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 Pharmacokinetics of Ridaforolimus (MK-8669) in Chinese Participants (MK-8669-059) (NCT01380184), a na phase 1 study. The primary outcome is Lag Time (Tlag) of Ridaforolimus: Day 1.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01380184 |
| Phase | Phase 1 |
| Condition | Cancer, Advanced |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Merck Sharp & Dohme LLC |
| Results posted | Yes |
2. Clinical Question
Population
Chinese participants with advanced cancer were enrolled. The primary pharmacokinetic endpoint applies to the Pharmacokinetic Evaluable population, which the registry record describes in terms of protocol compliance and qualifying blood samples. Further details of that population are not reported in the registry record.
Intervention
Participants received oral ridaforolimus, also identified as MK-8669. Part 1 assessed pharmacokinetics, safety, and tolerability after a single dose and multiple 40 mg doses. The optional second part allowed participants to continue treatment using a weekly regimen.
Comparator
There was no separate comparator group: the registry describes a single-group, unmasked study. The design therefore does not provide a like-for-like control comparison. Changes over time or comparisons with other treatments cannot be established from the design described in the registry record.
Primary question
The primary endpoint concerns the lag time, or Tlag, for ridaforolimus to appear in systemic circulation after oral administration. The registry specifies the median and full range after the single dose on Cycle 1 Day 1. A numerical endpoint result is not reported in the registry record, so the observed lag time cannot be characterized from that record.
The study’s focus is pharmacokinetic: it asks about the timing of drug appearance in circulation, rather than whether treatment changes a cancer outcome. The record also lists a secondary outcome concerning participants who experienced clinical and laboratory adverse events. These safety outcomes address a different question from Tlag and should be read separately.
3. Trial Design
The registry identifies this as a Phase 1 study of ridaforolimus in Chinese participants with advanced cancer. It was a single-group study with no masking and no randomized allocation. The registry lists enrollment of 15 participants and a completed status. The study dates recorded are 2011-07-05 for the start and 2011-10-20 for primary completion.
Part 1 was intended to assess pharmacokinetics, safety, and tolerability after single and multiple 40 mg doses. In the arm description, participants received an oral enteric-coated 40 mg tablet on Day 1, had no study treatment on Days 2-7, and then received two weekly sequences during Days 8-19. Each sequence consisted of 5 consecutive days of 40 mg ridaforolimus followed by 2 consecutive days off treatment. Part 2 was optional; the brief description says participants could continue with a weekly regimen of daily oral 40 mg doses for five consecutive days followed by two days off treatment.
Because there is no separate control group, the study describes observations among participants receiving ridaforolimus, not a randomized contrast between treatments. The unmasked design means masking is not part of the comparison framework. The registry record does not report a statistical analysis plan, an allocation procedure beyond listing allocation as not applicable, or a rationale for the enrollment target.
The primary outcome is Day 1 Tlag, recorded in hours and summarized as a median with a full range. Its scheduled observation period began before dosing and included various postdose time points up to 24 hours on Cycle 1 Day 1. The registry lists eight outcome measures overall, but a numerical result for the primary outcome is 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.
Part 1 (19 Days)
| Stage | Ridaforolimus 40 mg |
|---|---|
| STARTED | 15 |
| COMPLETED | 14 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Ridaforolimus 40 mg |
|---|---|
| Adverse Event | 1 |
Washout Period (≥2 Days)
| Stage | Ridaforolimus 40 mg |
|---|---|
| STARTED | 14 |
| COMPLETED | 14 |
| NOT COMPLETED | 0 |
Part 2
| Stage | Ridaforolimus 40 mg |
|---|---|
| STARTED | 14 |
| COMPLETED | 0 |
| NOT COMPLETED | 14 |
Reasons for not completing this period:
| Reason | Ridaforolimus 40 mg |
|---|---|
| Adverse Event | 5 |
| Disease Progression | 8 |
| Lost to Follow-up | 1 |
Analysis population for the primary outcome, as defined in the registry: The Pharmacokinetic Evaluable population consisted of all participants who complied with the protocol sufficiently to ensure that these data will likely exhibit the effects of treatment according to the underlying scientific model and who had blood samples with concentrations above the lower limit of quantitation.
