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 Cyproheptadine Hydrochloride and Nutritional Supplementation in Treating Young Patients With Weight Loss With Cancer (NCT00949117), a randomized phase 2 study. The primary outcome is Difference Between Measures of Weight at Baseline and at Week 24.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00949117 |
| Phase | Phase 2 |
| Condition | Leukemia, Lymphoma, Malnutrition |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | 2 |
| Lead sponsor | University of South Florida |
| Results posted | Yes |
2. Clinical Question
Population
Young patients with cancer or cancer-treatment-related weight loss were the intended population. The registry lists leukemia, lymphoma, myelodysplastic syndromes, unspecified childhood solid tumors, malnutrition, and weight changes among the conditions. It does not report an age range or other detailed eligibility criteria in the record.
Intervention
One group received oral cyproheptadine hydrochloride twice daily. The other received the same drug together with oral nutritional supplementation twice daily: PediaSure for patients aged 2 to 10 years and Ensure for those > 10 years. Treatment could continue for up to 24 weeks in the absence of weight loss or unacceptable toxicity.
Comparator
The comparator was cyproheptadine hydrochloride without the listed supplement. The randomized, parallel-group design therefore asked whether adding nutritional supplementation was associated with a different change in weight than using cyproheptadine alone. The study was open label, with no masking reported.
Primary question
The primary outcome was the mean difference in weight, in kilograms, between baseline and week 24. The registry’s description concerns participants continuing study treatment for the full 24-week period. It does not specify the subtraction direction for the change score. The posted arm results do not provide a usable two-group comparison: one arm has a mean based on 1 participant, while the other has no endpoint value.
3. Trial Design
This was an open-label, randomized phase II study with a parallel-group design. The registry identifies the sponsor as the University of South Florida. The recorded start was 2009-09, primary completion was 2011-11, and the current status is terminated. The reason for termination is not reported in the registry record.
The stated enrollment was 9. The two assigned regimens were cyproheptadine hydrochloride alone and cyproheptadine hydrochloride with PediaSure or Ensure. Both regimens used oral cyproheptadine twice daily, with treatment planned for up to 24 weeks in the absence of weight loss or unacceptable toxicity. The supplement was given orally twice daily; the supplement type depended on age as described in the registry.
Randomization indicates that assignment to the study groups was intended to be determined by a chance-based allocation process. The allocation sequence, implementation details, and any stratification factors are not reported in the registry record. Because masking was listed as none, participants and study personnel were not reported as blinded to assignment. The record does not describe blinded outcome assessment.
The registry lists five outcome measures in total, including the primary weight measure and outcomes concerning body mass index, pre-albumin and body composition, quality of life, and weight-for-age Z-score. Four secondary outcomes are listed. The timing and measures for those secondary outcomes are described in the registry, but their results and analyses are not reported in the posted results. Details of the intended statistical analysis plan and 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.
Overall Study
| Stage | Arm I- Cyproheptadine Hydrochloride | Arm II Cyproheptadine HCl and PediaSure or Ensure |
|---|---|---|
| STARTED | 4 | 5 |
| COMPLETED | 1 | 0 |
| NOT COMPLETED | 3 | 5 |
Reasons for not completing this period:
| Reason | Arm I- Cyproheptadine Hydrochloride | Arm II Cyproheptadine HCl and PediaSure or Ensure |
|---|---|---|
| Lack of Efficacy | 2 | 3 |
| Protocol Violation | 1 | 1 |
| Adverse Event | 0 | 1 |
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 | Arm I- Cyproheptadine Hydrochloride | Arm II Cyproheptadine HCl and PediaSure or Ensure | Total |
|---|---|---|---|---|
| Participants analysed | 4 | 5 | 9 | |
| Age, Categorical: <=18 years | Participants | 4 | 5 | 9 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Age, Continuous | years | 9.1 (SD 5.5) | 13.3 (SD 5.1) | 11.4 (SD 5.1) |
| Sex: Female, Male: Female | Participants | 2 | 0 | 2 |
| Sex: Female, Male: Male | Participants | 2 | 5 | 7 |
| Region of Enrollment: United States | participants | 4 | 5 | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Difference Between Measures of Weight at Baseline and at Week 24, measured over 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | kilograms |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was the difference between weight measured at baseline and weight measured at week 24, expressed in kilograms. The registry specifies a mean as the summary measure and identifies the assessment timeframe as 24 weeks. Its description refers to continuing study treatment for the full 24-week period, but it does not state which time point is subtracted from the other. That convention matters because it determines whether a positive change represents weight gain or weight loss.
