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 Advanced Mesothelioma (NCT00003508), a na phase 2 study. The primary outcome is Number of Participants With Objective Response, Stable Disease, Progressive Disease or Not Evaluable.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00003508 |
| Phase | Phase 2 |
| Condition | Malignant Mesothelioma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Burzynski Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Patients with malignant mesothelioma were eligible for the study. The registry describes the target group as patients with advanced mesothelioma, and identifies the primary-outcome population as all patients enrolled. Enrollment was 8.
Intervention
The experimental intervention was antineoplaston therapy, listed as Atengenal + Astugenal. The arm description says the drugs were administered by IV infusion every four hours for at least 12 months, with increasing dosages until the maximum tolerated dose was reached.
Comparator
There was no comparator arm. The study is listed as single-group, with allocation recorded as not applicable and masking as none. Consequently, the registry record does not provide a concurrent control group for a like-for-like treatment comparison.
Primary question
The primary outcome asks how many participants were classified as having objective response, stable disease, progressive disease, or being not evaluable. The registry gives response-category definitions, but category counts and an inferential result are not reported in the registry record.
The study’s stated purpose was to determine the good and bad effects of antineoplaston therapy in advanced mesothelioma. Its primary outcome combines disease-response classifications with a not-evaluable category, so it is intended to describe participants’ recorded disease status rather than estimate a comparison against another treatment. The record does not report a control group, random assignment, or masking. The available information therefore addresses what was recorded among enrolled participants, not whether outcomes differed from what would have happened under another treatment or without treatment. The small enrolled group and lack of reported outcome counts also limit how much can be concluded about the distribution of response categories.
3. Trial Design
This was a Phase 2 study listed as single-group and non-masked. The registry records allocation as not applicable, consistent with the absence of a randomized allocation between study arms. There was one experimental arm, described as antineoplaston therapy. The record lists enrollment of 8 and gives malignant mesothelioma as the condition. The study status is terminated. The reason for termination is not reported in the registry record.
The arm description specifies IV infusion every four hours for at least 12 months. Dosages of Atengenal and Astugenal were to increase until the maximum tolerated dose was reached. The registry title refers to Antineoplastons A10 and AS2-1, while the intervention and arm description use the names Atengenal and Astugenal; the record does not explain the naming relationship. The study start is recorded as 1996-03-08, and primary completion as 2005-04-26.
In a single-group study, the observed outcomes describe the enrolled participants under the study intervention. Without a concurrent control group, the design cannot separate the intervention’s effects from other explanations for participants’ outcomes, such as the course of disease or changes occurring over time. That limitation is distinct from the issue of sample size: even a larger single-group study would not supply a randomized control comparison. No protocol details beyond those recorded, including the rationale for the design or the reason the study ended, are reported in the registry record.
The record identifies one primary outcome measure and lists no analysis results. It does not report a statistical analysis plan, any interim analyses, or a justification for the planned enrollment. The registry status and completion dates are descriptive record details; they do not explain why the trial was terminated or establish when outcome analyses were conducted.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Arm: Experimental: Antineoplaston Therapy |
|---|---|
| STARTED | 8 |
| COMPLETED | 3 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | Arm: Experimental: Antineoplaston Therapy |
|---|---|
| Not evaluable | 5 |
Analysis population for the primary outcome, as defined in the registry: All patients enrolled in the study.
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.
Population: Eight patients were enrolled in the study, only 3 patient was evaluable.
| Characteristic | Unit | Arm: Experimental: Antineoplaston Therapy |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 60.0 (SD 8.9527) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Objective Response, Stable Disease, Progressive Disease or Not Evaluable, measured over 109 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is the number of participants classified as having objective response, stable disease, progressive disease, or being not evaluable, among all patients enrolled. Its timeframe is listed as 109 months. The measure is categorical: it assigns a participant to a disease-status category rather than reporting a continuous change in tumor size or a time-to-event estimate.
The registry describes objective response using the 1999 International Working Group response criteria. Complete response means disappearance of all disease sustained for at least four weeks. Partial response means a >=50% decrease in the sum of the products of the greatest perpendicular diameters of measurable lesions, sustained for at least four weeks. Stable disease is described as a < 50% change in that sum, sustained for at least twelve weeks. The registry does not give a definition for progressive disease in the outcome description.
Response categories can provide a structured way to describe tumor status, while the not-evaluable category signals that a participant could not be assigned to the listed disease-status groups. However, the registry record does not report the number of participants in any category. It therefore does not establish the observed response distribution or allow the precision of category-specific estimates to be assessed. The endpoint’s stated timeframe does not, by itself, explain when or how often assessments were made.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies 8 enrolled participants and one primary outcome measure, but it does not report the outcome counts for objective response, stable disease, progressive disease, or not evaluable. No effect estimate, confidence interval, or test result for the primary outcome is reported in the registry record. The primary outcome therefore cannot be interpreted as evidence for a particular response distribution from these posted results. The endpoint definitions explain how some categories were intended to be classified, but definitions are not outcome findings.
