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 Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases (NCT03988426), a na phase 3 study. The primary outcome is Number of Serious Bacterial Infections Per Person-Year on Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03988426 |
| Phase | Phase 3 |
| Condition | Primary Immune Deficiency Disorder |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Octapharma |
| Results posted | Yes |
2. Clinical Question
Population
Patients with primary immunodeficiency diseases. The registry record does not report additional eligibility details or a more specific description of the analyzed population.
Intervention
Octanorm, human normal immunoglobulin for subcutaneous administration. The record describes a liquid formulation at a concentration of 16.5%, administered by subcutaneous infusion at weekly intervals.
Comparator
There was no separate comparator group: the study is recorded as single-group, with allocation listed as not applicable. The registry record therefore does not describe a randomized or like-for-like control comparison.
Primary question
The primary endpoint was the number of serious bacterial infections per patient-year on treatment during the primary treatment period of 24 weeks. The registry record does not report the observed rate or its statistical uncertainty.
In practical terms, the endpoint asks how frequently a specified set of serious bacterial infections occurred relative to time receiving treatment. The definition includes bacteraemia or sepsis, bacterial meningitis, osteomyelitis or septic arthritis, bacterial pneumonia, and visceral abscess. It is an event-rate question, rather than simply a question about whether each participant experienced at least one infection.
The study does not frame that question as a comparison between Octanorm and another treatment, placebo, or usual care. A single-group rate can describe the observed experience under the study conditions, but it cannot by itself estimate a between-group treatment effect. The registry record does not report a primary endpoint estimate, a confidence interval, or a hypothesis test. Accordingly, it does not establish how the observed rate compares with a rate under another care strategy.
3. Trial Design
This was a phase 3 clinical study of Octanorm in patients with primary immunodeficiency diseases. The registry record lists a single-group model, no masking, and allocation as not applicable. It records enrollment of 25 and a completed status. The listed start date is 2017-03-07, and the primary completion date is 2018-01-26.
The intervention was human normal immunoglobulin for subcutaneous administration. The arm description identifies a liquid formulation with a concentration of 16.5%, delivered as a subcutaneous infusion at weekly intervals. The record notes administration at the study center during initial training sessions and then for every fourth administration, but the available arm-description text is incomplete. Further details of administration are not reported in the registry record.
With one treatment group and no masking, the study design is descriptive for the posted results rather than a randomized comparative design. The absence of a comparator matters when interpreting the primary event rate: any observed rate would describe the study group over its recorded treatment exposure, not a contrast attributable to treatment assignment. The registry record does not report a control group or a concurrent comparator.
The primary treatment period is listed as 24 weeks. The record also lists 19 outcome measures and several secondary outcomes concerning infections, infection resolution, antibiotic use, infection-related hospitalizations, and fever. It does not report the detailed eligibility criteria, the analysis population for the primary endpoint, or the statistical methods used to analyze that endpoint. Although the study is marked completed, completion status alone does not provide the numerical results or show which participants contributed to each outcome.
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 | Participants Administered Octanorm |
|---|---|
| STARTED | 25 |
| COMPLETED | 24 |
| NOT COMPLETED | 1 |
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 | All Patients |
|---|---|---|
| Participants analysed | 25 | |
| Age, Continuous | years | 35.24 (18 to 64) |
| Sex: Female, Male: Female | Participants | 15 |
| Sex: Female, Male: Male | Participants | 10 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 25 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Russia | participants | 25 |
| Height | centimeters | 170 (SD 8.68) |
| Weight | kilograms | 66.49 (SD 10.54) |
| BMI (Body Mass Index) | kg/m^2 | 23.02 (SD 3.48) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Serious Bacterial Infections Per Person-Year on Treatment, measured over Primary Treatment Period (24 Weeks).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | SBI per patient year |
| Endpoint classification | Event-Rate Endpoint |
The primary endpoint is the number of serious bacterial infections per patient-year on treatment during the primary treatment period of 24 weeks. It is an event rate: the count of qualifying infection events is interpreted relative to the time participants contribute while receiving treatment. This differs from the proportion of participants who experience at least one event, because an event-rate measure can account for more than one event in a participant and for differences in observed exposure time.
The endpoint uses a defined set of serious bacterial infections: bacteraemia or sepsis, bacterial meningitis, osteomyelitis or septic arthritis, bacterial pneumonia, and visceral abscess. Restricting the outcome to specified serious infections makes the event definition clinically focused and supports consistent classification, although the registry record does not describe adjudication procedures or how uncertain cases were handled.
