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 Mocetinostat (MGCD0103) Plus Brentuximab Vedotin (SGN-35) in Patients With Relapsed or Refractory Hodgkin Lymphoma (NCT02429375), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated Dose (MTD).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02429375 |
| Phase | Phase 1, Phase 2 |
| Condition | Hodgkin Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with relapsed or refractory Hodgkin lymphoma were the intended population. The registry record reports an enrollment of 7. It does not give further eligibility details or describe the characteristics of the enrolled participants.
Intervention
The study evaluated mocetinostat combined with brentuximab vedotin. The record describes a phase I dose-escalation portion and a phase II portion. The phase I portion was intended to evaluate sequential mocetinostat dose levels in combination with brentuximab vedotin.
Comparator
There was no separate control group: the registered model is single-group, and allocation is listed as not applicable. The record therefore does not describe a randomized comparison with another treatment or with usual care. The dose levels are not a like-for-like control comparison.
Primary question
The primary objective was to identify the maximum tolerated dose of mocetinostat in the combination, using a dose-escalation scheme based on dose-limiting toxicities. The registry record gives a timeframe of 1 year for this outcome, but states that data were not collected. Consequently, the posted record does not provide an observed maximum tolerated dose or a quantitative estimate of the primary outcome.
3. Trial Design
This was a phase IB/II, single-group study of mocetinostat plus brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma. The registry lists the study as completed and reports an enrollment of 7. It lists no masking and no applicable allocation, consistent with a design that did not assign participants between randomized treatment groups. There is no concurrent control group in the registry record.
The stated phase I design used sequential dose cohorts and a standard 3+3 dose-escalation scheme. Participants were to enter cohorts of 3, starting at dose level 1. The description says that the dose would be escalated if none of the 3 participants in a cohort experienced a dose-limiting toxicity. The registry record does not report the full decision rules for other toxicity patterns, the definition of a dose-limiting toxicity, or further details of how phase I findings would guide the phase II portion.
The listed dose levels were 50mg, 70mg, and 90mg of mocetinostat. The registry reports the combination as the intervention; it does not provide a separate intervention arm for either drug alone. The study summary describes the combination as the subject of the trial and notes that this was the first study giving the two drugs together.
The primary outcome was maximum tolerated dose, with a listed timeframe of 1 year. Overall response rate was listed as a secondary outcome. Although the record includes adverse-event summaries by dose level, it states that primary outcome data were not collected and reports no analyses. The registry record does not describe the final analysis plan or give results for the secondary outcome.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Dose Level 1: 50mg Mocetinostat | Dose Level 2: 70mg Mocetinostat | Dose Level 3: 90mg Mocetinostat |
|---|---|---|---|
| STARTED | 3 | 3 | 1 |
| COMPLETED | 3 | 3 | 1 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Data were not collected
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.
Population: Patients with relapsed or refractory Hodgkin lymphoma
| Characteristic | Unit | Dose Level 1: 50mg Mocetinostat | Dose Level 2: 70mg Mocetinostat | Dose Level 3: 90mg Mocetinostat | Total |
|---|---|---|---|---|---|
| Participants analysed | 3 | 3 | 1 | 7 | |
| Age, Continuous | years | 43 (24 to 62) | 30 (25 to 33) | 43 (43 to 43) | 33 (24 to 62) |
| Sex: Female, Male: Female | Participants | 1 | 3 | 0 | 4 |
| Sex: Female, Male: Male | Participants | 2 | 0 | 1 | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 1 | 0 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 2 | 1 | 6 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 3 | 3 | 1 | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | Participants | 3 | 3 | 1 | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (MTD), measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was maximum tolerated dose (MTD). In this study, MTD was the dose-finding objective for mocetinostat when given with brentuximab vedotin. The registry describes a 3+3 dose-escalation scheme: cohorts of 3 were to start at dose level 1, and escalation was planned if none of the 3 participants at a dose experienced a dose-limiting toxicity (DLT). The endpoint therefore concerns tolerability under the study’s dose-escalation rules, rather than a comparison of clinical outcomes between treatment groups.
