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 Topotecan to Treat Recurrent or Persistent Solid Tumors (NCT00382733), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated Dose (MTD) of Single Agent Metronomic Oral Topotecan.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00382733 |
| Phase | Phase 1, Phase 2 |
| Condition | Tumors |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 26 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Accelerated Community Oncology Research Network |
| Results posted | Yes |
2. Clinical Question
The study asks whether a metronomic schedule of oral topotecan can be administered at different dose levels to people with recurrent or persistent solid tumors, and how tolerable those doses are. Its named primary outcome is the maximum tolerated dose (MTD) of single-agent metronomic oral topotecan, expressed in mg/day. The registry describes assessment of the MTD during the first treatment cycle, days 1-28, using dose-limiting toxicities (DLTs).
The study also describes evaluating the effectiveness of oral topotecan, collecting information about how the medication is broken down and absorbed in the body, and assessing quality of life during treatment. The listed secondary outcomes are DLTs and best overall response. The registry record does not report a final MTD value or results for these secondary outcomes.
The population is described broadly as people with recurrent or persistent solid tumors; the registry condition is listed as tumors. More specific eligibility criteria and participant characteristics are not reported in the registry record. The intervention is oral topotecan, given at different dose levels. There is no separate comparator arm: the record identifies a single-group model and lists allocation as NA. Consequently, the central question is dose-finding within a treated group, not a randomized comparison of topotecan with another treatment or with no treatment.
For interpretation, the distinction between the question and the available results is important. The registry names a dose-finding endpoint and provides a description of the planned escalation logic, but the posted record does not give the endpoint value. The available information therefore identifies what the study set out to assess, rather than establishing the dose ultimately selected or describing the primary outcome observed.
3. Trial Design
This is a Phase I/Phase II, single-group study of oral topotecan. The registry lists enrollment as 26 and the status as completed. The start is recorded as 2006-11, and primary completion as 2011-03. The sponsor is Accelerated Community Oncology Research Network. Masking is listed as none, and allocation is listed as NA.
The intervention is oral topotecan, administered across dose levels. The primary-outcome description says the MTD was assessed during the first cycle of treatment, days 1-28, using a standard 3+3 dose-escalation cohort design. Under the described rule, if none of 3 subjects experiences a DLT at a dose level, enrollment proceeds at the next higher dose level. If at least 2 subjects experience a DLT at a dose level, escalation stops. If 1 of 3 experiences a DLT, 3 additional subjects are enrolled at that same dose level.
The registry reports DLT information for 3 subjects at 0.25 mg/day, 3 at 0.50 mg/day, 3 at 0.75 mg/day, and 3 at 1.0 mg/day; the final dose-level statement is incomplete, giving 2 subjects who received topotecan without specifying the dose in the available text. Separately, the safety groups list dose-level group sizes of 3, 4, 3, 14, and 2 for 0.25, 0.50, 0.75, 1.0, and 1.25 mg daily, respectively. These are registry entries from different parts of the record and should not be assumed to represent identical analysis populations.
Because the study is single-group and has no randomized comparator, the dose groups are not like-for-like control groups. Participants at different dose levels may have entered at different points in the escalation process. The registry does not report further details about the conduct of dose assignment, eligibility, or follow-up.
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.
Before assignment: Informed consent was obtained from all subjects. All subjects underwent a screening period that could last up to 4 weeks during which pre-study assessments were completed. During the dose finding portion of the study, subjects were assigned to a Dose Level during enrollment. Once the MTD was determined, additional subjects were treated at the MTD.
