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 Open-Label Study of OTO-201 for Treatment of AOMT (NCT02408796), a na phase 2 study. The primary outcome is Number of Ears With Otorrhea (Drainage From the Middle Ear).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02408796 |
| Phase | Phase 2 |
| Condition | Acute Otitis Media, AOMT |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 39 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Otonomy, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric subjects with acute otitis media with tympanostomy tubes in place (AOMT), involving either one affected ear or both. The primary outcome population is described as the safety analysis set: enrolled and treated subjects.
Intervention
A single administration of 6 mg OTO-201 to the affected ear or ears. The study is described as open-label.
Comparator
There is no comparator group in this single-group study. Allocation is recorded as not applicable, and the registry record does not describe a randomized comparison.
Primary question
At Day 29, how many affected ears have otorrhea, or middle-ear drainage, after OTO-201 administration? The registry record does not report the observed result for this endpoint, so it does not establish the number of ears with drainage at that assessment.
The study also set out to characterize safety, procedural factors and clinical effect during a one-month study. Its primary endpoint is an ear-level count, rather than a participant-level measure: a subject could have one or both ears affected. That distinction matters when interpreting the outcome, because the count concerns affected ears while the described analysis population concerns enrolled and treated subjects.
The registry description states that all subjects had otorrhea in the affected ear at baseline and that the assessment followed administration. The endpoint therefore concerns the presence of drainage at the later assessment, not a comparison between treatment groups. Without a reported endpoint count, and without a control group, the registry record cannot show the magnitude of any change or separate a treatment-associated pattern from other explanations.
3. Trial Design
This was a prospective, multicenter, open-label Phase 2 study of OTO-201 for AOMT. It used a single-group design, with no comparator arm described in the registry record. The study status is completed. The recorded enrollment is 39.
The intervention is described as a single 6 mg administration of OTO-201 to affected ears. Subjects could have unilateral or bilateral AOMT. Because there is no control group, the study is not a randomized comparison of OTO-201 against another treatment or against no treatment. The registry record does not report a masking procedure; masking is recorded as none.
The study is described as lasting one month, with the primary outcome assessed at Day 29. The registry record gives a start of 2015-03 and primary completion of 2015-05. It does not report a more detailed visit schedule or the timing of assessments for every listed outcome.
The primary outcome population is described as the safety analysis set, comprising all enrolled and treated subjects. The adverse-event group also reports 39 subjects at risk. The record does not give a separate primary efficacy analysis population or explain how subjects with missing endpoint assessments were handled.
The study description says it was designed to characterize safety, procedural factors and clinical effect. The registry record does not report a sample-size justification, a planned statistical analysis protocol, or a comparator-based design. No randomized treatment contrast can be estimated from a single-group study, and the posted primary outcome does not include a reported observed count or inferential result.
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 | OTO-201 |
|---|---|
| STARTED | 39 |
| COMPLETED | 39 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety analysis set: All enrolled and treated subjects; subjects could have either unilateral or bilateral (one ear or both ears) acute otitis media with tympanostomy tubes in place (AOMT)
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 | OTO-201 |
|---|---|---|
| Participants analysed | 39 | |
| Age, Continuous | years | 2.16 (SD 1.170) |
| Sex: Female, Male: Female | Participants | 17 |
| Sex: Female, Male: Male | Participants | 22 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 16 |
| Race (NIH/OMB): White | Participants | 22 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 39 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Ears With Otorrhea (Drainage From the Middle Ear), measured over Day 29.
| Element | Registry entry |
|---|---|
| Result type | Count Of Units |
| Measure of spread | not applicable |
| Unit | affected ears |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of affected ears with otorrhea at Day 29. Otorrhea is described in the registry record as fluid drainage from the middle ear. The assessment is made during otoscopic examination of each affected ear; affected ears are those with the infection.
This endpoint is used to describe whether drainage is present at the specified follow-up assessment. The registry description says all subjects had otorrhea in the affected ear at baseline and that the follow-up assessment was conducted after OTO-201 administration to check for remaining infection or a new infection. The endpoint is therefore a count of ears meeting the stated condition at Day 29, not a direct measure of the amount of drainage or a participant-level symptom score.
