Treatment Modification to Reduce Symptom Burden in Hemodialysis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Treatment Modification to Reduce Symptom Burden in Hemodialysis (NCT01775800), a randomized na study. The primary outcome is Number of Participants Recruited, Consented, Randomized and Completed.

2. Clinical Question

In statistical terms, the record describes a randomized comparison of a treatment-management strategy with usual care, alongside an outcome focused on participant flow. It does not report a numerical estimate comparing symptom burden between the arms. Therefore, the registry record cannot establish the size or precision of any difference in symptoms associated with the modified targets. The participant-count outcome also needs arm-specific results to show how many people progressed through recruitment, consent, randomization and completion; those values are not reported in the registry record.

3. Trial Design

The study is registered as a randomized, parallel-group trial with no masking. It lists two arms: treatment modification and usual care. The treatment-modification arm used different targets for blood pressure, parathyroid hormone and serum phosphorus; the usual-care arm received hemodialysis care without those modifications. The record gives an enrollment of 16. It does not state the allocation ratio, the method used to generate the random sequence, or how assignment was concealed.

The study is listed as completed, with a start date of 2013-01 and a primary completion date of 2014-09. Each participant was assessed for 6 weeks, and the total recruitment period is described as 15 months. The record does not give additional visit schedules, follow-up beyond that assessment period, or details about adherence to the assigned targets.

The primary outcome is titled “Number of Participants Recruited, Consented, Randomized and Completed.” Its unit is participants, and its timeframe description refers to participant assessment and the recruitment period. However, the registry record does not report the counts for those stages or define how completion was determined. The listed secondary outcome is symptom burden. The record does not provide a numerical symptom-burden result, the instrument used to measure it, or a detailed assessment schedule for that outcome.

The study is marked as not applicable for phase. The sponsor is The Rogosin Institute. The registry record does not report a detailed statistical analysis plan, sample-size justification, or the specific process used to implement the treatment targets. Consequently, the design can be described as randomized and parallel, but important details for assessing conduct and interpreting comparative results remain unavailable.

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.

Overall Study

Analysis population for the primary outcome, as defined in the registry: This number represents the entire dialysis population

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants Recruited, Consented, Randomized and Completed, measured over Each participant was assessed for 6 weeks; total recruitment period was 15 months.

The listed primary endpoint is a participant count covering recruitment, consent, randomization and study completion. Counts at these stages can describe whether a study enrolled people and how far participants progressed through its conduct. They are useful for understanding feasibility and participant flow, but they are not themselves a measure of symptom burden or of the effect of changing dialysis treatment targets.

The endpoint description says the count represents the entire dialysis population. It also states that each participant was assessed for 6 weeks and that recruitment lasted 15 months. The record does not report separate counts for recruitment, consent, randomization or completion, nor does it specify the operational definition of completion. Thus, the endpoint's label and timeframe are available, but the endpoint results cannot be reconstructed from the record.

Symptom burden is listed as a secondary outcome, consistent with the study's stated aim of examining symptoms such as pain, depression, anxiety and difficulty breathing. The registry record does not identify the measurement instrument, scoring method, or numerical results. Without those details, the reported record does not support a quantitative interpretation of symptom change or a comparison between treatment modification and usual care.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The record identifies the primary outcome as a count of participants at recruitment, consent, randomization and completion. Count outcomes describe how many participants meet a specified event definition or reach a study stage. They differ from continuous outcomes, such as a symptom score, and from time-to-event outcomes, which account for when an event occurs. The listed unit is participants, but the record does not provide stage-specific counts or fully define each stage.

What does randomization contribute?

In general, randomization assigns participants to treatment groups using a chance-based process. Its purpose is to reduce systematic differences between groups at the start of a trial, so that a comparison can be interpreted more fairly. Randomization does not guarantee that groups will be identical, especially in a small trial. The registry record identifies the allocation as randomized but does not describe sequence generation, concealment, stratification or the allocation ratio. Those implementation details therefore cannot be assessed.

What does a parallel design mean?

In general, a parallel design assigns participants to one group and compares outcomes across groups. Here, the recorded groups are treatment modification and usual care. The study is also listed as unmasked, meaning the registry does not identify masking. The record does not state who knew the assignments, whether outcome assessors were masked, or what procedures were used to reduce bias in outcome measurement.

How should count outcomes be read?

