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 MLN0264 in Previously Treated Asian Participants With Advanced Gastrointestinal Carcinoma or Metastatic or Recurrent Gastric or Gastroesophageal Junction Adenocarcinoma Expressing Guanylyl Cyclase C (NCT02391038), a non-randomized phase 1 study. The primary outcome is Phase 1: Cycle 1- Cmax: Maximum Observed Plasma Concentration for MLN0264.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02391038 |
| Phase | Phase 1 |
| Condition | Advanced Gastrointestinal Carcinoma, Gastroesophageal Junction Adenocarcinoma, Recurrent Gastric Adenocarcinoma |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Millennium Pharmaceuticals, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Previously treated Asian participants with advanced gastrointestinal carcinoma, or metastatic or recurrent gastric or gastroesophageal junction adenocarcinoma expressing guanylyl cyclase C (GCC). The Phase 1 pharmacokinetic population was narrower: participants had to receive at least one dose of MLN0264 and have sufficient concentration-time data for reliable exposure estimation.
Intervention
MLN0264, an intravenous drug. The Phase 1 description begins with a 1.2 mg/kg starting dose on Day 1 of 3-week cycles and describes planned dose escalation; the remaining dose-escalation description is incomplete in the registry record.
Comparator
No comparator arm is identified. The study is listed as nonrandomized, unmasked, and parallel, and the intervention list names MLN0264. It is therefore not a randomized treatment-versus-control comparison.
Primary question
In Phase 1, what was the maximum observed plasma concentration (Cmax) of MLN0264 during Cycle 1 after dosing? The registry identifies Cmax as the primary outcome, but does not report its numerical result.
The study’s broader purpose was to evaluate MLN0264 in previously treated Asian participants with advanced gastrointestinal carcinoma in Phase 1, and in participants with metastatic or recurrent gastric or gastroesophageal junction adenocarcinoma expressing GCC in Phase 2. The registry lists the study phase as Phase 1, while its official title describes both phases. The primary outcome available in the record is specifically a Phase 1 pharmacokinetic endpoint; it is not a tumor-response measure.
The registry lists disease response and antitherapeutic antibodies among Phase 1 secondary outcomes, and safety and time-to-event outcomes among Phase 2 secondary outcomes. Those listed outcomes describe the broader research questions, but the registry record does not provide numerical results for the primary Cmax endpoint. It also does not establish a controlled comparison that could attribute differences to treatment.
3. Trial Design
This was a nonrandomized, unmasked, parallel-model study of MLN0264. The registry records an enrollment of 12 and lists the study as terminated. It identifies the study phase as Phase 1, although the trial title and stated purpose describe a Phase 1/2 program. The listed primary outcome is a Phase 1 pharmacokinetic measure.
The Phase 1 intervention description states that MLN0264 was given intravenously, starting at 1.2 mg/kg on Day 1 of 3-week cycles. It describes treatment for up to 1 year or until disease progression or unacceptable toxicity, and indicates dose escalation to 1.5 mg/kg and then 1.8 mg/kg using a 3 + 3 design. The description ends before completing its statement of what the escalation was intended to determine. No further detail about dose assignment or cohort structure is reported in the registry record.
Because the study was nonrandomized and no comparator is identified, its design does not provide a like-for-like control comparison. Unmasked means that masking is listed as none; the record does not describe additional procedures intended to conceal treatment assignment. A parallel model indicates that the record classifies the study under a parallel design model, but it does not supply further details about how participants were organized across dose levels.
The study started in 2014-12, and the primary completion date is recorded as 2015-10. These dates describe the record’s study timeline; they do not give the duration of follow-up for any participant. The registry record does not report why the study was terminated, whether all planned phases were conducted, or how enrollment and follow-up differed from the original plan.
The stated Phase 1 population for the primary pharmacokinetic outcome comprised participants who received at least one dose and had sufficient MLN0264 concentration-time data to permit reliable exposure estimation. The record does not report the number meeting that definition. The enrollment figure should not be read as the number included in every outcome analysis.
