Stem Cell Radiotherapy and Temozolomide for Newly Diagnosed High-grade Glioma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Stem Cell Radiotherapy and Temozolomide for Newly Diagnosed High-grade Glioma (NCT02039778), a na na study. The primary outcome is Overall Survival.

2. Clinical Question

3. Trial Design

The STRONG trial (NCT02039778) is described as a prospective Phase I/II trial in its official title. The phase field itself is listed as NA, so the registry record does not provide a phase classification in that field. The study model is single group, allocation is NA, and masking is none. There is no randomized concurrent comparison in the registry record.

The recorded intervention combined stem cell radiotherapy with temozolomide for newly diagnosed high-grade glioma. The radiation schedule is described as 2.0 Gy daily, 5 days per week, for a total of 60.0 Gy over 6 weeks. Intensity modulated radiation therapy was mandated, and proton therapy, preferably intensity-modulated proton therapy, was an acceptable modality. The primary outcome description says treatment was followed by post-radiation temozolomide. The full temozolomide schedule is not reported in the registry record.

Enrollment was 4. The registry lists Beth Israel Medical Center as sponsor, a start date of 2013-12, primary completion of 2015-12, and status as terminated. The reason for termination is not reported in the registry record. A target enrollment, recruitment history, and explanation of whether enrollment ended as planned are not reported in the registry record.

The primary outcome was overall survival, with a timeframe of 12 months and a measurement described as a count of participants. Secondary outcomes listed are adverse events as a measure of safety and tolerability, neurocognition, and quality of life. The record lists 5 outcome measures, but other outcome-measure details are not reported in the registry record. It does not provide a detailed statistical analysis plan or explain how the historical comparison mentioned in the primary outcome description would be conducted.

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.

6. Primary Endpoint

The registry lists the primary outcome as Overall Survival, measured over 12 months.

The registered primary endpoint is overall survival at 12 months, expressed as a count of participants. Overall survival is used in clinical research to characterize survival over a specified follow-up period. As a general statistical concept, it is clinically interpretable, but the estimate depends on a clear definition of the analysis population, the time origin, follow-up, and how incomplete observation is handled.

The registry record names the endpoint and timeframe but does not report a survival count, an estimate, a confidence interval, or a statistical test. It also does not report an operational definition of the endpoint, the time origin, or details about censoring and follow-up completeness. The outcome description says the result would be compared with historical controls, but those controls and the comparison method are not reported in the registry record.

A count alone would not describe precision or establish how the result compares with another group. General statistical interpretation would usually consider the denominator and uncertainty around an estimate, as well as whether the groups are sufficiently comparable. Those details are not reported for the primary outcome. Therefore, the endpoint can be described from the registry record, but its outcome cannot be evaluated from the record’s reported results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a prospective study with a single-group model. Allocation is listed as NA, and masking as none. These fields indicate that the record does not describe a randomized allocation or masking procedure. General statistical context: randomization can help balance known and unknown factors between concurrent groups, while masking can reduce some forms of biased outcome assessment. Neither feature is documented for this study as a controlled comparison. The registry’s stated single-group model means the treatment cohort itself is the basis of the study description.

What does a single-group design allow?

A single-group study can document treatment delivery, follow-up, and outcomes among enrolled participants. It can also identify practical issues and describe observed events. General statistical context: without a concurrent control group, differences from a historical benchmark can be influenced by differences in patient selection, care, outcome definitions, follow-up, or other circumstances. The STRONG outcome description mentions comparison with historical controls, but the control source, comparability criteria, and analysis are not reported in the registry record. The historical comparison therefore cannot be appraised from the record.

How should overall survival be understood?

Overall survival is the named primary outcome, with a timeframe of 12 months and a measurement described as a count of participants. General statistical context: survival outcomes concern the time until an event, commonly death, and analyses may account for participants whose follow-up ends before the observation period is complete. A confidence interval, when reported, describes statistical uncertainty around an estimate under the analysis assumptions; it does not remove biases in design or measurement. Here, the registry does not report a survival count, estimate, interval, time origin, censoring approach, or analysis population. It is therefore not possible to assess the precision or analytic handling of this endpoint from the registry record.

What does the analysis information say?

The record lists 0 analyses. It does not report a statistical analysis plan, model, test, adjustment strategy, or hypothesis-testing procedure for the primary endpoint. General statistical context: inferential methods are most interpretable when the analysis is prespecified and the outcome definition, population, and handling of incomplete observations are clear. Those details are not reported in the registry record. No p-value or confidence interval for the primary outcome is reported, so there is no reported inferential result to interpret.

