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 Gemcitabine Hydrochloride and Cisplatin With or Without Veliparib or Veliparib Alone in Treating Patients With Locally Advanced or Metastatic Pancreatic Cancer (NCT01585805), a randomized phase 2 study. The primary outcome is Optimal Dose of Veliparib With Gemcitabine Hydrochloride and Cisplatin (Non-randomized Part I).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01585805 |
| Phase | Phase 2 |
| Condition | Locally Advanced Pancreatic Adenocarcinoma, Metastatic Pancreatic Adenocarcinoma, Stage III Pancreatic Cancer AJCC v8 |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 83 |
| Arms in the primary outcome | 4 |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
The program concerns people with locally advanced or metastatic pancreatic adenocarcinoma. Part I focuses on pancreatic adenocarcinoma with a known BRCA or PALB2 mutation; Part II concerns previously treated pancreatic adenocarcinoma. The primary endpoint applies only to participants in the Part I lead-in cohorts.
Intervention
In the randomized portion of Part I, one group receives veliparib with gemcitabine hydrochloride and cisplatin. A non-randomized lead-in evaluates veliparib dose levels with gemcitabine and cisplatin. Part II is a separate single-arm study of veliparib alone.
Comparator
The randomized comparison in Part I is the combination of veliparib, gemcitabine hydrochloride, and cisplatin versus gemcitabine hydrochloride and cisplatin. The Part II single-agent study is not described as a randomized comparison.
Primary question
The posted primary endpoint asks for the optimal veliparib dose with gemcitabine hydrochloride and cisplatin in the non-randomized Part I lead-in. The registry reports 80 mg for the Part I Lead In group, with 17 participants. This is a dose-selection result, not an estimate of comparative effectiveness.
The trial record brings together distinct components: a lead-in dose evaluation, a randomized treatment comparison, and a separate single-arm study. These components address related but different questions. The lead-in tests dose levels in combination therapy; the randomized portion is intended to compare the combination with chemotherapy alone; the single-agent portion studies veliparib in previously treated disease.
The title and description identify pancreatic adenocarcinoma and known BRCA or PALB2 mutation for Part I. The listed conditions include locally advanced and metastatic pancreatic adenocarcinoma and stage III and stage IV pancreatic cancer. Eligibility details beyond those descriptions are not reported in the registry record. The posted primary result is restricted to the lead-in population, so it cannot by itself answer the randomized comparison or the Part II question.
3. Trial Design
This is a phase II study sponsored by the National Cancer Institute. Its record describes randomized allocation, no masking, and a parallel model, while also specifying a non-randomized Part I lead-in and a single-arm Part II study. The overall record therefore contains more than one design component; the randomized design description applies to the comparative portion and does not make the lead-in or single-arm study randomized.
The randomized Part I comparison is between veliparib plus gemcitabine hydrochloride and cisplatin, and gemcitabine hydrochloride plus cisplatin alone. In the lead-in, participants receive escalating veliparib doses with fixed doses of gemcitabine and cisplatin. The dose levels listed are 20 mg, 40 mg, and 80 mg twice daily on days 1–12; an additional schedule evaluates 80 mg twice daily on days 1–21 if no dose-limiting toxicity is observed at the preceding schedule. The description says three to six participants are evaluated per dose level. These details describe the dose-finding plan, not the randomized treatment comparison.
Part II is a phase II single-arm study of single-agent veliparib in previously treated pancreatic adenocarcinoma. The arm descriptions list veliparib on days 1–28 of each cycle. For the combination arms, cycles repeat every 21 days; for the single-agent arm, cycles repeat every 28 days. Treatment is described as continuing in the absence of disease progression or unacceptable toxicity.
The record lists overall enrollment of 83 and status as active, not recruiting. The study start date is 2012-05-15, and the primary completion date is 2024-12-31. These record dates and overall enrollment do not identify how many participants were randomized to each comparative arm. Arm-level primary results for the randomized groups are not posted in the primary endpoint results. Masking is listed as none, so the study is open-label according to the registry design information.
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: Prior to randomization, some participants either withdrew consent or were excluded because of ineligibility. Two participants withdrew consent after they were randomized to Arm B.
Only the first 6 of 7 groups are shown.
