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 An Online Health Tool About Down Syndrome: Down Syndrome Clinic to You (NCT04227197), a randomized na study. The primary outcome is Number of Participants With Indicated Evaluations That Were Completed or Recommended by the PCP.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04227197 |
| Phase | NA |
| Condition | Down Syndrome |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 281 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Massachusetts General Hospital |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned caregivers of people with Down syndrome. Its primary outcome counted people with Down syndrome whose PCP completed or recommended indicated evaluations. The registry record lists enrollment of 281 participants, but further participant characteristics are not reported in the registry record.
Intervention
Caregivers assigned to Down Syndrome Clinic to You (DSC2U) completed a questionnaire and received online access to a personalized Caregiver Checklist and PCP plan. They were encouraged to share and discuss that plan at their next wellness visit with their PCP.
Comparator
Caregivers assigned to the control group received usual care for 7 months and did not receive DSC2U during that period. The registry description says they received the online personalized health assessment tool after 7 months.
Primary question
The primary outcome concerned whether the PCP completed or recommended five evaluations indicated by national guidelines for Down syndrome: celiac screen, sleep study, thyroid test, audiogram, and ophthalmology evaluation. The registry record does not report a single summarized comparison between groups for this outcome.
3. Trial Design
The registry describes a randomized, controlled, parallel-group trial with no masking. Its brief summary characterizes it as a national, two-arm trial of caregivers of people with Down syndrome. Massachusetts General Hospital is listed as the sponsor. The record lists the trial as completed, with a start date of 2017-10-03, a primary completion date of 2019-05-15, and enrollment of 281.
Caregivers in the intervention group completed the DSC2U questionnaire and received online access to a personalized Caregiver Checklist and PCP plan. They were encouraged to share and discuss the PCP plan with their PCP at their next wellness visit. The control group received usual care for 7 months and did not receive DSC2U during that period; the record says the tool was made available to them after 7 months.
The primary outcome was assessed 7 months after the participant's scheduled PCP visit. It counted participants for whom the PCP completed or recommended the specified evaluations. The registry identifies five evaluations: celiac screen, sleep study, thyroid test, audiogram, and ophthalmology evaluation. The record does not explain how randomization was generated, whether assignment was concealed, or how the study handled deviations from assigned groups.
The phase is listed as not applicable. Additional details about recruitment sites, eligibility criteria, participant characteristics, or the rationale for the enrollment target are not reported in the registry record.
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: We assessed 645 caregivers for eligibility through the study website, and 281 were consented. After consent, there were 51 consented caregivers who were excluded from the study prior to randomization because they did not complete the baseline survey, changed their primary care provider visit to a date outside of the study window, or self-withdrew. As such, 230 caregivers were randomized into assignment groups.
Randomization
| Stage | Intervention Group | Control Group |
|---|---|---|
| STARTED | 117 | 113 |
| COMPLETED | 115 | 113 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Intervention Group | Control Group |
|---|---|---|
| Protocol Violation | 1 | 0 |
| Discontinued participation because uncomfortable sharing information | 1 | 0 |
Primary Care Provider (PCP) Visit
| Stage | Intervention Group | Control Group |
|---|---|---|
| STARTED | 115 | 113 |
| COMPLETED | 113 | 113 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Intervention Group | Control Group |
|---|---|---|
| Did not complete PCP visit | 2 | 0 |
2-week Follow-up Survey for Caregivers
| Stage | Intervention Group | Control Group |
|---|---|---|
| STARTED | 113 | 113 |
| COMPLETED | 101 | 108 |
| NOT COMPLETED | 12 | 5 |
Reasons for not completing this period:
| Reason | Intervention Group | Control Group |
|---|---|---|
| Not responsive | 7 | 5 |
| Lost to Follow-up | 5 | 0 |
2-week Follow-up Survey for PCP's
| Stage | Intervention Group | Control Group |
|---|---|---|
| STARTED | 114 | 113 |
| COMPLETED | 94 | 88 |
| NOT COMPLETED | 20 | 25 |
Reasons for not completing this period:
| Reason | Intervention Group | Control Group |
|---|---|---|
| Not responsive | 20 | 25 |
Analysis population for the primary outcome, as defined in the registry: People with Down syndrome
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: Baseline characteristics of people with Down syndrome
| Characteristic | Unit | Intervention Group | Control Group | Total |
|---|---|---|---|---|
| Participants analysed | 117 | 113 | 230 | |
| Age, Customized: < 2 years | Participants | 10 | 8 | 18 |
| Age, Customized: 2 to 4 years | Participants | 29 | 28 | 57 |
| Age, Customized: 5 to 7 years | Participants | 17 | 16 | 33 |
