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 Study of Fentanyl-Propofol-EMLA of L.M.X4 Technique for Bone Marrow Aspiration (NCT00187135), a randomized phase 3 study. The primary outcome is Pain(Yes/No).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00187135 |
| Phase | Phase 3 |
| Condition | Bone Marrow Disease, Pain |
| Allocation | Randomized |
| Design model | Crossover |
| Masking | Quadruple |
| Enrollment | 168 |
| Arms in the primary outcome | n/a |
| Lead sponsor | St. Jude Children's Research Hospital |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric patients undergoing procedures that included bone marrow aspiration. The primary pain analysis used a subset of the 162 randomized participants: participants had to complete at least Visit 2 and receive each of the two treatments being compared. The exact number included in each pain analysis is not reported in the registry record.
Intervention
The study concerns combinations involving fentanyl and propofol with a topical numbing cream, EMLA or L.M.X4. The registry also lists fentanyl doses of 0.5 mcg/kg and 1mcg/kg, with normal saline as the placebo arm. The precise mapping of the topical creams and propofol to the listed arms is not reported in the registry record.
Comparator
The listed arm descriptions include fentanyl 0.5mcg/kg, fentanyl 1mcg/kg, and normal saline. The study summary describes three anesthetic combinations used across three procedures for each patient. The specific treatment pairings used in each primary comparison are not reported in the registry record.
Primary question
During sedation recovery after the procedure, did participants experience any pain, defined as a score greater than 0 on an age-appropriate pediatric pain scale? The registry identifies pain as yes/no and describes monitoring from the end of the procedure until recovery, for a maximum of 65 min. The pain-comparison estimates and their uncertainty are not reported in the registry record.
This is a within-participant clinical question: the record says each enrolled participant was intended to have three anesthetic combinations for three different procedures. That structure allows treatment experiences from the same child to be compared, rather than treating every procedure as an observation from a different child. The record also says treatment on each visit was randomized. A crossover design can make within-person contrasts informative because stable characteristics of a participant are shared across their treatment experiences. That general advantage depends on the analysis recognizing that observations from the same participant are related.
The registry describes the study as Phase III and randomized, with an enrollment of 168. Its summary says researchers aimed to study pain control during and after procedures such as bone marrow aspiration and lumbar puncture with intrathecal chemotherapy. The primary endpoint described in the results record, however, is pain during sedation recovery. The primary pain comparison results and the treatment-specific estimates are not reported in the registry record, so the clinical question can be stated but cannot be answered from the posted outcome details.
3. Trial Design
The registry identifies this as a Phase III, randomized, quadruple-masked crossover study sponsored by St. Jude Children's Research Hospital. Enrollment is listed as 168, while the primary-outcome description refers to 162 randomized participants. The registry does not explain the difference between those enrollment figures. The study began in 2002-03, primary completion is listed as 2008-08, and status is recorded as terminated. The reason for termination is not reported in the registry record.
The crossover structure is central to interpreting the study. According to the brief summary, each patient would have three different anesthetic combinations for three different procedures. The primary-outcome description further states that treatment on each visit was randomized. The listed arm descriptions are fentanyl 1mcg/kg in 3 ml of normal saline, fentanyl 0.5 mcg/kg in 3 ml normal saline, and normal saline. The interventions list also includes EMLA, L.M.X4, and propofol. The exact treatment sequence, which topical cream was paired with which regimen, and the role of propofol in the listed arms are not reported in the registry record.
In a crossover trial, a participant may contribute observations under more than one treatment. This creates a paired or repeated-measures structure: observations from the same participant are generally not independent. General statistical practice is to use an analysis that accounts for this dependence and for the randomized treatment sequence. The specific statistical model, sequence handling, and any adjustment for period or carryover effects are not reported in the registry record.
Quadruple masking indicates that the registry classified the study as masked at four levels, but it does not identify the roles of those masked or describe how masking was maintained. The randomization method, allocation concealment, and masking procedures are not reported in the registry record. Those details would help readers assess how expectations and procedural knowledge may have influenced treatment delivery or pain scoring.
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: 168 participants were enrolled and were expected to receive 3 Bone Marrow Aspirations/Lumbar Puncture Intrathecal Chemotherapy procedures; 6 patients withdrew (eg: medical withdrawal, withdrawal of consent and other). 162 were randomized, of which 77 completed all 3 treatments.