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: Participants with a historical diagnosis of gastrointestinal carcinoma were enrolled in 1 of 3 treatment groups: MLN0264 1.2 milligram per kilogram (mg/kg), MLN0264 1.5 mg/kg or MLN0264 1.8 mg/kg during Phase 1.
Overall Study
| Stage | Phase 1: MLN0264 1.2 mg/kg | Phase 1: MLN0264 1.5 mg/kg | Phase 1: MLN0264 1.8 mg/kg |
|---|---|---|---|
| STARTED | 3 | 3 | 6 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 3 | 3 | 6 |
Reasons for not completing this period:
| Reason | Phase 1: MLN0264 1.2 mg/kg | Phase 1: MLN0264 1.5 mg/kg | Phase 1: MLN0264 1.8 mg/kg |
|---|---|---|---|
| Progressive disease | 3 | 3 | 6 |
Analysis population for the primary outcome, as defined in the registry: Safety population includes all participants who received any amount of study drug.
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: Safety population included all participants who received any amount of study drug.
| Characteristic | Unit | Phase 1: All Participants |
|---|---|---|
| Participants analysed | 12 | |
| Age, Customized: Between 45 to 78 years | participants | 12 |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 7 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 12 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Japan | participants | 4 |
| Region of Enrollment: Taiwan | participants | 1 |
| Region of Enrollment: Korea, Republic of | participants | 7 |
| Histological classification: Colon cancer | participants | 2 |
| Histological classification: Rectal cancer | participants | 2 |
| Histological classification: Gastric cancer | participants | 2 |
| Histological classification: Lymphovascular invasion | participants | 1 |
| Histological classification: Invasive ductal carcinoma of the pancreas | participants | 1 |
| Histological classification: Moderately differentiated ductal adenocarcinoma | participants | 1 |
| Histological classification: Adenocarcinoma not otherwise specified | participants | 1 |
| Histological classification: Tubular adenocarcinoma | participants | 1 |
| Histological classification: Moderately differentiated and descending colon | participants | 1 |
| Participants With Prior Medical Condition | participants | 12 |
| Participants With Surgical History: With Surgical History | participants | 8 |
| Participants With Surgical History: Without Surgical History | participants | 4 |
| Participants With Prior Anticancer Therapy | participants | 12 |
| Participants With Prior Radiation Therapy: With prior radiation therapy | participants | 2 |
| Participants With Prior Radiation Therapy: Without prior radiation therapy | participants | 10 |
| Eastern Cooperative Oncology Group (ECOG) Therapy: 0 | participants | 8 |
| Eastern Cooperative Oncology Group (ECOG) Therapy: 1 | participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Phase 1: Cycle 1- Cmax: Maximum Observed Plasma Concentration for MLN0264, measured over Day 1 of Cycle 1: predose and at multiple time points (up to 336 hours) post-dose.
| Element | Registry entry |
|---|---|
| Result type | Geometric Mean |
| Measure of spread | Standard Deviation |
| Unit | microgram per milliliter (mcg/mL) |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is Cycle 1 Cmax: the maximum observed plasma concentration of MLN0264. Concentration is expressed in micrograms per milliliter. The planned sampling description begins with a predose measurement on Day 1 of Cycle 1 and includes multiple post-dose time points through 336 hours. The registry names the summary measure as a geometric mean with standard deviation.
Cmax is a pharmacokinetic measure: it describes the highest observed drug concentration in the sampled blood plasma during the measurement period. It is not a measure of tumor shrinkage, disease control, survival, or symptom change. Sampling time matters because the observed maximum depends on when measurements are taken; the listed repeated time points are intended to characterize concentration over time after dosing. The record does not report the actual concentration values, the resulting geometric mean, or its standard deviation.
