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 T-20 Plus a Selected Anti-HIV Treatment in HIV-Infected Children and Adolescents (NCT00022763), a non-randomized phase 2 study. The primary outcome is Area Under the Plasma Concentration Time Curve (AUC) From 0-12 Hours for Enfuvirtide and Its Metabolite (Ro 50-6343).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00022763 |
| Phase | Phase 2 |
| Condition | HIV Infections |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 52 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
Population
Children and adolescents with HIV infection enrolled in a study of enfuvirtide. The intensive pharmacokinetic analysis population included the first 12 participants enrolled per age group and all children aged 3 to 6 years who underwent intensive sampling at week 1.
Intervention
Enfuvirtide, also called T-20 in the study title, in combination with an optimized background treatment. The registry lists enfuvirtide as the drug intervention.
Comparator
No concurrent comparator is listed. The study is described as non-randomized and single-group, so it does not provide a randomized comparison between treatment groups.
Primary question
What was the area under the plasma concentration-time curve from 0 to 12 hours for enfuvirtide and its metabolite, measured during week 1?
The study’s main question was pharmacokinetic: it aimed to characterize the concentration of enfuvirtide and its metabolite in plasma over time after dosing. The primary endpoint was the area under the concentration-time curve from 0 to 12 hours. This is a measure of drug exposure across the specified sampling interval, rather than a measure of clinical benefit or disease control.
The registry describes the study as a pharmacokinetic and safety study in children and adolescents with HIV infection. It also lists safety outcomes, including adverse events, serious adverse events, laboratory abnormalities, deaths, and withdrawals attributed to adverse events. These outcomes address tolerability and observed safety events, but the registry record does not provide numerical results for the primary pharmacokinetic endpoint.
Because the study had no randomized comparator, its results cannot estimate a treatment contrast against an untreated or alternative-treatment group. The registry record also does not report a clinical efficacy endpoint. The question that can be addressed from the posted record is therefore limited to the study’s planned pharmacokinetic and safety assessments, and to the adverse-event counts and terms that are reported.
3. Trial Design
The registry identifies this as a Phase 2 study, while its official title describes a Phase I/II pharmacokinetic and safety study. It was non-randomized, unmasked, and single-group, with an enrollment of 52. The intervention was enfuvirtide in combination with an optimized background, as described in the official title. No separate control group is listed.
The study concerned children and adolescents with HIV infection. The primary endpoint was based on intensive pharmacokinetic sampling during week 1, at pre-dose (time 0) and 2, 4, 8, and 12 hours post-dose. The intensive pharmacokinetic analysis population was defined as the first 12 participants enrolled per age group and all children aged 3 to 6 years who underwent intensive pharmacokinetic sampling at week 1. This is a specified analysis population, not necessarily the same as every participant enrolled.
The registry lists Hoffmann-La Roche as the sponsor. The recorded study start is 2001-08, primary completion is 2004-12, and status is completed. These dates describe the registry record; they do not establish when each participant entered or completed follow-up.
A single-group design can describe measurements and events observed among participants receiving the study intervention, but it does not separate treatment effects from other influences using a randomized control. The record does not report the full treatment schedule, the composition of the optimized background, eligibility criteria, follow-up duration, or a detailed statistical analysis plan. Those design details are not reported in the registry record and should not be inferred from the study title or outcome list.
The registry includes pharmacokinetic outcomes and several safety outcomes. It lists ten outcome measures and no analysis records. Numerical values for the primary endpoint are not reported in the registry record. The available safety counts and serious-event terms are descriptive and do not constitute a randomized comparison.
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 | Stratum A | Stratum B |
|---|---|---|
| STARTED | 24 | 28 |
| COMPLETED | 15 | 11 |
| NOT COMPLETED | 9 | 17 |
Reasons for not completing this period:
| Reason | Stratum A | Stratum B |
|---|---|---|
| Adverse Event | 1 | 2 |
| Injection Site Reactions | 0 | 2 |
| Death | 0 | 1 |
| Lost to Follow-up | 0 | 3 |
| Withdrawal by Subject | 4 | 7 |
| Insufficient Therapy | 1 | 0 |
| Admin | 3 | 2 |
Analysis population for the primary outcome, as defined in the registry: Intensive PK Analysis Population: The first 12 participants enrolled per age group and all children aged 3 to 6 years who underwent intensive pharmacokinetic sampling at week 1 were included within the intensive PK analysis population.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Stratum A | Stratum B | Total |
|---|---|---|---|---|
| Participants analysed | 24 | 28 | 52 | |
| Age, Continuous | years | 8.7 (SD 1.8) | 13.9 (SD 1.6) | 11.5 (SD 3.1) |
| Sex: Female, Male: Female | Participants | 15 | 9 | 24 |
| Sex: Female, Male: Male | Participants | 9 | 19 | 28 |
6. Primary Endpoint
The registry lists the primary outcome as Area Under the Plasma Concentration Time Curve (AUC) From 0-12 Hours for Enfuvirtide and Its Metabolite (Ro 50-6343), measured over Pre-dose (time 0), and 2, 4, 8, and 12 hours post-dose (Week 1).
