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 PCI Treatment/Gemcitabine & Chemotherapy vs Chemotherapy Alone in Patients With Inoperable Extrahepatic Bile Duct Cancer (NCT04099888), a randomized phase 2 study. The primary outcome is Progression-free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04099888 |
| Phase | Phase 2 |
| Condition | Cholangiocarcinoma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 41 |
| Arms in the primary outcome | 2 |
| Lead sponsor | PCI Biotech AS |
| Results posted | Yes |
2. Clinical Question
Population
Participants with inoperable cholangiocarcinoma, described in the registry record as extrahepatic bile duct cancer. The trial enrolled 41 participants. The primary endpoint analysis used a modified intent-to-treat population: randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline.
Intervention
Fimaporfin-induced photochemical internalisation of gemcitabine, complemented by systemic gemcitabine/cisplatin chemotherapy. The registry describes study treatment for 6 months, followed by assessments every 3 months, as applicable.
Comparator
Gemcitabine/cisplatin chemotherapy alone, described as standard of care. The study was randomized, open-label, and used a parallel-group design.
Primary question
How did progression-free survival compare between the two treatment groups? PFS was measured from randomisation to objective disease progression or death, whichever came first, with the primary endpoint assessed up to 18 months.
The clinical question is comparative: whether the observed time until progression or death differed between participants assigned to the PCI-containing regimen and those assigned to chemotherapy alone. The registry reports median PFS for each group, with a 95% confidence interval for each median. Those summaries describe the observed time-to-event experience, but they do not by themselves quantify a relative treatment effect across the full follow-up period.
The registry also identifies overall survival, response, tumor-size change, disease control, biliary events, and adverse events among the secondary outcomes. These outcomes can provide context about survival, tumor response, disease course, and safety, but they are distinct questions from the primary PFS comparison. The registry record does not report a numerical sponsor analysis comparing the groups for the primary endpoint.
3. Trial Design
This was a randomized, open-label, parallel-group phase 2 study sponsored by PCI Biotech AS. One group received fimaporfin-induced photochemical internalisation of gemcitabine alongside gemcitabine/cisplatin chemotherapy; the comparator group received gemcitabine/cisplatin chemotherapy alone. The registry describes the study as multicenter and its official title identifies it as a safety, tolerability, and efficacy study in people with inoperable cholangiocarcinoma.
The trial enrolled 41 participants. For the primary endpoint, the registry reports 19 participants in the PCI plus standard-of-care group and 15 in the standard-of-care group in the modified intent-to-treat analysis set. That set included randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline. The registry does not report why the primary analysis set contains fewer participants than the enrollment total.
The registry gives a treatment period of 6 months, followed by assessments every 3 months, as applicable. The primary PFS timeframe was up to 18 months. The secondary overall-survival timeframe was up to 24 months, while the registry lists several other secondary outcomes with timeframes up to 18 months.
The study status is terminated. The registry lists a start date of 2019-05-23 and a primary completion date of 2022-04-28. The reason for termination, the planned enrollment target, and details about any changes to the trial plan are not reported in the registry record. Randomization helps create groups for a comparative study, while open-label conduct means treatment assignment was not masked. The registry record does not describe additional procedures to limit assessment or analysis bias.
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: Of the 41 enrolled participants, all met inclusion criteria and were randomized and included in the intent-to-great (ITT) analysis set.
Overall Study
| Stage | PCI Treatment in Conjunction With Standard of Care (SoC) | Standard of Care (SoC) |
|---|---|---|
| STARTED | 21 | 20 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 21 | 20 |
Reasons for not completing this period:
| Reason | PCI Treatment in Conjunction With Standard of Care (SoC) | Standard of Care (SoC) |
|---|---|---|
| Study terminated by sponsor | 9 | 11 |
| Lost to Follow-up | 1 | 1 |
| Withdrawal by Subject | 5 | 6 |
| Death | 5 | 2 |
| Other | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Modified intent-to-treat (mITT) analysis set (all randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline).
