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 CAE Plus LAI in Individuals With Bipolar Disorder at Risk for Treatment Non-adherence (BD-CAEL) (NCT03408873), a na phase 4 study. The primary outcome is Change in Tablets Routine Questionnaire (TRQ, Past Week).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03408873 |
| Phase | Phase 4 |
| Condition | Bipolar Disorder, Medication Adherence, Medication Non Adherence |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Martha Sajatovic |
| Results posted | Yes |
2. Clinical Question
Population
Poorly adherent patients with bipolar disorder who received long-acting injectable antipsychotic medication and a customized adherence enhancement intervention. The study enrolled 30 participants described as being at risk for treatment non-adherence.
Intervention
Participants received Abilify Maintena, a long-acting injectable antipsychotic, together with Customized Adherence Enhancement (CAE), a behavioral intervention. The study considered this combined treatment approach rather than either component on its own.
Comparator
The registry record describes a single-group study and does not identify a separate comparison group. It therefore does not provide a concurrent control condition against which to compare the combined intervention.
Primary question
The primary outcome was change in the percentage of bipolar disorder medications not taken during the past week, assessed from screening to Week 24. The clinical question is whether this measure changed among participants receiving the combined approach. The record does not report the observed change or its uncertainty, so the direction and precision of any change cannot be assessed from the registry record.
3. Trial Design
This was a prospective Phase 4 study with a single-group model. The registry record lists an enrollment of 30 and describes participants with bipolar disorder at risk for treatment non-adherence. All participants in the study arm were to receive both Abilify Maintena and Customized Adherence Enhancement (CAE). The record does not describe a randomized allocation, and allocation is listed as NA. There was no masking.
The study was marked completed. Its listed start date is 2018-04-01, and its primary completion date is 2020-05-06. These dates describe the registry record; they do not establish when individual participants began or completed treatment. The record does not report the treatment schedule or the timing of all assessments beyond the primary outcome timeframe.
The primary outcome was change in Tablets Routine Questionnaire (TRQ, Past Week), expressed as the percentage of missed bipolar disorder medications, from screening to Week 24. Secondary outcomes covered psychiatric symptoms, medication attitudes, and social and occupational functioning. The record lists 15 outcome measures and 19 analyses, but does not give enough detail to determine how those analyses were organized or which were prespecified.
A single-group design can describe changes observed over time in participants receiving the study approach. Without a separate comparison group, however, the design cannot distinguish intervention-related change from changes that might occur over time for other reasons. The registry record does not report a sample-size justification or a detailed statistical analysis plan.
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.
Visit 1 Assessment (Baseline)
| Stage | CAE-L |
|---|---|
| STARTED | 30 |
| COMPLETED | 27 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | CAE-L |
|---|---|
| Lost to Follow-up | 3 |
Visit 2 Assessment
| Stage | CAE-L |
|---|---|
| STARTED | 27 |
| COMPLETED | 26 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | CAE-L |
|---|---|
| Lost to Follow-up | 1 |
Visit 3 Assessment
| Stage | CAE-L |
|---|---|
| STARTED | 26 |
| COMPLETED | 25 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | CAE-L |
|---|---|
| Withdrawal by Subject | 1 |
Visit 4 Assessment
| Stage | CAE-L |
|---|---|
| STARTED | 25 |
| COMPLETED | 23 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | CAE-L |
|---|---|
| Lost to Follow-up | 2 |
Analysis population for the primary outcome, as defined in the registry: Poorly adherent patients with bipolar disorder who received long-acting injectable antipsychotic medication and a customized adherence enhancement intervention (CAE). Descriptive statistics (means and standard deviations or counts and percentages) were generated for all outcomes measures using complete cases.
