, allocation, statistical model and target power. They are not a measure of the probability that a completed study’s finding is true. The planning illustration associated with this trial is based on the observed effect and specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes planned sample sizes for hypothetical comparisons, not the design actually recorded for this single-group study. Such illustrations are not observed power and do not change the fact that no comparator is recorded.

The registry record does not report a sample-size justification for the actual study, the statistical analysis plan, interim analyses, or the methods used for missing data. Without those details, readers cannot determine from the record whether the study’s enrollment was selected for a particular precision target, how analyses were prespecified, or how incomplete observations were handled.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Octagam 10%")
n      <- c(5)
events <- c(4)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Octagam 10%  5  4  80.0  28.4  99.5  37.6  96.4  35.9  95.7

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists multiple outcome measures spanning safety, laboratory findings, antibody levels, and therapeutic efficacy. It reports 8 outcome measures and 0 analyses. Thus, the listed outcomes indicate what was intended to be assessed, but do not provide analyzed results for those measures.

General statistical explanation: when several outcomes or comparisons are tested, the chance of obtaining at least one apparently unusual result can increase. Statistical plans may address this through a prespecified hierarchy, adjustment, or careful distinction between primary and exploratory outcomes. The registry record does not report a multiplicity strategy, so it is not possible to determine whether or how that issue was addressed.

The primary outcome is identified as adverse events. The record does not report analysis details for that endpoint, including an analysis population or a method for handling participants with incomplete outcome information. Missing-data methods and the numbers of participants with missing outcome data are not reported in the registry record. The record also does not specify whether analyses were planned for all enrolled participants, treated participants, or another population.

These omissions limit interpretation of the listed outcomes. Without analysis results, denominators for each outcome, and information about missing observations, outcome definitions alone cannot establish the extent or direction of any efficacy, safety, or kinetics findings.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

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: surgery for inguinal condyloma (20.0%).

The posted safety figures report serious adverse events among 5 participants at risk, with 1 participant affected, and other adverse events among 5 participants at risk, with 5 affected. The serious adverse event term listed most prominently is “surgery for inguinal condyloma,” at 20.0%.

These entries describe reported events; they do not establish causation. The registry record does not state that the surgery was caused by Octagam 10%, and an event’s appearance in a safety listing is not by itself evidence of a treatment relationship. Nor should the serious-event term be taken as a complete clinical account of the event.

The adverse-event endpoint is defined as events during infusion or within 72 hours after the end of infusion, but the safety summaries are not explicitly tied to that timeframe in the registry record. The registry record also does not provide confidence intervals, event narratives, attribution assessments, or an analysis of the safety findings. With such limited counts and no control group, these figures describe what was reported in this study but do not establish comparative safety or the frequency expected in a wider population.

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

The result supports a limited descriptive statement: the registry record lists serious and other adverse-event counts for a single group receiving Octagam 10%. It records 1 affected participant among 5 at risk for serious adverse events and 5 affected among 5 at risk for other adverse events. It also lists “surgery for inguinal condyloma” as a serious adverse event term at 20.0%.

Those counts do not establish whether Octagam 10% caused an event. There is no recorded control group, and the available record does not report an attribution analysis or uncertainty estimates. The serious-event term should be read as a recorded safety classification, not as proof of a causal relationship.

The study’s stated aims included efficacy and kinetics, and the registry lists outcomes related to infections, treatment use, laboratory measures and immunoglobulin levels. However, the record reports 0 analyses. The presence of these outcomes in the record is not a substitute for results. No conclusion about efficacy, kinetics, or changes in those measures can be drawn from the outcome list alone.

The registry status is terminated. The reason for termination is not reported in the registry record, so it cannot be interpreted as evidence of a safety concern or any other specific issue. Overall, the posted information documents a small single-group study and a limited set of safety counts. It does not provide a controlled estimate of treatment effect, a complete safety assessment, or an explanation of the study’s termination.

14. Why This Trial Matters Statistically

This study is statistically notable because its design and the amount of reported information sharply bound what can be concluded. A phase 3 label does not itself establish that a study has a comparator or that its results are precise. This record describes a single-group study with enrollment of 5 and no masking, so interpretation depends on descriptive evidence rather than a randomized between-group comparison.

The difference between an outcome being listed and an outcome being analyzed is important. The registry identifies adverse events as the primary outcome and lists a range of secondary measures, but reports 0 analyses. Readers should not treat planned or registered outcome definitions as evidence of results. This distinction is especially relevant when the study’s aims include efficacy and kinetics but the available record does not report analyses of those domains.

The safety counts also illustrate why denominators and context matter. Counts describe the participants recorded as at risk and affected, while the small study size limits how much those observations can say about broader event frequency. The lack of a control group further means that the counts cannot establish comparative risk or causation.

Finally, the status “terminated” is a registry fact, but it is not an explanation. Without a reported reason, it should not be interpreted as a signal about safety, efficacy, feasibility, or study conduct. Together, these features make careful separation of recorded observations from unsupported conclusions central to reading this trial.

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 study’s single-group design is a major interpretive limitation. Without a control group, there is no concurrent basis for comparing event counts or other outcomes. The absence of masking is also recorded, but the registry record does not describe procedures that might have reduced assessment bias.

Enrollment was 5, which limits the scope and precision of descriptive safety findings. No confidence intervals or inferential analyses for the reported adverse-event counts are included in the registry record. The record also does not specify whether the safety summaries use the same timeframe as the primary outcome definition.

The registry lists outcomes for safety, laboratory measures, immunoglobulin levels and therapeutic efficacy, but reports 0 analyses. Results for the secondary outcomes, and analysis details for the primary outcome, are therefore not reported in the registry record. Information about missing data, analysis populations, treatment schedule, eligibility details, and statistical methods is also not reported in the registry record.

The study status is terminated, but the reason for termination is not reported in the registry record. The available information therefore cannot clarify whether the study ended as planned, how much follow-up was completed, or whether termination affected outcome reporting. The recorded serious adverse event term does not establish treatment causality, and no attribution information is reported.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases — NCT00811174.
• Registry record last updated 2017-07-14; status last verified 2017-06.
• Results first posted 2017-07-14.
• 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

This phase 3 study evaluated Octagam 10% as replacement therapy for primary immunodeficiency diseases. It was single-group, had no masking, and recorded enrollment of 5. The sponsor was Octapharma; the recorded start was 2009-01, primary completion was 2010-09, and registry status is terminated.

The primary outcome was the number of participants with adverse events during infusion or within 72 hours after infusion. The safety data list 1 participant affected by serious adverse events among 5 at risk, and 5 affected by other adverse events among 5 at risk. The registry lists 8 outcome measures and 0 analyses. The reason for termination, analysis methods, analysis populations, and results for the listed efficacy and kinetics outcomes are not reported in the registry record.