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 Glyburide Advantage in Malignant Edema and Stroke Pilot (NCT01268683), a na phase 1, phase 2 study. The primary outcome is Rate of Enrollment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01268683 |
| Phase | Phase 1, Phase 2 |
| Condition | Ischemic Stroke |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Remedy Pharmaceuticals, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Adults with severe anterior circulation ischemic stroke who met the study’s stated eligibility criteria. The brief summary specifies ages 18–80, a baseline diffusion weighted imaging lesion volume of 82–210 cm3, and a start of study infusion no more than 10 hours after symptom onset. Participants could have received standard-of-care intravenous rtPA or not.
Intervention
Glyburide for injection, given as a bolus followed by continuous infusion for 72 hours. The trial was open label, so masking was recorded as none.
Comparator
There was no concurrent comparator arm. The registry describes a single-group study. Its objectives refer to comparison of clinical and MRI outcomes with benchmark data from published literature, but the details of any benchmark comparison are not reported in the registry record.
Primary question
Could the study enroll, evaluate, and treat the intended severe-stroke participants, with the primary outcome defined as the number of months to enroll 10 participants? The registry record gives the outcome definition but does not report the observed enrollment time.
The study also set out to assess initial safety and tolerability and glyburide pharmacokinetics and pharmacodynamics. Clinical and MRI outcomes were secondary interests, including measures of infarcted hemisphere and diffusion weighted imaging lesion volumes, midline shift, ipsilateral ventricle volume, hemorrhagic events, and NIHSS score. Those questions are distinct from the primary feasibility question: measurements of clinical status or imaging do not, by themselves, establish whether enrollment was feasible or whether any observed change was caused by the intervention. With no concurrent control group, the trial’s clinical and imaging observations are descriptive and cannot isolate treatment effects from the course of stroke or other care.
3. Trial Design
This was a completed, prospective, multicenter, open-label Phase IIa pilot, with registry phase categories of Phase 1 and Phase 2. The study used a single-group design and recorded allocation as not applicable. Ten participants were enrolled. The intervention arm received glyburide for injection as a bolus followed by continuous infusion for 72 hours; masking was recorded as none.
The intended population was patients with severe anterior circulation ischemic stroke thought likely to experience clinically significant brain swelling. The brief summary describes eligibility criteria of age 18–80, baseline DWI lesion volume 82–210 cm3, and study infusion beginning within 10 hours of symptom onset. Receipt of standard-of-care intravenous rtPA was permitted but was not required. The registry record does not report how many participants received rtPA or provide participant-level characteristics.
The primary outcome was the number of months needed to enroll 10 participants, with a listed timeframe of Day 1. Its description is about recruitment feasibility rather than a clinical outcome. Secondary outcomes covered adverse events and serious adverse events, imaging measures, hemorrhagic events, and NIHSS score. The registry lists 20 outcome measures, but detailed results for these measures are not part of the record.
The study’s stated intention to compare clinical and MRI outcomes with literature-derived benchmark data does not create a randomized or concurrent control group. The benchmark source, comparison method, and any adjusted analyses are not reported in the registry record. Consequently, the design supports a description of recruitment and observed outcomes in the enrolled group, not a controlled estimate of the effect of glyburide.
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 | RP-1127 (Glyburide for Injection) |
|---|---|
| STARTED | 10 |
| COMPLETED | 8 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | RP-1127 (Glyburide for Injection) |
|---|---|
| Lost to Follow-up | 1 |
| Death | 1 |
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 | RP-1127 (Glyburide for Injection) |
|---|---|---|
| Participants analysed | 10 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 7 |
| Age, Categorical: >=65 years | Participants | 3 |
| Age, Continuous | years | 50.5 (25 to 73) |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 3 |
| Region of Enrollment: United States | Participants | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Rate of Enrollment, measured over Day 1.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Months |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of months to enroll 10 participants. It is a recruitment-feasibility measure: it asks how long enrollment took to reach the specified enrollment target. It does not measure brain swelling, neurological status, safety, or treatment effect. The registry lists the timeframe as Day 1 and describes the endpoint as the number of months to enroll 10 participants, but does not report the observed number of months.
