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 Special Survey on Parkinson's Disease (PD) Patients Without Concomitant Use of L-Dopa (NCT00613301), a registered phase not stated study. The primary outcome is Proportion of Adverse Events, Adverse Drug Reactions, Serious Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00613301 |
| Phase | Phase not stated |
| Condition | Parkinson Disease |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 416 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Boehringer Ingelheim |
| Results posted | Yes |
2. Clinical Question
Population
People with Parkinson’s disease treated in daily clinical settings in Japan, without concomitant L-Dopa supplementation. The registry record reports 416 enrolled participants, 364 patients with available case report forms, and a safety group of 346 in the adverse-event results. The record’s description of how these groups relate is incomplete.
Intervention
Pramipexole treatment without concomitant L-Dopa. The survey was a post-marketing surveillance effort intended to collect safety and effectiveness information during routine clinical care. The record does not report treatment dose, dose changes, adherence, or the detailed treatment protocol.
Comparator
No comparator group is reported. The allocation is not stated, and the record explicitly indicates that this is not a randomized comparison. The safety results therefore describe the pramipexole group; they do not provide a like-for-like control comparison.
Primary question
What proportions of participants experienced adverse events, adverse drug reactions, or serious adverse events during 36 months? The primary outcome is framed as safety surveillance in Parkinson’s disease patients without concomitant levodopa. The posted results give affected-participant counts for serious and other adverse events, but do not provide a complete set of outcome proportions for all three categories.
The registry also lists Clinical Global Impression of Improvement, change from baseline in Unified Parkinson’s Disease Rating Scale Part III total score, and change from baseline in Modified Hoehn & Yahr Rating Scale as secondary outcomes. Those outcomes could describe clinical status or change during treatment, but their results are not reported in the registry record. The primary question is consequently narrower than the survey’s stated aim to collect both safety and effectiveness information. The available numerical results support a description of recorded adverse events in the listed safety group, not an estimate of comparative benefit or harm.
3. Trial Design
This is a post-marketing surveillance survey, not a randomized controlled trial. Its stated purpose was to collect safety and effectiveness information for Parkinson’s disease patients treated with pramipexole without concomitant L-Dopa in daily clinical settings in Japan. The registry identifies Boehringer Ingelheim as the sponsor. The record gives a start date of 2004-02 and a primary completion date of 2009-09, and lists the status as completed.
The registry reports enrollment of 416. Its primary-outcome description says that 364 patients had available case report forms, then lists several exclusions: 5 patients with no visit since the first prescription, 12 irregularly enrolled patients, 9 excluded from analysis according to regulatory requirement, and 1 patient with no safety data available. The description then says that there were 346 patients in a further group, but the sentence is incomplete. The adverse-event results also list 346 participants at risk for each reported event category. The record does not provide a complete participant-flow account explaining the relationship among these counts.
There is one listed treatment group, pramipexole. No allocation method is stated, and the registry notes that the arms are not a like-for-like control comparison. No untreated, placebo, or active-comparator group is reported. The treatment dose, the rules for changing or stopping treatment, visit schedule, and detailed data-collection procedures are not reported in the registry record.
The primary outcome concerns the proportion of participants with adverse events, adverse drug reactions, and serious adverse events during 36 months. Three secondary outcomes are named: Clinical Global Impression of Improvement, change from baseline in UPDRS Part III total score, and change from baseline in Modified Hoehn & Yahr Rating Scale. Although the record identifies four outcome measures in total, it reports no statistical analyses for these outcomes. The protocol’s sample-size justification, any planned interim analyses, and a detailed statistical analysis plan are not reported in the registry record.
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 | Pramipexole |
|---|---|
| STARTED | 416 |
| COMPLETED | 188 |
| NOT COMPLETED | 228 |
Reasons for not completing this period:
| Reason | Pramipexole |
|---|---|
| Adverse Event | 49 |
| Lost to Follow-up | 88 |
| Withdrawal by Subject | 5 |
| Death | 6 |
| Lack of Efficacy | 13 |
| Physician Decision | 2 |
| No data was collected | 53 |
| Irregularly enrolled patients | 12 |
Analysis population for the primary outcome, as defined in the registry: 364 patients had available case report forms. Patients excluded: 5 patients with no visit since the first prescription, 12 irregularly enrolled patients, 9 excluded from analysis according to regulatory requirement, and 1 patient with no safety data available. As a result, there were 346 patients in the safety analysis set.
