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 A Study of Mycophenolate Mofetil (CellCept) in Lung Transplant Recipients (NCT01014442), a non-randomized phase 3 study. The primary outcome is Maximum Concentration (Cmax) of Mycophenolic Acid (MPA), Mycophenolic Acid Glucuronide (MPAG) and Acyl Glucuronide Metabolite of Mycophenolic Acid (AcMPAG) at Day 4.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01014442 |
| Phase | Phase 3 |
| Condition | Lung Transplantation |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 68 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
Population
Recipients of a new lung allograft were enrolled and grouped according to the original disease. Group A comprised participants with cystic fibrosis. Group B comprised participants with COPD, emphysema, idiopathic pulmonary fibrosis, or alpha-1 antitrypsin deficiency. The study enrolled 68 participants overall. The primary analysis population is described as a per-protocol population derived from participants who received at least one dose and had the primary variable measured under study drug, with participants who had major protocol violations excluded; the record states a total of 46 participants for this population.
Intervention
All participants received oral mycophenolate mofetil. The planned regimen was 1.5 g twice daily from Days 2 through 30 post-transplantation, followed by 1 g twice daily from Days 31 through 90. The study was open-label, so participants and investigators were not masked to treatment.
Comparator
There was no untreated or placebo comparator. Both disease-defined groups received mycophenolate mofetil, and allocation was non-randomized. The groups therefore describe recipients with different original diseases rather than randomized treatment arms designed to estimate a treatment contrast.
Primary question
The primary endpoint concerned the maximum concentration of mycophenolic acid, mycophenolic acid glucuronide, and the acyl glucuronide metabolite of mycophenolic acid at Day 4 after transplantation. Concentrations were expressed in mg/L, with sampling scheduled predose and at several post-dose times through 12 hours. The registry record does not report numerical primary endpoint results, so the observed concentrations and their precision cannot be described.
3. Trial Design
This completed Phase 3 study, NCT01014442, is titled A Study of Mycophenolate Mofetil (CellCept) in Lung Transplant Recipients. Its official title describes pharmacokinetics in de novo lung allograft recipients. The registry record identifies Hoffmann-La Roche as sponsor and describes the study as open-label, single-center, non-randomized, and parallel-group. Enrollment was 68. The recorded study start was 2010-01, and primary completion was 2012-06.
The two groups were defined by original disease, not by randomized assignment to different treatments. Participants with cystic fibrosis formed one group; the other included participants with COPD, emphysema, idiopathic pulmonary fibrosis, or alpha-1 antitrypsin deficiency. Both groups were assigned the same reported mycophenolate mofetil regimen: 1.5 g orally twice daily from Days 2 through 30 after transplantation, then 1 g orally twice daily from Days 31 through 90. Anticipated time on study treatment was 90 days.
The study aimed to assess pharmacokinetics, efficacy, and safety. Its primary endpoint was a pharmacokinetic measure on Day 4. Listed secondary outcomes include FEV1, percent predicted FEV1, FVC, changes in intracellular ATP and T-cell phenotype, and the percentage of participants with opportunistic infections. The record lists 54 outcome measures and 125 analyses, but those totals do not establish how many analyses were planned in advance, how they were prioritized, or how results were selected for reporting. Those details are not reported in the registry record.
Because there was no randomized control group, the design does not support a like-for-like causal comparison between treatment and no treatment. Group comparisons would also reflect the underlying disease categories and any other differences between participants. The registry record does not report further details about recruitment procedures, center characteristics, or the basis for the group definitions beyond the diagnoses listed.
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
| Stage | MMF - Cystic Fibrosis | MMF - COPD, Emphysema, IPF, or A1AD |
|---|---|---|
| STARTED | 33 | 35 |
| COMPLETED | 26 | 16 |
| NOT COMPLETED | 7 | 19 |
Reasons for not completing this period:
| Reason | MMF - Cystic Fibrosis | MMF - COPD, Emphysema, IPF, or A1AD |
|---|---|---|
| Adverse Event | 0 | 8 |
| Withdrawal by Subject | 4 | 4 |
| Protocol Violation | 2 | 5 |
| Lack of Compliance | 0 | 1 |
| Unspecified Reason | 1 | 1 |
Analysis population for the primary outcome, as defined in the registry: Per Protocol (PP) Population: Intent-to treat (ITT) population (received at least one dose of study drug and where the primary variable was measured at least once under study drug) excluding participants with major protocol violations (total 46 participants). Number of participants analyzed=participants who were evaluable for this outcome measure.
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.
