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 Botulism Toxin Injection as a Treatment for Arthritis of the Basal Thumb Joint (NCT01045694), a randomized phase 4 study. The primary outcome is Pain.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01045694 |
| Phase | Phase 4 |
| Condition | Arthritis Multiple Joint |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Missouri-Columbia |
| Results posted | Yes |
2. Clinical Question
Population
Participants with basal thumb joint arthritis. The registry record reports that 8 participants were randomized, and that 2 withdrew voluntarily. Further eligibility criteria and participant characteristics are not reported in the registry record.
Intervention
Botulinum toxin type A injection, investigated as a treatment for basal thumb joint arthritis.
Comparator
The trial included triamcinolone acetonide steroid injection and lidocaine injection. The record describes steroid as an active comparator and lidocaine as a baseline comparator. How many participants were assigned to each group is not reported in the registry record.
Primary question
The intended primary outcome was pain, assessed at twenty-four hours, ten days, twelve weeks, six months, and one year. The study was terminated due to lack of funds; pain data were not collected or analyzed. The record therefore does not establish how pain outcomes compared among the injection groups.
The clinical question is whether pain outcomes following botulinum toxin type A injection differ from those following steroid or lidocaine injection in people with basal thumb joint arthritis. The registry describes a randomized, double-blind, placebo-controlled clinical trial, while listing the three injections as interventions. It also records quadruple masking. These descriptions indicate an intention to reduce bias, but do not supply the allocation details or explain how masking was implemented.
The registry’s summary explains the rationale for examining botulinum toxin alongside steroid and lidocaine. It describes steroid injection as standard care while noting that its efficacy was considered uncertain in the trial rationale. Those statements explain the investigators’ motivation; they are not findings from this trial. Since the trial collected no pain data, the clinical comparison remains unanswered by its results.
3. Trial Design
The registry identifies this as a randomized, parallel-group, phase 4 trial sponsored by the University of Missouri-Columbia. Its official title describes a randomized, double-blind, placebo-controlled clinical trial comparing botulinum toxin with steroid injection for basal joint arthritis of the thumb. The record lists botulinum toxin type A, triamcinolone acetonide, and lidocaine as drug interventions. It describes the steroid arm as an active comparator and the lidocaine arm as a baseline comparator.
The masking designation is quadruple. The record does not explain which participants or study personnel were masked, how the injections were made indistinguishable, or whether masking was assessed. It also does not report the randomization sequence, allocation concealment, planned group sizes, or whether allocation was equal. Although the trial is described as parallel, the number randomized to each treatment group is not reported.
Enrollment was 8. The record states that 2 participants withdrew voluntarily, that the study was terminated due to lack of funds, and that unblinding did not occur. The start date is 2011-03 and the primary completion date is 2012-08; the overall status is terminated. The registry does not explain whether the listed primary completion date reflects the planned or actual timing of outcome assessment.
Range of motion and strength are listed as secondary outcomes. The record does not report results for these outcomes. Most importantly, it states that data were not collected or analyzed. Thus, the trial design defines intended treatment comparisons and outcomes, but the registry record contains no analyzed treatment-effect estimates.
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.
Before assignment: 8 participants were randomized; 2 withdrew voluntarily. Study was terminated due to lack of funds, and unblinding did not occur - it is not known how many participants were placed in each group.
Overall Study
| Stage | Arthritis Treatment |
|---|---|
| STARTED | 8 |
| COMPLETED | 6 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Arthritis Treatment |
|---|---|
| Withdrawal by Subject | 2 |
Analysis population for the primary outcome, as defined in the registry: 8 participants were randomized; 2 withdrew voluntarily. Study was terminated due to lack of funds, and unblinding did not occur - it is not known how many participants were placed in each group. Data was not collected or analyzed.
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: 8 participants were randomized; 2 withdrew voluntarily. Study was terminated due to lack of funds, and unblinding did not occur - it is not known how many participants were placed in each group.
| Characteristic | Unit | Arthritis Treatment |
|---|---|---|
| Participants analysed | 8 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 8 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 0 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: United States | participants | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Pain, measured over twenty-four hours, ten days, twelve weeks, six months, and one year.
| 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 is listed as pain. Pain is an outcome that can capture how participants experience their condition, and in general it may be assessed using a rating scale, a questionnaire, or another defined measure. The registry record does not identify the instrument, scoring method, unit, or threshold for interpreting a change in pain. Those measurement details are therefore not reported in the registry record.
