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 Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Premature Neonates (NCT02074059), a randomized phase 2 study. The primary outcome is Peri-Dosing Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02074059 |
| Phase | Phase 2 |
| Condition | Respiratory Distress Syndrome |
| Allocation | Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 80 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Windtree Therapeutics |
| Results posted | Yes |
2. Clinical Question
Population
Preterm neonates described in the trial record as 29 to 34 weeks gestational age and receiving nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome. The official title describes the range as 29 to 34 weeks PMA. The registry does not give further eligibility details in the information reported for this trial.
Intervention
Lucinactant for inhalation administered with nCPAP in escalating doses: 25, 50, 75, 100, or 150 mg/kg total phospholipids. Repeat dosing was possible in the 100 mg/kg and 150 mg/kg groups if criteria were met; the criteria are not reported in the registry record.
Comparator
nCPAP therapy alone. The registry describes the trial as randomized and controlled, but does not report the allocation procedure or the details of how participants were assigned among the dose groups and comparator.
Primary question
The stated primary objective was to evaluate the safety and tolerability of aerosolized lucinactant administered in escalating inhaled doses compared with nCPAP alone. The primary outcome is described as peri-dosing events within 48 hours after initiation of study treatment, with pre-specified adverse events occurring during treatment administration. The record does not report a single primary comparison estimate.
3. Trial Design
This was a Phase 2, multicenter, randomized, open-label, controlled trial with a sequential model. The registry lists an enrollment of 80 and a completed status. The recorded start was 2014-02, and primary completion was 2015-10. The sponsor was Windtree Therapeutics.
Five lucinactant dose groups were listed: 25, 50, 75, 100, and 150 mg/kg total phospholipids. Each was administered with nCPAP. The comparator group received nCPAP alone. Repeat dosing was possible for the 100 mg/kg and 150 mg/kg doses if criteria were met. Those criteria, and the precise sequence or rules for moving between dose groups, are not reported in the registry record. The trial’s sequential model and escalating doses describe its broad structure, but do not establish specific dose-escalation procedures.
Randomization means that assignment was described as randomized, but the record does not report how the random sequence was generated, whether allocation was concealed, or the allocation ratio. Masking was listed as none, so the trial was open-label. The registry does not report additional operational details that would clarify how treatment knowledge was handled during outcome assessment.
The study’s stated purpose was to evaluate safety and tolerability in preterm neonates with respiratory distress syndrome who were receiving nCPAP. PCO2 was listed as a secondary outcome. The record lists 5 outcome measures, but does not report 5 outcome results in the information summarized for this analysis. A detailed protocol, eligibility criteria, treatment schedule, and sample-size justification 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 | Aerolized Lucinactant (25 mg/kg) | Aerosolized Lucinactant (50 mg/kg) | Aerosolized Lucinactant (75 mg/kg) | Aerosolized Lucinactant (100 mg/kg) | Aerosolized Lucinactant (150 mg/kg) | nCPAP Alone |
|---|---|---|---|---|---|---|
| STARTED | 8 | 8 | 8 | 8 | 8 | 40 |
| COMPLETED | 8 | 8 | 8 | 8 | 7 | 39 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 1 | 1 |
Reasons for not completing this period:
| Reason | Aerolized Lucinactant (25 mg/kg) | Aerosolized Lucinactant (50 mg/kg) | Aerosolized Lucinactant (75 mg/kg) | Aerosolized Lucinactant (100 mg/kg) | Aerosolized Lucinactant (150 mg/kg) | nCPAP Alone |
|---|---|---|---|---|---|---|
| Death | 0 | 0 | 0 | 0 | 1 | 1 |
Analysis population for the primary outcome, as defined in the registry: Safety Population
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: All randomized subjects
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | Aerolized Lucinactant (25 mg/kg) | Aerosolized Lucinactant (50 mg/kg) | Aerosolized Lucinactant (75 mg/kg) | Aerosolized Lucinactant (100 mg/kg) | Aerosolized Lucinactant (150 mg/kg) | nCPAP Alone |
|---|---|---|---|---|---|---|---|
| Participants analysed | 8 | 8 | 8 | 8 | 8 | 40 | |
| Age, Categorical: <=18 years | Participants | 8 | 8 | 8 | 8 | 8 | 40 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 3 | 5 | 4 | 3 | 6 | 21 |
