Phase 1/2 Study to Evaluate Safety, PK and Efficacy of the MYC-Inhibitor OMO-103 in Solid Tumours: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Phase 1/2 Study to Evaluate Safety, PK and Efficacy of the MYC-Inhibitor OMO-103 in Solid Tumours (NCT04808362), a na phase 1, phase 2 study. The primary outcome is Phase 1: Safety and Tolerability.

2. Clinical Question

3. Trial Design

This was an open-label, two-part, first-in-human Phase 1/2 dose-finding study of OMO-103 in patients with advanced solid tumours. The stated aims were to evaluate safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and proof-of-concept. The registry record lists a single OMO-103 group, no masking and allocation as not applicable. It does not describe a control group or a randomized comparison.

The intervention was OMO-103, administered intravenously as a 30 min infusion once weekly. The record does not list the dose levels, escalation or dose-selection procedures, treatment stopping criteria, or the number of participants assigned to each dose level. It also does not explain how the two parts were organized or whether the study proceeded through specific dose cohorts.

Enrollment is listed as 22. The study start date is 2021-04-28, primary completion is listed as 2022-12-15, and the status is terminated. The reason for termination is not reported in the registry record. These dates and status describe the registry entry; they do not, on their own, explain the conduct or completion of individual assessments.

The primary outcome is labeled Phase 1: Safety and Tolerability, and its population is the safety population. The listed timeframe says the DLT period was 3 weeks and that adverse events were assessed for each patient until progression, which was in average 3 months. A secondary outcome is Phase 1 elimination half-life (t1/2). The registry record lists 2 outcome measures and 0 analyses. It does not report an analysis plan or the results for the primary outcome.

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

Analysis population for the primary outcome, as defined in the registry: safety population

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 Phase 1: Safety and Tolerability, measured over DLT period was 3 weeks and AEs were assessed for each patient until progression which was in average 3 months;.

The primary endpoint is described as the number of patients with a DLT and the number with IRRs, adverse events or serious adverse events according to NCI CTCAE v 5. Its title is Phase 1: Safety and Tolerability, its unit is participants, and its named population is the safety population. The DLT observation period is listed as 3 weeks; adverse events were assessed until progression, which was in average 3 months.

Counting participants with events is a way to describe how many people experienced specified safety outcomes in the population under observation. For a dose-finding study, such counts can characterize the observed safety experience and inform evaluation of tolerability. That is a general explanation of the role of these measures, not a statement about the study’s results or the investigators’ specific rationale.

The registry record does not report the primary outcome counts for DLTs, IRRs, adverse events or serious adverse events as the primary endpoint. The adverse-event figures listed separately do not supply all of those endpoint-specific counts or establish how they relate to the defined DLT period. A count of participants with at least one event also differs from a count of all event occurrences: one participant may experience more than one event, and the registry record does not give event-level totals for the primary endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation depends on matching each reported quantity to its definition, population and observation period. For this study, the primary outcome is a participant count in the safety population. The registry description specifies DLTs, IRRs, adverse events and serious adverse events, but does not provide primary endpoint counts or inferential analyses. The adverse-event counts listed separately are descriptive registry data; they do not fill in every missing endpoint result.

What a participant count means

A count of participants with an event answers how many people in the group experienced at least one event meeting the stated definition. It is different from a count of event episodes, because an individual can experience multiple events. It is also different from a rate based on time at risk, which requires information about follow-up time for each participant or an appropriate aggregate. These are general distinctions; the registry record does not provide the event-level detail needed to calculate additional measures for this study.

In the adverse-event listing, 9 of 22 participants are recorded as affected by serious adverse events, and 22 of 22 are recorded as affected by other adverse events. The record lists 3 deaths. These counts describe what is recorded for the single OMO-103 group, not a treatment contrast. No calculations beyond the registry’s reported numbers are needed to interpret them as counts. The number of affected participants does not reveal how many separate events occurred, how long participants were followed individually, or whether any event was judged related to treatment.

Why the single-group design matters

A randomized comparison can estimate a difference between treatment groups under its design assumptions. In contrast, a single-group study describes outcomes among people receiving the intervention but has no concurrent control group to distinguish treatment-associated outcomes from the background risks of the disease or other factors. This is a general design principle. In this record, allocation is not applicable, masking is none, and only one group is listed. No causal comparison with another treatment can therefore be drawn from the listed counts.

Open-label means the study is not masked according to the registry listing. In general, knowledge of treatment assignment can affect reporting or assessment of outcomes, particularly outcomes involving judgment. That does not establish that bias occurred in this study. The registry record does not describe assessment procedures, independent review or steps used to reduce assessment bias.