Population: The safety population included all participants who received at least one dose of study drug and a safety follow-up, whether withdrawn prematurely or not.
| Characteristic | Unit | MMF - Cystic Fibrosis | MMF - COPD, Emphysema, IPF, or A1AD | Total |
|---|---|---|---|---|
| Participants analysed | 33 | 35 | 68 | |
| Age, Continuous | years | 31.3 (SD 7.42) | 54.3 (SD 7.93) | 43.1 (SD 13.86) |
| Gender: Female | Participants | 13 | 14 | 27 |
| Gender: Male | Participants | 20 | 21 | 41 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Concentration (Cmax) of Mycophenolic Acid (MPA), Mycophenolic Acid Glucuronide (MPAG) and Acyl Glucuronide Metabolite of Mycophenolic Acid (AcMPAG) at Day 4, measured over Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 after transplantation.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | mg/L |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was the maximum concentration, or Cmax, of three measured compounds: mycophenolic acid (MPA), mycophenolic acid glucuronide (MPAG), and the acyl glucuronide metabolite of mycophenolic acid (AcMPAG). The endpoint was assessed on Day 4 after transplantation and expressed in mg/L. Scheduled samples were taken predose and at 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose.
In general, a maximum concentration describes the highest concentration observed or derived over a specified sampling interval. Pharmacokinetic measures such as Cmax characterize drug exposure over time; they are distinct from clinical outcomes such as lung function or infection. The concentration-time profile can depend on when samples are collected, so the scheduled sampling times are important context for interpreting a reported maximum.
The registry record does not report the Cmax values, means, standard deviations, or other numerical results for any of the three compounds. It therefore does not allow a reader to assess the magnitude or variability of the endpoint, or to compare concentrations between the disease-defined groups. The listed endpoint description says the measure is a mean with standard deviation, but the numerical summary itself is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limit is that the registry record does not report numerical results for the primary pharmacokinetic endpoint. It identifies the compounds, units, sampling schedule, and analysis population, but gives no mean Cmax values or standard deviations. Consequently, the magnitude and precision of the day-four concentrations cannot be assessed from the record. No conclusion about the observed pharmacokinetic profile can be drawn from an endpoint title and measurement schedule alone.
The study enrolled 68 participants overall, while the primary endpoint description identifies a per-protocol population of 46 participants. That population is described as participants who received at least one dose and had the primary variable measured under study drug, excluding participants with major protocol violations. The registry record does not report the number with a usable measurement for each compound, each sampling time, or each group. It also does not report the reasons for exclusion or the handling of measurements that were absent or unusable.
The study groups represent different original diseases and both received mycophenolate mofetil. Since allocation was non-randomized and there was no untreated comparator, a difference between those groups, if numerical results were available, would not by itself isolate a treatment effect. Disease category and other group differences could be relevant to interpretation. Likewise, a within-study concentration measurement does not establish a comparison with another regimen or treatment.
The registry lists several secondary outcomes related to lung function, immune measures, and opportunistic infections. Their numerical results are not reported in the registry record. The record also does not report confidence intervals, hypothesis tests, or other uncertainty measures for these outcomes. Without those results, it is not possible to describe the direction, size, or precision of any observed secondary outcome.
Safety information is reported as counts for serious and other adverse events in the two disease-defined groups, along with a list of serious event terms and percentages. These data are descriptive. The design has no randomized control group, and the record does not report event-level timing, exposure-adjusted rates, or a causal assessment for the listed events. The counts should not be interpreted as evidence that the study drug caused or prevented an event.
In general, estimates are more informative when accompanied by uncertainty measures and a clear account of the analysis population. Since numerical primary results and their uncertainty are absent from the registry record, neither the observed effect size nor its statistical precision can be evaluated. Any interpretation should therefore be limited to the design, planned measurements, and the safety counts that are explicitly posted.
9. Statistical Methods Explained
The registry describes the primary measure as a mean of a continuous variable with standard deviation. These are summary statistics for numeric measurements: the mean describes the arithmetic average, while the standard deviation describes how dispersed individual measurements are around that average. In general, the mean and standard deviation do not show the full shape of a distribution. Pharmacokinetic concentrations can be unevenly distributed, so the distribution and the methods used to summarize or analyze them can matter. The registry record does not report the numerical summaries or distributional diagnostics for this endpoint.
What does Cmax represent?
Cmax is the maximum concentration within the stated sampling period. For this study, the specified period included predose sampling and measurements through 12 hours after the dose on Day 4. Sampling at defined times provides observations of concentration over time; the maximum depends on the recorded values and the sampling schedule. If concentrations peak between scheduled samples, the observed maximum and the true peak may differ. The registry record does not describe whether Cmax was determined directly from measured samples or by another method, so that detail is not reported in the registry record.
