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 Bicarbonate for Tumor Related Pain (NCT01350583), a na na study. The primary outcome is Percent of Patients With Improvement.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01350583 |
| Phase | NA |
| Condition | Neoplasm Related Pain (Acute) (Chronic) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
People with neoplasm-related acute or chronic pain were the intended population. The registry record reports enrollment of 2, but does not describe participant characteristics or provide a detailed account of eligibility in the posted results. The planned outcome description refers to patients with tumor-related pain.
Intervention
The intervention was oral sodium bicarbonate, described as a dose-escalation therapy. The study aimed to examine tolerance of doses higher than those usually used for heartburn and whether pain intensity might improve. The registry record does not report the dose levels or how dose escalation was carried out.
Comparator
No comparator group is listed. The study is recorded as a single-group design, with allocation and masking both listed as NA or none. Consequently, the design does not provide a like-for-like control comparison for separating a treatment effect from other explanations for changes over time.
Primary question
The primary outcome was the percent of patients with greater than 30% improvement in pain intensity by visual assessment scale over 4 weeks per participant. This was a planned measure, not a reported result: the registry says outcome data were not calculated because accrual was low and the goal of 25 evaluable participants was not reached.
The clinical question therefore had two related parts: whether higher-dose oral sodium bicarbonate could be tolerated, and whether pain intensity might improve. The registry also identifies reduction in pain-medication requirements as an aim in its study description, but does not report a result for that question. With a single group and no posted primary outcome calculation, the record cannot establish a comparative treatment effect. It documents the intended questions and the limited amount of information available from the terminated pilot.
3. Trial Design
This was a pilot study of oral sodium bicarbonate as an adjunct for tumor-related pain, sponsored by H. Lee Moffitt Cancer Center and Research Institute. The registry lists the phase as NA, the model as single group, allocation as NA, and masking as none. The treatment group is described as sodium bicarbonate therapy with dose escalation. There is no listed placebo or other control arm, so the design was not a randomized comparison.
The record lists enrollment of 2. The study started in 2010-08, had a primary completion date of 2012-05, and is marked terminated. A reason for termination is not reported in the registry record. The planned outcome description says that outcome measures were based on 25 evaluable participants and that this goal was not reached. The record specifically attributes the lack of calculated results to low accrual.
The primary outcome timeframe was 4 weeks per participant. The intended primary measure was the percent of patients whose pain intensity improved by greater than 30% on a visual assessment scale. Secondary outcomes concerned the percent of patients for whom treatment was well tolerated and the number of participants with improvement in pain indices. The registry lists three outcome measures, but does not report calculated outcome results.
There is no posted control-group estimate, masking procedure, allocation process, or detailed schedule of dose escalation. The protocol’s statistical plan, including any planned interim analyses or sample-size justification, is not reported in the registry record. The design description supports reading this as a small, noncomparative pilot intended to explore tolerability and possible pain changes, rather than as a trial capable of estimating a controlled treatment effect.
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 | Sodium Bicarbonate Therapy |
|---|---|
| STARTED | 2 |
| COMPLETED | 2 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Results data was not calculated due to low accrual. Outcome Measures were based on 25 evaluable participants. That goal was not reached.
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 | Sodium Bicarbonate Therapy |
|---|---|---|
| Participants analysed | 2 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Percent of Patients With Improvement, measured over 4 weeks per participant.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the percent of patients with greater than 30% improvement in pain intensity by visual assessment scale. Its timeframe was 4 weeks per participant. The threshold defines the kind of change the study planned to count as improvement; the endpoint was not simply any change in the visual assessment score. The record does not provide the scale’s full scoring method, assessment schedule, or a calculated percentage.
As a general statistical concept, a threshold-based endpoint can make a clinical outcome easier to summarize: each participant is classified according to whether their change meets the stated improvement criterion, and the group result can be expressed as a percentage. That summary is meaningful only when the relevant observations and analysis population are available. A percentage without its underlying count and denominator can also be difficult to assess for precision.
