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 Ready to Sail: Evaluating Sailing's Feasibility as Ergotherapy (NCT06397443), a na na study. The primary outcome is Health-related Quality of Life.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT06397443 |
| Phase | NA |
| Condition | Multiple Osteochondroma, Ollier Disease, Osteogenesis Imperfecta |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Istituto Ortopedico Rizzoli |
| Results posted | Yes |
2. Clinical Question
Population
People with rare skeletal disorders, with multiple osteochondroma, Ollier disease, and osteogenesis imperfecta listed as conditions. The registry describes an enrollment of 8 participants.
Intervention
A 5-day sailing activity, described as sailing-based occupational therapy. The study rationale concerns feasibility and possible changes in physical, social, and psychological well-being.
Comparator
There is no separate control group in this single-group study. The record lists allocation as NA and masking as none, so the design does not provide a like-for-like comparison between treatment groups.
Primary question
The stated primary outcome is health-related quality of life, measured with the EQ-5D instrument at T0: Baseline. The broader study question concerns the feasibility of sailing as an activity for people with rare skeletal disorders. The registry also describes hypotheses about movement confidence, mental health, and social interaction. However, it does not report primary-outcome values or results for those broader questions.
3. Trial Design
Ready to Sail is registered as a prospective, longitudinal, single-group study of sailing as ergotherapy for people with rare skeletal disorders. The intervention is described as a 5-day sailing activity. The record lists enrollment as 8 and names multiple osteochondroma, Ollier disease, and osteogenesis imperfecta among the conditions of interest. The study is marked completed. Its start date is 2024-05-16 and its primary completion date is 2024-09-13.
There is no randomized allocation or separate control arm listed. Allocation is NA, masking is none, and the model is single group. Consequently, the design is suited to describing participants’ experience and measurements over time, but it does not isolate the effect of sailing from changes that could occur for other reasons. A before-and-after comparison, if conducted, would still be a within-group comparison rather than a controlled estimate of treatment effect.
The registry describes comprehensive pre- and post-intervention assessments in its study summary, but the listed primary outcome has the timeframe T0: Baseline. The record does not report a post-intervention primary-outcome measurement or a statistical analysis of change. The full assessment schedule and operational details of the intervention are not reported in the registry record.
The study lists 30 outcome measures, while the record reports no analyses. The phase is NA, and the study type is listed as other. A sample-size justification, a protocol-level statistical plan, and details of any interim analyses are not reported in the registry record. The enrollment figure should therefore be read as a description of the study, not as evidence that a particular precision or ability to detect change was achieved.
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: Of 10 eligibility participants, 8 met inclusion criteria and were included in the study.
Overall Study
| Stage | Patients With Rare Skeletal Disorders |
|---|---|
| STARTED | 8 |
| COMPLETED | 8 |
| NOT COMPLETED | 0 |
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 | Patients With Rare Skeletal Disorders |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 14 (12 to 18) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 4 |
| Race/Ethnicity, Customized: Caucasian | participants | 8 |
| Region of Enrollment: Italy | participants | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Health-related Quality of Life, measured over T0: Baseline.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | score on a scale |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is health-related quality of life, measured using the EQ-5D instrument at T0: Baseline. The registry describes two components: a descriptive system and a visual analog scale. The descriptive system asks about mobility, self-care, usual activities, pain or discomfort, and anxiety or depression. Responses create a health profile that is converted to an Index Value, with the stated scale running from 0, described as absence of life/death, to 1, described as perfect health. The visual analog scale records overall health status from 0, “The worst health you can imagine,” to 100, “The best health you can imagine.”
These measures are used because they capture a participant-reported view of health across several domains, rather than focusing on a single physical task or symptom. The registry identifies the primary outcome’s summary as median with full range. A median describes the central observation in a set of measurements, while a full range describes its lowest and highest values; neither is reported numerically for this outcome in the registry record.
The listed timeframe is baseline, not a change score or a post-intervention assessment. The registry record therefore does not establish whether health-related quality of life changed during the activity. The age-appropriate response options described in the record include a 5-level version for adults and a 3-level version for adolescents and children.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study’s purpose and design, but it does not provide numerical results for the primary health-related quality-of-life outcome. It lists the EQ-5D at T0: Baseline and specifies median with full range as the summary approach. No median, range, post-intervention value, or change estimate is reported. The primary outcome therefore cannot be interpreted as evidence of improvement, worsening, or no change.
