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 Pemetrexed and Carboplatin in Treating Patients With Recurrent Ovarian, Primary Peritoneal, or Fallopian Tube Cancer (NCT01001910), a na phase 2 study. The primary outcome is Overall Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors and by Rustin's Criteria (RECIST).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01001910 |
| Phase | Phase 2 |
| Condition | Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Albert Einstein College of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent ovarian, primary peritoneal, or fallopian tube carcinoma. The official title describes the disease as platinum sensitive. The registry lists enrollment of 22.
Intervention
Pemetrexed disodium and carboplatin, administered together. The treatment description specifies intravenous administration on day 1, with treatment repeating every 21 days for at least 6 courses unless progression or unacceptable toxicity occurs.
Comparator
There is no separate comparator group in this single-group study. The registry lists allocation as not applicable and masking as none, so the treatment group is not compared with a concurrent control arm.
Primary question
The primary endpoint is overall objective response rate, based on RECIST V1.1 or Rustin's criteria as appropriate. The registry record does not report the number or proportion of participants who achieved a response.
The statistical question is whether objective responses were observed under the study's specified response definitions among the population included in the primary endpoint analysis. RECIST classifies response using changes in target lesions on CT scans. Rustin's criteria define response using a reduction in CA125 from a pretreatment sample, with confirmation and maintenance for at least 28 days. These are alternative assessment approaches within the endpoint description, not a randomized comparison between assessment methods.
Because the design is single-group, the record cannot estimate a randomized contrast between this regimen and another treatment. A response count, if reported, would describe outcomes in the treated study group; it would not by itself establish what would have occurred under a different treatment or without treatment. The registry record does not provide the primary response count, response proportion, or an uncertainty interval, so the magnitude and precision of the observed response result cannot be evaluated from the posted results.
3. Trial Design
This is a phase II trial with a single-group model. The registry lists enrollment of 22 and identifies Albert Einstein College of Medicine as sponsor. The study status is completed. Its recorded start is 2008-07 and its primary completion is 2015-01. Masking is listed as none, and allocation is not applicable. There is no concurrent control arm described.
The treatment group received pemetrexed disodium and carboplatin. The registry describes both drugs as intravenous, administered on day 1: pemetrexed disodium over 8-15 minutes and carboplatin over 30-60 minutes. Treatment was to repeat every 21 days for at least 6 courses in the absence of disease progression or unacceptable toxicity. Laboratory biomarker analysis is also listed as an intervention, but further details of that activity are not reported in the registry record.
The primary endpoint is overall objective response rate, assessed according to RECIST V1.1 or Rustin's criteria as appropriate, with a timeframe of 4.5 years. Secondary outcomes listed are incidence of toxicities, overall survival, and progression-free interval. The registry identifies four outcome measures, but no analysis is listed. A statistical analysis plan, including details of planned estimation or hypothesis testing, is not reported in the registry record.
The primary endpoint population description says that patients were included if they completed 3 cycles of therapy, while also describing the population as intent to treat. The record's statement about the population ends without a complete description. Consequently, the exact analysis population and how participants who did not complete 3 cycles were handled are not fully specified. These details matter when interpreting an endpoint that may be calculated from fewer participants than the enrollment figure.
The sample-size assumptions describe a planning illustration using equal allocation between two arms, whereas the trial itself is recorded as single-group. Such an illustration is not a description of this trial's allocation or observed results. A trial-specific sample-size justification is 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 | Treatment (Pemetrexed Disodium, Carboplatin) |
|---|---|
| STARTED | 28 |
| COMPLETED | 22 |
| NOT COMPLETED | 6 |
Reasons for not completing this period:
| Reason | Treatment (Pemetrexed Disodium, Carboplatin) |
|---|---|
| Did not meet protocol defined eligibility s7.1.1 for primary outcome | 6 |
Analysis population for the primary outcome, as defined in the registry: Intent to Treat Patients were included for primary endpoint analysis of ORR if they completed 3 cycles of therapy. Response was categorized as complete response (CR), partial response (PR), stable disease (SD) or progressive disease (PD) by RECIST criteria. All patients had
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: This is a open-labeled, phase II trial evaluating the combination of intravenous pemetrexed and carboplatin in patients with confirmed recurrent epithelial ovarian, primary peritoneal, or fallopian tube carcinoma. There is no comparison group.
