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 Pharmacokinetic Study of Intranasal Dexmedetomidine in Pediatric Patients With Congenital Heart Disease (NCT03417999), a non-randomized phase 1 study. The primary outcome is Number of Samples Obtained Per Subject.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03417999 |
| Phase | Phase 1 |
| Condition | Dexmedetomidine, Congenital Heart Disease |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 28 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Children's Hospital of Philadelphia |
| Results posted | Yes |
2. Clinical Question
Population
Children with congenital heart disease undergoing an elective diagnostic or interventional cardiac catheterization procedure were the population of interest. The study description covers children aged ≥1 month to ≤6 years. The registry describes age subgroups, including children aged >2 years to ≤6 years and children aged ≥1 month to ≤2 years. The primary-endpoint population includes subjects who received study drug, whether evaluable or non-evaluable.
Intervention
The intervention was atomized intranasal dexmedetomidine. The record describes a 2 μg/kg dose in Cohort 1 and a 4 μg/kg dose in Cohort 2. Cohort 1 includes descriptions for administration under general oral endotracheal anesthesia and under sedation with a natural airway.
Comparator
This was a non-randomized, sequential study, not a randomized comparison. Its cohort descriptions vary by dose, age group, and anesthesia or airway context. These groups therefore should not be treated as interchangeable control arms. A like-for-like control comparison is not described in the registry record.
Primary question
The study’s stated objectives include characterizing peak plasma drug concentration and the corresponding time after intranasal administration. The registered primary endpoint, however, is the number of samples obtained per subject, summarized as a median and inter-quartile range. It concerns sample collection over up to 5 hours after administration. A numerical result for this endpoint is not reported in the registry record, so the posted information does not quantify the observed sample count or its distribution.
The distinction between the study objective and the registered primary endpoint matters: sample counts describe the availability of collected measurements, whereas peak concentration and time to peak are pharmacokinetic quantities. The record does not provide numerical estimates for those quantities or a reported analysis connecting sample counts to them.
3. Trial Design
This is a phase 1 pharmacokinetic study listed as non-randomized, unmasked, and sequential. The registry lists an enrollment of 28 and identifies Children's Hospital of Philadelphia as the sponsor. The study start date is 2018-06-14, and the primary completion date is 2021-10-12. Its status is terminated. The reason for termination is not reported in the registry record.
The cohort descriptions involve different dosing and clinical contexts. Cohort 1A is described as receiving dexmedetomidine 2 μg/kg under general oral endotracheal anesthesia, while Cohort 1B is described as receiving 2 μg/kg under sedation with a natural airway. Each description includes age groups aged >2 years to ≤6 years and aged ≥1 month to ≤2 years. Cohort 2 is described as receiving 4 μg/kg under general oral endotracheal anesthesia in subjects aged >2 years to ≤6 years. These features make the cohorts descriptive groups rather than randomized alternatives.
Sequential enrollment means the record does not describe allocation by random assignment. The order, decision rules, or progression criteria for moving between cohorts are not reported in the registry record. The masking field indicates none, so the record does not describe blinding. Further operational details, including dose-escalation criteria and the rationale for selecting the cohort structure, are not reported in the registry record.
The primary outcome is the number of samples obtained per subject, with the stated summary measure being the median and inter-quartile range. The record gives a sampling schedule extending through 300 minutes after administration. Although the stated study objective concerns peak plasma concentration and the time at which it occurs, the registry lists four outcome measures and no analyses. Numerical pharmacokinetic results and a description of how they were derived are not reported in the registry record.
Because dose, age, and anesthesia or airway context vary across cohort descriptions, differences between groups would not isolate a dose effect or an anesthesia effect. The record itself cautions that the arms are not a like-for-like control comparison. Enrollment is a registry-level figure and should not be assumed to equal the number with an available result for every outcome.
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: Some of our subjects were scheduled for a cardiac MRI prior to their heart catheterization. If the MRI answered the clinical question, the heart catheterization was not performed and therefore study drug was not administered.
