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 Dacarbazine and Carmustine in Metastatic Melanoma (NCT01692691), a na phase 2 study. The primary outcome is Progression Free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01692691 |
| Phase | Phase 2 |
| Condition | Melanoma Metastatic |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Western Regional Medical Center |
| Results posted | Yes |
2. Clinical Question
The study was intended to examine progression-free survival in people with Stage IV melanoma whose disease had progressed after at least one prior systemic therapy. The registry record identifies an 8 weeks timeframe for this primary outcome. Its brief summary describes the purpose as determining whether the dacarbazine and carmustine doses and schedule used in the study would help increase tumor shrinkage. The listed secondary outcomes are response rate and median duration of response.
Population
People with Stage IV metastatic melanoma and disease progression on at least one prior systemic therapy. The record reports enrollment of 1; the primary outcome population is described as having no data collected.
Intervention
A single-group regimen listed as dacarbazine IV on Day 1, carmustine IV on Day 2, and Neulasta SC on Day 3.
Comparator
No separate comparison group is reported. The study is identified as single group, with allocation recorded as NA.
Primary question
What was progression-free survival over the stated 8 weeks timeframe in the intended population? No primary outcome data were collected, so the record does not provide an answer.
This is a question about outcomes observed under a regimen, not a randomized comparison of that regimen against another treatment or control. Without a comparator, any outcome data would describe the treated group but would not by themselves isolate the regimen's effect from other explanations. In this record, the absence of collected primary outcome data means even a descriptive estimate of progression-free survival is unavailable. The intended question and the evidence retained in the registry therefore need to be kept distinct.
3. Trial Design
The registry identifies this as a phase II, single-group study with no masking. Allocation is recorded as NA. There is no separate control arm in the record, so the design is not a like-for-like comparison between treatment groups. Enrollment is reported as 1. The study status is terminated.
The listed regimen sequences three drugs: dacarbazine by IV on Day 1, carmustine by IV on Day 2, and Neulasta by SC on Day 3. The record does not report further details about dosing, treatment cycles, duration of therapy, or rules for changing or stopping treatment. Those protocol details should not be inferred from the sequence alone.
The study start is recorded as 2012-08 and primary completion as 2014-10. The registry states that the study was terminated because the principal investigator was no longer employed at CTCA and did not have rights to the trial information. It also states that, after much effort, results could not be retained. These statements explain why the record lacks outcomes; they do not establish what participants experienced during the study.
There are three outcomes listed in the record: progression-free survival as the primary outcome, and response rate and median duration of response as secondary outcomes. No analyses are reported. The record does not report a statistical analysis plan, a sample-size justification, interim analyses, or a protocol-defined comparator. It is therefore not possible to reconstruct how the investigators planned to analyze the outcomes or whether the planned procedures changed during the study.
For a single-group trial, the observed outcomes, if collected, would characterize the participants enrolled and treated under the study regimen. They would not provide the same causal comparison as outcomes from separately assigned treatment and control groups. Here, the enrollment of 1 and lack of collected outcome data also prevent a meaningful summary of clinical outcomes within the study itself.
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 | Dacarbazine Carmustine |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Data not collected. The study has been terminated due to the PI no longer being employed at CTCA and not having rights to the trial information."After much effort, results were not able to be retained.
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 | Received Treatment |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival, measured over 8 weeks.
| 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 is titled progression-free survival, with an 8 weeks timeframe. The description identifies the intended population as patients with Stage IV melanoma whose disease progressed on at least one prior systemic therapy. In general, progression-free survival is a time-to-event measure: it describes the time until disease progression or death, according to the study's operational definitions. That general description should not be mistaken for the trial's exact endpoint rules. The registry record does not report how progression or death was defined, how disease assessments were scheduled, or how the endpoint was calculated.
In general, progression-free survival is used to capture how long participants remain alive without documented disease progression. It can be informative when tumor response alone does not describe the timing of disease worsening. Its interpretation depends on clear event definitions, assessment schedules, follow-up, and handling of participants whose event status is unknown. Those details are not reported in the registry record.
Most importantly, the primary outcome population field states that data were not collected and that results could not be retained. No progression-free survival estimate, event count, uncertainty interval, or analysis is reported. The endpoint is therefore identifiable as the planned primary measure, but its value in this study cannot be determined from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that this record contains no primary outcome data. The primary outcome population field explicitly says data were not collected and explains that results could not be retained. The registry also reports 0 analyses. Consequently, there is no progression-free survival estimate to interpret, no reported response rate, and no reported median duration of response. The study's stated clinical question remains unanswered by the posted results.
