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 Niraparib and Dostarlimab for the Treatment of Germline or Somatic BRCA1/2 and PALB2 Mutated Metastatic Pancreatic Cancer (NCT04493060), a na phase 2 study. The primary outcome is Disease Control Rate at 12 Weeks (DCR12).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04493060 |
| Phase | Phase 2 |
| Condition | Metastatic Pancreatic Ductal Adenocarcinoma, Stage IV Pancreatic Cancer AJCC v8 |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Mayo Clinic |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic pancreatic cancer and germline or somatic BRCA1/2 or PALB2 mutations. The registry record also identifies metastatic pancreatic ductal adenocarcinoma and stage IV pancreatic cancer as conditions of interest. It does not report further eligibility details or a description of the participants enrolled.
Intervention
The treatment was niraparib together with dostarlimab. The registry describes niraparib taken by mouth once daily on days 1–21 and dostarlimab given intravenously on day 1. Dostarlimab was scheduled every three weeks for cycles 1–4 and every six weeks for subsequent cycles. Cycles were planned to repeat every 21 days for up to two years, unless disease progression or unacceptable toxicity occurred.
Comparator
There was no separate comparator group. The study is recorded as single-group, with allocation listed as not applicable. Its results therefore do not provide a randomized comparison with another treatment or a control group.
Primary question
The primary endpoint asks how many participants had disease control at 12 weeks, assessed using iRECIST. The registry record does not report that count or a corresponding response estimate. It therefore does not establish from the posted primary endpoint result how often disease control occurred in this study.
3. Trial Design
This completed phase II study evaluated niraparib and dostarlimab in patients with metastatic pancreatic cancer associated with germline or somatic BRCA1/2 or PALB2 mutations. The registry lists Mayo Clinic as the sponsor, an enrollment of 22, a start date of 2020-12-28, and a primary completion date of 2023-12-07. The study status is recorded as completed.
The design is recorded as single-group, and allocation is listed as not applicable. Masking is listed as none. Participants received the combination regimen rather than being assigned to separate treatment and control groups. Consequently, the study can describe outcomes among participants receiving the regimen, but it cannot by itself estimate a randomized treatment contrast or separate treatment effects from other explanations for observed outcomes.
The regimen description specifies oral niraparib on days 1–21 and intravenous dostarlimab on day 1, with dostarlimab scheduled every three weeks for cycles 1–4 and every six weeks in subsequent cycles. Cycles were to repeat every 21 days for up to two years in the absence of disease progression or unacceptable toxicity. This is the planned schedule in the registry record; it does not report how closely participants followed it or how long they actually received treatment.
The primary endpoint was disease control rate at 12 weeks, assessed using iRECIST. Secondary outcomes include objective response rate, time to next treatment, overall survival, time to and duration of confirmed response, progression-free survival, and adverse events. The registry record lists 13 outcome measures overall. It does not report the primary endpoint result or details of the statistical analysis plan.
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 | Treatment (Niraparib, Dostarlimab) |
|---|---|
| STARTED | 22 |
| COMPLETED | 18 |
| NOT COMPLETED | 4 |
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 | Treatment (Niraparib, Dostarlimab) |
|---|---|---|
| Participants analysed | 18 | |
| Age, Continuous | years | 61.3 (SD 8.49) |
| Sex: Female, Male: Female | Participants | 11 |
| Sex: Female, Male: Male | Participants | 7 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 18 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 17 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Number of Prior Treatment(s): 1 | Participants | 3 |
| Number of Prior Treatment(s): 2 | Participants | 7 |
| Number of Prior Treatment(s): 3+ | Participants | 8 |
| Mismatch Repair Status: Deficient | Participants | 2 |
| Mismatch Repair Status: Proficient | Participants | 15 |
| Deleterious Mutation: BRCA 1 | Participants | 5 |
| Deleterious Mutation: BRCA 2 | Participants | 12 |
| Deleterious Mutation: PALB2 | Participants | 1 |
| Platinum Tx Exposure: Yes | Participants | 16 |
| Platinum Tx Exposure: No | Participants | 2 |
| Refractory To Cancer Tx: Yes | Participants | 5 |
| Refractory To Cancer Tx: No | Participants | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Disease Control Rate at 12 Weeks (DCR12), measured over At 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is disease control rate at 12 weeks, described as a count of participants and assessed using the standard immune-modified Response Evaluation Criteria in Solid Tumors, or iRECIST. A disease-control endpoint is intended to capture participants meeting the study’s criteria for disease control at a specified assessment time. The registry record does not define the particular response categories counted as disease control, so those details should not be inferred from the endpoint name alone.