Weight is a direct measure of the outcome the study sought to track: weight loss associated with cancer or its treatment. Comparing change from baseline, rather than relying only on an end-of-study weight, can describe how the measured weight shifted during follow-up. A group comparison could then address whether the average change differed between the regimen with supplementation and the regimen without it. This is a general explanation of the endpoint’s statistical purpose, not a claim that the posted data establish a treatment effect.
The posted primary results are highly incomplete. The cyproheptadine-alone arm has a reported mean of 4.3 kilograms based on 1 participant, with no dispersion estimate. The combination arm has no reported endpoint value and an analyzed sample size of 0. Consequently, the registry results do not support estimating or interpreting a between-group difference in mean weight change.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary endpoint is a continuous measure summarized as a mean. For the cyproheptadine-alone arm, the posted mean is 4.3 kilograms, based on 1 participant. No standard deviation or other measure of spread is reported for that arm. The combination arm has 0 participants with a posted primary endpoint value, and its mean is not reported. These results are not a comparison of the two randomized groups.
A mean based on 1 participant describes that participant’s recorded outcome; it cannot show how outcomes varied among participants in the arm. Without a spread estimate, the precision of that arm’s mean cannot be assessed from the posted summary. More importantly, the absent endpoint value for the combination arm means there is no posted arm-to-arm contrast for the primary outcome. The record also reports no analyses. A confidence interval, p-value, or adjusted comparison is not reported in the registry record.
The stated enrollment of 9 and the endpoint sample sizes should not be treated as interchangeable. Enrollment counts participants entering the trial, whereas the primary endpoint results indicate how many participants contribute a reported value to that outcome. The registry does not explain why the primary endpoint has a value for only 1 participant in one arm and none in the other, or how those endpoint counts relate to the enrolled participants. Reasons for missing endpoint values, withdrawals, and exclusions are not reported in the registry record.
Interpretation is further limited by the lack of a reported analysis population, missing-data approach, or statistical analysis plan. The registry does not say whether a particular method was used to handle incomplete follow-up or whether any adjustment was planned. It also does not report a sponsor analysis for the primary endpoint. Since there is no numerical two-arm result to compare, no recalculated unadjusted treatment contrast is available. The record does not identify a discrepancy between a sponsor analysis and a recalculation.
The outcome is described in kilograms and at a specified follow-up time, but the direction convention for the change is unclear. That ambiguity affects how a signed result would be read. Even so, it does not alter the central limitation: only one arm has a numerical mean, and that mean rests on 1 participant. The result therefore cannot establish whether the groups differed or quantify the precision of such a difference.
Overall, the posted data show a very limited primary outcome record rather than evidence that can resolve the randomized comparison. The numeric mean should be reported with its sample size and missing dispersion information, not treated as a stable estimate of an arm’s typical response. A conclusion about the relative outcomes of the two regimens is not supported by these posted primary results.
9. Statistical Methods Explained
The registry identifies this as a randomized, open-label, parallel-group phase II trial. Randomization is a design method intended to reduce systematic differences between groups at assignment. In general, it can support a fair comparison when outcome data are adequately collected and analyzed, but randomization alone does not guarantee that the observed groups will be comparable in a small trial or when outcome information is incomplete. The registry does not report how the random sequence was generated, how assignments were concealed, or whether randomization used strata or blocks.
What does a mean summarize?