Counting ears can be informative for a condition that may affect one or both ears. However, the unit counted differs from the unit in the stated safety population. When a subject contributes two affected ears, those observations belong to the same subject and may be related. General statistical practice would account for such within-subject dependence when estimating uncertainty from ear-level observations; the registry record does not describe whether or how that was done.
The observed Day 29 count, its uncertainty, and any comparison with baseline are not reported in the registry record. Consequently, the endpoint definition is available, but the recorded result cannot be interpreted as a quantified change or treatment effect.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically interpretable outcome: the number of affected ears with otorrhea at Day 29. It also describes the population, intervention and assessment method. However, the record does not report the observed primary endpoint count. It therefore does not show how many ears had drainage at the assessment, nor does it provide an estimate of the precision of that result.
There are no reported analyses in the registry record. No confidence interval, p-value, model estimate or other inferential result is given for the primary endpoint. The record also does not report a numerical comparison with baseline. The description that all subjects had otorrhea at baseline is contextual information about eligibility or baseline status; it is not itself a follow-up result.
The single-group, open-label design is important to interpretation. Because there is no concurrent comparator, any observed follow-up pattern would be descriptive of the treated group. It would not, by itself, isolate the effect of OTO-201 from changes over time, other influences or the way outcomes were assessed. Open-label status also means the registry does not describe masking of treatment or outcome assessment. The record does not provide details that would permit assessment of how expectations may have affected assessment.
The endpoint is counted by ear, while subjects could contribute one or both affected ears. General statistical reasoning cautions that ears from the same subject may not be independent. If they are treated as independent when they are not, uncertainty estimates can be too narrow. The registry record does not report a method for handling this possible clustering, and no numerical uncertainty estimate is available to evaluate.
The safety counts are reported separately. Among 39 subjects at risk, 0 are recorded as having a serious adverse event and 0 deaths are recorded; 23 are recorded in the other adverse-event category. These counts describe the registry entries, but no event terms are listed among the top serious terms. The record does not give further detail on the nature, timing or severity of the other adverse events.
Overall, the posted information characterizes the design, endpoint definition and selected safety counts, but it does not provide the primary efficacy result or an inferential analysis. It supports a description of what the study intended to assess, not a quantified conclusion about the endpoint or a causal estimate of treatment effect.
9. Statistical Methods Explained
The registry record describes a count outcome and a single-group study, but reports no analyses. The statistical concepts below are general explanations of how such data may be considered; they are not claims about methods used in this study.
What does an ear count represent?
The primary endpoint counts affected ears with otorrhea at Day 29. A count records how many units meet a condition. Here the unit is an affected ear, while the outcome population is described in terms of subjects. A subject with bilateral AOMT can contribute observations from both ears, so the number of ears is not necessarily the same as the number of subjects.
Counts need to be interpreted with their eligible population and assessment rules in mind. The registry record explains which ears are considered affected and when the outcome is assessed, but it does not report the observed count. It also does not give a denominator for the primary endpoint result or describe whether every affected ear was assessed at Day 29. Those details would be needed to understand a reported count fully.
Why can ears from the same subject be related?
General: observations from the same person can share characteristics, so paired ears may not behave like observations from unrelated people. Statistical methods that assume independence can misstate uncertainty when this dependence is present. Methods for clustered or paired data can account for the relationship, depending on the estimand and design. The registry record does not report whether a method of this kind was used.
That issue concerns the uncertainty around an estimate, not the basic definition of the endpoint. The result is still an ear count. But if an analysis treats every ear as an independent observation, its uncertainty calculation may not reflect the way ears are grouped within subjects. No analysis results or method descriptions are reported to determine how this issue was addressed.
What would a confidence interval add?
General: a confidence interval expresses uncertainty around an estimated quantity under a specified statistical model and sampling framework. It gives a range of values compatible with the data and assumptions; it does not guarantee that the true value lies within that particular interval. A narrow interval indicates greater precision under those assumptions, while a wide interval indicates less precision.
No confidence interval is reported for the primary endpoint. Without one, readers cannot assess the statistical precision of an endpoint estimate from the registry record. Since the endpoint estimate itself is also not reported, there is no numerical result to which an interval could be attached.
What is the role of a comparator?
General: a comparator helps distinguish a treatment-group outcome from changes that might also occur without the intervention or under another intervention. A randomized comparison can support causal interpretation when the design and analysis are appropriate. This trial is recorded as single-group, with allocation not applicable, so it has no concurrent control arm.
