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 Testing Olaparib and AZD6738 in IDH1 and IDH2 Mutant Tumors (NCT03878095), a na phase 2 study. The primary outcome is Objective Response Rate (ORR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03878095 |
| Phase | Phase 2 |
| Condition | Malignant Solid Neoplasm, Refractory Cholangiocarcinoma, Refractory Malignant Solid Neoplasm |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 24 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Patients with IDH-mutant solid tumors, including cholangiocarcinoma, are the population of interest. The registry lists malignant solid neoplasm and refractory cholangiocarcinoma among the conditions. It records an enrollment of 24. The record does not report how participants were distributed across tumor types or provide further detail about the population included in the primary outcome analysis.
Intervention
The treatment is olaparib with ceralasertib, also identified as AZD6738. The treatment description specifies oral olaparib twice daily on days 1-28 of each cycle and oral ceralasertib once daily on days 1-7. Cycles repeat every 28 days while treatment continues under the conditions described in the record.
Comparator
There is no separate comparator group in the registry design. The study is listed as single-group, with allocation marked NA and masking listed as none. The treatment outcomes therefore cannot be read as a randomized comparison against another treatment or a control group.
Primary question
The primary question is whether participants receiving the combination have an objective tumor response, expressed as an objective response rate. The registry specifies exact two-sided 95% confidence intervals for this outcome. It also states that no responses were recorded to calculate the statistics outlined at registration, so the posted record does not provide an estimable response rate with its specified interval.
3. Trial Design
This is a phase II, single-group study sponsored by the National Cancer Institute (NCI). Its official title describes a study of olaparib and AZD6738 in IDH-mutant solid tumors. The registry lists malignant solid neoplasm, refractory malignant solid neoplasm, and refractory cholangiocarcinoma as conditions. Enrollment is listed as 24. The record does not report enrollment by tumor type, so the composition of the study population cannot be established from these details.
Participants in the treatment group receive olaparib and ceralasertib. Olaparib is taken by mouth twice daily on days 1-28 of each cycle; ceralasertib is taken by mouth once daily on days 1-7. Cycles repeat every 28 days in the absence of disease progression, unacceptable toxicity, withdrawal of consent, or death. Tumor biopsies are described during screening and on study. The listed interventions also include imaging, biospecimen collection, and bone marrow procedures, but the registry record does not describe their use for each participant.
Allocation is listed as NA, masking as none, and the model as single-group. Thus, the design does not include a like-for-like control comparison in the posted record. The registry lists the study as active, not recruiting. It records a start date of 2020-01-30 and a primary completion date of 2025-03-12. These dates and status describe the registry record; they do not establish how many participants completed treatment or contributed data to each outcome.
The primary outcome is objective response rate, with a timeframe extending up to 30 days post treatment after removal from study. Secondary outcomes listed are progression-free survival, overall survival, duration of response, and incidence of adverse events. The record identifies seven outcome measures overall. Detailed operational definitions, analysis populations, and statistical procedures beyond the stated confidence-interval plan are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Treatment (Olaparib, Ceralasertib) |
|---|---|
| STARTED | 24 |
| COMPLETED | 24 |
| NOT COMPLETED | 0 |
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 (Olaparib, Ceralasertib) |
|---|---|---|
| Participants analysed | 24 | |
| Age, Continuous | years | 59 (29 to 83) |
| Sex: Female, Male: Female | Participants | 9 |
| Sex: Female, Male: Male | Participants | 15 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 22 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 3 |
| 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 | 20 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 24 |
| ECOG Performance Status Scale: 0 | Participants | 7 |
| ECOG Performance Status Scale: 1 | Participants | 17 |
| Tumor Type: Cholangiocarcinoma | Participants | 14 |
| Tumor Type: Chondrosarcoma | Participants | 4 |
| Tumor Type: Other | Participants | 6 |
| IDH Mutation Status: IDH1 | Participants | 17 |
| IDH Mutation Status: IDH2 | Participants | 7 |
| Lines of Systemic Therapy | Prior lines of systemic therapy | 3 (0 to 6) |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response Rate (ORR), measured over Up to 30 days post treatment after removal from study.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
Objective response rate (ORR) is the proportion of participants who meet the study’s criteria for an objective tumor response during the defined assessment period. It is a binary outcome at the participant level: each participant is classified according to whether a response is recorded. The registry lists the unit as participants and gives the timeframe as up to 30 days post treatment after removal from study.
