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 Ramucirumab + Pembrolizumab in Patients With Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (NCT03650764), a non-randomized phase 1, phase 2 study. The primary outcome is Recommended Phase 2 Dose (RP2D) of Ramucirumab Combined With Fixed Dose Pembrolizumab (Phase I Participants Only).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03650764 |
| Phase | Phase 1, Phase 2 |
| Condition | Head and Neck Squamous Cell Carcinoma |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 43 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Washington University School of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
People with recurrent or metastatic head and neck squamous cell carcinoma. The study record describes a Phase I dose-finding part and a Phase II part for participants receiving the combination as first-line therapy.
Intervention
Ramucirumab combined with pembrolizumab. The Phase II regimen used ramucirumab at 10 mg/kg and pembrolizumab at 200 mg, administered intravenously on Day 1 of each 21-day cycle.
Comparator
There was no randomized control group. The study was nonrandomized, and the Phase II portion was single arm. The registry’s hypothesis compares the combination conceptually with PD-1 inhibition alone, but that comparison was not tested within this trial.
Primary question
The Phase I primary outcome was the recommended Phase 2 dose of ramucirumab when combined with fixed-dose pembrolizumab. The record defines that dose using dose-limiting toxicities during the first treatment cycle. The Phase II objective was to assess complete and partial tumor responses with the combination, but response results are not reported in the registry record.
The study therefore addresses two related but distinct questions. First, can a ramucirumab dose be selected for use with pembrolizumab under the protocol’s toxicity rule? Second, what tumor response rates are observed when that combination is given in the Phase II group? The first question is a dose-selection problem, not a comparison of treatment effects. The second is an estimation question in a single treatment group; without a concurrent control group, it cannot establish how outcomes compare with another regimen.
3. Trial Design
This was a prospective, nonrandomized, unmasked study with a sequential Phase I and Phase II structure. The registry lists enrollment of 43 and the condition as head and neck squamous cell carcinoma. The listed status is completed. The record gives a start date of 2019-05-29 and a primary completion date of 2026-05-11.
The Phase I portion used a dose-de-escalation approach to select a recommended Phase 2 dose of ramucirumab while pembrolizumab was fixed. The protocol description says three participants would be treated at each dose level. De-escalation to the next dose level was to occur if 2 or more participants experienced dose-limiting toxicities attributed to ramucirumab during cycle 1. The primary endpoint defines the recommended dose as the highest dose level at which fewer than 2 participants in a cohort of three experience a dose-limiting toxicity during the first cycle.
The Phase II portion was a single-arm study using the dose identified as the recommended Phase 2 dose. Its arm description lists ramucirumab at 10 mg/kg and pembrolizumab at 200 mg, both intravenously on Day 1; each cycle was 21 days. The Phase II primary objective was to determine complete and partial tumor response rates among participants receiving the combination as first-line therapy.
Because there was no random allocation or control group, comparisons between the Phase I and Phase II groups are not like-for-like treatment comparisons. They served different parts of a sequential study and may differ in eligibility, treatment purpose, or follow-up. Details of eligibility criteria beyond the broad cancer population, allocation procedures beyond the nonrandomized designation, and reasons for individual participants’ inclusion in each phase are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab |
|---|---|---|
| STARTED | 3 | 40 |
| COMPLETED | 3 | 33 |
| NOT COMPLETED | 0 | 7 |
Reasons for not completing this period:
| Reason | Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab |
|---|---|---|
| Death | 0 | 2 |
| Did not start treatment - infection | 0 | 1 |
| Did not start treatment - withdrawal by subject | 0 | 2 |
| Withdrawal by Subject | 0 | 2 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab | Total |
|---|---|---|---|---|
| Participants analysed | 3 | 40 | 43 | |
| Age, Continuous | years | 63 (51 to 72) | 64 (37 to 80) | 64 (37 to 80) |
| Sex: Female, Male: Female | Participants | 0 | 4 | 4 |
| Sex: Female, Male: Male | Participants | 3 | 36 | 39 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 40 | 43 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 4 | 4 |
| Race (NIH/OMB): White | Participants | 3 | 35 | 38 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 3 | 40 | 43 |
6. Primary Endpoint
The registry lists the primary outcome as Recommended Phase 2 Dose (RP2D) of Ramucirumab Combined With Fixed Dose Pembrolizumab (Phase I Participants Only), measured over Completion of first cycle of treatment (each cycle is 21 days) for all participants enrolled in Phase I portion of study (estimated to be 2.5 months).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg/kg |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the recommended Phase 2 dose of ramucirumab when combined with fixed-dose pembrolizumab, expressed in mg/kg and assessed for Phase I participants. The registry defines the recommended dose as the highest dose level at which fewer than 2 participants in a cohort of three experience a dose-limiting toxicity during the first 21-day cycle. The stated timeframe is completion of the first cycle for all Phase I participants, estimated in the record to be 2.5 months.