Visit One
| Stage | Fentanyl 0.5 / Placebo / Fentanyl 1 | Fentanyl 0.5 /Fentanyl 1 / Placebo | Placebo / Fentanyl 0.5 /Fentanyl 1 | Placebo /Fentanyl 1 / Fentanyl 0.5 | Fentanyl 1 / Fentanyl 0.5 / Placebo | Fentanyl 1 / Placebo /Fentanyl 0.5 |
|---|---|---|---|---|---|---|
| STARTED | 30 | 28 | 25 | 26 | 25 | 28 |
| COMPLETED | 26 | 27 | 25 | 26 | 25 | 26 |
| NOT COMPLETED | 4 | 1 | 0 | 0 | 0 | 2 |
Reasons for not completing this period:
| Reason | Fentanyl 0.5 / Placebo / Fentanyl 1 | Fentanyl 0.5 /Fentanyl 1 / Placebo | Placebo / Fentanyl 0.5 /Fentanyl 1 | Placebo /Fentanyl 1 / Fentanyl 0.5 | Fentanyl 1 / Fentanyl 0.5 / Placebo | Fentanyl 1 / Placebo /Fentanyl 0.5 |
|---|---|---|---|---|---|---|
| Withdrawal by Subject | 1 | 0 | 0 | 0 | 0 | 1 |
| Physician Decision | 3 | 1 | 0 | 0 | 0 | 1 |
Visit Two
| Stage | Fentanyl 0.5 / Placebo / Fentanyl 1 | Fentanyl 0.5 /Fentanyl 1 / Placebo | Placebo / Fentanyl 0.5 /Fentanyl 1 | Placebo /Fentanyl 1 / Fentanyl 0.5 | Fentanyl 1 / Fentanyl 0.5 / Placebo | Fentanyl 1 / Placebo /Fentanyl 0.5 |
|---|---|---|---|---|---|---|
| STARTED | 26 | 27 | 25 | 26 | 25 | 26 |
| COMPLETED | 21 | 24 | 23 | 18 | 19 | 18 |
| NOT COMPLETED | 5 | 3 | 2 | 8 | 6 | 8 |
Reasons for not completing this period:
| Reason | Fentanyl 0.5 / Placebo / Fentanyl 1 | Fentanyl 0.5 /Fentanyl 1 / Placebo | Placebo / Fentanyl 0.5 /Fentanyl 1 | Placebo /Fentanyl 1 / Fentanyl 0.5 | Fentanyl 1 / Fentanyl 0.5 / Placebo | Fentanyl 1 / Placebo /Fentanyl 0.5 |
|---|---|---|---|---|---|---|
| Death | 0 | 0 | 0 | 1 | 0 | 1 |
| Physician Decision | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrawal by Subject | 1 | 1 | 1 | 0 | 1 | 1 |
| Study Discontinued | 3 | 2 | 1 | 7 | 5 | 6 |
Visit Three
| Stage | Fentanyl 0.5 / Placebo / Fentanyl 1 | Fentanyl 0.5 /Fentanyl 1 / Placebo | Placebo / Fentanyl 0.5 /Fentanyl 1 | Placebo /Fentanyl 1 / Fentanyl 0.5 | Fentanyl 1 / Fentanyl 0.5 / Placebo | Fentanyl 1 / Placebo /Fentanyl 0.5 |
|---|---|---|---|---|---|---|
| STARTED | 21 | 24 | 23 | 18 | 19 | 18 |
| COMPLETED | 14 | 17 | 14 | 9 | 11 | 12 |
| NOT COMPLETED | 7 | 7 | 9 | 9 | 8 | 6 |
Reasons for not completing this period:
| Reason | Fentanyl 0.5 / Placebo / Fentanyl 1 | Fentanyl 0.5 /Fentanyl 1 / Placebo | Placebo / Fentanyl 0.5 /Fentanyl 1 | Placebo /Fentanyl 1 / Fentanyl 0.5 | Fentanyl 1 / Fentanyl 0.5 / Placebo | Fentanyl 1 / Placebo /Fentanyl 0.5 |
|---|---|---|---|---|---|---|
| Death | 0 | 0 | 1 | 0 | 1 | 0 |
| Withdrawal by Subject | 0 | 1 | 1 | 2 | 0 | 0 |
| Study Discontinued | 7 | 6 | 7 | 7 | 7 | 6 |
Analysis population for the primary outcome, as defined in the registry: Participants in this analysis are a subset of the 162 participants randomized since not all completed 3 visits on study. If a participant completed at least Visit 2 and received each of the 2 treatments compared they are included. Treatment on each visit was randomized; therefore these numbers cannot be derived directly from the participant flow.