In this study, the registry states that the results data were not calculated because accrual was low and the goal of 25 evaluable participants was not reached. Thus the endpoint describes the intended primary question, not an observed estimate of improvement. There is no reported count meeting the threshold, group percentage, or uncertainty estimate. The study description also mentions pain-medication requirements, but that is not the stated primary endpoint and no result for it is reported.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the primary outcome was not calculated. The registry says this was because of low accrual, and states that the outcome measures were based on 25 evaluable participants, a goal that was not reached. Enrollment is listed as 2. These registry entries leave no reported primary estimate to interpret: the record does not give the number of patients whose pain improved by greater than 30%, the corresponding percentage, or an uncertainty interval.
The study’s single-group structure also limits what a pain change, if one had been reported, could establish. Without a control group, changes over time could not be contrasted with changes in a comparable untreated or differently treated group. This is a general limitation of noncomparative designs, not evidence that any particular alternative explanation occurred in this study. Masking is listed as none, and the record does not describe a blinded outcome assessment.
The registry identifies tolerability and improvement in pain indices as secondary outcomes, but does not post their calculated results. It also describes an interest in whether sodium bicarbonate could reduce pain-medication requirements; a result for that question is not reported in the registry record. No inferential analyses are listed. Accordingly, the record does not provide an estimated treatment effect, a test of a treatment difference, or a basis for assessing statistical precision for the primary endpoint.
The safety entries are also sparse. For the sodium bicarbonate therapy group, serious adverse events are listed for 0 of 2 at risk, while other adverse events are listed for 1 of 2 at risk. These are event counts in a very small group, not estimates of a stable event rate. The registry does not list serious adverse event terms, and the death count is not reported in the registry record. The entries do not establish whether an event was caused by the intervention.
The study is marked terminated, but the reason is not reported in the registry record. The posted information can therefore describe the intended endpoint, the study’s limited accrual, and the recorded adverse-event counts. It cannot resolve whether the intervention reduced pain, altered medication requirements, or was generally tolerable in a broader population. A missing calculated outcome is not evidence of either improvement or no improvement; it means the registry record does not provide that result.
9. Statistical Methods Explained
The registry describes a single-group dose-escalation study, not a randomized comparison. This distinction determines what statistical questions can be answered. A single group can be described over time, but a comparison against a concurrent control group is not available in the listed design. Allocation is NA and masking is none. The registry record does not report a more detailed statistical analysis plan.
What does the primary endpoint summarize?
The primary outcome was the percent of patients with greater than 30% improvement in pain intensity by visual assessment scale, over 4 weeks per participant. This is a threshold-based endpoint: the intended summary classifies participants according to whether their observed improvement passes the specified threshold. It does not report the average change in a pain score, and those two summaries would answer different questions. The registry does not report a calculated result for either one.
In general, a percentage is formed from a count meeting a definition and a denominator drawn from a defined analysis population. The record says outcome measures were based on 25 evaluable participants, but that goal was not reached; it attributes the lack of calculated data to low accrual. It does not give a count meeting the improvement criterion or a denominator used for a final estimate. No percentage can therefore be interpreted from the posted primary outcome.
What can a confidence interval add?
In general, a confidence interval expresses the uncertainty around an estimated quantity under a stated statistical method and its assumptions. It helps readers distinguish a relatively precise estimate from one compatible with a wide range of values. A confidence interval does not correct for a weak design, missing observations, or a lack of a comparator. No confidence interval for the primary outcome is reported in the registry record, and without a calculated estimate there is no primary interval to interpret.
Why does the design matter for inference?
For a comparative treatment-effect question, a control group provides a reference for what might have happened over the same period without the study intervention or under another intervention. This trial has no listed comparator. As a general matter, a change within a single group can reflect multiple influences, and the design does not separate those influences from an intervention effect. This is why a within-group observation should not be read as the same quantity as a randomized between-group treatment contrast.
Masking is listed as none. In general, knowledge of treatment assignment can influence reports, assessment, or other aspects of study conduct, depending on the outcome and procedures. The registry does not specify whether any independent outcome assessment or other safeguards were used, so those details are not reported in the registry record.
How should planning calculations be read?
Any sample-size figures associated with planning illustrations are not observed results and are not the trial’s own design justification. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. The trial itself is listed as a single-group study, so the hypothetical two-arm allocation assumption does not describe its registered design.
