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 A Study to Determine Best Tumor Response With Trastuzumab Emtansine in Human Epidermal Growth Factor Receptor 2 (HER2) Overexpressing Solid Tumors (NCT02999672), a non-randomized phase 2 study. The primary outcome is Best Overall Response (BOR) Assessed by the Investigator Using Response Evaluation Criteria in Solid Tumors [RECIST] 1.1)..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02999672 |
| Phase | Phase 2 |
| Condition | Bladder Cancer, Pancreas Cancer, Cholangiocellular Carcinoma |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 20 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
Population
Participants with HER2-overexpressing urothelial bladder cancer (UBC), pancreatic cancer, or cholangiocarcinoma. The study summary describes locally advanced or metastatic disease; for the bladder cancer and pancreatic cancer/cholangiocarcinoma groups, it specifies disease that was not treatable with curative intent or where cure was no longer possible. The summary also describes no other treatment options as available for the pancreatic cancer/cholangiocarcinoma group.
Intervention
Trastuzumab emtansine, given by intravenous infusion under Regimen A or Regimen B. Regimen A is 2.4 mg/kg weekly; Regimen B is 3.6 mg/kg every 3 weeks. Treatment was to continue until unacceptable toxicity, withdrawal of consent, disease progression, or death, whichever occurred first.
Comparator
There was no randomized control group. The registry describes a non-randomized, single-agent study organized into disease cohorts. Comparisons between the cohorts or regimens would not be like-for-like randomized comparisons.
Primary question
What proportion of treated participants had a best overall response of complete response or partial response, assessed by the investigator using RECIST 1.1 and confirmed at least 28 days after the initial response? The record defines this endpoint for the UBC and pancreatic cancer/cholangiocarcinoma cohorts, but does not report the response proportion.
The study is NCT02999672, titled “A Study to Determine Best Tumor Response With Trastuzumab Emtansine in Human Epidermal Growth Factor Receptor 2 (HER2) Overexpressing Solid Tumors.” It was a multicenter Phase II exploratory study. Its central statistical question is descriptive: whether treated participants were recorded as having a confirmed complete or partial response. Without a randomized comparator, the design does not estimate a treatment effect against another therapy or against no treatment. The registry record describes two dosing schedules and disease-specific cohorts, but does not report numerical response results, so the primary question cannot be answered from the posted outcome information.
3. Trial Design
This was a completed, multicenter, non-randomized, single-agent Phase II study sponsored by Hoffmann-La Roche. The registry lists enrollment of 20. The recorded start date is 2016-12-23, and the primary completion date is 2018-04-10. The listed conditions are bladder cancer, pancreas cancer, and cholangiocellular carcinoma. The study model is parallel, with cohorts for UBC and pancreatic cancer/cholangiocarcinoma; allocation was not randomized, and masking was none.
Participants were to receive trastuzumab emtansine by intravenous infusion as Regimen A, 2.4 mg/kg weekly, or Regimen B, 3.6 mg/kg every 3 weeks. The study summary says treatment continued until unacceptable toxicity, withdrawal of consent, disease progression, or death, whichever occurred first. It also describes a steering committee and an Independent Data Monitoring Committee (iDMC) making decisions about expansion based on tolerability and safety. The cohort descriptions refer to an iDMC assessment among the first six participants in each cohort and possible dose changes. The detailed dose-assignment and switching procedures are not fully reported in the registry record.
The primary endpoint was best overall response (BOR), assessed by the investigator under RECIST 1.1. Secondary outcomes listed include progression-free survival, overall survival, adverse events and serious adverse events, drug-induced liver injury meeting Hy’s Law criteria, and trastuzumab emtansine concentrations. The registry lists 6 outcome measures and 0 analyses. Although the study status is completed, the registry record does not report numerical results for the primary endpoint or the listed secondary outcomes. The protocol, full statistical analysis plan, and sample-size justification 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.
