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 Stage IV Adrenal Gland Cancer (NCT00003453), a na phase 2 study. The primary outcome is Number of Participants With Objective Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00003453 |
| Phase | Phase 2 |
| Condition | Stage IV Adrenocortical Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Burzynski Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
People with Stage IV adrenocortical carcinoma were eligible for this study. The registry record lists an enrollment of 6 participants. Further details about eligibility criteria or the characteristics of participants enrolled are not reported in the registry record.
Intervention
The intervention was antineoplaston therapy, identified as Atengenal and Astugenal. The record describes intravenous infusion every four hours for at least 12 months, with increasing doses until the maximum tolerated dose was reached.
Comparator
This was a single-group study, with no comparator group described. Allocation is listed as not applicable, and the study was not masked. The design therefore does not provide a like-for-like control group for comparison.
Primary question
The primary outcome was the number of participants with an objective response at 12 months. Response was defined using stated criteria for complete or partial response. The registry record does not report the observed number of participants meeting those criteria.
The study’s stated purpose was to determine the good and bad effects of antineoplaston therapy in people with Stage IV adrenal gland cancer. Statistically, the primary question is descriptive: how many participants in this group met the response definition during the specified timeframe? Without a comparator, the study cannot directly estimate how response differs from what would have occurred under another treatment or under no study intervention.
3. Trial Design
This was a Phase II study with a single-group design. The registry record lists 6 participants enrolled, no masking, and allocation as not applicable. The study status is terminated. Its listed start date is 1996-08-21, and its primary completion date is 2011-07-05. The reason for termination and the circumstances surrounding the long interval between those dates are not reported in the registry record.
Participants were assigned to antineoplaston therapy, identified as Atengenal plus Astugenal. The intervention description specifies intravenous infusion every four hours for at least 12 months. Doses were to increase until the maximum tolerated dose was reached. The registry record does not report how many participants received the planned treatment duration, reached a maximum tolerated dose, or discontinued treatment, nor does it provide individual treatment histories.
A single-group design can describe what happened among enrolled participants, but it does not provide a concurrent comparison group. As a result, observed outcomes cannot be directly contrasted with outcomes under a different treatment within this study. Lack of masking also means that participants and study personnel were not blinded to treatment assignment. The registry record does not describe procedures that might have reduced assessment bias.
The record identifies a primary outcome and a secondary survival outcome, but does not report analysis results for either. It also does not report a protocol-level statistical analysis plan, sample-size justification, interim analysis procedures, or rules for handling participants who did not complete follow-up. Those details cannot be inferred from the study’s phase or design label.
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 | 6 |
| COMPLETED | 5 |
| NOT COMPLETED | 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 | 6 | |
| Age, Continuous | years | 45.0 (0.67 to 64.75) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 3 |
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 | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with an objective response, assessed at 12 months. The registry definition refers to response assessment for target lesions by MRI using Response Assessment in Neuro-Oncology criteria. A complete response is described as disappearance of all disease sustained for at least four weeks. A partial response is described as a decrease of at least 50% in the sum of the products of the greatest perpendicular diameters of measurable enhancing lesions, sustained for at least four weeks.
A binary response endpoint classifies each participant as meeting or not meeting the response criteria during the stated assessment period. Counting participants who meet the criteria gives a direct description of response in the enrolled group. Such endpoints are often used in Phase II studies to characterize whether responses were observed, though the interpretation depends on consistent assessment and adequate follow-up.
The registry record does not report the observed response count, the number assessed at 12 months, or a confidence interval around the response result. It also does not report how missing or unevaluable assessments were handled. Therefore, the endpoint definition is available, but the posted registry record does not allow the response frequency or its precision to be evaluated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small, single-group Phase II study and lists an enrollment of 6 participants. It identifies objective response at 12 months as the primary outcome, but does not report the number of participants with an objective response or any analysis result. Consequently, the primary outcome cannot be quantified from the registry record. The absence of a reported result is not evidence that the outcome was zero, nor does it establish that the outcome was favorable or unfavorable.
