OPT101 in Type 1 Diabetes Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for OPT101 in Type 1 Diabetes Patients (NCT05428943), a randomized phase 1 study. The primary outcome is Number of Treatment-related AE's as Assessed by CTCAE v4.0.

2. Clinical Question

The study record describes a randomized, triple-masked, parallel-group Phase 1b trial. Its stated aims extend beyond the primary safety outcome: secondary outcomes include pharmacokinetic measures and measurements related to HbA1C, C-peptide, and glucose. The registry lists 7 secondary outcomes. The posted results do not include analyses comparing treatment groups, so the central statistical question that can be answered from the primary outcome is descriptive: what event counts were recorded, and how much uncertainty remains about any underlying group difference? The record does not provide enough information to quantify that uncertainty with a confidence interval or to establish whether the observed counts reflect a consistent difference between groups.

3. Trial Design

This was a Phase 1b study of OPT101 in participants with type 1 diabetes. The registry describes randomized allocation, triple masking, and a parallel-group model. Its stated purpose was to assess safety, pharmacokinetics, immunological effects, and clinical effects of multiple ascending doses. The study is marked completed. The recorded start date is 2022-09-27, and the primary completion date is 2024-02-21.

The active treatment groups received OPT101 at 1.1 mg/kg or 2.8 mg/kg, with placebo listed as the comparator. The arm descriptions say that each dose cohort comprised 9 subjects, including 6 who received investigational product and 3 who received placebo. In the posted primary-outcome results, the group sizes are 6, 6, and 8 for the lower-dose, higher-dose, and placebo groups, respectively. The registry does not explain the difference between these descriptions and the outcome denominators, nor does it report participant disposition in sufficient detail to reconcile them.

The primary outcome is the number of treatment-related adverse events assessed by CTCAE v4.0 over 42 days. The registry lists a count of 1 in each OPT101 group and 0 in the placebo group. The record also includes secondary outcome descriptions for serum pharmacokinetics, HbA1C, C-peptide, and glucose measurements. Seven secondary outcomes are posted. Although the study objectives cover several domains, the registry record does not provide statistical analyses for the posted outcomes. It therefore supports a description of the reported counts and study design, but not a formal estimate of comparative treatment effects across the groups.

The registry does not report the randomization procedure, allocation concealment, masking procedures, dose-escalation decision rules, or the basis for the enrollment target. It also does not report a protocol-level statistical analysis plan. These details cannot be inferred from the labels randomized, triple-masked, and parallel-group; those labels identify broad design features, not how they were implemented.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Before assignment: Subjects will be randomized to OPT101 or placebo within each dosing group in a 2:1 ratio. Each patient will be assigned a subject ID and will only be identified by this unique ID. If, as an exception, it is necessary for safety or regulatory reasons to identify the subject, all parties will be bound to keep this information confidential. The subject ID will be used for patient identification throughout the study. The numbers will be assigned sequentially. The subject ID should be used on al

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.

Population: 3 females received the 1.1 mg/kg dose, 3 and females received the 2.8 mg/kg dose. 3 males received the 1.1 mg/kg dose, and 3 males received the 2.8 mg/kg dose. 5 females and 3 males were administered placebo between the 2 cohorts included in the study.

6. Primary Endpoint

The registry lists the primary outcome as Number of Treatment-related AE's as Assessed by CTCAE v4.0, measured over 42 days.

The primary endpoint is the number of treatment-related adverse events assessed using CTCAE v4.0 over 42 days. It is a count outcome: it records events rather than giving a continuous measurement or a time until an event. Safety counts are relevant in an early-phase study whose stated purpose includes assessing safety while studying multiple ascending doses.

A count can describe the number of events recorded in a group, but it does not by itself indicate how those events were distributed among participants. A participant may experience more than one event, so an event count and the number of participants affected are distinct quantities. The registry does not provide participant-level details for the primary endpoint or a separate estimate of the number of people with at least one treatment-related event. The safety record separately reports participants affected by other adverse events, but those entries should not be treated as interchangeable with the primary event count.