Overall Study
| Stage | 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo |
|---|---|---|---|
| STARTED | 10 | 5 | 13 |
| Evaluable Subjects | 7 | 3 | 6 |
| COMPLETED | 7 | 5 | 9 |
| NOT COMPLETED | 3 | 0 | 4 |
Reasons for not completing this period:
| Reason | 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo |
|---|---|---|---|
| Withdrawal by Subject | 1 | 0 | 0 |
| Procedure cancelled; subject did not receive study drug | 2 | 0 | 2 |
| Met exclusion criteria after obtaining consent | 0 | 0 | 1 |
| Inadequate study staff available to process samples | 0 | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Population includes all subjects who received study drug. This included evaluable and non-evaluable subjects.
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.
| Characteristic | Unit | 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo | Total |
|---|---|---|---|---|---|
| Participants analysed | 10 | 5 | 13 | 28 | |
| Age, Continuous | months | 44.5 (38 to 73) | 14 (6.5 to 18) | 40 (28.5 to 64.5) | 38.5 (27 to 59.5) |
| Age, Customized: >2 yo and ≤ 6 yo | Participants | 10 | 0 | 13 | 23 |
| Age, Customized: ≥1 mo and ≤2 yo | Participants | 0 | 5 | 0 | 5 |
| Sex: Female, Male: Female | Participants | 5 | 3 | 8 | 16 |
| Sex: Female, Male: Male | Participants | 5 | 2 | 5 | 12 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 | 1 | 4 | 8 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 7 | 4 | 9 | 20 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Black or African American | Participants | 0 | 2 | 1 | 3 |
| Race/Ethnicity, Customized: Asian | Participants | 1 | 0 | 1 | 2 |
| Race/Ethnicity, Customized: White or Caucasian | Participants | 7 | 1 | 6 | 14 |
| Race/Ethnicity, Customized: Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Other | Participants | 1 | 2 | 5 | 8 |
| Race/Ethnicity, Customized: Unknown | Participants | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: More than one race | Participants | 1 | 0 | 0 | 1 |
| Region of Enrollment: United States | participants | 10 | 5 | 13 | 28 |
| Weight | kilograms | 15.2 (13.7 to 20.7) | 8.8 (6.1 to 10.9) | 16.1 (13.8 to 17.7) | 14.7 (12.6 to 17.7) |
| Mixing lesion present | Participants | 5 | 2 | 8 | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Samples Obtained Per Subject, measured over Up to 5 hours - 0, 10, 15, 20, 30, 45, 60, 90, 120, 180, 240, and 300 minutes post drug administration.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | Samples |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of samples obtained per subject after atomized intranasal dexmedetomidine administration. The registered summary measure is the median with an inter-quartile range. The endpoint population includes all subjects who received study drug, including evaluable and non-evaluable subjects.
Serum sampling is described at 0, 10, 15, 20, 30, 45, 60, 90, 120, 180, 240, and 300 minutes after administration, over a timeframe of up to 5 hours. The endpoint therefore concerns how many scheduled samples were obtained for each treated subject. It is not itself a measurement of drug concentration, peak concentration, or time to peak.
General statistical context: a sample-count endpoint can help describe the completeness of the measurement record available for pharmacokinetic characterization. If samples are missing or not obtained at all scheduled times, the available concentration-time information may be less complete. The extent and handling of missing samples are not reported in the registry record.
No median, inter-quartile range, or other numerical result for the number of samples obtained per subject is reported in the registry record. As a result, the posted results do not establish how many samples subjects typically contributed or how variable sample collection was. The study’s broader objective includes peak concentration and corresponding time, but numerical results for those objectives are also not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a phase 1 study of intranasal dexmedetomidine in children with congenital heart disease. The design is non-randomized and sequential, and the cohort descriptions vary by dose, age, and anesthesia or airway context. This structure is suited to describing the study groups and collected measurements, but it does not provide a randomized, like-for-like comparison. Any apparent difference across the described groups could reflect more than one difference between them.
