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 High-Risk Neuroblastoma Chemotherapy Without G-CSF (NCT02786719), a na na study. The primary outcome is Incidence of Infection.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02786719 |
| Phase | NA |
| Condition | Neuroblastoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 13 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Baylor College of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
Children with high-risk neuroblastoma were the intended population. The record describes the condition as an aggressive childhood cancer and says the study was limited to the induction phase of treatment. Enrollment was 13; the primary endpoint description refers to patients evaluable for that endpoint, without reporting their number or the criteria used to determine evaluability.
Intervention
The study examined induction chemotherapy without prophylactic administration of G-CSF. The registry describes induction as six courses of chemotherapy, with drugs given in different combinations every 3 weeks. The listed arm description also includes tumor resection and stem cell collection. The study concerned induction, rather than the full treatment course described in the record.
Comparator
There was a single group and no like-for-like control arm. The registry lists allocation as NA and masking as none. The study therefore does not provide a randomized comparison of induction chemotherapy without G-CSF against induction chemotherapy with G-CSF. Any contrast with routine use described in the background is contextual, not a concurrent control comparison.
Primary question
The primary question was whether infections occurred during chemotherapy cycles that were not followed by hematopoietic growth factors. The primary endpoint is described as the incidence of infections, measured as a number, through study completion, approximately 5 months. The registry does not report the number of infections or an estimate of uncertainty for this endpoint. The record also lists secondary questions about chemotherapy delay due to prolonged neutrophil recovery and response after induction.
3. Trial Design
This was a completed, single-group safety pilot study of induction chemotherapy for high-risk neuroblastoma without prophylactic G-CSF. The record lists enrollment of 13, allocation as NA, and masking as none. It lists the phase as NA. Because there was no randomized control group, the design can describe events in the study group but cannot provide a direct randomized estimate of the difference between treatment strategies.
The treatment context is a longer course of care involving induction, consolidation, and maintenance. The study was limited to induction. The registry describes six chemotherapy courses, with combinations administered every 3 weeks; the listed arm description additionally names tumor resection and stem cell collection. The background says G-CSF is commonly used after chemotherapy to stimulate white blood cell production and shorten the period of low absolute neutrophil count. The study question was whether induction could be delivered without that prophylactic use.
The primary endpoint was infection incidence in chemotherapy cycles not followed by hematopoietic growth factors, through study completion, approximately 5 months. The record identifies the endpoint population as patients evaluable for the endpoint but does not define evaluability or report its size. Secondary outcomes concern delay in chemotherapy administration due to prolonged neutrophil recovery and response after induction without prophylactic G-CSF.
The registry marks the study completed, with enrollment of 13. It does not report a detailed statistical analysis plan, a sample-size justification, or interim-analysis methods. The listed drugs include chemotherapy agents and sargramostim; the record does not explain the role of each listed intervention in relation to the primary endpoint. The listed interventions alone therefore do not establish that a growth factor was administered in a particular cycle or to a particular participant.
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 | High Risk Neuroblastoma Patients |
|---|---|
| STARTED | 13 |
| COMPLETED | 12 |
| NOT COMPLETED | 1 |
Analysis population for the primary outcome, as defined in the registry: Includes all patients evaluable for endpoint
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: all participants evaluable for infection endpoint
| Characteristic | Unit | Single Arm |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 12 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 2.9 (1.1 to 6.7) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 8 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 11 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 12 |
| International Neuroblastoma Risk Group Stage: L2 (localized with image defined risk factors) | Participants | 2 |
| International Neuroblastoma Risk Group Stage: M (metastatic) | Participants | 10 |
| MYCN gene amplification: Yes | Participants | 6 |
| MYCN gene amplification: No | Participants | 6 |
| Marrow involvement: Yes | Participants | 9 |
| Marrow involvement: No | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Incidence of Infection, measured over through study completion, approximately 5 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | infections |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the incidence of infections in chemotherapy cycles not followed by hematopoietic growth factors. It is specified as a number of infections, with observation through study completion, approximately 5 months. The endpoint population is described as all patients evaluable for the endpoint. The registry does not report the observed number of infections, the number of evaluable patients, or the criteria for endpoint evaluation.
In general, an infection endpoint is relevant to a safety pilot when the study is assessing treatment delivery in a context where chemotherapy can affect blood-cell counts. The background explains that G-CSF is used to stimulate white blood cell production and shorten the time when ANC is low after chemotherapy. Counting infections during cycles without growth-factor follow-up addresses an event of clinical interest under the regimen being studied; it does not, by itself, establish how infection risk compares with another regimen.
