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 Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism (NCT01990560), a na phase 4 study. The primary outcome is A1C Level.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01990560 |
| Phase | Phase 4 |
| Condition | Mild Hypercortisolism |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Icahn School of Medicine at Mount Sinai |
| Results posted | Yes |
2. Clinical Question
Population
Patients with mild hypercortisolism. The registry record lists enrollment of 8; it does not report how many participants contributed data to each outcome analysis.
Intervention
Mifepristone, starting at 300mg once daily, with dose increases of no more than 300mg once monthly and a maximum of 1200mg daily as indicated by symptom response.
Comparator
There is no concurrent control group listed. The record describes a single-group study and reports allocation as NA, so it is not a randomized comparison.
Primary question
Whether HbA1c, a measure of glycated hemoglobin, changes from baseline during mifepristone treatment. The primary outcome timeframe is baseline, 3 months, and 6 months.
The study’s stated purpose was to assess mifepristone for hyperglycemia associated with mild hypercortisolism. Its summary also describes planned assessment of metabolic syndrome features, including waist circumference, lipid profile, body mass index, blood pressure, and insulin resistance measured by HOMA-IR. It describes quality of life, depression, and anxiety as additional areas of interest.
Those aims frame the clinical question, but they are not themselves evidence of an observed change. The registry record does not report the HbA1c values or change estimates, nor does it report numerical results for the other listed measures. It therefore cannot establish from the posted results whether any measure changed, how large a change was, or how precisely it was estimated. The single-group design also means that a change over time, if reported, would not by itself separate a treatment-associated change from other explanations for change.
3. Trial Design
The registry describes a Phase 4, single-group study of mifepristone in patients with mild hypercortisolism. Enrollment is listed as 8. Allocation is recorded as NA, and masking is listed as none. The study status is completed. The recorded start is 2013-11 and the primary completion date is 2016-09.
All participants are assigned to the listed mifepristone intervention in the study description; there is no concurrent comparator arm. The regimen begins with 300mg tablets taken once daily. The dose could increase by no more than 300mg once monthly, up to a maximum of 1200mg daily, as indicated by symptom response. The registry record does not report how many participants received each dose, how long each participant remained on treatment, or their adherence.
The primary outcome is A1C level, described as change in hyperglycemia assessed by HbA1c, also known as glycated hemoglobin. Its listed timeframe is baseline, 3 months, and 6 months. The outcome is continuous, with the mean and standard deviation specified as the summary measures. The registry also lists secondary outcomes concerning waist circumference, body mass index, fasting lipid profile, weight, CushingQoL, Nottingham Health Profile, Hospital Anxiety and Depression Scale, and quality of life.
A single-group design can describe measurements over time within the enrolled group, but it does not provide a randomized between-group contrast. Without a control group, observed changes cannot isolate the effect of mifepristone from changes related to time, co-interventions, measurement conditions, or other factors. The registry record does not report a protocol-level statistical plan or a sample-size justification. Any planning illustration based on a hypothetical two-arm comparison should therefore be distinguished from the actual single-group design.
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 | Mifepristone |
|---|---|
| STARTED | 8 |
| COMPLETED | 7 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Mifepristone |
|---|---|
| Withdrawal by Subject | 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 | Mifepristone |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 65.5 (SD 10.6) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 5 |
6. Primary Endpoint
The registry lists the primary outcome as A1C Level, measured over Baseline, 3 months, and 6 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | percentage of red blood cells |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is A1C level, expressed as a percentage of red blood cells. The registry describes it as HbA1c, or glycated hemoglobin, and says it is used to assess change in hyperglycemia. Measurements are scheduled at baseline, 3 months, and 6 months. The endpoint is continuous, and the registry specifies the mean and standard deviation as its summary form.
In general, HbA1c reflects glucose exposure over time rather than a single momentary glucose reading. That makes it useful for assessing longer-term glycemic status in clinical research. A change from baseline can describe whether the group’s measured level shifted during follow-up. Interpreting such a change requires the actual measurements or a change estimate, along with information about variability and the number of participants contributing data.
The registry record does not report mean HbA1c values at the listed timepoints, a change estimate, a confidence interval, or a test result. It therefore does not show the direction, size, or precision of any primary-endpoint change. It also does not report a specific definition of the primary analysis population or how missing HbA1c measurements were handled. Those details matter because a mean based on available measurements may represent a different set of participants at different timepoints.
