Boiled Peanut Oral Immunotherapy: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Boiled Peanut Oral Immunotherapy (NCT04090203), a na phase 1 study. The primary outcome is Increase in Tolerance to Peanut in Pediatric Patients With Peanut Hypersensitivity Reported as the Percentage of Participants Successfully Desensitized..

2. Clinical Question

The investigators’ stated hypothesis was that the proportion successfully desensitized with boiled peanut oral immunotherapy would exceed the theoretical placebo rate of 20%. That is a hypothesis against a theoretical reference, not a randomized comparison with a placebo group. The registry record does not report the observed proportion successfully desensitized, the confidence interval, or a test result, so it does not establish how the observed outcome compared with that reference.

The endpoint applies a specific threshold and challenge condition: participants had to tolerate a single dose of 300 mg or greater of peanut protein without dose-limiting symptoms at the exit food challenge. Participants unable to tolerate that dose, or who did not complete the exit challenge for any reason, were classified as not successfully desensitized in the endpoint definition. The listed timeframe is 18 weeks.

This question is about a measured challenge outcome in a small, single-group Phase 1 study. It is not a comparison of outcomes between randomized groups, and the registry record does not report an estimate of comparative benefit.

3. Trial Design

The registry describes a prospective Phase 1 clinical trial of boiled peanut oral immunotherapy as proof-of-concept research in children with peanut allergy. The study is marked completed. Its recorded start date is 2019-11-05, and its primary completion date is 2021-05-20. Enrollment is listed as 8.

The design is identified as single group, allocation is recorded as NA, and masking is listed as none. The intervention is Boiled Peanut Powder. No concurrent control group is described, so the study does not provide a like-for-like comparison between participants assigned to treatment and participants assigned to control. The theoretical placebo rate of 20% appears in the stated hypothesis, but it is not the outcome of a placebo group in this record.

The primary outcome is a binary classification: successfully desensitized or not successfully desensitized under the endpoint definition. The registry identifies one outcome measure and one analysis. The endpoint population includes all participants who enrolled, and participants who did not complete the exit food challenge are classified as not successfully desensitized.

The record does not report details of the treatment schedule, dose escalation, adherence, eligibility criteria beyond pediatric peanut hypersensitivity, or the timing of assessments beyond the 18-week endpoint timeframe. It also does not report a sample-size justification, a prespecified statistical analysis plan, or interim analyses. Those details should not be inferred from the study’s phase or proof-of-concept description.

The study sponsor is listed as Alton Melton. Because the design is single group and unmasked, the main endpoint is a descriptive proportion under the stated challenge definition. It cannot, by itself, distinguish an intervention effect from other explanations for an observed outcome.

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

Analysis population for the primary outcome, as defined in the registry: All participants who enrolled in the study.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

6. Primary Endpoint

The registry lists the primary outcome as Increase in Tolerance to Peanut in Pediatric Patients With Peanut Hypersensitivity Reported as the Percentage of Participants Successfully Desensitized., measured over 18 weeks.

The primary endpoint is the percentage of participants classified as successfully desensitized at the exit food challenge. Success required consuming a single dose of 300 mg or greater of peanut protein without dose-limiting symptoms. Participants who could not tolerate the dose, or who did not complete the exit food challenge for any reason, were classified as not successfully desensitized. The recorded timeframe is 18 weeks, and the analysis population is all participants who enrolled.

A binary endpoint turns each participant’s outcome into one of two categories according to a prespecified rule. Reporting a percentage is a direct way to describe how common the defined outcome was in the enrolled group. The confidence interval is intended to convey uncertainty around that percentage, rather than treating the observed proportion as an exact estimate of the underlying probability.

The threshold-based challenge outcome gives the endpoint a clear operational definition, but the result represents performance under that particular challenge and classification rule. It does not measure every aspect of peanut allergy or establish how outcomes compare with a concurrent control group. The registry record does not report the observed percentage or its confidence interval, so the endpoint’s numerical result cannot be interpreted from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary participant-level classification. Each participant is considered successfully desensitized if the stated challenge criterion is met; otherwise, the participant is classified as not successfully desensitized. A percentage for this kind of outcome is a proportion expressed on a percentage scale. Its interpretation depends on the definition of success, the population included, and how missing or incomplete outcome assessments are handled.

What does the confidence interval describe?

In general, a confidence interval communicates statistical uncertainty around an estimate. For a binary outcome, the estimate is the proportion meeting the endpoint definition, and the interval reflects uncertainty arising from observing outcomes in a finite group rather than the entire population of interest. A wider interval indicates less precision; a narrower interval indicates greater precision under the method used. Neither width nor location can be assessed for this trial because the registry record does not report the estimated percentage or interval bounds.

The endpoint description identifies the lower bound of a one-sided 95% confidence interval, calculated as the lower bound of a two-sided 90% exact confidence interval. An exact interval is a method for quantifying uncertainty in a binomial proportion. In general, exact methods derive bounds from the probability model for binary outcomes rather than relying solely on a large-sample normal approximation. The method is specified, but its numerical result is not reported in the registry record.