In general, dose-finding studies use toxicity observations to guide which dose levels are evaluated and whether escalation should continue. This can help identify a dose for further study, but an MTD estimate is specific to the treatment combination, study population, toxicity definitions, and decision rules used. It is not by itself an estimate of treatment benefit.
The registry lists a timeframe of 1 year for the primary outcome, but says that data were not collected. It does not report an observed MTD, DLT definition, or the number of participants with DLTs. As a result, the endpoint’s planned role can be described, but its value cannot be estimated from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive constraint is that the registry record does not report a primary endpoint result. MTD was the primary outcome, but the record states that data were not collected. No observed MTD, dose-limiting toxicity results, confidence interval, or statistical test for that endpoint is reported. The registry also lists no analyses. This means that the study’s primary objective cannot be assessed quantitatively from the posted record.
The enrollment was 7, and the design was single-group. Those details describe the size and structure of the study, not the magnitude or precision of any treatment effect. Without a control group, a difference between the combination and another treatment cannot be estimated from this design. Dose-level observations can describe what was recorded at each level, but the dose levels are not randomized comparison groups and should not be interpreted as if they were equivalent arms in a controlled trial.
The record lists overall response rate as a secondary outcome, but gives no posted response result or analysis. It therefore does not support a quantitative conclusion about response in the enrolled participants. Likewise, the brief study summary describes the purpose as assessing safety and effectiveness, but a stated purpose is not a result.
Adverse-event summaries are posted by dose level. They report serious-event affected counts of 1 among 3 at dose level 1, 0 among 3 at dose level 2, and 1 among 1 at dose level 3. The corresponding other-event counts are 3 among 3, 3 among 3, and 1 among 1. Deaths are listed as 0, 0, and 1, respectively. These are descriptive registry entries from small groups, not adjusted comparisons. The record does not establish whether events were caused by treatment, nor does it provide enough detail to interpret the death’s cause or timing.
In general, estimates from small samples are often imprecise, and a single observation can strongly affect a percentage or summary. Here, no primary endpoint estimate or inferential analysis is posted, so statistical precision for the main objective cannot be evaluated. Any planning illustration based on an observed effect should be treated as a planning calculation, not as observed power; the registry does not report a primary effect estimate to connect to such a calculation.
9. Statistical Methods Explained
The registry record describes a dose-finding design rather than a randomized efficacy comparison. Its stated primary endpoint was MTD, and its planned approach was a standard 3+3 dose-escalation scheme. The interpretation of that plan should be kept separate from the results: the record states that primary outcome data were not collected and reports no analyses.
What does a 3+3 dose-escalation scheme do?
In general, a 3+3 scheme evaluates doses in sequential cohorts. The registry description says that cohorts of 3 would begin at dose level 1 and that escalation would occur if none of the 3 participants at that dose exhibited a DLT. This makes toxicity observations part of a prespecified decision process: the cohort’s observed DLT pattern informs whether the next dose is considered. The registry description does not give the full set of decision rules for patterns other than zero DLTs, so those rules cannot be reconstructed from the record.
In general, a dose-escalation design is intended to guide dose selection, not to estimate a treatment effect relative to a control group. Its output depends on the toxicity definition, observation period, dose sequence, and escalation rules. The registry names a 1-year timeframe for the primary outcome, but does not specify the DLT definition or provide the observations needed to identify an MTD.
What can be concluded from a single-group design?
The registered model is single-group, with no applicable allocation and no masking. In general, a single-group study can describe outcomes observed among participants receiving the study intervention. It cannot, on its own, distinguish an intervention effect from other explanations for the observed outcomes. A randomized control group would support a different kind of comparison, but none is described in this record.
The study includes several mocetinostat dose levels, but sequential dose cohorts are not interchangeable with randomized arms. Participants were not described as being assigned concurrently to dose levels under random allocation. Dose-level summaries can be read descriptively, but comparisons between them may reflect the dose-escalation process and the small numbers at each level. They do not provide a controlled estimate of dose-related benefit or harm.
