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 STP206 for the Prevention of Necrotizing Enterocolitis (NEC) (NCT01954017), a randomized phase 1 study. The primary outcome is Number and Severity of Adverse Events Experienced by Subjects in Low-dose Treatment Groups.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01954017 |
| Phase | Phase 1 |
| Condition | Necrotizing Enterocolitis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 103 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Leadiant Biosciences, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Premature very low birth weight and extremely low birth weight neonates were the intended population. The registry lists necrotizing enterocolitis as the condition and records an enrollment of 103. The safety outcome identifies a safety population, but the registry record does not report detailed eligibility criteria or the number analyzed in each outcome group.
Intervention
The study assessed once-daily STP206 at low and high doses. The registry describes a sequential dose-escalation study intended to assess safety, tolerability, and preliminary preventive efficacy. The available primary outcome concerns adverse events among participants randomized to low-dose treatment groups; the record does not give dose amounts or dosing duration.
Comparator
The control intervention is described as sterile water. The trial is identified as randomized and placebo controlled, with a parallel-group model and quadruple masking. The registry does not report the allocation ratio or detailed masking procedures.
Primary question
The primary outcome measures the number and severity of adverse events, adverse events leading to study-drug discontinuation, and deaths among participants in low-dose treatment groups. The stated observation period is 30 days after the last dose of blinded study treatment. The registry does not reduce this multidimensional outcome to one comparative estimate.
These features frame the interpretation. The trial’s stated aim included preliminary NEC-preventative efficacy, but the primary outcome is a safety measure. The registry lists secondary outcomes concerning suspected and confirmed necrotizing enterocolitis, sepsis, and feeding intolerance in low- and high-dose groups. Their outcome titles alone do not establish the size or direction of any treatment effect. The posted safety counts are organized by dose tier and birth-weight band, with a separate control group in each band. Because the record does not provide a single adjusted or unadjusted comparison for the primary outcome, the clinical question that can be answered descriptively is narrower: what adverse-event and death counts are recorded for the listed groups, and how much uncertainty remains about comparisons between them?
3. Trial Design
The registry identifies STP206 for the prevention of necrotizing enterocolitis as a Phase Ib randomized, placebo-controlled study. It describes a parallel-group design with quadruple masking and a sequential dose-escalation approach. The brief study description says that two doses of STP206 were assessed against control in very low birth weight and extremely low birth weight neonates. The registry lists enrollment as 103 and status as completed.
The safety results distinguish low-dose groups, high-dose groups, and corresponding controls across birth-weight bands. The low-dose bands are labeled 2000-1501 g, 1500-1000 g, 999-750 g, and 749-500 g. The high-dose bands use the corresponding group labels. Each listed group has a separate recorded number at risk for serious adverse events, other adverse events, and deaths. The control intervention is described as sterile water. The record does not report the STP206 dose amounts, allocation ratio, sequence or timing of escalation, or whether each dose level was opened only after a particular safety review.
The study start is recorded as 2014-01-30, and primary completion as 2018-02-28. These dates describe the registry record and do not supply further information about recruitment pace, follow-up completeness, or changes to the study. The registry lists 26 outcome measures and 0 analyses. The primary outcome is a safety outcome in low-dose treatment groups, while the listed secondary outcomes cover suspected and confirmed NEC, sepsis, and feeding intolerance in low- and high-dose groups. The record does not report the detailed statistical analysis plan, sample-size justification, planned interim analyses, or a prespecified method for combining the separate weight and dose groups. Consequently, the design can be described from its registered features, but some operational and inferential details remain unreported 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 | Low Dose STP206 | High Dose STP206 | Control |
|---|---|---|---|
| STARTED | 36 | 33 | 34 |
| COMPLETED | 31 | 25 | 21 |
| NOT COMPLETED | 5 | 8 | 13 |
Reasons for not completing this period:
| Reason | Low Dose STP206 | High Dose STP206 | Control |
|---|---|---|---|
| Adverse Event | 0 | 0 | 1 |
| Death | 2 | 1 | 1 |
| Withdrawal by Subject | 0 | 1 | 0 |
| Protocol Violation | 0 | 3 | 2 |
| Necrotizing enterocolitis | 1 | 0 | 1 |
