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 Refametinib (BAY86-9766) in Combination With Regorafenib (Stivarga, BAY73-4506) in Patients With Advanced or Metastatic Cancer (NCT02168777), a non-randomized phase 1 study. The primary outcome is Maximum Drug Concentration in Plasma After Multiple Dose (Cmax,md) for Refametinib.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02168777 |
| Phase | Phase 1 |
| Condition | Neoplasms |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 20 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Bayer |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled patients with advanced or metastatic cancer. The Phase 1b dose-escalation portion allowed eligible participants with different tumor types; the planned Phase 2 portion was to evaluate the combination in patients with breast cancer, lung cancer, or colorectal cancer. The registry lists Neoplasms as the condition and records enrollment of 20.
Intervention
Participants received refametinib in combination with regorafenib. The dose-escalation cohorts used different doses of the two drugs. The listed schedules were refametinib 30mg twice daily with regorafenib 80mg once daily; refametinib 20mg twice daily with regorafenib 80mg once daily; and refametinib 20mg twice daily with regorafenib 120mg once daily. Each schedule is described as 3 weeks on/1 week off.
Comparator
There was no randomized control group. Allocation is recorded as non-randomized, and the study is described as uncontrolled and open-label. Cohorts reflect different dose combinations within the dose-escalation study, not a like-for-like comparison of treatments. Differences between cohort results therefore could not be attributed to dose alone from this design.
Primary question
The primary listed outcome concerns refametinib concentration in plasma after multiple dosing: its maximum concentration, reported as a geometric mean with a geometric coefficient of variation. This is a pharmacokinetic question about drug exposure, not a direct measure of tumor response. The registry also describes an intended dose-escalation aim of identifying a maximum tolerated dose and a recommended Phase 2 dose. The primary outcome result itself is not reported in the registry record.
3. Trial Design
The registry describes NCT02168777 as a Phase 1b/2, multicenter, uncontrolled, open-label dose-escalation study of refametinib with regorafenib in advanced or metastatic cancer. Its phase field, however, lists PHASE1. The study is marked non-randomized, with no masking and a parallel model. No randomized treatment comparison is described.
The stated Phase 1 aim was to determine the maximum tolerated dose of the combination through dose escalation. The registry says all tumor types meeting eligibility criteria could enter this part. After a recommended dose was determined, the planned Phase 2 portion was to assess tolerability and efficacy separately in patients with breast cancer, lung cancer, or colorectal cancer. The record does not establish that this planned Phase 2 evaluation was conducted or report its results.
The listed Phase 1b cohorts received different dose combinations: refametinib 30mg twice daily with regorafenib 80mg once daily; refametinib 20mg twice daily with regorafenib 80mg once daily; or refametinib 20mg twice daily with regorafenib 120mg once daily. Each regimen is described as 3 weeks on/1 week off. Because allocation was not randomized and there was no control arm, these cohorts should not be treated as equivalent groups assigned to a comparative trial.
The registry records enrollment of 20, a start date of 2014-06-23, a primary completion date of 2015-10-23, and a status of terminated. The reason for termination is not reported in the registry record. The record names Bayer as sponsor. It does not report why the planned Phase 2 portion was or was not undertaken.
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: The remaining 20 participants were assigned to and started treatment in one of three dose escalation cohorts. The Phase 2 part was not conducted.
Overall Study
| Stage | Ph1b-Refametinib/Regorafenib Cohort 0 | Ph1b-Refametinib/Regorafenib Cohort -1 | Ph1b-Refametinib/Regorafenib Cohort -1a |
|---|---|---|---|
| STARTED | 2 | 7 | 11 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 2 | 7 | 11 |
Reasons for not completing this period:
| Reason | Ph1b-Refametinib/Regorafenib Cohort 0 | Ph1b-Refametinib/Regorafenib Cohort -1 | Ph1b-Refametinib/Regorafenib Cohort -1a |
|---|---|---|---|
| Withdrawal by Subject | 1 | 0 | 2 |
| Adverse Event | 1 | 0 | 1 |
| Disease recurrence | 0 | 0 | 1 |
| Radiological disease progression | 0 | 5 | 5 |
| Clinical disease progression | 0 | 1 | 2 |
| Other reason | 0 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic (PK) analysis set for multiple dose.