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | microgram hour per milliliter (mcg.h/mL) |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the area under the plasma concentration-time curve from 0 to 12 hours for enfuvirtide and its metabolite, Ro 50-6343. The stated unit is microgram hour per milliliter (mcg.h/mL). Measurements were scheduled at pre-dose (time 0), and 2, 4, 8, and 12 hours post-dose during week 1. The registry describes the endpoint as a mean with standard deviation and says the AUC was calculated from plasma concentration-time data on Day 7 using standard non-compartmental pharmacokinetic methods.
In general, an area under a concentration-time curve summarizes exposure across a defined period by combining concentration measurements over time. Pharmacokinetic researchers use it to characterize drug absorption and exposure; it is not itself a direct measure of clinical benefit. The metabolite was assessed alongside enfuvirtide, allowing the study to characterize both compounds’ concentration-time profiles.
The registry record does not report the endpoint’s mean, standard deviation, sample size contributing to the endpoint, or a numerical estimate for either compound. It therefore does not permit a description of the observed exposure level or its precision. The defined intensive pharmacokinetic population also matters: its measurements pertain to participants selected for intensive sampling, not automatically to every participant enrolled.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a pharmacokinetic study with a single-group design, and it identifies the area under the plasma concentration-time curve from 0 to 12 hours as the primary endpoint. However, it does not report the numerical primary-endpoint results. Without a mean, standard deviation, or endpoint-specific sample size, the magnitude and variability of the observed drug exposure cannot be interpreted from the registry record.
The record does include descriptive safety information. For the enfuvirtide group, 31 of 52 participants were recorded as affected by serious adverse events, and 52 of 52 were recorded in the other adverse-event category. These are participant counts within the registry’s safety reporting fields. They do not indicate how many events occurred, when events occurred, how long participants were observed, or whether investigators judged events to be related to treatment. Those details are not reported in the registry record.
The serious-event terms listed with percentages include Pneumonia nos at 7.7%, Cellulitis at 5.8%, Gastroenteritis nos at 3.8%, Pyrexia at 3.8%, Bronchiolitis at 1.9%, and Bronchitis nos at 1.9%. The record does not give further detail about event timing, severity for each term, recurrence, or attribution. These percentages describe the posted terms; they should not be interpreted as comparative risks against another treatment.
The registry also lists outcomes for treatment-emergent Grade 3 or Grade 4 laboratory abnormalities, deaths, and premature withdrawal due to adverse events, but numerical results for these outcomes are not reported in the registry record. Deaths are recorded as not reported in the safety summary. The absence of a numerical result in the record is not evidence that no such events occurred.
There is no randomized comparator, and the record lists no analyses. Accordingly, the reported safety counts are descriptive, not estimates of a causal treatment effect. The record does not provide the information needed to assess whether the observed counts differ from those expected in a comparable group, or to account for background illness, concomitant treatment, or follow-up time.
The sample-size assumptions describe a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions do not match the registry’s single-group design and do not establish the trial’s actual planning rationale. Such illustrations are planning calculations, not observed power. No sponsor analysis is available in the registry record to compare with a recalculation.
Overall, the record establishes the study’s design, planned pharmacokinetic measurements, analysis population definition, and selected safety counts. It does not establish a numerical primary pharmacokinetic result, a comparative treatment effect, or a complete safety profile. Interpretation should remain limited to those descriptive details that are actually reported.
9. Statistical Methods Explained
The registry states that the primary endpoint was summarized as a mean with standard deviation. These are descriptive statistics for a continuous measurement. In general, the mean describes the central level of a set of measurements, while the standard deviation describes how dispersed individual measurements are around that mean. Neither statistic is reported numerically for the primary endpoint in the registry record, so the endpoint’s observed level and variability cannot be assessed.
