A Study to Evaluate Safety, Tolerability and Preliminary Efficacy of FP-1305 in Cancer Patients (MATINS): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study to Evaluate Safety, Tolerability and Preliminary Efficacy of FP-1305 in Cancer Patients (MATINS) (NCT03733990), a non-randomized phase 1, phase 2 study. The primary outcome is Dose Limiting Toxicities (DLT) in the Trial Subjects..

2. Clinical Question

This is a dose-finding question, rather than a conventional treatment-versus-control question. The study description frames FP-1305 as a first-in-human investigation and says the work was intended to assess safety, tolerability, and preliminary efficacy. The primary endpoint focuses specifically on dose-limiting toxicities (DLTs), which are used in early-phase development to inform whether doses can be considered tolerable for further study.

The registry identifies the condition broadly as cancer and gives a list of advanced tumour types in the summary. It does not provide a detailed primary endpoint population definition. The registry record also does not report a primary endpoint result, a selected tolerable dose, or an efficacy comparison. Consequently, the stated question can be described from the protocol summary, but the answer to that question cannot be established from the posted results in this record.

3. Trial Design

MATINS is registered as a Phase I/II, open-label, non-randomized study with sequential allocation. Its official title describes a three-part dose-finding trial and a separate cohort expansion. The registry lists enrollment of 216 and status as completed. The recorded study start is 2018-12-03, and primary completion is dated 2023-09-06.

The intervention is FP-1305, also named bexmarilimab. Listed dose groups are 0.1 mg/kg, 0.3 mg/kg, 1 mg/kg, 3 mg/kg, 10 mg/kg, and 30 mg/kg. The descriptions indicate that 0.3 mg/kg was administered at Q3W intervals; the 0.1 mg/kg group used three-week intervals; and the 1 mg/kg, 3 mg/kg, 10 mg/kg, and 30 mg/kg groups could use Q3W, Q2W, or Q1W intervals. The record associates different doses and schedules with different parts of the study. It does not give a complete account of how participants were assigned among these schedules.

Because the study was non-randomized and unmasked, dose groups do not function as randomized treatment and control arms. Dose escalation and cohort expansion serve distinct purposes: dose-finding assesses tolerability across dose levels, while expansion can gather additional information in selected cohorts. The registry title identifies these components, but detailed allocation rules and the operational transition between parts are not reported in the registry record.

The record does not report a concurrent comparator, a masking procedure, or a conventional between-group efficacy hypothesis. It also does not report the rationale for the enrollment target, the intended number of participants per dose group, or detailed eligibility criteria beyond the advanced cancer context in the summary. The registered design therefore supports interpretation as an early-phase safety and dose-finding investigation, not as a randomized estimate of comparative treatment effect.

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.

Only the first 6 of 7 groups are shown.

6. Primary Endpoint

The registry lists the primary outcome as Dose Limiting Toxicities (DLT) in the Trial Subjects., measured over Up to one year.

The primary outcome is the number of dose-limiting toxicities in trial subjects, with a timeframe of up to one year. The registry describes tolerable dose or doses as being determined using TITE-CRM, based on whether DLTs occur. The endpoint is therefore intended to guide dose selection rather than to measure a comparative clinical benefit against a control group.

In general, a DLT endpoint is used in early-phase studies because unacceptable toxicity can limit the dose considered suitable for further investigation. A count records how many DLTs are observed, while the dose-finding procedure uses the observed occurrence or non-occurrence to inform which dose level may be tolerable. This is a general description of the role of a DLT endpoint; the registry record does not give the trial-specific DLT definition, severity grading rules, attribution criteria, or rules for handling events with uncertain timing.

