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 Cannabidiol Oral Solution in Pediatric Participants With Treatment-resistant Seizure Disorders (NCT02324673), a non-randomized phase 1, phase 2 study. The primary outcome is Maximum Plasma Concentration (Cmax) for Cannabidiol and Metabolite 7-hydroxy (7-OH) Cannabidiol.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02324673 |
| Phase | Phase 1, Phase 2 |
| Condition | Seizures |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 61 |
| Arms in the primary outcome | n/a |
| Lead sponsor | INSYS Therapeutics Inc |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric participants aged 1–17 years, inclusive, with treatment-resistant seizures who satisfied the study’s inclusion and exclusion criteria. The primary endpoint was defined for a pharmacokinetic population: participants who received at least one dose and had at least one post-dose measured plasma concentration of cannabidiol and/or 7-OH cannabidiol with evaluable pharmacokinetic data.
Intervention
Pharmaceutical-grade, nonplant-based cannabidiol oral solution, administered in sequential low-, mid-, and high-dose groups. The target daily doses were 10 mg/kg/day, 20 mg/kg/day, and 40 mg/kg/day.
Comparator
There was no randomized control group. The registry describes a non-randomized, open-label, sequential study, so the dose groups should not be treated as like-for-like randomized comparators.
Primary question
What were the mean maximum plasma concentrations of cannabidiol and its 7-hydroxy metabolite after dosing, as measured at age-specific sampling times on Day 1? Numerical results for these endpoint summaries are not reported in the registry record.
The study also aimed to assess safety, tolerability, pharmacokinetics, and preliminary efficacy across multiple ascending doses. The registry record identifies seizure disorders as the condition of interest and describes participants as having treatment-resistant seizures. It does not report a randomized treatment-effect question comparing cannabidiol with placebo or another treatment. Instead, the primary endpoint concerns measured drug concentrations, which describe pharmacokinetics rather than directly quantifying seizure outcomes.
The sequential dose groups give context for the study’s dose-escalation framework, but group membership was not randomized. Any differences across groups could therefore reflect differences between participants, their sequence through the study, or other factors in addition to dose. The registry record does not report numerical primary endpoint estimates, so the magnitude and precision of the pharmacokinetic summaries cannot be assessed from the recorded information.
3. Trial Design
This was a Phase 1/2, completed, open-label study of cannabidiol oral solution in pediatric participants with treatment-resistant seizure disorders. The registry records a non-randomized allocation and a sequential model. Enrollment was 61. The study began on 2015-04-13 and reached primary completion on 2016-05-09. The sponsor was INSYS Therapeutics Inc.
The brief study description states that it was designed to assess pharmacokinetics, safety, tolerability, and preliminary efficacy using three multiple ascending doses in a sequential fashion. The intervention was pharmaceutical-grade, nonplant-based cannabidiol oral solution. The low-dose group had a target of 10 mg/kg/day, the mid-dose group 20 mg/kg/day, and the high-dose group 40 mg/kg/day. Each group’s regimen began with a morning dose on Day 1, followed by the stated total daily dose from Days 4 to 10, divided between morning and evening doses.
These features matter when interpreting any apparent differences between dose groups. Because allocation was non-randomized and the design was sequential, participants were not assigned by random chance to otherwise comparable arms. The registry record explicitly cautions that the arms are not a like-for-like control comparison. Thus, dose-group summaries may describe the participants and observations within each group, but they do not by themselves establish a causal contrast between doses.
The record describes an open-label study, meaning masking was absent. It does not report a separate control intervention. Although the study description includes preliminary efficacy among its aims, the primary endpoint identified in the record is pharmacokinetic: maximum plasma concentration for cannabidiol and 7-OH cannabidiol. Numerical results for that endpoint, and detailed efficacy estimates, are not reported in the registry record.
The record lists 32 outcome measures, while no analysis entries are recorded. The detailed protocol, statistical analysis plan, sample-size justification, and procedures for interim analyses are not reported in the registry record. The sequential structure and stated aims should not be taken as evidence of any additional design or analysis features beyond those recorded.