As a general statistical concept, response rate is used to describe how often a prespecified response occurs in a study population. It can provide a concise summary of tumor response, but it does not by itself describe how long a response lasts, whether disease progression is delayed, or survival. The registry separately lists duration of response, progression-free survival, and overall survival as secondary outcomes.
The registry states that exact two-sided 95% confidence intervals for ORR will be reported. In general, a confidence interval expresses uncertainty around an estimate and indicates which values are compatible with the observed data under the method’s assumptions. In this record, no responses were recorded to calculate the statistics outlined at registration. Consequently, the posted results do not give an ORR estimate or its specified confidence interval. That absence should not be interpreted as a calculated response rate of zero; the record does not provide the response data needed to make that calculation.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The most important interpretive feature is that the registry does not provide a calculated primary outcome. It identifies ORR as the primary endpoint and specifies exact two-sided 95% confidence intervals, but the outcome description says no responses were recorded to calculate the statistics outlined at registration. A point estimate and interval are therefore not available in the posted record. Without them, the precision of an estimated response rate cannot be evaluated.
The wording about no recorded responses is not equivalent to a reported estimate showing that the response rate was zero. It indicates that the record lacks response observations from which the planned statistics could be calculated. The reason that response data were not recorded is not reported in the registry record. Nor does the record give an alternative primary analysis, an explanation of whether data collection was incomplete, or a revised endpoint analysis.
The study is single-group and has no registered comparator arm. Any description of outcomes among treated participants would therefore be descriptive, not a randomized estimate of the difference between the combination and another treatment. In general, a single-group study can describe outcomes in its enrolled population, but it cannot isolate a treatment effect from other influences in the way a randomized comparison is intended to do. This matters especially when considering tumor types grouped under a broad solid-tumor description.
The registry lists an enrollment of 24, but it does not report the number included in each outcome analysis. Enrollment should not be treated as the denominator for ORR or any secondary outcome unless the analysis population and available outcome data are specified. The posted record does not report those details. It also does not report outcome estimates for progression-free survival, overall survival, or duration of response in the information described by the primary outcome entry.
Safety information includes counts of participants affected by serious and other adverse events, a list of serious-event terms with percentages, and one death recorded in the treatment group. These entries describe safety reporting in the registry; they do not establish that the treatment caused each event or death. Attribution and fuller context are not reported in the registry record.
The record’s summary gives a laboratory rationale for studying the combination in IDH-mutant tumors. That rationale is not clinical outcome evidence. The results described in the registry support neither a conclusion that the combination is effective nor a conclusion that it is ineffective. The primary response statistics are unavailable, and the design lacks a concurrent comparator. Interpretation must remain limited to the registry’s design and the outcome information it records.
9. Statistical Methods Explained
The registry describes a planned primary analysis of objective response rate (ORR), with exact two-sided 95% confidence intervals. It does not provide a response estimate or interval: the outcome description states that no responses were recorded to calculate the statistics outlined at registration. The methods below distinguish what the record specifies from general statistical principles; additional analytic details are not reported in the registry record.
What does a response rate estimate?
As a general statistical concept, a response rate is a proportion: the number of participants meeting a response definition relative to the number evaluated under the analysis rules. The outcome is binary for each participant, but the response criteria, assessment schedule, confirmation requirements, and handling of unevaluable participants are not reported in the registry record. Those details matter because they determine who is counted in the numerator and denominator.
The record identifies participants as the unit of the primary outcome. It does not identify an analysis population or state whether all enrolled participants, all treated participants, or another group would contribute to the estimate. The enrollment of 24 cannot be assumed to be the denominator for the response analysis. In general, a proportion depends on both the number meeting the outcome definition and the population included in the calculation.
Why specify a confidence interval?
