Lundbeck TOMs Orthostatic Hypotension: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Lundbeck TOMs Orthostatic Hypotension (NCT04510922), a non-randomized phase 4 study. The primary outcome is Orthostatic Hypotension Questionnaire (OHQ) Score.

2. Clinical Question

Orthostatic hypotension is described in the registry as a reduction in blood pressure upon standing from a seated position. The record notes symptoms such as dizziness, lightheadedness, feeling faint, or feeling as though one might black out while standing, and describes potential effects on balance and walking. These clinical features motivate measuring symptoms and daily activities as well as assessing gait and postural stability.

The study's stated focus is feasibility and effect-size finding, rather than a randomized comparison of treatments. Its listed primary outcome captures participants' reported symptoms and activity limitations, while the study aim also highlights objective technology-based measures. The registry record does not report how those measures were compared with questionnaire scores or how any change was evaluated. It therefore does not establish whether the devices improved detection or treatment response.

3. Trial Design

The registry describes NCT04510922, titled “Lundbeck TOMs Orthostatic Hypotension,” as a phase 4 study with a non-randomized allocation, no masking, and a single-group model. The record lists an enrollment of 9 and a completed status. The study start date is 2019-02-11, and the primary completion date is 2020-12-31. These dates describe the registry record; they do not indicate when individual participants were assessed.

The listed conditions are orthostatic hypotension and idiopathic Parkinson disease. The brief summary frames the project around technology-based assessment: a computerized system for analyzing walking abnormalities in clinical settings and a wearable sensor for detecting postural unsteadiness in the home environment. It states that the main aim is to evaluate whether these technologies may improve detection of complications and response to medical therapies.

The intervention record lists “Droxidopa 100 MG [Northera]” and describes the arm as “100-600mg droxidopa TID.” Both details are retained as recorded; the registry does not explain the relationship between the intervention label and the broader dose range. The primary outcome is an Orthostatic Hypotension Questionnaire score with a 1 month timeframe.

A single-group, non-randomized design does not include the randomized control comparison needed to separate intervention effects from other explanations for changes over time. No control arm is listed, and the record does not describe a comparison with usual care or another treatment. Masking is listed as none. Further details about visit schedules, device procedures, treatment duration, or the order of assessments are not reported in the registry record. The record labels the study as a feasibility and effect-size finding study, but does not report a protocol rationale or sample-size justification.

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 Orthostatic Hypotension Questionnaire (OHQ) Score, measured over 1 month.

The primary endpoint is the Orthostatic Hypotension Questionnaire (OHQ) score, recorded as a mean with standard deviation and assessed at 1 month. The registry describes the measure through two components: the Orthostatic Hypotension Symptom Assessment (OHSA) and the Orthostatic Hypotension Daily Activities Scale (OHDAS). Each is described as having a range of 0-10, and the record states that 10 items are measured on a Likert scale, with 10 representing the worst possible score.

Symptom and daily-activity measures address different aspects of orthostatic hypotension. Symptoms capture the participant's reported experience, while daily-activity items concern the effect of symptoms on functioning. This kind of patient-reported endpoint can complement objective measures of walking or postural stability: a device-based signal and a report of symptoms need not represent the same aspect of a condition.

The registry does not report the observed OHQ mean, standard deviation, change from baseline, or a comparison value. It also does not explain how the OHSA and OHDAS items are combined into the reported OHQ score, whether higher scores were analyzed as worsening, or how incomplete item responses were handled. Although the description identifies the worst score for the item scale, those additional scoring and analysis details are not reported in the registry record. Without numerical endpoint results, the magnitude and precision of any change cannot be assessed.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The design determines what a statistical result can support. Here, the registry describes a single-group, non-randomized study with no masking. A single-group design summarizes observations from one group rather than comparing randomized groups. If measurements are taken at more than one time, a within-group change can describe what happened over time in that group. It cannot, by itself, distinguish an intervention effect from other changes that could occur during the same period. The registry record does not report a control group or a randomized treatment allocation.

What does a mean and standard deviation describe?

The primary outcome is specified as a mean with standard deviation. A mean is a measure of the central location of observed scores. The standard deviation describes how dispersed individual scores are around that mean. These two summaries answer different questions: the mean describes an average score, while the standard deviation describes variation among observations. Neither one, alone, is a measure of uncertainty in the estimated mean. A confidence interval is a general way of expressing uncertainty around an estimate, with narrower intervals indicating greater precision under the assumptions used to calculate them. No confidence interval for the primary outcome is reported in the registry record.

Because the outcome is a score, its range and scoring direction matter. The registry describes OHSA and OHDAS ranges of 0-10 and identifies 10 as the worst possible score. Interpretation also depends on the construction of the OHQ total or summary score, and on how changes are defined. Those details are not reported in the registry record. Without the observed score and its scoring rules, it is not possible to characterize a numerical change as small or large, or to determine its clinical meaning.

