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 Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD) (NCT01814800), a na phase 3 study. The primary outcome is Number of Serious Bacterial Infections (SBIs) Per Subject Per Year (FDA Guidance for Industry (2008)).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01814800 |
| Phase | Phase 3 |
| Condition | Primary Immune Deficiency Disorder |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 59 |
| Arms in the primary outcome | n/a |
| Lead sponsor | ADMA Biologics, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled subjects with primary immunodeficiency diseases, with the primary endpoint description specifying primary humoral immunodeficiency. The primary analysis included 59 treated subjects. The registry’s brief summary describes the intended study population as approximately 60 subjects.
Intervention
Participants received RI-002, an intravenous immunoglobulin product, by infusion. The arm description gives a dose of 300-800 mg/kg, administered once every 3 to 4 weeks. The brief summary describes administration every 21 or 28 days.
Comparator
There was no concurrent comparison group. The study is described as single-group, open-label, and without masking. Its allocation is listed as not applicable. Consequently, the posted results do not provide a randomized comparison with another treatment or with a control group.
Primary question
The primary objective was to assess whether RI-002 reduced the frequency of serious bacterial infections, or SBIs, as defined by the Diagnostic Criteria for Serious Infection Types guideline. The primary endpoint was the number of SBIs per subject per year over one year. The registry record does not report the observed endpoint value or a statistical analysis of it, so the stated objective cannot be evaluated from the posted primary result.
3. Trial Design
This was a Phase III, multicenter, open-label study of RI-002 in subjects with primary immunodeficiency diseases. The study record identifies a single treatment group and no masking. Allocation is listed as not applicable, consistent with the absence of a randomized comparison. The registry lists ADMA Biologics, Inc. as the sponsor and the study status as completed.
The record gives a study start of 2014-02 and a primary completion of 2014-12. It records an enrollment of 59. The primary endpoint population description states that the analysis included 59 treated subjects and that they contributed a total of 55.88 subject-years of treatment with RI-002. Those figures describe the treated population and treatment exposure used in the endpoint description; they do not supply the observed SBI count or rate.
RI-002 was administered by intravenous infusion. The arm description specifies a dose of 300-800 mg/kg once every 3 to 4 weeks, while the brief summary describes dosing every 21 or 28 days. The study record lists several secondary outcomes concerning infections, infection-related lost days, unscheduled medical visits, time to resolution, and hospitalizations. It lists 34 outcome measures overall.
Because the study had a single group and was open-label, its design can describe outcomes during treatment but does not create a concurrent untreated or active-control comparison. The registry record does not report a statistical analysis plan, sample-size justification, interim-analysis plan, or details of any planned adjustments. Although the study is completed, the primary endpoint result and its inferential analysis 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
| Stage | RI-002 Treatment |
|---|---|
| STARTED | 59 |
| COMPLETED | 54 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | RI-002 Treatment |
|---|---|
| Adverse Event | 2 |
| Pregnancy | 1 |
| Sponsor decision | 1 |
| Subject relocation | 1 |
Analysis population for the primary outcome, as defined in the registry: Analysis included 59 treated subjects, with a total of 55.88 subject-years of treatment with RI-002
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 | RI-002 Treatment |
|---|---|---|
| Participants analysed | 59 | |
| Age, Categorical: <=18 years | Participants | 13 |
| Age, Categorical: Between 18 and 65 years | Participants | 35 |
| Age, Categorical: >=65 years | Participants | 11 |
| Age, Continuous | years | 41.8 (3 to 74) |
| Sex: Female, Male: Female | Participants | 31 |
| Sex: Female, Male: Male | Participants | 28 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 56 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| 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 | 1 |
| Race (NIH/OMB): White | Participants | 58 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 59 |
| PIDD Type: Common Variable Immunodeficiency | participants | 45 |
| PIDD Type: X-Linked Agammaglobulinemia | participants | 6 |
| PIDD Type: Antibody Deficiencies | participants | 4 |
| PIDD Type: Other | participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Serious Bacterial Infections (SBIs) Per Subject Per Year (FDA Guidance for Industry (2008)), measured over One year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | SBIs/subject/year |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of serious bacterial infections per subject per year. It is a count-rate measure: it concerns how many qualifying infections occurred in relation to the time participants were observed under treatment, rather than simply whether a participant ever had an infection. The registry specifies a one-year timeframe and defines SBIs by reference to the Diagnostic Criteria for Serious Infection Types guideline.