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 | CAE-L |
|---|---|---|
| Participants analysed | 30 | |
| Age, Continuous | years | 49.47 (SD 9.28) |
| Sex: Female, Male: Female | Participants | 13 |
| Sex: Female, Male: Male | Participants | 17 |
| Race/Ethnicity, Customized: White | Participants | 9 |
| Race/Ethnicity, Customized: Black or African American | Participants | 17 |
| Race/Ethnicity, Customized: Other or Multiracial | Participants | 4 |
| Race/Ethnicity, Customized: Hispanic | Participants | 1 |
| Race/Ethnicity, Customized: Non Hispanic | Participants | 29 |
| Marital Status: Single, never married | Participants | 17 |
| Marital Status: Married | Participants | 1 |
| Marital Status: Separated/Divorced/Widowed | Participants | 12 |
| Mean Years of Education Attained | years | 13.63 (SD 2.85) |
| Occupation Status: Full time employment/homemaker | Participants | 0 |
| Occupation Status: Part time employment/ homemaker or student | Participants | 4 |
| Occupation Status: Unemployed or on disability | Participants | 25 |
| Occupation Status: Other | Participants | 1 |
| Number of Participants Who Live Alone | Participants | 16 |
| Age at Bipolar onset | years | 27.63 (SD 11.80) |
| Duration of bipolar illness | years | 21.56 (SD 11.74) |
| Number of Participants With Current Substance Use Problems | Participants | 6 |
| Mean lifetime psychiatric hospitalizations | hospitalizations | 3.07 (SD 3.43) |
| Mean lifetime substance abuse hospitalizations | hospitalizations | 1.06 (SD 2.00) |
| Number of Participants With History of Physical Abuse | Participants | 16 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Tablets Routine Questionnaire (TRQ, Past Week), measured over Screen to Week 24.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | percentage of missed BD medications |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change in the Tablets Routine Questionnaire (TRQ, Past Week), measured from screening to Week 24. It records the percentage of bipolar disorder medications not taken during the past week at the time of assessment. The listed scale ranges from 0% to 100%, and higher scores indicate poorer treatment adherence.
This endpoint focuses on recent medication-taking behavior rather than relying only on a general impression of adherence. A change in the score can describe whether the reported percentage of missed medication moved over the assessment period. Because the study used a single group, such a change would describe the participants’ course during the study; it would not, by itself, show what would have happened under a different treatment or without the intervention.
The record identifies the outcome and timeframe, but does not report the observed mean at either assessment, the mean change, its standard deviation, or an interval estimating uncertainty. The registry record therefore does not establish the magnitude or direction of the observed change. It also does not provide enough information to assess the completeness of TRQ measurements at the assessment points.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the study as completed and describes the intended primary outcome, but the outcome values needed to interpret the result are not reported in the registry record. In particular, the record does not report the primary outcome’s observed mean change or its dispersion. It is therefore not possible to say from this record whether missed-medication percentage decreased, increased, or remained similar between screening and Week 24, or how precisely any estimated change was measured.
The study enrolled 30 participants in a single group receiving Abilify Maintena and CAE. That design allows description of outcomes over time among participants receiving the combined approach. It does not provide a randomized or concurrent control group. As a result, even if a change were reported, the change alone would not identify the effect of the combined approach relative to another treatment or to no intervention. Other influences over time cannot be separated from the intervention using a single-group comparison alone.
The primary measure is the percentage of medications not taken in the past week; higher scores mean poorer adherence. This direction helps interpret a reported score, but no score or change is available in the registry record. The record also does not report a confidence interval or other estimate of uncertainty for the primary outcome. A point estimate without uncertainty can be hard to judge, and the point estimate itself is not reported.
Secondary outcomes include measures of psychiatric symptoms, medication attitudes, and functioning. The record lists these outcome areas, but does not report their numerical results. It also lists 15 outcome measures and 19 analyses without enough detail to establish which estimates correspond to each planned question, whether analyses were adjusted, or how multiple analyses were handled. Those omissions limit assessment of the breadth and reliability of any outcome claims.