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 | Ridaforolimus 40 mg |
|---|---|---|
| Participants analysed | 15 | |
| Age, Continuous | years | 51.3 (SD 12.4) |
| Sex: Female, Male: Female | Participants | 10 |
| Sex: Female, Male: Male | Participants | 5 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 15 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: China | Participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Lag Time (Tlag) of Ridaforolimus: Day 1, measured over Cycle 1 Day 1: Predose and at various time points up to 24 hours postdose on Day 1; Cycle 1 is 19 days.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | Hours |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the lag time, Tlag, for ridaforolimus to appear in systemic circulation after oral administration. It is measured in hours. The endpoint description defines Tlag as the time taken for the drug to appear in systemic circulation following an oral dose. The registry specifies the median and full range, meaning the minimum and maximum, as the summary statistics for the single dose on Day 1.
Pharmacokinetic endpoints describe how a drug behaves in the body. Tlag focuses on the timing of initial appearance in circulation, rather than the amount of drug present over the entire dosing period or a clinical outcome. It is useful for characterizing the timing of absorption into systemic circulation after an oral dose. The scheduled measurements were taken before dosing and at various points up to 24 hours postdose on Cycle 1 Day 1.
A median gives a central value that is less affected by unusually early or late observations than a mean can be. The full range conveys the observed endpoints of the data, but does not show how observations are distributed between those endpoints. This distinction matters because a range can be wide even when most observations cluster closely, or narrow while leaving other features of the distribution unclear.
The registry record does not report the observed median or its range. It also does not report a confidence interval or another measure of uncertainty for this endpoint. Therefore, the endpoint definition and planned summary can be described, but the observed timing and its precision cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record establishes the study’s context and its primary pharmacokinetic question, but it does not provide a numerical result for Day 1 Tlag. The median and full range are the stated summaries, yet their observed values are not reported in the registry record. Without those values, it is not possible to describe the observed lag time, its range, or how variable it was among participants in the endpoint population.
The registry identifies enrollment of 15 participants, while the primary endpoint is specified for a Pharmacokinetic Evaluable population. Those are not necessarily interchangeable descriptions: the registry defines the endpoint population by protocol compliance and blood-sample criteria. The number included in the primary endpoint analysis is not reported in the registry record. No estimate of precision for Tlag, such as a confidence interval, is reported either.
The study has a single-group design. Its observations can characterize the participants who received ridaforolimus, but they do not estimate a treatment contrast against a concurrent control group. The record’s design therefore cannot by itself distinguish a treatment-related pattern from other explanations or establish how the pharmacokinetic profile compares with another population, dose, or treatment. Such comparisons would require evidence not described in this record.
The safety information is separate from the primary pharmacokinetic endpoint. The registry lists 4 participants at risk for serious adverse events and 4 affected, with 15 at risk and 15 affected for other adverse events. It also lists 3 deaths. These are counts recorded for the ridaforolimus group, not a controlled comparison. The serious-event terms listed include Neoplasm malignant at 13.3%, Disease progression at 6.7%, and Tracheal cancer at 6.7%. The record does not establish from these figures alone whether the terms overlap or how individual events relate to treatment.
These safety counts should be interpreted in the context of a study involving participants with advanced cancer and without a comparator group. The counts describe what the registry reports; they do not establish causation. The registry does not report detailed timing, event-level narratives, exposure-adjusted rates, or a comparative safety analysis in the registry record for this record.
In short, the available information supports a description of the study design, dose schedule, endpoint definition, and recorded safety counts. It does not support a numerical conclusion about the primary pharmacokinetic endpoint or a controlled assessment of treatment effects. The sponsor’s analysis is not presented in the registry record, and no adjusted or unadjusted primary analysis result is listed.
9. Statistical Methods Explained
Pharmacokinetic studies examine the time course of a drug in the body. In this study, the primary endpoint is Tlag after an oral dose. The endpoint is a timing measure: it concerns when ridaforolimus first appears in systemic circulation, according to the study’s measurement approach. It is not itself a measure of how much drug remains in the body, how long concentrations stay elevated, or whether a clinical condition changes.
What does the median describe?