This endpoint is used in early-phase combination studies to guide dose selection. Rather than asking whether the regimen improves an outcome relative to another treatment, it applies a prespecified toxicity rule to identify a dose for later study. The rule is cohort-based: observed toxicities during the defined cycle determine whether a dose level meets the criterion. Such a result is specific to the regimen and protocol conditions; it is not, by itself, an estimate of antitumor benefit.
The Phase II arm description identifies 10 mg/kg as the recommended Phase 2 dose determined during Phase I. However, the registry does not report the Phase I dose-limiting toxicity counts by dose level or a separate numerical primary endpoint result. Thus the dose used in Phase II is stated, but the underlying dose-selection observations cannot be independently evaluated from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a study intended to progress from dose selection to an initial assessment of tumor response. Its primary endpoint is a dose, not a response rate, survival measure, or direct estimate of comparative treatment effect. The Phase II arm description states that 10 mg/kg ramucirumab was the recommended Phase 2 dose and was used with 200 mg pembrolizumab. The underlying Phase I toxicity counts and a numerical primary endpoint result are not reported in the registry record.
The Phase II objective was to determine complete and partial response rates, but response counts and response-rate estimates are not reported in the record. Nor are numerical estimates reported for the listed secondary outcomes, which include duration of response, progression-free survival, overall survival, and quality-of-life measures. Consequently, the registry entry does not permit assessment of the magnitude or precision of tumor response or other clinical outcomes. A stated objective is not the same as a posted result.
The study’s nonrandomized, single-arm Phase II design also limits interpretation. An observed outcome in a single treatment group can describe what occurred among the participants analyzed, but it does not show what would have occurred under a different treatment. The sponsor’s stated hypothesis compares inhibition of angiogenesis and PD-1 with PD-1 inhibition alone. The trial does not include a randomized PD-1-only arm, so that hypothesis is not directly tested by a between-group comparison within this study.
The adverse-event summaries report serious adverse events for 2 of 3 participants in the Phase I Dose Level 1 group and 26 of 37 participants in the Phase II group. Other adverse events are listed for 3 of 3 and 37 of 37 participants, respectively. Deaths are listed for 3 and 32 participants. These are descriptive registry entries, not a randomized safety comparison. The registry record does not establish whether an event or death was caused by study treatment, and the Phase I and Phase II groups should not be treated as equivalent comparison arms.
The registry lists 12 outcome measures and 0 analyses. This means the entry names outcomes but does not post analytic estimates for them. The absence of results in the registry record is not evidence that an outcome did not occur or that the treatment had no effect. It means the outcome’s size, uncertainty, and analysis cannot be assessed from this record. The status is listed as completed, but completion status alone does not supply efficacy estimates, toxicity attribution, or the statistical details needed to interpret them.
9. Statistical Methods Explained
How does the dose-selection rule work?
The Phase I rule is a protocol decision rule based on dose-limiting toxicity during the first cycle. The record specifies cohorts of three and a threshold of 2 or more participants with qualifying toxicity for de-escalation. It defines the recommended dose as the highest dose level at which fewer than 2 participants in a cohort of three experience a dose-limiting toxicity. In general, a rule of this kind converts observed events in small cohorts into a dose-selection decision. It does not estimate a comparative treatment effect, and its result depends on the toxicity definition, observation window, attribution process, and dose levels used.
The registry describes the rule but does not report the observed dose-limiting toxicities by dose level, the full dose-level sequence, or the details of how incomplete first-cycle observation was handled. The Phase II description identifies 10 mg/kg as the recommended dose selected during Phase I. Without the underlying observations, readers cannot audit how the rule led to that selection. The timeframe is completion of the first cycle for Phase I participants, with 2.5 months given as the estimate for all such participants.
What does the sequential design contribute?