How should counts and percentages be understood?
Counts describe the number of participants recorded as affected within the listed group, while the at-risk count gives the group size for that category. In general, a percentage calculated from a small group can change substantially when the status of a single participant differs. Percentages should therefore be read alongside the number at risk, and not as precise estimates of event probability. The adverse-event record also includes listed serious-event terms with percentages, but does not explain their denominator or provide additional event-level detail.
Confidence intervals, in general, express the statistical uncertainty around an estimate under a specified analysis. No confidence interval is reported for the primary outcome or response rate. Consequently, precision cannot be evaluated for those outcomes. The record’s adverse-event counts are descriptive and do not include comparative uncertainty estimates.
What does the analysis record establish?
The registry lists overall response rate as a secondary outcome and reports 2 outcome measures, but does not post a response estimate or an analysis. It records 0 analyses. This does not establish which analysis methods were planned or whether analyses occurred outside the registry; those details are not reported in the registry record. No adjustment strategy, hypothesis-testing framework, or inferential model is described.
In general, multiplicity arises when several outcomes, doses, time points, or statistical tests are considered. If many tests are performed, the chance of at least one apparently notable result can increase unless the analysis plan addresses that issue. The record does not report a multiplicity strategy. It also does not describe missing-data methods, analysis populations, or a sample-size justification. Any sample-size illustration based on an observed effect is a planning illustration, not observed power, and should not be treated as evidence that the trial had a particular probability of detecting an effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists MTD as the primary outcome and overall response rate as a secondary outcome. It also reports 2 outcome measures, but does not provide posted results for the secondary outcome or analyses for either endpoint. The record lists 0 analyses. The registry does not report whether additional outcomes, dose-specific comparisons, or time points were considered in the analysis.
In general, multiplicity is a concern when several hypotheses are tested or when results can be selected from multiple outcomes or analyses. Without a stated analysis plan, readers cannot determine whether any multiplicity adjustment was intended or how the primary and secondary outcomes were prioritized in analysis. The multiplicity strategy is not reported in the registry record.
The primary outcome population is described as data not collected. The record does not give an analysis population for the secondary outcome, define which participants would be included in a response analysis, or state how missing observations were handled. It also does not describe withdrawals, exclusions from analysis, or imputation methods. These details are not reported in the registry record, so the handling of missing data and the relationship between enrollment and outcome denominators cannot be assessed.
In general, missing-data methods matter because conclusions can depend on why measurements are unavailable and how the analysis treats them. A sample-size justification and assumptions about anticipated outcomes can also clarify what level of precision a study aimed to achieve. The registry record does not report a sample-size justification, an interim-analysis plan, or a statistical analysis plan. Any planning illustration should be distinguished from observed power, and it cannot replace missing endpoint results.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Dose Level 1: 50mg Mocetinostat | 1 | 3 | 33.3% |
| Dose Level 2: 70mg Mocetinostat | 0 | 3 | 0.0% |
| Dose Level 3: 90mg Mocetinostat | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dose Level 1: 50mg Mocetinostat | 0 of 3 |
| Dose Level 2: 70mg Mocetinostat | 0 of 3 |
| Dose Level 3: 90mg Mocetinostat | 1 of 1 |
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 |
|---|---|
| Dose Level 1: 50mg Mocetinostat | 3 of 3 |
| Dose Level 2: 70mg Mocetinostat | 3 of 3 |