In general, an event rate can be useful when follow-up or treatment exposure varies, because the denominator reflects time at risk rather than participants alone. Interpretation still depends on complete event ascertainment, appropriate exposure-time measurement, and a suitable statistical model. The registry record does not report the observed event count, total exposure time, estimated rate, confidence interval, or analysis method for this endpoint. Its numerical result and precision therefore cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a primary endpoint and its time frame, but it does not report the primary endpoint result. There is no posted serious bacterial infection rate, event count, exposure-time denominator, confidence interval, or p-value for that endpoint. As a result, the record does not allow readers to assess the observed frequency of the defined infections or the precision of an estimated rate.
The design also limits what any single-group result could establish. A rate observed among participants receiving Octanorm would describe their experience in the study, conditional on how events and treatment exposure were collected. Without a comparator group, it would not estimate a difference from another treatment or from no treatment. The registry record does not report a benchmark rate for comparison, and one should not infer a comparative effect from the study design.
The safety information is a separate part of the record. For the Octanorm group, serious adverse events were reported for 0 of 25 participants at risk; other adverse events were reported for 18 of 25 participants at risk; deaths were recorded as 0. These safety counts do not supply the missing primary infection-rate result. Serious bacterial infections in the primary endpoint and serious adverse events in the safety record are distinct outcome concepts, even though a serious infection could also meet adverse-event criteria under a study's reporting rules. The registry record does not explain any overlap or classification process.
The record lists 19 outcome measures and 0 analyses. This means the registry record does not give analysis-specific results for interpreting the listed outcomes. The study's completed status and enrollment figure do not substitute for endpoint results, information about analysis populations, or measures of uncertainty. Without those details, statistical evidence about the primary endpoint cannot be evaluated.
Overall, the available record supports a description of the intended study question, the single-group design, and the safety counts recorded for the treatment group. It does not support a conclusion about the primary infection rate, its precision, or a comparative treatment effect. Those limits concern what is reported in the registry record, not necessarily what may have been collected or analyzed outside it.
9. Statistical Methods Explained
Interpreting a rate endpoint requires attention to both its numerator and its denominator. The numerator is the number of qualifying events; the denominator is time on treatment, expressed in patient-years. In general, a rate normalizes the event count by exposure time, which can be useful when participants contribute different amounts of follow-up. Here, the registry record gives the endpoint definition and a primary treatment period of 24 weeks, but does not report the event count, the total time on treatment, or the estimated rate. The endpoint therefore cannot be reconstructed from the posted information.
What does a confidence interval convey?
In general, a confidence interval expresses the statistical uncertainty around an estimate under the assumptions of the method used. For an event rate, the interval would help readers judge how precisely the observed rate has been estimated. It does not describe the range of outcomes for individual participants, and it does not by itself establish whether an effect is clinically important. No confidence interval for the primary endpoint is reported in the registry record, so the precision of that rate is unknown.
Why does the analysis model matter?
For event counts, a statistical model must reflect how events arise and how exposure time enters the analysis. A common starting point for counts is a Poisson model, which assumes that the event-count variability is compatible with the mean under the model. In general, over-dispersion occurs when variability is greater than that assumption allows. Ignoring over-dispersion can make standard errors too small and confidence intervals too narrow. Alternative approaches can account for extra variability, but their suitability depends on the data and analysis plan. The registry record does not report the model, any dispersion assessment, or adjustments used for the primary endpoint. No particular model should be attributed to this trial on the basis of the posted record.
The distinction between an event rate and a participant-level risk is also important. An event rate counts events in relation to exposure time; a risk or proportion instead asks whether participants experienced an event during a defined period. If participants can have repeated infections, these summaries answer different questions. The primary endpoint is explicitly an event-rate measure. The registry record does not provide a corresponding participant-level summary for serious bacterial infections.
Can this design estimate a treatment contrast?
A single-group study can estimate or describe outcomes within its treated group if the relevant results are reported. A treatment contrast, by comparison, requires a comparator and a defined method for comparing groups. Since this study is recorded as single-group, with allocation not applicable and no masking, there is no randomized comparison described in the registry record. A within-group rate should not be interpreted as a treatment effect relative to another strategy. Any external comparison would require information about the source population, outcome definitions, follow-up, and analysis that is not reported in the registry record.
In general, masking can reduce some risks of biased outcome assessment, depending on the outcome and how it is measured. The registry record lists masking as none. It does not describe event adjudication or other safeguards that might have been used to support consistent assessment. The effect of this design feature on the primary endpoint cannot be determined from the reported details alone.
