Sodium Fluorescein-Guided Resection of Pediatric Neurosurgical Tumors: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sodium Fluorescein-Guided Resection of Pediatric Neurosurgical Tumors (NCT03752203), a na na study. The primary outcome is Percent of Tumor Resected: Intracranial Lesions.

2. Clinical Question

The study’s stated rationale is that sodium fluorescein, described as useful for intraoperative guidance in adult neurosurgical tumors, could aid resection of pediatric neurosurgical tumors. That rationale motivates the clinical question but is not a finding from this study. With no comparator and no reported primary-outcome measurements, the record cannot establish whether the approach changes resection extent relative to another method. It also cannot establish how well any observed extent of resection would generalize to pediatric neurosurgical patients beyond this study.

3. Trial Design

This registry record describes NCT03752203, titled “Sodium Fluorescein-Guided Resection of Pediatric Neurosurgical Tumors.” The sponsor is the University of Colorado, Denver. The study is classified as an other type of study, with phase listed as NA. Its design is single-group; allocation is NA and masking is none. These entries describe a study without a randomized control arm or a listed comparator group.

The recorded intervention includes sodium fluorescein and microscopic resection. The arm description combines sodium fluorescein with an FDA-approved operative microscope equipped with excitation and barrier filters. The record does not provide a separate arm receiving an alternative procedure, nor does it describe a controlled comparison. Accordingly, differences between treatment groups cannot be estimated from this design.

Enrollment is listed as 2. The recorded study start date is 2021-08-11, and primary completion is listed as 2023-06-29. The study status is terminated. The primary-outcome description states that the study was terminated due to lack of patients and funding. The record does not report the date of termination or further details about the reasons.

The primary outcome was percent of tumor resected for intracranial lesions, assessed from pre-operative to post-operative imaging, with the timeframe described as pre-op assessment to outpatient follow up, up to 6 weeks. The registry says the data were not collected or analyzed, and the outcome data table could not be completed because of lack of data. Although 3 outcome measures are listed, the record does not report analyzed results for the primary outcome. It lists 0 analyses. Details such as the intended analysis population, a statistical analysis plan, and any sample-size justification are not reported in the registry record.

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

Analysis population for the primary outcome, as defined in the registry: Data not collected or analyzed and therefore cannot be reported . Study was terminated due to lack of patients and funding. The Outcome Measure Data Table is not able to be completed due to lack of data.

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 Percent of Tumor Resected: Intracranial Lesions, measured over Pre-op assessment to outpatient follow up, up to 6 weeks.

The primary endpoint is titled “Percent of Tumor Resected: Intracranial Lesions.” It is intended to quantify the extent of tumor resection using pre-operative and post-operative imaging. The registry specifies volumetric imaging tools for intracranial lesions and gives a timeframe from pre-op assessment to outpatient follow up, up to 6 weeks.

In general, an imaging-based measure of resection extent can describe the amount of tumor removed as assessed from scans. Such a measure is clinically relevant to a surgical study because it focuses on the intended procedural outcome rather than relying only on a general impression of the operation. The registry does not specify the endpoint’s calculation formula, measurement units, image-review procedures, or rules for handling imaging that may be unavailable or uninterpretable; these details are not reported in the registry record.

Most importantly, the registry states that the outcome data were not collected or analyzed. It therefore gives no participant-level measurements, summary estimate, measure of variability, confidence interval, or statistical test for this endpoint. The endpoint is described as a planned measure, not a reported result. No conclusion about the extent of tumor resection in the enrolled participants can be drawn from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design estimate?

The registry classifies this study as single-group, with allocation listed as NA and masking as none. In general, a single-group study can describe outcomes among participants who received the intervention. It does not directly estimate how those outcomes would have differed under a separate comparator treatment. A control group helps distinguish an intervention-associated outcome from changes or influences that might also occur without that intervention. No such group is listed in this record.