The primary endpoint is the number of samples obtained per subject. Although its summary measure is specified as the median and inter-quartile range, neither value is reported in the registry record. The record also does not report a numerical concentration-time profile, peak concentration, or time to peak. Thus, the registry information does not permit a quantitative interpretation of the central pharmacokinetic objective, nor does it show how completely the planned sampling schedule was achieved.
The registry lists an enrollment of 28, but does not report outcome-specific analysis counts. The primary endpoint population is described as all subjects who received study drug, including evaluable and non-evaluable subjects; that definition does not supply the number who actually contributed endpoint data. The registry also lists no analyses. In the absence of reported estimates, uncertainty intervals, or hypothesis tests, there is no posted inferential result to interpret as evidence of a difference or association.
The safety entries list zero subjects with serious adverse events in each of the three groups shown. They also list other adverse events affecting 1 subject in the 2 μg/kg group aged >2 years to ≤6 years, 2 subjects in the 2 μg/kg group aged ≥1 month to ≤2 years, and 3 subjects in the 4 μg/kg group aged >2 years to ≤6 years. These are descriptive counts in groups with different denominators and cohort characteristics. They do not support a causal comparison between dose groups. No deaths are listed in those groups, and no serious adverse-event terms are listed.
General statistical context: estimates describe the observed participants, while confidence intervals, when reported, communicate uncertainty about an estimate under a specified analysis. Neither an interval nor a numerical endpoint estimate appears in the registry record. Consequently, the precision of the pharmacokinetic findings and the sample-count endpoint cannot be assessed from the posted information. The appropriate interpretation is limited to what is explicitly recorded: the cohort structure, endpoint definition, and listed safety counts.
The study is marked terminated. The reason for termination, whether the termination affected data collection, and whether all intended analyses were completed are not reported in the registry record. Termination alone does not establish why the study ended or what its data show. The record’s lack of a numerical primary-endpoint result should be distinguished from a finding of no effect; it means the result is not reported in the registry record.
9. Statistical Methods Explained
What does a median and inter-quartile range describe?
The primary endpoint is summarized using a median and inter-quartile range. General statistical context: the median is the midpoint of an ordered set of observations. The inter-quartile range describes the middle half of those observations, bounded by the lower and upper quartiles. Together, these summaries describe a typical value and the spread around it without requiring the values to follow a normal distribution. They do not, by themselves, show the full distribution or explain why individual observations differ.
For this study, the observations are counts of samples obtained per subject. A median and inter-quartile range could describe the center and spread of those subject-level counts. However, their numerical values are not reported in the registry record. It is therefore not possible to characterize the observed center or variation in sample collection from the posted results.
Why does the sampling schedule matter?
The registry describes serum collection at 0, 10, 15, 20, 30, 45, 60, 90, 120, 180, 240, and 300 minutes after administration. General statistical context: pharmacokinetic characterization uses concentration measurements over time. Measurements at different times contribute information about the concentration-time pattern, including the peak and when it occurs. A subject-level count of samples indicates how much sampling information was obtained, but it does not identify which time points were missed or provide the concentrations themselves.
The registry does not report individual sampling patterns, concentration measurements, or an analysis of missing time points. It also does not report the numerical primary-endpoint summary. Accordingly, neither completeness at particular time points nor the precision of any concentration-time estimate can be assessed from the registry record.
What does a non-randomized sequential design imply?
The study is listed as non-randomized and sequential. General statistical context: random assignment can help balance measured and unmeasured characteristics across groups, on average. Without randomization, group membership may be related to factors such as age, clinical context, or the order in which participants enter the study. Sequential enrollment also means the cohort structure may reflect a progression through groups rather than concurrent assignment to comparable arms. The record does not describe the order or rules governing that progression.
Here, the cohort descriptions differ in more than one respect. The 2 μg/kg descriptions include two age groups and two anesthesia or airway contexts, whereas the 4 μg/kg description is limited to the older age group under general oral endotracheal anesthesia. A contrast between these groups would therefore not isolate dose from age or clinical context. The registry identifies the arms as not being a like-for-like control comparison.
