Merotocin in Mothers With Inadequate Milk Production and Infants Delivered Prematurely: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Merotocin in Mothers With Inadequate Milk Production and Infants Delivered Prematurely (NCT02545127), a randomized phase 2 study. The primary outcome is Volume of Mother's Own Milk (MoM).

2. Clinical Question

3. Trial Design

This was a phase 2, randomized, double-blind, placebo-controlled, multicenter trial with a parallel-group design. The registry record identifies merotocin and placebo as the two interventions and describes merotocin as a selective oxytocin-receptor agonist. The official title specifies intra-nasal administration. These design labels describe the planned trial structure; they do not establish that enough outcome information was available to evaluate the intervention.

Enrollment was 4. The record states that the trial was prematurely terminated due to slow recruitment. It does not report a reason for the slow recruitment, a planned or achieved recruitment rate, or whether the enrollment figure represents all participants who began treatment. Further details about eligibility criteria, allocation procedures, treatment dose and duration, masking procedures, and site participation are not reported in the registry record.

The primary outcome was daily volume of Mother's Own Milk, measured on days 1 to 14. It was measured by weight in grams and converted to milliliters. The registry record also lists secondary outcomes related to milk volume, milk fed to infants, formula or donor milk fed to infants, time to specified daily milk-volume thresholds, and merotocin concentration in milk. Although 17 outcome measures are listed, the record reports no analyses.

The most consequential design fact for interpretation is that the primary outcome data are not presented. The registry record says that data, if collected, are withheld to protect the privacy of the trial participants. Therefore, the registered design can be described, but the randomized comparison cannot be evaluated from the posted outcome results.

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.

Before assignment: In total, 15 participants were screened. Of these, 11 were screening failures and 4 participants were randomized. All the randomized participants were exposed to the investigational medicinal product (IMP): 2 participants to Merotocin and 2 participants to Placebo.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The trial was prematurely terminated due to slow recruitment. Data (if collected) are not presented in order to protect the privacy of the trial participant(s).

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.

6. Primary Endpoint

The registry lists the primary outcome as Volume of Mother's Own Milk (MoM), measured over Daily days 1 to 14.

The primary endpoint was Volume of Mother's Own Milk (MoM), with daily measurement during days 1 to 14. The registry record says milk weight in grams was converted to volume in milliliters. It does not provide endpoint values, a summary statistic, or a comparison between the merotocin and placebo groups.

As a general statistical rationale, a direct measure of daily milk volume can describe the amount of milk produced and how that amount changes over a specified assessment period. Repeated daily values can capture variation over time that a single measurement may not represent. A treatment comparison would need to account for the structure of those observations and specify how the daily measurements contribute to the primary analysis. The statistical model and summary method are not reported in the registry record.

The endpoint is clinically connected to the trial's stated aim of supporting lactation in mothers with preterm delivery and inadequate milk production. However, a measure of milk volume alone does not show how much milk an infant consumed, whether the milk was fed to the infant, or how feeding was supplemented. The listed secondary outcomes address some related questions, but no analyses are reported for them either.

Because the registry record does not present the primary data, the endpoint definition explains what the trial intended to measure, not what the trial found. No treatment effect, uncertainty interval, or inference about a difference in daily volume can be drawn from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a randomized, double-blind, placebo-controlled, parallel-group phase 2 trial. These terms describe the planned method of comparison. Randomization is generally used to reduce systematic differences between groups at allocation, while masking is generally intended to limit the influence of treatment knowledge on care or assessment. The registry record does not provide details of the randomization sequence, allocation concealment, who was masked, or whether masking was maintained.

What would the primary comparison estimate?

The stated primary outcome is daily MoM volume, assessed on days 1 to 14. A statistical comparison would ordinarily need to define the outcome summary or model, the participants whose measurements contribute, and how measurements over time are handled. The registry record does not report those analysis details. It describes measurement by weighing milk in grams and converting the result to milliliters, but it does not state how a treatment effect would be calculated or which time-point or summary would anchor the comparison.

In general, repeated daily measurements from the same participant are related rather than independent observations. An analysis of such data may need to account for within-participant correlation and changes over time. This is a general statistical consideration, not a description of the method used in this trial. The registry record reports no analysis, so it is not possible to determine whether a repeated-measures model, a summary measure, or another method was used.

Why are estimates and uncertainty important?

