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 Oral ONC201 in Relapsed/Refractory Multiple Myeloma (NCT02863991), a randomized phase 1, phase 2 study. The primary outcome is Response of Participants at Last On-study Visit (End of Treatment/Follow-up).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02863991 |
| Phase | Phase 1, Phase 2 |
| Condition | Multiple Myeloma |
| Allocation | Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 17 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Jazz Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Adults with relapsed/refractory multiple myeloma. The registry record reports enrollment of 17, but does not give further eligibility details or describe the characteristics of participants enrolled.
Intervention
Oral ONC201 once every week in combination with dexamethasone. The arm descriptions specify 375 mg ONC201 with 20 mg dexamethasone in Phase 1 and 625 mg ONC201 with 20 mg dexamethasone in Phase 2.
Comparator
No separate control treatment is described. The record identifies randomized allocation, but also labels the model as single-group and describes two dose groups. It does not explain how participants were allocated or whether the dose groups were intended as a direct comparison.
Primary question
The stated primary objective was to evaluate antitumor efficacy. The primary measure counts participants by response status at the last on-study visit, at the end of treatment or follow-up. Response was assessed using International Myeloma Working Group criteria. The registry record does not report the response counts or an analysis comparing response between groups.
In practical terms, the clinical question is whether participants receiving ONC201 with dexamethasone had response statuses recorded at their final on-study assessment. The record describes the treatment and response definitions, but it does not provide the primary outcome values needed to describe what responses occurred. Nor does it provide an estimate of a treatment effect, a confidence interval, or a statistical test for the primary measure. The question therefore remains a description of the study objective rather than a result that can be answered from the registry record.
3. Trial Design
This was a Phase 1/2, open-label study of oral ONC201 with dexamethasone in adults with relapsed/refractory multiple myeloma. ONC201 was administered once every week. The record describes a Phase 1 group receiving 375 mg ONC201 with 20 mg dexamethasone and a Phase 2 group receiving 625 mg ONC201 with 20 mg dexamethasone. It reports randomized allocation, no masking, and a single-group model. Those design labels and the two dose-group descriptions are not reconciled in the registry record, so the nature of any intended comparison between the dose groups is unclear.
Enrollment was 17. The study began on 2017-04-19, and primary completion was recorded as 2019-09-17. Its status is terminated. The record notes that the study was completed by predecessor company, Oncoceutics, Inc.; Jazz Pharmaceuticals is listed as sponsor. The stated primary objective was to evaluate antitumor efficacy.
The primary response assessment took place at the last on-study visit, defined as the end of treatment or follow-up, with assessments reported up to a maximum of 7 months following treatment initiation. The description says response assessments used magnetic resonance imaging, computed tomography, or positron emission tomography/computed tomography scans when applicable. It does not report the reason for termination, the schedule of visits, treatment exposure for each participant, or the allocation procedure. Those details are not reported in the registry record, and should not be inferred from the phase labels or dose groups.
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 | Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | Phase 2: 625 mg ONC201 + 20 mg Dexamethasone |
|---|---|---|
| STARTED | 2 | 15 |
| COMPLETED | 0 | 3 |
| NOT COMPLETED | 2 | 12 |
Reasons for not completing this period:
| Reason | Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | Phase 2: 625 mg ONC201 + 20 mg Dexamethasone |
|---|---|---|
| Death | 0 | 3 |
| Lack of Efficacy | 0 | 1 |
| Study Terminated | 2 | 8 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | Phase 2: 625 mg ONC201 + 20 mg Dexamethasone | Total |
|---|---|---|---|---|
| Participants analysed | 2 | 15 | 17 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 11 | 12 |
| Age, Categorical: >=65 years | Participants | 1 | 4 | 5 |
| Sex: Female, Male: Female | Participants | 1 | 8 | 9 |
| Sex: Female, Male: Male | Participants | 1 | 7 | 8 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 | 7 | 9 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 0 | 8 | 8 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): White | Participants | 1 | 12 | 13 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 2 | 3 |
| Number of participants who were not a candidate for established therapy | Participants | 2 | 15 | 17 |
6. Primary Endpoint
The registry lists the primary outcome as Response of Participants at Last On-study Visit (End of Treatment/Follow-up), measured over The response assessment data reported was conducted at the last on-study visit (end of treatment/follow-up), up to a maximum of 7 months following treatment initiation..
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants according to response at the last on-study visit, at the end of treatment or follow-up. The timeframe allows for the assessment to occur up to a maximum of 7 months after treatment initiation. The record says response was evaluated using International Myeloma Working Group criteria, with imaging by magnetic resonance imaging, computed tomography, or positron emission tomography/computed tomography when applicable.
