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 and Pharmacokinetics Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) (NCT02014051), a na phase 1 study. The primary outcome is Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02014051 |
| Phase | Phase 1 |
| Condition | Myelodysplastic Syndrome |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | SymBio Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with myelodysplastic syndrome described as recurrent, relapsed or refractory. The registry record lists an enrollment of 10. It does not report further eligibility details or describe the characteristics of participants who entered each dose group.
Intervention
The intervention was SyB C-1101, an oral drug. The study description says it was administered twice daily for 14 consecutive days. The registry record identifies dose groups of 280 mg/Dose and 560 mg/Dose; it does not state the actual dose received by every participant or provide a fuller schedule.
Comparator
There was no concurrent control group identified. The record describes a single-group study, with allocation listed as not applicable and masking as none. The two dose groups are not a like-for-like randomized comparison, so differences between their recorded counts cannot establish a causal dose effect.
Primary question
The primary outcome is adverse events, measured as the number of participants affected, over up to 18 weeks. The study description emphasizes tolerability, dose-limiting toxicity and maximum tolerated dose, and estimating a dose for phase II studies. It also says pharmacokinetics and antitumor effects would be investigated. The registry record lists hematologic remission and hematologic improvement as secondary outcomes, but does not post results for those outcomes in the information.
3. Trial Design
This was a phase I clinical trial of SyB C-1101 in patients with myelodysplastic syndrome. The registry record classifies the model as single-group, reports allocation as not applicable, and reports no masking. It lists enrollment of 10 and identifies SymBio Pharmaceuticals as the sponsor. The study status is completed, with a start date of 2013-03 and primary completion in 2014-11.
The study description states that SyB C-1101 was to be given orally twice daily for 14 consecutive days. The registry’s adverse-event information separates participants into a 280 mg/Dose group and a 560 mg/Dose group. These dose groups do not make the study a randomized, controlled comparison: the record does not identify random allocation, a control treatment, or masking. The reason participants were assigned to the listed doses and the process used to make that decision are not reported in the registry record.
The stated aims were to investigate tolerability, determine dose-limiting toxicity and maximum tolerated dose, and estimate a recommended dose for phase II studies. Pharmacokinetics and antitumor effects were also to be investigated. The primary outcome is the number of participants affected by adverse events, with a timeframe of up to 18 weeks. Two secondary outcomes are listed: hematologic remission under IWG 2006 criteria, with responses sustained for at least 4 weeks, and hematologic improvement under IWG 2006 criteria, with responses lasting at least 8 weeks.
The registry record does not report a statistical analysis plan, dose-escalation rules, sample-size justification, or details of how the dose-limiting toxicity and maximum tolerated dose were defined.
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 | SyB C-1101 280 mg/Dose Group | SyB C-1101 560 mg/Dose Group |
|---|---|---|
| STARTED | 3 | 6 |
| COMPLETED | 2 | 5 |
| NOT COMPLETED | 1 | 1 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | All Participants |
|---|---|---|
| Participants analysed | 9 | |
| Age, Customized: 50-59 years | participants | 1 |
| Age, Customized: 60-69 years | participants | 3 |
| Age, Customized: 70-79 years | participants | 4 |
| Age, Customized: 80- years | participants | 1 |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Adverse Events, measured over Up to 18 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is adverse events expressed as the total number of participants affected by any adverse event, with participants as the unit and a timeframe of up to 18 weeks. This is a binary participant-level outcome: each participant either is or is not counted as affected for the specified measure. It is not a count of how many separate events each participant experienced. The record directs readers to the adverse-event information for details.
In a phase I study, adverse-event monitoring is relevant to describing tolerability and identifying safety concerns during exposure. The study description also states that the trial aimed to determine dose-limiting toxicity and maximum tolerated dose. Those aims require clear definitions, observation rules and dose-escalation procedures, which are not reported in the registry record.
