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 Study of Genistein in Pediatric Oncology Patients (UVA-Gen001) (NCT02624388), a randomized phase 2 study. The primary outcome is Time to Neutrophil Count Recovery Following Myelosuppressive Chemotherapy.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02624388 |
| Phase | Phase 2 |
| Condition | Lymphoma, Childhood Lymphoma, Solid Tumor |
| Allocation | Randomized |
| Design model | Crossover |
| Masking | Triple |
| Enrollment | 4 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Virginia |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric cancer patients receiving myelosuppressive chemotherapy were the intended population. The registry lists lymphoma, solid tumors, neuroblastoma, Ewing sarcoma, Hodgkin and non-Hodgkin lymphoma, rhabdomyosarcoma, soft tissue sarcoma, medulloblastoma, germ cell tumor, Wilms tumor, brain neoplasms, and primitive neuroectodermal tumors among the conditions. Enrollment was 4. The registry record does not report how many participants had each diagnosis or further eligibility details.
Intervention
The intervention was 30 mg genistein daily during chemotherapy cycles 1 and 2 for one randomized sequence, followed by placebo during cycles 3 and 4. Genistein is described in the study summary as the major isoflavone component in soybeans. The intended contrast concerns short-term effects during chemotherapy cycles with genistein supplementation versus cycles with placebo.
Comparator
The comparator was placebo, with the order reversed for the other randomized sequence: placebo during cycles 1 and 2, followed by 30 mg genistein daily during cycles 3 and 4. Because the study used a crossover design, participants were assigned to a sequence that included both treatments rather than remaining in a single treatment group throughout.
Primary question
The primary endpoint was time to neutrophil count recovery following myelosuppressive chemotherapy, measured in days. The central question was whether recovery time differed between chemotherapy cycles accompanied by genistein and cycles accompanied by placebo. The study summary describes a broader hypothesis about short-term therapy-related side effects, but the registry identifies neutrophil recovery time as the primary outcome. Numerical primary outcome results and an estimated treatment contrast are not reported in the registry record.
3. Trial Design
This was a randomized, placebo-controlled pilot study registered as a phase 2 trial. It used a crossover model and triple masking. Participants were assigned to one of two sequences: genistein during chemotherapy cycles 1 and 2 followed by placebo during cycles 3 and 4, or placebo during cycles 1 and 2 followed by genistein during cycles 3 and 4. Each active-treatment period used genistein 30 mg daily. The registry does not state which specific groups of people were masked.
The study was sponsored by the University of Virginia. It began in 2016-08, had a primary completion date of 2021-09, and is listed as terminated. Enrollment was 4. Although the study summary describes the intended treatment sequence and rationale, the registry record does not report why the study was terminated or how termination affected data collection and analysis.
The crossover structure means treatment comparisons could draw on observations from the same participants under both conditions. In general, such designs can account for stable differences between participants, but interpretation may depend on period effects, treatment order, and whether effects from an earlier period persist into a later one. The registry record does not report a washout period, an assessment of carryover, or the statistical method used to account for sequence and period.
The stated primary outcome was time to neutrophil count recovery, summarized as a mean with standard deviation and measured in days. The registry describes its timeframe as from the first date study drug is taken until the last date study drug is taken, about 8 - 12 weeks if there are no chemotherapy delays. The record does not provide the numerical outcome values, a definition of the recovery threshold, or a numerical comparison between treatment conditions. It lists 12 outcome measures, including secondary outcomes related to inflammation markers, adverse events, hospitalization, treatment delays, dose reductions, and antimicrobial treatment.