What a confidence interval would add

In general, a confidence interval expresses the statistical uncertainty around an estimate under a specified model and method. A narrow interval usually indicates greater precision than a wide interval, although an interval does not remove limitations in design or measurement. The registry record does not report confidence intervals for the primary endpoint or the listed adverse-event counts. No interval can be interpreted as a study result from the registry entry.

Similarly, a p-value addresses compatibility between observed data and a specified statistical model or null hypothesis; it is not a measure of clinical importance or the probability that a hypothesis is true. The registry record lists 0 analyses and does not report primary endpoint tests or p-values. No significance claim is supported by the information in the record.

What planning illustrations mean

Sample-size calculations based on an observed effect are planning illustrations. They are not observed power, and they do not describe the number of participants actually needed or enrolled under the study’s own protocol. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation and a chosen fraction of an observed effect. Those assumptions do not match the listed single-group study design. The registry record does not report the trial’s own sample-size justification or planning assumptions; those are not reported in the registry record.

For a binary outcome, a planning calculation also depends on the event proportions assumed for the groups and the target effect. If those assumptions do not correspond to the trial design or the scientific question, a calculated sample size has limited relevance to this study. No new planning estimate should be inferred from the registry description alone.

Dispersion and event data

Dispersion describes variability around a measure, and the relevant form depends on the data type. For binary participant counts, the number of participants at risk and the number with the event determine the observed proportion, while uncertainty depends on the statistical method. For recurrent event counts, variability may exceed what a simple model assumes; in general, over-dispersion can make standard errors too small if it is ignored. The record does not report recurrent-event modeling, dispersion estimates or an analysis plan, so these considerations are general rather than claims about the study’s analyses.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists 2 outcome measures, including the Phase 1 safety and tolerability endpoint and a secondary outcome for elimination half-life (t1/2). It lists 0 analyses. It does not report a statistical analysis plan, a hierarchy of endpoints, a multiplicity adjustment, or a prespecified approach for interpreting multiple safety outcomes. These details are not reported in the registry record.

Multiplicity is a general concern when several hypotheses or outcomes are tested: the more tests considered, the greater the chance of at least one apparently notable result under a set of null hypotheses. A prespecified testing strategy or adjustment can address that issue, but the registry entry does not say whether one was used. Since it reports no analyses or p-values, there is no stated family of tests to evaluate.

Missing-data methods are also not reported in the registry record. The entry does not state how incomplete assessments, withdrawals, missed visits or participants with limited follow-up were handled. The adverse-event timeframe says events were assessed until progression, which was in average 3 months, but does not describe each participant’s follow-up duration or completeness.

The named primary analysis population is the safety population. How that population was defined, how many participants contributed to each endpoint, and whether any participants were excluded from particular analyses are not reported in the registry record. The record does not report imputation methods or sensitivity analyses. These omissions limit assessment of how complete the outcome data were and whether the reported adverse-event counts represent all enrolled participants under a consistent follow-up definition.

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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: bile duct obstruction (4.5%), pleuritic pain (4.5%), hydrocephalus (4.5%), abdominal pain (4.5%), epigastralgia (4.5%).

The registry lists 22 participants at risk for serious adverse events, with 9 affected. For other adverse events, it lists 22 at risk and 22 affected. It also lists 3 deaths. These are counts for the OMO-103 group, not results from a controlled comparison. They do not establish that treatment caused an event or death.

The serious adverse-event terms listed are Bile Duct Obstruction, Pleuritic pain, Hydrocephalus, Abdominal pain, Epigastralgia and IRR. Each term is displayed with 4.5%. The record does not explain whether a participant could contribute to more than one term, give event-level counts beyond these listed terms, or provide details about timing and causal assessment. The listed term percentages should not be interpreted as mutually exclusive categories or used to infer unreported totals.

Serious adverse events and other adverse events are distinct registry groupings, and their affected-participant counts should be read according to those labels. The primary outcome description also refers to DLTs, IRRs, AEs and SAEs, but the registry record does not report the primary endpoint counts for those measures. The adverse-event listing therefore gives limited descriptive safety information, not a complete quantitative report of the primary safety endpoint.

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

The registry record establishes that OMO-103 was studied in an open-label, single-group, first-in-human Phase 1/2 dose-finding study for advanced solid tumours. It describes weekly intravenous administration and identifies safety, tolerability, PK, PD and proof-of-concept as study aims. It also records adverse-event counts and selected serious adverse-event terms for the OMO-103 group.

