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 Conivaptan for the Reduction of Cerebral Edema in Intracerebral Hemorrhage- A Safety and Tolerability Study (NCT03000283), a na phase 1 study. The primary outcome is Patient Tolerance of Conivaptan.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03000283 |
| Phase | Phase 1 |
| Condition | Cerebral Hemorrhage, Cerebral Edema, Intracerebral Hemorrhage |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Jesse Corry |
| Results posted | Yes |
2. Clinical Question
Population
Patients with cerebral edema from intracerebral hemorrhage and pressure were the intended population. The registry lists cerebral hemorrhage, cerebral edema, intracerebral hemorrhage, and stroke among the conditions. The enrolled group was seven participants; further participant characteristics are not reported in the registry record.
Intervention
The intervention was conivaptan, described in the study summary as frequent and early use at 40mg/day. The record’s stated focus was safety and tolerability, with a further preliminary goal of estimating whether cerebral edema could be reduced.
Comparator
There was no comparator arm. The study is listed as a single-group design, and the registry identifies the conivaptan treatment group as the only arm. It was not a randomized comparison, so the record does not provide a concurrent control group for estimating a treatment contrast.
Primary question
The primary outcome concerns the number of participants with abnormal seizure activity, abnormal laboratory values, an increase in infection rate, or any drug-related adverse events from baseline to 168 hours post-enrollment. This is a tolerability question, not a direct test of comparative efficacy. Although the study summary also describes an exploratory cerebral-edema goal, the primary endpoint is framed around safety-related events.
3. Trial Design
The registry describes a phase 1, single-group study of conivaptan for cerebral edema associated with intracerebral hemorrhage. The listed enrollment is 7, and the arm description says all seven patients in the conivaptan treatment group would receive conivaptan. Allocation is listed as not applicable, and masking is listed as none. With no comparison arm, outcomes describe the treated group rather than a randomized treatment contrast.
The study title identifies safety and tolerability as its focus. The brief summary says the study aimed to preliminarily assess whether frequent and early conivaptan use at 40mg/day was feasible, safe, and well-tolerated. It also describes a preliminary aim of estimating whether that dose could reduce cerebral edema. The primary endpoint is patient tolerance, while listed secondary outcomes include in-hospital mortality, change in cerebral edema, cost, and Modified Rankin Scale score. Cost appears twice in the registry’s list.
The study is marked completed. Its listed start date is 2017-03-22, and its primary completion date is 2019-02-12. These dates describe the registry record; they do not establish when each participant was enrolled or how long each participant was followed. The primary outcome timeframe is baseline to 168 hours post-enrollment.
The registry does not report a randomized control condition or masking procedures, and the design fields indicate a single group without masking. Details about eligibility criteria, recruitment, treatment administration beyond the summary’s dose statement, and the statistical analysis plan are not reported in the registry record. In a study of this design, results can describe observed events in the enrolled group, but cannot by themselves separate treatment effects from the course of illness, concurrent care, or other influences.
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 | Conivaptan Treatment Group |
|---|---|
| STARTED | 7 |
| COMPLETED | 7 |
| 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 | Conivaptan Treatment Group |
|---|---|---|
| Participants analysed | 7 | |
| Age, Continuous | years | 58.5 (36 to 69) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 6 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 7 |
| 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 | 2 |
| Race (NIH/OMB): White | Participants | 5 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Patient Tolerance of Conivaptan, measured over Baseline to 168 hours post-enrollment.
| 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 any of the specified tolerance events: abnormal seizure activity, abnormal laboratory values, an increase in infection rate, or a drug-related adverse event. Its timeframe is baseline to 168 hours post-enrollment. The registry labels the outcome unit as participants and defines the measure as a count.
A composite endpoint combines multiple event types under one outcome. In general, this can make an endpoint responsive to a range of safety concerns, which may be useful when a study is focused on early tolerability. However, the categories can differ in seriousness, frequency, and clinical meaning. A single overall count would not show which component occurred or whether events differed in severity. Interpretation therefore depends on component-level information as well as the composite definition.
The endpoint is appropriate to the stated safety-and-tolerability aim in the sense that it tracks the specified adverse changes during the stated follow-up window. It is not itself a direct measure of cerebral edema reduction. The registry record does not report a result for this primary endpoint, such as the number of participants meeting its definition. It also does not report how each component was operationalized, how an increase in infection rate was determined, or how drug relationship was assessed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a small, single-group study whose primary aim was safety and tolerability. Its listed enrollment is 7, and no comparator arm is reported. The record therefore supports a descriptive reading of events observed in the conivaptan group, not an estimate of how outcomes would differ from an untreated or differently treated group. Without a concurrent comparator, the observed course cannot be attributed uniquely to conivaptan.