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 | Ph1b-Refametinib/Regorafenib Cohort 0 | Ph1b-Refametinib/Regorafenib Cohort -1 | Ph1b-Refametinib/Regorafenib Cohort -1a | Total |
|---|---|---|---|---|---|
| Participants analysed | 2 | 7 | 11 | 20 | |
| Age, Continuous | Years | 57.0 (SD 14.1) | 56.6 (SD 5.7) | 58.6 (SD 7.3) | 57.8 (SD 7.1) |
| Sex: Female, Male: Female | Participants | 1 | 4 | 4 | 9 |
| Sex: Female, Male: Male | Participants | 1 | 3 | 7 | 11 |
| Cancer types: Colon and rectal cancer | Participants | 1 | 0 | 4 | 5 |
| Cancer types: Other | Participants | 1 | 2 | 2 | 5 |
| Cancer types: Non-small cell lung cancer | Participants | 0 | 3 | 1 | 4 |
| Cancer types: Breast cancer | Participants | 0 | 2 | 2 | 4 |
| Cancer types: Pancreatic adenocarcinoma | Participants | 0 | 0 | 2 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Drug Concentration in Plasma After Multiple Dose (Cmax,md) for Refametinib, measured over Cycle 1 Day 21 at 0 (pre-dose), 0.5, 1, 2, 4, 8 and 12 hours post-dose.
| Element | Registry entry |
|---|---|
| Result type | Geometric Mean |
| Measure of spread | Geometric Coefficient of Variation |
| Unit | μg/L |
| Endpoint classification | not reduced to a single comparison |
The primary listed endpoint is the maximum plasma concentration of refametinib after multiple dosing, abbreviated in the registry as Cmax,md. It is measured in μg/L, with the assessment scheduled for Cycle 1 Day 21 at 0 (pre-dose), 0.5, 1, 2, 4, 8 and 12 hours post-dose. The stated analysis population is the pharmacokinetic analysis set for multiple dose.
The endpoint is summarized using a geometric mean and a geometric coefficient of variation. A concentration-time measure describes drug exposure in plasma; the maximum concentration characterizes the highest measured concentration in the specified sampling period. The geometric mean is commonly used for positive measurements that are right-skewed or vary multiplicatively. The geometric coefficient of variation describes relative variability on a geometric scale. These are general statistical explanations, not additional findings from this study.
Pharmacokinetic measurements can characterize exposure and help inform dose selection in an early-phase study. They do not, by themselves, establish clinical benefit or harm, nor do they directly measure tumor response. The registry lists the endpoint and its measurement schedule, but the actual geometric mean and variability are not reported in the registry record. Thus the endpoint’s intended role can be described, but its observed concentration results cannot be interpreted from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive point is that the registry identifies a pharmacokinetic primary outcome but does not report its numerical result. It names the measure, unit, sampling schedule, summary statistics, and analysis population; it does not give the geometric mean or geometric coefficient of variation. The record therefore does not allow readers to assess the observed refametinib concentration or its variability.
The study’s design also limits comparative interpretation. It was open-label, uncontrolled, and non-randomized. Its dose cohorts were part of dose escalation rather than randomized treatment groups. The registry explicitly cautions that the arms are not a like-for-like control comparison. Any differences between cohorts could reflect differences in the enrolled participants, dose selection, or other factors; the record does not support isolating a causal dose effect.
The stated enrollment is 20, while safety information is presented separately for the three named cohorts. These figures serve different purposes: enrollment is a study-level registry value, while safety denominators are cohort-specific. They should not be treated as interchangeable. The registry does not report a patient flow account that would explain how enrollment relates to each analysis population or to each outcome.
