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 Oral Tarceva Study for Recurrent/Residual Glioblastoma Multiforme and Anaplastic Astrocytoma (NCT00301418), a na phase 1, phase 2 study. The primary outcome is Safety of Twice a Day Oral 150 mg Erlotinib Dosing.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00301418 |
| Phase | Phase 1, Phase 2 |
| Condition | Glioblastoma Multiforme, Anaplastic Astrocytoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 11 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Northwell Health |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned participants with relapsed or refractory glioblastoma multiforme or anaplastic astrocytoma. These are the conditions named in the registry record. The record lists enrollment of 11 participants; additional eligibility details are not reported in the registry record.
Intervention
The intervention was oral erlotinib, also called Tarceva or OSI-774. The study description proposed testing whether it could be used safely at a dose of 150 mg two times a day for 12 months. The record does not establish that every participant received that dose or treatment duration.
Comparator
This was a single-group study, with no like-for-like control group recorded. Allocation is listed as not applicable and masking as none. The results therefore do not provide a randomized comparison of erlotinib with another treatment or with no treatment.
Primary question
The primary outcome concerned the number of participants with a Grade 2 or higher adverse event during the trial, in the context of twice-a-day oral 150 mg erlotinib dosing. The registry also names six-month progression-free survival and overall survival as secondary outcomes. Outcome results for these questions are not reported in the registry record.
3. Trial Design
The registry identifies this as a Phase I/II trial of oral erlotinib for relapsed or refractory glioblastoma multiforme and anaplastic astrocytoma. It is described as a single-group study, with enrollment of 11 participants. The allocation field is not applicable, and the masking field indicates none. There is no randomized comparator in the recorded design, so differences between treatment groups cannot be estimated from this trial.
The intervention is listed as erlotinib, under the name Tarceva. The study description states an intention to test whether erlotinib could be used safely up to 150 mg two times a day for 12 months and to study associations between response and genetic alterations, including EGFRvIII amplification and PTEN loss. These statements describe the study aims; they do not establish what dose each participant received or report the results of genetic analyses.
The sponsor is Northwell Health. The record lists the study start as 2006-03, primary completion as 2014-05, and status as completed. The primary outcome is a participant count for Grade 2 or higher adverse events over the duration of the trial. The listed secondary outcomes are six-month progression-free survival and overall survival. Although the record lists three outcome measures, outcome results are not reported in the registry record. Further details of eligibility, treatment administration, follow-up, and the analysis plan are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Tarceva (Erlotinib) |
|---|---|
| STARTED | 11 |
| COMPLETED | 11 |
| 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 | Tarceva (Erlotinib) |
|---|---|---|
| Participants analysed | 11 | |
| Age, Continuous | years | 50 (SD 10.7) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 7 |
| Region of Enrollment: United States | participants | 11 |
6. Primary Endpoint
The registry lists the primary outcome as Safety of Twice a Day Oral 150 mg Erlotinib Dosing, measured over duration of the trial.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is recorded as the number of participants experiencing a Grade 2 or higher adverse event during the duration of the trial. It is framed as a safety outcome for twice-a-day oral 150 mg erlotinib dosing. A participant count describes how many people met the stated event criterion; it is not itself a measure of how long an event lasted or how severe an event was beyond the specified grade threshold.
In general, safety endpoints are used to characterize unwanted events observed during treatment and follow-up. The grading threshold matters because it defines which events qualify for the endpoint. The registry record does not report the primary endpoint result, the grading criteria, or the method used to determine whether an event met the threshold. The adverse-event summary separately reports serious and other adverse-event counts; those categories should not be assumed to be interchangeable with the primary endpoint definition.