The endpoint names three compounds and gives the unit mg/L. Interpreting a concentration requires knowing which compound is being described and the time point of measurement. Comparing group summaries would require the numerical estimates and details of the analysis. Neither the estimates nor a group-specific statistical analysis for the primary endpoint is reported in the registry record.
What does a standard deviation add?
In general, a standard deviation expresses variability among individual observations, while a confidence interval expresses uncertainty about an estimated quantity such as a mean. They answer related but different questions. A standard deviation is not a confidence interval, and it does not on its own show how precisely a group mean is estimated. Confidence intervals and the method used to calculate them for the primary endpoint are not reported in the registry record. The posted endpoint description names standard deviation as the dispersion measure, but the numerical standard deviations are absent.
Pharmacokinetic concentration data may show greater variability than a simple normal model anticipates. In general, over-dispersion means the observed spread is larger than expected under a model; if a model ignores it, uncertainty estimates and tests can be misleading. This is a general statistical concern, not a claim that over-dispersion occurred in this study. The registry record does not report model diagnostics or an analysis method that would allow that issue to be assessed.
What does the design imply for comparisons?
Allocation was non-randomized, and the two groups were defined by original disease. Random assignment, in general, can help balance measured and unmeasured participant characteristics across treatment groups. That protection is not established by this design. Moreover, both groups received mycophenolate mofetil, so the group structure is not a treated-versus-untreated comparison. Any difference between groups could reflect disease category or other group differences as well as pharmacokinetic variation. The record does not report an adjusted comparative model or covariates used for adjustment.
The study is also described as open-label. In general, lack of masking can affect reporting or measurement of outcomes that depend on judgment or participant reporting. It is less directly relevant to a laboratory concentration measurement than to a subjective outcome, but the registry record does not describe assay procedures, laboratory masking, or quality-control procedures. Those details are not reported in the registry record.
How should planning illustrations be read?
In general, a sample-size calculation estimates how many participants would be needed under stated assumptions to detect a specified effect with a chosen significance level and power. It is a planning exercise, not a measure of what the completed study actually achieved. The registry record does not report the trial's own sample-size justification. A planning illustration based on an observed effect is distinct from the original design rationale and should not be called observed power. The assumptions described for such an illustration are a two-sided significance level of 0.05, equal allocation, a normal approximation, and an effect defined as a fraction of the observed effect. Those assumptions do not establish that the original trial used them.
General planning calculations also depend on the outcome scale, variability, target effect, analysis, and allocation structure. When an observed effect is uncertain, planning from it can yield an illustration that is sensitive to that estimate. No numerical planning results are described because they are not reported in the registry record. The illustration cannot substitute for the trial's original sample-size rationale, which is not reported in the registry record.
What can be inferred from absent results?
The registry record's descriptions of outcomes and methods do not supply an estimate of treatment effect. A statistical test, confidence interval, or p-value requires an analysis result; none for the primary endpoint is reported in the registry record. The absence of a posted numerical result is not evidence of a particular concentration, a null effect, or a lack of variability. It means that those quantities cannot be evaluated from this record.
10. Multiplicity, Missing Data and Analysis Populations
The record lists 54 outcome measures and 125 analyses, with a primary pharmacokinetic endpoint and multiple secondary outcomes. In general, testing many outcomes or many comparisons increases the opportunity for apparently notable findings to arise by chance. Statistical plans may address this through a prespecified hierarchy, adjustment for multiple testing, or emphasis on estimation rather than a collection of separate significance tests. The registry record does not report whether such a strategy was specified or used, and it does not report an interim-analysis plan.
The posted primary endpoint description identifies a per-protocol population derived from participants who received at least one dose and had the primary variable measured at least once under study drug, excluding participants with major protocol violations. It states a total of 46 participants. The enrollment was 68. The record's wording for the number analyzed is incomplete, so a more specific analyzed count is not reported in the registry record. Participant counts by compound, sampling time, and disease-defined group are also not reported.
In general, missing measurements can affect an analysis if the likelihood of missingness is related to participant characteristics or outcomes. The analysis population definition indicates that having a primary measurement was part of inclusion, but the registry record does not describe missing-data methods, reasons for missing measurements, or sensitivity analyses. It also does not report whether every enrolled participant completed the scheduled sampling or the planned study treatment.
The registry record does not provide enough detail to assess whether the analysis population, outcome hierarchy, missing-data approach, and multiplicity handling were prespecified. These omissions limit evaluation of potential selection and reporting effects. They do not establish that any particular bias occurred.