What is the role of the planning illustration?

The planning description says calculations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect set as a stated fraction of an observed effect. It distinguishes between event totals for time-to-event planning and participants per arm for other planning illustrations. Those are planning assumptions, not observed outcomes. General statistical context: a sample-size calculation estimates the sample required for a chosen design and assumptions; it is not an estimate of the probability that a completed study found an effect. It must not be called observed power. The described equal-allocation, two-arm assumptions also differ from the registry’s single-group model. The underlying observed effect and the resulting planning values are not reported in the registry record.

Why does dispersion matter for some outcomes?

General statistical context: for count outcomes, variability may exceed what a simple model assumes; this is called over-dispersion. If it is ignored, standard errors and confidence intervals may be misleading. The registry identifies the primary measure as a count of participants, but does not report a model, a dispersion assessment, or an uncertainty estimate. There is no basis in the record to say whether an over-dispersion adjustment was relevant or used for any outcome.

What can be concluded from the safety counts?

The registry records adverse-event counts, including serious adverse events and deaths. General statistical context: event counts and the number at risk describe frequency within the recorded group, while causal attribution requires more information about timing, clinical review, and alternative explanations. The registry does not report such details for the listed events. The recorded safety figures therefore should be read as descriptive counts, not as a causal estimate or a comparison with another treatment group.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Stem Cell Radiotherapy and Temozolomide")
n      <- c(4)
events <- c(1)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Stem Cell Radiotherapy and Temozolomide  4  1  25.0  0.6  80.6  4.6  69.9  5.3  71.6

11. Multiplicity, Missing Data and Analysis Populations

The registry lists overall survival as the primary outcome and names secondary outcomes concerning adverse events, neurocognition, and quality of life. It records 5 outcome measures in total. Other outcome-measure details are not reported in the registry record. The record does not explain how the outcomes were prioritized beyond naming the primary endpoint, or how conclusions would account for testing more than one outcome.

General statistical context: when researchers examine several outcomes or perform multiple statistical tests, the chance of at least one apparently unusual result can increase. A prespecified hierarchy or multiplicity adjustment can help clarify how evidence should be interpreted. No multiplicity strategy is reported in the registry record. Since no outcome analyses are listed, the record does not show whether or how multiplicity affected any reported inference.

Missing-data methods and analysis populations are also not reported in the registry record. There is no description of how incomplete follow-up, unavailable neurocognitive assessments, or missing quality-of-life information would be handled. The record does not say whether analyses were intended to include all enrolled participants or a defined subset. These details are important because the included participants and the handling of unavailable observations can affect estimates and their uncertainty.

The record also does not report a statistical analysis plan, a sample-size justification for the trial, or interim-analysis procedures. The planning illustration has stated assumptions, but it does not fill in these missing protocol details or establish what analysis was conducted. In the absence of reported results and methods, no conclusions about multiplicity control, missing-data bias, or analysis-population choices can be drawn.

12. 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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: death (50.0%).

The registry records 2 participants with serious adverse events among 4 at risk, and 4 participants with other adverse events among 4 at risk. It also records 2 deaths. “Death” is listed as the leading serious adverse-event term at 50.0%. These are the counts and percentage entered in the registry; the record does not provide a fuller event-level breakdown.

These figures are descriptive. General statistical context: an adverse event occurring during a study is not, by itself, proof that the intervention caused it. Establishing attribution requires clinical judgment and details about timing, alternative causes, and how the event was assessed. Such details are not reported in the registry record. The record also does not clarify whether participants counted among those with serious adverse events overlap with those counted for other adverse events, so the categories should not be treated as mutually exclusive.

The small enrolled group and the absence of a concurrent control mean these counts cannot establish a comparative safety rate. The record does not report event timing, severity grading beyond the serious/non-serious categories, treatment attribution, or follow-up details for deaths. The counts therefore describe recorded safety experience, but they do not estimate comparative risk or establish causation.

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

The registry record establishes that the STRONG trial studied ScRT with temozolomide in newly diagnosed high-grade glioma and set overall survival at 12 months as its primary outcome. It also documents a single-group design, enrollment of 4, termination status, and selected adverse-event counts. These registry entries describe the study and its recorded safety information.

The record does not report the primary survival result, an effect estimate, a confidence interval, or an analysis. It therefore does not show how many participants met the endpoint, how precisely survival was estimated, or how the result compared with historical controls. The absence of a reported result is not evidence that the treatment had a particular effect in either direction.

