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 Megestrol Acetate in Treating Weight Loss or Anorexia in Young Patients With Malignancies Who Are Undergoing Radiation Therapy and/or Chemotherapy (NCT01200602), a randomized phase 2 study. The primary outcome is Proportion of Patients Who Maintain Weight or Experience Weight Gain.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01200602 |
| Phase | Phase 2 |
| Condition | Anorexia, Weight Changes |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Mayo Clinic |
| Results posted | Yes |
2. Clinical Question
Population
Young patients with malignancies who had weight loss or anorexia and were undergoing radiation therapy and/or chemotherapy. The registry does not report age limits, eligibility details, or baseline characteristics.
Intervention
Oral megestrol acetate, given 1-2 times daily for 4 weeks in Arm A.
Comparator
Clinical observation for weight loss and gain for 4 weeks in Arm B. The official title uses placebo-controlled wording, but the arm description identifies clinical observation as the comparator. Masking is recorded as none.
Primary question
What proportion of patients maintain or gain weight at the end of initial treatment compared with baseline at study entry? The registry defines this as success, but does not report the outcome for the accrued patient.
The clinical question concerns a short-term weight outcome in a specific setting: young patients with malignancies receiving cancer treatment and experiencing weight loss or anorexia. The trial record describes a randomized, parallel comparison. It does not provide a reportable estimate of the proportion meeting the weight-maintenance or weight-gain definition in either arm, nor an estimate of the difference between arms.
The registry lists BMI trends, caloric intake, weight maintenance over time, and toxicity profile as secondary outcomes. Their results are not reported in the registry record. The study therefore identifies outcomes of interest but does not allow readers to evaluate the observed pattern across them. The clinical question is clear at the level of the stated endpoint; the available record does not supply the outcome information needed to answer it.
3. Trial Design
The registry identifies this as a Phase 2 randomized trial with a parallel-group model and no masking. It lists Mayo Clinic as sponsor. The trial title and brief description concern megestrol acetate for weight loss or anorexia in young patients with malignancies undergoing radiation therapy and/or chemotherapy. The record lists the conditions as anorexia and weight changes.
Arm A is described as oral megestrol acetate 1-2 times daily for 4 weeks. Arm B is described as clinical observation for weight loss and gain for 4 weeks. Although the official title includes placebo-controlled wording, the arm description names clinical observation, and masking is recorded as none. The registry does not report further details about allocation procedures, treatment adherence, co-interventions, or how clinical observation was conducted.
The registry records enrollment as 1. It gives a start date of 2011-03 and a primary completion date of 2011-11; the status is terminated. A reason for termination is not reported in the registry record. Nor does the record explain whether recruitment stopped early, how the enrollment was distributed between arms, or whether any planned protocol changes occurred.
The primary outcome was assessed at 4 weeks. Secondary outcomes listed are BMI trends, caloric intake, weight maintenance over time, and toxicity profile. The record lists 5 outcome measures, but provides no analyses. It does not report a detailed statistical analysis plan, including the planned comparison method or adjustment strategy. The available design information establishes the broad structure of the study, but not the operational details needed to judge how closely conduct followed a prespecified plan.
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 | Arm A | Arm B |
|---|---|---|
| STARTED | 0 | 0 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.
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: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.
| Characteristic | Unit | Arm A | Arm B | Total |
|---|---|---|---|---|
| Participants analysed | 0 | 0 | 0 | |
| Sex: Female, Male: Female | Participants | — | — | — |
| Sex: Female, Male: Male | Participants | — | — | — |
| Region of Enrollment: United States | participants | — | — | — |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Patients Who Maintain Weight or Experience Weight Gain, measured over 4 weeks.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the proportion of patients who maintain weight or experience weight gain. The registry defines a patient as a success if weight is maintained or increased at the end of initial treatment compared with baseline at study entry. The stated assessment timeframe is 4 weeks. The endpoint therefore turns a change in weight into a categorical outcome: success under the registry definition, or not meeting that definition.
Using a threshold-based endpoint can make a clinical question easier to interpret. Instead of focusing only on the size of a weight change, it asks how often a specified weight outcome occurs. The choice of definition matters: maintaining weight and gaining weight both count as success, while the record does not provide further detail on measurement procedures, allowable variation, or how weights were handled if a measurement was unavailable.