The study summary describes feasibility as the primary aim and mentions hypotheses about movement confidence, mental health, and social interaction. It also describes pre- and post-intervention assessments. However, no feasibility result or numerical result for those proposed areas is reported in the registry record. The stated hypotheses are not results. Likewise, the fact that the study is marked completed does not by itself establish whether the activity was feasible under the study’s criteria.
The single-group design matters for interpretation. A change observed within participants, if it were reported, would not on its own show that sailing caused the change. Without a separate control group, the design cannot distinguish an intervention-related change from other explanations, such as ordinary variation over time, expectations, or measurement context. These are general interpretive concerns for uncontrolled before-and-after studies, not claims about what occurred in this study.
The enrollment of 8 indicates a small study context. Small studies can be informative for feasibility and for describing measurements, but estimates may be imprecise and sensitive to individual observations. The registry does not report confidence intervals or other uncertainty estimates for the primary outcome. It also does not report a numerical analysis of the study’s proposed psychological, social, or movement-related outcomes.
The listed safety results show zero serious adverse events, zero other adverse events, and zero deaths in the group described, with 8 at risk in each adverse-event category. These figures describe the registry’s reported safety record; they do not establish that an activity has no risks. The record does not list serious adverse-event terms.
Any sample-size planning scenario should be interpreted as an illustration rather than an observed study result. The stated assumptions refer to a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a fraction of an observed effect. This study is single group and has no randomized comparison, and no observed effect estimate is reported. Such planning assumptions therefore do not describe the study’s actual allocation or provide evidence of observed power.
9. Statistical Methods Explained
Statistical methods help determine what a study’s measurements can support. In this record, the main interpretive constraint is that the study is single group and the registry reports no analyses. There is no reported treatment-versus-control estimate to interpret, and the primary outcome is listed at baseline only. The concepts below are general explanations of how these methods apply to a study with this structure; they do not imply that any unreported analysis was conducted.
What does a median with full range describe?
A median is a measure of the center of ordered observations. It is less affected by an unusually high or low value than a mean, which can make it a useful summary when a sample is small or a measure is skewed. The full range gives the lowest and highest observed values, conveying the spread from one end of the observed data to the other. A range is sensitive to extreme observations and does not describe how values are distributed between its endpoints.
These summaries do not show the uncertainty around an estimate in the way a confidence interval is intended to. A confidence interval is a range of values compatible with the data and a specified statistical procedure; its width conveys precision. General interpretation should distinguish the observed spread among participants from uncertainty about a population-level estimate. The registry identifies median and full range for the primary outcome but reports neither its values nor an interval estimate.
Why does the single-group design limit causal interpretation?
A single-group longitudinal study can describe measurements in participants over time, provided measurements at relevant time points are collected and reported. It has no concurrent control group to indicate what might have happened over the same period without the activity. As a general statistical principle, a pre-to-post difference in an uncontrolled study can reflect the intervention, but it can also reflect other time-related influences, changes in circumstances, measurement variability, or participant expectations. The design alone cannot separate these explanations.
Random allocation, when used in a comparative trial, aims to make groups comparable in expectation at the start of a study. This record lists allocation as NA and does not describe a randomized comparison. Thus, methods that estimate a contrast between randomized arms do not describe the design recorded for this study. A comparison with a participant’s own baseline is not the same as a comparison with a control group.
What would a confidence interval add?
In general, a confidence interval around a change estimate would indicate how precisely that change was estimated under the chosen analysis. A wide interval would be consistent with substantial uncertainty; a narrow interval would indicate greater precision under the model. It would not, by itself, resolve the lack of a control group or prove that a change was caused by the intervention. No confidence interval or change estimate is reported in the registry record.
Statistical significance and practical importance are also distinct concepts. A p-value summarizes how compatible data are with a statistical null model under specified assumptions; it does not measure the size or importance of an effect. An estimate and its uncertainty, interpreted alongside a meaningful threshold set in advance, are needed for a fuller assessment. A threshold for meaningful change is not reported in the registry record.
Why can over-dispersion matter?
