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 Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma (NCT02212574), a na early/phase 1 study. The primary outcome is Progression Free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02212574 |
| Phase | EARLY/Phase 1 |
| Condition | Medulloblastoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
| Results posted | Yes |
2. Clinical Question
Population
The intended population was children with newly diagnosed, non-metastatic, standard risk, Wnt positive medulloblastoma. The registry record describes this as the target group for the feasibility study. It does not report further eligibility details or participant characteristics, so the degree to which those enrolled matched particular clinical subgroups cannot be assessed from this record.
Intervention
The study evaluated an approach based on chemotherapy without radiation therapy or the weekly chemotherapy given during radiation therapy. Listed drugs include lomustine, vincristine, cisplatin, cyclophosphamide, and mesna. The arm description gives details for Chemotherapy Cycle A: lomustine and cisplatin on Day 1, with vincristine on Days 1, 8, and 15; the cycle lasts 42 days. That description does not establish the full treatment schedule for every participant.
Comparator
This was a single-group study, with allocation listed as not applicable and no masking. The registry record does not report a concurrent control group or a randomized comparison. The question is therefore not a like-for-like comparison of chemotherapy alone against another assigned treatment within this trial. Any broader comparison with other care would require information not reported in the registry record.
Primary question
The primary objective was to determine the feasibility of the chemotherapy-only approach, with progression-free survival as the stated primary outcome. The outcome timeframe was 3 years. The record says this outcome was not assessed because some patients had a relapse and the study was abruptly terminated. Thus, the intended question is clear, but the registry record does not provide an observed progression-free survival result with which to answer it.
3. Trial Design
This was an early phase 1, single-group pilot study registered as NCT02212574. The record lists an enrollment of 6, describes allocation as not applicable, and reports no masking. The study was intended to examine the feasibility of a chemotherapy-only approach for children with Wnt positive standard risk medulloblastoma. The stated approach omitted radiation therapy and the weekly chemotherapy given during radiation therapy.
The treatment listing includes lomustine, vincristine, cisplatin, cyclophosphamide, and mesna. The arm description provides a schedule for Chemotherapy Cycle A: lomustine by mouth on Day 1; vincristine intravenously on Days 1, 8, and 15; and cisplatin intravenously over 8 hours on Day 1. It says the cycle lasts 42 days. The listing also includes vincristine twice; the registry record does not explain that repetition. The Cycle A description should not be taken as a complete account of all treatment participants may have received.
The study start date was 2017-04-04, and the primary completion date was 2018-11-09. Its status is terminated. These dates and status describe the registry record, but do not explain the circumstances or timing of each participant’s treatment. The record states that the primary outcome was not assessed because some patients had a relapse and the study was abruptly terminated.
No control arm is described, so the design cannot isolate a treatment contrast against a concurrent assigned alternative. The record does not report a protocol-level statistical analysis plan, sample-size justification, or interim analysis plan. The stated enrollment and the primary endpoint timeframe therefore do not, by themselves, show whether the intended feasibility or survival questions were adequately evaluated.
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 | Chemotherapy |
|---|---|
| STARTED | 6 |
| COMPLETED | 6 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: This outcome was not assessed because some patients had a relapse and study was abruptly terminated.
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 | Chemotherapy |
|---|---|---|
| Participants analysed | 6 | |
| Age, Categorical: <=18 years | Participants | 6 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 7 (5 to 12) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 5 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 3 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival, measured over 3 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled Progression Free Survival, with a timeframe of 3 years. The registry description links the endpoint to feasibility of treating newly diagnosed children with non-metastatic, standard risk, Wnt positive medulloblastoma using chemotherapy alone. It describes the outcome as the number of participants with progression-free survival. The registry record does not give an endpoint definition, an analysis method, or an observed outcome value.
In general, progression-free survival is used to describe how long participants remain free of a specified disease-progression event under a study’s definition. It can capture disease control over follow-up rather than only an event count at a single visit. The exact event rules, censoring approach, and handling of participants without complete follow-up matter to interpretation; those study-specific details are not reported in the registry record.
