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 The Safety and Efficacy of a Peanut Immunotherapy Dissolving Film for Peanut Allergy (NCT01897077), a non-randomized phase 1, phase 2 study. The primary outcome is The Proportion of Subjects That Discontinue the Study for Treatment Related Reasons.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01897077 |
| Phase | Phase 1, Phase 2 |
| Condition | Peanut Allergy |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Johns Hopkins University |
| Results posted | Yes |
2. Clinical Question
Population
The intended population included people with peanut allergy, who were to receive gradually increasing doses of the dissolving peanut film. Healthy volunteers were also included to receive active film doses in an expedited manner for safety assessment. The registry record reports that no peanut-allergic subjects enrolled. Enrollment was 5, and the safety results list 5 healthy volunteers at risk.
Intervention
The intervention was a peanut dissolving film, a sublingual immunotherapy product. The study description identifies the film as the method of administration under investigation. The peanut-allergy group was intended to receive gradually increasing doses; the healthy volunteers were to receive active films in an expedited manner.
Comparator
No comparator group is recorded. The study was non-randomized and single-group, and the registry notes that the arms are not a like-for-like control comparison. Healthy volunteers had a different role and dosing approach from the intended peanut-allergy group, so their results cannot serve as a controlled comparison for allergic participants.
Primary question
The primary outcome concerned the proportion of subjects who discontinued the study for treatment-related reasons, with an outcome timeframe listed as 18 months. The record reports no treatment-related discontinuations among healthy volunteers and no peanut-allergic subjects enrolled. It therefore does not establish a discontinuation proportion for the intended peanut-allergy population.
3. Trial Design
The registry describes a Phase 1 and Phase 2, non-randomized, unmasked, single-group study. The enrollment listed is 5. The study began in 2012-11, had a primary completion date of 2013-02-03, and is recorded as terminated. The registry attributes the premature end to manufacturing issues with the study product.
The study was intended to examine a dissolving peanut film as a new way to administer peanut sublingual immunotherapy. The planned participant categories had different purposes. Peanut-allergic subjects were to receive gradually increasing doses. Healthy volunteers were to receive active films in an expedited manner to assess safety. The record reports that no peanut-allergic subjects enrolled, while the adverse-event results identify 5 healthy volunteers at risk.
There was no random allocation and no masking. The registry explicitly cautions that the arms are not a like-for-like control comparison. In particular, the healthy-volunteer group cannot be treated as a control arm for the intended peanut-allergy group: the groups differed in population and planned dosing approach, and the record does not report allergic participants who received the product.
The primary outcome was discontinuation for treatment-related reasons. Secondary outcomes concerned tolerance of a full 10 Gram peanut protein challenge, adverse effects, medication use, and changes in peanut-specific IgE and IgG, basophil reactivity, and salivary biomarkers. The record lists 6 outcome measures and 0 analyses. It does not report results for the planned allergy-related efficacy outcomes. Details of the protocol, including the intended statistical analysis plan and sample-size justification, are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Peanut Allergic | Healthy Volunteers |
|---|---|---|
| STARTED | 0 | 5 |
| COMPLETED | 0 | 5 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: There were no peanut allergic subjects enrolled because the study ended prematurely due to manufacturing issues with the study product.
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: The study ended prematurely before any peanut allergic subjects were enrolled.
| Characteristic | Unit | Peanut Allergic | Healthy Volunteers | Total |
|---|---|---|---|---|
| Participants analysed | 0 | 5 | 5 | |
| Age, Categorical: <=18 years | Participants | — | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | — | 5 | 5 |
| Age, Categorical: >=65 years | Participants | — | 0 | 0 |
| Sex: Female, Male: Female | Participants | — | 3 | 3 |
| Sex: Female, Male: Male | Participants | — | 2 | 2 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | — | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | — | 2 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | — | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | — | 2 | 2 |
| Race (NIH/OMB): White | Participants | — | 1 | 1 |
| Race (NIH/OMB): More than one race | Participants | — | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | — | 0 | 0 |
| Region of Enrollment: United States | participants | — | 5 | 5 |
6. Primary Endpoint
The registry lists the primary outcome as The Proportion of Subjects That Discontinue the Study for Treatment Related Reasons, measured over 18 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint concerns the proportion of subjects who discontinue the study for treatment-related reasons. Discontinuation is an outcome about whether participants remain in the study when treatment-related problems arise; it is not a direct measure of peanut challenge tolerance or a biomarker response. The registry lists participants as the unit and gives 18 months as the timeframe.