The record states a timeframe of up to one year but does not report the observed primary endpoint count, the number of participants evaluable for DLT, or the dose determination made by TITE-CRM. It also does not report a numerical estimate of tolerability by dose. The safety event counts posted for the dose groups should not be substituted for the primary DLT result: the registry does not state that those adverse event counts are the DLT endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registered primary endpoint is a count of DLTs, and the stated dose-selection method is TITE-CRM. TITE-CRM stands for a time-to-event version of the continual reassessment method. In general, the continual reassessment method is a model-based approach used in dose-finding: observed toxicity information at dose levels informs estimates of how toxicity relates to dose, and those estimates can guide dose selection. The time-to-event extension is designed for settings in which the toxicity outcome may take time to occur and participants may have different amounts of follow-up at a given analysis.

This general description does not establish the exact implementation in MATINS. The registry record does not report the model form, prior assumptions, target DLT probability, dose-assignment algorithm, decision thresholds, or rules for pausing or stopping escalation. It says only that tolerable dose or doses would be determined by TITE-CRM based on the occurrence or non-occurrence of DLTs. The DLT definition itself is not reported in the registry record, so the clinical events counted for the primary outcome cannot be independently identified from this information.

In general, a time-to-event dose-finding method can make use of partial follow-up rather than treating every participant whose follow-up is incomplete as if the outcome were already known. That matters because a DLT might occur after an initial observation period. The actual follow-up model, event window, censoring rules, and treatment of participants who discontinue are not stated in this record. The specified primary timeframe is up to one year, but the record does not indicate whether all participants were observed for that entire timeframe or how incomplete follow-up was reflected in the dose-finding analysis.

The design is sequential and non-randomized. Sequential allocation means dose decisions and later enrollment can depend on the stage of dose-finding; this is a general feature of sequential dose-escalation designs, not a description of undocumented operational rules in this particular trial. Non-randomized assignment also means that dose groups may differ in participant characteristics or study timing. Such differences limit direct comparisons between their observed event counts. Open-label status means the registry lists no masking; the record does not describe additional procedures to reduce assessment bias.

For a randomized comparison, an analysis might estimate a between-group contrast under an allocation scheme intended to make groups comparable. That is not the question answered by this study design. Here, the key statistical aim is dose selection from toxicity data, and the primary result should be interpreted in relation to the dose-finding method and its decision criteria. Since the record does not give the analysis result or those criteria, the registered method can be described, but the selected dose and the uncertainty of that selection cannot be reconstructed.

The registry lists four outcome measures but does not present statistical analysis results. No primary endpoint estimate, standard error, confidence interval, test statistic, or p-value is reported. These are not necessarily required to present a dose-finding conclusion, but without the outcome and decision result, the record does not allow readers to assess how the observed data informed tolerability. Statistical significance testing is also not the main framework implied by the stated TITE-CRM dose-finding objective.

In general, over-dispersion occurs when outcome variability is greater than a chosen statistical model expects. It can make standard errors too small and confidence intervals too narrow if the model does not account for that extra variation. This is a general concept; the registered primary outcome is a toxicity count, but the record does not state that an over-dispersion model was used or report data from which it could be assessed. The appropriate interpretation is not to assume an over-dispersion issue, but to note that the model specification and diagnostic information are not reported in the registry record.

Sample-size illustrations based on observed effects answer a different question from the trial's original dose-finding design. In general, such calculations can show the number of participants per arm that a conventional comparison might require under stated assumptions, but they are planning illustrations rather than observed power. The registry does not report a randomized comparison, and its primary outcome is not reduced to one comparison. Any planning calculation based on an observed effect would therefore not explain the original enrollment target or establish the power of the completed dose-finding study. The record does not report a sample-size justification or the trial's own design assumptions.

Finally, the event summaries should be distinguished from the primary endpoint analysis. Adverse event counts by dose group are descriptive safety information, whereas the primary endpoint is specifically DLTs used in a dose decision. Unless the registry states that an event meets the DLT definition and was included in the model, it cannot be treated as a DLT merely because it appears in an adverse event summary. The record does not provide the mapping between those summaries and the primary dose-finding dataset.

10. Multiplicity, Missing Data and Analysis Populations

The record lists four outcome measures, but the primary outcome described in the available information is DLT count up to one year. The other outcome definitions and their result details are not reported in the registry record. It is therefore not possible to determine which outcomes were designated as key secondary measures, how they were ordered, or whether any outcome was adjusted for multiple testing.