As a general statistical concept, a confidence interval conveys uncertainty around an estimate. A wider interval indicates less precision, while a narrower interval indicates greater precision under the method’s assumptions. The registry specifies an exact two-sided 95% interval. Exact methods are commonly used for proportions, particularly when data are sparse, because they avoid relying on some large-sample approximations. The exact method named beyond this description, and any choices about its implementation, are not reported in the registry record.
A confidence interval requires outcome data. Here, the registry says no responses were recorded to calculate the planned statistics, so there is no reported response estimate around which to interpret an interval. It would be inappropriate to substitute an assumed response count or construct an interval from enrollment alone. The absence of a calculated interval does not reveal how precise a response estimate would have been if outcome data had been available.
What does the design imply for comparisons?
The study model is single-group, allocation is listed as NA, and masking is listed as none. In general, a single-group design describes outcomes within the treated group; it does not directly estimate a contrast against a concurrent control group. This means that no treatment-versus-control effect can be derived from the registered design. Comparisons with external experience, if any, are not described in the registry record and should not be assumed.
Because the study includes IDH-mutant solid tumors and lists cholangiocarcinoma among the conditions, the population may encompass different tumor diagnoses. The registry record does not give a tumor-type breakdown or specify whether response would be analyzed separately by tumor type. As a general matter, pooling heterogeneous populations can make an overall estimate difficult to interpret if the groups differ in baseline characteristics or response patterns. Whether that issue arose in this study cannot be determined from the recorded information.
How should the secondary outcomes be understood?
The registry lists progression-free survival, overall survival, duration of response, and incidence of adverse events as secondary outcomes. These outcomes answer different questions. In general, time-to-event outcomes concern the time until a defined event, while duration of response concerns the period a response is maintained among participants who respond. Their interpretation depends on event definitions, censoring rules, follow-up, and analysis populations. Those details and corresponding estimates are not reported in the registry record.
Adverse-event outcomes also require clear definitions of the event, severity, attribution, observation period, and denominator. The safety entry gives participant counts at risk and affected, and lists selected serious-event terms with percentages. It does not supply the additional analytic details needed to interpret all possible event comparisons. The record identifies seven outcome measures overall and zero analyses. The precise relationship between the listed outcomes and the analyses that would have been conducted is not reported.
What methods remain unknown?
The registry does not report a detailed statistical analysis plan, sample-size justification, interim-analysis plan, missing-data method, or analysis populations. It also does not describe the operational response criteria or provide outcome-specific denominators. These are not minor technicalities: they can affect which observations enter an analysis and how uncertainty is represented. Their absence from the registry record means that readers cannot reconstruct the intended analysis beyond the stated exact two-sided 95% confidence intervals for ORR.
As a general statistical principle, sample-size planning and observed results serve different purposes. A planning calculation describes assumptions about a future study; it is not a substitute for observed outcomes, and it is not observed power. No trial-specific sample-size justification is reported in the registry record. The planned enrollment alone does not show how much precision the primary endpoint achieved, particularly when the response statistics were not calculated.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists ORR as the primary outcome and identifies progression-free survival, overall survival, duration of response, and incidence of adverse events as secondary outcomes. It also records seven outcome measures overall. When a study evaluates multiple outcomes, multiplicity can matter: as a general statistical concept, examining many hypotheses can increase the chance of obtaining an apparently notable result by chance. A prespecified strategy may address this issue, but a multiplicity adjustment or testing hierarchy is not reported in the registry record. The record also gives zero analyses, so it does not present a set of outcome analyses from which a multiplicity strategy can be assessed.
The primary outcome description specifies exact two-sided 95% confidence intervals for ORR, but the record says no responses were recorded to calculate the statistics outlined at registration. It does not state how missing or unevaluable response assessments would be handled, or whether any participants lacked assessments. Missing-data procedures and outcome-specific analysis populations are not reported in the registry record.
The enrollment listed is 24. That figure does not identify how many participants contributed to each endpoint, whether every enrolled participant received treatment, or which population would be used for a particular analysis. Those distinctions are important in general because excluding participants or observations can change both an estimated outcome and its precision. The registry record does not report whether analyses were intended to include all enrolled participants, treated participants, or another defined group.
