Long Term Safety Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Long Term Safety Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study (NCT02002936), a na phase 1 study. The primary outcome is Adverse Events.

2. Clinical Question

The clinical question concerns long-term safety and efficacy of SyB C-1101 in patients with recurrent, relapsed, or refractory myelodysplastic syndrome who had completed 6 cycles in study 2012002. The extension study description specifies oral administration every 3 weeks, twice daily for 14 consecutive days. The registry record identifies adverse events as the primary outcome, assessed up to 3 years.

The study is not a comparison of SyB C-1101 with another treatment or with no treatment. It is a single-group extension study, and the registry lists no allocation to different arms. The posted results therefore describe the experience of participants receiving SyB C-1101 in this extension; they do not provide a concurrent control group for separating treatment effects from other influences.

The primary outcome is a count of participants affected by any adverse event. The record reports 1 participant affected among 1 participant at risk. The serious adverse-event count is 0 among 1 participant at risk, while the record also lists 1 participant affected in the other adverse-event category. These are descriptive observations from a very small enrolled group.

The listed secondary outcomes concern hematologic remission and improvement under IWG 2006 criteria, cytogenetic response, overall survival, and changes in clinical laboratory test results. Results for these outcomes are not reported in the registry record. The record also contains no analysis results. Consequently, the central question the posted data can address is limited to what adverse-event counts were recorded for the participant in this extension, not whether the treatment changes outcomes relative to an alternative.

3. Trial Design

The registry classifies this as a Phase I, single-group extension study of SyB C-1101. Allocation is recorded as not applicable, and masking is listed as none. The study is marked completed. The recorded start is 2013-08 and primary completion is 2014-12. Enrollment was 1.

The extension was intended for patients who had completed 6 cycles in study 2012002. Its stated purpose was to investigate long-term safety and efficacy of oral SyB C-1101 in patients with recurrent, relapsed, or refractory myelodysplastic syndrome. The described schedule was every 3 weeks, twice daily for 14 consecutive days. The registry does not report additional details about the extension's eligibility criteria, treatment modification rules, or follow-up procedures.

Because there is a single treatment group and no masking, the record describes an open-label, uncontrolled design. A single-group design can document events observed during treatment and follow-up, but it cannot make a like-for-like comparison with a separate control group. The registry explicitly identifies the arms as unsuitable for a control comparison. No causal treatment contrast can be estimated from the posted design and results.

The primary outcome is adverse events, measured as the total number of participants affected, with a timeframe of up to 3 years. The record lists secondary outcomes for hematologic remission, hematologic improvement, cytogenetic response, overall survival, and changes in clinical laboratory test results. Although six outcome measures are listed, the registry reports no analyses, and results for the secondary outcomes are not reported in the registry record.

Enrollment of 1 makes the study's empirical scope particularly narrow. Its posted counts describe one participant; they cannot establish how frequently events would occur across a broader population. The record does not report a sample-size justification or a planned enrollment target, so the reason for the enrollment size is 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

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.

6. Primary Endpoint

The registry lists the primary outcome as Adverse Events, measured over Up to 3 years.

The primary endpoint is adverse events, expressed as the total number of participants affected by any adverse event during follow-up of up to 3 years. This is a participant count rather than a count of the total number of separate event episodes. The posted result is 1 participant affected out of 1 at risk.

Adverse-event monitoring is used in clinical research to describe harms observed while participants receive an intervention. Counting participants affected provides a straightforward summary of whether any event was recorded for each participant. It does not, by itself, describe the event's severity, duration, timing, cause, or relationship to treatment. Those details would be needed for a fuller account of the safety experience, and they are not presented in the primary endpoint result.

In this extension, the endpoint is especially limited by the size of the enrolled group. The observation that the participant experienced an adverse event is a description of that participant's record, not a precise estimate of an event rate in a larger patient population. It also does not show what would have happened to a similar participant under another treatment or without the study intervention.

The registry lists adverse events as the primary outcome, but it does not report a statistical analysis, uncertainty interval, or analysis population beyond the count and denominator. The record's separate serious adverse-event figures provide additional descriptive safety information, but they do not replace the primary endpoint or establish comparative safety.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a single-group Phase I extension study, with allocation not applicable and masking none. Its primary result is reported as a participant count: the number affected by any adverse event. The numerator is 1 and the denominator is 1 participant at risk. This is a descriptive summary rather than a model-based estimate or a treatment comparison. No analyses are reported in the registry record.

What does a participant count describe?

A participant count records how many people in the study group had an event, rather than how many separate episodes occurred. The distinction matters because a participant can experience more than one event, but the primary outcome is framed as the total number of participants affected. The posted result therefore supports a statement about whether the enrolled participant was recorded as having any adverse event. It does not reveal the number of event episodes or their clinical details.

