Safety And Efficacy Of Rescriptor In Patients For Human Immunodeficiency Virus (HIV) Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Safety And Efficacy Of Rescriptor In Patients For Human Immunodeficiency Virus (HIV) Patients (NCT00823966), a registered phase not stated study. The primary outcome is Number of Participants Who Reported Unlisted Adverse Drug Reaction..

2. Clinical Question

The registry record describes a drug-use investigation intended to collect safety and efficacy information about Rescriptor, or delavirdine mesilate, in people with HIV-1 infection. Its primary listed endpoint concerns participants reporting an adverse drug reaction that was not listed in the Japanese Package Insert, also described as the Local Product Document. The stated timeframe for that endpoint is one year.

The record’s objective also refers to collecting information on the incidence of adverse drug reactions and factors considered to affect safety or efficacy. Those objectives do not, by themselves, establish that comparative efficacy questions were answered. The registry does not report a numerical primary-endpoint result in the information available for this analysis. It also does not report details of the second outcome measure. Consequently, the clinical question can be described from the stated objective and endpoint, but the endpoint’s observed result and any efficacy findings cannot be characterized from the registry record.

3. Trial Design

This record is for a drug-use investigation and regulatory post-marketing commitment concerning Rescriptor. The listed condition is HIV-1 infection, and the intervention is delavirdine mesilate. The registry records enrollment of 3 participants and labels the study status completed. The recorded start is 2003-12, and primary completion is 2009-03.

The record does not describe a randomized comparison. Allocation is not stated, and the listed arm is not a like-for-like control comparison. The arm description says that patients were administered delavirdine mesilate. No comparator arm or allocation procedure is reported in the registry record. Therefore, the design information does not support treating the reported group as one side of a randomized treatment contrast.

The stated surveillance objective was to collect information about unlisted adverse drug reactions, the incidence of adverse drug reactions, and factors considered to affect safety and/or efficacy. The primary endpoint is a participant count for unlisted adverse drug reactions, with a timeframe of one year. The safety analysis population is described as enrolled subjects who had received at least 1 confirmed administration of delavirdine mesilate. The registry record does not report the recruitment process, eligibility criteria beyond the stated population information, follow-up procedures, or how adverse reactions were ascertained.

Two outcome measures are listed, but the record details only the primary endpoint in the registry record for interpretation. No statistical analysis records are listed. Information about the planned statistical methods, sample-size justification, interim analyses, or handling of incomplete follow-up is not reported in the registry record. The recorded enrollment is therefore a description of the study population, not evidence of a prespecified statistical target or a basis for judging the study’s intended precision.

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

Analysis population for the primary outcome, as defined in the registry: The safety analysis population included enrolled subjects who had received at least 1 confirmed, administration of delavirdine mesylate.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants Who Reported Unlisted Adverse Drug Reaction., measured over One Year.

The primary endpoint is the number of participants who reported an unlisted adverse drug reaction. The registry defines the reaction as one not listed in the Japanese Package Insert, also called the Local Product Document, and gives a timeframe of one year. The endpoint is expressed as a participant count rather than as a continuous measurement. In general, counting participants with an event can describe how often the event was reported in the defined safety population, provided the population and observation period are clear.

The endpoint is relevant to the stated surveillance objective because it focuses on reactions outside the listed product information. Monitoring for such reports is one way a post-marketing investigation can collect safety information in clinical use. A count alone, however, does not describe the nature, severity, timing, or certainty of each suspected reaction. Those details are not reported for the primary endpoint in the registry record.

The safety analysis population included enrolled subjects who had received at least 1 confirmed administration of delavirdine mesilate. The record states a one-year timeframe, but does not report further details about the observation window, completeness of follow-up, or rules for attributing an event to treatment. Most importantly, a numerical result for this primary endpoint is not reported in the registry record. Without that result, the endpoint’s observed frequency and its precision cannot be assessed from the available information.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies the primary outcome as a participant count: the number of participants reporting an unlisted adverse drug reaction. The outcome is binary at the participant level in the sense that each participant is classified according to whether the specified event was reported. A count of participants with an event can be useful for describing safety observations, but interpretation depends on a clear denominator, a defined observation period, and consistent event ascertainment. The record defines the safety analysis population and a one-year timeframe, while further details about ascertainment and follow-up are not reported in the registry record.