The posted primary values are 1 for 1.1 mg/kg OPT101, 1 for 2.8 mg/kg OPT101, and 0 for placebo. No measure of dispersion, confidence interval, or statistical comparison is posted for this endpoint. Without those analyses, the counts are descriptive and do not show how precisely the underlying event experience is estimated. The registry also does not define in further detail how treatment relatedness was determined or how events were counted when multiple events occurred in a participant.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies a randomized, triple-masked, parallel-group Phase 1b study. Those design terms describe intended features of how participants were assigned and followed. They do not, by themselves, supply the statistical methods used to analyze the outcomes. No outcome analyses are posted in the registry record, and details of the statistical analysis plan are not reported in the registry record.

What does a count outcome represent?

The primary outcome is a count of treatment-related adverse events during 42 days. Count outcomes record how many events occur in a group. Unlike a measurement such as a laboratory value, a count is restricted to whole event totals and can include multiple events for the same participant. That distinction matters because the number of events is not necessarily the same as the number of participants affected. The posted primary values are 1, 1, and 0 across the listed groups, while the safety listing separately identifies the number of participants affected by other adverse events.

Statistical analysis of counts often considers both the number of events and the amount of observation represented by each group. In general, count data may show over-dispersion when their variability exceeds what a simple count model assumes. Over-dispersion matters because an analysis that understates variability can make uncertainty appear smaller than it is. The registry does not report a count model, an over-dispersion assessment, or any adjustment for observation time. There is therefore no basis to say which method, if any, was used for the primary endpoint.

How should group comparisons be understood?

A comparison of groups would ordinarily estimate a contrast, such as a difference in mean event counts or a ratio of event rates, together with uncertainty. These are general examples of possible statistical summaries, not estimates reported for this study. The choice depends on the endpoint definition, the distribution of counts, exposure or follow-up information, and the analysis plan. The registry reports counts by group but no comparative effect estimate, confidence interval, or p-value. It is not possible to reconstruct an official analysis from the posted summaries.

A confidence interval, as a general statistical concept, represents a range of values compatible with the data under a specified method and its assumptions. It conveys uncertainty around an estimate; it is not a guarantee that the true value lies within a particular interval. No confidence interval is reported for this primary outcome. With sparse counts, uncertainty can be substantial, and a zero observed count does not imply that the true event rate is zero.

What does randomization add?

Random allocation can reduce systematic differences between groups at the point of assignment, on average, when implemented as intended. It does not ensure that groups are identical in a small study, nor does it resolve questions about missing data, outcome ascertainment, analysis populations, or how an analysis was conducted. The record identifies the study as randomized and triple-masked, but does not report the sequence-generation method, allocation concealment, masking procedures, or checks of masking. These implementation details cannot be inferred from the design labels.

In a parallel-group design, assigned groups are followed as distinct groups rather than being compared through a crossover sequence. That broad structure is consistent with the listed OPT101 dose groups and placebo group. The registry does not state whether the primary endpoint analysis followed all randomized participants, only participants who received study treatment, or another population. Nor does it explain how deviations from assigned treatment or incomplete follow-up were handled.

What can the planning illustrations say?

Planning sample sizes are hypothetical calculations rather than observed trial results. The methodological description for the planning illustrations states a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation, with the effect to detect defined as a stated fraction of the observed effect. For time-to-event settings, it describes total event counts using the Schoenfeld method; other settings are expressed as participants per arm. These calculations are not the study's original design justification and do not represent observed power. The registry does not report the trial's own sample-size justification.

Planning calculations based on an observed effect are especially sensitive to how uncertain and variable that observed effect is. They can illustrate the sample size associated with specified assumptions, but should not be read as evidence that the study had a particular probability of detecting a true effect. In this study, the primary outcome is a count, and the record does not post a two-arm comparison or a comparative effect estimate. Any planning illustration therefore cannot replace a prespecified analysis of the observed study data.

What is not available for independent verification?

