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 Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants (NCT02205034), a non-randomized phase 1, phase 2 study. The primary outcome is Occurence of Adverse Event.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02205034 |
| Phase | Phase 1, Phase 2 |
| Condition | Prader Willi Syndrome |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 18 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University Hospital, Toulouse |
| Results posted | Yes |
2. Clinical Question
The study addresses whether repeated intranasal oxytocin administration is tolerated in infants with Prader-Willi syndrome (PWS), and whether suckling and swallowing measures can be evaluated in this setting. The registry describes severe neonatal hypotonia and impaired suckling as concerns in PWS, and gives the investigators’ hypothesis that early oxytocin administration might improve suckling and possibly infant-mother interactions. Those statements describe the study rationale, not demonstrated effects.
Population
Infants younger than 5 months with Prader-Willi syndrome. The registry records 18 participants enrolled, with 6 at risk in each of the three arm entries.
Intervention
Repeated intranasal oxytocin administered on one of three schedules: 4IU every other day, 4 IU daily, or 4IU twice daily.
Comparator
The registry describes a non-randomized, parallel-arm study and does not identify a placebo or untreated control group. The dose groups are not a like-for-like randomized comparison.
Primary question
What adverse events occur during the 7 days following the first administration, including their description, severity, and attribution to repeated intranasal oxytocin, up to day 8 (Visit 8)?
The registry also lists NOMAS Score and Videofluoroscopy of Swallowing Score as secondary outcomes. Their result values and analyses are not reported in the registry record. Therefore, the record can describe the recorded safety counts, but it cannot answer whether the dose schedules changed suckling or swallowing outcomes.
3. Trial Design
This was a Phase 1/Phase 2, non-randomized, unmasked, parallel study sponsored by University Hospital, Toulouse. The registry records 18 participants enrolled and a completed status. The recorded study start is 2013-05, and primary completion is 2014-07. The study population was infants younger than 5 months with Prader-Willi syndrome.
The intervention was intranasal oxytocin. The registry describes three dose schedules: 4IU every other day in the First Arm, 4 IU daily in the Second Arm, and 4IU twice daily in the Third Arm. The brief summary characterizes the study as an escalating dose study conducted in three steps. The registry does not report enough detail to establish the timing or operational rules for moving between schedules, so those details should not be inferred.
Because allocation was non-randomized, the arms should not be read as randomized treatment groups. The record does not identify a placebo or untreated comparator. Differences in recorded adverse events between arms may reflect differences in participant characteristics or other factors, as well as the dose schedule; the registry does not report an adjustment strategy that would separate these possibilities.
The primary outcome concerns adverse events during the 7 days following the first administration, with assessment up to day 8 (Visit 8). It includes occurrence, description, severity, and attribution to repeated intranasal oxytocin. NOMAS Score and Videofluoroscopy of Swallowing Score are listed as secondary outcomes. Their numerical findings are not reported in the registry record. The record also does not report a detailed protocol for allocation, follow-up, or outcome assessment.
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 | First Arm | Second Arm | Third Arm |
|---|---|---|---|
| STARTED | 6 | 6 | 6 |
| COMPLETED | 6 | 6 | 6 |
| NOT COMPLETED | 0 | 0 | 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 | First Arm | Second Arm | Third Arm | Total |
|---|---|---|---|---|---|
| Participants analysed | 6 | 6 | 6 | 18 | |
| Age, Categorical: <=18 years | Participants | 6 | 6 | 6 | 18 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 3 | 1 | 4 | 8 |
| Sex: Female, Male: Male | Participants | 3 | 5 | 2 | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Occurence of Adverse Event, measured over up to day 8 (Visit 8).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | number of adverse events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the occurrence of adverse events, with their description and quantification of severity and their imputability to repeated intranasal oxytocin. The registry specifies assessment during the 7 days following the first administration, up to day 8 (Visit 8). This endpoint is used to characterize short-term tolerability across the three dosing schedules.