The listed response categories are complete response, partial response, progressive disease, and stable disease. Complete response is defined as disappearance of any soft tissue plasmacytomas. Partial response requires a more than 50% reduction in their size. Progressive disease involves definite development of new plasmacytomas or a definite increase in the size of existing ones. Stable disease means the criteria for complete response, partial response, or progressive disease are not met.
General explanation: A response-status endpoint can describe participants’ disease status at an assessment point. It does not, by itself, describe how long a response lasted or what happened after the assessment. The registry record does not give counts for the response categories, so the observed response distribution and any summary of it are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes the primary outcome and the criteria used to classify response, but it does not report the observed primary outcome values. Although one outcome measure is listed, the record gives no counts for complete response, partial response, progressive disease, or stable disease. It also gives no estimate of response, confidence interval, p-value, or analysis comparing the dose groups. Consequently, the posted information does not establish what response statuses were observed or how precisely any response rate was estimated.
This distinction matters because an endpoint definition is not an endpoint result. The response criteria explain how participants could be categorized; they do not show how many participants fell into any category. The assessment time is tied to the last on-study visit, up to a maximum of 7 months following treatment initiation. That is not the same as a common assessment time for every participant, and the record does not provide individual follow-up or assessment timing.
The design labels also call for caution. Allocation is recorded as randomized, while the model is recorded as single-group; meanwhile, two dose groups are described. The registry record does not explain this combination. It is therefore not possible to infer whether the study was designed to make a randomized comparison between doses, or to specify what comparison the primary analysis was intended to estimate. The fact that the study was open-label is relevant to interpretation of response assessments, but the record does not describe any blinded assessment process.
The posted safety figures are counts for the groups as labeled in the adverse-event record. They should not be treated as a substitute for the missing efficacy results. The record does not provide event timing, exposure duration, participant-level detail, or a basis for attributing an event to a particular treatment. Nor does it report a statistical comparison of safety between groups.
General explanation: A confidence interval, when reported, conveys the precision of an estimate across values compatible with the data and the analysis. A significance test addresses a different question and does not on its own describe the size or precision of an effect. Neither can be interpreted for the primary outcome because no response estimate, interval, or test is reported in the registry record. This absence should be read as missing statistical information, not as evidence of no response or of a particular treatment effect.
9. Statistical Methods Explained
The registry record identifies a binary outcome type and describes the primary endpoint as a count of participants. It defines response using four clinical categories, but the record does not report category counts or a statistical analysis of those counts. A binary endpoint can be analyzed in different ways depending on how categories are grouped and what contrast is targeted. The registry record does not specify a reduction of the response categories to a single comparison, and no primary analysis result is reported.
What does a count endpoint tell the reader?
A count records how many participants meet a defined condition or fall into a defined category. For a response endpoint with multiple categories, the category definitions matter: the count for each category answers a different descriptive question. A count alone does not show the uncertainty around a proportion, establish a comparison between groups, or account for differences in follow-up. Those interpretations require a defined analysis and its corresponding results. The response counts and such analyses are not reported in the registry record.
What would a comparison require?
General explanation: A comparative analysis needs a clear contrast, such as a difference in a response probability between groups, and a prespecified way to handle the categories and assessment timing. The design information should identify which participants belong to each comparison group and how allocation was conducted. Here, the record says allocation was randomized and also labels the model single-group, while describing two dose groups. It does not explain the relationship between those details. With no reported response results or contrast, no comparative estimate can be interpreted.
How should uncertainty be understood?
General explanation: A confidence interval expresses statistical uncertainty around an estimate under the specified method and assumptions. Wider intervals generally indicate less precision, while narrower intervals indicate greater precision. A p-value, if reported, addresses compatibility of the data with a stated null hypothesis under a statistical model; it does not measure the clinical importance of an effect. The registry record reports neither a primary endpoint estimate nor a confidence interval or p-value, so the precision and statistical evidence for response cannot be assessed.
What does the planning illustration represent?
The record describes planning illustrations based on a stated fraction of an observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation. For outcomes other than time-to-event outcomes, the illustrated quantity is participants per arm for 80% or 90% power; for time-to-event outcomes, it is the total number of events using the Schoenfeld approach. These are planning calculations, not observed power. The registry record contains no primary response effect from which to interpret a particular illustration, and it does not report a trial-specific sample-size justification or design calculation. The illustrations therefore cannot be used as evidence that the study had a particular probability of detecting an effect.
Why can over-dispersion matter?
General explanation: Over-dispersion occurs when observed variability in count data exceeds what a statistical model assumes. If it is ignored, standard errors may be too small and intervals or tests may convey too much certainty. It is not possible to assess over-dispersion from this record: the primary measure is a participant count with multiple response categories, and no outcome values or statistical model are reported. This is a general consideration, not a claim that over-dispersion was present in this study.
