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 VM202 to Treat Amyotrophic Lateral Sclerosis (NCT02039401), a na phase 1, phase 2 study. The primary outcome is Number of Subjects With Serious and Non Serious Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02039401 |
| Phase | Phase 1, Phase 2 |
| Condition | Amyotrophic Lateral Sclerosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 18 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Helixmith Co., Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
People with amyotrophic lateral sclerosis enrolled in a Phase I/II study. The registry reports an enrollment of 18 participants and identifies the safety analysis set as the population for the primary outcome.
Intervention
Intramuscular VM202, described as a total dose of 64 mg administered over visits on Day 0, Day 7, Day 14, and Day 21. The study purpose was to assess safety and tolerability at different injection sites.
Comparator
There was no concurrent comparator group. The registry describes a single-group, open-label study, with allocation listed as not applicable. The results therefore do not provide a like-for-like comparison with an untreated group or another intervention.
Primary question
How many participants experienced serious and non-serious adverse events during the nine month follow up? The primary outcome was a count of participants with such events, assessed throughout follow-up, as part of the study’s safety and tolerability evaluation.
The clinical question is descriptive rather than a test of whether VM202 differs from another treatment. The registry also names changes in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, Medical Research Council muscle-strength scores, and forced vital capacity as secondary outcomes. Those measures may describe function or respiratory status, but their results are not reported in the registry record. The posted safety counts can describe events recorded among enrolled participants; without a comparator, they cannot isolate how much of the observed event experience is attributable to the intervention rather than the underlying condition or other circumstances.
3. Trial Design
This was a completed Phase I/II, open-label, single-group study of VM202 in people with amyotrophic lateral sclerosis. The registry lists Helixmith Co., Ltd. as the sponsor and an enrollment of 18 participants. Allocation is listed as not applicable, and masking is listed as none. There is no randomized comparison or concurrent control group in the recorded design.
The brief study description identifies safety and tolerability of intramuscular injections at different injection sites as the purpose. The arm description states that a total dose of 64 mg was administered across visits on Day 0, Day 7, Day 14, and Day 21, with the final dose for each target muscle group divided and administered 2 weeks apart. The registry records a start date of 2014-03-11, primary completion on 2015-08-03, and a completed status.
The primary outcome was the number of participants with serious and non-serious adverse events throughout the nine month follow up. The outcome description includes serious adverse events and events leading to treatment discontinuation, and says descriptive statistics would characterize safety parameters. The registry also lists three secondary outcome measures: the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, change in mean muscle-strength Medical Research Council scores, and change from baseline in forced vital capacity. Results for those secondary measures are not reported in the registry record.
The single-group, open-label structure is important to interpretation. Event counts describe what was recorded among participants in this study, but there is no randomized control arm for estimating a comparative treatment effect. Details of eligibility criteria, dose-selection rationale, and the basis for the enrollment target are not reported in the registry record.
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 | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 |
|---|---|---|
| STARTED | 9 | 9 |
| COMPLETED | 9 | 8 |
| NOT COMPLETED | 0 | 1 |
Reasons for not completing this period:
| Reason | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 |
|---|---|---|
| Adverse Event | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Safety analysis set
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: Safety analysis set
| Characteristic | Unit | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Total |
|---|---|---|---|---|
| Participants analysed | 9 | 9 | 18 | |
| Age, Continuous | years | 49.1 (SD 11.9) | 55.8 (SD 3.0) | 52.4 (SD 9.1) |
| Sex: Female, Male: Female | Participants | 1 | 2 | 3 |
| Sex: Female, Male: Male | Participants | 8 | 7 | 15 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 9 | 9 | 18 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 9 | 9 | 18 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Subjects With Serious and Non Serious Adverse Events, measured over Throughout the nine month follow up.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants who experienced serious and non-serious adverse events during the nine month follow up. It is a participant-count outcome: the relevant quantity is how many people had an event, rather than the total number of separate event episodes. The registry’s endpoint description also includes adverse events leading to treatment discontinuation and identifies the safety analysis set as the population of interest.
Such an endpoint is used to characterize safety and tolerability. Counting participants provides a direct description of how many people in the safety population had recorded events. It does not, by itself, describe the number of repeated events each person experienced, the duration or severity of every event, or whether an event was caused by the study intervention. Those details require separate event-level information and attribution criteria, which are not reported in the registry record.
