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 Dose Escalation Study MORAb-066 Targeting Tissue Factor (TF)-Expressing Malignancies Including Breast, Pancreatic, Colorectal, NSCLC (NCT01761240), a randomized phase 1 study. The primary outcome is Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01761240 |
| Phase | Phase 1 |
| Condition | Breast Cancer, Pancreatic Cancer, Colorectal Cancer |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 27 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Morphotek |
| Results posted | Yes |
2. Clinical Question
Population
Patients with advanced or metastatic breast, pancreatic, colorectal, or non-small-cell lung cancer (adenocarcinoma) were eligible for this first-in-human study. The registry record lists 27 participants enrolled. The safety analysis set included participants who received at least 1 dose of study drug.
Intervention
The intervention was MORAb-066, a humanized monoclonal antibody targeting human tissue factor. It was administered by intravenous infusion once weekly, on Days 1, 8, 15, and 22 of each 28-day cycle. The five listed dose groups were 0.1 mg/kg, 0.3 mg/kg, 1 mg/kg, 2 mg/kg, and 3 mg/kg.
Comparator
The registry lists MORAb-066 dose groups, but no placebo, standard-care, or untreated comparator. It reports randomized allocation, without giving the method of randomization or allocation ratio. Comparisons between doses therefore cannot be interpreted as comparisons with usual care or no treatment.
Primary question
The primary outcome was the number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), assessed from the first dose through 30 days after the last dose. The study also aimed to assess tolerability and pharmacokinetics and identify the maximum tolerated dose. The registry record does not report a single statistical comparison for the primary outcome.
This is a safety-focused, early-phase question rather than a confirmatory test of clinical benefit. The record identifies malignancies of several types, so the study population spans distinct cancer diagnoses. It does not report how participants were distributed by diagnosis or provide separate outcome results by cancer type. The main interpretive task is consequently to understand what the reported safety counts describe, and what cannot be concluded from them.
3. Trial Design
NCT01761240 is registered as a Phase I, open-label, parallel study with randomized allocation and no masking. The sponsor was Morphotek. The record lists the study as completed, with a start date of 2013-06-19 and a primary completion date of 2016-02-09. Enrollment was 27.
The study evaluated MORAb-066 at five dose levels: 0.1 mg/kg, 0.3 mg/kg, 1 mg/kg, 2 mg/kg, and 3 mg/kg. Each group description specifies intravenous infusion on Days 1, 8, 15, and 22 of each 28-day treatment cycle. Treatment was to continue until disease progression, unacceptable toxicity, participant withdrawal, or a study physician decision to discontinue, as applicable in the group descriptions.
The stated purpose was to assess safety, tolerability, and pharmacokinetics, and to identify the maximum tolerated dose when MORAb-066 was given weekly. The secondary outcomes include dose-limiting toxicity and maximum tolerated dose, along with pharmacokinetic measures: maximum observed serum concentration, time to maximum concentration, terminal elimination half-life, areas under the serum concentration-time curve, and volume of distribution.
Although the registry describes randomized allocation, it does not report how randomization was carried out, how participants were assigned among dose groups, or whether the groups were enrolled concurrently. It does not report a protocol-level sample-size justification or a detailed dose-escalation algorithm. Those elements should not be inferred from the dose groups alone. The registry record also does not list a separate comparator arm or a masking procedure; it explicitly describes the study as open-label.
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: A total of 27 participants were enrolled and treated in the study.
Overall Study
| Stage | MORAb-066 0.1 mg/kg | MORAb-066 0.3 mg/kg | MORAb-066 1 mg/kg | MORAb-066 2 mg/kg | MORAb-066 3 mg/kg |
|---|---|---|---|---|---|
| STARTED | 3 | 4 | 5 | 7 | 8 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 3 | 4 | 5 | 7 | 8 |
Reasons for not completing this period:
| Reason | MORAb-066 0.1 mg/kg | MORAb-066 0.3 mg/kg | MORAb-066 1 mg/kg | MORAb-066 2 mg/kg | MORAb-066 3 mg/kg |
|---|---|---|---|---|---|
| Progressive disease:radiology assessment | 3 | 3 | 0 | 0 | 0 |
| Progressive disease:clinical assessment | 0 | 0 | 3 | 1 | 0 |
| Withdrawal by Subject | 0 | 0 | 0 | 3 | 1 |
| Adverse Event | 0 | 0 | 0 | 1 | 6 |
| Death | 0 | 1 | 1 | 2 | 1 |
| Non-compliance | 0 | 0 | 1 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The safety analysis set was the group of participants who received at least 1 dose 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: The safety analysis set included all participants who had received at least 1 dose of study drug.