Recruitment time is useful in a pilot because the ability to identify and enroll a defined clinical population is part of determining whether a larger study can be conducted. In this trial, eligibility required severe anterior circulation ischemic stroke, a specified baseline DWI lesion volume, and rapid initiation of infusion after symptom onset. The feasibility of enrolling people who meet such criteria is a different question from whether an intervention changes clinical or imaging outcomes.
As a general statistical point, a recruitment endpoint should be interpreted against a prespecified target and a clearly described observation period. The registry record does not report a recruitment target beyond the stated 10 participants, a benchmark for acceptable enrollment time, or the observed time to reach the target. It therefore does not allow the recruitment result to be quantified or judged against a success threshold. The endpoint definition is available, but its result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a pilot enrolling 10 participants in a single glyburide group. Its primary endpoint was recruitment time, defined as the number of months to enroll 10 participants. No observed value for that endpoint is reported in the registry record. This means the principal feasibility result cannot be interpreted as a measured duration or compared with any stated target. The study’s completed status does not substitute for the missing numerical result.
The registry lists safety and clinical or imaging outcomes as secondary measures. It records 3 participants at risk for serious adverse events as having such events, with 10 at risk; it also records 10 participants at risk for other adverse events as having such events. One death is recorded. These counts describe events reported for the intervention group. They do not establish that glyburide caused an event, and the absence of a concurrent comparator prevents a like-for-like estimate of background event rates. The serious-event terms listed include brain herniation, respiratory failure, cerebrovascular accident, dehydration, and hypotension, each with a registry percentage of 10.0%. These term entries should not be treated as mutually exclusive participant categories.
The registry also names MRI outcomes and NIHSS score, but the registry entries reported for this record do not include their numerical results. Although the brief summary states that the study aimed to compare clinical and MRI outcomes with literature-derived benchmarks, the benchmark data and comparison results are not reported in the registry record. No effect estimate, confidence interval, or test result for those outcomes is available in the stated results. The registry lists 20 outcome measures and 0 analyses; those counts do not supply outcome values or clarify how measurements were analyzed.
As a general statistical principle, a confidence interval describes the range of effects reasonably compatible with data under a specified model, while a p-value addresses compatibility with a null hypothesis under that model. Neither can be interpreted without an estimate, its uncertainty, and the analysis details. Those quantities are not reported for the primary endpoint or the named clinical and imaging outcomes in the record. No claim about precision or statistical significance for those outcomes is therefore supported.
The single-group, open-label design is suited to describing what happened among enrolled participants and documenting the conduct of a pilot. It cannot separate an intervention effect from the natural course of a severe stroke, other care, or differences in participant characteristics. Accordingly, the posted results support a limited descriptive reading, particularly of the recorded adverse-event counts; they do not establish a causal treatment effect or a comparative safety profile.
9. Statistical Methods Explained
The registry identifies the study as prospective, multicenter, open label, and single group. These design features determine what can be learned from the results. A prospective study follows its planned observations forward in time. An open-label study does not mask the assigned intervention from participants or study personnel; in this record, masking is listed as none. A single-group study has no concurrent control arm. These features do not invalidate descriptive observations, but they limit causal comparisons.
What a single-group result can describe
For participants who received glyburide, the record can describe recruitment, recorded adverse events, and any outcome measurements that are reported. Without a concurrent control group, however, an observed clinical or imaging outcome cannot show what would have happened to comparable participants without the intervention. In general, an uncontrolled before-and-after comparison can reflect the intervention, the underlying disease course, other care, measurement variation, or a combination of these factors. A benchmark based on published literature may provide context, but it is not automatically equivalent to a control group. The benchmark source, comparability criteria, and analysis are not reported in the registry record.
