Impact of CardiolRxTM on Recurrent Pericarditis (MAvERIC-Pilot): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Impact of CardiolRxTM on Recurrent Pericarditis (MAvERIC-Pilot) (NCT05494788), a na phase 2 study. The primary outcome is 11-point NRS Pain Score.

2. Clinical Question

3. Trial Design

MAvERIC-Pilot is a phase 2, open-label, single-group study of CardiolRx in recurrent pericarditis. The registry lists 27 enrolled participants, one intervention, and no masking. Allocation is listed as not applicable, consistent with a design that does not assign participants between randomized treatment arms. The trial is marked completed; its listed start date is 2023-01-09 and its primary completion date is 2024-06-15.

The registry describes CardiolRx as pharmaceutically produced cannabidiol. Its study summary frames the population as people with recurrent pericarditis who are refractory or intolerant to existing therapeutic management, or who need long-term corticosteroids for disease control. The stated aims include assessing tolerance and safety during resolution of symptoms, changes in objective disease measures, and the feasibility of weaning background therapy during an extension period. Those aims do not, by themselves, establish that any improvement or weaning occurred.

A single-group design can describe what happened among participants receiving the intervention, but it does not supply a concurrent untreated or active-control group. Because the study is also open label, participants and investigators were not masked to treatment assignment. These design features matter when interpreting changes in symptoms, which may also reflect the natural course of disease, expectations, co-interventions, or measurement variation. The registry record does not report a randomized comparison, a control arm, or a detailed protocol for handling background treatments.

The registry lists seven outcome measures and zero analyses. It does not report numerical results for the primary endpoint, details of a statistical analysis plan, or a sample-size justification. The record therefore gives the design and endpoint definition, but does not support a quantitative estimate of the observed change in pain score.

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: 8-week follow up; 18-week extension period

Initial Treatment Period

Reasons for not completing this period:

Extension Period

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All patients enrolled

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 11-point NRS Pain Score, measured over 8 weeks.

The primary endpoint is change from baseline in the 11-point NRS pain score after 8 weeks of treatment. The registry defines the scale as running from 0, no pain, to 10, maximum pain, and identifies the endpoint summary as a mean with standard deviation. It specifies the primary population as all patients enrolled.

Pain is a patient-reported symptom, so an NRS provides a structured way to record its intensity and compare scores over time. A change-from-baseline measure focuses on within-participant movement rather than the score at the endpoint alone. The mean describes the average change in the group, while the standard deviation describes how dispersed individual changes are around that average. In general, the mean can conceal variation: participants may have different directions or magnitudes of change even when the group average is stable.

The registry record defines the measure and timing but does not report the numerical baseline and follow-up values, the mean change, its standard deviation, or a confidence interval. It therefore does not show the size or precision of the observed change. A symptom-score endpoint also addresses pain specifically; it should not be treated as a direct measure of every objective disease feature or of the feasibility of reducing background therapy.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the primary outcome as a continuous measure summarized by a mean and standard deviation. These statistics answer different questions. The mean represents the group’s average change in NRS pain score; the standard deviation describes how much individual changes vary around that average. Neither is reported numerically for the primary outcome, so the group’s average change and participant-level spread cannot be evaluated.

What does a confidence interval communicate?

In general, a confidence interval expresses the uncertainty around an estimated quantity, such as a mean change. A narrower interval indicates greater precision under the assumptions of the analysis, while a wider interval indicates less precision. It is not a guarantee that the true value lies within the interval, nor does it describe the range of individual participant outcomes. No confidence interval for the primary endpoint is reported in the registry record.

How should a p-value be read?

In general, a p-value measures how compatible the observed data are with a specified null hypothesis under the statistical model. It does not measure the probability that the null hypothesis is true, the size of an effect, or whether a change is important to participants. A p-value cannot replace the estimated change and its uncertainty. The registry record lists zero analyses and does not report a primary-endpoint p-value.

Why does the single-group design affect interpretation?

A within-group change compares participants with their own baseline measurements. That can be useful for describing the observed course over time, but it does not create the counterfactual comparison needed to determine what would have happened over the same period without the intervention. In general, symptoms can change over time for multiple reasons. A comparison group can help account for common time-related influences, whereas this trial’s single-group model cannot provide that control.

The open-label design adds a separate consideration. In general, masking can reduce the influence of treatment knowledge on expectations, reporting, and assessment. Because masking is listed as none, such influences cannot be ruled out by blinded assessment. This is particularly relevant when the endpoint is a self-reported pain score, although it does not establish that bias occurred.

What can a standard deviation tell readers?