The analysis population is defined as participants who received at least one dose and had sufficient concentration-time data for reliable exposure estimation. This definition makes the endpoint dependent on both treatment exposure and usable sampling data. The number meeting the definition, and any reasons participants may not have contributed usable data, are not reported in the registry record.
Geometric means are generally used for positive measurements that are better described on a multiplicative scale than by an arithmetic average. This is general statistical context, not a claim about the observed distribution in this study. The registry does not explain why this summary was selected or state whether any transformation or additional pharmacokinetic modeling was used.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record identifies a Phase 1 pharmacokinetic question: the Cycle 1 maximum observed plasma concentration of MLN0264. It specifies a geometric mean and standard deviation as the summary, but does not report the numerical Cmax result. Consequently, the posted record does not establish the observed concentration level or the precision of its estimate. Without those values, a statistical interpretation of the primary endpoint’s magnitude is not possible.
The record also does not report a confidence interval for Cmax, an individual-level distribution, or an inferential test for the primary outcome. A standard deviation describes variability among observations when reported, whereas a confidence interval describes uncertainty in an estimated population quantity; these are different summaries. This is general statistical context. Since neither the endpoint result nor an interval is posted, the uncertainty around the Cmax estimate cannot be assessed from this registry entry.
The study is listed as nonrandomized and has no identified comparator. Thus, the design does not support a randomized comparison between MLN0264 and another treatment or control condition. The listed Phase 1 disease-response outcome does not change the primary pharmacokinetic focus, and no response results are reported in the registry record. Phase 2 secondary outcomes include progression-free survival and duration of response, but the entry does not provide numerical results for these measures either.
The adverse-event summary records 4 in the serious-event affected column among 12 at risk, and 10 in the other-event affected column among 12 at risk. Four serious-event terms are listed at 8.3% each: tumor hemorrhage and ascites, asthenia, abdominal distension, and adrenal insufficiency. Those entries describe the registry’s posted safety summary. They do not establish that an event was caused by MLN0264, and the record does not provide event-level timing, severity grading, or a comparison group. The death value is not reported.
These results must be interpreted in the context of the registry’s stated enrollment and study status. The record lists enrollment of 12 and a terminated status, but does not report the reason for termination or the number of participants contributing to each outcome. It is therefore not possible to infer that every enrolled participant completed the planned treatment or sampling schedule. Nor can the record establish whether the listed outcomes represent complete follow-up.
Overall, the registry describes the purpose, design, primary measure, and some safety counts, but does not post the numerical primary endpoint result. It therefore supports a description of what was planned and what safety figures were recorded, rather than a conclusion about the size or precision of the pharmacokinetic result.
9. Statistical Methods Explained
The registry identifies Cmax as the primary outcome and specifies a geometric mean with standard deviation. It does not report a statistical analysis plan, an inferential model, or a numerical Cmax estimate. The following explanations describe general statistical concepts and should not be read as descriptions of analyses confirmed for this study.
What does Cmax represent?
Cmax is the highest observed concentration among the samples collected during a defined period. Here the stated period begins with predose sampling on Day 1 of Cycle 1 and extends through post-dose measurements up to 336 hours. It is an observed maximum, rather than necessarily the exact underlying peak concentration in the body: the true peak could occur between scheduled samples. The record does not give the individual sampling times or the observed concentrations, so the sampling density and the actual peak values cannot be evaluated.
Pharmacokinetic measures describe drug concentration over time. Cmax focuses on the maximum observed concentration, whereas other pharmacokinetic quantities can characterize exposure across time or the rate at which concentration changes. The registry names no additional primary pharmacokinetic measure in the posted primary outcome description. It also does not report whether concentration profiles were analyzed using a particular pharmacokinetic model.
Why use a geometric mean?
As general statistical context, a geometric mean is calculated on a multiplicative scale and is often used for positive measurements that are skewed or vary proportionally. It can be useful when a typical value is better described by relative rather than additive variation. A standard deviation conveys spread around the reported summary, but the registry does not clarify whether it is calculated on the original or transformed scale. It would be inappropriate to assume a specific transformation or distribution from the choice of summary alone.