Overall Study
| Stage | Cohort 1 (UBC) | Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) |
|---|---|---|
| STARTED | 13 | 7 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 13 | 7 |
Reasons for not completing this period:
| Reason | Cohort 1 (UBC) | Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) |
|---|---|---|
| Death | 0 | 1 |
| Study Terminated by Sponsor | 0 | 1 |
| Progressive Disease | 9 | 5 |
| Adverse Event | 4 | 0 |
Analysis population for the primary outcome, as defined in the registry: Urothelial bladder cancer (UBC) and Pancreatic Cancer/Cholangiocarcinoma cohorts
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 | Cohort 1 (UBC) | Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) | Total |
|---|---|---|---|---|
| Participants analysed | 13 | 7 | 20 | |
| Age, Continuous | Years | 57.4 (SD 14.1) | 62.9 (SD 7.2) | 59.3 (SD 12.2) |
| Sex: Female, Male: Female | Participants | 1 | 3 | 4 |
| Sex: Female, Male: Male | Participants | 12 | 4 | 16 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 | 0 | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 9 | 7 | 16 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 | 0 | 2 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 12 | 7 | 19 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Best Overall Response (BOR) Assessed by the Investigator Using Response Evaluation Criteria in Solid Tumors [RECIST] 1.1)., measured over Baseline up to PD/recurrence or death, whichever occurs first (up to approximately 18 months).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Percentage of Treated Participants |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the percentage of treated participants whose best overall response was complete response (CR) or partial response (PR), as assessed by the investigator using RECIST 1.1. A response had to be confirmed at least 28 days after the initial response. The assessment period ran from baseline up to disease progression or recurrence, or death, whichever occurred first, with the timeframe described as up to approximately 18 months.
CR was defined as disappearance of all target and non-target lesions. Any pathological lymph nodes, whether target or non-target, had to decrease to normal size, defined as a short axis < 10 mm. PR was defined as a 30% decrease in the sum of target-lesion diameters, using the baseline sum as the reference. The endpoint combines these two response categories; it does not count stable disease as a response under the stated definition.
As a general statistical concept, a response proportion gives a direct description of how many participants in a defined treated group met the response criteria. Its interpretation depends on the response rules, confirmation requirement, assessment period, and participants included in the denominator. The record names the denominator as treated participants, but does not report the observed percentage, the number meeting response criteria, or a confidence interval. Therefore, the endpoint definition is available, but the magnitude and precision of the observed response are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and endpoint definition, but does not provide the numerical primary outcome. It does not report the number of participants with CR or PR, the percentage of treated participants with BOR, or an uncertainty interval for that percentage. As a result, the posted results do not establish the observed response frequency or how precisely it was estimated. The completed status and the enrollment figure do not substitute for an endpoint result.
The non-randomized design is important to interpretation. Participants were treated in disease cohorts, and there was no randomized control group. Any response proportion, if reported, would describe outcomes among the treated participants in the relevant cohort under the study’s assessment rules. It would not, by itself, quantify how outcomes would differ under another treatment or without trastuzumab emtansine. Nor would a difference between cohorts establish a regimen effect: the cohorts concern different cancer types and are not randomized comparison groups.
The record describes two schedules: 2.4 mg/kg weekly and 3.6 mg/kg every 3 weeks. It also says committee decisions could affect study expansion and describes possible dose switching in the cohort descriptions. The complete assignment and switching details are not reported in the registry record. Without detailed exposure and outcome information by regimen, comparisons between schedules cannot be interpreted as randomized evidence about the schedules.
The secondary outcomes cover survival, safety, liver injury, and drug concentrations, but no numerical results for these outcomes are reported in the registry record. The adverse-event entries do include cohort-level counts for serious adverse events, other adverse events, and deaths. Those counts describe the record’s reported categories; they do not establish that an event was caused by treatment. The serious-event terms and percentages also need to be read in light of the different cohort denominators.
In general, a confidence interval communicates the range of estimates compatible with the data under a specified method, and its width reflects statistical precision. No confidence interval for BOR is reported in the registry record, so precision cannot be assessed from an interval. The absence of an analysis entry also means there is no reported inferential test to interpret. The available record supports a description of the design, endpoint, and some safety counts, not a numerical conclusion about the primary response outcome or a causal treatment effect.
9. Statistical Methods Explained
What does the primary endpoint measure?
The endpoint is a response proportion: the percentage of treated participants whose best overall response was CR or PR. The investigator assessed response using RECIST 1.1, and a response had to be confirmed at least 28 days after the initial response. The definition matters because response rates depend on which outcomes qualify, when lesions are assessed, and what denominator is used. Here, the registry specifies treated participants as the denominator and defines CR and PR, but does not report the resulting count or percentage.
In general, a proportion is a descriptive statistic for a defined group. It does not indicate how much an individual participant’s tumor changed beyond the response rules, and it does not alone compare outcomes with a control group. For this study, the endpoint is also limited to the UBC and pancreatic cancer/cholangiocarcinoma cohorts named in the primary outcome description.
What role does RECIST 1.1 play?
