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 Riluzole in Treating Patients With Stage III or Stage IV Melanoma That Cannot Be Removed by Surgery (NCT00866840), a na phase 2 study. The primary outcome is Tumor Response as Measured by RECIST Criteria.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00866840 |
| Phase | Phase 2 |
| Condition | Melanoma (Skin) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 13 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Rutgers, The State University of New Jersey |
| Results posted | Yes |
2. Clinical Question
Population
Patients with stage III or stage IV melanoma that could not be removed by surgery. The registry record lists melanoma (skin) as the condition and records enrollment of 13 participants. It does not report further eligibility details or a description of the participants included in the primary outcome analysis.
Intervention
Riluzole, listed as a drug and described in the arm record as 100 mg orally twice daily. The registry record identifies the trial as phase II. It does not report additional details about treatment duration, adherence, dose changes, or discontinuation.
Comparator
There was no comparator arm: the model is single group, masking is none, and allocation is listed as NA. The registry record therefore does not support a direct comparison with another treatment or a control group.
Primary question
The primary outcome asks how many participants had tumor response assessed using RECIST criteria. Tumor imaging was performed after 6 weeks. The registry record describes how complete response, partial response, progressive disease, and stable disease were defined, but does not report the number of participants in those categories or the primary outcome result.
3. Trial Design
This was a phase II, single-group trial of riluzole in patients with advanced melanoma that could not be removed by surgery. The registry record identifies Rutgers, The State University of New Jersey, as the sponsor. The study is marked completed, with 13 participants enrolled. Its recorded start was 2009-04 and its primary completion was 2013-07.
The intervention was riluzole, described as 100 mg orally twice daily. Allocation is listed as NA, and masking as none. The trial model is single group. These features mean the record describes an intervention group without a concurrent comparison arm. It is not a randomized comparison, and any planning illustrations that assume two arms should not be mistaken for the trial’s actual design.
The primary outcome is tumor response assessed by RECIST criteria, using CT or MRI imaging. The outcome description specifies assessment of target lesions and states that imaging was performed after 6 weeks. The record also lists two secondary outcomes: number of participants with at least one adverse event and overall survival.
The registry record does not report the rationale for the planned enrollment, the statistical analysis plan, or details of the analysis population. It also does not give the observed primary outcome count. The completion status and enrollment describe the trial record, but do not by themselves indicate what the tumor-response findings were.
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 | Riluzole |
|---|---|
| STARTED | 13 |
| COMPLETED | 13 |
| NOT COMPLETED | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Riluzole |
|---|---|---|
| Participants analysed | 13 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 4 |
| Age, Categorical: >=65 years | Participants | 9 |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 7 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 13 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 13 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Tumor Response as Measured by RECIST Criteria, measured over Imaging for tumor assessments was performed after 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with tumor response measured using RECIST criteria. The record says target lesions were assessed by CT or MRI, with imaging performed after 6 weeks. It defines complete response as disappearance of all target lesions; partial response as a >=30% decrease in the sum of the longest diameter of target lesions; progressive disease as at least a 20% increase in that sum; and stable disease as neither enough shrinkage for partial response nor enough increase for progressive disease.
As a general concept, standardized response criteria provide a consistent way to classify changes in measurable tumors. Categories can distinguish shrinkage, growth, and changes that do not meet either threshold. A participant count is a straightforward way to report how many people fall into a defined category. The clinical interpretation still depends on knowing the actual counts and the analysis population.
The registry record does not report the number of participants with complete response, partial response, progressive disease, or stable disease. It therefore does not establish the observed primary endpoint result. It also does not report whether the primary analysis combined any response categories into a single definition of response.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The trial enrolled 13 participants and used a single-group design. The registry record identifies tumor response by RECIST as the primary outcome, with imaging performed after 6 weeks. However, it does not report the observed count for the primary endpoint or the distribution of participants across the RECIST categories. Without those results, the tumor-response finding cannot be described or quantified from the registry record.