| Age, Customized: 8 to 12 years | Participants | 24 | 24 | 48 |
| Age, Customized: 13 to 17 years | Participants | 12 | 11 | 23 |
| Age, Customized: 18 to 25 years | Participants | 13 | 11 | 24 |
| Age, Customized: 26 years or older | Participants | 12 | 15 | 27 |
| Sex: Female, Male: Female | Participants | 54 | 52 | 106 |
| Sex: Female, Male: Male | Participants | 63 | 61 | 124 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 15 | 13 | 28 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 101 | 98 | 199 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 | 2 | 3 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Asian | Participants | 3 | 1 | 4 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Black or African American | Participants | 5 | 7 | 12 |
| Race (NIH/OMB): White | Participants | 101 | 100 | 201 |
| Race (NIH/OMB): More than one race | Participants | 5 | 3 | 8 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 2 | 3 |
| Primary health care insurance: Public (e.g. Medicaid) | Number of participants | 32 | 29 | 61 |
| Primary health care insurance: Private (e.g. Blue Cross) | Number of participants | 84 | 83 | 167 |
| Primary health care insurance: None (e.g. self-pay) | Number of participants | 1 | 1 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Indicated Evaluations That Were Completed or Recommended by the PCP, measured over 7 months after the participant's scheduled PCP visit.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with Down syndrome for whom the PCP completed or recommended indicated evaluations. The registry describes five evaluations, covering screening or assessment related to celiac disease, sleep, thyroid function, hearing, and vision. The time frame is 7 months after the participant's scheduled PCP visit.
This endpoint focuses on guideline-related care processes rather than a direct measure of a health condition. A completed evaluation and a recommended evaluation are both included in the stated outcome. These are distinct actions: a recommendation does not establish that an evaluation was subsequently completed. The registry record does not describe how the outcome combined or categorized these actions, or how it handled a participant with more than one indicated evaluation.
Counting participants can give a practical view of whether PCPs addressed recommended evaluations during the specified follow-up period. However, a count alone does not show which specific evaluations were addressed, how many were addressed for each person, or the reasons an evaluation was not completed or recommended. Those details are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes the trial question and its design but does not report a single primary-outcome comparison. It therefore does not establish from the posted results whether the intervention and control groups differed in the number of participants whose PCP completed or recommended the indicated evaluations. An effect estimate, confidence interval, and p-value for a primary comparison are not reported in the registry record.
The primary outcome encompasses five different evaluations and includes either completion or recommendation by the PCP. The record notes that the outcome has several categories or time points and was not reduced to one comparison. Without a clearly stated summary contrast and its results, the clinical importance and statistical precision of a group difference cannot be assessed from the registry record. Nor can the contribution of each evaluation or of recommendation versus completion be distinguished.
Enrollment of 281 is a trial-level figure, not a reported outcome denominator for the primary comparison. The safety record lists 117 participants at risk in the intervention group and 113 in the control group; those figures should not be treated as the primary-outcome analysis populations because the registry does not identify them as such. The number analyzed for the primary endpoint, and the handling of missing outcome data, are not reported in the registry record.
For context, the general meaning of a confidence interval is a range expressing uncertainty around an estimated effect. A narrow interval indicates greater precision than a wide one, while an interval spanning values consistent with different possible effects can leave the result uncertain. No interval is reported for this primary outcome, so its precision cannot be described.
The posted record does not give a basis for claiming that the intervention changed care, or that the groups were equivalent. The absence of a reported primary comparison is not itself evidence of no effect. The interpretation must remain limited to the design and endpoint definitions recorded, alongside the fact that the registry lists the trial as completed.
9. Statistical Methods Explained
The registry describes a randomized, parallel-group study with no masking. In general, random allocation is intended to make assigned groups comparable in expectation, reducing the risk that systematic differences at enrollment explain an observed contrast. Randomization does not guarantee that groups will be alike in every characteristic, and the record does not provide enough detail to assess the randomization procedure or baseline balance. No masking means the record does not describe blinding of participants or study personnel; the effect of this on outcome assessment cannot be determined from the registry entry.
