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 Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System (NCT00003469), a na phase 2 study. The primary outcome is Number of Participants With Objective Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00003469 |
| Phase | Phase 2 |
| Condition | Rhabdoid Neoplasm of CNS |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 4 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Burzynski Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Children with a rhabdoid neoplasm of the central nervous system were the study population. The registry summary describes eligibility as children > 6 months of age. Four participants were enrolled. The registry record does not give further participant characteristics or a detailed description of the population used for the primary outcome analysis.
Intervention
Participants received antineoplaston therapy, identified as Atengenal plus Astugenal. The listed regimen was intravenous infusion every four hours for at least 12 months, with increasing dosages until the maximum tolerated dose was reached.
Comparator
No separate control group is listed. This was a single-group study, and allocation is recorded as not applicable. Consequently, the study does not make a like-for-like comparison between the intervention and another treatment or control condition.
Primary question
The primary outcome was the number of participants with an objective response at 12 months. Response was assessed by MRI using RANO criteria for target lesions. The registry defines complete and partial response, but does not report how many participants met either definition.
The study therefore set out to describe the effects, both good and bad, of antineoplaston therapy in children with this condition. Its primary question concerns whether participants had an objective response by the stated assessment criteria, rather than whether the treatment performed better than a comparator. A secondary outcome was the percentage of participants who survived. Results for these outcomes are not reported in the registry record, so the clinical question cannot be answered from the posted outcome data.
3. Trial Design
This was a Phase II, single-group study of antineoplastons A10 and AS2-1 in children with a rhabdoid tumor of the central nervous system. The registered intervention is named antineoplaston therapy (Atengenal plus Astugenal). The registry describes intravenous infusion every four hours for at least 12 months, with dosages increased until the maximum tolerated dose was reached.
The study has no listed control arm. Its allocation is recorded as not applicable, and masking is listed as none. In this design, outcome observations would be gathered among participants receiving the intervention, without a concurrent randomized group to provide a direct comparison. The record therefore does not support an estimate of a treatment contrast against an alternative therapy or control condition.
The recorded enrollment was 4. The study began in 1996-02, had a primary completion date of 2003-05, and is listed as terminated. The registry record does not report why the study was terminated, how many participants completed treatment or follow-up, or whether the planned treatment duration was reached by any participant.
The primary outcome was the number of participants with objective response at 12 months. A secondary outcome was the percentage of participants who survived. The record lists two outcome measures but no analyses. It does not report primary or secondary outcome results, participant-level outcome data, an analysis population, or a statistical analysis plan. The reason for the small enrollment, the original recruitment target, and any sample-size justification are not reported in the registry record. These omissions limit assessment of the study's precision and of how closely its conduct followed any planned design.
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 | Antineoplaston Therapy |
|---|---|
| STARTED | 4 |
| COMPLETED | 4 |
| NOT COMPLETED | 0 |
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 | Antineoplaston Therapy |
|---|---|---|
| Participants analysed | 4 | |
| Age, Continuous | Years | 3.7 (2.0 to 6.8) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Objective Response, measured over 12 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with an objective response at 12 months. The registry describes response assessment by MRI for target lesions using Response Assessment in Neuro-Oncology (RANO) criteria. A complete response is defined as disappearance of all disease sustained for at least four weeks. A partial response is defined as a >=50% decrease in the sum of the products of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks.
Objective response is a categorical outcome: each assessed participant would be classified according to whether the specified response criteria were met. Counting participants with a response can describe observed tumor response within a single-group study. The MRI-based criteria and required duration help define what qualifies as a response, rather than relying on an unspecified impression of improvement. The endpoint is time-specific: the registry names 12 months as the timeframe.
The record does not report the number of participants who had a complete response, partial response, or any objective response. It also does not report whether all enrolled participants had an MRI assessment at 12 months, how missing assessments were handled, or which participants were included in the endpoint analysis. Thus, the endpoint definition is available, but its observed result and the precision of that result are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a Phase II, single-group study with four participants and a primary endpoint of objective response at 12 months. It lists no statistical analyses, and the registry record does not report the number of participants with an objective response. The central efficacy result therefore cannot be quantified from the posted information. There is no reported response estimate, confidence interval, or p-value to interpret.