| Characteristic | Unit | Treatment (Pemetrexed Disodium, Carboplatin) |
|---|---|---|
| Participants analysed | 28 | |
| Age, Continuous | years | 64 (46 to 86) |
| Sex: Female, Male: Female | Participants | 28 |
| Sex: Female, Male: Male | Participants | 0 |
| Race/Ethnicity, Customized: Race – White | Participants | 21 |
| Race/Ethnicity, Customized: Race – African American | Participants | 1 |
| Race/Ethnicity, Customized: Race – Hispanic | Participants | 3 |
| Race/Ethnicity, Customized: Race – Asian | Participants | 3 |
| Site of Disease: Ovary | Participants | 23 |
| Site of Disease: Primary Peritoneal | Participants | 4 |
| Site of Disease: Fallopian Tube | Participants | 1 |
| FIGO Stage at Diagnosis: Stages I/II | Participants | 3 |
| FIGO Stage at Diagnosis: Stages III/IV | Participants | 25 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors and by Rustin's Criteria (RECIST), measured over 4.5 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
Overall objective response rate is the proportion of participants meeting the definition of complete or partial response. For RECIST V1.1, complete response means disappearance of all target lesions. Partial response requires at least a 30% decrease in the sum of target-lesion diameters. Progressive disease is defined by at least a 20% increase in that sum, using the smallest sum on study as the reference. Stable disease is neither sufficient shrinkage for partial response nor sufficient increase for progressive disease. The endpoint description defines overall response as complete response plus partial response.
Under Rustin's criteria, a CA125 response requires at least a 50% reduction from a pretreatment sample, confirmed and maintained for at least 28 days. The registry says RECIST or Rustin's criteria were used as appropriate; it does not explain which participants were assessed by each approach or how results across approaches were combined.
Response rate is used to describe the frequency of specified tumor-response outcomes in the analyzed group. It is a binary outcome at the participant level: a participant either meets the response definition or does not. It does not, on its own, describe how long a response lasts, survival, or the comparative effect of treatment versus a control. The registry record does not report the observed response count or rate, so the endpoint's result and its precision cannot be assessed from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically defined primary endpoint and a single treated group, but it does not report the primary endpoint result. In particular, there is no posted count of complete or partial responses, no overall response proportion, and no confidence interval for that proportion. It is therefore not possible to describe the observed response frequency or how precisely it was estimated. The absence of a reported response result should not be interpreted as evidence that no responses occurred.
The design limits what a reported response rate could establish. With no concurrent comparator arm, the result would describe the treated group under the registry's response criteria. It would not provide a randomized estimate of the difference between this regimen and another intervention. Differences in patient characteristics, assessment, or time could not be separated from treatment effects through a within-trial randomized comparison because no such comparison is recorded.
The enrollment figure is 22, but the endpoint population description says that patients were included in the primary endpoint analysis if they completed 3 cycles. The record also calls this population intent to treat, and does not fully explain the inclusion rule. Thus enrollment alone cannot establish how many participants contributed to the primary endpoint calculation. The number analyzed and handling of participants without the required follow-up are not reported in the registry record.
The safety information lists 22 at risk for serious adverse events, 11 affected by serious adverse events, 22 at risk for other adverse events, and 0 affected in that other category; deaths are listed as 0. The serious-event terms include hematologic at 36.4% and non-hematologic at 13.6%. The registry does not provide enough detail to determine from these percentages alone the precise event-counting method, the denominator used for each term, or the severity breakdown within those categories. These figures are descriptive safety data, not a randomized safety comparison.
General statistical interpretation: an estimate is more informative when accompanied by an interval showing its uncertainty and by a clear definition of the analysis population. A confidence interval conveys a range of values reasonably compatible with the observed data under the method used; it does not remove uncertainty or account automatically for bias. No confidence interval or inferential analysis for the primary response endpoint is reported in the registry record.
