This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for A Phase 1b/2a Study of the Safety and Pharmacokinetics of Rhu-plasma Gelsolin in Hospitalized Subjects With CAP (NCT03466073), a randomized phase 1, phase 2 study. The primary outcome is Number of Participants With Treatment-Emergent Adverse Events (TEAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03466073 |
| Phase | Phase 1, Phase 2 |
| Condition | Community-acquired Pneumonia |
| Allocation | Randomized |
| Design model | Sequential |
| Masking | Quadruple |
| Enrollment | 33 |
| Arms in the primary outcome | n/a |
| Lead sponsor | BioAegis Therapeutics Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Hospitalized subjects with acute community-acquired pneumonia (CAP) were eligible for the study. The registry describes the condition as community-acquired pneumonia. Further eligibility criteria and a detailed description of the population analyzed for the primary endpoint are not reported in the registry record.
Intervention
The intervention was recombinant human plasma gelsolin (rhu-pGSN), given intravenously in addition to standard of care. The dose-escalation regimens included a single dose of 6 mg/kg and once-per-day dosing for 3 days at 6 mg/kg, 12 mg/kg, or 24 mg/kg.
Comparator
The comparator was normal saline placebo, also given in addition to standard of care. The listed arms included single-dose and multiple-dose placebo groups. Details of the standard-care treatments are not reported in the registry record.
Primary question
The primary endpoint was the number of participants with treatment-emergent adverse events (TEAEs) during 0-28 days. The registry defines these as adverse events occurring after enrollment. The clinical question is whether the posted safety counts differ across the rhu-pGSN dose regimens and placebo groups; the record does not report a single statistical comparison answering that question.
3. Trial Design
This was a Phase 1b/2a study of recombinant human plasma gelsolin in subjects hospitalized for acute community-acquired pneumonia. Its official title describes a double-blind, placebo-controlled, dose-escalation study of safety, pharmacokinetics, and pharmacodynamics when the study drug was added to standard of care. The registry identifies the phases as Phase 1 and Phase 2, allocation as randomized, masking as quadruple, and the model as sequential.
The listed regimens comprised a single intravenous dose of 6 mg/kg and multiple-dose regimens of 6 mg/kg, 12 mg/kg, or 24 mg/kg once per day for 3 days. Each was compared with a saline placebo regimen, with treatment added to standard of care. The registry lists a single-dose placebo arm and a multiple-dose placebo arm. The exact sequence or rules for moving between dose levels are not reported in the registry record; the word sequential alone does not establish those details.
Enrollment was 33. The study start date was 2018-08-28, and the primary completion date was 2019-04-02. Its status is completed. BioAegis Therapeutics Inc. is listed as the sponsor.
The secondary outcomes listed concern pharmacokinetics: area under the rhu-pGSN concentration-time curve and maximum observed rhu-pGSN plasma concentration (Cmax). The record lists five outcome measures, but does not provide statistical analyses for a primary group comparison. Details of randomization procedures, allocation concealment, masking roles, treatment adherence, and the protocol’s statistical analysis plan are not reported in the registry record. The design therefore describes randomized, masked, dose-escalation comparisons, but not enough detail to reconstruct how every design feature was implemented.
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 | 6 mg/kg Rhu-pGSN Single Dose | Placebo Single Dose | 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 24 mg/kg Rhu-pGSN Multiple Ascending Dose | Placebo Multiple Ascending Dose |
|---|---|---|---|---|---|---|
| STARTED | 6 | 2 | 6 | 6 | 6 | 7 |
| COMPLETED | 5 | 2 | 6 | 6 | 6 | 6 |
| NOT COMPLETED | 1 | 0 | 0 | 0 | 0 | 1 |
Reasons for not completing this period:
| Reason | 6 mg/kg Rhu-pGSN Single Dose | Placebo Single Dose | 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 24 mg/kg Rhu-pGSN Multiple Ascending Dose | Placebo Multiple Ascending Dose |
|---|---|---|---|---|---|---|
| Death | 1 | 0 | 0 | 0 | 0 | 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.