The endpoint is relevant to the trial’s stated focus on weight loss, but it cannot be evaluated from the posted results. The registry says that only one patient was accrued and that patient confidentiality prevents reporting the patient’s result. Consequently, neither the success proportion nor a comparison between arms is available. The registry does not report an endpoint estimate, uncertainty interval, or test of the difference. A defined endpoint is not itself evidence about the observed outcome; the result remains unavailable in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive constraint is that the registry does not disclose the primary outcome. It reports enrollment as 1 and explains that confidentiality prevents reporting the accrued patient’s result. Because the outcome is withheld, readers cannot determine whether the patient met the definition of success, estimate a proportion for either arm, or compare megestrol acetate with clinical observation. No treatment effect estimate or measure of its precision is reported.
The registry also lists 0 analyses. This means there is no posted statistical comparison to interpret. It does not establish what analyses may have been planned, whether any analysis was attempted, or why none is listed. Those details are not reported in the registry record. The trial’s randomized, parallel design describes how a comparison was intended to be structured; it does not replace the missing outcome data or provide evidence about the direction or size of an effect.
There are listed secondary outcomes—BMI trends, caloric intake, weight maintenance over time, and toxicity profile—but their results are not reported in the registry record. The absence of results means that no conclusion about those measures can be drawn from this record. Likewise, the record does not report a confidence interval or other uncertainty measure for the primary outcome. In general, uncertainty intervals help show how precisely an estimate is supported; without an estimate and interval, precision cannot be assessed.
The adverse-event entries list zero patients at risk and zero affected for serious and other events in each arm. The death entries are not reported. These fields should be read as registry entries, not as a complete account of safety experience: the record also reports an accrued patient but does not disclose that patient’s outcome because of confidentiality. The entries do not support a comparative safety estimate or a claim that no events occurred.
The registry includes assumptions for planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It says these illustrations are not the trial’s own design calculations. Since the patient-level outcome is withheld and no observed effect estimate is reportable, planning illustrations cannot supply an observed treatment result. They are not observed power and do not resolve the uncertainty created by the unavailable endpoint.
Overall, the record documents a trial design and a defined clinical outcome, but it does not provide analyzable efficacy results. The proper interpretation is limited to what is documented: the comparison was randomized and unmasked, enrollment was recorded as 1, the study status is terminated, and the principal outcome is not disclosed. Any claim about benefit, lack of benefit, or comparative safety would go beyond the posted information.
9. Statistical Methods Explained
The statistical information in the registry is sparse. It identifies a randomized parallel-group design, a categorical primary outcome based on maintaining or gaining weight, and a 4-week timeframe. It does not report an analysis method, an effect estimate, an uncertainty interval, or a statistical test. The explanations below describe general concepts that would apply to a trial of this type; they do not describe analyses reported for this study.
What does the primary endpoint represent?
In general, a proportion describes the share of an analyzed group meeting a defined outcome. Here, the registry’s success definition combines maintaining weight and gaining weight by the end of initial treatment compared with baseline. A proportion would require an outcome classification for the patients included in the analysis and a clearly defined denominator. The registry withholds the accrued patient’s result, so the success classification and a reportable proportion are unavailable. The listed enrollment alone does not establish an outcome value.
A comparison between randomized arms could be expressed in several ways, such as a difference in proportions or a ratio of proportions. These measures answer related but distinct questions and have different interpretations. The registry does not identify which measure was planned or report any comparison. It would therefore be inappropriate to choose one as though it were the trial’s reported analysis or to calculate one from information that is not disclosed.
Why do estimates need uncertainty measures?
In general, an estimate describes the observed data, while a confidence interval conveys the range of values reasonably compatible with those data under the method’s assumptions. A narrow interval indicates greater precision than a wide interval, although interpretation also depends on the study design and analysis. A p-value, when used, addresses compatibility of data with a specified null hypothesis under a statistical model; it does not measure the size or clinical importance of an effect. This registry record reports no estimate, confidence interval, or p-value for the primary endpoint, so neither precision nor statistical evidence can be assessed.
With very limited enrollment, estimates would generally be highly sensitive to the outcome of each included patient. That is a general property of sparse data, not a calculated result for this trial. In this record the more fundamental issue is that the outcome itself is withheld. Even an otherwise suitable analysis method cannot recover a result that is not reported.