RECIST 1.1 provides standardized rules for classifying tumor response. In this record, CR requires disappearance of all target and non-target lesions, with pathological lymph nodes decreasing to a short axis < 10 mm. PR requires a 30% decrease in the sum of target-lesion diameters from baseline. These operational definitions support consistent classification, but do not remove all potential variation in imaging, lesion selection, or investigator assessment. The registry does not report whether response assessments were independently reviewed or blinded; masking is listed as none.
Confirmation at least 28 days after initial response is part of the endpoint definition. In general, confirmation requirements distinguish a recorded response from a finding that persists at a subsequent assessment. The posted information does not give individual assessment dates or response classifications, so the confirmation rule cannot be checked against participant-level data.
How should precision be assessed?
In general, a confidence interval around a response proportion expresses uncertainty about the underlying response frequency for a population represented by the study participants. A wider interval indicates less precise estimation; a narrower interval indicates more precision under the chosen method and assumptions. No BOR estimate or confidence interval is reported in the registry record. Consequently, neither the estimated response frequency nor its statistical precision can be evaluated from the posted endpoint information.
The registry lists 0 analyses. It therefore does not report an inferential test for the primary endpoint, a p-value, or an analysis model for BOR. This does not establish that no analyses were conducted outside the registry; it means they are not reported in the registry record. The statistical analysis plan and details of the analysis population are also not reported in the registry record.
What do the planning calculations represent?
General sample-size calculations are planning tools: they describe the number of participants or events that might be needed under specified assumptions. They are not observed results and are not a measure of the trial’s achieved power. The planning assumptions associated with this record use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. For time-to-event calculations, the stated convention is a total number of events using Schoenfeld; other calculations are expressed as participants per arm for 80% power or another stated power level. The numeric planning outputs are not needed to interpret the clinical outcome and are not reported in the registry record as part of the trial’s own design.
These calculations should not be confused with this study’s actual design. The trial was non-randomized and organized by disease cohorts, so an equal-allocation, two-arm illustration is not a description of its enrollment structure. The registry record does not report the sponsor’s sample-size justification or protocol assumptions. In general, planning calculations depend on the effect size, allocation, outcome model, and other assumptions; a change in those assumptions changes the resulting planning requirement. Such calculations cannot replace observed outcome data.
Why does over-dispersion matter in some analyses?
In general, over-dispersion occurs when observed variability in data exceeds what a statistical model assumes. If it is ignored, standard errors may be too small and uncertainty may be understated. This concept can matter for count outcomes or clustered data, but the record does not report a model for BOR or a count-based analysis. It would not be appropriate to claim that over-dispersion was assessed or corrected in this study. The absence of analysis details means the relevant model assumptions cannot be evaluated.
What is the limit of the available statistical evidence?
The endpoint definition is specific, but the endpoint result is absent. The registry also does not report estimates and uncertainty for the listed survival outcomes, or an analysis population, missing-data procedure, or multiplicity strategy. Statistical interpretation is therefore limited to the design and definitions that are recorded, plus the adverse-event counts. No effect estimate should be inferred from the planning illustration, cohort enrollment, treatment schedule, or study completion status.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and several secondary outcomes, including progression-free survival, overall survival, adverse events and serious adverse events, drug-induced liver injury meeting Hy’s Law criteria, and trastuzumab emtansine concentrations. It does not report a multiplicity strategy, such as a prespecified ordering of tests or adjustment of significance thresholds. The statistical analysis plan is not reported in the registry record. Without those details, it is not possible to determine how the study addressed the chance of false-positive findings across multiple outcomes or comparisons.
Missing-data methods are also not reported in the registry record. The endpoint definition gives an assessment period and a treated-participant denominator, but does not explain how participants without an evaluable assessment, who withdrew, or who had incomplete follow-up were handled in the BOR analysis. The record does not report how missing survival follow-up or incomplete safety information was treated. These omissions matter because analysis choices can affect both the denominator and the interpretation of estimates.
The analysis populations are not reported in the registry record. In particular, the registry does not specify whether the primary outcome used all treated participants, an evaluable population, or another defined group beyond stating that the endpoint is a percentage of treated participants. It also does not describe how participants who changed dosing schedules were assigned to an analysis group.
In general, multiplicity, missing data, and analysis-population definitions are parts of the statistical plan that help readers understand which estimates were targeted and how they were calculated. Their absence does not prove that no procedures were used, but it prevents assessment of those procedures from the registry record. With 0 analyses listed and no numerical primary outcome, there is no reported inferential result to interpret in relation to these issues.