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 | Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | High Dose Cannabidiol Oral Solution [40 mg/kg/Day] |
|---|---|---|---|
| STARTED | 20 | 20 | 21 |
| COMPLETED | 20 | 20 | 21 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic population (PK), all participants who received ≥1 dose of study drug and had ≥1 post-dose measured plasma concentration of cannabidiol and/or 7-OH cannabidiol with evaluable PK data available for analysis. The number of participants in each category is the number of participants with evaluable PK data available for Cmax.
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: Safety Analysis Population (SAF), all participants who received ≥1 dose of the investigational product.
| Characteristic | Unit | Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | High Dose Cannabidiol Oral Solution [40 mg/kg/Day] | Total |
|---|---|---|---|---|---|
| Participants analysed | 20 | 20 | 21 | 61 | |
| Age, Continuous | years | 7.5 (SD 5.32) | 7.7 (SD 5.17) | 7.8 (SD 5.39) | 7.6 (SD 5.21) |
| Age, Customized: Infants = 1 to <2 years | Participants | 5 | 5 | 5 | 15 |
| Age, Customized: Children = 2 to <12 years | Participants | 9 | 9 | 10 | 28 |
| Age, Customized: Adolescents = 12 to ≤17 years | Participants | 6 | 6 | 6 | 18 |
| Sex: Female, Male: Female | Participants | 10 | 7 | 11 | 28 |
| Sex: Female, Male: Male | Participants | 10 | 13 | 10 | 33 |
| Region of Enrollment: United States | Participants | 20 | 20 | 21 | 61 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Plasma Concentration (Cmax) for Cannabidiol and Metabolite 7-hydroxy (7-OH) Cannabidiol, measured over Day 1 at age-specific times.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | nanograms/milliliter (ng/mL) |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is maximum plasma concentration, or Cmax, for cannabidiol and its metabolite 7-hydroxy cannabidiol (7-OH cannabidiol), measured in nanograms per milliliter. The endpoint is summarized using a mean and standard deviation. Cmax is a pharmacokinetic measure: it describes the highest concentration recorded in a participant’s sampled blood profile. It is used to characterize systemic drug exposure after administration and can help describe how concentrations vary across participants and dose groups.
The record specifies sampling on Day 1 at age-specific times. Participants aged 1 to <2 years had samples at 2, 4, 8, and 12 hours post-dose. Those aged 2 to <6 years had pre-dose sampling and samples at 1, 2, 3, 4, 8, 12, 16, 24, and 48 hours post-dose. Participants aged 6 through 17 years had pre-dose sampling and samples at 1, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours post-dose.
As a general statistical point, the observed maximum depends on when samples are collected: a measured maximum is the highest concentration captured at the scheduled sampling times, rather than a continuous observation of concentration at every moment. The age-specific schedules therefore define the observation windows used for this endpoint. The registry record does not report numerical mean or standard deviation results, confidence intervals, or the number of evaluable participants in each endpoint category.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary endpoint is a pharmacokinetic summary, not a direct measure of seizure frequency or clinical benefit. It concerns the mean maximum plasma concentration of cannabidiol and 7-OH cannabidiol, with standard deviation used to describe variability around each mean. The registry record identifies the endpoint, measurement unit, timing, and pharmacokinetic population, but numerical endpoint results are not reported in the registry record. Consequently, the recorded information does not establish the observed concentration levels, the degree of variability, or the precision of a group estimate.
The study used sequential, non-randomized dose groups and had no masking. That design can describe observations within each dose group, but it does not provide the protection against confounding that random allocation is intended to provide. If group summaries differ, the design alone cannot determine whether the difference is attributable to dose or to differences between participants or study sequence. The registry itself notes that the arms are not a like-for-like control comparison. No randomized treatment contrast should be inferred.