Multiplicity arises when several outcomes, subgroups, doses, or comparisons are tested. In general, conducting many statistical tests can increase the chance of finding an apparently notable result by chance. A prespecified hierarchy or adjustment can address some multiplicity concerns, but the registry record does not report a multiplicity strategy, an inferential testing plan, or analysis results. The dose-finding design also differs from a single confirmatory comparison, so the intended interpretation of multiple dose-level results would need to be understood from the protocol and analysis plan, which are not reported in the registry record.

Missing-data procedures are not reported in the registry record. This includes the handling of incomplete follow-up for the up-to-one-year DLT timeframe, withdrawal, missing outcome assessments, and participants who could not be classified for DLT. These details matter because TITE-CRM is a time-to-event method, and the amount and timing of follow-up can affect how observed and not-yet-observed outcomes are interpreted.

Analysis populations are also not reported. The record does not identify which participants were included in the primary dose-finding analysis, how evaluability was defined, or whether the safety summaries use the same population. No analysis results are listed. As a result, the registry record does not support a full assessment of outcome multiplicity, missingness, or the relationship between enrolled participants and those analyzed for the primary question.

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: ascites (40.0%), pyrexia (20.0%), general physical health deterioration (20.0%), infected seroma (20.0%), adrenocortical insufficiency acute (11.1%).

The registry reports serious adverse event counts, other adverse event counts, and deaths for each listed dose group. For 0.3 mg/kg, it lists 1 participant affected by serious adverse events among 13 at risk, 13 affected by other adverse events among 13 at risk, and 0 deaths. For 1 mg/kg, the corresponding figures are 57 among 130, 122 among 130, and 12 deaths. For 3 mg/kg, they are 13 among 41, 38 among 41, and 3 deaths. For 10 mg/kg, they are 10 among 18, 17 among 18, and 3 deaths. For 0.1 mg/kg, they are 3 among 5, 5 among 5, and 0 deaths. For 30 mg/kg, they are 4 among 9, 9 among 9, and 0 deaths.

These are descriptive group-level figures, not randomized comparisons. The registry record does not report the event attribution, event-level timing, exposure duration, or whether the same individuals experienced multiple events. A count of participants affected does not indicate how many separate events occurred. Deaths are listed by group, but the record does not establish their cause or relationship to study treatment.

The listed serious adverse event terms include Ascites (40.0%), Pyrexia (20.0%), General physical health deterioration (20.0%), Infected seroma (20.0%), Adrenocortical insufficiency acute (11.1%), and Intestinal obstruction (11.1%). The record does not give the denominator or further context for these percentages in the information reported. They should be read as registry-listed terms, not as causal findings or as rates suitable for comparing doses.

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

The registry record establishes that MATINS was completed as a non-randomized, open-label Phase I/II study, with a dose-finding component and separate cohort expansion described in the official title. Its registered primary objective was to determine tolerable dose or doses based on DLT occurrence, using TITE-CRM. The study therefore addresses an early-phase dose-selection question rather than a randomized comparative efficacy question.

The posted record does not report the primary DLT result, the dose or doses selected as tolerable, or a statistical analysis result. It also does not report an efficacy estimate. The registry summary describes preliminary efficacy as an objective, but that statement alone is not evidence of an observed benefit. Without outcome estimates and their precision, the record cannot establish the size or reliability of an efficacy signal.

The adverse event figures describe the participants affected within each dose group, along with deaths listed by group. They are useful for understanding what safety information the registry posted, but they do not establish that FP-1305 caused an event. Nor do differences among the group counts establish that one dose is safer or less safe: the groups were not formed by random allocation, and the registry does not provide the detail needed to account for follow-up, exposure, or participant differences.

More generally, a completed study status does not itself mean that its primary question has been answered in the registry record. Completion is a status label, while an interpretable statistical conclusion depends on reported outcome data, analysis methods, and uncertainty. The record supports a description of study design and selected adverse event summaries; it does not support a definitive dose-selection conclusion or a treatment-effect estimate.

