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 Doravirine Concentrations and Antiviral Activity in Cerebrospinal Fluid in HIV-1 Infected Individuals (NCT04079452), a na phase 3 study. The primary outcome is Total Doravirine Concentrations in Cerebrospinal Fluid.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04079452 |
| Phase | Phase 3 |
| Condition | HIV-1-infection |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fundación FLS de Lucha Contra el Sida, las Enfermedades Infecciosas y la Promoción de la Salud y la Ciencia |
| Results posted | Yes |
2. Clinical Question
Population
People with HIV-1 infection receiving antiretroviral therapy. The registry describes a descriptive analysis for all participants, identified as 15 participants.
Intervention
Doravirine, 100 mg orally once daily, combined with tenofovir alafenamide and emtricitabine as Descovy® TAF/FTC, 25/200 mg orally once daily during 4 weeks.
Comparator
There is no separate comparator group. The record identifies a single-group study and does not describe a randomized allocation or a like-for-like control arm.
Primary question
What are the total doravirine concentrations in cerebrospinal fluid after 4 weeks of treatment? The primary outcome is specified as a median and interquartile range. Numerical concentration results are not reported in the registry record.
The study description also identifies interest in doravirine’s pharmacokinetic profile in cerebrospinal fluid and the maintenance of HIV suppression in that compartment. Those aims provide context for measuring drug concentrations in cerebrospinal fluid, but the primary outcome listed is concentration, not a direct measure of viral suppression. The registry record does not report a primary outcome value or a comparison of suppression between treatment groups.
This is therefore a descriptive question about drug exposure in cerebrospinal fluid during a specified treatment regimen. It is not a question about whether doravirine is better than another regimen: no comparator is listed, and the record does not support a comparative treatment-effect estimate.
3. Trial Design
This is a phase 3, completed, single-group study of doravirine with FTC/TAF in people with HIV-1 infection. The registry lists enrollment of 15 and no allocation scheme. Masking is listed as none. There is one described arm: doravirine 100 mg orally once daily with tenofovir alafenamide and emtricitabine coformulated as Descovy® TAF/FTC 25/200 mg, also taken orally once daily during 4 weeks.
The study began on 2020-02-18, reached primary completion on 2020-08-31, and is listed as completed. These dates describe the registry record’s study timeline; they do not establish when each participant was assessed. The outcome timeframe for the primary measure is 4 Weeks.
The primary outcome is total doravirine concentration in cerebrospinal fluid, expressed in ng/ml and summarized using a median and interquartile range. The registry describes a descriptive analysis for all participants (15). The record lists 8 outcome measures, but details of the other measures are not included in the registry record for this account.
Because this is a single-group study, there is no concurrent randomized control group for a like-for-like treatment comparison. The record does not report a sample-size justification, a statistical analysis plan, interim analyses, or methods for handling deviations from the treatment regimen. It also does not report how the study population was selected beyond the stated condition and treatment description. These absences limit what can be concluded about the design and analysis beyond the details explicitly 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 | Doravirie+TAF/FTC Arm (Single Arm) |
|---|---|
| STARTED | 15 |
| COMPLETED | 15 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Descriptive analysis was performed for all participants (15), defining median and IQR of Doravirine concentrations in CSF
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 | Doravirine + Descovy® TAF/FTC |
|---|---|---|
| Participants analysed | 15 | |
| Age, Continuous | years | 47 (27 to 65) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 13 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 15 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Spain | participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Total Doravirine Concentrations in Cerebrospinal Fluid, measured over 4 Weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | ng/ml |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is total doravirine concentration in cerebrospinal fluid, measured in ng/ml at 4 weeks. The registry specifies that the result is to be described by the median and interquartile range for all participants (15). These summaries describe the distribution of measured concentrations: the median represents its central value, while the interquartile range describes the spread of the middle portion of the observations. This is a general explanation of these statistical summaries.
Measuring a drug in cerebrospinal fluid addresses exposure in a compartment relevant to the study’s stated interest in the central nervous system. Concentration is a pharmacokinetic measure; it is not, by itself, a direct measure of antiviral activity or clinical benefit. The study description also mentions maintaining HIV suppression in cerebrospinal fluid, but the primary endpoint is concentration rather than a stated suppression outcome.
The registry record does not report the median, interquartile range, individual concentration values, or a numerical primary endpoint result. Consequently, the specified measure and summary method can be described, but the size and variability of the observed concentrations cannot be interpreted from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The design and the outcome definition identify what the study set out to describe: total doravirine concentration in cerebrospinal fluid after 4 weeks of doravirine with FTC/TAF. The primary outcome is intended to be summarized across all participants (15) by a median and interquartile range. However, the registry record does not report the numerical median or interquartile range. It therefore does not permit an interpretation of the observed concentration level, its variability, or the precision of its estimate.