The design is important to interpretation. Because there was no comparator arm and allocation is listed as NA, the record does not provide a randomized estimate of the difference between riluzole and another treatment. A response count in a single group, if available, would describe outcomes among participants in that group; it would not alone establish what would have happened under a different treatment or no treatment.
Precision also cannot be evaluated for the primary outcome from the information recorded. The registry record gives no confidence interval, standard error, or other measure of uncertainty for tumor response. A confidence interval is a general statistical way to express the range of effect values compatible with the observed data under a specified method. Without the endpoint result and its analysis details, that range cannot be interpreted.
The registry lists number of participants with at least one adverse event and overall survival as secondary outcomes. It gives safety counts and serious adverse-event terms, but does not report an overall-survival result. The safety counts should be read as descriptive summaries in a single group, not as comparisons with another treatment. The record does not state whether events were considered related to riluzole.
Planning illustrations use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and fractions of the observed effect. These are planning assumptions, not features of the enrolled single-group trial and not evidence of observed power. The registry record does not report the trial’s own sample-size justification. Since the primary effect estimate is not reported, no numerical planning interpretation should be inferred from the record alone.
9. Statistical Methods Explained
Outcome type and reporting. The primary outcome is a participant count, so it is a binary-style endpoint when the question is whether a participant meets a defined response criterion or does not. The record’s description also names four RECIST categories: complete response, partial response, progressive disease, and stable disease. It does not state which category or combination of categories was used as the final primary response definition, nor does it provide the counts for those categories. That distinction matters: a response rate requires a specified numerator and a defined denominator.
General concept: uncertainty around a count. Counts describe the participants observed in each outcome category. An estimate based on a small group can be imprecise because a small change in the observed count may materially alter the estimated proportion. A confidence interval communicates uncertainty around an estimate, subject to the assumptions of its method. No confidence interval or primary outcome estimate is reported in the registry record, so the precision of the tumor-response finding cannot be assessed.
Single-group interpretation. The trial model is single group, with allocation listed as NA and masking listed as none. General statistical interpretation of a single-group study focuses on the outcomes observed in that group. There is no concurrent control group from which to estimate a between-group treatment contrast. Even if a response count were reported, it would not by itself distinguish an intervention effect from other explanations for the observed outcomes. The record does not report a comparator or a randomized analysis.
Sample-size planning. The planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They also describe planning to detect a stated fraction of the observed effect. These assumptions concern planning illustrations, not the observed design: the registered trial is single group. The record does not report its own sample-size justification, target effect, or statistical power calculation. A planning sample size is not observed power and does not show whether the trial’s findings are conclusive.
General concept: effect size and planning. A sample-size calculation depends on the outcome, the effect the study aims to detect, the chosen error rate, and the analysis method. Planning for a smaller fraction of an observed effect asks about a different target than planning for the full observed effect. Such illustrations are conditional on their assumptions; they should not be read as proof that an effect exists or as a substitute for an uncertainty interval around trial results. The registry record does not provide enough primary-outcome information to interpret a numerical planning result as a trial finding.
Over-dispersion and event counts. Over-dispersion is a general concern when count data vary more than a chosen statistical model expects. It can make uncertainty estimates too narrow if the model assumes less variability than the data contain. The primary endpoint is described as a participant count with a binary interpretation, and the registry record does not report a model for over-dispersed data. It would therefore be inappropriate to claim that an over-dispersion adjustment was used or required.
Time-to-event methods. Overall survival is listed as a secondary outcome, but no survival result or analysis method is reported in the registry record. General time-to-event analyses account for the timing of events and may also account for participants whose event status is not observed during follow-up. The record does not state follow-up duration, censoring rules, survival estimates, or any model used. No survival conclusion can be drawn from the outcome name alone.
What is not documented. The registry record does not report the statistical analysis plan, confidence intervals, hypothesis tests, adjusted analyses, interim analyses, or the primary analysis population. These details should not be inferred from the phase designation or from the outcome description. The available information supports a description of the registered design and endpoint definitions, but not a reconstruction of the unreported analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists three outcome measures: tumor response as the primary outcome, plus number of participants with at least one adverse event and overall survival as secondary outcomes. It records one analysis. It does not report a multiplicity strategy, such as whether the primary outcome was formally prioritized over secondary outcomes for hypothesis testing or whether any adjustment was planned for multiple comparisons.