The primary outcome is a count of participants with an event, with the event defined as a PCP having completed or recommended indicated evaluations. This is a binary-type outcome at the participant level: a participant either meets a specified outcome definition or does not. The registry also describes five constituent evaluations. It does not state in the available primary outcome description precisely how the participant-level count is constructed across those evaluations, so a specific aggregation rule should not be assumed.
In general, a comparison of binary outcomes can be expressed using a difference in proportions, a ratio, or another model-based measure. Each expresses a different aspect of the contrast, and interpretation depends on the outcome definition and analysis method. The registry record does not report a primary effect estimate or identify the model used for a primary comparison. No particular measure should therefore be attributed to the trial.
A confidence interval gives information about the uncertainty and precision of an estimate, whereas a p-value addresses compatibility between observed data and a specified statistical model under a null hypothesis. Neither replaces the estimated effect or its clinical context. The registry record does not report a confidence interval or p-value for a single primary-outcome comparison. The significance level of 0.05 appears in the stated assumptions for a planning illustration; that is not enough to establish the trial's own analysis threshold, which is not reported in the registry record.
The record lists 5 outcome measures and 13 analyses. It also identifies several categories or time points for the primary outcome and states that these were not reduced to one comparison. These details matter because multiple outcomes, comparisons, or time points can create multiple opportunities to observe a result that appears notable. In general, multiplicity procedures address that issue by controlling or describing the consequences of making multiple inferences. The registry record does not report whether such procedures were planned or used.
The stated sample-size assumptions describe a planning illustration based on the observed effect, not the trial's own design. They specify a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation, with the effect to detect defined as a stated fraction of the observed effect. In general, such an illustration asks how many participants per arm would be needed under chosen assumptions; it is not observed power and does not establish the original enrollment rationale. The registry record does not report the trial's sample-size justification or the numerical results of that illustration.
Over-dispersion is a general concern when counts vary more than a statistical model expects; it can make standard errors too small if ignored. The primary endpoint is a participant count for an outcome, not a reported model for repeated counts. Whether an analysis involved over-dispersed data or required a correction is not reported in the registry record. The record also does not describe the primary analysis population, missing-data methods, covariate adjustment, or any interim analyses.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 5 outcome measures and 13 analyses, as well as secondary outcomes concerning caregiver experience, quality of life at 2-week and 7-month follow-up, and PCP experience. The primary outcome itself includes several categories or time points and was not reduced to one comparison in the registry record. These features raise questions about which outcomes and comparisons were designated as primary for inference and how multiplicity was addressed. The multiplicity plan is not reported in the registry record.
In general, when many outcomes, comparisons, or time points are examined, the chance of obtaining an apparently unusual result can increase. Statistical methods may adjust for multiple testing, or investigators may identify a limited set of primary comparisons in advance. The record does not say whether either approach was used, so no adjustment or hierarchy should be assumed.
The registry does not report the number of participants included in the primary-outcome analysis, the amount or pattern of missing outcome data, or the method used to handle missingness. It also does not identify an intention-to-treat, per-protocol, or other analysis population. These omissions limit assessment of whether the reported enrollment corresponds to the population contributing to any outcome analysis. The safety at-risk figures do not establish the primary analysis denominators.
In general, missing data can affect an estimate if the likelihood of missingness is related to group assignment or outcome status. The direction and size of any such effect depend on the data and assumptions used. Since the registry record does not report missing-data information or methods, the potential influence of missingness cannot be evaluated.
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 |
|---|---|---|---|
| Intervention Group | 0 | 117 | 0.0% |
| Control Group | 0 | 113 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Intervention Group | 0 of 117 |
| Control Group | 0 of 113 |
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 |
|---|---|
| Intervention Group | 0 of 117 |
| Control Group | 0 of 113 |
The safety record lists 117 participants at risk in the intervention group and 113 in the control group. It reports 0 serious adverse events, 0 other adverse events, and 0 deaths for each group. No top serious adverse event terms are listed.
These figures describe what the registry safety entry records; they do not provide a detailed account of how events were defined, collected, or reviewed. The registry record does not provide event narratives, severity criteria, or an explanation of the at-risk denominators. They should not be assumed to match the analysis populations for the primary outcome.
In general, observing no recorded events does not establish that an event is impossible or that risk is absent. The ability to assess uncommon harms depends on factors such as the number followed, duration of follow-up, and completeness of event ascertainment. The available registry figures alone do not resolve those questions, and they do not support a broader safety conclusion.
