Molecular-Guided Therapy for Childhood Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Molecular-Guided Therapy for Childhood Cancer (NCT02162732), a na na study. The primary outcome is Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers..

2. Clinical Question

The central statistical question is about completing a process within clinical care, rather than estimating a treatment effect against a concurrent control. The primary endpoint is days to treatment, with a mean and full range specified. The registry does not report the observed mean or range, so the timing and its variability cannot be assessed from the posted primary outcome information.

3. Trial Design

The registry describes a completed, single-group study of molecular-guided therapy for relapsed and refractory childhood cancers. Allocation is listed as NA and masking as none. No randomized comparison is described, and there is no like-for-like control arm in the record. The listed intervention is “Guided Therapy,” classified as a device. Its description says that treatment can use any therapeutic combination of up to 4 agents, provided the medications are contained in the study report.

The study purpose is to test the feasibility of technologies that assess a tumor’s molecular makeup. The brief summary describes a genomic report based on DNA exomes and RNA sequencing, intended to support understanding of cancers and potentially inform future treatment selection. The primary endpoint definition follows the pathway from enrollment through genomic analysis and reporting, a tumor board decision, treatment review and treatment start, ending with completion of one cycle of therapy.

The record lists an enrollment of 186. Separately, the arm description says that a total of 200 neuroblastoma, brain tumor and rare tumor patients will be treated. These are different figures in the registry record; it does not explain their relationship. The safety entry reports 144 participants at risk for each of its adverse-event categories, which is another count and should not be assumed to represent the enrolled population as a whole.

The study start is listed as 2014-07-08, primary completion as 2024-01-18, and status as COMPLETED. The registry lists one primary outcome and three secondary outcomes: unexpected adverse events, overall response rate among participants with radiologically assessable disease, and progression-free survival compared with previous chemotherapy regimens since relapse for each participant. The registry record does not report a protocol-level allocation scheme, detailed statistical analysis plan, or explanation of the enrollment figures.

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

Reasons for not completing this period:

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: Subjects that started at least one dose of study therapy

6. Primary Endpoint

The registry lists the primary outcome as Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers., measured over 2 years.

The primary endpoint is days to treatment, summarized as a mean with a full range. Its timeframe is listed as 2 years. The feasibility definition is a sequence of milestones: enrollment onto the study, completion of genomic profiling and report generation, a tumor board with a treatment decision, treatment review, treatment start and completion of one cycle of therapy. The endpoint therefore concerns the time required to move through the specified profiling and treatment process.

In general, a time measure can describe how quickly a process is completed, while a mean gives an average across participants and a full range gives the lowest and highest observed values. These summaries answer different questions: the mean characterizes average timing, while the range conveys the spread between the most extreme observations. A range alone does not show how observations are distributed between those extremes.

The registry specifies the endpoint’s unit, summary measure and feasibility milestones, but the observed mean and full range are not reported in the registry record. The record therefore does not establish how long the process took for participants, how variable the timing was, or what proportion completed every milestone. It also does not report a benchmark defining an acceptable number of days. Without those results and a stated threshold, the endpoint definition can be described, but the degree of feasibility cannot be judged from the posted primary outcome information.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The statistical information in the registry record is limited. It identifies the primary outcome as a mean number of days, with a full range, and lists secondary outcome topics. It does not report analysis entries or a detailed statistical plan. The explanations below are general descriptions of the methods and concepts named in the record, not claims about analyses that were performed.

What does a mean and full range describe?

In general, the mean is the arithmetic average of observed values. It can summarize the typical timing across participants, but it may be influenced by unusually short or long times. A full range reports the lowest and highest observed values, indicating the span of the observations. The range does not reveal how many participants had values near either endpoint or how values were distributed in between. For a time-to-treatment outcome, the mean and range can therefore provide complementary descriptions, but neither alone describes the full shape of the data.

The record specifies these summaries but does not post their observed values. It also does not report the number of participants contributing to the primary endpoint, how incomplete milestone sequences were handled, or whether participants who did not complete a cycle were included in the timing summary. Those analysis details are not reported in the registry record.

How should the feasibility definition be understood?

In general, a feasibility outcome evaluates whether a specified process can be carried out, rather than directly measuring comparative treatment benefit. Here, the stated sequence includes enrollment, genomic profiling and report generation, tumor board discussion and treatment decision, treatment review, treatment start and completion of one cycle. These milestones make the operational definition concrete. But the record does not give separate completion counts for each milestone or specify a threshold for success. A defined process endpoint is not, by itself, evidence that the process was completed within an acceptable time or for a particular proportion of participants.

What do the planning assumptions mean?

The registry describes planning illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. Those assumptions are not a description of this trial’s design: the record describes a single group and no randomized comparator. The illustrations should therefore be understood as hypothetical planning scenarios, not as evidence that the study used equal allocation or as observed power. The record says that the effect to detect is expressed as a stated fraction of the observed effect, but it does not report a trial design justification or a sample-size calculation as part of the trial’s own plan.