Overall Study
| Stage | Part I Lead In: Veliparib 20mg | Part I Lead In: Veliparib 40mg | Part I Lead In: Veliparib 80mg | Part I Lead In: Veliparib 80mg Cont | Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | Arm B (Gemcitabine Hydrochloride, Cisplatin) |
|---|---|---|---|---|---|---|
| STARTED | 3 | 3 | 6 | 5 | 27 | 23 |
| COMPLETED | 3 | 3 | 6 | 5 | 27 | 23 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Only applicable to participants in Part I Lead In Cohorts. Data is reported as per study design and as published. Pre-specified in the study protocol to collect, analyze, and report as a single Arm/Group
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.
Only the first 6 of 8 groups are shown.
| Characteristic | Unit | Part I Lead In: Veliparib 20mg | Part I Lead In: Veliparib 40mg | Part I Lead In: Veliparib 80mg | Part I Lead In: Veliparib 80mg Cont | Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | Arm B (Gemcitabine Hydrochloride, Cisplatin) |
|---|---|---|---|---|---|---|---|
| Participants analysed | 3 | 3 | 6 | 5 | 27 | 23 | |
| Age, Continuous | years | 66 (62 to 70) | 60 (57 to 64) | 55.5 (49 to 70) | 68 (50 to 71) | 64 (48 to 82) | 63 (37 to 81) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 4 | 3 | 12 | 10 |
| Sex: Female, Male: Male | Participants | 3 | 3 | 2 | 2 | 15 | 13 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 3 | 6 | 5 | 27 | 21 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 1 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 1 | 2 | 1 | 1 |
| Race (NIH/OMB): White | Participants | 3 | 3 | 5 | 3 | 25 | 21 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 1 |
| Region of Enrollment: Canada | participants | 0 | 0 | 1 | 0 | 1 | 0 |
| Region of Enrollment: United States | participants | 3 | 3 | 5 | 5 | 22 | 20 |
| Region of Enrollment: Israel | participants | 0 | 0 | 0 | 0 | 4 | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Optimal Dose of Veliparib With Gemcitabine Hydrochloride and Cisplatin (Non-randomized Part I), measured over 21 days.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the optimal dose of veliparib with gemcitabine hydrochloride and cisplatin in the non-randomized Part I lead-in. It is expressed as a dose in mg, with a stated timeframe of 21 days. This is a dose-selection endpoint rather than a measure of tumor response, survival, or a treatment difference between randomized groups.
Dose-finding studies use a structured escalation process to identify a dose suitable for further study while monitoring dose-limiting toxicity. In this record, the lead-in description lists ascending veliparib dose levels and specifies escalation conditional on whether dose-limiting toxicity is observed. It also describes evaluation of an 80 mg twice-daily schedule over days 1–21 after the days 1–12 schedule. The rationale is to assess the selected dose and schedule alongside fixed gemcitabine and cisplatin doses.
The registry reports 80 mg for the Part I Lead In group, which includes 17 participants. That posted result should be read in the context of the stated endpoint and population: it records the selected dose for the lead-in, not a comparative estimate of efficacy or safety. The registry record does not post a confidence interval or other measure of uncertainty for this dose result. It also does not provide a primary endpoint result for the randomized arms in the listed endpoint data.
Choosing a dose is useful because later comparative evaluation depends on having a regimen specified for study. A selected dose alone does not establish how the regimen compares with chemotherapy alone or what outcomes occurred in either randomized group.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive distinction is between the result that is posted and the broader questions raised by the trial. The primary endpoint result is 80 mg for the Part I Lead In group, with 17 participants. It pertains to dose selection in a non-randomized lead-in. It is not the result of the randomized comparison between combination therapy and gemcitabine hydrochloride with cisplatin alone.
The endpoint data list the randomized Arm A and Arm B with zero participants and no endpoint values, and the Arm C result is also not populated in that endpoint record. This does not mean that no participants entered those arms: the safety data list participants at risk in each arm. It means that arm-level values for this primary endpoint are not reported in the posted endpoint results. The randomized arms are not the population for the primary endpoint as defined in the record.
The program also lists secondary outcomes: progression-free survival, adverse events, disease control rate and duration of response, and overall survival. The record identifies four secondary outcomes, but no analyses are posted. An outcome being named in a registry is not the same as a result being available, and the listed outcome names alone do not provide estimates, uncertainty intervals, or treatment comparisons.
For the posted dose result, precision is difficult to assess because no interval or other uncertainty measure accompanies the value. A sample of 17 in the lead-in is not equivalent to a randomized comparison with an estimate of treatment effect. Dose escalation is intended to inform dose selection; it does not estimate the effect of adding veliparib on survival or disease control.