Safety outcomes can include events that differ in seriousness and clinical description. Recording both occurrence and severity helps distinguish the presence of an event from its seriousness; attribution records whether the event is considered related to the intervention. The registry does not report the rules used to judge severity or imputability, so those judgments cannot be independently interpreted from the posted record alone.
The group-level adverse-event entries list participants at risk and participants affected by serious and other adverse events. These are not necessarily counts of every event: a participant may experience more than one event, and the registry record does not clarify whether the primary endpoint's event counts correspond to the participant counts in those entries. NOMAS Score and Videofluoroscopy of Swallowing Score are listed as secondary outcomes, but their results are not reported in the registry record. The safety endpoint therefore does not, by itself, establish an effect on suckling or swallowing.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The adverse-event entries list 6 participants at risk in each arm. In the First Arm, 1 participant was affected by serious adverse events and 4 by other adverse events. In the Second Arm, 0 participants were affected by serious adverse events and 5 by other adverse events. In the Third Arm, 0 participants were affected by serious adverse events and 4 by other adverse events. No deaths are listed in any arm. The registry lists pneumopathy as a serious adverse event term at 16.7%; its denominator and arm-specific attribution are not reported in the registry record.
These figures describe recorded affected participants within a small, non-randomized study. They should not be treated as a direct comparative estimate of risk between schedules. With no random allocation, differences between arms cannot be attributed to dose alone. The registry does not report baseline characteristics or adjusted comparisons that would help assess whether the groups were comparable. Nor does it report uncertainty intervals or hypothesis tests for the safety findings.
The primary endpoint concerns events during a short follow-up window: the 7 days following the first administration, up to day 8 (Visit 8). It therefore describes the recorded short-term safety experience over that specified period, not events outside it. The record does not report longer-term safety findings. The event counts also do not establish the number of distinct event episodes, because the posted group entries identify participants affected, and further event-level detail is not reported.
The study rationale refers to possible improvement in suckling and infant-mother interactions, and the registry lists NOMAS Score and Videofluoroscopy of Swallowing Score as secondary outcomes. However, their numerical results and analyses are not reported in the registry record. Thus, the posted results cannot establish whether either outcome changed, whether any change differed among schedules, or how precisely such changes were estimated.
Overall, the record supplies descriptive safety counts for the three dose groups. It does not report a randomized comparison, an inferential analysis of the adverse-event counts, or numerical findings for the listed secondary outcomes. Those absences limit the conclusions that can be drawn from the posted results; they do not imply that an unreported outcome had no effect or that an unreported analysis was conducted.
9. Statistical Methods Explained
Statistical methods describe how study observations are organized and how conclusions are drawn from them. In this record, the available results are descriptive adverse-event counts by arm. No analysis results are reported in the registry record. The explanations below distinguish what the design and posted counts show from general statistical concepts that help interpret such data.
What does non-randomized allocation mean for comparisons?
The registry identifies the study as non-randomized and parallel. In a randomized study, a chance-based assignment process is intended to balance participant characteristics across groups, on average. With non-randomized allocation, that balancing is not guaranteed. The people in the three dose groups may differ in ways related to both the schedule they received and their outcomes. Consequently, a contrast between recorded counts cannot be interpreted as an unbiased causal effect of one schedule versus another without additional design or analysis support.
The registry does not report the allocation procedure or adjusted statistical comparisons. It is therefore not possible to determine from the record whether the groups were similar at entry, how assignment was decided, or whether any measured characteristics were accounted for. The arm counts are descriptive, not proof that schedule differences caused any observed difference in adverse events.
What do the adverse-event counts represent?
General statistical distinction: a count of participants affected is different from a count of event episodes. One participant could experience multiple events, and an event could recur. The arm entries report participants at risk and participants affected, while the primary endpoint description refers to occurrence and quantification of adverse events. The registry does not report whether the group entries also provide the full number of event episodes or how repeated events were handled. These figures should therefore be read as affected-participant counts, rather than assumed to be a complete event-by-event tally.