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | 6 mg/kg Rhu-pGSN Single Dose | Placebo Single Dose | 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 24 mg/kg Rhu-pGSN Multiple Ascending Dose | Placebo Multiple Ascending Dose |
|---|---|---|---|---|---|---|---|
| Participants analysed | 6 | 2 | 6 | 6 | 6 | 7 | |
| Age, Continuous | years | 62.2 (35 to 81) | 64 (62 to 66) | 69.3 (56 to 83) | 55.3 (45 to 70) | 40 (22 to 66) | 64 (39 to 78) |
| Sex: Female, Male: Female | Participants | 2 | 0 | 5 | 3 | 4 | 3 |
| Sex: Female, Male: Male | Participants | 4 | 2 | 1 | 3 | 2 | 4 |
| Race/Ethnicity, Customized: White | Participants | 6 | 2 | 6 | 6 | 6 | 7 |
| Region of Enrollment: Georgia | Participants | 1 | 1 | 6 | 6 | 6 | 7 |
| Region of Enrollment: Australia | Participants | 5 | 1 | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-Emergent Adverse Events (TEAEs), measured over 0-28 days.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with treatment-emergent adverse events during 0-28 days. The registry defines a treatment-emergent adverse event as an adverse event occurring subsequent to enrollment. It also states that seriousness was judged by the site investigator. The endpoint is expressed as a count of participants, rather than a measure of the intensity or duration of events.
Counting participants with an event is a way to describe how many people experienced a safety event within the stated observation period. It can support a descriptive comparison across treatment groups, but a count alone does not show whether events were related to study treatment, how severe non-serious events were, or how many events each participant experienced. The endpoint title refers to TEAEs generally, while the posted safety groups separately list counts for serious and other adverse events. The registry does not report a single primary-endpoint estimate comparing rhu-pGSN with placebo.
The endpoint is useful in a dose-escalation study because safety is a central question when regimens at different dose levels are being evaluated. However, interpretation depends on the event definitions, how events were collected, which participants were included in each analysis, and whether missing observations affected ascertainment. Those details are not reported in the registry record. The endpoint should therefore be read as a bounded description of recorded events over 0-28 days, not as a complete account of every possible safety outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports safety counts by dose regimen and placebo group, but does not report an inferential result for a single comparison of the primary endpoint. In the 6 mg/kg single-dose rhu-pGSN group, the serious-adverse-event count is 1 among 6 at risk, the other-adverse-event count is 3 among 6 at risk, and the death count is 1. In the single-dose placebo group, the corresponding counts are 0 among 2 at risk for serious events, 2 among 2 at risk for other events, and 0 deaths.
For multiple dosing, the 6 mg/kg rhu-pGSN group has 0 serious events among 6 at risk and 4 other events among 6 at risk, with 0 deaths. The 12 mg/kg group has 0 serious events among 6 at risk and 3 other events among 6 at risk, with 0 deaths. The 24 mg/kg group has 0 serious events among 6 at risk and 2 other events among 6 at risk, with 0 deaths. The multiple-dose placebo group has 1 serious event among 7 at risk, 4 other events among 7 at risk, and 1 death. These are descriptive counts; they do not establish a treatment effect or a causal relationship between a treatment and an event.
The registry also lists Pneumonia at 16.7% and Pulmonary Embolism at 14.3% as top serious terms. These percentages are presented without a group-specific breakdown or a stated denominator in the record. They should not be used to infer comparative risks between regimens.
No p-value, confidence interval, adjusted estimate, or formal comparison for the primary endpoint is reported in the registry record. The record indicates that the primary outcome has several categories or time points and was not reduced to one comparison; it lists no analyses. Consequently, the posted counts do not quantify the precision of a treatment contrast. Small group sizes also mean that each recorded participant can have a visible influence on a group’s count, but the record does not provide an uncertainty estimate to characterize that variation.