For a proportion to be interpretable, the eligible group and its denominator need to be clear, along with the number meeting the event definition. The record states that no healthy volunteers discontinued for treatment-related reasons. It also states that no peanut-allergic subjects enrolled because the study ended prematurely due to manufacturing issues. These statements do not yield a treatment-related discontinuation proportion for the intended allergic population. The registry does not clarify a separate denominator for the primary outcome across all participants.
In general, discontinuation can be used to describe treatment acceptability or problems that interfere with continued participation. It is only informative about those issues in the population actually enrolled and exposed, and its interpretation depends on how reasons for stopping were defined and assessed. Those operational details are not reported in the registry record. The endpoint also cannot substitute for the planned challenge outcome: remaining in a study does not itself establish a change in peanut tolerance.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that no peanut-allergic subjects enrolled. The registry says the study ended prematurely because of manufacturing issues with the study product. Consequently, the record cannot answer whether the dissolving film changed peanut allergy outcomes in the population the study intended to treat. It reports no efficacy estimate for allergic participants, and no conclusion about the effect of the film on challenge tolerance can be drawn from this record.
The primary endpoint is treatment-related discontinuation. The outcome description says no healthy volunteers discontinued for treatment-related reasons, but this is not a result in peanut-allergic participants. The record separately gives no allergic participants enrolled. Those details need to remain distinct: the absence of an allergic cohort means the intended population’s discontinuation experience was not observed. The registry does not provide an overall endpoint proportion or a confidence interval for that population.
For safety, the record lists 5 healthy volunteers at risk for serious and other adverse effects, with 0 affected in each category and 0 deaths. The peanut-allergy group lists 0 at risk. These observations describe the recorded healthy-volunteer experience; they do not establish safety in people with peanut allergy. The zero counts in a group with no allergic participants reflect the lack of an exposed allergic cohort, not evidence that adverse events could not occur in that population.
The study was non-randomized, unmasked, and single-group. There is no randomized comparator and the registry warns that the participant groups are not a like-for-like control comparison. As a result, the record offers no controlled estimate of a treatment effect. Nor does the record provide inferential analyses: it lists 0 analyses. The secondary outcome list includes challenge tolerance, medication use, adverse effects, and biomarkers, but results for those outcomes are not reported in the registry record.
In general, a confidence interval describes the range of values compatible with an estimate under a statistical model. No relevant efficacy estimate or interval for peanut-allergic participants is reported, so precision cannot be assessed for that intended question. A numerical result from the healthy volunteers cannot repair that gap because the population and dosing purpose differ. The appropriate reading is therefore narrow: the record describes study termination, the absence of peanut-allergic enrollment, and the recorded safety counts for healthy volunteers. It does not establish efficacy or characterize treatment-related risks in peanut-allergic subjects.
9. Statistical Methods Explained
What does the design allow?
The registry identifies a non-randomized, unmasked, single-group design. In general, random allocation can help make groups comparable at baseline, while masking can reduce the influence of knowledge about treatment assignment on some assessments. Neither feature is recorded for this study. More importantly, the record does not describe a usable comparison group: the registry explicitly says the arms are not a like-for-like control comparison.
The study also had distinct participant roles. Peanut-allergic subjects were intended to receive gradually increasing doses, while healthy volunteers were to receive active film in an expedited manner for safety assessment. Because no peanut-allergic subjects enrolled, the intended group-level treatment response cannot be estimated from the observed record. Healthy-volunteer observations do not provide a substitute estimate for allergic participants.
How should a proportion be understood?
In general, an event proportion is the count of participants experiencing the specified event relative to the eligible participants being assessed. Interpretation requires a clear event definition, an appropriate denominator, and information about whether participants had an opportunity to experience the event during the stated follow-up. The primary endpoint refers to discontinuation for treatment-related reasons, and the registry gives 18 months as its timeframe. It does not report the operational event definition or clarify the denominator for the endpoint across the study.
The registry’s outcome description reports no treatment-related discontinuations among healthy volunteers and says no active participants enrolled; it also explains that no peanut-allergic subjects enrolled. The registry entries therefore support describing those statements, but not constructing a proportion for the intended allergic population. An absent event among a group that was not enrolled is not an observed zero event rate in that group.
Why do uncertainty estimates matter?
In general, a point estimate alone does not show how much uncertainty surrounds it. A confidence interval conveys statistical uncertainty around an estimate under its model and assumptions. For event proportions, the amount of information depends on the number of eligible participants and the number of events. When no allergic participants enrolled, there is no observed allergic-population endpoint estimate to accompany with an interval. The registry reports no efficacy estimate or confidence interval for the intended population.