The primary outcome is continuous weight change in kilograms, summarized by a mean. In general, a mean represents the arithmetic average of the observed values in a group. It can be useful for describing the center of a continuous outcome, but its interpretation depends on the number of observations and the variability among them. Here, the registry reports a mean of 4.3 kilograms for 1 participant in the cyproheptadine-alone group. With only 1 observation, the mean is that participant’s value; there is no within-group variation to estimate from that arm’s posted result. The combination group has no reported primary endpoint observation.
Why does dispersion matter?
A standard deviation describes how spread out individual values are around their group mean. It helps readers understand whether a mean represents tightly clustered observations or a wide range of outcomes. The registry identifies standard deviation as the dispersion measure for the primary endpoint, but no standard deviation is posted for either arm. Without observed variation and adequate endpoint data, the precision of the group estimates and any contrast cannot be judged. This is a general statistical explanation; no confidence interval is reported in the registry record.
What would a group comparison require?
For a continuous endpoint, a basic two-group comparison would require outcome summaries for both groups, including the number of participants contributing data and information about variability. An unadjusted comparison would use those summaries to estimate the difference in group means and its uncertainty. More complex analyses might account for baseline measurements or other prespecified factors, but the registry does not report whether such an approach was planned. No arm-to-arm primary endpoint estimate, confidence interval, or p-value is posted, and no analysis results are reported.
The registry’s skipped comparison reflects the absence of estimable values for a two-arm continuous endpoint. No recalculated comparison can be derived from the posted primary summaries because the second arm has no mean or spread, and the first arm has only one observation with no spread estimate. It would be inappropriate to infer a group difference from the single-arm mean.
What does a confidence interval communicate?
General: a confidence interval expresses uncertainty around an estimated quantity, such as a difference between group means. A narrower interval indicates greater precision under the assumptions of the method; a wider interval indicates less precision. It is not a range containing a known treatment effect with certainty. Because the posted results do not contain the data needed for a two-arm estimate, there is no interval to interpret for the primary comparison. The registry does not report an interval for the single-arm mean either.
What is the role of a significance test?
General: a significance test assesses how compatible observed data are with a specified null hypothesis, under the assumptions of the chosen model. A p-value does not measure the size or clinical importance of an effect, and it does not establish that a hypothesis is true or false. No p-value or inferential test result is reported for the primary outcome. The statistical test, if any, planned by the investigators is not reported in the registry record.
Why can over-dispersion matter?
General: over-dispersion occurs when observed variability is greater than a statistical model expects. If a model assumes less variability than the data contain, standard errors may be too small and confidence intervals or tests may give an overly confident impression. This concept is especially relevant to count outcomes, such as event counts, and to models that impose a particular variance structure. The primary endpoint in this trial is continuous weight change, and the registry does not report a model or an over-dispersion assessment. Over-dispersion should not be asserted as a feature of these results.
The record describes a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The target effect is expressed as a stated fraction of the observed effect. Such sample-size calculations are planning illustrations, not observed power and not evidence that the trial achieved a particular probability of detecting an effect. They rely on assumptions about the effect and analysis. Given the incomplete primary outcome summaries, those planning assumptions should not be mistaken for a valid estimate of the trial’s observed comparative result. The registry does not report its own sample-size justification.
Masking was listed as none. General: when participants or study personnel know treatment assignment, subjective outcomes and decisions about reporting or assessment can be more susceptible to expectations. Weight itself is a measured outcome, but the registry does not describe assessment procedures or whether the assessor knew assignments. The record also does not describe interim analyses, multiplicity adjustments, or the planned handling of missing data. Those methods therefore cannot be attributed to this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four secondary outcomes in addition to the primary weight outcome. They concern body mass index at baseline and 24 weeks; pre-albumin and body composition; quality of life assessed by the Peds-FAACT questionnaire at baseline and at weeks 4 and 24; and change in weight-for-age Z-score through 24 weeks. The posted results do not include analyses for these outcomes. A multiplicity strategy—such as prioritizing outcomes, adjusting significance thresholds, or treating secondary findings as exploratory—is not reported in the registry record.