Recruitment as an endpoint
The primary endpoint concerns the number of months to enroll 10 participants. Recruitment time is a feasibility outcome, not a measure of clinical benefit. Its interpretation depends on the recorded start and end of the recruitment interval, the target, and any criterion for acceptable recruitment. The target appears in the endpoint definition, but the observed recruitment duration and any success threshold are not reported in the registry record. Thus, the endpoint’s definition can be explained, but its result cannot be reconstructed.
Outcome estimates and uncertainty
In general, an outcome estimate gives a numerical summary of the observations, while a confidence interval expresses uncertainty around that estimate under the chosen statistical model. A wider interval generally indicates less precision; a narrower interval indicates greater precision, conditional on the model and data. A p-value is not an effect size and does not describe clinical importance. For this record, numerical results and uncertainty estimates for the primary endpoint and the listed MRI and clinical outcomes are not reported in the registry record. No such statistics should be inferred from the number of measures or participants.
The registry lists 20 outcome measures and 0 analyses. These counts do not tell readers whether every measure was collected, which time points were analyzed, or whether estimates were adjusted. The analysis populations, handling of missing observations, and statistical models are not reported in the registry record. As a general point, those choices matter because exclusions, incomplete measurements, and repeated observations can affect both the estimate and its uncertainty.
Safety counts and denominators
The adverse-event entries give the number at risk and the number affected for the glyburide group. Reading a count alongside its denominator makes clear which participants contributed to that entry. Such counts are descriptive and should not be interpreted as comparative risks in the absence of a control group. A participant can experience more than one adverse event, and the listed serious-event terms should not be assumed to represent distinct people unless the registry states that they do. The record does not report event timing, duration, severity grading beyond serious status, or a causal assessment for each event.
Planning illustrations
General sample-size calculations ask how much information a future study might need to detect a chosen effect under stated assumptions. The planning assumptions associated with the illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is expressed as a stated fraction of the observed effect. For time-to-event outcomes, the calculation is described in terms of total events; other calculations are described in participants per arm. These are planning illustrations, not observed power for this single-group pilot. The trial did not have equal allocation between two arms, and its actual design had no comparator arm. The assumptions and resulting planning counts therefore should not be mistaken for the design or results of the enrolled study.
As a general statistical concern, over-dispersion occurs when outcome variability exceeds what a simple model assumes. If it is present, standard errors from an under-dispersed model may be too small, making uncertainty look less than it is. The registry record does not report a model for count outcomes or an assessment of over-dispersion, so no conclusion about that issue can be drawn for this study.
10. Multiplicity, Missing Data and Analysis Populations
The study lists a broad set of secondary outcomes, including adverse events, MRI measures, hemorrhagic events, and NIHSS score. When many outcomes or time points are examined, the chance of at least one apparently unusual result can increase. General approaches to multiplicity include defining a primary outcome in advance, specifying a hierarchy among outcomes, or adjusting inferential procedures. The multiplicity strategy for this study is not reported in the registry record. The primary outcome is identified as enrollment time, but the record does not describe inferential testing or adjustment for the secondary outcomes.
The registry lists 20 outcome measures and 0 analyses. Those entries do not establish that every planned measure was collected or analyzed, nor do they identify which observations were included in any analysis. Analysis populations, definitions of evaluable participants, and rules for excluding observations are not reported in the registry record. The record also does not describe how missing outcome data were handled, whether any measurements were incomplete, or whether sensitivity analyses were conducted.
As a general matter, missing data can affect estimates when the probability of a missing measurement is related to participant status or outcome. Complete-case summaries may describe only those with recorded measurements, while imputation or model-based approaches require explicit assumptions. The appropriate interpretation depends on the pattern and reasons for missingness, neither of which is reported in the registry record. No missing-data method can therefore be attributed to this study.