12. What the Result Does, and Does Not, Mean
The registry record describes a randomized comparison of an online caregiver tool with usual care and defines a primary outcome related to PCP completion or recommendation of five evaluations for people with Down syndrome. It also records the follow-up time frame and reports no serious adverse events, other adverse events, or deaths in the safety entries for either group.
However, no single primary-outcome comparison or corresponding statistical result is reported. The posted information therefore does not show the direction or magnitude of a group difference, nor how precisely such a difference was estimated. It cannot support a conclusion that DSC2U improved, worsened, or left unchanged the primary outcome. A missing result in the registry is not equivalent to a finding of no difference.
The outcome combines recommendation and completion, which represent different points in care. It also spans multiple evaluations. Without separate results, it is not possible to determine whether any particular evaluation, or one type of PCP action, accounted for a result. The registry record does not report whether an evaluation was completed after recommendation, or the reasons for actions not taken.
The safety figures are limited to the registry's recorded counts and at-risk groups. They do not establish the absence of all possible harms. Likewise, trial enrollment does not reveal how many participants contributed to each outcome analysis.
In general, a trial result is most informative when an outcome definition, group estimates, an effect measure, and uncertainty are all available. Here, the record supplies important design and endpoint information but not the primary comparison needed to quantify the trial's findings.
13. Why This Trial Matters Statistically
This trial addresses a statistical question about a care process: whether a personalized online tool for caregivers is associated with PCP completion or recommendation of guideline-indicated evaluations. The random allocation and parallel-group design provide a framework for comparing intervention and usual-care groups. The primary endpoint also illustrates that a clinically relevant process outcome can combine multiple evaluations and different actions by a clinician.
That structure makes clear why precise endpoint definition matters. A participant-level count can be useful, but its interpretation depends on how the component evaluations are combined and whether recommendation and completion are treated identically. The registry record names the five evaluations but does not report a single primary comparison or enough analysis detail to clarify all of those points.
Statistically, the trial also highlights the importance of distinguishing an enrolled population from an analysis population and of reporting the handling of missing data. Enrollment is listed, but primary-outcome denominators and missing-data methods are not. The record lists multiple outcomes and analyses without reporting a multiplicity plan, leaving uncertainty about how inferential claims were organized.
The posted record is therefore useful for understanding the intervention, comparison, endpoint, and safety counts, while also showing the limits of interpreting a completed trial without its primary effect estimate and uncertainty. Those reporting details are necessary to assess what the data support and how precisely they support it.
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 interpretation is that the registry record does not report a single primary-outcome comparison, effect estimate, confidence interval, or p-value. It also does not report the primary analysis population or missing-data handling. As a result, the magnitude, direction, and precision of any group contrast cannot be assessed from the posted results.
The endpoint includes five distinct evaluations and counts either completion or recommendation by the PCP. The registry description does not explain the exact participant-level aggregation rule, or give separate results for each evaluation and type of action. This limits assessment of what the endpoint would mean in practice even if a combined comparison were available.
The record lists no masking, but does not describe how outcome information was collected or assessed. It does not report how randomization was generated or concealed, or provide participant characteristics that would help readers understand the enrolled population. These details are not reported in the registry record and should not be inferred from the trial design label.
The registry lists multiple outcome measures and analyses but does not report a multiplicity plan. It also does not report the trial's sample-size justification or statistical analysis plan. Although planning assumptions are described for an illustration based on the observed effect, those assumptions do not establish the original design rationale or the precision of a primary result.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: An Online Health Tool About Down Syndrome: Down Syndrome Clinic to You — NCT04227197.
- Registry record last updated 2022-06-14; status last verified 2022-03.
- Results first posted 2022-06-14.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry describes NCT04227197, “An Online Health Tool About Down Syndrome: Down Syndrome Clinic to You,” as a completed randomized, controlled, parallel-group trial with no masking. Massachusetts General Hospital is listed as sponsor. Enrollment is listed as 281; the record gives a start date of 2017-10-03 and a primary completion date of 2019-05-15.
The intervention was DSC2U, an online health tool for caregivers that included a personalized Caregiver Checklist and PCP plan. The control group received usual care for 7 months before receiving the tool. The primary outcome counted participants whose PCP completed or recommended five indicated evaluations, 7 months after the participant's scheduled PCP visit.
The registry record does not report a single primary-outcome comparison or statistical result. Safety entries report 0 serious adverse events, 0 other adverse events, and 0 deaths in each group.