A Trial to Measure the Difference in All-cause Hospitalizations for Participants Who Are Using Abilify MyCite Versus Virtual Matched Controls in Adults With Schizophrenia, Bipolar 1 Disorder, and Major Depressive Disorder: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Trial to Measure the Difference in All-cause Hospitalizations for Participants Who Are Using Abilify MyCite Versus Virtual Matched Controls in Adults With Schizophrenia, Bipolar 1 Disorder, and Major Depressive Disorder (NCT03643159), a non-randomized phase 4 study. The primary outcome is Difference In The Number Of Participants With All-cause Hospitalizations For Participants Using Abilify MyCite Versus Virtual Matched Controls From Baseline To Day 180.

2. Clinical Question

3. Trial Design

This was a Phase 4, multicenter, 180-day pragmatic clinical trial sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc. The registry record lists a non-randomized allocation, no masking, and a single-group model. The study began on 2018-06-28, had a primary completion date of 2018-10-17, and is recorded as terminated. Enrollment was 2.

The intended comparison was not a conventional trial with participants assigned to two enrolled arms. Participants using Abilify MyCite were to be compared with virtual matched controls, who were not to be enrolled in the study and were to be identified from health records. The registry record does not specify the matching method, the data source details, or how differences between the participant and control groups were to be addressed. It therefore does not establish that the groups were comparable at baseline.

The Abilify MyCite description specifies treatment during Months 1-3 and prohibits its use during Months 4-6. It also describes an optional interventional period of up to 6 months beginning at Day 180. Virtual matched controls were to receive treatment as usual throughout the trial, defined as any product other than Abilify MyCite. These descriptions indicate the planned structure, not what participants actually received; details of treatment exposure beyond the registry descriptions are not reported in the registry record.

The stated primary objective concerned all-cause hospitalizations, with secondary and exploratory objectives concerning medication adherence, healthcare utilization and costs, and patient-reported outcomes. The record identifies a secondary measure of the difference in the number of participants with at least 80% proportion of days covered with antipsychotic medication from baseline to Day 180. However, efficacy data were not collected due to early termination. The registry record reports no efficacy analyses.

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.

Before assignment: Virtual matched controls were not to be enrolled into the study, but identified from health insurance claims data and matched to the enrolled Abilify MyCite participants at the end of the study for analysis. Since the study was terminated early, no matching occurred.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Efficacy Analysis: All participants who received at least 1 dose of Abilify MyCite or who were a virtual matched control and had efficacy data. Both participants were excluded from efficacy analysis because they discontinued study therapy early. In addition, efficacy data were not collected due to early study termination.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: Safety: All participants who received at least 1 dose of Abilify MyCite or who were a virtual matched control.

6. Primary Endpoint

The registry lists the primary outcome as Difference In The Number Of Participants With All-cause Hospitalizations For Participants Using Abilify MyCite Versus Virtual Matched Controls From Baseline To Day 180, measured over Baseline through Day 180.

The primary endpoint was the difference in the number of participants with at least one all-cause hospitalization between participants using Abilify MyCite and virtual matched controls from baseline through Day 180. All-cause hospitalizations include hospitalizations for any reason, rather than only those attributed to a particular diagnosis or treatment. The registry record gives the endpoint timeframe but does not provide a measured event count, an effect estimate, or a statistical analysis.

A participant-based hospitalization endpoint can describe whether people experienced any hospitalization during a defined observation period. In general, such an outcome may be relevant to healthcare utilization because it captures hospital stays regardless of cause. It does not, by itself, describe the number of admissions per participant, how long admissions lasted, or why they occurred. Those distinctions matter when interpreting what an endpoint represents, but the registry record does not report such additional hospitalization details.

The primary outcome was not observed in the posted results: the record states that efficacy data were not collected because of early study termination. Both participants were excluded from the efficacy analysis after discontinuing study therapy early. As a result, no estimate of the difference in participants with hospitalizations can be interpreted, and no conclusion about the magnitude or precision of a group contrast is available.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies the outcome of interest and its timeframe, but does not report a statistical analysis for that outcome. The concepts below are general explanations of how such an analysis might be interpreted; they are not claims about methods used in this trial.