| Enteral intake stopped >7 days | 0 | 1 | 1 |
| All other reasons | 2 | 2 | 7 |
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: Intent-to-treat Population
| Characteristic | Unit | Low Dose STP206 | High Dose STP206 | Control | Total |
|---|---|---|---|---|---|
| Participants analysed | 36 | 33 | 34 | 103 | |
| Age, Customized: <=28 | Participants | 24 | 16 | 23 | 63 |
| Age, Customized: >28 | Participants | 12 | 16 | 10 | 38 |
| Sex: Female, Male: Female | Participants | 12 | 12 | 14 | 38 |
| Sex: Female, Male: Male | Participants | 24 | 21 | 20 | 65 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 1 | 1 | 2 | 4 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 11 | 8 | 9 | 28 |
| Race (NIH/OMB): White | Participants | 23 | 23 | 23 | 69 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 1 | 0 | 2 |
| 1-minute Apgar Score | units on a scale | 5.1 (SD 2.62) | 5.0 (SD 2.04) | 4.6 (SD 2.68) | 4.9 (SD 2.44) |
| 5-minute Apgar Score | units on a scale | 7.1 (SD 1.95) | 7.5 (SD 1.67) | 6.9 (SD 2.18) | 7.1 (SD 1.93) |
| Birth Weight | grams | 1110.8 (SD 422.37) | 1110.8 (SD 408.14) | 1141.0 (SD 434.12) | 1120.8 (SD 415.69) |
6. Primary Endpoint
The registry lists the primary outcome as Number and Severity of Adverse Events Experienced by Subjects in Low-dose Treatment Groups, measured over 30 days after the last dose of blinded study treatment.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome counts participants experiencing adverse events in the low-dose treatment groups. Its description includes the number and severity of adverse events, adverse events leading to study-drug discontinuation, and deaths. The stated timeframe is 30 days after the last dose of blinded study treatment, and the population is identified as the safety population.
Safety outcomes are used to characterize harms and tolerability during and after treatment exposure. Recording severity and discontinuations adds context beyond simply counting whether an event occurred, while deaths are a distinct clinically important outcome. For a small or dose-escalation study, such information can help describe the kinds of events observed across dose groups and birth-weight bands. That general purpose does not turn descriptive counts into proof that an intervention caused or prevented an event.
The registered primary outcome combines several types of information rather than specifying one event definition or one contrast. The posted record gives serious and other adverse-event counts by group, along with death counts, but it does not provide a single overall primary-outcome estimate, a formal comparison, or a confidence interval. It also does not report how severity categories were defined or how events leading to discontinuation were tabulated. The data therefore support a descriptive reading of the recorded group counts, not a complete reconstruction of the endpoint’s planned analysis.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results are group-specific counts, not a single treatment-effect estimate. In the low-dose groups, serious adverse events affected 1 of 8 participants in Group 1a, 2 of 9 in Group 2a, 3 of 9 in Group 3a, and 5 of 8 in Group 4a. In the corresponding controls, serious adverse events affected 1 of 4, 2 of 4, 3 of 5, and 1 of 4 participants. These figures describe the recorded groups separately; they do not adjust for birth-weight band or establish a pooled comparison.
For the high-dose groups, serious adverse events affected 1 of 8 participants in Group 1b, 2 of 8 in Group 2b, 2 of 8 in Group 3b, and 4 of 8 in Group 4b. Serious adverse events affected 0 of 4, 0 of 4, 1 of 4, and 4 of 4 participants in the corresponding controls. The small group denominators mean that each participant’s event status has substantial influence on a group’s observed count. Differences between these observed counts should not be read as precise estimates of underlying risk.
Other adverse events were recorded in all listed groups: the posted counts range from 3 of 4 to 9 of 9 in the low-dose and control groups, and from 3 of 4 to 8 of 8 in the high-dose and control groups. Death counts are also listed separately by group, including 2 in the Group 4a low-dose group, 1 in the Group 2a low-dose group, 1 in the Group 1a control group, 1 in the Group 4b high-dose group, and 1 in the Group 4b control group; the other listed groups have 0 deaths. These are descriptive records, not evidence by themselves that treatment caused or prevented deaths.
The serious adverse-event terms reported most often include necrotising colitis and convulsion, each listed at 50.0%; pneumonia, pneumonia staphylococcal, sepsis, and bacterial infection are each listed at 25.0%. The registry does not provide the event-level denominator or further detail needed to interpret those percentages as participant-level risks. It also does not provide a primary-outcome test, confidence interval, or comparative estimate. The safety information is therefore useful for describing what was recorded, but its precision and comparative meaning are limited. The registry’s secondary-outcome titles do not supply the corresponding NEC, sepsis, or feeding-intolerance results.