Over-dispersion is a general feature of count data in which observed variation exceeds what a simple probability model assumes. When it is present and ignored, standard errors can be too small and confidence intervals too narrow, making results appear more precise than they are. Analysts may use models that allow extra variation, but the suitable approach depends on the outcome and study design. The primary EQ-5D outcome is described as a score, and the registry does not report a count-data analysis or an analysis model. Over-dispersion is therefore a general concept, not a stated feature of these results.
How should planning calculations be read?
Sample-size calculations are planning tools: they estimate the amount of information a future design might need under specified assumptions. The stated assumptions refer to a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They also describe detecting a stated fraction of an observed effect. These assumptions do not match the registry’s single-group design with allocation listed as NA. The registry record does not report the study’s own sample-size justification, and the reported enrollment is not evidence of observed power.
A planning calculation based on an observed effect is especially dependent on that effect estimate and on the assumed design. No observed effect estimate or analysis is reported in the registry record. A planning illustration should not be treated as a forecast of what this completed study detected, nor as evidence that its measurements had a particular level of power.
In general, statistical interpretation also depends on a prespecified analysis plan: how outcomes are summarized, how missing measurements are handled, which participants are included, and how multiple outcomes are addressed. Those details are not reported in the registry record. The absence of an analysis description limits reproducibility and makes it impossible to assess whether the reported outcome approach followed a prespecified plan.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 30 outcome measures and repeats labels for self-esteem and motor coordination among the secondary outcomes. It does not report how the outcomes were organized into families, which were considered confirmatory, or whether any adjustment for multiple comparisons was planned. When many outcomes are tested, the chance of at least one apparently notable result can increase. This is a general statistical issue; without reported analyses, there are no p-values or findings to evaluate for multiplicity in this record.
Missing-data handling is not reported in the registry record. The record does not state whether every enrolled participant completed each assessment, how incomplete questionnaires would be scored, or whether any observations were excluded. In general, missingness can affect both the precision and interpretation of an estimate, particularly if the likelihood of missing data is related to a participant’s health or experience. No missingness assumptions or sensitivity analyses are reported.
The analysis population is also not reported in the registry record. The enrollment figure alone does not indicate which participants contributed to each outcome analysis. The record does not describe eligibility for an analysis set, handling of withdrawals, or whether analyses followed an intention-to-treat principle. In a single-group study, the specific population included in a before-and-after analysis would matter because paired measurements require both relevant observations, but no such analysis is reported.
These omissions do not establish that no analysis or data-handling decisions were made; they mean those details are not reported in the registry record. They constrain assessment of robustness and reproducibility.
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 |
|---|---|---|---|
| Patients With Rare Skeletal Disorders | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Patients With Rare Skeletal Disorders | 0 of 8 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Patients With Rare Skeletal Disorders | 0 of 8 |
The registry reports the adverse-event figures for the group titled Patients With Rare Skeletal Disorders. It lists 8 at risk for serious adverse events and 0 affected, and 8 at risk for other adverse events and 0 affected. Deaths are listed as 0. No serious adverse-event terms are listed.
These are the safety figures recorded for this study, not a comparison of event rates between sailing and a control activity. Because the study is single group, the figures cannot establish whether any event would be more or less likely under another approach. A zero count in a small study is compatible with an event not occurring among the recorded participants; it does not show that the event is impossible or that the activity is risk-free.
The registry record does not describe the method or timing of adverse-event collection, nor does it provide further detail about the safety-monitoring process. It is therefore appropriate to report the posted counts as recorded while avoiding broader conclusions about safety beyond this study’s observed registry figures.
12. What the Result Does, and Does Not, Mean
The record is useful for understanding the intended scope of a feasibility study: a 5-day sailing activity for people with rare skeletal disorders, with the broader aim of assessing well-being and quality of life. It identifies health-related quality of life measured by EQ-5D at baseline as the primary outcome, and describes additional areas of interest including movement confidence, mental health, and social interaction. These descriptions explain the research questions, but they do not constitute evidence of an outcome.
The registry marks the study completed and records enrollment of 8. It does not report numerical primary-outcome results, post-intervention EQ-5D values, a change estimate, or analyses of the proposed secondary outcomes. As a result, the record cannot establish whether quality of life or any other outcome changed, how large a change might have been, or how precise an estimate would be.