The distinction between a count of infections and the proportion of participants experiencing at least one infection matters. A participant could contribute more than one infection, whereas a participant-level proportion would count each participant once. The registry specifies a number of infections, not a participant-level risk measure. It does not report whether repeat infections were handled as separate events or how observation time was incorporated. These details limit what can be inferred about the rate or distribution of infections.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record identifies a focused safety question: infections during induction chemotherapy cycles not followed by hematopoietic growth factors. It establishes that the study was a completed pilot with enrollment of 13, and it describes the primary endpoint and its approximate observation period. However, it does not report the primary endpoint result. Without the number of infections or an estimate of uncertainty, the observed infection experience cannot be quantified from the registry entry.
The registry lists serious adverse event summaries for a group with 12 participants at risk for serious events and 11 affected, and 12 at risk for other events and 12 affected. It reports 0 deaths. The listed serious-event terms include febrile neutropenia at 75.0%, bacteremia at 33.3%, skin/soft tissue infection at 25.0%, urinary tract infection at 16.7%, respiratory failure at 8.3%, and sinusoidal obstruction syndrome at 8.3%. These safety summaries are distinct from the primary endpoint result: the registry does not establish that the listed serious events occurred specifically during cycles without growth-factor follow-up.
These data should be read as descriptive safety information from a small, single-group study, rather than as a comparative estimate. There is no randomized control group, so differences from routine treatment or other studies cannot be attributed to the absence of prophylactic G-CSF on the basis of this record. The registry also does not report confidence intervals or another uncertainty measure for the primary endpoint or the serious-event summaries.
In general, a pilot study can provide information about feasibility and observed events that may help frame later research questions. It does not automatically establish how common an event would be in a broader population. The enrollment of 13 and the safety group sizes reported in the record are important context for the limited precision that descriptive summaries can offer, but they do not permit a new estimate of precision without the underlying endpoint data.
The record lists secondary outcomes for chemotherapy delay due to prolonged neutrophil recovery and response following induction without prophylactic G-CSF. It does not report results or analyses for those outcomes. Consequently, this entry supports a description of the questions the study addressed and of the posted safety summaries, but it does not support a conclusion about the primary infection endpoint, secondary outcomes, or comparative effects.
9. Statistical Methods Explained
The registry describes the primary outcome as a number of infections, rather than as a mean, percentage, or time-to-event outcome. No statistical analysis is listed for that endpoint. The record provides no infection count, dispersion estimate, confidence interval, or model result. Statistical interpretation must therefore remain at the level of the endpoint definition and the information that is explicitly recorded.
What does a count endpoint represent?
In general, a count endpoint records how many events occur during a defined observation period. A count is not the same as the number of participants who experience an event: a participant may experience repeated events, depending on the study definition. Here, the registry names infections as the unit and specifies chemotherapy cycles not followed by hematopoietic growth factors as the relevant setting. It does not state whether each infection was counted separately, how repeated infections were distinguished, or how differences in observation time were handled.
In general, a count can be summarized as a total, but a total alone may not describe how events are distributed across participants or over time. Statistical models for counts can account for variation beyond what a simple model assumes; that extra variation is often called over-dispersion. Over-dispersion matters because a model that assumes too little variation can make uncertainty look smaller than it is. The registry does not report a count model, an over-dispersion assessment, or any other analysis choice for the primary endpoint. It is therefore not possible to say whether such methods were used.
How does the single-group design affect inference?
In general, a single-group study estimates or describes outcomes among those enrolled, without a concurrent randomized group to estimate what would have happened under an alternative treatment strategy. This study lists one group and allocation as NA. Its results, if reported, could characterize events under the study conditions, but they would not by themselves isolate the effect of omitting prophylactic G-CSF from other influences. There is no like-for-like control comparison in the registry record.
In general, confidence intervals express the uncertainty around an estimate under the assumptions of the analysis. A narrow interval indicates greater statistical precision, while a wide interval indicates less. The registry does not report a confidence interval for the primary endpoint or a numerical primary estimate around which one could interpret an interval. No inferential test or p-value is listed; the registry reports zero analysis entries.
What do the planning assumptions mean?
The record describes planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For time-to-event tables, the described quantity is the total number of events; other planning quantities are participants per arm. These are planning assumptions, not a description of this trial's design. The actual study is listed as single-group, and the registry does not report a sample-size justification for the pilot. Any such planning illustration should not be read as observed power or as evidence that the study was designed with a control arm.
In general, power is a design-stage probability tied to assumptions about an effect, variability, sample size, and analysis. It is not a measure of whether an observed finding is reliable, and a post hoc calculation based on observed results does not substitute for an uncertainty interval. The registry record does not report the primary endpoint result needed to assess the observed infection count. It also does not provide the effect estimate underlying the planning illustration in the information available for this trial.
What can be learned from the safety summaries?