| Sex: Female, Male: Male | Participants | 5 | 3 | 4 | 5 | 2 | 19 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 4 | 2 | 2 | 2 | 11 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 7 | 4 | 6 | 6 | 6 | 29 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 3 | 0 | 0 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 | 0 | 0 | 1 | 0 | 4 |
| Race (NIH/OMB): White | Participants | 4 | 5 | 4 | 5 | 6 | 30 |
| Race (NIH/OMB): More than one race | Participants | 1 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 3 | 1 | 2 | 2 | 4 |
| Region of Enrollment: United States | participants | 8 | 8 | 8 | 8 | 8 | 40 |
| Gestational Age | weeks | 30.5 (SD 0.81) | 31.8 (SD 1.50) | 33.3 (SD 2.11) | 31.7 (SD 2.10) | 32.3 (SD 1.95) | 31.5 (SD 1.56) |
6. Primary Endpoint
The registry lists the primary outcome as Peri-Dosing Events, measured over Within 48 Hours after Initiation of Study Treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is titled Peri-Dosing Events and is measured as participants. Its timeframe is within 48 hours after initiation of study treatment. The description specifies pre-specified adverse events that occurred during treatment administration, and says that nCPAP alone is not included. The outcome population is identified as the Safety Population.
A safety endpoint focused on events around treatment administration can describe whether adverse events were recorded in connection with that period. Counting participants with an event is different from counting every event: a participant-level measure indicates how many people experienced an event, but does not by itself describe the number of repeated events, their duration, or their severity. Those additional details are not reported in the primary outcome description.
The record does not reduce this endpoint to a single comparison or provide a single primary result estimate. It identifies several categories or time points, so the posted endpoint description should not be read as though it were one uniform binary result contrasting every dose with nCPAP alone. The adverse-event summaries reported by group are relevant safety information, but the registry does not establish that those summaries are the complete numerical results for the named primary endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The trial record describes a randomized Phase 2 study of inhaled lucinactant given with nCPAP across five dose groups, with nCPAP alone as comparator. Its primary objective was safety and tolerability, and its primary outcome description concerns peri-dosing adverse events. The posted numerical safety summary reports serious adverse events and other adverse events separately, by group. These summaries are counts of participants affected among the participants at risk in each group, as labeled in the record.
For the serious adverse-event summary, the number at risk was 8 in each lucinactant dose group, and 40 in the nCPAP-alone group. The numbers affected were 2 at 25 mg/kg, 3 at 50 mg/kg, 2 at 75 mg/kg, 3 at 100 mg/kg, 3 at 150 mg/kg, and 7 with nCPAP alone. For other adverse events, the number at risk was also 8 in each dose group and 40 with nCPAP alone; the numbers affected were 8, 8, 8, 7, and 8 across the dose groups in increasing dose order, and 37 with nCPAP alone.
These counts describe the posted group summaries, not a formal comparison of treatment effects. The trial is open-label, and the registry record does not report an analysis adjusting for group differences or a statistical comparison between any dose and nCPAP alone. The dose groups are small in the safety summary, and a count based on a small group can change substantially with the experience of one participant. The summaries therefore warrant careful attention to their denominators and should not be interpreted as precise estimates of relative risk.
The registry does not report a single result for the primary endpoint, and the record lists 0 analyses. That means there is no reported primary estimate, confidence interval, or hypothesis test to interpret from the registry information. The serious and other adverse-event counts should not be substituted for an absent primary endpoint analysis without confirmation that they match the endpoint’s definitions, timeframe, and population.
The record also lists serious adverse-event terms with percentages, including Pneumothorax at 25.0%, Necrotising Enterocolitis Neonatal at 12.5%, Cadio-respiratory Arrest at 12.5%, Apnoea Neonatal at 12.5%, Pneumomediastinum at 12.5%, and Hydrocephalus at 2.5%. The denominator and calculation basis for these percentages are not reported in the registry record. They should therefore be read as registry-listed figures rather than as group-specific incidence estimates. Death counts are not reported.