The record does not include the geometric mean or its standard deviation. It therefore gives no basis for judging whether observed concentrations were tightly or widely dispersed, whether the sample distribution was skewed, or whether the summary was representative of individual participants. No alternative summaries, such as a median or range, are reported for this endpoint.
What does the analysis population mean?
The stated pharmacokinetic evaluable population includes participants who received at least one MLN0264 dose and had sufficient concentration-time data for reliable exposure estimation. This is an endpoint-specific definition: eligibility for the analysis depends on both receiving the intervention and having usable sampling information. It does not necessarily equal the study’s enrollment, and the record does not report how many participants satisfied the definition.
In general, restricting a pharmacokinetic analysis to participants with evaluable concentration data can make the endpoint interpretable for those with adequate measurements, but it can also mean that the analyzed group differs from the enrolled group. The direction or importance of any such difference cannot be assessed without participant-level accounting. Reasons for missing or unusable samples, and the handling of incomplete concentration profiles, are not reported in the registry record.
How should uncertainty be assessed?
A confidence interval is a general way to express uncertainty around an estimated quantity. Its width depends on factors such as variability and the amount of usable information. A standard deviation is not a substitute for a confidence interval: it describes dispersion in observations, while an interval describes uncertainty in an estimate under specified assumptions. The registry does not post a confidence interval, standard error, or other uncertainty interval for Cmax. No precision claim can therefore be made from the primary endpoint entry.
The record also does not report a hypothesis test, p-value, or prespecified threshold for interpreting the Cmax result. For a descriptive pharmacokinetic endpoint, an inferential test may not be the central aim, but the registry does not state the intended analysis beyond the named summary statistic. Any claim about the use or absence of a particular test would go beyond what is reported.
What do the design labels imply?
Nonrandomized means the registry does not identify random allocation. No comparator arm is identified, so the study does not permit a controlled estimate of the difference between MLN0264 and another assigned treatment. Unmasked indicates that masking is listed as none. These design features are relevant when interpreting outcomes: descriptive observations can characterize participants who received the drug, but they do not by themselves isolate a treatment effect from other influences.
The intervention description mentions a 3 + 3 dose-escalation design and lists a starting dose and subsequent dose levels. The registry description is truncated before completing the purpose of escalation. No dose-limiting toxicity definition, escalation decision rule, or statistical dose-selection method is reported. The design label alone does not justify assumptions about the actual conduct of escalation or the dose ultimately selected.
In sum, the statistical information reported for the primary endpoint is limited to its definition, timing, summary measure, and analysis-population criteria. Numerical results and detailed analysis methods are not reported in the registry record. General concepts about Cmax, geometric means, variability, uncertainty, and analysis populations help explain the endpoint, but cannot fill those reporting gaps.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 51 outcome measures, including the Phase 1 primary Cmax endpoint and secondary outcomes covering antitherapeutic antibodies, investigator-assessed disease response, safety, laboratory values, vital signs, progression-free survival, and duration of response. The number of analyses is listed as 0. These record entries do not explain how outcomes were prioritized across phases or how multiple statistical comparisons, if any, were handled.
When many outcomes or comparisons are tested, the chance of at least one apparently unusual result can increase. As general statistical context, prespecifying a primary endpoint and controlling multiplicity can help distinguish confirmatory questions from exploratory ones. The registry record does not report a multiplicity adjustment, an endpoint hierarchy beyond the primary designation, or a statistical plan describing the interpretation of multiple tests. It would be inappropriate to infer that an adjustment was or was not used in analyses that are not described.
Missing-data methods are also not reported in the registry record. The primary endpoint population requires sufficient concentration-time data, but the record does not state how incomplete profiles were handled or give reasons for missing measurements. It does not report methods for missing data in the listed disease-response or safety outcomes.