Using a fixed assessment time gives the endpoint a clear time reference: the outcome concerns disease status at 12 weeks rather than an unspecified point during follow-up. iRECIST is an immune-modified response framework. As a general statistical point, a prespecified response framework helps standardize how observed disease status is classified, which matters when interpreting a count based on assessments.
The registry record does not report the number of participants who met the endpoint, the number assessed at 12 weeks, or an uncertainty interval. It also does not report how missing or unevaluable assessments were handled. Without those details, the endpoint’s observed frequency and its precision cannot be described from the registry record.
A count is not itself a treatment comparison. In this single-group study, the primary endpoint could describe the experience of participants receiving the combination, but there is no randomized comparator in the record against which to estimate a between-group difference. The registry record also does not report a threshold for interpreting the disease-control result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical limitation is that the registry record identifies the primary endpoint but does not report its result. Disease control at 12 weeks was to be assessed with iRECIST, yet no count of participants meeting that endpoint, denominator assessed, or estimate of uncertainty is reported. Accordingly, the posted record does not allow a reader to quantify the primary outcome or judge its precision.
The study is recorded as phase II and single-group, with no masking and no separate comparator. Those design features define what the data could describe: outcomes among participants treated with niraparib and dostarlimab. Without a concurrent randomized control group, any observed outcome would not by itself establish what would have happened to comparable participants under a different treatment or no study treatment. Differences between the observed participants and an external group could also complicate comparisons, and no such external comparison is reported in the registry record.
The enrollment is recorded as 22. That enrollment figure does not tell how many participants were included in a particular endpoint analysis, how many had an evaluable assessment at 12 weeks, or how many contributed follow-up for time-to-event outcomes. These analysis-specific numbers are not reported in the registry record. It would therefore be inappropriate to treat enrollment as the denominator for every outcome.
The record lists several secondary outcomes, including objective response rate, time to next treatment, overall survival, progression-free survival, duration of confirmed response, and adverse events. Their names indicate the kinds of outcomes considered, not their observed results. Except for the adverse-event counts and terms recorded in the safety data, outcome estimates and their uncertainty are not reported in the registry record.
The safety figures describe recorded events in the treatment group, including two participants affected by a serious event and two deaths. The record also reports other adverse events for the group. These are descriptive registry counts; the information does not establish the cause of deaths, their relationship to treatment, or whether a recorded event was more common than it would have been without the regimen.
Statistical interpretation depends on both the estimate and how precisely it is measured. A confidence interval, when reported, describes uncertainty around an estimate under the analysis assumptions; it does not resolve limitations such as the lack of a control group or incomplete outcome reporting. No primary-endpoint confidence interval or statistical test result is reported. Thus, the available record supports a description of the design and reported safety counts, but not a quantitative conclusion about the primary endpoint or a comparative treatment effect.
9. Statistical Methods Explained
The registry record identifies the study as phase II, single-group, and unmasked. It does not report a detailed statistical analysis plan. The explanations below describe general statistical concepts relevant to interpreting the listed design and endpoints; they do not establish which specific methods were used beyond what the record states.
What does a single-group design estimate?
In a single-group study, outcome estimates describe participants who received the study regimen. A count or rate can summarize what was observed in that group, but it does not directly estimate a difference between the regimen and a control treatment. Random assignment in a comparative study helps make groups comparable on average; without that feature, differences from another population may reflect differences in participant characteristics, clinical setting, follow-up, or outcome measurement as well as treatment.
For this study, allocation is listed as not applicable and the model as single-group. There is no separate comparator arm in the registry record. A comparison with historical experience would require a suitable external source and careful attention to comparability; no such analysis is reported in the registry record.
