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 Erlotinib in Treating Patients With Recurrent Malignant Glioma or Recurrent or Progressive Meningioma (NCT00045110), a non-randomized phase 1, phase 2 study. The primary outcome is Number of Dose Limiting Toxicity (DLT) Each Dose Level Phase I.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00045110 |
| Phase | Phase 1, Phase 2 |
| Condition | Adult Anaplastic Astrocytoma, Adult Anaplastic Oligodendroglioma, Adult Giant Cell Glioblastoma |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 136 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned patients with recurrent malignant glioma or recurrent or progressive meningioma. The primary endpoint description specifies patients with recurrent disease or newly diagnosed disease after radiation therapy without tumor progression, on enzyme-inducing antiepileptic drugs. The registry lists adult glioma and meningioma conditions, as well as recurrent adult brain tumor.
Intervention
The study intervention was oral erlotinib hydrochloride. The registry also lists laboratory biomarker analysis and a pharmacological study. In Phase I, the dose-escalation description concerns patients concurrently receiving enzyme-inducing antiepileptic drugs. The Phase II description concerns patients not concurrently receiving them.
Comparator
This was a non-randomized trial, not a like-for-like comparison of treatment and control arms. The registry describes dose escalation in Phase I and a Phase II group, but does not report a randomized control group. Differences between those groups therefore cannot be read as randomized treatment effects.
Primary question
The primary endpoint asks how many dose-limiting toxicities occurred at each Phase I dose level during 28 days. It is a safety and dose-finding question, not a direct measure of tumor response or survival. The registry record does not report the dose-level counts.
The title describes erlotinib in recurrent malignant glioma and recurrent or progressive meningioma. The official title emphasizes a Phase I/II trial in recurrent malignant gliomas and malignant gliomas after radiation therapy. These descriptions indicate the disease settings under study, but they do not establish that every listed condition contributed participants to every endpoint analysis.
The registry states that erlotinib may stop tumor-cell growth by blocking enzymes necessary for growth. That is the stated rationale, not a trial result. The primary endpoint instead focuses on dose-limiting toxicity during dose escalation. The record also lists secondary outcomes involving adverse events, survival, response, and pharmacokinetic measurements. Results for those outcomes cannot be inferred from their listing.
The central statistical distinction is between asking whether a dose can be tolerated and asking whether treatment changes a clinical outcome. The primary endpoint addresses the former. The registry record does not report a control comparison or the primary endpoint values, so it cannot establish a comparative treatment effect from the information recorded.
3. Trial Design
This was a completed Phase I/II, non-randomized, parallel-design trial with no masking. The registry lists an enrollment of 136 and names the National Cancer Institute as sponsor. The recorded start is 2002-08 and primary completion is 2010-12. These dates describe the registry record; they do not supply details about recruitment timing or follow-up for individual participants.
The Phase I description concerns dose escalation among patients concurrently receiving enzyme-inducing antiepileptic drugs. Cohorts of 3-6 patients were to receive escalating erlotinib doses until the maximum tolerated dose was determined. Courses were described as repeating every 4 weeks in the absence of disease progression or unacceptable toxicity. The primary endpoint is the number of dose-limiting toxicities at each dose level over 28 days.
The Phase II description concerns patients not concurrently receiving enzyme-inducing antiepileptic drugs and specifies a predetermined erlotinib dose of 150mg/day. It also describes patients requiring surgery as treated 7 days before tumor removal, along with pharmacokinetic analysis and assessment of effects on epidermal growth factor receptor. The registry wording does not provide a complete account of all Phase II procedures or the full dose-escalation stopping rules.
Because allocation was non-randomized, Phase I and Phase II groups should not be treated as interchangeable arms in a controlled comparison. Their descriptions differ, including their relation to enzyme-inducing antiepileptic drugs and their study purpose. Any difference in recorded outcomes across them could reflect participant or design differences as well as treatment exposure.
The registry lists 18 outcome measures, including the primary dose-limiting toxicity count and secondary outcomes related to adverse events, survival, response, and drug concentration. It lists no statistical analyses. The statistical analysis plan, sample-size justification, interim analyses, and details of analysis populations 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.