The single-group design is important when interpreting any observed outcomes. If category counts were available, they would describe the enrolled group under the study intervention. They would not, on their own, quantify a treatment effect relative to another therapy or a no-treatment condition. There is no randomized or concurrent control arm in the record, and no allocation comparison is available. The absence of masking is also recorded, although the registry does not describe how outcome assessments were conducted or whether assessors knew the treatment exposure.
Precision matters even for descriptive estimates. In general, a confidence interval conveys the range of values compatible with the data under a specified statistical method; smaller samples commonly yield less precise estimates. Here, no primary-outcome estimate or interval is reported, so the precision cannot be evaluated. The enrolled sample was 8, but it would be inappropriate to infer a response rate or its uncertainty without the category counts.
The safety information is separate from the primary endpoint. The registry lists 2 participants affected by serious adverse events among 8 at risk, and 8 participants affected by other adverse events among 8 at risk. It also lists three serious adverse-event terms, each with a reported percentage of 12.5%. These data describe recorded safety events in this group; without details about event timing, recurrence, severity grading, or attribution, they do not establish how the events relate to treatment. A death count is not reported in the registry record.
The study is marked terminated, but the reason is not reported in the registry record. Termination should not be interpreted as evidence of benefit, lack of benefit, harm, or a particular operational problem. Taken together, the registry supports a description of the study design, planned outcome classification, and selected safety counts. It does not report enough primary-outcome information to assess the trial’s observed disease-response findings or their statistical uncertainty.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study describes outcomes observed among participants receiving the study intervention. It does not directly estimate the difference between outcomes under the intervention and outcomes under a comparator. A randomized comparison can help distinguish treatment effects from other influences by creating groups whose differences are attributable, on average, to the assigned treatment. This study has no such comparison: it is listed as single-group, with allocation not applicable. Any outcome counts, if reported, would therefore be descriptive of this enrolled group rather than a controlled estimate of comparative efficacy.
The study is also listed as non-masked. In general, masking can reduce the influence of expectations on care, reporting, or outcome assessment, depending on who is masked and how outcomes are measured. The registry does not describe masking procedures beyond recording none, nor does it specify assessment procedures. The potential effect of this design feature on outcome classification cannot be quantified from the record.
What does a categorical endpoint tell the reader?
A categorical endpoint places participants into distinct outcome groups. Here, the named groups are objective response, stable disease, progressive disease, and not evaluable. In general, category counts describe how many participants fall into each classification. They do not automatically provide a comparative treatment effect, and interpretation depends on the definitions, assessment timing, and handling of participants who cannot be evaluated.
The registry gives criteria for complete response, partial response, and stable disease, including the required changes in measured lesions and the duration over which those changes must be sustained. It does not describe the progressive-disease criterion in the outcome text. Nor does it report the category counts. The outcome measure is therefore identifiable, but the result itself and its precision are not available in the registry record. The listed timeframe of 109 months should be read as the endpoint timeframe recorded, not as a description of a specific assessment schedule; that schedule is not reported in the registry record.
Why does uncertainty matter?
In general, an estimate is more informative when accompanied by a measure of uncertainty. A confidence interval communicates the range of estimates compatible with the observed data under the chosen method. It is not a guarantee that the true value lies in that interval, and its interpretation depends on the statistical assumptions and study design. A small enrolled group can make estimates unstable, but no interval can be evaluated because the registry record does not report primary-outcome counts or an estimate.
A p-value, when reported, measures how surprising data would be under a specified null hypothesis and statistical model. It does not measure the probability that a treatment works, nor does statistical significance alone establish clinical importance. No primary-outcome p-value or inferential analysis is reported in the registry record. There is consequently no basis in these results for a significance claim.
How should planning illustrations be understood?
General sample-size planning asks how many participants would be needed to detect a specified effect under chosen assumptions. The stated planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect defined as a stated fraction of an observed effect. Such calculations are illustrations, not observed trial findings and not the trial’s own design justification. They do not represent observed power, which cannot be inferred from a completed study’s result.
The current trial is single-group, whereas those assumptions describe equal allocation between two arms. That contrast makes the planning framework a hypothetical comparison rather than a description of this trial’s design. Further, the registry record does not report the primary-outcome category counts or an observed effect from which a reader could judge the planning target. The trial’s sample-size justification is not reported in the registry record. Any illustration based on an effect and selected assumptions should therefore be read as conditional planning, not as evidence about the results or adequacy of the study.
