Increasing Documentation and Disclosure of Sickle Cell Trait Status: An Implementation Science Approach: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Increasing Documentation and Disclosure of Sickle Cell Trait Status: An Implementation Science Approach (NCT05387564), a randomized na study. The primary outcome is Acceptability.

2. Clinical Question

3. Trial Design

The registry describes a randomized, unmasked, 2-arm trial using a sequential model and an interrupted time series approach. Its focus is an implementation question: how strategies for supporting SCT documentation and disclosure are delivered in pediatric primary care, and how providers respond to those strategies. The record does not describe the randomization procedure, allocation ratio, or any stratification factors.

The intervention is the SCT Documentation and Disclosure Toolkit. In the "All-in" arm, pediatric primary care physicians receive all toolkit components at once. In the "Add-in" arm, components are added sequentially in 6 week increments. This creates a contrast in the timing and sequencing of the toolkit, not a stated comparison with no toolkit. The record does not specify the individual components or explain whether delivery differed in other respects.

The primary endpoint is assessed by survey at the conclusion of the study, defined as 18 weeks after initial intervention rollout. The record lists knowledge as a secondary outcome and identifies 5 outcome measures, but does not provide their individual definitions or results. Enrollment is reported as 114. The registry status is Completed, with a study start date of 2024-01-18 and primary completion date of 2024-08-31.

Although the design is described as an interrupted time series trial, the registry record does not describe the measurement schedule, the interruption points, or how time trends were to be modeled. It also does not state whether measurements were repeated for individual providers or summarized at another level. Those details matter for understanding how the sequential implementation strategy was evaluated, and cannot be inferred from the design label alone.

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.

Clinician

Caregiver Survey From Retrospective Time

Caregiver Survey From Intervention Time

Analysis population for the primary outcome, as defined in the registry: At conclusion of study: 18 weeks after initial roll-out of interventions.

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: Two sets of participants included: 1) Physicians who were the primary target of the intervention, 2) Caregivers who consented and completed surveys. As physicians were the primary interest, demographic characteristics and the majority of study outcomes were based on physicians enrolled.

6. Primary Endpoint

The registry lists the primary outcome as Acceptability, measured over At conclusion of study: 18 weeks after initial roll-out of interventions.

The primary endpoint is titled Acceptability. It measures the number of pediatric primary care providers who answer Agree or Strongly Agree when asked whether they liked a toolkit component. Responses are collected by survey at the study conclusion, defined as 18 weeks after initial rollout. The response options are Strongly disagree, Disagree, Agree, and Strongly Agree, described as a 4-point Likert scale.

Acceptability is a measure of providers’ perceptions of the intervention. In implementation research, this kind of measure can help characterize whether intended users view a strategy favorably, which is relevant when considering how an intervention is received in practice. It is distinct from a measure of actual documentation or disclosure behavior, and it does not by itself establish that a strategy changed clinical records or communication.

The endpoint is expressed as a count of participants meeting the response criterion, rather than as a mean score on the Likert scale. The registry record does not provide the observed counts, a breakdown by toolkit component, or an analysis comparing the arms. It also does not explain how unanswered survey items or providers who did not complete the survey were handled. Therefore, the endpoint’s definition is available, but its observed value and precision are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The methods description contains several labels that identify broad design features, but a label does not specify every analytic decision. The registry calls the trial randomized, unmasked, sequential, and an interrupted time series study. It identifies an acceptability endpoint but does not report the statistical model or a primary outcome analysis. The following explains the general meaning of these design and statistical concepts without attributing unreported methods to this trial.

What does randomization contribute?

In general, random assignment aims to make comparison groups similar, on average, with respect to factors that could affect outcomes. This can strengthen causal interpretation of a contrast between assigned strategies. It does not guarantee that groups will be identical, especially in a study with a limited enrollment, and it cannot address differences introduced after assignment. The registry record does not describe how assignments were generated or concealed, the allocation ratio, or whether any additional design features were used.

Here the assigned contrast is between delivery of all toolkit components at once and sequential addition of components in 6 week increments. This is a comparison of implementation approaches. The record does not state that either arm receives no toolkit, so the described contrast should not be framed as toolkit versus no toolkit.

What does an interrupted time series approach mean?

In general, an interrupted time series examines outcome patterns over time in relation to an intervention or implementation change. Its interpretation depends on the number and timing of observations, the pattern before and after an interruption, and the assumptions used to distinguish an intervention-associated change from an ongoing time trend or other changes occurring at the same time. When over-dispersion is present in count data, variability exceeds what a simple model assumes; accounting for it can prevent uncertainty from being understated. These are general considerations, not claims about the model used in this trial.