The design also limits what can be inferred comparatively. General statistical context: observed outcomes in a single group can reflect the intervention, the characteristics of participants, other care, and the natural course of disease. Without a concurrent randomized comparison or clearly described historical controls, those influences cannot be separated using the information in the registry record. The outcome description’s reference to historical controls does not itself establish a valid comparative analysis.

The adverse-event counts indicate what the registry records within the treated group. They do not establish that ScRT or temozolomide caused the events, and they do not provide a comparative estimate of harms. Details needed to examine attribution and event timing are not reported in the registry record.

Accordingly, the record supports a careful account of the trial’s intended question, design, and reported safety counts. It does not support a conclusion about treatment benefit, comparative effectiveness, or comparative safety. It also does not provide enough statistical detail to judge the precision of the primary endpoint or the handling of missing observations and multiple outcomes.

14. Why This Trial Matters Statistically

The trial is statistically notable for the gap between a clinically important question and the information available for evaluating its answer. Overall survival at 12 months was named as the primary endpoint, but the registry record does not report an outcome count or analysis. A clearly stated endpoint helps define what a study intends to assess; without a reported result and uncertainty measure, readers cannot judge the observed outcome or its precision.

The design is also central to interpretation. The registry identifies the study as prospective and single group, with allocation listed as NA and no masking. A treatment cohort can describe observed outcomes, but it does not by itself provide a randomized comparison. The outcome description mentions historical controls, yet the control data and comparison method are not reported in the registry record. This distinction matters because a historical benchmark can differ from the treated cohort in ways that affect outcomes.

Enrollment was 4, and the record lists serious and other adverse-event counts. Small cohorts can offer descriptive information, but general statistical context is that estimates from a small amount of observed data may be imprecise. The registry provides no uncertainty estimates for these counts or for the primary endpoint, and it does not report a comparative safety analysis.

The registry also records secondary interests in neurocognition and quality of life, outcomes that complement survival and safety when assessing patient experience. Yet their results and methods are not reported in the registry record. The listed outcome domains show the breadth of questions the study intended to address, while the absence of reported analyses limits conclusions about each domain.

These features make transparent outcome reporting, clear analysis methods, and explicit treatment of missing data and multiplicity especially important. The registry record does not contain those details. Its statistical importance lies in showing how design and reporting determine what can—and cannot—be inferred from a clinical study.

15. 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.

Several trial-specific limitations constrain interpretation. The registry describes a single-group study, with allocation listed as NA and no masking. There is no concurrent randomized control group. Although the primary outcome description mentions historical controls, details of those controls and the planned or completed comparison are not reported in the registry record. Any comparative interpretation would therefore require information not available in the record.

Enrollment was 4, and the study status is terminated. The reason for termination, recruitment history, and whether enrollment or follow-up ended as intended are not reported in the registry record. Participant characteristics, eligibility details, and follow-up completeness are also not reported. Without these details, it is difficult to judge the context of the observed safety counts or the population to which any results might apply.

The primary endpoint is identified as overall survival at 12 months, but its result and analysis are not reported. The registry does not specify the endpoint’s operational definition, time origin, censoring method, or analysis population. The record lists secondary outcomes in safety, neurocognition, and quality of life, but does not report their results or detailed measurement methods.

Statistical methods, missing-data handling, multiplicity procedures, and a trial-specific sample-size justification are not reported in the registry record. The planning illustration has assumptions for a two-arm calculation, while the registered study model is single group; those planning assumptions do not establish the trial’s design or its statistical precision. These gaps mean that the record can describe the study and selected safety counts, but cannot support a full assessment of outcome reliability or comparative effects.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Stem Cell Radiotherapy and Temozolomide for Newly Diagnosed High-grade Glioma — NCT02039778.
• Registry record last updated 2017-06-05; status last verified 2017-04.
• Results first posted 2017-06-05.
• 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.

19. Record Summary

NCT02039778, titled the STRONG trial, is a prospective Phase I/II study in its official title of stem cell radiotherapy and temozolomide for newly diagnosed high-grade glioma. The registry lists a single-group model, allocation as NA, masking as none, enrollment of 4, and status as terminated. The sponsor is Beth Israel Medical Center; the recorded start is 2013-12 and primary completion is 2015-12.

The primary outcome is overall survival at 12 months, measured as a count of participants. Listed secondary outcomes concern adverse events, neurocognition, and quality of life. The registry records 2 participants with serious adverse events among 4 at risk, 4 with other adverse events among 4 at risk, and 2 deaths; “Death” is listed as the leading serious-event term at 50.0%.

No primary outcome result or analysis is reported in the registry record. Details of the historical comparison, statistical analysis plan, missing-data methods, and reason for termination are not reported.