The posted adverse-event counts distinguish serious events from other events within each dose group. Those categories provide some detail about the reported safety information, but they do not by themselves establish a complete result for the primary endpoint. The record does not explain how overlapping serious and other events were handled in the primary total, or provide a separately stated overall count for participants affected by any adverse event across the full study. The outcome’s timeframe is up to 18 weeks; the registry record does not report individual follow-up duration or whether every participant was observed for the full timeframe.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted results describe adverse-event counts in two dose groups, rather than a randomized comparison. In the 280 mg/Dose group, 3 participants were at risk for other adverse events and 3 were affected; 3 were at risk for serious adverse events and 1 was affected. In the 560 mg/Dose group, 6 participants were at risk for other adverse events and 6 were affected; 6 were at risk for serious adverse events and 3 were affected. These are group-specific counts, and the record does not present a comparative statistical analysis.
The counts should be read as descriptive information about the recorded groups. Because allocation was not applicable and the study is classified as single-group, the dose groups cannot be assumed to be exchangeable. The record does not report how participants came to receive either dose, whether the groups differed at baseline, or whether dose decisions depended on earlier safety observations. Consequently, a difference in event counts cannot be attributed to dose alone.
The enrollment is 10, whereas the adverse-event information lists 3 and 6 participants at risk in the two dose groups. The reason the listed at-risk counts do not account for the full enrollment is not reported in the registry record. The record also does not report confidence intervals, hypothesis tests, adjusted estimates, or an inferential analysis for the adverse-event outcome. The results therefore do not quantify statistical uncertainty around the group-specific counts.
The registry lists serious adverse-event terms with percentages: Type 2 diabetes mellitus and delirium at 33.3% each; cholecystitis, periodontitis, pneumonia, and soft tissue infection at 16.7% each. These are presented as serious-event terms, not as evidence that every listed event occurred in every dose group. The record does not state the denominator or clarify whether a participant could contribute more than one term. It also does not provide a death count.
The outcome information should be interpreted alongside its scope: a small phase I study, no concurrent control, limited group counts, and no posted analysis of the listed hematologic secondary outcomes. The counts describe what the registry reports; they do not establish comparative safety, efficacy, or a definitive dose recommendation.
9. Statistical Methods Explained
The outcomes described in the registry are counts of participants, not measurements on a continuous scale. For an event outcome, each participant is classified according to whether an event was recorded during the stated observation period. A participant-level count is distinct from an event count: one participant might have more than one event, but the participant is counted once in a measure that asks how many people were affected. The primary outcome is phrased as the total number affected by any adverse event, while the posted safety details separately give counts for serious and other adverse events.
What a participant count can tell readers
A count gives the number of people recorded in a category. Its interpretation depends on the population eligible for counting and the number observed. The registry reports at-risk denominators of 3 in the 280 mg/Dose group and 6 in the 560 mg/Dose group for both serious and other adverse events. Those denominators are essential context: the counts should not be read in isolation or treated as estimates for a broader patient population. The registry does not explain why the at-risk totals differ from the enrollment of 10.
For binary outcomes, a proportion can often help describe the frequency relative to a denominator. No new proportions are calculated. The posted safety record already gives counts and, for selected serious-event terms, percentages. The record does not explain the denominator used for those term percentages. Without that context, the percentages should be retained as reported rather than reinterpreted as rates for a particular dose group or for all enrolled participants.
Why the dose groups are not a controlled contrast
A randomized comparison uses a defined allocation process to support a fair comparison between treatment groups. This study is classified as single-group, with allocation listed as not applicable and masking listed as none. Although the adverse-event details are divided into dose groups, the record does not describe random assignment between those doses. Nor does it report a concurrent control group. The groups may therefore differ for reasons other than dose, and a numerical contrast would not isolate a dose effect.
In general, confounding occurs when a factor related to group assignment is also related to the outcome. In a dose-finding study, dose decisions may be linked to study progression or earlier observations, but the specific process is not reported in the registry record. The appropriate reading is descriptive: the posted counts summarize events recorded in each listed group, without treating the groups as equivalent or estimating a causal effect.