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: Intent-to-treat (ITT) Analysis Set
| Characteristic | Unit | PCI Treatment in Conjunction With Standard of Care (SoC) | Standard of Care (SoC) | Total |
|---|---|---|---|---|
| Participants analysed | 21 | 20 | 41 | |
| Age, Continuous | years | 68.1 (SD 7.30) | 64.4 (SD 11.43) | 66.3 (SD 9.61) |
| Age, Customized: <50 years of age | Participants | 0 | 2 | 2 |
| Age, Customized: 50 to <65 years of age | Participants | 6 | 7 | 13 |
| Age, Customized: >=65 years of age | Participants | 15 | 11 | 26 |
| Sex: Female, Male: Female | Participants | 7 | 11 | 18 |
| Sex: Female, Male: Male | Participants | 14 | 9 | 23 |
| Race/Ethnicity, Customized: White | Participants | 11 | 11 | 22 |
| Race/Ethnicity, Customized: African American | Participants | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Asian | Participants | 10 | 9 | 19 |
| Race/Ethnicity, Customized: Other | Participants | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Hispanic or Latino | Participants | 1 | 1 | 2 |
| Race/Ethnicity, Customized: Not Hispanic or Latino | Participants | 20 | 19 | 39 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival (PFS), measured over Up to 18 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | Months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival measures the time from randomisation until objective disease progression or death, whichever occurs first. The registry expresses this endpoint in months and specifies assessment up to 18 months. It uses a modified intent-to-treat analysis set: randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline.
PFS is a time-to-event endpoint. Unlike a simple count of participants with an event, it accounts for when progression or death occurs during follow-up. A median summarizes the time by which half of the analyzed group has experienced the event, subject to the endpoint and analysis procedures. The registry reports median PFS of 7.29 months in the PCI plus standard-of-care group, with a 95% confidence interval from 6.0 to 12.0 months. For standard of care alone, it reports 8.08 months, also with a 95% confidence interval from 6.0 to 12.0 months.
Researchers use PFS to describe disease control over time without waiting for death as the only event. It is not interchangeable with overall survival, which counts death, or with response outcomes, which describe tumor response. The registry record does not report the sponsor’s numerical between-group hazard ratio, confidence interval, or p-value for PFS. The two group medians therefore describe the posted results, but do not establish the size or precision of a relative treatment effect.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary endpoint results are medians of 7.29 months for PCI plus standard of care and 8.08 months for standard of care alone. Each median has the same reported 95% confidence interval, 6.0 to 12.0 months. These are group-level summaries of time to progression or death in the modified intent-to-treat analysis set, not estimates of the proportion of participants who benefited or of an individual participant’s expected outcome.
The registry record does not report a numerical sponsor comparison for PFS. In particular, it does not provide the hazard ratio, its confidence interval, or a p-value. A comparison of medians alone is not a substitute for those analyses: time-to-event comparisons use information across follow-up, including event timing, and may incorporate censoring. No unadjusted recalculation is presented for this endpoint because values needed to perform the specified two-arm comparison are missing or not estimable from the posted median summaries.
The secondary outcomes offer additional descriptive context, but are not a replacement for a missing primary comparison. The posted secondary results include counts for overall survival and objective response, and means for change in tumor size. Simple unadjusted recalculations give a risk-difference estimate of 0.235 for overall survival, with a 95% confidence interval from -0.042 to 0.512 and p = 0.123. For objective response rate, the estimate is 0.042, with a 95% confidence interval from -0.218 to 0.303 and p = 0.749. For mean change in tumor size, the recalculated difference is -2.67 percentage points, with a 95% confidence interval from -19.13 to 13.79 and p = 0.751. These are unadjusted recalculations from posted summary statistics, not reported sponsor estimates.