For a continuous endpoint, the standard deviation describes dispersion of participant-level values around the mean. A small standard deviation would indicate that values are more concentrated near the mean; a larger one would indicate more variation. In general, the standard deviation is not the standard error: the standard error concerns the precision of the estimated mean and depends on both variability and the amount of information. Neither the endpoint standard deviation nor an uncertainty estimate is reported for the primary result.

How do planning illustrations differ from observed results?

The registry’s stated planning assumptions describe an illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. For time-to-event outcomes, the assumptions specify total events; for other outcomes, they specify participants per arm. These are planning concepts, not trial findings and not observed power.

The current study is single group, and the registry record does not report the observed primary-endpoint effect or numerical planning illustration values. The stated equal-allocation, two-arm assumption is not a description of the trial’s actual design. A planning calculation based on an observed effect would not serve as the trial’s original sample-size justification; that justification is not reported in the registry record. In general, planning estimates depend on their assumptions and should not be interpreted as evidence that a study did or did not detect a treatment effect.

Finally, the outcome definition names all patients enrolled as the primary population. The record does not explain how missing endpoint values were handled, how deviations from treatment were addressed, or whether the endpoint analysis included every enrolled participant. Those details matter because the analyzed data and the assumptions used for missing observations can affect estimates and their uncertainty. They are not reported in the registry record.

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("CardiolRx")
n   <- c(27)
m   <- c(-3.7)
sp  <- c(2.07)
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  CardiolRx  27  -3.700  2.070  -4.519  -2.881

11. Multiplicity, Missing Data and Analysis Populations

The registry lists seven outcome measures and zero analyses. It does not report a multiplicity adjustment, a hierarchy of outcomes, or a prespecified strategy for distinguishing confirmatory from exploratory analyses. When a study assesses several outcomes or makes multiple statistical comparisons, the chance of at least one apparently unusual result can increase. In general, multiplicity methods can address that issue, but the record does not say whether any such method was planned or used. With no numerical endpoint results or analysis details reported, the implications for interpretation cannot be assessed.

The primary outcome population is described as all patients enrolled. That wording identifies the intended population but does not establish how many participants contributed an 8-week pain score or how missing measurements were treated. The registry record does not report the amount or pattern of missing primary-endpoint data, reasons for missingness, imputation methods, or sensitivity analyses. In general, missingness can affect an estimate if participants with observed outcomes differ from those without them, and the assumptions used to address missing data should be made clear.

The record also does not report a detailed analysis population, such as rules for participants who discontinued treatment or had protocol deviations. It does not specify whether the primary analysis used only observed cases or another approach. These details are not reported in the registry record and should not be inferred from the phrase all patients enrolled.

Because the study has a single-group model, the multiplicity and missing-data questions are distinct from the absence of a control comparison. Neither an adjustment for multiple testing nor a missing-data method would create a concurrent comparator. The registry record therefore leaves both the analysis procedures and the basis for interpreting any within-group change unclear.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: chest pain (3.7%), enteritis infectious (3.7%), tooth fracture (3.7%), drug reaction with eosinophilia and systemic symptoms (3.7%).

The registry reports 27 participants at risk for serious adverse events and one participant affected. It reports 27 participants at risk for other adverse events and 14 participants affected. Deaths are listed as zero. These figures are counts of participants in the CardiolRx group, not comparative estimates against another arm.

The registry lists Chest pain, ENTERITIS INFECTIOUS, Tooth fracture, and DRUG REACTION WITH EOSINOPHILIA AND SYSTEMIC SYMPTOMS among the top serious adverse-event terms, each at 3.7%. These listed terms describe reported events; the record does not establish from the figures alone whether CardiolRx caused them. The figures also do not provide a complete account of event timing, duration, severity beyond serious-event classification, or clinical course.

In general, adverse-event counts are interpreted in relation to who was monitored, the observation period, and how events were collected and coded. The registry record does not give enough detail to compare risks across treatments or to attribute events causally. The counts are descriptive safety information for this enrolled group and should not be read as proof of either causation or absence of risk.

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

The study is designed to describe CardiolRx use in an open-label, single-group pilot involving recurrent pericarditis. Its primary endpoint is a defined measure of change in pain score after 8 weeks, and the registry also describes aims concerning safety, objective disease measures, and the feasibility of reducing background therapy during an extension period. The design and endpoint definition clarify what the investigators intended to assess.

However, the registry record does not report the numerical primary endpoint result. It therefore does not show whether the mean pain score increased, decreased, or remained similar, or how variable participant changes were. The record also does not provide numerical results for the other stated aims. Without those findings, the intended questions cannot be answered quantitatively from this record.