The secondary outcomes include toxicity incidence, overall survival, and progression-free interval. Their names indicate outcomes of interest, but no numerical results or analysis details for them are included in the registry record. The record therefore does not support conclusions about their observed values or precision.
9. Statistical Methods Explained
General statistical explanation: a clinical endpoint translates a study question into an outcome that can be recorded for participants. A binary endpoint, such as whether a participant meets a response definition, is commonly summarized by the number meeting the definition and the proportion of the analysis population meeting it. The response definition must be applied consistently, and the population included in the calculation must be clear. In this trial record, the primary response definition is described, but the resulting count and proportion are not reported.
What does a confidence interval add?
General statistical explanation: a confidence interval expresses the uncertainty around an estimate arising from sampling variation under the method's assumptions. For a response proportion, a narrow interval would indicate greater statistical precision than a wide interval, while neither interval would establish that a treatment caused the observed outcomes. Interpretation also depends on whether the enrolled participants and analysis population are representative of the question of interest. No confidence interval for the primary response endpoint is reported in the registry record, so its precision cannot be evaluated.
Why does the analysis population matter?
The registry describes the primary endpoint population as intent to treat, but also says participants were included if they completed 3 cycles of therapy. The record does not explain how those statements were reconciled or how people who did not complete 3 cycles were handled. General statistical explanation: excluding participants based on events occurring after treatment begins can change the population represented by an estimate. If the chance of completing treatment is related to health status, tolerability, or disease course, a completer-based estimate may not describe all enrolled participants. This is a methodological consideration, not a claim about what happened in this trial.
General statistical explanation: a missing outcome is different from an outcome that is observed and classified as no response. Statistical analysis needs to state whether missing assessments were treated as nonresponse, excluded, imputed, or handled another way. Each approach has assumptions and can affect interpretation. The missing-data method for the primary endpoint is not reported in the registry record, nor is the number of participants with missing response assessments.
What is the role of a comparator?
This study is recorded as single-group, with allocation not applicable and masking none. General statistical explanation: random allocation to a comparator group can support a causal contrast by balancing, on average, measured and unmeasured characteristics at baseline. A single-group study instead describes outcomes in the group receiving the regimen. Without a concurrent randomized comparator, differences from historical expectations can be influenced by differences in eligibility, follow-up, assessment, and patient characteristics. The registry record does not identify a control group or a statistical adjustment intended to create a controlled comparison.
How should the response criteria be understood?
RECIST V1.1 classifies response using target-lesion measurements: complete response is disappearance of all target lesions; partial response requires at least a 30% decrease in their summed diameters; progressive disease requires at least a 20% increase relative to the smallest sum on study; and stable disease meets neither the shrinkage nor increase threshold. Overall response is defined as complete or partial response. Rustin's criteria use CA125 reduction: a response requires at least a 50% decrease from a pretreatment sample, confirmed and maintained for at least 28 days. These definitions create explicit rules for classification, but the record does not state how assessments were assigned between the criteria or report the classifications observed.
General statistical explanation: when more than one response assessment approach is allowed, transparent reporting should clarify which participants were evaluated by each approach and how the endpoint was assembled. Without that detail, readers cannot fully assess whether the response categories were applied comparably across the analysis population. This information is not reported in the registry record.
What does over-dispersion mean for count data?
General statistical explanation: over-dispersion occurs when variation in count outcomes is greater than a statistical model's assumptions would predict. If present, a model that assumes too little variation can produce uncertainty estimates that are too small. This concept is relevant to some count-based analyses, but the primary endpoint is described as a participant response count and no statistical model or dispersion estimate is reported. There is no basis in the registry record to say that over-dispersion was assessed or affected the trial analysis.
Can the planning assumptions be read as the trial's analysis plan?
No. The record's sample-size assumptions describe an illustration using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They describe planning for a fraction of an observed effect and specify that non-time-to-event calculations are expressed as participants per arm. However, this trial is recorded as a single-group study, and no specific sample-size justification or trial-specific statistical plan is reported in the registry record. Those generic assumptions should not be treated as the trial's design or as evidence of observed power. Any sample-size illustration is a planning exercise, not a measure of the study's observed ability to detect an effect.