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: Data Set for Safety Analysis
| Characteristic | Unit | Pramipexole |
|---|---|---|
| Participants analysed | 346 | |
| Age, Continuous | years | 66.2 (SD 9.7) |
| Sex: Female, Male: Female | Participants | 187 |
| Sex: Female, Male: Male | Participants | 159 |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Adverse Events, Adverse Drug Reactions, Serious Adverse Events, measured over during 36 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Proportion (percentage of participants) |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is described as the proportion of participants with adverse events, adverse drug reactions, and serious adverse events during 36 months. It is a safety endpoint: it aims to describe how often these categories were recorded among patients treated with pramipexole without concomitant levodopa. A participant-based proportion can be useful in surveillance because it expresses the number of people affected relative to the group being followed. It does not, by itself, establish that treatment caused an event.
The posted adverse-event results list 346 participants at risk, with 29 affected by serious adverse events and 106 affected by other adverse events. The record does not give a corresponding overall count for adverse drug reactions, nor does it report a complete set of endpoint percentages for the primary outcome. It does not specify in the posted results whether the serious and other categories are mutually exclusive or how repeat events in one participant were handled. The record’s primary-outcome description gives a 36-month timeframe, but detailed follow-up and event-ascertainment procedures are not reported in the registry record.
Safety surveillance is used to characterize events observed during treatment in the setting under study. General statistical interpretation requires attention to the population followed, the period of observation, and the rules used to define and collect events. Without a control group, these counts describe what was recorded in the pramipexole group; they do not estimate how the same patients would have fared under another treatment or without treatment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports a single pramipexole group and lists 346 participants at risk for the adverse-event results. Among those listed, 29 were affected by serious adverse events and 106 by other adverse events. These are counts in the registry record. They should be read as descriptions of recorded events in this group, not as comparative estimates. The record does not report a control group, random allocation, or a treatment contrast.
The primary outcome is titled as proportions of adverse events, adverse drug reactions, and serious adverse events, with a 36-month timeframe. However, the posted results do not give complete proportions for each named category. Counts are available for serious and other adverse events, while a count for adverse drug reactions is not reported in the registry record. No confidence intervals, adjusted estimates, or formal tests are reported. As a result, the precision of the safety estimates cannot be assessed from the registry results alone. A count is informative about how many participants were recorded as affected, but does not show the uncertainty around the underlying event frequency.
The serious-event terms with reported percentages include cerebral infarction at 0.9%, pneumonia aspiration at 0.9%, lung neoplasm malignant at 0.6%, neuroleptic malignant syndrome at 0.6%, pneumonia at 0.3%, and septic shock at 0.3%. These percentages are reported for individual terms. The record does not state whether participants could contribute to more than one term, how terms were selected for listing, or whether these are all serious-event terms. They should not be added together or treated as a combined risk estimate.
The registry names three secondary outcomes: Clinical Global Impression of Improvement, change from baseline in UPDRS Part III total score, and change from baseline in Modified Hoehn & Yahr Rating Scale. It reports no analyses for these outcomes. Therefore, the stated survey aim includes effectiveness information, but effectiveness findings cannot be interpreted from the posted results. The number of outcome measures is four, while the number of analyses reported is zero.
General interpretation of an uncontrolled surveillance record is descriptive. Events observed during treatment may reflect the underlying condition, other aspects of care, participant characteristics, or treatment; this record does not separate those possibilities. The absence of a comparator also means that differences between treatment strategies cannot be estimated. These limitations do not make the observed counts meaningless: they describe recorded events in the listed group. They do limit what can be concluded about causation, relative risk, and comparative effectiveness.
The participant accounting needs caution. Enrollment is 416, the narrative refers to 364 patients with available case report forms, and the adverse-event results use 346 at risk. The listed exclusions and the truncated sentence do not provide a complete, readily reconstructable flow from enrollment to the safety group. The registry does not report enough information to resolve that accounting or to determine whether all participants had the same duration of observation. Thus, the record supports a bounded reading: specific event counts and term percentages are posted, but a complete account of outcome estimation and its precision is not.
9. Statistical Methods Explained
The registry record presents a surveillance outcome as a proportion of participants, but the posted adverse-event results are principally counts. General statistical explanation helps distinguish these kinds of summaries. A count states how many participants were recorded in a category. A proportion relates that count to a defined group at risk. A confidence interval, when reported, describes statistical uncertainty around an estimate under specified assumptions. The registry does not report confidence intervals for the primary outcome, so the precision of its estimates cannot be read from the posted results.
