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 Gene Therapy for Ischemic Heart Disease in Korea (NCT01422772), a non-randomized phase 1, phase 2 study. The primary outcome is The Incidence of Adverse Events - Total Adverse Events (AE).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01422772 |
| Phase | Phase 1, Phase 2 |
| Condition | Ischemic Heart Disease |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Helixmith Co., Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
Patients with ischemic heart disease whose affected coronary artery territory could not be completely revascularized even after coronary artery bypass grafting (CABG). The registry lists an enrollment of 9.
Intervention
VM202 gene medicine injected into cardiac muscle in three dose-escalation cohorts: 0.5 mg in 1 mL at 4 sites, 1 mg in 2 mL at 8 sites, or 2 mg in 4 mL at 8 sites.
Comparator
There was no control group. The registry describes a single-group, non-randomized, non-comparative study. The dose cohorts therefore are not randomized or like-for-like comparator arms.
Primary question
What adverse events were observed after VM202 administration during the 24-week safety assessment? The primary outcome is the total number of adverse events, with assessment of adverse reactions, vital signs, physical examination, and laboratory test results.
The clinical question is principally about safety, not a controlled estimate of treatment benefit. The registry describes direct injection into cardiac muscle in the coronary territory that remained incompletely revascularized after CABG. The study title identifies it as open-label and dose-escalation, and the registry lists Phase 1 and Phase 2.
The listed secondary outcomes concern cardiac function and myocardial measurements: left ventricular ejection fraction assessed by magnetic resonance imaging; cardiac function assessed by echocardiography; viable myocardium thickness in end-systolic and end-diastolic conditions; and myocardial ischemic area in stress and resting conditions. These measures can describe changes over time, but the registry record does not report their numerical results or a between-group comparison. Without a control group, changes in these outcomes cannot by themselves distinguish a treatment effect from other explanations for change over time.
3. Trial Design
This was an open-label, non-randomized, single-group, non-comparative, dose-escalation trial at a single center, according to the official title and design fields. The registry lists Phase 1 and Phase 2. Enrollment was 9, and the record is marked completed. The listed study start was 2007-01 and primary completion was 2010-02.
VM202 was administered by intramyocardial injection, with the dose and injection pattern varying across three cohorts. Cohort I received 0.5 mg in 1 mL injected into 4 sites. Cohort II received 1 mg in 2 mL injected into 8 sites. Cohort III received 2 mg in 4 mL injected into 8 sites. The registry describes this as gene medicine injected into cardiac muscle in an area that remained incompletely revascularized after CABG.
Dose escalation is a design in which exposure levels are considered in sequence, often with attention to safety. That general description does not establish the decision rules used in this particular study: those rules are not reported in the registry record. The record also does not report how participants were assigned to cohorts, the timing of escalation decisions, or whether any formal stopping criteria were used.
Because there was no random allocation and no control group, the cohorts are not a randomized comparison of doses. Differences between cohort observations could reflect dose, participant characteristics, chance, or other factors. The study can describe what was recorded during this small safety evaluation, but it cannot provide the controlled treatment-effect estimate that a randomized comparison is designed to support. The registry record does not report the sample-size justification or a prespecified statistical analysis plan.
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 | Group 1 - 0.5 mg/1 mL | Group 2 - 1.0 mg/2 mL | Group 3 - 2 mg/4 mL |
|---|---|---|---|
| STARTED | 3 | 3 | 3 |
| COMPLETED | 3 | 3 | 3 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Total Number of Adverse Events (ITT 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.
Population: Intent-to-Treat
| Characteristic | Unit | Group 1 - VM202 - 0.5 mg/ 1mL | Group 2 - VM202 - 1.0 mg/2 mL | Group 3 - VM202 - 2 mg/4 mL | Total |
|---|---|---|---|---|---|
| Participants analysed | 3 | 3 | 3 | 9 | |
| Age, Continuous | years | 61.00 (SD 12.77) | 59.00 (SD 6.93) | 67.67 (SD 6.11) | 62.56 (SD 8.60) |
| Sex: Female, Male: Female | Participants | 1 | 0 | 2 | 3 |
| Sex: Female, Male: Male | Participants | 2 | 3 | 1 | 6 |
| Region of Enrollment: South Korea | participants | 3 | 3 | 3 | 9 |
6. Primary Endpoint
The registry lists the primary outcome as The Incidence of Adverse Events - Total Adverse Events (AE), measured over 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Total AEs |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled the incidence of adverse events and is expressed as total adverse events over 24 weeks. The record describes a safety assessment over a 6-month period and says that subjects administered the investigational drug are included regardless of protocol violations or adherence to visit schedules. Assessments include adverse reactions, vital signs, physical examination, and laboratory test results. The stated population is Total Number of Adverse Events (ITT Safety).