Because the trial has one treatment group and no reported analysis details for the clinical and imaging outcomes, the registry record does not support claims about confirmatory testing or a prespecified multiplicity-controlled conclusion. These gaps limit the interpretation of outcome-level evidence beyond the descriptive safety counts that are recorded.
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 |
|---|---|---|---|
| RP-1127 (Glyburide for Injection) | 3 | 10 | 30.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| RP-1127 (Glyburide for Injection) | 1 of 10 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| RP-1127 (Glyburide for Injection) | 10 of 10 |
Most frequent serious adverse event terms
| Event term | Organ system | RP-1127 (Glyburide for Injection) |
|---|---|---|
| Brain herniation | Nervous system disorders | 1 of 10 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 10 |
| Cerebrovascular accident | Nervous system disorders | 1 of 10 |
| Dehydration | Metabolism and nutrition disorders | 1 of 10 |
| Hypotension | Vascular disorders | 1 of 10 |
Most frequent non-serious adverse event terms
| Event term | Organ system | RP-1127 (Glyburide for Injection) |
|---|---|---|
| Hypomagnesemia | Metabolism and nutrition disorders | 8 of 10 |
| Hypokalaemia | Metabolism and nutrition disorders | 8 of 10 |
| Hypocalcemia | Metabolism and nutrition disorders | 6 of 10 |
| Pain | General disorders | 5 of 10 |
| Anxiety | Psychiatric disorders | 4 of 10 |
| Hypophosphatemia | Metabolism and nutrition disorders | 4 of 10 |
| Nausea | Gastrointestinal disorders | 4 of 10 |
| Urinary tract infection | Infections and infestations | 4 of 10 |
| Agitation | Nervous system disorders | 3 of 10 |
| Hyponatremia | Metabolism and nutrition disorders | 3 of 10 |
Serious events with the highest arm-level rate: brain herniation (10.0%), respiratory failure (10.0%), cerebrovascular accident (10.0%), dehydration (10.0%), hypotension (10.0%).
For the glyburide group, the record lists 10 participants at risk for serious adverse events and 3 affected. It lists 10 participants at risk for other adverse events and 10 affected, and records 1 death. The serious-event terms include brain herniation, respiratory failure, cerebrovascular accident, dehydration, and hypotension; each term is listed with a percentage of 10.0%.
These entries are descriptive counts, not evidence that the intervention caused an event. Severe stroke itself and other care may also be relevant, and this single-group study has no concurrent control group for comparison. The listed terms should not be added together or interpreted as distinct participants: an individual may have experienced more than one event, and the registry record does not specify overlap. The term percentages likewise do not provide an event rate over time or a comparison with another group.
The registry record does not report event timing, duration, severity details beyond serious status, or a causal assessment for each event. The safety counts should therefore be read as the events recorded among the enrolled group, with those limitations, rather than as comparative evidence about risk.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed, open-label, single-group pilot enrolled 10 participants and studied glyburide for injection. It defines the primary feasibility outcome as the number of months required to enroll 10 participants. It also records adverse-event counts and identifies clinical and MRI outcomes of interest. These details describe the study’s scope and the types of observations it sought to make.
The primary endpoint result itself is not reported in the registry record. Without the observed enrollment duration, the feasibility question cannot be answered quantitatively from this record. The record also does not report numerical results for the named MRI measures or NIHSS score. Although the stated objectives include comparison with literature-derived benchmarks, the benchmark data and comparison results are not reported in the registry record.
The serious and other adverse-event counts describe what was recorded in the intervention group. They do not identify whether the intervention caused an event, and, without a concurrent comparison group, they cannot establish how event occurrence compares with usual care or another treatment. The death count is likewise a descriptive observation, not a causal finding.
As a general statistical principle, a treatment effect is best interpreted using a suitable comparison, a clearly specified outcome, an estimate with its uncertainty, and transparent analysis methods. Those elements are not reported for the clinical and imaging outcomes in this record. The findings therefore should not be read as proving benefit, lack of benefit, or comparative safety. They are limited to the design, enrollment, and event information recorded for this pilot, with many outcome results and analysis details not reported in the registry record.