The record lists 24 outcome measures and no analyses. A count of listed measures is not evidence that results are available for each measure. Although secondary outcomes include pharmacokinetic measures for refametinib, regorafenib, and metabolites, their numerical findings are not included in the registry record. The stated study aims also mention tolerability and efficacy, but the record does not provide tumor response results or a reported efficacy comparison.
The status is terminated, but the reason is not reported in the registry record. That status alone does not explain whether termination reflected safety, recruitment, strategic decisions, or another cause. Nor does it establish that every planned study component was completed. The listed Phase 1b/2 title and the phase field PHASE1 do not, by themselves, resolve which planned components reached completion.
Overall, the record documents a dose-escalation design, treatment schedules, an intended pharmacokinetic endpoint, and selected safety counts. It does not provide enough outcome information to estimate drug exposure, compare doses reliably, evaluate efficacy, or explain study termination. The registry entries should be read as a description of the registered study and its reported safety figures, not as a complete account of all trial findings.
9. Statistical Methods Explained
Statistical methods help distinguish what a study was designed to measure from what its results can establish. The registry gives some design and endpoint details, but it does not report a full statistical analysis plan. The explanations below describe general statistical concepts and identify which study-specific details are not reported in the registry record.
What does the primary pharmacokinetic measure represent?
The primary outcome is maximum plasma concentration after multiple dosing, summarized by a geometric mean and geometric coefficient of variation. Concentration is measured at scheduled times on Cycle 1 Day 21, including a pre-dose measurement and post-dose samples through 12 hours. In general, measuring concentrations over time can describe exposure patterns; the maximum concentration is one feature of that pattern. It does not summarize every aspect of exposure, such as total exposure across an interval, and it is not a clinical tumor outcome.
Geometric summaries are often used for positive measurements whose variation is multiplicative or whose distribution is skewed. A geometric mean is calculated on a logarithmic scale and transformed back to the original scale. A geometric coefficient of variation expresses spread relative to the geometric mean. This is a general explanation; the record does not provide the observed values, the underlying distribution, or details of any transformations used in analysis.
Why does the dose-escalation design matter?
Dose escalation is used in early-phase research to evaluate tolerability across dose combinations and inform subsequent dose selection. This study lists three refametinib/regorafenib schedules. The cohorts are not randomized controls, and the registry states that the arms are not a like-for-like control comparison. As a general principle, non-randomized groups can differ in ways that affect observed outcomes. A numerical difference between cohort summaries would not automatically demonstrate that a dose change caused the difference.
Open-label status means masking is recorded as absent. In general, lack of masking can influence assessments that depend on judgment or reporting. Pharmacokinetic concentrations are laboratory measurements, but the record does not describe assay procedures, laboratory blinding, quality-control methods, or other safeguards. Those details are not reported in the registry record.
What can be inferred from the analysis populations?
The primary endpoint names a pharmacokinetic analysis set for multiple dose. This indicates the intended population for that endpoint, but the registry does not give its size, membership rules, or the number with measurements at each sampling time. It also does not specify how missed samples, concentrations below a quantification limit, protocol deviations, or withdrawals were handled. Without these details and the outcome values, the precision and representativeness of the primary estimate cannot be assessed.
The registry separately gives numbers at risk and affected for serious and other adverse events within each cohort. Those denominators describe the reported safety figures; they are not necessarily the same as the primary pharmacokinetic analysis population. The record does not define the event-collection period or provide patient-level exposure time. Consequently, the safety counts should not be interpreted as rates per unit of treatment time.
What role do confidence intervals and variability measures play?
In general, a confidence interval describes the uncertainty around an estimated quantity under the assumptions of the analysis. A narrow interval indicates greater precision than a wide interval, while neither interval width nor statistical significance alone determines clinical importance. The registry record does not provide confidence intervals for the primary endpoint or the listed safety outcomes. The geometric coefficient of variation is a variability summary, not a confidence interval, and the two should not be conflated.