9. Statistical Methods Explained
Statistical methods help distinguish a descriptive count from an estimate of a treatment effect. In this record, the posted safety information is organized by dose and birth-weight group. A count answers how many participants in a listed group were recorded with an event. A denominator indicates the number of participants at risk for that outcome in that group. Neither alone expresses the uncertainty around a group’s underlying event probability, and neither establishes whether an observed difference between groups is attributable to treatment.
What does randomization contribute?
In general, randomization is intended to make treatment allocation independent of participant characteristics, helping reduce confounding in comparisons. Its protection depends on how the allocation was implemented and on maintaining the intended analysis. The registry identifies the study as randomized, but the allocation ratio, sequence-generation details, concealment procedures, and group-level baseline characteristics are not reported in the registry record. The separate dose tiers and birth-weight bands also matter: a comparison across all participants would need a clearly specified approach to account for those design features. No such pooled or adjusted analysis is reported.
What does masking contribute?
In general, masking can reduce the influence of knowledge about assigned treatment on care, event assessment, and reporting. The record labels the trial quadruple-masked and placebo controlled, and describes control as sterile water. It does not specify which roles were masked, how masking was maintained, or whether it was assessed. Masking is a design feature, not a substitute for transparent outcome definitions and analysis methods.
Why are confidence intervals useful?
In general, a confidence interval communicates the statistical uncertainty around an estimated quantity, such as a difference between event risks. A narrow interval indicates greater precision than a wide interval, given the model and assumptions used; an interval that includes values on both sides of no difference may leave the direction and size of an effect uncertain. The registry record does not report confidence intervals or a comparative primary-outcome estimate. No interval can therefore be attributed to the posted primary outcome from these registry entries.
Why does the small group structure matter?
In general, when groups are small, one participant’s outcome can move the observed proportion noticeably. Estimates based on few participants are commonly imprecise, and rare outcomes may be absent from a group even when their underlying risk is not zero. The results should therefore be read as observed counts within the recorded groups, not as stable predictions for a broader population. A formal model could potentially combine information across groups, but its assumptions and analysis plan would need to be stated; the registry does not report such a model.
What can be said about multiplicity?
Multiplicity arises when many outcomes, groups, time points, or comparisons are examined. In general, the more inferential tests performed, the greater the opportunity for apparently notable findings to arise by chance if no adjustment or hierarchy is specified. The registry lists 26 outcome measures, including safety and secondary outcomes across dose groups. It records 0 analyses. The record does not report a testing hierarchy, multiplicity adjustment, or prespecified primary contrasts. This absence does not show that no analyses were conducted elsewhere; it means the registry record does not document them.
How should sample-size illustrations be interpreted?
In general, a planning illustration based on an observed effect describes how a future study might be designed under stated assumptions. It is not a measure of the probability that the current trial detected an effect, and it does not establish that the observed effect is reliable. The registry does not report the trial’s own sample-size justification in the registry record for this analysis. Any planning illustrations associated with the posted results should consequently be treated as prospective scenarios, not as observed power or as evidence about efficacy.
Finally, outcome interpretation depends on definitions and handling of incomplete data. The registry does not report detailed adverse-event coding rules, severity criteria, missing-data procedures, or a full analysis population definition beyond naming the safety population. These omissions prevent a complete assessment of reproducibility and of the sensitivity of results to analytic choices. General statistical principles can clarify why these details matter, but they cannot replace methods that are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The record lists 26 outcome measures, including the primary safety outcome and secondary outcomes concerning suspected and confirmed NEC, sepsis, and feeding intolerance across dose groups. It records 0 analyses. A long list of outcomes can create many possible comparisons. In general, if investigators test numerous outcomes or group contrasts, the chance of at least one apparently unusual result increases unless the testing strategy accounts for multiplicity. The registry record does not report a hierarchy of tests, a multiplicity adjustment, or which secondary outcomes were intended to support confirmatory conclusions. The absence of this information limits interpretation of any apparent pattern across outcomes.
Missing-data handling is not reported in the registry record. In particular, the registry entries do not explain whether participants with incomplete follow-up were included in the safety counts, how missing adverse-event information was handled, or whether any participants were excluded after randomization. The record identifies the primary population as the safety population, but does not provide its operational definition or a full accounting of participants by analysis population. These details matter because exclusion or incomplete observation can affect descriptive counts as well as treatment comparisons.
