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 Monoclonal Antibody Ch14.18, Sargramostim, Aldesleukin, and Isotretinoin After Autologous Stem Cell Transplant in Treating Patients With Neuroblastoma (NCT01041638), a na phase 3 study. The primary outcome is Percentage of Patients Who Experienced a Significant (CTC Grade 3-5) Nonhematologic Toxicity of Interest (Pain, Hypotension, Allergic Reactions, Capillary Leak Syndrome, or Fever)..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01041638 |
| Phase | Phase 3 |
| Condition | High Risk Neuroblastoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 105 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with high-risk neuroblastoma following myeloablative therapy and autologous stem cell transplant. The registry record reports enrollment of 105 participants. One participant did not receive treatment and was excluded from the primary outcome tabulation.
Intervention
The treatment group received a regimen combining Ch14.18, sargramostim, aldesleukin, and isotretinoin after transplant. The trial description emphasizes comprehensive safety and toxicity data, alongside additional efficacy data. The registry record does not provide a complete, usable account of every treatment schedule detail.
Comparator
There was no comparator group: this was a single-group study, with allocation listed as NA and masking listed as none. The study therefore does not provide a concurrent control for comparing the combination regimen with another treatment or with no treatment.
Primary question
The primary question was how often participants experienced a significant nonhematologic toxicity of interest—pain, hypotension, allergic reactions, capillary leak syndrome, or fever—at CTC grade 3-5 during up to 6 courses of therapy. The registry describes a descriptive assessment of these toxicities, including reporting by course, but does not report the primary endpoint result.
3. Trial Design
NCT01041638 is recorded as a phase III, single-group trial with no masking. Allocation is listed as NA, consistent with the absence of a randomized comparison. The registry record identifies the National Cancer Institute (NCI) as sponsor and reports enrollment of 105. The recorded start date is 2009-12-21, the primary completion date is 2013-12-31, and the status is completed.
The study evaluated Ch14.18 with sargramostim, aldesleukin, and isotretinoin after autologous stem cell transplant in patients with high-risk neuroblastoma. The registry’s arm description identifies this as a treatment regimen, but its schedule text is incomplete. A full schedule cannot be reconstructed from the registry record.
The primary outcome was a percentage of participants with specified severe nonhematologic toxicities during up to 6 courses. The registry describes descriptive summaries of adverse events and reporting of the percentage with each unacceptable toxicity code by course. One participant did not receive treatment and was not included in the primary outcome tabulation. The adverse-event summary separately lists 104 participants at risk for serious events and 104 at risk for other events.
Event-free survival and overall survival are listed as secondary outcomes, but their results are not reported in the registry record. The record also lists 3 outcome measures and 0 analyses. These details do not establish what statistical procedures were actually applied beyond the descriptive approach specified for the primary objective.
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 | Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin |
|---|---|
| STARTED | 105 |
| COMPLETED | 78 |
| NOT COMPLETED | 27 |
Reasons for not completing this period:
| Reason | Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin |
|---|---|
| Adverse Event | 4 |
| Death | 1 |
| Lack of Efficacy | 7 |
| Physician Decision | 4 |
| Withdrawal by Subject | 11 |
Analysis population for the primary outcome, as defined in the registry: There was one patient that did not receive treatment, represented on the baseline form as "withdrawal by patient", not included in outcome measure tabulation.
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 | Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin |
|---|---|---|
| Participants analysed | 105 | |
| Age, Continuous | years | 4.1 (1.1 to 27.5) |
| Age, Categorical: <=18 years | Participants | 103 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 46 |
| Sex: Female, Male: Male | Participants | 59 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 |
| Race (NIH/OMB): Asian | Participants | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 10 |
| Race (NIH/OMB): White | Participants | 82 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 10 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 9 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 87 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 9 |
| Region of Enrollment: United States | participants | 105 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Patients Who Experienced a Significant (CTC Grade 3-5) Nonhematologic Toxicity of Interest (Pain, Hypotension, Allergic Reactions, Capillary Leak Syndrome, or Fever)., measured over Up to 6 courses of therapy.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint concerns the percentage of participants who experienced at least one of the specified CTC grade 3-5 nonhematologic toxicities: pain, hypotension, allergic reactions, capillary leak syndrome, or fever. The assessment period was up to 6 courses of therapy. The endpoint description also states that reporting was intended to cover each unacceptable toxicity code by course.
A percentage is a way to express how common an event was within a defined group. Its interpretation depends on a clear numerator, denominator, event definition, and observation period. In general, a toxicity endpoint can characterize the safety experience during treatment, while separating event types and courses can show whether the reporting varies over treatment. These descriptions do not by themselves establish why an event occurred or whether it was caused by a particular treatment component.
