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 Effect of Proactive Management of Side Effects on Treatment Compliance in Malignant Melanoma Patients on High-dose Intron A Therapy (Study P04600) (NCT00723710), a registered phase not stated study. The primary outcome is Number of Participants Who Completed Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00723710 |
| Phase | Phase not stated |
| Condition | Melanoma |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 299 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Merck Sharp & Dohme LLC |
| Results posted | Yes |
2. Clinical Question
Population
Patients with malignant melanoma who had undergone surgery and were free of disease after surgery but considered at high risk for systemic recurrence. The primary outcome population is defined as participants who started the induction phase. The registry record lists enrollment of 299.
Intervention
Intron A, identified in the registry record as interferon alfa-2b, in an adjuvant treatment plan described as high-dose therapy for one year. The study title and official title frame the question around proactive side-effect management and a nursing support program, but the registry record does not describe the program's specific procedures or components.
Comparator
No like-for-like control comparison is described. The record does not state an allocation method, and it explicitly notes that the arms are not a randomized comparison. The registry record does not report a comparator treatment or a control arm suitable for estimating a comparative effect.
Primary question
How many participants who started induction completed treatment, defined as completing both the induction and maintenance phases, within the timeframe of up to one year? The study objective is described as maximizing compliance through proactive management of side effects in day-to-day care. The completion result and an analysis linking side-effect management to completion are not reported in the registry record.
This clinical question is about treatment completion, not a reported comparison of clinical outcomes between management strategies. The record names Intron A as the intervention and describes the target population as patients at high risk for recurrence after surgery. It does not establish what support each participant received, whether support differed between participants, or how adherence was assessed beyond the stated completion definition. Consequently, the registered objective can be described, but the available record does not show whether the objective was achieved or whether proactive management changed completion.
3. Trial Design
The registry record identifies NCT00723710 as a completed study sponsored by Merck Sharp & Dohme LLC. Its brief description concerns patients with malignant melanoma after surgery who were to receive adjuvant high-dose Intron A therapy for one year. The stated aim was to maximize treatment compliance through proactive side-effect management in day-to-day healthcare. The official title presents the study as a nursing support program for patients with high-risk melanoma receiving high-dose Intron A.
The record lists an enrollment of 299, a start date of 2006-04, and a primary completion date of 2012-09. The named intervention is Intron A (interferon alfa-2b; SCH 30500). The listed arm description concerns participants who were free of disease after surgery but at high risk for systemic recurrence. The record does not describe another treatment arm suitable as a control. It also does not state the allocation method; the record specifically cautions that this is not a randomized comparison and that the arms are not a like-for-like control comparison.
The primary outcome is the number of participants who completed treatment, with completion defined as completing both induction and maintenance. Its timeframe is up to one year, and its stated population consists of participants who started induction. The registry record lists one outcome measure and no analysis results. Details such as the setting, eligibility criteria beyond the population description, planned enrollment rationale, treatment delivery procedures, and follow-up procedures are not reported in the registry record. The titles indicate a focus on supportive management, but the specific support procedures are not described in the registry record.
Because there is no reported randomized comparison or analyzed comparator, the design information does not support a causal contrast between proactive management and another strategy. The posted information is best read as a description of the study objective, registered treatment, population, and completion endpoint, rather than as evidence from a comparative treatment-effect analysis.
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.
Before assignment: 299 participants were enrolled. Of these, 294 were considered efficacy evaluable (5 participants were excluded from efficacy analyses: 2 moved, 1 required radiation, 1 not eligible and 1 missing data).
Overall Study
| Stage | Intron A |
|---|---|
| STARTED | 294 |
| COMPLETED | 120 |
| NOT COMPLETED | 174 |
Reasons for not completing this period:
| Reason | Intron A |
|---|---|
| Disease progression | 48 |
| Adverse Event | 88 |
| Withdrawal by Subject | 36 |
| Physician Decision | 2 |
Analysis population for the primary outcome, as defined in the registry: The number of participants who started the induction phase
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 | Intron A |
|---|---|---|
| Participants analysed | 294 | |
| Age, Continuous | years | 52 (19 to 78) |
| Sex: Female, Male: Female | Participants | 124 |
| Sex: Female, Male: Male | Participants | 170 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants Who Completed Treatment, measured over Up to 1 year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants who completed treatment. The registry defines completion as completing both the induction and maintenance phases. The stated timeframe is up to one year, and the population for the measure is participants who started induction. The registry record does not report the number who met this definition.