What can be inferred from the absence of analysis results?
The registry lists four outcome measures and zero analyses. This does not establish that no analyses were conducted outside the registry; it means no analysis results are reported in the registry record. No numerical primary-endpoint estimate, confidence interval, p-value, or concentration-time result is listed. Without an estimate and its uncertainty, readers cannot assess the magnitude or precision of the reported primary outcome.
General statistical context: a confidence interval gives a range of values compatible with an estimate under the assumptions of the analysis and conveys uncertainty. A p-value, when used, addresses compatibility between observed data and a specified null model; it is not a measure of clinical importance. Neither type of inferential statistic can be interpreted when it is not reported. No hypothesis-testing approach or model specification is reported in the registry record.
How should the planning assumptions be understood?
The registry describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a specified fraction of the observed effect. For time-to-event settings, the illustration is described as using a total number of events; other settings are described in participants per arm.
These are assumptions for a planning illustration, not evidence of the study’s own sample-size justification or an observed power calculation. No numerical effect, fraction, or sample-size illustration is reported in the registry record. The trial itself is non-randomized and does not describe a like-for-like two-arm comparison, so an equal-allocation illustration should not be mistaken for the design actually used. The registry does not report the protocol’s sample-size justification, whether such a calculation was performed, or whether any interim analyses were planned.
General statistical context: planning calculations depend on assumptions about the target effect, variability, allocation, and analysis. Changing those assumptions changes the resulting planning estimate. Such calculations do not demonstrate that a study had a particular probability of detecting an effect after data collection, and they do not replace the observed estimate and its uncertainty. Here, the numerical inputs and outputs needed to evaluate a planning illustration are not reported in the registry record.
What role does over-dispersion have?
General statistical context: over-dispersion occurs when count data vary more than a simple statistical model assumes. If a count endpoint has substantial heterogeneity, a model that assumes too little variation can yield uncertainty estimates that are too narrow. The median and inter-quartile range offer a descriptive summary of a count distribution, but assessing over-dispersion would require the observed distribution and an applicable model. The registry record does not report such a model or enough numerical endpoint information to assess over-dispersion for sample counts.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four outcome measures and zero analyses. It does not report a multiplicity strategy, such as how inference across multiple outcomes or comparisons would be handled. It also does not report a hierarchy of confirmatory and exploratory outcomes, adjustment procedure, or prespecified set of statistical tests. These details are not reported in the registry record, so the consequences of testing more than one outcome cannot be evaluated.
Missing-data handling is also not reported in the registry record. The primary endpoint’s population is defined as all subjects who received study drug, including evaluable and non-evaluable subjects, but this definition does not explain how unavailable sample counts or incomplete sampling schedules were handled. The record does not describe imputation, exclusions, sensitivity analyses, or the amount and pattern of missing data. It also does not provide outcome-specific numbers analyzed.
The record does not report whether analyses followed an intention-to-treat, per-protocol, or other named analysis population for outcomes beyond the stated primary-endpoint population. Nor does it provide a statistical analysis plan or details on covariate adjustment. Because the design is non-randomized and sequential, information on the analysis population and any adjustment would be relevant to understanding group comparisons; that information is not reported in the registry record.
General statistical context: multiplicity can increase the chance of at least one false-positive result when multiple hypotheses are tested without an appropriate strategy. Missing data can also affect estimates, particularly when the reasons for missingness relate to the measurements or participant characteristics. These are general considerations, not claims about what occurred in this study. The posted information is insufficient to determine whether either issue affected the results.