For that reason, a follow-up count in this study, if reported, would describe outcomes in the treated group. It would not provide a between-group treatment contrast. The record does not report a statistical analysis that estimates a causal effect against a control condition.
What do the planning assumptions mean?
General: sample-size calculations for a future comparative study use assumptions about the effect to detect, the significance level, power, allocation and analysis method. A planning calculation is an illustration of design requirements under those assumptions; it is not a result observed in the current study, nor does it show the probability that the current study detected an effect.
The registry record’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a stated fraction of the observed effect. It also distinguishes planning outputs as participants per arm, except for time-to-event calculations, which are described as total events. Those are planning assumptions, not the design of this single-group trial. The record does not report a sample-size justification used to plan this study, and no observed primary effect is reported in the registry record for readers to evaluate against such planning assumptions.
Why does over-dispersion matter for count data?
General: some count outcomes vary more than a simple statistical model expects. This is called over-dispersion. If a model assumes less variability than the data actually have, its uncertainty estimates can be too optimistic. A suitable analysis may need to account for the observed variation, clustering or excess zeros, depending on the data and study question.
The registry record does not report a model for the ear count, a dispersion estimate or an analysis result. There is therefore no basis to say that over-dispersion was present or that a particular count model was used. These are considerations for analysis, not conclusions about this trial.
In short, the registry record provides an endpoint definition and a design description, but no reported primary endpoint analysis. Statistical interpretation is consequently limited to explaining what the endpoint would measure and what kinds of assumptions could matter, rather than evaluating a fitted model or quantified treatment contrast.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists three outcome measures and identifies the number of analyses as 0. It names the primary outcome as the number of affected ears with otorrhea at Day 29 and lists a secondary outcome concerning ears with no more otorrhea. The remaining outcome-measure details are not reported in the registry record.
When a study assesses multiple outcomes or makes multiple statistical comparisons, the chance of at least one apparently notable result can increase. General statistical practice may address this multiplicity through a prespecified testing hierarchy or another adjustment strategy. The registry record does not report a multiplicity plan, a testing hierarchy or an adjustment method. With no analyses reported, there are no posted p-values to interpret in light of multiple testing.
Missing-data procedures are also not reported in the registry record. The record does not state how missing Day 29 assessments were handled, whether any endpoint values were excluded, or whether any imputation was used. The primary outcome population is described as the safety analysis set of enrolled and treated subjects, but this does not specify the analysis denominator for the ear-level endpoint or establish that every subject or affected ear contributed a Day 29 observation.
The registry record does not report a separate intention-to-treat analysis population, a per-protocol population, or sensitivity analyses for missing data. These omissions leave uncertainty about how the primary endpoint would have been analyzed if observations were incomplete. They do not establish that data were missing or that any particular handling method was applied.
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 |
|---|---|---|---|
| OTO-201 | 0 | 39 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| OTO-201 | 0 of 39 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| OTO-201 | 23 of 39 |
Most frequent non-serious adverse event terms
| Event term | Organ system | OTO-201 |
|---|---|---|
| Pyrexia | General disorders | 9 of 39 |
| Device Extrusion | General disorders | 4 of 39 |
| Sinusitis | Infections and infestations | 3 of 39 |
| Otitis Media | Infections and infestations | 2 of 39 |
| Otitis Media Acute | Infections and infestations | 2 of 39 |
| Pharyngitis Streptococcal | Infections and infestations | 2 of 39 |
| Diarrhea | Gastrointestinal disorders | 2 of 39 |
| Seasonal Allergy | Immune system disorders | 2 of 39 |
The registry record reports one OTO-201 group, with 39 subjects at risk for serious adverse events, 0 subjects affected by serious adverse events, 39 at risk for other adverse events, and 23 affected by other adverse events. It also records 0 deaths. These are the reported safety counts, not a comparison between treatment groups.
The record does not list any terms among the top serious adverse events. A count of 0 affected subjects in the serious-event field is distinct from a complete account of all possible safety outcomes: the registry entry does not provide detailed event descriptions, timing, severity or a narrative assessment. The 23 subjects recorded in the other-adverse-event category are not described further in the registry record in the registry record.