Over-dispersion is a general issue when observed variability exceeds what a simple statistical model expects. In count or event data, an inadequate model for variability can make standard errors too small and uncertainty appear less than it is. The record does not report a model for count outcomes, an over-dispersion assessment, or any inferential analysis of the adverse-event counts, so no such analysis should be presumed.
What do the planning assumptions say?
The registry describes a sample-size planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It specifies that the effect to detect is a stated fraction of the observed effect; for time-to-event planning, the stated quantity is total events, while other illustrations are participants per arm. These are planning assumptions, not observed power. They are not reported as the original trial’s design justification. Moreover, this trial is non-randomized and has no like-for-like control comparison, so a two-arm planning illustration should not be mistaken for a description of the study’s actual cohort design. The registry does not report a sample-size justification for the trial itself.
The record does not report inferential tests, estimation methods beyond the endpoint summaries, confidence intervals, adjustment covariates, interim analyses, or a prespecified statistical analysis plan. Accordingly, no unreported model or hypothesis-testing procedure should be attributed to the study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 24 outcome measures, including the primary refametinib concentration measure and secondary pharmacokinetic outcomes for refametinib, regorafenib, and their metabolites. Several outcomes concern different concentration summaries or sampling times. When many outcomes are examined, the chance of finding an apparently notable result by chance can increase. General approaches to multiplicity include prespecifying a primary outcome, adjusting significance thresholds, or interpreting secondary analyses as exploratory. The registry record does not report a multiplicity strategy, a hierarchy among secondary outcomes, or a family of hypotheses for formal testing.
The record lists no analyses. It therefore does not provide outcome estimates, test results, confidence intervals, or a clear account of which endpoints were analyzed. The absence of reported analyses does not establish whether analyses were performed outside the registry; it means their methods and results are not reported in the registry record.
Missing-data handling is not reported. The primary endpoint names a pharmacokinetic analysis set, but eligibility criteria for that set, the number with usable samples, and the handling of missing or unusable measurements are not given. The record does not say how incomplete sampling schedules or withdrawals affected analyses. No imputation method or sensitivity analysis is described.
The registry states that the primary endpoint uses the pharmacokinetic analysis set for multiple dose. It gives cohort-specific safety denominators, but does not define other analysis populations or explain whether they differ from enrollment. The statistical analysis plan, including any planned interim analyses, is not reported in the registry 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 |
|---|---|---|---|
| Ph1b - Refametinib/Regorafenib Cohort 0 | 0 | 2 | 0.0% |
| Ph1b - Refametinib/Regorafenib Cohort -1 | 4 | 7 | 57.1% |
| Ph1b - Refametinib/Regorafenib Cohort -1a | 5 | 11 | 45.5% |
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 |
|---|---|
| Ph1b - Refametinib/Regorafenib Cohort 0 | 2 of 2 |
| Ph1b - Refametinib/Regorafenib Cohort -1 | 7 of 7 |
| Ph1b - Refametinib/Regorafenib Cohort -1a | 11 of 11 |
Most frequent serious adverse event terms
| Event term | Organ system | Ph1b - Refametinib/Regorafenib Cohort 0 | Ph1b - Refametinib/Regorafenib Cohort -1 | Ph1b - Refametinib/Regorafenib Cohort -1a |
|---|---|---|---|---|
| Blood creatine phosphokinase increased | Investigations | 0 of 2 | 0 of 7 | 2 of 11 |
| Cholangitis | Hepatobiliary disorders | 0 of 2 | 1 of 7 | 0 of 11 |
| Urinary tract infection | Infections and infestations | 0 of 2 | 1 of 7 | 0 of 11 |
| Wound infection | Infections and infestations | 0 of 2 | 0 of 7 | 1 of 11 |
| Troponin increased | Investigations | 0 of 2 | 1 of 7 | 0 of 11 |
| Dehydration | Metabolism and nutrition disorders | 0 of 2 | 1 of 7 | 0 of 11 |
| Hypokalaemia | Metabolism and nutrition disorders | 0 of 2 | 0 of 7 | 1 of 11 |