What does a comparison of participants with hospitalizations represent?

For a binary endpoint, each person is classified according to whether the defined event occurred during the observation period. A group comparison then describes how the event occurrence differs between groups. The present endpoint is phrased as the difference in the number of participants with all-cause hospitalizations, from baseline through Day 180. In general, counts alone depend on how many people are observed in each group. When group sizes differ, rates or proportions can provide context, but the registry record does not report an analysis or an outcome value for either group.

The endpoint also focuses on whether a participant had a hospitalization, not on the total number of admissions. A participant with more than one hospitalization may still contribute only as a participant with at least one event if the analysis is based on the stated participant count. The record does not provide further details on event-level counting or repeated admissions. No assumptions about those details should be made.

What does a confidence interval tell a reader?

In general, a confidence interval expresses the statistical uncertainty around an estimated effect under the assumptions of the analysis. A narrower interval indicates greater precision, while a wider interval indicates less precision. An interval can also show which effect sizes remain compatible with the observed data under the specified method. The registry record reports no estimate or confidence interval for the primary endpoint, so precision cannot be assessed.

A p-value, when reported, addresses how compatible the observed data are with a specified null hypothesis under an analysis model. It does not measure the probability that a treatment is beneficial, and it does not describe the size or practical importance of an effect. No primary-outcome p-value or test result is reported in the registry record. Statistical significance therefore cannot be assessed, and the lack of a reported test is not evidence that the groups were equivalent.

Why does the comparator design matter?

Random assignment is a general method for balancing measured and unmeasured characteristics between groups on average. This trial is recorded as non-randomized, and virtual matched controls were not to be enrolled. A matched-control approach can seek to make groups more alike on selected characteristics, but its interpretation depends on what was matched, how the matching was conducted, and which data were available. These details are not reported in the registry record. Even if a comparative estimate had been posted, the design would require careful consideration of possible residual differences between groups.

The single-group model listed in the record sits alongside a stated objective that compares Abilify MyCite participants with virtual controls. The controls were virtual rather than an enrolled study arm. This distinction matters: the registry description of the objective does not itself demonstrate that comparable data were available or that a valid comparison was completed. The record reports no efficacy analysis.

How should early termination and excluded participants be understood?

Early termination means the study ended before the planned study course was completed, but it does not, on its own, specify why the study stopped or how much follow-up each participant contributed. The registry record gives the status as terminated and states that efficacy data were not collected due to early termination. It also says both participants were excluded from the efficacy analysis because they discontinued study therapy early. The reasons for discontinuation and any outcomes observed before discontinuation are not reported in the registry record.

In general, excluding participants after treatment discontinuation can affect interpretation because those who discontinue may differ from those who remain in a study. An analysis that includes participants according to their original group assignment, or an analysis using another clearly specified approach, can address different questions and relies on different assumptions. The registry record does not report an analysis population beyond the stated efficacy population description, nor does it provide a statistical method for handling discontinuation or missing outcomes. It is not possible to determine what an alternative analysis would have shown.

What is over-dispersion?

General count-data methods sometimes assume that variation in event counts follows a particular distribution. Over-dispersion occurs when the observed variability is greater than that assumed by a simpler model. If present, it can make standard errors too small and confidence intervals too narrow unless the method accounts for the extra variation. This concept is relevant to some analyses of hospitalization counts, but the stated primary endpoint is the number of participants with hospitalizations, and the registry record reports no count model or evidence of over-dispersion. No claim can be made about whether it affected this trial.

What does the record say about the statistical plan?

The registry record reports zero analyses and does not describe a planned model, covariate adjustment, hypothesis-testing procedure, confidence-interval method, or interim analysis. It also does not report a sample-size justification or an account of how the planned comparison would be estimated. These omissions prevent readers from checking whether an analysis followed a prespecified plan or assessing the assumptions behind any inferential result. Because no efficacy result is posted, there is no sponsor analysis to compare with a recalculation from summary statistics.

