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 Pembrolizumab and BCG Solution in Treating Patients With Recurrent Non-Muscle-Invasive Bladder Cancer (NCT02808143), a na phase 1 study. The primary outcome is Maximum Tolerated Dose (MTD).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02808143 |
| Phase | Phase 1 |
| Condition | Recurrent Bladder Carcinoma, Stage 0a Bladder Urothelial Carcinoma, Stage 0is Bladder Urothelial Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Northwestern University |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled patients with high-risk or BCG-refractory non-muscle-invasive bladder cancer. The registry record lists recurrent bladder carcinoma and several bladder cancer stages among the conditions, including stage 0a, stage 0is, and stage I. Enrollment was 9. The record does not report further eligibility details or a fuller description of the primary-endpoint population.
Intervention
The intervention combined intravesical pembrolizumab with BCG solution. The arm description specifies pembrolizumab on day -14 before induction, then weekly BCG during weeks 0-5 with pembrolizumab at weeks 0, 2, and 4. It also names a maintenance phase, but the maintenance schedule is not reported in the registry record. Laboratory biomarker analysis and a pharmacological study are also listed.
Comparator
There is no separate control arm described. The study is listed as single-group, with allocation recorded as not applicable and no masking. The two phase 1 cohorts used different pembrolizumab dose levels alongside BCG, but they are not a like-for-like control comparison. Differences between these cohorts cannot, by themselves, establish what would have happened with a different treatment or without the combination.
Primary question
The primary question is what maximum tolerated dose of intravesical pembrolizumab can be identified when it is administered with BCG. The record defines the MTD by the occurrence of dose-limiting toxicities during follow-up of up to 9 weeks. It does not report the MTD result, the number of dose-limiting toxicities by cohort, or a conclusion about which dose met the definition. The study also lists tolerability, adverse events, and tumor characteristics associated with efficacy as interests, but the registry record does not report results for those questions.
3. Trial Design
This was a phase 1 dose-escalation study of intravesical pembrolizumab and BCG in patients with high-risk or BCG-refractory non-muscle-invasive bladder cancer. The registry lists a single-group model, no masking, and allocation as not applicable. Enrollment was 9, and the study status is completed. The listed dates are 2017-02-10 for study start and 2020-05-15 for primary completion.
The posted arm description identifies two cohorts: one with pembrolizumab at 1 mg/kg and BCG at 50 mg, and another with pembrolizumab at 2 mg/kg and BCG at 50 mg. These are dose cohorts within a dose-escalation study, not randomized treatment groups or an external control. Their sizes and adverse-event counts are described in the registry record, but a direct contrast between them would not have the protections of random allocation. The record does not report a statistical comparison between cohorts.
The treatment schedule includes a pre-induction pembrolizumab administration, an induction period with weekly BCG and pembrolizumab every 2 weeks, and a maintenance phase. The maintenance description is incomplete in the registry record, so its schedule cannot be characterized from the available entry. The record also lists laboratory biomarker analysis and pharmacological study interventions.
The primary endpoint is maximum tolerated dose, assessed over up to 9 weeks. Secondary outcomes include dose-limiting toxicities and the incidence of adverse events. The record lists 15 outcome measures, but detailed results and analysis methods for the outcomes beyond the named primary and secondary measures are not reported in the registry record. Sample-size justification, details of the dose-escalation decision process, and any interim-analysis plan are not reported in the registry record.
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.