A denominator gives the number of participants at risk for the outcome. Here, the registry lists 1 participant at risk for the primary adverse-event outcome. With such a small denominator, the description applies directly to the enrolled participant and gives little information about variation across patients. General statistical principle: estimates based on small samples are typically imprecise for a wider target population, and a single observed outcome cannot characterize the distribution of possible outcomes.

Why does the design limit comparisons?

In a randomized comparison, allocation can support a like-for-like contrast between assigned groups, subject to the trial's conduct and analysis. This record does not describe a randomized comparison: allocation is not applicable, and there is one group. The treatment group has no concurrent control group against which to compare event counts or other outcomes. Consequently, no between-group treatment effect can be calculated from the posted design.

Masking is recorded as none. In general, masking can reduce the influence of knowledge about treatment assignment on assessment or reporting, depending on the outcome and the people involved. The record does not report any masking procedures, so the safety observations should be read as open-label observations. It is not possible to determine from the registry entry how event ascertainment was conducted beyond the outcome description.

What do the adverse-event categories show?

The registry reports 0 serious adverse events among 1 participant at risk and 1 participant affected in the other adverse-event category among 1 at risk. The primary outcome records 1 participant affected by any adverse event. These are counts in separate registry categories, not a comparison between groups. The record does not list serious adverse-event terms, and death counts are not reported in the registry record. The categories do not provide details about event timing, severity beyond the serious classification, or causal attribution.

How should the planning illustrations be read?

The sample-size illustrations use a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. The assumptions describe an effect to detect as a stated fraction of the observed effect. These are planning illustrations, not the design of this single-group extension. They are not observed power and do not change the interpretation of the observed count. The record does not report a sample-size justification for the extension.

In general, power is a property of a specified study design and assumed effect under a statistical model; it is not a measure of whether a result is true. Sample-size calculations can illustrate how many participants per arm might be needed under specified assumptions, but those illustrations are not results from the participants actually enrolled. They cannot compensate for the absence of a comparator or for the enrollment of 1.

What can be inferred from the posted analysis?

The registry reports no analyses. No inferential test, confidence interval, adjusted estimate, or model-based result is reported. General statistical methods can quantify uncertainty or compare outcomes when the design and data support those calculations, but no such result is posted for this trial. The appropriate reading is descriptive: 1 participant was recorded with any adverse event, and 0 serious adverse events were recorded among 1 participant at risk. The listed secondary outcomes do not have reported results in the registry record, so their statistical interpretation is not possible from the posted information.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("SyB C-1101")
n      <- c(1)
events <- c(1)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  SyB C-1101  1  1  100.0  2.5  100.0  20.7  100.0  15.8  98.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists six outcome measures, including the primary adverse-event outcome and secondary outcomes for hematologic remission, hematologic improvement, cytogenetic response, overall survival, and changes in clinical laboratory test results. It reports no analyses. Results for the secondary outcomes are not reported in the registry record, so there is no set of posted secondary estimates or tests to interpret.

Multiplicity arises when several outcomes or statistical tests are considered, because the chance of at least one apparently notable result can increase as more tests are conducted. General statistical practice may address this through a prespecified hierarchy or adjustment, depending on the study objectives. The registry does not report whether any multiplicity strategy was planned or used. It would be inappropriate to infer a strategy from the list of outcomes alone.

Missing-data methods and analysis populations are also not reported in the registry record. The entry gives enrollment of 1 and an at-risk count of 1 for the adverse-event results, but it does not describe whether any data were missing, how missing observations were handled, or which analysis population was intended for each outcome. The actual follow-up duration for the participant is not reported.

These omissions matter because the interpretation of an outcome can depend on its analysis population, the completeness of follow-up, and how missing measurements are treated. For instance, a count among participants with recorded information can have a different meaning from a result based on all enrolled participants, if those groups differ. No such distinction can be evaluated from this record. The posted adverse-event counts remain descriptive, and unreported procedures should not be assumed.

12. 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 registry reports 0 serious adverse events among 1 participant at risk. It also reports 1 participant affected in the other adverse-event category among 1 participant at risk, while the primary endpoint reports 1 participant with any adverse event among 1 at risk. These are participant counts, not counts of event episodes.

A serious-event count of 0 means that no serious adverse event was recorded in the result for the enrolled participant. It does not demonstrate that the risk of serious events is absent in a broader population. The record lists no serious adverse-event terms, and death counts are not reported in the registry record. Details about the event in the other category, including its type, severity, timing, and relationship to treatment, are not reported in the primary result.

General statistical interpretation: a zero count in a very small group is weak evidence about how often an event might occur outside that group. The denominator is essential context; with 1 participant at risk, these figures describe only the observed study experience. There is no control group to provide a comparative safety estimate. The counts therefore should be read as a limited record of events observed during this extension, not as proof of safety or as a comparison with another treatment.

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

The posted primary result means that the registry recorded an adverse event for the only participant enrolled in this extension. It also records no serious adverse events for that participant. These observations provide a concise description of the safety data reported for this study, but the enrolled group is too small to characterize outcomes across a broader population with precision.

