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 Nivolumab (Opdivo®) Plus ABI-009 (Nab-rapamycin) for Advanced Sarcoma and Certain Cancers (NCT03190174), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated Dose of ABI-009.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03190174 |
| Phase | Phase 1, Phase 2 |
| Condition | Ewing Sarcoma, PEComa, Epithelioid Sarcoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 34 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Sarcoma Oncology Research Center, LLC |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns people with advanced cancers named in the registry record, including Ewing sarcoma, PEComa, epithelioid sarcoma, desmoid tumor, chordoma, and a range of carcinomas and other malignancies. The record also describes tumors with genetic mutations sensitive to mTOR inhibitors. Its brief summary includes small cell lung cancer, while the listed conditions include non-small cell lung cancer. The primary endpoint population is narrower: participants assigned to the Phase I dose-escalation part.
Intervention
The interventions are nivolumab and nab-rapamycin, also called ABI-009. The brief summary describes sequential administration, and the Phase 1b arm description says a defined dose of nivolumab is given with escalating doses of nab-rapamycin intravenously. The registry record does not report a numerical dose result for the primary endpoint.
Comparator
This is a single-group study with no like-for-like control arm. Allocation is listed as not applicable, and masking is listed as none. The dose-escalation cohorts and expansion group are parts of the study design, not randomized comparison groups. As a result, the record does not support a controlled estimate of how the combination compares with another treatment.
Primary question
The primary question is the maximum tolerated dose of ABI-009 at Week 6, using the registry’s definition based on dose-limiting toxicity at dose levels. This is a dose-finding question, rather than a comparative question about tumor response or survival. Disease control rate, progression-free survival, and overall survival are listed as secondary outcomes, but numerical results for those outcomes are not reported in the registry record.
3. Trial Design
The registry describes a phase 1/2, single-group study of nivolumab and ABI-009, also named nab-rapamycin. It is marked completed, with enrollment of 34. The study started on 2017-08-24, and its primary completion date is 2021-12-01. The listed sponsor is Sarcoma Oncology Research Center, LLC. The study title and summary describe advanced sarcoma and several other cancers, including tumors with mutations sensitive to mTOR inhibitors.
The design combines dose escalation with an expansion phase. The arm description identifies an open-label, dose-seeking Phase 1b study using a defined nivolumab dose and escalating ABI-009 doses given intravenously. It describes a standard “cohort of three” approach for the dose-escalation portion. The registry record does not report the full dose-escalation sequence, the doses tested, or a numerical maximum tolerated dose.
Allocation is listed as not applicable, masking as none, and the model as single group. These details mean that the record does not describe a randomized, masked comparison against a control treatment. Cohort labels and the expansion group do not create a randomized control comparison. Consequently, interpretation should focus on the dose-finding objective and the results reported for the study, rather than treating groups as comparable treatment arms.
The primary outcome is maximum tolerated dose of ABI-009 at Week 6. Disease control rate, progression-free survival, and overall survival are listed as secondary outcomes. Although the record reports four outcome measures, numerical results for the primary endpoint and the listed secondary outcomes are not reported in the registry record. The record also does not report a statistical analysis for those outcomes.
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 | Dose Escalation Phase 1 Dose Level I | Dose Escalation Phase 1 Dose Level II | Dose Escalation Phase 1 Dose Level III | Expansion Phase 1b Part |
|---|---|---|---|---|
| STARTED | 3 | 3 | 3 | 25 |
| COMPLETED | 3 | 3 | 3 | 25 |
| NOT COMPLETED | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Intention-to-treat population (all participant assigned to the Phase I dose escalation part of the study).
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 1: 56 mg/m^2 | Phase 1: 75mg/m^2 | Phase 1: 100 mg/m^2 | Phase 1b: 100 mg/m^2 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 3 | 3 | 3 | 25 | 34 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 3 | 2 | 25 | 31 |
| Age, Categorical: >=65 years | Participants | 2 | 0 | 1 | 0 | 3 |
| Sex: Female, Male: Sex – Female | Participants | 1 | 2 | 1 | 11 | 15 |
| Sex: Female, Male: Sex – Male | Participants | 2 | 1 | 2 | 14 | 19 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 3 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 3 | 3 | 3 | 22 | 31 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose of ABI-009, measured over Week 6.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg/m^2 |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the maximum tolerated dose of ABI-009, measured in mg/m^2 at Week 6. The registry defines this as the highest dose considered safely tolerated under a dose-limiting-toxicity rule: not more than one patient experiences dose-limiting toxicity at that dose level, while at least two patients experience dose-limiting toxicity at the next higher dose level. The endpoint is therefore a dose-selection measure based on observed toxicity across dose levels, not a measure of tumor response or survival.