100mg Phase 1 Dose Escalalation
| Stage | Phase 1 Dose Escalation | Phase 2 - Recurrent Malignant Glioma | Phase 2 - Newly Diagnosed GBM Post RT |
|---|---|---|---|
| STARTED | 6 | 59 | 45 |
| COMPLETED | 6 | 53 | 43 |
| NOT COMPLETED | 0 | 6 | 2 |
Reasons for not completing this period:
| Reason | Phase 1 Dose Escalation | Phase 2 - Recurrent Malignant Glioma | Phase 2 - Newly Diagnosed GBM Post RT |
|---|---|---|---|
| did not initiate treatment | 0 | 2 | 1 |
| prior treatment history | 0 | 4 | 0 |
| ineligible histology | 0 | 0 | 1 |
200mg Phase 1 Dose Escalation
| Stage | Phase 1 Dose Escalation | Phase 2 - Recurrent Malignant Glioma | Phase 2 - Newly Diagnosed GBM Post RT |
|---|---|---|---|
| STARTED | 4 | 0 | 0 |
| COMPLETED | 4 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 |
275mg Phase 1 Dose Escalation
| Stage | Phase 1 Dose Escalation | Phase 2 - Recurrent Malignant Glioma | Phase 2 - Newly Diagnosed GBM Post RT |
|---|---|---|---|
| STARTED | 3 | 0 | 0 |
| COMPLETED | 3 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 |
400mg Phase 1 Dose Escalation
| Stage | Phase 1 Dose Escalation | Phase 2 - Recurrent Malignant Glioma | Phase 2 - Newly Diagnosed GBM Post RT |
|---|---|---|---|
| STARTED | 3 | 0 | 0 |
| COMPLETED | 3 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Patients were eligible if they had recurrent disease or if they were newly diagnosed post RT and did NOT have tumor progression (nonprogression glioblastoma) on Enzyme-inducing Antiepileptic Drugs
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.
Population: enrolled between 8/2002 and 8/2005. All recruited from outpatient setting.
| Characteristic | Unit | Phase 1 Dose Escalation | Phase 2 w/ Recurrent Malignant Glioma | Phase 2 Newly Diagnosed GBM Post RT | Total |
|---|---|---|---|---|---|
| Participants analysed | 32 | 59 | 45 | 136 | |
| Age, Continuous | years | 45 (19 to 76) | 54 (29 to 78) | 52 (19 to 72) | 52 (19 to 78) |
| Sex: Female, Male: Female | Participants | 12 | 25 | 10 | 47 |
| Sex: Female, Male: Male | Participants | 20 | 34 | 35 | 89 |
| Histology: Glioblastoma | participants | 21 | 42 | 44 | 107 |
| Histology: Anaplastic Astrocytoma | participants | 8 | 14 | 1 | 23 |
| Histology: Anaplastic oligodendroglioma | participants | 2 | 1 | 0 | 3 |
| Histology: Atypical meningioma | participants | 1 | 0 | 0 | 1 |
| Histology: Pleomorphic Xanthoastrocytoma | participants | 0 | 1 | 0 | 1 |
| Histology: Oligodendroglioma | participants | 0 | 1 | 0 | 1 |
| Prior Radiation Treatment: Yes | participants | 32 | 55 | 45 | 132 |
| Prior Radiation Treatment: No | participants | 0 | 4 | 0 | 4 |
| Prior Number of Chemotherapy Regimens: 0 treatments | participants | 3 | 4 | 0 | 7 |
| Prior Number of Chemotherapy Regimens: 1 treatment | participants | 13 | 31 | 45 | 89 |
| Prior Number of Chemotherapy Regimens: 2 treatments | participants | 14 | 21 | 0 | 35 |
| Prior Number of Chemotherapy Regimens: 3 treatments | participants | 2 | 3 | 0 | 5 |
| Karnofsky Performance Status Scale | units on a scale | 90 (60 to 100) | 80 (60 to 100) | 90 (60 to 100) | 80 (60 to 100) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Dose Limiting Toxicity (DLT) Each Dose Level Phase I, measured over 28 days.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | dose limiting toxicities |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of dose-limiting toxicities at each Phase I dose level during 28 days. The registry defines a dose-limiting toxicity as any grade 3 thrombocytopenia, grade 4 anemia or neutropenia, any non-hematologic grade 3 toxicity, or failure to recover from toxicities enough to be eligible for erlotinib retreatment within 2 weeks of the last dose.