In general, a planning calculation asks how much information might be needed to detect a specified effect under particular assumptions. It does not establish that a study had that amount of information, that its assumptions were met, or that a result is likely to be true. Sample-size illustrations are not observed power. Because the study is single-group, a two-arm illustration should not be read as a description of the enrolled study or a direct analysis of its participants.

What does the time-to-event planning description mean?

The planning note says that time-to-event illustrations use the total number of events, with the Schoenfeld method, while other illustrations give participants per arm. In general, time-to-event methods use event information and account for the timing of events and censoring, rather than relying only on a count of participants. Censoring occurs when an event time is not observed during the relevant follow-up. The record does not state the assumptions, event counts or methods used for an actual time-to-event analysis of progression-free survival. Nor does it report an analysis comparing progression-free survival between treatment periods.

Why can over-dispersion matter in general?

For count outcomes, over-dispersion means that observed variability is greater than a model assumes. If it is ignored, uncertainty estimates can be too narrow and tests can be misleading. This is a general statistical issue; the registry record does not state that an over-dispersion assessment or count model was used in this study. The primary endpoint is described as days, not as a count outcome, and no model for it is reported.

What can be inferred from the absence of analysis entries?

The record lists zero analysis entries. This means the registry record does not present analysis results in that area; it does not establish why results are absent or whether analyses were conducted outside the registry. No inferential procedure, confidence interval, hypothesis test, covariate adjustment, or subgroup analysis is described in the posted record. In general, a confidence interval expresses uncertainty around an estimate under a specified method. Since no primary endpoint estimate or interval is posted, the precision of the timing estimate cannot be evaluated. The record’s outcome definitions and planning assumptions should not be mistaken for observed statistical results.

10. Multiplicity, Missing Data and Analysis Populations

The registry identifies a primary outcome and three secondary outcomes, but it does not report a multiplicity strategy. In general, multiplicity arises when several outcomes or hypotheses are assessed: interpreting many tests without an appropriate plan can increase the chance of a chance-positive finding. A prespecified hierarchy, adjustment method or clear distinction between confirmatory and exploratory analyses can help frame interpretation. None of those details is reported in the registry record, so it is not possible to determine how any multiple testing was handled.

Missing-data procedures are also not reported in the registry record. The primary endpoint requires completion of several milestones, but the record does not say how participants with incomplete profiling, missing dates, no treatment start or incomplete cycle information were handled. It does not describe censoring rules or the treatment of participants who could not be evaluated for radiologic response. For progression-free survival, the record does not provide follow-up or censoring details. In general, missingness can affect both the number of participants contributing to an estimate and the interpretation of that estimate; the impact depends on why information is missing and how the analysis handles it.

The analysis populations are not described. The record does not state whether outcomes were analyzed among everyone enrolled, only participants with completed profiling, those who began guided therapy, or another defined group. The enrollment figure of 186, the arm description’s stated total of 200, and the adverse-event risk-group count of 144 are not reconciled in the registry record. These counts should not be treated as interchangeable analysis denominators.

Without a reported analysis plan, missing-data rules, or defined analysis populations, the reader cannot assess whether reported outcome estimates would represent all enrolled participants or a selected subset. Since outcome estimates are not posted, that assessment is not possible for the primary or secondary outcomes.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry reports 0 participants with serious adverse events among 144 at risk in the Guided Therapy group. For other adverse events, it reports 6 affected among 144 at risk. It separately lists 3 deaths. The serious-adverse-event terms list is empty. These counts describe what is entered in the registry record; they are not comparative risk estimates, and the record does not provide a control group for comparison.

In general, “at risk” gives the denominator used for a reported event category, while “affected” gives the number of participants counted with that category. Such counts do not describe event severity beyond the category label, timing, duration, recurrence, or attribution unless those details are reported. The record does not explain whether the deaths were related to adverse events, treatment, cancer progression or another cause. It also does not explain why the safety denominator is 144 rather than the separate enrollment figure of 186 or the arm description’s total of 200.

The reported absence of participants with serious adverse events should not be expanded into a claim about all possible harms or about safety relative to another treatment. The registry provides no serious-event terms and no comparative safety analysis. These figures are limited to the categories, counts and denominators entered in the record.

12. What the Result Does, and Does Not, Mean

The record describes a study designed to test whether molecular profiling could be used in a process intended to support real-time treatment decisions for children with relapsed or refractory cancers. Its primary endpoint definition focuses on the time needed to progress from enrollment through profiling, report generation, tumor board review, treatment decision and treatment start, ending with completion of one cycle. This provides a clear description of the process the study intended to assess.

