Randomized, Double-Blind Clinical Trial for Parkinson's Disease (Early and Moderate): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Randomized, Double-Blind Clinical Trial for Parkinson's Disease (Early and Moderate) (NCT04928287), a randomized phase 2 study. The primary outcome is Change From Baseline in MDS-UPDRS Part II..

2. Clinical Question

3. Trial Design

This was a randomized, parallel-group, single-center, phase 2 study listed as completed. It compared autologous HB-adMSCs with placebo in people with Parkinson disease. The registry record lists an enrollment of 24 and dates the study start as 2021-06-28 and primary completion as 2023-02-06. These registry entries describe the planned and recorded study, but do not establish how many participants completed each assessment.

The study description includes screening of up to 4 weeks, a 32-week treatment period, and a safety follow-up period of 20 weeks after the last investigational product administration. The primary endpoint timeframe is described separately: baseline through Week 32, follow-up at week 42, and end of study at week 52. The record does not provide visit-level completion counts or explain how the endpoint timing relates to the last administration for each participant.

Randomization is reported, but the sequence-generation method, allocation concealment, and allocation ratio are not reported in the registry record. The masking field says quadruple, while the study description also calls the trial double-blind. The record does not identify the masked parties or provide details about blinding procedures. It lists HB-adMSCs as a biological intervention and placebo as sterile saline solution 0.9%; the description says multiple HB-adMSCs administrations but does not report the dose or schedule.

The registry record includes secondary outcomes for MDS-UPDRS Parts I, III, and IV and several Neuro-QOL domains. It lists 44 outcome measures overall. The record does not report the results for the primary endpoint, and its analysis listing contains no analyses. Completion status therefore should not be mistaken for a reported efficacy result.

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

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 Change From Baseline in MDS-UPDRS Part II., measured over Baseline through Week 32, Follow-up at week 42 and End of Study at week 52.

The primary endpoint is change from baseline in MDS-UPDRS Part II. The registry describes this as a mean continuous measure with standard deviation as its dispersion measure. Part II assesses motor aspects of experiences of daily living. The record says it contains 13 items, each rated from 0 to 4, and that its score ranges from 0 to 52. Higher values represent a worse outcome. The principal investigator evaluates the answers during study visits.

In general, a change-from-baseline endpoint compares a participant’s follow-up score with that participant’s starting score. Comparing these changes between randomized groups can describe whether the groups’ score trajectories differ over the specified period. The interpretation depends on the way change is summarized and on the uncertainty around the group comparison. Those result statistics are not reported in the registry record, so the direction, size, and precision of the primary comparison cannot be described.

Part II focuses on daily-living motor experiences, while the registry lists other MDS-UPDRS parts as secondary outcomes. This endpoint therefore addresses a particular aspect of the scale rather than every aspect of Parkinson disease or every outcome included in the study. The record’s description of score meaning supports interpreting higher scores as worse, but it does not report a threshold for judging whether a change is important to participants.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the primary endpoint as a mean change from baseline on a continuous score and names standard deviation as the measure of dispersion. It does not report the statistical analysis used to compare groups, the endpoint values, or measures of uncertainty. The explanations below describe general statistical concepts; they should not be read as a description of methods confirmed for this study.

What does a mean change represent?

In general, a mean change summarizes the average difference between follow-up and baseline scores within a group. A comparison between groups can then focus on the difference in these average changes. This approach is distinct from reporting only the score at follow-up: change incorporates the starting measurement, while a follow-up-only comparison does not express the result in that way. The registry record does not report which specific calculation or model was used for the primary comparison.

A mean is useful for summarizing a continuous measure, but it does not show how individual participants’ scores were distributed. The standard deviation describes variation among observations around the mean. It is not the same as the uncertainty around the mean or around a between-group effect. The registry names standard deviation as the endpoint’s dispersion measure but does not provide its observed value.

Why does the between-group estimate matter?

In a randomized study, the central efficacy comparison is generally the difference between groups, rather than whether one group changed from its own baseline. A within-group change alone cannot separate changes associated with the intervention from changes that might also occur under placebo or over time. The registry record does not report a between-group estimate for MDS-UPDRS Part II, so the comparison that addresses the primary question is unavailable.

In general, an effect estimate gives the magnitude and direction of a comparison on the outcome’s scale. A confidence interval conveys uncertainty in that estimate. These quantities should be read together: an estimate alone can look precise while uncertainty remains substantial, and a small p-value does not measure the size of an effect. No such primary endpoint estimate or uncertainty measure is reported in the registry record.