The record does not report an interim-analysis plan, stopping rule, or detailed statistical analysis plan. Their absence from the record should be read as missing documentation, not evidence that a particular approach was or was not used. No assumptions about handling incomplete follow-up or multiple outcomes can be verified from the posted information.
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 (Olaparib, Ceralasertib) | 13 | 24 | 54.2% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment (Olaparib, Ceralasertib) | 1 of 24 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Treatment (Olaparib, Ceralasertib) | 24 of 24 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Olaparib, Ceralasertib) |
|---|---|---|
| Nausea | Gastrointestinal disorders | 2 of 24 |
| Platelet count decreased | Investigations | 2 of 24 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 2 of 24 |
| Anemia | Blood and lymphatic system disorders | 1 of 24 |
| Pericardial effusion | Cardiac disorders | 1 of 24 |
| Abdominal pain | Gastrointestinal disorders | 1 of 24 |
| Ascites | Gastrointestinal disorders | 1 of 24 |
| Vomiting | Gastrointestinal disorders | 1 of 24 |
| Disease progression | General disorders | 1 of 24 |
| Fatigue | General disorders | 1 of 24 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Olaparib, Ceralasertib) |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 17 of 24 |
| Nausea | Gastrointestinal disorders | 12 of 24 |
| Constipation | Gastrointestinal disorders | 9 of 24 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 9 of 24 |
| Vomiting | Gastrointestinal disorders | 8 of 24 |
| Fatigue | General disorders | 8 of 24 |
| Lymphocyte count decreased | Investigations | 8 of 24 |
| Platelet count decreased | Investigations | 8 of 24 |
| Anorexia | Metabolism and nutrition disorders | 8 of 24 |
| White blood cell decreased | Investigations | 7 of 24 |
Serious events with the highest arm-level rate: nausea (8.3%), platelet count decreased (8.3%), hypoxia (8.3%), anemia (4.2%), pericardial effusion (4.2%).
The safety entry lists one treatment group, with 24 participants at risk for serious adverse events and 13 affected. For other adverse events, it lists 24 at risk and 24 affected. These are participant counts, not counts of the total number of event episodes. A participant may experience more than one event, and the registry entry does not provide a full event-level accounting.
The listed serious adverse-event terms include nausea, platelet count decreased, and hypoxia, each with a reported percentage of 8.3%. Anemia, pericardial effusion, and abdominal pain are each listed at 4.2%. These percentages are quoted as recorded; the registry entry does not provide additional detail about event timing, recurrence, severity grading beyond the serious-event classification, or attribution to treatment.
One death is recorded in the treatment group. The entry does not state whether the death was considered related to treatment or provide its cause. Counts and percentages describe what the registry records, but do not establish causation. The treatment group is single-arm, so the safety figures cannot be interpreted as a comparative excess relative to a control group.
12. What the Result Does, and Does Not, Mean
The registry record describes a phase II investigation of olaparib and ceralasertib in IDH-mutant solid tumors, including cholangiocarcinoma. It identifies ORR as the primary endpoint and specifies an exact two-sided 95% confidence interval. However, the record says no responses were recorded to calculate the statistics outlined at registration. As a result, the primary response estimate and its precision are not available in the posted record.
This does not establish a response rate of zero, nor does it establish that participants had no clinical benefit. The record does not give the response observations or explain why the planned statistics could not be calculated. It therefore cannot support a conclusion about the magnitude of the response rate. It also does not provide the outcome estimates needed to draw conclusions about progression-free survival, overall survival, or duration of response.
The single-group design is another important boundary on interpretation. There is no concurrent randomized comparator in the registry record, so outcomes cannot be used to estimate a direct treatment effect relative to another group. Even if outcomes were available, descriptive results in a single group would need to be interpreted in light of the enrolled population, follow-up, and outcome definitions. Several of those details are not reported.
The safety counts provide information about participants recorded as affected by serious and other adverse events, and the record lists selected serious-event terms and one death. These entries do not by themselves establish that treatment caused an event. The registry does not provide a comparative safety estimate or the full context needed to assign causation.