The median is a measure of the center of a distribution. In general, it is the value that divides ordered observations into lower and upper portions. It can be a useful summary when data are skewed or contain unusually large or small observations, because it is less sensitive to extreme values than the arithmetic mean. Here, the registry says that Tlag is summarized by its median and full range. It does not report the observed median.
What does the full range add?
The full range is the minimum and maximum observed values. It shows the outer limits of the recorded observations, but not how densely observations fall within those limits. It does not by itself quantify precision, and it can be strongly influenced by extreme observations. For a fuller description of a distribution, analysts often consider additional summaries or a plot, but the registry record’s stated primary summary is the median and full range. Other distribution summaries for Tlag are not reported in the registry record.
What is a confidence interval?
In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the analysis method. Its width reflects both the amount of information available and the variability in the data, as well as the method used to construct it. A confidence interval is not the same as the range of individual observations: the range describes observed data values, while an interval for an estimate describes uncertainty about a population quantity. No confidence interval for the primary endpoint is reported in the registry record.
How does the design shape the analysis?
This is a single-group study, not a randomized comparison. Statistical summaries can describe the measured pharmacokinetic values among participants meeting the endpoint population criteria. In contrast, a treatment comparison would require a defined comparator and an analysis contrasting groups. Neither a comparator arm nor such a contrast is described in the registry record. The study is also unmasked, so masking does not provide a safeguard against awareness of treatment assignment; however, the primary endpoint is a pharmacokinetic measurement, and the record does not detail any additional procedures used to limit measurement or analysis bias.
The registry describes the Pharmacokinetic Evaluable population as participants who complied with the protocol sufficiently for the data to reflect treatment according to the underlying scientific model and who had qualifying blood samples. The exact lower-limit criterion is not fully available in the registry description, and the number analyzed for Tlag is not reported in the registry record. A population definition matters because endpoint summaries depend on which participants and samples qualify for inclusion.
What can the dose schedule tell us?
The schedule establishes when dosing and sampling were intended to occur. The primary endpoint uses observations from Cycle 1 Day 1, from predose through various points up to 24 hours postdose. Later dosing in Part 1 followed a sequence of treatment days and off-treatment days. This schedule provides context for the intended observations but does not substitute for the measured concentration data or the endpoint result. The actual sampling times and completeness of sampling are not reported in the registry record.
What are sample-size planning illustrations?
In general, a sample-size calculation describes the size of a future study under specified assumptions. It is a planning exercise, not a description of what the completed study observed and not proof that an analysis had a particular probability of detecting an effect. The registry describes its planning illustrations as based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also states that the effect to detect is a stated fraction of the observed effect. For time-to-event tables, the described quantity is a total event count; for other outcomes, it is participants per arm.
Those are general planning assumptions, not evidence that this single-group pharmacokinetic study used a two-arm design. The registry record does not report a trial-specific sample-size justification or the numerical values of the planning illustrations in the registry record for this record. Such illustrations should not be described as observed power. The observed effect itself is not reported for the primary endpoint, so it cannot be evaluated from the record.
Why does over-dispersion matter?
In general, over-dispersion occurs when the variability in data exceeds what a statistical model expects. If a model assumes less variability than the data contain, estimated uncertainty can be too small and inferential results can appear more precise than warranted. Analysts may use models or standard errors that account for extra variability when the outcome and design call for them. The registry record does not describe an over-dispersion assessment or a model for the primary endpoint, and it does not report an inferential analysis of Tlag.
The registry lists eight outcome measures and no analyses. Consequently, the method used to estimate or compare endpoint values, any adjustment for covariates, and any handling of unusual observations are not reported in the registry record. The endpoint’s stated descriptive summary can be explained, but a more detailed account of the analysis cannot be reconstructed from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists eight outcome measures, including the primary Tlag endpoint and a secondary outcome concerning clinical and laboratory adverse events. It lists no analyses. A multiplicity strategy for handling several outcomes or several statistical tests is not reported in the registry record. Without such a strategy, it is not possible to determine whether any family of tests was defined or how the chance of false-positive findings was addressed. No inferential result is reported for the primary endpoint in any case.