The PK-evaluable population is defined by receipt of at least one dose and adequate concentration-time data. The record does not report its size, participant flow into other analysis populations, or whether outcomes were analyzed according to additional population definitions. Enrollment should not be treated as the denominator for every endpoint without that information. These omissions limit assessment of selection, completeness, and the relationship between the planned outcomes and the analyses actually conducted.
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 1: All Participants | 4 | 12 | 33.3% |
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 |
|---|---|
| Phase 1: All Participants | 10 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase 1: All Participants |
|---|---|---|
| Tumor hemorrhage and ascites | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 12 |
| Asthenia | Respiratory, thoracic and mediastinal disorders | 1 of 12 |
| Abdominal distension | Gastrointestinal disorders | 1 of 12 |
| Adrenal insufficiency | Endocrine disorders | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase 1: All Participants |
|---|---|---|
| Nausea | Gastrointestinal disorders | 4 of 12 |
| Neutrophil count decreased | Investigations | 4 of 12 |
| Decreased appetite | Metabolism and nutrition disorders | 4 of 12 |
| Vomiting | Gastrointestinal disorders | 3 of 12 |
| Abdominal pain | Gastrointestinal disorders | 2 of 12 |
| Constipation | Gastrointestinal disorders | 2 of 12 |
| Diarrhoea | Gastrointestinal disorders | 2 of 12 |
| Asthenia | General disorders | 2 of 12 |
| Pyrexia | General disorders | 2 of 12 |
| Myalgia | Musculoskeletal and connective tissue disorders | 2 of 12 |
Serious events with the highest arm-level rate: tumor hemorrhage and ascites (8.3%), asthenia (8.3%), abdominal distension (8.3%), adrenal insufficiency (8.3%).
The registry’s Phase 1 safety summary records 12 participants at risk for serious adverse events and 4 in the serious-event affected column. It also records 12 at risk for other adverse events and 10 in the other-event affected column. The record does not provide event-level details that would establish whether the categories overlap, how events were counted across participants, or how long participants were observed.
The listed serious-event terms are tumor hemorrhage and ascites, asthenia, abdominal distension, and adrenal insufficiency, each reported at 8.3%. These percentages should be read as the registry’s reported figures; the record does not separately provide participant-level details for each term. The deaths entry is not reported in the registry record.
Serious adverse events are a safety classification, not a statement that the event was caused by the study drug. The summary does not report event onset, duration, severity grade, outcome, relatedness assessment, or a control-group rate. It also does not provide a detailed account of adverse-event collection or follow-up. The figures describe what the registry posts, but cannot establish comparative risk or causality.
12. What the Result Does, and Does Not, Mean
The record establishes that the study evaluated MLN0264 in previously treated Asian participants with advanced gastrointestinal carcinoma or specified gastric and gastroesophageal junction adenocarcinomas expressing GCC. The listed primary outcome is Phase 1 Cycle 1 Cmax, a pharmacokinetic measure of maximum observed plasma concentration. The registry describes the measurement period and the intended summary statistic, but does not report the numerical Cmax result.
That absence means the primary endpoint’s magnitude and precision cannot be interpreted from this entry. It is not evidence that the concentration was high or low, nor does it establish that the endpoint was not measured. The record simply does not report the numerical result. The same distinction applies to listed outcomes for disease response, progression-free survival, and duration of response: their presence in the outcome list does not itself supply results.
The safety summary reports affected counts and four serious-event terms with percentages. These are descriptive entries, not proof of causation, and the lack of a comparator prevents a controlled safety comparison. The record also does not report why the study was terminated or whether termination was related to safety, efficacy, recruitment, or another reason.
Because the study is nonrandomized and no comparator is identified, its design cannot support a randomized estimate of benefit or harm relative to another treatment. The posted record is also limited in its reporting of analysis populations, missing data, uncertainty, and outcome-specific results. Those gaps constrain what can be concluded from the registry entry. They do not justify filling in results from assumptions about the study’s aims or design.