What can a count show?

A count communicates the number of people meeting an outcome definition. In general, a count is not interchangeable with the number of events: one participant may have more than one event, while a participant-level endpoint counts that participant once if the event definition is met. The primary endpoint is explicitly expressed in participants, so it concerns participants reporting an unlisted reaction rather than a total count of every reaction episode. The registry does not report the primary count, so no event frequency can be described from that endpoint.

For a participant-level outcome, a proportion can describe the observed share of a defined population with the event. That proportion is most informative when its numerator and denominator are available and the population has a consistent observation opportunity. The safety population is defined as enrolled subjects who had received at least 1 confirmed administration of delavirdine mesilate. However, the primary endpoint’s numerator is not reported, and no uncertainty interval is reported. It is therefore not possible to assess the observed primary-outcome proportion or its precision.

Why does precision matter?

In general, estimates from small groups are often imprecise because each observation can have a substantial influence on the estimate. An uncertainty interval conveys a range of values compatible with the data under a specified statistical method. It does not remove limitations in the data or guarantee that the interval captures every source of uncertainty. Since the primary endpoint estimate and interval are not reported, the precision of that outcome cannot be evaluated. The enrollment of 3 participants describes the recorded study size, but does not establish how precisely the primary endpoint was estimated.

The registry contains no statistical analysis records. The specific statistical plan, including any adjustment, model, or inferential procedure for the primary outcome, is not reported in the registry record. No p-value or confidence interval is reported for the primary endpoint. In general, a p-value addresses compatibility of data with a specified null model; it is not a measure of clinical importance or a substitute for an effect estimate. With no posted primary analysis, there is no basis for interpreting a significance test.

How should planning illustrations be understood?

The stated assumptions for planning illustrations describe two-sided significance level 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. The record describes these as planning illustrations computed from the observed effect, not as the study’s own design. Such illustrations are hypothetical planning calculations, not evidence about the probability that the completed study detected an effect. They should not be described as observed power. No specific planning values are available in the registry entries used for this interpretation.

In general, sample-size planning depends on assumptions about the outcome, effect size, variability, allocation, and the analysis method. Changing an assumption can change the resulting planning estimate. A calculation based on a fraction of an observed effect is especially dependent on the effect used as its starting point; it does not create new evidence or correct limitations in the observed data. The record does not report a protocol sample-size justification, so the planning illustration should not be treated as the original enrollment target or as proof that the study had adequate precision.

Why does over-dispersion matter?

Over-dispersion is a general issue for count data when observed variation exceeds what a chosen statistical model assumes. If present, it can make standard errors too small and uncertainty intervals too narrow unless the analysis accounts for it. The primary endpoint is a participant count, not a reported repeated-event rate, and the registry does not report a model or analysis that can be assessed for over-dispersion. This concept is therefore a general statistical consideration, not a finding about this investigation.

For safety surveillance, descriptive counts and event terms can support a transparent account of what was recorded. They do not by themselves establish causation, quantify comparative risk, or resolve whether an event was expected or unexpected under every relevant clinical circumstance. The registry’s description of an unlisted reaction refers to its status relative to the Japanese Package Insert. Details about event adjudication, causal assessment, missing observations, and coding are not reported in the registry record. These omissions constrain how far statistical interpretation can go beyond the recorded counts and labels.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Delavirdine Mesilate")
n      <- c(3)
events <- c(0)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Delavirdine Mesilate  3  0  0.0  0.0  70.8  0.0  56.1  0.6  60.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures, but only the primary endpoint is described in the information available for interpretation. Details of the other outcome measure are not reported in the registry record. No statistical analysis records are listed. The record does not report a multiplicity strategy, such as whether or how analyses across outcomes were prioritized or adjusted. In general, multiplicity refers to the increased chance of finding an apparently noteworthy result when many outcomes or comparisons are examined. Without the analysis plan and results, it is not possible to determine whether multiplicity was relevant to any reported inference.