Pre-Induction Period
| Stage | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BCG) |
|---|---|---|
| STARTED | 3 | 6 |
| COMPLETED | 3 | 6 |
| NOT COMPLETED | 0 | 0 |
Induction Period
| Stage | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BCG) |
|---|---|---|
| STARTED | 3 | 6 |
| COMPLETED | 3 | 6 |
| NOT COMPLETED | 0 | 0 |
Maintenance Phase
| Stage | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BCG) |
|---|---|---|
| STARTED | 3 | 6 |
| COMPLETED | 1 | 1 |
| NOT COMPLETED | 2 | 5 |
Reasons for not completing this period:
| Reason | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BCG) |
|---|---|---|
| Disease recurrence | 1 | 3 |
| Disease progression | 1 | 2 |
Survival Follow-Up
| Stage | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BCG) |
|---|---|---|
| STARTED | 3 | 6 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 3 | 6 |
Reasons for not completing this period:
| Reason | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BCG) |
|---|---|---|
| Still in survival Follow-up | 2 | 3 |
| Disease recurrence | 0 | 2 |
| Adverse Event | 0 | 1 |
| Death | 1 | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BGC) | Total |
|---|---|---|---|---|
| Participants analysed | 3 | 6 | 9 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 3 | 6 | 9 |
| Sex: Female, Male: Female | Participants | 0 | 1 | 1 |
| Sex: Female, Male: Male | Participants | 3 | 5 | 8 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 5 | 8 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): White | Participants | 2 | 4 | 6 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 1 |
| Cancer Stage at Baseline: TaHG | Participants | 0 | 2 | 2 |
| Cancer Stage at Baseline: TaHG + CIS | Participants | 1 | 1 | 2 |
| Cancer Stage at Baseline: Tis | Participants | 1 | 2 | 3 |
| Cancer Stage at Baseline: T1 | Participants | 1 | 0 | 1 |
| Cancer Stage at Baseline: T1HG | Participants | 0 | 1 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (MTD), measured over Up to 9 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg/kg Pembrolizumab |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is maximum tolerated dose, expressed as mg/kg of pembrolizumab. It asks which dose level meets the study's toxicity-based definition when pembrolizumab is given intravesically with BCG. The registry defines the MTD as the highest dose that causes dose-limiting toxicities in <2 of 6 patients, graded using the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.03. The endpoint timeframe is up to 9 weeks.
In general, an MTD endpoint is used in early-phase dose-escalation research to characterize tolerability and guide decisions about doses for further study. It does not directly measure comparative efficacy. The definition recorded for this study combines a dose level, a specified toxicity threshold, and a follow-up period; each part matters when interpreting the endpoint.
The registry record does not report the observed MTD, the dose-limiting toxicity counts used to determine it, or whether the stated definition was met at either cohort dose. Therefore, the endpoint's intended meaning can be described, but its result cannot be inferred from the listed enrollment or serious adverse-event figures. Serious adverse events are not interchangeable with dose-limiting toxicities: the endpoint uses a specified toxicity definition and grading process, while the registry's serious-event summary does not establish which events counted as DLTs.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed phase 1 dose-escalation study with enrollment of 9 and a primary endpoint of maximum tolerated dose. The record does not report the MTD result. It also does not report the dose-limiting toxicity counts by dose level, so the endpoint definition cannot be applied to determine a result from the information available. A completed study status is not itself evidence that a particular dose met the endpoint criterion.
The adverse-event summary separates results by cohort. For the cohort listed as 1 mg/kg pembrolizumab with 50 mg BCG, the record lists 3 at risk for serious adverse events and 2 affected; for the cohort listed as 2 mg/kg pembrolizumab with 50 mg BCG, it lists 6 at risk and 2 affected. The corresponding other-adverse-event counts are 3 at risk and 3 affected in the first cohort, and 6 at risk and 6 affected in the second. The registry also lists deaths of 1 and 3, respectively. These counts do not establish whether events were related to treatment, whether they met the DLT definition, or when they occurred. The record does not report those details.
The serious-event terms include Myocardial Infarctrion, Hematuria, and Sinus Bradycardia, each listed at 33.3%, and Autoimmune Disorder, Pelvic Infection, and Tremor, each listed at 16.7%. These are entries in the registry's serious-event summary, not a complete account of all possible clinical events or a treatment-effect estimate. The record does not clarify event overlap or provide additional context for interpreting the percentages.
Because there is no randomized comparator, the cohort summaries should not be read as a causal comparison of the dose levels. The cohorts may differ in ways not captured by the record, and their small enrollment limits how precisely event frequencies can characterize the wider patient population. No confidence intervals, hypothesis tests, or adjusted effect estimates are reported in the registry record.
The brief summary describes efficacy and tolerability as study aims and mentions tumor characteristics associated with efficacy. However, the posted results represented in the registry record do not give an efficacy estimate or biomarker result. The appropriate statistical conclusion is therefore limited: the record describes the study design and selected adverse-event counts, while leaving the primary MTD result and efficacy findings unreported.
9. Statistical Methods Explained
Interpreting a phase 1 dose-escalation study requires separating the design question from a comparative treatment-effect question. The design question concerns the maximum tolerated dose of intravesical pembrolizumab in combination with BCG. The registry describes two dose cohorts, but it does not describe randomized allocation or an independent control group. As a result, the cohort summaries are descriptive; they are not a randomized estimate of the effect of one dose versus another.