What does a participant-based safety measure represent?
In general, a participant-based measure counts people who experienced an event at least once during a defined observation period. This differs from counting every event occurrence: one participant with repeated events may still count once in a participant-based measure. The record describes the primary outcome using the proportion of participants, and the adverse-event results provide affected counts. However, the detailed counting rules are not reported in the registry record. It is therefore not possible to determine from the record how repeat events, events in multiple categories, or incomplete observation were handled.
The primary timeframe is described as during 36 months. General interpretation of a fixed-period proportion depends on whether participants were observed for the full period and how time outside follow-up was treated. If observation duration varies, a simple participant count may not represent the same amount of follow-up for every person. The registry does not report individual follow-up durations, censoring rules, or a time-to-event analysis. No event rate per person-time is reported. The posted outcome should therefore be read in the terms the record gives: affected-participant counts, rather than an event rate adjusted for differing time under observation.
Why does the lack of a comparator matter?
General statistical comparisons require a defined contrast, such as outcomes under one treatment versus outcomes under another. Here, the registry lists a pramipexole group and says that allocation is not stated and the arms are not a like-for-like control comparison. There is no reported control group with which to estimate a treatment difference. Consequently, the adverse-event counts are descriptive of the group under surveillance. They cannot isolate a treatment effect from other explanations for events, including the condition being treated or other features of routine care.
Randomization, when used, can help make comparison groups similar on average at the start of a study. This record does not report random allocation. It would be inappropriate to assume a randomized design or to apply an interpretation that depends on randomized groups. Likewise, no adjusted comparison is reported. The registry record does not describe a model that accounts for participant characteristics or other factors.
How should the event counts and term percentages be read?
The adverse-event results list 346 participants at risk, 29 affected by serious adverse events, and 106 affected by other adverse events. These figures are counts, not inferential comparisons. The listed serious-event terms have percentages: cerebral infarction and pneumonia aspiration are each 0.9%; lung neoplasm malignant and neuroleptic malignant syndrome are each 0.6%; pneumonia and septic shock are each 0.3%. Those term-level percentages describe the posted categories. The record does not report confidence intervals around them, the full list of terms, or the rule for choosing which terms to display.
In general, uncertainty tends to be greater for less frequent outcomes because fewer observations inform their estimates. A confidence interval would help communicate that uncertainty, but no such intervals are reported. Likewise, a percentage without a comparator does not show whether an event is more or less common than it would be under another treatment. The term-level figures should not be summed: the registry does not report whether categories overlap or whether a participant could appear under more than one term.
What do the planning assumptions mean?
The record describes planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a specified fraction of the observed effect. General sample-size illustrations answer a hypothetical design question: how many participants might be needed under stated assumptions to detect a target effect with a chosen statistical power. They are not results from participants and are not observed power. The record’s planning assumptions do not turn this single-group surveillance survey into a two-arm randomized comparison, and the registry’s own sample-size justification is not reported.
For time-to-event planning, the record says illustrations use the total number of events under a Schoenfeld approach; other illustrations use participants per arm. These are descriptions of planning methods, not evidence that a corresponding event-driven or two-arm design was used in the survey. No planning outputs are interpreted as findings about the observed safety group.
What is not available for statistical review?
The registry reports zero analyses. It does not give inferential results for the primary endpoint, analysis methods for the secondary outcomes, missing-data procedures, model specifications, or a prespecified multiplicity strategy. These omissions prevent a reader from checking whether reported estimates follow a stated analysis plan or how uncertainty was handled. The secondary outcome names indicate what was intended for collection, but do not establish that results were analyzed or reported.
General principles of reporting call for a clear definition of the analysis population, outcome rules, follow-up period, missing-data handling, and statistical methods. Those details are particularly relevant in surveillance, where records and follow-up can be incomplete. The registry gives some participant counts and exclusion reasons, but its population description is truncated and does not fully explain the final group. The resulting interpretation should remain descriptive and limited to the counts and percentages actually recorded.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four outcome measures: the primary safety outcome and three secondary outcomes. The secondary outcomes are Clinical Global Impression of Improvement, change from baseline in UPDRS Part III total score, and change from baseline in Modified Hoehn & Yahr Rating Scale. The registry reports zero analyses. Results for the secondary outcomes, including estimates and uncertainty, are not reported in the registry record.