As a general statistical concept, a completion endpoint captures whether participants reached a prespecified treatment milestone. It can be relevant to a study concerned with treatment compliance because it reflects persistence through the phases defined in the protocol or registry description. It is not, by itself, a complete account of medication exposure: completion status does not tell a reader about dose interruptions, dose changes, time spent on treatment, or the reasons for stopping unless those details are reported separately. Such details are not reported in the registry record.
A count endpoint can describe how many participants completed treatment in the defined population. For interpretation, the denominator matters: the registry specifies participants who started induction as the population, while the record also lists enrollment of 299. The posted information does not provide the completion count or an analyzed denominator, so a completion proportion cannot be assessed from the registry record. Nor is a comparison of completion between different side-effect management approaches reported. The endpoint definition is clear at the level of treatment phases, but the outcome result needed to answer the primary question is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study's stated purpose and primary endpoint, but it does not provide an analysis of treatment completion. The primary measure is the number of participants completing both induction and maintenance among those who started induction, with a timeframe of up to one year. Although the record lists enrollment of 299, it does not report how many participants completed both phases. The central outcome therefore cannot be quantified from the posted results.
The record lists no analyses. It does not provide an estimate of completion, a confidence interval, a hypothesis test, or an adjusted analysis for the primary endpoint. A confidence interval is a statistical way to express the uncertainty around an estimated quantity; without a reported estimate and interval, the precision of the completion result cannot be evaluated. A p-value would address compatibility with a specified statistical comparison under a model, but no such analysis is reported. No conclusion about the size or certainty of an effect on compliance can be drawn from the stated study objective alone.
The registry also does not describe a randomized, like-for-like comparison. Its note says allocation was not stated and that the arms are not a like-for-like control comparison. This matters because a difference in completion between groups, if one were reported, could reflect differences other than the side-effect management approach. The record does not provide an analyzed comparison that could address those concerns. It is therefore not possible to attribute a completion outcome to proactive management from the information available.
The safety information is descriptive. The Intron-A group lists 21 participants affected by serious adverse events among 299 at risk, and 297 participants affected by other adverse events among 299 at risk. The registry lists headache at 1.0% as a leading serious event term, and nausea, vomiting, cellulitis, myalgia, and metastases to central nervous system at 0.7% each. These figures describe the posted safety record, not a comparative safety effect. The record does not provide a control group for a like-for-like comparison, nor does it report deaths in the listed field.
In short, the registry supplies the endpoint definition, the treatment context, and descriptive safety counts, but not the primary completion result or an inferential analysis. The study's objective should not be treated as evidence that compliance was maximized. The posted record supports description of what was intended and of the safety counts that were recorded; it does not establish the magnitude, precision, or cause of any effect on treatment completion.
9. Statistical Methods Explained
The primary measure is a count: the number of participants who completed both induction and maintenance. The registry defines the population as participants who started induction and gives a timeframe of up to one year. For a count to be interpretable, readers need both the number meeting the endpoint and the number eligible for that endpoint. The registry record provides the population definition and lists enrollment of 299, but it does not report the number completing treatment or the analyzed denominator. A count alone also does not describe the uncertainty in the result.
What a confidence interval would add
In general, a confidence interval expresses the precision of an estimate under the statistical method used to calculate it. A narrow interval indicates that the estimate is more precisely determined by the data and model than an estimate with a wide interval. The registry record does not report a completion estimate or confidence interval, so the precision of the primary outcome cannot be judged. An interval would not remove concerns about study design or establish causation; it would describe uncertainty around an estimate, conditional on the analysis and its assumptions.
What a hypothesis test would answer
In general, a hypothesis test evaluates data relative to a stated null hypothesis and statistical model. A p-value is not the probability that a treatment works, nor does it measure the size or importance of an effect. Interpreting one requires knowing the comparison, analysis population, and methods used. The registry record lists no analyses and does not report a p-value for treatment completion. Since no like-for-like comparator is described, the record also does not supply a comparative question for a conventional treatment-effect test.