For this study, the endpoint is descriptive. With a single group and no concurrent comparator, the counts cannot establish whether event experience was higher or lower than it would have been under another treatment or no study intervention. The endpoint can still describe the safety events recorded during the specified follow-up, but its interpretation must remain bounded by that design and by the detail available in the registry.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports that 3 of 18 participants were affected by serious adverse events and that 17 of 18 were affected by other, non-serious adverse events. These are participant counts from a safety-focused, single-group study. They describe the recorded event experience among enrolled participants; they are not estimates of a contrast between treatments. The registry also records 1 death. It does not report the cause of death or its relationship to VM202, so neither should be inferred from the count alone.
The serious-event terms listed include small intestinal obstruction, Bronchitis, Urinary tract infection, Pneumonia aspiration, and Respiratory failure, each shown as 5.6%. The registry listing does not establish from those labels alone whether different terms occurred in the same participant, how many episodes occurred, or whether each event was judged related to treatment. The event descriptions and percentages should therefore be read as reported, without adding them together or treating them as mutually exclusive categories.
The primary outcome is a count of participants experiencing events over the nine month follow up. Such a count offers a direct summary but compresses important information: it does not distinguish one event from repeated events in a participant, and it does not show event timing or duration. The registry’s endpoint description states that descriptive statistics would characterize safety parameters. No inferential analysis results are reported in the registry record; it lists no analyses. Consequently, there is no reported confidence interval, hypothesis test, or adjusted comparative estimate to interpret.
General statistical point: a confidence interval, when reported, communicates the precision of an estimated quantity across values compatible with the data and the analysis assumptions. No interval is reported for these safety counts, so their precision cannot be evaluated from an interval in the registry record. Counts from a small safety population can also be sensitive to individual participants, but that general consideration does not justify calculating or assuming unreported uncertainty measures.
The open-label design and absence of a comparator limit causal interpretation. Events observed during follow-up may reflect the underlying illness, other circumstances, the intervention, or more than one factor. The registry record does not provide a comparative analysis that can separate these possibilities. The results therefore support a description of recorded events in this enrolled group, not a claim that the intervention caused or prevented a particular outcome.
The registry also lists secondary functional and respiratory outcomes, but their numerical results are not reported in the registry record. Their absence means the posted information does not support interpretation of change in function or forced vital capacity. The safety results should not be extended to those outcomes, nor should the event counts be read as a comprehensive account of all clinical effects.
A planning illustration based on an observed effect is not an observed study result or a measure of observed power. The stated illustration assumptions concern a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. Those assumptions do not match this trial’s single-group design. Any such illustration is hypothetical planning information, not evidence from a randomized two-arm comparison.
9. Statistical Methods Explained
The primary measure is a count of participants with events, not a continuous measurement or a time-to-event outcome. The registry says descriptive statistics would be used to characterize safety parameters. This is consistent with the stated safety purpose: report what events were recorded in the safety analysis set, without implying a treatment-versus-control test that the design cannot support.
What does a participant count represent?
A participant count indicates how many people in the defined group experienced at least one event meeting the outcome definition. It does not count every event episode. If a participant experienced repeated events, a participant-level count would still treat that person as one affected participant for the relevant category. The registry reports 3 participants with serious adverse events and 17 with other, non-serious adverse events, among 18 participants at risk in each category. The registry does not give event-level counts or explain whether categories overlap.
General statistical point: participant counts can be easier to interpret than a long list of event episodes, but they omit recurrence and timing. Two groups with the same number of affected participants could have different numbers of total events or different event patterns. Those distinctions require additional summaries. The posted registry record does not report them in the information available for this endpoint.
Why is the design relevant to the analysis?
The recorded design is open label and single group, with no randomized comparator. A descriptive summary can state how many participants had recorded events during the defined follow-up. A comparative treatment estimate would require a contrast between groups or another specified comparison, and none is identified in the registry record. For that reason, the counts should not be interpreted as a risk difference, relative effect, or causal estimate.
General statistical point: randomization and masking address different sources of bias in comparative studies. Random allocation can help make groups comparable at baseline, while masking can reduce the influence of knowledge of treatment assignment on care or assessment. The registry lists allocation as not applicable and masking as none; it does not describe a blinded assessment process. In a single-group open-label study, these design features cannot create a control comparison that is absent.
What does descriptive analysis tell the reader?
Descriptive statistics organize observed outcomes, for example by reporting the number of participants in whom an event was recorded. They do not automatically test a null hypothesis or show that an observed event was caused by the intervention. The endpoint description explicitly says descriptive statistics would characterize safety parameters. The registry record does not report inferential analyses, confidence intervals, or a statistical model for the primary outcome.
General statistical point: an estimate’s precision depends on the information available and on the method used to quantify uncertainty. A confidence interval is one way to express that uncertainty, but no interval is reported for these participant counts. It would therefore be inappropriate to present an unreported interval or to treat the counts as more precise than the record establishes.