Enrollment was 1, which is the reported number of participants enrolled, not an outcome estimate. It does not establish whether the person completed treatment, had an endpoint assessment, or contributed to a particular analysis. The registry's adverse-event group is titled Received Treatment, but additional treatment exposure details are not reported in the registry record. Enrollment and analysis populations should not be treated as interchangeable.
The single-group design would limit causal interpretation even if outcomes had been retained. A treated-group outcome can describe what was observed among participants in that group, but without a concurrent comparator it cannot show what would have happened under another treatment or no study treatment. Differences from other studies or historical outcomes would not remove that limitation unless the comparison methods and assumptions were carefully specified; no such analysis is reported.
The registry's absence of an analysis is not evidence that the endpoint was favorable, unfavorable, or unchanged. It means an outcome result is unavailable. Likewise, the lack of retained results does not establish that participants had no progression, no response, or no adverse events beyond the categories and counts recorded. It is important to distinguish a zero event count in the adverse-event entry from missing efficacy results: the former is a posted count for a recorded category, whereas the latter reflects the stated lack of collected or retained outcome data.
No confidence interval or p-value is available for the primary endpoint. In general, a confidence interval conveys the range of values compatible with an estimate under the analysis assumptions, and a p-value assesses compatibility with a specified null model. Neither can be interpreted when an estimate and analysis are absent. A precise or uncertain treatment effect cannot be inferred from the enrollment count alone.
The record lists response rate and median duration of response as secondary outcomes, but gives no values for either. It also does not report the outcome definitions, assessment procedures, or missing-data approach. Thus, the limitation is not merely that the number of participants is small; the key issue is that outcome information and analyses are unavailable. Conclusions about efficacy, comparative effectiveness, or the relationship between treatment and outcomes cannot be drawn from this record.
9. Statistical Methods Explained
The registry record identifies a phase II, single-group study, but it does not report the statistical methods used to analyze outcomes. It reports 0 analyses and states that the primary outcome data were not collected and results could not be retained. The concepts below explain how methods would generally be interpreted; they are not a description of an analysis performed in this trial.
What does a single-group design estimate?
In general, a single-group study can describe outcomes among participants who receive the regimen. If outcomes are collected consistently, a report might give the number of participants with an event, a response proportion, or a time-to-event summary. Such estimates characterize the observed group. They do not, by themselves, estimate the difference between the regimen and a comparator, because there is no concurrent comparison group to show what outcomes might have occurred under an alternative.
The registry identifies one group and no masking, and allocation is recorded as NA. These design features matter to interpretation. In a comparative randomized study, assignment helps create groups that can be contrasted under the study design. That contrast is not available. Lack of masking can also matter in general when outcome assessment or decisions may be influenced by knowledge of treatment, although the record does not report how outcomes were assessed or whether that issue affected this trial.
How is progression-free survival generally analyzed?
In general, progression-free survival is analyzed as a time-to-event outcome. The analysis requires a defined starting point, an event definition, and a rule for participants without a recorded event by the end of their available follow-up. Participants whose event has not been observed are commonly treated as censored at an appropriate last assessment under a specified method. Censoring is not the same as confirming that no event ever occurred; it indicates that event status is not observed beyond a point in follow-up.
For this trial, the primary outcome is labeled progression-free survival and its timeframe is 8 weeks. The registry does not report the time origin, event criteria, assessment schedule, censoring rules, or a time-to-event estimator. It also reports that data were not collected. There is therefore no basis for describing a survival curve, a median, an event rate, or the handling of censored observations as an actual trial method.
What do uncertainty measures ordinarily add?
In general, an estimate summarizes the data, while an uncertainty interval communicates how much precision the data and model support. A wide interval indicates less precision than a narrow interval, subject to the method's assumptions. A confidence interval does not guarantee that the true value lies within its limits, nor does it correct for bias, an unsuitable endpoint definition, or a design without a comparator. The registry record reports no endpoint estimate or interval, so precision cannot be assessed for the primary outcome.
In general, a hypothesis test compares observed data with a null hypothesis under specified assumptions. A p-value is not the probability that a treatment works, nor is it a measure of clinical importance. Its interpretation requires knowing the tested contrast, model, analysis population, and prespecified decision rules. No such test or p-value is reported for this study. The absence of a p-value should not be read as a nonsignificant result; there is no reported analysis from which to make that judgment.
What is over-dispersion, and is it relevant?
In general, over-dispersion occurs when count or proportion data vary more than a statistical model assumes. If ignored in a setting where it is present, uncertainty estimates can be too small and tests can be misleading. Whether it matters depends on the outcome, data structure, and chosen model. The registry does not report count outcome data or a statistical model, so it is not possible to assess over-dispersion in this trial or say that a method addressing it was used.