Counting these events during dose escalation is useful because the purpose of that phase is to characterize tolerability across dose levels and determine a maximum tolerated dose. The endpoint is tied to a specific observation window and a stated toxicity definition. Those details matter: the count depends on which events meet the definition and occur within the timeframe, as well as on the number of participants assessed at each dose level.
A count is not a rate unless its denominator is also specified. The primary endpoint title calls for counts by dose level, but the registry record does not report those counts, the number assessed at each dose level, or a summary of the dose-escalation decision. It therefore does not support a numerical description of the observed dose-limiting toxicity burden or the maximum tolerated dose.
This endpoint is distinct from tumor response, progression, or survival. It measures a safety outcome used in dose finding; it does not by itself establish whether treatment changes disease outcomes. The registry lists response and survival among secondary outcomes, but their results are not reported in the information recorded.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry establishes that the trial was completed and records its broad design, interventions, outcomes, and some adverse-event figures. It does not report the primary endpoint values. In particular, no dose-level counts of dose-limiting toxicities are recorded, even though that count is the primary endpoint and its timeframe is stated as 28 days. The registry also does not report a maximum tolerated dose determination.
That absence limits interpretation at the point the Phase I endpoint is meant to inform. Without dose-level counts and their corresponding assessed populations, readers cannot describe how often the defined toxicities occurred across doses or evaluate the pattern used for dose finding. No numerical conclusion about the primary safety endpoint follows from the endpoint definition alone.
The record lists secondary outcomes for adverse events, survival, response, and pharmacokinetics, but a listed outcome is not a reported result. The posted serious adverse-event figures show 0 affected participants among 32 at risk in the Phase I dose-escalation group and 2 among 99 at risk in the Phase II recurrent malignant gliomas and nonprogressive GBM group. Other adverse events are recorded as affecting 27 of 32 and 11 of 99, respectively. These are descriptive registry counts, not a randomized comparison and not a substitute for the missing primary endpoint results.
The serious-event terms listed are seizure and hypomagnesemia, each at 1.0%. The registry does not supply enough detail in these figures to establish event timing, recurrence, relationship to erlotinib, or the basis for the term-specific percentages. Death counts are not reported in the adverse-event information. Such omissions should not be interpreted as evidence that events did not occur.
The trial was non-randomized and had no masking. It therefore does not provide a randomized control contrast. The Phase I dose-escalation group and Phase II group had different descriptions and should not be compared as if allocation had made them similar. Any apparent difference in the posted adverse-event counts is descriptive; it does not identify a causal effect of treatment or a dose effect.
The registry describes an enrollment of 136, while the adverse-event groups have their own at-risk counts. Those counts should be read in their stated group context, not assumed to represent a single analysis population for every outcome. The record does not explain the relationship between enrollment, endpoint-specific populations, and missing observations.
Overall, the record supports a description of the study’s design and selected safety figures, but not a complete statistical assessment of the primary endpoint or the listed efficacy outcomes. The absence of posted analyses means estimates, uncertainty intervals, and adjusted comparisons are not reported in the registry record.
9. Statistical Methods Explained
Phase I dose escalation and Phase II outcome assessment answer related but different questions. Dose escalation examines safety across dose levels and uses dose-limiting toxicities to inform selection of a dose for further study. A later-phase group can assess outcomes such as response or survival, but conclusions require outcome data and an analysis appropriate to the design. The registry describes both phases, yet does not report statistical analyses for either.
Why a dose-limiting toxicity count needs context
A count records the number of qualifying events or affected participants, depending on the endpoint’s operational definition. The primary endpoint is phrased as the number of dose-limiting toxicities at each dose level, and its definition specifies qualifying toxicities and a 28-day timeframe. To interpret counts, readers also need to know how many participants were evaluated at each level, whether participants could contribute more than one event, and how incomplete observation was handled. Those details are not reported in the registry record.
