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 Peanut Oral Immunotherapy in Children With Peanut Allergy (Peanut Flour) (NCT02203799), a na phase 1 study. The primary outcome is Determine the Percentage of Patients Tolerating the 12-month DBPCFC.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02203799 |
| Phase | Phase 1 |
| Condition | Peanut Allergic Subjects |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Baylor College of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled peanut-allergic subjects. The registry lists an enrollment of 15. It reports that three subjects withdrew before the maintenance phase, and that 12 subjects were analyzed for the primary objective. The registry does not report further eligibility details or demographic characteristics in the information available for this analysis.
Intervention
The intervention was peanut oral immunotherapy (POIT) using peanut flour. The registry describes one daily dose, taken from a small cup, with the amount increased during a build-up phase. Subjects returned every 2 weeks during build-up until they achieved a maintenance dose of 3900 mg.
Comparator
There was no parallel control group: the study is listed as single-group, with allocation recorded as NA and masking as none. The double blind placebo controlled food challenge was used as an assessment, not as evidence that the treatment groups were randomized or comparable.
Primary question
The primary objective was to determine the percentage of subjects tolerating a cumulative dose of 26255 mg of peanut flour without clinical symptoms during the challenge at month 12 after the first dose. The registry does not report the number tolerating that dose or the resulting percentage, so the primary result cannot be described from the registry record.
3. Trial Design
This was a phase 1, single-group study of peanut oral immunotherapy in peanut-allergic subjects. The registry records the study as completed, with enrollment of 15. The listed intervention, peanut oral immunotherapy (POIT), used peanut flour. According to the intervention description, a dose was taken once per day in a small cup, with the amount increased during a build-up phase.
The build-up phase is described as month 0-12. Subjects returned every 2 weeks during this period until achieving a maintenance dose of 3900 mg. The primary challenge was performed on achieving the maintenance dose, at month 12 after the first dose of peanut flour. The objective concerned tolerating a cumulative dose of 26255 mg without clinical symptoms during the double blind placebo controlled food challenge.
The record reports that three subjects withdrew before the maintenance phase and that only 12 subjects were analyzed for the primary objective. It does not report why those subjects withdrew, whether other participants had missing assessments, or how missing outcome information was handled.
There was no randomized comparator: allocation is listed as NA, masking as none, and the model as single-group. The challenge’s placebo-controlled format does not create a randomized comparison between treatment arms. Consequently, the primary objective is an assessment within the treated study group, not a between-group estimate of treatment effect.
The registry lists a secondary outcome of sustained unresponsiveness after 1 month of treatment discontinuation. A result for that outcome is not reported in the registry record. Detailed eligibility criteria, the protocol’s statistical plan, and any sample-size justification are also 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.
Before assignment: Participants were brought in for a screening visit to assess for eligibility in the study. 28 subjects were seen for screening visit and 13 subjects were ineligible due to failure to complete food challenge (1), FEV1\<90% predicted on spirometry (4), Peanut IgE \< 7 kU/L (1), and were not interested due to time commitment (5), unable to tolerate initial dose (2).
Overall Study
| Stage | Peanut Oral Immunotherapy (POIT) |
|---|---|
| STARTED | 15 |
| COMPLETED | 12 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Peanut Oral Immunotherapy (POIT) |
|---|---|
| Withdrawal by Subject | 3 |
Analysis population for the primary outcome, as defined in the registry: Three (3) subjects withdrew from the trial prior to maintenance phase. Only 12 subjects were analyzed for this objective.
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 | Peanut Oral Immunotherapy (POIT) |
|---|---|---|
| Participants analysed | 15 | |
| Age, Continuous | years | 8.74 (SD 2.42) |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 8 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 13 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 12 |
| Race (NIH/OMB): More than one race | Participants | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Number of Participants that Completed the Protocol: Withdrawal | Participants | 3 |
| Number of Participants that Completed the Protocol: Completed | Participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Determine the Percentage of Patients Tolerating the 12-month DBPCFC, measured over Month 12 after first dose of peanut flour.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint asks how many participants tolerated a cumulative dose of 26255 mg of peanut flour without clinical symptoms during the 12-month double blind placebo controlled food challenge. The registry expresses the objective as a percentage of patients tolerating the challenge. It identifies the timeframe as month 12 after the first dose of peanut flour and states that 12 subjects were analyzed for this objective.
This endpoint uses a defined challenge and a specified symptom criterion to describe the observed response at the assessment. In general, a binary endpoint classifies each participant according to whether the defined event or condition occurred. A count and a percentage can then communicate how often that condition was observed in the analysis population. The percentage result itself, and the number tolerating the challenge, are not reported in the registry record.