What does an endpoint count convey?
The primary endpoint is a count of participants with disease control at 12 weeks under iRECIST. A count conveys how many participants met the endpoint only when the count and the relevant analysis denominator are available. A rate or proportion would also depend on the denominator and its definition. Neither the primary count nor its denominator is reported in the registry record, so neither can be interpreted from the listed endpoint description alone.
In general, a confidence interval communicates statistical uncertainty around an estimated quantity. A wider interval indicates less precision than a narrower interval, under the method’s assumptions. It does not indicate whether the study design eliminates bias, and it does not substitute for a control group. No confidence interval for the primary endpoint is reported in the registry record.
How should response criteria be understood?
iRECIST is the response framework named for the primary endpoint. In general, standardized criteria help define how clinical observations are classified, so that endpoint results can be interpreted using stated rules rather than informal judgment. Interpretation still requires knowing which categories count toward the endpoint, how assessments were timed, and how absent or unevaluable assessments were handled. Those details are not reported in the registry record beyond the 12-week timeframe and the use of iRECIST.
The registry lists objective response rate and time to and duration of confirmed response as secondary outcomes. These are distinct from disease control at a fixed time. A response endpoint concerns participants meeting response criteria, while time-to-event outcomes concern how long it takes for an event to occur or whether it occurs during follow-up. The outcome names alone do not specify the exact analysis rules or results.
What do time-to-event outcomes require?
Overall survival, progression-free survival, and time to next treatment are listed secondary outcomes. In general, time-to-event analyses use follow-up time and account for participants whose event has not been observed by the end of their available follow-up. The resulting estimate depends on definitions of the event, the start of follow-up, censoring rules, and the amount of follow-up. Those details and results are not reported in the registry record. A listed time-to-event outcome therefore should not be mistaken for a reported survival estimate.
For time-to-event data, events and censoring both matter. A participant without an observed event by the last available assessment may contribute information up to that point, but is not equivalent to a participant followed until an event. In general, informative loss to follow-up can affect interpretation if the chance of being lost is related to prognosis or outcome. The registry record does not describe follow-up completeness or censoring methods.
What are the planning assumptions?
The record describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. These are assumptions for a planning illustration, not a description of the actual single-group trial design. The record specifies that time-to-event planning uses a total number of events, while other planning outputs use participants per arm. The planning result values are not reported in the registry record.
As a general concept, a sample-size calculation estimates how much information a proposed study might need under specified assumptions. It is not an observed result and is not observed power. A planning calculation depends on assumptions about the effect, variability or event rate, statistical threshold, allocation, and analysis method. If those assumptions differ from what occurs, the resulting precision can differ from the plan. Here the two-arm equal-allocation assumption is not the recorded single-group design, so the illustration should not be read as a description of the enrollment or analysis actually used.
Why do missing data and analysis populations matter?
Endpoint counts can change in meaning depending on which participants are included and how missing assessments are treated. An analysis restricted to participants with an evaluable 12-week assessment may not describe everyone enrolled if some have no such assessment. Other rules could count missing observations differently. The record does not report an analysis population or a missing-data rule for the primary endpoint, so neither should be assumed.
Similarly, the enrollment figure does not establish the denominator for safety, response, or time-to-event analyses. Each outcome can have a different evaluable population or follow-up structure. Clear reporting of those details helps readers assess the reach and precision of each estimate. The registry record lists zero analyses, and it does not provide the corresponding statistical tests, estimates, or uncertainty measures.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 13 outcome measures, including the primary disease-control endpoint and secondary response, timing, survival, and safety outcomes. When a study examines multiple outcomes, multiplicity can arise if several statistical tests are used to support claims. In general, testing many outcomes without an appropriate plan can increase the chance of at least one apparently noteworthy result arising by chance. The registry record does not report whether any multiplicity adjustment was planned or used, so no such adjustment should be assumed.