Overall Study
| Stage | Oral Topotecan 0.25 mg Daily | Oral Topotecan 0.50 mg Daily | Oral Topotecan 0.75 mg Daily | Oral Topotecan 1.0 mg Daily | Oral Topotecan 1.25 mg Daily |
|---|---|---|---|---|---|
| STARTED | 3 | 4 | 3 | 14 | 2 |
| COMPLETED | 3 | 3 | 3 | 14 | 2 |
| NOT COMPLETED | 0 | 1 | 0 | 0 | 0 |
Reasons for not completing this period:
| Reason | Oral Topotecan 0.25 mg Daily | Oral Topotecan 0.50 mg Daily | Oral Topotecan 0.75 mg Daily | Oral Topotecan 1.0 mg Daily | Oral Topotecan 1.25 mg Daily |
|---|---|---|---|---|---|
| Withdrawal by Subject | 0 | 1 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: DLT information was available for 3 subjects who received a topotecan dose of 0.25 mg/day, 3 subjects who received a topotecan dose of 0.50 mg/day, 3 subjects who received a topotecan dose of 0.75 mg/day, 3 subjects who received a topotecan dose of 1.0 mg/day, and 2 subjects who received a topotecan dose of 1.25 mg/day.
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 | Metronomic Oral Topotecan |
|---|---|---|
| Participants analysed | 26 | |
| Age Continuous | years | 65.6 (SD 8.99) |
| Sex: Female, Male: Female | Participants | 20 |
| Sex: Female, Male: Male | Participants | 6 |
| Region of Enrollment: United States | participants | 26 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (MTD) of Single Agent Metronomic Oral Topotecan, measured over MTD was assessed during the first cycle of treatment (days 1-28)..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg/day |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the maximum tolerated dose of single-agent metronomic oral topotecan, measured in mg/day. The registry says that this endpoint was assessed during the first treatment cycle, days 1-28, using DLT information. It describes DLT-based dose escalation as the method for identifying a dose level at which escalation should stop or continue.
In general, an MTD endpoint is used in early dose-finding research to characterize tolerability across dose levels and to guide selection of a dose for further study. The 3+3 approach described in this record uses small cohorts and prespecified DLT counts to make escalation decisions. Its aim is not to estimate a comparative treatment effect against a control group. The resulting MTD is therefore an endpoint about dose and short-term tolerability under the study’s definitions and observation period, rather than a direct measure of effectiveness.
The registry describes the decision rules but does not provide the final MTD value, the DLT count at each dose level, or a complete account of which dose was selected. The dose information in the population description is incomplete for its final entry. Accordingly, the endpoint’s purpose and measurement window can be described, but the posted record does not allow the reader to state the observed MTD or independently reconstruct the dose-escalation outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a completed single-group Phase I/Phase II study of oral topotecan and lists enrollment as 26. It describes a dose-escalation design in which DLTs during the first treatment cycle inform decisions about moving to a higher dose, stopping escalation, or enrolling additional subjects at the same dose. These design details clarify how the primary question was intended to be addressed.
However, the primary outcome is named without a reported numerical result. The registry record does not report the final MTD, a complete DLT distribution by dose, or an estimate with uncertainty. It therefore does not support a conclusion about which dose met the MTD criterion. The available dose-level population text is incomplete, and the safety-group counts should not be substituted for missing primary-endpoint results: those groups are listed under adverse events and are not explicitly identified as the DLT analysis population.
The record also names best overall response as a secondary outcome, but does not report its results. It does not provide a treatment-effect estimate, confidence interval, or comparative analysis. Since this is a single-group study with no randomized comparator, comparisons among dose groups would not constitute randomized treatment comparisons. In general, differences across dose cohorts can reflect the way participants entered the escalation sequence as well as dose, and the registry does not report an adjusted analysis that would address such concerns.
The safety information gives counts of participants at risk and affected for each dose group, plus a list of serious adverse-event terms with percentages. These figures describe the entries recorded for those groups. They do not provide a complete clinical profile of event timing, duration, severity grading, relationship to treatment, or statistical uncertainty. The registry’s listed safety figures should therefore be read as descriptive rather than as estimates from a controlled comparison.