The registry record says that descriptive analyses of the number and type of adverse events would address the primary objective. However, it does not report the primary endpoint percentage, counts for the specified toxicity categories, or results by course. It is therefore not possible to characterize the observed frequency of the primary endpoint from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a safety-focused, single-group study and defines a primary toxicity question, but it does not report the result for that endpoint. No percentage or participant count for the specified grade 3-5 nonhematologic toxicities is available in the record. The endpoint’s intended observation period was up to 6 courses, and the description says that toxicity reporting was organized by course. Without the corresponding results, the primary safety experience cannot be quantified from the registry entry.
The adverse-event summary does contain a list of serious-event terms with percentages. The listed terms include lymphocyte count decreased (75.0%), fever (66.3%), anemia (62.5%), platelet count decreased (55.8%), neutrophil count decreased (52.9%), and hypokalemia (49.0%). These are distinct from the unreported primary endpoint result: several are hematologic or laboratory terms, while the primary endpoint specifies selected nonhematologic toxicities. The registry record does not establish that the serious-event list is a substitute for the primary endpoint analysis.
The adverse-event group summary reports 104 participants at risk and 103 affected for serious events; for other events it reports 104 at risk and 104 affected. These group-level figures describe the adverse-event summary and should not be mistaken for the primary endpoint’s count. The record does not explain the relationship between those aggregate counts and the percentages listed for individual serious-event terms. A death count is not reported in the registry record.
Event-free survival and overall survival appear among the secondary outcomes, but no results for either are reported. The entry therefore does not support a description of survival outcomes. More broadly, because the study has no concurrent comparator, its data cannot directly estimate how the regimen’s safety or efficacy differs from another treatment. Descriptive data can report what was recorded in the treated group; they do not, on their own, isolate treatment effects from other influences.
Precision also cannot be assessed for the primary endpoint from the registry record: no confidence interval or other measure of uncertainty is reported for it. In general, a confidence interval describes a range of values compatible with the data under a statistical model. Without a reported estimate and its uncertainty, the primary toxicity frequency cannot be judged for precision. The listed adverse-event percentages should likewise be interpreted as registry summaries, not as evidence of a comparative treatment effect.
9. Statistical Methods Explained
The registry describes the primary objective as collecting comprehensive safety and toxicity data, as well as additional efficacy data. It states that descriptive analyses summarizing the number and type of adverse events would be performed. The stated reporting approach for the primary endpoint was to give the percentage reporting each unacceptable CTC toxicity code, by course. No inferential model, adjustment procedure, or detailed statistical analysis plan is reported in the registry record.
What a descriptive percentage communicates
In general, a percentage expresses the number of participants meeting an event definition relative to a specified denominator. The event definition matters: a percentage for any adverse event is not interchangeable with a percentage for a selected set of grade 3-5 nonhematologic toxicities. The denominator also matters. It may depend on who received treatment, who was eligible for a particular assessment, and whether each participant had adequate observation during the relevant period. The registry identifies one untreated participant as excluded from the primary outcome tabulation, but does not give the primary endpoint’s actual numerator or denominator.
Course-by-course reporting can help describe how adverse events were recorded over the treatment period. In general, however, course-specific percentages require clear definitions of which participants were considered at risk in each course and how repeated events in the same participant were handled. Those details are not reported in the registry record. The record’s reference to reporting by course does not establish that each course was analyzed as an independent group.
Uncertainty and comparison
In general, an estimate is more informative when accompanied by a measure of uncertainty, such as a confidence interval. A confidence interval conveys the range of values that remain reasonably compatible with the observed data under the chosen method. It does not guarantee that the true value falls inside that particular interval, and it does not address bias from missing observations or an unsuitable design. No confidence interval for the primary endpoint is reported in the registry record.
This trial used a single-group design. In general, a concurrent comparator can help distinguish outcomes associated with one treatment strategy from outcomes that might also occur under another strategy. Without such a group, observed events can be described in the enrolled group, but a between-treatment difference cannot be estimated directly. The registry lists allocation as NA and the model as single-group; it does not report a randomized comparison.
Over-dispersion and repeated observations
In general, over-dispersion means that observed variation is greater than a statistical model expects. It can arise when observations are more alike within participants or clusters than a simple model assumes. For repeated course-level toxicity data, observations from the same participant may be related; treating them as independent can understate uncertainty. This is a general statistical consideration, not a claim that over-dispersion was present or that a particular model was used in this trial. The registry record does not report a model for repeated observations or an assessment of over-dispersion.
Planning illustrations are not trial results
General planning calculations may explore how many participants would be needed to detect an effect under stated assumptions. Such calculations are not observed outcomes and do not constitute observed power. The registry’s planning assumptions describe an illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect set as a stated fraction of the observed effect. These are assumptions for a planning illustration, not a description of this trial’s single-group design. The registry record does not report the trial’s own sample-size justification.
