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 Music Therapy in Sickle Cell Transition Study (NCT02466555), a na na study. The primary outcome is Change (T1 - T5) From Baseline in Scores on the Sickle Cell Self-Efficacy Scale (SCSES).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02466555 |
| Phase | NA |
| Condition | Sickle Cell Disease |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University Hospitals Cleveland Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
Young adults with sickle cell disease participating in the music therapy intervention during transition. The registry record lists an enrollment of 30, while the number who completed the intervention or contributed endpoint data is not reported in the registry record.
Intervention
The BEATS Music Therapy Program. The record describes music therapy as a clinical use of music interventions within a therapeutic relationship, delivered by a credentialed professional. Further detail about the intervention schedule and content is not reported in the registry record.
Comparator
There is no separate comparison group. The primary hypothesis concerns change from baseline within the group receiving music therapy. The clinic appointment adherence hypothesis compares the study period with the year before it, but details of that historical comparison are not reported in the registry record.
Primary question
Did participants’ Sickle Cell Self-Efficacy Scale scores change from baseline at T1 to 12 months at T5? The registry record identifies this as a change in least-squares mean and describes higher scores as indicating higher self-efficacy. The result itself is not reported in the registry record.
The study’s stated broader aim is to investigate self-efficacy, trust in health care providers, sickle cell disease knowledge, and adherence during transition. The summary also lists questions about clinic appointments, transfusion attendance, health care utilization, and hydroxyurea adherence. These are research questions, not evidence that an outcome changed. The available record describes a single-group design, so the primary question is about change over time among participants receiving music therapy, not a randomized comparison with usual care or another intervention.
3. Trial Design
The registry record identifies NCT02466555 as a completed study titled Music Therapy in Sickle Cell Transition Study. It lists an enrollment of 30 and a study period beginning in 2015-06, with primary completion in 2016-08. The condition is sickle cell disease, and the sponsor is University Hospitals Cleveland Medical Center.
The study is described as single-group, with one Music Therapy Group. Allocation is listed as NA, and masking is listed as NONE. Thus, the record does not describe random assignment or a masked comparison. The primary endpoint is framed as a within-participant change from baseline, rather than a difference between intervention and control groups. The appointment adherence hypothesis separately refers to comparing the study period with the one year before the study period; that historical comparison does not create a concurrent control group.
The intervention is identified as behavioral music therapy. The summary names the BEATS Music Therapy Program and describes intended areas of interest, including self-efficacy, trust, knowledge, and adherence. It does not report the intervention’s session schedule, duration, or participant-level exposure. Those implementation details are not reported in the registry record.
The record lists a primary outcome measured at baseline (T1), 3 months (T2), 6 months (T3), 9 months (T4), and 12 months (T5), as well as a change from T1 to T5. The number of participants contributing measurements at each time point is not reported in the registry record. The record also lists one secondary outcome concerning change from baseline in the percentage of attended clinic appointments during the one-year study period. Four outcome measures are listed, but the registry record does not report numerical results for them.
The record does not report a phase beyond NA, a sample-size justification, or a detailed statistical analysis plan. Completion status indicates that the study is marked completed; it does not establish that every enrolled participant completed follow-up or that all planned outcomes were analyzed.
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: Thirty-three participants with SCD were approached for recruitment. Three declined due to scheduling conflicts or disinterest. Thirty (90.9%) participants agreed to participate and completed written consent.
Overall Study
| Stage | Music Therapy Group |
|---|---|
| STARTED | 30 |
| COMPLETED | 28 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Music Therapy Group |
|---|---|
| Lost to Follow-up | 2 |
Analysis population for the primary outcome, as defined in the registry: All participants who participated in the music therapy intervention.
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 | Music Therapy Group |
|---|---|---|
| Participants analysed | 28 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 28 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 21.04 (SD 1.40) |
| Sex: Female, Male: Female | Participants | 14 |
| Sex: Female, Male: Male | Participants | 14 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 28 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 28 |
| Type of Sickle Cell Disease: HbSS | Participants | 19 |
| Type of Sickle Cell Disease: HbSC | Participants | 6 |
| Type of Sickle Cell Disease: HbSBeta+ Thalassemia | Participants | 2 |
| Type of Sickle Cell Disease: HbSBeta0 Thalassemia | Participants | 1 |
| Sickle Cell Disease Treatment: Hydroxyurea | Participants | 7 |
| Sickle Cell Disease Treatment: Chronic transfusions | Participants | 9 |
| Sickle Cell Disease Treatment: None | Participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Change (T1 - T5) From Baseline in Scores on the Sickle Cell Self-Efficacy Scale (SCSES), measured over Baseline (T1), 3 months (T2), 6 months (T3), 9 months (T4), 12 months (T5), change (T1-T5) in least square mean from T1 to T5 reported.
| Element | Registry entry |
|---|---|
| Result type | Least Squares Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | score on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change from baseline in scores on the Sickle Cell Self-Efficacy Scale (SCSES), expressed as a least-squares mean. The record identifies the scale as a nine-item Likert measure developed for adults with sickle cell disease and later revised for adolescents. Its total score ranges from 9 to 45, with higher scores representing higher self-efficacy.