The result does not establish the frequency of adverse events among all patients with myelodysplastic syndrome, or among all patients who might receive SyB C-1101. It does not show whether the event was caused by the intervention, how severe or prolonged it was, or when it occurred. Those details are not reported in the registry record. Nor does the serious adverse-event count of 0 mean that serious events cannot occur.

The design also limits what can be concluded about treatment effects. There is no comparator group, so the posted data do not show how outcomes would differ under another treatment or no treatment. The record's description of long-term safety and efficacy as a study purpose does not itself establish either. Results for the listed hematologic, cytogenetic, survival, and laboratory outcomes are not reported in the registry record.

General statistical interpretation: observed data and population conclusions are different. A study can report exactly what occurred among its participants while still leaving substantial uncertainty about outcomes beyond the study. In this case, the count is informative as a record of the single participant's reported experience, but it cannot provide a reliable estimate of event rates or comparative effects. No inferential analysis is reported.

The study should therefore be understood as a very small, uncontrolled extension with descriptive adverse-event counts. It documents 1 participant affected by any adverse event and 0 participants with a serious adverse event among 1 at risk. Broader conclusions about efficacy, comparative safety, or the likelihood of future events go beyond what the registry record reports.

14. Why This Trial Matters Statistically

This trial is statistically notable because it illustrates the difference between a recorded observation and a population-level conclusion. The registry reports 1 participant enrolled, with 1 participant affected by any adverse event and 0 serious adverse events among 1 at risk. These counts are unambiguous descriptions of the posted results, but their scope is limited to the enrolled participant.

The extension context is also important. The study was intended for patients who had completed 6 cycles in study 2012002, and the stated aim was to investigate long-term safety and efficacy. This is a distinct question from evaluating an intervention in a randomized comparison. Its single-group design can record experiences during follow-up but cannot supply a like-for-like control contrast. The record explicitly identifies the arms as not suitable for such a comparison.

Statistically, the case highlights why sample size, comparator structure, outcome definitions, and reporting completeness all shape interpretation. A participant count is easy to understand, yet a denominator of 1 cannot convey the range of outcomes across a population. A zero serious-event count is similarly limited: it describes what was recorded, not the absence of risk in general.

The record also demonstrates the limits of an outcome list without corresponding analysis results. Several secondary outcomes are named, but their results and the analyses are not reported in the registry record. That prevents statistical interpretation of those endpoints. The study remains meaningful as a documented extension with a stated safety focus, while its posted data support only narrow descriptive conclusions rather than comparative or broadly generalizable ones.

15. 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.

Enrollment was 1, so the adverse-event findings describe only one participant. This sharply limits the ability to assess how representative the observation might be of other patients or to characterize variation in outcomes. The record does not report a sample-size justification, and the reason for the enrollment size is not reported in the registry record.

The study is single-group, with allocation not applicable and no masking. There is no concurrent control group. As a result, the posted counts cannot distinguish the experience associated with treatment from other influences, and they do not support a comparative treatment effect. The record itself notes that the arms are not a like-for-like control comparison.

The primary result gives the number of participants affected by any adverse event, but the registry does not report event details in that outcome description. The serious adverse-event list contains no terms. Event timing, severity details beyond the serious classification, relatedness to treatment, and actual duration of follow-up are not reported in the registry record. Although the stated timeframe is up to 3 years, the record does not show whether the participant was followed for the full timeframe.

The registry lists secondary outcomes but reports no analyses. Their outcome results are not reported in the registry record. Statistical methods for missing data, analysis populations, multiplicity, and any prespecified inferential analyses are also not reported. Without those details, it is not possible to evaluate the completeness or analysis of the secondary outcomes.

Finally, the registry's stated purpose includes both long-term safety and efficacy, but the posted result available for interpretation is the adverse-event count. The purpose statement should not be mistaken for evidence that all intended questions were answered. The record supports a limited descriptive account, not a broad assessment of safety or efficacy.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Long Term Safety Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study — NCT02002936.
• Registry record last updated 2017-02-23; status last verified 2017-01.
• Results first posted 2016-12-12.
• 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.

19. Record Summary

This completed Phase I extension study evaluated SyB C-1101 in patients with myelodysplastic syndrome. It was single-group, with allocation not applicable and no masking. Enrollment was 1. The recorded study start was 2013-08, and primary completion was 2014-12.

The extension description concerns patients with recurrent, relapsed, or refractory disease who had completed 6 cycles in study 2012002. The specified oral schedule was every 3 weeks, twice daily for 14 consecutive days. The primary outcome was the number of participants affected by any adverse event, with a timeframe of up to 3 years.

The registry reports 1 participant with any adverse event among 1 at risk, 0 serious adverse events among 1 at risk, and 1 participant affected in the other adverse-event category among 1 at risk. No analyses are reported. Results for the listed secondary outcomes, details of the events, and the analysis and missing-data methods are not reported in the registry record. The findings are descriptive and do not provide a control-group comparison.