9. Statistical Methods Explained
Statistical interpretation depends on separating the study’s design, its outcome definitions, and the numerical summaries that are actually reported. The trial was randomized, but the registry record does not report the randomization procedure, the allocation ratio, or allocation concealment. Randomization can help balance known and unknown participant characteristics on average, but it does not guarantee that small groups will be balanced. The safety summary includes 8 participants at risk in each dose group and 40 in the nCPAP-alone group, so group counts need to be read in the context of these different group sizes.
What does a participant count tell the reader?
The adverse-event figures are counts of participants affected, rather than a tally of every event. A participant-level count answers how many people were recorded with at least one event in the specified category, subject to the registry’s definitions. It does not show how many separate events a person experienced, how long an event lasted, or whether several events occurred in the same participant. The record does not provide enough detail to reconstruct those features.
The reported serious adverse-event counts are 2, 3, 2, 3, and 3 across the five lucinactant dose groups, with 7 in the nCPAP-alone group. The other adverse-event counts are 8, 8, 8, 7, and 8 across those dose groups, with 37 in the comparator group. These are descriptions of observed counts, not estimates that account for uncertainty. No confidence intervals or inferential tests for these group summaries are reported in the registry record.
Why do denominators and definitions matter?
A count alone is hard to interpret without knowing how many participants could contribute to it. The registry gives the numbers at risk for the serious and other adverse-event summaries: 8 in each lucinactant group and 40 in the nCPAP-alone group. The denominators are not interchangeable, and comparisons based only on the raw numbers affected would overlook their different group sizes. This explanation is general: proportions can help put counts in context, but no new proportion or comparative effect is calculated in this interpretation.
Outcome definitions matter just as much. The named primary outcome is peri-dosing events within 48 hours after initiation of study treatment, described as pre-specified adverse events that occurred during treatment administration. The adverse-event group summaries are labeled serious and other adverse events. The registry record does not establish in the reported information that these group summaries use precisely the same definitions, time window, or event categories as the primary endpoint. It would be inappropriate to assume that they are interchangeable.
What can be concluded without a reported model?
The registry lists 0 analyses. It does not report a model, an adjusted treatment effect, a standard error, a confidence interval, or a p-value for the primary outcome in the information described. Consequently, there is no reported statistical estimate from which to assess the precision of a contrast between lucinactant and nCPAP alone. A confidence interval, in general, represents the range of values compatible with a statistical estimate under the assumptions of the method used. Without an estimate and its interval, the precision of a treatment contrast cannot be assessed from the registry record.
For event-count data, statistical methods may need to account for the fact that event counts can vary more widely than a simple model assumes. General methods for count outcomes may address over-dispersion, meaning variability beyond that expected under a basic count model. But this trial’s posted participant counts are not enough to identify an appropriate model, and no count model or over-dispersion assessment is reported in the registry record. The primary measure is described in participants, not as a rate of recurrent events, so a recurrent-event analysis should not be presumed.
How should planning calculations be read?
Sample-size illustrations are planning exercises, not observed findings and not evidence that a study achieved a particular power. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. For non-time-to-event outcomes, the stated convention is participants per arm. These assumptions describe the planning illustration, not the trial’s own design choices. The trial’s sample-size justification is not reported in the registry record. A planning result based on an observed effect should not be described as observed power or as proof that the trial was adequately powered.
More generally, statistical conclusions depend on the prespecified analysis, the outcome definitions, the population analyzed, and the handling of incomplete observations. Those details are not reported for the primary endpoint in the registry information described. The posted counts remain useful descriptive information, but they cannot replace a complete analysis plan or a reported comparative estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry record does not report how multiplicity was handled. Multiplicity arises when several outcomes, dose groups, time points, or comparisons are examined: the more tests conducted, the greater the chance of finding an apparently notable result by chance. This is a general statistical concern, not evidence that an unreported multiplicity procedure was or was not used. The record lists 5 outcome measures, but the reported information does not identify a hierarchy among them, state which comparisons were prespecified, or describe any adjustment for multiple testing.
The primary outcome description refers to several categories or time points, and the registry information does not reduce it to one comparison. No single primary estimate or analysis result is reported. A multiplicity strategy, such as selecting one primary comparison or adjusting inferential thresholds, cannot be inferred from the trial’s randomized design or its sequential dose structure. The statistical analysis plan is not reported in the registry record.