13. Why This Trial Matters Statistically
This trial matters statistically as an example of the role and limits of a pilot study. Its primary endpoint focuses on whether participants could be enrolled, rather than on a comparative clinical effect. Recruitment feasibility is a meaningful study question when eligibility requires severe stroke, imaging criteria, and timely treatment initiation. Yet the observed time to enroll the target is not reported in the registry record, so the central feasibility outcome remains unquantified.
The study also illustrates why design determines the strength of interpretation. A prospective, open-label, single-group study can document treatment delivery and describe outcomes among enrolled participants. It cannot, on its own, separate the intervention’s contribution from the natural course of illness or other care. This distinction is important when clinical and imaging measures are collected alongside safety observations: having multiple outcomes does not create a comparison group.
Safety reporting is also statistically informative when read with its denominator and design context. The record gives counts for serious and other adverse events among 10 participants, but a small group and the lack of a concurrent comparator constrain what those observations can establish. The event terms and death count are observations to characterize, not causal conclusions.
Finally, the registry identifies many outcome measures but lists 0 analyses and does not report the statistical analysis plan, missing-data methods, or multiplicity approach. These omissions make it difficult to assess precision and the risk of selective or chance findings. The study is therefore instructive not because the available record resolves treatment-effect questions, but because it shows how feasibility aims, descriptive safety results, comparator structure, and analysis transparency jointly shape what a clinical trial can support.
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 main limitation for interpreting the primary question is that the registry record defines enrollment time but does not report the observed number of months. It also does not state a success threshold for recruitment feasibility. Completed status alone does not convey whether enrollment met a particular operational target.
The single-group design has no concurrent control arm, and masking is recorded as none. That structure limits causal interpretation of clinical outcomes and safety observations. The stated intention to use published-literature benchmarks does not resolve this limitation because the benchmark source, participant comparability, and comparison method are not reported in the registry record.
The enrolled group consisted of 10 participants. This is a pilot-scale cohort, and the record does not provide enough outcome estimates and uncertainty measures to judge precision for clinical or imaging results. The record lists 20 outcome measures, but numerical results for the named imaging and neurological measures are not reported in the registry record described for this analysis. It also lists 0 analyses.
Analysis populations, missing-data handling, multiplicity procedures, and statistical models are not reported in the registry record. The record does not describe event timing or the causal attribution process for adverse events. It also does not report how many participants received rtPA. These omissions constrain subgroup interpretation and the assessment of how fully outcomes were observed. Together, the design and reporting limits support a descriptive reading of the recorded information, not a definitive comparative assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Glyburide Advantage in Malignant Edema and Stroke Pilot — NCT01268683.
- Registry record last updated 2024-11-22; status last verified 2024-11.
- Results first posted 2014-06-06.
- 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
NCT01268683, Glyburide Advantage in Malignant Edema and Stroke Pilot, is listed as completed. The study was a prospective, multicenter, open-label Phase IIa trial, with registry phase categories of Phase 1 and Phase 2. It used a single-group design and enrolled 10 participants with severe anterior circulation ischemic stroke. The intervention was glyburide for injection, administered as a bolus followed by continuous infusion for 72 hours.
The primary outcome was the number of months to enroll 10 participants, with a listed timeframe of Day 1. Its observed result is not reported in the registry record. The registry lists secondary safety, MRI, hemorrhagic-event, and NIHSS outcomes, 20 outcome measures, and 0 analyses. For the intervention group, 3 of 10 participants at risk were recorded with serious adverse events, 10 of 10 with other adverse events, and 1 death was recorded. These are descriptive counts from a single-group study, not comparative or causal estimates. Statistical analysis details and numerical results for the primary, clinical, and imaging outcomes are not reported in the registry record.