The record also does not report an analysis plan specifying the primary contrast, treatment of the separate dose tiers and weight bands, or adjustment for baseline characteristics. No primary-outcome confidence interval or formal test is reported. This does not establish that analyses were absent outside the registry; it identifies what is not reported in the registry record. Without prespecified methods and clear reporting of missingness, multiplicity, and analysis populations, the posted group counts should be treated as descriptive rather than as a complete inferential evaluation.
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 |
|---|---|---|---|
| Group 1a (Birth wt: 2000-1501 g) Low Dose | 1 | 8 | 12.5% |
| Group 1a (Birth wt:2000-1501 g) Control | 1 | 4 | 25.0% |
| Group 2a (Birth wt:1500-1000 g) Low Dose | 2 | 9 | 22.2% |
| Group 2a (Birth wt:1500-1000 g) Control | 2 | 4 | 50.0% |
| Group 3a (Birth wt:999-750 g) Low Dose | 3 | 9 | 33.3% |
| Group 3a (Birth wt:999-750 g) Control | 3 | 5 | 60.0% |
| Group 4a (Birth wt: 749-500 g) Low Dose | 5 | 8 | 62.5% |
| Group 4a (Birth wt:749-500 g) Control | 1 | 4 | 25.0% |
| Group 1b (Birth wt: 2000-1501 g) High Dose | 1 | 8 | 12.5% |
| Group 1b (Birth wt:2000-1501 g) Control | 0 | 4 | 0.0% |
| Group 2b (Birth wt: 1500-1000 g) High Dose | 2 | 8 | 25.0% |
| Group 2b (Birth wt:1500-1000 g) Control | 0 | 4 | 0.0% |
| Group 3b (Birth wt: 999-750 g) High Dose | 2 | 8 | 25.0% |
| Group 3b (Birth wt:999-750 g) Control | 1 | 4 | 25.0% |
| Group 4b (Birth wt: 749-500 g) High Dose | 4 | 8 | 50.0% |
| Group 4b (Birth wt:749-500 g) Control | 4 | 4 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group 1a (Birth wt: 2000-1501 g) Low Dose | 0 of 8 |
| Group 1a (Birth wt:2000-1501 g) Control | 1 of 4 |
| Group 2a (Birth wt:1500-1000 g) Low Dose | 1 of 9 |
| Group 2a (Birth wt:1500-1000 g) Control | 0 of 4 |
| Group 3a (Birth wt:999-750 g) Low Dose | 0 of 9 |
| Group 3a (Birth wt:999-750 g) Control | 0 of 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 |
|---|---|
| Group 1a (Birth wt: 2000-1501 g) Low Dose | 7 of 8 |
| Group 1a (Birth wt:2000-1501 g) Control | 4 of 4 |
| Group 2a (Birth wt:1500-1000 g) Low Dose | 7 of 9 |
| Group 2a (Birth wt:1500-1000 g) Control | 3 of 4 |
| Group 3a (Birth wt:999-750 g) Low Dose | 9 of 9 |
| Group 3a (Birth wt:999-750 g) Control | 5 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Group 1a (Birth wt: 2000-1501 g) Low Dose | Group 1a (Birth wt:2000-1501 g) Control | Group 2a (Birth wt:1500-1000 g) Low Dose | Group 2a (Birth wt:1500-1000 g) Control | Group 3a (Birth wt:999-750 g) Low Dose | Group 3a (Birth wt:999-750 g) Control |
|---|---|---|---|---|---|---|---|
| Necrotising colitis | Gastrointestinal disorders | 1 of 8 | 1 of 4 | 0 of 9 | 0 of 4 | 0 of 9 | 0 of 5 |
| Pneumonia | Infections and infestations | 0 of 8 | 0 of 4 | 0 of 9 | 1 of 4 | 1 of 9 | 0 of 5 |
| Sepsis | Infections and infestations | 0 of 8 | 1 of 4 | 0 of 9 | 0 of 4 | 1 of 9 | 0 of 5 |
| Convulsion | Nervous system disorders | 0 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 2 of 9 | 0 of 5 |
| Cardio-respiratory arrest neonatal | Cardiac disorders | 0 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 0 of 9 | 1 of 5 |
| Patent ductus arterioles | Congenital, familial and genetic disorders | 0 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 0 of 9 | 1 of 5 |
| Necrotising enterocolitis neonatal | Gastrointestinal disorders | 0 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 1 of 9 | 0 of 5 |
| Vomiting | Gastrointestinal disorders | 0 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 0 of 9 | 1 of 5 |
| Pneumonia staphylococcal | Infections and infestations | 0 of 8 | 0 of 4 | 0 of 9 | 1 of 4 | 0 of 9 | 0 of 5 |
| Bronchiolitis | Infections and infestations | 0 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 1 of 9 | 0 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group 1a (Birth wt: 2000-1501 g) Low Dose | Group 1a (Birth wt:2000-1501 g) Control | Group 2a (Birth wt:1500-1000 g) Low Dose | Group 2a (Birth wt:1500-1000 g) Control | Group 3a (Birth wt:999-750 g) Low Dose | Group 3a (Birth wt:999-750 g) Control |