Here, the essential limitation is that the planned outcome was not assessed. The record attributes this to some patients having a relapse and to abrupt termination of the study. It does not report a three-year estimate, participant-level times, or a confidence interval. As a result, the endpoint’s clinical purpose can be described, but its observed value and precision cannot be evaluated from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record sets out a feasibility study of chemotherapy without radiation for children with a defined medulloblastoma subgroup. It identifies progression-free survival at 3 years as the primary outcome. However, it explicitly states that this outcome was not assessed because some patients had a relapse and the study was abruptly terminated. No progression-free survival estimate, event count for this endpoint, or measure of uncertainty is reported. The record therefore does not support a quantitative conclusion about the endpoint.
The study’s single-group design is important to what can be inferred. Without a concurrent control group, the trial cannot directly estimate a between-group treatment effect. Observations from a single group can describe what occurred among participants who enrolled and were followed, but causal comparison with another treatment requires a suitable comparison and assumptions that are not established by this design. The registry record does not report a comparative analysis.
The enrollment is listed as 6. That figure alone does not indicate how many participants had evaluable follow-up, whether all met the intended eligibility criteria, or how much outcome information was available. Those details are not reported in the registry record. Nor does the record provide participant-level follow-up, censoring information, or a completed endpoint analysis. Consequently, no estimate of precision can be assessed.
The registry’s adverse-event summary lists the group as All Participants and records zeros for participants at risk, participants affected, and deaths. The record does not explain the meaning or coverage of these zero entries. They should not be interpreted as proof that participants experienced no adverse events, especially without information about collection, follow-up, or reporting. The record also does not provide enough detail to assess treatment exposure or safety outcomes comprehensively.
Overall, the record documents the study objective, design, planned endpoint, treatment approach, enrollment, and termination status, but not a completed primary outcome analysis. It is therefore possible to explain the statistical question and design constraints, but not to quantify an effect, assess its precision, or determine whether the intended feasibility objective was met. Any stronger conclusion would require results or analysis details not reported in the registry record.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study describes outcomes observed among people who receive the study approach. Its estimates do not directly compare outcomes under assigned alternatives. A randomized comparison is designed to make treatment groups comparable on average through allocation; no such allocation is reported for this trial. Here, allocation is listed as not applicable and the model is single group. Consequently, the record does not support a randomized treatment contrast or an estimate of the difference between chemotherapy alone and another assigned strategy.
A single-group design can still be useful for a pilot question, such as whether a treatment approach can be delivered or whether further study is practicable. Those questions require prespecified definitions and observable measures. The registry record identifies feasibility as the objective, but it does not report a separate feasibility criterion or a completed analysis against such a criterion. The primary outcome named is progression-free survival, and the record states that it was not assessed.
How should progression-free survival be understood?
In general, a time-to-event endpoint records the time from a defined starting point to a specified event. Progression-free survival commonly concerns time until disease progression or another protocol-defined event, but the exact rules must come from the trial’s endpoint definition. This registry entry does not report the operational event definition, the time origin, censoring rules, or the statistical method used for the planned endpoint. It gives a 3-year timeframe and describes a participant count with progression-free survival, but does not provide an estimate.
Time-to-event analysis can use information from participants with different follow-up durations, rather than treating all follow-up as identical. In general, censoring means that the event time is not observed during the available follow-up; proper interpretation depends on how censoring is defined and handled. The registry record does not report censoring information or participant-level follow-up. Since the outcome was not assessed, there is no posted survival estimate or confidence interval to interpret.
What does a confidence interval contribute?
In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the analysis. It helps distinguish a relatively precise estimate from one compatible with a broad range of values. It does not repair limitations such as the absence of a control group, incomplete outcome assessment, or an unsuitable analysis population. No confidence interval for progression-free survival is reported in the registry record, so the precision of the intended endpoint cannot be judged.
Likewise, a p-value would address compatibility between data and a specified statistical null model; it would not establish clinical importance or remove design limitations. The registry record reports no analyses for this outcome and no analysis count. No p-value or hypothesis test result is available to interpret. It would be inappropriate to infer statistical significance or lack of significance from the stated enrollment or termination status.
What are the sample-size calculations for?
The sample-size assumptions describe a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. For time-to-event calculations, the stated quantity is total events using the Schoenfeld approach; other calculations are expressed as participants per arm. These assumptions describe an illustration, not the design or analysis of this single-group trial.
