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 Safety Study of Botulinum Toxin Type A in Post-Upper Limb Stroke Patients With Reduced Lung Function (NCT00076687), a randomized phase 2 study. The primary outcome is Change From Baseline in Forced Vital Capacity (FVC).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00076687 |
| Phase | Phase 2 |
| Condition | Stroke, Muscle Spasticity, Motor Neuron Disease |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 155 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Allergan |
| Results posted | Yes |
2. Clinical Question
Population
Patients with reduced lung function and focal upper-limb post-stroke spasticity. The primary outcome population is identified as Safety.
Intervention
The registry lists botulinum toxin Type A groups at 240 U and 360 U.
Comparator
The adverse-event groups include placebo. The registry also lists saline as an intervention; further arm descriptions are not reported in the registry record.
Primary question
How did observed forced vital capacity change from baseline to Week 6 in the listed treatment groups?
The study’s stated purpose was to evaluate the safety of botulinum toxin Type A injections in patients with reduced lung function and focal upper-limb post-stroke spasticity. Its primary outcome was change from baseline in forced vital capacity (FVC), measured in liters of air. FVC is the maximum amount of air exhaled after taking the deepest breath possible.
The registry record identifies a primary outcome measure and its timeframe, but it does not report numerical FVC changes, group comparisons, confidence intervals, or p-values. It therefore does not establish how the groups’ FVC measurements changed or how precisely any difference was estimated. The primary outcome population is labelled Safety, but further details about that population are not reported in the registry record.
The listed secondary outcomes are change from baseline in the FEV1/FVC ratio and change from baseline in the Ashworth Scale. Their numerical results and analyses are not reported in the registry record. The question is therefore defined by the study’s stated safety purpose and outcome measures, while the posted information does not permit a quantitative answer about the primary or secondary outcomes.
3. Trial Design
The study is registered as a randomized, quadruple-masked, parallel-group Phase 2 trial. The registry lists enrollment as 155 and status as completed. Its recorded start is 2003-10, and primary completion is recorded as 2009-08. The sponsor is Allergan.
The intervention listings include botulinum toxin Type A twice and saline once. In the adverse-event groups, the named groups are Placebo, Botulinum Toxin Type A 240 U, and Botulinum Toxin Type A 360 U. The arm descriptions themselves are blank, so details beyond those group labels are not reported in the registry record. The registry does not specify in the posted information how allocation was generated or concealed, or what procedures were used to maintain masking.
Randomization is intended to create groups that can be compared without assigning treatment according to participant characteristics. Masking is intended to limit the influence of treatment knowledge on care, assessment, or reporting; the record’s label of quadruple masking does not identify which roles were masked or how masking was implemented. A parallel design means participants are assigned to a group rather than described as crossing between treatments, but details of the treatment schedule are not reported in the registry record.
Although the registry marks the study completed, completion status alone does not supply outcome results. The recorded primary outcome is change from baseline in observed FVC at Baseline and Week 6, for the Safety population. Numerical outcome summaries and a statistical analysis are not reported in the registry record. The registry lists four outcome measures, but its analysis count is 0. That distinction matters: the existence of a registered endpoint does not mean an analyzable comparison or its results are posted.
The record does not report the sample-size justification, planned enrollment by group, detailed eligibility criteria, treatment administration schedule, or a protocol for follow-up. Those design details cannot be inferred from the enrollment figure, intervention labels, or completion status.
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 | Placebo | Botulinum Toxin Type A 240 U | Botulinum Toxin Type A 360 U |
|---|---|---|---|
| STARTED | 48 | 52 | 55 |
| COMPLETED | 42 | 47 | 51 |
| NOT COMPLETED | 6 | 5 | 4 |
Analysis population for the primary outcome, as defined in the registry: Safety
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 | Placebo | Botulinum Toxin Type A 240 U | Botulinum Toxin Type A 360 U | Total |
|---|---|---|---|---|---|
| Participants analysed | 48 | 52 | 55 | 155 | |
| Age Continuous | years | 58 (18 to 80) | 55.5 (21 to 83) | 55.8 (19 to 81) | 56.4 (18 to 83) |
| Sex: Female, Male: Female | Participants | 19 | 13 | 21 | 53 |
| Sex: Female, Male: Male | Participants | 29 | 39 | 34 | 102 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Forced Vital Capacity (FVC), measured over Baseline, Week 6.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Liters of air |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change from baseline in forced vital capacity (FVC), measured in liters of air. FVC is the maximum amount of air a person can exhale after taking the deepest breath possible. The registry describes patients performing 3 to 8 exhalations into a spirometer, with the highest value recorded at Baseline and Week 6. The named population is Safety.