The sequential structure lets the study address dose selection before the Phase II assessment. Phase I’s primary task was to select a ramucirumab dose with fixed-dose pembrolizumab; Phase II then used the selected dose and aimed to estimate tumor response. This is not a randomized treatment comparison. In general, sequential phases have different purposes and can involve different participant groups, so their results should be interpreted within each phase rather than as if the groups were concurrent control and treatment arms.
The Phase II study was single arm and unmasked. A single-arm response estimate can describe observed responses under the study regimen, but it cannot separate treatment effects from other influences on outcomes. Unmasked treatment assignment can also matter for outcomes involving judgment or participant reporting. The registry record does not report an independent blinded response review or other procedures to address this issue.
What can time-to-event methods tell readers?
The registry lists Kaplan-Meier estimates of median progression-free survival and overall survival among Phase II participants, and duration of response as a secondary outcome. In general, the Kaplan-Meier method estimates the pattern of time to an event while accounting for participants whose event time is not observed during follow-up through censoring. The median is the time by which the estimated event-free proportion reaches the midpoint of the distribution. Its interpretation depends on the event definition, follow-up, censoring, and analysis population.
Those general properties do not supply a result for this study. The registry record does not report the median estimates, confidence intervals, event counts for these endpoints, censoring details, or the analysis populations used. It also does not report the event definitions beyond the endpoint names. The degree of precision and maturity of these time-to-event outcomes therefore cannot be assessed from the record.
What does statistical precision mean?
In general, a confidence interval expresses the uncertainty around an estimated quantity under the assumptions of the method used. A narrower interval indicates greater statistical precision, while a wider interval indicates less precision; neither property alone establishes clinical importance or eliminates bias. For a response proportion, uncertainty depends in part on the number of participants with an evaluable response and the number of observed responses. For time-to-event estimates, precision depends on the event and follow-up information as well as the analysis method. The registry record does not post confidence intervals for the primary or secondary outcomes.
The registry lists 0 analyses, so no analysis results, model estimates, or inferential tests are posted in the record. It does not report a statistical analysis plan, hypothesis-testing procedure, confidence-interval method, or prespecified handling of response unevaluability. These details are not reported in the registry record and should not be inferred from the named outcomes.
How should planning calculations be understood?
The planning-illustration description says calculations use the observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also says the effect to detect is a stated fraction of the observed effect; for time-to-event outcomes, the calculation concerns the total number of events, while other calculations are expressed as participants per arm. Such illustrations are planning exercises, not observed power and not evidence that the trial was designed with those assumptions. The registry record does not report that these assumptions were the trial’s original design justification.
In general, a planning calculation translates assumptions about a target effect, variability, allocation, and error rates into a projected study size. Changing the assumed effect changes the projected size, and the calculation may not reflect the actual design or available information. The study itself is nonrandomized and its Phase II portion is single arm, whereas the described planning assumptions refer to equal allocation between two arms. The planning description therefore should not be read as turning the trial into a two-arm comparison. No sample-size justification for the trial is reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists outcomes spanning dose selection, adverse events, tumor response, duration of response, progression-free survival, overall survival, and several quality-of-life measures. Multiple outcomes create interpretive choices: the more outcomes examined, the greater the chance that at least one result may appear notable by chance if formal significance testing is used. In general, a prespecified hierarchy or multiplicity adjustment can address that issue, but the registry record does not report a multiplicity strategy or a hierarchy for interpreting secondary outcomes.
The Phase I dose-selection endpoint and Phase II response objective are distinct. The primary Phase I outcome is the recommended dose, while Phase II’s primary objective concerns complete and partial response rates. The registry does not provide the Phase II response analysis, the response assessment criteria, or the statistical method used to calculate response rates. It also does not report confidence intervals or hypothesis tests for those outcomes.
Missing-data and analysis-population details are not reported in the registry record. This includes how participants with no post-baseline assessment, incomplete cycles, missing quality-of-life questionnaires, or incomplete follow-up were handled. The named Phase II measures apply to Phase II participants, but precise analysis-set definitions, exclusions, and censoring rules are not provided. The record does not state whether analyses were planned for all enrolled participants, only evaluable participants, or another defined group.