In general, a planning calculation estimates how much information may be needed to detect a specified effect under chosen assumptions. Its output depends on the effect size, variability or event process, allocation, analysis method, and target power. Planning for a fraction of an observed effect is a scenario analysis, not evidence that a future study will achieve the stated performance. These calculations are not observed power and do not establish what this trial could detect. The registry record does not report the numerical effect used or a trial-specific sample-size justification.
Over-dispersion is a general issue when count outcomes vary more than a model assumes; ignoring it can make uncertainty estimates too narrow. That concept does not establish that over-dispersion applies to this endpoint. No count-model analysis or over-dispersion assessment is reported for the primary outcome. More broadly, statistical methods must match the endpoint, follow-up, and observed data. Since the endpoint was not assessed, there are no reported model results or diagnostics to evaluate.
The central methodological conclusion is therefore limited: the registry specifies a planned survival endpoint and includes general planning assumptions, but does not report the analysis that would be needed to quantify progression-free survival. Statistical concepts can clarify what such an analysis would ordinarily require, yet they cannot substitute for outcome data, a defined analysis plan, or a completed result.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists Progression Free Survival as the primary outcome and Patterns of Failure as a secondary outcome. It reports two outcome measures and no analyses. It does not describe a multiplicity strategy, such as prioritizing tests, adjusting significance thresholds, or distinguishing confirmatory from exploratory analyses. Without that information and without reported results, the impact of testing more than one outcome cannot be assessed.
In general, multiplicity arises when several outcomes, comparisons, time points, or subgroups are examined. Repeated testing can increase the chance of a nominally unusual result if no plan accounts for the number and relationship of analyses. The relevant concern depends on what was prespecified and what was actually analyzed. Neither a detailed prespecified analysis plan nor completed analyses are reported in the registry record, so it is not possible to determine how multiplicity was handled.
Missing outcome information also matters. The record says the primary outcome was not assessed because some patients had a relapse and the study was abruptly terminated. It does not report participant-level outcome status, follow-up duration, missingness reasons beyond that statement, or a missing-data approach. In general, missing follow-up can affect both the estimate and its precision, particularly when loss of information is related to prognosis or study conduct. The available record does not allow those possibilities to be evaluated.
The analysis population is not described for the primary endpoint. The record does not say whether analyses were intended to include all enrolled participants, only eligible participants, or another defined group. It also does not report exclusions, protocol deviations, or outcome ascertainment procedures. These omissions prevent assessment of how the enrolled group relates to any analyzed population. The absence of an endpoint analysis means there is no basis for examining the effect of alternative missing-data or population definitions.
11. What the Result Does, and Does Not, Mean
The record establishes that a chemotherapy-only approach was studied as a pilot for children with Wnt positive standard risk medulloblastoma, with radiation therapy and the weekly chemotherapy given during radiation omitted from the intended approach. It also establishes that the planned primary outcome was progression-free survival at 3 years and that the study was terminated. These registry entries describe the purpose and conduct recorded for the trial; they are not a result showing the value of the primary endpoint.
The record says the primary outcome was not assessed because some patients had a relapse and the study was abruptly terminated. That statement does not provide a complete event history, a survival estimate, or an account of what happened to every participant. It also does not state whether the planned feasibility objective was met. Without outcome results and analysis details, the registry record cannot quantify the observed clinical course or the uncertainty around it.
The single-group design means the study does not provide a randomized within-trial comparison against another treatment. Even if an outcome estimate had been reported, interpretation would need to distinguish a description of this enrolled group from a causal claim about how outcomes differ under alternative approaches. The registry record does not report comparative data that would answer the latter question.
More generally, an absent result is not evidence that an effect is absent. It is also not evidence that the approach is effective. The distinction is between a result that estimates an outcome with stated uncertainty and a record that says the outcome was not assessed. For this trial, the latter applies to the primary endpoint. The registry entry cannot support a conclusion about benefit, lack of benefit, or the magnitude of any treatment effect.