FVC is a continuous measurement of lung volume during a forced exhalation. Comparing a participant’s follow-up measurement with their baseline measurement focuses on change over the study period rather than the follow-up measurement alone. In general, a continuous endpoint retains more information about the measured quantity than a classification that places results into broad categories. That can be useful when the scientific question concerns the magnitude and direction of change.
The registry record identifies the endpoint and measurement schedule, but does not report group means, standard deviations, changes, or a between-group estimate. It also does not report how missing measurements or repeated spirometry readings were handled in the analysis. Consequently, the endpoint’s definition is available, but the observed FVC changes and their precision are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record makes the study question and primary endpoint identifiable: the trial evaluated safety in patients with reduced lung function and focal upper-limb post-stroke spasticity, with change from baseline in FVC as the primary outcome. It records measurements at Baseline and Week 6 and identifies the primary outcome population as Safety. These details describe what was intended to be measured; they do not give the result of that measurement.
No numerical FVC results are reported in the registry record. There are no posted group means, standard deviations, mean changes, between-group estimates, confidence intervals, or p-values for the primary endpoint. The registry’s analysis count is 0. Thus, no estimate of the size or direction of an FVC difference can be interpreted from the posted results. It is also not possible to assess the precision of an estimate or distinguish a small, uncertain difference from a larger, more precisely estimated one.
The listed secondary outcomes are change from baseline in the FEV1/FVC ratio and change from baseline in the Ashworth Scale. Their numerical results and inferential analyses are not reported in the registry record. The record notes four outcome measures overall, but the remaining measures and their results are not detailed in the information available. The presence of registered outcomes should not be mistaken for posted outcome findings.
Adverse-event counts are posted for three named groups. In the Placebo group, 7 of 48 participants are listed with serious adverse events and 18 of 48 with other adverse events. For Botulinum Toxin Type A 240 U, the corresponding counts are 9 of 52 and 15 of 52. For Botulinum Toxin Type A 360 U, they are 6 of 55 and 17 of 55. These are descriptive counts in the registry record. They do not, on their own, establish whether any event was caused by treatment or whether differences between groups are statistically reliable.
The record does not report death counts. That means deaths cannot be characterized from this entry; it does not establish that no deaths occurred. The listed serious-event terms and percentages are also descriptive: the registry does not provide enough information in this record to infer the denominator or a comparative treatment effect for each term.
Overall, the posted information supports a description of the design, endpoint definition, and adverse-event counts. It does not support a numerical conclusion about change in FVC, a quantified comparison of primary outcomes, or a causal interpretation of adverse-event differences. The study’s completed status does not change this limitation in the posted results.
9. Statistical Methods Explained
The registry identifies a continuous primary outcome summarized by a mean and standard deviation. It also describes the outcome as change from baseline. These labels tell readers what kind of measurement was planned and how it was intended to be summarized. They do not provide the numerical summaries or specify every step of the analysis.
What does a mean change describe?
In general, a mean is an average that provides a central summary of observations. For a change-from-baseline outcome, each participant’s follow-up measurement is considered in relation to that participant’s baseline measurement, and group-level summaries can describe the pattern of change. A group mean change alone does not show how much participants’ results varied, whether all participants changed in the same direction, or how different two treatment groups were. The record names mean as the summary parameter, but numerical group means and changes are not reported in the registry record.
What does the standard deviation add?