The primary endpoint is defined as the number of participants with any of several tolerance events during baseline to 168 hours post-enrollment. Yet a primary-endpoint result is not reported in the registry record. The adverse-event information is not interchangeable with that endpoint: serious adverse events and other adverse events are presented separately, while the primary endpoint also includes specified seizure, laboratory, and infection findings. The record does not establish which serious events, if any, met the primary endpoint definition.
The adverse-event data list 7 participants at risk for serious adverse events and 7 affected, with 0 deaths. For other adverse events, the record lists 7 at risk and 0 affected. It also lists serious-event terms with percentages, including Catheterization, Central Venous (PICC) at 100.0%, Tracheostomy at 42.9%, Pulmonary Edema at 28.6%, Gastostomy (PEG) at 28.6%, Deep Vein Thrombosis at 28.6%, and Intracranial Hemorrhage Expansion at 14.3%. These are descriptions in the registry, not evidence that the events were caused by conivaptan. The record does not report event-level timing, severity grading beyond the serious-event classification, or causal assessments.
The study summary identifies reduction in cerebral edema as a further preliminary objective, and change in cerebral edema is listed as a secondary outcome. A corresponding numerical result is not reported in the registry record. Thus, the stated exploratory goal should not be mistaken for evidence of a measured effect. The registry’s list of outcomes and adverse events provides context about what was monitored or recorded, but it does not supply a comparative estimate of efficacy.
Precision is also a central concern. In general, estimates based on a small group can vary substantially with the outcomes of individual participants. A confidence interval, when reported, expresses uncertainty around an estimate; no confidence interval for the primary endpoint is reported in the registry record. There is consequently no registry-based interval from which to judge the precision of that endpoint estimate. The study’s design and the missing primary result limit conclusions more fundamentally than an uncertainty interval alone would.
The posted planning illustrations, if consulted, should be read as hypothetical design calculations based on an observed effect and stated assumptions, not as observed trial findings or evidence of achieved power. The registry record does not report an analysis count beyond listing zero analyses. It does not report the primary endpoint result, an inferential comparison, or a statistical plan that would support stronger claims.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study describes outcomes among people who receive the study intervention. It can document observed events and generate preliminary estimates, but it does not provide a randomized comparison between treatment conditions. This study is listed as single-group, with allocation not applicable and masking none. Consequently, any change in a participant’s condition over time could reflect the underlying illness, concurrent care, measurement variation, the intervention, or several influences together. A before-and-after observation does not isolate the intervention’s effect in the way a randomized concurrent comparison is intended to do.
What does the primary measure count?
The registry defines the primary measure as a count of participants who experience at least one of a set of tolerance events during baseline to 168 hours post-enrollment. In general, participant-level composite outcomes count a participant once under the overall definition even if more than one component occurs, unless a different counting rule is specified. The registry record does not report the detailed counting rules or a component-level breakdown for this endpoint. It also does not report the outcome value itself. It is therefore not possible to reconstruct how many participants met the complete primary definition from the listed adverse-event summaries.
How should a confidence interval be understood?
In general, a confidence interval describes the statistical uncertainty around an estimated quantity under a specified method and its assumptions. A narrow interval indicates greater precision than a wide interval; neither interval width nor statistical significance alone establishes clinical importance or causation. For event counts or proportions, interval methods may be selected to account for the nature of the data, particularly when the group is small or events are uncommon. No confidence interval for the primary endpoint is reported in the registry record, so its precision cannot be evaluated from that outcome.
Why does the lack of a comparator matter?
In general, random assignment to concurrent groups helps balance measured and unmeasured factors on average, supporting a more credible estimate of a treatment contrast. This registry record describes no randomized comparison and no control arm. The observed serious-event counts therefore describe the group’s experience; they do not show what would have occurred under another treatment or without conivaptan. Confounding by illness severity, other interventions, and changes over time cannot be ruled out through a between-group comparison in this design.
What does over-dispersion mean in general?
For count outcomes, some statistical models assume that variability follows a particular relationship to the average count. Over-dispersion means the observed variability exceeds that assumed by the model. If present and ignored, it can make standard errors too small and confidence intervals too narrow, overstating precision. This is a general statistical issue, not a reported feature of this study. The registry record does not report a count model, an over-dispersion assessment, or a statistical analysis plan, so no claim about the handling of this issue is supported.
How should planning calculations be read?