The most defensible reading is therefore descriptive: the registry records the study’s population, intervention, primary pharmacokinetic question, design labels, and selected safety figures. It does not report enough numerical primary-outcome information to assess Cmax, and it does not provide a controlled comparison that would establish a treatment effect.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between a trial’s stated clinical purpose and its posted primary outcome. The overall purpose includes evaluating MLN0264 in gastrointestinal cancers, while the recorded Phase 1 primary endpoint is a pharmacokinetic quantity: maximum observed plasma concentration during Cycle 1. Pharmacokinetic characterization and tumor-response assessment address different questions and require different interpretations.
The record also illustrates why design labels matter. A nonrandomized study without an identified comparator can describe what was observed among participants who received the intervention, but it cannot provide a randomized treatment-versus-control contrast. This is particularly important when reading safety counts, which do not by themselves identify causation or comparative risk.
Another statistical issue is the distinction between enrollment and an endpoint’s analysis population. The primary pharmacokinetic population requires both drug exposure and sufficient concentration-time information. Without the number meeting that definition or a participant flow account, readers cannot determine how the endpoint population relates to enrollment.
Finally, a clearly named endpoint is not the same as a fully interpretable result. The registry defines Cmax, its sampling period, and the planned summary, but does not report the numerical estimate or its uncertainty. This makes reporting completeness central to interpretation: the endpoint can be understood conceptually, while its observed magnitude cannot be assessed. The study therefore highlights how design, analysis-population definitions, and outcome reporting all shape the statistical conclusions a registry record 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.
Several limitations are specific to the available registry record. The numerical result for the primary Cmax endpoint is not reported, and the record gives no estimate of uncertainty such as a confidence interval. Although the outcome specifies a geometric mean and standard deviation, neither value is posted. The primary pharmacokinetic question is therefore defined but not quantitatively answerable from this entry.
The registry lists enrollment of 12 but does not state how many participants contributed to the primary analysis or to each secondary outcome. Its PK-evaluable definition requires sufficient concentration-time data, yet the number meeting that definition and the reasons for any unavailable data are not reported. No participant-flow details or outcome-specific denominators are available in the record.
The design is nonrandomized, unmasked, and has no identified comparator. This limits causal and comparative interpretation. The record does not describe the reason for termination, whether all planned study phases were carried out, or how the termination affected follow-up or reporting. The intervention description is incomplete at its final sentence, so the purpose of the dose escalation is not fully stated there.
The safety entry does not include detailed event-level information, follow-up duration, relatedness assessments, or a reported deaths value. The registry also does not report a statistical analysis plan, missing-data approach, multiplicity handling, or methods for the listed time-to-event outcomes. These omissions should be treated as reporting limitations, not as evidence that a particular procedure or result was absent from the conduct of the study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: MLN0264 in Previously Treated Asian Participants With Advanced Gastrointestinal Carcinoma or Metastatic or Recurrent Gastric or Gastroesophageal Junction Adenocarcinoma Expressing Guanylyl Cyclase C — NCT02391038.
- Registry record last updated 2017-03-29; status last verified 2017-02.
- Results first posted 2017-03-29.
- 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
The registry describes a nonrandomized, unmasked, parallel-model study of MLN0264 in previously treated Asian participants with advanced gastrointestinal carcinoma or specified gastric or gastroesophageal junction adenocarcinoma expressing GCC. Enrollment is recorded as 12, the study status as terminated, and the phase as Phase 1; the title and stated purpose refer to Phase 1 and Phase 2.
The primary outcome is Phase 1 Cycle 1 maximum observed plasma concentration, summarized as a geometric mean with standard deviation. The PK-evaluable population requires at least one dose and sufficient concentration-time data. The numerical primary result and the size of that analysis population are not reported in the registry record.
The safety summary records serious- and other-event affected counts and lists four serious-event terms at 8.3% each. The reason for termination, detailed analysis methods, missing-data handling, and comparative results are not reported in the registry record.