The safety record contains arm-specific counts of participants at risk and participants with serious or other adverse events, together with deaths. These counts describe what was recorded in the safety groups but do not establish comparative risk on their own. Group sizes differ, and the registry does not give a full statistical comparison or adjusted analysis. Serious adverse-event terms are listed with percentages, but the denominator and method used to produce those percentages are not reported in the registry record.
No p-value or confidence interval for a randomized treatment contrast is posted. Consequently, the available record does not support a conclusion about the magnitude or precision of a comparative treatment effect. The most defensible reading is limited: the registry posts a lead-in dose result and safety counts across study groups, while the randomized primary endpoint comparison and analyses of the named secondary outcomes are not posted.
9. Statistical Methods Explained
Statistical interpretation depends first on matching each result to the design component and population it belongs to. This study includes a non-randomized dose-finding lead-in, a randomized parallel comparison, and a single-arm study. The primary endpoint is explicitly assigned to the Part I lead-in cohorts. Its reported value therefore answers a dose-selection question within that cohort, not a treatment-effect question across randomized arms.
What does a dose-selection endpoint measure?
In general, a dose-selection endpoint records the dose chosen for continued evaluation after planned dose levels have been assessed. Escalation rules use observed tolerability information to guide whether a higher dose or a different schedule should be examined. Such a design is different from a trial powered to estimate a difference in response or survival. The registry describes dose levels, a conditional escalation approach, and a 21-day endpoint timeframe. It reports the selected dose as 80 mg for the lead-in group, but does not report an interval quantifying uncertainty around that selection.
General statistical caution is important for small dose cohorts: each participant’s experience can have substantial influence on the observed pattern, and the selected dose does not guarantee that the same balance of tolerability and activity will be seen in a larger study. The record’s description says three to six participants are evaluated per dose level, while the posted lead-in endpoint group contains 17 participants. The record does not give a dose-by-dose primary endpoint analysis in the endpoint data, so the 17-person group result should not be interpreted as a separate estimate for every dose level.
Why does randomization matter?
General explanation: random allocation helps make treatment groups comparable, on average, with respect to measured and unmeasured baseline characteristics. In a randomized comparison, the treatment contrast is the primary quantity of interest, together with its uncertainty. Here, the registry records a randomized comparison of the combination regimen against gemcitabine hydrochloride and cisplatin. However, the posted primary endpoint is not a comparative endpoint, and the registry’s endpoint results do not provide estimates for those randomized arms.
Masking is listed as none. In general, lack of masking can affect behavior, reporting, and outcome assessment, especially for outcomes that involve judgment or participant reporting. The record does not state whether particular outcome assessments were independently reviewed or whether other procedures were used to reduce assessment bias. Those details are not reported in the registry record.
How should uncertainty be assessed?
General explanation: a confidence interval describes a range of values compatible with the data and the statistical model at a stated confidence level. It helps readers distinguish a precise estimate from one with considerable uncertainty. A point estimate alone does not show how much the result might vary across repeated studies. No confidence interval is posted for the lead-in dose result, and no randomized treatment-effect estimate is posted in the primary endpoint data.
General explanation: a p-value summarizes how incompatible observed data are with a specified null model, under its assumptions. It does not measure the probability that a treatment works, and it does not convey the size or clinical importance of an effect. No p-value for a randomized treatment comparison appears in the posted analyses; the registry lists zero analyses.
What do the planning illustrations mean?
General explanation: sample-size planning asks how many participants or events may be needed to detect a chosen effect under stated assumptions. The registry-associated planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the target effect as a stated fraction of the observed effect; time-to-event illustrations use total events, while other illustrations use participants per arm. These are planning assumptions, not observed outcomes, and they are not evidence of the trial’s original design or of achieved power.
The record does not provide a posted observed comparative effect to which these planning assumptions can be applied in the primary endpoint results. Planning calculations based on an observed effect are especially difficult to interpret when the corresponding treatment contrast is not posted. They should not be read as an analysis of the completed sample or as proof that the study had adequate power.
Why does over-dispersion matter in general?
General explanation: for count outcomes, over-dispersion means the data vary more than a simple model assumes. If it is ignored, standard errors can be too small and uncertainty can be understated. The primary endpoint is a dose in mg, not a count of events or a continuous treatment contrast, so a count-model explanation does not supply an analysis of this endpoint. The registry does not report a model, variance estimate, or inferential procedure for the posted dose result.