In general, the standard deviation describes how dispersed individual measurements are around a group mean. It is not the same as a confidence interval: the standard deviation describes variability among measurements, while a confidence interval describes uncertainty around an estimated quantity. Both can help readers understand results, but neither can be interpreted for this endpoint without the relevant numerical summaries. The registry specifies standard deviation as the dispersion measure, but does not report its value.
How would a group comparison be interpreted?
In general, a comparison of changes between randomized groups estimates how much the groups’ average changes differ. A confidence interval gives a range of values compatible with the data and the analysis assumptions, and helps convey estimation precision. A p-value addresses how compatible the observed data are with a specified null hypothesis under the chosen statistical model; it does not measure the size or clinical importance of an effect. These concepts explain how a complete report might be read, not what happened in this trial: the registry record does not report a group contrast, confidence interval, or p-value for FVC.
What do randomization and masking contribute?
In general, random allocation helps make treatment groups comparable at the start with respect to measured and unmeasured factors, though it does not guarantee perfect balance in any one trial. Masking can reduce the risk that knowledge of assigned treatment changes care, outcome assessment, or reporting. The record describes this study as randomized and quadruple-masked. It does not report the allocation procedure, the identities of the masked roles, or the practical masking procedures. Those labels describe the design, but do not substitute for details about implementation.
What does the measurement schedule tell readers?
The primary outcome is defined as observed FVC at Baseline and Week 6, with the highest value recorded from 3 to 8 spirometry exhalations at each stated time. This indicates the intended measurement approach and timing. It does not reveal how many participants contributed measurements at either time, whether all scheduled measurements were obtained, or how the analysis handled missing values. Those details are not reported in the registry record.
How should planning illustrations be understood?
In general, a sample-size calculation estimates how many participants may be needed under specified assumptions to detect a target effect with a chosen significance level and power. A planning illustration based on an observed effect is not the same as the trial’s original design justification, and it is not observed power. The registry’s stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. The record does not report the trial’s own sample-size justification. Without the numerical illustration values and the observed outcome estimate, the assumptions alone do not establish how suitable the original enrollment was for the primary comparison.
The general statistical principles above are useful for interpreting a complete continuous-outcome analysis. For this study, however, the registry record contains no primary-outcome analysis. The choice of statistical model, any adjustment variables, handling of repeated measurements, and any robustness or sensitivity analyses are not reported in the registry record. They should not be presumed from the endpoint’s mean-and-standard-deviation labels or from the trial design.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists change from baseline in FEV1/FVC ratio and change from baseline in Ashworth Scale as secondary outcomes, in addition to the primary FVC outcome. It records four outcome measures overall, but does not detail all of them in the information available. Numerical results and analyses for the listed outcomes are not reported in the registry record.
When several outcomes or comparisons are tested, the chance of at least one apparently notable result can increase. In general, multiplicity procedures may adjust how results are evaluated or reported; whether and how to do so depends on the prespecified analysis plan. The registry record does not report a multiplicity strategy, a hierarchy among secondary outcomes, or whether any adjustment was applied. It also does not report interim analyses or a statistical analysis plan. No such procedures should be inferred from the outcome list.
The primary outcome population is labelled Safety, but the analysis population details are not reported in the registry record. The record does not specify whether analyses used all randomized participants, participants who received treatment, or another defined set. It also does not report missing-data handling, reasons for missingness, imputation methods, or sensitivity analyses addressing missing outcomes.
These omissions affect how fully the results could be assessed even if numerical estimates were available. In this record, no outcome analyses are posted, so multiplicity, missing-data methods, and analysis-population definitions cannot be checked against reported findings.