Uncertainty and inferential analysis
A confidence interval is a statistical range used to express uncertainty around an estimate under a specified method and assumptions. A hypothesis test evaluates data against a defined statistical null hypothesis. Neither type of result is reported for the primary adverse-event outcome. The registry record lists no analyses, and it does not provide an adjusted model, a test statistic, a p-value, or a confidence interval for the dose groups. This absence means the posted results do not quantify inferential uncertainty. It does not mean that the groups are identical or that an effect is absent.
With small group denominators, each participant’s classification has a substantial influence on the group count. General statistical reasoning therefore cautions against treating the observed counts as precise estimates of event frequency in a larger population. No numerical precision measure can be inferred from the counts alone without selecting a method and assumptions; the registry does not report such an analysis.
How the intended dose-finding aims relate to analysis
The study description identifies dose-limiting toxicity and maximum tolerated dose as objectives. These are dose-finding concepts, not automatically conclusions that can be read from any adverse-event count. Their determination depends on protocol definitions, timing, dose escalation and rules for evaluating participants. Those methods are not reported in the registry record. The study also aimed to estimate a recommended phase II dose, but the posted information does not identify a recommended dose or the decision process behind one.
Sample-size calculations are planning exercises based on chosen assumptions and a target analysis. They are not observed outcomes or evidence that a study had a particular probability of detecting an effect. The registry record does not report the study’s sample-size justification. Any planning illustration should therefore be kept separate from the trial’s observed counts and should not be described as observed power.
Finally, the registry lists secondary hematologic outcomes with response-duration requirements. The outcomes are not accompanied by participant counts, estimates, or analyses. Their criteria indicate how the outcomes were named in the record, but do not establish that responses occurred. A statistical interpretation of those endpoints would require posted results and information about their analysis populations and missing observations.
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.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| SyB C-1101 280 mg/Dose Group | 3 | 3 | 100.0% | 29.2% to 100.0% | 43.9% to 100.0% | 39.8% to 99.4% |
| SyB C-1101 560 mg/Dose Group | 6 | 6 | 100.0% | 54.1% to 100.0% | 61.0% to 100.0% | 59.0% to 99.6% |
R code and output
arm <- c("SyB C-1101 280 mg/Dose Group", "SyB C-1101 560 mg/Dose Group")
n <- c(3, 6)
events <- c(3, 6)
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 280 mg/Dose Group 3 3 100.0 29.2 100.0 43.9 100.0 39.8 99.4
ARM SyB C-1101 560 mg/Dose Group 6 6 100.0 54.1 100.0 61.0 100.0 59.0 99.611. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary adverse-event outcome and two secondary hematologic outcomes: remission under IWG 2006 criteria with responses sustained for at least 4 weeks, and hematologic improvement under IWG 2006 criteria with responses lasting at least 8 weeks. It does not report how many hypotheses were planned, whether any outcomes were prioritized beyond their primary or secondary labels, or whether a multiplicity adjustment was planned. Multiplicity refers generally to the increased chance of at least one false-positive finding when several statistical questions are tested. No hypothesis tests are reported for these outcomes, so the record does not support an assessment of multiplicity control.
The registry record does not report an analysis plan, analysis populations, or rules for handling missing outcomes. It does not state how participants who discontinued, missed assessments, or had incomplete follow-up were handled. The adverse-event timeframe is up to 18 weeks, but individual follow-up duration and completeness are not reported. The distinction between participants enrolled and the at-risk denominators shown in the adverse-event details is also unexplained.
For the hematologic outcomes, the record names response-duration criteria but does not post outcome counts or describe how missing assessments or responses with insufficient duration were classified. No imputation method or sensitivity analysis is reported. These omissions limit assessment of whether the observed safety and response information represents all enrolled participants, a subset, or a particular analysis population. They also prevent evaluation of how missing observations might affect interpretation.