For time-to-event planning, the record notes that illustrations use a total number of events, while other planning illustrations use participants per arm. This distinction is general: event-driven time-to-event designs focus on accumulated events, whereas other designs may be expressed as participant counts. It does not supply results for the listed event-free or overall survival outcomes. No such survival results are reported in the registry record.
Analysis choices that remain unclear
The registry does not report detailed rules for handling missing toxicity assessments, repeated events, changes in treatment exposure, or deviations from the planned assessment schedule. It also does not report whether any adjustment was made for multiple outcomes or comparisons. These omissions limit the ability to reproduce or fully evaluate the statistical analysis. The stated descriptive approach is the extent of the primary analysis method documented in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary toxicity outcome and two secondary outcomes: event-free survival and overall survival. It does not report whether the analysis plan controlled for multiplicity across these outcomes or across individual toxicity types. In general, testing many outcomes can increase the chance of finding a result that appears noteworthy by chance. A prespecified strategy for handling multiple tests helps clarify how evidence should be interpreted. No such strategy is reported in the registry record, and no survival results are reported.
Missing-data methods are also not reported. The registry notes that one participant did not receive treatment, described as a withdrawal by patient, and that this participant was excluded from the primary outcome tabulation. It does not state how other missing or incomplete toxicity assessments, if any, were handled. In general, missingness can affect an estimate when participants with recorded outcomes differ from those without recorded outcomes. The record does not provide information to assess whether this concern affected the primary endpoint.
The analysis population is not otherwise fully described. The adverse-event summary reports 104 participants at risk for serious events and 104 at risk for other events, while the primary outcome description identifies one untreated participant excluded from tabulation. The record does not explain the full relationship between these populations or provide a detailed analysis-population definition. The sample-size justification for the trial is not reported in the registry record; the described planning illustration is not the trial’s design rationale.
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 |
|---|---|---|---|
| Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin | 103 | 104 | 99.0% |
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 |
|---|---|
| Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin | 104 of 104 |
Most frequent serious adverse event terms
| Event term | Organ system | Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin |
|---|---|---|
| 53700-Lymphocyte count decreased | Investigations | 78 of 104 |
| 33900-Fever | General disorders | 69 of 104 |
| 13200-Anemia | Blood and lymphatic system disorders | 65 of 104 |
| 65800-Platelet count decreased | Investigations | 58 of 104 |
| 58300-Neutrophil count decreased | Investigations | 55 of 104 |
| 43100-Hypokalemia | Metabolism and nutrition disorders | 51 of 104 |
| 60600-Pain | General disorders | 46 of 104 |
| 43300-Hyponatremia | Metabolism and nutrition disorders | 42 of 104 |
| 20500-Catheter related infection | Infections and infestations | 33 of 104 |
| 43600-Hypotension | Vascular disorders | 33 of 104 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Chimeric Antibody 14.18 With GM-CSF, IL-2 and Isotretinoin |
|---|---|---|
| 33900-Fever | General disorders | 102 of 104 |
| 42600-Hypoalbuminemia | Metabolism and nutrition disorders | 101 of 104 |
| 13200-Anemia | Blood and lymphatic system disorders | 99 of 104 |
| 65800-Platelet count decreased | Investigations | 97 of 104 |
| 42700-Hypocalcemia | Metabolism and nutrition disorders | 96 of 104 |
| 43300-Hyponatremia | Metabolism and nutrition disorders | 95 of 104 |
| 15000-Aspartate aminotransferase increased | Investigations | 91 of 104 |
| 11600-Alanine aminotransferase increased | Investigations | 90 of 104 |
| 88500-White blood cell decreased | Investigations | 89 of 104 |
| 53700-Lymphocyte count decreased | Investigations | 87 of 104 |
Serious events with the highest arm-level rate: 53700-lymphocyte count decreased (75.0%), 33900-fever (66.3%), 13200-anemia (62.5%), 65800-platelet count decreased (55.8%), 58300-neutrophil count decreased (52.9%).
The adverse-event summary lists 104 participants at risk for serious events and 103 affected. It also lists 104 participants at risk for other events and 104 affected. The registry’s most frequently listed serious-event terms, with their reported percentages, are lymphocyte count decreased (75.0%), fever (66.3%), anemia (62.5%), platelet count decreased (55.8%), neutrophil count decreased (52.9%), and hypokalemia (49.0%).