The record does not report the observed primary endpoint mean or full range. It therefore does not show how long the process took or how much timing varied. The definition also does not provide a posted completion rate or an explicit threshold for acceptable feasibility. The registry identifies secondary outcomes for adverse events, response and progression-free survival, but does not report analysis results for them. As a result, the posted information does not establish the level of feasibility, response, or progression-free survival observed in the study.

The single-group design is important. Without a concurrent control group, the registry record cannot support a randomized comparison of guided therapy with another treatment approach. The description of comparing progression-free survival with previous chemotherapy regimens is a within-participant historical comparison, not a concurrent randomized comparison. In general, changes across treatment periods can reflect differences in patient status, timing, follow-up or other factors, in addition to treatment.

The safety counts should also be kept within their limits: 0 serious adverse events among 144 at risk, 6 other adverse events among 144 at risk and 3 deaths. The record does not describe the relationship among these categories or provide event terms for serious events. These figures do not establish comparative safety or explain the causes of deaths.

Thus, the registry record communicates the intended feasibility question and some descriptive safety counts, but its posted results do not permit a quantitative conclusion about the primary endpoint or the listed secondary outcomes. A study purpose, endpoint definition and planning illustration are not substitutes for reported observed results.

13. Why This Trial Matters Statistically

This study raises statistical questions that are important when molecular testing is linked to treatment decisions. The feasibility endpoint is a process measure: it tracks whether profiling, reporting, clinical review and treatment steps can be completed, and how many days the sequence takes. Such outcomes can help distinguish the practical delivery of a testing strategy from its potential clinical effects. They also make clear that a molecular report alone is not the whole process; the endpoint definition includes review and treatment milestones.

The design also illustrates why the target question and comparison structure need to be kept distinct. A single-group study can describe what happened among participants in the study, but it does not provide a concurrent randomized estimate of what would have happened under another approach. The registry’s reference to previous chemotherapy regimens for progression-free survival makes the importance of comparison methods especially apparent. Historical comparisons can be informative, but interpretation depends on how periods and participants are made comparable, and those methods are not reported in the registry record.

Another statistical issue is the distinction between a defined outcome and a reported result. A precise endpoint definition is useful, but it cannot establish the observed time to treatment, variability, completion rate or treatment outcome. The posted record lists outcome topics without analysis entries, leaving the principal feasibility result and secondary outcome estimates unavailable for interpretation.

Finally, the record contains different participant counts for enrollment, the arm description and safety risk groups. Clear denominators are essential for interpreting outcomes and understanding which participants contributed data. The record does not reconcile these counts. Together, the design and reporting details show why registry descriptions, observed results and analysis methods each serve different purposes in statistical interpretation.

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 of the posted record is that it does not report the primary endpoint result. The endpoint is defined as mean days to treatment with a full range, but neither summary is posted. The registry also provides no analysis entries, so numerical results for the listed secondary outcomes are not available in the record.

The single-group design does not provide a concurrent control comparison. The description of comparing progression-free survival with previous chemotherapy regimens does not make the study randomized or create a like-for-like control group. The registry does not report methods for accounting for differences between treatment periods or participants.

Several operational details remain unclear. The enrollment count is 186, while the arm description states that a total of 200 patients will be treated; the record does not explain the difference. The adverse-event denominator is 144, and its relationship to the other counts is not reported. The record does not specify analysis populations, missing-data handling, endpoint completion counts, a feasibility threshold, or the approach to follow-up and censoring.

The registry describes the intervention as guided therapy, including combinations of up to 4 agents when the medications are contained in the study report. It does not provide the treatment combinations received by participants or analysis results by regimen. The record also does not report a detailed statistical plan, multiplicity handling, interim analyses, or the rationale for the study’s planned enrollment. These absent details limit assessment of both the design and the precision or robustness of any results.

These limitations concern what can be interpreted from the registry record. They do not establish whether additional information exists outside the record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Molecular-Guided Therapy for Childhood Cancer — NCT02162732.
• Registry record last updated 2025-11-03; status last verified 2025-10.
• Results first posted 2024-04-05.
• 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.

18. Record Summary

The registry describes NCT02162732, “Molecular-Guided Therapy for Childhood Cancer,” as a completed, single-group study of molecular-guided therapy for relapsed and refractory childhood cancers. Its stated purpose is to assess the feasibility of tumor profiling using DNA exomes and RNA sequencing to support treatment decisions. The primary outcome is days to treatment, summarized by a mean and full range, with a feasibility definition that ends after completion of one cycle.

The record lists enrollment of 186, while the arm description states a total of 200 patients; the relationship between those figures is not explained. The safety entry reports 0 serious adverse events among 144 at risk, 6 other adverse events among 144 at risk, and 3 deaths. The primary endpoint result, secondary outcome analyses and detailed statistical methods are not reported in the registry record. Consequently, the posted information describes the study question and some safety counts but does not establish the observed feasibility outcome or comparative treatment effects.