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 |
|---|---|---|---|
| Cohort 1 (UBC) | 6 | 13 | 46.2% |
| Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) | 2 | 7 | 28.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Cohort 1 (UBC) | 7 of 13 |
| Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) | 1 of 7 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Cohort 1 (UBC) | 11 of 13 |
| Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) | 7 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | Cohort 1 (UBC) | Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) |
|---|---|---|---|
| Urinary tract infection | Infections and infestations | 2 of 13 | 0 of 7 |
| Diarrhoea | Gastrointestinal disorders | 1 of 13 | 0 of 7 |
| Vomiting | Gastrointestinal disorders | 1 of 13 | 0 of 7 |
| Pulmonary sepsis | Infections and infestations | 1 of 13 | 0 of 7 |
| Sepsis | Infections and infestations | 1 of 13 | 0 of 7 |
| Craniocerebral injury | Injury, poisoning and procedural complications | 1 of 13 | 0 of 7 |
| Confusional state | Psychiatric disorders | 1 of 13 | 0 of 7 |
| Haematuria | Renal and urinary disorders | 1 of 13 | 0 of 7 |
| Pancreatitis | Gastrointestinal disorders | 0 of 13 | 1 of 7 |
| Pyrexia | General disorders | 0 of 13 | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cohort 1 (UBC) | Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma) |
|---|---|---|---|
| Pyrexia | General disorders | 5 of 13 | 3 of 7 |
| Asthenia | General disorders | 4 of 13 | 2 of 7 |
| Anaemia | Blood and lymphatic system disorders | 3 of 13 | 1 of 7 |
| Abdominal pain | Gastrointestinal disorders | 0 of 13 | 4 of 7 |
| Aspartate aminotransferase increased | Investigations | 3 of 13 | 1 of 7 |
| Platelet count decreased | Investigations | 2 of 13 | 2 of 7 |
| Decreased appetite | Metabolism and nutrition disorders | 1 of 13 | 3 of 7 |
| Constipation | Gastrointestinal disorders | 2 of 13 | 1 of 7 |
| Vomiting | Gastrointestinal disorders | 2 of 13 | 1 of 7 |
| Influenza like Illness | General disorders | 2 of 13 | 1 of 7 |
Serious events with the highest arm-level rate: urinary tract infection (15.4%), pancreatitis (14.3%), pyrexia (14.3%), device-related sepsis (14.3%), diarrhoea (7.7%).
The registry reports serious adverse-event counts by cohort. In Cohort 1 (UBC), 6 of 13 participants at risk were recorded as affected by a serious adverse event. In Cohort 2 (Pancreatic Cancer/Cholangiocarcinoma), 2 of 7 participants at risk were recorded as affected. The record also lists other adverse-event counts: 11 of 13 in Cohort 1 and 7 of 7 in Cohort 2. These are cohort-specific counts, with different denominators; they should not be read as a direct comparison of safety between the cohorts.
The listed serious-event terms include urinary tract infection at 15.4%, pancreatitis at 14.3%, pyrexia at 14.3%, device-related sepsis at 14.3%, diarrhoea at 7.7%, and vomiting at 7.7%. The registry record does not provide enough detail in these entries to establish whether each event was considered related to treatment. The term “serious” refers to regulatory seriousness criteria and is not simply another word for symptom intensity. In general, serious adverse events and other adverse events are distinct reporting categories, and counts may represent participants rather than unique episodes unless the record states otherwise.
Deaths are listed for both cohorts: 7 in Cohort 1 and 1 in Cohort 2. The death counts alone do not indicate cause or treatment attribution. The registry does not report the associated timing or circumstances in these entries. These figures should therefore be read as reported counts, not as evidence that treatment caused an event or death.
12. What the Result Does, and Does Not, Mean
The record describes a clinical study designed to assess best overall response, safety, and pharmacokinetics of trastuzumab emtansine in HER2-overexpressing solid tumors. It gives a detailed primary endpoint definition based on investigator assessment under RECIST 1.1, including confirmation of CR or PR at least 28 days after an initial response. It also reports cohort-level adverse-event and death counts. These details clarify what outcomes the study intended to assess and what some safety entries record.
However, the numerical BOR result is not reported in the registry record. There is no reported number or percentage of treated participants with CR or PR, and no confidence interval or statistical test for that endpoint. The listed completion status and enrollment of 20 do not show how many participants responded. The registry’s secondary outcome list likewise does not provide numerical survival or pharmacokinetic results.