The registry record does not give the measurement schedule, the number or timing of observations, or a model specification. It therefore does not allow readers to determine how time, intervention timing, or within-provider correlation were handled. It also does not clarify how the interrupted time series component was combined with the randomized arm comparison.

How should the acceptability measure be understood?

The primary outcome counts providers who select Agree or Strongly Agree about liking a toolkit component. This converts responses on an ordered Likert scale into a favorable-response count. Such a count is straightforward to describe, but it does not preserve distinctions between Agree and Strongly Agree, and it treats other response options as outside the stated favorable category. The record does not say whether results would be reported separately by component, combined across components, or compared between arms in a particular way.

For a count or proportion to be interpreted, the denominator and handling of missing responses are important. For example, a count among survey respondents answers a different question from a count calculated using everyone assigned to an arm. The registry record does not report the response denominator or a missing-item rule. No outcome counts are listed, so the measure cannot be recalculated from the registry information.

What can a significance level and a sample-size illustration tell readers?

The record describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It also describes considering the stated fraction of the observed effect. In general, a significance level is a threshold used in a hypothesis-testing procedure; it does not measure the size or importance of an effect. A two-sided test considers departures in either direction. Equal allocation and a normal approximation are assumptions for the illustration, not evidence that the trial used those assumptions for its primary analysis.

A sample-size illustration addresses how many participants per arm might be needed under specified assumptions. It is not a description of the number enrolled, and it is not observed power. The registry record does not report the trial’s own sample-size justification or statistical analysis plan. The record’s planning statement also distinguishes time-to-event illustrations, which use total events, from other illustrations, which use participants per arm. No calculation should be mistaken for a result of the randomized comparison.

What is missing for statistical interpretation?

The registry record reports 0 analyses and does not provide an effect estimate, standard error, confidence interval, or p-value for the primary endpoint. It does not specify a statistical model, adjustment variables, the analysis population, treatment of repeated measures, or missing-data methods. These omissions do not demonstrate that the study team did not use such methods; they mean those methods are not reported in the registry record. Without outcome results and analysis details, precision and robustness cannot be assessed.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 5 outcome measures, with Acceptability identified as the primary outcome and Knowledge listed as a secondary outcome. It does not report the full set of outcome definitions or a plan for managing multiplicity. When a study evaluates several outcomes, components, time points, or contrasts, the chance of at least one apparently notable result can increase if multiple tests are conducted. In general, a prespecified strategy may define which comparisons are primary and whether adjustment is appropriate. No such strategy is reported in the registry record, so it is not possible to determine whether or how multiplicity was addressed.

The primary endpoint description refers to liking a toolkit component and gives a survey time point of 18 weeks after initial rollout. The record does not say how many components were evaluated in the survey, whether each generated a separate outcome, or whether any additional time points were analyzed. It also does not identify which outcomes or comparisons were included in the reported analyses. The registry lists 0 analyses, so inferential results and any multiplicity adjustments are not available there.

Missing-data handling is also not reported in the registry record. There is no description of survey nonresponse, unanswered items, incomplete follow-up, or whether such observations were excluded, imputed, or handled in another way. The analysis population is not specified: the record does not state whether the endpoint would be analyzed according to randomized assignment, among respondents, or using another population definition. These details matter because the denominator and the method of handling unavailable responses can affect interpretation of a count endpoint.

These are reporting gaps, not proof that the study lacked a prespecified plan or that missing responses occurred. The registry record does not provide enough information to assess those possibilities.

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 5% reporting threshold.

The registry lists serious adverse event counts separately for the "All-in" and "Add-in" groups. It reports 0 serious events among 16 at risk in the "All-in" group and 0 among 12 at risk in the "Add-in" group. Deaths are reported as 0 in both groups. No serious adverse event terms are listed.

These figures describe the safety counts recorded for the listed groups; they are not the primary endpoint and do not measure acceptability, documentation, or disclosure. A zero count means no serious events are reported in these registry entries. It does not establish that an intervention has no risk, and it should not be generalized beyond the scope of the recorded groups and monitoring. The record does not explain how adverse events were defined, what surveillance procedures were used, or whether non-serious events were collected. The at-risk counts are not described as the denominator for the acceptability survey, and should not be used as such.

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

The registry record documents a completed randomized trial that compared two ways of delivering a toolkit intended to support SCT documentation and disclosure in pediatric primary care. It defines an acceptability endpoint based on providers’ survey responses at the study conclusion. This is useful information about the question the trial set out to study and how the primary measure was framed.

However, the record does not report the observed acceptability counts or a statistical comparison between the arms. It also does not report results for documentation, disclosure, or the secondary knowledge outcome. Accordingly, the record does not establish whether providers favored either delivery strategy, whether documentation or disclosure changed, or whether outcomes differed between the arms. A trial’s stated objective and completed status are not themselves evidence of an effect.