The wording of the record distinguishes the lower confidence bound from the percentage estimate: the percentage is presented with a corresponding lower bound. A lower bound can be useful when the scientific question focuses on whether the proportion might plausibly exceed a reference value. To make that assessment, however, the estimated proportion and its lower bound must be available. Neither is included in the registry record recorded for this endpoint.

Why does the endpoint rule matter?

General statistical principle: an endpoint is interpretable only when its operational definition is clear. Here, success requires tolerating a single dose of 300 mg or greater of peanut protein without dose-limiting symptoms at the exit food challenge. The rule also explicitly classifies participants who do not tolerate that dose or do not complete the exit challenge for any reason as not successfully desensitized. This avoids leaving those cases undefined within the stated endpoint, but it combines challenge failure and challenge noncompletion in the same outcome category. The registry record does not report the counts or reasons behind those classifications.

The analysis population is defined as all enrolled participants. In general, defining a population before analysis helps clarify which participants contribute to an estimate. The record does not report the endpoint numerator, the number of completed challenges, or participant-level outcome details, so the stated population and rule cannot be used to reconstruct the result.

What does the design allow statistically?

The study is identified as single group, with allocation NA and no masking. As a general matter, a single-group proportion describes outcomes in the participants observed under that study’s conditions. It does not provide a randomized estimate of the difference between an intervention and a concurrent control. The stated theoretical placebo rate of 20% is part of the investigators’ hypothesis, not a measured control-group outcome. The record does not report the statistical test, if any, used to assess that hypothesis.

The registry lists one outcome measure and one analysis. It does not report whether there were additional analyses outside those listings, whether adjustments for multiple testing were planned, or the statistical plan for the primary endpoint. A specified confidence-interval method is not enough to establish all other analysis decisions.

How should planning calculations be read?

The planning assumptions describe an illustration based on the observed effect, not the trial’s own design. They specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. These are assumptions for a planning calculation, not evidence that this study used two arms or was designed with those features. The registry record does not report a sample-size justification for the trial.

General statistical principle: planning calculations estimate how many participants might be needed under specified assumptions. They depend on the assumed effect, variability or event rate, significance level, allocation, and analysis method. Such calculations are prospective illustrations, not observed study results and not observed power. The specific planning values and any resulting participant requirements are not reported in the registry record details available for this interpretation. A normal approximation used in a planning illustration should not be confused with the exact confidence-interval method named for the primary endpoint.

Over-dispersion is a general issue when observed variability exceeds what a simple probability model assumes. It can make standard errors too small if the model fails to account for the extra variability, leading to intervals or tests that appear more precise than warranted. The registry record does not report a model for over-dispersion, and the binary endpoint description does not establish that such a model was used.

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.

R code and output

arm    <- c("Boiled Peanut Powder")
n      <- c(8)
events <- c(6)
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  Boiled Peanut Powder  8  6  75.0  34.9  96.8  40.9  92.9  40.0  92.5

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and one analysis. That does not, by itself, establish whether multiplicity adjustments were unnecessary or whether other outcomes or analyses were considered elsewhere. A multiplicity plan, including any adjustment to account for testing several hypotheses, is not reported in the registry record.

The primary analysis population is stated to include all participants who enrolled. The endpoint definition classifies participants who did not tolerate the specified dose or did not complete the exit food challenge for any reason as not successfully desensitized. This is a specific missing-outcome rule for the primary endpoint: noncompletion is incorporated into the not-successfully-desensitized category. The record does not report the number of participants with incomplete challenges or the reasons for noncompletion.

Other missing-data details are not reported in the registry record. These include whether any other study measurements were missing, whether data were imputed, whether sensitivity analyses were conducted, and whether the primary endpoint was analyzed exactly as registered. No participant-level outcome data are given to assess the influence of the classification rule.

In general, missing-data handling matters because conclusions can depend on whether missing outcomes are treated as failures, excluded, or addressed through a model. Here, the endpoint description specifies the classification for exit-challenge noncompletion, but it does not supply the counts needed to understand how much that rule affected the result. The observed endpoint estimate and confidence interval are not reported, so neither the estimate nor its sensitivity to missingness can be evaluated.

12. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: anaphylaxis - hives; redness and swelling of eyes; cough; difficulty breathing (12.5%), coughing and wheezing (12.5%), globus sensation; audible change in voice; dysphagia (12.5%).

The safety entry lists Boiled Peanut Powder with 8 participants at risk for serious adverse events and 2 affected. It records 0 deaths. The listed serious adverse-event terms are “Anaphylaxis - Hives; redness and swelling of eyes; cough; difficulty breathing,” “Coughing and wheezing,” and “Globus sensation; audible change in voice; dysphagia.” Each term is accompanied by 12.5%.

These percentages should be read as separate listed figures, not added together. The record does not state whether a participant could contribute to more than one term, nor does it provide event dates, duration, severity details beyond the serious-event listing, treatment, outcome, or the investigator’s assessment of relatedness. The percentages alone do not establish the number of events represented by each term.

For other adverse events, the record lists 8 participants at risk and 8 affected. The registry record does not provide further detail about those events in the registry record for this entry. Serious and other adverse-event counts are descriptive safety information; they do not provide a comparison with a control group and do not establish causation.