The registry marks the endpoint as a count-other type and indicates that values are missing or not estimable for a two-arm comparison. That classification is consistent with the endpoint being a single dose-selection result rather than a two-arm effect estimate. No inferential analysis should be inferred from the endpoint label alone. The record does not describe adjustments, covariates, a formal dose-selection model, or uncertainty estimation; those methods are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple outcomes, including progression-free survival, adverse events, disease control rate and duration of response, and overall survival. When a study evaluates multiple outcomes, time points, or treatment contrasts, the chance of a false-positive finding can increase if each is tested without accounting for multiplicity. General approaches include designating a primary hypothesis, controlling an overall error rate, or treating secondary analyses as exploratory. The registry record does not report a multiplicity strategy, testing hierarchy, or adjustment method.
Four secondary outcomes are listed, but the registry reports zero analyses. Their presence in the record does not establish that estimates or hypothesis tests are available. Details on which outcomes were prespecified for each study part, how multiple measures were handled, and whether analyses were planned separately by part are not reported in the registry record.
Missing-data methods are not reported in the registry record. The record does not explain how participants with incomplete follow-up, unavailable tumor assessments, treatment discontinuation, or missing outcome information were handled. General explanation: missingness can affect an estimate when the likelihood of having an observed outcome is related to prognosis, treatment, or the unobserved outcome itself. The effect depends on why data are missing and on the analysis method; it cannot be resolved from group counts alone.
The analysis population is described for the primary endpoint as participants in the Part I Lead In Cohorts, with the result reported as a single arm/group. The registry does not report whether the randomized outcomes were analyzed by intention to treat, per protocol, or another population definition. It also does not report exclusions after randomization or the number included in each randomized efficacy analysis. These omissions limit interpretation of any treatment comparison because the population contributing to an estimate is central to its meaning.
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 |
|---|---|---|---|
| Part I Lean In: Veliparib 20 mg | 1 | 3 | 33.3% |
| Part I Lean In: Veliparib 40 mg | 2 | 3 | 66.7% |
| Part I Lean In: Veliparib 80 mg | 4 | 6 | 66.7% |
| Part I Lean In: Veliparib 80 mg Cont | 3 | 5 | 60.0% |
| Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | 10 | 27 | 37.0% |
| Arm B (Gemcitabine Hydrochloride, Cisplatin) | 5 | 23 | 21.7% |
| Arm C (Veliparib) | 8 | 16 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Part I Lean In: Veliparib 20 mg | 2 of 3 |
| Part I Lean In: Veliparib 40 mg | 2 of 3 |
| Part I Lean In: Veliparib 80 mg | 6 of 6 |
| Part I Lean In: Veliparib 80 mg Cont | 3 of 5 |
| Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | 20 of 27 |
| Arm B (Gemcitabine Hydrochloride, Cisplatin) | 21 of 23 |
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 |
|---|---|
| Part I Lean In: Veliparib 20 mg | 3 of 3 |
| Part I Lean In: Veliparib 40 mg | 3 of 3 |
| Part I Lean In: Veliparib 80 mg | 6 of 6 |
| Part I Lean In: Veliparib 80 mg Cont | 5 of 5 |
| Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | 27 of 27 |
| Arm B (Gemcitabine Hydrochloride, Cisplatin) | 19 of 23 |
Most frequent serious adverse event terms
| Event term | Organ system | Part I Lean In: Veliparib 20 mg | Part I Lean In: Veliparib 40 mg | Part I Lean In: Veliparib 80 mg | Part I Lean In: Veliparib 80 mg Cont | Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | Arm B (Gemcitabine Hydrochloride, Cisplatin) |
|---|---|---|---|---|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | 0 of 3 | 1 of 3 | 1 of 6 | 1 of 5 | 2 of 27 | 2 of 23 |
| Biliary tract infection | Infections and infestations | 0 of 3 | 0 of 3 | 1 of 6 | 0 of 5 | 2 of 27 | 2 of 23 |
| Nausea | Gastrointestinal disorders | 0 of 3 | 0 of 3 | 2 of 6 | 0 of 5 | 1 of 27 | 1 of 23 |