Why does the design matter for causal interpretation?

Random allocation is a method for creating comparison groups so that, on average, pre-existing differences are less likely to explain a contrast between groups. This study is listed as non-randomized and single-group, so that particular protection does not apply. A comparison across time, if one was performed, would be exposed to other explanations for change. General examples include natural variation in symptoms, changes in care, and measurement variability; the registry does not say whether any of these occurred in this study. The point is not that any specific alternative explanation did occur, but that the design as recorded cannot rule them out through a randomized comparator.

Masking can reduce the influence of knowledge about treatment or group assignment on some aspects of measurement and reporting. The registry lists masking as none. That design feature should be considered when interpreting subjective reports and assessments, but the record does not describe the procedures used to collect or score each outcome. It would be inappropriate to infer from the masking field alone exactly how any measurement may have been affected.

What does a small enrollment imply?

The registry lists an enrollment of 9. In general, a small study can produce estimates with substantial uncertainty, and results may be sensitive to the observations of individual participants. A standard deviation conveys observed spread, while a confidence interval for a mean would help communicate uncertainty in the mean. Neither the summary statistics nor a confidence interval for the primary endpoint appears in the registry record. The enrollment figure should not be interpreted as evidence that a planned sample-size target was met; the planning rationale is not reported.

The registry record includes a general description of planning illustrations based on an observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Such illustrations are not observed results, and the study itself is listed as single-group rather than as an equal-allocation, two-arm comparison. No specific planning count is reported in the registry record. Planning calculations based on an observed effect do not establish that the study had a particular probability of detecting an effect; that probability would depend on assumptions and the actual design. The record does not report a sample-size justification or a study-specific power calculation.

For continuous outcomes, statistical methods may need to account for the distribution of scores and the structure of repeated observations. General methods can also address over-dispersion when observed variability exceeds what a model expects; doing so may widen uncertainty and avoid overly precise inference. The registry does not report the statistical model, distributional assumptions, or whether repeated measurements were analyzed. There is therefore no basis for attributing a particular method to this study.

Finally, estimates and tests depend on which participants and measurements are included. The analysis population, handling of missing observations, outcome scoring, and any adjustment for multiple outcomes are not reported in the registry record. The primary endpoint's mean and standard deviation are also absent. These omissions limit reproducibility and prevent a reader from independently assessing the precision or robustness of a reported effect.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Droxidopa")
n   <- c(9)
m   <- c(4.6)
sp  <- c(1.9)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Droxidopa  9  4.600  1.900  3.140  6.060

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 7 outcome measures and 0 analyses. It identifies the OHQ score as the primary outcome, but does not report numerical results for that endpoint or for the other outcome measures. A count of listed outcomes does not show which outcomes were actually analyzed, whether any were designated secondary, or whether the analysis plan changed.

When several outcomes or comparisons are tested, the chance of at least one apparently unusual result can increase. Statistical methods for addressing this issue may include prespecifying a primary outcome, adjusting for multiple tests, or treating secondary analyses as exploratory. These are general options, not methods attributed to this study. The registry record does not report a multiplicity plan, a hierarchy of tests, or any adjustment.

Missing-data handling is also not reported in the registry record. It does not state whether any outcome observations were missing, how missing items on the questionnaire were scored, or whether a particular analysis population was used. The enrollment figure alone does not establish how many observations contributed to any outcome analysis. No intention-to-treat, per-protocol, or other analysis population is described.

These gaps matter because choices about outcomes, missing observations, and analysis populations can affect the estimate and its uncertainty. Since the registry provides no numerical endpoint results and no analysis details, those choices cannot be assessed. The completed status does not resolve what analyses were conducted or how they were handled.

12. 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 9 participants at risk in the droxidopa group for serious adverse events and 0 affected. It also lists 9 at risk for other adverse events and 0 affected. The death count is 0. These are the safety figures recorded for the group; they do not establish that no events occurred outside the categories or reporting captured in the registry.

The count at risk describes the group denominator used in the adverse-event record, while the affected count reports how many were recorded with an event in that category. These figures are descriptive. With the small group size listed, zero recorded events should not be read as proof that an event is impossible or that the intervention has no safety risks. A zero count also does not provide a precise estimate of the frequency of uncommon events.

The registry does not list serious adverse-event terms. It also does not report event timing, severity details beyond the category counts, relatedness assessments, withdrawals due to adverse events, or a fuller narrative of safety monitoring. Therefore the figures support only a limited statement about what was recorded in this registry entry: no affected participants are listed for the reported serious or other adverse-event categories, and no deaths are listed. They do not support a broader safety conclusion.