The entry lists 12 outcome measures and 0 analyses. This supports a distinction between outcomes named for collection and results available for interpretation: the record lists the former but does not post the latter. Interim-analysis procedures, stopping rules beyond the Phase I dose-de-escalation criterion, and a full statistical analysis plan are 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 |
|---|---|---|---|
| Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | 2 | 3 | 66.7% |
| Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab | 26 | 37 | 70.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | 3 of 3 |
| Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab | 32 of 37 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | 3 of 3 |
| Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab | 37 of 37 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab |
|---|---|---|---|
| Lung infection | Infections and infestations | 0 of 3 | 10 of 37 |
| Oral hemorrhage | Gastrointestinal disorders | 0 of 3 | 4 of 37 |
| Dysphagia | Gastrointestinal disorders | 0 of 3 | 3 of 37 |
| Death due to disease progression | General disorders | 0 of 3 | 3 of 37 |
| Sepsis | Infections and infestations | 0 of 3 | 3 of 37 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 3 of 37 |
| Colonic obstruction | Gastrointestinal disorders | 0 of 3 | 2 of 37 |
| Gastritis | Gastrointestinal disorders | 0 of 3 | 2 of 37 |
| Infusion related reaction | General disorders | 0 of 3 | 2 of 37 |
| Dehydration | Metabolism and nutrition disorders | 0 of 3 | 2 of 37 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase I Dose Level 1 : Ramucirumab (10 mg/kg) + Pembrolizumab | Phase II: Ramucirumab (10 mg/kg) + Pembrolizumab |
|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | 2 of 3 | 36 of 37 |
| Lymphocyte count decreased | Investigations | 3 of 3 | 34 of 37 |
| Fatigue | General disorders | 3 of 3 | 31 of 37 |
| Proteinuria | Renal and urinary disorders | 2 of 3 | 29 of 37 |
| Dysphagia | Gastrointestinal disorders | 3 of 3 | 24 of 37 |
| Hyponatremia | Metabolism and nutrition disorders | 2 of 3 | 23 of 37 |
| Weight loss | Investigations | 3 of 3 | 21 of 37 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 2 of 3 | 22 of 37 |
| Hypertension | Vascular disorders | 3 of 3 | 19 of 37 |
| Hypothyroidism | Endocrine disorders | 3 of 3 | 18 of 37 |
Serious events with the highest arm-level rate: hemolytic uremic syndrome (33.3%), catheter related infection (33.3%), pneumocephalus (33.3%), lung infection (27.0%), oral hemorrhage (10.8%).
The serious-adverse-event summaries list 2 of 3 participants in Phase I Dose Level 1 and 26 of 37 participants in Phase II as affected. The registry also lists other adverse events for 3 of 3 participants in Phase I and 37 of 37 in Phase II. These are descriptive counts within the registry’s two study groups; they are not a randomized comparison, and the groups should not be interpreted as treatment and control arms.
The record lists deaths for 3 participants in Phase I Dose Level 1 and 32 in Phase II. A death count in an adverse-event summary does not by itself show that the death was caused by treatment. The causal relationship between reported events or deaths and study treatment is not reported in the registry record. Follow-up duration and the timing of these events are also not given in the adverse-event summary.
The listed serious-event terms include hemolytic uremic syndrome (33.3%), catheter related infection (33.3%), and pneumocephalus (33.3%), followed by lung infection (27.0%), oral hemorrhage (10.8%), and dysphagia (8.1%). These percentages are reproduced as registry entries; the record does not clarify their denominator or provide event counts for each listed term. They should not be treated as estimates of treatment-attributable risk without additional information on definitions, exposure, follow-up, and attribution.
12. What the Result Does, and Does Not, Mean
The registry record establishes the study’s intended sequence: Phase I dose selection followed by a single-arm Phase II assessment of the combination. It identifies 10 mg/kg ramucirumab as the recommended Phase 2 dose used with pembrolizumab at 200 mg. It also describes the first-cycle toxicity rule that was to guide selection. These details explain the treatment regimen and decision framework, but the underlying Phase I toxicity observations are not reported in the record.
The Phase II objective was to determine complete and partial response rates in people receiving the combination as first-line therapy for recurrent or metastatic disease. The registry does not report response counts or rates. It also names survival, response-duration, adverse-event, and quality-of-life outcomes without posting their estimates. Therefore, the record does not show the magnitude or precision of efficacy outcomes, and it does not allow readers to determine how outcomes varied over follow-up.
Even if response or survival estimates were available for this single treatment group, they would describe outcomes among the analyzed participants rather than establish a causal advantage over another treatment. The nonrandomized design and lack of a control group mean that the sponsor’s hypothesis about adding angiogenesis inhibition to PD-1 inhibition is not directly tested against PD-1 inhibition alone within this trial.
