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 Patients With Meningioma (NCT00003483), a na phase 2 study. The primary outcome is Number of Participants With Objective Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00003483 |
| Phase | Phase 2 |
| Condition | Meningioma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Burzynski Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Adults with meningioma. The registry record does not report further eligibility details or describe the participants’ disease characteristics.
Intervention
Antineoplaston therapy, identified as Atengenal plus Astugenal, administered by intravenous infusion every four hours. The protocol description says doses increased until the maximum tolerated dose was reached, with treatment intended for at least 12 months.
Comparator
There is no comparator group in this single-group study. The registry record does not describe a randomized comparison or a like-for-like control arm.
Primary question
How many participants had an objective response by 12 months, using MRI assessment of target lesions under the stated RANO definitions?
The study was intended to assess the effects of antineoplaston therapy in adults with meningioma. The primary outcome focuses on objective response, a classification based on changes in measurable lesions. The registry record also lists the percentage of participants who survived as a secondary outcome, but it does not post results for that outcome.
The record lists three enrolled participants and a terminated status. It does not report why the study was terminated, how many participants completed the planned treatment, or whether all participants had evaluable MRI assessments at the specified time. Those details matter when interpreting an outcome framed as a response count: the number enrolled does not by itself establish how many were assessed, or how missing assessments were handled.
Because the study has one treatment group and no comparator, it can describe observations among participants receiving the regimen, if those observations are reported. It cannot provide a randomized between-group estimate of treatment effect. In this record, the primary response result is not reported, so the question cannot be answered from the posted outcome data.
3. Trial Design
This is a Phase II, single-group study of antineoplaston therapy in participants with meningioma. The registry lists allocation as not applicable and masking as none. No randomized comparison or control group is described; the record identifies one treatment group, Antineoplaston Therapy.
The intervention is named as Antineoplaston therapy (Atengenal plus Astugenal). The regimen description specifies intravenous infusion every four hours and dose escalation until the maximum tolerated dose was reached. Treatment was described as continuing for at least 12 months. The registry record does not report how dose escalation was conducted in practice, what dose levels were used, or how many participants received treatment for the intended duration.
The study enrolled three participants. Its status is terminated, with a start date of 1998-06 and primary completion recorded as 2004-03. The reason for termination is not reported in the registry record. Nor does the record explain whether the recorded primary completion date reflects completion of follow-up for all enrolled participants or another administrative milestone.
The primary outcome is the number of participants with objective response at 12 months, assessed by MRI under the stated RANO criteria. A secondary outcome is the percentage of participants who survived. The record lists two outcome measures but no statistical analyses. It does not report a statistical analysis plan, an intended enrollment target, eligibility criteria beyond the broad meningioma population, or details about follow-up and analysis populations.
With one group and no control condition, the design is descriptive for the posted data. Any observed changes would not be separated from the course of disease, measurement variation, or other influences by a concurrent randomized comparison. In this case, even descriptive primary outcome results are not posted, limiting what can be concluded about the response question.
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 | 3 |
| COMPLETED | 2 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Antineoplaston Therapy |
|---|---|
| Not evaluable | 1 |
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 | 3 | |
| Age, Continuous | Years | 41.8 (30.2 to 49.8) |
| Sex: Female, Male: Female | Participants | 2 |
| 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 objective response at 12 months. It uses MRI assessment of target lesions and classifies response according to the RANO definitions in the registry record. Complete response requires disappearance of all disease sustained for at least four weeks. Partial response requires at least a 50% decrease in the sum of the products of the greatest perpendicular diameters of measurable enhancing lesions, sustained for at least four weeks. Stable disease is defined as less than a 50% decrease and less than a 25% increase in that sum, sustained for at least eight weeks. Progressive disease is defined as at least a 25% increase.
Objective-response criteria provide a structured way to describe changes in measurable disease. In general, explicit criteria help make assessments more consistent than an informal judgment, although interpretation still depends on assessment timing, image quality, lesion measurement, and application of the definitions. The registry record does not report who assessed the scans, whether assessments were independently reviewed, or how disagreements or missing scans were handled.
The endpoint is a count of participants with objective response, not a comparative treatment-effect estimate. The registry record gives the endpoint definition and timeframe but does not report the observed response count. Therefore, the posted data do not establish how many participants met the response criteria.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small, single-group Phase II study that enrolled three participants and was terminated. It names objective response at 12 months as the primary outcome and supplies MRI-based RANO criteria, but the posted results do not include the number of participants with objective response. The record lists no statistical analyses. Thus, there is no posted estimate of the primary outcome to interpret, and no reported inferential result such as a confidence interval or hypothesis test.