Self-efficacy refers, in the outcome description, to a person’s conviction that they can carry out behavior needed to produce an outcome. In a transition-care study, measuring this construct can describe participants’ reported confidence in managing behavior related to their condition. A self-report scale captures the participant’s responses to its items; it is not itself a measure of clinical events, appointment attendance, or treatment adherence.
The planned assessment schedule includes baseline (T1), 3 months (T2), 6 months (T3), 9 months (T4), and 12 months (T5). The named primary change compares T1 with T5. The record does not report the observed score at either time point, the estimated change, or a confidence interval. It also does not describe which covariates, if any, were used to obtain the least-squares mean. Consequently, the endpoint’s definition and timing can be described, but its magnitude and precision cannot be interpreted from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study’s design and its planned primary measure, but it does not report a numerical primary endpoint result. It lists 0 analyses. There is therefore no posted estimate of change in SCSES score, no reported confidence interval for that change, and no reported statistical test or p-value to interpret. The absence of these results means the direction, magnitude, and precision of the observed change cannot be assessed from the registry record.
The record’s hypothesis is directional: compared with baseline, participants receiving music therapy were expected to report higher self-efficacy. A hypothesis describes what investigators set out to evaluate; it is not a finding. Likewise, the outcome description says that higher SCSES scores represent higher self-efficacy, but this scoring direction does not establish that scores rose during the study.
Even if a within-group change estimate were available, it would describe change over time in the group receiving the intervention. In a single-group study, that change alone cannot separate the intervention’s contribution from other explanations, such as changes over time or other experiences during follow-up. This is a general limitation of interpreting before-and-after comparisons without a concurrent control group. The record does not report a randomized or otherwise concurrent comparison group.
A confidence interval, if reported, would describe uncertainty around an estimated quantity under the analysis used. It would not by itself show whether the change was caused by the intervention or whether it matters to participants. Here, the registry record identifies 95% confidence intervals as the planned dispersion format for the primary measure, but no interval values are reported. No conclusions about statistical significance, clinical importance, or consistency across participants can be drawn from an absent estimate.
The record’s summary also names hypotheses about trust and disease knowledge, and questions about clinic appointments, transfusions, health care utilization, and hydroxyurea adherence. It lists a secondary clinic-appointment outcome, but numerical results for these outcomes are not reported in the registry record. The number of outcome measures listed is not a substitute for their results. The record should therefore be read as documenting study aims and endpoint definitions, not as demonstrating changes in self-efficacy or other outcomes.
Planning illustrations based on an observed effect are described in the study information, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are not observed power and do not establish the design or analysis used in this single-group study. No numerical illustration values are stated in the registry record, and the illustrations cannot fill the gap left by the missing outcome estimates.
9. Statistical Methods Explained
The registry record names a least-squares mean and a 95% confidence interval for the primary endpoint, but it does not report an analysis model, covariate adjustments, test statistic, or numerical estimate. The concepts below explain how such summaries are generally used; they do not identify methods that the study necessarily used beyond those expressly named in the record.
What does a least-squares mean represent?
In general, a least-squares mean is a model-based estimate of an outcome mean. Depending on the analysis model, it may account for specified factors or repeated measurements. It can differ from a simple arithmetic average when the model adjusts for covariates, handles an unbalanced set of observations, or otherwise structures the data. The registry record does not state which model or adjustments produced the primary least-squares mean. Its label alone is not enough to reconstruct the analysis.
The endpoint is defined as change from baseline between T1 and T5, with intervening assessments at T2, T3, and T4. Repeated measurements from the same participant are generally related rather than independent. In general, a longitudinal analysis may account for this within-person relationship and use measurements from more than one time point. The record does not say whether the intermediate measurements were included in the primary analysis, how repeated observations were modeled, or how participants with incomplete follow-up were handled.
What would a confidence interval convey?
In general, a confidence interval gives a range of values compatible with an estimate under the statistical model and its assumptions. A narrower interval indicates greater precision than a wider interval, while neither width nor location alone establishes causation. Interpreting an interval requires knowing what quantity was estimated, how the interval was calculated, and which observations were included. Although the registry record names 95% confidence intervals as the dispersion measure, it reports no interval endpoints for the primary outcome.
A confidence interval around a within-group change would address uncertainty in that change estimate. It would not remove the design limitation of lacking a concurrent comparison group. Statistical precision and causal identification are different issues: an estimate can be precise yet still not isolate the effect of an intervention from other changes over time. This is a general distinction, not a result reported for this study.