An event count is relevant to safety because one participant can experience more than one event. A count can therefore capture repeated events, whereas a count of participants affected answers a different question: how many people experienced at least one event. These measures should not be treated as interchangeable. The posted results list serious and other adverse-event figures by cohort, but do not report the primary endpoint as a total-event count. The primary outcome's numerical result and any statistical uncertainty around it are not reported in the registry record.
Safety outcomes are commonly used in early-phase research to characterize observed events and identify issues that may warrant further evaluation. That general purpose does not mean that an event count alone establishes causality. The registry does not report a control group, so events observed after administration cannot be compared against an untreated or otherwise controlled background within this study. Nor does the endpoint title alone establish that every event was caused by VM202.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted record supports a narrow descriptive reading. It documents a completed, open-label dose-escalation study of VM202 in patients with ischemic heart disease after CABG when complete revascularization could not be achieved. It lists enrollment of 9 and describes three dose cohorts. Its principal stated objective was safety assessment, with the primary outcome defined as the total number of adverse events during 24 weeks.
The posted adverse-event figures distinguish serious events from other events and report participants affected by cohort. The serious-event participant count is 1 in the 0.5 mg cohort and 0 in each of the 1.0 mg and 2 mg cohorts. The other-event participant count is 3 in each cohort. The record lists no deaths in any cohort. These figures describe recorded participants affected; they do not give the total number of event episodes. In particular, the primary endpoint is a total-event count, and that numerical result is not reported.
The serious adverse-event terms listed in the record are Worsening Spinal Stenosis and Infectious Spondylitis, each reported as 33.3%. Those percentages are not enough to determine the total-event count, the timing of events, or whether an event was considered related to VM202. The registry record does not report those details. The presence of a serious event in a cohort also does not by itself establish a dose relationship or a causal connection to the intervention.
These cohort figures should not be read as evidence that a higher dose has a lower or higher risk. Participants were not randomized between doses, each cohort is small, and the record does not report a comparative analysis. With few observations, a single participant's experience can have substantial influence on the displayed cohort summary. Differences between cohorts may be due to chance or differences in participants as well as dose.
The registry lists secondary measures of ejection fraction, echocardiographic cardiac function, myocardial thickness, and ischemic area. It does not report their numerical results. Consequently, the posted record does not permit an assessment of changes in those measures or their precision. The count of 8 outcome measures indicates that outcomes were registered, but does not supply their findings.
In statistical terms, the record offers descriptive safety summaries rather than an effect estimate against a control. It does not report confidence intervals, hypothesis tests, adjusted estimates, or a primary total-event result. The findings should therefore be interpreted as limited observations from this study, not as a precise estimate of risk, proof of causation, or evidence of comparative benefit.
9. Statistical Methods Explained
The design determines what statistical conclusions can be drawn. This study is registered as non-randomized, single-group, and non-comparative. Randomization can help balance measured and unmeasured characteristics between groups; without it, dose cohorts may differ for reasons other than dose. A control group can help separate changes associated with an intervention from changes that might occur over time or under other care. Neither feature is present in this registry description, so a causal dose comparison is not supported by design.
What does an event count represent?
General statistical explanation: count outcomes record how many events occur within a defined observation period. They differ from binary outcomes, which record whether a participant had at least one event, and from rates, which relate events to time at risk. One participant may contribute multiple events to a count, while contributing only once to a count of participants with an event. The registry's primary endpoint is a total adverse-event count, but the posted cohort summaries report participants affected by serious and other events. The record does not state the primary total-event counts, so those participant summaries cannot substitute for the primary endpoint.
General statistical explanation: count data are often analyzed with models designed for counts. A Poisson model is one possibility when its assumptions are suitable, including an assumption about the relationship between the mean and variance. Over-dispersion occurs when the observed variability is greater than such a model allows; ignoring it can make uncertainty look too small. Alternatives may be considered when assumptions do not fit. This is general context only. The registry record reports no analysis for the primary outcome and does not identify a count model, an over-dispersion assessment, or any other analysis method.