A participant count is meaningful only in relation to a clear event definition and the group at risk of experiencing or reaching that event. Recruitment, consent, randomization and completion are distinct stages in the outcome title, but the record gives no separate values or detailed definitions for them. Without stage-specific results, it is not possible to determine where participants may have left the study or to compare progression between arms. Counts alone also do not measure the degree of symptom burden.

What is not reported about the analysis?

The registry record does not report a detailed statistical analysis plan, a prespecified primary contrast, a method for handling missing outcome data, or an analysis population. It does not report whether analyses were adjusted for baseline factors or how repeated symptom measurements, if any, were handled. These choices can affect how estimates are constructed and interpreted. Without the analysis plan and outcome values, no adjusted or unadjusted clinical effect can be evaluated.

In general, a confidence interval communicates uncertainty around an estimated effect. A narrower interval indicates greater precision, while a wider interval indicates less precision, conditional on the model and data. A hypothesis test addresses compatibility with a specified null hypothesis, but a p-value alone does not describe the size or clinical importance of an effect. The record reports no confidence intervals or p-values for the primary or symptom outcomes, so neither precision nor statistical evidence for a comparative clinical effect can be assessed.

What do the planning assumptions describe?

The registry record describes planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between arms and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not observed trial results or observed power. A planning illustration estimates the sample size that would be needed under its assumptions; it does not establish the precision or power achieved by a completed study. The record does not report the underlying observed effect or a sample-size justification for the trial's own design.

For event outcomes in general, over-dispersion occurs when variability exceeds what a simple count model assumes. If present, it can make standard errors too small under an overly restrictive model, resulting in confidence intervals that are too narrow. The record does not report a fitted count model or the information needed to assess over-dispersion. This is a general consideration, not a finding about this trial.

Likewise, general methods for binary outcomes may compare proportions or model the probability of an event, while methods for continuous symptom scores may compare means or model changes from baseline. Choosing a method depends on the outcome scale, study design, missingness and analysis plan. The record does not provide enough information to identify a particular inferential method for symptom burden or to reproduce a comparative analysis.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists symptom burden as a secondary outcome and reports two outcome measures, but it does not give the full outcome definitions or a detailed analysis plan. It does not report whether symptom burden included multiple scales, symptom domains, assessment times or comparisons. Nor does it describe a method for controlling the chance of false-positive findings across multiple analyses. Multiplicity therefore cannot be evaluated from the registry information.

Missing-data handling is also not reported. The record does not provide the number of participants with observed symptom assessments, reasons for missing assessments, or a method for incorporating incomplete data. For the primary participant-count outcome, the record gives no stage-specific results or definition of completion, so attrition cannot be characterized from the listed enrollment alone.

The analysis population is not reported. The registry record does not state whether analyses were planned according to randomized assignment, restricted to participants who completed treatment, or defined in another way. It also does not report whether any participants were excluded from outcome analyses or how such exclusions were handled. These details matter because different analysis populations can yield different estimates and answer different questions.

Without outcome counts, analysis populations and missing-data methods, the record does not permit an assessment of potential bias from incomplete follow-up or selective reporting. These omissions should be treated as uncertainty about what can be inferred from the registry record, not as evidence that missing data or multiplicity problems necessarily occurred.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

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

The registry lists zero serious adverse events and zero other adverse events in the treatment-modification arm, with eight participants at risk for each category. It lists the same counts and at-risk numbers for the usual-care arm. The death count is zero in each arm. These entries describe the recorded event counts and the groups identified as at risk.

A zero count means that no event was entered for that category in the registry record; it does not prove that an event is impossible or that a treatment is free of risk. Safety interpretation depends on how events were defined and collected, the length and completeness of monitoring, and the number of participants observed. The record does not report further details about event ascertainment, severity criteria, follow-up completeness or specific event terms.

The adverse-event entries should be read separately from the symptom-burden outcome. The record's zero adverse-event counts do not provide symptom scores and do not show whether the treatment targets changed pain, depression, anxiety or difficulty breathing. Nor do they establish a difference in safety between groups. With no comparative safety analysis or uncertainty estimates reported, the entries support only a description of the recorded counts.

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

The registry record establishes that a randomized, parallel-group study compared modified blood-pressure, parathyroid hormone and serum-phosphorus targets with usual hemodialysis care. It describes symptom burden as a secondary outcome and participant progression through recruitment, consent, randomization and completion as the primary outcome. It also records enrollment of 16 and the study's completed status.