Even if a within-group change estimate were available, it would describe change over time in this group rather than a controlled treatment effect. In general, comparison with a concurrent control group helps distinguish treatment-associated change from other influences. No such comparator is listed for this study.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record supports a description of study design, intended outcomes, and selected safety counts, but it does not report primary or secondary outcome estimates. In particular, the primary HbA1c endpoint is identified, yet the registry does not report its values at baseline, 3 months, or 6 months, a change from baseline, a measure of uncertainty, or an inferential test. No numerical result for the listed metabolic, quality-of-life, depression, or anxiety outcomes is reported either. The study’s stated hypotheses should not be mistaken for findings.
With no reported endpoint estimates, the direction and magnitude of any observed change are unknown from the registry record. Its precision is also unknown: there is no confidence interval or standard error to indicate how compatible the data are with different values of the underlying change. A confidence interval is a general statistical tool for communicating uncertainty around an estimate; it cannot be interpreted when the estimate and interval are not reported.
The design further constrains interpretation. The study is listed as single-group, with allocation NA and no masking. There is no listed randomized comparator. In general, a before-and-after observation can show that a measurement changed between assessments, but without a concurrent control it cannot establish that the intervention caused the change. Factors other than treatment may change over time, and an unmasked study can also be more vulnerable to differences in measurement or reporting. These are design considerations, not evidence that any particular bias occurred in this study.
The enrollment of 8 is a study-level registry figure, not a reported endpoint analysis denominator. The record does not state how many participants had measurements available at each timepoint or whether any participants were excluded from analyses. It would be inappropriate to assume that all enrolled participants contributed to every outcome.
The safety record lists 8 at risk for serious adverse events, 0 affected by serious adverse events, 8 at risk for other adverse events, 2 affected by other adverse events, and 0 deaths. These are descriptive counts for the recorded group. They do not provide detailed event terms or timing in the information recorded here, and the record lists no top serious adverse event terms. The counts do not substitute for the missing efficacy outcomes, nor do they establish a comparative safety profile.
Overall, the posted information is insufficient to quantify changes in HbA1c or the other intended outcomes, to judge their statistical precision, or to attribute any change to mifepristone. The appropriate conclusion is limited to what the record documents: a completed single-group study with enrollment of 8, a stated measurement schedule, and the listed safety counts, but no posted numerical outcome analyses.
9. Statistical Methods Explained
The registry identifies HbA1c as a continuous primary outcome and specifies a mean with standard deviation. It does not report an analysis result for that endpoint. Understanding what those summaries would mean helps distinguish a description of the planned measurement from evidence about an observed effect.
What does a mean describe?
In general, the mean is the arithmetic average of the observed measurements in the analysis group. For a continuous endpoint such as HbA1c, it provides a compact description of the group’s level at a particular assessment. A mean at follow-up can be compared descriptively with a baseline mean, but a difference between group averages is not automatically a treatment effect. In a single-group study, changes may also reflect time trends, changes in care, participant circumstances, or measurement variation.
The standard deviation describes how dispersed individual measurements are around the mean. It is not the same as the uncertainty in the estimated mean. In general, the standard error and a confidence interval reflect uncertainty in an estimate, and their calculation depends on the analysis and the available observations. The registry specifies mean and standard deviation as descriptive summaries, but it does not post those values or a confidence interval for the primary outcome.
Why does the design matter for inference?
Randomization to a concurrent control group can support a comparison that balances, on average, factors that might influence outcomes. This study is listed as single-group, with no comparator. A within-group change, if it had been reported, would answer a descriptive question about measurements over time in the group. It would not directly answer how those measurements would have changed under an alternative treatment or no study intervention.
Masking is listed as none. In general, masking can reduce the influence of knowledge about treatment assignment on certain aspects of care, outcome assessment, or reporting. Its relevance depends on the endpoint and measurement process. The registry record does not detail how HbA1c was measured or whether laboratory assessment was separated from other study procedures, so further claims about possible measurement bias are not warranted.
What would uncertainty measures add?
A point estimate gives one best-fitting summary under a particular analysis, while a confidence interval conveys uncertainty around it. A narrow interval generally indicates more precision than a wide one, although the interpretation depends on the design and assumptions. A p-value, when reported, addresses compatibility between observed data and a specified null hypothesis under an analysis model; it is not the probability that a hypothesis is true, and it does not measure clinical importance. The registry record reports no primary-endpoint estimate, confidence interval, or p-value, so none can be interpreted.
For repeated measurements, an analysis may compare each follow-up value with baseline or model the measurements jointly. General approaches can account for within-person correlation, but the appropriate model depends on the protocol and data structure. The statistical analysis plan, covariance structure, and method for estimating change are not reported in the registry record. No particular approach should be assumed.