| Characteristic | Unit | MORAb-066 0.1 mg/kg | MORAb-066 0.3 mg/kg | MORAb-066 1 mg/kg | MORAb-066 2 mg/kg | MORAb-066 3 mg/kg | Total |
|---|---|---|---|---|---|---|---|
| Participants analysed | 3 | 4 | 5 | 7 | 8 | 27 | |
| Age, Continuous | years | 61.0 (SD 7.21) | 57.8 (SD 9.39) | 55.2 (SD 6.06) | 62.4 (SD 7.98) | 64.3 (SD 8.75) | 60.8 (SD 8.18) |
| Sex: Female, Male: Female | Participants | 2 | 2 | 3 | 4 | 4 | 15 |
| Sex: Female, Male: Male | Participants | 1 | 2 | 2 | 3 | 4 | 12 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 3 | 5 | 6 | 7 | 24 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 0 | 1 | 1 | 3 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 1 | 0 | 0 | 1 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 2 | 1 | 0 | 3 |
| Race (NIH/OMB): White | Participants | 3 | 4 | 2 | 5 | 8 | 22 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), measured over First dose of study drug (Baseline) up to 30 days after last dose of study drug (Up to approximately 2 years 7 months).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is the number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). The assessment period began with the first dose of study drug and continued until 30 days after the last dose; the registry gives the overall timeframe as up to approximately 2 years 7 months. The safety analysis set was defined as participants who received at least 1 dose.
Counting participants with events is a direct way to describe how many people in a group experienced a recorded safety event. It differs from counting every event: one participant may experience more than one event, while the participant count records that person as having an event. The registry describes safety monitoring through recording adverse events and serious adverse events, regular laboratory and vital-sign monitoring, periodic electrocardiograms and performance-status assessments, and physical examinations.
For an early-phase dose-escalation study, safety outcomes are used to characterize tolerability and inform dose selection, including assessment of dose-limiting toxicity and the maximum tolerated dose. This outcome does not, by itself, measure tumor response, survival, or comparative benefit. The registry record names TEAEs and SAEs as the primary outcome but does not provide a single combined estimate, a statistical comparison, or a complete set of category-specific primary-outcome results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record presents safety counts for the five MORAb-066 dose groups. The number at risk for serious adverse events was 3 in the 0.1 mg/kg group, 4 in the 0.3 mg/kg group, 5 in the 1 mg/kg group, 7 in the 2 mg/kg group, and 8 in the 3 mg/kg group. The corresponding numbers with serious adverse events were 0, 1, 3, 4, and 3. These are descriptive counts within the listed groups, not estimates from a stated adjusted comparison.
The posted counts for other adverse events were 3 of 3, 4 of 4, 5 of 5, 7 of 7, and 8 of 8 across the dose groups, respectively. Death counts in those groups were 0, 1, 1, 2, and 1. The registry does not state in these summaries whether deaths were considered related to study drug, caused by cancer, or otherwise attributable. Counts alone cannot establish causation.
These group sizes are small, and the number of participants differs across dose groups. A count in one group should therefore not be read as evidence that its dose has a reliably higher or lower risk than another dose. A participant count also does not show how many events an individual experienced, how long each participant was exposed, or how event timing differed. The registry does not report exposure-adjusted rates or a statistical model comparing dose groups.
The primary outcome is named as the number of participants with TEAEs and SAEs, but the record does not report one overall primary-endpoint result or a formal inferential comparison. It lists zero analyses. No confidence interval, p-value, or adjusted estimate for the primary outcome is reported in the registry record. Accordingly, the posted counts support a descriptive account of events in the safety analysis set, not a precise estimate of the difference between doses.