These figures are adverse-event summaries, not the primary endpoint result. The primary endpoint was restricted to selected grade 3-5 nonhematologic toxicities and was intended to be reported by course. The individual serious-event percentages should not be interpreted as percentages for that endpoint. The registry record does not explain whether the listed percentages refer to participants with at least one event, event counts, or another calculation, so that denominator detail is not reported in the registry record.
The serious-event and other-event affected counts describe the group-level summary; they do not mean that every affected participant had the same event or severity. The record does not report a death count.
12. What the Result Does, and Does Not, Mean
The record describes a completed phase III, single-group study of a combination regimen after autologous stem cell transplant in patients with high-risk neuroblastoma. Its primary objective was safety-focused: to summarize selected serious nonhematologic toxicities by course over up to 6 courses of therapy. The record also lists event-free survival and overall survival as secondary outcomes.
What can be stated is limited to the study design, endpoint definition, and adverse-event summaries recorded in the registry. The primary toxicity percentage and the survival results are not reported in the registry record. The listed serious adverse-event terms and percentages provide a separate safety summary, but do not fill that gap or establish the primary endpoint frequency.
In general, a single-group study can describe outcomes among participants who received the regimen. Without a concurrent comparison group, it cannot show how those outcomes would differ under another treatment strategy. Even a clearly reported event frequency would be a description of this group and observation period, not by itself proof that the regimen caused or prevented an event.
The results also cannot establish comparative efficacy from the available record. Although additional efficacy data were an objective and survival outcomes are listed, their results are not reported. The entry therefore does not support conclusions about survival or about whether one treatment is better than another. No medical recommendation follows from these registry summaries.
13. Why This Trial Matters Statistically
This trial illustrates why the endpoint definition and the reported result must be read together. The primary outcome is specific: selected CTC grade 3-5 nonhematologic toxicities during up to 6 courses, with intended reporting by course. A general adverse-event summary is not interchangeable with that endpoint. Keeping those summaries distinct prevents broader adverse-event figures from being mistaken for the result of the prespecified safety question.
The design also matters to interpretation. A single-group study can document adverse events in the treated population, but it does not supply a concurrent treatment comparison. That limits the kinds of statistical claims available, regardless of the number of events recorded. A descriptive percentage answers how often something was recorded in a defined group; a comparative estimate requires a suitable comparison.
Finally, the entry shows the importance of reporting counts, denominators, uncertainty, and analysis populations. The registry identifies an untreated participant excluded from primary outcome tabulation and gives group-level adverse-event counts, yet it does not report the primary endpoint estimate or its uncertainty. These gaps make it harder to judge the precision and scope of the safety findings. Clear reporting is essential for researchers who need to distinguish observed summaries from conclusions about comparative effects.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The main limitation for statistical interpretation is that the primary endpoint result is not reported in the registry record. The endpoint definition and planned descriptive approach are available, but the corresponding percentage and counts are not. Confidence intervals or other measures of uncertainty for the primary endpoint are also not reported. As a result, the primary safety question cannot be answered quantitatively from the registry entry.
The single-group design has no concurrent comparator. Consequently, it cannot support a direct estimate of how the regimen’s outcomes differ from those under another treatment. The lack of masking is recorded, but the registry does not provide details about outcome assessment procedures that would clarify how toxicity reporting was conducted.
The registry identifies one participant who did not receive treatment and was excluded from primary outcome tabulation. It does not describe other missing assessments, the handling of incomplete follow-up, or the full analysis-population rules. The relationship between the primary endpoint population and the adverse-event summary populations is not fully explained.
Event-free survival and overall survival are listed as secondary outcomes, but their results are not reported. The record does not report multiplicity procedures, a detailed model for repeated course-level observations, or the trial’s sample-size justification. The adverse-event summary does not explain the denominator basis for the individual serious-event percentages, and a death count is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Monoclonal Antibody Ch14.18, Sargramostim, Aldesleukin, and Isotretinoin After Autologous Stem Cell Transplant in Treating Patients With Neuroblastoma — NCT01041638.
- Registry record last updated 2026-06-24; status last verified 2026-05.
- Results first posted 2015-02-19.
- 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
NCT01041638 is recorded as a completed phase III, single-group trial sponsored by the National Cancer Institute (NCI). It enrolled 105 participants with high-risk neuroblastoma and evaluated Ch14.18, sargramostim, aldesleukin, and isotretinoin after autologous stem cell transplant. One participant did not receive treatment and was excluded from primary outcome tabulation.
The primary endpoint concerned selected CTC grade 3-5 nonhematologic toxicities over up to 6 courses. The registry describes descriptive reporting by course but does not report the primary endpoint result. Event-free survival and overall survival are listed as secondary outcomes, without results. Serious and other adverse-event group summaries are recorded, as are percentages for several serious-event terms. The record has no comparator group, and the listed survival and primary endpoint results are not reported in the registry record.