|---|---|---|---|---|---|---|---|
| Dermatitis diaper | Skin and subcutaneous tissue disorders | 3 of 8 | 1 of 4 | 3 of 9 | 1 of 4 | 2 of 9 | 2 of 5 |
| Anaemia neonatal | Blood and lymphatic system disorders | 1 of 8 | 0 of 4 | 2 of 9 | 1 of 4 | 5 of 9 | 2 of 5 |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | 3 of 8 | 1 of 4 | 4 of 9 | 0 of 4 | 2 of 9 | 1 of 5 |
| Vomiting | Gastrointestinal disorders | 2 of 8 | 1 of 4 | 3 of 9 | 1 of 4 | 0 of 9 | 1 of 5 |
| Anaemia | Blood and lymphatic system disorders | 0 of 8 | 0 of 4 | 1 of 9 | 0 of 4 | 2 of 9 | 3 of 5 |
| Inguinal hernia | Gastrointestinal disorders | 1 of 8 | 1 of 4 | 0 of 9 | 0 of 4 | 3 of 9 | 1 of 5 |
| Apnoea neonatal | Respiratory, thoracic and mediastinal disorders | 1 of 8 | 0 of 4 | 0 of 9 | 0 of 4 | 2 of 9 | 3 of 5 |
| Nasopharyngitis | Infections and infestations | 2 of 8 | 1 of 4 | 2 of 9 | 0 of 4 | 0 of 9 | 0 of 5 |
| Pneumonia | Infections and infestations | 0 of 8 | 0 of 4 | 1 of 9 | 1 of 4 | 3 of 9 | 0 of 5 |
| Atrial septal defect | Congenital, familial and genetic disorders | 0 of 8 | 0 of 4 | 1 of 9 | 0 of 4 | 3 of 9 | 0 of 5 |
Serious events with the highest arm-level rate: necrotising colitis (50.0%), convulsion (50.0%), pneumonia (25.0%), pneumonia staphylococcal (25.0%), sepsis (25.0%).
For each group, the serious-adverse-event results give the number at risk and the number affected; the same structure is used for other adverse events. For example, the Group 1a low-dose record lists 8 at risk for serious adverse events and 1 affected. This is a group-level count, not a rate adjusted for follow-up or a comparison that accounts for study design. The other adverse-event counts are a separate category and should not be added to serious-event counts, because the record does not say whether participants could appear in both.
Death counts are listed separately. They should not be assumed to be mutually exclusive with adverse-event counts or attributed to study treatment from these data alone. The record also lists serious-event terms with percentages, including necrotising colitis and convulsion at 50.0% and several other terms at 25.0%. The denominator and counting rule for those percentages are not reported in the registry record, so they should not be interpreted as risks among all participants in a treatment group.
Because the group counts are small and split across dose and weight categories, comparisons are inherently limited in precision. The registry does not provide a formal test, comparative effect estimate, or confidence interval for the primary safety outcome. The most defensible reading is therefore descriptive: these are the events recorded in the listed groups, not conclusive evidence of a treatment-related difference.
12. What the Result Does, and Does Not, Mean
The results document serious and other adverse events and deaths in the listed low-dose, high-dose, and control groups. That information is relevant to the trial’s safety focus and to its stated interest in tolerability. The reported counts also show why separate group-level reporting matters: events are not uniformly distributed across the dose and birth-weight categories. Such variation is descriptive and, without a prespecified comparative analysis, does not establish a dose response or a treatment effect.
The record does not establish whether STP206 prevents NEC. Although the study summary describes preliminary NEC-preventative efficacy as an aim, the primary outcome is about safety, and the registry information does not include numerical results for the listed secondary NEC outcomes. The outcome titles alone cannot show whether suspected or confirmed NEC differed between groups. Nor can the safety counts serve as a substitute for those efficacy results.