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.
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | Fentanyl 0.5/Placebo/Fentanyl 1 | Fentanyl 0.5/Fentanyl 1/Placebo | Placebo/Fentanyl 0.5/Fentanyl 1 | Placebo/Fentanyl 1/Fentanyl 0.5 | Fentanyl 1/Fentanyl 0.5/Placebo | Fentanyl 1/Placebo/Fentanyl 0.5 |
|---|---|---|---|---|---|---|---|
| Participants analysed | 30 | 28 | 25 | 26 | 25 | 28 | |
| Age, Continuous | years | 6.4 (SD 4.3) | 7.2 (SD 4.5) | 7.6 (SD 4.8) | 7.1 (SD 4.1) | 7.3 (SD 4.7) | 7.1 (SD 4.4) |
| Sex: Female, Male: Female | Participants | 15 | 11 | 12 | 10 | 9 | 13 |
| Sex: Female, Male: Male | Participants | 15 | 17 | 13 | 16 | 16 | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Pain(Yes/No), measured over The participant was monitored from the end of the procedure until sedation recovery, which lasted a maximum of 65 min. Discharge from the recovery area was determined by hospital standards of care..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is recorded as “Pain(Yes/No)” among participants. During recovery from sedation, pain was assessed using one of three validated pediatric scales: the Numerical Pain Scale, the FACES pain scale, or the FLACC scale. The registry says scale choice depended on the participant’s developmental ability. Each scale ranges from 0 - 10 units, and the registry describes them as interchangeable for comparison purposes. A score greater than 0 was classified as pain; a score of 0 was classified as no pain. This produced one binary score for each participant’s procedure.
The observation period began at the end of the procedure and continued until sedation recovery, which lasted a maximum of 65 min. Discharge from recovery was determined by hospital standards of care. The binary threshold makes the outcome straightforward to interpret as the occurrence or absence of any recorded pain during that recovery period. It also discards information about the magnitude of a nonzero score: scores just above 0 and scores nearer the top of the scale both count as pain. That is a general feature of converting a scale into a yes/no outcome, not a finding about this trial’s participants.
The registry does not report treatment-specific pain counts, proportions, effect estimates, confidence intervals, or p-values. Consequently, the endpoint definition is available, but the size and precision of any treatment contrast cannot be assessed from the posted pain results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record supplies the definition of the primary pain outcome and describes the population eligible for the analysis, but it does not report the results of the pain comparisons. No treatment-specific pain counts or effect estimates are present in the registry record for interpretation. Confidence intervals and p-values for pain are also not reported in the registry record. It is therefore not possible to describe the direction, magnitude, or precision of a primary pain contrast.
The primary analysis population was not simply every randomized participant. The record says it was a subset of the 162 randomized participants, including those who completed at least Visit 2 and received each of the two treatments compared. That condition makes inclusion depend on having repeated treatment information. How many participants met the condition for each analysis is not reported in the registry record. Nor is the handling of participants with incomplete visits, missing pain scores, or other missing data described.
Because this is a crossover study, observations from a participant across visits are connected. General statistical interpretation should distinguish a comparison that uses within-participant information from one that treats treatment groups as independent. The registry establishes randomized treatment on each visit but does not report the model used to estimate pain contrasts or whether the repeated-measures structure was incorporated. Without that information and the estimates themselves, a statistically sound account of the result is not possible.
The endpoint is binary because every scale score greater than 0 is coded as pain, while a score of 0 is coded as no pain. A treatment contrast for such an endpoint would ordinarily describe how often pain was recorded in one condition relative to another. The registry entries do not include the counts needed to state such a contrast. It would also be inappropriate to infer that the creams, fentanyl doses, or placebo differed based on the study’s rationale or its title. A hypothesis is not an outcome.
The registry lists six outcome measures and seven analyses, but the available details do not identify all analysis methods or explain how the primary comparisons were selected. The record’s skip note says the primary outcome has several categories or time points and was not reduced to one comparison. That caveat limits any attempt to treat the endpoint description as a complete account of the primary analysis. The distinctions among comparisons and time points, as well as their results, are not reported in the registry record.