The serious adverse event record lists zero participants affected in the serious-event category, while the other adverse-event category lists 23 affected. These counts should be read alongside the recorded at-risk denominators and the absence of reported serious-event terms. They do not, on their own, settle questions about tolerability or establish how events were related to treatment.
Overall, the registry record describes a prospective study of a combined behavioral and injectable medication approach, not a controlled estimate of comparative effectiveness. The posted information does not support a conclusion about the magnitude, direction, or precision of the primary outcome change.
9. Statistical Methods Explained
The registry record describes the primary outcome as a mean of a continuous measure: change in the percentage of bipolar disorder medications missed during the past week. The listed dispersion measure is the standard deviation. The record says descriptive statistics, including means and standard deviations or counts and percentages, were generated for outcomes. It does not report the numerical primary outcome summary or specify a model used to estimate change.
What does a mean change describe?
General statistical explanation: A mean summarizes the average value in a group. For a change outcome, the estimate describes the average difference between measurements as defined by the analysis. Its interpretation depends on the scale and direction: for this TRQ measure, a higher score indicates poorer adherence. A mean is a group summary, not a statement that every participant changed by that amount. Individual responses can vary around the group average.
The standard deviation describes how spread out individual values are around a mean. It is not the same as the uncertainty in the mean estimate. An interval estimate, such as a confidence interval, can communicate uncertainty in an estimated group mean or change. The registry record does not report a confidence interval for the primary outcome, so its precision cannot be assessed from the posted results described in the record.
What can a single-group comparison establish?
General statistical explanation: Measuring a group at more than one time can show whether the group’s observed outcome changed over time. A comparison with a separate control group is usually needed to estimate how that change differs from what might have happened under another condition. Without such a comparator, time-related influences, changes in participants, and other concurrent factors may contribute to observed change. The registry lists a single-group model and no separate control group, so its design does not support a direct treatment-versus-control contrast.
Allocation is listed as NA and masking as NONE. The record does not report random assignment or blinded outcome assessment. These entries describe design features; they do not establish whether any particular source of bias occurred. The record also does not report a detailed statistical plan, so the analysis approach beyond descriptive summaries is not reported in the registry record.
How should planning illustrations be understood?
General statistical explanation: A sample-size calculation estimates how many participants a hypothetical study might require under chosen assumptions. It is a planning exercise, not evidence that an observed effect is reliable or that a study had a particular probability of detecting an effect. The registry record’s planning assumptions describe a two-arm illustration with equal allocation, a two-sided significance level of 0.05, and a normal approximation, using a stated fraction of an observed effect. These assumptions do not match the registered single-group model. Any resulting illustration should therefore be read as hypothetical planning information, not as the design actually used or as observed power.
The record does not report a trial-specific sample-size justification, the rationale for the enrollment target, or whether any interim analyses were conducted. No assumptions about those matters can be drawn from the planning illustration. The observed effect needed to interpret such an illustration is also not reported in the registry record.
What does over-dispersion mean in general?
General statistical explanation: Over-dispersion occurs when variability in observed data is greater than a statistical model expects. If not accounted for in a setting where it applies, standard errors can be too small and intervals too narrow. This concept is relevant to some types of count or categorical data, but the primary endpoint is described as a continuous percentage summarized with a mean and standard deviation. The registry record does not report an over-dispersion assessment or model, so it is not possible to say whether this issue arose in any analysis.
The record lists multiple secondary measures and a larger number of analyses than outcome measures, but does not describe the statistical tests, model assumptions, adjustment choices, or missing-data procedures. Those methods cannot be inferred from the outcome list. For the primary endpoint, the most important missing interpretive elements are the observed summary, an estimate of uncertainty, and enough analysis detail to understand how the change was calculated.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 15 outcome measures and 19 analyses. The listed secondary outcomes span psychiatric symptom scores, medication attitudes, and functioning, in addition to the primary adherence measure. When many outcomes or analyses are examined, the chance of finding an apparently notable result by chance can increase. General statistical explanation: Multiplicity procedures can address this issue by controlling error rates or by distinguishing confirmatory questions from exploratory ones. The registry record does not report whether any such procedure was used, which outcomes were treated as primary or secondary for inference, or how the 19 analyses map to the outcome measures.
