Effectiveness of Chinese Herbal Therapy for Asthma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Effectiveness of Chinese Herbal Therapy for Asthma (NCT00712296), a randomized phase 1, phase 2 study. The primary outcome is Glucose, Urea and Creatinine Phase I.

2. Clinical Question

3. Trial Design

This was a randomized, quadruple-masked, parallel-group study listed as Phase I and Phase II. The registry record gives an enrollment of 66 and identifies asthma as the condition. The sponsor is Icahn School of Medicine at Mount Sinai. The study started in 2008-08, had a primary completion date of 2009-09-29, and is listed with a terminated status. The reason for termination is not reported in the registry record.

The listed interventions are ASHMI 4, ASHMI 12 and placebo. For the Phase II arm descriptions, ASHMI 4 and ASHMI 12 are each described as capsules taken twice a day; placebo is described as 6 capsules twice a day. The adverse-event record also distinguishes Phase I dose groups, including active ASHMI groups and placebo groups, as well as three Phase II groups. Those group labels and counts are useful for reading the safety information, but they do not establish a complete account of the randomization process or allocation by phase.

Randomization and masking describe aspects of the design, not the results. The registry record does not report the method used to generate the random sequence, conceal allocation, or maintain masking. It also does not report the intended sample size by arm, a sample-size justification, or a detailed protocol for handling deviations from assigned treatment. The overall enrollment alone does not show how many participants were included in each analysis.

The registry lists 10 outcome measures and no posted analyses. Its named primary outcome is laboratory data for glucose, urea and creatinine in Phase I, summarized as means and standard deviations one week after receiving active drug or placebo. The study purpose refers to efficacy, but the available primary outcome description is a laboratory summary rather than a reported asthma efficacy comparison.

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: Phase I: Time frame 1 week; Phase II: Time frame 28 weeks. Participants from Phase I were invited to participate in Phase II and were re-randomized to all three arms of Phase II.

Phase I ( 1 Week)

Phase II (28 Weeks)

Analysis population for the primary outcome, as defined in the registry: For the Phase I study we used descriptive statistics to report the frequency of adverse events (AEs) among patients treated with ASHMI or placebo. Analysis was per intention to treat.

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: ASHMI/Placebo was discontinued on 9/29/10. 20 participants were re-randomized into the Phase II.

6. Primary Endpoint

The registry lists the primary outcome as Glucose, Urea and Creatinine Phase I, measured over 1 week after receiving active drug or placebo.

The named primary outcome is “Glucose, Urea and Creatinine Phase I.” It concerns laboratory measurements of glucose, urea and creatinine, each expressed in mg/dL. The stated timeframe is one week after receiving active drug or placebo. The outcome is described for subjects enrolled in Phase I and uses the mean as its summary measure, with standard deviation as the stated measure of dispersion.

These laboratory tests describe measured values in blood. As general statistical context, a mean gives a summary of the central level of a continuous measurement, while a standard deviation describes how much individual measurements vary around that mean. Together they can characterize the observed laboratory values, but they do not by themselves establish whether groups differ, whether a difference is precise, or whether an observed change is caused by treatment.

Laboratory measurements can be included to describe safety or physiological status during a study. The registry description identifies this outcome as a summary of Phase I laboratory data, but it does not state a specific rationale for choosing these measures as the primary endpoint. The numerical means and standard deviations are not reported in the registry record. Nor does the record give a treatment contrast, confidence interval, or test result for these measurements. Consequently, the endpoint definition is available, but its observed values and any comparative interpretation cannot be recovered from the posted record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The study is described as randomized, quadruple-masked and parallel-group. In general, randomization assigns participants to study groups using a chance-based process, which can reduce systematic differences between groups at the point of assignment. Masking is intended to limit the influence of treatment knowledge on participant experience, care, assessment or analysis. “Quadruple” is the registry’s masking designation; the specific people covered by masking and the procedures used are not reported in the registry record. A parallel design means participants are assigned to a group for comparison rather than being described as crossing through multiple treatments.

What do the mean and standard deviation describe?

The primary outcome is continuous: glucose, urea and creatinine are reported in mg/dL, with a mean and standard deviation specified. In general, a mean condenses a set of observations into a central summary. The standard deviation describes dispersion of observations around that mean. A small standard deviation indicates that observations are more concentrated around the mean than when the standard deviation is larger. These summaries do not show the distribution’s full shape, and they do not establish a treatment effect without group-specific values and an appropriate comparison.

The record does not include the actual laboratory means or standard deviations. It also does not state whether values were adjusted for baseline measurements, how repeated measurements were handled, or whether any transformations were applied. Those analysis details are not reported in the registry record. The stated measurement time is one week after active drug or placebo, but the record does not provide further details about laboratory collection or assessment procedures.

What can a comparison estimate?