The acceptability measure captures providers’ expressed views about liking toolkit components. It is not interchangeable with observed provider behavior, families’ knowledge, or the accuracy or completeness of an electronic health record. Even a favorable response would not, by itself, establish that the toolkit changed documentation or disclosure; those outcomes require their own reported measures and analyses.

Likewise, absence of a reported result is not evidence that no effect occurred. Without estimates and uncertainty measures, readers cannot distinguish a small effect from an imprecisely estimated one, or assess whether results are compatible with different possible effects. The registry record also does not contain the analysis details needed to evaluate time trends, missing responses, or multiple comparisons.

The most supportable conclusion is therefore limited: the registry describes the trial’s design, intervention contrast, and planned primary measure, and reports serious-event counts for the listed groups. It does not report enough outcome or analysis information to support a conclusion about the trial’s effectiveness or the relative acceptability of its two implementation strategies.

13. Why This Trial Matters Statistically

This trial addresses an implementation gap described in the registry: SCT status can be incidentally identified through newborn screening, while the registry’s background states that documentation in pediatric electronic health records is low and few adults report knowing their status despite universal screening. The trial’s focus is not simply the clinical condition; it is how providers receive strategies intended to support documentation and disclosure. That makes the distinction between implementation measures and behavioral outcomes especially important.

Statistically, the design combines randomization with a sequential implementation approach and an interrupted time series description. Those features raise questions about both between-arm differences and changes over time. Interpreting such a design requires clear reporting of when measurements occurred, how the time pattern was modeled, and how the randomized contrast was estimated. The registry record does not provide those details or the analyses, so the design’s statistical contribution cannot be evaluated from the registry alone.

The primary endpoint also highlights an important measurement distinction. Provider-reported acceptability can help characterize whether an intervention is viewed favorably, but it is not a substitute for evidence about changes in documentation or disclosure. Reporting each outcome with its denominator, estimate, and uncertainty would allow readers to see whether perceptions and practice outcomes align or differ.

Finally, transparent reporting of missing responses, analysis populations, and multiplicity is consequential when outcomes include provider surveys and potentially multiple toolkit components. Those details support a fair reading of results and their precision. The registry record gives the framework for asking these questions, but not the results needed to answer them.

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 this particular record is that it does not include outcome results or statistical analyses. The primary endpoint is defined, but no count of favorable responses, arm-level estimate, confidence interval, or p-value is reported. The registry lists 0 analyses. As a result, the relative acceptability of the delivery approaches cannot be assessed, and the stated goals concerning documentation and disclosure cannot be evaluated from the posted results.

Important design details are also absent. The record does not describe the randomization procedure, allocation ratio, toolkit components, measurement schedule for the interrupted time series, or statistical model. It does not explain how the randomized comparison and time-based analysis were combined. These omissions make it difficult to assess the assumptions that would support an effect estimate or its precision.

Survey interpretation is limited by the absence of response denominators and missing-data procedures. The registry does not specify how providers who did not respond, or respondents with incomplete items, were treated. It also does not state the analysis population or whether acceptability was assessed separately for each component. Since the endpoint counts favorable responses, those definitions are necessary to understand the meaning of any eventual count.

The record identifies 5 outcome measures but does not fully describe their definitions or report a multiplicity strategy. It does not provide a sample-size justification for the trial itself. The safety counts are limited to the serious-event figures and deaths listed; further information about event definitions and monitoring is not reported in the registry record. These limitations concern what can be established from the registry record, and do not establish that the study team did not collect additional information or conduct analyses elsewhere.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Increasing Documentation and Disclosure of Sickle Cell Trait Status: An Implementation Science Approach — NCT05387564.
• Registry record last updated 2026-01-07; status last verified 2026-01.
• Results first posted 2026-01-07.
• 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 NCT05387564, a completed randomized, unmasked, 2-arm implementation trial of the SCT Documentation and Disclosure Toolkit. The "All-in" arm received all components at once, while the "Add-in" arm received sequential additions in 6 week increments. Enrollment is reported as 114. The study start date is 2024-01-18, and the primary completion date is 2024-08-31.

The primary outcome is provider acceptability, measured by survey 18 weeks after initial rollout as the count answering Agree or Strongly Agree that they liked a toolkit component. Knowledge is identified as a secondary outcome, and 5 outcome measures are listed. The registry reports 0 analyses and does not report primary or secondary outcome results. Serious adverse event counts are 0 in both listed groups, and deaths are 0 in both. Statistical estimates, analysis methods, missing-data procedures, and multiplicity handling are not reported in the registry record.