What do the planning assumptions mean?
The registry describes planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. These are assumptions for planning illustrations, not evidence that the trial was designed with those parameters. The registry explicitly distinguishes the illustrations from the trial’s own design. It does not report the trial’s sample-size justification, a prespecified target, or its statistical plan.
In general, planning calculations explore how much information a future study might need under stated assumptions about the effect to detect and the analysis. They depend on those assumptions; changing the assumed effect or method changes the resulting planning estimate. Such calculations are not observed power. Observed power is not a substitute for an effect estimate and uncertainty interval, and planning illustrations do not demonstrate that a completed trial detected an effect. No planning result should be interpreted as the outcome of this trial.
The record’s assumptions specify equal allocation for the planning exercise, but do not establish that the actual enrollment was equally allocated. The enrollment recorded for the trial cannot be translated into arm-specific denominators because these are not reported. The record also does not provide an observed effect that can be interpreted, given the confidentiality statement. Therefore, the planning description cannot be used to reconstruct a group comparison.
Why can sparse data challenge standard methods?
In general, statistical approximations work best when the data contain enough information for the approximation to be credible. With a binary outcome and sparse observations, some standard approximations may be unreliable, and exact or other methods may be considered depending on the design and estimand. This is a general methodological point, not a description of an analysis conducted. The registry does not report a method, and the outcome cannot be evaluated from the posted record.
For outcomes measured repeatedly over time, observations from the same person are usually related rather than independent. In general, an analysis of trends should account for that dependence and specify how timing and incomplete measurements are handled. The registry lists BMI trends and weight maintenance over time, but does not report their results or the methods used to analyze them. No conclusions about trajectories or within-person change can be drawn.
What can the posted analysis entries establish?
The registry lists 0 analyses. That entry indicates that no analysis is posted in the record; it is not evidence that a particular statistical procedure was used or that a specific hypothesis was tested. The record does not report prespecified analysis populations, covariate adjustment, handling of protocol deviations, or a method for missing measurements. Those matters are not reported in the registry record. Without those details and without the endpoint result, a statistical reanalysis is not possible.
In short, the design and endpoint permit a general description of the intended comparison, but the registry lacks the data and methods needed for statistical interpretation of an effect. General explanations of proportions, uncertainty, planning, and sparse-data methods should not be mistaken for findings from this trial.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome and secondary outcomes involving BMI trends, caloric intake, weight maintenance over time, and toxicity profile. It records 5 outcome measures, but does not report results for the secondary outcomes or analyses. A multiplicity strategy—such as a hierarchy of outcomes, adjustment for multiple testing, or identification of outcomes as exploratory—is not reported in the registry record. Without that information and without posted analyses, the possibility of multiple comparisons cannot be evaluated in relation to the study’s conclusions.
The primary endpoint has a stated success definition and a 4-week timeframe. The record does not report whether alternative definitions, additional time points, or subgroup comparisons were prespecified. It also does not report a statistical analysis plan that would clarify how multiple outcomes or comparisons were to be handled. The absence of this information should not be filled by assuming a particular protocol.
Missing-data methods and analysis populations are not reported in the registry record. There is no description of how unavailable weights, incomplete follow-up, treatment discontinuation, or protocol deviations were handled. The confidentiality statement is distinct from an ordinary missing-data analysis: it says the patient’s result is not reported, not how an analysis treated an unobserved measurement.
The registry reports 0 analyses and does not provide an outcome estimate. As a result, there is no basis to assess whether all randomized patients were included in an analysis, whether analyses followed an intention-to-treat principle, or whether any sensitivity analysis was conducted. These are unresolved reporting limitations, not evidence that a particular approach was or was not used.
11. What the Result Does, and Does Not, Mean
The record documents a randomized, unmasked comparison of oral megestrol acetate with clinical observation over 4 weeks, with weight maintenance or gain specified as the primary outcome. It also records enrollment as 1 and status as terminated. These registry entries describe the trial record, but they do not reveal the primary outcome or the comparative result.
The registry explicitly states that the accrued patient’s result is withheld to protect confidentiality. Consequently, the posted information cannot show whether the success definition was met, how the arms compared, or how precise any effect estimate would be. It does not support a conclusion that megestrol acetate improved weight, failed to improve weight, or differed from observation. A missing result is not equivalent to a finding of no effect.