The endpoint should not be read as a randomized comparison. The trial is single-group and has no listed control arm. Nor does a challenge result at the specified assessment, by itself, describe outcomes beyond the assessment or establish what would have happened without the intervention. The named secondary outcome concerns sustained unresponsiveness after 1 month of treatment discontinuation, but its result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clear primary objective: assess tolerance of a cumulative peanut-flour dose of 26255 mg without clinical symptoms during the challenge at month 12 after the first dose. However, it does not report the primary outcome count or percentage. Therefore, the record does not permit a description of how many analyzed subjects met the endpoint, or how common that response was in the analysis population.
The reported analysis population is 12 subjects. The record also says three subjects withdrew before the maintenance phase. These statements describe who entered the primary analysis and an important point of attrition, but do not reveal the outcomes of the participants who withdrew. Reasons for withdrawal and the method used to handle missing outcome data are not reported in the registry record. The absent result should not be interpreted as evidence that no subject tolerated the challenge, nor as evidence that all subjects did.
Because this is a single-group study without a randomized comparator, its primary objective is descriptive rather than a direct estimate of a difference between treatment and control. A challenge conducted after the build-up phase can characterize outcomes among those assessed under the study conditions. It cannot, on its own, separate an intervention-related change from other explanations or establish a comparative treatment effect.
No inferential analysis is listed: the registry records zero analyses. A confidence interval, p-value, or other measure of uncertainty for the primary endpoint is not reported in the registry record. In general, a confidence interval indicates the precision of an estimated quantity under the assumptions of the analysis; without a reported estimate and interval, the precision of the primary result cannot be assessed.
The registry names a secondary outcome, sustained unresponsiveness after 1 month of treatment discontinuation, but does not report its result. It also lists safety counts: zero subjects affected by serious adverse events among 15 at risk, 15 affected by other adverse events among 15 at risk, and zero deaths. Those counts are descriptive and do not replace the missing primary outcome. The serious-event terms are not listed.
Thus, the registry record supports a description of the design, challenge endpoint, analysis population, and recorded safety counts. It does not support a conclusion about the observed primary response, statistical precision, comparative effectiveness, or the secondary outcome.
9. Statistical Methods Explained
The registry describes a binary primary outcome in terms of participants tolerating or not tolerating the specified challenge dose without clinical symptoms. The record lists the primary measure as a count of participants, while the objective is worded as a percentage. These are related summaries: a count describes the number meeting a condition, and a percentage expresses that count relative to the analysis population. The count and percentage results are not reported in the registry record.
What does a single-group design estimate?
A single-group design can describe outcomes observed among participants who receive the intervention and undergo assessment. It does not provide a concurrent randomized control group for estimating a contrast between intervention and control. Here, the registry records a single-group model, allocation as NA, and masking as none. The challenge’s double blind placebo controlled format is part of the outcome assessment; it does not turn the study into a randomized, parallel-arm comparison.
In general, a comparison intended to estimate a treatment effect needs a suitable comparator and a design that supports interpreting differences between groups. Without those features, changes or outcomes observed within one group cannot be attributed to the intervention alone. This design limitation matters when interpreting the primary endpoint, even if its outcome count were available.
How should a confidence interval be read?
In general, a confidence interval describes the uncertainty around an estimated quantity under the analysis assumptions. A narrower interval indicates greater precision, while a wider interval indicates less precision. For a participant-level binary endpoint, an interval could communicate uncertainty around the estimated proportion meeting the endpoint. The registry record does not report a primary estimate or a confidence interval, so neither the observed response nor its statistical precision can be evaluated from this record.
A p-value, if reported, would address compatibility between data and a specified statistical null hypothesis under stated assumptions; it would not measure the probability that a treatment works. No p-value or inferential analysis is listed in the registry record. The absence of a reported analysis is not itself evidence for or against the endpoint occurring.
What can the analysis population tell us?
The primary objective was analyzed in 12 subjects. The registry also reports three withdrawals before the maintenance phase. The analysis count therefore identifies the group used for the stated objective, but does not explain how participants were selected for analysis or what happened to their outcome data after withdrawal. Reasons for withdrawal, missing-data methods, and an intention-to-treat analysis are not reported in the registry record.
In general, missing outcome data can affect interpretation when the participants with observed outcomes differ systematically from those without them. The direction and size of any such effect depend on the missingness pattern and the assumptions used to address it. Since those details are not reported, the effect of withdrawal or any other missing data cannot be assessed.