What does the MTD definition mean?
The registry defines the MTD as the highest dose that causes dose-limiting toxicities in <2 of 6 patients, with toxicity graded according to NCI CTCAE version 4.03. In general, a threshold rule translates observed toxicity at dose levels into a dose-selection decision. Its interpretation depends on which events qualify as DLTs, how severity is graded, the period in which events count, and how decisions are made when observed counts are near the threshold. The endpoint timeframe is up to 9 weeks. The registry record does not report the cohort-specific DLT counts or the final dose decision, so the rule cannot be evaluated from the adverse-event summary alone.
A DLT is a protocol-defined endpoint, whereas a serious adverse event is a safety classification. The categories may overlap, but they are not synonymous. A serious event does not automatically qualify as a DLT, and the serious-event counts do not show which events were included in the MTD determination. The registry record does not report the relationship between the listed serious events and the DLT endpoint.
Why does the single-group design matter?
In general, random assignment helps balance measured and unmeasured factors between groups, supporting a causal comparison. This study is listed as single-group, with allocation not applicable and no masking. The two dose cohorts are part of the escalation design rather than a randomized control comparison. Any difference in their descriptive counts could reflect dose, participant characteristics, exposure, chance, or other differences; the registry record does not provide information that separates these explanations.
The absence of a control arm also means there is no direct estimate of what event frequency or tumor outcome would have occurred without the study combination. That limits comparative interpretation. It does not prevent the study from addressing its early-phase dose and tolerability aims, but those aims require the prespecified dose-escalation results, which are not reported in the registry record.
How should precision be considered?
In general, a confidence interval gives a range of values compatible with the observed data under a statistical model. Wider intervals indicate greater uncertainty, and small cohorts commonly yield imprecise estimates of event frequencies. The registry record does not report confidence intervals for the primary endpoint or adverse-event summaries. The listed counts should therefore be read as descriptive observations, not as precise estimates for a broader population.
The enrollment figure is 9. The cohort-level at-risk counts and event counts are reported separately. No cohort-specific inferential analyses are listed, and the registry record does not report a statistical model for comparing dose levels. No adjusted analysis is reported. The numerical summaries alone do not quantify uncertainty about a treatment effect.
What do the planning illustrations represent?
General sample-size planning uses assumptions about the effect to be detected, the statistical significance threshold, allocation, and the model used to approximate the sampling distribution. The planning illustration associated with this trial is described as using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. These are planning assumptions, not details of the trial's actual design. The stated assumption of equal allocation between two arms does not change the fact that the registry lists a single-group dose-escalation study.
Such planning values are illustrations for a hypothetical future comparison, not observed power and not evidence that the present study was adequately powered for a treatment-effect question. The sample-size justification for the actual study is not reported in the registry record. No calculated planning values are quoted because the registry entries do not list them.
What remains unavailable?
The registry lists 15 outcome measures and names dose-limiting toxicities and incidence of adverse events as secondary outcomes. It reports no analyses in the record's analysis count. That does not establish that no analyses were conducted outside the registry; it means the registry record does not report them. The primary endpoint result, detailed DLT results, efficacy findings, analysis populations, missing-data handling, and statistical analysis plan are not reported in the registry record.
In general, a statistical analysis plan clarifies which outcomes and populations will be analyzed, how missing observations will be handled, and whether comparisons are adjusted for multiple testing. Those details help distinguish prespecified analyses from exploratory ones and make uncertainty easier to evaluate. Their absence from the registry record prevents assessment of those practices for this study. It is not appropriate to infer unreported methods or results from the study's phase or design label.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 15 outcome measures, names maximum tolerated dose as the primary endpoint, and identifies dose-limiting toxicities and incidence of adverse events among the secondary outcomes. It does not provide a complete account of the outcome-specific analysis plan. The record's analysis count is 0, and detailed outcome analyses are not reported in the registry record.
In general, multiplicity arises when several outcomes, time points, subgroups, or statistical tests are examined. The more opportunities there are to identify a result by chance, the more important it is to specify which analyses are primary and whether any adjustment or hierarchy is used. The registry record does not report a multiplicity strategy, so it is not possible to determine how the listed outcomes relate analytically or whether inferential claims were adjusted.