Rates are useful when participants contribute different amounts of observation time. Expressing a count in relation to person-time can make the amount of observed time explicit and can support comparison of event frequency across groups or periods. This is a general statistical rationale; the registry record does not describe the calculation details used for this endpoint.
The endpoint was chosen to address the stated objective of assessing the frequency of serious bacterial infections in subjects with primary humoral immunodeficiency receiving RI-002. Its clinical interpretation depends on the event definition, the amount of time under observation, and the analysis used to estimate the rate. The record reports that 59 treated subjects contributed a total of 55.88 subject-years, but it does not report the number of SBIs, an estimated rate, uncertainty around that estimate, or a hypothesis test. Therefore, the endpoint’s observed result and precision cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes the primary question and the population contributing treatment exposure, but it does not post the primary outcome value. In particular, it gives no observed number of serious bacterial infections, estimated SBI rate per subject per year, confidence interval, or test result. The stated objective is therefore not accompanied by the result needed to judge how the observed infection frequency compared with any benchmark or expected frequency.
The primary endpoint population description reports 59 treated subjects and a total of 55.88 subject-years of treatment. These details establish the amount of experience included in the endpoint description, but exposure time alone does not determine the infection rate. That rate also requires the event count, which is not reported in the registry record. A confidence interval would additionally be needed to show how much uncertainty surrounds the estimated rate. Neither is available in the posted primary result.
The study’s single-group, open-label design also limits what can be concluded from a treatment-period outcome. Without a concurrent comparison group, the study does not directly estimate a difference between RI-002 and another treatment or control condition. General changes over time, participant characteristics, and ascertainment practices can affect observed outcomes in a single group. These are design considerations, not evidence that any particular influence occurred in this study.
The record lists 34 outcome measures, including several secondary infection-related outcomes, but reports no analyses. The existence of outcome descriptions does not establish what their observed values were or whether they support a particular interpretation. The record also does not state how results for multiple outcomes were handled statistically.
Accordingly, the registry supports a description of the study design, population, treatment exposure, and intended endpoint. It does not support a quantitative assessment of the primary SBI result or the precision of an estimated treatment-period rate. No conclusion about a treatment effect can be drawn from the endpoint information posted in the registry record alone.
9. Statistical Methods Explained
What does a count-rate endpoint represent? A count endpoint records how often events occur. When observation time differs across participants, a rate relates the event count to the time at risk or under observation. This general approach distinguishes an event frequency from a simple proportion of participants who experienced an event. The primary outcome in this study is specified as SBIs per subject per year. The registry record gives the total treatment exposure used in the endpoint description, but does not give the SBI count or explain the exact rate-estimation procedure.
Why does person-time matter? Participants may contribute different amounts of observation time, for example if follow-up ends at different points. A rate denominator accounts for the time contributing to observation, while the count in the numerator represents qualifying events. In general, the interpretation requires clear definitions of when observation begins and ends, how time at risk is handled after an event, and whether repeated events can contribute. Those implementation details are not reported in the registry record. The reported total of 55.88 subject-years describes treatment exposure but cannot substitute for the missing event count.
How should uncertainty be assessed? A point estimate alone would not show how precisely a rate has been estimated. A confidence interval is a range of values compatible with the data under the specified statistical method; a wider interval generally signals less precision. For event counts, uncertainty depends on both the number of events and the model assumptions. The registry record does not report a primary rate estimate or a confidence interval, so the precision of the primary endpoint cannot be assessed. No p-value or other inferential result for the primary endpoint is reported either.
Why can event-count methods need special care? General count models often begin with assumptions about how event counts vary in relation to exposure. If observed counts vary more than a chosen model expects, this is called over-dispersion. Ignoring over-dispersion can make uncertainty appear smaller than it should. Repeated events within a participant may also be related rather than independent, so an analysis may need to account for clustering. Whether the study assessed over-dispersion, within-participant dependence, or alternative count models is not reported in the registry record. No model should be attributed to the study without that information.
What does the single-group design imply for statistical interpretation? A single-group study can estimate outcomes observed among treated participants, subject to the endpoint definition and analysis. It does not, by itself, estimate a randomized between-group treatment contrast. A comparison with a historical threshold or external data would require a stated benchmark and careful consideration of differences in population, event definitions, follow-up, and ascertainment. The registry record does not report such a benchmark or a comparator analysis. The objective’s wording about reducing infection frequency does not establish that a particular comparison was performed.