Serious and other adverse events are listed separately. These categories communicate different aspects of safety and should not be combined or treated as interchangeable. The record reports no deaths in the arms. It also identifies pneumopathy as a serious adverse event term at 16.7%, but does not report its denominator or arm-specific distribution. That percentage should not be assigned to a particular arm or interpreted as a rate in the absence of those details.
How should uncertainty be understood?
General statistical explanation: a confidence interval describes a range of values compatible with the data under a specified statistical model and procedure. Narrower intervals generally indicate greater precision, while wider intervals indicate less precision. The registry record does not report confidence intervals for the adverse-event counts, so the precision of comparative risk estimates cannot be assessed from the record. The small number of participants at risk in each arm also means that each affected participant represents an important part of the observed group count, but no new rate or uncertainty estimate should be inferred from that observation.
A p-value, when reported, describes how incompatible the data are with a specified null hypothesis under the assumptions of a test. It does not give the probability that a treatment works, and it does not measure the size or importance of an effect. The registry record reports no hypothesis tests or p-values for these safety findings. No inferential conclusion about differences between schedules can be drawn from a test result that is not reported.
Why do statistical models matter for event data?
General statistical explanation: models for event counts rely on assumptions about how observations arise. A simple count model may be inadequate when variation in event counts is greater than the model expects, a feature called over-dispersion. In such settings, standard errors and uncertainty estimates from a too-restrictive model can be misleading. For this study, however, the registry does not report a count model, model diagnostics, or event-level data. It is not possible to say whether over-dispersion was present or whether any model was used.
Time-to-event methods answer a different question: they use information about when an event occurs and account for follow-up duration and censoring. Although the primary endpoint has a defined follow-up window, the record does not report event times or a time-to-event analysis. The available information should not be described as a survival analysis.
What can planning calculations tell us?
General statistical explanation: a sample-size calculation estimates how many participants would be needed under specified assumptions to detect a chosen effect with a chosen statistical procedure. It is a planning exercise, not a measure of the probability that a completed study has found a true effect. A retrospective calculation based on an observed effect is also not observed power; it depends on the effect estimate and assumptions used. The registry record does not report the trial's own sample-size justification or a statistical plan. Any sample-size planning illustration should therefore be distinguished from the trial's observed results and should not be treated as evidence of efficacy or safety.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when a study evaluates several outcomes, comparisons, time points, or statistical tests. If many tests are conducted, the chance of obtaining at least one apparently unusual result can increase unless the analysis plan accounts for the number and relationship of the tests. General statistical approaches may control that risk, but the appropriate method depends on the planned questions and analysis structure.
The registry lists the primary adverse-event endpoint and two secondary outcomes: NOMAS Score and Videofluoroscopy of Swallowing Score. It does not report a multiplicity strategy, a hierarchy of tests, or analysis results for the secondary outcomes. The statistical plan is not reported in the registry record. It is therefore not possible to determine which comparisons were planned, whether any adjustment was used, or how secondary outcomes were intended to inform conclusions.
Missing-data handling is also not reported in the registry record. The record does not state whether all enrolled participants completed outcome assessment, how missing safety or secondary-outcome observations were handled, or whether any imputation or sensitivity analysis was conducted. Analysis populations, such as a defined set of participants used for a particular outcome analysis, are not reported either.
These are reporting limitations, not evidence that missing data or multiple testing necessarily affected the findings. Without the analysis plan and outcome-level data, readers cannot assess their possible influence. The record reports 18 enrolled participants and 6 at risk in each arm's safety entry, but it does not provide a participant-flow account or clarify the relationship between enrollment and each outcome-specific analysis population.