For this study, the described endpoint uses imaging before and after resection. In general, paired measurements relate two observations from the same participant, which can make each participant’s pre-operative assessment a reference for their post-operative assessment. That within-participant structure is not the same as a between-group comparison. The registry does not state a statistical procedure for analyzing these measurements, and the outcome data were not collected or analyzed.

What would an endpoint estimate mean?

A statistical estimate is a numerical summary of the quantity being studied. For an imaging-based resection endpoint, an estimate would summarize the extent of resection for the relevant participants under the specified measurement approach. A measure of uncertainty, such as a confidence interval, would describe the range of values reasonably compatible with the data under the analysis assumptions. In general, a wider interval indicates less precision, while a narrower interval indicates greater precision. This record reports no endpoint estimate or confidence interval, so neither the amount nor the precision of any resection result can be assessed.

The title names the outcome as percent of tumor resected, but the registry does not specify the formula used to derive that percentage. It describes volumetric imaging tools, but does not report image segmentation procedures, who assessed the images, whether assessments were blinded, or how disagreements were resolved. Those measurement and analysis details are not reported in the registry record. They matter in general because measurement choices can influence the resulting estimate.

What can a hypothesis test tell a reader?

In general, a hypothesis test evaluates how compatible observed data are with a specified statistical null model. A p-value is conditional on that model and the analysis assumptions; it does not measure the probability that a treatment works, nor does it indicate the size or clinical importance of an effect. Interpretation also depends on the estimate and its uncertainty, not on a threshold alone. No p-value, test statistic, or effect estimate for the primary outcome is reported. The registry lists 0 analyses, and the endpoint data were not collected or analyzed.

The record does not report whether inferential testing was planned, which model would have been used, or how any covariates would have been handled. It also does not report interim analyses or a statistical analysis plan. Those matters cannot be inferred from the fact that the study had a stated primary outcome.

Why does the lack of a comparator matter?

General causal interpretation depends on comparing outcomes under different conditions in a way that controls confounding. A single-group study has no concurrent comparator in the registry, so it cannot directly estimate a contrast between fluorescein-guided resection and another approach. Even a complete outcome summary would primarily describe what happened within the studied group; it would not, without additional design or assumptions, establish what would have happened to the same participants under a different approach.

The single-group classification also means that planning assumptions referring to equal allocation between two arms do not describe the recorded study structure. Such assumptions can be used in hypothetical planning exercises, but they should not be mistaken for the actual allocation scheme or evidence from a comparative trial.

How should sample-size planning be understood?

In general, sample-size calculations are prospective planning tools. They depend on assumptions about the effect to detect, variability, statistical test, significance level, allocation, and desired power. A planning illustration is not an observed result and does not establish the probability that a completed study found a real effect. The registry does not report a sample-size justification for this trial. Its stated illustration assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the record describes these as planning assumptions, not the trial’s own design.

The illustration description refers to an effect based on an observed effect, but the registry reports no analyzed primary-outcome effect. Therefore, the record does not provide a primary endpoint result with which to evaluate such a planning assumption. The resulting planning figures should not be treated as trial findings, observed power, or evidence about the intervention. They also cannot fill the gap left by missing outcome measurements.

What can the posted statistical record support?

It supports a description of the registered design, enrollment, endpoint plan, termination status, and adverse-event counts. It does not support an efficacy estimate, a comparison of treatments, or a precise account of the primary outcome. In general, missing data can reduce precision and can also introduce bias if the availability of measurements is related to participant characteristics or outcomes. Here, the registry states that outcome data were not collected or analyzed, but does not describe a missing-data analysis or explain whether any partial outcome information exists. No imputation, sensitivity analysis, or analysis population is reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 3 outcome measures, but the record does not provide analyzed results for the primary endpoint. It also lists 0 analyses. The names and analysis results for the other outcome measures are not detailed in the available record, and any multiplicity strategy is not reported in the registry record.