How should planning illustrations be read?
In general, a sample-size calculation estimates how many observations a future study might need under chosen assumptions about the target effect, variability, significance level, power, and design. Such calculations are planning tools, not evidence that a study had a particular probability of detecting its observed result. The registry’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with effects expressed as fractions of an observed effect. It also distinguishes total events for time-to-event calculations from participants per arm for other calculations.
Those stated assumptions describe a planning illustration, not the recorded design of this study: the study is single-group and has no concurrent control. A hypothetical equal-allocation, two-arm calculation does not turn a single-group study into a randomized comparison. It should not be described as observed power, and it cannot establish the strength or reliability of the study’s findings. No protocol justification for the trial’s enrollment is reported in the registry record.
What can be concluded from the posted analyses?
The record lists zero analyses. Thus, the available information identifies the endpoint and design but does not provide numerical outcome analyses to interpret. The stated study aims, outcome names, and measurement schedule are not substitutes for estimates. Without results and analysis details, readers cannot assess the size or precision of primary-endpoint change, compare outcomes across time, or evaluate a statistical test. General statistical principles explain how those results might be interpreted if reported; they do not supply missing findings.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome and multiple secondary outcomes, including anthropometric measures, fasting lipids, weight, several quality-of-life instruments, and anxiety and depression assessments. Considering many outcomes creates a multiplicity issue in general: if each is tested separately, the chance of at least one apparently notable result can increase. Researchers may address this by prespecifying a hierarchy, adjusting significance thresholds, or treating secondary analyses as exploratory. The registry record does not report a multiplicity strategy, outcome hierarchy beyond identifying the primary endpoint, or a statistical analysis plan. No claim about adjustment or confirmatory status can therefore be made.
The record lists zero analyses and does not report numerical results for the primary or secondary outcomes. It also does not report missing-data counts, reasons for missingness, imputation methods, or sensitivity analyses. Those omissions prevent assessment of whether available measurements differed from missing ones and whether the handling of incomplete follow-up could affect estimates.
The enrollment figure is 8, but the number contributing to each endpoint and timepoint is not reported in the registry record. Enrollment should not be treated as the denominator for every analysis. In general, complete-case analysis uses participants with the required measurements, while other approaches may use additional information under assumptions about missingness. The selected population, analysis set, and missing-data assumptions can affect both estimates and uncertainty. Without those details or results, the registry record does not support conclusions about the robustness of any outcome finding.
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 |
|---|---|---|---|
| Mifepristone | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Mifepristone | 0 of 8 |
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 |
|---|---|
| Mifepristone | 2 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Mifepristone |
|---|---|---|
| Drug Rash | Skin and subcutaneous tissue disorders | 1 of 8 |
| Hypokalemia | Metabolism and nutrition disorders | 1 of 8 |
The registry’s adverse-event record lists the mifepristone group with 8 at risk for serious adverse events and 0 affected. For other adverse events, it lists 8 at risk and 2 affected. Deaths are listed as 0. These are counts, not rates calculated for a particular duration of exposure, and no additional rate or percentage should be inferred from them.
The serious adverse-event count describes what is recorded for this group; it is not a comparison with another treatment or an untreated group. The record lists no top serious adverse event terms. It also does not provide event timing, severity details beyond the serious-versus-other categories, exposure duration, or a detailed account of how events were collected. The listed other-adverse-event count does not identify the specific events in the information recorded.
In general, safety counts are interpreted alongside the number at risk, follow-up duration, event definitions, and completeness of reporting. Counts from a single group cannot establish whether an event frequency is higher or lower than it would have been under another condition. The recorded figures should therefore be read as descriptive registry information, not as a comparative safety estimate or a complete account of all possible harms.
12. What the Result Does, and Does Not, Mean
The study record documents an investigation of mifepristone in patients with mild hypercortisolism, with HbA1c change as the primary outcome and measurements scheduled at baseline, 3 months, and 6 months. It also describes interest in metabolic and quality-of-life outcomes. That information clarifies what the study set out to assess, but it does not reveal what happened to those outcomes.
The registry record does not report the primary HbA1c results or numerical results for the secondary outcomes. It therefore does not establish whether HbA1c or any other measure increased, decreased, or remained stable. It also provides no estimate of the size or precision of a change. The stated hypotheses are research questions, not evidence that the anticipated changes occurred.