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 |
|---|---|---|---|
| MMF - Cystic Fibrosis | 13 | 33 | 39.4% |
| MMF - COPD, Emphysema, IPF, or A1AD | 14 | 35 | 40.0% |
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 |
|---|---|
| MMF - Cystic Fibrosis | 27 of 33 |
| MMF - COPD, Emphysema, IPF, or A1AD | 29 of 35 |
Most frequent serious adverse event terms
| Event term | Organ system | MMF - Cystic Fibrosis | MMF - COPD, Emphysema, IPF, or A1AD |
|---|---|---|---|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 0 of 33 | 3 of 35 |
| Ileus | Gastrointestinal disorders | 2 of 33 | 0 of 35 |
| Pneumonia | Infections and infestations | 1 of 33 | 1 of 35 |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 2 of 33 | 0 of 35 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 0 of 33 | 2 of 35 |
| Leukopenia | Blood and lymphatic system disorders | 1 of 33 | 0 of 35 |
| Atrial tachycardia | Cardiac disorders | 1 of 33 | 0 of 35 |
| Tachyarrhythmia | Cardiac disorders | 0 of 33 | 1 of 35 |
| Deafness | Ear and labyrinth disorders | 1 of 33 | 0 of 35 |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | 1 of 33 | 0 of 35 |
Most frequent non-serious adverse event terms
| Event term | Organ system | MMF - Cystic Fibrosis | MMF - COPD, Emphysema, IPF, or A1AD |
|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | 1 of 33 | 12 of 35 |
| Anaemia | Blood and lymphatic system disorders | 3 of 33 | 8 of 35 |
| Leukopenia | Blood and lymphatic system disorders | 8 of 33 | 3 of 35 |
| Fibrinous bronchitis | Respiratory, thoracic and mediastinal disorders | 5 of 33 | 6 of 35 |
| Nausea | Gastrointestinal disorders | 5 of 33 | 3 of 35 |
| Vomiting | Gastrointestinal disorders | 4 of 33 | 4 of 35 |
| Diarrhoea | Gastrointestinal disorders | 3 of 33 | 4 of 35 |
| Bacterial infection | Infections and infestations | 3 of 33 | 4 of 35 |
| Bronchial polyp | Respiratory, thoracic and mediastinal disorders | 5 of 33 | 2 of 35 |
| Sleep disorder | Psychiatric disorders | 0 of 33 | 6 of 35 |
Serious events with the highest arm-level rate: pulmonary embolism (8.6%), ileus (6.1%), pneumothorax (6.1%), respiratory failure (5.7%), leukopenia (3.0%).
The registry reports serious adverse event counts for the disease-defined groups. In the cystic fibrosis group, 33 participants were listed as at risk and 13 as affected. In the group with COPD, emphysema, idiopathic pulmonary fibrosis, or alpha-1 antitrypsin deficiency, 35 were listed as at risk and 14 as affected. For other adverse events, the corresponding counts were 33 at risk and 27 affected in the cystic fibrosis group, and 35 at risk and 29 affected in the other group. These are counts, not comparative effect estimates.
The listed serious event terms and percentages are pulmonary embolism, 8.6%; ileus, 6.1%; pneumothorax, 6.1%; respiratory failure, 5.7%; leukopenia, 3.0%; and atrial tachycardia, 3.0%. The record does not report the denominator or further details needed to interpret these percentages as rates. Death counts are not reported in the registry record.
In general, adverse event counts describe events recorded during a specified study context; they do not establish causation. This non-randomized study has no untreated control group, and the groups differ by original disease. The registry record does not report event timing, exposure-adjusted rates, severity details for each term, or attribution to study drug. The counts therefore should be read descriptively, not as estimates of comparative risk or proof that treatment caused or prevented an event.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed Phase 3 study enrolled 68 lung transplant recipients and assessed mycophenolate mofetil pharmacokinetics, efficacy, and safety. It describes a Day 4 primary endpoint based on maximum concentrations of MPA, MPAG, and AcMPAG, with concentration sampling scheduled from predose through 12 hours post-dose. It also describes two disease-defined groups receiving the same regimen and reports selected adverse event counts.
Those design and measurement details do not answer how large the measured concentrations were. Numerical primary endpoint results, including means and standard deviations, are not reported in the registry record. The record also does not report numerical results for the listed secondary outcomes. No conclusion about the direction or size of a pharmacokinetic or clinical outcome can be drawn from the endpoint descriptions alone.
The study's two groups are not a randomized comparison of mycophenolate mofetil against an alternative or no treatment. Both received the study drug, and group membership followed original disease. Even if group-specific results were available, they would not by themselves establish that a difference was caused by the drug or by disease category. The absence of a randomized comparator also prevents a direct estimate of how outcomes would have differed without the regimen.