The study's stated aims also included identifying the maximum tolerated dose and describing pharmacokinetics. The registry lists these as outcomes, but the results summarized in this record do not state a maximum tolerated dose or provide pharmacokinetic results. The absence of those results in the registry record should not be converted into an assumption about what the investigators observed. The dose groups and safety counts alone do not establish which dose was selected or how exposure varied with dose.
9. Statistical Methods Explained
Because the primary outcome counts participants with events, its basic data structure is binary at the participant level: each person either did or did not meet a particular event definition during the assessment period. Counts are useful for showing the observed number of people affected in each group. They do not, on their own, describe the number of separate events, event severity, duration, timing, or relationship to treatment.
For a binary outcome, a comparative analysis would ordinarily define the groups being compared and a measure of difference or relative frequency, then quantify uncertainty around that estimate. A confidence interval describes a range of values compatible with the data under the statistical model and its assumptions; it is not a guarantee that the true value lies within that range. With small groups, uncertainty can be substantial, and estimates can change noticeably when a participant's outcome changes. The registry record does not report a confidence interval or a comparative estimate for the primary outcome.
The record lists randomized allocation, but it does not describe the randomization method, allocation ratio, or the analysis used to account for the five dose groups. Randomization can support fair comparisons when assignment procedures are properly implemented and the comparison is defined in advance. It does not by itself establish that a particular comparison was made or that a reported difference is precise. Here, the registry does not report a primary-endpoint comparison among doses or against a control group.
The groups have different numbers at risk: 3, 4, 5, 7, and 8 across the listed dose levels. With such small group counts, descriptive totals should not be treated as stable estimates of event probability. A formal analysis could use methods appropriate for sparse binary data, but the statistical method used for the primary outcome is not reported in the registry record. It would be inappropriate to claim that a specific model or correction was applied.
Over-dispersion is a general statistical concept that applies when observed variability is greater than a chosen model expects. For count data, ignoring over-dispersion can make uncertainty appear smaller than it is. That issue is distinct from the participant-level binary outcome, although repeated events per participant or heterogeneity between participants can also complicate analyses. The registry does not report an event-count model, a repeated-measures analysis, or an adjustment for such features. No conclusion about whether over-dispersion was assessed is possible from the record.
The primary outcome timeframe runs from the first dose to 30 days after the last dose, with an overall period stated as up to approximately 2 years 7 months. Participants may have different lengths of treatment and observation within such a study. A simple participant count treats the specified assessment period as the outcome window; it does not account for differing time at risk. Time-adjusted rates or time-to-event analyses could address different questions, but their use is not reported in the registry record. The death counts likewise should not be interpreted as time-to-event estimates.
The registry defines the safety analysis set as participants who received at least 1 dose. This is an exposure-based population, rather than a stated intention-to-treat population. For safety interpretation, including only participants who received study drug is a common descriptive approach, since those participants had exposure. The record does not describe how participants who were enrolled but never dosed were handled in other analyses, nor does it give the number in any alternative analysis population.
In a dose-escalation study, dose-limiting toxicity and maximum tolerated dose are important concepts. A dose-limiting toxicity is a protocol-defined safety event used in dose evaluation, while maximum tolerated dose is a dose-selection outcome based on study criteria. The registry lists both as secondary outcomes but does not provide the operational definitions, evaluation window for dose-limiting toxicity, escalation rules, or the resulting maximum tolerated dose. These details cannot be reconstructed from the dose-group summaries.
The pharmacokinetic outcomes concern concentration over time. Maximum observed serum concentration describes the highest measured concentration; time to maximum concentration records when it occurred; terminal half-life characterizes decline in the terminal elimination phase; and areas under the curve summarize concentration over specified intervals. Volume of distribution is a pharmacokinetic parameter describing the apparent distribution of a drug. These are general interpretations of the named measures, not findings for MORAb-066. Their numerical results and analysis methods are not reported in the registry record.