Why the comparison structure matters
Random allocation, when used and properly implemented, can help make groups comparable in expectation, supporting interpretation of differences as effects of the assigned strategies. The registry record does not state an allocation method and cautions that the arms are not a like-for-like control comparison. This means any unreported or hypothetical difference between groups should not be read as a randomized treatment effect. Differences could reflect group composition, care circumstances, or other factors; the record does not provide information to assess or adjust for these possibilities.
The study title emphasizes proactive side-effect management and a nursing support program, while the listed intervention is Intron A. The record does not detail the support program components or indicate how support was measured. Without those details, it is not possible to identify a specific exposure contrast or determine how consistently the proposed management approach was implemented. The study objective is useful context, but it is not a statistical result.
Planning illustrations are not observed evidence
The record describes planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also states that the effect to detect is a stated fraction of the observed effect. These are assumptions for planning illustrations, not a description of the study's actual design or an observed analysis. The record does not report the observed effect needed to evaluate such illustrations, and no calculated sample-size values are stated in the registry information. An illustration of participants per arm for 80% power, if shown with the results, should be read as a planning quantity under its assumptions, not as evidence that the study had a particular probability of finding an effect. It is not observed power.
More generally, a planning calculation depends on its target effect, significance level, allocation assumptions, and statistical approximation. Changing an assumption can change the planned sample size. Equal allocation between two arms is specifically a planning assumption in the record; it does not establish that the study actually enrolled participants in two comparable arms. The planning description also does not replace a protocol or sample-size justification. The protocol rationale and the trial's own sample-size justification are not reported in the registry record.
Counts, rates, and over-dispersion
As a general concept, a count records how many participants or events meet a definition, while a rate incorporates a time-at-risk denominator. A participant-level count can answer a question about the number affected, but it does not necessarily describe how often events occurred per unit of time. The posted safety figures are participant counts at risk and affected; the registry record does not provide person-time or event-level totals. Over-dispersion is a general concern for count models when observed variability exceeds what a chosen model assumes. The record does not report a count-model analysis or a dispersion estimate, so no conclusion about over-dispersion or its handling can be made for this study.
For the primary endpoint, the key statistical limitation is not an unusual model result but the absence of a reported analysis. Without the number who completed treatment, an analyzed denominator, and a stated analysis method, readers cannot evaluate the endpoint estimate, its uncertainty, or how the analysis treated participants whose outcome was unknown. Those details are not reported in the registry record.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Intron A | 294 | 120 | 40.8% | 35.1% to 46.7% | 35.4% to 46.5% | 35.3% to 46.5% |
R code and output
arm <- c("Intron A")
n <- c(294)
events <- c(120)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Intron A 294 120 40.8 35.1 46.7 35.4 46.5 35.3 46.511. Multiplicity, Missing Data and Analysis Populations
The registry record lists one outcome measure and no analyses. It does not describe a statistical plan for controlling multiplicity, such as how multiple outcomes, time points, or subgroup comparisons would be handled. The number of registered outcome measures alone does not establish whether additional outcomes or exploratory analyses were planned or conducted. Such plans are not reported in the registry record.
The primary outcome population is defined as participants who started induction, but the record does not explain how participants with missing completion status were handled. Missing-data methods, imputation procedures, sensitivity analyses, and reasons for missing outcome information are not reported in the registry record. Without those details, the extent to which an analysis might depend on assumptions about participants with unknown outcomes cannot be assessed.
The registry does not describe an intention-to-treat population or another prespecified analysis population beyond the population stated for the primary outcome. It also does not report exclusions from analysis, protocol deviations, withdrawals, or the number retained through follow-up. These are relevant because different analysis populations can yield different interpretations, particularly for a completion endpoint where treatment discontinuation and unavailable status information may matter. The record does not provide enough information to assess those issues.
Interim analyses, stopping boundaries, and a multiplicity adjustment plan are not reported in the registry record. Their absence from the record should not be interpreted as proof that none were used; it means they cannot be evaluated from the posted information. No multiplicity-adjusted or missing-data analysis is listed among the reported results.