12. 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 |
|---|---|---|---|
| SyB C-1101 280 mg/Dose Group | 1 | 3 | 33.3% |
| SyB C-1101 560 mg/Dose Group | 3 | 6 | 50.0% |
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 |
|---|---|
| SyB C-1101 280 mg/Dose Group | 3 of 3 |
| SyB C-1101 560 mg/Dose Group | 6 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | SyB C-1101 280 mg/Dose Group | SyB C-1101 560 mg/Dose Group |
|---|---|---|---|
| Cholecystitis | Hepatobiliary disorders | 0 of 3 | 1 of 6 |
| Periodontitis | Infections and infestations | 0 of 3 | 1 of 6 |
| Pneumonia | Infections and infestations | 0 of 3 | 1 of 6 |
| Soft tissue infection | Infections and infestations | 0 of 3 | 1 of 6 |
| Urinary tract infection | Infections and infestations | 0 of 3 | 1 of 6 |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | 1 of 3 | 0 of 6 |
| Delirium | Psychiatric disorders | 1 of 3 | 0 of 6 |
| Haematuria | Renal and urinary disorders | 0 of 3 | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | SyB C-1101 280 mg/Dose Group | SyB C-1101 560 mg/Dose Group |
|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 0 of 3 | 2 of 6 |
| Vomiting | Gastrointestinal disorders | 1 of 3 | 1 of 6 |
| Nasopharyngitis | Infections and infestations | 0 of 3 | 2 of 6 |
| Alanine aminotransferase increased | Investigations | 1 of 3 | 1 of 6 |
| Aspartate aminotransferase increased | Investigations | 1 of 3 | 1 of 6 |
| Lymphocyte count decreased | Investigations | 1 of 3 | 1 of 6 |
| Neutrophil count decreased | Investigations | 1 of 3 | 1 of 6 |
| Abdominal pain | Gastrointestinal disorders | 1 of 3 | 0 of 6 |
| Constipation | Gastrointestinal disorders | 1 of 3 | 0 of 6 |
| Diarrhoea | Gastrointestinal disorders | 0 of 3 | 1 of 6 |
Serious events with the highest arm-level rate: type 2 diabetes mellitus (33.3%), delirium (33.3%), cholecystitis (16.7%), periodontitis (16.7%), pneumonia (16.7%).
The serious adverse-event information is presented as participant counts by dose group. In the 280 mg/Dose group, 1 of 3 participants at risk was affected by a serious adverse event. In the 560 mg/Dose group, 3 of 6 were affected. These are descriptive counts, not results of a randomized comparison. The record does not report a statistical test or uncertainty interval for the difference between groups, and the study design does not establish that the groups are comparable.
For other adverse events, the record reports 3 of 3 participants affected in the 280 mg/Dose group and 6 of 6 in the 560 mg/Dose group. Serious and other adverse events are separate reporting categories in the registry entries; the record does not explain whether a participant could be represented in both. The primary outcome is described more broadly as participants affected by any adverse event, but a separate overall total is not stated in the information.
The listed serious-event terms and percentages are Type 2 diabetes mellitus, 33.3%; delirium, 33.3%; cholecystitis, 16.7%; periodontitis, 16.7%; pneumonia, 16.7%; and soft tissue infection, 16.7%. The denominator for these percentages is not reported in the registry record. The record also does not report a death count; an unreported count should not be read as zero.
13. What the Result Does, and Does Not, Mean
The registry record describes a phase I study focused on tolerability, dose-limiting toxicity, maximum tolerated dose and estimating a dose for phase II studies. It reports adverse-event counts for participants in two dose groups and names hematologic remission and improvement as secondary outcomes. These details indicate the kinds of safety and response questions the study was intended to address.
The posted counts do not establish that one dose is safer than the other. The study is classified as single-group, allocation is not applicable, and no concurrent control is identified. The record does not explain dose assignment or report an adjusted comparison. The counts therefore describe events among the listed participants rather than an isolated effect of dose.
The record does not establish whether SyB C-1101 reduced disease activity or produced hematologic responses. Although the study description says antitumor effects would be investigated and the registry names two hematologic secondary outcomes, results for those outcomes are not reported in the information. The listed outcome criteria alone are not evidence of a response.
The small at-risk group counts and absence of reported confidence intervals or statistical tests limit conclusions about precision. The enrollment is 10, while the two listed adverse-event denominators are 3 and 6; the record does not explain the difference. It also does not report complete follow-up, the analysis populations, or how missing assessments were handled. These gaps make it difficult to judge how fully the posted counts represent all enrolled participants.