What can sample-size calculations say? A sample-size calculation is a planning exercise based on specified assumptions; it is not an observed result and is not observed power. The planning assumptions described for the illustrations use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. Those assumptions describe planning illustrations, not the single-group trial design. The registry record does not report the trial’s own sample-size justification or planned power calculation. It also does not provide the observed primary effect needed to interpret any effect-based illustration. Any planning estimate must therefore be kept separate from evidence about the study outcome.
What does the record reveal about analysis conduct? The registry record lists 34 outcome measures and no analyses. It does not describe the statistical model, covariate adjustment, confidence-interval method, handling of recurrent infections, or sensitivity analyses. It also does not report whether any interim analyses were planned or performed. Without these details and without the primary result, readers cannot verify how the stated endpoint was operationalized or judge whether the analysis accounted for relevant features of the data.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 34 outcome measures, including the primary SBI rate and secondary measures related to infections, time lost, unscheduled visits, infection resolution, and hospitalization. When a study assesses multiple outcomes, the chance of at least one apparently unusual result can increase if each is tested separately without accounting for multiplicity. This is a general statistical issue, not a statement that a particular adjustment was required or performed in this study. The record does not report a multiplicity strategy, a hierarchy of outcomes, or which outcomes were intended for confirmatory inference.
The registry lists no analyses. It therefore does not provide results for the primary endpoint or the secondary outcomes, nor does it indicate whether any multiplicity adjustment was applied. The record also does not describe missing-data procedures, participant withdrawal handling, event adjudication completeness, or sensitivity analyses. Whether outcome data were missing, and how any missingness was addressed, is not reported in the registry record.
The endpoint description states that the analysis included 59 treated subjects and a total of 55.88 subject-years. This describes the population and exposure reported for the primary endpoint, but the record does not label the population as intention-to-treat, per-protocol, or another analysis set. It does not explain whether all treated subjects contributed complete endpoint information or how person-time was assigned. Those details should not be inferred from the participant count alone.
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 |
|---|---|---|---|
| RI-002 Treatment | 2 | 59 | 3.4% |
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 |
|---|---|
| RI-002 Treatment | 58 of 59 |
Most frequent serious adverse event terms
| Event term | Organ system | RI-002 Treatment |
|---|---|---|
| Postoperative wound infection | Infections and infestations | 1 of 59 |
| Migraine | Nervous system disorders | 1 of 59 |
Most frequent non-serious adverse event terms
| Event term | Organ system | RI-002 Treatment |
|---|---|---|
| Sinusitis | Infections and infestations | 16 of 59 |
| Gastroenteritis viral | Infections and infestations | 13 of 59 |
| Nasopharyngitis | Infections and infestations | 13 of 59 |
| Upper respiratory tract infection | Infections and infestations | 13 of 59 |
| Nausea | Gastrointestinal disorders | 12 of 59 |
| Bronchitis | Infections and infestations | 12 of 59 |
| Acute sinusitis | Infections and infestations | 11 of 59 |
| Cough | Reproductive system and breast disorders | 9 of 59 |
| Fatigue | General disorders | 8 of 59 |
| Viral upper respiratory tract infection | Infections and infestations | 8 of 59 |
Serious events with the highest arm-level rate: postoperative wound infection (1.7%), migraine (1.7%).
The registry’s adverse-event summary lists 59 participants at risk for serious events and 2 affected in the serious-event count. It lists 59 at risk for other events and 58 affected in that count. These are registry summary counts; they do not give event-level detail, timing, duration, or a comparison with another group. The record does not report a death count, so the missing death value should not be read as evidence that no deaths occurred.
The listed serious-event terms are postoperative wound infection and migraine, each reported as 1.7%. These are the terms and percentages identified in the registry summary. The registry record does not provide additional details about these events or establish their relationship to RI-002. A term appearing in an adverse-event listing is not, by itself, evidence of causation.
Safety counts in a single-group, open-label study describe what was recorded among participants receiving treatment. Without a concurrent comparator, the counts do not quantify whether an event was more or less frequent than it would have been under another condition. The summary also does not replace a detailed review of event definitions and reporting methods, which are not described in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a completed, open-label, single-group Phase III study of RI-002 in subjects with primary immunodeficiency diseases. It identifies serious bacterial infections per subject per year as the primary endpoint and reports the treated population and treatment exposure used in the endpoint description. These registry entries clarify what the study set out to measure and the broad context in which the primary outcome was intended to be interpreted.