The study’s single-group design is important to what any reported change could mean. In general, measurements before and after treatment can describe change over time, but without a concurrent control they cannot isolate the intervention’s contribution from other influences. This limitation applies even when a change is measured precisely; precision does not by itself address causal attribution.
The safety counts describe serious adverse events, other adverse events, and deaths in the recorded group, but they do not provide a comparative assessment or a complete event narrative. The enrollment figure of 8 also does not tell how many participants posted data at each assessment. The registry record does not report the analysis populations, missing-data handling, or detailed statistical methods needed to evaluate those issues.
Accordingly, the record supports a cautious description of the study’s design, planned outcomes, and recorded safety counts, but not a conclusion about the size, reliability, or cause of outcome changes. It does not establish that mifepristone did or did not alter HbA1c, metabolic measures, or quality of life. Those conclusions would require outcome estimates and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable as a single-group investigation of a continuous clinical outcome measured at baseline and follow-up. That structure highlights the distinction between describing change and estimating a comparative treatment effect. A change over time can be informative as a record of what occurred within the group, but a control group is generally needed to strengthen attribution of that change to an intervention.
The registry identifies a range of intended outcomes beyond HbA1c, covering metabolic measures and patient-reported domains. Such breadth can be useful for mapping questions a study intends to address, while also making prespecification and multiplicity management important. The registry record does not report the analysis plan or a multiplicity strategy, so it is not possible to tell how these outcomes would be prioritized or interpreted together.
The record also illustrates why outcome definitions and results must be kept separate. It states the primary measure, unit, summary form, and assessment schedule, but does not report the associated estimates. A clear endpoint description makes the question understandable; it does not answer the question without observations and statistical results.
Finally, the planning description uses a two-arm equal-allocation framework, whereas the recorded study design is single-group. This distinction matters because planning assumptions must match the design and analysis they are intended to support. A planning illustration is not a retrospective measure of evidential strength and cannot substitute for a control group or an outcome analysis.
For biostatistical interpretation, the central lesson is to separate study aims, design features, planning assumptions, safety counts, and outcome evidence. The record contains the first four in varying detail, but it does not report the numerical efficacy analyses needed to quantify change or its uncertainty.
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 limitations are specific to the information recorded for this study. Enrollment is listed as 8, and the study has a single-group design without a concurrent comparator. Consequently, the registry describes no randomized between-group contrast. Any within-group pattern, if results were available, would have limited ability to distinguish intervention-associated change from other changes over time.
The primary endpoint is defined as HbA1c at baseline, 3 months, and 6 months, but the registry record does not report outcome values, change estimates, confidence intervals, or p-values. Numerical results for the secondary outcomes are also not reported. This prevents assessment of direction, magnitude, precision, or consistency across measures.
The record does not report the number of participants analyzed at each timepoint, missing-data methods, analysis populations, adherence, dose received, or a detailed statistical analysis plan. The dosing description allows increases based on symptom response, but the record does not say how many participants received each dose or how treatment exposure varied. No assumptions about those details are warranted.
Masking is listed as none, and allocation is listed as NA. These design features should be considered when interpreting any subjective or assessment-dependent outcomes, while recognizing that the registry does not describe the measurement procedures in enough detail to determine whether a specific bias occurred. The record also does not report a multiplicity approach for the several listed outcomes.
The safety information is limited to group-level counts and does not include event terms for the other adverse events, timing, or exposure-adjusted summaries. These gaps constrain interpretation of both benefit-related outcomes and safety. They are limitations of what is reported in the registry record and should not be treated as evidence that unreported outcomes or methods had a particular value.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism — NCT01990560.
- Registry record last updated 2018-03-02; status last verified 2018-01.
- Results first posted 2018-03-02.
- 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 describes a completed Phase 4, single-group study of mifepristone in patients with mild hypercortisolism. Enrollment is listed as 8. The intervention began at 300mg once daily, with increases of no more than 300mg once monthly up to 1200mg daily as indicated by symptom response. The primary outcome was HbA1c, assessed at baseline, 3 months, and 6 months.
Secondary outcomes include measures of body size, fasting lipids, weight, quality of life, anxiety, and depression. The record reports no numerical outcome analyses; it does not provide primary or secondary estimates or their precision. It lists 0 serious adverse events among 8 at risk, 2 other adverse events among 8 at risk, and 0 deaths.
The single-group design has no concurrent comparator. The registry record does not report the statistical analysis plan, analysis populations, or missing-data methods. Its documented information therefore describes the study’s design and selected safety counts, but does not establish the size, precision, or cause of any change in the intended clinical outcomes.