The registry also names six-month progression-free survival and overall survival as secondary outcomes. Their results are not reported in the registry record. Without the endpoint results and their analysis details, the primary safety question and the survival questions cannot be quantified from the posted record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The study is recorded as completed, with enrollment of 11 participants in a single group receiving the listed intervention, erlotinib. The posted adverse-event summary reports 0 participants affected in the serious adverse-event category, among 11 at risk, and 9 participants affected in the other adverse-event category, among 11 at risk. These are counts in the registry’s adverse-event summary; they are not reported as results of the primary Grade 2 or higher adverse-event endpoint. The registry does not list serious adverse-event terms, and the death count is not reported in the registry record.
The primary outcome is defined as the number of participants with a Grade 2 or higher adverse event over the duration of the trial. Its result is not reported in the registry record. Nor are results reported for the named secondary outcomes of six-month progression-free survival and overall survival. Thus, the posted information does not quantify the primary endpoint or the survival outcomes, and it does not provide an estimate of treatment effect for those outcomes.
In general, a count gives the observed number of participants meeting a definition, while a rate or proportion places that count in relation to the population assessed. A confidence interval, when reported, communicates uncertainty around an estimate; narrower intervals generally indicate greater precision, while wider intervals indicate less precision. No confidence interval or inferential analysis result is reported in the registry record. With no comparator group, the record also cannot supply a randomized between-group treatment effect.
The adverse-event counts should be read within the scope of their registry categories and the small enrolled group, not as proof that events outside those categories did or did not occur. The registry does not report the observation time for each participant, event timing, event attribution, or detailed definitions for the adverse-event categories. The 9 participants listed for other adverse events should not be treated as the primary endpoint count, because the record does not establish that this category uses the same Grade 2 threshold. These posted results describe reported counts and missing outcome information; they do not establish whether erlotinib is beneficial or harmful.
9. Statistical Methods Explained
The registry identifies a single-group study and records participant counts as the primary outcome format. It lists no analyses. The outcome description names the number of participants with a Grade 2 or higher adverse event during the trial. The result for that endpoint is not reported in the registry record, so there is no posted primary-outcome estimate to interpret statistically.
What does a participant count represent?
In general, a count is the number of people who meet a specified outcome definition. Counts depend on both the event definition and the group being observed. A count alone does not describe the time each person was followed, and it does not show whether events occurred early or late in follow-up. A proportion can express a count relative to the number assessed, but interpreting it requires a clear denominator and a consistent assessment rule. The primary endpoint result and its analysis details are not reported in the registry record.
Why do the outcome definitions matter?
In general, an endpoint is useful only when its criteria are clear enough to determine which observations qualify. The primary endpoint specifies Grade 2 or higher adverse events, while the registry’s adverse-event summary separates serious events from other events. Those labels represent different descriptions unless the record states otherwise. The registry does not report whether the other-event count uses the primary endpoint’s grade threshold, so that count cannot be substituted for the primary endpoint result. Event grading rules and the process for attributing events to treatment are also not reported in the registry record.
What can a single-group design estimate?
In general, a single-group study can describe outcomes observed among participants receiving the intervention, but it does not create a concurrent control group for estimating a randomized treatment contrast. Outcomes can also reflect the underlying condition, prior care, participant selection, follow-up, or other influences. Without a control group, observed outcomes alone cannot separate these influences from any effect associated with treatment. The recorded allocation is not applicable, masking is none, and the study model is single group.
The registry lists a sample-size planning illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; for time-to-event outcomes, the illustration is framed in terms of a total number of events, while other outcomes are framed as participants per arm. These are planning assumptions, not a description of this trial’s recorded design. The trial is single group, and the registry does not report a trial-specific sample-size justification. Any sample-size illustration is not observed power and does not show that a result was likely or unlikely to be statistically significant.
What does uncertainty add?
In general, a point estimate gives a single summary of an observed outcome, while a confidence interval communicates the range of values compatible with the data under the specified statistical method. A p-value, when used, summarizes how unusual data would be under a stated null model; it does not by itself measure clinical importance or the probability that a hypothesis is true. No confidence intervals, p-values, or analysis results are reported in the registry record. The record also lists no analyses, so the methods used to estimate uncertainty or assess outcomes are not reported in the registry record.