The adverse-event counts describe reported events in the Phase I and Phase II groups. They do not establish that the events were caused by the study drugs. The record’s completed status indicates the study is listed as completed, but completion does not substitute for posted outcome results, analysis details, or causal attribution. The results support only the factual description of the study’s design, selected Phase II dose, and registry-listed safety counts; broader treatment conclusions are not established by this record.
13. Why This Trial Matters Statistically
This trial is statistically notable for combining a dose-selection question with a later outcome-assessment question in a sequential design. The Phase I portion uses a cohort toxicity rule to select a dose, while Phase II uses that dose to pursue an estimate of tumor response. These are different inferential tasks: one makes a protocol decision based on early toxicity, and the other aims to describe outcomes in a treatment group.
The design also illustrates why a stated hypothesis and a tested comparison are not interchangeable. The investigators hypothesized that inhibiting angiogenesis and PD-1 would be more effective than inhibiting PD-1 alone. The Phase II portion was single arm and the trial was nonrandomized, so it does not contain the concurrent randomized comparator needed to directly estimate that contrast. Statistical interpretation must follow the actual design rather than the motivation for conducting the study.
The primary endpoint is itself a useful reminder that early-phase outcomes may be dose-oriented rather than efficacy-oriented. The endpoint’s operational definition makes the decision rule clear, but meaningful appraisal also requires the observed toxicity data and details of the dose-level process. The record identifies 10 mg/kg as the Phase II dose while not reporting the dose-specific toxicity counts that led to its selection.
Finally, naming a set of secondary outcomes is not equivalent to reporting their results. The registry lists 12 outcome measures but 0 analyses. Without numerical estimates and uncertainty measures, readers cannot judge the precision of response, survival, or quality-of-life findings. That gap matters statistically because design, endpoint definitions, and analysis results are all needed to distinguish an intended question from an answer supported by data.
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 most important limitation for interpreting the registry record is the absence of numerical outcome analyses. Although the study is listed as completed and describes 12 outcome measures, the record lists 0 analyses. No response rates, survival estimates, quality-of-life changes, confidence intervals, or formal tests are reported. A completed study status cannot fill that evidence gap.
The Phase I dose-selection process is described, but dose-limiting toxicity counts by dose level and the details of the dose-level sequence are not reported. The Phase II arm description identifies 10 mg/kg as the recommended dose, but readers cannot reconstruct the dose-selection decision from the registry entry. The definition and adjudication procedures for dose-limiting toxicity are not further detailed in the record.
The Phase II portion is single arm and the trial is nonrandomized, so there is no concurrent control group for estimating a comparative treatment effect. The Phase I and Phase II groups have different roles and are not interchangeable comparison groups. The registry also does not report response assessment criteria, analysis populations, missing-data handling, follow-up completeness, or detailed censoring rules for time-to-event outcomes.
The safety summaries list counts by study group and selected serious-event terms with percentages, but do not report event-level attribution, timing, exposure-adjusted rates, or the denominator for the listed term percentages. The counts should therefore be read descriptively. A statistical analysis plan, multiplicity approach, interim analyses, and trial sample-size justification are not reported in the registry record. These omissions limit independent assessment of both the planned analyses and the reliability and precision of any conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Ramucirumab + Pembrolizumab in Patients With Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma — NCT03650764.
- Registry record last updated 2026-08-17; status last verified 2026-08.
- Results first posted 2026-08-17.
- 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
NCT03650764 is listed as a completed, nonrandomized, unmasked, sequential Phase I and Phase II trial sponsored by Washington University School of Medicine. It enrolled 43 participants with recurrent or metastatic head and neck squamous cell carcinoma. The study started on 2019-05-29, with primary completion listed as 2026-05-11.
Phase I was designed to select the recommended Phase 2 dose of ramucirumab using first-cycle dose-limiting toxicity. The Phase II arm used ramucirumab at 10 mg/kg with pembrolizumab at 200 mg, intravenously on Day 1 of 21-day cycles. Phase II was single arm and aimed to determine complete and partial tumor response rates.
The registry lists 12 outcome measures and 0 analyses. It identifies 10 mg/kg as the recommended Phase 2 dose but does not report the dose-specific toxicity observations or numerical efficacy estimates. Serious adverse-event and death counts are listed by group, without treatment-causality information. Accordingly, the record describes the design and regimen but does not provide enough results to assess efficacy estimates or their precision.