These counts should not be read as evidence that an event cannot occur, or as a comparison with another treatment. General statistical interpretation recognizes that observed safety counts describe what was recorded in a particular study population; they do not by themselves determine the frequency of events in a broader population. No additional safety analysis or uncertainty estimate is reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study examined OTO-201 in pediatric subjects with AOMT, using a prospective, multicenter, open-label, single-group design. It defines the primary outcome as the number of affected ears with otorrhea at Day 29 and reports selected safety counts. It does not report the primary outcome result, a numerical treatment-effect estimate, or inferential analyses.
Accordingly, the posted information does not establish how many ears had otorrhea at Day 29. It does not quantify a change from baseline, because no follow-up count or baseline-to-follow-up comparison is reported. Nor does the single-group design provide a controlled contrast that could separate the treatment-associated course from other possible explanations for outcomes over time.
The safety entries report 0 subjects with serious adverse events and 0 deaths, with 23 subjects affected in the other adverse-event category among 39 at risk. These counts are limited to the registry’s recorded categories and do not include detailed event terms for the other events. They should be interpreted as descriptive entries, not as a comparison or a complete characterization of risk.
General: a study’s design and endpoint can define a useful question even when results are not available in the registry record. But an endpoint description is not an endpoint result, and an intended assessment is not evidence of its observed outcome. Readers cannot judge the size, direction or precision of the primary finding from the recorded information.
The study therefore documents an investigation and the questions it was designed to address. It does not, through the registry results, establish a quantified clinical effect or support a causal claim about OTO-201. That distinction is especially important because there is no comparator group and no reported analysis of the primary endpoint.
13. Why This Trial Matters Statistically
This study is statistically notable for the way its outcome unit and population unit differ. The primary endpoint counts affected ears, while the population description refers to subjects, who could have unilateral or bilateral AOMT. General analysis of such data may need to account for the possibility that two ears from one subject are more alike than ears from different subjects. The registry record does not describe how that issue was handled.
The design also illustrates the limits of single-group evidence. A treated-group outcome can describe what was observed after treatment, but without a concurrent comparator it cannot by itself isolate a treatment effect. This limitation remains even when an endpoint is clinically direct and assessed at a specified follow-up time.
Another statistical lesson is the distinction between a complete endpoint definition and a reported endpoint result. The record explains what was assessed at Day 29, yet does not give the resulting count or an uncertainty estimate. Without those, readers cannot evaluate the magnitude or precision of the primary finding.
Finally, the study’s stated planning assumptions are described as illustrations based on an observed effect, not as the trial’s own design justification. General sample-size planning depends on assumptions about a future comparison and should not be confused with observed power or a result from this single-group study. The registry record does not report its sample-size justification or primary analysis methods.
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 are specific to the information and design recorded for this study. It is open-label and single-group, with no comparator arm. Those features mean that the registry record cannot support a controlled estimate of the effect of OTO-201, and it does not describe masking that might reduce influence on outcome assessment.
The primary endpoint is assessed at Day 29, within a study described as one month in duration. The record does not report later follow-up for this endpoint, so it cannot describe whether any observed status persisted beyond the stated assessment. The actual Day 29 endpoint count is not reported in the registry record, which prevents assessment of the outcome’s magnitude and precision.
The endpoint counts ears, but subjects could have one or both ears affected. The registry record does not state how analyses handled possible dependence between ears from the same subject. It also does not report the endpoint denominator, missing-data handling, statistical model or sensitivity analyses.
The study description identifies pediatric subjects, but the registry record does not provide age details in the registry record. It gives no detailed account of the other adverse events and lists no serious-event terms. These limits concern what can be interpreted from this record; they do not establish that any particular data, events or analyses were absent from the full study documentation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Open-Label Study of OTO-201 for Treatment of AOMT — NCT02408796.
- Registry record last updated 2020-10-19; status last verified 2020-09.
- Results first posted 2020-09-23.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This completed Phase 2 study evaluated a single 6 mg administration of OTO-201 in pediatric subjects with acute otitis media with tympanostomy tubes. It was prospective, multicenter, open-label and single-group, with recorded enrollment of 39. The primary endpoint was the number of affected ears with otorrhea at Day 29.
The registry record does not report the observed primary endpoint count, a confidence interval, a p-value or any primary outcome analysis. It lists 0 subjects with serious adverse events, 23 with other adverse events and 0 deaths among the recorded safety counts. The design has no comparator, and details on missing-data handling, multiplicity and the primary analysis method are not reported in the registry record.