The age-specific sampling schedules are relevant to interpretation. The timing of blood collection differed by age category, so measured concentration profiles were not observed at an identical set of time points for every participant. As a general statistical consideration, the observed maximum can depend on sampling times: a concentration peak between scheduled samples may not be captured. The registry defines the sampling schedule but does not report how incomplete profiles or missing samples were handled.
Safety information is recorded separately from the primary pharmacokinetic endpoint. Serious adverse event counts and other adverse event counts are listed by dose group, and deaths are recorded as zero in each group. These are descriptive safety figures; because participants were not randomized to comparable arms, they should not be interpreted as a controlled estimate of relative risk. The registry does not provide a statistical comparison for these counts.
The study description also names tolerability and preliminary efficacy as aims, but the primary endpoint reported is Cmax. Numerical results for efficacy outcomes, uncertainty estimates, and analysis methods are not reported in the registry record. In the absence of those details, the record supports a description of the study’s aims and structure, not a conclusion about the magnitude or reliability of any clinical effect.
Any planning illustration based on an observed effect must be interpreted separately from the trial’s results. Such calculations are not observed power and do not replace the trial’s own design rationale. The registry record does not report a sample-size justification or the numerical planning results. The assumptions recorded for the planning illustration do not change the non-randomized sequential design or create a randomized comparison.
9. Statistical Methods Explained
The primary endpoint is a continuous pharmacokinetic measure summarized as a mean and standard deviation. A mean describes the arithmetic average of observed values. A standard deviation describes how dispersed individual values are around that average. These summaries answer different questions: the mean locates the group’s average concentration, while the standard deviation indicates variability among measured values. Neither summary, on its own, gives the uncertainty in the estimated mean.
What does Cmax represent?
Cmax is the maximum plasma concentration recorded for the drug or metabolite over the relevant sampling profile. In general, pharmacokinetic measures such as Cmax describe drug exposure in the body. A higher or lower concentration is not, by itself, a measure of clinical benefit or harm. Its interpretation depends on the study question and the rest of the evidence. In this registry record, the endpoint is stated for cannabidiol and 7-OH cannabidiol, with concentration measured in nanograms per milliliter.
The endpoint is based on serial blood samples on Day 1, with collection times defined by age category. The schedules differ across participants’ age groups. As a general measurement issue, sampling at discrete times can miss a peak that occurs between scheduled collections. The observed maximum is therefore linked to the sampling schedule. The record does not report whether a model was used to estimate concentrations between samples or whether Cmax was defined only from measured concentrations.
What do mean and standard deviation tell the reader?
The mean gives a central summary, while the standard deviation describes spread. A standard deviation is not a confidence interval. A confidence interval, when reported, expresses uncertainty around an estimated quantity under a specified statistical method. It is often used to show a range of estimates compatible with the data and the analysis assumptions. No confidence interval for the primary endpoint is reported in the registry record, so the precision of its mean estimates cannot be assessed from the record.
Means and standard deviations can be useful summaries, but they may not fully describe a distribution if values are strongly skewed or include unusually high observations. As a general statistical consideration, concentration data can have distributions that are not symmetric. The registry identifies the planned summary as mean with standard deviation, but it does not report distributional assessments, transformations, alternative summaries, or a model for the endpoint. Those choices should not be inferred.
Why does the study design matter?
Randomization is a method for assigning participants to groups in a way that can balance measured and unmeasured characteristics on average. This study was non-randomized and sequential, so the dose groups are not equivalent to randomized arms. The record specifically cautions that they are not a like-for-like control comparison. A difference in group means would therefore be descriptive unless supported by an analysis addressing the design and relevant sources of confounding. Numerical mean estimates and an adjusted comparison are not reported in the registry record.
Open-label status means masking was absent. As a general matter, masking can reduce the influence of expectations on measurements or reporting, particularly for outcomes involving judgment or participant report. The primary endpoint is a measured plasma concentration, but open-label status remains part of the design context. The registry record does not report additional procedures to limit bias in measurement or analysis.
How should planning calculations be read?