| Fever | General disorders | 0 of 3 | 0 of 3 | 0 of 6 | 1 of 5 | 2 of 27 | 1 of 23 |
| Small intestinal obstruction | Gastrointestinal disorders | 1 of 3 | 1 of 3 | 0 of 6 | 0 of 5 | 2 of 27 | 0 of 23 |
| Fatigue | General disorders | 0 of 3 | 0 of 3 | 2 of 6 | 1 of 5 | 0 of 27 | 0 of 23 |
| Sepsis | Infections and infestations | 0 of 3 | 0 of 3 | 1 of 6 | 1 of 5 | 1 of 27 | 0 of 23 |
| Anemia | Blood and lymphatic system disorders | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 2 of 27 | 0 of 23 |
| AST | Investigations | 0 of 3 | 0 of 3 | 0 of 6 | 1 of 5 | 1 of 27 | 0 of 23 |
| ALT | Investigations | 0 of 3 | 0 of 3 | 0 of 6 | 1 of 5 | 1 of 27 | 0 of 23 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Part I Lean In: Veliparib 20 mg | Part I Lean In: Veliparib 40 mg | Part I Lean In: Veliparib 80 mg | Part I Lean In: Veliparib 80 mg Cont | Arm A (Veliparib, Gemcitabine Hydrochloride, Cisplatin) | Arm B (Gemcitabine Hydrochloride, Cisplatin) |
|---|---|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 22 of 27 | 19 of 23 |
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 23 of 27 | 16 of 23 |
| Leukopenia | Investigations | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 19 of 27 | 16 of 23 |
| Hyperglycemia | Metabolism and nutrition disorders | 3 of 3 | 1 of 3 | 2 of 6 | 0 of 5 | 11 of 27 | 11 of 23 |
| Fatigue | General disorders | 1 of 3 | 1 of 3 | 1 of 6 | 1 of 5 | 10 of 27 | 8 of 23 |
| Increased alkaline phosphatase | Investigations | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 9 of 27 | 5 of 23 |
| Constipation | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 0 of 6 | 1 of 5 | 5 of 27 | 6 of 23 |
| Lymphopenia | Blood and lymphatic system disorders | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 6 of 27 | 6 of 23 |
| ALT | Investigations | 0 of 3 | 0 of 3 | 0 of 6 | 0 of 5 | 4 of 27 | 8 of 23 |
| Neutrophil count decreased | Investigations | 2 of 3 | 2 of 3 | 5 of 6 | 3 of 5 | 0 of 27 | 0 of 23 |
Serious events with the highest arm-level rate: nausea (33.3%), fatigue (33.3%), colon perforation (33.3%), abdominal pain (33.3%), small intestinal obstruction (33.3%).
The safety record lists, for each group, the number at risk and the number affected by serious adverse events, other adverse events, and deaths. In the lead-in groups, the serious adverse-event counts are 1 of 3 at 20 mg, 2 of 3 at 40 mg, 4 of 6 at 80 mg, and 3 of 5 for the 80 mg continuous schedule. In Arm A, 10 of 27 participants had serious adverse events; in Arm B, 5 of 23; and in Arm C, 8 of 16. These are descriptive counts, not adjusted comparisons.
The record lists deaths as 2, 2, 6, and 3 across the four lead-in groups, and 20, 21, and 14 in Arms A, B, and C. The registry does not specify in this summary whether these deaths were treatment-related, nor does it provide a comparative time-to-event analysis for them. Counts should not be interpreted as causal attribution.
Other adverse events are listed for 3 of 3, 3 of 3, 6 of 6, and 5 of 5 participants across the lead-in groups, and 27 of 27, 19 of 23, and 10 of 16 across Arms A, B, and C. Group sizes and exposure durations may differ; exposure-adjusted rates are not reported in the registry record. The listed serious-event terms each carry 33.3%, but their denominator and calculation are not reported in the registry record. These figures describe the safety entries and do not alone establish a treatment difference.
12. What the Result Does, and Does Not, Mean
The posted primary result means that the registry records 80 mg as the optimal veliparib dose with gemcitabine hydrochloride and cisplatin for the Part I Lead In group. The group contains 17 participants, and the endpoint timeframe is 21 days. This result belongs to a non-randomized dose-selection component. It does not estimate how much adding veliparib changes response, progression-free survival, or overall survival compared with gemcitabine hydrochloride and cisplatin alone.
The randomized component is designed to compare those regimens, but the primary endpoint results do not provide values for the randomized arms. The registry also lists a single-agent veliparib study in previously treated pancreatic adenocarcinoma. A result from the lead-in cannot be transferred to that separate population or design. Four secondary outcomes are named, but no analyses are posted in the registry record.