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: Genistein Followed by Placebo | Arm B: Placebo Followed by Genistein |
|---|---|---|
| STARTED | 2 | 2 |
| COMPLETED | 2 | 2 |
| NOT COMPLETED | 0 | 0 |
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 A: Genistein Followed by Placebo | Arm B: Placebo Followed by Genistein | Total |
|---|---|---|---|---|
| Participants analysed | 2 | 2 | 4 | |
| Age, Categorical: <=18 years | Participants | 2 | 2 | 4 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Age, Continuous | years | 14 (12 to 16) | 10 (8 to 12) | 12 (8 to 16) |
| Sex: Female, Male: Female | Participants | 2 | 2 | 4 |
| Sex: Female, Male: Male | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 2 | 2 | 4 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 2 | 2 | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Time to Neutrophil Count Recovery Following Myelosuppressive Chemotherapy, measured over From the first date study drug is taken until the last date that study drug is taken, about 8 - 12 weeks if there are no chemotherapy delays.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | days |
| Endpoint classification | Continuous Endpoint |
Time to neutrophil count recovery following myelosuppressive chemotherapy is the primary endpoint. It measures elapsed time in days from the study-defined starting point to recovery of the neutrophil count. Neutrophils are a type of white blood cell, and recovery time is a clinical measure of how long a participant takes to reach the recovery criterion after chemotherapy. The registry record does not define the count threshold used to determine recovery, so the operational endpoint definition cannot be evaluated from the record.
As general context, a recovery-time endpoint can capture the duration of a treatment-related change rather than only whether a change occurred. It may therefore be relevant to a study focused on short-term effects during chemotherapy. Its interpretation depends on a clearly specified start and end point, consistent measurement, and an analysis that reflects the study design. In this record, the outcome is identified as a mean with standard deviation, but the numerical means, standard deviations, and treatment contrast are not reported in the registry record.
The listed timeframe extends from the first date study drug is taken until the last date it is taken, about 8 - 12 weeks if there are no chemotherapy delays. This describes the intended observation period, not the observed recovery time. Neither the number of participants contributing endpoint data nor the handling of missing measurements is reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and design, but it does not provide a numerical result for the primary endpoint. There is no reported mean time to neutrophil recovery for either treatment condition, standard deviation, treatment contrast, confidence interval, or p-value. The primary endpoint therefore cannot be interpreted as showing the size, direction, or precision of a difference between genistein and placebo from the posted results.
Enrollment was 4, and the study is listed as terminated. With such a small enrolled group, any estimate based on the participants would be expected, as a general statistical matter, to be highly sensitive to individual observations. That point does not establish what the results would have been; the necessary endpoint values are not reported in the registry record. The record also does not state the number of participants with complete primary endpoint data.
The study's crossover design was intended to expose participants to both genistein and placebo in a specified order. General interpretation of a crossover comparison depends on the within-participant data and on whether sequence, period, or carryover effects were considered. The registry record does not report the analysis approach or the data needed to assess those features. Accordingly, the existence of a crossover design alone does not establish that a treatment contrast was estimated appropriately or precisely.
The adverse-event entries report small numbers at risk within the listed genistein-cycle and placebo-cycle groupings. These entries are descriptive and do not supply a reliable basis for comparing safety between treatments. The record reports febrile neutropenia as the top serious adverse event term at 50.0%, but it does not provide enough contextual detail to infer a comparative risk from that figure. Safety interpretation is further limited by the very small enrollment and absence of reported analysis details.
Overall, the posted record documents a randomized pilot study of genistein supplementation during pediatric chemotherapy and lists several outcomes of interest. It does not report primary outcome estimates or analyses, so no conclusion about the magnitude or precision of the primary comparison can be drawn from the registry record. The record's design and rationale describe the question the investigators intended to study, not a demonstrated treatment effect.
9. Statistical Methods Explained
The registered primary outcome is continuous: time to neutrophil count recovery, measured in days and summarized using a mean and standard deviation. The registry record does not report an analysis result for this endpoint. The following concepts are general explanations of how such data and designs are commonly interpreted, not descriptions of an analysis confirmed in the registry record.
What does a mean describe?
General: a mean is an average that summarizes the observed values in a group or treatment condition. For recovery time, it would describe the average number of days to recovery among the observations included in the analysis. A mean by itself does not show how spread out individual recovery times were, whether there were unusually long times, or how uncertain the estimated average is. The registry specifies that the outcome is a mean with standard deviation, but numerical values for either are not reported in the registry record.
What does the standard deviation add?
General: the standard deviation describes the spread of observations around their mean. A larger standard deviation indicates greater variability among observed values, while a smaller one indicates values closer to the mean. It is not the same as a confidence interval: a standard deviation describes variation among observations, whereas a confidence interval expresses uncertainty about an estimated quantity. No standard deviation or confidence interval for neutrophil recovery is reported in the registry record.
