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 Nelfinavir in Recurrent Adenoid Cystic Cancer of the Head and Neck (NCT01065844), a na phase 2 study. The primary outcome is Tumor Progression.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01065844 |
| Phase | Phase 2 |
| Condition | Carcinoma, Adenoid Cystic, Head and Neck Neoplasms |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Iowa |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent symptomatic adenoid cystic cancers of the head and neck were the intended population. The record reports enrollment of 15. The primary outcome population excluded people whose adverse events required interruption of the intervention and removal from the study; the number assessed for the outcome is not reported in the registry record.
Intervention
The intervention was nelfinavir, described as 1250 mg twice daily Monday-Sunday. The brief summary says the study evaluated the drug as an oncologic agent and describes clinical follow-up and assessment of quality of life.
Comparator
This was a single-group study with no masking and no comparator arm. The registry lists allocation as NA. Consequently, the study does not provide a concurrent control group for separating changes during treatment from changes that might occur over time for other reasons.
Primary question
The primary question was whether tumor progression, assessed using RECIST version v1.1 categories, was observed during follow-up. The outcome description lists complete response, partial response, stable disease, and progressive disease, with assessment every 1 to 3 months. The registry does not report how many participants fell into each category or an analysis of the outcome.
3. Trial Design
This was a phase II, single-group trial of nelfinavir in patients with recurrent symptomatic adenoid cystic cancers of the head and neck. The University of Iowa is listed as sponsor. The registry reports a start date of 2009-10, primary completion in 2015-05, and overall status as completed. Enrollment was 15. These record details describe the study's registration and conduct; they do not establish that outcome results were reported.
The intervention was nelfinavir at 1250 mg twice daily Monday-Sunday. The record states that participants would follow up with their medical oncologist as clinically indicated while taking the medication. Tumor progression was to be assessed every 1 to 3 months. The brief summary also describes use of the EORTC QLQ-C30 questionnaire for quality-of-life assessment. The record does not report quality-of-life results or a separate quality-of-life outcome measure.
Masking was listed as none, and allocation was listed as NA. The study therefore was not a randomized comparison and had no like-for-like control arm. In a single-group design, observed outcomes describe participants treated in the study, but cannot by themselves establish what would have occurred in a comparable untreated or differently treated group. The registry does not report a statistical analysis plan, sample-size justification, interim analyses, or the number of participants who completed treatment or outcome assessment.
The brief summary says the study remained unfunded and that participants would need to arrange self-travel, with no reimbursement for time, travel, or physician costs. These details describe participation conditions in the summary, but the record does not quantify their effect on enrollment, retention, or the observed outcomes.
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 | Nelfinavir |
|---|---|
| STARTED | 15 |
| COMPLETED | 10 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | Nelfinavir |
|---|---|
| Adverse Event | 5 |
Analysis population for the primary outcome, as defined in the registry: Those with adverse events necessitating interruption of intervention and removal from study were not assessed for outcome measures
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 | Nelfinavir |
|---|---|---|
| Participants analysed | 15 | |
| Age, Customized: <=18 years | Participants | 0 |
| Age, Customized: Between 18 and 65 years | Participants | 12 |
| Age, Customized: >=65 years | Participants | 3 |
| Sex/Gender, Customized: Female | Participants | 9 |
| Sex/Gender, Customized: Male | Participants | 6 |
| Region of Enrollment: United States | participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Tumor Progression, measured over Every 1 to 3 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is titled Tumor Progression and is measured in participants. Its description defines assessment using RECIST version v1.1 and lists complete response, partial response, stable disease, and progressive disease. Assessment was scheduled every 1 to 3 months. These categories describe the status of measurable disease over follow-up; a participant count by category would indicate how many people were recorded in each specified state.
Such an endpoint is used to characterize tumor status under a defined assessment framework. In general, repeated assessments can help distinguish a documented response or stable disease from progressive disease during the observation period. Interpretation depends on the assessment rules, timing, and availability of measurements. The registry does not report the actual counts, the number assessed, or a time-specific result.