| Dose Level 3: 90mg Mocetinostat | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Dose Level 1: 50mg Mocetinostat | Dose Level 2: 70mg Mocetinostat | Dose Level 3: 90mg Mocetinostat |
|---|---|---|---|---|
| Anorexia | Metabolism and nutrition disorders | 0 of 3 | 0 of 3 | 1 of 1 |
| Dehydration | Metabolism and nutrition disorders | 0 of 3 | 0 of 3 | 1 of 1 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 0 of 3 | 1 of 1 |
| Hypokalemia | Metabolism and nutrition disorders | 0 of 3 | 0 of 3 | 1 of 1 |
| Infections and infestations - Other, specify | Infections and infestations | 1 of 3 | 0 of 3 | 0 of 1 |
| Malaise | General disorders | 0 of 3 | 0 of 3 | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dose Level 1: 50mg Mocetinostat | Dose Level 2: 70mg Mocetinostat | Dose Level 3: 90mg Mocetinostat |
|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | 2 of 3 | 2 of 3 | 1 of 1 |
| Fatigue | General disorders | 2 of 3 | 2 of 3 | 0 of 1 |
| Neutrophil count decreased | Investigations | 0 of 3 | 3 of 3 | 1 of 1 |
| White blood cell decreased | Investigations | 0 of 3 | 2 of 3 | 0 of 1 |
| Hypocalcemia | Metabolism and nutrition disorders | 0 of 3 | 1 of 3 | 1 of 1 |
| Lymphocyte count decreased | Investigations | 1 of 3 | 1 of 3 | 0 of 1 |
| Hyperglycemia | Metabolism and nutrition disorders | 0 of 3 | 0 of 3 | 1 of 1 |
| Platelet count decreased | Investigations | 0 of 3 | 1 of 3 | 0 of 1 |
| Alanine aminotransferase increased | Investigations | 0 of 3 | 1 of 3 | 0 of 1 |
| Aspartate aminotransferase increased | Investigations | 0 of 3 | 1 of 3 | 0 of 1 |
Serious events with the highest arm-level rate: anorexia (100.0%), dehydration (100.0%), dyspnea (100.0%), hypokalemia (100.0%), malaise (100.0%).
The adverse-event entries are presented by mocetinostat dose level. At dose level 1, 3 participants were at risk for the serious-event category and 1 was listed as affected; 3 were at risk for other events and 3 were affected; deaths were listed as 0. At dose level 2, the corresponding counts were 3 at risk and 0 affected for serious events, 3 at risk and 3 affected for other events, and 0 deaths. At dose level 3, 1 participant was at risk and 1 affected in each event category, with 1 death listed.
The registry also lists serious-event terms with percentages: Anorexia, Dehydration, Dyspnea, Hypokalemia, and Malaise are each listed as 100.0%; Infections and infestations - Other, specify is listed as 33.3%. The record does not explain the denominator for these term percentages or give further event-level context.
In general, these entries are descriptive counts, not evidence that an event was caused by the combination. The record does not provide causality assessments, event timing, severity grading details, or an explanation of the death. The small at-risk counts also limit how precisely these entries characterize safety beyond the participants represented in the registry.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase IB/II study of mocetinostat plus brentuximab vedotin was conducted as a single-group study in patients with relapsed or refractory Hodgkin lymphoma, with an enrollment of 7. It describes a phase I dose-escalation plan and lists MTD as the primary outcome. It also records adverse-event counts by dose level and names overall response rate as a secondary outcome.
It does not establish an observed MTD. The primary outcome is explicitly described as data not collected, and no dose-limiting toxicity results or analysis are posted. The record also does not report an overall response rate result. The study’s stated purpose of evaluating safety and effectiveness should therefore not be mistaken for evidence about either outcome.
Because there was no control group, the posted information does not provide a randomized comparison of the combination with another treatment. The dose-level entries are not a substitute for a control group: they arise within a dose-escalation design and involve small, sequential cohorts. Descriptive counts can show what the registry recorded in those cohorts, but they cannot establish a comparative treatment effect.
In general, a result and its precision are distinct. A point estimate describes the observed value, while measures such as confidence intervals describe uncertainty around that estimate. For the main clinical outcomes, neither an estimate nor its precision is available in the registry record. The adverse-event summaries offer counts but not comparative estimates or uncertainty measures.