The record describes a planning illustration based on a fraction of an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. These are calculation assumptions, not evidence that the trial used those features. In particular, equal allocation in an illustration is not the same as the recorded sequential, non-randomized design. Planning sample sizes are hypothetical inputs for future-study planning; they are not observed power and do not measure the probability that the completed study detected an effect.
For a continuous outcome, a planning calculation generally depends on the effect size to be detected, variability, chosen error rate, allocation, and analysis assumptions. If based on an observed effect, the calculation inherits uncertainty in that observed estimate. The registry record does not report numerical values for the planning results or a trial-specific sample-size justification. It also does not report numerical primary endpoint results that would allow readers to assess the observed effect used in the illustration.
What can be concluded from the recorded analysis information?
The record defines a pharmacokinetic population as participants who received at least one dose and had at least one post-dose measured concentration of cannabidiol and/or 7-OH cannabidiol with evaluable pharmacokinetic data. This definition makes clear that the endpoint population is based on both exposure to study drug and availability of evaluable post-dose data. It should not be assumed to include every enrolled participant. The numbers included in each endpoint category are not reported in the registry record.
The record lists 32 outcome measures, but no analysis entries. It does not report the statistical model for Cmax, any covariate adjustment, handling of repeated measurements, confidence interval method, or diagnostic checks. These are not details that can be reconstructed from the endpoint’s mean-and-standard-deviation designation. As a general principle, analysis methods should be interpreted from a prespecified statistical plan or a clear results report; neither is described in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 32 outcome measures, including the primary pharmacokinetic endpoint, but does not report a multiplicity strategy. Multiplicity concerns the increased chance of a false-positive finding when many outcomes or comparisons are examined. General approaches can include defining a hierarchy of outcomes or adjusting statistical inference, but no such procedure is reported in the registry record. It is also not reported whether the listed outcomes were all analyzed or which were considered exploratory beyond the study description’s reference to preliminary efficacy.
Missing-data methods are not reported in the registry record. The primary endpoint population requires at least one dose and at least one post-dose measured concentration with evaluable pharmacokinetic data. This criterion describes who could contribute to the pharmacokinetic analysis; it does not explain how missing blood samples, incomplete profiles, or participants without evaluable data were handled. The number of participants contributing to each endpoint category is not reported in the registry record.
The sampling schedule varied by age group. That is a planned feature of measurement, not necessarily missing data. However, the record does not say how departures from the scheduled sampling times were treated, whether values were imputed, or whether participants with incomplete sampling were included in Cmax summaries. The extent and pattern of missing data are also not reported.
The record does not report an analysis population for outcomes beyond the stated pharmacokinetic population, nor does it describe an intention-to-treat analysis. The detailed statistical plan, sensitivity analyses, and procedures for handling multiplicity or missingness are not reported in the registry record. Without these details, readers cannot determine how robust any endpoint estimate would be to alternative assumptions about missing measurements or multiple outcomes.
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 |
|---|---|---|---|
| Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | 0 | 20 | 0.0% |
| Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | 1 | 20 | 5.0% |
| High Dose Cannabidiol Oral Solution [40 mg/kg/Day] | 2 | 21 | 9.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | 0 of 20 |
| Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | 0 of 20 |
| High Dose Cannabidiol Oral Solution [40 mg/kg/Day] | 0 of 21 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | 13 of 20 |
| Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | 9 of 20 |
| High Dose Cannabidiol Oral Solution [40 mg/kg/Day] | 17 of 21 |