What does the AUC measure?
In general, an area under a concentration-time curve combines concentration measurements across a specified time interval. Here, the defined interval was 0 to 12 hours, with sampling at pre-dose (time 0), and 2, 4, 8, and 12 hours post-dose during week 1. The registry says the calculation used plasma concentration-time data on Day 7 and standard non-compartmental pharmacokinetic methods. AUC is used to characterize drug absorption and exposure. It should not be confused with a clinical outcome or with a direct measure of how a participant feels or functions.
The endpoint covers both enfuvirtide and its metabolite, Ro 50-6343. The registry does not report the separate numerical AUC results for either compound. It also does not state how many participants contributed usable concentration data to each calculation, or how missing samples were handled. Those details are not reported in the registry record.
Why is the analysis population important?
The registry defines an intensive pharmacokinetic analysis population. It includes the first 12 participants enrolled per age group and all children aged 3 to 6 years who underwent intensive pharmacokinetic sampling at week 1. This definition identifies participants relevant to the detailed sampling analysis. It should not be treated as interchangeable with the full enrollment of 52. The record does not report the number in each age group, the total number with complete sampling, or whether all eligible participants contributed to each endpoint estimate.
In general, a population definition helps readers understand which participants contribute to an analysis and how closely that group matches the enrolled population. If participants with incomplete or unavailable measurements are excluded, the resulting summary may describe only those with analyzable data. The registry does not report exclusions or missing-data procedures, so the extent of any such influence cannot be determined.
What can a single-group design establish?
The study was non-randomized, unmasked, and single-group. In general, a single-group pharmacokinetic study can characterize measured concentrations among participants who receive the intervention. It does not create a concurrent randomized comparison that can distinguish the intervention’s effect from other causes of observed differences. Unmasked status means masking was absent according to the registry; the record does not describe any additional steps used to limit measurement or assessment bias.
The intervention is described as enfuvirtide in combination with an optimized background. The registry does not report the specific components of that background or their dosing. Consequently, any description of the treatment context must remain at the level given in the record, and the observed findings cannot be attributed to enfuvirtide alone on the basis of the listed design.
How should the planning illustration be read?
The sample-size assumptions describe an illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. In general, planning calculations estimate how many participants might be needed under stated assumptions to detect a selected effect with a chosen design. They are not measurements of what happened in the trial and are not observed power.
Those assumptions describe a hypothetical two-arm setting, whereas this registry record describes a single-group study. The illustration therefore should not be read as the study’s actual allocation or as evidence that the trial was designed around a two-arm comparison. The registry record does not report the original sample-size justification or the assumptions used by the sponsor when planning the study. No discrepancy between a sponsor analysis and a recalculated analysis is reported.
What do the analysis records say?
The registry lists no analysis records. This means the record does not provide statistical analysis entries or numerical primary endpoint results that can be evaluated in this account. It does not show whether analyses were conducted outside the registry record. The endpoint description alone is not enough to reconstruct an analysis or its results.
In general, estimates are more informative when reported with a measure of uncertainty, such as a confidence interval. A confidence interval describes the range of values compatible with the data and analysis under the method’s assumptions; it is not a guarantee that the true value lies in that range. No primary-endpoint confidence interval is reported in the registry record. Since the endpoint estimate itself is also not reported, its precision cannot be evaluated.
Pharmacokinetic measurements may show variation between participants and across sampling times. General statistical methods can account for the form of such variation, but the registry does not describe a model for repeated measurements, transformation of concentrations, handling of values below assay limits, or adjustment for participant characteristics. These methodological details are not reported in the registry record and should not be inferred.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary pharmacokinetic outcome and several secondary outcomes, including additional concentration measures, adverse events, laboratory abnormalities, deaths, and withdrawals due to adverse events. When a study examines multiple outcomes, there is a general statistical concern that interpreting many tests can increase the chance of apparently notable findings arising by chance. The registry record does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or a prespecified testing strategy. It is therefore not possible to determine how multiple outcomes were handled analytically.
No analysis records are listed, and numerical primary endpoint results are not reported in the registry record. The record also does not describe interim analyses or stopping rules. These aspects of the statistical plan are not reported in the registry record.