Each of those secondary confidence intervals includes the value corresponding to no difference on its respective scale. The estimates are also imprecise, as indicated by the breadth of their intervals. The p-values do not establish that the groups are equivalent, nor do they resolve the uncertainty in the estimates. In particular, a non-small p-value is not proof that there is no effect; it indicates that the analysis did not provide strong evidence against the tested no-difference value under its assumptions.
The trial enrolled 41 participants, and the primary analysis set includes 19 and 15 participants by group. With a small study population, estimates can be unstable and confidence intervals can remain broad. The record’s terminated status is also relevant context, but the reason for termination and whether it affected the intended analysis are not reported in the registry record. The posted results support a description of the observed groups, while leaving the comparative primary PFS effect unquantified in the registry record.
9. Statistical Methods Explained
Interpreting the posted results requires distinguishing a group summary from a formal comparison, and a registry analysis from an unadjusted recalculation. The primary endpoint is time-to-event, whereas several secondary outcomes are counts or continuous measurements. Each type of outcome has a different scale and calls for different methods.
What does a median and its confidence interval describe?
The PFS median is a time summary: it locates the point in the observed time-to-event distribution at which the event experience reaches the median. A confidence interval expresses uncertainty around an estimated quantity under the analysis method. It is not a range containing every participant’s PFS time, nor does overlap or non-overlap of separate group intervals provide a complete formal test of the treatment contrast.
The registry reports a 95% confidence interval for each median, but no sponsor hazard ratio or confidence interval for the comparison. A hazard ratio is a relative measure of event rates over follow-up under the model used for the analysis. Its interpretation depends on the model and assumptions, and it is not derivable from the two medians alone. The record does not report the method used for the primary comparison. The median-only endpoint was not recalculated because the values needed for a two-arm comparison are missing or not estimable from the posted summaries.
How should the unadjusted binary comparisons be read?
For overall survival and objective response rate, participants are counted as having or not having the outcome over the stated timeframe. A risk difference describes a contrast between the observed proportions in the two groups, on an absolute scale. Its confidence interval communicates uncertainty around that contrast. If the interval includes zero, the data are compatible with no difference as well as with values in either direction; this does not demonstrate that the groups are equivalent.
The reported recalculations use posted participant counts and are unadjusted. For overall survival, the estimate is 0.235, the 95% confidence interval is -0.042 to 0.512, and p = 0.123. For objective response rate, the estimate is 0.042, the interval is -0.218 to 0.303, and p = 0.749. These recalculations do not account for any covariates or multiplicity adjustments. The registry record does not give a numerical sponsor comparison for either outcome, despite identifying a superiority analysis.
What does the continuous outcome comparison add?
Change in tumor size is reported as a mean percentage change and standard deviation for each group. A mean summarizes the average observed change; the standard deviation describes variability among participant measurements. The recalculated unadjusted mean difference is -2.67, with a 95% confidence interval from -19.13 to 13.79 and p = 0.751. The wide interval reflects considerable uncertainty in the contrast. The registry record does not report a numerical sponsor comparison or its method for this outcome.
Over-dispersion is a general concern when count data vary more than a chosen statistical model expects. It can make standard errors too small if the extra variability is ignored. That issue is relevant in general to some count outcomes, but the listed recalculations concern binary proportions and a continuous measure; the registry record does not report an over-dispersion assessment or a statistical model for the primary PFS result. No such analysis should be inferred.
How should the planning illustrations be understood?
Planning illustrations use the observed secondary-outcome estimates as reference effects and show how hypothetical sample sizes change when the assumed effect is smaller. For overall survival, the listed scenarios range from 53 participants per arm for 80% power and 70 for 90% power when planning for 100% of the observed effect, to 242 and 324 when planning for 50% of that effect. For objective response, the corresponding listed values range from 1,300 and 1,740 to 4,960 and 6,639. For change in tumor size, they range from 1,291 and 1,728 to 5,161 and 6,909. The intermediate scenario plans for 75% of each observed effect.