The record identifies the primary endpoint population as Pharmacokinetic Evaluable and describes eligibility in terms of protocol compliance and qualifying blood samples. It does not report how many participants contributed to the primary endpoint summary or how many samples were available at each planned time point. A detailed missing-data approach, including whether incomplete profiles were excluded or handled in another way, is not reported in the registry record.
The record also does not state whether any participants withdrew, whether any measurements were below the assay’s lower limit, or how such observations were handled. These details matter for pharmacokinetic summaries because incomplete or non-quantifiable measurements may affect which profiles can be evaluated. No assumptions about missingness should be inferred from the population definition alone.
The statistical plan, including any prespecified analysis methods, sensitivity analyses, or interim analyses, is not reported in the registry record. Therefore, readers can identify the outcome definitions and population description but cannot assess whether the posted results follow a prespecified plan or how robustness to missing data was examined.
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 |
|---|---|---|---|
| Ridaforolimus 40 mg | 4 | 15 | 26.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Ridaforolimus 40 mg | 3 of 15 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Ridaforolimus 40 mg | 15 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Ridaforolimus 40 mg |
|---|---|---|
| Neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 15 |
| Disease progression | General disorders | 1 of 15 |
| Tracheal cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ridaforolimus 40 mg |
|---|---|---|
| Stomatitis | Gastrointestinal disorders | 12 of 15 |
| Proteinuria | Renal and urinary disorders | 11 of 15 |
| White blood cell count decreased | Investigations | 8 of 15 |
| Hyperglycaemia | Metabolism and nutrition disorders | 7 of 15 |
| Pyrexia | General disorders | 6 of 15 |
| Gamma-glutamyltransferase increased | Investigations | 6 of 15 |
| Anaemia | Blood and lymphatic system disorders | 5 of 15 |
| Blood cholesterol increased | Investigations | 5 of 15 |
| Platelet count decreased | Investigations | 5 of 15 |
| Hypoalbuminaemia | Metabolism and nutrition disorders | 5 of 15 |
Serious events with the highest arm-level rate: neoplasm malignant (13.3%), disease progression (6.7%), tracheal cancer (6.7%).
The registry reports safety counts for the Ridaforolimus 40 mg group. For serious adverse events, 4 participants were listed at risk and 4 affected. For other adverse events, 15 were listed at risk and 15 affected. The record also lists 3 deaths. These figures describe the posted registry counts; they are not a comparison with an untreated or active-control group.
The serious adverse-event terms listed are Neoplasm malignant at 13.3%, Disease progression at 6.7%, and Tracheal cancer at 6.7%. The record does not explain whether a participant could contribute to more than one term, so the terms should not be treated as mutually exclusive or added together. Nor do the terms establish that an event was caused by ridaforolimus.
Adverse-event counts are not equivalent to rates over time unless exposure and follow-up are incorporated into the calculation. Those details are not reported in the registry record. The counts also do not show severity distribution beyond the serious-event classification, timing, duration, or event-level clinical context. A single-group study cannot provide a controlled estimate of excess risk relative to a comparator.
12. What the Result Does, and Does Not, Mean
The study addresses a specific pharmacokinetic question: how long it takes ridaforolimus to appear in systemic circulation after oral administration. Its stated primary summary is the median and full range of Tlag after a single dose on Cycle 1 Day 1. The record also describes a dosing schedule and reports safety counts for the ridaforolimus group.
The primary endpoint result itself is not reported in the registry record. Accordingly, the recorded information does not establish the observed median lag time, the range of observed values, or the precision of an estimate. It does not support a conclusion about the distribution of Tlag in the endpoint population. The enrollment figure is not a substitute for the number analyzed for that endpoint, which is also not reported.
The single-group design does not estimate a treatment difference against a concurrent control. Thus, the record cannot establish comparative pharmacokinetics or comparative safety from this study alone. It also does not establish whether ridaforolimus changes a cancer outcome: the primary endpoint is pharmacokinetic, and the listed safety outcome is not an efficacy comparison.
The serious adverse-event figures and listed terms are descriptive registry data. They do not, on their own, determine causation or provide comparative risk estimates. The registry does not report the event-level context and exposure information needed for a fuller safety interpretation.