How should a before-and-after contrast be read?
The primary endpoint compares baseline with the 12-month assessment. In general, a before-and-after contrast summarizes how the measured outcome differs between two times among those contributing data. The interpretation depends on the population included, measurement consistency, missing observations, and the model used. Without a separate control group, the contrast does not by itself reveal what would have happened over the same period in the absence of the intervention.
The record’s historical appointment-adherence hypothesis compares the study period with the preceding year. General concerns for historical comparisons include changes in record-keeping, opportunity to attend, participant circumstances, or other care processes across periods. The registry record does not report details of this comparison, the data source for appointments, or an analysis method. These are considerations for interpreting such a design, not claims that any particular change occurred in this study.
What do the planning assumptions mean?
The record describes planning illustrations calculated from an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. These are assumptions for illustrating sample-size requirements under a hypothetical two-arm setup. The record distinguishes participants per arm for non-time-to-event outcomes from total event counts for time-to-event outcomes. The illustrations are not the trial’s observed results, and they are not observed power. They do not show that the actual study used two arms; the study is identified as single-group.
In general, sample-size planning depends on the effect a future study aims to detect, outcome variability, significance level, allocation, and analysis assumptions. An illustration based on an observed effect is especially dependent on that effect estimate and its uncertainty. It cannot serve as a substitute for a reported analysis, a design justification, or evidence that the study had a particular probability of detecting an effect. The registry record does not report its own sample-size justification or the numerical planning illustration values.
Why do outcome distributions matter?
The primary outcome is a continuous score, so its analysis concerns a numerical scale rather than a count or time-to-event measure. In general, continuous-outcome models rely on assumptions about the distribution of residuals and the relationship between the outcome and modeled factors. Those assumptions matter for uncertainty estimates and confidence intervals. The record does not report diagnostic checks or a description of the model’s assumptions.
Over-dispersion is a general concern for count outcomes when the observed variability exceeds what a simple count model assumes. It can make standard errors too small if not handled appropriately. The registry record names outcomes involving appointment attendance and health care utilization, but it does not provide their event counts or analysis methods. It is therefore not possible to determine whether over-dispersion arose or how any count outcome was analyzed.
These general explanations clarify the kinds of information needed to evaluate a statistical result. Because the registry record lists 0 analyses and supplies no numerical endpoint estimates, no study-specific assessment of model fit, precision, or statistical significance is possible.
10. Multiplicity, Missing Data and Analysis Populations
The study summary describes multiple questions: the primary hypotheses include self-efficacy, trust, and disease knowledge; it also describes clinic appointment adherence and additional questions about transfusion attendance, health care utilization, and hydroxyurea adherence. The registry record identifies SCSES change as the primary outcome measure and lists clinic appointment attendance as a secondary outcome. It also lists four outcome measures. The record does not explain how these questions were prioritized for statistical testing or whether any adjustment for multiple comparisons was planned.
In general, evaluating multiple outcomes or time points can increase the chance of finding at least one apparently notable result by chance when each is tested separately. Statistical plans may address this through prespecified hierarchies or other approaches. No such approach is reported in the registry record, so it is not possible to determine how multiplicity was handled. The record lists 0 analyses and reports no tests or outcome estimates.
Missing-data methods are also not reported in the registry record. The primary population is described as all participants who participated in the music therapy intervention, but the record does not state how many contributed primary endpoint measurements, whether all scheduled measurements were available, or how incomplete observations were handled. Enrollment is not equivalent to the number included in every analysis. No imputation method, complete-case rule, or sensitivity analysis is reported.
The record does not provide a detailed analysis population definition for the secondary outcome or the additional questions. It is therefore unclear which participants or records contributed to those assessments. These omissions limit assessment of bias and precision, especially for outcomes measured repeatedly or drawn from historical records. They do not establish that data were missing or that any particular method was used.
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 |
|---|---|---|---|
| Music Therapy Group | 0 | 30 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Music Therapy Group | 0 of 30 |
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 |
|---|---|
| Music Therapy Group | 0 of 30 |
The registry record lists 30 participants at risk for serious adverse events and 0 participants affected. For other adverse events, it also lists 30 at risk and 0 affected. Deaths are listed as 0, and no serious adverse event terms are listed.
These figures describe what was entered in the registry record; they do not establish that no adverse events of any kind occurred. The record does not report a safety observation window, definitions used for event collection, or details about how events were solicited and verified. It also does not provide event-level descriptions beyond the absence of listed serious-event terms.