The record describes planning illustrations computed from an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not a description of the trial’s observed design, which is single-group. In general, sample-size planning calculations estimate the number of participants needed under specified assumptions to target a chosen level of power for a hypothetical future comparison. They do not measure the power achieved by a completed study, and they are not observed outcomes. The record does not report the trial’s own sample-size justification. Any sample-size illustrations should therefore be kept separate from interpretation of this study’s enrolled group and results.
What can be concluded from the registry’s analysis fields?
The record lists zero analyses. This field does not establish which analyses may have been performed outside the registry or whether results exist elsewhere; those details are not reported in the registry record. The statistical plan, handling of missing data, analysis population definitions, and multiplicity procedures are also not reported in the registry record. Without these details and a reported primary-endpoint estimate, readers cannot assess whether analyses followed a prespecified approach or how sensitive conclusions might be to alternative methods.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists six outcome measures and names several secondary outcomes, including in-hospital mortality, change in cerebral edema, cost, and Modified Rankin Scale score. Cost appears twice in the listed outcomes. The primary endpoint is a composite tolerance measure. When a study assesses multiple outcomes or multiple components, the chance of at least one apparently notable result can increase if many statistical tests are performed. In general, multiplicity procedures can control or describe this issue, but the appropriate approach depends on the study’s objectives and analysis plan.
The registry record does not report a statistical plan, a multiplicity adjustment, a hierarchy among secondary outcomes, or an inferential analysis for these outcomes. It lists zero analyses. It is therefore not possible to determine which outcomes were intended for formal testing or how any multiplicity was handled. The exploratory cerebral-edema objective should be interpreted as an aim described in the summary, not as a confirmed finding.
Missing-data handling is not reported in the registry record. The record does not state whether all enrolled participants contributed data to each outcome, how missing measurements were treated, or whether any participants were excluded from an analysis. Analysis populations are not reported in the registry record. These omissions matter because the interpretation of outcome counts depends on which participants were assessed and how incomplete follow-up was handled. No assumptions about completeness or analysis populations can be made from the listed enrollment alone.
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 |
|---|---|---|---|
| Conivaptan Treatment Group | 7 | 7 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Conivaptan Treatment Group | 0 of 7 |
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 |
|---|---|
| Conivaptan Treatment Group | 0 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | Conivaptan Treatment Group |
|---|---|---|
| Catheterization, Central Venous (PICC) | Surgical and medical procedures | 7 of 7 |
| Tracheostomy | Surgical and medical procedures | 3 of 7 |
| Pulmonary Edema | Respiratory, thoracic and mediastinal disorders | 2 of 7 |
| Gastostomy (PEG) | Surgical and medical procedures | 2 of 7 |
| Deep Vein Thrombosis | Vascular disorders | 2 of 7 |
| Intracranial Hemorrhage Expansion | Nervous system disorders | 1 of 7 |
| Urinary Tract Infection | Infections and infestations | 1 of 7 |
| Increase in Cerebral Edema | Nervous system disorders | 1 of 7 |
Serious events with the highest arm-level rate: catheterization, central venous (picc) (100.0%), tracheostomy (42.9%), pulmonary edema (28.6%), gastostomy (peg) (28.6%), deep vein thrombosis (28.6%).
The registry lists 7 participants at risk for serious adverse events and 7 affected; it lists 0 deaths. For other adverse events, it lists 7 at risk and 0 affected. These summaries are event classifications as recorded in the registry. They should not be confused with the primary endpoint result, which is not reported in the registry record and has its own definition.
The listed serious-event terms include Catheterization, Central Venous (PICC) at 100.0%, Tracheostomy at 42.9%, Pulmonary Edema at 28.6%, Gastostomy (PEG) at 28.6%, Deep Vein Thrombosis at 28.6%, and Intracranial Hemorrhage Expansion at 14.3%. The registry does not report whether each term was considered related to the intervention. Nor does it explain whether people could contribute more than one term, so the term percentages should not be added or treated as mutually exclusive categories.
Serious adverse events may arise from the underlying condition, procedures, other care, or an intervention. In general, an adverse-event listing documents events and does not by itself establish causation. The record’s event summaries also do not provide enough detail to infer timing, duration, severity within each term, or individual clinical courses.
12. What the Result Does, and Does Not, Mean
This trial record documents a phase 1, single-group study that focused on the feasibility and tolerability of conivaptan in patients with cerebral edema from intracerebral hemorrhage and pressure. It identifies a primary safety-oriented outcome and gives a follow-up timeframe from baseline to 168 hours post-enrollment. The registry also records serious and other adverse-event summaries for the treatment group. These details describe the study’s scope and the safety information entered in the record.