What do the planning illustrations mean?
Sample-size planning is different from analyzing observed outcomes. The stated planning framework describes hypothetical calculations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it also describes the target effect as a stated fraction of the observed effect. For time-to-event calculations, the framework refers to a total number of events, while other calculations are expressed as participants per arm. These are planning assumptions, not evidence that the trial used a two-arm design. The actual trial is recorded as single-group.
Any sample-size figure based on an observed effect is a planning illustration, not observed power and not a result of this trial. Its interpretation depends on the assumed effect, variability, design, allocation, and analysis method. The registry record does not report a sample-size justification for the actual study, the observed primary effect needed for such a calculation, or an analysis linking the planning assumptions to this single-group design. Therefore, hypothetical two-arm calculations should not be treated as the study's design or as a test of its findings.
Finally, a registry entry listing 0 analyses does not provide a substitute for a statistical analysis plan. The method used to handle repeated events, variable exposure, missing outcome information, or any deviations from planned analysis is not reported in the registry record. Without those details and a numerical estimate with uncertainty, readers cannot independently evaluate the primary rate analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 19 outcome measures and names secondary outcomes relating to other infections, infection resolution, antibiotic use, hospitalizations due to infection, and fever. It lists 0 analyses. No analysis-specific estimates, tests, or adjustments for multiple comparisons are reported in the registry record.
In general, testing multiple outcomes or making multiple comparisons can increase the chance of at least one apparently unusual result arising by chance. A prespecified strategy can define which outcome is primary and how secondary results will be interpreted, including whether multiplicity adjustments are used. The registry record identifies a primary endpoint, but does not report a multiplicity strategy, a hierarchy among secondary outcomes, or an analysis plan. It is therefore not possible to determine how the listed outcomes were handled statistically.
The record also does not report the amount or pattern of missing outcome information, the approach to missing data, or the population included in the primary endpoint analysis. Enrollment of 25 does not establish that every participant contributed complete primary endpoint data or the same amount of exposure time. In general, missingness can affect both event counts and time-at-risk denominators, and different assumptions about unavailable information can lead to different estimates. No such assumptions or sensitivity analyses are reported in the registry record.
These omissions prevent assessment of the completeness and robustness of the analysis. They do not establish that missing data or multiplicity problems occurred; they mean that the registry record does not report how either issue was addressed.
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 |
|---|---|---|---|
| Octanorm | 0 | 25 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Octanorm | 0 of 25 |
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 |
|---|---|
| Octanorm | 18 of 25 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Octanorm |
|---|---|---|
| Respiratory Tract Infection | Infections and infestations | 6 of 25 |
| Condition Aggravated | General disorders | 5 of 25 |
| Bronchitis | Infections and infestations | 4 of 25 |
| Dizziness | Nervous system disorders | 2 of 25 |
| Food Poisoning | Gastrointestinal disorders | 2 of 25 |
| Rhinitis | Infections and infestations | 2 of 25 |
| Pharyngitis | Infections and infestations | 2 of 25 |
| Blood and Lymphatic Infection | Blood and lymphatic system disorders | 2 of 25 |
| Investigations | Investigations | 2 of 25 |
For the Octanorm group, the registry record reports serious adverse events for 0 of 25 participants at risk, other adverse events for 18 of 25 participants at risk, and 0 deaths. These are safety figures for the group recorded in the registry; they are not the primary serious bacterial infection endpoint and should not be substituted for its missing result.
In general, a participant count indicates how many people were recorded as affected, not how many separate event episodes occurred. The safety figures posted in the registry record do not state the number of individual adverse-event episodes, event severity breakdown beyond the serious-event category, timing, or attribution details. The record also does not list top serious adverse-event terms. It is therefore not possible to describe the nature or course of any other adverse events from these figures alone.
Because there is no comparator group, these counts do not estimate a difference in safety outcomes between treatments. They also do not establish that events were or were not caused by Octanorm. The registry record does not report additional detail needed to assess event ascertainment, follow-up completeness, or causal attribution.
12. What the Result Does, and Does Not, Mean
The study record describes a phase 3, single-group evaluation of subcutaneous Octanorm in patients with primary immunodeficiency diseases. Its primary question concerns the rate of a defined group of serious bacterial infections per patient-year on treatment during a 24-week primary treatment period. This is a clinically specific event-rate endpoint, but the registry record does not report its numerical result.
