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 Compassionate Use of Brontictuzumab for Adenoid Cystic Carcinoma (ACC) (NCT02662608), a na na study. The primary outcome is Number of Participants With Disease Progression.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02662608 |
| Phase | NA |
| Condition | Adenoid Cystic Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
A person with adenoid cystic carcinoma (ACC) and NOTCH1 mutations. The official title specifies two activating NOTCH1 mutations. The registry record reports one participant enrolled.
Intervention
Brontictuzumab, described as a single agent given intravenously at 1.5 mg/Kg every three weeks.
Comparator
None is listed. This is a single-group study, and the registry record does not identify a control group or a comparative treatment.
Primary question
How many participants had disease progression by the six-week assessment, using the stated RECIST v1.0 criteria? The progression result is not reported in the registry record.
The registry description says the treatment was intended as an investigational attempt to control tumors and prolong survival. That statement describes the study’s aim, not a demonstrated treatment effect. The clinical question is therefore descriptive: what disease-progression outcome was recorded for the participant during the stated timeframe? With no comparator, the study cannot estimate how outcomes would differ under another treatment or under no study treatment.
The record identifies ACC and describes the treatment in relation to NOTCH1 mutations. It does not report further participant characteristics, such as disease history or other eligibility details. Those details cannot be used to characterize how broadly the observation might apply. The single participant also means that the study’s result, if reported, would describe an individual observation rather than a group-level estimate supported by multiple participants.
3. Trial Design
This completed study is listed as a single-group investigation of brontictuzumab for adenoid cystic carcinoma. The registry identifies M.D. Anderson Cancer Center as the sponsor, lists the phase as NA, and records enrollment of 1 participant. The study start date is 2015-12-29, and the primary completion date is 2016-03-06. The overall status is COMPLETED.
The intervention is brontictuzumab, a drug. The arm description specifies 1.5 mg/Kg as a single agent, administered intravenously every three weeks. Allocation is listed as NA, and masking is listed as NONE. Because there is a single group and no comparator listed, this is not a randomized, like-for-like treatment comparison. The record does not describe a control arm, random assignment, or blinded assessment.
The primary outcome measure is the number of participants with disease progression, assessed over 6 weeks. Its description refers to tumor response for measurable disease using RECIST v1.0. Although the record gives the outcome definition, it does not report the observed count or an analysis of that outcome. It also does not report a protocol-level sample-size justification, an interim-analysis plan, or a detailed statistical analysis plan.
The study title and summary frame brontictuzumab as an investigational treatment for ACC associated with NOTCH1 mutations. The registry record’s design details support interpretation as a small, single-group clinical observation. They do not support a treatment-effect comparison or a conclusion about outcomes in a broader population.
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 | Brontictuzumab |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Brontictuzumab |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Disease Progression, measured over 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with disease progression during the stated 6 weeks. The registry describes progression using RECIST v1.0: a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions. These criteria define the event being counted; the endpoint is not a continuous measurement of tumor size or a time-to-event estimate.
General: A binary outcome records whether an event occurred for each participant. Summing those participant-level outcomes gives a count of participants with the event. Such an endpoint can make an assessment rule explicit, but the count alone does not show the timing or magnitude of each participant’s change. The registry record does not describe how measurements were obtained, who assessed them, or how a case with unevaluable measurements would be handled.
Progression is used as a measure of disease status in the study’s stated timeframe. Its interpretation depends on the specified criteria and the availability of assessments. The record lists the assessment timeframe and criteria, but does not report the progression count, a denominator for the endpoint analysis, or any supporting measurements. Consequently, the endpoint definition is available, while the observed endpoint result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed study with one participant enrolled and one primary outcome measure. The listed outcome concerns disease progression at 6 weeks, but the observed progression count is not reported in the registry record. No statistical analyses are listed. Thus, the record does not provide an outcome estimate, an uncertainty interval, or a hypothesis-test result for the primary endpoint.
The absence of a reported endpoint result is distinct from a result of no progression. It is not possible to infer whether progression occurred, whether the participant completed the assessment, or whether the outcome was unevaluable. Those details are not reported in the registry record. The study’s completion status does not itself establish that the primary outcome was observed or that an analysis was conducted.