How should planning illustrations be read?
In general, sample-size calculations are planning tools that estimate how many participants or events may be needed under stated assumptions. The record describes planning illustrations based on a fraction of an observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event outcomes, the stated illustrations use a total number of events; other illustrations are expressed as participants per arm. These are assumptions for planning, not observed trial results.
The record itself describes a single group, enrollment of 1, and no retained primary outcome data or analysis. The illustrations should not be called observed power or mistaken for the size of a completed two-arm comparison. In general, power calculations describe design performance under assumed effects and methods; they do not establish the probability that a particular observed result is true. Because no primary outcome estimate is reported, these illustrations cannot supply the missing treatment effect or answer the clinical question.
What can be reconstructed from the record?
The design labels, intended outcome, timeframe, enrollment, status, and sequence of the listed regimen are reported. The analysis model, endpoint definitions, analysis population, missing-data procedures, multiplicity approach, interim monitoring, and sample-size justification are not reported in the registry record. The statement that data were not collected and results could not be retained places a firm limit on reconstruction: no statistical result should be inferred from the intended methods or planning assumptions.
10. Multiplicity, Missing Data and Analysis Populations
The record lists one primary outcome, progression-free survival, and two secondary outcomes, response rate and median duration of response. Considering several outcomes can raise multiplicity questions in general. If multiple hypotheses are tested, the chance of at least one apparently noteworthy result can increase unless the analysis plan accounts for the number and relationship of tests. Approaches to multiplicity depend on the study objectives and prespecified analysis plan.
No multiplicity procedure, hierarchy for interpreting outcomes, or statistical analysis plan is reported in the registry record. The record also reports 0 analyses and no collected primary outcome data. It is therefore not possible to determine whether multiplicity adjustments were planned, whether any outcome was analyzed, or how results would have been prioritized.
The primary outcome population description says data were not collected, and the record explains that results could not be retained. This is not enough information to characterize the missingness mechanism. In general, missing data may arise for different reasons, and conclusions can depend on whether missingness is related to observed or unobserved outcomes. No missing-data methods or sensitivity analyses are reported, and the available information does not support assumptions about why outcome data were unavailable beyond the registry's stated explanation about the loss of trial information.
An analysis population is also not reported. The enrollment count of 1 does not show whether that participant was treated, followed for the primary outcome, included in any analysis, or eligible for an endpoint assessment. The registry does not identify an intention-to-treat, per-protocol, or safety population. Those labels should not be assigned by inference. With outcome collection and analysis absent, neither the handling of missing observations nor the population underlying an efficacy estimate can be evaluated.
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 |
|---|---|---|---|
| Received Treatment | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Received Treatment | 0 of 1 |
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 |
|---|---|
| Received Treatment | 0 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Received Treatment |
|---|---|---|
| Nausea | Gastrointestinal disorders | 0 of 1 |
| Arthalgia | General disorders | 0 of 1 |
| gastroesophageal reflux | Gastrointestinal disorders | 0 of 1 |
| Leukopenia | Investigations | 0 of 1 |
The adverse-event entry lists a group titled Received Treatment with 1 participant at risk. It reports 0 serious adverse events, 0 other events in the posted category, and 0 deaths. These are the counts in the registry record; they do not provide a detailed account of event definitions, collection procedures, or observation time.
In general, a zero count means that no events in that category were recorded for the stated group and reporting context. It does not establish that an event is impossible, nor does it show that the regimen is free of risk. With only 1 participant at risk, the record provides very limited information about how often adverse events might occur in a broader population. The counts also cannot be compared with another treatment because no comparator group is reported.
The record does not report a list of serious event terms, and it does not provide additional context such as the duration of safety follow-up or the criteria used to classify events. The adverse-event figures should therefore be read narrowly as recorded counts, not as a comprehensive safety profile. They do not fill the gap in the uncollected primary outcome data or establish whether the planned treatment was completed.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II, single-group study of sequenced dacarbazine, carmustine, and Neulasta in previously treated metastatic melanoma. It reports enrollment of 1, identifies progression-free survival over 8 weeks as the primary outcome, and lists response rate and median duration of response as secondary outcomes. It also reports that the trial was terminated and that primary outcome data were not collected and results could not be retained.
Those details describe the study's intended question and the limits of the available record. They do not show whether participants experienced progression, responded to treatment, or had a particular duration of response. No efficacy estimate or analysis is reported. A statement that the study sought to increase tumor shrinkage describes its purpose, not an observed result.