The record identifies disease control at 12 weeks as the primary endpoint, but it does not report a statistical analysis plan, a prespecified hypothesis test, a decision threshold for the endpoint, or an estimate with a confidence interval. It also does not report whether any interim analyses were conducted. These items are not reported in the registry record; their absence should not be filled in by guessing.
Missing-data methods and analysis populations are also not reported. The registry record does not state how participants without a 12-week assessment were handled, how unevaluable assessments were classified, or which participants were included in each outcome analysis. The enrollment of 22 cannot be substituted for outcome-specific denominators. Without these details, it is not possible to assess whether missing observations could affect the reported endpoint or to determine the precise population represented by an estimate.
In general, analysis-population definitions and missing-data rules clarify which participants contribute to an estimate and how incomplete follow-up is treated. These choices can affect interpretation, particularly for a fixed-time endpoint and for time-to-event outcomes. The registry record lists zero analyses and does not report results for the primary endpoint. As a result, neither multiplicity handling nor the impact of missing outcome data can be evaluated from the posted 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 |
|---|---|---|---|
| Treatment (Niraparib, Dostarlimab) | 2 | 20 | 10.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment (Niraparib, Dostarlimab) | 2 of 20 |
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 |
|---|---|
| Treatment (Niraparib, Dostarlimab) | 20 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Niraparib, Dostarlimab) |
|---|---|---|
| Asystole | Cardiac disorders | 1 of 20 |
| Infections and infestations - Oth spec | Infections and infestations | 1 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Niraparib, Dostarlimab) |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 19 of 20 |
| Fatigue | General disorders and administration site conditions | 17 of 20 |
| Pain | General disorders and administration site conditions | 13 of 20 |
| Hyperglycemia | Metabolism and nutrition disorders | 11 of 20 |
| Platelet count decreased | Investigations | 8 of 20 |
| Aspartate aminotransferase increased | Investigations | 6 of 20 |
| Diarrhea | Gastrointestinal disorders | 5 of 20 |
| Creatinine increased | Investigations | 5 of 20 |
| Lymphocyte count decreased | Investigations | 5 of 20 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 5 of 20 |
Serious events with the highest arm-level rate: asystole (5.0%), infections and infestations - oth spec (5.0%).
The registry record lists adverse-event information for the treatment group. It reports 20 participants at risk for serious events and 2 affected, along with 20 at risk for other events and 20 affected. It records 2 deaths. These are counts as listed in the registry record; they should not be combined or treated as interchangeable measures. The record does not provide further details about the event definitions, timing, severity grading, or relationship to treatment.
The listed serious-event terms include “Asystole” at 5.0% and “Infections and infestations - Oth spec” at 5.0%. These are reported terms and percentages, not evidence by themselves about cause or treatment attribution. The registry figures do not establish whether a death was due to an adverse event, whether an event was caused by treatment, or whether its frequency differs from that under another treatment.
In general, safety counts are descriptive and depend on how events are defined, collected, and attributed. A count of affected participants is not necessarily the same as a count of individual events, since one participant may experience more than one event; the record does not clarify event-level totals. With no comparator group, the figures do not support a randomized comparison of safety outcomes.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase II, single-group study of niraparib and dostarlimab was completed for metastatic pancreatic cancer associated with germline or somatic BRCA1/2 or PALB2 mutations. It identifies disease control at 12 weeks under iRECIST as the primary endpoint and lists several secondary outcomes. It also records safety counts for the treatment group.
Those details do not provide the primary endpoint result. The disease-control count, its denominator, and an uncertainty interval are not reported in the registry record. The record therefore does not support a numerical statement about the frequency or precision of disease control at 12 weeks. The listed secondary outcomes likewise should not be read as results; their estimates are not reported in the registry record.
The study’s single-group design also limits what any observed outcome could establish. An outcome among treated participants would describe their experience, but without a randomized comparator it would not directly quantify how outcomes differ from another treatment or from no study treatment. The registry record cannot by itself establish that the combination caused an outcome or separate treatment effects from other factors.
The safety counts and serious-event terms are descriptive registry information. They do not establish the cause of the recorded deaths or events, nor do they establish whether the regimen changes risk relative to a comparator. The record does not report the detail needed to assess event attribution or compare safety between groups.