What role does randomization play?

Randomization is intended to make assignment to study groups independent of participants’ measured and unmeasured characteristics, on average. This supports a comparison that is less vulnerable to systematic differences at the outset. It does not guarantee that every group characteristic will be balanced, especially in a small study, and it does not remove the need to report how outcomes were analyzed. The record gives the allocation as randomized but does not report the sequence-generation process, concealment procedures, or allocation ratio.

Blinding can reduce the influence of expectations on participant reports, clinical assessments, and study decisions. The registry records quadruple masking and describes the trial as double-blind, but does not identify the masked roles or describe the procedures. The record therefore does not permit a detailed assessment of how masking was implemented or whether it was maintained.

How should planning calculations be read?

In general, sample-size planning calculations describe how many participants might be needed under specified assumptions about an effect, variability, significance level, allocation, and statistical model. They are design illustrations, not evidence of the effect actually observed. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation, with the effect to detect defined as a stated fraction of the observed effect. No resulting sample-size values are reported in the registry record.

The planning description says that the illustration is based on the observed effect, but the primary outcome result is not reported. The record also flags the primary outcome as having several categories or time points and says it was not reduced to one comparison. In the absence of a reported primary estimate and a single comparison, planning illustrations cannot be interpreted as observed power or as a test of the study’s actual findings.

Why do missing-data and analysis choices matter?

In general, missing follow-up measurements can affect an analysis if participants with observed data differ from those without it. Statistical approaches make assumptions about the missing outcomes, and those assumptions can influence estimates and uncertainty. The registry record does not report the amount or pattern of missing primary endpoint data, the missing-data method, or whether sensitivity analyses were conducted.

The analysis population also matters. An analysis of everyone as randomized answers a different operational question from an analysis limited to participants who completed treatment or provided particular measurements. The analysis population for the primary endpoint is not reported in the registry record. Without that information, and without endpoint results, readers cannot assess the analysis basis or judge the robustness of the primary comparison.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary endpoint of change from baseline in MDS-UPDRS Part II and secondary outcomes including MDS-UPDRS Parts I, III, and IV and Neuro-QOL domains. It reports 44 outcome measures overall. Looking at multiple outcomes or time points can increase the chance that at least one result appears notable by chance. In general, a prespecified testing strategy or multiplicity adjustment can help control that issue, but the registry record does not report a multiplicity plan or adjustment.

The registry also flags the primary outcome as having several categories or time points and not being reduced to one comparison. The primary measure’s timeframe includes baseline through Week 32, follow-up at week 42, and end of study at week 52. The record does not explain which time point or comparison was intended to carry the primary statistical conclusion, or how results across those time points would be handled.

Missing-data methods are not reported in the registry record. The record does not give the number of missing primary measurements, reasons for missingness, or any method for addressing incomplete observations. The analysis population is also not reported. These omissions make it difficult to assess whether the planned comparison would include all randomized participants or a subset defined by treatment exposure or measurement availability.

No primary analysis results are listed, and the analysis listing contains no analyses. The record does not report an analysis plan, interim analyses, or sensitivity analyses. These items should be treated as unreported rather than inferred from the phase, design, or completion status. As a result, readers cannot determine how multiplicity, incomplete data, or analysis-population choices affected the primary finding.

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.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: dyspnea (6.7%).

The registry reports serious and other adverse-event counts by group. For HB-adMSCs, 1 serious adverse event was reported among 15 participants at risk; for placebo, 0 were reported among 9 participants at risk. Other adverse events were reported for 15 participants at risk in the HB-adMSCs group and 9 participants at risk in the placebo group. Deaths were reported as 0 in each group. These are counts, not a comparison adjusted for follow-up time or other factors.

Dyspnea is listed as the top serious-event term at 6.7%. The registry’s top-term summary does not identify the group associated with that entry. The term and percentage should therefore be read separately from the group-level serious-event counts, rather than assigned to a particular group based on that summary.

In general, serious adverse events are classified by seriousness, which is distinct from how severe a symptom is. Event counts also do not describe duration, recurrence, timing, or the relation of an event to treatment. Those details are not reported in the registry record. The observed counts are descriptive safety information; they do not establish that an intervention caused or prevented an event, and they do not provide a measure of the primary efficacy outcome.

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

The registry record establishes that this was a completed, randomized, parallel-group phase 2 study comparing autologous HB-adMSCs with saline placebo in people with Parkinson disease. It identifies change from baseline in MDS-UPDRS Part II as the primary endpoint and describes its score direction and assessment timeframe. The posted results do not include the primary endpoint estimate or its statistical uncertainty.