In general, multiplicity arises when several outcomes, comparisons, or statistical tests are considered. If many tests are conducted, the chance of obtaining at least one apparently notable result by chance can increase. Statistical plans may address this through a prespecified primary endpoint, adjustment procedures, or a clearly defined hierarchy of tests. The record identifies a primary outcome but does not report a plan for controlling multiplicity or a hierarchy among outcomes. Since no outcome analyses are reported, there is no set of test results to interpret or adjust.

The endpoint description states that data were not collected or analyzed. The reason given is lack of patients and funding, and the study was terminated. The registry does not describe whether any partial measurements were obtained, why outcome data were not collected, or how participants with unavailable imaging would have been handled. A missing-data method, imputation approach, or sensitivity analysis is not reported in the registry record.

The registry also does not identify an intention-to-treat, per-protocol, or other analysis population. Such populations define which participants contribute to an analysis and can affect how results are interpreted. Enrollment is listed as 2, but this does not establish that both participants had evaluable primary endpoint data; the record explicitly says the outcome data were not collected or analyzed. Consequently, no analyzed denominator for the primary endpoint can be determined from the record.

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 5% reporting threshold.

The adverse-event entry lists the sodium-fluorescein resection group with 2 participants at risk. It records 0 participants affected by serious adverse events, 0 affected by other adverse events, and 0 deaths. No top serious-event terms are listed. These are the counts in the registry record; they should not be read as an estimate of comparative risk because the study has no listed control group.

In general, zero recorded events in a small group does not establish that an event cannot occur or precisely determine its frequency. The range of possible risks compatible with a count depends on the amount and quality of follow-up and the number of participants observed. The registry does not provide detailed information on the adverse-event collection period, event definitions, ascertainment procedures, or participant-level follow-up. The primary outcome timeframe is stated separately as pre-op assessment to outpatient follow up, up to 6 weeks; the record does not establish that this was the safety observation period.

The safety counts therefore describe what is recorded, but are limited in their ability to characterize uncommon events or compare safety with another approach. They should not be interpreted as proof of absence of harm or as a treatment recommendation.

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

The record establishes that a study of sodium-fluorescein-guided resection of pediatric neurosurgical tumors was registered as a single-group study, enrolled 2, and was terminated. It documents a planned primary endpoint based on pre-operative and post-operative imaging of intracranial lesions, with volumetric imaging tools intended to assess resection extent. It also records the group’s adverse-event counts.

What the record does not establish is the amount of tumor resected, the precision of any estimate, or whether the intervention changed resection extent. The registry states that the primary outcome data were not collected or analyzed. Without those measurements, an effect estimate and its uncertainty cannot be calculated from the record. Without a comparator, even a measured outcome in the single group would not by itself show what would have happened under a different surgical approach.

The study’s rationale mentions the use of sodium fluorescein for intraoperative guidance in adult neurosurgical tumors and the possibility that it could aid pediatric tumor resection. That statement explains the motivation for studying the approach. It does not constitute evidence from the enrolled participants. The record cannot confirm or refute the underlying rationale.

The safety entry records no serious adverse events, other adverse events, or deaths among the participants listed at risk. These counts are limited to the study record and do not establish that the intervention is free of risk. The number of participants and the lack of a comparator limit the conclusions that can be drawn from the counts.

In general, a terminated study with unavailable primary outcome data can document its design and status but cannot answer its primary statistical question. The appropriate reading is that the primary result is unknown from this registry record, rather than positive, negative, or equal to zero. The record offers no basis for medical advice or for a comparative claim about treatment effects.

13. Why This Trial Matters Statistically

This study illustrates an important distinction between registering an outcome and reporting evidence about that outcome. A clearly named primary endpoint and a planned imaging timeframe identify what investigators intended to measure. They do not supply an estimate when the registry states that data were not collected or analyzed. Statistical interpretation depends on observed measurements, not on the endpoint description alone.