The design also limits what any response proportion could establish. As a non-randomized, single-agent study with disease cohorts, it does not provide a randomized comparison with another treatment. A response proportion would describe outcomes among participants treated in the relevant cohort, not the causal difference between treatments. The disease cohorts and dosing schedules should not be treated as interchangeable comparison groups.
In general, a clinical endpoint can be precisely defined while its result remains unavailable. The definition supports consistent interpretation of a result if one is reported; it does not supply that result. Similarly, safety counts can describe reported events without showing whether the study drug caused them. The record supports discussion of the study’s design, endpoint rules, and reported safety counts, but not a conclusion about the size or precision of the primary response outcome, comparative efficacy, or causal safety effects.
13. Why This Trial Matters Statistically
This study is statistically notable for the gap between a clearly specified response endpoint and the absence of a reported numerical response result in the registry record. Best overall response is defined using explicit RECIST 1.1 criteria, a confirmation requirement, and a stated treated-participant denominator. Those features give the endpoint an interpretable structure, but they cannot establish the response frequency without the underlying result.
The non-randomized design also makes the distinction between description and comparison essential. Results from the UBC and pancreatic cancer/cholangiocarcinoma cohorts would describe different clinical groups. Because participants were not randomized between regimens or disease cohorts, differences in observed outcomes would not isolate a causal effect of dose schedule or cancer type. The study summary’s committee-led safety and expansion decisions add design context, while the detailed procedures are not fully reported in the registry record.
For biostatisticians and clinical researchers, the record illustrates why endpoint definitions, analysis populations, missing-data rules, multiplicity plans, and numerical estimates all matter. A study may list multiple outcomes and a primary endpoint, yet without reported estimates and analysis details readers cannot judge magnitude, precision, or the handling of incomplete observations. Planning calculations are also conceptually separate from outcomes: they cannot stand in for an observed response estimate or be interpreted as achieved power.
The safety entries provide a further lesson in denominator-aware reading. Counts are reported separately by cohort, and the cohort denominators differ. Serious-event terms and death counts should be interpreted as recorded events, not as proof of causation. In this study, careful statistical interpretation means distinguishing what is defined, what is counted, what is not reported, and what the design can support.
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 record does not report the primary endpoint result, despite specifying how BOR was defined. It also does not report confidence intervals or inferential analyses for the primary endpoint. This prevents assessment of the observed response frequency and its precision. The record lists 0 analyses, and the statistical analysis plan is not reported in the registry record.
The non-randomized design has no randomized control group. It therefore cannot support a like-for-like randomized comparison of trastuzumab emtansine against another treatment. The cohorts represent different cancer types, and the record describes two dosing schedules with possible committee-directed changes. Detailed assignment, switching, exposure, and regimen-specific outcome information are not fully reported in the registry record. These features limit interpretation of any apparent differences between cohorts or schedules.
The record does not report the analysis population, missing-data approach, or multiplicity strategy. It also does not provide numerical results for the listed secondary outcomes. The adverse-event entries give counts and selected serious-event terms, but do not establish treatment attribution or provide detailed timing and circumstances for events and deaths.
The registry reports enrollment of 20, but enrollment alone does not establish the number assessed for BOR or the number included in each analysis. The protocol and sample-size justification are not reported in the registry record. Planning illustrations should not be taken as evidence of the study’s original design or as observed power. These limitations mean the available information is useful for understanding the intended endpoint and selected safety counts, but insufficient for a full assessment of efficacy, precision, or comparative effects.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study to Determine Best Tumor Response With Trastuzumab Emtansine in Human Epidermal Growth Factor Receptor 2 (HER2) Overexpressing Solid Tumors — NCT02999672.
- Registry record last updated 2019-08-28; status last verified 2019-07.
- Results first posted 2019-08-28.
- 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
NCT02999672 is a completed, multicenter, non-randomized Phase II study of trastuzumab emtansine in HER2-overexpressing solid tumors. The registry lists enrollment of 20 and identifies UBC and pancreatic cancer/cholangiocarcinoma cohorts. Participants were to receive one of two intravenous dosing schedules, with treatment ending at specified clinical or participant-related stopping events.
The primary endpoint was the percentage of treated participants with investigator-assessed CR or PR under RECIST 1.1, with response confirmation required at least 28 days after initial response. The registry lists 6 outcome measures and 0 analyses. It does not report a numerical BOR result, a confidence interval, or the statistical analysis plan. Cohort-specific adverse-event and death counts are recorded, but those counts do not establish treatment causation or allow a randomized safety comparison.