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 Pilot Study Evaluating the Safety and Performance of the GeNO NITROsyl Delivery System for Inhaled Nitric Oxide (NCT01092559), a na phase 2 study. The primary outcome is Incidence and Severity of Treatment Emergent Adverse Events; Unanticipated Adverse Device Effects and Changes From Baseline to End-of-study in Clinical Lab Parameters and Vital Signs..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01092559 |
| Phase | Phase 2 |
| Condition | Pulmonary Arterial Hypertension |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Geno LLC |
| Results posted | Yes |
2. Clinical Question
Population
Participants with pulmonary arterial hypertension undergoing right heart catheterization. The registry lists enrollment as 10. Further eligibility criteria and participant characteristics are not reported in the registry record.
Intervention
Inhaled nitric oxide delivered with the GeNO Nitrosyl system by nasal cannula. The registry summary says participants receive nitric oxide in oxygen or nitric oxide in air. It does not report how those delivery conditions were assigned or whether each participant received both.
Comparator
There is no separate comparator group in the registry design: the study is listed as single-group, with allocation recorded as NA. It is not a randomized or like-for-like controlled comparison.
Primary question
The primary question concerns safety and device performance, including treatment-emergent adverse events, unanticipated adverse device effects, and changes from baseline to end-of-study in clinical laboratory parameters and vital signs. The registry outcome description gives the relevant assessment periods, but the primary outcome results are not reported in the registry record.
This design frames the study as an initial evaluation in a defined clinical setting, rather than a test of comparative benefit. Right heart catheterization provides the setting in which the delivery system is evaluated; the registry summary also lists hemodynamic, laboratory, and clinical assessment data as collected for safety evaluation. It does not report a separate efficacy hypothesis or a control condition against which to interpret those measurements.
The question is therefore descriptive: what safety observations and device-related findings are recorded during the stated treatment and follow-up periods? Without reported outcome values, the registry record cannot establish the frequency or severity of events or the direction and size of changes in laboratory tests or vital signs. Those details are necessary to interpret the observed safety profile.
3. Trial Design
The registry describes NCT01092559 as an open-label Phase 2 pilot study evaluating preliminary safety and performance of the GeNO Nitrosyl delivery system. Its design is listed as single-group, and allocation is recorded as NA. Masking is listed as none. There is no separate control arm in the record, so the design does not support a randomized comparison between nitric oxide delivery and another treatment or device.
The registry lists enrollment as 10 and the status as COMPLETED. The recorded study start is 2010-10, and primary completion is listed as 2011-07. These are registry dates and status details; they do not supply outcome results or explain the conduct of individual visits.
Participants with pulmonary arterial hypertension were to receive inhaled nitric oxide in oxygen or nitric oxide in air, delivered by nasal cannula during right heart catheterization. The registry identifies the intervention as nitric oxide generated by the GeNO Nitrosyl system. It does not report how the oxygen or air delivery condition was selected, whether the same participant experienced both conditions, or the sequence of any exposure.
The study summary says that hemodynamics, clinical laboratory data, and clinical assessment data were collected on all participants to evaluate safety. It does not specify the instruments, measurement schedule beyond the endpoint timeframes, or criteria for classifying an abnormal finding. Detailed eligibility criteria, participant baseline characteristics, and protocol deviations are not reported in the registry record.
Because the study is single-group and open-label, its results, if reported, would be interpreted as observations from participants receiving the device-delivered intervention under the study conditions. They would not by themselves isolate the effect of nitric oxide or the delivery system from other influences, nor provide a direct estimate against a concurrent control group. The record does not report a statistical analysis plan that would clarify how any such limitations were addressed.
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 | Nitric Oxide Via GeNO Nitrosyl System |
|---|---|
| STARTED | 10 |
| COMPLETED | 10 |
| NOT COMPLETED | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Nitric Oxide Via GeNO Nitrosyl System |
|---|---|---|
| Participants analysed | 10 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 9 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age Continuous | years | 51 (SD 15.17) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 2 |
| Region of Enrollment: United States | participants | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Incidence and Severity of Treatment Emergent Adverse Events; Unanticipated Adverse Device Effects and Changes From Baseline to End-of-study in Clinical Lab Parameters and Vital Signs., measured over through Day 30 Follow-up Period.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint combines several kinds of safety and device observations. Its title covers the incidence and severity of treatment-emergent adverse events, unanticipated adverse device effects, and changes from baseline to end-of-study in clinical laboratory parameters and vital signs. The endpoint unit is participants, and its parameter is described as a number. The registry classifies the outcome as binary, with the stated meaning of participants with an event (counts).