For the safety outcomes, the listed groups have zero serious adverse events and zero other adverse events recorded. These are descriptive registry counts, not a demonstration that adverse events could not occur. The corresponding at-risk counts are 107, 104, and 105 for the fentanyl 0.5mcg/kg, fentanyl 1mcg/kg, and placebo groups, respectively. Death counts are null, which means they are not reported in the registry record rather than being established as zero. These safety entries do not supply evidence about the primary pain endpoint.
Overall, the posted information supports a description of the study structure, pain measurement, and recorded safety counts. It does not support a claim about which regimen had fewer pain reports, whether any contrast was statistically distinguishable from no difference, or how precisely treatment effects were estimated.
9. Statistical Methods Explained
What does the crossover design imply?
General statistical explanation: a crossover trial evaluates more than one treatment condition within the same participant. The participant can serve as their own reference, which can reduce the influence of stable differences between people on a treatment contrast. In this study, the registry summary describes three anesthetic combinations across three procedures, and the primary-outcome description says treatment on each visit was randomized. Thus, the intended data have a repeated-measures structure rather than a simple set of independent observations.
That structure has an important analytical consequence. Responses from the same child may be more alike than responses from different children, so treating each procedure as independent can misstate uncertainty. A suitable analysis generally accounts for within-participant association and the treatment assignment sequence. It may also need to consider visit or period effects and the possibility that an earlier procedure or treatment influences a later observation. These are general considerations for crossover designs. The statistical model, period adjustment, sequence analysis, and any assessment of carryover are not reported in the registry record.
How should the binary pain measure be understood?
The registry defines a binary endpoint from three pediatric pain scales. Scale selection depended on developmental ability, and a score greater than 0 was classified as pain; a score of 0 was classified as no pain. The reported endpoint therefore answers whether any pain was recorded during the defined recovery period, not how high the pain score was among participants with nonzero scores. General statistical explanation: dichotomizing a graded measure can simplify interpretation, but it loses distinctions in severity and can make the result sensitive to the chosen threshold. The threshold used in this study is specified in the registry.
For a binary outcome, treatment results can be presented as counts and proportions in each condition, followed by a contrast and an uncertainty interval. The contrast might be expressed on different statistical scales, but the registry entries do not identify the scale or provide the pain results. No treatment-specific pain counts, estimates, confidence intervals, or p-values are reported in the registry record. Those quantities should not be reconstructed from the endpoint definition alone.
What does randomization contribute?
The registry classifies allocation as randomized and states that treatment on each visit was randomized. General statistical explanation: random assignment is intended to reduce systematic differences in treatment allocation, supporting a fair comparison of assigned conditions. In a crossover design, the order of exposure matters as well as the treatment itself. The registry does not describe the sequence-generation method, allocation concealment, or whether the analysis followed assigned treatment or treatment received. Those details are important for assessing whether the planned randomization is reflected in the analysis.
The record classifies masking as quadruple, but does not specify who was masked or the procedures used to maintain masking. General statistical explanation: masking can reduce the influence of expectations on care, outcome assessment, and reporting. Its relevance is especially concrete for a pain endpoint that depends on scale-based assessment. Still, the masking label alone does not establish how effectively masking was maintained.
How should the analysis population be read?
The pain-outcome description identifies a subset of 162 randomized participants. A participant was included in a particular comparison if they completed at least Visit 2 and received each of the two treatments compared. That definition reflects the need for repeated observations in a crossover contrast, but it also means that the analyzed participants may not equal all randomized participants. The number included in each comparison, reasons for missing visits, and reasons for exclusion are not reported in the registry record.
General statistical explanation: an analysis limited to participants with usable observations under both conditions can lose precision and may be biased if completion is related to treatment response or other characteristics. Methods can address missingness in different ways, but no imputation method, missing-data assumption, or sensitivity analysis is reported in the registry record. The intended analysis population and the population used for each final estimate should be distinguished; the latter is not described in the available record.
What do the planning calculations mean?
The registry describes sample-size calculations as planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. For time-to-event outcomes, the stated convention is a total number of events; for other outcomes, it is participants per arm. These are planning illustrations, not observed power and not the trial’s original design justification. The actual effect fractions and resulting sample-size values are not reported in the registry record.
General statistical explanation: a planning calculation asks what sample size might be needed to detect a specified effect under stated assumptions. It does not establish the probability that the completed study found a real effect, and an observed effect used for planning can be an imprecise basis for future planning. The trial’s own sample-size justification, target power, and assumptions at the design stage are not reported in the registry record.
What can be inferred from the adverse-event counts?