The registry record does not report an observed primary effect estimate from which a planning illustration could be interpreted as a trial-specific basis for future sample-size planning. Such calculations, when based on an effect estimate, are sensitive to the estimate chosen and to assumptions about the future study. They are planning tools, not evidence that the current study had a particular probability of detecting an effect. They are not observed power, and they do not supply a result for the uncalculated endpoint.
What is known about the analysis plan?
The record lists three outcome measures and zero analyses. It does not report a statistical analysis plan, an interim-analysis plan, or a sample-size justification. No model, adjustment strategy, or inferential procedure for the primary outcome is described. For that reason, statistical choices beyond the posted endpoint definition and the general planning assumptions should not be inferred. In general, choices such as the analysis population, handling of missing observations, and method for estimating uncertainty can affect interpretation; details for those choices are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists three outcome measures: the primary improvement endpoint and secondary outcomes concerning tolerability and improvement in pain indices. It lists zero analyses. The record does not report whether a hierarchy among outcomes was planned, whether any multiplicity adjustment was intended, or how secondary outcomes would be interpreted alongside the primary endpoint. These details are not reported in the registry record.
In general, examining multiple endpoints can increase the chance that an apparently notable finding arises by chance if each is tested separately. A prespecified outcome hierarchy or adjustment method can address aspects of that issue, but neither should be assumed for this study. Since calculated outcome results are not posted, multiplicity cannot be evaluated from observed analyses.
The primary outcome description states that the planned results were based on 25 evaluable participants, and that this goal was not reached because of low accrual. Enrollment is listed as 2. The registry does not provide participant-level disposition, the number with outcome observations at the planned assessment, reasons for any missing measurements, or an imputation method. These details are not reported in the registry record.
In general, missing outcome data can affect both the estimate and its uncertainty, particularly if the people with missing measurements differ from those assessed. No assumptions about why data may be unavailable or how that would affect the result can be made from this record. It also does not define a separate intention-to-treat or per-protocol analysis population. With no calculated endpoint result and no reported analysis population beyond the planned evaluable-participant basis, neither missing-data handling nor robustness to alternative analysis populations 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 |
|---|---|---|---|
| Sodium Bicarbonate Therapy | 0 | 2 | 0.0% |
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 |
|---|---|
| Sodium Bicarbonate Therapy | 1 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sodium Bicarbonate Therapy |
|---|---|---|
| Nausea | Gastrointestinal disorders | 1 of 2 |
| Vomiting | Gastrointestinal disorders | 1 of 2 |
| Edema: head and neck | Blood and lymphatic system disorders | 1 of 2 |
| Edema: limb | Blood and lymphatic system disorders | 1 of 2 |
| Pain - Head/headache | General disorders | 1 of 2 |
The registry lists the sodium bicarbonate therapy group as having 2 participants at risk for serious adverse events, with 0 affected. For other adverse events, 2 are listed at risk and 1 affected. These are the posted counts; they should not be converted into a claim about a stable event rate, particularly given the small group and the pilot context.
The registry does not list any serious adverse event terms. The number of deaths is not reported in the registry record. The record also does not provide descriptions of the other adverse event, its severity, timing, duration, or a judgment about whether it was related to sodium bicarbonate. A count of participants affected does not itself establish causality.
As a general statistical point, sparse safety counts provide limited information about how often an event might occur in a wider population. Zero serious events recorded in this group does not establish that serious events cannot occur, and one participant with another adverse event does not establish a population frequency. The safety entries are descriptive registry data, not a comparative safety analysis: there is no control group listed, and no inferential analysis is reported.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study asked whether oral sodium bicarbonate, given by dose escalation, could be tolerated and might improve tumor-related pain. It also establishes that the study was single group, had enrollment of 2, and was terminated. The planned primary measure was the percent of patients with greater than 30% improvement in pain intensity by visual assessment scale over 4 weeks per participant.
It does not establish a numerical result for that endpoint. The registry says outcome data were not calculated because accrual was low, and that the goal of 25 evaluable participants was not reached. There is no reported primary percentage, count meeting the threshold, effect estimate, or uncertainty interval. The lack of a calculated result should not be interpreted as evidence of benefit, lack of benefit, or no change.