Those details do not establish a treatment effect. There is no comparator group, and the record does not report a randomized contrast. Counts of participants with adverse events describe observations in the study group; without a control, they cannot show how outcomes would have differed under another treatment or no study treatment. The registry listing alone also cannot attribute every event or death to OMO-103.

The primary endpoint results are not reported in the registry record. In particular, the record does not give primary counts for DLTs, IRRs, adverse events or serious adverse events under the endpoint definition. It therefore does not permit an estimate of the primary endpoint’s precision, a statistical test, or a quantitative assessment of that endpoint’s results.

The status is terminated, but the reason for termination is not reported in the registry record. Termination status alone does not explain whether the decision reflected safety, recruitment, operational issues or another reason. The record also does not provide dose-level outcomes, detailed follow-up, an analysis plan or missing-data methods. Conclusions should remain limited to the registered design and the descriptive adverse-event information actually listed, without extending them to unreported efficacy or causal claims.

13. Why This Trial Matters Statistically

This study is statistically notable as a single-group, first-in-human dose-finding study whose registered primary focus is safety and tolerability. Early studies of this type are generally designed to characterize observations under treatment and inform further investigation, rather than to establish comparative effects. That is a general description of the role of dose-finding research, not a statement about a protocol decision that is absent from the registry record.

The distinction between a descriptive safety count and a comparative effect is important. The adverse-event listing records affected participants in the OMO-103 group, but a single group cannot provide a concurrent estimate of what would have occurred in a control group. The counts can therefore be read as observations, not as treatment-attributable excess risk. Detailed event definitions, timing and causal assessments would be needed for fuller interpretation and are not reported in the registry record.

It also matters that the registry lists a primary safety endpoint but does not report its outcome counts or analyses. The absence of those results prevents readers from assessing the registered primary question quantitatively. Reporting the endpoint definition without its outcome values is not equivalent to reporting evidence that the endpoint was or was not met.

Finally, the record’s 0 analyses and absence of a stated analysis plan leave important statistical questions unanswered, including endpoint-specific populations, missing-data handling and multiplicity. The stated sample-size illustration assumptions concern two-arm planning, whereas the trial itself is listed as a single group. Keeping those planning assumptions separate from the actual study design avoids treating a hypothetical calculation as evidence about observed power or as the trial’s own design rationale.

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 registry entry leaves several trial-specific details unresolved. It does not report dose levels, the dose-escalation procedure, cohort sizes, or how the two study parts were organized. These details would help readers understand which exposure levels were studied and how safety information may have accumulated, but they cannot be reconstructed from the listed weekly administration schedule.

The primary safety and tolerability endpoint is defined in terms of participant counts, yet its results are not reported in the registry record. The listed adverse-event figures do not provide all of the primary outcome counts, and no endpoint-specific estimates or confidence intervals are reported. The registry lists 0 analyses and does not describe statistical methods for the outcomes.

The record names the safety population but does not define it or state how many participants were included in each analysis. It does not report missing-data handling, individual follow-up, event-level detail, or causal assessments. Although the adverse-event timeframe says assessment continued until progression, which was in average 3 months, the record does not provide participant-specific observation times.

The study is open-label and single-group, with no listed control. This limits causal interpretation and leaves no concurrent basis for comparison. The status is terminated, but the reason is not reported in the registry record. These gaps restrict interpretation to the registered design and the adverse-event counts and terms actually listed; they do not establish why the study stopped or what unreported outcomes would have shown.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Phase 1/2 Study to Evaluate Safety, PK and Efficacy of the MYC-Inhibitor OMO-103 in Solid Tumours — NCT04808362.
• Registry record last updated 2024-04-25; status last verified 2024-04.
• Results first posted 2024-04-25.
• 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

NCT04808362 is a Phase 1/2, open-label, single-group study of OMO-103 in patients with advanced solid tumours. The registry describes it as a two-part, first-in-human dose-finding study intended to evaluate safety, tolerability, PK, PD and proof-of-concept. OMO-103 was administered intravenously as a 30 min infusion once weekly. Enrollment is listed as 22; the study started on 2021-04-28, primary completion is listed as 2022-12-15, and status is terminated.

The primary endpoint concerns the number of participants with DLTs, IRRs, adverse events and serious adverse events. A secondary outcome is elimination half-life (t1/2). The record lists 22 participants at risk and 9 affected for serious adverse events, 22 at risk and 22 affected for other adverse events, and 3 deaths. It does not report primary endpoint results, analyses, the termination reason or a statistical analysis plan.