The secondary measure concerns the number of participants reaching at least 80% proportion of days covered with antipsychotic medication from baseline to Day 180. The record lists that measure, but does not report results or an analysis. The listed secondary objective does not supply evidence about the primary hospitalization endpoint. In general, outcome definitions, analysis populations, missing-data methods, and the handling of multiple outcomes all influence interpretation; the record does not report those methods in sufficient detail to evaluate them.

10. Multiplicity, Missing Data and Analysis Populations

The registry record identifies a primary hospitalization outcome and a secondary measure of the number of participants with at least 80% proportion of days covered with antipsychotic medication. It also describes secondary and exploratory objectives concerning medication adherence, healthcare utilization and costs, and patient-reported outcomes. The record does not report a multiplicity adjustment, a hierarchy for testing outcomes, or a statistical plan specifying how multiple comparisons would be handled. These details are not reported in the registry record.

In general, testing several outcomes can increase the chance of obtaining at least one apparently notable result by chance. A prespecified hierarchy or another multiplicity strategy can clarify which findings are confirmatory and how uncertainty should be interpreted. Since the registry record reports no efficacy analyses, there are no posted outcome tests to evaluate for multiplicity.

The record states that efficacy data were not collected due to early termination and that both participants were excluded from the efficacy analysis after early discontinuation of study therapy. It does not describe the amount or pattern of missing outcome information, reasons for discontinuation, or a method for handling missing data. Missing-data methods and assumptions are not reported in the registry record.

The efficacy population is described as participants who received at least 1 dose of Abilify MyCite or were virtual matched controls and had efficacy data. Both participants were excluded because they discontinued study therapy early. The record gives no efficacy results for an alternative population and does not report an intention-to-treat analysis. It is therefore not possible to assess how different analysis-population choices might have affected a result.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry record lists safety figures for the Abilify MyCite group. Among 2 participants at risk for serious adverse events, 0 were affected; among 2 at risk for other adverse events, 1 was affected. The record lists 0 deaths. These are participant counts, not a comparative estimate of safety between Abilify MyCite and virtual matched controls. Corresponding control-group safety figures are not reported in the registry record.

The serious adverse event count should be read as a descriptive observation within a very small enrolled group. A count of 0 affected participants does not establish that an event cannot occur or quantify the risk in a broader population. With 2 participants at risk, the record offers limited information about uncommon events and does not report a comparative statistical analysis. It also does not identify any top serious adverse-event terms.

The other-adverse-event figure is distinct from the serious adverse-event figure. The record reports 1 participant affected among 2 at risk for other adverse events, but does not provide event details in the listed safety summary. These safety counts do not fill the gap in efficacy data or answer the primary hospitalization question.

12. What the Result Does, and Does Not, Mean

The registry record supports a limited set of conclusions about the conduct and reporting of the trial. It identifies a Phase 4, non-randomized, 180-day pragmatic study intended to compare all-cause hospitalizations among participants using Abilify MyCite with virtual matched controls. It records enrollment of 2 participants and a terminated status. It also states that efficacy data were not collected because of early termination and that both participants were excluded from efficacy analysis after discontinuing study therapy early.

Those details do not establish whether Abilify MyCite was associated with more, fewer, or the same number of participants with hospitalizations as the virtual controls. No primary-outcome counts, effect estimate, uncertainty interval, or statistical test are reported. The absence of a result is not a finding of no difference, and it is not evidence of equivalence. There is no estimate whose size or precision can be evaluated.

The design further limits the interpretation of the intended comparison. Allocation was non-randomized, the record lists a single-group model, and virtual controls were not to be enrolled. The record does not describe the matching procedure or report comparative outcomes. As a result, it is not possible to judge whether the planned groups were sufficiently comparable or whether any potential differences were addressed.

The safety entries provide only descriptive counts for the Abilify MyCite group: 0 participants with serious adverse events among 2 at risk, 1 participant with other adverse events among 2 at risk, and 0 deaths. They are not comparative evidence and should not be treated as a substitute for the missing efficacy outcome.