Why does the crossover design matter?
General: in a crossover trial, a participant may receive each study condition in different periods. An analysis can compare conditions within the same participant, which can reduce the influence of stable participant characteristics. But a valid interpretation also depends on the timing of measurements, the order in which treatments are received, and whether an earlier treatment has an effect that continues into a later period. Period effects can occur if outcomes change over time for reasons unrelated to treatment. These are design considerations, not findings about this trial. The record does not report a washout period, a carryover assessment, or an analysis that addresses sequence and period.
Here the assigned sequences were genistein in cycles 1 and 2 followed by placebo in cycles 3 and 4, or placebo in cycles 1 and 2 followed by genistein in cycles 3 and 4. The registry gives the sequences but not participant-level measurements or the statistical method used to compare them. It therefore does not establish whether the analysis used paired observations, adjusted for period, or treated the sequences in another way.
What does masking contribute?
General: masking is intended to limit the influence of knowledge about assigned treatment on conduct, measurement, or assessment. The record labels the study triple-masked. It does not specify which roles were masked, whether masking was maintained, or how it was assessed. The placebo comparator and masking label describe features of the design, but do not by themselves demonstrate that bias was absent.
Why are uncertainty estimates important?
General: an estimated difference is more informative when accompanied by a confidence interval, which gives a range of values compatible with the data under the method's assumptions. A narrow interval indicates greater precision than a wide interval. A p-value addresses how compatible data are with a specified null hypothesis under a statistical model; it does not state the probability that a treatment works or the size of an effect. Neither a confidence interval nor a p-value for the primary comparison is reported in the registry record. Without an effect estimate and uncertainty measure, the magnitude and precision of a primary comparison cannot be assessed.
How should planning assumptions be read?
General: a sample-size calculation is a planning exercise based on assumptions about a target effect, variability, significance level, allocation, and analysis. It is not an observed result and is not observed power. The registry describes a planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. It also states that time-to-event calculations use the total number of events under the Schoenfeld method, while other calculations give participants per arm. These are described as planning assumptions, not as the trial's own design justification. The registry record does not report a sample-size justification for the trial. Numerical planning estimates are not reported in the registry record.
The distinction between a planning calculation and observed evidence is especially important for a pilot study with enrollment of 4. A planning illustration cannot replace an estimate from observed data, and it cannot show that the study had adequate precision. Since no primary outcome analysis is reported, the posted record supports no numerical assessment of the treatment contrast or its uncertainty.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 12 outcome measures, including the primary neutrophil recovery endpoint and secondary measures involving inflammation, adverse events, hospitalization, treatment delays, dose reductions, and antimicrobial treatment. When a study examines multiple outcomes or multiple time points, general statistical practice considers multiplicity: the chance of at least one apparently notable result can increase as the number of statistical tests grows. Methods to address this can include prespecifying a primary outcome, adjusting inference, or interpreting secondary analyses as exploratory. The registry record does not report a multiplicity adjustment or a detailed analysis plan.
The registry reports 0 analyses. It does not report a primary endpoint estimate, statistical test, confidence interval, or analysis population. The number of enrolled participants is 4, but the number with evaluable data for each outcome is not reported in the registry record. The record does not describe how missing outcome measurements, incomplete treatment periods, withdrawals, or protocol deviations were handled.
In general, missingness can affect an estimate if the availability of observations is related to a participant's outcome or treatment experience. A crossover study also requires clarity about which participants contributed measurements under both conditions and how incomplete sequences were treated. These are important analysis questions, but the registry record does not provide enough information to determine the approach used. It also does not report whether the primary analysis followed randomized assignment, used only participants who completed both treatment periods, or applied another analysis population definition.