The absence of a reported response count is distinct from a finding of no response. The registry record does not say that the response count was zero; it does not report the count. The same distinction applies to the secondary survival outcome: a survival percentage is listed as an outcome, but no result is posted. These missing results prevent readers from assessing the observed response pattern or survival experience from this record.
Even if outcome counts were available, a single-group study would describe outcomes in the enrolled participants without a randomized comparator. It would not isolate the effect of treatment from other explanations for observed outcomes. The small enrollment also means that each participant’s outcome would have substantial influence on a group summary. General statistical principle: estimates based on very few observations are typically imprecise, and uncertainty should be made visible rather than obscured by a point estimate alone. No uncertainty interval is reported.
The record does provide safety counts. In the Antineoplaston Therapy group, three of three participants are listed as having serious adverse events, and three of three are listed as having other adverse events. The listed serious adverse-event terms each have a reported percentage of 33.3%. These data describe recorded events in this group; they do not establish that therapy caused the events. The record does not report deaths in the adverse-event summary, so a death count should not be inferred from the blank entry.
The study status is terminated, but the reason is not reported in the registry record. Termination alone does not explain whether the reason was recruitment, safety, funding, administrative, or another consideration. Without the primary and secondary outcome results, participant disposition, analysis methods, and termination reason, the registry record supports a description of the design and reported safety counts, but not a conclusion about efficacy or a full assessment of the study’s conduct.
9. Statistical Methods Explained
The registry record identifies a binary outcome type and names the primary measure as the number of participants with objective response. The record lists no statistical analyses. Consequently, the methods that can be described are the outcome definition and the general statistical principles relevant to interpreting such a result; an analysis model or inferential procedure used by the investigators is not reported in the registry record.
What does a count endpoint show?
A count of participants meeting a response definition reports how many people in the analyzed group had the outcome. It is a direct summary, but its interpretation depends on the denominator: the number enrolled, the number assessed, and the number included in the analysis may not be the same. The registry record gives enrollment of three but does not report the number assessed for response or the analysis population. It also does not post the response count. The denominator and handling of participants without an evaluable assessment are therefore unknown.
In general, a proportion or count from a small group can change noticeably when the outcome of one participant changes. That is why the count should be interpreted alongside the denominator and an uncertainty interval when those are available. A confidence interval expresses a range of values compatible with the data under the assumptions of the method; it is not a guarantee that the true value lies within that range. No confidence interval is reported in the registry record.
Why does the study design matter?
The study is single-group, with no randomized comparator. A one-group result can describe what was observed among participants in that group, but it does not estimate a randomized contrast against another treatment or control condition. General statistical principle: a comparison group helps distinguish differences associated with the assigned intervention from changes that may occur for other reasons. Without one, causal interpretation is limited. The registry record does not report an adjusted or historical comparison.
The study has three enrolled participants. With so few observations, estimates would be particularly sensitive to individual outcomes and uncertainty would be substantial. This is a general consideration, not a calculation of power or precision for this study. The record does not report a sample-size justification, a target enrollment, or a formal precision objective.
What can be said about planning calculations?
Any sample-size planning illustration associated with these results is a hypothetical planning calculation, not observed power. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a fraction of the observed effect. The trial itself is single-group, so those two-arm assumptions do not describe its design. Planning calculations do not show that a study achieved a particular power, and they cannot replace a reported outcome analysis.
The registry record does not provide the numeric primary outcome result or an inferential analysis from which to judge the observed effect. It also does not report the investigators’ planned statistical methods, any adjustments, or assumptions used for the primary analysis. Those omissions mean that no claim about a planned or completed statistical test can be made from the record.
How should the MRI response categories be read?
The response endpoint is based on change in measurable enhancing lesions and requires stated durations for complete response, partial response, and stable disease. This makes the endpoint dependent on both the amount of measured change and the period over which the classification is sustained. General measurement principle: repeated imaging assessments can vary, and rules about measurement, timing, and confirmation help define what counts as a response. The registry record does not describe imaging schedules beyond the 12-month timeframe for the primary outcome, reader masking, central review, or procedures for resolving assessment differences.