These are planning illustrations, not observed power and not the trial’s own sample-size justification. The stated assumptions are a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The registry record does not report the trial’s actual sample-size assumptions or justification. Event-based planning for time-to-event outcomes would concern total events, but the posted primary PFS comparison does not include a sponsor hazard ratio on which to base such a calculation.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple secondary outcomes in addition to the primary PFS endpoint. When many outcomes are examined, the chance of at least one apparently unusual result can increase even if there is no true difference for any outcome. A prespecified testing hierarchy or adjustment procedure can help control this issue, but the multiplicity strategy is not reported in the registry record. The record also does not describe whether secondary analyses were intended to be confirmatory or exploratory. The recalculated p-values for the posted secondary summaries are unadjusted, so they should not be interpreted as accounting for the full set of outcomes.
Missing-data handling is also not reported in the registry record. The primary analysis population is defined as randomized participants who received at least 1 dose of study treatment and had a RECIST assessment at baseline. The record gives 19 and 15 participants in that analysis set, while overall enrollment was 41. It does not explain the difference between enrollment and the analysis population, report the reasons for exclusion, or describe how missing follow-up, censoring, or incomplete assessments were handled.
These details matter because the analysis population and missing-data procedures can affect which participants contribute to an estimate and how uncertainty is assessed. For a time-to-event endpoint, censoring rules and follow-up completeness are especially important. The registry record does not report the censoring details or the sponsor’s method for comparing PFS. It also does not report the analysis method for the secondary superiority comparisons, or the sponsor’s numerical comparison results for the primary and listed secondary endpoints.
The study is marked terminated, but the reason for termination and its implications for the planned analyses are not reported in the registry record. The registry also does not report interim analyses, a statistical analysis plan, or changes to the analysis plan. Without those details, readers cannot determine whether the posted analyses followed a prespecified strategy or how unreported outcomes and missing observations were handled.
11. 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 |
|---|---|---|---|
| PCI Treatment in Conjunction With Standard of Care (SoC) | 12 | 18 | 66.7% |
| Standard of Care (SoC) | 8 | 16 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| PCI Treatment in Conjunction With Standard of Care (SoC) | 7 of 19 |
| Standard of Care (SoC) | 2 of 15 |
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 |
|---|---|
| PCI Treatment in Conjunction With Standard of Care (SoC) | 18 of 18 |
| Standard of Care (SoC) | 16 of 16 |
Most frequent serious adverse event terms
| Event term | Organ system | PCI Treatment in Conjunction With Standard of Care (SoC) | Standard of Care (SoC) |
|---|---|---|---|
| Cholangitis | Hepatobiliary disorders | 6 of 18 | 3 of 16 |
| Stent malfunction | Product Issues | 2 of 18 | 1 of 16 |
| Pyrexia | General disorders | 1 of 18 | 1 of 16 |
| Cholangitis acute | Hepatobiliary disorders | 0 of 18 | 2 of 16 |
| Biliary tract infection | Infections and infestations | 1 of 18 | 1 of 16 |
| Duodenal ulcer | Gastrointestinal disorders | 1 of 18 | 0 of 16 |
| Pancreatitis acute | Gastrointestinal disorders | 1 of 18 | 0 of 16 |
| Aspartate aminotransferase increased | Investigations | 1 of 18 | 0 of 16 |
| Transaminases increased | Investigations | 1 of 18 | 0 of 16 |
| Biliary obstruction | Hepatobiliary disorders | 1 of 18 | 0 of 16 |
Most frequent non-serious adverse event terms
| Event term | Organ system | PCI Treatment in Conjunction With Standard of Care (SoC) | Standard of Care (SoC) |
|---|---|---|---|
| Gastrointestinal disorders | Gastrointestinal disorders | 10 of 18 | 12 of 16 |
| General disorders and administration site conditions | General disorders | 9 of 18 | 8 of 16 |
| Skin and subcutaneous tissue disorders | Skin and subcutaneous tissue disorders | 8 of 18 | 3 of 16 |
| Metabolism and nutrition disorders | Metabolism and nutrition disorders | 3 of 18 | 6 of 16 |
| Investigations | Investigations | 6 of 18 | 1 of 16 |
| Hepatobiliary disorders | Hepatobiliary disorders | 4 of 18 | 3 of 16 |
| Infections and infestations | Infections and infestations | 5 of 18 | 1 of 16 |
| Nervous system disorders | Nervous system disorders | 6 of 18 | 0 of 16 |
| Blood and lymphatic system disorders | Blood and lymphatic system disorders | 1 of 18 | 3 of 16 |
| Injury, poisoning and procedural complications | Injury, poisoning and procedural complications | 4 of 18 | 0 of 16 |
Serious events with the highest arm-level rate: cholangitis (33.3%), cholangitis acute (12.5%), stent malfunction (11.1%), pyrexia (6.2%), cholecystitis (6.2%).