How should the planning assumptions be understood?
The registry’s planning-illustration description refers to a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and detecting a stated fraction of an observed effect. These assumptions describe a hypothetical two-arm planning calculation, not the recorded single-group trial design. The illustrations are not observed study outcomes and must not be described as observed power. No planning result or sample-size justification is reported in the registry record.
General statistical point: a planning calculation depends on its assumed effect, variability or event frequency, analysis method, and design. Altering those assumptions can change a planning estimate. A calculation framed around two arms does not demonstrate that a one-arm study had a specified power for a comparative question. In particular, planning figures are not a substitute for an analysis of collected data.
Why can over-dispersion matter in other count analyses?
General statistical point: in some count-data models, over-dispersion means that observed variability exceeds what a model’s assumptions allow. If ignored, uncertainty estimates from such a model can be too small. That issue applies to model-based analyses of event counts; it should not be mistaken for a feature established by this registry record. The primary outcome is described as a count of participants with events, and no count model or over-dispersion assessment is reported.
Finally, the analysis population is identified as the safety analysis set, but the record does not describe its membership rules beyond the reported 18 participants at risk. The method used to handle missing outcome information, if any, is not reported in the registry record. These omissions limit reproducibility and make it important to keep interpretation close to the posted descriptive counts.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety outcome and secondary outcomes covering the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, mean muscle-strength Medical Research Council scores, and forced vital capacity. The number of outcome measures is recorded as 4. Considering several outcomes can raise a multiplicity question: if many effects are tested, the chance of at least one apparently notable result can change. The registry record does not report a multiplicity adjustment, a testing hierarchy, or a statistical analysis plan specifying how these outcomes would be interpreted together. The listed outcomes should therefore not be treated as if a correction or prespecified hierarchy had been documented.
The registry identifies the safety analysis set for the primary endpoint. Its inclusion criteria and how participants with incomplete follow-up were handled are not reported in the registry record. The method for handling missing values for the secondary outcomes is also not reported. There is no basis in the record for assuming that missing observations were absent, ignored, imputed, or handled by a particular model.
The record states that descriptive statistics would characterize safety parameters and lists no analyses. It does not report an interim-analysis plan, stopping rule, or adjustment for repeated looks at accumulating data. Those procedures are not reported in the registry record; their absence from the record should not be converted into an assumption that they were or were not used. The available information supports a cautious reading of the reported safety counts, not a reconstruction of unreported analysis choices.
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 |
|---|---|---|---|
| Total | 3 | 18 | 16.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Total | 1 of 18 |
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 |
|---|---|
| Total | 17 of 18 |
Most frequent serious adverse event terms
| Event term | Organ system | Total |
|---|---|---|
| small intestinal obstruction | Gastrointestinal disorders | 1 of 18 |
| Bronchitis | Infections and infestations | 1 of 18 |
| Urinary tract infection | Infections and infestations | 1 of 18 |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | 1 of 18 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 18 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Total |
|---|---|---|
| Fall | Injury, poisoning and procedural complications | 11 of 18 |
| Nasopharyngitis | Infections and infestations | 4 of 18 |
| Headache | Nervous system disorders | 3 of 18 |
| Skin laceration | Injury, poisoning and procedural complications | 2 of 18 |
| Depression | Psychiatric disorders | 2 of 18 |
| diarrhoea | Gastrointestinal disorders | 1 of 18 |
| Gastroesophageal reflux disease | Gastrointestinal disorders | 1 of 18 |
| Asthenia | General disorders | 1 of 18 |
| Fatigue | General disorders | 1 of 18 |
| Pyrexia | General disorders | 1 of 18 |
Serious events with the highest arm-level rate: small intestinal obstruction (5.6%), bronchitis (5.6%), urinary tract infection (5.6%), pneumonia aspiration (5.6%), respiratory failure (5.6%).
Among the 18 participants at risk, the registry reports serious adverse events affecting 3 participants and other, non-serious adverse events affecting 17 participants. It separately records 1 death. The death count does not establish cause or treatment relationship, neither of which is reported in the registry record.
The serious-event listing names small intestinal obstruction, Bronchitis, Urinary tract infection, Pneumonia aspiration, and Respiratory failure, each with a reported value of 5.6%. These are event terms as recorded; the registry information does not establish that the terms occurred in different people or that they are mutually exclusive. They should not be added together to infer a participant total.
General statistical point: serious adverse events are a safety category, while the participant count indicates how many people were affected. Neither the category nor the count by itself establishes causality. The registry’s nine month follow up defines the stated observation period, but event timing, recurrence, severity details beyond the serious classification, and attribution to treatment are not reported in the registry record. These results describe recorded events in this study group and do not supply a controlled comparison.