General explanation: statistical evidence is most informative when an estimate is paired with a measure of uncertainty and interpreted in light of the study design. The posted dose value has no confidence interval in the registry record. The safety counts provide some descriptive information, but they do not include a complete comparative analysis or account for differing group sizes and exposure. These data therefore do not support a precise estimate of comparative benefit or harm.
The registry status is active, not recruiting, and the primary completion date is 2024-12-31. The record’s status and dates describe the study’s registry history; they do not substitute for posted outcome analyses. The result does not establish that a regimen is effective or ineffective, and it does not establish that the randomized groups had equivalent outcomes. It identifies a dose selected in the lead-in and records safety information for the listed groups. Further interpretation of comparative outcomes would require results and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically distinctive because its registry record combines a dose-finding lead-in, a randomized comparison, and a single-arm study. The primary endpoint belongs to the lead-in, whereas the broad treatment question concerns a randomized comparison. Keeping these questions separate prevents a dose-selection result from being mistaken for an estimate of comparative efficacy.
The study also illustrates why registry design labels need to be read alongside the specific study-part descriptions. Randomized allocation and a parallel model characterize the comparative component, but the lead-in is explicitly non-randomized and Part II is single arm. An overall enrollment number cannot describe the size of each component or provide the denominator for every analysis. The primary endpoint population is stated separately as the Part I lead-in cohorts.
Another statistical lesson is the difference between outcomes being named and analyses being available. The record identifies several secondary outcomes but posts no analyses. Without estimates, uncertainty intervals, and clearly defined analysis populations, the outcome list cannot answer the comparative questions that motivate the randomized design.
Finally, safety counts are not interchangeable with treatment-effect estimates. Counts of participants affected can be useful descriptions, but comparisons require attention to group size, exposure, outcome definitions, and uncertainty. The posted record supports a limited account of the lead-in dose and adverse-event counts, while leaving the randomized outcome comparisons unresolved. Recognizing that distinction is essential when reading a complex phase II trial 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 primary endpoint result is limited to the Part I lead-in, and the registry does not provide a randomized-arm result for that endpoint. The endpoint data list no values for the randomized arms, even though the safety record includes participants in those arms. The registry does not explain the absence of comparative endpoint values or provide a separate randomized primary outcome estimate.
The record gives no confidence interval, standard error, or other precision measure for the 80 mg dose result. It also does not post a dose-by-dose endpoint analysis that would allow readers to assess how each dose level contributed to the selected dose. Although the design description lists dose escalation and a conditional schedule evaluation, the statistical decision procedure and uncertainty around the decision are not reported in the registry record.
The overall enrollment is 83, but arm-specific enrollment for the randomized endpoint analysis is not posted. The adverse-event groups have different listed numbers at risk, and the record does not give exposure-adjusted safety rates or a statistical comparison. The serious adverse-event terms are accompanied by percentages whose denominator and calculation are not reported in the registry record. The death counts are not accompanied by a stated attribution or comparative survival analysis.
Although progression-free survival, disease control, duration of response, and overall survival are listed as secondary outcomes, no analyses are posted. The registry record also does not report multiplicity handling, missing-data methods, analysis populations for the randomized outcomes, or whether particular analyses were adjusted. These omissions limit reproducibility and make it impossible to assess the precision and robustness of comparative conclusions from this record alone.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Gemcitabine Hydrochloride and Cisplatin With or Without Veliparib or Veliparib Alone in Treating Patients With Locally Advanced or Metastatic Pancreatic Cancer — NCT01585805.
- Registry record last updated 2026-09-11; status last verified 2026-09.
- Results first posted 2026-05-19.
- 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
NCT01585805 is a phase II program sponsored by the National Cancer Institute. It concerns pancreatic adenocarcinoma and includes a non-randomized Part I lead-in, a randomized comparison of veliparib with gemcitabine hydrochloride and cisplatin against gemcitabine hydrochloride and cisplatin alone, and a separate single-arm Part II study of veliparib. The record lists overall enrollment of 83, no masking, and active, not recruiting status.
The posted primary endpoint is optimal veliparib dose in the Part I lead-in. The registry reports 80 mg for the Part I Lead In group, with 17 participants. This result does not supply a treatment-effect estimate for the randomized comparison. Four secondary outcomes are listed, but no analyses are posted. Safety counts are available for lead-in and treatment groups; they are descriptive and do not include a reported comparative analysis.