The adverse-event summary gives participants at risk and affected for serious and other events, plus a death count and selected serious-event terms with percentages. In general, participant-level affected counts and event-term percentages answer different questions from a total infection count. The registry does not clarify whether event-term percentages represent participants or events, whether participants could appear under multiple terms, or the observation rules for those summaries. No new rate or comparison can be derived from the posted numbers without those definitions.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary infection endpoint and secondary outcomes concerning delay in chemotherapy administration due to prolonged neutrophil recovery and response following induction without prophylactic G-CSF. It records three outcome measures and zero analysis entries. The registry record does not report how the primary and secondary outcomes were prioritized in statistical testing, whether any multiplicity adjustment was planned, or whether secondary outcomes were intended to be descriptive or inferential.
In general, testing several outcomes can increase the chance that at least one result appears notable by chance. A statistical plan can address this through a prespecified hierarchy, adjustment, or a clearly descriptive interpretation. None of those approaches is documented in the registry record. Because outcome results and analyses are not reported, no conclusion about multiplicity handling can be drawn from the listed outcomes alone.
The primary endpoint population is described as patients evaluable for the endpoint, but the number evaluable and the criteria for evaluability are not reported in the registry record. It also does not report whether all enrolled participants contributed to each outcome, how missing observations were handled, or whether any participants were excluded from an analysis. There is no reported intention-to-treat or per-protocol analysis population.
In general, missing outcome data can affect interpretation when the likelihood of missingness is related to participants' health or outcome experience. Statistical methods may use different assumptions for missing values, but the record does not identify any such method. The safest reading is that the available registry entry describes the intended endpoint population but does not document the analysis population or missing-data procedure.
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 |
|---|---|---|---|
| High Risk Neuroblastoma Patients | 11 | 12 | 91.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| High Risk Neuroblastoma Patients | 0 of 13 |
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 |
|---|---|
| High Risk Neuroblastoma Patients | 12 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | High Risk Neuroblastoma Patients |
|---|---|---|
| Febrile Neutropenia | Blood and lymphatic system disorders | 9 of 12 |
| bacteremia | Infections and infestations | 4 of 12 |
| Skin/soft tissue infection | Infections and infestations | 3 of 12 |
| Urinary tract infection | Infections and infestations | 2 of 12 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 12 |
| Sinusoidal obstruction syndrome | Hepatobiliary disorders | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | High Risk Neuroblastoma Patients |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 12 of 12 |
| Neutropenia | Blood and lymphatic system disorders | 12 of 12 |
| Thrombocytopenia | Blood and lymphatic system disorders | 12 of 12 |
| WBC decreased | Blood and lymphatic system disorders | 12 of 12 |
| Pain | Musculoskeletal and connective tissue disorders | 2 of 12 |
| ALT increased | Hepatobiliary disorders | 1 of 12 |
| Alk phos increased | Hepatobiliary disorders | 1 of 12 |
| AST increased | Hepatobiliary disorders | 1 of 12 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 12 |
| Rash | Skin and subcutaneous tissue disorders | 1 of 12 |
Serious events with the highest arm-level rate: febrile neutropenia (75.0%), bacteremia (33.3%), skin/soft tissue infection (25.0%), urinary tract infection (16.7%), respiratory failure (8.3%).
The registry lists 12 participants at risk for serious adverse events, with 11 affected, and 12 participants at risk for other adverse events, with 12 affected. It reports 0 deaths. These are descriptive safety summaries; they do not establish a comparative risk because the study has a single group and no concurrent control arm.
The listed serious adverse event terms and percentages are febrile neutropenia, 75.0%; bacteremia, 33.3%; skin/soft tissue infection, 25.0%; urinary tract infection, 16.7%; respiratory failure, 8.3%; and sinusoidal obstruction syndrome, 8.3%. The registry does not report the denominator or counting rules for each term, whether participants could have multiple terms, or whether these events occurred during cycles without growth-factor follow-up. Those percentages should not be treated as the primary infection endpoint.
In general, adverse-event summaries describe events recorded under the registry's reporting definitions. They do not, on their own, show that a treatment caused an event or indicate how event frequency would compare under another treatment strategy. The record does not provide comparative safety estimates or uncertainty intervals for these summaries.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this completed pilot examined induction chemotherapy for high-risk neuroblastoma without prophylactic G-CSF and specified infection incidence as its primary endpoint. It also records serious and other adverse-event summaries. Those details describe the study's scope and the safety information posted, but the primary endpoint's observed infection count is not reported in the registry record.
For that reason, the primary question cannot be answered quantitatively from the registry entry. The number of infections, the number of evaluable participants, and an uncertainty estimate are absent. The secondary outcomes are named, but their results are not reported in the registry record either. It would be inappropriate to treat the absence of a posted result as evidence that no infections, delays, or response events occurred.
The serious-event summaries show that adverse events were recorded in the safety group, and the record reports 0 deaths. The listed percentages convey the registry's reported summaries, but details about term-specific denominators and overlap are not reported. These figures cannot be used as a comparison with usual care because the design has no randomized control arm. Nor do they directly answer whether infections occurred during the specific cycles defined by the primary endpoint.