These results describe the registry’s reported safety information for the stated observation period. They do not establish that rhu-pGSN caused or prevented an event, nor do they establish equivalence between any dose regimen and placebo. A difference in counts, or the absence of a recorded event in a group, is not by itself a statistical test or evidence of no risk. Interpretation is further limited by the lack of reported analysis populations, missing-data handling, event-level detail, and a prespecified comparison plan.
9. Statistical Methods Explained
For a binary safety outcome, the basic descriptive quantity is the count of participants recorded as having an event, interpreted alongside the number at risk. Such a count is not the same as a count of all event episodes: one participant may experience more than one event, while the endpoint as worded concerns participants with events. The registry’s safety information distinguishes serious adverse events from other adverse events. Without further definitions or participant-level data, those categories should be read as the categories reported, not reclassified or combined.
What does a confidence interval add?
In general, a confidence interval describes the uncertainty around an estimated quantity under a statistical model. It can show which values are reasonably compatible with the observed data and the method used. A point estimate alone does not communicate that uncertainty. The registry record does not report a confidence interval for the primary endpoint or a comparative estimate with an interval. Therefore, the precision of a difference between treatment groups cannot be assessed from an inferential result in the record.
What would a treatment comparison require?
In general, a comparative analysis needs a clearly defined outcome, a population for analysis, a contrast between specified groups, and a statistical method appropriate to the outcome and design. Randomization can support a causal comparison when the analysis preserves the randomized groups and the assumptions of the analysis are reasonable. It does not remove the need to report the analysis method, the data included, and the uncertainty around the estimate. The registry identifies randomization but does not report the primary endpoint’s statistical analysis plan or an estimate for a specified contrast.
The posted groups include different dose regimens and placebo groups, rather than a single dose-versus-placebo contrast. Combining dose levels or choosing a particular comparison would require an analysis decision that is not specified in the record. A trend across dose levels also cannot be established by simply reading the separate counts; a dose-response analysis would require a stated method and suitable data. Neither a dose-response result nor a formal comparison is reported in the registry record.
Why does event classification matter?
Seriousness and treatment-emergent timing describe different features of an adverse event. The registry says that TEAEs occurred after enrollment and that the site investigator judged seriousness. A serious event is not automatically caused by study treatment, and an event occurring after enrollment is not automatically attributable to treatment. Attribution, severity grading, event duration, and event-level narratives are not reported in the registry record. The two labels should not be treated as evidence of causation.
Counts are especially sensitive to how events are collected and coded. In general, studies can use prespecified definitions, clinical review, and consistent follow-up to improve comparability. The record does not describe its coding rules or adjudication procedures. The listed serious terms, Pneumonia at 16.7% and Pulmonary Embolism at 14.3%, add descriptive context, but their denominators and group assignments are not reported in the registry record. Those terms therefore cannot be turned into a reliable between-group contrast from the information reported.
How should planning calculations be interpreted?
In general, sample-size calculations estimate how many participants a future study might need under stated assumptions. They are planning illustrations, not evidence about the probability that a completed study detected an effect. The registry’s stated assumptions describe 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. The record also specifies that non-time-to-event calculations are expressed as participants per arm. Those assumptions do not establish the trial’s own design rationale, and the registry does not report a sample-size justification for this study.
Over-dispersion is a general concern when counts vary more than a chosen model expects. If a count-based outcome is analyzed with a model that assumes less variability than the data contain, uncertainty can be understated. This study’s primary endpoint is described as a count of participants with an event, not a count of repeated event episodes; the record does not report an inferential count model or dispersion estimate. It would therefore be inappropriate to claim that over-dispersion was assessed or addressed.
Finally, no statistical analyses are listed for the study’s primary endpoint. That means there is no reported model, adjustment set, hypothesis test, or confidence interval to interpret. General statistical principles can explain what such methods would contribute, but they cannot substitute for analyses not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple dose regimens, placebo groups, safety categories, and pharmacokinetic outcomes. When analyses make several comparisons or examine several outcomes, the chance of obtaining an apparently notable finding by chance can increase. Statistical methods may address this multiplicity, but the appropriate approach depends on which comparisons were prespecified and what claims the analysis is intended to support. A multiplicity strategy is not reported in the registry record.