The intended assessment times were twenty-four hours, ten days, twelve weeks, six months, and one year. Repeated assessments can describe how an outcome changes over follow-up, but they also require a clear analysis plan specifying how the measurements will be compared and handled together. The record does not report such a plan or explain whether any one assessment time was intended to drive the primary comparison.
The registry states that 8 participants were randomized, 2 withdrew voluntarily, and the study was terminated due to lack of funds. It further states that data were not collected or analyzed. Consequently, no pain scores, changes from baseline, between-group comparisons, or uncertainty estimates are reported. The endpoint was planned, but its results cannot be interpreted from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that this trial has no analyzed pain outcome data in the registry record. Although pain was designated as the primary outcome and assessment times were listed, the record explicitly says that data were not collected or analyzed. There is therefore no reported treatment effect for botulinum toxin, steroid, or lidocaine, and no statistical comparison among those interventions.
The registry reports that 8 participants were randomized and 2 withdrew voluntarily. It also states that the study was terminated due to lack of funds and that unblinding did not occur. These details describe the study’s conduct and explain why the intended comparison did not yield reported outcome analyses. They do not show whether the treatments differed, nor do they establish that the treatments were equivalent. In particular, an absence of analyzed results is not evidence of no effect.
Randomization is a design feature intended to support fair comparisons by reducing systematic differences between groups. Its benefits depend on having outcome information and a suitable analysis. Here, the registry does not report group sizes or the number assigned to each intervention, and no outcome data were analyzed. The randomized design alone cannot supply a treatment estimate.
No confidence interval, p-value, or other estimate of statistical uncertainty is reported. In general, a confidence interval conveys a range of effect values compatible with the data and analysis, while a p-value addresses how compatible the data are with a specified null hypothesis under an assumed model. Neither can be interpreted when the trial reports no outcome analysis. No claim about precision or statistical significance is supported.
The record’s adverse-event figures report zero serious adverse events, zero other adverse events, and zero deaths among 8 participants at risk in the aggregate group. These descriptive counts do not replace efficacy results. The record does not give treatment-specific safety counts, and the small overall enrollment and lack of group allocation details limit what can be concluded from the aggregate figures.
Overall, the registry documents a planned randomized comparison that ended without collected or analyzed pain data. The trial record therefore neither supports nor rules out differences among the injections. Its contribution is chiefly to document the design, early termination, and absence of outcome analyses, rather than to estimate comparative effects.
9. Statistical Methods Explained
The registry describes a randomized, parallel-group trial with quadruple masking. These are design characteristics, not evidence that a treatment has a particular effect. Because the record reports no collected or analyzed outcome data, the statistical methods that would have been used to estimate treatment differences are not reported in the registry record.
What does randomization contribute?
In general, randomization assigns participants to treatment groups using a chance-based process. Its purpose is to reduce systematic differences between groups at the start of a study, including differences that investigators may not anticipate. Randomization does not guarantee that groups will be identical, especially in a small study. It also cannot prevent bias arising after assignment, such as incomplete outcome collection or differences in how outcomes are measured.
The record does not report the sequence-generation method, allocation concealment, or group sizes. It is therefore not possible to assess those aspects of the randomization process. The stated total enrollment of 8 does not reveal how many participants were assigned to botulinum toxin, steroid, or lidocaine.
What does masking mean?
Masking is intended to limit the influence of treatment knowledge on participants’ reports, care, or outcome assessment. The registry labels the study quadruple masked and its official title calls it double blind. The record does not clarify how these terms were operationalized, who was masked, or whether masking remained intact. It also says unblinding did not occur. That statement does not supply an assessment of masking success; it reports that unblinding was not carried out.
How would a treatment comparison be estimated?
In general, a trial analysis compares outcomes between the assigned treatment groups using a method appropriate to the outcome and design. For a pain outcome, the suitable method would depend on the measurement instrument, the timing of assessments, the distribution of the measurements, and the prespecified question. The registry record does not report the pain instrument, a statistical model, an effect measure, or an adjustment strategy. It also does not report an analysis population, such as whether the intended analysis would follow randomized assignment regardless of treatment received.