The outcome metadata classifies the measure as a binary count of participants, while the outcome description lists four RECIST categories. The record does not explain how those categories were grouped for any binary analysis. No mapping or outcome calculation should therefore be inferred. The listed population also excludes people removed from the study after adverse events requiring interruption of treatment, which is important when interpreting who could contribute to the endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed trial that enrolled 15 participants, but it does not report the primary outcome results or any analyses. There is no reported count of participants with tumor progression, complete response, partial response, or stable disease, and no reported number assessed for this outcome. Completion status and enrollment alone do not supply evidence about the outcome. The record therefore does not support a numerical estimate of tumor status in the study.
The trial used one treatment group and had no masking. Results from such a design, if reported, would describe outcomes among participants who received the intervention under the study conditions. Without a concurrent comparator, a change observed during follow-up cannot be attributed to the intervention alone. Disease course, patient selection, follow-up, and other factors could also be relevant. This design limitation does not make the study's observations meaningless, but it limits what a causal comparison could establish.
The primary endpoint's description includes four RECIST categories, but the record's outcome metadata calls it a binary participant count. Because the record does not explain how those categories would be converted into a binary outcome, it is not possible to identify a particular event definition or calculate a proportion from the available results. The outcome population also excludes participants removed after adverse events that necessitated treatment interruption. The number excluded and the number otherwise missing are not reported.
The adverse-event information reports 0 serious adverse events affecting participants among 15 at risk in the Nelfinavir group. It reports other adverse events affecting 5 among 15 at risk. These are descriptive registry counts, not comparisons with another treatment group. Deaths are not reported in the registry record. The record provides no serious adverse event terms, so the types of serious events cannot be described from the listed information.
In short, the record documents the question, intervention, design, and some safety counts, but not the primary outcome estimate or its precision. No treatment-effect estimate, confidence interval, or inferential test is reported. Any conclusion about whether tumor outcomes differed from a comparator or were caused by treatment would go beyond the recorded results.
9. Statistical Methods Explained
The registry describes a phase II single-group study with no masking. It does not report the statistical analysis plan, a sample-size justification, interim analyses, or any statistical analyses. The following concepts are general explanations of how the design and outcome would ordinarily be interpreted; they are not claims that these methods were used in this trial.
What does a single-group design estimate?
In general, a single-group study can describe the outcomes observed among participants who receive an intervention. For a participant-count endpoint, an analysis might report the number meeting a prespecified outcome definition and the number assessed. The registry does not give those outcome counts or the assessed denominator for tumor progression. It is therefore not possible to reconstruct an outcome estimate from the enrollment figure alone.
A single-group study lacks a concurrent comparison group. A change in disease status during follow-up can be described, but the design does not show what would have happened to comparable participants under another treatment or no treatment. Random allocation in a comparative study can help balance measured and unmeasured factors on average; that feature is not present. No causal effect relative to a control can be calculated from the posted record.
What does a confidence interval communicate?
General statistical explanation: a confidence interval expresses the uncertainty around an estimated quantity under a specified statistical model. A narrow interval indicates greater precision than a wide interval, while both the estimate and interval depend on the data and assumptions. An interval is not a guarantee that the true value lies within its limits, and it does not correct design limitations such as lack of a comparator or selective availability of outcome measurements. No confidence interval for tumor progression is reported in the registry record.
For participant counts, a proportion and its interval require a clearly defined event, a count of participants with that event, and an appropriate denominator. Here, the listed outcome description names four RECIST categories, while the outcome metadata classifies the measure as a binary count. The registry does not explain the binary event definition or report the category counts. Neither a proportion nor its precision can be inferred without those details.
How should the sample-size illustrations be read?
The planning assumptions describe illustrations based on a stated fraction of an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are planning illustrations, not the trial's own design and not observed power. The actual trial is single-group, so the equal-allocation, two-arm assumption does not describe its allocation. Moreover, the primary outcome estimate needed to identify an observed effect is not reported in the registry record. The illustrations should not be taken as evidence that the trial had a particular probability of detecting an effect.