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 |
|---|---|---|---|
| First Arm | 1 | 6 | 16.7% |
| Second Arm | 0 | 6 | 0.0% |
| Third Arm | 0 | 6 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| First Arm | 0 of 6 |
| Second Arm | 0 of 6 |
| Third Arm | 0 of 6 |
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 |
|---|---|
| First Arm | 4 of 6 |
| Second Arm | 5 of 6 |
| Third Arm | 4 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | First Arm | Second Arm | Third Arm |
|---|---|---|---|---|
| Pneumopathy | Infections and infestations | 1 of 6 | n/a of 6 | n/a of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | First Arm | Second Arm | Third Arm |
|---|---|---|---|---|
| Pyrexia | General disorders | 3 of 6 | 0 of 6 | 0 of 6 |
| Gastro-oesophageal reflux disease | Gastrointestinal disorders | 1 of 6 | 1 of 6 | 0 of 6 |
| Conjunctivitis bacterial | Infections and infestations | 0 of 6 | 2 of 6 | 0 of 6 |
| Urinary tract infection | Infections and infestations | 0 of 6 | 2 of 6 | 0 of 6 |
| Diarrhoea | Gastrointestinal disorders | 0 of 6 | 1 of 6 | 1 of 6 |
| Culture urine positive | Investigations | 0 of 6 | 0 of 6 | 2 of 6 |
| Hypothyroidism | Endocrine disorders | 0 of 6 | 0 of 6 | 2 of 6 |
| constipation | Gastrointestinal disorders | 1 of 6 | 0 of 6 | 0 of 6 |
| Nasopharyngitis | Infections and infestations | 1 of 6 | 0 of 6 | 0 of 6 |
| Teething | Gastrointestinal disorders | 1 of 6 | 0 of 6 | 0 of 6 |
Serious events with the highest arm-level rate: pneumopathy (16.7%).
The registry's adverse-event entries list 6 participants at risk in each arm. Serious adverse events affected 1 participant in the First Arm, 0 in the Second Arm, and 0 in the Third Arm. Other adverse events affected 4 participants in the First Arm, 5 in the Second Arm, and 4 in the Third Arm. The recorded number of deaths is 0 in each arm.
These are descriptive counts, not evidence from a randomized comparison. The study is non-randomized, and the registry does not report an inferential comparison or uncertainty intervals. The counts should not be used to conclude that one schedule is safer than another. The record lists pneumopathy as a serious adverse event term at 16.7%, but does not report the denominator or which arm it pertains to.
Participants affected are not necessarily the same as the total number of event episodes. The registry does not clarify whether individuals could contribute multiple events to the primary endpoint count. The safety window is the 7 days following the first administration, up to day 8 (Visit 8); longer-term safety is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The posted results show that adverse events were recorded in each of the three dose groups during the specified short-term assessment. Serious adverse events were recorded for 1 participant in the First Arm and for 0 participants in the Second and Third Arms. Other adverse events were recorded for 4, 5, and 4 participants in the First, Second, and Third Arms, respectively. No deaths are listed. These are the descriptive observations in the registry, not estimates from a randomized comparison.
The record does not establish that the dose schedules caused or prevented the recorded events. Non-randomized allocation allows group differences unrelated to dose, and the registry does not report adjusted analyses or uncertainty estimates. The serious adverse event term pneumopathy is listed at 16.7%, but the record does not provide enough information to assign that percentage to an arm or interpret its denominator.
The findings also do not answer whether oxytocin changed suckling or swallowing. The study rationale describes those outcomes as important questions, and NOMAS Score and Videofluoroscopy of Swallowing Score are listed as secondary outcomes. Their numerical findings and analyses are not reported in the registry record. The stated hypothesis about suckling and infant-mother interactions should therefore be understood as a rationale, not a result.
The assessment window ends at day 8 (Visit 8), covering the 7 days following the first administration. The record does not report longer-term outcomes. The absence of a reported outcome or analysis is not evidence that no event or change occurred; it means that the registry record does not permit that question to be answered. Overall, the data support a description of recorded short-term adverse-event counts, but not a definitive comparison among schedules or a conclusion about the secondary outcomes.