Why is the study not a dose comparison?
General statistical explanation: an observed difference between groups is not automatically a treatment effect. Random allocation supports a fairer comparison by reducing systematic differences at baseline, while adequate sample size supports precision. Here, allocation is non-randomized, the trial is non-comparative, and cohort assignment details are not reported in the registry record. It would therefore be inappropriate to treat the cohort summaries as a controlled estimate of how risk changes with dose. The record does not report adjusted analyses that might address differences between cohorts.
What would uncertainty measures add?
General statistical explanation: a confidence interval gives a range of values compatible with the data under a specified statistical procedure and its assumptions. It expresses uncertainty around an estimate; it does not guarantee that the true value lies in the interval or resolve bias caused by study design. For small samples, estimates are typically less precise, and intervals can be wide. The registry record does not report confidence intervals for the adverse-event summaries or the primary endpoint. No precision estimate for the primary total-event count can therefore be described.
General statistical explanation: hypothesis tests address a specified question under a statistical model. A p-value is not the probability that a treatment works or that a finding is due to chance. It does not measure clinical importance and does not correct for a weak comparison design. The registry record reports no analysis for outcomes, no p-values, and no analysis plan. It is not possible to infer which hypotheses, if any, were tested or how they were handled.
How should the dose-escalation structure be understood?
Dose escalation organizes exposure into cohorts, but the label alone does not reveal how decisions were made. A protocol might specify rules for moving to another dose, pausing enrollment, or reviewing safety; those rules are not reported in the registry record. Nor is the sequence or timing of cohort review described. General statistical explanation: in small early-phase studies, safety summaries are often primarily descriptive because estimates from sparse data are imprecise. That general principle does not establish the intended analysis for this trial.
The registry record also does not identify a statistical method for the secondary outcomes. Those outcomes involve different measurements, including imaging and echocardiographic measures. General statistical explanation: continuous measurements can be summarized by changes from baseline, but interpretation depends on how values are measured, which time points are used, how missing measurements are handled, and what analysis is prespecified. The registry lists percentage change and changes in myocardial measurements, but it does not report numerical findings, analysis details, or confidence intervals.
Sample-size calculations elsewhere associated with observed effects, if presented, are planning illustrations rather than evidence about the study's observed power. General statistical explanation: observed power is not a useful substitute for examining the estimate and its uncertainty. The registry record does not report a sample-size justification, and the cohort summaries should not be interpreted as establishing a minimum effective sample size for future studies.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple secondary outcomes in addition to the primary safety outcome. These include imaging-based ejection fraction, echocardiographic cardiac function, two measures of viable myocardium thickness, and ischemic area under stress and resting conditions. Assessing many outcomes can increase the chance of finding an apparently notable result by chance if multiple statistical tests are performed. General statistical explanation: a prespecified multiplicity strategy can control or describe that risk, for example by identifying a primary outcome or adjusting inference across a defined family of tests. The registry record does not report a multiplicity strategy, and it lists no outcome analyses.
Missing-data handling is also not reported in the registry record. The primary outcome description says participants administered the investigational drug are included regardless of protocol violations or adherence to visit schedules. This describes the intended safety population, but does not explain how incomplete adverse-event follow-up or missing laboratory, imaging, or cardiac-function measurements were handled. It also does not establish that every participant contributed complete observations at every assessment.
The record does not report a statistical analysis plan, an imputation method, a sensitivity analysis for missing outcomes, or details of how participants were counted when follow-up was incomplete. The phrase ITT Safety appears in the population description for the primary outcome, but the record does not provide further operational definitions for that population. Therefore, the analysis population and missing-data procedures cannot be reconstructed from the registry description.