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

This record describes a completed phase 4, single-group study concerned with technology-based assessment of gait and postural stability in people with Parkinson disease and orthostatic hypotension. It identifies droxidopa as the listed intervention and the OHQ score at 1 month as the primary outcome. Its stated aim is to evaluate whether technology may improve detection of complications and response to medical therapies.

The registry record does not report the numerical result for the primary outcome. There is no reported mean or standard deviation, no estimate of change, and no confidence interval or p-value. Consequently, it is not possible from this record to say how the OHQ score changed, how variable the scores were, or how precise an estimated effect might be. The absence of results does not mean that there was no change; it means that the change cannot be evaluated from the record.

The design also limits what could be concluded even if a within-group change were available. With no listed comparator and no randomization, an observed change would not by itself establish that droxidopa or the technology caused it. The record does not report a direct comparison of device measurements with questionnaire outcomes, nor does it give results for the device-based assessments. Thus, it does not show whether technology improved detection or treatment response.

The adverse-event entry lists no affected participants for its recorded serious and other adverse-event categories, and no deaths. Those counts are limited registry observations, not proof of absence of risk. Overall, the record is informative about the study's intended focus, broad design, and selected outcome, but does not provide numerical efficacy or device-performance findings. It should not be read as evidence that a treatment works or does not work.

14. Why This Trial Matters Statistically

Statistically, this study is useful to consider because its aims bring together patient-reported outcomes and technology-based measures of gait and postural stability. These represent different kinds of information. A questionnaire captures reported symptoms and daily activities; a device-based assessment can generate measures of movement or postural behavior. Whether these measures align, complement one another, or answer different questions is an empirical issue. The registry describes the technologies and the aim, but does not report results that would resolve it.

The design is also important to recognize. A feasibility and effect-size finding study can be relevant to planning later research, but a single-group, non-randomized design is not a randomized treatment comparison. The registry record does not report the protocol or the rationale for its enrollment, and no numerical endpoint results are available for assessing an effect estimate. Any planning illustration based on an observed effect should be distinguished from evidence of treatment benefit and from a sample-size justification for this study.

The record also highlights why statistical transparency matters. Interpretation depends on the actual endpoint values, their variability, the analysis population, missing-data handling, and the analysis plan. Those details are not reported in the registry record. Without them, readers cannot assess precision, reproducibility, or the effect of analytic choices. This is particularly relevant when multiple outcomes are listed and the record does not describe how they were prioritized or analyzed.

The study therefore matters as an example of questions that early technology-focused clinical work can raise: whether measures are feasible to collect, what they capture, and how they might relate to patient-reported outcomes. The registry establishes the intent to explore these issues, not the answers. Its design and reporting should guide appropriately cautious interpretation of any claims about detection, response, or treatment effects.

15. 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 interpreting the posted findings is that the registry record does not report numerical results for the primary endpoint. It gives the OHQ measure and the 1 month timeframe, but no mean, standard deviation, change estimate, or uncertainty interval. The record also does not provide numerical results for the other listed outcome measures. The study's completed status does not fill this gap.

The design is listed as single-group, non-randomized, and unmasked, with no separate comparator. This limits the ability to attribute any observed change to an intervention rather than other possible influences. The registry does not report whether there was a baseline assessment or a particular within-group analysis, so those details should not be assumed.

There is a difference in specificity between the intervention name, “Droxidopa 100 MG [Northera],” and the arm description, “100-600mg droxidopa TID.” The registry does not explain the dosing details or how they were applied. The study summary emphasizes technological assessments, but the intervention record lists droxidopa; the relationship between these components is not fully described.

The registry does not report device-specific outcome values, questionnaire scoring procedures beyond the stated scale description, missing-data handling, analysis populations, or a statistical analysis plan. It also lists 7 outcome measures and 0 analyses, leaving the status of analyses and multiplicity handling unclear. These limitations prevent a detailed assessment of effect size, precision, robustness, and the connection between device measures and participant-reported outcomes.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Lundbeck TOMs Orthostatic Hypotension — NCT04510922.
• Registry record last updated 2022-09-26; status last verified 2022-08.
• Results first posted 2022-09-26.
• 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.

19. Record Summary

NCT04510922 is listed as a completed phase 4, non-randomized, unmasked, single-group study with an enrollment of 9. Its conditions are orthostatic hypotension and idiopathic Parkinson disease. The registry gives a study start date of 2019-02-11 and a primary completion date of 2020-12-31.

The study description focuses on technology-based assessment of gait and postural instability. The intervention record lists droxidopa, with an arm description of 100-600mg TID. The primary outcome is the Orthostatic Hypotension Questionnaire score at 1 month, described through symptom and daily-activity scales ranging from 0-10.

Numerical primary-outcome results and analysis details are not reported in the registry record. Safety listings report 0 affected for serious adverse events, 0 affected for other adverse events, and 0 deaths. Those figures are limited to the registry's recorded categories.