In general, counts often increase as the number of people observed increases, even when the underlying event risk is unchanged. A rate or proportion can make groups with different denominators more interpretable, but it still depends on a clearly defined population and complete outcome ascertainment. The registry’s primary endpoint is a count; it does not report the denominators or a rate for each dose level.
What a non-randomized design permits
Random allocation is a method for balancing measured and unmeasured participant characteristics across comparison groups, on average. Without randomization, group differences may reflect differences in eligibility, clinical status, co-medication, phase of study, or other factors, in addition to any treatment exposure. This is a general design principle. In this trial, the registry specifically says allocation was non-randomized and describes Phase I and Phase II groups with different study roles. Their adverse-event figures are therefore descriptive, not evidence of a causal contrast between groups.
Masking can reduce some forms of bias in outcome assessment or participant reporting. The record lists no masking. It does not describe additional safeguards for outcome assessment, so their use is not reported in the registry record. This absence does not establish that no safeguards existed; it limits what can be assessed from the registry.
Understanding uncertainty
In general, a confidence interval expresses the range of values compatible with an estimate under a statistical model and its assumptions. Wider intervals indicate less precision, while narrower intervals indicate greater precision. No confidence intervals or other uncertainty estimates are reported for the trial outcomes in the registry record. Consequently, the precision of the posted adverse-event counts cannot be assessed from those counts alone.
In general, statistical tests assess compatibility between data and a specified null hypothesis under model assumptions. A p-value is not the probability that a hypothesis is true, nor does it measure clinical importance. No p-values or inferential analyses are listed for the outcomes in this record. There is therefore no basis in the registry information for describing statistical significance.
Planning calculations are not trial findings
The planning material associated with the results describes an illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. It states that the effect to detect is a specified fraction of the observed effect; time-to-event calculations use a total event count, while other calculations express participants per arm. Such calculations are planning illustrations, not observed trial outcomes and not observed power. Their assumptions do not establish that the trial itself used that design or had that statistical plan.
In general, power calculations describe the chance of detecting an effect of a specified size under assumed design and model conditions. After a study is complete, a retrospective calculation based on an observed effect does not measure what the trial was capable of detecting independently of the result. The registry’s own sample-size justification and statistical planning are not reported in the registry record. The planning illustration should not be used to fill that gap.
Why over-dispersion can matter
In general, count data can vary more than a simple model assumes. This is called over-dispersion. If it is present and ignored, estimated uncertainty may be too small and inferential conclusions may be overly confident. Whether over-dispersion is relevant depends on the outcome distribution, exposure time, and analysis model. The registry record does not describe a count model or report the dose-level data needed to evaluate this issue. It would not be appropriate to assert that over-dispersion occurred in this trial.
For time-to-event outcomes such as survival, general analyses account for both the timing of events and censoring, when a participant’s event time is not observed during follow-up. The registry lists survival outcomes but does not report event counts, follow-up distributions, censoring details, survival estimates, or analysis methods. The listed survival outcomes alone do not permit reconstruction of a time-to-event analysis.
For response outcomes, a response rate requires a response definition, an analysis population, and observed response counts. The registry names modified RECIST criteria for the response outcome but does not report the response results or analysis details. As a result, neither a response estimate nor its precision can be described from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 18 outcome measures, including a primary dose-limiting toxicity endpoint and secondary measures covering adverse events, survival, response, and pharmacokinetic characteristics. When a study assesses multiple outcomes, the chance of at least one apparently unusual statistical result can increase if many tests are performed. General approaches to multiplicity include designating a primary outcome, using a testing hierarchy, or adjusting inference; which approach is suitable depends on the study questions and analysis plan.
The registry record does not report whether multiplicity was addressed, how outcomes were prioritized analytically, or whether any secondary outcomes were exploratory. It lists no statistical analyses. Therefore, no claim about multiplicity adjustment or the interpretation of multiple tests can be made from the record.
Missing-data handling is also not reported in the registry record. For the primary endpoint, the record does not state how participants with incomplete observation during the 28-day timeframe were treated, whether every participant was evaluable, or how missing toxicity assessments affected the dose-level counts. For survival and response, details of missing outcomes, follow-up, and censoring are not reported.