The registry reports serious adverse events among 12 of 18 participants in the PCI plus standard-of-care group and 8 of 16 in the standard-of-care group. It also reports other adverse events among 18 of 18 and 16 of 16 participants, respectively. Death counts are 7 and 2. These are descriptive counts for the registry’s safety groups; they do not establish that an event or death was caused by a study treatment. The registry record does not provide a causal attribution for these figures.
The serious adverse event terms listed most often include cholangitis at 33.3%, acute cholangitis at 12.5%, and stent malfunction at 11.1%. Pyrexia, cholecystitis, and biliary tract infection are each listed at 6.2%. These percentages describe the registry’s listed terms and should not be read as comparative treatment effects. The record does not provide a formal statistical comparison of serious adverse events, nor does it explain the denominator or event-counting rules for each listed term in the information.
Safety counts can be affected by exposure time, event definitions, reporting practices, and the number of participants followed. The registry describes a treatment period of 6 months, but detailed exposure-adjusted rates and a statistical analysis plan for safety are not reported in the registry record. The counts are therefore useful as descriptive context, not as proof of a difference in risk or of treatment causation.
12. What the Result Does, and Does Not, Mean
The posted primary results describe median PFS of 7.29 months in the PCI plus standard-of-care group and 8.08 months in the standard-of-care group, with a 95% confidence interval from 6.0 to 12.0 months for each median. These estimates characterize the observed groups in the modified intent-to-treat analysis set. The registry does not report the sponsor’s numerical comparative PFS estimate or its uncertainty, so the record does not quantify the relative effect between groups.
The results do not establish that the treatments are equivalent. Similar-looking medians, or overlapping confidence intervals, are not an equivalence analysis. Demonstrating equivalence or noninferiority generally requires a prespecified margin and an analysis designed for that purpose; such details are not reported in the registry record. Likewise, the absence of a reported PFS p-value is not evidence that the groups are the same.
The secondary recalculations are also uncertain and unadjusted. Their confidence intervals include no difference, but that does not establish absence of an effect. The available participant counts and estimates cannot show what would have been observed with a larger study or different follow-up. The planning illustrations are hypothetical sample-size scenarios based on observed secondary effects; they are not observed power, and they are not evidence about the study’s original design rationale.
The safety counts describe reported events in the registry’s safety groups. They do not determine whether an event was caused by treatment, and they do not by themselves support a comparative safety conclusion. The record does not report the reason for termination, missing-data methods, or the full statistical analysis strategy. Those omissions limit interpretation of how the posted findings relate to the original analysis plan.
Overall, the registry record supports a careful description of the recorded outcomes and their uncertainty. It does not support a definitive claim about the comparative effect on PFS, prove equivalence, or establish that one regimen is safer or more effective. These are statistical limits of the posted results, not advice about treatment decisions.