What cannot be reconstructed?
Statistical methods depend on details such as the analysis population, endpoint coding, missing-data rules, and planned estimand. An estimand is the precise treatment-effect question an analysis is intended to answer, including how events such as discontinuation or unavailable assessments are handled. The registry identifies the primary population as all patients enrolled, but it does not give a detailed analysis plan, missing-data strategy, or estimand. It also does not report a model, uncertainty interval, or outcome analysis.
These omissions prevent a reader from reproducing an inferential analysis or checking whether the analysis followed a prespecified plan. They do not prove that analyses were never performed; they mean such details and results are not reported in the registry record. In general, methods should be interpreted alongside the design and reporting completeness, rather than inferred from the endpoint title or safety summaries.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study tests or estimates several outcomes, subgroups, time points, or treatment comparisons. In general, carrying out many statistical tests can increase the chance of a seemingly notable result arising by chance, so an analysis plan may specify how multiple comparisons will be handled. The registry identifies one primary outcome measure, but it does not report the number of planned statistical tests or a multiplicity strategy. No interim analyses or inferential results are reported in the registry record. It is therefore not possible to assess how multiplicity was addressed.
The primary outcome includes four possible classifications, including not evaluable. The registry describes the population as all patients enrolled, but it does not report category counts or explain how participants with unavailable assessments would be treated analytically. The not-evaluable category is part of the endpoint description; its presence does not reveal how many participants were not evaluable, whether any data were missing, or why an assessment might be unavailable.
In general, missing-data methods can affect estimates when the probability of missingness is related to participant characteristics or outcomes. A complete-case analysis, imputation, or another approach makes different assumptions. The record does not identify any such method, and it does not say whether missing outcome data occurred. No missing-data mechanism should be inferred from the outcome categories alone.
The registry identifies all enrolled patients as the primary-outcome population. It does not state whether any separate population was used for safety or other analyses, or how discontinuations and protocol deviations were handled. No statistical analysis plan or population-specific analysis details are reported in the registry record. As a result, neither the completeness of the analysis population nor the robustness of findings to missing-data assumptions can be assessed.
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 |
|---|---|---|---|
| Arm: Experimental: Antineoplaston Therapy | 2 | 8 | 25.0% |
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 |
|---|---|
| Arm: Experimental: Antineoplaston Therapy | 8 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm: Experimental: Antineoplaston Therapy |
|---|---|---|
| Hemorrhage, pulmonary: Bronchopulmonary NOS | Respiratory, thoracic and mediastinal disorders | 1 of 8 |
| Infection NOS | Infections and infestations | 1 of 8 |
| Seizure | Nervous system disorders | 1 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm: Experimental: Antineoplaston Therapy |
|---|---|---|
| Fatigue (asthenia, lethargy, malaise) | General disorders | 5 of 8 |
| Hypernatremia | Investigations | 4 of 8 |
| Edema/Fluid retention | General disorders | 3 of 8 |
| Diarrhea | Gastrointestinal disorders | 3 of 8 |
| Nausea | Gastrointestinal disorders | 3 of 8 |
| Allergic reaction/hypersensitivity (including drug fever) | General disorders | 2 of 8 |
| Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip) | General disorders | 2 of 8 |
| Hemoglobin | Blood and lymphatic system disorders | 2 of 8 |
| Lymphopenia | Blood and lymphatic system disorders | 2 of 8 |
| Albumin, serum-low (hypoalbuminemia) | Investigations | 2 of 8 |
Serious events with the highest arm-level rate: hemorrhage, pulmonary: bronchopulmonary nos (12.5%), infection nos (12.5%), seizure (12.5%).
The safety record lists 8 participants at risk for serious adverse events and 2 affected. For other adverse events, it lists 8 at risk and 8 affected. These are participant counts, not a measure of the total number of events or their duration. One participant may experience more than one event, and the registry information does not specify recurrence or event-level counts.
The listed serious adverse-event terms are “Hemorrhage, pulmonary: Bronchopulmonary NOS,” “Infection NOS,” and “Seizure.” Each term is accompanied by a reported percentage of 12.5%. The registry does not clarify the denominator or whether participants could contribute to more than one term, so the percentages should be read as recorded rather than converted into participant counts or added together.
The record does not report a death count. It also does not provide details about event onset, duration, severity grading beyond serious-event classification, resolution, or attribution to study treatment. These figures describe recorded safety events in the study group; they do not by themselves establish causation or permit a comparison with another treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase 2, single-group study of antineoplaston therapy enrolled 8 participants with malignant mesothelioma and was terminated. It identifies a primary outcome based on disease-response categories and provides definitions for some of those categories. It also records selected participant-level safety counts and serious adverse-event terms. These are useful descriptive elements for understanding what the study set out to observe and what safety information was entered in the registry.