Missing-data handling is also not reported in the registry record. The available entry does not state how participants with incomplete follow-up or unavailable measurements were treated in the primary endpoint or secondary outcomes. It does not describe imputation, exclusion rules, or sensitivity analyses. The analysis populations are not reported in the registry record, so the population used for each outcome cannot be identified.
These omissions do not establish that the study lacked an analysis plan or that data were missing. They limit what can be assessed from the registry entry. In particular, the available information does not show whether all enrolled participants contributed to each endpoint, whether follow-up was complete, or how uncertainty was handled when outcomes were unavailable.
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 |
|---|---|---|---|
| Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | 2 | 3 | 66.7% |
| Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BGC) | 2 | 6 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | 1 of 3 |
| Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BGC) | 3 of 6 |
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 |
|---|---|
| Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | 3 of 3 |
| Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BGC) | 6 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BGC) |
|---|---|---|---|
| Myocardial Infarctrion | Cardiac disorders | 1 of 3 | 0 of 6 |
| Hematuria | Renal and urinary disorders | 1 of 3 | 0 of 6 |
| Sinus Bradycardia | Cardiac disorders | 1 of 3 | 0 of 6 |
| Autoimmune Disorder | Immune system disorders | 0 of 3 | 1 of 6 |
| Pelvic Infection | Infections and infestations | 0 of 3 | 1 of 6 |
| Tremor | Nervous system disorders | 0 of 3 | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase I: Cohort 1 (1 mg/kg Pembrolizumab, 50 mg BGC) | Phase I: Cohort 2 (2 mg/kg Pembrolizumab, 50 mg BGC) |
|---|---|---|---|
| Hematuria | Renal and urinary disorders | 3 of 3 | 6 of 6 |
| Hypertension | Vascular disorders | 3 of 3 | 6 of 6 |
| Anemia | Blood and lymphatic system disorders | 3 of 3 | 4 of 6 |
| Creatinine increased | Investigations | 2 of 3 | 5 of 6 |
| Proteinuria | Renal and urinary disorders | 2 of 3 | 5 of 6 |
| Hyperuricemia | Metabolism and nutrition disorders | 2 of 3 | 3 of 6 |
| Diarrhea | Gastrointestinal disorders | 1 of 3 | 3 of 6 |
| Fatigue | General disorders | 1 of 3 | 2 of 6 |
| Urinary tract infection | Infections and infestations | 1 of 3 | 2 of 6 |
| Back Pain | Musculoskeletal and connective tissue disorders | 1 of 3 | 1 of 6 |
Serious events with the highest arm-level rate: myocardial infarctrion (33.3%), hematuria (33.3%), sinus bradycardia (33.3%), autoimmune disorder (16.7%), pelvic infection (16.7%).
The registry reports adverse-event summaries separately for the two dose cohorts. In the 1 mg/kg pembrolizumab and 50 mg BCG cohort, 3 were listed at risk for serious adverse events and 2 were affected; the other-adverse-event summary lists 3 at risk and 3 affected. In the 2 mg/kg pembrolizumab and 50 mg BCG cohort, the corresponding serious-event figures are 6 at risk and 2 affected, and the other-event figures are 6 at risk and 6 affected. Deaths are listed as 1 and 3, respectively.
The listed serious-event terms and percentages are Myocardial Infarctrion, Hematuria, and Sinus Bradycardia at 33.3% each, and Autoimmune Disorder, Pelvic Infection, and Tremor at 16.7% each. These entries should be read as the registry's event summary. The record does not report the event-level timing, severity grades, attribution to treatment, overlap among terms, or whether any listed event met the DLT definition. The death counts are also not described in terms of timing or cause. These summaries alone do not establish causation or provide a complete safety profile.
12. What the Result Does, and Does Not, Mean
This record describes a completed phase 1 study that combined intravesical pembrolizumab and BCG in patients with high-risk or BCG-refractory non-muscle-invasive bladder cancer. Its central statistical purpose was dose and tolerability assessment, with maximum tolerated dose as the primary endpoint. The registry provides the endpoint definition and cohort descriptions but does not report the MTD result or the DLT counts needed to evaluate it.
The adverse-event figures provide descriptive information for the listed cohorts, including counts at risk, counts affected, deaths, and selected serious-event terms with percentages. They do not show whether events were caused by treatment, how events were distributed over time, or how many qualified as dose-limiting toxicities. The percentages should not be interpreted as comparative risk estimates across treatments, because there is no randomized control arm and the record does not report a formal cohort comparison.