The listed secondary outcomes likewise have no reported results, and the registry lists 0 analyses. The adverse-event entries show zero at-risk and affected values in the arm records, while death entries are not reported. Those registry entries do not establish a complete comparative safety profile, particularly when the record does not provide a reportable outcome for the accrued patient.
Planning illustrations, if considered alongside the registry, must remain planning exercises based on assumptions rather than observations from a completed comparison. They are not observed power and cannot compensate for the withheld outcome. The registry also does not explain the reason for termination or report whether its design assumptions were met.
The appropriate conclusion is narrow: the study asked a defined clinical question, but the posted record does not contain the outcome information required to answer it. No treatment recommendation or claim about efficacy or safety follows from these data. The record is informative about the planned structure and the limits of reporting, not about the direction or size of a treatment effect.
12. Why This Trial Matters Statistically
This trial illustrates an important distinction between a study’s design and the evidence available for interpretation. Randomization and a clearly stated endpoint can create a framework for a comparison, but they do not by themselves provide an estimate. Without a reportable endpoint, readers cannot evaluate either the observed outcome or its uncertainty.
Confidentiality can constrain reporting when enrollment is very limited. In this record, the registry states that the accrued patient’s outcome cannot be disclosed. That protects privacy, but it also prevents readers from determining the result for the primary endpoint. The consequence is not merely a wide interval or an imprecise comparison; the essential outcome information is unavailable in the registry record.
The trial also highlights why registry entries should be interpreted carefully. A title describes a placebo-controlled trial, while the listed comparator arm is clinical observation and masking is recorded as none. The record does not explain this wording difference. Readers should distinguish the official title from the arm descriptions rather than assume details not documented in the record.
Finally, planning calculations and observed results answer different questions. Planning illustrations can examine assumptions for a future design, but they cannot replace a reported comparison or be treated as observed power. Where outcome data and analyses are not available, the statistical lesson is one of evidentiary limits: the intended question may be clinically relevant, yet the public record may not permit a quantitative answer.
13. 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 is the absence of a reportable primary outcome. The registry states that only one patient was accrued and that confidentiality prevents disclosure of that patient’s result. This prevents calculation or interpretation of a success proportion and precludes a comparison between arms. No confidence interval, p-value, or effect estimate is reported.
Enrollment is recorded as 1, but arm-specific enrollment and outcome denominators are not reported. The registry does not explain the reason for termination, and the study status alone cannot establish why it ended or whether the stated design was completed as intended. Information about eligibility criteria, baseline characteristics, adherence, and the conduct of clinical observation is also not reported in the registry record.
The official title uses placebo-controlled wording, whereas the comparator arm is described as clinical observation; masking is recorded as none. The record does not clarify the relationship between these descriptions. The statistical analysis plan, sample-size justification, missing-data approach, and analysis populations are not reported. The planning assumptions are explicitly described as illustrations rather than the trial’s own design calculations.
Although secondary outcomes and adverse-event fields are listed, results are not reported for the secondary outcomes, and no analyses are listed. The adverse-event entries report zero at-risk and affected values, while death entries are not reported; these entries do not provide a complete, interpretable safety comparison. Taken together, the record permits description of the intended question and broad design, but not estimation of efficacy, precision, or comparative safety.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Megestrol Acetate in Treating Weight Loss or Anorexia in Young Patients With Malignancies Who Are Undergoing Radiation Therapy and/or Chemotherapy — NCT01200602.
- Registry record last updated 2015-11-25; status last verified 2015-10.
- Results first posted 2013-05-24.
- 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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17. Record Summary
NCT01200602 is a randomized, unmasked, parallel Phase 2 trial sponsored by Mayo Clinic. It compared oral megestrol acetate 1-2 times daily with clinical observation for 4 weeks in young patients with malignancies and weight loss or anorexia who were undergoing radiation therapy and/or chemotherapy. The registry records enrollment as 1, a start date of 2011-03, a primary completion date of 2011-11, and status as terminated.
The primary outcome was the proportion maintaining or gaining weight at the end of initial treatment compared with baseline. The registry says the accrued patient’s result is withheld for confidentiality. It lists secondary outcomes but reports no analyses. The result and reason for termination are not reported in the registry record, so the posted information does not permit an efficacy or comparative safety conclusion.