The endpoint was scheduled at multiple follow-up times. In general, repeated measurements can be analyzed by comparing a particular time point, by modeling the course over time, or by using another prespecified summary. These approaches answer different questions and rely on different assumptions. The registry does not report which approach was intended. Since no pain data were collected or analyzed, none of these possible approaches can be attributed to this trial.
Why do uncertainty estimates matter?
In general, an effect estimate describes the observed contrast between groups, while a confidence interval communicates its statistical uncertainty under the chosen model. A narrow interval indicates greater precision than a wide interval, but interpretation also depends on the measure and clinical context. A p-value is not an effect size and does not describe the probability that a treatment works. These concepts require actual outcome data and a defined analysis. No effect estimate, confidence interval, or p-value is reported for the trial.
What is over-dispersion?
In general, over-dispersion occurs when observed variability is greater than a statistical model expects. If it is ignored, uncertainty estimates may be too small and tests may be misleading. The relevance of this issue depends on the outcome type and analysis model. The registry does not report a model or analyzed outcome data, so there is no basis for assessing over-dispersion in this trial.
Can a sample-size calculation recover the missing results?
No. A planning calculation can illustrate the number of participants needed under specified assumptions, but it cannot substitute for observed outcomes or establish what this trial found. The registry record does not report the trial’s sample-size justification or statistical plan. With no collected or analyzed primary outcome data, there is no observed effect on which to base a trial-specific interpretation.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists pain as the primary outcome and range of motion and strength as secondary outcomes. Pain was intended to be assessed at twenty-four hours, ten days, twelve weeks, six months, and one year. Considering several outcomes or assessment times can raise a multiplicity issue: in general, making many separate statistical comparisons can increase the chance of finding an apparently notable result by chance. A prespecified strategy may define a primary comparison or otherwise account for multiple testing. Such a strategy is not reported in the registry record.
The record states that no data were collected or analyzed. Consequently, no outcome-specific analysis, missing-data adjustment, or multiplicity correction is reported. The record does not describe a planned method for handling missing observations, define analysis populations, or state how withdrawals would have been included in an analysis. The reasons for the two voluntary withdrawals beyond that description, their assigned groups, and the timing of withdrawal are not reported in the registry record.
In general, missing outcomes can affect both the amount of information available and the validity of a comparison, particularly if missingness differs among groups or relates to outcomes. Here, this is not a case where a reported analysis has imputed or otherwise handled missing outcome measurements: the registry explicitly says the data were not collected or analyzed. The impact of missingness on any treatment estimate cannot be quantified because no estimate exists.
The record also says unblinding did not occur. It does not report whether a separate planned analysis population or interim analysis was specified. No assumptions about these matters can be made from the trial’s design labels alone.
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 |
|---|---|---|---|
| Arthritis Treatment | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Arthritis Treatment | 0 of 8 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Arthritis Treatment | 0 of 8 |
The registry’s adverse-event record reports 8 participants at risk in the aggregate arthritis-treatment group, with 0 serious adverse events, 0 other adverse events, and 0 deaths. These are descriptive counts as reported in the registry. The record does not provide treatment-specific counts or identify how many participants were in each intervention group, so the figures cannot be used to compare safety among botulinum toxin, steroid, and lidocaine.
In general, an event count of zero does not establish that an event is impossible or that a treatment is free of risk. The information conveyed depends on the number of participants observed, the duration and completeness of follow-up, and how events were defined and collected. The registry does not report those details for the adverse-event figures. It also does not list serious adverse-event terms.
The study was terminated due to lack of funds, and the record states that 2 participants withdrew voluntarily. The adverse-event figures should therefore be read as the registry’s aggregate report, not as a complete comparative safety analysis. They do not address efficacy, and they do not establish that the interventions have equivalent safety profiles.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a randomized, parallel-group trial was intended to compare injections for basal thumb joint arthritis, with pain as the primary outcome. It lists botulinum toxin type A, triamcinolone acetonide, and lidocaine, and identifies range of motion and strength as secondary outcomes. It also documents enrollment of 8 participants, 2 voluntary withdrawals, termination due to lack of funds, and no unblinding.