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 |
|---|---|---|---|
| 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 0 | 7 | 0.0% |
| 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 0 | 5 | 0.0% |
| 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 0 | 9 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 0 of 7 |
| 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 0 of 5 |
| 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 0 of 9 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 1 of 7 |
| 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 2 of 5 |
| 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 3 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 2 μg/kg; Subjects Age >2 yo and ≤ 6 yo | 2 ug/kg; Subjects Age ≥1 mo and ≤2 yo | 4 μg/kg; Subjects Age >2 yo and ≤ 6 yo |
|---|---|---|---|---|
| Bradycardia not requiring intervention | Cardiac disorders | 1 of 7 | 1 of 5 | 1 of 9 |
| Maximum Plasma Concentration | Cardiac disorders | 0 of 7 | 0 of 5 | 3 of 9 |
| Hypotension not requiring intervention | Cardiac disorders | 0 of 7 | 1 of 5 | 0 of 9 |
| New Intraventricular conduction delay | Cardiac disorders | 0 of 7 | 0 of 5 | 0 of 9 |
The registry lists serious adverse events for three groups. In the 2 μg/kg group aged >2 years to ≤6 years, 0 of 7 subjects are listed as affected. In the 2 μg/kg group aged ≥1 month to ≤2 years, 0 of 5 are listed as affected. In the 4 μg/kg group aged >2 years to ≤6 years, 0 of 9 are listed as affected. No serious adverse-event terms are listed, and deaths are listed as 0 in each group.
For other adverse events, the registry lists 1 of 7 subjects affected in the 2 μg/kg, older-age group; 2 of 5 in the 2 μg/kg, younger-age group; and 3 of 9 in the 4 μg/kg, older-age group. These are counts of subjects affected, not a description of event severity or a comparison adjusted for cohort differences. The groups differ in age, dose, or clinical context, and they are not a randomized, like-for-like comparison.
General statistical context: zero recorded events in a group does not establish that an event is impossible or that the underlying risk is zero. The precision of an event estimate depends in part on the amount of observed information. The registry does not report additional safety analyses or a detailed account of event definitions, timing, or ascertainment. These figures should therefore be read as descriptive registry counts, not as proof of comparative safety.
12. What the Result Does, and Does Not, Mean
The record establishes that this was a phase 1, non-randomized, sequential study of intranasal dexmedetomidine in pediatric patients with congenital heart disease. It describes 2 μg/kg and 4 μg/kg cohorts in specified age and anesthesia or airway contexts. It also defines the primary endpoint as the number of samples obtained per subject and gives a serum sampling schedule through 300 minutes after administration.
The record does not report the numerical median or inter-quartile range for the primary endpoint. It does not report numerical peak plasma concentrations, time to peak, confidence intervals, or inferential tests. Therefore, the posted information does not quantify the pharmacokinetic results or the precision of those results. Absence of a posted numerical result is not a finding that there was no effect, no difference, or no pharmacokinetic signal.
The safety entries show zero serious adverse events and zero deaths in each listed group, alongside the group-specific counts of subjects affected by other adverse events. These counts are descriptive. Because the study is non-randomized and the cohorts differ in age, dose, and clinical context, they do not establish that one dose or administration context caused a different safety outcome from another.
The registry’s enrollment figure is 28, but it does not report how many participants contributed data to each outcome. The endpoint population includes subjects who received study drug, whether evaluable or non-evaluable, but the record does not provide a numerical endpoint result or a detailed account of missing observations. The reason the study was terminated is not reported in the registry record.
General statistical context: conclusions about an estimate depend on both its observed value and its uncertainty, as well as the design and analysis that produced it. Here, the missing numerical endpoint and analysis results prevent evaluation of magnitude, precision, and between-group inference. The appropriate scope is therefore descriptive: the record documents the intended population, cohort structure, endpoint definition, sampling schedule, and listed safety counts, but it does not support a quantified conclusion about the primary pharmacokinetic objective.
13. Why This Trial Matters Statistically
This trial is statistically notable because the registry’s stated pharmacokinetic objective and its registered primary endpoint address different aspects of the study. The objective concerns peak plasma concentration and corresponding time after intranasal administration. The primary endpoint is the number of samples obtained per subject. Sample counts can describe the amount of measurement information collected, but they do not substitute for concentration estimates or a time-to-peak result.