The absence of a comparator is important to interpretation. Any outcome observed in a single group would describe participants in that group; it would not, by itself, distinguish an intervention effect from other explanations for the observed course. The registry lists allocation as not applicable and masking as none. It does not report a randomized or blinded comparison, so a comparative treatment effect cannot be assessed from this record.
The enrollment of 4 also means that each participant's outcome could have substantial influence on a group summary. This is a general feature of small studies, not a calculation of this study's response rate. Because the response count and analysis population are not reported, no response proportion or precision measure should be inferred. The study's planned duration, completed treatment exposure, and completeness of 12-month assessments are likewise not reported in the registry record.
A secondary outcome, the percentage of participants who survived, is listed, but its result and the methods used to determine survival are not reported. No time-to-event estimate or survival percentage can be interpreted. The registry also records safety information for four participants: three are listed as affected by serious adverse events, and four as affected by other adverse events. Those summaries do not establish that the intervention caused the events. The record does not provide enough detail to assess event timing, severity beyond the serious classification, or attribution.
The listed status is terminated, but the reason for termination is not reported. It would be inappropriate to infer that termination resulted from safety concerns, lack of efficacy, recruitment difficulty, or any other cause. Likewise, no sample-size rationale or statistical analysis plan is reported. Taken together, the record documents the study question, design, intervention, and some safety counts, but it does not include the primary or secondary outcome results needed to judge the observed response or survival experience.
9. Statistical Methods Explained
The statistical information in the registry is limited. It identifies a single-group Phase II design and a count-based primary outcome, but lists no analyses. The primary and secondary outcome results are not reported in the registry record. The following concepts explain how such outcomes are commonly interpreted in general; they do not describe unreported procedures or results from this study.
What does a participant count measure?
The primary endpoint is a count: the number of participants meeting the objective response definition at 12 months. A count is most informative when its denominator and analysis population are also clear. For example, interpretation depends on knowing how many participants were eligible for assessment, how many were actually assessed, and what happened to participants without an assessment. Those details are not reported in the registry record. The response count itself is also not reported, so neither the count nor a response proportion can be interpreted.
For a binary outcome such as response, a confidence interval is commonly used to express the uncertainty around an estimated proportion. In general, a narrower interval indicates greater precision, while a wider interval indicates more uncertainty. With a small group, estimates are often imprecise, and the outcome of each participant can have a noticeable influence on the estimate. No confidence interval or response estimate is reported for this study, so its precision cannot be evaluated from the registry.
Why does the design matter?
In a single-group study, participants receive the listed intervention without a concurrent comparator group. The outcome count can describe what was recorded among those participants, if results are available. It cannot directly establish how outcomes would have differed under another treatment or no intervention. Randomized comparison is one way studies seek to support a treatment contrast, but no such comparison is listed. Because masking is recorded as none, the record also does not indicate masked outcome assessment. The registry does not describe any additional procedures intended to reduce assessment bias.
These design features do not make a descriptive count meaningless; they define what it can answer. A count of responses would be an observation within the study group, not a comparative effect estimate. In this record, the outcome count is absent, so even that descriptive result is unavailable.
What can be learned from the response definition?
The registry specifies MRI assessment of target lesions using RANO criteria. Complete response requires disappearance of all disease sustained for at least four weeks; partial response requires a >=50% decrease in a specified measure of measurable enhancing lesions, also sustained for at least four weeks. Such criteria make the endpoint more operational by stating what types of change qualify and for how long. They do not, on their own, establish that assessments were performed consistently or that all participants had complete follow-up. The registry does not report assessment procedures beyond the definition, central review, assessor details, or handling of missing measurements.
What is over-dispersion, and is it relevant?
Over-dispersion is a general issue for count outcomes when variation exceeds what a chosen statistical model expects. It can affect standard errors and confidence intervals if the model assumes less variability than the data contain. The listed primary outcome is a participant count, but no model or analysis is reported. The record gives no basis to conclude that an over-dispersion adjustment was needed or used. In particular, a count of participants with a binary response is not, by itself, evidence that an over-dispersed count model was applied.
What do analysis and planning results mean?
The registry lists zero analyses. It does not report a prespecified statistical plan, hypothesis test, model, interim analysis, or sample-size justification. A sample-size illustration based on an observed effect is a planning exercise, not evidence about what the study was designed to detect, and it is not observed power. In this case, the primary outcome result needed to define an observed effect is not reported in the registry record. No study-specific planning rationale or detectable-effect claim can therefore be drawn from the posted record.