The safety listings give the numbers at risk and the numbers affected for serious and other adverse events in each group. The affected counts are zero in all listed groups. General statistical explanation: an observed count of zero is a description of the recorded data, not proof of zero underlying risk. Precision depends on the amount of information and on how events were collected and classified. The registry does not report a detailed safety-analysis method, event-collection procedures, or confidence intervals for these counts. Death counts are null and are not interpretable as observed zeroes.
In sum, the registry permits a clear account of the endpoint and broad design, while leaving the principal inferential methods and pain results unspecified. General statistical principles clarify why crossover dependence, missing observations, binary coding, and uncertainty matter; they do not fill in the trial-specific methods that are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists six outcome measures and seven analyses. It does not provide enough detail to identify the complete set of comparisons, how they were grouped, or which were designated primary for statistical testing. Its skip note says the primary outcome has several categories or time points and was not reduced to one comparison. The exact relationship among those categories, time points, analyses, and any reported outcome values is not reported in the registry record.
General statistical explanation: when several outcomes, treatment contrasts, or time points are tested, the chance of at least one apparently unusual result can increase if every test is interpreted without regard to the number of tests. Methods such as prespecifying a primary contrast or adjusting for multiple comparisons can address that issue, but the registry does not report whether such methods were planned or used. It also does not report a testing hierarchy, multiplicity adjustment, or a rationale for selecting analyses. No conclusion about multiplicity control can therefore be drawn.
The analysis population requires particular care. Participants were included in a comparison if they completed at least Visit 2 and received both treatments being compared. The number included for each contrast and the number with incomplete observations are not reported in the registry record. The record does not describe missing-data methods, assumptions about why observations were missing, or sensitivity analyses. In a crossover study, incomplete visit data can affect which within-participant contrasts are available; the scale of that issue cannot be evaluated without participant-flow and analysis counts.
The registry records 168 enrolled and describes a subset of 162 randomized participants for the primary pain analysis. It does not explain the relation between these figures, give the count analyzed for each comparison, or provide a complete account of exclusions. The distinction between randomized participants and the analysis subset should therefore remain explicit when interpreting any outcome claim.
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 |
|---|---|---|---|
| Fentanyl 0.5mcg/kg | 0 | 107 | 0.0% |
| Fentanyl 1mcg/kg | 0 | 104 | 0.0% |
| Placebo | 0 | 105 | 0.0% |
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 |
|---|---|
| Fentanyl 0.5mcg/kg | 0 of 107 |
| Fentanyl 1mcg/kg | 0 of 104 |
| Placebo | 0 of 105 |
The registry lists three safety groups: fentanyl 0.5mcg/kg, fentanyl 1mcg/kg, and placebo. Their respective at-risk counts are 107, 104, and 105. The serious adverse event counts are zero in all three groups, and the counts for other adverse events are also zero in all three groups. These values describe the entries recorded for the listed groups; they should not be read as proof that no event could occur or as evidence that the groups are equivalent in safety.
The record lists no serious adverse event terms. Death counts are null rather than zero, so deaths are not reported in the registry record. The safety information does not describe how events were solicited, how long participants were followed for safety, or how events were attributed to treatment. Those details are not reported in the registry record.
General statistical explanation: a zero event count can still be compatible with uncertainty about the underlying event rate. The size of that uncertainty depends on the number observed, follow-up, and event ascertainment. No confidence intervals or comparative safety estimates are reported. These safety counts are separate from the primary pain endpoint and do not establish a difference in pain during recovery.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a randomized, quadruple-masked crossover study of pediatric procedural sedation, with a stated enrollment of 168 and a primary endpoint based on pain during recovery. It also describes a subset of the 162 randomized participants for the pain analysis and provides safety counts for three listed groups. These registry entries define the context in which the study should be read.
The record does not include the treatment-specific pain counts or comparative estimates needed to tell whether pain was more or less frequently recorded under any listed regimen. It also does not provide confidence intervals or p-values for the pain endpoint. Therefore, no statement about the direction or size of a treatment difference, statistical compatibility with no difference, or precision of a treatment estimate can be supported by the posted information.
The study rationale describes an expectation that a combination of fentanyl and propofol with a topical numbing cream would provide better pain control. That expectation is a hypothesis, not a result. The title and rationale cannot substitute for the outcome data. Likewise, the safety entries with zero recorded events do not establish that risks are absent or that treatments have the same safety profile. Death counts are not reported.