In general, a prospective sample-size calculation asks how many participants a planned study would need under assumptions about the effect, variability, significance threshold, and design. Observed power calculated after a study is completed does not replace the estimate and its uncertainty; it largely re-expresses the observed result. Planning scenarios based on assumed fractions of an observed effect are conditional on those assumptions and do not establish that the effect is real or clinically important.
Why does the endpoint definition matter?
RECIST version v1.1 provides a framework for classifying tumor response and progression. The record lists complete response, partial response, stable disease, and progressive disease and states that assessment occurred every 1 to 3 months. A statistical analysis still needs to specify the participant-level outcome used, such as which category or categories count as an event and the period over which that event is assessed. The registry does not report those analysis details. The four listed categories should not be collapsed into a binary outcome without a stated rule.
Repeated assessments can produce different states across follow-up. A single count at a time point and a time-to-event analysis answer different questions: the former describes status at that time, while the latter concerns the timing of an event and usually needs event times and handling of participants without an observed event. The record reports no event times, censoring information, or time-to-event analysis. It is not possible to infer that such an analysis was conducted.
How do outcome availability and adverse events affect interpretation?
The primary outcome population excludes participants whose adverse events required interruption of intervention and removal from the study. General statistical explanation: if missing outcome measurements are related to health status, treatment experience, or the probability of progression, the observed participants may not represent everyone enrolled. This is sometimes called informative missingness. The direction and size of any resulting bias cannot be determined without the number and outcomes of excluded or otherwise unassessed participants.
The record does not report a method for handling missing data, an analysis population, or sensitivity analyses. It also does not report a statistical test or model for the primary outcome. These omissions prevent assessment of whether the planned analysis accounted for follow-up gaps, protocol departures, or uncertainty in the endpoint classification. They do not justify assuming a particular method was used.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure, Tumor Progression, and reports no analyses. The brief summary also describes quality-of-life assessment using the EORTC QLQ-C30 questionnaire, but quality-of-life results and a separate registered outcome measure are not reported in the registry record. No hierarchy among outcomes, multiplicity adjustment, or prespecified set of statistical tests is reported. With no analysis results available, the effect of multiple testing cannot be assessed.
The primary outcome population excludes participants whose adverse events required interruption of the intervention and removal from the study. The number excluded for this reason is not reported in the registry record. The record also does not state how many participants had missing or unevaluable measurements for other reasons, how missing outcome data were handled, or whether sensitivity analyses were conducted.
The record does not define an intention-to-treat, treated, or evaluable analysis population. Enrollment of 15 is not necessarily the same as the number assessed for the primary endpoint, particularly given the stated exclusion from outcome assessment. The number assessed and the denominator for any outcome estimate are not reported. These gaps limit interpretation of both the endpoint and its representativeness.
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 |
|---|---|---|---|
| Nelfinavir | 0 | 15 | 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 |
|---|---|
| Nelfinavir | 5 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Nelfinavir |
|---|---|---|
| Investigations - Other, liver enzymes increased, grade 3 | Investigations | 2 of 15 |
| platelet count decreased | Blood and lymphatic system disorders | 1 of 15 |
| dizziness, grade 3 | Nervous system disorders | 1 of 15 |
| hyponatremia, grade 4 | Metabolism and nutrition disorders | 1 of 15 |
For the Nelfinavir group, the registry reports 0 participants affected by serious adverse events among 15 at risk. It reports 5 participants affected by other adverse events among 15 at risk. These are counts recorded in the registry, not rates calculated across follow-up time, and they do not compare nelfinavir with another treatment.
The record lists no serious adverse event terms, so the kinds of serious events cannot be identified from the available information. Deaths are not reported in the registry record; a missing death count should not be read as a count of zero. The record does not give event-level timing, duration, severity beyond the serious/non-serious grouping, or attribution to the intervention. It also does not describe how many events occurred among the affected participants.
The counts are descriptive and should be read alongside the study's single-group design and the stated removal of some participants from outcome assessment after adverse events requiring treatment interruption. They do not establish comparative safety or determine whether an event was caused by nelfinavir.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study of nelfinavir was conducted for recurrent symptomatic adenoid cystic cancers of the head and neck. It describes the regimen, the intended schedule of tumor assessment, and a primary endpoint based on RECIST version v1.1 categories. It also records enrollment of 15 and provides limited adverse-event counts.