The record does not report the primary statistical method, covariate adjustment, assumptions for a comparison, or handling of repeated or differently timed assessments. Those choices cannot be reconstructed from the endpoint definition alone. In particular, the use of established response criteria does not specify the statistical analysis used to summarize or compare responses.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure, but that measure has several response categories and is assessed at the last on-study visit. The record does not reduce those categories to one reported comparison or provide a primary endpoint analysis. It also reports zero analyses. No multiplicity procedure, hierarchy of hypotheses, or handling of multiple comparisons is reported in the registry record. It is therefore not possible to assess whether multiple statistical tests were planned or how any multiplicity would have been addressed.
The record does not describe missing-data rules, how participants without an end-of-treatment or follow-up assessment were handled, or whether any imputation or sensitivity analysis was used. The population included in the primary efficacy analysis is not reported in the registry record. Enrollment alone does not establish how many participants contributed to an endpoint analysis.
General explanation: Missing outcome data can affect both the denominator and the interpretation of a response summary. The impact depends on why assessments are absent and on the analysis rule used; neither can be determined from the information recorded. Likewise, a named analysis population, such as all randomized participants or participants who received treatment, would clarify which participants contribute to an estimate. No such population is specified in the registry 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 |
|---|---|---|---|
| Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | 0 | 2 | 0.0% |
| Phase 2: 625 mg ONC201 + 20 mg Dexamethasone | 5 | 15 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | 0 of 2 |
| Phase 2: 625 mg ONC201 + 20 mg Dexamethasone | 3 of 15 |
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 |
|---|---|
| Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | 2 of 2 |
| Phase 2: 625 mg ONC201 + 20 mg Dexamethasone | 15 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | Phase 2: 625 mg ONC201 + 20 mg Dexamethasone |
|---|---|---|---|
| Disease progression | General disorders | 0 of 2 | 1 of 15 |
| Abscess limb | Infections and infestations | 0 of 2 | 1 of 15 |
| Arthritis bacterial | Infections and infestations | 0 of 2 | 1 of 15 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 0 of 2 | 1 of 15 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 0 of 2 | 1 of 15 |
| Anaemia | Cardiac disorders | 0 of 2 | 1 of 15 |
| Hypercalcaemia | Metabolism and nutrition disorders | 0 of 2 | 1 of 15 |
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 2 | 1 of 15 |
| Bone pain | Musculoskeletal and connective tissue disorders | 0 of 2 | 1 of 15 |
| Cellulitis | Skin and subcutaneous tissue disorders | 0 of 2 | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase 1: 375 mg ONC201 + 20 mg Dexamethasone | Phase 2: 625 mg ONC201 + 20 mg Dexamethasone |
|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 0 of 2 | 13 of 15 |
| Fatigue | General disorders | 0 of 2 | 11 of 15 |
| Neuropathy peripheral | Nervous system disorders | 1 of 2 | 10 of 15 |
| Leukopenia | Blood and lymphatic system disorders | 0 of 2 | 9 of 15 |
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 2 | 8 of 15 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 2 | 7 of 15 |
| Diarrhoea | Gastrointestinal disorders | 1 of 2 | 5 of 15 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 0 of 2 | 6 of 15 |
| Back pain | Musculoskeletal and connective tissue disorders | 2 of 2 | 4 of 15 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 0 of 2 | 6 of 15 |
Serious events with the highest arm-level rate: disease progression (6.7%), abscess limb (6.7%), arthritis bacterial (6.7%), hypoxia (6.7%), pulmonary embolism (6.7%).
The adverse-event record lists two dose groups. For the Phase 1 group labeled 375 mg ONC201 with 20 mg dexamethasone, the serious-event figures list 2 at risk and 0 affected; the other-event figures list 2 at risk and 2 affected, with 0 deaths. For the Phase 2 group labeled 625 mg ONC201 with 20 mg dexamethasone, the serious-event figures list 15 at risk and 5 affected; the other-event figures list 15 at risk and 15 affected, with 3 deaths. These are recorded counts, not estimates of comparative risk.
The registry lists Disease progression, Abscess limb, Arthritis bacterial, Hypoxia, Pulmonary embolism, and Anaemia among the leading serious-event terms, each at 6.7%. The record does not connect these terms to a specific dose group in the information available for interpretation, and it does not provide event-level details or timing.