As a general statistical concept, testing several outcomes can increase the chance of at least one apparently notable finding when each is assessed separately. A prespecified testing strategy can clarify how such results should be interpreted. The registry record does not provide enough information to determine whether a strategy was used. The listed number of analyses alone does not establish how multiplicity was handled.
Missing-data methods and analysis populations are also not reported in the registry record. It does not state how participants without an evaluable scan were handled, whether all enrolled participants were included in the primary analysis, or whether any participants were excluded. These omissions matter because the denominator is needed to interpret a participant count or a proportion. The record also does not describe missing follow-up information for overall survival.
No assumptions about imputation, censoring, or analysis populations should be inferred. The primary outcome result itself is not reported, so the possible influence of missing data on that result cannot be assessed.
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 |
|---|---|---|---|
| Riluzole | 10 | 13 | 76.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Riluzole | 4 of 13 |
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 |
|---|---|
| Riluzole | 11 of 13 |
Most frequent serious adverse event terms
| Event term | Organ system | Riluzole |
|---|---|---|
| Muscle weakness, generalized or specific area | General disorders | 2 of 13 |
| Dehydration | General disorders | 2 of 13 |
| Dizziness | General disorders | 1 of 13 |
| Death | General disorders | 1 of 13 |
| Infection | Infections and infestations | 1 of 13 |
| Speech impairment (e.g., dysphasia or aphasia) | Nervous system disorders | 1 of 13 |
| Pain - Extremity-limb | General disorders | 1 of 13 |
| Neuropathy: motor | Nervous system disorders | 1 of 13 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Riluzole |
|---|---|---|
| Constitutional Symptoms | Gastrointestinal disorders | 8 of 13 |
| Gastrointestinal | Gastrointestinal disorders | 7 of 13 |
| Neurology | Nervous system disorders | 7 of 13 |
| Pain | General disorders | 5 of 13 |
| Metabolic/Laboratory | Metabolism and nutrition disorders | 3 of 13 |
| Musculoskeletal/Soft Tissue | Musculoskeletal and connective tissue disorders | 3 of 13 |
| Blood/Bone Marrow | Blood and lymphatic system disorders | 2 of 13 |
| Pulmonary/Upper Respiratory | Respiratory, thoracic and mediastinal disorders | 2 of 13 |
| Cardiac General | Cardiac disorders | 1 of 13 |
| Dermatology/Skin | Skin and subcutaneous tissue disorders | 1 of 13 |
Serious events with the highest arm-level rate: muscle weakness, generalized or specific area (15.4%), dehydration (15.4%), dizziness (7.7%), death (7.7%), infection (7.7%).
The registry safety record lists 13 participants at risk in the riluzole group. Its serious adverse-event fields record 10 participants affected, and its other adverse-event fields record 11 participants affected. These are separate registry summaries; they should not be added together, since the same participant may appear in more than one category. The record does not explain event overlap or provide further definitions in the information recorded.
The listed serious adverse-event terms include muscle weakness, generalized or specific area, at 15.4%; dehydration at 15.4%; and dizziness, death, infection, and speech impairment (e.g., dysphasia or aphasia), each at 7.7%. The record also lists 4 deaths. It does not specify in this information whether those deaths were judged related to treatment, or how the death count relates to the listed adverse-event terms.
These figures are descriptive for the enrolled group. They do not establish comparative risk because there was no comparator arm. The registry record does not provide event timing, severity grades, exposure duration, or attribution to riluzole. The percentages should be interpreted as the registry’s reported figures, not as a causal estimate or a comparison with another treatment.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study of riluzole enrolled 13 participants with advanced melanoma that could not be removed by surgery. It specifies a dose of 100 mg orally twice daily and a primary outcome based on tumor response assessed using RECIST criteria after imaging at 6 weeks. It also records safety outcomes and lists overall survival as a secondary outcome.