In general, a treatment comparison is most informative when the outcome data, analysis population, methods, and uncertainty are reported clearly. Here, the record describes the intended question but does not provide the evidence needed to answer it. The findings cannot support a conclusion about hospitalization differences or treatment effects.

13. Why This Trial Matters Statistically

This trial is statistically important as an example of the distinction between a research question and an answerable comparison. The registry record clearly states a primary question about all-cause hospitalizations through Day 180, but the trial ended without efficacy data. A defined endpoint and a planned comparator do not generate an estimate when the necessary outcome observations and analysis are unavailable.

The record also illustrates how design labels need to be read alongside the practical comparison. It lists non-randomized allocation and a single-group model, while describing virtual matched controls as the intended comparator. Those controls were not to be enrolled. Without details about how they were selected and matched, readers cannot evaluate the comparability of the groups or the assumptions required for a valid estimate.

Enrollment of 2 is relevant to the scope of the record, but the main obstacle to interpreting the primary endpoint is that efficacy data were not collected and both participants were excluded from efficacy analysis. A small enrollment alone would not tell a reader whether an effect estimate was large or small; here, there is no efficacy estimate to assess at all. This is why a missing result must not be mistaken for a null result.

Finally, the safety counts and the listed secondary outcome do not resolve the primary question. They represent different outcomes and, in this record, do not include a reported comparative efficacy analysis. Clear reporting of outcome data, analysis populations, missing-data handling, and statistical methods is essential for readers to judge both the magnitude and precision of any clinical-trial comparison. The registry record does not report those elements for an efficacy result.

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 most direct limitation is that the study was terminated and efficacy data were not collected. Both participants were excluded from the efficacy analysis because they discontinued study therapy early. The registry record does not report the reasons for discontinuation, the outcomes observed before discontinuation, or whether any efficacy information was available outside the reported analysis population. No primary-outcome estimate or comparison can therefore be assessed.

Enrollment was 2, and the registry record does not report participant characteristics or the distribution of diagnoses. The intended population included adults with schizophrenia, Bipolar 1 Disorder, and Major Depressive Disorder, but the record does not show how many enrolled participants had each condition. The extent to which the enrolled group reflects the intended population cannot be judged.

The control group was virtual and was not to be enrolled. The record does not describe the health data source, matching variables, matching procedure, control-group size, follow-up completeness, or outcome ascertainment. The design is listed as non-randomized, so the record does not establish comparability between the intervention group and controls. No adjustment methods are reported.

The registry record also does not report an analysis plan, a sample-size justification, a missing-data method, a multiplicity strategy, or inferential results. It does not provide a hospitalization count or information about admissions beyond the endpoint definition. The listed safety counts are limited to the Abilify MyCite group and do not provide a comparative safety analysis. These limitations prevent assessment of the primary effect, its precision, and the robustness of any intended comparison.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Trial to Measure the Difference in All-cause Hospitalizations for Participants Who Are Using Abilify MyCite Versus Virtual Matched Controls in Adults With Schizophrenia, Bipolar 1 Disorder, and Major Depressive Disorder — NCT03643159.
• Registry record last updated 2019-11-08; status last verified 2019-10.
• Results first posted 2019-11-08.
• 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.

18. Record Summary

The registry record describes NCT03643159, a Phase 4, multicenter, 180-day pragmatic clinical trial sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc. It was non-randomized, had no masking, used a single-group model, enrolled 2 participants, and is recorded as terminated. The intended primary comparison concerned participants with all-cause hospitalizations from baseline through Day 180, comparing Abilify MyCite users with virtual matched controls receiving treatment as usual.

Virtual controls were not to be enrolled. Participants using Abilify MyCite were to receive it during Months 1-3, with use prohibited during Months 4-6; an optional period beginning at Day 180 was also described. The record states that efficacy data were not collected because of early termination and that both participants were excluded from efficacy analysis after early discontinuation of study therapy. No efficacy analysis or primary-outcome result is reported. The safety summary lists 0 participants with serious adverse events, 1 with other adverse events, and 0 deaths in the Abilify MyCite group.