That distinction defines what can and cannot be concluded. The study’s completion status, planned enrollment, intervention description, and endpoint definition do not reveal the observed difference between groups. Without a reported estimate, the size and direction of any change cannot be described. Without a confidence interval or other uncertainty measure, the precision of a comparison cannot be assessed. The record therefore does not support a conclusion about whether the groups differed on the primary measure.

The registry’s safety counts describe adverse events recorded in the groups, including serious-event counts and deaths. They are not a substitute for a primary efficacy result. Nor do these counts, on their own, establish whether an event was caused by an intervention. The record does not provide detailed event timing, attribution, or participant-level context.

In general, a trial result applies most directly to the participants, interventions, and outcome measurements studied, and its interpretation depends on design and analysis details. Here, eligibility criteria, participant characteristics, the primary analysis population, missing-data methods, and the statistical model are not reported in the registry record. Those gaps limit assessment of both applicability and robustness.

The appropriate reading is not that the intervention did or did not have an effect, but that the registry record does not report the primary outcome result needed to evaluate that question. Further claims about benefit, lack of benefit, or the likely result of a larger study would go beyond the posted information.

13. Why This Trial Matters Statistically

This study is statistically relevant because it sets up a randomized comparison of an intervention with placebo and identifies a patient-relevant daily-living measure as its primary endpoint. Randomization can support a fair comparison, while the MDS-UPDRS Part II score provides a defined outcome with an explicit direction: higher values represent a worse outcome. These design features make clear what the primary question was intended to address.

At the same time, the registry record illustrates why design information and outcome reporting must be considered separately. A trial can be marked completed and still lack a posted primary estimate, confidence interval, and analysis description. Without those results, the design cannot be translated into an assessment of the observed effect or its precision. The enrollment figure and adverse-event counts do not fill that gap.

The record also identifies multiple secondary outcomes and several primary-endpoint time points, while not reporting a multiplicity strategy or a single primary comparison. In general, specifying which comparison is primary helps readers distinguish the main confirmatory question from additional exploratory outcomes. Clear reporting of missing-data methods and analysis populations is also needed to understand how the randomized groups were represented in the final analysis.

These statistical considerations matter beyond this study: estimates, uncertainty, and transparent analysis methods are necessary to interpret clinical-trial findings. In this case, the registry describes the intended comparison and some safety counts, but does not report enough primary-outcome information to judge the result.

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 trial-specific limitation of the registry record is that it does not report the primary MDS-UPDRS Part II result. There are no posted group changes or between-group estimate, and no confidence interval or p-value for that endpoint. The analysis listing contains no analyses. Thus the primary comparison cannot be evaluated from the registry information.

Important design and analysis details are also absent. The record does not report participant characteristics, detailed eligibility criteria, the randomization method, allocation concealment, the allocation ratio, the primary analysis population, the statistical model, or the handling of missing measurements. Although the study is described as double-blind and the masking field says quadruple, the masked roles and blinding procedures are not reported.

The primary endpoint covers multiple assessment times, and the registry notes that the outcome has several categories or time points without reducing it to one comparison. The record does not explain how those time points were handled in analysis or report a multiplicity strategy. It lists 44 outcome measures, but does not provide analysis results for them.

The adverse-event information is limited to group counts, deaths, and a top serious-event term. The record does not provide event timing, duration, attribution, or detailed participant-level context. The top-term summary does not identify the associated group. These reporting limits prevent a fuller interpretation of safety patterns and should not be replaced with assumptions.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Randomized, Double-Blind Clinical Trial for Parkinson's Disease (Early and Moderate) — NCT04928287.
• Registry record last updated 2026-04-15; status last verified 2026-03.
• Results first posted 2024-05-20.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

The registry lists a completed, randomized, parallel-group, phase 2 study of autologous HB-adMSCs versus saline placebo for Parkinson disease. It describes a single-center trial with planned enrollment of 24, a start date of 2021-06-28, and primary completion on 2023-02-06. The primary endpoint is change from baseline in MDS-UPDRS Part II, assessed through the stated follow-up periods.

The record includes adverse-event counts by group, with 0 deaths reported in each group, and lists dyspnea at 6.7% as the top serious-event term. It also lists multiple secondary outcomes. However, the primary endpoint result, statistical uncertainty, analysis methods, missing-data approach, and analysis population are not reported in the registry record. The posted information therefore describes the trial’s design and selected safety counts, but does not permit assessment of the primary efficacy comparison.