The outcome definition distinguishes response categories, but the named primary measure is the number of participants with objective response. The registry record does not explain whether this count combines complete and partial responses, how stable disease is treated in the analysis, or how participants with missing assessments are classified. These analytic details are not reported in the registry record, so the written criteria alone do not establish how the endpoint was operationalized in the final analysis.
What do the posted analysis counts mean?
The record lists no analyses. This means it supplies no analysis result to assess, rather than demonstrating that an analysis was conducted and found no effect. No p-value, confidence interval, model estimate, or adjusted result is reported. The statistical interpretation must therefore remain descriptive of the record: a defined primary endpoint, three enrolled participants, and no posted primary outcome analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists two outcome measures: the primary objective-response count and the secondary percentage of participants who survived. It does not report a statistical analysis plan, a hierarchy of hypotheses, or any procedure for controlling multiplicity. Multiplicity concerns arise when several outcomes or comparisons are tested, because looking across multiple tests can increase the chance of a chance-positive finding. No inferential analyses are posted, so the record does not permit evaluation of how this issue was handled.
Missing-data methods are also not reported. The registry record does not state whether any participants lacked an MRI assessment, how incomplete follow-up was addressed, or how missing survival status would be treated. It does not provide participant flow or identify an analysis population. Enrollment of three should not be assumed to mean that all three contributed data to each outcome.
The record does not distinguish an intention-to-treat population from a treated or evaluable population, and it does not say whether participants who stopped treatment remained in outcome analyses. It also does not describe how treatment discontinuation, protocol deviations, or unavailable measurements were handled. These details are important because the analyzed group and missing-data rules can affect the interpretation of a response count.
Without the outcome results and these analysis details, readers cannot determine whether results were selectively available, whether all planned outcomes were reported, or how incomplete observations influenced the estimates. Such possibilities should not be assumed; the relevant information is simply not reported in the registry record.
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 | 3 | 100.0% |
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 | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Antineoplaston Therapy |
|---|---|---|
| Allergic reaction/hypersensitivity (including drug fever) | Immune system disorders | 1 of 3 |
| Infection (documented clinically): Skin (cellulitis) | Infections and infestations | 1 of 3 |
| Confusion | Nervous system disorders | 1 of 3 |
| Somnolence/depressed level of consciousness | Nervous system disorders | 1 of 3 |
| Pain: Head/headache | Nervous system disorders | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Antineoplaston Therapy |
|---|---|---|
| Nausea | Gastrointestinal disorders | 3 of 3 |
| Allergic reaction/hypersensitivity (including drug fever) | Immune system disorders | 2 of 3 |
| Central Venous Catheter Infection | Infections and infestations | 2 of 3 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 2 of 3 |
| Lymphopenia | Blood and lymphatic system disorders | 1 of 3 |
| Weight gain | General disorders | 1 of 3 |
| Edema/Fluid retention | General disorders | 1 of 3 |
| Dry mouth/salivary gland (xerostomia) | Gastrointestinal disorders | 1 of 3 |
| Vomiting | Gastrointestinal disorders | 1 of 3 |
| Infection (documented clinically): Mucosa | Infections and infestations | 1 of 3 |
Serious events with the highest arm-level rate: allergic reaction/hypersensitivity (including drug fever) (33.3%), infection (documented clinically): skin (cellulitis) (33.3%), confusion (33.3%), somnolence/depressed level of consciousness (33.3%), pain: head/headache (33.3%).
For Antineoplaston Therapy, the adverse-event summary lists three participants at risk and three participants affected by serious adverse events. It also lists three at risk and three affected by other adverse events. These are group-level counts; they do not show how many individual events occurred, how long participants were observed, or whether the same participant experienced multiple events.
The most frequently listed serious adverse-event terms each have a reported percentage of 33.3%: allergic reaction or hypersensitivity (including drug fever), infection (documented clinically), skin (cellulitis), confusion, somnolence or depressed level of consciousness, and pain: head or headache. The percentage attached to a term describes the reported frequency in this small group, not the severity of every event or the likelihood of an event in another population.
General safety principle: an event occurring during a study is not automatically caused by the intervention. The registry record does not report attribution, event timing, duration, outcome, or whether events overlapped within participants. The deaths entry is blank, so deaths are not reported in the registry record; a blank entry is not evidence of zero deaths. These summaries describe recorded safety information but do not support a causal comparison.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II, single-group study of antineoplaston therapy enrolled three participants with meningioma and was terminated. It defines a 12-month MRI-based objective-response endpoint and lists survival as a secondary outcome. It also reports serious and other adverse-event counts for the treatment group. These registry entries describe the study design and the safety entries that are present.