In general, a treatment comparison asks how an outcome differs between groups assigned to different interventions. A point estimate describes the observed contrast, while a confidence interval describes statistical uncertainty around that estimate under the analysis assumptions. A p-value, when used, describes how compatible the data are with a specified null hypothesis under a statistical model; it does not measure the probability that a treatment works. No point estimate, confidence interval or p-value for the primary laboratory outcome is reported in the registry record, so no such comparison can be interpreted for this study from the posted information.

Randomization can support a causal interpretation when the analysis preserves the randomized comparison and other relevant assumptions are met. However, the registry record does not give a complete statistical analysis plan, the allocation counts by group for the primary analysis, or details about adherence and follow-up. The outcome description says the Phase I adverse-event analysis was per intention to treat and used descriptive statistics. That statement applies to the described Phase I adverse-event reporting; it does not establish how every outcome was analyzed.

How should planning calculations be read?

General sample-size planning uses assumptions about the effect to detect, the significance level, allocation, and the statistical method. The record’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect set as a stated fraction of the observed effect. For time-to-event tables, it describes total events using the Schoenfeld method; other planning values are participants per arm.

Such calculations are planning illustrations based on the observed effect, not observed power. In general, a planning illustration asks how many participants might be needed under stated assumptions to detect a chosen effect. It does not show the probability that the completed trial detected an effect, and it cannot replace an analysis of the observed outcomes. The registry record does not report the trial’s own sample-size justification. No numeric planning result should be interpreted as an observed trial result.

Why does over-dispersion matter in general?

For count outcomes, over-dispersion means observed variability exceeds what a simple model assumes. In general, ignoring it can make uncertainty estimates too small and statistical evidence appear stronger than warranted. This concept is not a reported feature of the continuous laboratory endpoint, and the registry record does not say that an over-dispersion model was used. The broader point is that model choice and assumptions matter when interpreting any estimate; the analysis details for the primary outcome are not reported in the registry record.

The record lists 10 outcome measures and no posted analyses. It therefore does not permit a reader to verify which statistical tests were used, how any model assumptions were checked, or whether estimates were adjusted. The available description supports explaining the planned form of the primary endpoint and the general meaning of its summaries, but not reconstructing an unreported analysis.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 10 outcome measures. When a study evaluates multiple outcomes or multiple comparisons, general statistical practice recognizes a possibility of chance findings across the collection of tests. A prespecified approach can address that issue, for example by identifying a primary comparison or adjusting interpretation across tests. The registry record does not report a multiplicity strategy, a hierarchy of outcomes, or a statistical analysis plan specifying how multiple comparisons were handled.

The primary outcome description itself includes three laboratory measures: glucose, urea and creatinine. The record notes that this primary outcome has several categories or time points and was not reduced to one comparison. This means there is no single comparative result in the registry record to interpret as the primary treatment contrast. It does not establish whether the study team conducted analyses that were not posted.

Missing-data methods are not reported in the registry record. There is no account of the number of missing observations, exclusions, withdrawals, or how missing outcome measurements were handled. The total enrollment of 66 does not show the analysis population for any specific endpoint. The Phase I adverse-event description states that analysis was per intention to treat, but the record does not provide enough detail to determine how that population was defined or whether the same approach applied to other outcomes.

These omissions matter because the results can depend on which participants and observations enter an analysis and how multiple outcomes are interpreted. Without the analysis plan, missing-data information and posted outcome analyses, it is not possible to evaluate those choices for this trial. The reason for termination is also not reported in the registry record, so its relationship, if any, to outcome availability cannot be assessed.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event record separates serious events from other events and lists the number at risk and the number affected in each group. Across all listed Phase I and Phase II groups, the serious-event affected count is 0. The group sizes at risk vary: the Phase I groups list 2 or 4 participants, while the Phase II groups list 15 or 16. The counts of participants affected by other events are also shown for each group. These are descriptive counts and do not constitute a formal comparison between treatments.

Zero serious events in the posted groups means none were recorded among the participants counted at risk in those groups. It does not establish that the risk of serious events is zero, particularly when the listed groups are small. The registry record does not report confidence intervals or a statistical comparison for these safety counts. It also does not provide event terms in the serious-event list.

The deaths field is blank rather than a numeric count for the listed groups. Deaths are therefore not reported in the registry record; a blank field should not be read as a recorded zero. The adverse-event group labels distinguish Phase I dose cohorts and Phase II groups. They should be retained as labeled rather than combined into a new total or treated as directly interchangeable groups.

12. What the Result Does, and Does Not, Mean

The result information in the registry record is limited. It identifies the study design, enrollment, intervention labels, primary endpoint definition and adverse-event counts. It does not report numerical results for the primary laboratory endpoint or a comparative analysis of ASHMI and placebo. The study’s stated purpose is efficacy in adult asthmatics, but the information posted does not quantify an asthma outcome or estimate an efficacy effect.