A treatment-effect estimate describes the observed contrast between groups according to a specified analysis. A confidence interval, in general, communicates the precision of that estimate and the range of effects compatible with the data and method. A significance test, if planned and reported, would address a related but different question about compatibility with a null hypothesis. None of these primary comparison results is reported in the registry record. The outcome values themselves are not presented, so no estimate, confidence interval, or test result can be interpreted.

The record reports 17 outcome measures but zero analyses. This means the listed outcomes cannot be treated as evidence of a result. The secondary outcome descriptions include threshold-based measures, milk-volume measures, feeding volumes, and milk concentration. In general, threshold outcomes and continuous volume outcomes answer different questions: a threshold outcome concerns reaching a specified level, while a volume outcome describes the measured quantity. No analysis method or estimate for either type is reported.

What can be said about power and sample size?

The registry record does not report the trial's own sample-size justification, target enrollment rationale, or planned statistical power. It is therefore not possible to assess whether the original design was considered adequate for a planned treatment effect. Any planning illustration based on an observed effect is a hypothetical calculation, not observed power and not evidence that a trial can detect an effect. In this case, no primary treatment-effect estimate is posted from which to interpret such an illustration. The trial's enrollment and early termination do not substitute for a reported design justification.

How should the safety counts be interpreted?

Adverse-event entries are descriptive counts by group. General statistical interpretation would depend on the event definitions, the observation period, completeness of reporting, and the number of participants contributing safety information. The registry record gives counts at risk and counts with events, but does not report an inferential comparison, adjustment, or uncertainty interval. Given the limited enrollment, these counts should not be read as a precise estimate of comparative risk.

Over-dispersion is a general concern when event counts vary more than a simple statistical model expects; it can make standard errors too small if the model does not account for that extra variation. The registry record does not report a count model or any analysis of adverse-event rates. There is therefore no basis for claiming that over-dispersion was assessed or that a particular model was used.

In short, the registry record gives design descriptors, outcome definitions, termination information, and selected safety counts. It does not give the statistical analysis plan, analysis populations, handling of missing outcome measurements, or inferential results. General methods can explain what such analyses might address, but they cannot fill in the unreported methods or results for this trial.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists 17 outcome measures, including the primary daily MoM volume and secondary measures of milk volume, feeding, threshold attainment, and merotocin concentration in milk. It reports zero analyses. The record does not report a multiplicity strategy, such as a prespecified hierarchy, adjustment procedure, or rule for interpreting multiple outcome tests. Without results and an analysis plan, it is not possible to determine how any multiplicity concerns were handled or whether selected outcomes were emphasized.

The primary outcome description says that data, if collected, are not presented to protect participant privacy. This statement should not be rewritten as a claim that data were definitely missing: the record leaves open whether the data were collected. It does mean that no primary outcome values or analysis are available in the registry record. The reasons for any uncollected or unavailable measurements, and any methods used to address missingness, are not reported.

Analysis populations are also not reported in the registry record. It does not specify who was included in an efficacy analysis, whether participants were analyzed according to randomized assignment, or how participants with incomplete measurements would have been handled. These details matter because different analysis populations and missing-data approaches can lead to different estimates. With no analyses posted, none can be assessed for this trial.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: infections nec (50.0%).

The registry record reports serious adverse events for 2 participants at risk in the merotocin group and 2 at risk in the placebo group. It records 0 serious adverse events in the merotocin group and 1 in the placebo group. Deaths are reported as 0 in both groups. The leading serious-event term listed is Infections NEC (50.0%). The record does not identify the group for that listed term in the summary, so it should not be assigned to a group without further information.

For other adverse events, the record reports 0 affected participants among 2 at risk in the merotocin group and 1 among 2 at risk in the placebo group. These are small descriptive counts, not a statistically adjusted comparison. The registry record does not provide event-level details, timing, duration, severity definitions beyond the serious-event category, or a formal test of group differences.

Counts from a trial with limited enrollment cannot establish a stable estimate of the frequency of uncommon events. Nor do these entries alone demonstrate that an event was caused by a treatment. The reported counts should be read as registry safety information, with the absence of a posted analysis and the early termination kept in view.

12. What the Result Does, and Does Not, Mean

The record establishes that a phase 2 randomized, double-blind, placebo-controlled, multicenter trial of merotocin was conducted for mothers with preterm delivery and inadequate milk production. It identifies daily Mother's Own Milk volume as the primary endpoint and states that the trial was terminated because recruitment was slow. These are meaningful details about the trial's design and conduct.