For survival outcomes, general methods account for the time until an event and may also account for participants whose event status is not observed through the end of follow-up. The registry names six-month progression-free survival and overall survival, but the analysis definitions, censoring rules, follow-up summaries, and outcome results are not reported in the registry record. Therefore, no survival estimate or precision measure can be interpreted from this record.
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 |
|---|---|---|---|---|---|---|
| Tarceva (Erlotinib) | 11 | 9 | 81.8% | 48.2% to 97.7% | 52.3% to 94.9% | 51.6% to 94.5% |
R code and output
arm <- c("Tarceva (Erlotinib)")
n <- c(11)
events <- c(9)
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 Tarceva (Erlotinib) 11 9 81.8 48.2 97.7 52.3 94.9 51.6 94.511. Multiplicity, Missing Data and Analysis Populations
The registry names a primary safety outcome and two secondary outcomes: six-month progression-free survival and overall survival. It records three outcome measures in total, but does not report the outcome results or analyses. The analysis plan, including any prespecified hierarchy among outcomes, is not reported in the registry record.
In general, testing or interpreting several outcomes creates a multiplicity question: the more separate questions considered, the greater the chance that some findings may appear noteworthy by chance. Study plans may address this through a hierarchy, adjustment, or other prespecified approach. No multiplicity procedure is reported in the registry record, so it is not possible to tell whether or how this issue was handled.
Details about missing outcome data, withdrawals, follow-up completeness, and the handling of participants with incomplete observations are not reported in the registry record. The analysis population definitions are also not reported. In general, these details matter because the participants included in an analysis and the way missing observations are handled can affect estimates and their interpretation. The enrollment count alone does not establish how many participants contributed data to each outcome analysis.
The record lists no analyses. Consequently, it does not provide information about adjustment for other variables, sensitivity analyses, interim analyses, or the statistical procedures used for the primary and secondary outcomes. These aspects of the study’s statistical plan are not reported in the registry record.
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 |
|---|---|---|---|
| Tarceva (Erlotinib) | 0 | 11 | 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 |
|---|---|
| Tarceva (Erlotinib) | 9 of 11 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Tarceva (Erlotinib) |
|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | 9 of 11 |
| Diarrhea | Gastrointestinal disorders | 6 of 11 |
The registry adverse-event summary lists 11 participants at risk for serious adverse events and 0 affected; it also lists 11 at risk for other adverse events and 9 affected. These are separate categories in the posted summary. The serious adverse-event terms are not listed, and the death count is not reported in the registry record. An unreported death count should not be read as a count of zero.
These figures are not identified as the result of the primary endpoint, which is defined as the number of participants with a Grade 2 or higher adverse event during the trial. The registry does not say that the other-event category uses that grade threshold. Nor does it report event timing, the number of events per participant, exposure duration, or how events were attributed to treatment. In general, adverse-event counts describe what was recorded under specified categories; they do not by themselves establish cause or provide a full account of safety. The reported counts should therefore be interpreted within the limits of the definitions and details available in the registry record.
13. What the Result Does, and Does Not, Mean
The record documents a completed, single-group Phase I/II study of erlotinib in participants with relapsed or refractory glioblastoma multiforme or anaplastic astrocytoma. It lists enrollment of 11 participants and reports adverse-event summary counts: 0 of 11 affected in the serious category and 9 of 11 affected in the other category. These figures describe the registry’s adverse-event categories and should not be recast as the primary Grade 2 or higher endpoint result.
The record does not report the primary safety outcome result, six-month progression-free survival, or overall survival. It also does not report confidence intervals, p-values, or analyses. Therefore, the posted information does not quantify the principal safety endpoint, estimate survival outcomes, or show the precision of any treatment effect. The study’s stated interest in genetic alterations is an aim, not a reported finding; genetic results are not reported in the registry record.