What the record does not establish is whether pain differed between treatment groups. It states that data were not collected or analyzed. There are no reported group-specific outcomes, effect estimates, confidence intervals, or significance tests. As a result, the trial cannot support a conclusion that one injection was better, worse, or equivalent to another. The lack of a reported difference is not the same as evidence that no difference exists.
In general, an under-informative or incomplete trial can leave a clinical question unresolved even when its design was randomized and masked. A randomized design describes how assignment was intended to work; it does not generate a result in the absence of outcome observations. Likewise, an intervention’s rationale in the registry summary is not a trial finding.
The aggregate adverse-event counts are limited descriptive information, not a comparative safety result. Because the record does not report treatment-specific exposure or event counts, the figures cannot distinguish safety profiles across the interventions. The listed secondary outcomes also have no reported results.
The appropriate interpretation is therefore narrow: the registry documents the trial’s planned comparisons and its termination before outcome data were analyzed. The comparative effects of the injections remain unknown from this trial record. That uncertainty should not be converted into a claim of benefit, lack of benefit, or equivalence.
13. Why This Trial Matters Statistically
This trial is statistically notable because its design and its results have to be kept distinct. Randomization, masking, parallel groups, and defined outcomes describe a structure for making comparisons. But statistical inference requires outcome data, and the registry states that data were not collected or analyzed. The design cannot answer the treatment question by itself.
The record also illustrates how trial completion affects the information available to readers. It reports enrollment of 8 participants, 2 voluntary withdrawals, and termination due to lack of funds. It does not report group sizes or any pain analysis. Thus, even basic features needed to judge the planned comparison—such as the number assigned to each intervention—are unavailable in the registry record.
The listed pain assessments span multiple follow-up times, with range of motion and strength also named as secondary outcomes. In general, multiple outcomes and assessment times make a clear analysis plan important. Without such a plan, readers cannot know which comparisons were intended to be primary or how statistical uncertainty would have been handled. The plan is not reported, and no outcome results are available to interpret.
Finally, the record’s aggregate adverse-event counts should not be mistaken for a comparative safety analysis. They report zero events in a small overall enrolled group but do not break the counts down by treatment. The statistical lesson is not that the interventions are alike, but that the available record is insufficient to estimate how they compare.
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 main limitation is that the registry states that data were not collected or analyzed. No primary pain result is available, and no results are reported for range of motion or strength. This prevents estimation of treatment effects and their precision, regardless of the randomized design.
The study was terminated due to lack of funds. The record reports 8 randomized participants and 2 voluntary withdrawals, but does not identify their treatment assignments or the timing of withdrawal. It also does not report how many participants were placed in each group. The extent to which any intended follow-up was completed is not reported in the registry record.
Measurement details for pain are absent: the record gives no instrument, scale, unit, or definition of the primary comparison. Although assessment times are listed, the intended approach for analyzing repeated measurements is not reported. The registry also does not report a statistical analysis plan, sample-size justification, analysis populations, missing-data methods, or multiplicity strategy.
The trial is described as quadruple masked and as double blind in its official title, but the record does not explain who was masked or how masking was maintained. Unblinding did not occur, according to the primary outcome description. The registry does not provide treatment-specific adverse-event information, and its aggregate safety counts cannot support comparisons between groups. Together, these limitations mean the record documents a planned study but does not provide evidence about comparative efficacy or safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Botulism Toxin Injection as a Treatment for Arthritis of the Basal Thumb Joint — NCT01045694.
- Registry record last updated 2017-10-06; status last verified 2017-09.
- Results first posted 2017-07-13.
- 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 4, randomized, parallel-group trial sponsored by the University of Missouri-Columbia. It planned to compare botulinum toxin type A, triamcinolone acetonide steroid, and lidocaine injections for basal thumb joint arthritis. The official title describes a double-blind, placebo-controlled trial; the masking designation is quadruple.
Enrollment was 8. The record reports that 2 participants withdrew voluntarily, the study was terminated due to lack of funds, and unblinding did not occur. Pain was the primary outcome, with assessments intended at twenty-four hours, ten days, twelve weeks, six months, and one year. Range of motion and strength were secondary outcomes.
The primary outcome description states that data were not collected or analyzed. No treatment-effect estimates or secondary outcome results are reported. Aggregate adverse-event figures list 0 serious adverse events, 0 other adverse events, and 0 deaths among 8 at risk; treatment-specific counts are not reported.