The primary result itself is not reported in the registry record. The number of participants who met the objective-response criteria is absent, as is a survival result. Therefore, the posted information does not show whether any participant had an objective response, how survival was observed, or how precisely either outcome was estimated. Missing results must not be read as a negative result or as evidence of no effect.
The single-group design does not provide a randomized comparison. General statistical principle: observations in a treatment group alone cannot separate intervention-related changes from other possible causes. The very small enrollment also limits the stability and precision of any group summary. No inferential analyses or uncertainty intervals are posted to quantify that uncertainty.
The safety entries indicate that three of three participants were recorded as affected by serious adverse events and three of three by other adverse events. The listed serious terms each have a reported percentage of 33.3%. These observations are important to describe, but they do not establish attribution, and the blank deaths entry does not establish that no deaths occurred.
The study status is terminated, but the reason is not reported. Taken together, the record permits a cautious account of the study’s intended question, design, enrollment, and posted safety counts. It does not support a conclusion that the therapy is effective or ineffective, or a complete assessment of benefit and risk.
13. Why This Trial Matters Statistically
This trial illustrates why a well-defined endpoint and an available result are both necessary for statistical interpretation. The registry record specifies an objective-response definition based on MRI and RANO criteria, including thresholds and durations for response categories. Yet it does not report the primary response count. A precise endpoint definition cannot substitute for the observed outcome data.
The study also shows how design constrains the claims that data can support. A single-group study can describe outcomes among enrolled participants, but it has no randomized comparator to provide a between-group contrast. Even a complete outcome count would need cautious interpretation, particularly with only three participants enrolled. General statistical principle: design and sample size determine how much uncertainty surrounds an estimate and how far an interpretation can reasonably extend.
Registry completeness is another statistical issue. The record lists a secondary survival outcome but provides no result, and it lists no analyses. It also does not explain the termination or report analysis populations and missing-data handling. These omissions leave important questions about outcome reporting and analysis unresolved without proving why information is absent.
The safety summary offers a separate example of careful reading: participant counts and term-specific percentages describe recorded events, but do not establish causation or allow a comparison with an untreated group. The record is therefore useful for understanding what was intended and what was recorded, while also showing why absent results and limited design information sharply restrict inference.
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 enrolled group is very small: three participants. This limits the amount of information available and makes any group summary sensitive to individual outcomes. The record does not report the primary response count, so even a descriptive estimate cannot be assessed. It also does not provide a confidence interval or another measure of precision.
The single-group design has no randomized comparator, and the record does not describe a like-for-like control. This restricts causal interpretation. The absence of masking is recorded as none; the registry does not describe whether outcome assessors were masked or whether images received independent review. The effect of assessment procedures cannot be evaluated from the record.
The trial is marked terminated, but the reason is not reported. The record does not say how many participants completed the planned treatment, whether all participants had outcome assessments at 12 months, or how incomplete follow-up was addressed. Eligibility details, baseline participant characteristics, treatment received in practice, and analysis populations are also not reported in the registry record.
The endpoint definitions are present, but the registry does not explain how response categories were combined for the primary count, who made the MRI assessments, or how missing scans were classified. No statistical analysis plan, sample-size justification, or analysis results are reported. The death entry in the adverse-event summary is blank, which cannot be interpreted as a zero count. Together, these omissions prevent a full appraisal of outcome estimation, safety attribution, and the reasons the study ended.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Antineoplaston Therapy in Treating Patients With Meningioma — NCT00003483.
- Registry record last updated 2017-08-24; status last verified 2017-07.
- Results first posted 2017-02-17.
- 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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18. Record Summary
NCT00003483 is a Phase II, single-group study titled “Antineoplaston Therapy in Treating Patients With Meningioma.” It enrolled three participants and is listed as terminated. The study dates recorded are 1998-06 for start and 2004-03 for primary completion.
The intervention was Antineoplaston therapy (Atengenal plus Astugenal), described as intravenous infusion every four hours with increasing doses until the maximum tolerated dose, for at least 12 months. The primary outcome was the number of participants with objective response at 12 months by MRI using stated RANO criteria. Survival percentage was listed as a secondary outcome.
The registry record does not report either outcome result or any statistical analyses. It lists three of three participants as affected by serious adverse events and three of three by other adverse events. The reason for termination, deaths, missing-data handling, and analysis populations are not reported in the registry record.