The primary outcome’s description says that descriptive statistics were generated. It does not specify the inferential procedure, if any, used to assess change. The record also does not report whether results were adjusted for baseline values or other variables. These details are not reported in the registry record, so it is not possible to evaluate the intended handling of multiple testing or covariate adjustment.
Missing-data handling is also not described. The record does not report how many participants contributed measurements at each assessment, how missing measurements were treated, or whether any sensitivity analyses addressed missingness. The enrollment figure does not establish the number with complete outcome data. The analysis population is not defined beyond the description of poorly adherent patients who received the study interventions; an intention-to-treat, per-protocol, or other analysis population is not reported in the registry record.
These omissions matter because the interpretability of a change estimate depends not only on its value but also on who contributed data, how incomplete measurements were handled, and how multiple questions were addressed. The posted record does not provide enough detail to resolve those issues.
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 |
|---|---|---|---|
| CAE-L | 0 | 30 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| CAE-L | 0 of 30 |
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 |
|---|---|
| CAE-L | 23 of 30 |
Most frequent non-serious adverse event terms
| Event term | Organ system | CAE-L |
|---|---|---|
| Injection site pain or reaction | Skin and subcutaneous tissue disorders | 7 of 23 |
| Drowsiness | Nervous system disorders | 7 of 23 |
| Tremor | Nervous system disorders | 6 of 23 |
| Sexual dysfunction | Reproductive system and breast disorders | 3 of 23 |
| Drooling | General disorders | 2 of 23 |
| Muscle Movement complaints | Musculoskeletal and connective tissue disorders | 2 of 23 |
| Weight gain | Metabolism and nutrition disorders | 2 of 23 |
The serious adverse event record lists 30 participants at risk and zero affected in the serious-event category. The other adverse-event category lists 30 at risk and 23 affected. Deaths are recorded as zero, and no top serious adverse event terms are listed. These are registry counts for the CAE-L group; the record does not provide event-level descriptions for the serious-event category.
Counts of participants affected are not the same as counts of individual events: one participant may experience more than one event, and the record does not provide the event totals or descriptions needed to examine that distinction. The registry record also does not report whether events were judged related to treatment, their severity beyond the serious-event classification, their timing, or how long participants were monitored. The listed at-risk denominators should be read as recorded and do not establish that every participant had identical follow-up.
General statistical explanation: A recorded zero is an observed count in the specified category, not proof that the underlying risk is zero. Safety interpretation depends on the size and follow-up of the group, how events were collected and classified, and the completeness of reporting. The registry figures alone do not establish comparative safety because the study has no separate control group.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed prospective study of Abilify Maintena combined with Customized Adherence Enhancement in people with bipolar disorder at risk for treatment non-adherence. Its primary question concerns change in a past-week measure of missed medication from screening to Week 24. The record establishes the outcome definition, scale direction, and study structure, but does not report the numerical primary outcome result.
Accordingly, the posted information does not show the direction or size of change in the adherence measure. It also does not report an uncertainty interval for that change. No claim about the observed primary outcome can be supported from the registry record alone. The same limitation applies to the listed secondary measures: their names are recorded, but numerical results are not reported in the material.
Even if a before-and-after change were available, the single-group design would describe change during participation rather than provide a controlled comparison. It could not by itself distinguish the contribution of CAE, the injectable medication, their combination, or other influences over time. A separate comparator would be needed to estimate a comparative effect under the study conditions.
The safety record reports zero affected participants in the serious-event category and 23 in the other-event category, with zero deaths. These counts should be understood within the limits of the registry’s reporting and the absence of a control group. They do not establish that adverse events were caused by treatment, nor do they answer comparative safety questions.