| Pancreatic carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 2 | 0 of 7 | 1 of 11 |
| Acute kidney injury | Renal and urinary disorders | 0 of 2 | 0 of 7 | 1 of 11 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 0 of 2 | 0 of 7 | 1 of 11 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ph1b - Refametinib/Regorafenib Cohort 0 | Ph1b - Refametinib/Regorafenib Cohort -1 | Ph1b - Refametinib/Regorafenib Cohort -1a |
|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 2 of 2 | 4 of 7 | 9 of 11 |
| Fatigue | General disorders | 2 of 2 | 4 of 7 | 8 of 11 |
| Anaemia | Blood and lymphatic system disorders | 1 of 2 | 3 of 7 | 4 of 11 |
| Aspartate aminotransferase increased | Investigations | 2 of 2 | 0 of 7 | 6 of 11 |
| Constipation | Gastrointestinal disorders | 1 of 2 | 3 of 7 | 3 of 11 |
| Blood creatine phosphokinase increased | Investigations | 0 of 2 | 2 of 7 | 5 of 11 |
| Hypoalbuminaemia | Metabolism and nutrition disorders | 2 of 2 | 0 of 7 | 5 of 11 |
| Decreased appetite | Metabolism and nutrition disorders | 1 of 2 | 1 of 7 | 5 of 11 |
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | 2 of 2 | 2 of 7 | 3 of 11 |
| Rash | Skin and subcutaneous tissue disorders | 1 of 2 | 1 of 7 | 5 of 11 |
Serious events with the highest arm-level rate: blood creatine phosphokinase increased (18.2%), cholangitis (14.3%), urinary tract infection (14.3%), troponin increased (14.3%), dehydration (14.3%).
The registry reports serious adverse events and other adverse events separately for each dose cohort. For Cohort 0, 2 were at risk of serious events and 0 were affected; for Cohort -1, 7 were at risk and 4 were affected; for Cohort -1a, 11 were at risk and 5 were affected. For other adverse events, the recorded at-risk and affected counts are 2 and 2 in Cohort 0, 7 and 7 in Cohort -1, and 11 and 11 in Cohort -1a. These are cohort-specific counts, not a randomized comparison.
The record also lists serious adverse-event terms with percentages: Blood creatine phosphokinase increased, 18.2%; Cholangitis, 14.3%; Urinary tract infection, 14.3%; Troponin increased, 14.3%; Dehydration, 14.3%; and Hypertension, 14.3%. The registry excerpt does not give the denominator or further context for these term percentages, so they should not be assigned to a particular cohort or treated as comparable rates without that context.
Adverse-event counts describe events recorded under the study’s reporting definitions; they do not by themselves establish causation. The registry does not report event timing, duration, severity grading beyond the serious-event classification, exposure-adjusted rates, or a causal assessment. Death counts are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
This record establishes that a non-randomized, open-label dose-escalation study of refametinib with regorafenib was registered for patients with advanced or metastatic cancer. It describes three dose schedules and names a pharmacokinetic primary endpoint. It also reports selected cohort-level adverse-event counts and serious adverse-event terms. These registry entries describe the study’s scope and the material recorded, not a complete evaluation of the combination.
The primary endpoint is a measure of refametinib plasma concentration after multiple dosing. Because its numerical result is not reported in the registry record, readers cannot determine the observed geometric mean or variability from this information. The record likewise does not provide efficacy results, a randomized comparison, or an inferential analysis that would support conclusions about tumor outcomes or relative treatment effects.
The safety information can be read as counts of participants at risk and affected within the listed cohorts, alongside terms and percentages for serious adverse events. The design and reporting details do not establish whether an event was caused by treatment, how long participants were exposed, or whether the cohorts differed in ways relevant to event occurrence. Cohort counts should not be converted into a comparative claim about dose or treatment.
The terminated status is a registry status, not an explanation of why the study ended. Its reason is not reported in the registry record. The stated Phase 2 plan should also be distinguished from evidence that a Phase 2 evaluation occurred: the record’s title describes a Phase 1b/2 study, while its phase field lists PHASE1, and no Phase 2 efficacy results are included.