The safety counts give a descriptive account of reported events, but the registry record does not provide information needed to attribute those events to treatment or compare them with an untreated group. Counts and listed percentages should not be mistaken for causal estimates.
In general, results are best interpreted alongside estimates, uncertainty intervals, analysis populations, and prespecified methods. Here, the primary numerical estimates and several details of the statistical plan are not reported in the registry record. The appropriate conclusion is limited: the record describes the study design, endpoint, treatment schedule, and selected safety counts, but does not provide enough outcome results to assess the magnitude or precision of the primary pharmacokinetic findings.
13. Why This Trial Matters Statistically
This study is statistically notable because its primary focus is pharmacokinetic measurement in lung allograft recipients, while its groups are organized by original disease rather than randomized treatment. The distinction matters: descriptive patterns across those groups may be useful for characterizing the enrolled population, but they do not create a controlled estimate of treatment effect. The design and the question must be kept separate when interpreting results.
The endpoint also illustrates how measurement schedules shape pharmacokinetic summaries. Cmax is defined over an observation window, and the planned samples range from predose to 12 hours after dosing on Day 4. In general, the timing and completeness of these samples affect what can be said about a concentration maximum. The registry record does not provide the numerical values or details of how maxima were derived, so the statistical properties of the reported endpoint cannot be evaluated.
The analysis-population description is another important consideration. The record identifies a per-protocol population of 46 participants after excluding major protocol violations from participants who received at least one dose and had the primary variable measured. That differs from the overall enrollment of 68. In general, exclusions and missing measurements can affect how representative an analysis is of everyone enrolled; the registry record does not report enough detail to assess that impact.
Finally, the record lists many outcomes and analyses but does not describe multiplicity handling or an interim-analysis plan. These omissions illustrate why statistical interpretation depends not only on outcome names but also on prespecified methods, available estimates, and uncertainty. Since numerical primary results are not reported in the registry record, the most defensible statistical account focuses on the design and the limits of what can be inferred rather than on an effect estimate.
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 principal limitation for interpretation is the absence of numerical results for the primary pharmacokinetic endpoint. Although the endpoint, compounds, units, and sampling times are described, means and standard deviations are not reported in the registry record. Numerical results for the listed secondary outcomes are also not reported. Thus, the size and precision of any outcome cannot be assessed from the record.
The non-randomized design and disease-defined groups limit comparative interpretation. All participants received mycophenolate mofetil, and there is no listed untreated or alternative-treatment group. Group differences, if any, could reflect the original disease or other differences rather than treatment. The registry record does not report adjustment methods or the factors considered in any comparative analysis.
The primary endpoint population is described as 46 participants, compared with overall enrollment of 68. The wording about the number analyzed is incomplete, and detailed participant flow is not reported in the registry record. Counts by group and sampling time, reasons for exclusion, completeness of sample collection, and handling of missing measurements are not reported.
The registry record does not report the original sample-size justification, the planned statistical analysis in sufficient detail, multiplicity procedures, interim analyses, or sensitivity analyses. It also does not describe laboratory assay procedures or methods for deriving Cmax. These omissions restrict assessment of the endpoint's measurement and analysis, but do not establish that the study methods were inadequate.
For safety, the record reports selected counts and serious event terms but does not report event-level timing, exposure-adjusted rates, attribution, or death counts. Without a randomized control group, these data are descriptive and cannot isolate treatment-related risk. These limitations are specific to the information and design described in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Mycophenolate Mofetil (CellCept) in Lung Transplant Recipients — NCT01014442.
- Registry record last updated 2017-02-10; status last verified 2015-07.
- Results first posted 2017-02-10.
- 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
NCT01014442 is a completed, open-label, single-center, non-randomized Phase 3 study sponsored by Hoffmann-La Roche. It enrolled 68 lung transplant recipients and grouped them by original disease: cystic fibrosis, or COPD, emphysema, idiopathic pulmonary fibrosis, or alpha-1 antitrypsin deficiency. Both groups received oral mycophenolate mofetil, with 1.5 g twice daily from Days 2 through 30 post-transplantation and 1 g twice daily from Days 31 through 90.
The primary endpoint was Day 4 Cmax for MPA, MPAG, and AcMPAG, measured in mg/L using scheduled samples from predose through 12 hours post-dose. The primary analysis population is described as 46 participants after specified eligibility and protocol-violation criteria. Numerical results for the primary and listed secondary outcomes are not reported in the registry record. The registry reports selected serious and other adverse event counts, but the design does not provide a randomized treatment comparison.