The outcome definition is based on specified imaging response criteria, including sustained complete or partial response. Those criteria define what would count as a response, but they do not supply the observed result. The record does not report the number of participants with an assessment at the relevant time, the number with missing assessments, or the handling of participants who left the study or did not complete treatment. These omissions prevent assessment of how much information contributed to the planned endpoint.
Even if a response count were available, a single-group result would describe outcomes among the enrolled participants rather than estimate a treatment effect against a concurrent control. Without a comparator, differences in underlying disease course, participant selection, supportive care, or other factors cannot be separated from the intervention’s contribution. This is a design limitation, not a conclusion about the therapy.
The registry lists survival as a secondary outcome, but does not report its result or analysis. It also lists 0 analyses. Accordingly, no estimate of response, survival, uncertainty, or statistical significance is available in the registry record. A statement about the treatment’s effect would require results and a suitable basis for comparison that are not reported.
The adverse-event record is a separate descriptive component. It lists 4 participants with serious adverse events among 6 at risk and 6 participants affected by other adverse events among 6 at risk. Several serious adverse event terms are each listed at 16.7%. These entries describe recorded safety events; they do not supply a comparative estimate of risk, and the listed terms should not be treated as proof that an event was caused by the intervention. The record does not report deaths in the adverse-event field.
Overall, the registry record supports a description of the planned endpoint, intervention, design, enrollment, and some adverse-event entries. It does not provide the primary outcome result or inferential analyses. The statistical interpretation is therefore constrained by missing results and by the absence of a control group.
9. Statistical Methods Explained
For a binary endpoint such as objective response, the outcome is classified into two categories: the participant meets the response definition or does not. A count describes how many participants fall into the response category. A proportion can also describe the share of assessed participants who responded, but its interpretation depends on knowing both the response count and the appropriate denominator. The registry record states the primary endpoint as a count and lists enrollment of 6; it does not report the endpoint result or the number assessed. No response proportion can therefore be interpreted from the registry record.
What would a confidence interval show?
In general, a confidence interval expresses the uncertainty around an estimate. For a response proportion, it would indicate a range of values compatible with the observed data under the method used. With a small dataset, estimates can be imprecise, and the interval can remain wide even when a point estimate appears clear. A confidence interval does not correct for a biased design, missing observations, or the lack of a comparison group. The registry record does not report a confidence interval for the primary endpoint.
What can a single-group study estimate?
In general, a single-group study can describe outcomes among participants who received the intervention. It cannot directly estimate a between-group treatment contrast because no concurrent comparator group is present. A before-and-after comparison, if one were available, would answer a different question and could be affected by changes over time or by how measurements were collected. No such analysis is reported in the registry record. The study is not a randomized comparison, so the observed group should not be read as if it were a treatment arm matched to a control arm.
How should event counts be read?
Counts are useful for conveying the number of participants or events recorded, but their meaning depends on the unit being counted. A count of participants with an event is different from a count of all event occurrences: one participant may experience more than one event. The safety entries specify participants at risk and affected, while the listed serious-event terms carry percentages. The registry record does not clarify whether individuals could appear under multiple terms, so those term entries should not be added together or interpreted as mutually exclusive categories.
Why does over-dispersion matter?
In general, over-dispersion occurs when observed variation in count data exceeds what a chosen statistical model expects. If a model assumes less variation than the data contain, standard errors may be too small and uncertainty may be understated. This issue can matter when analyzing event counts, particularly when participants have different observation times or event rates. The primary endpoint in this study is reported as a participant count with a binary response definition, and no count-model analysis or dispersion estimate is reported. Over-dispersion should not be presumed to have been assessed.
What do planning calculations mean?
In general, sample-size calculations are planning tools: they estimate how many participants would be needed under specified assumptions to detect a chosen effect with a chosen statistical procedure. They are not evidence that a study had a particular probability of success after enrollment, and they are not observed power. The stated planning assumptions for illustrations associated with these results use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and a target effect expressed as a fraction of the observed effect. For time-to-event calculations, the stated output is a total event count; other calculations are expressed as participants per arm. Those are generic planning assumptions, not evidence that this single-group trial used a two-arm design or followed those assumptions in its own protocol. The registry record does not report the trial’s own sample-size justification or statistical plan.