12. 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 |
|---|---|---|---|
| Intron-A | 21 | 299 | 7.0% |
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 |
|---|---|
| Intron-A | 297 of 299 |
Most frequent serious adverse event terms
| Event term | Organ system | Intron-A |
|---|---|---|
| HEADACHE | Nervous system disorders | 3 of 299 |
| NAUSEA | Gastrointestinal disorders | 2 of 299 |
| VOMITING | Gastrointestinal disorders | 2 of 299 |
| CELLULITIS | Infections and infestations | 2 of 299 |
| MYALGIA | Musculoskeletal and connective tissue disorders | 2 of 299 |
| METASTASES TO CENTRAL NERVOUS SYSTEM | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 299 |
| FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | 1 of 299 |
| NEUTROPENIA | Blood and lymphatic system disorders | 1 of 299 |
| PANCYTOPENIA | Blood and lymphatic system disorders | 1 of 299 |
| ATRIAL FIBRILLATION | Cardiac disorders | 1 of 299 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Intron-A |
|---|---|---|
| INFLUENZA LIKE ILLNESS | General disorders | 220 of 299 |
| FATIGUE | General disorders | 189 of 299 |
| NAUSEA | Gastrointestinal disorders | 159 of 299 |
| HEADACHE | Nervous system disorders | 145 of 299 |
| LIVER FUNCTION TEST ABNORMAL | Investigations | 117 of 299 |
| DEPRESSION | Psychiatric disorders | 97 of 299 |
| PYREXIA | General disorders | 86 of 299 |
| MYALGIA | Musculoskeletal and connective tissue disorders | 84 of 299 |
| DECREASED APPETITE | Metabolism and nutrition disorders | 80 of 299 |
| NEUTROPENIA | Blood and lymphatic system disorders | 69 of 299 |
Serious events with the highest arm-level rate: headache (1.0%), nausea (0.7%), vomiting (0.7%), cellulitis (0.7%), myalgia (0.7%).
The registry lists safety information for the Intron-A group. For serious adverse events, 299 participants were at risk and 21 were affected. For other adverse events, 299 were at risk and 297 were affected. These are participant counts, not a count of all individual event episodes. The record does not explain whether a participant could contribute to more than one listed term, so the term counts should not be added to derive a number of unique participants.
The leading serious adverse event terms are headache at 1.0%, and nausea, vomiting, cellulitis, myalgia, and metastases to central nervous system at 0.7% each. These are the percentages as listed in the registry record. The record does not provide a comparison group suitable for assessing whether these figures differ from another treatment or from a control condition. It also does not provide event-level detail, timing, duration, severity grading beyond the serious-event classification, or an assessment of relatedness for these terms.
The deaths field is not reported in the registry record. These safety counts are descriptive and do not establish that the intervention caused an event or that an event was unrelated to treatment. The record does not provide the information needed for a comparative safety analysis.
13. What the Result Does, and Does Not, Mean
The posted results describe a study intended to maximize treatment compliance through proactive management of side effects for patients receiving high-dose Intron A after surgery for high-risk melanoma. The primary endpoint is treatment completion, defined as completing both induction and maintenance. However, the completion count and any analysis of that endpoint are not reported in the registry record. The study objective cannot substitute for an observed outcome.
The record does show that the Intron-A group had 21 participants affected by serious adverse events among 299 at risk, and 297 affected by other adverse events among 299 at risk. It also lists several serious adverse event terms with their percentages. These data provide a descriptive account of the posted safety results, but do not establish a comparative effect or causal relationship. A like-for-like control comparison is not reported.
As a general statistical principle, a study's stated intention is different from evidence that an outcome changed. To assess whether a side-effect management strategy influenced completion, readers would need a reported completion result, a suitable comparison, and enough information about allocation, analysis populations, missing outcomes, and uncertainty. The registry record does not provide these elements. It is therefore not possible to determine from the posted results whether proactive management increased completion, how large any difference might have been, or how precisely it was estimated.
The record also does not establish how well the nursing support program was implemented or which elements participants received. The study titles convey the focus of the investigation, but the specific support procedures are not reported. The results should therefore be interpreted as limited registry information about a treatment-completion question and descriptive safety counts, rather than as proof of the benefit or lack of benefit of a support approach.