Information about missing data is not reported in the registry record. The registry does not describe whether all enrolled participants completed the one-year observation period, whether any primary-outcome status was unavailable, or how incomplete follow-up was handled. These details matter because missing outcome information can affect both the numerator and denominator used to describe an event frequency. The safety analysis population is defined by receipt of at least 1 confirmed administration, but that definition does not explain how missing outcome observations were treated.

The analysis population definition is stated for safety: enrolled subjects who had received at least 1 confirmed administration of delavirdine mesilate. The record does not report a separate efficacy analysis population or additional analysis-population rules. The primary endpoint result and its analysis are not reported, so it is not possible to verify how many participants contributed to that endpoint or whether the analysis followed the stated population definition.

In general, a prespecified analysis plan helps readers distinguish planned analyses from exploratory ones and understand how incomplete data and multiple outcomes were handled. A protocol or analysis plan is not described in the registry record. No interim-analysis information is reported in the registry record. These unknowns should be treated as limitations of the available documentation, not as evidence that a particular method was or was not used.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: pneumocystis jiroveci pneumonia (33.3%), hypoxia (33.3%).

The serious-event figures list 3 participants at risk and 2 affected in the delavirdine mesilate group. The registry also lists Pneumocystis jiroveci pneumonia and hypoxia among the top serious-event terms, each at 33.3%. These are descriptive entries. The registry information does not establish that the listed terms represent separate participants, nor does it provide event-level details that would allow a reader to reconstruct the counts by term.

A recorded serious event is not automatically an event caused by the study drug. In general, adverse-event reporting records events occurring during observation, while causal attribution requires additional assessment. The registry record does not report the causal assessment for these terms, their timing, clinical details, or the basis for classifying each event as serious. The percentages should therefore be read as the registry’s reported figures, not as comparative risk estimates or causal effects.

Deaths are not reported in the registry record. This is different from interpreting an unreported value as a confirmed count of zero. The information also does not give a comparator group suitable for assessing whether serious events were more or less frequent under another treatment. The figures describe the recorded safety information; they do not establish a treatment effect or provide a complete account of the participants’ clinical courses.

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

The record documents a completed post-marketing investigation of Rescriptor, identified as delavirdine mesilate, with enrollment of 3 participants with HIV-1 infection. It identifies a safety objective focused on adverse drug reactions not listed in the Japanese Package Insert and gives a one-year timeframe for the primary endpoint. It also provides selected serious-event counts and terms. These are the main descriptive contributions that can be drawn from the information in the registry record.

The record does not report a numerical result for the primary endpoint. It therefore does not show how many participants reported an unlisted adverse drug reaction, or how precise an estimate of that outcome might be. The serious-event entries do not substitute for the missing primary outcome: they concern a separate set of safety information and do not establish whether an event met the primary endpoint definition.

The investigation is not described as randomized, allocation is not stated, and no like-for-like control comparison is reported. Consequently, the record cannot support an estimate of a comparative treatment effect. The stated objective mentions safety, efficacy, and factors considered to affect both, but efficacy results and related analyses are not reported in the available registry information. No statistical analysis records are listed.

In general, post-marketing surveillance can contribute descriptive information about reports arising during drug use. Such information may help characterize what was recorded, but its interpretation depends on observation, reporting, definitions, and analytic methods. Here, details about event ascertainment, follow-up completeness, and causal assessment are not reported in the registry record. The small recorded enrollment also limits how much can be inferred about the range of outcomes in a broader population.

The appropriate reading is narrow: the registry describes the purpose, population definition, and selected safety figures, while leaving the primary endpoint result and several analytic details unreported. It does not establish that the drug caused the listed serious events, does not quantify the primary endpoint, and does not provide a controlled efficacy comparison. These limits distinguish what the posted information records from conclusions it cannot support.

14. Why This Trial Matters Statistically

This investigation matters statistically because it illustrates the difference between a stated surveillance objective and an interpretable result. The registry names a primary safety endpoint, defines it as participants reporting an unlisted adverse drug reaction, and gives a one-year timeframe. Yet a numerical result for that endpoint is not reported in the registry record. Without the result, readers cannot estimate the endpoint’s frequency or examine its precision.