In general, dose-finding studies use a maximum tolerated dose endpoint to identify a dose level for further study while accounting for toxicity. The decision depends on the observed pattern of dose-limiting toxicities at successive levels and on the rule specified for interpreting that pattern. It is not equivalent to proving that a dose is free of harm, nor does the endpoint alone establish clinical benefit.
The registry identifies the primary endpoint population as the intention-to-treat population, defined there as all participants assigned to the Phase I dose-escalation part. However, it does not report a numerical maximum tolerated dose, dose-level-specific toxicity results for this endpoint, or further details about endpoint analysis. The endpoint’s stated timeframe is Week 6; the record does not provide additional timing details.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical feature of this study is its dose-finding purpose. Its primary endpoint is not a between-treatment effect, but the maximum tolerated dose of ABI-009 under a specified dose-limiting-toxicity rule. The registry identifies the study as single group, with allocation not applicable and no masking. Therefore, the dose-escalation cohorts should not be interpreted as randomized treatment arms, and the record does not provide a controlled estimate of the combination’s effect relative to another treatment.
The endpoint definition connects toxicity at a dose level with toxicity at the next higher level. This is a decision rule for selecting a dose, not a conventional estimate of a treatment effect with a confidence interval. In general, dose-finding decisions are sensitive to which participants are observed at each level and how toxicities are classified and timed. The registry record does not report dose-specific participant counts, the observed dose-limiting toxicities, or the selected ABI-009 dose. Accordingly, the numerical primary result cannot be interpreted from the record.
The record lists disease control rate, progression-free survival, and overall survival as secondary outcomes. It does not report numerical results for these outcomes or statistical analyses. Thus, the posted record does not establish the magnitude or precision of any tumor-control or survival outcome. The study’s completed status and enrollment of 34 do not substitute for outcome estimates, and they do not indicate whether a particular endpoint was met.
The adverse-event information is descriptive. It lists serious and other adverse events at risk and affected within named study groups, along with deaths, and identifies thrombocytopenia and dehydration among the serious terms with registry-listed percentages. Those entries can describe reported safety events, but the record does not provide enough detail to attribute events to a particular intervention or to compare risk with a control group. The death counts are not evidence by themselves about cause or treatment relationship.
In general, uncertainty around an outcome estimate is assessed using its statistical interval and analysis details. Such estimates and intervals are not reported for the primary or secondary outcomes in this record. Interpretation should therefore remain bounded by the reported design, endpoint definition, and descriptive safety entries, without drawing an efficacy conclusion or treating unreported analyses as evidence of no effect.
9. Statistical Methods Explained
The registry describes a Phase 1b dose-seeking study with escalating doses of ABI-009 alongside a defined dose of nivolumab. It says the dose-escalation portion uses a standard “cohort of three” design. In general, cohort-based dose escalation observes toxicity among participants treated at a dose level and uses those observations to guide dose selection. The registry’s primary endpoint rule adds a specific criterion: the maximum tolerated dose is the highest dose with not more than one patient experiencing dose-limiting toxicity, when at least two patients experience dose-limiting toxicity at the next higher dose level. The registry does not report which dose levels were tested or how many dose-limiting toxicities occurred at each one.
What does the primary endpoint represent?
The endpoint is a dose-finding decision, stated in mg/m^2 and assessed at Week 6. It is not an estimate of the difference between nivolumab plus ABI-009 and another treatment. In general, an endpoint rule makes explicit how observed data are translated into a decision. That improves clarity about the target decision, but it does not remove uncertainty: the observed toxicity pattern may depend on the participants enrolled and the dose levels evaluated. The registry record gives the rule but not the numerical endpoint result or the detailed observations needed to reproduce the decision.
What does the single-group design allow?
The model is single group, allocation is listed as not applicable, and masking is listed as none. In general, random allocation to a control group can support a direct comparison by reducing some sources of systematic difference between groups. This study is not described that way. The dose-escalation parts and expansion group are design components, not like-for-like randomized controls. Any outcome rates reported for this study would need to be interpreted as observations in the study population, not as a controlled estimate of benefit or harm relative to another treatment.
How should the planning illustrations be understood?
The registry’s sample-size assumptions describe planning illustrations computed from an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. For time-to-event outcomes, the stated method gives the total number of events using Schoenfeld; other outcomes are expressed as participants per arm. These are planning assumptions, not a description of this trial’s single-group design. The numerical planning results are not reported in the registry record. In general, such sample-size illustrations ask how much information a hypothetical design might need under specified assumptions. They are not observed power, and they do not show that the actual study had a particular probability of detecting an effect.
What can confidence intervals and over-dispersion tell readers in general?