Accordingly, the record supports a narrow descriptive reading of adverse events in the reported groups. It does not support a definitive claim about comparative safety, treatment benefit, or a recommended dose. Those conclusions would require additional protocol and results information that is not reported in the registry record.
14. Why This Trial Matters Statistically
This trial illustrates why a dose-finding study should be interpreted according to its design and aims. Its stated focus was tolerability and identifying dose-limiting toxicity and maximum tolerated dose, rather than estimating a treatment effect against a control group. The adverse-event counts are therefore useful as descriptive safety information, but they do not become a controlled dose comparison merely because the registry reports two dose groups.
The distinction between enrollment and the denominators used in outcome reporting is also important. The registry reports enrollment of 10 but lists 3 and 6 participants at risk in the dose-specific adverse-event details. Without an explanation of this difference, readers cannot determine how the reported safety population relates to everyone enrolled. Clear reporting of analysis populations and follow-up would make that relationship easier to assess.
The record also demonstrates the limits of interpreting counts without uncertainty measures or analysis details. Counts convey what was recorded in the listed groups, while confidence intervals and other analyses can describe statistical precision under stated assumptions. Such analyses are not reported for the primary outcome. The absence of inferential results does not itself imply no effect; it means the registry record does not quantify one.
Finally, naming secondary endpoints is not the same as reporting their results. The criteria for hematologic remission and improvement are stated, but the outcomes are not accompanied by estimates or analyses in the information. Separating endpoint definitions from outcome evidence is essential when readers assess what a trial contributes.
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.
Several trial-specific limitations constrain interpretation of the registry results. The study is classified as single-group, with allocation listed as not applicable and masking as none. The two dose groups therefore do not provide a randomized, controlled comparison. The record does not describe the process used to assign participants to doses, the reasons for dose selection, or whether group characteristics differed.
The enrollment is 10, but the adverse-event details list 3 participants at risk in the 280 mg/Dose group and 6 in the 560 mg/Dose group. The registry record does not explain this difference. It also does not report baseline characteristics, individual follow-up, completeness of observation through the stated timeframe of up to 18 weeks, or the analysis populations. As a result, the connection between enrollment and the safety counts is unclear.
The primary outcome is described as the total number of participants affected by any adverse event, while the posted details separate serious from other events. A distinct overall count for the primary measure is not stated in the information, and overlap between categories is not explained. The percentages attached to serious-event terms are reported without a stated denominator. A death count is not reported in the registry record.
There are no reported statistical analyses, confidence intervals, hypothesis tests, or adjusted estimates. The sample-size justification, dose-escalation rules, definitions of dose-limiting toxicity and maximum tolerated dose, and methods for handling missing data are not reported in the registry record. Although secondary hematologic outcomes are listed, their results and analysis details are not reported in the information. These limitations leave the posted results descriptive and incomplete for comparative or inferential conclusions.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Safety and Pharmacokinetics Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) — NCT02014051.
- Registry record last updated 2022-11-21; status last verified 2022-11.
- Results first posted 2016-09-14.
- 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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19. Record Summary
The registry record describes a completed phase I study sponsored by SymBio Pharmaceuticals, with a start date of 2013-03 and primary completion in 2014-11. It reports enrollment of 10 and classifies the study as single-group, with allocation not applicable and no masking.
SyB C-1101 was described as an oral treatment administered twice daily for 14 consecutive days. The stated aims included tolerability, dose-limiting toxicity, maximum tolerated dose, a phase II dose estimate, pharmacokinetics and antitumor effects. The primary outcome was participants affected by any adverse event, over up to 18 weeks. Two hematologic outcomes are listed as secondary endpoints.
Adverse-event counts are reported for 280 mg/Dose and 560 mg/Dose groups. The record does not report statistical analyses, a sample-size justification, analysis populations, or results for the listed hematologic endpoints. The safety counts are descriptive and do not establish a controlled comparison between doses.