The study's brief summary describes efficacy and tumor characteristics as interests. The registry record does not report tumor-response results, biomarker findings, or an estimate of efficacy. Accordingly, the record cannot establish a comparative benefit, and its safety summaries cannot by themselves establish a complete account of harms. This is not a judgment about the treatment; it reflects the limited outcomes reported in the registry entry.
In general, early-phase dose-escalation findings are most informative when the dose-selection rule, toxicity definitions, follow-up, and observed DLT results are all available. Here, the design and rule are described, but the result is not. Readers should distinguish the study's stated aims from findings actually reported in the registry record, and should not treat the cohort summaries or the study's completed status as a substitute for the missing primary endpoint result.
13. Why This Trial Matters Statistically
This study is statistically notable because it asks a dose-finding question rather than a conventional randomized efficacy question. The primary endpoint, maximum tolerated dose, is defined through dose-limiting toxicity observed over up to 9 weeks. That makes the endpoint dependent on both the toxicity definition and the dose-escalation decision rule, not simply on an overall adverse-event count.
The distinction between endpoint types is important for readers of early-phase studies. Serious adverse events are clinically important safety observations, while DLTs are the events used under the study's stated rule to determine the MTD. The registry lists serious-event summaries but does not report the DLT counts or the resulting MTD. The entry therefore illustrates why a safety summary cannot be substituted for the primary dose-finding result.
The single-group design also shapes the claims that can be supported. Cohort-level summaries may describe what was observed at the listed dose levels, but without randomized allocation or a control arm they do not provide a like-for-like causal comparison. This is especially relevant when enrollment is limited and the record does not report inferential analyses or uncertainty estimates.
Finally, the registry's limited reporting of analysis methods, missing-data handling, analysis populations, and efficacy results constrains reproducibility and interpretation. In general, transparent reporting of these elements helps readers judge precision, identify the population behind each estimate, and distinguish confirmatory from exploratory findings. The record supplies the design context but leaves several of those interpretive elements unreported.
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 available registry entry does not report the primary MTD result, cohort-specific DLT counts, or the dose-selection decision. Those omissions prevent assessment of the central endpoint against its stated definition. The listed serious adverse-event counts are not a substitute because the record does not identify which events qualified as DLTs.
The study has no randomized comparator and is listed as single-group, with no masking. The dose cohorts are not a like-for-like control comparison. Consequently, their descriptive summaries cannot isolate the effect of pembrolizumab dose from other differences or estimate outcomes relative to an untreated or alternative-treatment group. The registry record does not report a formal statistical comparison between cohorts.
The enrollment was 9, and the cohort summaries include small at-risk counts. Such summaries offer limited precision for characterizing event frequencies beyond the study participants. Confidence intervals and other uncertainty estimates are not reported in the registry record. The record also does not provide detailed event timing, attribution, severity grades, or the relationship between serious events and DLTs.
The registry describes a maintenance phase but does not report its full schedule. It does not report the statistical analysis plan, sample-size justification, interim analyses, multiplicity strategy, missing-data methods, or analysis populations. Although efficacy and tumor characteristics are named as interests, corresponding results are not reported in the registry record. These limitations concern what can be evaluated from the entry and do not establish what analyses or additional results may exist elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pembrolizumab and BCG Solution in Treating Patients With Recurrent Non-Muscle-Invasive Bladder Cancer — NCT02808143.
- Registry record last updated 2024-06-26; status last verified 2024-06.
- Results first posted 2022-10-20.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT02808143 is a completed phase 1, single-group dose-escalation study sponsored by Northwestern University. It enrolled 9 patients with high-risk or BCG-refractory non-muscle-invasive bladder cancer and evaluated intravesical pembrolizumab with BCG. The registry lists two pembrolizumab dose cohorts, no masking, and no randomized comparator.
The primary endpoint was maximum tolerated dose, defined using dose-limiting toxicities graded by NCI CTCAE version 4.03 over up to 9 weeks. Secondary outcomes include dose-limiting toxicities and incidence of adverse events. The entry reports selected cohort-level adverse-event counts and serious-event terms, but does not report the MTD result, detailed DLT results, efficacy findings, or biomarker results. Statistical analysis methods, missing-data handling, and analysis populations are not reported in the registry record.