These omissions matter because the interpretation of a small study can depend strongly on which participants and observations are included. Without analysis details, it is not possible to assess whether missingness could have influenced the reported summaries or whether secondary-outcome findings were interpreted with multiplicity in mind. No claims about such procedures can be made from the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Group 1 - VM202 - 0.5 mg/ 1mL | 1 | 3 | 33.3% |
| Group 2 - VM202 - 1.0 mg/2 mL | 0 | 3 | 0.0% |
| Group 3 - VM202 - 2 mg/4 mL | 0 | 3 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group 1 - VM202 - 0.5 mg/ 1mL | 0 of 3 |
| Group 2 - VM202 - 1.0 mg/2 mL | 0 of 3 |
| Group 3 - VM202 - 2 mg/4 mL | 0 of 3 |
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 |
|---|---|
| Group 1 - VM202 - 0.5 mg/ 1mL | 3 of 3 |
| Group 2 - VM202 - 1.0 mg/2 mL | 3 of 3 |
| Group 3 - VM202 - 2 mg/4 mL | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Group 1 - VM202 - 0.5 mg/ 1mL | Group 2 - VM202 - 1.0 mg/2 mL | Group 3 - VM202 - 2 mg/4 mL |
|---|---|---|---|---|
| Worsening Spinal Stenosis | Musculoskeletal and connective tissue disorders | 1 of 3 | 0 of 3 | 0 of 3 |
| Infectious Spondylitis | Infections and infestations | 1 of 3 | 0 of 3 | 0 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group 1 - VM202 - 0.5 mg/ 1mL | Group 2 - VM202 - 1.0 mg/2 mL | Group 3 - VM202 - 2 mg/4 mL |
|---|---|---|---|---|
| Wound Complication | Injury, poisoning and procedural complications | 1 of 3 | 3 of 3 | 3 of 3 |
| Supraventricular extrasystoles | Cardiac disorders | 3 of 3 | 1 of 3 | 2 of 3 |
| Atrial fibrillation | Cardiac disorders | 0 of 3 | 0 of 3 | 3 of 3 |
| Sinus tachycardia | Cardiac disorders | 1 of 3 | 1 of 3 | 1 of 3 |
| Procedural Pain | Injury, poisoning and procedural complications | 2 of 3 | 0 of 3 | 1 of 3 |
| Nausea | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 1 of 3 |
| Dyspepsia | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 1 of 3 |
| Constipation | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 1 of 3 |
| Diarrhoea | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 1 of 3 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 1 of 3 | 0 of 3 | 1 of 3 |
Serious events with the highest arm-level rate: worsening spinal stenosis (33.3%), infectious spondylitis (33.3%).
The registry reports serious and other adverse-event participant figures separately for each dose cohort. For serious events, the figures are 1 affected participant in the 0.5 mg cohort and 0 in each of the other two cohorts. For other adverse events, the figure is 3 affected participants in each cohort. The record lists 0 deaths in each cohort. These are participant-level summaries, not totals of every event episode.
Two serious adverse-event terms are named: Worsening Spinal Stenosis and Infectious Spondylitis. Each is listed with 33.3%. The record does not explain the denominator used for these term percentages, provide event dates, or state whether the events were considered related to the intervention. They should not be converted into participant counts or interpreted as causal evidence.
Small cohort summaries are inherently limited in how precisely they describe uncommon events. A zero in a cohort means no event was recorded in that displayed category for that cohort; it does not establish that the underlying risk is zero. Conversely, an event occurring after administration does not, by itself, show that the intervention caused it. The study has no control group for comparison, and the registry does not provide uncertainty intervals for these figures.
The primary safety endpoint is the total number of adverse events over 24 weeks, which is different from the number of participants affected. The posted results do not report that total count. Thus, the cohort figures are useful as descriptive safety information but do not fully answer the registered primary-outcome question.
12. What the Result Does, and Does Not, Mean
The record establishes that VM202 was studied as an intramyocardial injection in patients with ischemic heart disease and incomplete revascularization after CABG, using three dose cohorts. It documents a safety-focused study with a 24-week primary outcome period and describes assessment of adverse reactions and clinical and laboratory measures. The record also lists cohort-level participant counts for serious and other adverse events, serious-event terms, and deaths.
Those observations are not a controlled estimate of the effect of VM202. There was no randomized comparator, and the registry describes the trial as non-comparative. It is not possible to attribute cohort differences to dose or intervention, or to exclude alternative explanations. The posted record does not report the primary total adverse-event count, confidence intervals, hypothesis tests, or numerical results for the listed cardiac and imaging outcomes.
The study also cannot establish that an absence of a recorded event in a cohort means an absence of risk. With limited observations, rare or delayed events may not be characterized precisely. The registry does not report details of longer-term safety follow-up beyond the stated safety period, and no inference about outcomes outside the described follow-up should be made.