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 A: Genistein Followed by Placebo - GENISTEIN Cycles | 1 | 2 | 50.0% |
| Arm B: Placebo Followed by Genistein - GENISTEIN Cycles | 1 | 2 | 50.0% |
| Arm A: Genistein Followed by Placebo - PLACEBO Cycles | 1 | 2 | 50.0% |
| Arm B: Placebo Followed by Genistein - PLACEBO Cycles | 1 | 2 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Arm A: Genistein Followed by Placebo - GENISTEIN Cycles | 0 of 2 |
| Arm B: Placebo Followed by Genistein - GENISTEIN Cycles | 1 of 2 |
| Arm A: Genistein Followed by Placebo - PLACEBO Cycles | 0 of 2 |
| Arm B: Placebo Followed by Genistein - PLACEBO Cycles | 0 of 2 |
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 A: Genistein Followed by Placebo - GENISTEIN Cycles | 2 of 2 |
| Arm B: Placebo Followed by Genistein - GENISTEIN Cycles | 2 of 2 |
| Arm A: Genistein Followed by Placebo - PLACEBO Cycles | 0 of 2 |
| Arm B: Placebo Followed by Genistein - PLACEBO Cycles | 0 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm A: Genistein Followed by Placebo - GENISTEIN Cycles | Arm B: Placebo Followed by Genistein - GENISTEIN Cycles | Arm A: Genistein Followed by Placebo - PLACEBO Cycles | Arm B: Placebo Followed by Genistein - PLACEBO Cycles |
|---|---|---|---|---|---|
| febrile neutropenia | Blood and lymphatic system disorders | 1 of 2 | 1 of 2 | 1 of 2 | 1 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm A: Genistein Followed by Placebo - GENISTEIN Cycles | Arm B: Placebo Followed by Genistein - GENISTEIN Cycles | Arm A: Genistein Followed by Placebo - PLACEBO Cycles | Arm B: Placebo Followed by Genistein - PLACEBO Cycles |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | 1 of 2 | 2 of 2 | n/a of 2 | n/a of 2 |
| Neutrophil count decreased | Investigations | 2 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Platelet count decreased | Investigations | 2 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| White blood cell count decreased | Investigations | 2 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Anemia | Blood and lymphatic system disorders | 1 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 1 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Vomiting | Gastrointestinal disorders | 1 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Fatigue | General disorders | 1 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Fever | General disorders | 1 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
| Headache | Nervous system disorders | 1 of 2 | 1 of 2 | n/a of 2 | n/a of 2 |
Serious events with the highest arm-level rate: febrile neutropenia (50.0%).
The registry reports serious adverse event counts separately for genistein-cycle and placebo-cycle groupings within each assigned sequence. For the Arm A genistein-cycle grouping, 2 participants were at risk and 1 was affected by a serious adverse event; for the Arm B genistein-cycle grouping, 2 were at risk and 1 was affected. For each placebo-cycle grouping, 2 were at risk and 1 was affected. The record lists 0 deaths for the Arm A genistein-cycle grouping, 1 for the Arm B genistein-cycle grouping, and 0 for both placebo-cycle groupings.
The top serious adverse event term is febrile neutropenia at 50.0%. The registry also reports other adverse events in the two genistein-cycle groupings as 2 at risk and 2 affected in each, and in both placebo-cycle groupings as 2 at risk and 0 affected. These are descriptive counts from a very small enrolled study. The crossover sequences mean the cycle groupings should not automatically be treated as independent sets of participants. The registry record does not provide a statistical comparison of adverse events between treatments, event-level details, or enough information to interpret the listed percentage as a comparative risk. These figures should therefore be read as reported counts, not as evidence of a treatment effect on safety.
12. What the Result Does, and Does Not, Mean
The record identifies a randomized, triple-masked crossover pilot study of genistein and placebo during myelosuppressive chemotherapy in pediatric cancer patients. Its primary question concerned time to neutrophil count recovery, and its study summary describes a broader interest in short-term treatment-related side effects. The registry's description of that rationale and hypothesis does not establish that genistein changed recovery time or reduced side effects.
The primary endpoint's numerical results are not reported in the registry record. There is no reported estimate of the difference between genistein and placebo, nor an uncertainty measure that would indicate how precisely a difference was estimated. The direction and size of any observed primary outcome contrast therefore cannot be determined from the posted results. The absence of a reported result is not evidence that the treatments were equivalent or that no effect occurred.
Enrollment was 4, and the trial is listed as terminated. Those details limit the amount of information available in the registry, but they do not explain why the study ended or what data were collected. The record does not report the number of participants with complete outcome data, reasons for missingness, or an analysis population. The adverse-event counts are also based on small groupings and do not support a robust treatment comparison.