Finally, the secondary outcome of survival is named, but the record does not state how survival was summarized or analyzed. General methods for time-to-event outcomes can account for differing follow-up lengths and censoring, but there is no reported survival analysis to interpret. The registry's outcome titles and definitions describe what was to be measured; they do not substitute for outcome data or an analysis report.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates multiple outcomes, comparisons, time points, or statistical tests. In general, testing several hypotheses can increase the chance of a false-positive finding unless the analysis plan addresses that issue. The registry lists two outcome measures: the primary outcome of participants with objective response and a secondary outcome of the percentage of participants who survived. It does not report statistical analyses, tests, or a multiplicity strategy. The record therefore does not establish whether any adjustment was planned or performed.
The primary outcome timeframe is 12 months. The registry does not describe other response assessment time points or repeated testing. It also does not report whether any interim analyses were planned or conducted. No assumptions about these procedures can be made from the record.
Missing data are especially relevant when the outcome requires assessment at a specified time. The registry does not report how many participants had an MRI assessment at 12 months, whether any outcome data were missing, why data might be missing, or how missing assessments were handled. It does not identify an intention-to-treat, per-protocol, or other analysis population. Although the enrollment is recorded as 4, the record does not state which participants were included in the primary outcome analysis.
These omissions make it impossible to judge whether the reported enrollment and endpoint denominator would match, how missingness might affect an estimate, or whether outcome reporting followed a prespecified hierarchy. Because the primary and secondary results are not reported in the registry record, there are no posted estimates against which to assess selective outcome reporting or multiplicity-adjusted inference.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Antineoplaston Therapy | 3 | 4 | 75.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Antineoplaston Therapy | 4 of 4 |
Most frequent serious adverse event terms
| Event term | Organ system | Antineoplaston Therapy |
|---|---|---|
| Central Venous Catheter Infection | General disorders | 1 of 4 |
| Vomiting | Gastrointestinal disorders | 1 of 4 |
| Hypernatremia | Investigations | 1 of 4 |
| Hypokalemia | Investigations | 1 of 4 |
| Pain: Head/headache | Nervous system disorders | 1 of 4 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Antineoplaston Therapy |
|---|---|---|
| Hypokalemia | Investigations | 4 of 4 |
| Hemoglobin | Blood and lymphatic system disorders | 3 of 4 |
| Vomiting | Gastrointestinal disorders | 3 of 4 |
| Hypernatremia | Investigations | 3 of 4 |
| Pain: Head/headache | Nervous system disorders | 3 of 4 |
| Non-functional central venous catheter | General disorders | 2 of 4 |
| Fatigue (asthenia, lethargy, malaise | General disorders | 2 of 4 |
| Allergic reaction/hypersensitivity (including drug fever) | Immune system disorders | 1 of 4 |
| Leukocytes (total WBC) | Blood and lymphatic system disorders | 1 of 4 |
| Lymphopenia | Blood and lymphatic system disorders | 1 of 4 |
Serious events with the highest arm-level rate: central venous catheter infection (25.0%), vomiting (25.0%), hypernatremia (25.0%), hypokalemia (25.0%), pain: head/headache (25.0%).
The registry safety summary lists four participants at risk for serious adverse events, with three affected. For other adverse events, it lists four participants at risk and four affected. These are participant counts within the registered safety summary. The record does not provide event-level dates, duration, severity details beyond the serious classification, or an attribution assessment linking events to the intervention.
The serious adverse event terms listed include Central Venous Catheter Infection, Vomiting, Hypernatremia, Hypokalemia, and Pain: Head/headache. Each is reported as 25.0%. These percentages describe the listed terms in the registry summary; the record does not clarify whether participants could contribute more than one event term or provide further detail on event recurrence. They should not be read as comparative risks, since there is no control group.
The deaths field is not reported in the registry record. The summary cannot establish whether an adverse event was caused by antineoplaston therapy, nor does it allow comparison with another treatment. The counts describe the safety information posted, not a causal conclusion.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II study of antineoplaston therapy enrolled four children with a rhabdoid neoplasm of the central nervous system. It identifies a single-group design without masking and defines an MRI-based objective response endpoint at 12 months. It also lists survival as a secondary outcome and reports safety counts for the enrolled group. These details help characterize what the study set out to examine.