Likewise, the listed secondary outcomes do not establish a clinical benefit. Their numerical findings are not reported in the registry record, and a before-and-after change without a control group would not, on its own, isolate an intervention effect. No treatment recommendation follows from these data.
The appropriate statistical interpretation is therefore descriptive and limited: the registry records what safety categories were observed in this study and what outcomes were intended to be assessed. It does not provide enough reported results to quantify the primary event burden or estimate comparative safety or efficacy. Further studies with a suitable comparison, transparent analysis methods, and precise outcome reporting would be needed to answer broader questions.
13. Why This Trial Matters Statistically
This trial is statistically informative because its design makes the distinction between description and comparison especially important. It is a small, non-randomized, single-group dose-escalation study rather than a controlled trial. Its cohort summaries can document observed events, but they cannot isolate a dose effect from participant differences, chance, or other influences.
The primary endpoint also illustrates why outcome definitions matter. A total adverse-event count captures event episodes, while the posted serious and other event figures count participants affected. These are different summaries and answer different questions. Because the registry does not report the total-event result, the registered primary outcome cannot be evaluated numerically from the posted results.
The record's safety focus is appropriate context for interpreting an early-phase study, but a safety-oriented purpose does not remove the need to report denominators, follow-up completeness, event definitions, and uncertainty. The record contains selected cohort-level figures and names serious-event terms, but does not report confidence intervals, analysis methods, or causality assessments. Such details are important when observations are sparse.
This study also shows how a list of registered secondary outcomes differs from evidence of findings. Cardiac function, ejection fraction, myocardial thickness, and ischemic area are listed as outcomes, but their numerical results are not reported. Without estimates and uncertainty, readers cannot gauge the direction or precision of changes.
The central statistical lesson is to match the strength of a conclusion to the design and available results. These data can describe recorded safety observations within the study, but they do not support a randomized dose comparison, a precise risk estimate, or a conclusion about treatment benefit.
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.
Important limitations arise from the trial's design and the level of detail in the registry record. The study is non-randomized and non-comparative, with no control group. Its dose cohorts therefore cannot provide a like-for-like estimate of differences between doses or between VM202 and an alternative. Cohort differences may reflect factors other than dose, and the registry does not report an adjusted comparative analysis.
The enrollment is 9, and the posted results report small cohort-level event summaries. Such data provide limited precision for characterizing safety, particularly for uncommon events. No confidence intervals are reported. The registry also does not report cohort assignment procedures, participant characteristics by cohort, or the criteria governing dose escalation, limiting assessment of comparability and study conduct.
The primary outcome is defined as total adverse events over 24 weeks, but the posted results do not give the total-event count. Instead, adverse-event summaries describe participants affected by serious and other events. The serious-event terms have percentages, but the denominator and relationship assessments are not reported. Event timing and duration are also not reported in the registry record.
Although secondary cardiac and imaging outcomes are listed, their numerical results and analyses are not reported. The record does not specify missing-data procedures, a statistical analysis plan, multiplicity handling, or the sample-size justification. The primary completion date is reported, but details of the follow-up completeness and any longer-term assessment are not available in the registry record.
These limitations mean that the posted findings are descriptive and incomplete for estimating the primary outcome, comparing doses, or evaluating changes in cardiac measures. They also constrain how confidently the observed adverse events can be attributed to the intervention.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety Study of Gene Therapy for Ischemic Heart Disease in Korea — NCT01422772.
- Registry record last updated 2025-10-03; status last verified 2025-09.
- Results first posted 2025-10-03.
- 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 record describes NCT01422772, titled Safety Study of Gene Therapy for Ischemic Heart Disease in Korea. It is marked completed and lists Phase 1 and Phase 2, with an open-label, non-randomized, non-comparative, single-group dose-escalation design at a single center. Enrollment was 9; the study start was 2007-01 and primary completion was 2010-02.
VM202 was injected into cardiac muscle in three dose cohorts for patients with ischemic heart disease and an area that remained incompletely revascularized after CABG. The primary outcome was total adverse events over 24 weeks. Posted safety summaries include serious and other adverse-event participants by cohort, two serious-event terms, and no deaths listed in any cohort.
The registry does not report the numerical primary total-event result, analyses for the outcomes, or numerical results for the listed secondary cardiac measures. With no control group and no randomized allocation, the available cohort summaries are descriptive and do not establish comparative effects.