The record also shows why study design matters to the question a result can answer. A single-group study can potentially describe outcomes among its participants, but a comparison of approaches requires an appropriate comparator. Here, allocation is listed as NA, masking as none, and no control arm is recorded. These features mean that the registry does not present a direct treatment contrast.

Enrollment of 2 and the absence of an analyzed primary outcome are especially relevant to precision. In general, fewer observations tend to leave less information for estimating a population outcome, although precision also depends on the variability and measurement process. In this case, there is no endpoint estimate at all, so the precision cannot be quantified. The issue is not simply that an estimate may be uncertain; the registry says the necessary data were not analyzed.

The terminated status and stated reasons of lack of patients and funding provide context for why the trial did not report its planned primary result. Such a record remains useful for understanding what was intended and what information is missing. It also cautions against treating hypothetical sample-size planning as evidence about the actual study. Statistical planning figures cannot replace outcome data or turn a single-group study into a comparison.

For clinical researchers, the record underscores the value of interpreting design, endpoint availability, and safety counts separately. Each answers a different question: the design indicates the possible scope of inference, the endpoint describes the intended measurement, and the safety entries record specific event counts. None can stand in for the absent primary analysis.

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 principal limitation is that the registry reports no collected or analyzed primary-outcome data. The intended imaging endpoint therefore has no reported estimate, uncertainty measure, or statistical test. The stated reason is that the study was terminated due to lack of patients and funding. The record does not provide further detail about the termination process or whether any partial outcome information was obtained.

Enrollment is listed as 2, and the study has a single-group design. The small recorded enrollment limits the scope for describing variation among participants, while the lack of a comparator prevents a direct comparison with another approach. No random allocation or control arm is listed. Masking is recorded as none, and the registry does not detail assessment procedures that might reduce measurement bias.

The primary endpoint description identifies imaging and volumetric assessment but does not give a calculation formula, image assessment protocol, or rules for missing or unusable scans. Analysis methods, covariate adjustment, analysis populations, and handling of missing data are not reported in the registry record. The record also does not report interim analyses or a statistical analysis plan. These omissions prevent evaluation of how the intended measurement and analysis would have been carried out.

The safety record contains counts for participants at risk but lacks detailed event terms and information about ascertainment and follow-up duration. With no comparison group, safety differences between approaches cannot be estimated. The registry’s planning assumptions refer to equal allocation between two arms, which does not match the recorded single-group structure; the assumptions are not a description of actual trial allocation.

Finally, the record lists 3 outcome measures but does not provide analyzed results for the primary endpoint. Multiplicity handling and the analysis of other outcomes are not reported. Together, these limitations mean the record supports a description of the trial and its status, but not an assessment of the primary outcome or comparative effectiveness.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Sodium Fluorescein-Guided Resection of Pediatric Neurosurgical Tumors — NCT03752203.
• Registry record last updated 2025-03-06; status last verified 2025-03.
• Results first posted 2024-02-14.
• 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

NCT03752203 is a University of Colorado, Denver study titled “Sodium Fluorescein-Guided Resection of Pediatric Neurosurgical Tumors.” It is recorded as a single-group study, with phase listed as NA, allocation as NA, and masking as none. Enrollment is listed as 2. The recorded start date is 2021-08-11, primary completion is 2023-06-29, and status is terminated.

The intervention description combines sodium fluorescein with microscopic resection using an operative microscope equipped with excitation and barrier filters. The primary endpoint concerns percent of tumor resected for intracranial lesions, assessed using pre-operative and post-operative imaging and volumetric imaging tools, with follow-up up to 6 weeks.

The registry states that primary-outcome data were not collected or analyzed because the study was terminated due to lack of patients and funding. It lists 0 analyses. The adverse-event record lists 2 participants at risk, 0 serious adverse events, 0 other adverse events, and 0 deaths. The record therefore documents the study design, planned endpoint, status, and recorded safety counts, but it does not report a primary outcome result.