The record does not report the number of participants with objective response, stable disease, progressive disease, or not evaluable. It therefore does not show the observed primary-outcome distribution. It also does not report an effect estimate, confidence interval, or p-value for that endpoint. Without these results, the statistical strength and precision of the primary outcome cannot be judged. The outcome definitions alone are not evidence that participants experienced any particular category.
The lack of a comparator means that even reported outcomes would describe this group rather than estimate a contrast with another treatment. A single-group design cannot rule out alternative explanations for observed changes. The absence of masking is recorded, but the record does not provide enough assessment detail to determine whether it influenced classification.
The safety counts indicate that serious and other adverse events were recorded among participants, but they do not establish that the intervention caused those events. The registry does not give the event-level detail or death count needed for a fuller safety interpretation. Termination is a study-status entry, not an explanation of the study’s outcomes. The reason for termination is not reported in the registry record. Overall, these data support cautious description of the study and its reporting limits, not a conclusion about treatment effectiveness or comparative safety.
13. Why This Trial Matters Statistically
This trial illustrates why study design and outcome reporting must be considered together. A clearly named endpoint and response criteria are not enough to interpret a result if the category counts and uncertainty estimates are absent. The endpoint description indicates what classifications were intended, but the registry does not show how participants were distributed among them. That distinction separates a planned measurement from a reported finding.
The single-group design also defines the kind of statistical question the study can answer. It can describe outcomes among enrolled participants, but it cannot provide a randomized comparison against another treatment. This limitation concerns causal interpretation, not simply the precision of a numerical estimate. More participants could improve precision for a descriptive result, but would not create a concurrent control group.
The safety record highlights a related reporting issue: participant counts, event terms, and percentages answer different questions. Counts of affected participants do not reveal the total number of events, while term percentages cannot be translated into participant counts without a specified denominator and counting convention. A useful safety interpretation depends on those details as well as information about timing and attribution.
Finally, planning assumptions should not be confused with trial design or observed evidence. Illustrative calculations under two-arm assumptions do not describe this single-group study, and they are not observed power. Statistical education benefits from keeping these categories separate: protocol design, observed outcomes, uncertainty, and hypothetical planning each have distinct meanings. The registry’s omissions make that distinction especially important for this record.
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 primary limitation for statistical interpretation is the absence of reported category counts for the primary outcome. Without those counts, the response distribution, any effect estimate, and its precision cannot be assessed. The registry lists one outcome measure, but no analysis result is reported. It is also not possible to determine from the record whether outcome data were complete or how participants who could not be evaluated were handled.
The single-group design provides no concurrent comparator. Outcomes therefore cannot be interpreted as a controlled treatment effect, and other explanations for disease status cannot be separated from the intervention. Masking is listed as none; the registry does not explain who assessed disease status or what procedures were used to reduce assessment bias. The progressive-disease criteria are not described in the primary outcome text, although that category is included in the outcome title.
Enrollment was 8, which limits the amount of descriptive information available from the trial. The record does not provide a sample-size justification, a statistical analysis plan, an interim-analysis plan, or details of missing-data handling. It does not state why the study was terminated. These gaps prevent assessment of prespecification, analysis choices, and the circumstances surrounding study completion.
Safety reporting is also limited: the death count, event-level counts, timing, and treatment attribution are not reported in the registry record. The denominator for the serious-event term percentages is not specified. These limitations are specific to the information recorded; they should not be taken as proof that the omitted procedures or information did not exist outside the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Antineoplaston Therapy in Treating Patients With Advanced Mesothelioma — NCT00003508.
- Registry record last updated 2021-04-27; status last verified 2021-01.
- Results first posted 2021-02-21.
- 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
The registry describes a Phase 2, single-group study of antineoplaston therapy in patients with malignant mesothelioma. Enrollment was 8, masking was none, and the study status is terminated. The intervention is listed as Atengenal + Astugenal, administered by IV infusion every four hours for at least 12 months, with dosage increases until the maximum tolerated dose was reached.
The primary outcome is the number of participants classified as having objective response, stable disease, progressive disease, or being not evaluable, with a timeframe of 109 months. The registry provides criteria for complete response, partial response, and stable disease, but does not report category counts or an inferential analysis. Safety figures list 2 of 8 participants affected by serious adverse events and 8 of 8 affected by other adverse events. A death count is not reported in the registry record. The reason for termination and the statistical analysis plan are also not reported in the registry record.