Missing-data handling is also not reported. The adverse-event summaries give the number at risk in each group, but that alone does not explain whether all randomized participants contributed to every outcome, whether any observations were missing, or how incomplete follow-up was handled. The analysis population is named as the Safety Population for the primary endpoint; criteria defining that population are not reported in the registry information described.
The record does not report an intention-to-treat population, a per-protocol population, or rules for exclusions from analysis. It also does not report whether the safety summaries include all participants who began assigned treatment, or how treatment discontinuations and unavailable observations were handled. These omissions limit interpretation of both the numerator and the denominator for outcomes beyond the explicit at-risk counts. In general, results can depend on population definitions and missing-data assumptions, so those details are necessary for a complete assessment of the analysis.
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 |
|---|---|---|---|
| Aerolized Lucinactant (25 mg/kg) | 2 | 8 | 25.0% |
| Aerosolized Lucinactant (50 mg/kg) | 3 | 8 | 37.5% |
| Aerosolized Lucinactant (75 mg/kg) | 2 | 8 | 25.0% |
| Aerosolized Lucinactant (100 mg/kg) | 3 | 8 | 37.5% |
| Aerosolized Lucinactant (150 mg/kg) | 3 | 8 | 37.5% |
| nCPAP Alone | 7 | 40 | 17.5% |
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 |
|---|---|
| Aerolized Lucinactant (25 mg/kg) | 8 of 8 |
| Aerosolized Lucinactant (50 mg/kg) | 8 of 8 |
| Aerosolized Lucinactant (75 mg/kg) | 8 of 8 |
| Aerosolized Lucinactant (100 mg/kg) | 7 of 8 |
| Aerosolized Lucinactant (150 mg/kg) | 8 of 8 |
| nCPAP Alone | 37 of 40 |
Most frequent serious adverse event terms
| Event term | Organ system | Aerolized Lucinactant (25 mg/kg) | Aerosolized Lucinactant (50 mg/kg) | Aerosolized Lucinactant (75 mg/kg) | Aerosolized Lucinactant (100 mg/kg) | Aerosolized Lucinactant (150 mg/kg) | nCPAP Alone |
|---|---|---|---|---|---|---|---|
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 2 of 8 | 2 of 8 | 1 of 8 | 2 of 8 | 2 of 8 | 4 of 40 |
| Necrotising Enterocolitis Neonatal | Gastrointestinal disorders | 0 of 8 | 1 of 8 | 0 of 8 | 1 of 8 | 0 of 8 | 1 of 40 |
| Cadio-respiratory Arrest | Respiratory, thoracic and mediastinal disorders | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 1 of 8 | 0 of 40 |
| Apnoea Neonatal | Respiratory, thoracic and mediastinal disorders | 0 of 8 | 0 of 8 | 1 of 8 | 0 of 8 | 0 of 8 | 0 of 40 |
| Pneumomediastinum | Respiratory, thoracic and mediastinal disorders | 0 of 8 | 1 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 40 |
| Hydrocephalus | Nervous system disorders | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 1 of 40 |
| Pulmonary Haemorrhage | Respiratory, thoracic and mediastinal disorders | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 1 of 40 |
| Cardio-respiratory Arrest | Cardiac disorders | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 0 of 8 | 1 of 40 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Aerolized Lucinactant (25 mg/kg) | Aerosolized Lucinactant (50 mg/kg) | Aerosolized Lucinactant (75 mg/kg) | Aerosolized Lucinactant (100 mg/kg) | Aerosolized Lucinactant (150 mg/kg) | nCPAP Alone |
|---|---|---|---|---|---|---|---|
| Jaundice Neonatal | Pregnancy, puerperium and perinatal conditions | 8 of 8 | 8 of 8 | 7 of 8 | 5 of 8 | 7 of 8 | 30 of 40 |
| Apnoea Neonatal | Respiratory, thoracic and mediastinal disorders | 6 of 8 | 3 of 8 | 4 of 8 | 2 of 8 | 4 of 8 | 23 of 40 |
| Constipation | Gastrointestinal disorders | 5 of 8 | 6 of 8 | 2 of 8 | 2 of 8 | 5 of 8 | 14 of 40 |
| Dermatitis Diaper | Skin and subcutaneous tissue disorders | 4 of 8 | 3 of 8 | 1 of 8 | 3 of 8 | 2 of 8 | 16 of 40 |
| Anaemia Neonatal | Blood and lymphatic system disorders | 5 of 8 | 3 of 8 | 1 of 8 | 1 of 8 | 1 of 8 | 15 of 40 |