There is no basis in the posted record to interpret the absence of a reported endpoint result as evidence that no DLTs occurred or that no response was observed. Missing result information is not a zero result. Likewise, the completed status indicates a registry status, not that all planned outcomes are reported in the record. The interpretation should remain limited to the design and the data entries that are explicitly available.
In general, confidence intervals convey a range of values compatible with an estimate under the assumptions of the analysis. No confidence interval for the MTD or a treatment effect is reported, so precision cannot be assessed from such an interval. The central limitation is not an imprecise reported estimate, but the lack of a reported primary-endpoint result.
9. Statistical Methods Explained
The methods described in the registry are tailored to dose finding. The primary outcome is the MTD of metronomic oral topotecan, assessed during the first cycle of treatment, days 1-28. The record specifies a standard 3+3 cohort design based on DLTs. It does not report a statistical analysis plan, a model-based dose-finding procedure, or a final dose-selection result.
How does the described escalation rule work?
As general statistical context, a cohort dose-escalation design uses observed toxicity in participants at one dose to guide whether to expose a later cohort to a higher dose. Here, the registry states that 3 subjects are enrolled at a dose level initially. If no DLT is observed, the design proceeds to the next higher dose. If at least 2 experience a DLT, escalation stops. If 1 of 3 experiences a DLT, 3 additional subjects are enrolled at the same dose level. These rules define decision points; they do not by themselves reveal the decisions actually made in this study.
The approach is rule-based rather than a comparison of randomized arms. Dose level is the organizing feature of the cohorts, and the goal is to assess tolerability according to the stated DLT rules. In general, such designs use relatively small cohorts at each level and can be sensitive to the particular toxicity observations in those cohorts. They should not be interpreted as estimating a precise dose-toxicity curve unless the analysis explicitly provides such a model. No dose-toxicity model is reported in the registry record.
What does the DLT window contribute?
The stated assessment window is the first treatment cycle, days 1-28. In general, a defined observation window makes the toxicity criterion interpretable by specifying when an event counts toward a dose-escalation decision. It also limits the scope of that endpoint: a DLT outcome assessed during the first cycle does not, on its own, describe all later tolerability or all adverse events over the full course of treatment. The registry does not report the detailed DLT definition, the grading criteria, or how events outside that window were handled.
The record does not provide the DLT result at each dose level. It lists DLT information for several dose groups, but the last statement is incomplete. The separate adverse-event group data cannot fill this gap because the registry does not identify those entries as the DLT counts used in the escalation decisions. Without complete DLT outcomes and the final decision, the MTD cannot be reconstructed from the available record.
What can the safety counts tell readers?
In general, counts of participants affected by an event are descriptive summaries. Their interpretation depends on the event definition, observation period, analysis population, and completeness of follow-up. The registry lists participants at risk and participants affected in each dose-level safety group, along with percentages for serious adverse-event terms. These entries can show what the record reports for the listed groups, but they do not supply a randomized contrast or an adjustment for differences between dose cohorts.
Over-dispersion is a general issue when counts vary more than a simple statistical model would expect. In event-count analyses, it can make standard errors too small if ignored and can therefore make uncertainty look narrower than it is. No event-count model or estimate of over-dispersion is reported in the registry record, and no inferential analysis of the safety counts is reported. This concept is relevant as general statistical context, not as a claim that over-dispersion occurred in this study.
Why is there no comparative effect estimate?
The registry identifies a single-group model and no allocation scheme. In general, a comparative effect estimate requires a defined comparison and an analysis that relates outcomes between groups. Dose cohorts in a dose-escalation study are not automatically suitable controls for one another: they are encountered sequentially under a toxicity-driven design and may differ in enrollment or other characteristics. The registry explicitly notes that the arms are not a like-for-like control comparison. No adjusted comparison is reported.