The stated enrollment and the adverse-event summary do not resolve these limitations. The record does not explain the adverse-event zeros or provide enough information to evaluate event ascertainment and follow-up. Thus, neither those entries nor the planned endpoint should be treated as a substitute for a completed analysis. Interpretation should remain confined to the design and status information actually recorded.
12. Why This Trial Matters Statistically
This trial is statistically notable because its clinical question centers on a planned survival outcome, while the registry record states that the outcome was not assessed. That distinction is fundamental: a trial objective is not an estimate, and a planned endpoint does not itself provide evidence about the outcome. Readers need to separate the intended analysis from the results actually reported.
The single-group pilot design also illustrates how design determines the scope of inference. A single group may describe outcomes under an approach, but does not provide a randomized comparison with an alternative. This limitation is especially important when a result could otherwise be read as evidence that one treatment strategy is better or worse than another. The record does not report a comparison, and no causal treatment contrast can be recovered from the stated design alone.
Termination and incomplete outcome assessment underscore why follow-up and analysis populations are part of statistical interpretation, not administrative details. For a time-to-event endpoint, the observed follow-up and handling of participants without a recorded event shape the estimate and its uncertainty. The registry record does not report these details or an endpoint analysis, so readers cannot assess how much information the planned outcome would have contained.
The entry also distinguishes planning exercises from trial evidence. Sample-size illustrations based on an observed effect are not observed power and are not a substitute for a prespecified, trial-specific design justification. Their assumptions describe hypothetical planning conditions. They do not overcome the lack of a reported primary outcome or create a comparison absent from the study design.
For clinical researchers, this record is a useful reminder that the stated question, trial design, outcome definition, follow-up, and analysis results must be considered together. Here, the objective and endpoint are identifiable, but the registry record does not provide the completed statistical evidence needed to estimate progression-free survival or its precision.
13. 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 primary limitation is explicit: progression-free survival, the primary outcome with a 3-year timeframe, was not assessed. The registry attributes this to some patients having a relapse and abrupt study termination. No estimate, event count for the endpoint, confidence interval, or participant-level follow-up is reported. Consequently, the primary clinical question cannot be answered quantitatively from the registry record.
The single-group model and absence of a randomized comparator limit comparative inference. Allocation is listed as not applicable, and the record reports no masking. These features do not invalidate descriptive information, but they mean that the trial does not provide a like-for-like control comparison. The registry record also does not report a separate feasibility criterion or an analysis showing whether that objective was achieved.
Enrollment is listed as 6, but the record does not give participant characteristics, treatment exposure, duration of follow-up, eligibility details, or the number with evaluable endpoint information. It does not define progression events, censoring, or the analysis population. These omissions make it impossible to assess whether endpoint ascertainment and follow-up were complete or how an analysis would have handled incomplete information.
The adverse-event summary lists zero values for participants at risk, participants affected, and deaths, but does not explain the coverage or meaning of these entries. They cannot establish that no adverse events occurred. The registry record does not provide a detailed safety analysis or adverse-event collection procedures.
Finally, the record does not report a protocol-level statistical plan, multiplicity approach, missing-data strategy, or trial-specific sample-size justification. The planning assumptions are not a substitute for those details. Together, these limitations mean that the registry entry supports description of the study’s purpose and status, but not a reliable estimate of the intended outcome or a comparative treatment conclusion.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma — NCT02212574.
- Registry record last updated 2021-11-05; status last verified 2021-10.
- Results first posted 2021-11-05.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
17. Record Summary
NCT02212574 was an early phase 1, single-group pilot study sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. It was designed to assess the feasibility of chemotherapy without radiation therapy for children with Wnt positive standard risk medulloblastoma. The listed enrollment was 6, and the registry status is terminated.
The primary outcome was Progression Free Survival at 3 years. The registry record states that this outcome was not assessed because some patients had a relapse and the study was abruptly terminated. It lists Patterns of Failure as a secondary outcome and reports no analyses. No primary outcome estimate or comparative result is reported.
The record lists lomustine, vincristine, cisplatin, cyclophosphamide, and mesna. It describes part of Chemotherapy Cycle A, but does not provide a complete treatment history. The study started on 2017-04-04, with primary completion dated 2018-11-09. The record supports a description of the planned question and design, not a conclusion about the observed progression-free survival outcome.