| Metabolic Acidosis | Metabolism and nutrition disorders | 4 of 8 | 2 of 8 | 0 of 8 | 1 of 8 | 1 of 8 | 11 of 40 |
| Neonatal Respiratory Distress Syndrome | Pregnancy, puerperium and perinatal conditions | 5 of 8 | 2 of 8 | 1 of 8 | 0 of 8 | 0 of 8 | 6 of 40 |
| Cardiac Murmur | Investigations | 1 of 8 | 2 of 8 | 2 of 8 | 0 of 8 | 1 of 8 | 6 of 40 |
| Bradycardia | Cardiac disorders | 1 of 8 | 1 of 8 | 1 of 8 | 2 of 8 | 0 of 8 | 6 of 40 |
| Hyponatraemia | Metabolism and nutrition disorders | 2 of 8 | 2 of 8 | 1 of 8 | 0 of 8 | 0 of 8 | 5 of 40 |
Serious events with the highest arm-level rate: pneumothorax (25.0%), necrotising enterocolitis neonatal (12.5%), cadio-respiratory arrest (12.5%), apnoea neonatal (12.5%), pneumomediastinum (12.5%).
The registry reports serious adverse events and other adverse events in separate group summaries. In each lucinactant dose group, 8 participants were listed at risk; 40 were listed at risk with nCPAP alone. Serious adverse events affected 2, 3, 2, 3, and 3 participants across the dose groups in ascending dose order, and 7 participants in the comparator group. Other adverse events affected 8, 8, 8, 7, and 8 participants across the dose groups, and 37 participants with nCPAP alone.
These figures are participant counts, not counts of all events. The registry also lists serious-event terms and percentages: Pneumothorax, 25.0%; Necrotising Enterocolitis Neonatal, 12.5%; Cadio-respiratory Arrest, 12.5%; Apnoea Neonatal, 12.5%; Pneumomediastinum, 12.5%; and Hydrocephalus, 2.5%. The denominator for those percentages is not reported in the registry record, so they cannot be assigned to a specific treatment group or interpreted as group-specific risks from the information available. Death counts are not reported; an unreported count should not be read as zero.
The counts are descriptive and do not establish a statistical difference between groups. The registry does not report confidence intervals, tests, or an adjusted analysis for these safety summaries.
12. What the Result Does, and Does Not, Mean
The posted results describe a safety-focused, randomized trial in which lucinactant for inhalation was studied with nCPAP at five dose levels and compared with nCPAP alone. The registry’s adverse-event summaries report the numbers of participants affected and at risk for serious and other adverse events. These figures offer a descriptive account of the recorded events in the groups, but they do not by themselves establish whether the groups differed beyond what could occur through sampling variability.
The registry record does not report a single primary endpoint estimate, a confidence interval, or a statistical test. It also does not report enough information to determine whether the adverse-event summaries reproduce the exact primary endpoint definition, which is limited to peri-dosing events within 48 hours after initiation and describes pre-specified events occurring during treatment administration. The summaries should therefore be interpreted as posted safety information, not as a complete comparative analysis of that endpoint.
The figures do not establish that lucinactant is effective or ineffective. The stated primary objective was safety and tolerability, and the secondary outcome listed was PCO2. A secondary outcome name without a reported result does not establish a treatment effect. Nor do the reported adverse-event counts establish that any specific event was caused by a study treatment; the registry summary does not provide causal attribution, event-level narratives, or adjusted comparisons.
Because each dose group’s safety summary includes 8 participants at risk, the counts are limited in their ability to characterize uncommon events or provide precise group-level estimates. This is a general limitation of small safety groups. The larger nCPAP-alone group has a different denominator, so raw counts across groups cannot be directly compared as though the groups were the same size. No comparative estimate is calculated.