It does not report the primary tumor-response count or the number of participants in the RECIST response categories. As a result, the record does not show how many participants had complete response, partial response, progressive disease, or stable disease. It also does not report an overall-survival result. A conclusion about either endpoint cannot be drawn from the outcome definitions alone.
The single-group design can describe outcomes among participants who received the intervention, but it does not provide a randomized comparison. Without a comparator, observed outcomes cannot be interpreted as a measured difference between riluzole and another treatment. The available safety summaries likewise do not establish comparative risk or whether events were caused by treatment.
In general, a clinical result is most informative when its estimate, uncertainty, denominator, and analysis methods are available. Several of these elements are not reported in the registry record for the primary endpoint. The completion status and enrollment count are study-record details, not evidence of a particular tumor-response result. The evidence described in the record is therefore limited to the design, endpoint definitions, and reported safety summaries.
13. Why This Trial Matters Statistically
This trial illustrates why study design and outcome reporting need to be read together. A phase II label and a defined primary endpoint identify the study’s purpose and measurement approach, but they do not supply the result. Here, the registry describes a RECIST-based tumor-response endpoint and an imaging assessment after 6 weeks, yet does not report the primary response count. The endpoint can be understood, but its observed value and precision cannot be assessed from the record.
The single-group design is also central to statistical interpretation. It can document what was observed among enrolled participants, but it does not create a randomized comparison with another treatment. This distinction prevents descriptive results from being mistaken for comparative treatment effects. The record’s planning assumptions involving two arms are planning illustrations and should not be confused with the actual single-group design.
Safety reporting raises a related issue: counts and percentages describe events in the recorded group, while causality and comparative risk require information the registry record does not give. The figures are still useful as descriptions of what the record lists, provided their limits are kept clear.
More broadly, this trial highlights the value of reporting endpoint counts, denominators, uncertainty estimates, analysis populations, missing-data handling, and prespecified methods. Without these details, readers cannot fully evaluate precision or reproduce the analysis. The statistical importance is not a conclusion about riluzole; it is the distinction between a clearly specified question and a result that is adequately reported.
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.
Several trial-specific limitations follow directly from the registry record. First, the study is single group, with allocation listed as NA and masking listed as none. There is no randomized comparator, so the record cannot support a direct between-group treatment contrast. The enrolled group contains 13 participants, and no further participant characteristics are reported in the information available.
Second, the primary endpoint result is not reported. Although RECIST categories and the imaging timeframe are described, the record gives no count for complete response, partial response, progressive disease, or stable disease. It also does not state the primary analysis denominator or provide an uncertainty estimate. This prevents assessment of the observed response and its precision.
Third, important analysis details are not reported in the registry record. These include the statistical analysis plan, sample-size justification, analysis population, handling of missing outcome data, and any interim analyses. The record does not describe the overall-survival analysis or report an overall-survival result.
Finally, the safety summaries do not describe event timing, severity grading, treatment attribution, or overlap between event categories. The record lists deaths but does not explain their relationship to treatment or to the serious adverse-event terms. These limitations constrain interpretation of the registry record; they do not establish that unreported procedures or analyses did or did not occur.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Riluzole in Treating Patients With Stage III or Stage IV Melanoma That Cannot Be Removed by Surgery — NCT00866840.
- Registry record last updated 2024-01-19; status last verified 2023-12.
- Results first posted 2022-09-28.
- 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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18. Record Summary
The registry record describes NCT00866840, a completed phase II, single-group trial sponsored by Rutgers, The State University of New Jersey. It enrolled 13 participants with stage III or stage IV melanoma that could not be removed by surgery. The intervention was riluzole, 100 mg orally twice daily. The recorded start was 2009-04, and primary completion was 2013-07.
The primary outcome was tumor response by RECIST criteria, with imaging performed after 6 weeks. Number of participants with at least one adverse event and overall survival were listed as secondary outcomes. The registry record reports safety counts and serious adverse-event terms, but does not report the primary tumor-response result or an overall-survival result. It does not report the statistical analysis plan, analysis populations, or missing-data methods.