In short, the record is informative about the study’s purpose, design, measures, and selected safety counts. It is not sufficient to determine whether the primary measure changed, how precisely any change was estimated, or whether the combined approach performed differently from an alternative.
13. Why This Trial Matters Statistically
This study raises a statistical question that is important for interpreting behavioral and medication approaches together: whether an adherence measure changes over time among people receiving the combined intervention. The primary outcome is defined in concrete terms as the percentage of bipolar disorder medications not taken during the past week, with higher scores indicating poorer adherence. That definition makes the direction of any reported change meaningful, provided the actual values and uncertainty are available.
The single-group design also makes a useful distinction between describing change and estimating a comparative effect. A before-and-after summary can characterize participants’ measured course, but cannot establish how that course differs from an alternative. This distinction is essential when translating a registry result into a statistical claim: the design determines which questions the data can answer, regardless of how the intervention is framed.
The record further illustrates why outcome labels alone are not enough for interpretation. Numerical estimates, uncertainty, analysis populations, missing-data handling, and multiplicity procedures all affect how confidently readers can interpret findings. The registry record lists numerous outcomes and analyses, yet those results and methods are not reported in the information available for this account.
Finally, the planning assumptions described for sample-size illustrations concern a hypothetical two-arm setting, while the registered study is single-group. Recognizing that distinction prevents a planning calculation from being mistaken for a feature of the actual study or for observed evidence about precision. These design and reporting considerations are central to a careful statistical reading of the record.
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 central limitation for interpreting the results is that numerical primary and secondary outcome findings are not reported in the registry record. Without observed means, changes, dispersion, or uncertainty intervals, the direction and precision of the primary endpoint cannot be assessed. The record’s description that descriptive statistics were generated does not substitute for the results themselves.
The single-group model and absence of masking limit what can be concluded about comparative effects. There is no registered control group to provide a concurrent estimate of what might have happened under another condition. The record does not report whether outcome assessment was otherwise standardized or whether any additional safeguards were used.
The primary outcome description gives a measure and timeframe, but the record does not report the number of participants with measurements at each assessment or how missing data were handled. It does not define the analysis population in sufficient detail to determine who contributed to each reported analysis. Completeness and representativeness of follow-up therefore cannot be assessed from the registry record.
Although the record lists 15 outcome measures and 19 analyses, it does not report which analyses were prespecified, how multiple analyses were addressed, or the full statistical methods. It also does not report a trial-specific sample-size justification. The planning assumptions listed for sample-size illustrations use a two-arm setup, which is not the registered single-group design, and should not be read as the study’s own design rationale.
The safety information is limited to category counts and does not include event descriptions, relatedness, or monitoring details. These reporting limitations do not show that a particular bias or problem occurred; they mean that important aspects of the results cannot be evaluated from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: CAE Plus LAI in Individuals With Bipolar Disorder at Risk for Treatment Non-adherence (BD-CAEL) — NCT03408873.
- Registry record last updated 2022-02-17; status last verified 2022-01.
- Results first posted 2022-02-17.
- 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
NCT03408873 is a completed Phase 4 prospective, single-group study of Abilify Maintena combined with Customized Adherence Enhancement in people with bipolar disorder at risk for treatment non-adherence. Enrollment was 30; allocation was listed as NA, and masking as NONE. The listed study start was 2018-04-01 and primary completion was 2020-05-06.
The primary outcome was change in the percentage of bipolar disorder medications missed in the past week, from screening to Week 24. The scale ranges from 0% to 100%, with higher scores indicating poorer adherence. The registry record does not report the observed primary outcome values or their uncertainty. It lists secondary symptom, attitude, and functioning measures but does not report their numerical results in the information.
The safety record lists zero serious-event-affected participants, 23 affected in the other-event category, and zero deaths. No serious adverse event terms are listed. The record does not provide a separate control group or enough statistical detail to assess comparative effects, missing-data handling, or multiplicity procedures.