13. Why This Trial Matters Statistically
This trial raises statistical questions that are especially important when studying a small group of infants and recording safety outcomes across dose schedules. The registry reports 18 enrolled participants and 6 at risk in each arm's adverse-event entries. With counts of this size, individual observations make a visible contribution to the group summaries. Estimates of between-group differences would require careful attention to uncertainty, but the registry does not report such estimates or intervals.
The non-randomized design is central to interpretation. A dose-escalation rationale does not by itself make dose groups comparable. If assignment is related to participant characteristics, those characteristics may also relate to observed outcomes. Descriptive arm counts remain useful for reporting what was recorded, but they cannot isolate the effect of schedule in the way a well-executed randomized comparison is intended to do.
The endpoint also illustrates the importance of defining the unit of analysis. The registry describes adverse-event occurrence and quantification, while the group entries report participants affected. A participant count and an event count answer different questions. Clear reporting of event definitions, repeated events, severity grading, attribution, and follow-up would help readers understand precisely what each count represents. Several of those details are not reported in the registry record.
Finally, the study's clinical rationale and its available results should be kept distinct. The record identifies suckling and swallowing outcomes as secondary measures, but does not report their findings. Statistical interpretation depends on the outcome data and analysis plan, not only on the motivation for collecting them. This record is therefore most informative as a description of the design and reported short-term safety observations, while leaving comparative and secondary-outcome questions unresolved.
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 limitations specific to this record constrain interpretation. The study is non-randomized and has no like-for-like randomized control comparison reported. Group differences in adverse events cannot be attributed to dose schedule alone, and the registry does not report an adjustment strategy or baseline comparisons.
The record reports 18 participants enrolled and 6 at risk in each safety arm, but does not provide a participant-flow account or explain whether the safety entries include every enrolled participant. It does not report event-level detail sufficient to distinguish the number of affected participants from the total number of event episodes. Definitions and procedures for grading severity and judging imputability are not reported in the registry record.
The primary safety assessment covers the 7 days following the first administration, up to day 8 (Visit 8). Longer-term safety findings are not reported. The registry also does not report numerical results for NOMAS Score or Videofluoroscopy of Swallowing Score, despite listing them as secondary outcomes. No uncertainty intervals, hypothesis tests, or adjusted comparisons are reported for the available safety counts.
The statistical plan, sample-size justification, missing-data approach, and analysis populations are not reported in the registry record. The record also does not explain the denominator for the 16.7% serious adverse event term pneumopathy or identify an arm-specific attribution. These omissions limit assessment of precision, completeness, and the scope of the recorded findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants — NCT02205034.
- Registry record last updated 2024-02-21; status last verified 2024-01.
- Results first posted 2023-12-04.
- 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.
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18. Record Summary
The registry record describes a completed Phase 1/Phase 2, non-randomized, unmasked, parallel study sponsored by University Hospital, Toulouse. It enrolled 18 infants with Prader-Willi syndrome and evaluated repeated intranasal oxytocin using three schedules: 4IU every other day, 4 IU daily, and 4IU twice daily. The recorded study start is 2013-05, and primary completion is 2014-07.
The primary outcome concerns adverse events during the 7 days following the first administration, up to day 8 (Visit 8). The arm entries report serious and other adverse-event affected-participant counts, and 0 deaths in each arm. Pneumopathy is listed as a serious adverse event term at 16.7%, without a reported denominator or arm attribution.
NOMAS Score and Videofluoroscopy of Swallowing Score are listed as secondary outcomes, but their results are not reported in the registry record. The record also does not report a statistical plan, adjusted comparisons, confidence intervals, missing-data methods, or analysis populations. The available findings are descriptive and do not establish comparative effects of the dose schedules.