Consequently, the record does not show the observed infection rate, how precisely it was estimated, or whether the data were compatible with a range of possible rates. It also does not report a test of the primary endpoint. Without those results, no statistical conclusion about the endpoint can be drawn from the registry record.
The single-group design further means that the record does not provide a randomized comparison with another treatment or a control condition. Even if a treated-group rate were reported, it would describe outcomes observed in that group rather than, by itself, establish what would have happened under a different strategy. A causal or comparative interpretation would require additional evidence and suitable comparison methods.
The safety information records 0 serious adverse events among 25 participants at risk, other adverse events among 18 of 25 participants at risk, and 0 deaths. Those counts describe the entries in the safety record; they do not establish a comparative safety profile or provide detail on event types and attribution. They are also distinct from the primary serious bacterial infection outcome.
Thus, the record is informative about the study's design, intervention, stated endpoint, and reported safety counts, but it does not provide the outcome analysis needed to judge the primary endpoint. A completed status indicates the study is recorded as completed; it is not itself evidence of a particular clinical or statistical result.
13. Why This Trial Matters Statistically
This trial is statistically notable for the contrast between a rate-based primary question and a single-group design. The endpoint is expressed per patient-year on treatment, making exposure time part of its definition. That choice can describe event frequency while accounting for time observed, but meaningful interpretation still requires the event count, exposure denominator, estimation method, and uncertainty. Those primary endpoint results are not reported in the registry record.
The design also clarifies the boundary between description and comparison. A single group can provide information about the events recorded among its participants, but it does not create a randomized reference group. Without a comparator, a treatment contrast cannot be estimated from the design described in the registry record. This distinction is essential when reading any rate from a one-group study and is especially important when no rate is posted.
The listed safety counts offer a limited additional description: serious adverse events were reported for 0 of 25 participants at risk, other adverse events for 18 of 25, and deaths as 0. They do not replace the primary outcome analysis, nor do they characterize comparative safety. The registry record's listing of 19 outcome measures alongside 0 analyses also illustrates why an outcome list should not be mistaken for a set of reported statistical results.
More broadly, the record shows how study registration details can identify a research question without supplying enough analysis information to evaluate its answer. Endpoint definitions, exposure time, comparator structure, missing-data handling, and uncertainty all affect what a result can establish. In this case, the design and endpoint are described, but the primary numerical result and its analysis are not reported in the registry record.
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 most consequential limitation for interpretation is that the registry record does not report the primary endpoint estimate, event count, exposure denominator, confidence interval, or statistical test. The endpoint is defined, but its observed value and precision cannot be assessed.
The study is recorded as single-group, with no masking and allocation listed as not applicable. There is no comparator group described, so the design does not provide a randomized estimate of a difference between Octanorm and another treatment. This constrains any interpretation to the treatment-group experience, even if a rate were available.
Further limitations arise from missing methodological detail. The registry record does not report the primary analysis population, the number of participants contributing endpoint data, the amount of missing information, the method for handling missing data, the statistical model, or procedures for assessing repeated events and variability. It also does not describe event adjudication or the analysis strategy for the listed outcome measures. The record lists 19 outcome measures and 0 analyses, leaving no analysis-specific results to assess.
The safety record gives participant counts but does not report event-level detail, attribution, or the nature of other adverse events. The primary treatment period is listed as 24 weeks, but the registry record does not give the actual exposure time used in the primary rate analysis. The intervention description is also incomplete in the registry record. These reporting limitations do not establish that the underlying study lacked these procedures or data; they limit what can be concluded from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases — NCT03988426.
- Registry record last updated 2020-03-10; status last verified 2020-02.
- Results first posted 2020-03-10.
- 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
NCT03988426 is recorded as a completed phase 3 study of Octanorm in patients with primary immunodeficiency diseases. It used a single-group model, had no masking, and lists allocation as not applicable. Enrollment is recorded as 25. The listed start date is 2017-03-07 and primary completion date is 2018-01-26.
The intervention was subcutaneous human normal immunoglobulin, described as a 16.5% liquid formulation administered at weekly intervals. The primary endpoint was the number of serious bacterial infections per patient-year on treatment during a primary treatment period of 24 weeks. The registry record does not report the endpoint result or its statistical analysis.
For the Octanorm group, the safety record lists serious adverse events for 0 of 25 participants at risk, other adverse events for 18 of 25 participants at risk, and 0 deaths. The record lists 19 outcome measures and 0 analyses. It does not report a comparator group or analysis details needed to evaluate the primary endpoint.