Multiplicity is the issue of interpreting several statistical tests or estimates together. In general, when many outcomes are examined, a prespecified approach can clarify which findings are primary and how uncertainty from multiple testing is addressed. The registry record does not report a multiplicity strategy, a hierarchy among secondary outcomes, or a detailed statistical analysis plan. Since no analyses are reported, there are no posted tests to assess for multiplicity adjustment.
The record contains some information about exclusions: 364 patients had available case report forms, and it lists 5 with no visit since the first prescription, 12 irregularly enrolled patients, 9 excluded according to regulatory requirement, and 1 with no safety data available. It then gives 346 as the group size in an incomplete sentence; the adverse-event results also list 346 at risk. The record does not provide a complete participant-flow account explaining the transition among these counts. The rules for missing outcome data, incomplete follow-up, or inclusion in each analysis population are not reported in the registry record.
General missing-data methods depend on why information is absent and on the analysis being conducted. Without a reported method or fuller follow-up accounting, readers cannot assess how missing information may affect the safety counts or the unreported effectiveness outcomes. The registry identifies some exclusions but does not establish that all missingness or exclusions were handled under a prespecified statistical approach.
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 |
|---|---|---|---|
| Pramipexole | 29 | 346 | 8.4% |
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 |
|---|---|
| Pramipexole | 106 of 346 |
Most frequent serious adverse event terms
| Event term | Organ system | Pramipexole |
|---|---|---|
| Cerebral infarction | Nervous system disorders | 3 of 346 |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | 3 of 346 |
| Lung neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 346 |
| Neuroleptic malignant syndrome | Nervous system disorders | 2 of 346 |
| Pneumonia | Infections and infestations | 1 of 346 |
| Septic shock | Infections and infestations | 1 of 346 |
| Colon cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 346 |
| Metastases to lung | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 346 |
| Large intestine carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 346 |
| Iron deficiency anaemia | Blood and lymphatic system disorders | 1 of 346 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Pramipexole |
|---|---|---|
| Somnolence | Nervous system disorders | 59 of 346 |
| Hallucination | Psychiatric disorders | 28 of 346 |
| Constipation | Gastrointestinal disorders | 25 of 346 |
| Nausea | Gastrointestinal disorders | 20 of 346 |
Serious events with the highest arm-level rate: cerebral infarction (0.9%), pneumonia aspiration (0.9%), lung neoplasm malignant (0.6%), neuroleptic malignant syndrome (0.6%), pneumonia (0.3%).
The adverse-event results list 346 participants at risk in the pramipexole group. Of these, 29 are listed as affected by serious adverse events and 106 by other adverse events. These are participant counts in the registry record, not a comparison between treatment groups. The record does not report a like-for-like control group, and it does not provide a complete set of percentages for all primary safety categories.
The listed serious-event terms and percentages are cerebral infarction, 0.9%; pneumonia aspiration, 0.9%; lung neoplasm malignant, 0.6%; neuroleptic malignant syndrome, 0.6%; pneumonia, 0.3%; and septic shock, 0.3%. These are term-specific figures. The registry does not report whether the terms are exhaustive, whether categories can overlap, or the method used to select terms for listing. They should not be combined into an overall percentage.
In general, serious adverse events are a safety category distinct from an assessment of whether treatment caused an event. The record does not provide event-level causal assessments or a comparator-based estimate. The posted counts therefore describe events recorded in the surveillance group; they do not establish how often such events would occur under another treatment or without treatment. Confidence intervals and detailed event-collection rules are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The posted results establish that the registry records a pramipexole group of 346 participants at risk for adverse events, with 29 affected by serious adverse events and 106 by other adverse events. Several serious-event terms are accompanied by percentages. These figures provide a descriptive account of safety events recorded in the surveillance group. They do not, on their own, estimate a treatment effect or show whether treatment caused any particular event.
The survey’s stated purpose included collecting effectiveness information, and the registry names three secondary measures: Clinical Global Impression of Improvement, change from baseline in UPDRS Part III total score, and change from baseline in Modified Hoehn & Yahr Rating Scale. But the registry reports zero analyses, and results for those measures are not reported in the registry record. The posted information therefore cannot establish what changes, if any, were observed on those measures or how precisely they were estimated.
There is no reported comparator group and no randomized allocation. This means the data do not provide a like-for-like contrast that would estimate what might have happened under a different treatment strategy. Even a well-recorded event count in a single group cannot separate treatment-related effects from other causes. General statistical interpretation of uncontrolled surveillance is descriptive rather than causal.