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

The record documents a prospective, completed Phase 1, single-group study of boiled peanut oral immunotherapy in pediatric patients with peanut hypersensitivity. It specifies a primary endpoint based on whether enrolled participants met a particular exit food-challenge threshold. It also records serious and other adverse-event counts. These are useful details about what was studied and how the primary outcome was defined.

The primary endpoint result itself is not reported in the registry record. Without the observed percentage and confidence interval, the degree of desensitization observed in the study and the precision of that estimate cannot be assessed. The stated hypothesis refers to a theoretical placebo rate of 20%, but there is no concurrent placebo group described. The record therefore does not provide a randomized treatment comparison, and the missing endpoint result prevents assessment even against the stated theoretical reference.

In general, a measured challenge outcome is specific to the challenge conditions and success rule used. It should not automatically be interpreted as a measure of every aspect of allergy or as evidence about outcomes beyond the recorded timeframe. The endpoint definition includes participants who did not complete the exit food challenge as not successfully desensitized, but the number and reasons for such cases are not reported.

The safety entries describe affected participants and listed adverse-event terms, but the limited detail does not establish timing, relatedness, or comparative risk. The study’s single-group design also means that the recorded counts cannot be contrasted with a concurrent control group in this record.

Accordingly, this registry entry permits a description of design, endpoint criteria, and selected safety counts. It does not report enough primary outcome information to determine the observed endpoint value, its precision, or whether the investigators’ stated hypothesis was supported.

14. Why This Trial Matters Statistically

This study illustrates why a clear endpoint definition and a reported result are both necessary for statistical interpretation. The registry specifies what counted as successful desensitization: tolerating a single dose of 300 mg or greater of peanut protein without dose-limiting symptoms at the exit food challenge. It also specifies how challenge noncompletion is classified. These details establish what the outcome means, but they do not reveal how often it occurred.

The confidence-interval method is also named: a one-sided 95% lower bound derived from a two-sided 90% exact confidence interval. That information makes the intended uncertainty method more transparent. Yet a method without its estimate and bounds cannot show the degree of precision or support a quantitative conclusion.

The single-group design makes the distinction between a theoretical reference and a concurrent control especially important. The stated hypothesis concerns a theoretical placebo rate of 20%, but the record does not describe a placebo group. A reference rate and a randomized comparison answer different questions and rely on different evidence.

Finally, the record’s safety counts demonstrate why event percentages require context. Listed terms, participant counts, and missing details about event overlap, timing, and relatedness all affect interpretation. Statistical reporting is strongest when endpoint results, uncertainty, analysis populations, missingness, and safety definitions are reported together. In this record, several design and definition details are available, while the primary endpoint estimate and important analysis details are not reported.

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 central limitation for interpreting the primary endpoint is that its observed percentage and confidence interval are not reported in the registry record. Without them, the outcome’s magnitude and precision cannot be assessed, and the stated hypothesis about exceeding the theoretical placebo rate of 20% cannot be evaluated from the reported endpoint information.

The study is listed as single group, with allocation NA and no masking. No concurrent control group is described. Accordingly, the record does not support a randomized comparison or a causal estimate relative to a control group. The theoretical reference in the hypothesis should not be treated as though it were a measured study arm.

The endpoint definition combines inability to tolerate the challenge threshold and failure to complete the exit challenge into the not-successfully-desensitized category. The number of participants in each circumstance and reasons for noncompletion are not reported. Other missing-data procedures, sensitivity analyses, and participant-level outcomes are also not reported in the registry record.

The registry does not report a sample-size justification, a complete statistical analysis plan, interim analyses, or multiplicity procedures. The planning assumptions describe an illustration rather than the trial’s own design, and specific planning results are not reported in the registry record details used.

Safety information is limited to participant counts, listed terms, and percentages. Details about event timing, duration, relatedness, overlap, and clinical course are not reported. These limitations restrict interpretation of both the primary outcome and the safety entries; they should not be filled in by assumptions about the protocol or study conduct.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Boiled Peanut Oral Immunotherapy — NCT04090203.
• Registry record last updated 2024-09-19; status last verified 2024-09.
• Results first posted 2024-09-19.
• 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.

19. Record Summary

NCT04090203 is a completed, prospective Phase 1, single-group study titled “Boiled Peanut Oral Immunotherapy.” It enrolled 8 pediatric patients with peanut hypersensitivity and evaluated Boiled Peanut Powder. Allocation is listed as NA, masking as none, and the sponsor as Alton Melton. The recorded start date is 2019-11-05, and the primary completion date is 2021-05-20.

The primary endpoint was the percentage of enrolled participants able to consume a single dose of 300 mg or greater of peanut protein without dose-limiting symptoms at an exit food challenge, with a timeframe of 18 weeks. Participants unable to tolerate that dose or not completing the challenge were classified as not successfully desensitized. The endpoint estimate and confidence interval are not reported in the registry record.

Safety entries list 2 participants affected by serious adverse events among 8 at risk, 0 deaths, and 8 participants affected by other adverse events among 8 at risk. The record lists three serious-event terms, each with 12.5%.