The registry does not report the statistical analysis plan, the analysis population, participant-level data, event-level data, model diagnostics, missing-data methods, or multiplicity procedures. It also does not report inferential analyses for the primary endpoint. These omissions prevent readers from verifying whether an analysis was prespecified, whether its assumptions were suitable, or how the reported counts relate to the complete randomized population. The posted summary can be interpreted descriptively, but it cannot support a detailed statistical audit or independent reanalysis.

10. Multiplicity, Missing Data and Analysis Populations

The study has several outcome domains. The registry lists 8 outcome measures overall and 7 secondary outcomes, including pharmacokinetic measures and measures related to HbA1C, C-peptide, and glucose. When multiple outcomes are evaluated, the chance of at least one apparently notable result can increase if each is tested without accounting for the multiplicity. Methods such as prespecifying a primary outcome, adjusting significance thresholds, or treating secondary outcomes as exploratory are general approaches to this issue. The registry record does not report a multiplicity procedure or how confirmatory and exploratory outcomes were distinguished beyond identifying a primary outcome.

No outcome analyses are posted in the registry record. As a result, there are no reported inferential results for which multiplicity adjustment can be evaluated. The absence of posted analyses should not be read as evidence that no analysis was conducted elsewhere; it means the registry record does not report one.

Missing-data handling is also not reported in the registry record. The enrollment entry is 24, whereas the primary-outcome entries list 6 participants in each OPT101 group and 8 in the placebo group. The arm descriptions state that each dose cohort included 9 subjects, with 6 assigned to investigational product and 3 to placebo. The registry does not explain the different denominators, give participant disposition, or state whether the primary outcome was unavailable for some participants. These differences alone do not identify the reason for the counts.

The analysis population is not reported in the registry record. It is therefore unclear whether the primary outcome entries represent all randomized participants, participants who received treatment, or another analysis set. The record also does not describe how partial follow-up, withdrawals, or participants without outcome data were handled. These gaps limit interpretation of both the endpoint counts and any attempt to compare groups.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: infusion site extravasation (0.0%).

The serious adverse-event entries report 0 participants with serious adverse events among the 6 at risk in each OPT101 group and among the 8 at risk in the placebo group. These are observed counts during the recorded safety reporting, not proof that serious adverse events could not occur. The record lists Infusion site extravasation with a value of 0.0% among the top serious-event terms. The listing does not provide additional detail to clarify the term's relationship to the zero serious-event counts, so it should not be interpreted as evidence of a serious event occurring in a participant.

The safety listing also reports other adverse events affecting 1 participant in each OPT101 group and 0 in the placebo group. These are participant counts, distinct from the primary endpoint's number of treatment-related adverse events. The primary outcome records event counts of 1, 1, and 0, respectively. The registry does not report enough detail to determine whether the event counts and participants affected refer to the same events or how relatedness was assigned.

Small group sizes make zero counts difficult to interpret as evidence of absence. They also leave limited information for estimating how frequently uncommon events might occur. The safety data are descriptive, and no comparative uncertainty estimates are posted.

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

The registry reports 1 treatment-related adverse event in each OPT101 dose group and 0 in the placebo group over the 42-day primary-outcome timeframe. It also reports no serious adverse events among the participants listed as at risk in any group. These observations describe the entries in the record. They do not by themselves establish a causal difference between OPT101 and placebo, nor do they establish that the underlying event frequency is equal across groups.

The study was a Phase 1b trial designed to assess safety, pharmacokinetics, immunological effects, and clinical effects of multiple ascending doses. Its early-phase setting and posted group sizes mean that the reported counts provide limited information about uncommon events or the precision of group comparisons. No confidence intervals, p-values, or comparative estimates are posted for the primary outcome. The registry also does not report analysis populations, missing-data methods, or an analysis plan. Consequently, readers cannot assess the statistical uncertainty or independently verify how the counts were analyzed.