General statistical explanation: a result is more informative when it includes an effect estimate and an uncertainty interval, not only a test result or a direction of effect. For this record, those primary pain quantities are not reported. The endpoint also collapses a graded pain scale into a yes/no classification, so even a complete binary result would address the occurrence of any recorded pain rather than the distribution of pain scores across their full range.
The trial structure suggests repeated observations within participants, but the statistical model and handling of incomplete visits are not reported in the registry record. That prevents assessment of how the analysis accounted for within-participant dependence and missing observations. The appropriate conclusion is limited: the design and endpoint are described, while the comparative pain result and several details needed to evaluate its analysis are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable because its crossover design asks participants to experience more than one treatment condition. Such designs can provide within-participant comparisons, which may control for stable differences between participants. They also require careful analysis: repeated observations from one person are not generally independent, and an incomplete sequence can limit the available paired information. The registry describes randomized treatment on each visit but does not report the model used to account for these features.
The endpoint illustrates a second important issue in clinical measurement. Pain was assessed with one of three validated pediatric scales chosen according to developmental ability, and the result was reduced to pain versus no pain using a score greater than 0 as the threshold. This permits a simple occurrence-based interpretation across scales, while losing information about the degree of pain. Readers should distinguish that binary question from questions about severity or change in the full score.
The registry also illustrates why an analysis population must be described in relation to the design. The primary pain analysis included a subset of randomized participants who completed at least Visit 2 and received both treatments in a comparison. Without counts by comparison or an account of missing observations, the reader cannot determine how much of the randomized experience is represented in each analysis.
Finally, the difference between a trial’s stated rationale and its reported results matters. The study summary presents a reason for evaluating combinations of analgesic and anesthetic medicines with topical creams, but the primary pain comparison results are not reported in the registry record. Statistical interpretation must rest on estimates and their uncertainty, not on the motivation for testing a treatment or the design label alone. The listed safety zeros are likewise descriptive counts, not a comparative 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 largest limitation for interpreting efficacy is that the primary pain comparison results are not reported in the registry record. Treatment-specific counts, effect estimates, confidence intervals, and p-values are unavailable in the registry record described. The endpoint can be explained, but its comparative result cannot be evaluated.
The primary analysis population is described as a subset of 162 randomized participants, requiring completion of at least Visit 2 and receipt of each treatment in a comparison. The number analyzed for each contrast and the reasons participants did not contribute are not reported. The registry also lists enrollment of 168 without explaining the difference from the randomized count. These gaps limit assessment of participant flow and potential selection related to incomplete visits.
Details needed to understand the crossover implementation are limited. Treatment was randomized on each visit and the study is classified as quadruple-masked, but the sequence-generation process, allocation concealment, masked roles, and masking procedures are not reported in the registry record. The exact mapping of fentanyl, propofol, EMLA, and L.M.X4 to the listed treatment arms is also not reported. Period effects, carryover considerations, and the statistical model for repeated observations are not described.
The endpoint combines three scales into a binary outcome at a score greater than 0. The record says they were interchangeable for comparison purposes, but provides no information about how scale choice or differences in developmental ability were handled in analysis. The full scale-score distributions are not reported.
Multiplicity procedures, missing-data methods, sensitivity analyses, and the trial’s original sample-size justification are not reported in the registry record. The status is terminated, but the reason is not reported. Safety information gives zero serious and other adverse events in the listed groups, while death counts are not reported; event-collection details and comparative safety estimates are absent.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Fentanyl-Propofol-EMLA of L.M.X4 Technique for Bone Marrow Aspiration — NCT00187135.
- Registry record last updated 2017-04-04; status last verified 2010-03.
- Results first posted 2010-03-19.
- 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
NCT00187135 is a Phase III randomized, quadruple-masked crossover study sponsored by St. Jude Children's Research Hospital. The registry lists enrollment of 168, while the primary pain analysis description refers to a subset of 162 randomized participants. The study began in 2002-03, primary completion is listed as 2008-08, and status is terminated.
The primary outcome is pain, coded yes/no from pediatric pain-scale scores during sedation recovery, for a maximum of 65 min. Participants were eligible for a comparison if they completed at least Visit 2 and received each of the two treatments being compared. Treatment-specific pain results and the analysis model are not reported in the registry record.
The listed groups are fentanyl 0.5mcg/kg, fentanyl 1mcg/kg, and placebo. Serious and other adverse event counts are zero in each listed group; death counts are not reported. The reason for termination, missing-data methods, multiplicity procedures, and the original sample-size justification are not reported in the registry record.