The record does not report the number of participants with an objective response or the percentage who survived. It therefore does not show an observed response result or a survival result. No effect estimate, confidence interval, or statistical test is reported. The missing results do not mean that no responses or survival events occurred; they mean those results are not reported in the registry record.
The single-group design also limits what could be concluded even if outcome counts were posted. Without a comparator, the study would not directly estimate how outcomes differed from those under another treatment or control condition. The record does not provide enough information to assess the completeness of follow-up, the analysis population, or possible missing outcome data.
The safety summary reports serious adverse events affecting three of four participants and other adverse events affecting four of four participants. It lists several serious event terms, each reported as 25.0%. These observations deserve to be read as descriptive safety information, not proof of causation. The record does not report the event-level context needed to attribute events to treatment.
The study status is terminated, but the reason is not reported. The posted information cannot establish why recruitment or study activity ended, nor can it establish a benefit or lack of benefit. Overall, the registry record documents a small study and its intended measures, but its absent outcome results prevent a substantive reading of response or survival.
13. Why This Trial Matters Statistically
This trial is statistically notable because it illustrates the difference between a clearly defined study question and an interpretable result. The registry specifies a primary endpoint, an assessment timeframe, and response criteria. Yet it does not report the number of participants meeting the endpoint or any statistical analysis. A precise definition helps readers understand what would count as a response, but it cannot replace the observed outcome data.
The single-group design also makes the distinction between description and comparison especially important. A response count, if reported, could describe outcomes in the treated group. It would not by itself estimate how outcomes compare with those under another condition. The absence of a control group is therefore central to the scope of any conclusion.
With enrollment of 4, the study also highlights why sample size and uncertainty matter. General statistical principles indicate that estimates based on very small groups can be sensitive to individual outcomes. Here, however, no response estimate or confidence interval is posted, so the magnitude and precision of any observed response cannot be assessed.
Finally, the registry's limited analysis reporting leaves important questions unanswered: the analysis population, missing-data handling, multiplicity approach, and sample-size rationale are not reported. These gaps demonstrate why outcome results and analysis methods are needed alongside design and endpoint definitions. The record provides useful information about the planned clinical question and safety summary, but not the results necessary for statistical evaluation of response or survival.
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.
Several trial-specific limitations arise from the registry information. Enrollment was 4, and the design was single-group, with allocation recorded as not applicable and masking recorded as none. There is no listed control group for a direct comparison. The study status is terminated, but the reason for termination is not reported in the registry record.
The primary outcome is defined as the number of participants with objective response at 12 months, assessed by MRI using RANO criteria. However, the registry does not report the response count, the number assessed, or the analysis population. The secondary outcome of survival is listed without a reported result or analysis method. Consequently, neither outcome can be evaluated for magnitude or precision.
The record does not report participant characteristics beyond the condition and the summary's age description. It does not state treatment exposure actually received, completion of the stated treatment duration, follow-up completeness, or handling of missing outcome assessments. It also does not report a statistical analysis plan, sample-size justification, interim analyses, or multiplicity approach. These details should not be inferred from the Phase II designation or the study's status.
The safety summary gives affected-participant counts and listed serious event terms, but lacks event-level context and attribution. The deaths field is not reported in the registry record. As a result, the available safety figures are descriptive and cannot support a comparison or causal assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System — NCT00003469.
- Registry record last updated 2017-08-24; status last verified 2017-07.
- Results first posted 2017-03-07.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This Phase II study examined antineoplaston therapy (Atengenal plus Astugenal) in children with a rhabdoid neoplasm of the central nervous system. It used a single-group design, had no masking, enrolled 4 participants, and is listed as terminated. The stated regimen was intravenous infusion every four hours for at least 12 months, with dosage increases until the maximum tolerated dose was reached.
The primary outcome was the number of participants with objective response at 12 months, assessed by MRI using RANO criteria. Survival was listed as a secondary outcome. The registry record does not report results for either outcome or any statistical analyses. It lists serious adverse events affecting three participants and other adverse events affecting four; these counts do not establish causation. The reason for termination, analysis population, missing-data handling, and sample-size rationale are not reported in the registry record.