The record lists zero analyses. It does not report a confidence interval, p-value, or fitted model for the primary endpoint. A confidence interval, in general, communicates uncertainty around an estimated quantity under an analysis model; without a reported estimate and interval, the precision of an MTD or treatment-effect estimate cannot be assessed. This does not mean the endpoint was measured without uncertainty; it means the registry record does not present an inferential result for it.
What is not specified?
The registry does not report the final MTD, the full DLT distribution, a complete description of the DLT criteria, or the final dose-escalation decision. It also does not report the statistical analysis plan, interim analyses, missing-data procedures, analysis populations, or multiplicity adjustment. Sample-size justification is not reported in the registry record. The planned 3+3 decision rules are available, but they should not be mistaken for a complete statistical methods account. Any broader methodological explanation in this discussion is general context rather than a description of unreported study procedures.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists DLTs as a secondary outcome and best overall response as another secondary outcome, alongside the primary MTD endpoint. It does not report results for the secondary outcomes. It also does not report a multiplicity strategy, such as adjustment of significance thresholds or a hierarchy for interpreting multiple outcomes. Because the record reports zero analyses and no inferential results, there are no posted p-values or confidence intervals for which a multiplicity adjustment can be evaluated.
In general, multiplicity matters when several outcomes or comparisons are tested because the chance of a false-positive finding can increase across repeated tests. A prespecified testing strategy can clarify which results are confirmatory and how uncertainty should be interpreted. The registry record does not report whether such a strategy was specified or used. It would be inappropriate to infer one from the outcome list alone.
Missing-data methods and analysis populations are also not reported in the registry record. The record does not state how missing DLT observations, incomplete follow-up, or unavailable response assessments were handled. It does not identify an intention-to-treat, safety, or per-protocol analysis population for the posted outcomes. The incomplete dose-level population statement further limits understanding of which participants contributed to the MTD assessment.
In general, conclusions can depend on how participants with missing outcomes are handled and on which population is analyzed. Those choices should be described to assess potential bias and to understand the meaning of an estimate. No such procedures or population definitions are reported. The enrollment figure and safety-group entries do not, by themselves, establish the analysis population for each endpoint.
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 |
|---|---|---|---|
| Oral Topotecan 0.25 mg Daily | 1 | 3 | 33.3% |
| Oral Topotecan 0.50 mg Daily | 0 | 4 | 0.0% |
| Oral Topotecan 0.75 mg Daily | 0 | 3 | 0.0% |
| Oral Topotecan 1.0 mg Daily | 7 | 14 | 50.0% |
| Oral Topotecan 1.25 mg Daily | 1 | 2 | 50.0% |
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 |
|---|---|
| Oral Topotecan 0.25 mg Daily | 3 of 3 |
| Oral Topotecan 0.50 mg Daily | 4 of 4 |
| Oral Topotecan 0.75 mg Daily | 3 of 3 |
| Oral Topotecan 1.0 mg Daily | 13 of 14 |
| Oral Topotecan 1.25 mg Daily | 2 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Oral Topotecan 0.25 mg Daily | Oral Topotecan 0.50 mg Daily | Oral Topotecan 0.75 mg Daily | Oral Topotecan 1.0 mg Daily | Oral Topotecan 1.25 mg Daily |
|---|---|---|---|---|---|---|
| Dehydration | Metabolism and nutrition disorders | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 1 of 2 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
| Anemia | Blood and lymphatic system disorders | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
| Ascites | Gastrointestinal disorders | 1 of 3 | 0 of 4 | 0 of 3 | 0 of 14 | 0 of 2 |
| Intestinal obstruction | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
| Nausea | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 0 of 3 | 0 of 14 | 1 of 2 |