The record does not report numerical results for the primary endpoint or symptom burden. Accordingly, it does not show how many participants reached each primary-outcome stage, whether completion differed between arms, or whether symptom burden changed or differed across groups. There is no reported effect estimate or confidence interval from which the size and precision of a clinical comparison could be judged.

The zero adverse-event counts are registry entries, not proof of absence of risk. The record does not include enough safety-monitoring detail to assess how comprehensively events were ascertained. Similarly, the absence of reported symptom results should not be interpreted as no effect. It means the registry information does not permit an estimate.

In general, statistical significance and clinical importance are separate considerations. A statistical test evaluates data relative to a model and a null hypothesis, while clinical importance concerns whether an effect is meaningful in context. Neither can be judged for the symptom outcome without its numerical results, measurement details and uncertainty. The record therefore supports an account of the trial's purpose and structure, but not a conclusion about whether modifying treatment targets reduced symptom burden or changed participant completion.

13. Why This Trial Matters Statistically

This trial raises a useful statistical distinction between a study's clinical aim and its registered primary outcome. The rationale focuses on symptom burden and treatment-target changes, whereas the primary outcome is a count of participants moving through recruitment, consent, randomization and completion. Participant-flow information can be important for understanding feasibility, but it does not directly quantify a symptom effect.

Clear outcome definitions and results are necessary to connect the design to the question. For participant counts, stage-specific values and a definition of completion would help readers understand progression through the study. For symptom burden, the instrument, scoring method, assessment timing and between-arm results would be needed to interpret the clinical question. The registry record does not report those details.

The design also highlights why a randomized label alone is not enough to evaluate a trial. Information about allocation procedures, analysis populations, missing-data handling and statistical methods helps determine how robust a comparison may be. Those details are not reported in the registry record. With enrollment of 16, the availability of estimates and uncertainty would be especially important for understanding how much the results can support, but numerical clinical estimates are absent.

Statistically, the record is therefore most informative about the intended comparison and outcome structure, rather than the magnitude or precision of a clinical result. The distinction matters because a completed trial status and a stated enrollment do not substitute for outcome reporting. Transparent reporting of outcomes and analysis methods is essential for readers to assess what the study can establish.

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 numerical results for the registered primary outcome are not reported. The outcome title includes recruitment, consent, randomization and completion, but there are no stage-specific counts or definitions that would allow participant flow to be assessed. Enrollment is recorded as 16, but that alone does not describe completion or retention.

The registry record also does not provide numerical results for symptom burden, despite identifying it as a secondary outcome and describing symptoms as central to the study rationale. It does not identify the measurement instrument or scoring approach. Without these details, neither the outcome's scale nor the size and precision of a comparison can be evaluated.

Important design and analysis details are unavailable, including the randomization procedure, allocation ratio, analysis population, missing-data methods and statistical analysis plan. The record does not report the basis for the trial's sample-size choice or whether interim analyses were planned. These absences limit assessment of bias, precision and the relationship between the registered design and any analysis.

The safety entries report no adverse events in the listed categories, but monitoring and ascertainment details are not reported. Thus, the scope of those counts is unclear. The record also provides no detailed description of usual care or operational guidance for applying the modified targets. These limitations mean that the registry information permits a broad description of the trial, but does not support a detailed evaluation of its clinical effects or safety.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Treatment Modification to Reduce Symptom Burden in Hemodialysis — NCT01775800.
• Registry record last updated 2018-10-09; status last verified 2018-09.
• Results first posted 2018-10-09.
• 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

NCT01775800, “Treatment Modification to Reduce Symptom Burden in Hemodialysis,” is listed as a completed randomized, unmasked, parallel-group study sponsored by The Rogosin Institute. It enrolled 16 participants and compared modified treatment targets for blood pressure, parathyroid hormone and serum phosphorus with usual hemodialysis care. The record lists a start date of 2013-01 and a primary completion date of 2014-09. Each participant was assessed for 6 weeks, and recruitment lasted 15 months.

The primary outcome is a participant count covering recruitment, consent, randomization and completion. Symptom burden is listed as a secondary outcome. Numerical results for these outcomes are not reported in the registry record. Adverse-event entries report zero serious events, zero other events and zero deaths in each arm, with eight participants at risk in each arm. The registry information describes the study question and design, but does not provide numerical clinical outcome estimates or enough analysis detail to assess a treatment effect.