The registry lists zero analyses, and it does not report p-values, confidence intervals, model specifications, covariate adjustments, interim analyses, or a detailed statistical analysis plan. A planned sample size and its justification are also not reported in the registry record. Any sample-size planning illustration should be understood as a planning exercise rather than an observed study result or a measure of the study's power after completion. No observed-power claim can be made from the counts.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple safety and pharmacokinetic outcomes. The primary outcome includes TEAEs and SAEs, while secondary outcomes include dose-limiting toxicity, maximum tolerated dose, and several pharmacokinetic measures. It does not report a multiplicity strategy, such as a hierarchy of hypotheses or an adjustment for testing multiple outcomes. The number of analyses is listed as zero, so the record does not supply a set of formal tests whose multiplicity can be evaluated.
Multiplicity matters when many outcomes or comparisons are tested: the chance of finding at least one apparently notable result can increase if tests are interpreted independently without a plan. This is a general statistical concern, not a claim that such testing occurred in this study. Without reported analyses or a statistical plan, it is not possible to determine how investigators handled multiple outcomes, dose comparisons, or repeated looks at accumulating data.
Missing-data methods are not reported in the registry record. The safety population is described as participants who received at least 1 dose, but the record does not explain how incomplete safety follow-up, missing laboratory measurements, withdrawal, or other missing observations were handled. It does not report the amount or pattern of missing data. The treatment descriptions allow discontinuation for several reasons, but they do not provide counts or outcome handling for each reason.
These omissions limit the interpretation of uncertainty and completeness. A count of participants with an event may depend on how long participants were observed and whether follow-up information was available. The registry does not report sensitivity analyses, imputation, or a comparison of participants with complete and incomplete follow-up. Therefore, the posted counts can be described, but the effect of missing observations on those counts cannot be assessed from this record.
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 |
|---|---|---|---|
| MORAb-066 0.1 mg/kg | 0 | 3 | 0.0% |
| MORAb-066 0.3 mg/kg | 1 | 4 | 25.0% |
| MORAb-066 1 mg/kg | 3 | 5 | 60.0% |
| MORAb-066 2 mg/kg | 4 | 7 | 57.1% |
| MORAb-066 3 mg/kg | 3 | 8 | 37.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| MORAb-066 0.1 mg/kg | 0 of 3 |
| MORAb-066 0.3 mg/kg | 1 of 4 |
| MORAb-066 1 mg/kg | 1 of 5 |
| MORAb-066 2 mg/kg | 2 of 7 |
| MORAb-066 3 mg/kg | 1 of 8 |
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 |
|---|---|
| MORAb-066 0.1 mg/kg | 3 of 3 |
| MORAb-066 0.3 mg/kg | 4 of 4 |
| MORAb-066 1 mg/kg | 5 of 5 |
| MORAb-066 2 mg/kg | 7 of 7 |
| MORAb-066 3 mg/kg | 8 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | MORAb-066 0.1 mg/kg | MORAb-066 0.3 mg/kg | MORAb-066 1 mg/kg | MORAb-066 2 mg/kg | MORAb-066 3 mg/kg |
|---|---|---|---|---|---|---|
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 0 of 4 | 1 of 5 | 1 of 7 | 0 of 8 |
| Small intestinal obstruction | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 0 of 5 | 1 of 7 | 1 of 8 |
| Haematuria | Renal and urinary disorders | 0 of 3 | 0 of 4 | 0 of 5 | 1 of 7 | 1 of 8 |
| Anaemia | Blood and lymphatic system disorders | 0 of 3 | 0 of 4 | 0 of 5 | 0 of 7 | 2 of 8 |
| Cholangitis | Hepatobiliary disorders | 0 of 3 | 1 of 4 | 0 of 5 | 0 of 7 | 0 of 8 |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 1 of 5 | 0 of 7 | 0 of 8 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 0 of 4 | 1 of 5 | 0 of 7 | 0 of 8 |