Most frequent serious adverse event terms
| Event term | Organ system | Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | High Dose Cannabidiol Oral Solution [40 mg/kg/Day] |
|---|---|---|---|---|
| Apnoea | Respiratory, thoracic and mediastinal disorders | 0 of 20 | 0 of 20 | 1 of 21 |
| Drug eruption | Skin and subcutaneous tissue disorders | 0 of 20 | 0 of 20 | 1 of 21 |
| Thrombophlebitis | Vascular disorders | 0 of 20 | 1 of 20 | 0 of 21 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Low Dose Cannabidiol Oral Solution [10 mg/kg/Day] | Mid Dose Cannabidiol Oral Solution [20 mg/kg/Day] | High Dose Cannabidiol Oral Solution [40 mg/kg/Day] |
|---|---|---|---|---|
| Somnolence | Nervous system disorders | 3 of 20 | 3 of 20 | 7 of 21 |
| Anaemia | Blood and lymphatic system disorders | 2 of 20 | 5 of 20 | 4 of 21 |
| Diarrhoea | Gastrointestinal disorders | 1 of 20 | 2 of 20 | 7 of 21 |
| Flatulence | Gastrointestinal disorders | 0 of 20 | 2 of 20 | 3 of 21 |
| Psychomotor hyperactivity | Nervous system disorders | 0 of 20 | 2 of 20 | 3 of 21 |
| Abdominal pain upper | Gastrointestinal disorders | 2 of 20 | 1 of 20 | 0 of 21 |
| Vomiting | Gastrointestinal disorders | 2 of 20 | 1 of 20 | 0 of 21 |
| Catheter site pruritus | General disorders | 0 of 20 | 2 of 20 | 1 of 21 |
| Weight increased | Investigations | 0 of 20 | 1 of 20 | 2 of 21 |
| Decreased appetite | Metabolism and nutrition disorders | 2 of 20 | 0 of 20 | 1 of 21 |
Serious events with the highest arm-level rate: thrombophlebitis (5.0%), apnoea (4.8%), drug eruption (4.8%).
The registry lists serious adverse events by dose group, alongside the number at risk, other adverse events, and deaths. In the low-dose group, 20 participants were at risk for serious adverse events and 0 were affected; 20 were at risk for other adverse events and 13 were affected. In the mid-dose group, 20 were at risk for serious adverse events and 1 was affected; 20 were at risk for other adverse events and 9 were affected. In the high-dose group, 21 were at risk for serious adverse events and 2 were affected; 21 were at risk for other adverse events and 17 were affected. Deaths were recorded as 0 in each group.
These figures describe affected participants as recorded for each category; they are not a randomized comparison. The registry does not define the “other” category in the information, so it should not automatically be read as synonymous with non-serious adverse events. The record lists thrombophlebitis at 5.0%, apnoea at 4.8%, and drug eruption at 4.8% among the top serious terms. The denominators and term-specific group assignments for those percentages are not reported in the registry record.
As a general statistical point, adverse event counts are descriptive unless the study design and analysis support a comparative estimate. Because allocation was non-randomized and sequential, the figures alone do not establish that one dose group had greater or lesser risk than another.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this completed study was designed to examine pharmacokinetics, safety, tolerability, and preliminary efficacy of cannabidiol oral solution in pediatric participants with treatment-resistant seizure disorders. It identifies three sequential dose groups and a primary endpoint based on maximum plasma concentrations of cannabidiol and 7-OH cannabidiol. It also records the sampling schedules and defines the pharmacokinetic population.
Those details do not establish the observed Cmax values. Numerical means, standard deviations, confidence intervals, and group-specific endpoint population counts are not reported in the registry record. Without these results, the concentration levels, their variability, and the precision of the estimates cannot be evaluated. The study’s aims should not be mistaken for reported findings.
The safety counts describe events recorded in each group, including serious adverse events, other adverse events, and deaths. They do not provide a controlled estimate of differences between doses. The study was open-label, non-randomized, and sequential, and the registry explicitly cautions that its arms are not like-for-like control groups. A descriptive difference across those groups would not by itself show that dose caused the difference.
The primary endpoint is pharmacokinetic rather than a direct measure of seizures or another clinical outcome. Pharmacokinetic information can characterize measured concentrations, but it does not by itself establish clinical efficacy or safety. The registry record mentions preliminary efficacy as an aim, but numerical efficacy results and their statistical uncertainty are not reported in the registry record.