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 |
|---|---|---|---|
| Placebo | 7 | 48 | 14.6% |
| Botulinum Toxin Type A 240 U | 9 | 52 | 17.3% |
| Botulinum Toxin Type A 360 U | 6 | 55 | 10.9% |
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 |
|---|---|
| Placebo | 18 of 48 |
| Botulinum Toxin Type A 240 U | 15 of 52 |
| Botulinum Toxin Type A 360 U | 17 of 55 |
Most frequent serious adverse event terms
| Event term | Organ system | Placebo | Botulinum Toxin Type A 240 U | Botulinum Toxin Type A 360 U |
|---|---|---|---|---|
| Convulsion | Nervous system disorders | 1 of 48 | 0 of 52 | 2 of 55 |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | 1 of 48 | 1 of 52 | 0 of 55 |
| Hypotension | Vascular disorders | 1 of 48 | 1 of 52 | 0 of 55 |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 48 | 0 of 52 | 1 of 55 |
| Abscess | Infections and infestations | 0 of 48 | 1 of 52 | 0 of 55 |
| Acute endocarditis | Infections and infestations | 0 of 48 | 0 of 52 | 1 of 55 |
| Appendicitis | Infections and infestations | 0 of 48 | 0 of 52 | 1 of 55 |
| Bacteraemia | Infections and infestations | 0 of 48 | 1 of 52 | 0 of 55 |
| Cellulitis | Infections and infestations | 0 of 48 | 1 of 52 | 0 of 55 |
| Gastroenteritis | Infections and infestations | 0 of 48 | 0 of 52 | 1 of 55 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Placebo | Botulinum Toxin Type A 240 U | Botulinum Toxin Type A 360 U |
|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | 3 of 48 | 4 of 52 | 6 of 55 |
| Diarrhoea | Gastrointestinal disorders | 4 of 48 | 1 of 52 | 2 of 55 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 2 of 48 | 3 of 52 | 2 of 55 |
| Headache | Nervous system disorders | 3 of 48 | 0 of 52 | 3 of 55 |
| Vomiting | Gastrointestinal disorders | 3 of 48 | 1 of 52 | 1 of 55 |
| Cough | Respiratory, thoracic and mediastinal disorders | 2 of 48 | 3 of 52 | 0 of 55 |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 1 of 48 | 0 of 52 | 3 of 55 |
| Muscle spasticity | Nervous system disorders | 0 of 48 | 3 of 52 | 0 of 55 |
Serious events with the highest arm-level rate: convulsion (3.6%), splenic abscess (2.1%), wound infection (2.1%), cerebral infarction (2.1%), syncope (2.1%).
The registry reports serious adverse-event counts and other adverse-event counts for Placebo, Botulinum Toxin Type A 240 U, and Botulinum Toxin Type A 360 U. Serious adverse events are listed for 7 of 48 participants in Placebo, 9 of 52 in the 240 U group, and 6 of 55 in the 360 U group. Other adverse events are listed for 18 of 48, 15 of 52, and 17 of 55, respectively.
These figures are descriptive counts, not adjusted comparisons. They do not show whether a particular event was related to treatment, how many events each participant experienced, or whether differences between groups are statistically reliable. The registry record does not report the statistical methods used for these safety data or provide confidence intervals for the group comparisons.
The listed top serious-event terms include Convulsion at 3.6%, Splenic abscess at 2.1%, Wound infection at 2.1%, Cerebral infarction at 2.1%, Syncope at 2.1%, and Angina Pectoris at 2.1%. These are the percentages attached to the terms in the registry record; the denominators and comparative estimates for the terms are not reported there. Death counts are not reported in the registry record, so the record does not establish whether deaths occurred.
12. What the Result Does, and Does Not, Mean
The record describes a randomized, quadruple-masked, parallel-group Phase 2 study intended to evaluate the safety of botulinum toxin Type A injections in patients with reduced lung function and focal upper-limb post-stroke spasticity. It defines change from baseline in FVC as the primary outcome, measured at Baseline and Week 6, and identifies the primary outcome population as Safety. These registry entries establish the trial’s stated question and measurement framework.
The posted information does not include numerical FVC results or an analysis of the primary endpoint. It therefore does not show the direction or magnitude of FVC change in any group, the difference between groups, or the precision of such a difference. The registry’s completed status is not a substitute for outcome data. Nor does a registered endpoint, by itself, establish that its results were analyzed or posted.
The adverse-event figures provide counts by named group, along with selected serious-event terms and percentages. They describe what the registry lists, but do not prove that an event was caused by a treatment or that apparent differences between groups are more than chance variation. Without an analysis, uncertainty estimates, and further event details, the counts cannot be used to make a quantified comparative safety conclusion. Death counts are not reported in the registry record.