| Small intestinal obstruction | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
| Asthenia | General disorders | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
| Infection | Infections and infestations | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
| Decreased appetite | Metabolism and nutrition disorders | 0 of 3 | 0 of 4 | 0 of 3 | 1 of 14 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Oral Topotecan 0.25 mg Daily | Oral Topotecan 0.50 mg Daily | Oral Topotecan 0.75 mg Daily | Oral Topotecan 1.0 mg Daily | Oral Topotecan 1.25 mg Daily |
|---|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | 2 of 3 | 2 of 4 | 1 of 3 | 8 of 14 | 0 of 2 |
| Fatigue | General disorders | 2 of 3 | 3 of 4 | 1 of 3 | 7 of 14 | 0 of 2 |
| Nausea | Gastrointestinal disorders | 2 of 3 | 1 of 4 | 0 of 3 | 6 of 14 | 1 of 2 |
| Vomiting | Gastrointestinal disorders | 1 of 3 | 0 of 4 | 0 of 3 | 7 of 14 | 1 of 2 |
| Constipation | Gastrointestinal disorders | 0 of 3 | 2 of 4 | 0 of 3 | 6 of 14 | 0 of 2 |
| Abdominal pain | Gastrointestinal disorders | 0 of 3 | 1 of 4 | 0 of 3 | 6 of 14 | 0 of 2 |
| Diarrhea | Gastrointestinal disorders | 1 of 3 | 1 of 4 | 0 of 3 | 3 of 14 | 1 of 2 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 3 | 1 of 4 | 0 of 3 | 3 of 14 | 0 of 2 |
| Abdominal pain upper | Gastrointestinal disorders | 2 of 3 | 0 of 4 | 0 of 3 | 2 of 14 | 0 of 2 |
| Decreased appetite | Metabolism and nutrition disorders | 0 of 3 | 0 of 4 | 0 of 3 | 4 of 14 | 0 of 2 |
Serious events with the highest arm-level rate: nausea (50.0%), dehydration (50.0%), hypokalemia (50.0%), ascites (33.3%), dyspnea (33.3%).
The safety entries list, for each oral topotecan dose group, the number at risk and the number affected for serious adverse events and for other adverse events. For serious adverse events, the listed at-risk and affected counts are 3 and 1 at 0.25 mg daily, 4 and 0 at 0.50 mg daily, 3 and 0 at 0.75 mg daily, 14 and 7 at 1.0 mg daily, and 2 and 1 at 1.25 mg daily. These are descriptive registry entries, not results from randomized comparisons.
The serious-event terms listed with percentages are nausea, 50.0%; dehydration, 50.0%; hypokalemia, 50.0%; ascites, 33.3%; dyspnea, 33.3%; and anemia, 7.1%. The registry record does not state the denominator or event-counting rule for each term in the list. The percentages should therefore be read as posted, without attempting to infer participant counts or denominators from them.
In general, an adverse-event percentage is meaningful only with its population, observation period, and event definition. The record does not report event timing, duration, severity grades, treatment relatedness, or a detailed safety-analysis population. Differences across dose groups should not be treated as causal dose effects, because the study is single-group and the groups are not like-for-like controls. These figures describe recorded safety entries; they do not, by themselves, establish the full safety profile.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study was designed to evaluate oral topotecan across dose levels in a dose-finding setting, with the MTD as the primary endpoint and DLTs during the first treatment cycle guiding escalation. It also records safety counts for several dose groups and names best overall response as a secondary outcome. Those details describe the study’s purpose and some reported safety data.
The record does not report the final MTD, a complete DLT result by dose, or the results for best overall response. It therefore does not establish which dose was selected under the stated escalation rules, nor does it provide an estimate of effectiveness. A completed registry status does not substitute for missing outcome results. The absence of a result in the record must not be interpreted as a finding of no toxicity, no response, or no effect.
Because there is no randomized comparator, the record cannot support a causal comparison of topotecan with another treatment. Nor should dose-level groups be treated as equivalent control groups. In general, a single-group dose-escalation study is directed toward dose and tolerability questions, while a controlled comparative study is needed to estimate differences between treatment strategies under a specified design.