13. Why This Trial Matters Statistically
This trial illustrates why reporting a group median is not the same as reporting a comparative time-to-event analysis. The primary endpoint is PFS, but the registry record gives medians and confidence intervals without a numerical sponsor hazard ratio, confidence interval for the contrast, or p-value. Readers can describe the observed medians, yet cannot assess the registry’s formal estimate of the relative event experience across follow-up.
The distinction matters in a small randomized phase 2 study. The trial enrolled 41 participants, while the modified intent-to-treat primary analysis set includes 19 and 15 participants in the respective groups. Estimates from small groups can be imprecise, and exclusion from an analysis population can affect interpretation. The record does not explain why the enrollment total differs from the primary analysis counts or report missing-data procedures.
The secondary results demonstrate another important principle: point estimates need to be read alongside their uncertainty. Recalculated differences for the posted secondary outcomes have confidence intervals that include no difference. Those intervals permit a range of plausible contrasts, so the estimates should not be reduced to a binary statement based only on p-values. Because the recalculations are unadjusted and many outcomes are listed, multiplicity and exploratory interpretation also matter.
Finally, the sample-size illustrations show how planning needs depend on the effect a future study aims to detect, but they are not the trial’s design justification or observed power. The registry does not report its original sample-size assumptions. Together, these features make the record a useful example of the limits of interpreting outcome summaries when the comparative analysis, analysis plan, and missing-data methods are not reported.
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 limitations are specific to the posted record. The primary endpoint is reported as group medians with confidence intervals, but the sponsor’s comparative PFS estimate, confidence interval, p-value, and analysis method are not reported in the registry record. Because PFS is time-to-event, the medians alone cannot convey the full comparative pattern of events over follow-up or explain how censoring was handled.
The primary endpoint analysis includes 19 participants in the PCI plus standard-of-care group and 15 in the standard-of-care group, while total enrollment is 41. The registry defines the modified intent-to-treat set but does not report why participants are absent from that set or how missing assessments were handled. This prevents readers from judging the possible impact of exclusions or incomplete follow-up.
The trial is terminated, but the reason and its relationship to recruitment, follow-up, or analysis are not reported in the registry record. The registry also does not report the original sample-size justification, interim analyses, statistical analysis plan, or whether analysis methods changed. These omissions make it difficult to assess whether the posted analyses follow an intended confirmatory strategy.
Only selected secondary outcomes have numerical summaries in the information. The secondary recalculations are unadjusted and do not account for multiple outcome testing. The safety results are descriptive, and the record does not provide a formal between-group safety analysis or detailed event-counting rules. Finally, the open-label design means treatment assignment was not masked; the record does not describe measures used to limit potential assessment or analysis bias. These limitations do not invalidate the posted numbers, but they constrain the conclusions that can be drawn from them.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: PCI Treatment/Gemcitabine & Chemotherapy vs Chemotherapy Alone in Patients With Inoperable Extrahepatic Bile Duct Cancer — NCT04099888.
- Registry record last updated 2023-09-11; status last verified 2023-04.
- Results first posted 2023-09-11.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT04099888 is a randomized, open-label, parallel-group phase 2 study sponsored by PCI Biotech AS in people with inoperable cholangiocarcinoma. It compared fimaporfin-induced photochemical internalisation of gemcitabine plus gemcitabine/cisplatin with gemcitabine/cisplatin alone. The registry lists 41 enrolled participants and a terminated status.
The primary endpoint was PFS up to 18 months in a modified intent-to-treat analysis set. Median PFS was 7.29 months with PCI plus standard of care and 8.08 months with standard of care alone; both 95% confidence intervals were 6.0 to 12.0 months. A numerical sponsor comparison for PFS is not reported in the registry record. Selected secondary outcomes and safety counts are also posted, but the available secondary recalculations are unadjusted and uncertain.
The registry record does not report the reason for termination, the sample-size justification, the statistical analysis plan, multiplicity procedures, or missing-data methods. Those omissions limit assessment of the comparative results and the intended analysis strategy.