The recorded enrollment of 3 participants is also important context. In general, a small study can describe observations, but estimates based on few participants are vulnerable to substantial uncertainty. A numerical estimate accompanied by an uncertainty interval would help readers understand precision; neither is reported for the primary endpoint. The enrollment count should not be mistaken for a statistical finding or proof of adequate information for every surveillance objective.

The study is not a randomized comparison, and no like-for-like control is described. That distinction matters because a descriptive safety count and a comparative treatment effect answer different questions. Without a suitable comparison and analysis, the observed safety entries cannot establish whether the drug changed the probability of an event. Nor can the broad objective to consider efficacy factors be treated as evidence of an efficacy result.

The registry’s adverse-event entries make the same point in another way: counts and listed terms are useful records of what was reported, but causal attribution and context require more information. The record does not report event-level details or causal assessments for the serious-event terms. It also does not list statistical analyses or describe missing-data handling. These absences limit reproducibility and make it difficult to evaluate how the recorded information maps onto the stated objectives.

The statistical lesson is not that descriptive surveillance is uninformative. Rather, its claims must match its design and reporting. Clear endpoint results, denominators, uncertainty measures, and methods would be needed to support stronger quantitative interpretation. The available registry entries permit a cautious description of the investigation and selected safety entries, but not a complete assessment of its primary endpoint or a comparative conclusion.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The principal limitation is that a numerical result for the primary endpoint is not reported in the registry record. Although the endpoint definition, population, and one-year timeframe are stated, the number of participants meeting the endpoint and any corresponding uncertainty measure are unavailable. The endpoint’s observed frequency and precision therefore cannot be assessed.

Enrollment was 3 participants. This is the recorded study size, but the registry does not report a protocol-based sample-size justification or a target enrollment rationale. The planning assumptions described for illustrations do not establish the original design rationale. No statistical analysis records are listed, and details of any analysis method are not reported in the registry record.

The investigation is not described as randomized, allocation is not stated, and no like-for-like control comparison is identified. The serious-event counts and terms are therefore descriptive rather than comparative. The record does not report event-level timing, clinical context, causal assessment, or the relation between each listed term and individual participants. Deaths are not reported in the registry record, which should not be read as a confirmed zero.

Other important design and reporting details are not reported in the registry record: recruitment and eligibility procedures beyond the stated population information; how adverse reactions were ascertained; completeness of follow-up; handling of missing observations; and the analysis plan. Two outcome measures are listed, but the other outcome is not described in the available information. No interim-analysis information is reported.

The record’s stated objectives include collecting information about safety, efficacy, and factors considered to affect them. The available details do not include an efficacy result or a quantitative analysis of such factors. These limits mean the registry supports a narrow descriptive account, not a full evaluation of the investigation’s stated objectives or a causal conclusion about treatment.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Safety And Efficacy Of Rescriptor In Patients For Human Immunodeficiency Virus (HIV) Patients — NCT00823966.
• Registry record last updated 2010-07-05; status last verified 2010-06.
• Results first posted 2010-07-05.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

The registry record describes a completed drug-use investigation and regulatory post-marketing commitment concerning Rescriptor, or delavirdine mesilate, in people with HIV-1 infection. Enrollment was 3. The recorded start was 2003-12, and primary completion was 2009-03.

The primary endpoint counts participants reporting an adverse drug reaction not listed in the Japanese Package Insert, with a one-year timeframe. The safety analysis population included enrolled subjects who had received at least 1 confirmed administration. The primary endpoint’s numerical result is not reported in the registry record.

For the delavirdine mesilate group, the serious-event information lists 3 participants at risk and 2 affected. Pneumocystis jiroveci pneumonia and hypoxia are each listed at 33.3%. No statistical analysis records are listed. The investigation is not described as randomized, and no like-for-like control comparison is reported. These details support a limited descriptive reading of the record, not an estimate of comparative efficacy or a causal interpretation of the listed events.