| Gastric haemorrhage | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 1 of 5 | 0 of 7 | 0 of 8 |
| Mental status changes | Psychiatric disorders | 0 of 3 | 0 of 4 | 0 of 5 | 1 of 7 | 0 of 8 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 0 of 4 | 0 of 5 | 1 of 7 | 0 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | MORAb-066 0.1 mg/kg | MORAb-066 0.3 mg/kg | MORAb-066 1 mg/kg | MORAb-066 2 mg/kg | MORAb-066 3 mg/kg |
|---|---|---|---|---|---|---|
| Haematuria | Renal and urinary disorders | 0 of 3 | 1 of 4 | 1 of 5 | 5 of 7 | 6 of 8 |
| Vomiting | Gastrointestinal disorders | 1 of 3 | 2 of 4 | 4 of 5 | 3 of 7 | 1 of 8 |
| Fatigue | General disorders | 2 of 3 | 2 of 4 | 3 of 5 | 2 of 7 | 2 of 8 |
| Anaemia | Blood and lymphatic system disorders | 0 of 3 | 2 of 4 | 2 of 5 | 1 of 7 | 3 of 8 |
| Decreased appetite | Metabolism and nutrition disorders | 1 of 3 | 1 of 4 | 3 of 5 | 1 of 7 | 1 of 8 |
| Urinary tract infection | Infections and infestations | 0 of 3 | 1 of 4 | 1 of 5 | 3 of 7 | 2 of 8 |
| Diarrhoea | Gastrointestinal disorders | 2 of 3 | 2 of 4 | 0 of 5 | 1 of 7 | 1 of 8 |
| Nausea | Gastrointestinal disorders | 1 of 3 | 1 of 4 | 4 of 5 | 0 of 7 | 0 of 8 |
| Pyrexia | General disorders | 1 of 3 | 0 of 4 | 1 of 5 | 2 of 7 | 2 of 8 |
| Constipation | Gastrointestinal disorders | 0 of 3 | 0 of 4 | 1 of 5 | 2 of 7 | 1 of 8 |
Serious events with the highest arm-level rate: cholangitis (25.0%), anaemia (25.0%), gastrointestinal haemorrhage (20.0%), pleural effusion (20.0%), dyspnoea (20.0%).
The adverse-event record gives, for each dose group, the number at risk and the number with serious adverse events, other adverse events, and deaths. For serious adverse events, the group counts were 0 of 3 at 0.1 mg/kg, 1 of 4 at 0.3 mg/kg, 3 of 5 at 1 mg/kg, 4 of 7 at 2 mg/kg, and 3 of 8 at 3 mg/kg. For other adverse events, the counts were 3 of 3, 4 of 4, 5 of 5, 7 of 7, and 8 of 8. The death counts were 0, 1, 1, 2, and 1, respectively.
The registry also lists serious-event terms with percentages: cholangitis and anaemia at 25.0% each, and gastrointestinal haemorrhage, pleural effusion, dyspnoea, and gastric haemorrhage at 20.0% each. The record does not give a denominator alongside these percentages in the summary, so they should be read as posted rather than converted into participant counts.
These figures are descriptive. They do not indicate whether events were caused by MORAb-066, and the death counts are not stated to be treatment-related. Small group sizes, differing observation time, and the absence of a comparator affect interpretation. The registry record does not provide event-level timing, exposure-adjusted rates, or an analysis comparing doses.
12. What the Result Does, and Does Not, Mean
The record establishes that NCT01761240 was a Phase I study intended to examine safety, tolerability, and pharmacokinetics of MORAb-066 in patients with advanced or metastatic malignancies. It reports five dose groups and describes safety counts among participants who received at least 1 dose. It also names dose-limiting toxicity and maximum tolerated dose as outcomes of interest.
The counts do not establish that MORAb-066 caused any particular event, nor do the deaths establish a causal relationship to study drug. The registry does not report attribution for the listed events or deaths. Likewise, these data do not show whether MORAb-066 reduced tumor burden, prolonged survival, or improved any clinical outcome. Those questions are not answered by the posted safety counts.
The lack of a placebo or standard-care arm in the listed interventions means the record does not provide a direct comparison with usual care or no treatment. The dose groups can be described, but without a reported inferential analysis, one should not interpret differences in counts as evidence of a dose-related effect. Group sizes are small and unequal, and the record does not provide a model or uncertainty estimates that would support a precise comparison.