The registry lists no analyses. It does not report a hypothesis test, p-value, confidence interval, adjusted estimate, or details of how the primary endpoint was calculated. The record therefore supports a description of the design and endpoint definitions, but not an independent reconstruction of the primary efficacy analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists overall objective response rate as the primary endpoint and identifies toxicity incidence, overall survival, and progression-free interval as secondary outcomes. It does not report a plan for controlling multiplicity across these outcomes or across any additional analyses. General statistical explanation: when multiple outcomes or repeated tests are considered, the chance of at least one apparently unusual result can increase. A prespecified strategy can clarify which outcomes are confirmatory and how findings should be interpreted. No such strategy is reported in the registry record, so it is not possible to determine whether any adjustment was planned or used.
Missing-data methods are not reported in the registry record. The primary population description says that patients were included if they completed 3 cycles, while also using the term intent to treat. The record does not give a complete explanation of eligibility for the primary endpoint analysis, the number with evaluable response assessments, or the handling of participants with incomplete follow-up. These omissions matter because the response proportion depends on both the response classification and the denominator used.
The registry does not list any statistical analyses, and it does not report interim-analysis procedures, stopping rules, or a detailed analysis plan. These items should be treated as not reported in the registry record rather than assumed to have been absent from all trial conduct. The posted information does not establish how the secondary endpoints were analyzed or whether the analyses were prespecified.
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 |
|---|---|---|---|
| Treatment (Pemetrexed Disodium, Carboplatin) | 11 | 22 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment (Pemetrexed Disodium, Carboplatin) | 0 of 22 |
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 |
|---|---|
| Treatment (Pemetrexed Disodium, Carboplatin) | 0 of 22 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Pemetrexed Disodium, Carboplatin) |
|---|---|---|
| Hematologic | Blood and lymphatic system disorders | 8 of 22 |
| Non-Hematologic | General disorders | 3 of 22 |
Serious events with the highest arm-level rate: hematologic (36.4%), non-hematologic (13.6%).
The registry lists 22 participants at risk for serious adverse events and 11 affected. For other adverse events, it lists 22 at risk and 0 affected. Deaths are listed as 0. These are counts associated with the treatment group; the record does not provide a separate control group for comparison.
The serious adverse event terms listed include hematologic at 36.4% and non-hematologic at 13.6%. The registry does not specify in this summary whether these percentages refer to participants, events, or another denominator, nor does it provide further detail about the event categories. They should therefore be read as the reported percentages attached to those terms, not recalculated or treated as a fully defined comparison.
General statistical explanation: safety counts are descriptive and depend on the observation period, event definitions, and whether repeated events in one participant are counted once or multiple times. A count of participants affected does not show the total number of events. The registry record does not provide those additional counting details. The reported figures describe the recorded safety experience and do not, by themselves, estimate comparative risk or establish causation.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a completed phase II, single-group study of pemetrexed disodium and carboplatin in recurrent ovarian, primary peritoneal, or fallopian tube cancer. It specifies a primary response endpoint and gives RECIST V1.1 and Rustin's criteria for classifying response. It also reports safety counts and lists secondary outcomes. These details clarify what the study set out to assess and the treatment schedule used.
The record does not report the observed overall response rate or its uncertainty. It therefore cannot support a statement about how often participants met the response definition or how precisely that rate was estimated. This absence is not the same as a reported result of zero response. The record also does not provide results for the listed secondary outcomes, including overall survival and progression-free interval.
Even if a response result were available, the single-group design would describe outcomes in the treated participants rather than establish a randomized treatment effect. No concurrent control arm is listed. The record also leaves uncertainty about the primary analysis population, since its description refers both to intent to treat and completion of 3 cycles, without fully explaining how these terms were applied.
General statistical explanation: statistical evidence and clinical interpretation answer different questions. An endpoint result describes observations according to a defined measure; a causal comparison requires a design and analysis that address what would have happened under an alternative. A result's precision also depends on the amount and completeness of usable data. These considerations are especially relevant when a registry record does not contain the outcome estimate, an interval, or a full account of the analysis population.