General: statistical significance and clinical importance are distinct. A small estimated difference can be statistically precise yet have limited practical relevance, while an imprecise estimate may leave a wide range of possible effects unresolved. Assessing either requires an outcome estimate and suitable uncertainty information, neither of which is reported for the primary comparison. The trial's stated design and outcome plan help define the research question, but the posted record does not provide the results required to answer it.
13. Why This Trial Matters Statistically
This trial is statistically notable as a crossover pilot study in a pediatric oncology setting. Participants were assigned to sequences that placed genistein and placebo in different chemotherapy-cycle periods, making the distinction between within-participant and between-sequence information relevant. In general, crossover designs can be useful when outcomes can be measured across periods, but interpretation depends on period effects, treatment order, and potential carryover. The registry record does not report how these issues were handled.
The endpoint also raises a fundamental reporting issue: a clearly named outcome is not enough to evaluate a treatment comparison. Time to neutrophil recovery is defined in the record as a mean in days, but the recovery threshold, observed means, variability, and estimated contrast are not reported. Without those details, readers cannot assess the result's magnitude or precision. The distinction between an outcome definition and an outcome finding is central to transparent statistical reporting.
Enrollment of 4 and the terminated status make completeness and analysis choices especially important to document. The record does not report participant flow, missing-data handling, or an analysis population. It also lists multiple outcomes but does not report analyses or a multiplicity strategy. These omissions leave readers unable to determine how much information each outcome contains or how findings across outcomes would have been interpreted.
The record therefore illustrates why design labels and a stated hypothesis must be separated from empirical evidence. Randomization, masking, and a placebo comparator describe intended safeguards; they do not replace reporting of estimates, uncertainty, and analysis methods. For clinical researchers and biostatisticians, the missing numerical results and analytic details are themselves consequential features of the evidence 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 most direct limitation is that the registry record does not report a numerical result for the primary endpoint. It gives no mean recovery time, standard deviation, treatment contrast, confidence interval, or p-value. The recovery threshold is not defined, so the endpoint cannot be independently operationalized from the record. Although the stated timeframe is about 8 - 12 weeks if there are no chemotherapy delays, observed follow-up duration is not reported.
Enrollment was 4, and the trial is listed as terminated. The registry does not report the reason for termination, participant flow, the number completing each treatment period, or how many had evaluable primary outcome data. The status and enrollment figures alone do not explain why the study ended or establish whether all planned observations were collected.
The crossover structure makes period, sequence, and carryover considerations relevant, yet the record does not report a washout period, an assessment of carryover, or methods for accounting for period effects. It identifies the study as triple-masked but does not specify which roles were masked or whether masking was maintained. Eligibility details and the number of participants within each listed cancer diagnosis are also not reported.
The registry lists 12 outcome measures and 0 analyses. It does not report a detailed statistical analysis plan, multiplicity approach, missing-data method, or analysis population. The serious adverse event entries are descriptive counts from small groupings, without a reported comparative analysis. Taken together, these limitations prevent assessment of the primary treatment contrast, its precision, and the robustness of safety comparisons.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Genistein in Pediatric Oncology Patients (UVA-Gen001) — NCT02624388.
- Registry record last updated 2024-05-06; status last verified 2024-05.
- Results first posted 2024-05-06.
- 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
NCT02624388, titled Study of Genistein in Pediatric Oncology Patients (UVA-Gen001), is listed as a phase 2 randomized, placebo-controlled, triple-masked crossover pilot study sponsored by the University of Virginia. It enrolled 4 pediatric cancer patients receiving myelosuppressive chemotherapy and is listed as terminated. The study began in 2016-08, with primary completion in 2021-09.
The treatment sequences were genistein 30 mg daily during chemotherapy cycles 1 and 2 followed by placebo during cycles 3 and 4, or the reverse sequence. The primary outcome was time to neutrophil count recovery, measured in days and summarized as a mean with standard deviation. The registry lists 12 outcome measures and 0 analyses. Numerical primary outcome results and detailed analysis methods are not reported in the registry record. Adverse-event entries include small counts by treatment-cycle grouping and febrile neutropenia at 50.0% as the top serious adverse event term.