The study’s design also does not support a controlled comparison. With no comparator group listed, the record cannot estimate how outcomes under sodium bicarbonate differed from outcomes under another condition. In general, a single-group observation cannot rule out other explanations for change over time. The registry lists masking as none, and does not report further information about assessment procedures.
The posted safety entries show 0 serious adverse events among 2 at risk and 1 other adverse event among 2 at risk. Those counts are descriptive and do not establish how often adverse events would occur in a broader group or whether the reported event was caused by the intervention. Deaths are not reported in the registry record.
The appropriate reading is therefore narrow: this is a terminated pilot with very low accrual, uncalculated primary outcome data, and limited descriptive safety information. It does not provide a basis for concluding whether sodium bicarbonate changed pain intensity or medication requirements. The reason for termination and the detailed statistical plan are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a planned endpoint and a result that can be interpreted. The primary endpoint is clearly described as a threshold-based measure of pain improvement, but the registry says it was not calculated because accrual was low. A precisely worded endpoint cannot substitute for observations sufficient to estimate it.
The gap between the intended 25 evaluable participants and the reported enrollment of 2 is central to interpretation. The registry directly states that the evaluable-participant goal was not reached. With no calculated outcome, there is no estimate whose size or precision can be considered, and no inferential result to use in answering the primary question.
The record also highlights why design and analysis must be considered together. The single-group design has no listed control, so it cannot estimate a randomized between-group contrast. The stated planning illustration assumptions involve equal allocation between two arms, while the registered study is single group. Such hypothetical assumptions do not change the actual design or create a comparative result.
Finally, the safety entries demonstrate the limited scope of very small descriptive counts. They report serious and other adverse-event counts, but do not provide a stable estimate for a wider population or a causal comparison. These features make transparent reporting especially important: a reader needs to distinguish what was planned, what was recorded, and what was not calculated. The registry supplies that distinction for the primary endpoint, while leaving the reason for termination and several analysis details unreported.
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 specific to this study constrain interpretation. First, the registry reports enrollment of 2, and the primary outcome data were not calculated because accrual was low. The planned outcome measures were based on 25 evaluable participants, a goal that was not reached. As a result, the key pain-improvement endpoint has no posted estimate or measure of precision.
Second, the study is listed as single group, with no comparator, allocation NA, and masking none. Thus, even if a within-group change had been reported, the registered design would not offer a control-based estimate of the intervention’s effect. The record does not describe whether outcome assessment had any independent or masked component.
Third, the registry gives limited operational detail. The dose levels and escalation schedule are not reported in the registry record. Participant characteristics, individual outcome observations, reasons for missing assessments, and the reason for termination are also not reported. Without those details, readers cannot assess how closely the participants and treatment implementation match other settings or evaluate possible sources of missingness.
Fourth, the registry lists three outcomes but no analyses. The statistical analysis plan, analysis population beyond the planned evaluable-participant basis, and approach to multiplicity and missing data are not reported. It is therefore not possible to assess whether analyses followed a prespecified strategy.
Finally, safety information consists of sparse counts without listed serious-event terms or a reported death count. The registry does not provide enough detail to assess event timing, severity, or relationship to treatment. These limitations do not imply a particular outcome; they define what the posted record can and cannot support.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bicarbonate for Tumor Related Pain — NCT01350583.
- Registry record last updated 2013-08-14; status last verified 2013-06.
- Results first posted 2013-08-08.
- 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 describes a pilot study of oral sodium bicarbonate as an adjunct for tumor-related pain, using a single-group dose-escalation design. It lists phase NA, no masking, enrollment of 2, and status as terminated. The study started in 2010-08 and had a primary completion date of 2012-05.
The primary outcome was the percent of patients with greater than 30% improvement in pain intensity by visual assessment scale over 4 weeks per participant. The record says results were not calculated because of low accrual and that the goal of 25 evaluable participants was not reached. Three outcome measures are listed and zero analyses are listed.
For the sodium bicarbonate therapy group, serious adverse events are listed for 0 of 2 at risk and other adverse events for 1 of 2 at risk. Serious event terms are not listed, and deaths are not reported in the registry record. The reason for termination, detailed statistical plan, and calculated primary outcome are not reported.