They do not establish that conivaptan caused the listed events, prevented them, or changed the course of cerebral edema. A single-group study cannot provide a randomized comparison, and the registry does not report a numerical result for the primary endpoint or for the secondary cerebral-edema outcome. The study summary’s hypothesis and objectives are statements of what investigators sought to examine, not results. Similarly, the serious-event terms are not a substitute for the primary endpoint’s specifically defined set of tolerance events.
In general, early-phase safety studies can provide preliminary descriptive information and help frame questions for later research. Their estimates may be imprecise, especially when the enrolled group is small. A finding in a small group may not represent outcomes in a broader patient population, and the absence of a reported event in a particular category does not prove that the event cannot occur. The registry record does not provide confidence intervals or a comparative analysis to quantify uncertainty for the primary endpoint.
The appropriate statistical reading is therefore limited: the record describes a completed, unmasked, single-group study with listed enrollment of 7, and it reports the adverse-event summaries stated above. It does not report the primary endpoint result, a comparator-based treatment effect, or a statistical plan. The study’s exploratory objective concerning cerebral edema should remain distinct from the safety endpoint and from any claim of efficacy. The registry record supports description of the design and recorded events, but not a causal conclusion about benefit or harm.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the difference between a safety-oriented objective and an efficacy comparison. The registry’s primary outcome is defined around tolerance events, while reduction in cerebral edema is described as a further preliminary objective and listed as a secondary outcome. Those questions require different outcome interpretations. A tally of safety events does not answer whether cerebral edema changed, and an exploratory change in edema would not by itself establish a comparative treatment effect.
The single-group design also makes clear why study structure matters. Descriptive evidence can record what occurred among treated participants, but the lack of a concurrent randomized comparator leaves the counterfactual outcome unknown. Statistical analysis cannot recover a randomized contrast that the design did not create. This limits causal interpretation even if an outcome is measured consistently.
The record also illustrates why endpoint definitions, analysis plans, and reporting completeness are important. The primary endpoint definition includes several possible event types, but its result is not reported in the registry record. The registry lists zero analyses, and it does not report the handling of missing data, multiplicity, or analysis populations. Without such details, readers cannot determine how the primary result would be summarized or how secondary findings would be interpreted.
Finally, planning illustrations based on an observed effect are not trial outcomes and should not be read as achieved power. Their assumptions concern a hypothetical design, including equal allocation between two arms, whereas the registered study is single-group. Separating planning calculations from observed evidence helps prevent design scenarios from being mistaken for results. The record’s value lies in its clearly stated early-stage aims and in the opportunity to identify what information is still needed for interpretable statistical conclusions.
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 principal limitation for interpretation is the single-group design. Allocation is listed as not applicable, masking as none, and no comparator arm is described. Consequently, observed events cannot be contrasted with outcomes under another treatment condition, and causal effects cannot be isolated from the course of intracerebral hemorrhage, concurrent care, or other influences.
The listed enrollment is 7. Estimates from a group of this size may be sensitive to individual outcomes, and the registry record does not report a confidence interval for the primary endpoint. More importantly, it does not report the primary endpoint result itself. The adverse-event summaries cannot fill that gap because the primary measure includes its own specified combination of seizure, laboratory, infection, and drug-related events.
The record does not report detailed eligibility criteria, participant characteristics, component-level primary-endpoint results, event timing, or causal assessments for the serious adverse-event terms. It also does not report missing-data methods, analysis populations, a statistical analysis plan, or multiplicity procedures. The registry lists zero analyses, but further information about analyses is not reported in the registry record.
The stated cerebral-edema objective is preliminary, and a corresponding numerical result is not reported in the registry record. The registry’s outcome list includes change in cerebral edema, but the outcome name alone does not establish a measured change or its precision. These limitations mean that the record is most useful for understanding the study’s design, aims, and recorded safety summaries, rather than for estimating a treatment effect.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Conivaptan for the Reduction of Cerebral Edema in Intracerebral Hemorrhage- A Safety and Tolerability Study — NCT03000283.
- Registry record last updated 2020-04-17; status last verified 2020-04.
- Results first posted 2020-04-17.
- 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
NCT03000283 is listed as a completed phase 1 study of conivaptan for cerebral edema associated with intracerebral hemorrhage. The design is single-group, with allocation not applicable and no masking. Listed enrollment is 7, and the treatment arm description says all seven patients would receive conivaptan. The study’s primary outcome is a count of participants with specified tolerance events from baseline to 168 hours post-enrollment.
The registry lists serious adverse events affecting 7 participants, other adverse events affecting 0, and 0 deaths. It does not report a numerical result for the primary endpoint or for the secondary change-in-cerebral-edema outcome. The record also does not report a comparator-based analysis, statistical plan, missing-data methods, or analysis populations. The results are therefore descriptive and do not establish a causal treatment effect.