Even if an individual outcome were available, a single-group design would remain descriptive. Without a comparator, the record cannot estimate a difference between brontictuzumab and another treatment or control condition. A single participant also offers very limited information about variation in outcomes across people. The study therefore cannot establish a group-level response pattern from its enrollment alone.
General: An estimate is more informative when accompanied by a measure of uncertainty, such as a confidence interval. A confidence interval expresses the range of values compatible with the data and the method used, under its assumptions. Neither an estimate nor a confidence interval for progression is reported. For a very small sample, uncertainty would be substantial even if a simple event count were available; the exact uncertainty cannot be stated without the actual outcome data and an analysis method.
The safety figures are also descriptive: the record reports one participant at risk for serious adverse events and zero participants affected, and one at risk for other adverse events and one affected. These figures do not identify the type, timing, severity, or relatedness of the other adverse event. Deaths are not reported in the registry record. The small enrollment and absence of a listed comparator limit any broader safety interpretation.
The study’s stated purpose was to attempt tumor control and prolong survival. That purpose should not be confused with evidence that either outcome was achieved. The posted information supports statements about the design, treatment schedule, endpoint definition, enrollment, and recorded adverse-event counts. It does not report the primary efficacy result or establish a treatment effect.
9. Statistical Methods Explained
The registry lists a count of participants with disease progression as the primary outcome. This is a binary-event endpoint at the participant level: each participant either meets the defined progression criteria during the stated assessment period or does not. A count aggregates those participant-level classifications. In this record, the outcome timeframe is 6 weeks and the criteria reference RECIST v1.0.
What can a count establish?
General: A count describes how many observed participants experienced a defined event. To interpret it fully, readers also need to know the number assessed and how missing or unevaluable observations were treated. A count does not, by itself, measure the size of a treatment effect. In a comparative study, a treatment contrast might be estimated from outcomes in separate groups. This study is listed as single-group, with no comparator, and the registry does not report an outcome count or analysis.
The record gives enrollment as 1, but does not state how many participants were included in the endpoint analysis. Enrollment and analysis population are not necessarily interchangeable: a participant may lack an evaluable assessment, for example. The registry does not report whether that occurred. No denominator for the primary endpoint is listed.
What would an uncertainty interval mean?
General: A confidence interval describes the precision of an estimate under a specified statistical method and its assumptions. A wide interval indicates that the data are compatible with a broad range of values; a narrow interval indicates greater precision under that method. An interval cannot supply information that the underlying data do not contain, and its interpretation depends on the design and analysis. The registry record does not report a confidence interval for the progression outcome, nor does it list an analysis.
With only one participant enrolled, a group-level estimate would have little capacity to characterize variation between participants. That is a design constraint, not evidence for or against a particular clinical outcome. No estimate of treatment effect, p-value, or other inferential result is reported in the registry record. The record also does not specify a statistical model for the primary endpoint.
How should planning calculations be read?
General: Sample-size calculations are planning exercises based on chosen assumptions. They are not observed results and are not evidence that a study had a particular probability of detecting an effect. The planning illustration associated with the record uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It frames the effect to detect as a stated fraction of the observed effect. For time-to-event outcomes, the illustration is defined in total events; for other outcomes, it is defined in participants per arm.
Those assumptions describe the planning illustration, not the design of this single-group study. The record does not report a protocol sample-size justification or a two-arm allocation. It also does not report a primary outcome analysis or observed effect on which readers could base a trial-specific interpretation of such planning assumptions. Any planning figure should therefore be understood as hypothetical, not as the study’s actual enrollment target, observed power, or evidence of efficacy.
Why does over-dispersion matter in general?
General: Over-dispersion arises when variability in count data exceeds what a chosen statistical model expects. If a count model assumes less variability than is present, standard errors can be too small and intervals or tests can be misleading. This endpoint, however, is defined as a count of participants with a binary event, rather than a recurrent-event rate or an unbounded count per person. The registry does not report a model or an analysis that would make an over-dispersion assessment relevant. The observed result is also not reported, so variability cannot be evaluated from the record.