The primary endpoint is described as the number of participants with TEAEs, and the record indicates that the primary outcome has several categories or time points and was not reduced to one comparison. No analyses are listed. Accordingly, there is no reported primary contrast, hierarchy of outcomes, or adjustment for multiple testing to interpret. The record does not report whether other analyses were planned or exploratory.
Missing-data handling and analysis populations are also not reported in the registry record. The posted at-risk counts describe the denominators associated with the adverse-event groups, but do not by themselves explain how these populations were defined or whether any observations were unavailable. It is not possible to establish from the record whether every randomized participant contributed to each endpoint, how withdrawals were handled, or whether any imputation or sensitivity analysis was used.
In general, missing safety observations can affect interpretation if follow-up or event reporting differs between groups. A complete-case summary may describe only participants with observed data, while other approaches make different assumptions about unobserved outcomes. Without reported population definitions and missing-data methods, neither the direction nor the size of any such influence can be determined. These reporting gaps limit the interpretation of both the descriptive counts and any planning calculation based on an observed effect.
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 |
|---|---|---|---|
| 6 mg/kg Rhu-pGSN Single Dose | 1 | 6 | 16.7% |
| Placebo Single Dose | 0 | 2 | 0.0% |
| 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 0 | 6 | 0.0% |
| 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 0 | 6 | 0.0% |
| 24 mg/kg Rhu-pGSN Multiple Ascending Dose | 0 | 6 | 0.0% |
| Placebo Multiple Ascending Dose | 1 | 7 | 14.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| 6 mg/kg Rhu-pGSN Single Dose | 1 of 6 |
| Placebo Single Dose | 0 of 2 |
| 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 0 of 6 |
| 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 0 of 6 |
| 24 mg/kg Rhu-pGSN Multiple Ascending Dose | 0 of 6 |
| Placebo Multiple Ascending Dose | 1 of 7 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| 6 mg/kg Rhu-pGSN Single Dose | 3 of 6 |
| Placebo Single Dose | 2 of 2 |
| 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 4 of 6 |
| 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 3 of 6 |
| 24 mg/kg Rhu-pGSN Multiple Ascending Dose | 2 of 6 |
| Placebo Multiple Ascending Dose | 4 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | 6 mg/kg Rhu-pGSN Single Dose | Placebo Single Dose | 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 24 mg/kg Rhu-pGSN Multiple Ascending Dose | Placebo Multiple Ascending Dose |
|---|---|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | 1 of 6 | 0 of 2 | 0 of 6 | 0 of 6 | 0 of 6 | 1 of 7 |
| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | 0 of 6 | 0 of 2 | 0 of 6 | 0 of 6 | 0 of 6 | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 6 mg/kg Rhu-pGSN Single Dose | Placebo Single Dose | 6 mg/kg Rhu-pGSN Multiple Ascending Dose | 12 mg/kg Rhu-pGSN Multiple Ascending Dose | 24 mg/kg Rhu-pGSN Multiple Ascending Dose | Placebo Multiple Ascending Dose |
|---|---|---|---|---|---|---|---|
| Headaches | Nervous system disorders | 1 of 6 | 0 of 2 | 1 of 6 | 0 of 6 | 0 of 6 | 1 of 7 |
| Hypotension | Vascular disorders | 1 of 6 | 1 of 2 | 0 of 6 | 0 of 6 | 0 of 6 | 1 of 7 |
| Nausea | Gastrointestinal disorders | 0 of 6 | 0 of 2 | 1 of 6 | 1 of 6 | 0 of 6 | 0 of 7 |
| Blood pressure increased | Investigations | 0 of 6 | 0 of 2 | 2 of 6 | 0 of 6 | 0 of 6 | 0 of 7 |
| Fatigue | General disorders | 0 of 6 | 0 of 2 | 1 of 6 | 0 of 6 | 0 of 6 | 1 of 7 |
| Pyrexia | General disorders | 0 of 6 | 0 of 2 | 1 of 6 | 0 of 6 | 0 of 6 | 1 of 7 |
| Hypertension | Vascular disorders | 0 of 6 | 0 of 2 | 0 of 6 | 1 of 6 | 0 of 6 | 1 of 7 |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 1 of 6 | 0 of 2 | 0 of 6 | 0 of 6 | 0 of 6 | 0 of 7 |
| Neck pain | Musculoskeletal and connective tissue disorders | 1 of 6 | 0 of 2 | 0 of 6 | 0 of 6 | 0 of 6 | 0 of 7 |
| Blood alkaline phosphatase increased | Investigations | 0 of 6 | 0 of 2 | 0 of 6 | 1 of 6 | 0 of 6 | 0 of 7 |
Serious events with the highest arm-level rate: pneumonia (16.7%), pulmonary embolism (14.3%).