The findings also do not establish that STP206 caused the adverse events or deaths recorded in the treatment groups. Events can occur during a trial for reasons unrelated to the assigned intervention, and a causal comparison requires an appropriate analysis of randomized groups, clear outcome definitions, and adequate precision. The registry does not report a comparative primary-outcome estimate, confidence interval, or detailed analysis plan. The small numbers within individual groups further limit the strength of conclusions that can be drawn from raw counts.
Accordingly, the posted information is best understood as a descriptive account of registered safety outcomes, rather than a definitive evaluation of efficacy or comparative safety. It can identify what was recorded and where uncertainty remains. It cannot establish the magnitude or certainty of a treatment effect, resolve how missing information was handled, or determine whether the observed event patterns would persist in a larger or different population.
13. Why This Trial Matters Statistically
This trial is statistically notable for combining randomization and masking with sequential dose escalation in a population split across dose and birth-weight groups. That structure is relevant because safety observations are reported within multiple small groups rather than as one simple two-arm comparison. A reader needs to distinguish the design’s stated purpose from the analysis that the registry actually documents.
The trial also illustrates why endpoint structure matters. Its primary outcome includes several safety components—event number and severity, discontinuations related to adverse events, and deaths—yet the registry does not reduce these to a single comparative estimate. Reporting counts by group gives useful descriptive detail, but it does not provide the same information as a prespecified contrast with an uncertainty interval. The absence of such an estimate makes it important not to treat visible differences among counts as definitive evidence.
Multiplicity is another central statistical issue. The registry lists 26 outcome measures across safety and other clinical topics, but records 0 analyses. Without a reported testing hierarchy or adjustment strategy, the inferential status of comparisons across outcomes is unclear. The same issue applies to decisions about combining dose tiers or weight bands: pooling can increase precision in general, but only if the grouping is justified and the analysis is specified.
Finally, the record shows the limits of registry-based interpretation. A completed status, enrollment figure, and group counts provide a useful account of registered study features and results, but they do not supply every detail needed to evaluate precision, missingness, or causal attribution. The trial therefore offers a practical example of how careful statistical reading separates recorded observations from the stronger claims that require documented methods and comparative estimates.
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.
Several trial-specific limitations constrain interpretation of the posted results. The primary outcome is a multidimensional safety measure, but the registry record does not report a single definition or summary estimate covering all its components. The counts are separated across dose levels and birth-weight bands, leaving small denominators within individual groups. Such a structure makes raw counts sensitive to individual outcomes and limits precision.
The record does not report dose amounts, allocation ratio, detailed randomization procedures, masking roles, or the rules governing sequential escalation. It also does not provide group-level baseline characteristics or explain whether dose groups were intended to be compared directly. Without these details, the balance and comparability of the listed groups cannot be assessed fully from the registry information.
For outcome interpretation, the registry does not report severity definitions, adverse-event coding procedures, the denominator for the serious-event term percentages, or detailed handling of missing observations. The safety population is named, but its definition and participant accounting are not reported. The record also does not provide formal comparative estimates, confidence intervals, a testing hierarchy, or the planned approach to multiplicity. Numerical results for the secondary NEC, sepsis, and feeding-intolerance outcomes are not present in the registry record for this account.
These limitations do not show that the trial lacked procedures or analyses; they identify information that is not reported in the registry record. They mean that the posted data support a cautious descriptive reading rather than a comprehensive assessment of treatment effects or the precision of those effects.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: STP206 for the Prevention of Necrotizing Enterocolitis (NEC) — NCT01954017.
- Registry record last updated 2020-03-24; status last verified 2020-03.
- Results first posted 2020-03-24.
- 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
NCT01954017 is a completed Phase Ib randomized, placebo-controlled, quadruple-masked, parallel-group study of STP206 for prevention of necrotizing enterocolitis. The registry describes sequential dose escalation and records enrollment of 103. Control is described as sterile water. The study start is recorded as 2014-01-30 and primary completion as 2018-02-28.
The primary outcome concerns adverse-event number and severity, adverse events leading to study-drug discontinuation, and deaths in low-dose treatment groups, during 30 days after the last dose of blinded study treatment. Posted safety results give serious-event, other-event, and death counts across low-dose, high-dose, and control groups within birth-weight bands. The registry lists 26 outcome measures and records 0 analyses.
The posted counts support description of recorded safety events, but the registry record does not report a formal primary-outcome comparison, confidence interval, detailed analysis plan, or numerical results for the listed secondary efficacy outcomes. Interpretation should remain descriptive and account for the small group counts and unreported methods.