The registry summary describes laboratory findings as the rationale for testing the combination. Laboratory rationale is distinct from clinical evidence. The study’s stated purpose and treatment schedule describe what was investigated; they do not establish a treatment effect. The appropriate statistical conclusion is limited: the registry records a single-group phase II study and a primary response analysis that could not be calculated from recorded responses, alongside safety counts with limited comparative context.
13. Why This Trial Matters Statistically
This trial illustrates how a clearly named primary endpoint can remain statistically uninformative when the outcome data needed to calculate it are not recorded. ORR is specified, and the registry states that exact two-sided 95% confidence intervals would be reported. Yet it also states that no responses were recorded to calculate the statistics outlined at registration. The endpoint’s name and planned interval do not substitute for an estimate and its uncertainty.
It also highlights the difference between enrollment and an analysis denominator. The registry lists enrollment of 24, but does not state how many participants contributed data to each outcome. Without those denominators, readers cannot reconstruct an outcome rate or assess its precision. This distinction is fundamental when reporting participant-level outcomes.
The single-group design makes the scope of inference equally important. The record does not establish a randomized comparison, so it cannot show how outcomes differ from those under another treatment or a control condition. A treatment rationale based on laboratory findings does not resolve that limitation. Statistical interpretation has to separate the biological rationale from what the clinical outcome record can support.
Finally, the record lists several secondary outcomes and safety information, but does not report detailed analysis populations, missing-data methods, or a multiplicity strategy. These omissions matter because choices about endpoint definitions, denominators, and multiple analyses affect interpretation. The trial is therefore a useful example of why transparent reporting requires not only an endpoint and study design, but also analyzable outcome data and enough methodological detail to understand how estimates were obtained.
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 main trial-specific limitation in the registry record is the unavailable primary response analysis. The record states that no responses were recorded to calculate the statistics outlined at registration, so it does not provide an ORR estimate or the specified confidence interval. It does not explain why the response observations were unavailable. The absence of a calculated result prevents assessment of the response estimate’s direction or precision.
The design is single-group, with no separate comparator. Therefore, the registered study cannot furnish a randomized comparison of the combination against another treatment. Masking is listed as none. These design features do not invalidate descriptive reporting, but they limit the kinds of causal or comparative conclusions that can be drawn.
The record lists enrollment as 24 but does not report the analysis denominator for the primary or secondary outcomes. It does not give enrollment or outcome data by tumor type, despite listing multiple solid-tumor conditions. This makes it difficult to judge which participants are represented by any outcome result and whether an overall summary would apply consistently across diagnoses.
Operational details are also limited. Response criteria, assessment schedules, handling of unevaluable participants, analysis populations, and missing-data procedures are not reported in the registry record. Neither a sample-size justification nor a detailed statistical analysis plan is reported. The record lists secondary outcomes, but does not provide their estimates in the primary outcome entry or describe how multiple outcomes would be addressed.
The safety entry includes participant counts, selected serious-event terms, percentages, and one death, but does not report attribution or enough detail to establish causation. These limitations are specific to the information recorded and should not be taken as evidence that additional data or documentation do not exist elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Testing Olaparib and AZD6738 in IDH1 and IDH2 Mutant Tumors — NCT03878095.
- Registry record last updated 2026-08-03; status last verified 2026-07.
- Results first posted 2026-04-08.
- 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 phase II, single-group study of olaparib and ceralasertib (AZD6738) in IDH-mutant solid tumors, including cholangiocarcinoma. The National Cancer Institute (NCI) is listed as sponsor, and enrollment is recorded as 24. The treatment schedule uses 28-day cycles, with olaparib taken orally on days 1-28 and ceralasertib on days 1-7.
ORR is the primary outcome, with exact two-sided 95% confidence intervals specified. The record states that no responses were recorded to calculate the statistics outlined at registration. Secondary outcomes listed are progression-free survival, overall survival, duration of response, and incidence of adverse events. The safety entry records 13 participants affected by serious adverse events among 24 at risk, 24 affected by other adverse events among 24 at risk, and one death. Allocation is NA, masking is none, and the study model is single-group. Further statistical analysis details are not reported in the registry record.