In general, results from a Phase 1 pharmacokinetic study can help characterize drug behavior under the studied conditions, while conclusions beyond those conditions require additional evidence. For this record, even the primary numerical result is not reported. The appropriate interpretation is therefore limited to the study’s design, endpoint definition, scheduled dosing and sampling context, and the safety counts recorded in the registry.
13. Why This Trial Matters Statistically
This trial is statistically distinctive because it focuses on a pharmacokinetic timing measure in a single-group Phase 1 study. Tlag is different from an efficacy endpoint: it describes the timing of drug appearance in systemic circulation after an oral dose. That makes the endpoint definition, sampling schedule, and analysis population central to understanding what the study intended to measure.
The use of a median and full range is also relevant. A median describes the center of the observed distribution, while the range communicates its extremes. Together they can offer a compact descriptive account, but they do not provide the same information as a confidence interval or a detailed distribution. Since the numerical values are not reported in the registry record, readers can assess the intended summary but not the observed data.
The record highlights the importance of separating enrollment from endpoint analysis. It lists enrollment of 15 participants and defines a Pharmacokinetic Evaluable population using compliance and blood-sample criteria. The number contributing to the endpoint result is not reported. That distinction is important in studies where measurement availability and protocol adherence determine which observations can be summarized.
The single-group design also sets a clear boundary on inference. It can describe measurements and adverse events observed among participants receiving ridaforolimus, but it does not provide a randomized comparator for treatment contrasts. The safety figures therefore remain descriptive, and the serious-event terms should not be read as causal findings or combined into a total.
Finally, the registry record illustrates the difference between having an outcome definition and having a reported result. Statistical interpretation depends on both: a clear endpoint identifies what was measured, while the estimate and its uncertainty show what the data indicate. Here, the endpoint definition is available, but its numerical result and analysis details are not reported.
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 direct limitation for interpreting the primary endpoint is that its numerical result is not reported in the registry record. The stated median and full range cannot be evaluated, and no confidence interval or other uncertainty measure is listed. The record therefore does not show the observed Tlag distribution or how precise its summary might be.
The endpoint population is described by protocol compliance and blood-sample criteria, but its size and the number of samples contributing to the analysis are not reported. The exact lower-limit criterion is not fully described in the available record. Actual sample timing, incomplete profiles, and the handling of missing or non-quantifiable measurements are also not reported.
The study is single-group and unmasked, with no like-for-like comparator. Its design cannot estimate a controlled difference in pharmacokinetics or safety. The registry does not report detailed statistical methods, a prespecified analysis plan, multiplicity handling, sensitivity analyses, or a trial-specific sample-size justification. It lists eight outcome measures and no analyses.
The safety record gives group counts, deaths, and selected serious adverse-event terms, but does not report detailed event timing, duration, participant-level overlap, exposure-adjusted rates, or comparative analyses. The listed terms may overlap; the record does not clarify that issue. These limitations restrict interpretation of the counts and do not support causal attribution.
Finally, the study’s stated population and dosing schedule define the context for its observations. The registry does not report enough numerical pharmacokinetic results to assess how those observations were distributed or to compare them with other settings. Conclusions should therefore remain limited to the design and descriptive information recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pharmacokinetics of Ridaforolimus (MK-8669) in Chinese Participants (MK-8669-059) — NCT01380184.
- Registry record last updated 2019-04-19; status last verified 2019-01.
- Results first posted 2019-04-19.
- 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
This completed Phase 1 study assessed ridaforolimus pharmacokinetics, safety, and tolerability in Chinese participants with advanced cancer. The registry lists enrollment of 15, a single-group design, and no masking. Part 1 included a single 40 mg oral dose followed by a multiple-dose schedule; Part 2 was optional.
The primary endpoint was Day 1 Tlag in hours, summarized by the median and full range. Sampling was scheduled from predose through various time points up to 24 hours postdose. The numerical primary endpoint result and analysis details are not reported in the registry record.
The registry lists 4 participants affected by serious adverse events, 15 affected by other adverse events, and 3 deaths. Selected serious-event terms and percentages are recorded, but the study has no control group for comparison. The safety figures are descriptive and do not establish causation.