In general, evidence from a single-group study can describe outcomes observed in the treated group, but without a concurrent comparator it cannot establish what would have happened under another treatment or no treatment. Interpretation is further constrained when outcome definitions, follow-up details, and analysis populations are unavailable. The record supports a limited description of its design and adverse-event counts; it does not establish that erlotinib improves or fails to improve outcomes, and it should not be read as a clinical recommendation.
14. Why This Trial Matters Statistically
This study is statistically notable because its registry record combines a single-group design with a stated safety question and secondary survival outcomes. A single group can provide descriptive information about events recorded among participants, but it cannot provide a randomized comparison against another care strategy. That distinction is important when readers encounter treatment-related language in a study description: an aim to assess safety or survival is not itself evidence of an estimated treatment effect.
The record also illustrates why outcome definitions should be kept distinct. The primary endpoint is specified as a participant count for Grade 2 or higher adverse events. The adverse-event summary separately reports serious and other categories. Without a documented link between those categories and the primary endpoint threshold, their counts cannot be used as substitutes for the primary result. The absence of a reported primary outcome is particularly consequential because it leaves the trial’s central safety measure unquantified in the registry record.
In general, statistical interpretation depends on more than the direction of an observed result. It also depends on the study design, outcome definition, available denominator, uncertainty, follow-up, and analysis plan. Here, analysis results and precision measures are not reported, while the named survival outcomes lack posted results. The sample-size planning assumptions are not the trial design and do not provide observed power. These distinctions help separate the study’s aims and recorded adverse-event counts from conclusions that the available registry information cannot support.
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.
Several trial-specific details needed to assess the posted results are not reported in the registry record. The eligibility criteria and participant characteristics are not described in the available record. Treatment administration details, including the dose actually received by each participant and treatment duration, are not reported; the study description’s intended dose and duration do not establish individual exposure.
The primary endpoint result is not reported, despite the endpoint being defined as the number of participants with a Grade 2 or higher adverse event. The listed secondary outcomes, six-month progression-free survival and overall survival, also have no reported results. The record lists no analyses, and it does not provide confidence intervals, p-values, analysis populations, or methods for handling missing data. Follow-up completeness and time under observation are not reported.
The adverse-event summary does not list serious-event terms, and the death count is not reported in the registry record. Event definitions, timing, duration, severity grading procedures, and attribution are not described. The relationship between the other-event category and the primary endpoint’s Grade 2 threshold is not reported. The record also does not give results for the planned examination of genetic alterations, including EGFRvIII amplification and PTEN loss.
The single-group design does not include a randomized comparator. Thus, even if outcome estimates were available, the design would not support a randomized between-group comparison. These limitations concern what can be interpreted from the registry record and do not establish that the study did or did not collect additional information elsewhere.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Oral Tarceva Study for Recurrent/Residual Glioblastoma Multiforme and Anaplastic Astrocytoma — NCT00301418.
- Registry record last updated 2016-02-10; status last verified 2016-01.
- Results first posted 2016-02-10.
- 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 identifies NCT00301418 as a completed Phase I/II, single-group study sponsored by Northwell Health. It concerns oral erlotinib (Tarceva) for relapsed or refractory glioblastoma multiforme and anaplastic astrocytoma. Enrollment is listed as 11; the start is recorded as 2006-03 and primary completion as 2014-05. Masking is listed as none.
The primary outcome is the number of participants with a Grade 2 or higher adverse event during the trial, in the context of twice-a-day oral 150 mg dosing. The registry also names six-month progression-free survival and overall survival. Their results, along with the primary endpoint result and analysis methods, are not reported in the registry record. The posted adverse-event summary lists 0 of 11 affected for serious events and 9 of 11 for other events; serious-event terms are absent and the death count is not reported. The record does not describe a randomized comparison.