The serious-adverse-event figures are reported by treatment group, with an at-risk count and an affected-participant count. For the single-dose rhu-pGSN group, 1 among 6 at risk had a serious adverse event; the single-dose placebo group had 0 among 2. For the multiple-dose groups, the counts were 0 among 6 for 6 mg/kg rhu-pGSN, 0 among 6 for 12 mg/kg, 0 among 6 for 24 mg/kg, and 1 among 7 for placebo. The registry reports 1 death in the single-dose 6 mg/kg group and 1 death in the multiple-dose placebo group, with 0 deaths in the other listed groups.
These counts should be read as descriptive registry results, not as proof that treatment caused or did not cause an event. The record does not state that the deaths were related to study treatment. The small group denominators mean that a count represents a limited set of observations, and the record does not provide confidence intervals or a formal comparison. Zero recorded serious events in a group does not establish that the regimen has no serious-event risk.
The registry lists Pneumonia at 16.7% and Pulmonary Embolism at 14.3% among the top serious terms. It does not report their group-specific distribution or denominator, so those percentages cannot be used to compare treatment arms. Seriousness was judged by the site investigator, according to the endpoint description.
12. What the Result Does, and Does Not, Mean
The posted results document adverse-event counts across the listed dose regimens and placebo groups during the study’s reported safety follow-up. They show that the registry recorded serious and other adverse events in some groups and no recorded serious events in others. The entries also report deaths in the single-dose 6 mg/kg rhu-pGSN group and the multiple-dose placebo group. These observations are relevant to describing what was recorded, but they are not a statistical estimate of a treatment effect.
The results do not establish that rhu-pGSN caused an adverse event, prevented one, or had a particular effect on mortality. They do not show that the dose regimens are equivalent to placebo. The absence of a recorded event in a group is not proof that an event could not occur. The record does not provide an inferential comparison, confidence intervals, or enough details about event attribution and analysis populations to support those conclusions.
The study also listed pharmacokinetic outcomes for area under the concentration-time curve and maximum observed plasma concentration. These outcomes address drug exposure, whereas the primary endpoint concerns participants with treatment-emergent adverse events. Their listing does not establish a pharmacokinetic result, and quantitative outcome results for them are not included in the information.
The registry’s planning assumptions describe illustrations based on a fraction of an observed effect, equal allocation between two arms, a normal approximation, and a two-sided significance level of 0.05. Such planning calculations should not be interpreted as observed power or as evidence that the completed study could reliably detect a safety difference. The registry does not report a sample-size justification for the trial.
Overall, these results are a limited descriptive account from a dose-escalation study. The completed status and the posted event counts are details about the registry record; they do not resolve the safety profile of each regimen or establish comparative efficacy. More detailed outcome definitions, follow-up information, analysis methods, and uncertainty estimates are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for the way its design and outcome reporting constrain interpretation. It used randomized, quadruple-masked allocation and a sequential, dose-escalation model, with rhu-pGSN regimens spanning a single dose and multiple-dose levels. Such a design can generate early descriptive safety and pharmacokinetic information across regimens, but interpretation depends on the specific comparisons and methods prespecified for the study.