The registry record does not state the maximum tolerated dose or report results for the pharmacokinetic outcomes, despite listing these as study aims or outcomes. It also does not report the protocol definitions and dose-escalation rules needed to interpret dose selection. These are absences in the registry record, not evidence that the study did not collect the information.
In short, the posted results permit a limited descriptive reading of adverse-event counts within the safety analysis set. They do not support a conclusion about treatment benefit, causation, comparative safety between doses, or the selected dose. Those questions require results and analysis details not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically relevant as an example of the challenges in interpreting early-phase dose-escalation data. The outcomes are primarily safety and pharmacokinetic measures, and the dose groups are small. Counts provide a transparent first description, but sparse data do not automatically support precise comparisons or a stable ranking of doses by safety.
The record also illustrates why study design labels need context. It reports randomized allocation, but does not describe the allocation procedure or present a formal comparison. It is open-label and lists only MORAb-066 dose groups. A design label alone does not tell readers which contrast was planned, how it was analyzed, or how uncertainty was quantified.
Several distinct questions are named: adverse events, dose-limiting toxicity, maximum tolerated dose, and pharmacokinetics. They require different definitions and analyses. A participant count with an event is not the same as an event rate over time; a pharmacokinetic concentration measure is not a clinical outcome; and dose selection requires protocol criteria that are not included in the registry summary.
Finally, the record makes clear the importance of reporting analysis populations, outcome definitions, missing-data handling, and statistical methods. Without these details, readers can accurately describe the posted counts but cannot assess how robustly they estimate risk or how the study reached dose-selection conclusions. The statistical value of the record is therefore in clarifying both what is reported and the limits of what can be inferred.
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 contains limited inferential information. It reports zero analyses and does not provide a statistical analysis plan, method of randomization, allocation ratio, or formal comparison between dose groups. It also does not report a sample-size justification, interim-analysis plan, confidence intervals, p-values, or adjusted effect estimates. The basis for selecting participants per dose group is not reported in the registry record.
Although dose-limiting toxicity and maximum tolerated dose are listed as outcomes, their definitions, assessment rules, and results are not given. The record does not state which dose, if any, was identified as the maximum tolerated dose. The pharmacokinetic outcomes are also listed without numerical results in the information. Thus the dose-escalation and pharmacokinetic aims cannot be evaluated from the posted adverse-event counts alone.
The record describes a safety population of participants who received at least 1 dose, but does not report the number or outcomes of participants who may have enrolled without receiving treatment. Follow-up completeness, reasons for withdrawal, and handling of missing observations are not described. Group exposure duration and event timing are also absent, limiting assessment of differing time at risk.
The population spans breast, pancreatic, colorectal, and non-small-cell lung cancer (adenocarcinoma), but diagnosis-specific enrollment and outcomes are not reported. The record also gives no event attribution for the listed serious adverse events or deaths. These limitations mean that the safety counts should remain descriptive and should not be generalized into causal or comparative conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Dose Escalation Study MORAb-066 Targeting Tissue Factor (TF)-Expressing Malignancies Including Breast, Pancreatic, Colorectal, NSCLC — NCT01761240.
- Registry record last updated 2024-03-29; status last verified 2017-02.
- Results first posted 2024-03-29.
- 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
NCT01761240 was a completed Phase I, open-label, parallel study with randomized allocation, sponsored by Morphotek. It enrolled 27 participants with advanced or metastatic breast, pancreatic, colorectal, or non-small-cell lung cancer (adenocarcinoma). The study evaluated weekly intravenous MORAb-066 at 0.1 mg/kg, 0.3 mg/kg, 1 mg/kg, 2 mg/kg, and 3 mg/kg.
The primary outcome was the number of participants with TEAEs and SAEs. The safety analysis set included participants who received at least 1 dose. The registry posts adverse-event counts by dose group, but lists zero analyses and does not provide a formal primary-endpoint comparison. Dose-limiting toxicity, maximum tolerated dose, and pharmacokinetic measures are listed as secondary outcomes; their results are not reported in the registry record.
The counts support a descriptive account of events in the study groups. They do not establish causation, comparative safety, clinical benefit, or the dose selected for further study.