Multiplicity matters when several outcomes or repeated assessments are tested. General: as the number of statistical comparisons grows, the chance of at least one apparently notable result can increase if each test is interpreted independently. A prespecified plan can help distinguish the main confirmatory question from additional exploratory questions. No such plan or adjustment is reported, so no claim about control of multiplicity is warranted.
Missing primary endpoint information is substantial in the posted results: the cyproheptadine-alone arm reports a value for 1 participant, while the combination arm has 0 participants with a posted value. The reasons for this pattern, the timing of any loss to follow-up, and the relationship to the stated enrollment are not reported in the registry record. The record also does not describe imputation, sensitivity analyses, or another missing-data method.
The analysis population is not reported. It is therefore unclear from the registry whether the results were intended to represent all randomized participants, those who received treatment, those who completed follow-up, or another defined group. The registry does not report how participants with incomplete observations were handled. These gaps prevent assessment of whether the posted endpoint summaries represent the randomized groups as assigned.
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 |
|---|---|---|---|
| Arm I- Cyproheptadine Hydrochloride | 0 | 4 | 0.0% |
| Arm II Cyproheptadine HCl and PediaSure or Ensure | 0 | 5 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Arm I- Cyproheptadine Hydrochloride | 1 of 4 |
| Arm II Cyproheptadine HCl and PediaSure or Ensure | 2 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm I- Cyproheptadine Hydrochloride | Arm II Cyproheptadine HCl and PediaSure or Ensure |
|---|---|---|---|
| somnolence | General disorders | 1 of 4 | 0 of 5 |
| relapse of cancer | General disorders | 0 of 4 | 1 of 5 |
| rash | Skin and subcutaneous tissue disorders | 0 of 4 | 1 of 5 |
| flatulence | Gastrointestinal disorders | 0 of 4 | 1 of 5 |
For serious adverse events, the registry reports 0 affected participants among 4 at risk in the cyproheptadine-alone group and 0 among 5 at risk in the group receiving cyproheptadine with PediaSure or Ensure. These are counts within the safety groups, not estimates of the primary weight outcome. The record lists no serious adverse-event terms.
For other, non-serious adverse events, 1 participant among 4 at risk was affected in the cyproheptadine-alone group, and 2 among 5 at risk were affected in the combination group. The registry does not provide event terms in the registry entries, so the kinds of events and their severity beyond the serious/non-serious classification cannot be described. Death counts are not reported in the registry record.
These small safety-group counts should be read descriptively. General: counts from small groups provide limited information about how often events might occur in a broader population, and a zero count does not establish that an event cannot occur. The registry figures alone also do not establish whether an event was caused by a study treatment. No causal conclusion should be drawn from these counts.
12. What the Result Does, and Does Not, Mean
The posted results establish that the registry contains a primary endpoint mean of 4.3 kilograms for the cyproheptadine-alone group, based on 1 participant. They also establish that no primary endpoint value was posted for the group receiving cyproheptadine with nutritional supplementation. No variability estimate accompanies the available mean. These details describe the limits of the result rather than demonstrating a comparative effect.
The results do not show whether adding PediaSure or Ensure changed weight differently from cyproheptadine alone. There is no two-arm estimate, uncertainty interval, or statistical test for that comparison in the registry record. The available information also does not show whether either regimen changed weight from baseline for the broader enrolled population. A lone mean cannot be assumed to represent a group’s typical result, especially when the number contributing data is 1.
General: an observed outcome in a randomized trial is interpreted through the design, the completeness of follow-up, the analysis population, and the uncertainty around the estimate. Random assignment is valuable, but incomplete results can prevent the intended comparison from being made. The record does not provide enough primary endpoint information to assess precision, missing-data bias, or the robustness of a treatment contrast.