The analysis populations are not described beyond the eligibility and group descriptions. The record does not state whether analyses used all enrolled participants, participants who received treatment, participants meeting an evaluability rule, or a different defined population. The enrollment of 136 should not be assumed to be the denominator for every outcome. Likewise, the adverse-event group counts should not be treated as the analysis populations for efficacy endpoints unless the registry specifies that relationship.
These omissions matter because analysis choices can affect both estimates and their uncertainty. Without a statistical plan, missing-data rules, and explicit analysis populations, readers cannot fully assess the robustness or reproducibility of results. These items are not reported in the registry record; their absence from the record does not establish what procedures were or were not used during the study.
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 |
|---|---|---|---|
| Phase 1 Dose Escalation | 0 | 32 | 0.0% |
| Phase 2 Recurrent Malignant Gliomas and Nonprogressive Gbm | 2 | 99 | 2.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Phase 1 Dose Escalation | 27 of 32 |
| Phase 2 Recurrent Malignant Gliomas and Nonprogressive Gbm | 11 of 99 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase 1 Dose Escalation | Phase 2 Recurrent Malignant Gliomas and Nonprogressive Gbm |
|---|---|---|---|
| seizure | Nervous system disorders | 0 of 32 | 1 of 99 |
| hypomagnesemia | Metabolism and nutrition disorders | 0 of 32 | 1 of 99 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase 1 Dose Escalation | Phase 2 Recurrent Malignant Gliomas and Nonprogressive Gbm |
|---|---|---|---|
| rash | Skin and subcutaneous tissue disorders | 19 of 32 | 11 of 99 |
| diarrhea | Gastrointestinal disorders | 9 of 32 | 2 of 99 |
| fatigue | General disorders | 5 of 32 | 3 of 99 |
| nausea | Gastrointestinal disorders | 3 of 32 | 0 of 99 |
| granulocytopenia | Investigations | 2 of 32 | 0 of 99 |
Serious events with the highest arm-level rate: seizure (1.0%), hypomagnesemia (1.0%).
The registry reports serious adverse-event figures separately for the Phase I dose-escalation group and the Phase II recurrent malignant gliomas and nonprogressive GBM group. In Phase I, 0 of 32 at-risk participants are recorded as affected by a serious adverse event. In Phase II, 2 of 99 at-risk participants are recorded as affected. The registry also records other adverse events affecting 27 of 32 and 11 of 99 participants, respectively.
These are descriptive figures. They do not show that one group was safer than the other, because the trial was non-randomized and the groups had different descriptions and roles. The figures also do not establish whether an event was caused by erlotinib. The registry does not report event timing, duration, recurrence, severity detail beyond the serious-event category, or individual-level exposure in these summaries.
The listed serious-event terms are seizure and hypomagnesemia, each reported at 1.0%. The record does not clarify from these figures whether a term represents a participant, an event, or a particular reporting convention. Death counts are not reported in the adverse-event information. A missing death count should not be interpreted as zero.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase I/II study of erlotinib was completed, with a non-randomized design, no masking, and a primary endpoint focused on dose-limiting toxicities during Phase I. It also records selected adverse-event counts and lists secondary outcomes addressing survival, response, and pharmacokinetic measurements. These details describe the study questions and some safety reporting; they are not a complete account of its results.
The primary endpoint result itself is not reported in the registry record. There are no dose-level toxicity counts, no reported maximum tolerated dose, and no reported statistical analysis. Thus, the endpoint definition does not reveal how many qualifying events occurred or how the dose-escalation findings were interpreted.
The serious adverse-event counts can be read as descriptive observations within the two named groups. They cannot establish a treatment effect, a comparison between groups, or a causal relationship between erlotinib and an event. Non-random allocation leaves potential differences between groups, and the group descriptions indicate distinct study settings.
The registry’s listing of survival and response outcomes does not demonstrate that those outcomes improved, worsened, or were unchanged. Outcome names are not findings. Without reported outcome estimates, analysis methods, and uncertainty measures, the magnitude and precision of any clinical outcome cannot be assessed from this record.
The planning illustration is also not evidence about what happened in the trial. Planning sample sizes are not observed results or observed power. The record does not report the trial’s own sample-size justification or statistical analysis plan, so the illustration cannot be taken as a description of the protocol’s rationale.