These method concepts explain what would be needed to interpret a numerical result. They do not fill in the missing endpoint result, specify an unreported analysis plan, or change the central design limitation: a single-group observation in one enrolled participant cannot provide a controlled treatment comparison.
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 |
|---|---|---|---|---|---|---|
| Brontictuzumab | 1 | 1 | 100.0% | 2.5% to 100.0% | 20.7% to 100.0% | 15.8% to 98.7% |
R code and output
arm <- c("Brontictuzumab")
n <- c(1)
events <- c(1)
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 Brontictuzumab 1 1 100.0 2.5 100.0 20.7 100.0 15.8 98.711. Multiplicity, Missing Data and Analysis Populations
The registry record lists one outcome measure and zero analyses. It does not report results for the primary endpoint, and it does not describe an analysis of secondary outcomes. The record does not state whether outcomes were collected but not posted, or whether any additional analyses were planned or conducted.
Multiplicity refers, in general, to the increased chance of false-positive findings when multiple hypotheses or outcomes are tested. Statistical plans sometimes address this by specifying a primary outcome, limiting tests, or adjusting inference across tests. The registry record does not report a multiplicity strategy, any adjustment, or a detailed statistical analysis plan. Since no analysis is listed, there is no reported set of test results from which to assess multiplicity in practice.
Missing-data handling is also not reported in the registry record. The record does not say whether the participant had an evaluable assessment at 6 weeks, how an absent or incomplete assessment would be classified, or whether any imputation method was used. The analysis population is not reported. Although enrollment is listed as 1, that figure alone does not establish the denominator used for the primary outcome.
General: Missing outcome data can affect interpretation because the observed participants may not represent all enrolled participants. The impact depends on why data are missing and how the analysis handles them. With no reported endpoint result, analysis population, or missing-data method, those issues cannot be evaluated for this study. The registry record also does not report interim analyses or stopping rules; no such plan should be assumed.
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 |
|---|---|---|---|
| Brontictuzumab | 0 | 1 | 0.0% |
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 |
|---|---|
| Brontictuzumab | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Brontictuzumab |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 1 of 1 |
The registry lists one participant at risk for serious adverse events and zero affected by serious adverse events. It lists one participant at risk for other adverse events and one affected. These are participant counts, not event counts: the record does not establish how many individual events occurred for the participant counted as affected.
No serious adverse-event terms are listed. The record does not name the other adverse event, describe its severity or timing, or state whether it was considered related to brontictuzumab. Deaths are not reported in the registry record. A blank or unreported death figure should not be read as a confirmed count of zero.
General: Adverse-event summaries depend on definitions, observation time, and collection procedures. Counts from a very small group provide a limited description of what was recorded among those participants; they cannot reliably characterize the range or frequency of events in a wider population. The registry record does not give further safety detail or a comparator. These figures should therefore be read as limited descriptive counts, not as a comparative safety estimate.
13. What the Result Does, and Does Not, Mean
The record establishes that a completed, single-group study of brontictuzumab for ACC was registered with enrollment of 1 participant. It specifies the dose and schedule, a six-week progression endpoint, and RECIST v1.0 criteria for defining progression. It also records participant counts for serious and other adverse events.
The record does not report the primary endpoint result, an analysis, or an estimate of treatment effect. It therefore does not show whether the participant experienced disease progression by the assessment timeframe. The missing result cannot be interpreted as either progression or absence of progression. The completed status is a registry status, not a substitute for an outcome report.
Because there is no control group, the design cannot distinguish outcomes associated with treatment from outcomes that might have occurred under another condition. Because only one participant was enrolled, even a reported outcome would be an individual observation, not a stable estimate of outcomes across a patient population. No conclusion about benefit or lack of benefit follows from the available endpoint information.
General: A clinical study can contribute useful information even when it cannot answer a comparative question. A clearly defined endpoint and treatment schedule can document what was intended to be measured and administered. But efficacy and safety conclusions require outcome information, appropriate context, and attention to design limitations. For this record, the endpoint count, analysis, participant characteristics, and additional safety details are not reported in the registry record.