The available safety figures are descriptive. The registry lists serious adverse events in 11 participants, no affected participants in the other adverse-event category, and no deaths. The serious-event term percentages are reported without enough denominator detail to establish exactly how they were calculated. These registry entries do not establish comparative safety, and they should not be extended beyond the population and reporting described in the record.
13. Why This Trial Matters Statistically
This trial illustrates why the design, endpoint definition, and results must be read together. The registry clearly identifies a phase II, single-group study and defines a response outcome using RECIST V1.1 or Rustin's criteria as appropriate. Those definitions make the intended classification rules more explicit than a general label such as tumor response, while the single-group design sets a limit on comparative interpretation.
The distinction between an endpoint's definition and its observed result is important. A detailed definition does not reveal how many participants met it. Here, the registry record reports the response rules but not the response count or proportion. It also does not report an interval describing uncertainty. Readers can therefore understand what was to count as an objective response without being able to assess the frequency or precision of the response result from the posted results.
The population wording is another statistically important feature. The record uses intent-to-treat language but also states that participants were included in primary endpoint analysis if they completed 3 cycles. Without a complete explanation of the analysis population, it is difficult to know how the enrollment figure relates to the denominator for the endpoint. This affects reproducibility and the interpretation of any response estimate.
The record also demonstrates why planning assumptions must be separated from trial design and observed evidence. Its planning illustration describes equal allocation between two arms, while the trial is recorded as single-group. A planning illustration is not evidence of a comparator, and it is not observed power. The registry does not report a trial-specific sample-size justification or a full statistical analysis plan.
These points matter for statistical reading because they identify what can be learned from the record and what remains unresolved: the regimen and endpoint definitions are documented, but the primary endpoint result, its precision, analysis population details, and comparative evidence are not reported in the registry record.
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 main limitation for interpreting efficacy is that the registry record does not report the primary response count, response proportion, or an uncertainty interval. Although the endpoint definition is detailed, readers cannot assess the observed response frequency or its statistical precision from the posted results. Numerical results for the listed secondary outcomes are also not reported in the registry record.
The study has a single-group design, with no concurrent control arm identified. This means the registry record does not provide a randomized comparison of the treatment regimen with another intervention. Any outcome described for the treated group would not, on its own, establish a treatment effect relative to an alternative.
The analysis population description is incomplete. It refers to intent-to-treat patients but also conditions inclusion in the primary endpoint analysis on completing 3 cycles. The record does not fully explain this wording, identify the number included in the endpoint analysis, or state how missing or unevaluable response assessments were handled. The assignment of participants to RECIST or Rustin assessment is also not described in detail.
For safety, the record provides counts and serious-event term percentages but does not fully specify percentage denominators, event-counting rules, or further category detail. It does not provide comparative safety data. A statistical analysis plan, multiplicity approach, trial-specific sample-size justification, and interim-analysis details are not reported in the registry record. These limitations describe what the record leaves unclear; they do not establish that the corresponding procedures were not conducted.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pemetrexed and Carboplatin in Treating Patients With Recurrent Ovarian, Primary Peritoneal, or Fallopian Tube Cancer — NCT01001910.
- Registry record last updated 2024-02-28; status last verified 2024-02.
- Results first posted 2021-10-20.
- 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.
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18. Record Summary
NCT01001910 is a completed phase II, single-group trial sponsored by Albert Einstein College of Medicine. The registry lists enrollment of 22, no masking, and allocation as not applicable. It describes pemetrexed disodium and carboplatin given intravenously on day 1, repeating every 21 days for at least 6 courses unless progression or unacceptable toxicity occurs.
The primary endpoint is overall objective response rate using RECIST V1.1 or Rustin's criteria as appropriate, with a stated timeframe of 4.5 years. Secondary outcomes include toxicity incidence, overall survival, and progression-free interval. The registry describes serious adverse events in 11 participants and lists 0 deaths.
The primary response result and its precision are not reported in the registry record. The primary analysis population description is incomplete, and the registry does not list statistical analyses or a trial-specific statistical plan. Accordingly, the record documents the study design and endpoint definitions but does not permit assessment of the observed response rate or a controlled treatment comparison.