These components address different concerns. Adverse-event monitoring records clinical events and their severity. Device-effect monitoring concerns system malfunction, damage, or nitric oxide threshold monitor alarms. Laboratory assessments include hematology, chemistry, coagulation-related testing, arterial blood gas, and methemoglobin. Vital-sign assessments include pulse, blood pressure, and respiratory rate. The registry lists these measures as part of the safety evaluation, not as evidence of a comparative treatment effect.
The recorded timeframes differ by component. Adverse-event severity is assessed through the Day 30 Follow-up Period. Unanticipated device effects, laboratory tests, and vital signs are described through discharge from the Treatment Period. Those distinctions matter: a result should be interpreted in relation to the specific observation window, and findings from different windows should not be treated as if they covered the same follow-up.
In general, safety endpoints are used to organize observations that may indicate tolerability concerns or device-related problems. Combining event reporting with laboratory and vital-sign monitoring can capture different types of potential safety signals. However, the endpoint title alone does not show what was observed. The primary outcome values and any participant-level changes are not reported in the registry record, so the endpoint's intended scope can be described but its results cannot be assessed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a safety-focused pilot study, but it does not report the primary outcome results. Consequently, the incidence or severity of treatment-emergent adverse events, the occurrence of unanticipated adverse device effects, and changes in laboratory parameters or vital signs cannot be characterized from this record. The absence of reported outcome values is not evidence that no events or changes occurred.
The adverse-event summary lists 0 in the at-risk and affected fields for both serious and other events. Since the at-risk fields are also 0, these entries do not establish that participants were assessed and had no events. The record lists no serious event terms, and deaths are recorded as null. These entries are incomplete for drawing a safety conclusion; the registry record does not report an evaluable participant denominator or event results that would resolve that limitation.
The single-group, open-label design also sets the limits of interpretation. Without a separate comparator group, observed measurements cannot be interpreted as differences attributable to the device relative to another delivery approach. The study can be understood as an evaluation conducted in participants with pulmonary arterial hypertension during right heart catheterization, but the record does not provide outcome data with which to characterize even the within-group findings.
For an event outcome, a count of participants with an event answers a different question from a count of all event episodes. A participant may experience more than one event, while a participant-level measure typically records whether the event criterion was met. The registry's stated outcome meaning is participants with an event (counts), but no outcome count is reported. The distinction therefore helps clarify what the endpoint is intended to capture without filling in missing results.
In general, estimates from a small pilot study can be imprecise, particularly for uncommon events. That is a general statistical consideration, not a calculated finding about this trial. The registry lists enrollment as 10, but supplies no confidence interval, event estimate, or analysis from which precision could be evaluated. A confidence interval, when reported, describes uncertainty around an estimate; a wide interval would indicate that a range of values remains compatible with the data. No such interval is reported.
Nothing in the record supports a conclusion about comparative efficacy or the magnitude of a treatment effect. No randomized comparison is listed, and no primary outcome analysis is reported. The study title and objective describe what investigators set out to evaluate; they are not substitutes for observed results. The statistically appropriate reading is therefore limited to the design and the scope of planned safety monitoring, with the actual endpoint findings remaining unreported.
9. Statistical Methods Explained
The record describes a single-group study with no masking and no allocation to a comparator. In a general parallel-group trial, allocation determines which intervention participants receive, and randomization can help balance measured and unmeasured participant characteristics on average. Those concepts do not describe this record: allocation is NA, and the study is not listed as a randomized comparison. A single-group study can describe measurements and events in the treated group, but it does not create a concurrent counterfactual showing what would have happened under another intervention.
What does an event count represent?
The primary outcome is classified as binary, and the record defines its meaning as participants with an event (counts). In general, binary event data classify each participant according to whether a specified event occurred during the relevant observation period. A participant-level count is not the same as a count of event episodes: repeated episodes in the same participant may be handled differently depending on the endpoint definition. The registry record does not report the outcome count, operational definitions for every component, or a fuller analysis description.
The endpoint also includes changes from baseline to end-of-study in laboratory parameters and vital signs. In general, a change outcome requires a defined baseline measurement, a later measurement, and a rule for comparing them. Analysis may use the measured follow-up value, the change from baseline, or a model that accounts for repeated measurements. These approaches answer related but not identical questions. The record names the change-from-baseline concept and assessment measures but does not report the statistical method used, the handling of multiple measurements, or any resulting estimates.
Why do timeframes matter?
An event can only be counted within an observation window that is defined for that outcome. The registry describes adverse-event severity through the Day 30 Follow-up Period. It describes unanticipated device effects, laboratory tests, and vital signs through discharge from the Treatment Period. In general, a longer observation period allows more time for an event to be detected, so event counts from different periods are not directly comparable unless their windows and definitions are considered. The record does not report participant-level follow-up completeness or the actual duration of observation for each participant.