Even if outcome data had been available, the single-group design would describe outcomes in the treated group rather than provide a randomized contrast with another regimen or control. The record's enrollment of 1 further limits what could be learned about outcomes in a broader population. But the primary obstacle to interpreting efficacy is more direct: the registry says the relevant data were not collected and the results could not be retained.
The adverse-event entry records 0 serious adverse events, 0 other events, and 0 deaths among 1 participant at risk. These counts should not be expanded into a general conclusion about safety. They are a limited record of event counts for this group, without reported event definitions or follow-up details.
Accordingly, the trial record supports a description of the design, regimen sequence, enrollment, status, planned outcomes, and stated reason results are unavailable. It does not support a conclusion that the regimen improved outcomes, failed to improve outcomes, or had a particular safety profile. An unavailable result is not equivalent to a negative result, and planning assumptions cannot substitute for clinical observations.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a planned question and an answerable analysis. The registry identifies a primary endpoint, a timeframe, and additional outcomes, but states that primary outcome data were not collected and that results could not be retained. Without observations, methods such as confidence intervals, hypothesis tests, or time-to-event estimation cannot produce an interpretable clinical result.
It also illustrates why study design and evidence claims need to be considered together. The study is single group, with no separate comparator. A single-group study can describe outcomes among enrolled participants if those outcomes are measured, but it cannot by itself establish how those outcomes compare with what would have happened under another treatment. Statistical analysis cannot create a control group that the design did not include.
The enrollment count of 1 makes the distinction between enrollment and information especially visible. Enrollment is a design and conduct count; it is not itself a measure of treatment effect. Even a fully retained outcome for a participant would describe that observation, not reliably characterize outcome patterns across a wider population. Here, no primary outcome data are reported at all.
Finally, planning calculations and observed evidence serve different purposes. Planning illustrations depend on assumptions about effects, allocation, and analysis. They are not results, and they are not observed power. The record's assumptions cannot replace the unretained outcome data or provide evidence of benefit, lack of benefit, or comparative effectiveness. Clear reporting of missing results is therefore essential: it helps readers distinguish an unanswered question from a finding that points in a particular direction.
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 consequential limitation is explicit: the registry states that primary outcome data were not collected and that results could not be retained. It attributes termination to the principal investigator no longer being employed at CTCA and not having rights to the trial information. This leaves no primary endpoint estimate and no reported analysis to evaluate.
Enrollment was 1, and no comparator group is reported. These features limit the scope of any possible inference. A single-group outcome, even if available, would not provide a randomized treatment contrast; the small enrollment would also leave little information about variability across participants. In this record, these design limitations are compounded by the complete absence of primary efficacy data.
Outcome definitions and measurement details are not reported in the registry record. For progression-free survival, the event criteria, time origin, assessment schedule, and censoring rules are unavailable. Response-rate criteria and the definition and calculation of duration of response are also not reported. Without these details, readers could not assess endpoint consistency or reproduce an analysis even if summary results were present.
The record does not report the analysis population, missing-data methods, multiplicity procedures, interim analyses, statistical analysis plan, or sample-size justification. It does not report further treatment exposure details, the safety observation period, or adverse-event classification criteria. These absences should not be filled by assumptions about usual practice. The listed treatment sequence and the counts in the safety entry are not enough to reconstruct those procedures.
The study dates are recorded as 2012-08 for start and 2014-10 for primary completion, and status is terminated. These registry entries do not establish the duration of an individual participant's follow-up or whether outcome assessments were completed. Overall, the record permits a limited description of design and conduct, but not an assessment of efficacy, endpoint precision, or comparative safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Dacarbazine and Carmustine in Metastatic Melanoma — NCT01692691.
- Registry record last updated 2018-04-04; status last verified 2018-03.
- Results first posted 2016-02-02.
- 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
NCT01692691 is a phase II, single-group, unmasked trial of dacarbazine, carmustine, and Neulasta in metastatic melanoma. The listed sequence is dacarbazine IV on Day 1, carmustine IV on Day 2, and Neulasta SC on Day 3. Enrollment is 1, and the status is terminated.
Progression-free survival over 8 weeks is the primary outcome; response rate and median duration of response are listed as secondary outcomes. The registry states that primary outcome data were not collected and results could not be retained. It reports 0 analyses. The safety entry lists 0 serious adverse events, 0 other events, and 0 deaths among 1 participant at risk.
The record supports no efficacy estimate or comparative treatment conclusion. Its principal contribution is documentation of the intended study question, single-group design, enrollment, termination, and unavailability of outcome results.