The safety counts in healthy volunteers are descriptive. The record lists 5 healthy volunteers at risk, 0 with serious adverse effects, 0 with other adverse effects, and 0 deaths. Such counts do not provide a controlled comparison, and the record does not report an inferential analysis. They also do not characterize risks among peanut-allergic participants, whose at-risk count is 0.
What does over-dispersion mean in general?
For count outcomes, a statistical model may assume a particular amount of variation. Over-dispersion means the observed variation is greater than that assumption allows; if ignored, uncertainty estimates can be too narrow. This is a general concept, not a reported feature of this study. The primary outcome is described as a participant count/proportion rather than a reported series of count observations, and the registry does not report a model, dispersion estimate, or analysis for it.
What can be said about multiplicity and planning?
The registry lists 6 outcome measures and 0 analyses. The secondary outcomes span challenge tolerance, adverse effects, medication use, and biomarker changes. When several outcomes are assessed, multiplicity can matter because repeated statistical testing may increase the chance of finding an apparently notable result by chance. In general, a prespecified analysis plan can state which outcomes are primary and how multiple comparisons will be handled. The registry does not report a multiplicity procedure or a statistical analysis plan, and no outcome analyses are listed.
The record does not report a sample-size justification or target precision for the intended peanut-allergy question. Any planning illustration based on an observed effect should be interpreted cautiously when the allergic group did not enroll and no relevant effect estimate is reported. Planning calculations describe assumptions about a future design; they are not observed power and cannot create evidence from an unobserved treatment response. The registry does not report interim analyses, analysis populations, missing-data methods, or a plan for handling deviations from the intended dosing schedule.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Healthy Volunteers | 5 | 0 | 0.0% | 0.0% to 52.2% | 0.0% to 43.4% | 0.4% to 45.9% |
R code and output
arm <- c("Healthy Volunteers")
n <- c(5)
events <- c(0)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Healthy Volunteers 5 0 0.0 0.0 52.2 0.0 43.4 0.4 45.911. Multiplicity, Missing Data and Analysis Populations
The registry lists 6 outcome measures, including the primary discontinuation outcome and secondary outcomes involving peanut challenge tolerance, adverse effects, medication use, and biomarker changes. It lists 0 analyses. A multiplicity adjustment or other approach to handling several outcomes is not reported in the registry record. Without reported analyses, there are no outcome-specific statistical tests or estimates to evaluate for selective interpretation.
Missing-data methods are not reported in the registry record. The record does explain why the study ended early: manufacturing issues with the study product. It also states that no peanut-allergic subjects enrolled. This is not simply a case of missing follow-up within an enrolled allergic cohort; the intended group was not enrolled at all. The record does not describe how incomplete observations among healthy volunteers, if any, were handled.
The analysis populations and any rules for inclusion in an analysis are not reported in the registry record. No interim analysis plan is reported. The registry does not provide a statistical analysis plan, a multiplicity procedure, or details of how the outcome timeframe would be applied to participants. These omissions limit assessment of whether the reported results followed prespecified methods. In general, prespecification helps distinguish planned analyses from choices made after observing results, but no such plan can be evaluated from the registry information available.
12. 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 |
|---|---|---|---|
| Healthy Volunteers | 0 | 5 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Peanut Allergic | 0 of 0 |
| Healthy Volunteers | 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 |
|---|---|
| Peanut Allergic | 0 of 0 |
| Healthy Volunteers | 0 of 5 |
The registry lists the Peanut Allergic group with 0 participants at risk, 0 serious adverse effects, 0 other adverse effects, and 0 deaths. It lists the Healthy Volunteers group with 5 participants at risk, 0 serious adverse effects, 0 other adverse effects, and 0 deaths. No serious adverse-event terms are listed.
These counts describe the safety data recorded for the enrolled healthy volunteers. They do not establish a safety profile for peanut-allergic participants, because none enrolled. A zero count in a group with 0 at risk is not evidence that the event was assessed and absent among treated allergic participants. Likewise, the healthy-volunteer findings should not be generalized to the allergic population: the registry describes different participant purposes and dosing approaches and does not present a like-for-like comparison.
In general, safety counts are most informative when the exposed population, observation period, event definitions, and completeness of reporting are clear. The registry gives 18 months as the primary endpoint timeframe, but does not report detailed safety follow-up or event-ascertainment methods. The counts should therefore be read as descriptive registry results, not as a controlled estimate of risk.
13. What the Result Does, and Does Not, Mean
The record establishes that the study was terminated and that no peanut-allergic subjects enrolled. It attributes the early end to manufacturing issues with the study product. It also records that 5 healthy volunteers were at risk for adverse effects, with 0 serious and 0 other adverse effects and 0 deaths. These are the specific observations available for interpreting the study’s posted results.