The trial’s stated purpose was to examine cyproheptadine with and without nutritional supplementation in young patients with cancer- or treatment-related weight loss. The posted results should not be read as proving that either regimen is effective or ineffective. They also do not provide a basis for choosing between the regimens. A comparative conclusion would require outcome data and analyses that are not reported in the registry record.
The record’s status is terminated, but the reason for termination is not reported. That status by itself does not explain why primary outcome data are incomplete or what happened to participants who did not contribute a result. Without that information, the status should not be used to infer the study’s conduct or its findings.
13. Why This Trial Matters Statistically
This trial raises a basic statistical issue in randomized research: the value of a comparison depends on having usable outcome information from both groups. Random assignment was intended to compare cyproheptadine alone with cyproheptadine plus nutritional supplementation. Yet the posted primary endpoint has a mean for only one participant in one group and no endpoint value in the other. The intended randomized comparison cannot be recovered from those summaries.
The distinction between enrollment and outcome availability is important. The registry reports enrollment of 9, while the primary outcome summaries reflect 1 participant in one arm and 0 in the other. Those figures concern different stages of a trial: entering the study and contributing a result for a particular endpoint. When their relationship is unexplained, readers cannot determine how complete follow-up was or whether the reported participants are representative of those randomized.
The record also illustrates why a mean should not be interpreted without its sample size and spread. A mean can look precise when printed as a single value, but the amount of information behind it depends on the observations contributing to it. With no variation estimate and no comparison-arm mean, the apparent numerical simplicity of the result conceals substantial uncertainty.
Finally, a registered outcome list and an analysis plan are not substitutes for posted results. The registry names several outcomes, but does not report analyses for them. Missing analysis details also limit assessment of multiplicity, missing data, and the population included. These are not merely technical reporting details: they determine how confidently a study can answer its stated question. The trial is statistically informative as an example of why transparent outcome reporting and clear accounting of participants matter.
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 direct limitation is the incompleteness of the primary outcome results. The cyproheptadine-alone arm reports a mean of 4.3 kilograms based on 1 participant, while the combination arm has no endpoint value. Neither arm has a posted standard deviation. The record therefore does not permit an estimate of the primary between-group difference or its precision.
The registry does not explain the discrepancy between stated enrollment of 9 and the sparse primary endpoint results. It does not report reasons for missing outcome values, participant flow by arm, withdrawals, treatment discontinuation, or exclusions from the endpoint analysis. The analysis population and missing-data approach are also not reported. Without these details, the extent and potential consequences of missing information cannot be assessed.
The trial was open label, with no masking reported. The registry does not describe whether outcome assessors were blinded or how weight was measured. The allocation sequence and concealment methods are not reported either. These omissions limit evaluation of the safeguards around assignment and outcome assessment.
The record does not report the reason for termination, a sample-size justification, the planned statistical analysis, interim analyses, or a multiplicity strategy. It lists secondary outcomes but does not report their analyses. The direction convention for calculating the primary change score is also not specified. Together, these limitations prevent a full appraisal of the trial’s intended analysis and the meaning of any signed change.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Cyproheptadine Hydrochloride and Nutritional Supplementation in Treating Young Patients With Weight Loss With Cancer — NCT00949117.
- Registry record last updated 2014-04-02; status last verified 2014-03.
- Results first posted 2014-03-04.
- 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
NCT00949117 is an open-label randomized phase II parallel-group study sponsored by the University of South Florida. It enrolled 9 participants and is listed as terminated. The registry’s primary outcome is mean weight change, in kilograms, from baseline to week 24. The comparison was cyproheptadine hydrochloride alone versus cyproheptadine with PediaSure or Ensure.
The posted primary outcome includes a mean of 4.3 kilograms for 1 participant in the cyproheptadine-alone arm. The combination arm has no reported primary endpoint value, and no dispersion estimate or analysis is posted. Four secondary outcomes are listed, but their analyses are not reported in the registry record. The reason for termination and the reasons for missing endpoint information are also not reported. The available results do not support a comparative conclusion about the two regimens.