Thus, the record is informative about the intended design, primary objective, enrollment, and posted safety counts. It does not support a quantitative conclusion about the primary dose-finding objective or response, and it does not determine causation for the listed events. Those limits reflect what is and is not reported, rather than a conclusion about outcomes outside the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable as a small, single-group dose-finding study whose registered primary endpoint is MTD. That combination of design and endpoint changes the questions that can be answered. The study was structured to use toxicity observations to guide dose escalation, not to estimate a treatment effect against a concurrent control group. Interpreting it as a comparative efficacy trial would therefore misread its design.
The record also illustrates why a planned endpoint and a reported result must be distinguished. It describes the MTD objective and the 3+3 scheme, yet states that primary outcome data were not collected. A design description explains how the endpoint was intended to be assessed; it does not supply an endpoint estimate. Without that estimate, statistical precision and the outcome of the dose-finding objective cannot be evaluated.
The enrollment of 7 and the dose-level safety counts make denominators especially important. In general, counts and percentages from small groups can be sensitive to individual observations. Reporting the at-risk counts alongside affected counts helps readers understand the scale of the observed groups, but it does not resolve questions about causality, representativeness, or uncertainty.
The registry lists response rate as a secondary outcome but posts no result or analysis. This further demonstrates that a registered outcome is not equivalent to a reported finding. The absence of a control group and the absence of posted outcome analyses also limit the scope for drawing inferential conclusions from the registry record.
For statistical reading, the key distinction is between descriptive information about trial conduct and evidence estimating an outcome. Here, the design and safety summaries are documented, while the primary endpoint result is unavailable. Maintaining that distinction prevents dose-level counts or the study’s stated aims from being treated as proof of an efficacy or safety conclusion.
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 limitation for interpretation is that the registry says primary outcome data were not collected. MTD was the primary endpoint, but no value, DLT count, or analysis is posted. The record lists overall response rate as a secondary outcome without giving its result. As a result, the registered clinical outcomes cannot be quantified from the posted information.
The study was single-group, with no applicable allocation and no masking. There is no randomized comparator, so the record cannot provide a controlled estimate of the combination’s effect relative to another treatment. The dose levels should not be treated as comparable control arms, particularly because the described approach was sequential dose escalation.
Enrollment was 7, and the safety summaries include very small at-risk groups at the listed dose levels. Such counts describe the participants represented, but give limited information about how well those observations characterize a broader population. The registry does not report participant characteristics, eligibility details, event timing, DLT definitions, or the cause of the death listed at dose level 3.
Important analysis details are also absent. The registry record does not report a statistical analysis plan, sample-size justification, analysis populations, missing-data methods, interim analyses, or a multiplicity strategy. It lists 0 analyses. These omissions make it impossible to assess how outcomes were intended to be analyzed or whether the posted adverse-event summaries reflect all relevant observations.
The registry lists a 1-year timeframe for MTD, but does not provide further endpoint assessment details. The status is completed, yet completion status does not supply missing outcome results. These limitations are specific to what the registry record documents and should constrain interpretation of the study’s design and posted safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Mocetinostat (MGCD0103) Plus Brentuximab Vedotin (SGN-35) in Patients With Relapsed or Refractory Hodgkin Lymphoma — NCT02429375.
- Registry record last updated 2022-05-12; status last verified 2022-03.
- Results first posted 2022-05-12.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT02429375 is a completed phase IB/II, single-group study of mocetinostat plus brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma. The registry reports an enrollment of 7 and no masking. Its phase I description specifies sequential dose cohorts and a 3+3 approach. The primary outcome was MTD, with a listed timeframe of 1 year, but the registry states that primary outcome data were not collected. Overall response rate was listed as a secondary outcome, with no result or analysis posted.
Adverse-event counts are reported by dose level, including serious events, other events, and deaths. These entries are descriptive and do not establish event causation or a treatment comparison. The registry does not report a sample-size justification, analysis plan, missing-data method, or multiplicity strategy. The record therefore documents the study design and selected safety counts, but does not provide quantitative results for its primary or secondary clinical outcomes.