In general, a confidence interval expresses the range of values compatible with an estimate under a statistical model and its assumptions. A narrow interval indicates greater precision than a wide interval, but neither guarantees that the model or study design is appropriate. The registry record does not report confidence intervals for the primary or secondary outcomes. Over-dispersion is a general concern when observed variation in count data exceeds what a model assumes; if ignored, uncertainty can be understated. The record does not report a count-data model or an over-dispersion assessment, so these concepts should not be mistaken for methods used in this study.
What does the record say about analysis?
The registry reports zero analyses. This means numerical analysis results are not reported in the record; it does not establish that no analyses were conducted outside the registry. The record names the primary endpoint and several secondary outcomes, but does not describe a statistical analysis plan, model specifications, adjustment methods, or confidence-interval procedures. General statistical principles can explain how those methods work, but cannot establish which methods were used in this study.
10. Multiplicity, Missing Data and Analysis Populations
The record lists a primary endpoint and secondary outcomes of disease control rate, progression-free survival, and overall survival. It does not report how many statistical hypotheses were tested, whether formal hypothesis tests were conducted, or whether any multiplicity adjustment was planned or used. In general, testing multiple outcomes can increase the chance of obtaining at least one apparently notable result by chance. Methods that adjust for multiplicity can address that issue, but the registry record does not report such a method. No claim about adjusted or unadjusted statistical significance can be made from the listed outcomes alone.
Missing-data handling is not reported in the registry record. The record does not explain how participants without endpoint assessments, incomplete follow-up, or unavailable outcome values were handled. In general, missingness can affect both the estimate and its uncertainty, and the consequences depend on why observations are missing and on the analysis approach. Without the relevant details, readers cannot determine whether missing data were excluded, imputed, or handled in another way.
The primary endpoint population is described as the intention-to-treat population, defined as all participants assigned to the Phase I dose-escalation part. This is the population specified for the primary endpoint; it should not be assumed to apply identically to every secondary outcome. The record does not report analysis populations for the secondary outcomes or explain how population membership was determined for each analysis.
The registry reports zero analyses, and numerical results for the stated outcomes are not reported in the registry record. This limits assessment of multiplicity, missingness, and the relationship between intended and analyzed populations. No statistical analysis plan or further population details are reported.
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 |
|---|---|---|---|
| Dose Escalation Phase 1 Part I | 0 | 3 | 0.0% |
| Dose Escalation Phase 1 Part II | 0 | 3 | 0.0% |
| Dose Escalation Phase 1 Part III | 0 | 3 | 0.0% |
| Expansion Phase 1b | 3 | 25 | 12.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dose Escalation Phase 1 Part I | 3 of 3 |
| Dose Escalation Phase 1 Part II | 3 of 3 |
| Dose Escalation Phase 1 Part III | 3 of 3 |
| Expansion Phase 1b | 21 of 25 |
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 |
|---|---|
| Dose Escalation Phase 1 Part I | 0 of 3 |
| Dose Escalation Phase 1 Part II | 0 of 3 |
| Dose Escalation Phase 1 Part III | 0 of 3 |
| Expansion Phase 1b | 2 of 25 |
Most frequent serious adverse event terms
| Event term | Organ system | Dose Escalation Phase 1 Part I | Dose Escalation Phase 1 Part II | Dose Escalation Phase 1 Part III | Expansion Phase 1b |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 3 | 0 of 3 | 0 of 3 | 3 of 25 |
| Dehydration | Metabolism and nutrition disorders | 0 of 3 | 0 of 3 | 0 of 3 | 1 of 25 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dose Escalation Phase 1 Part I | Dose Escalation Phase 1 Part II | Dose Escalation Phase 1 Part III | Expansion Phase 1b |
|---|---|---|---|---|---|
| Oral mucositis | Gastrointestinal disorders | 0 of 3 | 0 of 3 | 0 of 3 | 2 of 25 |
Serious events with the highest arm-level rate: thrombocytopenia (12.0%), dehydration (4.0%).
The safety entries are organized by study group and distinguish participants at risk from participants affected. For serious adverse events, the Dose Escalation Phase 1 Part I, Part II, and Part III groups each list 3 at risk and 0 affected. The Expansion Phase 1b group lists 25 at risk and 3 affected. For other adverse events, the first three groups each list 3 at risk and 0 affected, while the Expansion Phase 1b group lists 25 at risk and 2 affected. The registry also lists deaths of 3, 3, 3, and 21, respectively, across those groups.
These are descriptive registry entries, not a comparison against a control group. The counts do not by themselves establish that an event or death was caused by either intervention. The record does not report event-level details, timing, severity grades, relatedness assessments, or the causes of death in these entries. It lists thrombocytopenia at 12.0% and dehydration at 4.0% among the leading serious adverse-event terms; the registry record does not state the denominator or additional context for those percentages. These figures should be read as reported safety information, not as comparative risk estimates.