What do the planning assumptions mean?
The record describes sample-size assumptions as a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not evidence that the trial itself used a two-arm design: the registered study is single-group. The sample-size justification for the trial is not reported in the registry record.
In general, planning calculations ask how much information a future study might need to detect a prespecified effect under chosen assumptions. Such calculations are not observed power and do not establish that a completed study had sufficient power. The record does not list the resulting planning values in the material described for this analysis; no planning count can therefore be interpreted as an observed trial result.
Why does the event type matter?
The primary endpoint is a participant count for a binary condition, not a time-to-event measure or a repeated measurement summary. In general, binary outcomes focus on whether a participant meets a defined condition, whereas time-to-event methods account for when an event occurs and may incorporate censoring. The registry does not report a time-to-event analysis for the primary endpoint.
Over-dispersion is a concern in some count models when variability exceeds what the assumed model allows. In general, it can affect standard errors and uncertainty estimates if a model fails to account for extra variability. The primary measure is described as a count of participants meeting a binary endpoint, and no model or inferential analysis is reported. Over-dispersion modeling is therefore not something that can be evaluated from the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary objective and one secondary outcome: sustained unresponsiveness after 1 month of treatment discontinuation. It records two outcome measures and zero analyses. A statistical procedure for controlling multiplicity across outcomes, or a hierarchy specifying how outcomes should be tested, is not reported in the registry record. Since no inferential analyses are listed, there are no reported tests whose multiplicity adjustment can be evaluated.
The primary endpoint analysis population is stated as 12 subjects. The record reports that three subjects withdrew before the maintenance phase, but it does not report why they withdrew, whether any other outcome data were missing, or how missing data were handled. It does not describe imputation, sensitivity analyses, or a prespecified missing-data assumption. These details are not reported in the registry record.
In general, an analysis population defines which participants contribute to a particular estimate, while a missing-data strategy describes how unavailable measurements are addressed. Both affect how an observed result should be interpreted. Here, the stated analysis population is known, but the result and missing-data methods are not. The registry does not state whether the primary analysis followed an intention-to-treat principle or another analysis rule.
The record also does not report an interim analysis plan, a stopping rule, or a statistical analysis plan. Their absence from the registry record should not be taken to mean that a particular method was or was not used outside the information recorded.
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 |
|---|---|---|---|
| Frequency of Symptom Occurrence During Dosing | 0 | 15 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Frequency of Symptom Occurrence During Dosing | 0 of 15 |
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 |
|---|---|
| Frequency of Symptom Occurrence During Dosing | 15 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Frequency of Symptom Occurrence During Dosing |
|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | 13 of 15 |
| Skin Itchiness | Skin and subcutaneous tissue disorders | 13 of 15 |
| Hives | Skin and subcutaneous tissue disorders | 12 of 15 |
| Throat Symptoms | Respiratory, thoracic and mediastinal disorders | 10 of 15 |
| Gastrointestinal, Subjective Complaints | Gastrointestinal disorders | 9 of 15 |
| Sneezing/Itching | Respiratory, thoracic and mediastinal disorders | 8 of 15 |
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | 8 of 15 |
| Stuffy Nose | Respiratory, thoracic and mediastinal disorders | 7 of 15 |
| Gastrointestinal, Objective Complaints | Gastrointestinal disorders | 6 of 15 |
| Wheezing | Respiratory, thoracic and mediastinal disorders | 4 of 15 |
The safety summary lists the frequency of symptom occurrence during dosing. It records 15 subjects at risk for serious adverse events and zero affected; for other adverse events, it records 15 at risk and 15 affected. It records zero deaths. These are counts as listed in the registry, not estimates of comparative risk, because there is no control group.
The serious adverse event count describes the number recorded as affected in that category. The record lists no serious adverse event terms. The other-adverse-event count should be kept separate from the serious adverse event count; the registry summary does not provide the specific other-event terms in the registry record used for this analysis. The counts do not indicate how often events occurred per person or describe their severity, timing, duration, or relationship to treatment.
In general, a count of affected participants is a descriptive safety summary, not a complete characterization of safety. Interpretation also depends on event definitions, collection methods, follow-up, and details of each event. Those details are not reported in the registry record. Zero recorded serious adverse events does not establish that serious events are impossible, and the listed counts cannot be compared with an untreated group in this single-group study.