Nothing in the available record supports a recommendation about care. The meaningful statistical conclusion is narrower: the primary endpoint is defined, but its result and analysis are unavailable in the registry record; the safety data are reported descriptively without a comparable control group.
14. Why This Trial Matters Statistically
This study raises a statistical question that is important whenever treatment completion is an objective: how should completion be defined, and what data are needed to estimate it clearly? The registry specifies completion as finishing both induction and maintenance, and identifies the population as participants who started induction. That definition makes the endpoint interpretable in principle. Yet without the number completing treatment and the analyzed denominator, the central quantity remains unknown.
The record also illustrates why an objective and an outcome result must be kept separate. A study may aim to maximize compliance through proactive side-effect management, but the aim does not show that compliance changed. A reported estimate and an appropriate comparison are needed to assess an effect. In this record, no analysis is listed, and the registry cautions that the arms are not a like-for-like control comparison.
From a biostatistical perspective, those design details affect what can be inferred, not merely how results are presented. A non-comparable group structure limits causal interpretation, while missing details on analysis populations and missing outcomes limit assessment of the endpoint itself. The listed safety counts remain useful descriptive information, but they do not fill the gap in the primary completion analysis or support a comparative safety conclusion.
The case also demonstrates the value of transparent reporting. Explicitly reporting the endpoint estimate, its denominator, uncertainty, analysis population, and missing-data handling would help readers distinguish the observed result from the study's purpose and evaluate precision. Without those elements, the registry record supports only a constrained description of the study and its posted safety data. The design and reporting limitations are therefore central to the statistical meaning of the record.
15. 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 primary limitation is that the registry record does not report the result for its primary endpoint. Although treatment completion has a stated definition and timeframe, the number completing both phases and the analyzed denominator are not reported. The record lists no analyses, leaving the endpoint's magnitude and precision unknown.
The comparison structure is also limited for causal interpretation. The registry does not state allocation, and it notes that this is not a randomized comparison and that the arms are not a like-for-like control comparison. A comparator suitable for estimating the effect of proactive side-effect management is not reported. As a result, the record cannot establish whether any completion outcome was caused by the support approach.
The official title and study objective refer to a nursing support program and proactive management, but the registry record does not describe the program's components, delivery, uptake, or consistency. The listed intervention is Intron A. Without details about the support exposure, the question's intended management contrast cannot be evaluated from the registry record.
Other important methodological details are not reported in the registry record: eligibility criteria beyond the population description, the sample-size justification, outcome missingness and its handling, analysis populations, withdrawals, protocol deviations, interim analyses, and the statistical plan. The deaths field is also not reported. These omissions limit assessment of both the primary outcome and the descriptive safety information.
Finally, the safety data are not comparative. The record lists affected participants and leading serious event terms, but does not provide a like-for-like control group or the detail needed to evaluate event timing, relatedness, and participant-level overlap. The posted information can be described, but its scope should not be extended to claims about comparative effectiveness, comparative safety, or the effect of a support program.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Effect of Proactive Management of Side Effects on Treatment Compliance in Malignant Melanoma Patients on High-dose Intron A Therapy (Study P04600) — NCT00723710.
- Registry record last updated 2015-08-26; status last verified 2015-08.
- Results first posted 2013-10-28.
- 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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19. Record Summary
NCT00723710 is listed as completed and sponsored by Merck Sharp & Dohme LLC. The study concerns adjuvant high-dose Intron A for patients with malignant melanoma after surgery, with an objective of maximizing treatment compliance through proactive side-effect management. The registry lists enrollment of 299, a start date of 2006-04, and a primary completion date of 2012-09.
The primary endpoint is the number completing both induction and maintenance among participants who started induction, within up to one year. The completion result and analysis are not reported in the registry record. The record lists 21 participants affected by serious adverse events among 299 at risk and 297 affected by other adverse events among 299 at risk. Allocation is not stated, and the record cautions that the arms are not a like-for-like control comparison. The posted information therefore supports descriptive reporting, not a comparative estimate of the effect on treatment completion.