In general, a statistical result is most informative when its estimate, uncertainty, analysis population, and method are clear. Those details are not reported for the primary endpoint in this record. The appropriate reading is therefore limited: the design, intended endpoint, regimen, and listed adverse-event counts can be described, but a treatment-effect conclusion or a quantitative claim about the primary outcome cannot be drawn from the registry information reported.
13. Why This Trial Matters Statistically
This study raises a clear statistical question: what outcomes were observed when niraparib and dostarlimab were given together to participants with metastatic pancreatic cancer and germline or somatic BRCA1/2 or PALB2 mutations? The registry record describes the treatment schedule and identifies disease control at 12 weeks as the primary endpoint. That makes the intended outcome and assessment time explicit, even though the result itself is not reported.
The single-group design is important for interpreting the question. It can describe outcomes within the treated group, but it does not create a randomized contrast with another treatment. This distinction is central to clinical statistics: a within-group description and a treatment-effect estimate answer different questions. The registry record lists no comparator group, so its design cannot directly estimate a between-group difference.
The combination also makes attribution a consideration. Even if an outcome were reported, a single-group study of two agents would not, by itself, identify the separate contribution of each agent. This is a general interpretive limitation of evaluating a combination without comparative groups; the registry record does not report an analysis that separates component effects.
The record also illustrates why outcome reporting needs more than an endpoint name. A count, its analysis denominator, uncertainty, missing-data handling, and analysis population are needed to understand what an endpoint estimate represents. Those details are not reported for the primary endpoint. The registry’s safety counts similarly require careful distinction between descriptive reporting and evidence of comparative risk.
For biostatisticians and clinical researchers, the study is therefore useful as an example of the boundary between design information and outcome evidence. The record documents the planned clinical question and the single-group structure, but does not report the primary result or statistical analysis details needed for a precise efficacy interpretation.
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 of the registry information is that it does not report the primary endpoint result. Although disease control at 12 weeks is named and iRECIST is specified, the count of participants meeting the endpoint and the number assessed are not reported. No primary-endpoint uncertainty interval or statistical test result is reported either.
The study is single-group, with no comparator and no masking. This limits causal interpretation and prevents a direct randomized comparison with another treatment. Because niraparib and dostarlimab were administered together, the design also does not isolate the contribution of either component. These are design limitations, not evidence for or against a treatment effect.
The registry record reports an enrollment of 22, but does not report outcome-specific analysis populations or denominators. It also does not describe missing-data rules, the handling of unevaluable 12-week assessments, or follow-up completeness for time-to-event outcomes. Consequently, the information needed to understand who contributed to each estimate is not available.
Although 13 outcome measures are listed, the record does not report a detailed statistical analysis plan, multiplicity handling, or whether interim analyses occurred. It lists zero analyses. The status of completed does not replace reporting of the estimates and methods used to analyze outcomes.
Safety information is also limited. The record gives counts of affected participants and deaths, and lists two serious-event terms with percentages, but does not report further context about event-level counts, attribution, timing, or comparison with a control group. These omissions limit interpretation of both efficacy and safety beyond the descriptive details recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Niraparib and Dostarlimab for the Treatment of Germline or Somatic BRCA1/2 and PALB2 Mutated Metastatic Pancreatic Cancer — NCT04493060.
- Registry record last updated 2026-09-03; status last verified 2026-08.
- Results first posted 2025-08-07.
- 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 record describes a completed phase II study sponsored by Mayo Clinic, with an enrollment of 22. It evaluated niraparib and dostarlimab in a single group of patients with metastatic pancreatic cancer associated with germline or somatic BRCA1/2 or PALB2 mutations. Allocation is listed as not applicable and masking as none.
The primary endpoint was disease control rate at 12 weeks, assessed using iRECIST. Secondary outcomes included response, treatment timing, survival, and adverse events. The primary endpoint result and statistical analysis details are not reported in the registry record. Safety information includes 2 participants affected by serious events, 20 affected by other events, and 2 deaths; the record lists Asystole and “Infections and infestations - Oth spec” among serious-event terms at 5.0% each.