The cohort structure also illustrates why group labels alone do not guarantee a valid comparison. The described groups vary by dose, age, and anesthesia or airway context, and allocation is non-randomized and sequential. A numerical difference between groups, if reported, could not automatically be attributed to dose alone. This is why the record’s warning that the arms are not a like-for-like control comparison is important for interpretation.
The record gives a sampling schedule and defines the primary endpoint’s summary measure, but it does not report the endpoint summary or analyses. This leaves an important gap between a clearly specified measurement plan and an interpretable numerical result. Readers cannot assess the observed completeness of sample collection or the uncertainty around a pharmacokinetic estimate from the registry information.
The safety counts likewise demonstrate the distinction between descriptive observations and comparative inference. Counts are listed for serious and other adverse events, but the groups are small and differ in their characteristics. The counts should be read as recorded observations, not as a basis for attributing differences to dose or context.
General statistical context: transparent reporting of analysis populations, missing-data handling, uncertainty, and model assumptions allows readers to understand how observations support conclusions. Those details are not reported in the registry record. The study therefore provides a useful example of how design, endpoint definition, and completeness of reporting jointly constrain statistical interpretation.
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 most direct limitation for interpretation is that the registry record does not report a numerical result for the primary endpoint. Although the endpoint is defined as the number of samples obtained per subject and its summary measure is specified as the median and inter-quartile range, neither summary is listed. Numerical peak concentration and time-to-peak results are also not reported in the registry record.
The study is non-randomized and sequential, and its cohort descriptions differ in age, dose, and anesthesia or airway context. The record explicitly states that the arms are not a like-for-like control comparison. Consequently, the design does not support straightforward causal attribution of any group difference to one feature alone. Progression rules, cohort sequencing details, and the reason for termination are not reported in the registry record.
The registry lists an enrollment of 28, but outcome-specific analysis counts are not reported. The primary-endpoint population includes all subjects who received study drug, including evaluable and non-evaluable subjects; the record does not state how many contributed data or how incomplete sampling was handled. The amount and pattern of missing data are not reported in the registry record.
No analyses are listed. A statistical analysis plan, inferential methods, adjustment strategy, multiplicity handling, and interim-analysis plan are not reported in the registry record. The protocol’s sample-size justification is also not reported. The described planning assumptions do not give numerical planning estimates in the registry record and should not be treated as evidence of the study’s own design rationale.
The safety information is limited to group-level counts of subjects affected and does not include serious adverse-event terms. The registry record does not provide detailed event timing or further safety analyses. These limitations restrict interpretation to the design descriptions and reported counts rather than allowing a comprehensive assessment of efficacy, pharmacokinetic precision, or comparative safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pharmacokinetic Study of Intranasal Dexmedetomidine in Pediatric Patients With Congenital Heart Disease — NCT03417999.
- Registry record last updated 2024-03-22; status last verified 2023-09.
- Results first posted 2024-03-22.
- 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
The registry record describes NCT03417999 as a phase 1, non-randomized, unmasked, sequential study of intranasal dexmedetomidine in pediatric patients with congenital heart disease. The sponsor is Children's Hospital of Philadelphia. The listed enrollment is 28, the status is terminated, the study start date is 2018-06-14, and the primary completion date is 2021-10-12.
The cohort descriptions include 2 μg/kg and 4 μg/kg doses in specified age and anesthesia or airway contexts. The primary endpoint is the number of samples obtained per subject, summarized by median and inter-quartile range, with sampling described through 300 minutes after administration. The primary-endpoint result and numerical pharmacokinetic findings are not reported in the registry record.
Safety entries list zero serious adverse events and zero deaths in each of the three groups, with other adverse events affecting 1, 2, and 3 subjects in the respective groups. The registry lists four outcome measures and zero analyses. It also cautions that the arms are not a like-for-like control comparison. The reason for termination and the details of missing-data and analysis procedures are not reported in the registry record.