In general, a completed trial can contribute useful information even when a registry record is incomplete, but conclusions must stay within the data that are actually recorded. For this study, the available information supports a description of design, endpoint definitions, and selected safety figures. It does not support a numerical conclusion about the primary endpoint, a controlled treatment comparison, or the listed efficacy outcomes.
13. Why This Trial Matters Statistically
This trial illustrates why the study design and the endpoint must be read together. Its primary endpoint is a dose-level count of defined toxicities over 28 days, a measure suited to a Phase I dose-escalation question. It is not a measure of tumor response or survival. Separating safety and dose finding from efficacy outcomes prevents a common interpretive error: treating a safety endpoint as evidence about clinical benefit.
The non-randomized design is also important statistically. Phase I and Phase II serve different roles and have different group descriptions, including whether participants were concurrently receiving enzyme-inducing antiepileptic drugs. Comparing their outcomes as if they were randomized arms would ignore these design differences. The adverse-event counts are most appropriately read as descriptive group-specific observations.
The record further illustrates the distinction between an outcome being named and an outcome being reported. It identifies response and survival measures, but does not include their results or statistical analyses. A complete interpretation ordinarily depends on estimates, denominators, follow-up information, and measures of uncertainty. Without those items, readers cannot assess precision, compare outcomes reliably, or determine how missing observations were handled.
Finally, planning illustrations should be kept separate from evidence generated by the trial. Assumptions used for a planning calculation do not document the actual protocol, and a planning sample size is not observed power. The registry record does not report the study’s own sample-size justification or statistical plan. These distinctions are broadly useful when reading clinical trial registries: design descriptions, planned outcomes, posted results, and inferential conclusions are different kinds of information.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most consequential limitation in the registry record is that it does not report the primary endpoint results. Dose-limiting toxicity counts by dose level, the corresponding numbers assessed, and the maximum tolerated dose determination are not reported. The record therefore cannot support a numerical interpretation of the principal Phase I endpoint.
The record also lacks statistical analyses for the outcomes. It does not report estimates of response or survival, confidence intervals, p-values, analysis methods, or the handling of censoring and missing data. Although 18 outcome measures are listed, their results and analytic priority beyond the primary designation are not given. The statistical plan, interim analyses, and sample-size justification are not reported in the registry record.
The non-randomized design limits causal interpretation. Phase I and Phase II groups have distinct descriptions and are not a randomized treatment-control comparison. Their adverse-event figures should not be interpreted as evidence of relative safety or treatment effect. The record does not provide enough information to assess the comparability of participants across groups or potential confounding.
Safety summaries are limited as well. The registry records at-risk and affected counts, two serious-event terms with percentages, and no death counts. It does not provide detailed event timing, recurrence, attribution, or the basis for the term-level percentages in these figures. The adverse-event summaries cannot replace the unreported primary dose-limiting toxicity results.
Finally, the registry’s broad disease and outcome listings do not specify the analysis population for each result. Enrollment does not by itself establish the number included in every endpoint analysis. The relation between enrollment, group-specific at-risk counts, and outcome-specific populations is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Erlotinib in Treating Patients With Recurrent Malignant Glioma or Recurrent or Progressive Meningioma — NCT00045110.
- Registry record last updated 2017-08-17; status last verified 2017-07.
- Results first posted 2017-07-13.
- 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
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18. Record Summary
NCT00045110 is a completed, non-randomized Phase I/II trial sponsored by the National Cancer Institute. The registry records enrollment of 136, no masking, and a parallel model. The recorded start is 2002-08, with primary completion in 2010-12.
The study evaluated erlotinib hydrochloride in patients with recurrent malignant glioma or recurrent or progressive meningioma. Its primary endpoint was the number of dose-limiting toxicities at each Phase I dose level over 28 days, using a stated toxicity definition. The registry lists 18 outcome measures, including adverse events, survival, response, and pharmacokinetic measures.
The registry record does not report the primary endpoint counts or statistical analyses. It records serious adverse events affecting 0 of 32 participants in the Phase I group and 2 of 99 in the Phase II group, along with other adverse-event counts and two serious-event terms. Because the trial was non-randomized, these figures are descriptive rather than a controlled comparison.