This distinction is important for early-phase research. A dose-finding study can inform the design of later research without establishing comparative clinical benefit. The information in the registry should therefore be understood as a record of the study's stated aims, design, and posted safety counts, with the primary result and its statistical precision not reported in the registry record.

13. Why This Trial Matters Statistically

MATINS is statistically important as an example of a study whose primary question is dose selection rather than a treatment effect against a control. In early-phase research, choosing a dose requires balancing observed toxicity information with the need to learn about doses under investigation. A method such as TITE-CRM is intended to use dose and time-to-event toxicity information to inform that process. The registry identifies this method, making clear that the primary statistical target is tolerability.

The design also illustrates why dose groups in a non-randomized trial should not be interpreted like randomized arms. Sequential dose-finding can produce groups observed at different stages and under different dosing schedules. Observed counts can describe what was recorded in each group, but they do not by themselves isolate the effect of dose. This is particularly relevant when the registry lists several doses and schedules and provides no concurrent randomized control group.

The record makes a further statistical distinction visible: safety summaries and the primary DLT endpoint are not interchangeable. A serious adverse event listing may help describe safety experience, but the primary dose-finding result depends on the trial's DLT definition and analysis procedure. Those details and the resulting dose decision are not reported in the registry record. Readers should not infer the dose-finding conclusion from adverse event counts alone.

Finally, the study highlights the importance of complete results reporting. The registry marks the trial completed and describes its intended method, yet does not report the primary endpoint result or an efficacy estimate in the information available. Without those results, the statistical contribution that can be assessed is limited to the design description and posted adverse event counts. Reporting outcome estimates, analysis populations, follow-up, and uncertainty would be necessary to evaluate how strongly the study answers its planned questions.

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.

Several trial-specific limitations affect interpretation of the registry record. First, the primary DLT result is not reported, even though DLTs were the basis for the stated tolerable-dose determination. The record does not report the DLT definition, evaluable population, model specifications, decision criteria, or dose conclusion. Thus, the key dose-finding result cannot be assessed from the information posted.

Second, the study is non-randomized and has no concurrent randomized comparator. Dose groups should not be treated as equivalent groups differing only by dose. The listed groups also include different dosing intervals and correspond to different study parts. The record does not provide enough detail to separate the effects of dose, schedule, study stage, or participant characteristics.

Third, the registry does not report an efficacy analysis, confidence intervals, or other precision measures. The description of preliminary efficacy as an objective does not provide an estimate of efficacy. The record also does not report how missing data, incomplete follow-up, or analysis populations were handled. These omissions limit assessment of both outcome completeness and uncertainty.

Finally, safety summaries do not provide event-level context, attribution, duration of exposure, or detailed follow-up. The serious adverse event percentages are listed without the denominator context in the available record. Group counts and death counts are descriptive and cannot establish causality or comparative safety. These limitations apply to what can be concluded from the registry record; they do not establish what analyses may have been conducted outside the information reported there.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Study to Evaluate Safety, Tolerability and Preliminary Efficacy of FP-1305 in Cancer Patients (MATINS) — NCT03733990.
• Registry record last updated 2025-04-02; status last verified 2025-03.
• Results first posted 2025-04-02.
• 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

MATINS (NCT03733990) is a completed Phase I/II, open-label, non-randomized study of FP-1305 (bexmarilimab) in advanced solid tumours. The official title describes three study parts, including dose-finding and separate cohort expansion. The registry lists enrollment of 216, a start date of 2018-12-03, and primary completion on 2023-09-06.

The primary outcome was the number of DLTs up to one year, with tolerable doses to be determined using TITE-CRM. The registry record does not report the primary endpoint result, the selected dose, or an efficacy estimate. It posts serious and other adverse event counts and deaths for dose groups, but these are descriptive findings from a non-randomized study and do not provide a controlled comparison or establish causation.