12. What the Result Does, and Does Not, Mean
The record documents a completed phase 1/2, single-group study of nivolumab and ABI-009 in advanced sarcoma and selected other cancers. Its primary objective is dose finding: identifying the maximum tolerated dose of ABI-009 at Week 6 using the stated dose-limiting-toxicity rule. The registry’s design information and safety entries help describe what the study set out to assess and what event counts it recorded.
The record does not report a numerical maximum tolerated dose or numerical results for the listed secondary outcomes. Therefore, it does not establish from the posted information how the dose-finding endpoint turned out, or the magnitude and precision of disease control, progression-free survival, or overall survival. A completed status and enrollment of 34 do not provide those missing estimates. No conclusion about treatment benefit should be drawn from the study description alone.
The single-group design also sets limits on interpretation. Without a like-for-like control group, the record does not provide a randomized comparison of outcomes under the combination versus another treatment. The cohort labels within dose escalation and expansion are not a control arm. The safety counts describe events reported within study groups, but do not by themselves identify causes or establish comparative safety.
In general, a study can contribute useful information about dose selection and observed events even when it does not provide a controlled treatment-effect estimate. The strength and scope of any conclusion depend on the outcomes actually reported, their precision, and the design used to obtain them. Here, the registry record does not report the numerical primary result, secondary outcome analyses, confidence intervals, or detailed statistical methods. The appropriate interpretation is consequently limited to the study’s stated purpose, design, endpoint definition, and descriptive safety entries.
13. Why This Trial Matters Statistically
This trial is statistically important because its primary question is a dose-finding question, not a conventional comparison of treatment effects. The maximum tolerated dose is defined through toxicity observations at successive dose levels. That makes the decision rule and the dose-specific observations central to interpretation. The registry record supplies the endpoint definition and describes a cohort-of-three approach, but does not report the dose-level results or the selected dose.
The study also illustrates why design labels matter. It is listed as single group, with allocation not applicable and no masking. Cohorts within a dose-escalation design should not be mistaken for randomized treatment arms. This distinction prevents descriptive differences across groups from being interpreted as controlled evidence about treatment effects.
Another statistical issue is the separation between a prespecified endpoint and a reported result. The registry names the primary outcome and secondary outcomes, but numerical outcome analyses are not reported. A clear endpoint definition helps readers understand what was intended to be measured; it cannot replace the result itself. Without estimates, uncertainty intervals, or analysis details, readers cannot assess precision or evaluate whether the reported evidence supports a specific conclusion.
Finally, planning illustrations require care when they do not match the actual design. The record describes hypothetical calculations based on equal allocation between two arms, while the trial is listed as single group. Such illustrations can explain assumptions behind a planning scenario, but do not describe the study’s actual allocation or represent observed power. Keeping design, analysis, and interpretation distinct is essential when reading this record.
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 does not report the numerical maximum tolerated dose, dose-specific toxicity observations, or numerical results for disease control rate, progression-free survival, and overall survival. It also does not report confidence intervals or further statistical analyses. This makes it impossible to assess the magnitude or precision of the intended outcomes from the posted record.
The design is single group, with no like-for-like randomized control comparison. That limits what can be inferred about differences attributable to the interventions. The record’s dose-escalation and expansion groups should not be read as randomized controls, and the descriptive adverse-event counts do not provide comparative risk estimates.
Details that would help assess conduct and interpretation are not reported in the registry record, including dose levels tested, the dose-limiting toxicities observed at each level, missing-data handling, analysis populations for secondary outcomes, and methods for any statistical testing. The record also does not report a statistical analysis plan or how multiple outcomes were handled.
The safety entries list participants at risk and affected and deaths by group, but provide no event-level timing, attribution, or cause-of-death detail in the information reported. The denominator and context for the listed serious adverse-event percentages are not stated. These limitations mean that the counts should remain descriptive and should not be used to infer causality or comparative safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Nivolumab (Opdivo®) Plus ABI-009 (Nab-rapamycin) for Advanced Sarcoma and Certain Cancers — NCT03190174.
- Registry record last updated 2025-02-17; status last verified 2025-02.
- Results first posted 2025-02-17.
- 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
The registry describes a completed phase 1/2, single-group study of nivolumab and ABI-009 (nab-rapamycin) for advanced sarcoma and selected other cancers. Enrollment is listed as 34. The study began on 2017-08-24, and the primary completion date is 2021-12-01.
The primary endpoint is the maximum tolerated dose of ABI-009 at Week 6, defined using dose-limiting toxicity at a dose level and the next higher level. The record describes an open-label dose-escalation design and lists disease control rate, progression-free survival, and overall survival as secondary outcomes. Numerical outcome analyses are not reported in the registry record.
Safety entries report serious and other adverse events and deaths by study group. The study has no like-for-like randomized control comparison, and the registry does not report a numerical primary endpoint result or detailed statistical analysis methods.