The primary outcome description covers multiple domains. In general, a broad safety endpoint can be useful for organizing monitoring, but each component may have a different clinical meaning and frequency. Reporting component-specific counts and assessment results helps readers distinguish, for example, a device malfunction from a laboratory change. The registry record names the domains but does not give their separate results.
What can a confidence interval add?
In general, a confidence interval expresses the uncertainty around an estimated quantity under a specified statistical method. It helps readers assess the range of values reasonably compatible with the observed data, rather than focusing only on a point estimate. A point estimate without an uncertainty measure can give an incomplete account of precision. No confidence interval or primary outcome estimate is reported in the registry record, so precision cannot be judged from it.
Over-dispersion is a general issue for count data: observed variability may exceed what a chosen model assumes. When that happens, conventional standard errors can understate uncertainty unless the analysis accounts for the extra variability. This concept is relevant when outcomes are counts of repeated events or episodes. The registry's stated primary outcome meaning is participants with an event (counts), and it does not report a model for event episodes or any method for addressing over-dispersion. It would be inappropriate to infer that such a model was used.
How should the planning assumptions be read?
The registry describes a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. For time-to-event tables, the illustration uses a total number of events; for other outcomes, it uses participants per arm. These are planning assumptions, not evidence about how this study was designed or analyzed. In particular, equal allocation between two arms does not match the recorded single-group design. The record does not report a trial-specific sample-size justification, and the planning illustrations are not observed power.
In general, a sample-size calculation estimates how much information a future study might need under chosen assumptions. Its result depends on the target effect, variability or event rate, significance level, allocation, and analysis method. A calculation based on an observed effect can be especially uncertain when the observed estimate is imprecise. The registry record does not report the numeric planning outputs in the registry record for interpretation, so no required sample size can be stated.
No statistical analyses are listed in the record. It does not report whether results were adjusted, which analysis population was used, how missing observations were handled, or whether there were interim analyses. These are important features of an analysis plan, but their absence from the registry record does not establish what procedures were or were not used during trial conduct.
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.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Nitric Oxide Via GeNO Nitrosyl System | 10 | 0 | 0.0% | 0.0% to 30.8% | 0.0% to 27.8% | 0.2% to 28.5% |
R code and output
arm <- c("Nitric Oxide Via GeNO Nitrosyl System")
n <- c(10)
events <- c(0)
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 Nitric Oxide Via GeNO Nitrosyl System 10 0 0.0 0.0 30.8 0.0 27.8 0.2 28.511. Multiplicity, Missing Data and Analysis Populations
The registry lists 2 outcome measures, but only the primary outcome description is available in the record details for this analysis. The other outcome definitions and their results are not reported in the registry record. The record also lists 0 analyses. It therefore does not document a statistical analysis plan, a hierarchy among outcomes, or how multiple comparisons were addressed.
In general, multiplicity arises when many outcomes, time points, subgroups, or statistical tests are examined. Testing many questions can increase the chance of at least one apparently unusual result even when no real effect is present. Prespecifying a primary outcome and controlling or describing multiplicity can help readers distinguish confirmatory findings from exploratory ones. Here, the primary endpoint is identified, but no analysis details or results are reported from which to assess multiplicity handling.
Missing-data methods and analysis populations are also not reported in the registry record. It does not say whether every participant completed each assessment, how missing laboratory or vital-sign values were handled, or whether participants were excluded from particular analyses. In general, missing observations can affect an estimate when the availability of data is related to participant status or outcomes. Complete-case analysis, imputation, and model-based approaches rely on different assumptions, so a clear description is needed to evaluate their implications.
The adverse-event fields list 0 for both at-risk and affected entries in the serious and other categories. With the at-risk entries recorded as 0, those fields do not provide an interpretable participant denominator for event incidence. Deaths are recorded as null, and no serious event terms are listed. The record does not explain these entries or provide additional data that would clarify them. No conclusion about absent events or complete ascertainment should be drawn from these fields alone.
12. What the Result Does, and Does Not, Mean
The record establishes the broad purpose and design: an open-label Phase 2 pilot evaluating preliminary safety and device performance of the GeNO Nitrosyl system in participants with pulmonary arterial hypertension during right heart catheterization. It identifies the intervention, the single-group design, and the safety domains and observation periods named for the primary outcome. It also lists enrollment as 10 and status as COMPLETED.
It does not report the primary endpoint results. As a result, readers cannot determine from the record the number of participants with treatment-emergent adverse events, the severity of those events, whether unanticipated device effects occurred, or how laboratory parameters and vital signs changed. The adverse-event summary fields do not remedy this gap because the at-risk counts are recorded as 0. A blank or incomplete result should not be treated as a zero event rate.