Even if within-participant change estimates were available, the single-group design would limit causal conclusions. Without a control group, changes cannot be confidently attributed to the sailing activity rather than other influences. This is a general design limitation, not a judgment about whether participants experienced benefit.
The safety figures are reassuring only in the narrow descriptive sense that the registry reports zero serious and zero other adverse events, and zero deaths, for the listed group. They do not demonstrate that risks are absent. Overall, this record documents a small completed feasibility study and its intended measures, but it does not provide the outcome evidence needed to quantify change or assess a comparative treatment effect.
13. Why This Trial Matters Statistically
Rare skeletal disorders can involve varied physical and social experiences, and the study summary identifies well-being, quality of life, movement confidence, mental health, and social interaction as relevant areas of interest. A feasibility study can be a useful early step when an intervention is being explored in a population for which the registry describes limited treatment options. It can help frame the practical and measurement questions that later research may address, although the registry record does not report feasibility findings for this study.
The statistical importance of the record lies partly in the distinction between a registered research question and a reported result. The primary outcome is an EQ-5D measure at baseline, while the summary describes pre- and post-intervention assessment. Without numerical outcome values or a reported analysis, readers cannot determine what the observed health-status measurements were or whether they changed. Clear outcome reporting is essential even when the aim is exploratory.
The design also illustrates how study structure shapes interpretation. A single-group study can provide descriptive information, but it cannot make a controlled comparison. This distinction matters when interpreting any future estimate of change: precision and size would be relevant, but causal attribution would remain limited by the absence of a control group.
Finally, reporting the summary method, population included in analysis, handling of missing measurements, and approach to multiple outcomes would make the findings easier to assess. Those details are not reported in the registry record. The study’s topic may be important to its target population, but the available record supports only a description of its design, planned measures, and posted safety counts, not a conclusion about efficacy.
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 specific to interpreting this trial is the lack of reported outcome results. The primary outcome is specified at baseline, but the registry record does not report its median or full range, a post-intervention value, or a change estimate. The summary describes pre- and post-intervention assessments, yet the posted primary-outcome timeframe is T0: Baseline. This leaves the relationship between the stated assessment plan and the reported outcome timeframe unclear.
The design is single group, with allocation listed as NA and no masking. There is no separate control group for estimating what might have occurred without the sailing activity. Any within-group change, if reported elsewhere, would therefore have limited causal interpretation. The record also does not report the statistical analysis plan, outcome-specific analysis populations, missing-data procedures, or methods for addressing the number of outcomes.
Enrollment is listed as 8. Estimates from a study of this size may be imprecise, and individual observations can have substantial influence on descriptive summaries. No uncertainty estimates are reported for the primary outcome. The registry does not report a sample-size justification, a clinically meaningful threshold for change, or a protocol-level rationale for the selected measures.
The registry lists 30 outcome measures, but no analyses are reported. It also provides limited detail about intervention delivery, assessment procedures, or adverse-event collection. These reporting gaps prevent a fuller assessment of feasibility, reproducibility, missingness, and the interpretation of the safety counts. They should be understood as limitations of what is reported in the registry record, not proof that such procedures or analyses did not occur.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Ready to Sail: Evaluating Sailing's Feasibility as Ergotherapy — NCT06397443.
- Registry record last updated 2025-03-21; status last verified 2025-03.
- Results first posted 2025-03-21.
- 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
Ready to Sail is a completed, prospective, longitudinal, single-group study sponsored by Istituto Ortopedico Rizzoli. It evaluates a 5-day sailing activity described as sailing-based occupational therapy for people with rare skeletal disorders. The registry lists enrollment of 8, conditions including multiple osteochondroma, Ollier disease, and osteogenesis imperfecta, and no masking or randomized allocation.
The primary outcome is health-related quality of life measured with EQ-5D at T0: Baseline, summarized as median with full range. Although the study summary describes pre- and post-intervention assessments, numerical primary-outcome results and analyses are not reported in the registry record. The record lists zero serious adverse events, zero other adverse events, and zero deaths, with 8 at risk in each adverse-event category. The study is marked completed; the registry does not report an analysis plan or outcome estimates.