More generally, statistical significance tests require an explicit hypothesis, analysis method, and observed data. A p-value, when calculated, summarizes how incompatible the data are with a specified null model; it does not measure the probability that a treatment works, and it does not establish causation. No analysis results are reported in the registry record, so there is no reported test or p-value to interpret for the primary endpoint.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 2 outcome measures and identifies objective response as the primary outcome and survival as a secondary outcome. It reports 0 analyses. No statistical analysis results are reported for either outcome. The record does not describe a multiplicity strategy, such as how interpretation would account for testing more than one outcome or making repeated comparisons. It is therefore not possible to determine whether any formal adjustment was planned or applied.
Missing-data methods are also not reported in the registry record. The record does not state how participants without a 12-month response assessment would be classified, whether incomplete follow-up would be treated as nonresponse, or whether another analysis population was used. It does not give the number assessed for the primary endpoint. These details affect the denominator and interpretation of a binary response result, so their absence matters even though the response result itself is not reported.
The analysis population is not described. The registry record does not specify whether outcomes were to be analyzed for all enrolled participants, only those who received treatment, or a defined subset with evaluable assessments. Nor does it describe exclusions or protocol deviations. Without this information and without analysis outputs, readers cannot determine which participants contributed to any endpoint estimate.
These are reporting gaps, not proof that the study did not have internal procedures. The methods for multiplicity, missing data, and analysis populations are 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 | 4 | 6 | 66.7% |
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 | 6 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | Antineoplaston Therapy |
|---|---|---|
| Ascites (non-malignant) | Gastrointestinal disorders | 1 of 6 |
| Musculoskeletal/Soft Tissue - Other (Muscle Spasms) | Musculoskeletal and connective tissue disorders | 1 of 6 |
| Fever | General disorders | 1 of 6 |
| Central Venous Catheter Infection | General disorders | 1 of 6 |
| Somnolence/depressed level of consciousness | Nervous system disorders | 1 of 6 |
| Dizziness | Nervous system disorders | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Antineoplaston Therapy |
|---|---|---|
| Hypokalemia | Investigations | 6 of 6 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 5 of 6 |
| Nausea | Gastrointestinal disorders | 5 of 6 |
| Hemoglobin | Blood and lymphatic system disorders | 4 of 6 |
| Vomiting | Gastrointestinal disorders | 4 of 6 |
| Alkaline phosphatase | Investigations | 4 of 6 |
| Hypoglycemia | Investigations | 4 of 6 |
| Pain: Abdomen NOS | Gastrointestinal disorders | 4 of 6 |
| Hypertension | Cardiac disorders | 3 of 6 |
| Fever | General disorders | 3 of 6 |
Serious events with the highest arm-level rate: ascites (non-malignant) (16.7%), musculoskeletal/soft tissue - other (muscle spasms) (16.7%), fever (16.7%), central venous catheter infection (16.7%), somnolence/depressed level of consciousness (16.7%).
The registry record lists 4 participants with serious adverse events among 6 at risk, and 6 participants affected by other adverse events among 6 at risk. These are participant counts in the antineoplaston therapy group. The record does not provide a comparison group, so the counts do not estimate how safety outcomes differ from another treatment or from no study intervention.
The listed serious adverse event terms are Ascites (non-malignant), Musculoskeletal/Soft Tissue - Other (Muscle Spasms), Fever, Central Venous Catheter Infection, Somnolence/depressed level of consciousness, and Dizziness. Each term is listed as 16.7%. The registry record does not clarify whether the same participant could be represented under multiple terms, or whether the percentage refers to participants or events. The term percentages should therefore be read as separate entries, not added together.
Adverse-event reporting does not by itself establish causation. The record does not report an assessment attributing events to the intervention, event timing, duration, or outcomes. The deaths field is not reported in the registry record, which should not be interpreted as a count of zero deaths. These figures describe what the registry lists, with important limits on comparison and attribution.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II, single-group study of antineoplaston therapy was registered for Stage IV adrenocortical carcinoma, with 6 participants enrolled. It describes the intervention and defines objective response at 12 months as the primary endpoint. It also lists a secondary survival outcome and adverse-event entries.