General explanation: An at-risk count identifies the recorded group size for an event summary; an affected count is the number with the recorded event. Neither indicates causation. The record does not give exposure duration, event severity beyond the serious-event grouping, or an adjusted comparison. Differences in counts between groups should not be interpreted as a treatment effect or as evidence that treatment caused an event.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase 1/2 open-label study evaluated weekly oral ONC201 with dexamethasone in adults with relapsed/refractory multiple myeloma. It describes two ONC201 dose groups and gives a primary response endpoint based on International Myeloma Working Group criteria at the last on-study visit. It also records adverse-event counts. These details describe the study question and some safety reporting, but they do not supply the primary response results.
The record does not show how many participants had complete response, partial response, progressive disease, or stable disease. It provides no response estimate, confidence interval, or statistical test. It therefore cannot establish the magnitude or precision of an efficacy result, nor whether responses differed between the dose groups. The absence of those results is not evidence that there were no responses or that a treatment effect was absent.
The adverse-event counts describe recorded events within the group labels, but do not establish that a treatment caused an event. The registry does not provide the details needed to compare safety while accounting for exposure or follow-up. Its mixed design labels—randomized allocation and a single-group model—also leave the intended comparison unclear.
General explanation: A clinical trial result is interpreted by combining the outcome estimate, its uncertainty, the analysis population, and the design that generated the data. An endpoint definition and a set of event counts are only parts of that picture. In this record, the missing efficacy analysis and unresolved design description limit what can be concluded. The information should be read as a registry description with reported safety counts, not as a complete statistical account of efficacy.
13. Why This Trial Matters Statistically
Statistically, this record illustrates why an endpoint definition and a result must be distinguished. The response endpoint is described in detail, including the criteria for complete response, partial response, progressive disease, and stable disease. Yet no response-category counts or analysis are reported. Without the observed values, readers cannot assess the response distribution, estimate uncertainty, or evaluate a comparison.
The record also highlights the importance of coherent design reporting. Randomized allocation, a single-group model, and two dose-group descriptions appear together, without an explanation of how they relate. The intended estimand—the precise quantity the analysis was designed to estimate—is therefore not clear from the registry record. This affects how an eventual comparison, if reported elsewhere in the record, would need to be interpreted.
Safety counts likewise need context. Counts by group are descriptive, and do not establish treatment attribution or comparative risk. Event timing, exposure, analysis populations, and methods would be needed for a fuller statistical interpretation. The registry does not report those details.
General explanation: Transparent reporting connects a clinical question to a defined endpoint, a prespecified analysis, and results with uncertainty. When any link is missing, readers should avoid filling the gap with assumptions. Here, the registry supports a description of the study design as recorded and of the posted safety counts, but not a statistical conclusion about the primary efficacy question.
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 principal trial-specific limitation is that the registry record gives no observed results for the primary response endpoint. It lists one outcome measure and its assessment criteria, but reports no response-category counts, effect estimate, interval, or test. The record also reports zero analyses. As a result, efficacy cannot be statistically characterized from the posted results.
Interpretation of the design is limited by the combination of randomized allocation and a single-group model, alongside two dose-group descriptions. The registry record does not explain how allocation worked or specify a planned comparison. It does not report a statistical analysis plan, sample-size justification, interim analyses, covariate adjustment, missing-data approach, or analysis population.
The trial is marked terminated, but the reason for termination is not reported in the registry record. The response assessment timeframe extends to a maximum of 7 months following treatment initiation, but individual follow-up and assessment times are not described. The open-label design is recorded, while blinding of response assessment is not reported in the registry record.
Safety reporting is also limited: event timing, exposure duration, participant-level detail, and a comparative analysis are not given. These omissions prevent the counts from supporting causal or adjusted comparisons. The limitations concern what can be interpreted from the registry record; they do not establish that the unreported methods or results were absent from the conduct of the study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Oral ONC201 in Relapsed/Refractory Multiple Myeloma — NCT02863991.
- Registry record last updated 2024-07-03; status last verified 2024-06.
- Results first posted 2024-07-03.
- 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
NCT02863991 is a Phase 1/2 open-label study of oral ONC201 once every week with dexamethasone in adults with relapsed/refractory multiple myeloma. The record describes 375 mg ONC201 with 20 mg dexamethasone in Phase 1 and 625 mg ONC201 with 20 mg dexamethasone in Phase 2. Enrollment was 17; the study began on 2017-04-19, reached primary completion on 2019-09-17, and is listed as terminated.
The primary measure is participant response status at the last on-study visit, assessed using International Myeloma Working Group criteria, up to a maximum of 7 months following treatment initiation. Response results and statistical analyses are not reported in the registry record. Safety counts and serious-event terms are listed, but do not establish treatment causation or a comparative safety effect. The registry’s randomized-allocation and single-group labels are not reconciled.