12. What the Result Does, and Does Not, Mean
The study record describes a completed phase 1 investigation of peanut oral immunotherapy using peanut flour in peanut-allergic subjects. Its primary objective was to assess whether subjects tolerated a specified cumulative challenge dose without clinical symptoms at the stated assessment. The record identifies the analysis population and dosing schedule, but it does not report the primary endpoint count or percentage. The central outcome therefore cannot be summarized as an observed response from this registry information.
Even if the primary result were available, its interpretation would remain tied to the study design and population assessed. This was a single-group study, with no randomized comparator. A response observed after the build-up phase would describe the participants assessed under these study conditions, not a randomized difference attributable to the intervention. The registry also reports withdrawals before the maintenance phase, while reasons and missing-data handling are not reported.
The named secondary outcome concerns sustained unresponsiveness after 1 month of treatment discontinuation. Its result is not reported in the registry record. The safety summary records zero subjects affected by serious adverse events, 15 affected by other adverse events, and zero deaths, with 15 at risk in each category. Those counts are descriptive and do not provide event details or a controlled comparison.
In general, statistical uncertainty and clinical interpretation are different questions. An estimate describes the observed data, while an interval or other analysis can show how precisely the estimate is known under specified assumptions. Neither the primary estimate nor its uncertainty is reported. Consequently, the record does not establish the size or precision of the primary response, a comparative treatment effect, or the result of the secondary outcome. These limits reflect what is reported, not evidence that an unreported outcome did or did not occur.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its primary question is phrased as a percentage, while the record identifies the primary measure as a count of participants. Understanding the denominator matters for interpreting such a percentage. The registry states that 12 subjects were analyzed for the primary objective, but does not report the count meeting the endpoint or the resulting percentage. A clearly specified endpoint and analysis population do not replace the result itself.
The design also shows why outcome assessment and treatment comparison are distinct. A double blind placebo controlled food challenge is described as the primary assessment, but the registered study has a single-group model, no allocation to parallel arms, and no masking. The challenge procedure does not supply a randomized comparator for estimating a between-group effect.
Withdrawal is another important statistical feature. The record reports three withdrawals before maintenance and an analysis population of 12, but provides no reasons or missing-data strategy. In general, missingness can influence both the estimate and its interpretation, depending on who is missing and why. Without those details, the extent of any effect cannot be judged.
Finally, the record separates a stated clinical objective from the statistical evidence needed to evaluate it. No primary result, confidence interval, p-value, or inferential analysis is reported. The safety counts are available as descriptive information but do not resolve the missing efficacy endpoint or provide a comparison group. This combination underscores the value of complete outcome reporting alongside clear design and analysis descriptions.
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 most direct limitation for interpreting the primary objective is that its outcome count and percentage are not reported in the registry record. Without them, there is no observed primary estimate to interpret, and no reported confidence interval or other measure of precision. The registry lists zero analyses, so inferential results are not available in the record.
The trial is single-group, with allocation listed as NA and masking as none. It therefore does not provide a randomized comparison. The primary assessment’s placebo-controlled challenge format does not create a treatment control group. Outcomes observed in this design cannot be interpreted as a randomized treatment-versus-control contrast.
The analysis population is stated as 12 subjects, and three subjects are reported to have withdrawn before the maintenance phase. The reasons for withdrawal, missing-data handling, and any sensitivity analysis are not reported in the registry record. The effect of attrition on the endpoint cannot be assessed from the available details.
The registry names a secondary outcome, sustained unresponsiveness after 1 month of treatment discontinuation, but does not report its result. It also does not report a statistical plan, a sample-size justification, interim analyses, or multiplicity procedures. The safety summary gives counts, but serious-event terms are not listed and further event details are not reported in the registry record. These omissions limit assessment of outcome precision, robustness, and completeness.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Peanut Oral Immunotherapy in Children With Peanut Allergy (Peanut Flour) — NCT02203799.
- Registry record last updated 2024-01-19; status last verified 2023-04.
- Results first posted 2024-01-19.
- 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 records NCT02203799 as a completed phase 1, single-group study of peanut oral immunotherapy using peanut flour in peanut-allergic subjects. Enrollment is listed as 15. The primary objective concerned tolerance of a cumulative dose of 26255 mg without clinical symptoms during a challenge at month 12 after the first dose. Three subjects withdrew before maintenance, and 12 were analyzed for the primary objective.
The primary outcome count and percentage are not reported in the registry record. A secondary outcome is listed, but its result is also not reported. The safety summary records zero serious adverse events, 15 subjects affected by other adverse events, and zero deaths. The record lists zero analyses and does not report the statistical plan or missing-data methods.