Even a reported before-and-after change in this design would describe the course of the enrolled group, not isolate the effect of CardiolRx from other influences. In general, a single-group, open-label study cannot provide the same counterfactual evidence as a controlled comparison. Its observations may inform further study questions, but design limitations and the lack of endpoint results constrain what can be inferred.

The safety figures provide limited descriptive information: one participant was affected by a serious adverse event, 14 by other adverse events, and there were zero deaths among 27 participants at risk. These counts do not establish whether reported events were caused by the intervention, and they do not compare CardiolRx with another treatment.

Accordingly, the registry entry establishes the trial’s broad design, its primary measure, and selected safety counts. It does not establish a numerical pain-score effect, a causal treatment effect, or whether background therapy was successfully reduced. Those conclusions would require information not reported in the registry record.

14. Why This Trial Matters Statistically

This trial raises several statistical questions that are important in interpreting pilot studies. First, the endpoint is a patient-reported pain score measured over time. Such a measure can describe symptom change directly, but the mean alone would not show whether change was consistent across participants. Dispersion and uncertainty estimates are needed to understand how precisely the group average is estimated and how varied individual responses may be.

Second, the single-group design makes the distinction between observed change and treatment effect especially important. In general, an improvement from baseline can be real as a description of participants’ experience while still not identifying what caused that change. A concurrent comparison group can help address time-related changes and other shared influences; the registry lists no such group for this study.

Third, open-label assessment matters for subjective outcomes. General statistical reasoning recognizes that treatment knowledge can affect expectations and reporting. This consideration does not negate participant-reported outcomes, but it is part of the uncertainty around their interpretation when masking is absent.

Fourth, the study’s stated aims span pain, safety, objective measures, and background-therapy feasibility. Multiple aims can be useful in a pilot, but clear outcome definitions, analysis plans, and reporting are needed to distinguish exploratory observations from confirmatory evidence. The registry lists seven outcome measures and zero analyses, and numerical primary-outcome results are not reported in the registry record.

Finally, the safety counts offer a descriptive snapshot, not a comparative risk estimate. Taken together, the design illustrates why statistical interpretation depends on more than an endpoint label: the observed values, uncertainty, analysis population, missing-data handling, and comparison structure all matter. Several of those details are not reported in the registry record.

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 central limitation for interpreting efficacy is the absence of a numerical primary-endpoint result in the registry record. Although change in 11-point NRS pain score after 8 weeks is defined, the record gives no mean change, standard deviation, confidence interval, or p-value. The direction, magnitude, and precision of pain-score change therefore cannot be judged.

The single-group model has no concurrent control comparison, and the study is open label. In general, these features limit causal interpretation, particularly for a symptom endpoint that participants report themselves. The design can describe outcomes among those enrolled, but it cannot distinguish treatment-associated change from other changes over time through a control group.

The registry describes additional aims involving tolerance, safety, objective disease measures, and feasibility of weaning concomitant background therapy, including corticosteroids, during an extension period. Numerical findings for those aims are not reported in the registry record. The record does not provide details about the extension period’s duration, the criteria for weaning, or how feasibility was assessed.

Other important analysis details are also not reported in the registry record: missing-data handling, the number contributing endpoint measurements, analysis-population rules, and a detailed statistical plan. The record lists seven outcome measures but zero analyses, and it does not report a sample-size justification. These omissions limit assessment of how outcomes were analyzed and how precisely they might be estimated.

For safety, the registry provides selected counts and listed serious-event terms, but not a controlled comparison or enough information to infer causality. The findings should therefore be read as limited descriptive information rather than a comparative safety estimate.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Impact of CardiolRxTM on Recurrent Pericarditis (MAvERIC-Pilot) — NCT05494788.
• Registry record last updated 2025-10-20; status last verified 2025-10.
• Results first posted 2025-10-20.
• 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.

19. Record Summary

MAvERIC-Pilot is a completed phase 2, open-label, single-group study of CardiolRx in recurrent pericarditis, with 27 participants enrolled. The primary endpoint is mean change from baseline in 11-point NRS pain score after 8 weeks; the scale ranges from 0 for no pain to 10 for maximum pain. The primary population is described as all patients enrolled.

The registry reports one participant affected by a serious adverse event, 14 affected by other adverse events, and zero deaths among 27 at risk. It lists seven outcome measures and zero analyses. Numerical results for the primary endpoint and the additional stated aims are not reported in the registry record. The single-group, open-label design does not provide a like-for-like control comparison, limiting causal interpretation of any change.