In general, a pilot can inform descriptions of study conduct and the types of events observed, but it is not necessarily suited to provide a precise comparative effect. The enrolled group numbered 13, while the safety summaries list 12 participants at risk. These are distinct recorded quantities; the registry does not explain the difference. They should not be combined to infer how many participants contributed to the primary endpoint.
The most defensible interpretation is narrow: the study posed a safety question about omitting prophylactic G-CSF during induction, but the registry record does not provide the primary infection result needed to evaluate that question. The posted adverse-event summaries remain descriptive and do not establish causality, comparative safety, or the effectiveness of any treatment strategy.
13. Why This Trial Matters Statistically
This study is statistically notable because its question concerns safety under a treatment approach without a concurrent randomized comparison. The design is single-group, and the registry lists allocation as NA. That distinction matters: an observed event count can describe what was recorded in the study, but a comparison between treatment strategies requires a suitable comparator or other design and analysis capable of addressing differences between groups.
The primary endpoint also illustrates why outcome definitions matter. The record specifies a number of infections in chemotherapy cycles not followed by hematopoietic growth factors, rather than simply a proportion of participants with any infection. A count can include repeat events, while a participant-level summary treats each participant as a single unit. The registry does not explain the event-counting rules, so the exact interpretation of a potential count is limited.
The endpoint is tied to a particular treatment context and observation period: through study completion, approximately 5 months. The background explains the rationale for studying the approach in relation to white blood cell production and low ANC after chemotherapy. That context helps identify the question but does not substitute for an outcome estimate. The registry does not report the infection count or its uncertainty.
The record also demonstrates the separation between a trial's planned question and the results actually posted. It identifies secondary outcomes and a completed status, but lists zero analysis entries and does not report those outcome results. Completion status alone does not indicate whether the primary endpoint met a prespecified threshold or how precise any estimate might be.
Finally, the safety figures underscore the need to distinguish broad adverse-event summaries from an endpoint-specific analysis. The serious-event terms and affected-participant summaries provide descriptive information, but their relation to the primary endpoint's cycle-level definition is not stated. Without a comparator, event definitions, and endpoint results, the available evidence should be interpreted as a record of a small pilot's scope and posted safety summaries, not as a comparative treatment estimate.
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 central limitation for interpretation is that the primary infection result is not reported in the registry record. The endpoint's number of infections, the number of evaluable participants, and any estimate of uncertainty are absent. Although the endpoint timeframe is given as approximately 5 months, the record does not provide detailed follow-up or event-counting rules.
The study is single-group, with allocation listed as NA and masking as none. It therefore has no concurrent randomized control group. Comparisons with routine practice or other populations would not be like-for-like comparisons and cannot establish that any observed events were caused by omitting prophylactic G-CSF. The record does not report an adjusted observational comparison either.
Enrollment was 13, while the safety summaries list 12 participants at risk. The registry does not explain the distinction or state how many participants were evaluable for the primary endpoint. It also does not report the analysis population, exclusions, missing-data methods, or endpoint evaluability criteria. This prevents clear reconstruction of who contributed to each result.
The listed serious-event percentages lack reported term-specific denominators and counting rules. The registry does not state whether a participant could contribute to multiple terms or whether the listed events occurred during the cycles covered by the primary endpoint. The safety summaries cannot therefore replace a primary endpoint analysis.
The registry lists three outcome measures and zero analysis entries, and does not report a detailed statistical plan, multiplicity approach, sample-size justification, or interim-analysis methods. The presence of planning assumptions does not establish that the trial was designed around a two-arm comparison. These omissions limit assessment of precision, analysis choices, and the relationship between planned and reported outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: High-Risk Neuroblastoma Chemotherapy Without G-CSF — NCT02786719.
- Registry record last updated 2020-03-12; status last verified 2020-03.
- Results first posted 2020-03-12.
- 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
The registry record describes a completed, single-group safety pilot of induction chemotherapy for high-risk neuroblastoma without prophylactic G-CSF. Enrollment was 13; allocation is listed as NA, masking as none, and phase as NA. The treatment description includes six chemotherapy courses and is limited to induction.
The primary endpoint is the number of infections in chemotherapy cycles not followed by hematopoietic growth factors, through study completion, approximately 5 months. The record does not report the infection count, the number evaluable, or an uncertainty estimate. Secondary outcomes address chemotherapy delay due to prolonged neutrophil recovery and response after induction, but their results are not reported.
Safety summaries list 12 participants at risk for serious events and 11 affected, 12 at risk for other events and 12 affected, and 0 deaths. The registry lists selected serious-event terms with percentages but does not provide their counting details. There are zero analysis entries. The record supports description of the study design and posted safety information, not a comparative treatment estimate.