The design also does not answer a comparative question. Because there is no separate comparator group and no random allocation, the record cannot show whether outcomes differ from those under another device, delivery method, or usual care. It also does not establish that any observed change would have been caused by the intervention. These are design limits, not evidence that the intervention had a particular effect.
In general, a pilot study can inform planning and describe feasibility or safety observations, but conclusions depend on the actual measurements, their completeness, and the analysis. The registry entry names the evaluation goals, yet does not include outcome estimates, uncertainty intervals, analysis methods, or detailed participant characteristics. It therefore supports a description of what was intended to be monitored, not a conclusion about the observed safety profile or clinical benefit.
The findings should not be extended beyond the population and setting named in the record. Eligibility details and participant characteristics are not reported in the registry record, and the study is described in relation to participants with pulmonary arterial hypertension undergoing right heart catheterization. The posted information is insufficient to determine how well any findings would apply to other populations or settings.
13. Why This Trial Matters Statistically
This trial is statistically notable for the gap between a clearly stated safety-monitoring objective and the lack of reported outcome results. The registry defines a broad primary endpoint spanning adverse events, device effects, laboratory tests, and vital signs. Without values for those components, readers cannot estimate event frequency, describe changes, or assess precision. The endpoint definition alone is not a finding.
The single-group design makes the distinction between description and comparison particularly important. A safety observation in a treated group may be useful for describing what was recorded under the study conditions, but it does not by itself estimate a difference relative to a control. The record does not report a comparator or an analysis that could support a treatment contrast.
The entry also illustrates why denominators and outcome definitions matter. The adverse-event summary records 0 in the at-risk fields as well as the affected fields. Since the denominator information is not evaluable from those entries, they cannot support an incidence estimate. Clear counts of participants assessed, participants with an event, event definitions, and observation periods are necessary for meaningful interpretation.
Finally, the planning assumptions described in the registry should not be mistaken for the trial's own design or for observed evidence. The stated equal-allocation, two-arm illustration does not correspond to the single-group design. Separating planning calculations from trial results and analysis methods is essential to prevent a hypothetical sample-size exercise from being read as a result of the study. The record does not report a trial-specific sample-size justification or a statistical analysis plan.
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 important limitation for interpreting this trial record is that the primary outcome results are not reported. The endpoint is broad, but no event counts, severity results, device-effect findings, or laboratory and vital-sign changes are available. The adverse-event summary contains 0 in the at-risk fields, so it does not provide an evaluable denominator for estimating incidence.
The design is single-group and open-label, with allocation recorded as NA and masking listed as none. There is no separate control arm for a direct comparison. Any outcome findings, if available elsewhere, would need to be interpreted as observations in the study group rather than as a randomized estimate of a difference between interventions.
The registry record does not report detailed eligibility criteria, participant characteristics, assessment procedures, or participant-level follow-up completeness. It also does not describe missing-data handling, analysis populations, prespecified statistical methods, or adjustment for multiple outcomes. These omissions limit assessment of how complete and comparable the measurements were and how uncertainty was handled.
The primary endpoint spans components with different definitions and timeframes: adverse-event severity through the Day 30 Follow-up Period, and device effects, laboratory tests, and vital signs through discharge from the Treatment Period. The record does not provide component-specific results or explain whether each participant had complete measurements over those windows. The planning illustration's two-arm assumptions also differ from the listed single-group design and should not be treated as a trial-specific justification.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pilot Study Evaluating the Safety and Performance of the GeNO NITROsyl Delivery System for Inhaled Nitric Oxide — NCT01092559.
- Registry record last updated 2013-06-04; status last verified 2013-04.
- Results first posted 2013-06-04.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01092559 is listed as a completed, open-label Phase 2 pilot study evaluating preliminary safety and performance of the GeNO Nitrosyl delivery system. The registry describes a single-group study with no masking and enrollment of 10. The recorded study start is 2010-10, and primary completion is listed as 2011-07.
The intervention is inhaled nitric oxide generated by the GeNO Nitrosyl system and delivered by nasal cannula, with the summary describing nitric oxide in oxygen or in air. The population is participants with pulmonary arterial hypertension undergoing right heart catheterization. The primary outcome covers adverse events, unanticipated device effects, laboratory parameters, and vital signs, with component-specific observation periods.
The registry record does not report the primary outcome results, a statistical analysis plan, or an interpretable adverse-event denominator. Its design supports no direct randomized comparison, and the described two-arm planning assumptions are not the listed study design. Interpretation is therefore limited to the study's stated objective, design, and monitoring scope.