However, the record does not report the observed SBI count or rate, a confidence interval, or a statistical test. It therefore does not show the size or precision of the primary result. The stated aim of reducing infection frequency is an objective, not a reported finding. The registry information available cannot establish whether the observed rate was high or low, or how it related to a specified benchmark.
The single-group design also means there is no concurrent randomized control group against which to estimate a treatment difference. Any observed treatment-period frequency, if reported elsewhere, would need to be interpreted in light of that design and the way infections were defined and counted. The registry record does not report a comparator-based estimate.
The adverse-event summary provides counts for serious and other events and lists two serious-event terms with percentages. Those entries describe the registry’s safety summary; they do not establish that RI-002 caused the events or provide a comparative safety estimate. The record does not report a death count.
In short, the posted information describes study conduct and selected safety counts but leaves the primary efficacy result and its uncertainty unreported. No treatment-effect conclusion follows from the stated objective or the treatment-exposure figures alone.
13. Why This Trial Matters Statistically
This study illustrates a basic distinction in clinical-trial interpretation: a clearly stated endpoint is not the same as a reported endpoint result. The registry identifies a clinically framed count-rate outcome, serious bacterial infections per subject per year, and describes the population and treatment exposure included in its analysis. Yet without the event count, estimated rate, and measure of uncertainty, readers cannot assess the magnitude or precision of the primary finding.
It also highlights the importance of design when interpreting a treatment-period outcome. In a single-group, open-label study, the observed experience during treatment is not a randomized contrast against another treatment or control condition. The design can describe events recorded in the treated group, but causal interpretation requires caution. The registry’s allocation and masking information makes that limitation explicit.
The listing of many outcome measures makes multiplicity relevant as a general consideration. A prespecified outcome hierarchy and analysis plan can clarify which questions are confirmatory and how several tests should be interpreted. Those details are not reported in the registry record, and no analyses are listed. The case therefore shows why readers need both results and analysis documentation, not just endpoint names and enrollment information.
Finally, the distinction between planning calculations and observed evidence matters. Sample-size illustrations based on assumptions do not establish the precision achieved in the study, and they are not observed power. For this record, the primary result needed to connect the planned question to the observed data is not reported. Clear statistical interpretation depends on keeping design, analysis, and outcome evidence separate.
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 for interpreting this study from the registry record is that the primary endpoint result is not reported. The event count, estimated SBI rate, confidence interval, and inferential test are absent. Although the endpoint description reports 59 treated subjects and 55.88 subject-years of treatment exposure, those figures are insufficient to reconstruct the primary outcome.
The design has no concurrent comparator and is open-label. As a result, the registry information cannot provide a randomized estimate of the difference between RI-002 and another condition. Open-label status can also matter for how outcomes are observed and recorded, although the registry does not describe whether ascertainment was affected in this study.
The record does not report the statistical analysis plan, sample-size justification, model specification, handling of recurrent events, missing-data methods, interim analyses, or sensitivity analyses. It also does not state how the 34 listed outcome measures were prioritized or whether multiplicity was addressed. The analysis population is described as including treated subjects, but its formal designation and inclusion rules are not reported.
Safety interpretation is limited by the summary format. The registry gives affected counts and selected serious-event terms, but does not provide detailed event-level information or a concurrent comparison. A death count is not reported. These limitations concern what can be learned from the registry record; they should not be mistaken for evidence that unreported analyses or outcomes were absent from all other study materials.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD) — NCT01814800.
- Registry record last updated 2016-10-05; status last verified 2016-08.
- Results first posted 2016-10-05.
- 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
NCT01814800 is a completed Phase III, multicenter, open-label, single-group study of RI-002 in subjects with primary immunodeficiency diseases. The registry lists 59 enrolled participants and describes RI-002 as an intravenous infusion administered at 300-800 mg/kg once every 3 to 4 weeks. The primary endpoint was serious bacterial infections per subject per year over one year.
The endpoint description states that 59 treated subjects were included and contributed 55.88 subject-years of treatment. The registry lists 34 outcome measures and no analyses. It does not report the observed primary SBI result, its precision, or a comparative treatment-effect estimate. The adverse-event summary records 2 affected in the serious-event count and 58 in the other-event count, with 59 at risk in each category; a death count is not reported.