The registry’s primary endpoint is a participant count for TEAEs over 0-28 days. The posted safety results separate serious from other adverse events and give at-risk counts by group. That distinction makes it possible to describe the recorded events without claiming that a group-level difference is statistically established. The record does not report a single primary comparison, a confidence interval, a p-value, or a model. This illustrates why raw event counts and formal inference answer different questions.
The multiple arms also make the choice of comparison important. A conclusion about a particular dose, all doses combined, or a pattern across doses would require a clearly specified analysis. Without a reported multiplicity strategy or analysis plan, readers cannot determine which comparisons were intended to support confirmatory claims. The registry does not report such a strategy.
Finally, the record highlights the difference between planning illustrations and evidence from a completed trial. A sample-size calculation under stated assumptions can help plan future research, but it cannot replace a reported estimate with uncertainty, and it is not observed power. Enrollment was 33, while the group-level safety counts are reported separately. Statistical interpretation should stay with those reported details and avoid extending them into conclusions the registry does not support.
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 does not report detailed eligibility criteria, baseline characteristics, or the analysis population for the primary endpoint. Without that information, it is difficult to assess how representative the participants were or whether the analyzed groups correspond to all randomized participants. The listed at-risk counts provide group-specific denominators for the safety information, but the record does not explain their derivation.
The study was a dose-escalation study with single-dose and multiple-dose regimens. The rationale for the dose sequence, rules for escalation, and any stopping criteria are not reported in the registry record. The randomization method, allocation concealment, and the individuals covered by quadruple masking are also not described. The masking label alone does not establish how those procedures were implemented.
Safety reporting is limited to the listed counts and serious terms. The record does not provide event-level descriptions, attribution to treatment, severity grading beyond seriousness, timing by group, duration, resolution, or the method used to code events. The seriousness judgment is attributed to the site investigator, but additional adjudication information is not reported. The denominator and group distribution for Pneumonia at 16.7% and Pulmonary Embolism at 14.3% are not reported in the registry record.
No statistical analysis is listed for the primary endpoint. A prespecified comparison, statistical model, uncertainty estimate, multiplicity approach, missing-data procedure, and analysis-population definition are not reported. These omissions prevent assessment of the precision and robustness of any treatment contrast. The record also does not provide a sample-size justification, so the enrollment figure alone cannot establish whether the study was designed to estimate or detect a particular effect.
The reported safety period is 0-28 days. Longer-term follow-up and outcomes beyond that timeframe are not described in the primary endpoint information. These limitations mean the counts should be interpreted as a registry-based description of the reported follow-up, not a complete safety characterization.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase 1b/2a Study of the Safety and Pharmacokinetics of Rhu-plasma Gelsolin in Hospitalized Subjects With CAP — NCT03466073.
- 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
NCT03466073 is a completed randomized, quadruple-masked, sequential dose-escalation study of rhu-pGSN added to standard of care for hospitalized subjects with acute community-acquired pneumonia. The record lists Phase 1 and Phase 2, enrollment of 33, and BioAegis Therapeutics Inc. as sponsor. The study start date was 2018-08-28, and the primary completion date was 2019-04-02.
The primary endpoint was the number of participants with TEAEs during 0-28 days. The registry reports serious and other adverse-event counts and deaths by treatment group, as well as Pneumonia at 16.7% and Pulmonary Embolism at 14.3% among top serious terms. It lists pharmacokinetic outcomes for area under the concentration-time curve and maximum observed plasma concentration.
No primary-endpoint analysis or formal comparison is listed. Details of analysis populations, missing-data handling, multiplicity, and the statistical plan are not reported in the registry record. The reported counts therefore support descriptive reading only; they do not establish a causal or comparative treatment effect.