The record includes secondary measures related to pharmacokinetics, HbA1C, C-peptide, and glucose, but lists no outcome analyses. The presence of those outcomes indicates areas of assessment, not evidence of a particular clinical or statistical result. Likewise, a planning sample-size illustration is not an observed result and does not indicate the study's actual power.

The study results should therefore be understood as a limited descriptive record of event counts and safety summaries. They do not establish whether OPT101 changes clinical outcomes, whether one dose differs from another, or whether a population-level safety difference exists. Nor do the counts alone resolve how the enrollment target relates to the participants listed for the primary outcome. The registry record does not report those conclusions or the analyses needed to evaluate them.

13. Why This Trial Matters Statistically

This study illustrates a common statistical challenge in early-phase trials: safety outcomes can be reported as small event counts in relatively small groups, while the reader needs to distinguish event totals from the number of participants affected. For this primary outcome, the registry records counts of 1, 1, and 0. The separate safety entries report participants affected by other adverse events and participants with serious adverse events. Treating those measures as interchangeable would blur different questions: how many events occurred, how many people were affected, and how many experienced a serious event.

The record also shows why denominators matter. The registry lists enrollment of 24, the primary outcome entries list groups of 6, 6, and 8, and the arm descriptions state that each dose cohort included 9 subjects. These descriptions do not explain participant flow or the differences among the listed counts. Without clear denominators and analysis-population definitions, even straightforward descriptive comparisons require caution.

Finally, randomized design labels do not replace the reporting of estimates and uncertainty. A useful statistical interpretation would require an analysis method suited to the endpoint, a clear definition of the analysis population, and transparent handling of missing data and multiple outcomes. None of these analysis details is reported in the registry record. The study is therefore a useful example of how a registry can convey design and descriptive safety results while leaving important questions about comparative inference unanswered.

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 posted primary outcome is a count with small group sizes, and the registry does not provide confidence intervals or comparative analyses. The numerical values alone cannot show the precision of a group difference. The count endpoint also differs from the participant-based adverse-event summaries, and the record does not explain whether the same events contribute to both.

The enrollment entry is 24, while primary-outcome group sizes are listed as 6, 6, and 8. The arm descriptions give 9 subjects per dose cohort and describe the allocation of investigational product and placebo within each cohort. Participant flow, the reason for the differing denominators, and the analysis population are not reported in the registry record. It is not possible to determine from these entries whether the differences reflect noncompletion, eligibility, outcome availability, or another explanation.

The registry lists no outcome analyses. It does not report the statistical analysis plan, the method used to compare count outcomes, the handling of missing data, or procedures for addressing multiple outcomes. It also does not report event-level descriptions or how treatment relatedness was determined. These omissions limit both independent verification and interpretation of the observed event counts.

The study's stated aims include pharmacokinetic, immunological, and clinical effects, but the registry lists no analyses for the secondary outcomes. The posted record therefore does not support statistical conclusions about those domains. In addition, descriptive observations from a Phase 1b study with the listed group sizes cannot by themselves characterize uncommon safety events with precision. These limitations concern what can be concluded from the registry record; they do not establish what analyses or information may exist outside it.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: OPT101 in Type 1 Diabetes Patients — NCT05428943.
• Registry record last updated 2026-08-10; status last verified 2026-05.
• Results first posted 2026-08-10.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

NCT05428943 is a completed Phase 1b randomized, triple-masked, parallel-group study of OPT101 in participants with type 1 diabetes. The registry lists enrollment of 24 and describes the study as assessing safety, pharmacokinetics, immunological effects, and clinical effects of multiple ascending doses. The primary outcome is the number of treatment-related adverse events assessed using CTCAE v4.0 over 42 days.

The posted primary outcome counts are 1 for 1.1 mg/kg OPT101, 1 for 2.8 mg/kg OPT101, and 0 for placebo. The safety listing reports no serious adverse events in the listed groups. No outcome analyses are posted in the registry record. Participant flow, analysis populations, missing-data methods, and comparative estimates are not reported in the registry record, limiting conclusions to descriptive interpretation of the posted results.