The safety entries indicate that the registry records serious adverse events in some dose groups and lists several serious-event terms with percentages. They do not provide enough detail to characterize event timing, duration, severity, or relationship to treatment. No confidence intervals or inferential comparisons are reported, so the precision of these safety summaries cannot be assessed from the registry.
The most defensible interpretation is therefore limited: the record documents a completed early-phase dose-escalation study and its stated methods, but does not post the primary endpoint result or a complete account of the outcomes. The study’s stated aims should not be confused with results, and the available safety summaries should not be taken as a comprehensive or comparative assessment.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between a dose-finding design and a comparative treatment trial. The registry describes a single-group study using a 3+3 escalation rule, where DLT observations at a dose level direct the next step. The aim is to identify an MTD under the study’s specified toxicity criteria, rather than to estimate a treatment effect against a control group.
That distinction affects how the dose groups should be read. In general, dose cohorts in escalation research are formed as part of a sequential decision process. The record itself cautions that the arms are not a like-for-like control comparison. A numerical difference between cohort summaries would not automatically represent a dose effect, and no comparative analysis is reported.
The case also illustrates why a methods description is not a substitute for an outcome result. The registry gives the MTD endpoint and escalation rules, but does not report the final MTD or complete DLT results. Without those results, readers cannot assess the observed decision path or how much uncertainty surrounds the dose selection. The outcome is not recoverable merely from the listed safety counts, which are presented separately and do not identify DLT outcomes.
Finally, the record’s safety entries show the value and limits of descriptive reporting. Counts and serious-event percentages communicate what was entered for the listed groups, but interpretation also depends on event definitions, denominators, follow-up, analysis populations, and uncertainty. Those details are not fully reported. Statistically, the record therefore supports a clear account of the design and selected safety summaries, while leaving the key dose-finding result and several interpretive details unavailable.
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 important trial-specific limitation for interpreting the registry is that the primary endpoint is not accompanied by a reported result. The MTD is named, and the 3+3 decision rules are described, but the final MTD and complete DLT outcomes by dose are not reported. The population description ends with 2 subjects who received topotecan without identifying the dose in the available text. This prevents a complete reconstruction of the primary endpoint from the registry record.
The safety groups list different at-risk counts by dose from some of the DLT population counts described in the primary-outcome text. The registry does not explain whether these entries correspond to different analysis populations, different outcome definitions, or another distinction. They should not be combined or treated as interchangeable. The record also does not report the denominator or counting rule for each serious-event term percentage.
More detailed eligibility criteria, participant characteristics, DLT definitions, event grading, duration of follow-up, and treatment-relatedness assessments are not reported in the registry record. Without these details, readers cannot fully assess the context for the observed safety entries or how representative the study population may be.
The registry does not report statistical analysis methods beyond the described escalation design, a sample-size justification, missing-data procedures, analysis populations, multiplicity handling, or interim analyses. It lists zero analyses and does not post a final primary result or secondary-outcome results. These omissions limit assessment of both the outcome evidence and its precision. The single-group design also means there is no randomized comparator for causal treatment-effect interpretation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Oral Topotecan to Treat Recurrent or Persistent Solid Tumors — NCT00382733.
- Registry record last updated 2013-07-22; status last verified 2013-07.
- Results first posted 2013-07-18.
- 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
NCT00382733 is a completed Phase I/Phase II, single-group study of oral topotecan for recurrent or persistent solid tumors. The registry lists enrollment as 26, no masking, and no allocation scheme. The primary endpoint is the MTD of single-agent metronomic oral topotecan, assessed during the first cycle, days 1-28, using a 3+3 DLT-based dose-escalation design.
The record lists DLTs and best overall response as secondary outcomes and includes dose-group safety counts and serious adverse-event terms with percentages. It does not report a final MTD, complete DLT results, or secondary-outcome results. The available safety summaries are descriptive and do not constitute randomized comparisons. Missing details include the full primary-endpoint result, analysis populations, missing-data procedures, and multiplicity methods.