The intensive pharmacokinetic analysis population is defined as the first 12 participants enrolled per age group and all children aged 3 to 6 years who underwent intensive sampling at week 1. The registry does not give the number analyzed for each compound, the number with complete concentration-time data, or the handling of missing measurements. It also does not report whether participants were excluded from any safety outcome analyses or how incomplete follow-up was treated.
In general, missing-data procedures matter because the reasons data are absent can affect how well an analysis represents the intended population. No imputation method, sensitivity analysis, or missingness assumption is reported in the registry record. The posted information does not establish whether measurements were missing or how any missingness may have affected the results.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Enfuvirtide | 31 | 52 | 59.6% |
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 |
|---|---|
| Enfuvirtide | 52 of 52 |
Most frequent serious adverse event terms
| Event term | Organ system | Enfuvirtide |
|---|---|---|
| Pneumonia nos | Infections and infestations | 4 of 52 |
| Cellulitis | Infections and infestations | 3 of 52 |
| Gastroenteritis nos | Infections and infestations | 2 of 52 |
| Pyrexia | General disorders | 2 of 52 |
| Bronchiolitis | Infections and infestations | 1 of 52 |
| Bronchitis nos | Infections and infestations | 1 of 52 |
| Colitis pseudomembranous | Infections and infestations | 1 of 52 |
| Gastroenteritis viral nos | Infections and infestations | 1 of 52 |
| Kawasaki's disease | Infections and infestations | 1 of 52 |
| Lobar pneumonia nos | Infections and infestations | 1 of 52 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Enfuvirtide |
|---|---|---|
| Upper respiratory tract infection nos | Infections and infestations | 22 of 52 |
| Cough | Respiratory, thoracic and mediastinal disorders | 13 of 52 |
| Otitis media nos | Infections and infestations | 12 of 52 |
| Nausea | Gastrointestinal disorders | 11 of 52 |
| Vomiting nos | Gastrointestinal disorders | 11 of 52 |
| Pyrexia | General disorders | 11 of 52 |
| Dermatitis Nos | Skin and subcutaneous tissue disorders | 10 of 52 |
| Nasopharyngitis | Infections and infestations | 9 of 52 |
| Diarrhoea nos | Gastrointestinal disorders | 9 of 52 |
| Sinusitis nos | Infections and infestations | 8 of 52 |
Serious events with the highest arm-level rate: pneumonia nos (7.7%), cellulitis (5.8%), gastroenteritis nos (3.8%), pyrexia (3.8%), bronchiolitis (1.9%).
For the enfuvirtide group, the registry records 31 participants affected by serious adverse events among 52 at risk. It records 52 participants affected in the other adverse-event category among 52 at risk. These are counts of participants, not counts of individual events; one participant could potentially have experienced more than one event, but the registry record does not describe event recurrence or counting rules.
The listed serious-event terms and percentages are Pneumonia nos at 7.7%, Cellulitis at 5.8%, Gastroenteritis nos at 3.8%, Pyrexia at 3.8%, Bronchiolitis at 1.9%, and Bronchitis nos at 1.9%. The registry does not report further information about the timing, duration, severity by term, or attribution of these events. The percentages should be read as the posted descriptive values, not as comparisons with another group.
The record lists deaths as a safety outcome but does not report a numerical death result. It also does not report numerical results for treatment-emergent Grade 3 or Grade 4 laboratory abnormalities or premature withdrawal due to adverse events. Unreported results should not be interpreted as zero events. Since the study had no concurrent randomized comparator, these safety counts cannot show whether event frequency was higher or lower than under another treatment.
12. What the Result Does, and Does Not, Mean
The record documents a completed, single-group study of enfuvirtide in children and adolescents with HIV infection. It specifies a primary pharmacokinetic measure, the time points for intensive sampling, and the population defined for that analysis. It also provides selected safety counts and serious-event terms. These details describe what the study set out to assess and what safety information appears in the registry record.
The primary endpoint’s numerical results are not reported in the registry record. Therefore, the record does not show the mean AUC, its standard deviation, or a confidence interval for enfuvirtide or its metabolite. Without those statistics, the magnitude and precision of the measured exposure cannot be evaluated. The endpoint is a pharmacokinetic measure, not a clinical efficacy outcome.
The safety figures describe participants recorded as affected, but they do not establish that the intervention caused the events. A single-group study lacks a randomized control for separating effects of treatment from background illness, concomitant treatment, or other influences. The record also does not provide event timing, exposure duration, or detailed attribution information needed for a fuller safety interpretation.