In short, the registry supports a description of the planned early-phase dose-escalation work and some reported safety data. It does not establish whether the combination is effective, whether one dose is preferable, or why the study was terminated. Those questions require results and methodological details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable because its stated primary endpoint is pharmacokinetic rather than a direct measure of tumor response. Early-phase research often needs to characterize drug exposure while evaluating dose combinations. Here, the registered endpoint focuses on the maximum plasma concentration of refametinib after multiple dosing, with geometric summaries and a defined sampling schedule. Understanding what an endpoint measures prevents a concentration result from being mistaken for evidence of clinical efficacy.
The dose-escalation structure also matters. Cohorts received different combinations of refametinib and regorafenib, but allocation was non-randomized and the study was uncontrolled. These features mean the cohort structure is suited to describing dose-escalation experience, not to a clean causal comparison between doses. Any comparisons would need to account for the design and the possibility that cohort membership reflects more than the dose received.
The record illustrates the distinction between a trial’s planned objectives and its publicly reported results. It states an aim of identifying a maximum tolerated dose and a recommended Phase 2 dose, and describes a planned Phase 2 evaluation in specified cancer types. Yet the primary pharmacokinetic result and efficacy results are not reported in the registry record. Registry status and design details alone cannot fill that evidentiary gap.
Finally, the listed safety counts are informative as descriptive reporting but lack context needed for stronger comparisons, such as event timing and exposure duration. This is a useful reminder that denominators, analysis populations, endpoint definitions, and the completeness of reporting all shape what can be inferred. Statistical interpretation depends not only on the named outcomes, but also on the study design and the detail of the reported results.
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 gives limited outcome information for this study. It identifies the primary pharmacokinetic endpoint and its summary measures, but does not report its numerical result. It lists 24 outcome measures and no analyses. The listed efficacy objective is not accompanied by tumor response results, and no formal comparative estimates are reported.
The non-randomized, uncontrolled, open-label design limits comparisons across cohorts. Dose groups are not like-for-like control groups, and the registry does not give adjustment methods that might address differences among participants. The record also does not provide detailed eligibility criteria, baseline characteristics, or participant flow by cohort and analysis population.
For the primary outcome, the registry names a multiple-dose pharmacokinetic analysis set but does not report its size, inclusion rules, completeness of sampling, or missing-data handling. Assay details and the statistical analysis plan are also not reported. The registry does not state how multiplicity was handled across the listed outcomes or whether interim analyses took place.
Safety reporting is limited to cohort counts and listed serious adverse-event terms with percentages. Event timing, duration, exposure-adjusted incidence, severity grading beyond the serious classification, and causal assessment are not reported. The denominator context for the term percentages is not specified in the record, and death counts are not reported.
The study is marked terminated, but its reason is not reported in the registry record. The record does not clarify the completion of the planned Phase 2 portion. These limitations mean the available information can describe the registered design and selected safety figures, but cannot support a comprehensive assessment of exposure, efficacy, comparative safety, or the reasons for study completion.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Refametinib (BAY86-9766) in Combination With Regorafenib (Stivarga, BAY73-4506) in Patients With Advanced or Metastatic Cancer — NCT02168777.
- Registry record last updated 2018-01-26; status last verified 2017-06.
- Results first posted 2018-01-26.
- 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
NCT02168777 is a Bayer-sponsored, non-randomized, open-label, uncontrolled dose-escalation study of refametinib with regorafenib in advanced or metastatic cancer. The title describes a Phase 1b/2 study, while the phase field lists PHASE1. The registry records enrollment of 20 and a terminated status.
Three Phase 1b cohorts are described, using different dose combinations on a 3 weeks on/1 week off schedule. The primary listed outcome is refametinib maximum plasma concentration after multiple dosing, summarized by geometric mean and geometric coefficient of variation. Its numerical result is not reported in the registry record. The record includes cohort-level serious and other adverse-event counts, but does not report efficacy analyses or the reason for termination.