Overall, the registry supports a description of the study design and the posted safety counts, but not a definitive statistical conclusion about a difference between treatments, the size or precision of such a difference, or the cause of a particular event. The protocol, full analysis plan, and additional outcome results are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for combining a randomized comparison with a sequential dose-escalation structure. Randomization identifies the trial as a comparative design, while the multiple dose groups allow safety information to be described across increasing lucinactant doses. Those design features do not, on their own, reveal the dose-escalation rules, the allocation method, or the analysis used to compare groups. Those details are not reported in the registry record.
The safety summaries also illustrate why group denominators and outcome definitions matter. Each dose group has 8 participants at risk in the posted summaries, compared with 40 in the nCPAP-alone group. The reported counts can be read descriptively, but raw numbers alone do not provide a standardized comparison, an estimate of uncertainty, or evidence of a dose trend. A dose-related pattern should not be inferred simply from the ordering of dose groups and the observed counts.
Another statistical issue is the distinction between a clearly named primary endpoint and a reported primary analysis. The record names Peri-Dosing Events and describes its time window and population, but it does not report one primary result estimate. It also lists 0 analyses, and the primary outcome is not reduced to one comparison. This leaves readers without a reported inferential result that could connect the stated objective to a quantified treatment contrast.
The record’s sample-size illustrations are planning exercises based on stated assumptions, not a report of the trial’s original justification or evidence of achieved power. This distinction matters because a planning calculation cannot repair absent outcome results, and a small safety sample can remain imprecise even when numerical summaries are available. The trial is useful statistically as an example of why design labels, endpoint definitions, denominators, and analysis reporting need to be considered together rather than treated as substitutes for one another.
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.
Important design and analysis details are not reported in the registry record. These include the randomization method, allocation concealment, the allocation ratio, the dose-escalation decision rules, and the criteria for repeat dosing at 100 mg/kg and 150 mg/kg. The registry identifies a sequential model and escalating doses, but that information is not sufficient to reconstruct the treatment sequence or the decisions that governed progression between doses.
The primary endpoint is described, but the registry does not report a single primary result estimate or a statistical comparison. Although peri-dosing events are specified as occurring during treatment administration within 48 hours after initiation, the available information does not give the event-level detail needed to check how the endpoint was assembled. The relationship between that endpoint and the separately posted serious and other adverse-event summaries is not reported.
Further limitations include no reported confidence intervals, p-values, analysis model, multiplicity strategy, missing-data procedure, or criteria for defining the Safety Population. The record does not report the number of participants with unavailable outcome data, reasons for missingness, or analysis populations beyond the safety-population label. These gaps prevent a full assessment of selection, precision, and statistical uncertainty.
The serious-event term percentages are listed without a reported denominator or group attribution. Death counts are not reported. The record also does not report the trial’s sample-size justification, and planning illustrations should not be mistaken for that justification. Finally, the listed PCO2 secondary outcome has no result in the information described. These limits mean that the available record supports a bounded descriptive reading of design and safety counts, not a complete evaluation of all trial outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Premature Neonates — NCT02074059.
- Registry record last updated 2017-04-21; status last verified 2017-03.
- Results first posted 2017-04-21.
- 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
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18. Record Summary
NCT02074059 is a completed Phase 2, multicenter, randomized, open-label, controlled trial sponsored by Windtree Therapeutics. Its recorded start was 2014-02, primary completion was 2015-10, and enrollment was 80. The study evaluated inhaled lucinactant with nCPAP in preterm neonates described as 29 to 34 weeks, with nCPAP alone as comparator. Five lucinactant dose groups were listed, from 25 mg/kg through 150 mg/kg total phospholipids.
The primary objective concerned safety and tolerability. The primary outcome was Peri-Dosing Events, described as pre-specified adverse events during treatment administration within 48 hours after initiation, in the Safety Population; nCPAP alone was excluded from that endpoint description. PCO2 was listed as a secondary outcome. The registry posts participant counts for serious and other adverse events, but does not report a single primary outcome estimate, confidence interval, or analysis. The analysis plan, missing-data procedures, and sample-size justification are not reported in the registry record.