The record does not show whether the dissolving film improved peanut tolerance, whether it changed biomarkers, or whether it altered medication use in people with peanut allergy. Although those topics appear among the planned outcomes, results for them are not reported in the registry record. The primary discontinuation endpoint also cannot describe the allergic population’s experience when that population was not enrolled.
In general, absence of a reported effect estimate is different from a precise estimate showing no effect. Without enrolled peanut-allergic participants and corresponding outcome data, there is no allergic-population effect estimate to interpret as evidence for or against a treatment effect. Similarly, a small set of healthy-volunteer safety observations cannot rule out uncommon or population-specific adverse effects; this study record does not provide a controlled estimate of such risks.
The design further limits causal interpretation. There was no randomization or masking, and the registry cautions that the participant categories do not form a like-for-like control comparison. The record lists 0 analyses, so it supplies no statistical test or confidence interval that could quantify an outcome effect. These limitations mean the results are best understood as a record of study conduct and the available healthy-volunteer safety counts, not as an efficacy assessment in peanut allergy.
14. Why This Trial Matters Statistically
Statistically, this study illustrates why enrollment and outcome denominators are central to interpretation. The intended clinical question concerned peanut-allergic participants, but the registry reports that none enrolled. Without participants from that target population, the intended endpoint cannot produce an estimate for it, regardless of how the outcome is defined or what analysis might have been planned.
The record also makes clear that the healthy volunteers were not a control group for the allergic participants. They were included for safety assessment and had an expedited dosing approach, whereas allergic subjects were intended to receive gradually increasing doses. Treating these groups as interchangeable would obscure the different populations and study purposes. The registry explicitly warns that the arms are not a like-for-like comparison.
The trial’s early termination due to manufacturing issues also shows how operational constraints can prevent a study from answering its planned question. The registry lists a primary discontinuation endpoint and several secondary clinical and biomarker outcomes, but lists 0 analyses. Thus, the documented study question and the evidence actually available are not the same: planned outcomes do not become evidence when the relevant population and measurements are absent.
More generally, statistical interpretation depends on matching the estimate to the population and design that generated it. Descriptive safety counts among healthy volunteers should remain tied to those volunteers. They should not be treated as an efficacy result, a controlled comparison, or an estimate of safety for people with peanut allergy. Keeping that distinction explicit prevents the absence of data from being mistaken for a finding of no effect or no risk.
15. 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 main trial-specific limitation is the absence of peanut-allergic enrollment. The registry states that the study ended prematurely because of manufacturing issues with the study product. Consequently, the planned gradual-dose experience in allergic subjects is not represented in the posted results, and the record cannot answer the intended efficacy question.
The available safety results concern healthy volunteers, a different population with a different planned dosing approach. There is no randomized or like-for-like comparator. The study was non-randomized, unmasked, and single-group, so the record does not support a controlled treatment-effect estimate. No results for the planned challenge, medication-use, or biomarker outcomes are reported in the registry record.
The registry lists 0 analyses and does not report the statistical analysis plan, sample-size justification, missing-data approach, analysis populations, interim analyses, or multiplicity handling. It also does not provide detailed operational definitions for discontinuation or safety-event assessment. The outcome timeframe is listed as 18 months, but participant-level follow-up details are not reported in the registry record.
These limitations are specific to what can be interpreted from this terminated study record. They do not establish that the product is ineffective or unsafe; they mean that the recorded information does not provide an estimate of efficacy or safety for peanut-allergic participants.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: The Safety and Efficacy of a Peanut Immunotherapy Dissolving Film for Peanut Allergy — NCT01897077.
- Registry record last updated 2018-07-03; status last verified 2018-07.
- Results first posted 2018-07-03.
- 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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19. Record Summary
The registry describes a Phase 1 and Phase 2 non-randomized, unmasked, single-group study of a peanut dissolving film. It lists enrollment of 5, a start in 2012-11, primary completion on 2013-02-03, and terminated status. The study ended prematurely due to manufacturing issues, and no peanut-allergic subjects enrolled.
The primary outcome concerned treatment-related discontinuation. The record reports no such discontinuations among healthy volunteers. Safety results list 5 healthy volunteers at risk, with 0 serious adverse effects, 0 other adverse effects, and 0 deaths; the peanut-allergy group had 0 at risk. The registry lists 6 outcome measures and 0 analyses. It does not report an efficacy result for peanut-allergic participants, or a statistical analysis plan, sample-size justification, or methods for missing data and multiplicity.