More generally, an absence of posted results is not evidence that the groups were the same, and it is not evidence that an intervention had no effect. It means the record does not provide the estimates needed to evaluate those possibilities. The study’s stated safety purpose and its endpoint definitions should therefore be kept separate from claims about what the data showed. No treatment-effect conclusion can be drawn from the posted primary-outcome information.
13. Why This Trial Matters Statistically
This trial is statistically notable because its question links a treatment safety evaluation to a continuous lung-function outcome in a population described as having reduced lung function and focal upper-limb post-stroke spasticity. The primary endpoint is defined as change from baseline in FVC, with measurement at Baseline and Week 6. That definition makes clear what quantity was intended to be assessed, while the absence of numerical results leaves its observed behavior unknown.
The record also illustrates why registration details and outcome analyses serve different purposes. Registration identifies design features, outcome definitions, and timing. To evaluate a result, readers also need estimates, measures of uncertainty, and an account of the analysis population and methods. Here, the registry reports randomization, quadruple masking, and a parallel-group design, but the primary analysis is not reported. The study is marked completed, yet its posted record does not permit a statistical assessment of the primary endpoint.
Safety results also require careful interpretation. The registry gives counts for serious and other adverse events across named groups, and lists selected serious-event terms. Such counts are useful descriptive information, but without comparative estimates and uncertainty they do not establish whether group differences are reliable or attributable to treatment. This distinction is important in any trial: observed counts and causal interpretation are not interchangeable.
Finally, the record lists multiple outcomes while leaving multiplicity procedures, missing-data handling, and detailed analysis-population definitions unreported. These are not merely technical footnotes. They help determine what an estimate represents and how confidently results can be interpreted. The statistical lesson is not a conclusion about the treatments, but the importance of complete outcome reporting alongside clear endpoint and design definitions.
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 most consequential limitation of the posted record is the absence of numerical primary-outcome results. Although FVC and its measurement schedule are described, there are no group summaries, comparative estimates, confidence intervals, or p-values. The analysis count is 0. The registry therefore does not support evaluation of the size, direction, or precision of an FVC effect.
The primary outcome population is labelled Safety, but its operational definition is not reported in the registry record. Detailed arm descriptions are blank, and the record does not explain how the listed interventions map onto every study arm beyond the group labels in the adverse-event data. Allocation procedures and the roles covered by quadruple masking are also not described in the posted information.
The registry lists secondary outcomes and four outcome measures overall, but does not report numerical results or analyses for them. It does not report a multiplicity approach, missing-data methods, analysis-set definitions, or sensitivity analyses. Death counts are not reported. The top serious-event terms include percentages, but denominators and comparative estimates for those terms are not reported in the registry record.
Further study details that could inform interpretation, including detailed eligibility criteria, treatment schedules, and the original sample-size justification, are not reported in the registry record. The stated planning-illustration assumptions do not replace a trial-specific design justification. These limitations restrict what can be concluded from the posted entry; they do not establish that the study itself lacked these procedures or data.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety Study of Botulinum Toxin Type A in Post-Upper Limb Stroke Patients With Reduced Lung Function — NCT00076687.
- Registry record last updated 2011-10-03; status last verified 2011-08.
- Results first posted 2011-10-03.
- 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 identifies a completed randomized, quadruple-masked, parallel-group Phase 2 study sponsored by Allergan. Enrollment is recorded as 155; the recorded start is 2003-10 and primary completion is 2009-08. The study’s stated purpose was to evaluate the safety of botulinum toxin Type A injections in patients with reduced lung function and focal upper-limb post-stroke spasticity.
The primary outcome is change from baseline in observed FVC, measured in liters of air at Baseline and Week 6 in the Safety population. Listed secondary outcomes are change from baseline in FEV1/FVC ratio and change from baseline in Ashworth Scale. The registry record does not report numerical primary or secondary outcome results or an analysis. Adverse-event counts are posted for Placebo and botulinum toxin Type A groups at 240 U and 360 U; death counts are not reported in the registry record.