Some listed safety outcomes have no numerical results in the registry record, including deaths, treatment-emergent Grade 3 or Grade 4 laboratory abnormalities, and premature withdrawal due to adverse events. Their absence from the numerical summary does not mean that none occurred. The record does not provide enough information to characterize these outcomes.
The study’s design and reporting can support a description of its planned pharmacokinetic and safety assessments, alongside the adverse-event counts that are posted. It cannot support a randomized comparison, a causal estimate of treatment effect, or a conclusion about clinical benefit. The numerical primary result and several details needed to understand the analysis are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for the distinction between pharmacokinetic characterization and comparative clinical evaluation. Its primary endpoint was a concentration-time summary, designed to characterize exposure to enfuvirtide and its metabolite over a defined interval. That type of endpoint can answer a focused pharmacokinetic question, but it does not by itself establish a clinical treatment effect.
The study’s single-group, non-randomized design also defines what its estimates can mean. Measurements among participants receiving the intervention can be described, but there is no randomized control group to provide a like-for-like contrast. This makes it especially important to distinguish descriptive observation from causal comparison when reading the safety information.
The registry’s defined intensive pharmacokinetic analysis population is another important statistical feature. Detailed sampling was specified for a selected group, including the first 12 participants enrolled per age group and all children aged 3 to 6 years who underwent intensive sampling at week 1. That population definition helps delimit the intended analysis, but the record does not report how many participants contributed results or whether data were complete.
Finally, the difference between an endpoint being defined and its result being reported matters. The registry gives a measurement schedule and calculation method for the primary AUC endpoint, but no numerical estimate or uncertainty measure. This prevents readers from assessing the actual exposure values or their precision. The study therefore illustrates why interpretation depends on both design and complete numerical reporting, not merely on the presence of a clearly described endpoint.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation for statistical interpretation is that numerical results for the primary pharmacokinetic endpoint are not reported in the registry record. The record provides the endpoint definition, unit, sampling schedule, and calculation approach, but not the mean, standard deviation, number analyzed, or a measure of uncertainty. As a result, the primary result cannot be quantitatively interpreted.
The study was non-randomized and single-group, with no concurrent comparator listed. Consequently, the posted safety counts cannot be used to estimate a comparative risk or to distinguish treatment-associated events from other causes. The registry also identifies the study as unmasked, and does not report procedures for limiting assessment bias.
There is limited detail about the participants who contributed to the pharmacokinetic endpoint. The intensive analysis population is defined, but the record does not report its size, group composition by age, completeness of sampling, or handling of missing data. The full eligibility criteria, follow-up duration, dose schedule, and components of the optimized background are also not reported in the registry record.
Safety reporting is incomplete for several listed outcomes. Numerical results for deaths, treatment-emergent Grade 3 or Grade 4 laboratory abnormalities, and premature withdrawal due to adverse events are not reported in the registry record. The serious adverse-event counts and terms also lack details about event timing, duration, recurrence, and attribution. No comparative or adjusted safety analysis is listed.
The registry lists no analysis records and does not report a statistical analysis plan, multiplicity procedures, interim analyses, or missing-data methods. The planning illustration uses assumptions for equal allocation between two arms, which do not describe the recorded single-group design. The original sample-size justification is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: T-20 Plus a Selected Anti-HIV Treatment in HIV-Infected Children and Adolescents — NCT00022763.
- Registry record last updated 2016-02-15; status last verified 2016-01.
- Results first posted 2016-02-15.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record describes a completed Phase 2 study, whose official title calls it a Phase I/II pharmacokinetic and safety study, of enfuvirtide with an optimized background in children and adolescents with HIV infection. It was non-randomized, unmasked, and single-group, with enrollment of 52. Hoffmann-La Roche is listed as sponsor.
The primary endpoint was the 0-to-12-hour AUC for enfuvirtide and its metabolite, assessed during week 1 using intensive pharmacokinetic sampling. The endpoint’s numerical results are not reported in the registry record. For safety, 31 of 52 participants were recorded as affected by serious adverse events, and 52 of 52 were recorded in the other adverse-event category. Numerical results for several other listed safety outcomes are not reported.
The record supports a descriptive account of the design, endpoint, analysis population definition, and posted safety information. It does not provide a randomized comparison or enough numerical primary-endpoint information to assess exposure magnitude and precision.