The participant accounting also limits interpretation. The record reports enrollment of 416, 364 patients with available case report forms, and 346 at risk in the adverse-event results. The listed exclusions and incomplete description do not provide a fully traceable account of how the safety group was formed. Follow-up completeness and missing-data procedures are not reported.
The meaning is therefore specific and bounded: the record gives counts and selected term percentages for a surveillance group receiving pramipexole without concomitant L-Dopa, but it does not provide comparative safety estimates, complete endpoint proportions, uncertainty intervals, or analyzed effectiveness results. These limitations prevent stronger claims about relative risks, causes, or treatment effects. The data remain useful as a description of what the registry records for this group, provided that distinction is maintained.
13. Why This Trial Matters Statistically
This survey is statistically notable because it asks a safety question in routine clinical settings rather than reporting a randomized treatment comparison. Post-marketing surveillance can describe events recorded among people receiving a treatment in practice. Such descriptions complement controlled comparisons by addressing a different question: what events were recorded in the monitored group. Their interpretation depends on clear population definitions, follow-up, and event-collection rules.
The record makes the distinction between surveillance and comparison especially important. It names one pramipexole group, does not state an allocation method, and explicitly warns that the arms are not a like-for-like control comparison. Statistical estimates from this record therefore should not be read as treatment contrasts. The safety counts describe the group, while causal attribution and comparative effects remain beyond what the posted results establish.
It also illustrates why participant accounting matters. Enrollment is 416; the narrative refers to 364 case report forms and lists exclusions; the adverse-event results use 346 participants at risk. The registry’s incomplete population description does not fully explain that flow. Clear accounting is necessary to understand the population behind an estimate and to judge how exclusions and incomplete data may affect interpretation.
Finally, the record distinguishes intended outcomes from reported analyses. It names safety and effectiveness measures, but reports zero analyses. A list of outcome measures is not itself evidence of an outcome result. For statistical readers, this distinction helps prevent an intended measurement from being mistaken for a finding. The record’s value lies in the safety counts and the transparent recognition of its non-comparative design, while its missing analysis details constrain precision and inference.
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 primary outcome description is incomplete: it ends after stating that there were 346 patients in a further group. Although the adverse-event results also list 346 at risk, the record does not provide a complete narrative explaining the final analysis population. It reports enrollment of 416 and says that 364 patients had available case report forms, followed by listed exclusions, but does not give a fully reconstructable participant flow. This makes the relationship between enrollment, forms, exclusions, and safety results difficult to audit.
The primary endpoint names adverse events, adverse drug reactions, and serious adverse events, but the posted counts distinguish serious and other adverse events. The record does not provide a complete count or proportion for every named category, define how repeat or overlapping events were counted, or explain the displayed serious-event terms. It also does not report confidence intervals or other measures of uncertainty, so the precision of the counts and term percentages cannot be assessed from the registry results.
The design is non-randomized and lacks a reported like-for-like control group. Treatment dose, treatment changes, detailed follow-up procedures, and event-ascertainment rules are not reported in the registry record. The record does not describe whether participants contributed equal follow-up across the 36-month timeframe or how incomplete observation was handled. These omissions limit both reproducibility and interpretation.
Effectiveness outcomes are named but not analyzed in the registry record. Missing-data procedures, analysis populations for those outcomes, and multiplicity methods are also not reported. The record does not report a detailed statistical analysis plan, protocol sample-size justification, or interim analyses. Together, these gaps restrict the available evidence to the posted descriptive safety figures and prevent assessment of a broader statistical analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Special Survey on Parkinson's Disease (PD) Patients Without Concomitant Use of L-Dopa — NCT00613301.
- Registry record last updated 2014-06-06; status last verified 2014-05.
- Results first posted 2010-11-23.
- 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 a completed post-marketing surveillance survey of pramipexole use without concomitant L-Dopa in Parkinson’s disease patients in daily clinical settings in Japan. It lists Boehringer Ingelheim as sponsor, enrollment of 416, a start date of 2004-02, and primary completion of 2009-09.
The primary outcome concerns the proportion of participants with adverse events, adverse drug reactions, and serious adverse events during 36 months. The adverse-event results list 346 at risk, with 29 affected by serious adverse events and 106 by other adverse events. Selected serious-event terms are reported with percentages. The registry names three secondary effectiveness measures but reports zero analyses.
No comparator group, randomized allocation, complete analysis plan, confidence intervals, or full account of participant flow is reported in the registry record. The available results are descriptive safety counts and selected term percentages; comparative and effectiveness conclusions cannot be drawn from the posted analyses.