It does not establish whether merotocin increased, decreased, or had no effect on milk volume. The primary data are not presented, and the registry record reports no analyses. There is consequently no posted treatment-effect estimate, no measure of its precision, and no basis for a formal efficacy conclusion. Missing results should not be interpreted as a null result.

The same limitation applies to the listed secondary outcomes. Their presence in the registry record indicates what was intended for assessment, but does not show what happened to milk-volume thresholds, feeding volumes, or merotocin concentration in milk. No conclusions about these outcomes can be drawn from the outcome listings alone.

The safety counts offer limited descriptive information, but do not provide a robust comparison of harms. Small counts cannot clarify the frequency of events beyond the participants recorded, establish causality, or rule out events that were not observed. The early termination and limited enrollment further restrict what the record can show.

Accordingly, the appropriate reading is narrow: the trial's design and termination are documented, and selected safety counts are reported, but the primary efficacy result is unavailable. The registry record does not support a claim that merotocin works, does not work, or changes safety relative to placebo.

13. Why This Trial Matters Statistically

This trial matters statistically because it illustrates the difference between a registered study question and an answerable treatment comparison. The registry record describes randomization, masking, a placebo comparator, and a clear primary measurement: daily MoM volume over days 1 to 14. Yet those design features cannot produce an interpretable result in the absence of outcome data and analyses.

Early termination due to slow recruitment is also important context. A trial that ends before generating a reportable primary comparison may leave uncertainty rather than resolve it. The registry record does not report the reasons for recruitment difficulty beyond describing recruitment as slow, nor does it provide enough information to assess how termination affected the planned analysis.

The entry also distinguishes an outcome's registration from its reporting. Seventeen outcome measures are listed, but zero analyses are reported. That gap matters for readers who might otherwise mistake a detailed outcome list for evidence that results are available. It also makes it impossible to assess precision, multiplicity, or whether the intended outcomes were analyzed as planned.

Finally, the stated privacy rationale highlights a practical tension in trial reporting: participant privacy matters, while interpretable aggregate results are needed for scientific evaluation. The record does not explain whether any alternative aggregate reporting was considered. What can be learned is therefore mainly methodological: registered design details and safety counts do not replace a transparent, analyzable efficacy result.

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 central limitation is the absence of primary outcome data. The registry record says that data, if collected, are not presented in order to protect participant privacy. It therefore provides no daily milk-volume results, treatment-effect estimate, or uncertainty measure. It also reports zero analyses, leaving the intended statistical comparison unevaluable.

The trial was terminated due to slow recruitment, and enrollment was 4. The registry record does not provide a target enrollment, a sample-size justification, or a detailed account of recruitment. It is not possible to determine from the record how the termination affected data collection or whether any outcomes were available for analysis.

Important methodological details are not reported in the registry record. These include the eligibility criteria in detail, treatment dose and duration, implementation of randomization and masking, analysis populations, missing-data methods, and the statistical analysis plan. Multiplicity handling is also not reported despite the listing of 17 outcome measures.

The safety information is limited to small descriptive counts and does not include an inferential comparison or detailed event-level context. The listed serious-event term does not, in the record summary, identify a treatment group. These limitations prevent a precise assessment of comparative safety as well as efficacy.

These gaps should not be resolved by assuming that an unreported result was favorable, unfavorable, or null. They define what the registry record can support: a description of design, termination, and reported safety counts, but not an efficacy conclusion.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Merotocin in Mothers With Inadequate Milk Production and Infants Delivered Prematurely — NCT02545127.
• Registry record last updated 2024-02-07; status last verified 2022-11.
• Results first posted 2024-02-07.
• 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.

18. Record Summary

NCT02545127 was a phase 2 randomized, double-blind, placebo-controlled, multicenter, parallel-group trial of merotocin in mothers with preterm delivery and inadequate milk production. The primary outcome was daily Mother's Own Milk volume measured on days 1 to 14, with milk weight in grams converted to milliliters. Enrollment was 4, and the trial was terminated because recruitment was slow.

The registry record states that primary outcome data, if collected, are not presented to protect participant privacy. It lists 17 outcome measures and reports zero analyses. Thus, no primary efficacy estimate or comparison is available. Safety counts are reported descriptively, including serious adverse events and deaths, but no formal comparative safety analysis is reported.