What the record does not establish is the observed distribution of tumor outcomes. It gives no counts for complete response, partial response, stable disease, or progressive disease, no reported number assessed, and no statistical analysis. It therefore cannot support a numerical statement about the primary endpoint or its precision. The completed status does not substitute for outcome results.
Even if outcome counts were available, the single-group design would describe observations during treatment rather than provide a controlled estimate of treatment effect. Without a concurrent comparator, the record cannot distinguish an intervention-related effect from other explanations for outcomes over time. The absence of masking is another design feature to consider when interpreting assessments, although the record does not report whether or how it affected evaluation.
The serious and other adverse-event counts are likewise descriptive. They do not establish comparative risk, causal attribution, or a complete account of safety over time. Taken together, the information supports an account of study design and registry-reported safety counts, but not a conclusion that nelfinavir did or did not alter tumor progression.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a registered clinical question and a result that can be quantitatively interpreted. The record specifies a population, intervention, and tumor outcome framework, but does not report the primary outcome counts or analyses. A completed study with enrollment information is not, by itself, enough to estimate an outcome or its uncertainty.
The design also illustrates why a single-group phase II study and a controlled comparison answer different questions. A single-group study can describe what was observed among its participants. It cannot supply the counterfactual comparison needed to estimate how outcomes would differ under another treatment. This distinction is essential when interpreting a trial title or stated purpose as evidence of efficacy.
The record highlights the importance of making participant flow and endpoint rules explicit. Participants removed after adverse events requiring treatment interruption were not assessed for the primary outcome, yet the number affected and the endpoint denominator are absent. In addition, the outcome description lists four RECIST categories while the metadata classifies the outcome as a binary count. Without a stated mapping, the event definition is unclear for a binary analysis.
Finally, the record's planning assumptions should be separated from the actual study design and from evidence about observed outcomes. Planning illustrations based on an assumed effect do not demonstrate observed power or resolve missing results. Clear outcome counts, analysis methods, and handling of incomplete assessments would be needed for readers to judge precision and interpret the study's findings.
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 principal limitation for interpreting the primary endpoint is that no tumor-progression results or analysis are reported in the registry record. The number assessed is also not reported. Enrollment of 15 cannot be used as the endpoint denominator because the record states that participants removed after adverse events requiring treatment interruption were not assessed for outcome measures.
The endpoint description lists complete response, partial response, stable disease, and progressive disease, while the outcome metadata classifies the measure as a binary participant count. The record does not explain how the categories were to be combined, or what period or event definition would govern a binary analysis. That ambiguity prevents reconstruction of the intended statistical outcome.
The single-group design has no concurrent comparator, and the study had no masking. The record does not report an analysis plan, statistical model, confidence interval, interim analyses, missing-data method, or analysis population. The brief summary mentions quality-of-life assessment, but its results are not reported. These gaps mean that neither the primary outcome nor quality-of-life findings can be evaluated from the registry information.
Safety information is limited to participant counts, without event terms for serious adverse events, event timing, or a reported death count. The summary also describes self-funded travel and no reimbursement; the record does not state whether these participation conditions affected retention or follow-up. No inference about their effect on results is possible.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Nelfinavir in Recurrent Adenoid Cystic Cancer of the Head and Neck — NCT01065844.
- Registry record last updated 2019-10-09; status last verified 2019-09.
- Results first posted 2016-12-19.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01065844 is a completed phase II, single-group study sponsored by the University of Iowa, with 15 enrolled participants. It evaluated nelfinavir at 1250 mg twice daily Monday-Sunday in patients with recurrent symptomatic adenoid cystic cancers of the head and neck. The record reports a start date of 2009-10 and primary completion in 2015-05.
The primary outcome was tumor progression assessed using RECIST version v1.1 categories every 1 to 3 months. The registry does not report primary outcome counts or analyses. It reports 0 serious adverse events affecting participants among 15 at risk and other adverse events affecting 5 among 15 at risk. Deaths are not reported in the registry record. The single-group design provides no concurrent control comparison.