As a general principle, a study design and an outcome definition describe how a question was framed; they are not results. Likewise, a registry status of terminated is a status description, not an explanation for why the study ended. The reason is not reported in the registry record. The listed enrollment of 66 does not establish how many participants completed the study, had usable measurements, or entered a particular analysis.

The safety counts show no serious adverse events recorded among participants at risk in the listed groups, alongside reported counts for other events. Those observations describe what is posted, but they cannot establish that serious events are impossible or provide a precise comparison of safety. The death field is not numerically reported. Interpretation should keep these distinctions in view rather than treating missing information as a favorable or unfavorable result.

Planning illustrations, where presented, are not evidence that the completed trial had a particular probability of finding a result. They use assumptions to describe potential sample-size requirements, and they do not substitute for observed estimates, uncertainty intervals or a reported analysis. The record does not provide the trial’s own sample-size justification.

Accordingly, the registry information can be used to understand the broad structure and reporting limits of the study. It cannot support a numerical conclusion about the laboratory outcome, the efficacy question, or a comparative safety effect. Those conclusions would require results and analysis details that are not reported in the registry record.

13. Why This Trial Matters Statistically

This study illustrates why the distinction between a research question and a reported endpoint matters. The stated purpose is to determine efficacy in adult asthmatics, whereas the named primary outcome is a Phase I summary of glucose, urea and creatinine. A record can describe its intended purpose and list outcomes without presenting the results needed to answer the clinical question. Readers need to check what was actually measured and what was actually reported before interpreting a study as evidence for a treatment effect.

It also illustrates the limits of summary statistics without a comparison. A mean and standard deviation are useful ways to describe continuous laboratory measurements, but they do not show whether groups differed, how large a difference was, or how uncertain an estimate might be. Without numerical results and a comparative analysis, even a clearly defined endpoint cannot answer a treatment-effect question.

The safety record offers another statistical lesson: observed zero counts are not equivalent to a guarantee of no risk. The listed groups are limited in size, and the registry does not report uncertainty intervals or comparative testing. In addition, the death field is blank, which is distinct from a numeric zero. Careful reading depends on preserving the difference between an observed count and a value that was not reported.

Finally, this record shows why design labels should not be mistaken for a complete methods description. Randomization and quadruple masking are important design features, but details about allocation, outcome analysis, missing observations and multiplicity are not reported. Such details are needed to assess how a study’s data were converted into statistical conclusions. Here, no analyses are posted, so the registry record is more informative about study structure and reporting gaps than about estimated effects.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The primary outcome is defined but has no numerical results in the registry record. There are no posted means or standard deviations for glucose, urea or creatinine, and no comparative estimate or uncertainty interval. The primary outcome description includes several measures, and the record indicates that it was not reduced to one comparison. This prevents a single treatment contrast from being interpreted.

The record lists 10 outcome measures but no posted analyses. It does not report which outcomes were analyzed, which comparisons were prespecified, how multiple outcomes were handled, or whether results were adjusted for baseline values. Missing-data counts and methods are also not reported. Although the Phase I adverse-event description states that analysis was per intention to treat, the registry does not define the analysis population in detail or establish that the same approach was used for all outcomes.

Enrollment is reported as 66, but participant counts for the primary outcome analysis are not. The record does not give group-specific allocation totals for the laboratory analysis, participant flow, adherence, follow-up completion or reasons for withdrawal. The overall number enrolled therefore cannot be treated as the number analyzed for any particular endpoint.

The study is listed as terminated, with a primary completion date of 2009-09-29. The reason for termination is not reported in the registry record. The registry also does not report a sample-size justification or detailed statistical analysis plan. Finally, the blank death fields do not provide a numeric death count, and the safety information does not include uncertainty estimates or a formal treatment comparison. These reporting limits constrain both efficacy and safety interpretation.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Effectiveness of Chinese Herbal Therapy for Asthma — NCT00712296.
• Registry record last updated 2020-09-16; status last verified 2020-08.
• Results first posted 2020-08-25.
• 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.

18. Record Summary

NCT00712296 is a randomized, quadruple-masked, parallel-group study of ASHMI and placebo in asthma, listed as Phase I and Phase II. The registry reports enrollment of 66, a start date of 2008-08, a primary completion date of 2009-09-29, and a terminated status. The reason for termination is not reported in the registry record.

The named primary outcome is a Phase I summary of glucose, urea and creatinine in mg/dL, measured one week after active drug or placebo and described using means and standard deviations. Numerical outcome values and comparative analyses are not reported. The registry lists 10 outcome measures and no posted analyses.

For the listed adverse-event groups, the serious-event affected count is 0; other adverse events are also reported. Deaths are not numerically reported. The record supports describing the design and what was registered, but not estimating the primary treatment effect or drawing a comparative efficacy conclusion.