The registry summary states that the treatment was intended as an investigational attempt to control tumors and prolong survival. That is the study’s stated goal. It is not a finding that tumors were controlled or survival was prolonged. The posted information supports a description of the study, but not a claim about the treatment’s effect.
14. Why This Trial Matters Statistically
This study is statistically notable for the gap between a clearly described clinical question and the limited outcome information recorded. The registry identifies a population context, an investigational drug regimen, and a progression endpoint with a stated assessment timeframe and criteria. Yet it lists one participant, no comparator, zero analyses, and no reported primary endpoint result.
That combination makes the distinction between study purpose and evidence especially important. A registry description can show what investigators aimed to examine without showing what happened to participants. Likewise, a completed status does not imply that a numerical outcome or statistical analysis is available. Readers need the actual endpoint result and its analysis to assess what the study observed.
General: Study design determines which questions an analysis can answer. A controlled comparison can estimate a difference between groups under its assumptions; a single-group design cannot make that direct comparison. Small enrollment also limits the precision and generalizability of descriptive findings. These limits are not remedied by a planning calculation, which is conditional on assumptions and is not an observed result.
The record also illustrates why safety counts need context. The serious and other adverse-event figures are participant counts from a group of one, with no event descriptions for the other adverse event and no comparative group. They provide a narrow account of recorded events, not a broad safety profile.
For clinical researchers and biostatisticians, the central lesson is to separate endpoint definitions, registry status, analysis results, and planning assumptions. Each serves a different purpose. Here, the design and endpoint are described, but the outcome analysis needed to answer the primary question is not reported in the registry record.
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 principal limitation is the very small enrollment: the registry reports 1 participant. Findings from an individual observation cannot characterize the range of outcomes across people with ACC or support a reliable group-level estimate. The single-group design also has no listed comparator, so it cannot estimate a contrast with another treatment or control condition.
The primary outcome is defined, but its observed count and analysis are not reported in the registry record. The record does not report the endpoint analysis denominator, participant-level measurements, or whether the six-week assessment was completed. The absence of these details prevents interpretation of the primary clinical result.
Participant characteristics beyond the stated disease and NOTCH1 mutation context are not reported in the registry record. This limits assessment of how the participant’s circumstances compare with those of other people with ACC. The record also does not report adherence, treatment exposure beyond the listed regimen, or reasons for any treatment interruption.
Safety information is limited to participant counts. The other adverse event is not named, details about event timing and severity are not reported, and deaths are not reported in the registry record. No comparator is available to put these counts in a comparative context.
The statistical analysis plan, missing-data handling, analysis population, multiplicity approach, and protocol sample-size justification are not reported in the registry record. The phase is listed as NA, and allocation is listed as NA. These omissions mean that several aspects of the intended design and analysis cannot be assessed. The results and limitations should not be extended beyond what the registry record describes.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Compassionate Use of Brontictuzumab for Adenoid Cystic Carcinoma (ACC) — NCT02662608.
- Registry record last updated 2017-06-14; status last verified 2017-05.
- Results first posted 2017-05-16.
- 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.
19. Record Summary
The registry record describes a completed, single-group study of brontictuzumab for adenoid cystic carcinoma associated with NOTCH1 mutations. M.D. Anderson Cancer Center is listed as sponsor. Enrollment was 1, the phase is listed as NA, and masking is listed as NONE. The treatment regimen was 1.5 mg/Kg intravenously every three weeks.
The primary outcome was the number of participants with disease progression at 6 weeks, using RECIST v1.0 criteria. The registry lists one outcome measure and zero analyses, but does not report the progression result. Serious adverse events affected zero of one participant at risk; other adverse events affected one of one participant at risk. No serious-event terms are listed, and deaths are not reported in the registry record.
The single-group design, one-participant enrollment, and unreported primary outcome result limit clinical interpretation. The record describes the study question and regimen, but does not establish a treatment effect or provide a comparative safety assessment.