What the record does not establish is the observed objective-response result. It provides no count of participants with response, no response estimate, no confidence interval, and no analysis result. The absence of a registry result cannot be interpreted as no responses, as evidence of responses, or as a statistical finding. Survival results are also not reported.
The design limits what could be concluded even if an outcome count were available. With a single group and no comparator, the study could describe outcomes among participants enrolled, but it could not directly compare those outcomes with a control group in the same study. The record’s lack of masking is another design feature relevant to assessment, but there is no basis in the registry record for quantifying its effect on measurement.
The safety entries show recorded serious and other adverse events among the study group. They do not establish that the intervention caused those events, and they do not support a comparative safety estimate. The record does not report death counts in this field or provide enough detail to interpret overlap among event terms.
In short, the registry record documents the study’s purpose, design, endpoint definition, enrollment, and selected safety information. It does not contain the results needed to estimate response or survival, assess precision, or draw a comparative conclusion. Statistical interpretation should remain limited to those documented details.
13. Why This Trial Matters Statistically
This study illustrates why a clearly stated endpoint and a reported endpoint result are both necessary for statistical interpretation. The registry defines objective response using imaging criteria and a 12-month timeframe, but does not report how many participants met the definition. Without that result, readers cannot estimate the response frequency or its uncertainty.
The single-group design also shows the distinction between describing outcomes and estimating a treatment contrast. A count in a treated group may be informative as a description, but a comparison requires a credible reference group or another design that supports a counterfactual. The registry record lists no comparator, so it does not support a like-for-like treatment comparison.
The listed enrollment of 6 highlights the importance of denominator details and precision in small studies. A response count, number assessed, and uncertainty estimate would be needed to understand how much the observed data constrain the underlying response frequency. Those results are not reported in the registry record.
Finally, this record separates safety reporting from causal interpretation. Adverse-event counts and terms can document what was recorded, while still leaving attribution, timing, overlap, and comparison unresolved. The statistical value of a registry record depends not only on design descriptions but also on complete, interpretable outcome reporting. Here, the available entries do not answer the primary endpoint question.
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 limitations are specific to the information reported for this study. The enrollment is 6, and the study has a single-group design with no comparator. That limits both precision and the ability to distinguish treatment-associated outcomes from other explanations. The registry record does not report the primary objective-response result, despite specifying the endpoint and its 12-month timeframe.
The record does not report the number of participants assessed at 12 months, the number with incomplete assessments, or how missing outcome data were handled. It also does not describe the analysis population. As a result, even the relevant denominator for an endpoint estimate is not available.
The study status is terminated, but the reason for termination is not reported in the registry record. The record also does not report treatment completion, dose levels reached, individual follow-up, or the number of participants contributing to the survival outcome. These omissions prevent interpretation of treatment exposure and outcome ascertainment.
Safety reporting is limited to participant counts and listed event terms. Timing, duration, severity details beyond the serious-event designation, attribution, and overlap among terms are not described. The deaths field is not reported in the registry record.
Finally, the registry record does not report the trial’s statistical analysis plan, sample-size justification, multiplicity approach, or methods for missing data. These details should not be inferred from the Phase II designation. Together, the absent outcome results and limited design and analysis details substantially constrain what can be concluded statistically.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Antineoplaston Therapy in Treating Patients With Stage IV Adrenal Gland Cancer — NCT00003453.
- Registry record last updated 2018-05-09; status last verified 2018-04.
- Results first posted 2018-05-09.
- 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
The registry record describes a Phase II study of antineoplaston therapy, identified as Atengenal and Astugenal, in Stage IV adrenocortical carcinoma. It lists 6 participants enrolled, a single-group design, no masking, and a terminated status. The primary outcome is the number of participants with objective response at 12 months, defined using stated imaging response criteria.
The record does not report the primary endpoint result, a survival result, or analysis results. It lists serious adverse events in 4 participants among 6 at risk and other adverse events in 6 participants among 6 at risk; listed serious-event terms each carry a reported percentage of 16.7%. No comparator is described, and the record does not report the statistical plan, missing-data procedures, or analysis population. The available information supports description of the study design and selected safety entries, but not estimation of response or a comparative treatment effect.