Counts with no affected participants should be read in light of the number at risk and the available follow-up and reporting methods. In general, a zero count does not prove that the underlying risk is zero, and the record does not provide enough information to estimate a precise risk range. The figures also should not be interpreted as a comparison with another treatment, because this is a single-group study without a reported control group.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed, single-group investigation of music therapy for young adults with sickle cell disease during transition. It specifies a primary self-efficacy measure, its scoring direction, and assessments from baseline through 12 months. It also records an enrollment of 30 and describes broader questions concerning trust, knowledge, and adherence.
What the record does not provide is an outcome estimate. It lists 0 analyses and does not report the primary SCSES change, its confidence interval, or numerical results for the other outcomes. Consequently, it cannot show from the posted results whether self-efficacy changed, how large any change was, or how precisely it was estimated. The stated hypotheses are intended questions, not findings.
Even a reported change from baseline would need cautious interpretation. Because the design is single-group, a before-and-after difference would not on its own distinguish an intervention-related change from other influences over time. A concurrent comparison could help address that question, but none is described in the record. The listed historical comparison for clinic appointments is a separate question and does not provide a randomized concurrent control for the primary endpoint.
The endpoint itself is a self-reported scale score, not a direct measure of health care utilization or adherence. The record’s additional questions about appointments, transfusions, hospital utilization, and hydroxyurea adherence should therefore be treated as distinct outcomes with their own measurement and analysis requirements. Their results are not reported in the registry record.
These limitations do not mean the study has no descriptive value. The record defines an outcome and follow-up schedule relevant to a transition-period research question. But without reported estimates, participant counts at assessment times, and analysis details, the available information cannot establish the size or reliability of any observed change, nor support a causal conclusion about the music therapy intervention.
13. Why This Trial Matters Statistically
This study is statistically notable for asking a longitudinal question in a single-group setting. Repeated SCSES assessments at baseline, 3 months, 6 months, 9 months, and 12 months can describe how a measured outcome varies over time. The design also makes clear why a within-group trajectory and an intervention effect are not interchangeable: without a concurrent comparison group, observed change cannot by itself isolate the intervention’s contribution.
The registry record identifies a least-squares mean and a 95% confidence interval for the primary endpoint. Those terms signal that estimation and precision are important to the intended analysis, but the record does not report the model or the resulting estimates. This distinction is useful for readers: endpoint definitions tell what investigators planned to measure, while numerical estimates and methods are needed to judge what the data show.
The study also places a patient-reported self-efficacy scale alongside questions about care engagement and utilization. Statistically, these are different types of outcomes, and each needs clear definitions, denominators, time windows, and analysis choices. The registry record lists a clinic appointment attendance measure and additional questions, but does not report their numerical findings or detailed methods.
Finally, the record illustrates the limits of interpreting a completed study from registry information alone. Completion status and enrollment do not supply outcome results, explain missing observations, or resolve multiplicity across several questions. These distinctions matter when readers assess what can be learned: the record supports a description of the study’s aims and design, but not a quantitative conclusion about changes in its outcomes.
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 primary limitation for interpretation is that the registry record does not report numerical results for the primary endpoint. It lists 0 analyses, so there is no estimated SCSES change, confidence interval, or test result to assess. The same absence of numerical results applies to the listed secondary outcome and other questions.
The single-group design limits causal interpretation. The primary endpoint compares measurements over time among participants receiving music therapy, without a reported concurrent control group. Any observed before-and-after change could reflect the intervention, other changes during follow-up, or a combination; the record does not provide a design feature that separates these explanations.
Important analysis details are not reported in the registry record. These include the model behind the least-squares mean, any covariate adjustment, handling of repeated observations, missing-data methods, and multiplicity procedures. The number of participants contributing data at each assessment is also not reported. Although the enrollment is listed as 30, the number with complete primary outcome data cannot be inferred from that figure.
The intervention description does not report its schedule, duration, or participant-level exposure. The historical comparison for appointment adherence is not described in enough detail to evaluate the source or comparability of the two periods. The record also does not report numerical results for trust, knowledge, transfusion attendance, utilization, or hydroxyurea adherence.
These are limitations of what can be interpreted from the registry record; they do not establish that the study failed to collect or analyze these data outside the record. Further detail is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Music Therapy in Sickle Cell Transition Study — NCT02466555.
- Registry record last updated 2019-09-13; status last verified 2019-08.
- Results first posted 2019-09-13.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT02466555 is listed as a completed, single-group behavioral music therapy study in young adults with sickle cell disease during transition. The registry record lists an enrollment of 30, sponsor University Hospitals Cleveland Medical Center, a start date of 2015-06, and primary completion in 2016-08. Allocation is listed as NA and masking as NONE.
The primary outcome is change from baseline to 12 months in the least-squares mean SCSES score. The scale ranges from 9 to 45, with higher scores indicating higher self-efficacy. The record lists 0 analyses and does not report the primary estimate, its confidence interval, or numerical results for the other outcomes. Details of the statistical model, missing-data handling, and multiplicity procedures are not reported in the registry record.