12. What the Result Does, and Does Not, Mean
The posted data describe safety events recorded among participants in a completed, open-label, single-group study of VM202 in amyotrophic lateral sclerosis. The registry reports 3 participants with serious adverse events, 17 with other non-serious adverse events, and 1 death among an enrolled group of 18. It also lists serious-event terms with values of 5.6% each. These are descriptive findings for the recorded follow-up, not comparative treatment effects.
The numbers do not show whether the events were caused by VM202. Without a comparator, observed events cannot be separated from events associated with the underlying condition or other circumstances. The registry does not report cause or treatment relationship for the death, event-level timing, or whether participants contributed to more than one listed category. The counts also do not indicate how many repeated event episodes occurred.
The record does not report results for the listed functional and respiratory secondary outcomes, nor does it report inferential analyses or confidence intervals. As a result, the posted information cannot establish the precision of the safety counts through interval estimates, and it cannot describe the magnitude of changes in function or forced vital capacity.
General statistical point: an absence of a reported comparison is not evidence that two treatment strategies are equivalent, and a descriptive count alone is not a test of efficacy. The appropriate reading is narrower: the registry records these event counts in the study’s safety population during the specified follow-up. Conclusions about comparative benefit, comparative risk, causality, or broader populations are not established by these data.
13. Why This Trial Matters Statistically
This study illustrates why design details matter as much as the numerical result. An open-label, single-group safety study can document adverse events observed during follow-up, but without a concurrent comparator it cannot estimate how event experience differs under another treatment or no study intervention. That distinction is central when interpreting safety counts as descriptive evidence rather than a causal comparison.
The primary endpoint is a participant count, which is straightforward to state but does not preserve every detail about event recurrence, timing, duration, or attribution. The registry’s serious and non-serious counts therefore answer a limited question: how many participants were recorded as affected in each category. They do not convey the full event history for each person.
The record also illustrates the importance of reporting analysis details. It identifies a safety analysis set and says descriptive statistics would be used, but does not provide membership rules, missing-data handling, inferential analyses, or a multiplicity approach. Those details affect how readers judge reproducibility and how much confidence to place in interpretations extending beyond the counts themselves.
Finally, planning calculations must be matched to the design and question. The stated planning assumptions describe an equal-allocation, two-arm framework, whereas this trial is recorded as single group. Planning illustrations are not evidence that the study had a particular observed power or that a treatment difference was established. Keeping design, analysis, and interpretation aligned prevents descriptive safety evidence from being overstated.
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 enrollment was 18, and the study had no concurrent comparator. This means the event counts describe a small enrolled group and do not provide a controlled estimate of relative safety. The open-label design also means masking was not used according to the registry record. The registry does not report whether outcome assessment had any separate safeguards against knowledge of treatment.
The endpoint records participants with serious and non-serious adverse events throughout the nine month follow up. The registry does not report event-level timing, recurrence, duration, detailed severity beyond the serious classification, or treatment attribution. It also does not clarify whether the same participants appear in both event categories or across multiple serious-event terms. The recorded death count has no reported cause or relationship to the intervention.
The analysis information is limited. The safety analysis set is named, but its membership rules are not reported. Missing-data procedures, a statistical analysis plan, inferential results, confidence intervals, and multiplicity handling are not reported in the registry record. The functional and respiratory secondary outcome results are also not reported. These omissions restrict assessment of precision, reproducibility, and outcomes beyond the descriptive safety counts.
Eligibility details, the rationale for the enrollment target, and any interim-analysis or stopping procedures are not reported in the registry record. These limitations do not negate the counts as recorded, but they constrain the questions those counts can answer and discourage broader causal or comparative conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis — NCT02039401.
- Registry record last updated 2025-10-06; status last verified 2025-08.
- Results first posted 2023-09-21.
- 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 NCT02039401 as a completed Phase I/II, open-label, single-group study of VM202 in people with amyotrophic lateral sclerosis. It records an enrollment of 18 participants and describes intramuscular administration of a total dose of 64 mg across visits on Day 0, Day 7, Day 14, and Day 21. The primary outcome was the number of participants with serious and non-serious adverse events throughout the nine month follow up.
The posted safety results report 3 participants with serious adverse events, 17 with other non-serious adverse events, and 1 death. No randomized comparator is recorded. The registry says descriptive statistics would characterize safety parameters and lists no analyses. Results for the secondary functional and respiratory measures, analysis details beyond the safety analysis set, and the cause or treatment relationship of the death are not reported in the registry record.