The record also does not report the detailed statistical plan, missing-data methods, multiplicity procedures, or sample-size justification. Accordingly, readers can understand the study’s stated design and endpoint framework, but cannot judge from the registry information alone how precise the primary endpoint estimates were, how the planned analyses were conducted, or how conclusions would change under alternative assumptions.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of why design and endpoint type must be considered alongside dose-group summaries. Its primary endpoint is a pharmacokinetic measure: maximum plasma concentration for cannabidiol and 7-OH cannabidiol. That is distinct from a clinical outcome such as seizure frequency. A concentration endpoint can describe measured exposure, but it does not substitute for evidence about clinical outcomes.
The age-specific blood-sampling schedules also make measurement design important. Cmax is derived from a profile observed at scheduled times, and the schedules differed by age group. In general, a recorded maximum is tied to when samples were taken. This highlights why an endpoint definition needs to be read together with its timing and population criteria, rather than treated as a standalone label.
The sequential, non-randomized allocation is central to interpretation. Dose groups can provide descriptive information about participants who received those regimens, but they do not form a randomized comparison. This distinction is especially important when readers encounter summaries arranged by dose: a group contrast is not automatically a causal dose effect. The registry’s explicit warning against treating the arms as like-for-like controls reinforces that point.
The record also illustrates the limits of registry reporting. It describes the endpoint and adverse event counts, but numerical primary endpoint results, detailed statistical methods, and approaches to missing data and multiplicity are not reported in the registry record. Statistical interpretation depends not only on a named endpoint, but also on the estimates, uncertainty, population analyzed, and analysis procedures that produced them.
Finally, planning calculations should be kept separate from observed results. A sample-size illustration based on an observed effect is a planning tool under stated assumptions, not observed power and not a substitute for a trial-specific design rationale. The registry record does not report the trial’s sample-size justification. Recognizing these distinctions helps prevent descriptive pharmacokinetic and safety information from being overstated as comparative clinical evidence.
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 registry record does not report numerical results for the primary endpoint. It gives the endpoint definition, unit, summary type, sampling schedule, and pharmacokinetic population criteria, but not the group means, standard deviations, confidence intervals, or endpoint-specific participant counts. This prevents assessment of the observed concentrations and their precision.
The design limits comparative interpretation. Allocation was non-randomized, the study was sequential and open-label, and the record states that the arms are not a like-for-like control comparison. The number enrolled is recorded as 61, but enrollment alone does not establish how many participants contributed to each endpoint or adverse event analysis. Those group-specific analysis counts are not reported in the registry record.
Sampling times differed across age groups, which is part of the endpoint’s measurement context. The registry record does not report whether all scheduled samples were obtained, how incomplete profiles were handled, or whether Cmax was estimated by a model or taken from measured concentrations. It also does not report missing-data methods, multiplicity procedures, a detailed statistical analysis plan, or a sample-size justification.
The safety information is descriptive. The record lists affected counts by group, but does not provide a randomized comparison or statistical uncertainty for the counts. The context and denominators for the listed serious adverse event terms are not fully described. The record’s aims include preliminary efficacy, but numerical efficacy findings are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Cannabidiol Oral Solution in Pediatric Participants With Treatment-resistant Seizure Disorders — NCT02324673.
- Registry record last updated 2017-06-23; status last verified 2017-05.
- Results first posted 2017-06-23.
- 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
NCT02324673 is a completed Phase 1/2, open-label, non-randomized, sequential study of cannabidiol oral solution in pediatric participants with treatment-resistant seizure disorders. The registry records enrollment of 61 and three target daily dose groups: 10 mg/kg/day, 20 mg/kg/day, and 40 mg/kg/day. The study aimed to assess pharmacokinetics, safety, tolerability, and preliminary efficacy.
The primary endpoint was maximum plasma concentration of cannabidiol and 7-OH cannabidiol, summarized as a mean with standard deviation and measured using age-specific Day 1 sampling schedules. The record defines a pharmacokinetic analysis population but does not report numerical primary endpoint results or endpoint-specific population counts. Safety counts and deaths are recorded by dose group, but the non-randomized design does not support treating the groups as like-for-like controls. Detailed statistical methods, missing-data procedures, multiplicity handling, and sample-size justification are not reported in the registry record.