The single-group design also sets a boundary on causal interpretation. Without a separate comparison group, the record cannot establish how concentrations under this regimen differ from concentrations under another regimen or from a control condition. It also cannot isolate the contribution of doravirine from that of the coadministered FTC/TAF regimen. These are design limits, not evidence that the regimen has or lacks a particular effect.
The study description mentions antiviral activity and maintenance of HIV suppression in cerebrospinal fluid as motivating concerns. Those statements describe the study’s context, not a reported result. The available primary outcome is drug concentration. No numerical result for viral suppression in cerebrospinal fluid is reported in the registry record, and the posted details do not specify a suppression endpoint or its analysis. Concentration measurements should not be treated as proof of suppression.
The record lists enrollment of 15 and says the descriptive analysis was performed for all participants (15). It does not report concentration values or a confidence interval. A confidence interval, in general, expresses the statistical uncertainty around an estimate under a specified method; it is not reported for this primary outcome in the registry record. Nor does the record provide enough information to assess the influence of missing measurements, unusual values, or variation in assessment timing.
The adverse-event figures are separate from the primary concentration outcome. The registry lists 0 serious adverse events among 15 at risk and 1 other adverse event among 15 at risk. These counts do not establish a comparative safety profile, because there is no control group. The record does not provide further event descriptions in the registry record.
Any planning illustration based on an observed effect should be read as a hypothetical design calculation, not as evidence about the precision or success of this study. The registry describes its illustration as using a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and specified fractions of the observed effect. Such assumptions do not turn this single-group study into a two-arm comparison, and planning calculations are not observed power. Numerical planning outputs and a reported observed effect are not included in the registry record.
9. Statistical Methods Explained
The primary outcome is a concentration, reported in ng/ml, and the registry specifies the median and interquartile range as its descriptive summaries. No inferential test or adjusted analysis for this endpoint is specified in the registry record. The distinction matters: a descriptive summary characterizes the observed measurements, while an inferential analysis would use an explicit statistical method to quantify uncertainty or test a defined hypothesis.
Why use a median and interquartile range?
As a general statistical explanation, the median is the middle of an ordered set of observations. The interquartile range describes the span from the lower quartile to the upper quartile, capturing the middle portion of the distribution. These summaries can be useful when measurements are skewed or include extreme observations, since they are less influenced by extremes than the mean and standard deviation. This is general guidance about the statistics, not a claim about the shape of this study’s concentration data. The registry record does not report those data or their distribution.
The specified descriptive approach does not, on its own, describe how precisely the population’s typical concentration is estimated. Precision could be conveyed with an appropriate uncertainty interval, but no interval for the primary endpoint is reported in the registry record. The available description also does not identify a model or test for comparing concentrations across groups or timepoints.
What does a single-group design allow?
In general, a single-group study can describe outcomes observed while participants receive a regimen. It does not provide a concurrent control group to estimate what would have happened under an alternative regimen. In this record, the allocation is listed as NA and the model as single group; the arm description combines doravirine with FTC/TAF. Thus, any observed concentration would describe exposure during that regimen, not a contrast attributable to doravirine alone. No treatment-effect comparison is specified.
Masking is listed as none. The registry record does not report whether outcome measurement was blinded, the laboratory procedures used for concentration measurement, or how measurement quality was assessed. Those details would be relevant to understanding potential measurement variability, but they cannot be inferred from the listed design fields.
How should planning calculations be understood?
In general, a sample-size calculation uses assumptions about a target effect, variability, statistical error rates, allocation, and an analysis method to illustrate the size of a future study needed for a chosen operating characteristic. The registry describes a planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it also describes planning for stated fractions of that effect. Those are planning assumptions, not the trial’s own design or a reported analysis of the single group.
A planning illustration is not observed power. It does not show the probability that this completed study detected an effect, and it cannot substitute for the unreported primary outcome values or their uncertainty. The registry record does not report the sample-size justification used to plan the study, and the registry entries available do not give numerical planning results. No additional sample-size output can be interpreted from the stated assumptions alone.
What is not specified?
The registry record does not report a detailed statistical analysis plan, a hypothesis test for the primary endpoint, adjustment for covariates, handling of repeated measurements, or a method for missing concentration data. It also does not report an interim analysis plan. These omissions mean that readers can identify the intended descriptive summaries but cannot reconstruct a fuller analysis protocol or determine whether additional procedures were used.
The primary outcome description names all participants (15) as the population for descriptive analysis. Beyond that statement, the record does not give a detailed analysis-population definition, such as rules for eligibility of measurements or treatment adherence. It also does not describe the distribution of concentration values. Therefore, explanations of robust summaries and uncertainty are general statistical context, not a claim about specific properties of the observed data.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 8 outcome measures, while the primary outcome is total doravirine concentration in cerebrospinal fluid at 4 weeks. Details of the other outcome measures are not reported in the registry record. The record does not report a multiplicity strategy, such as designating a hierarchy among multiple outcomes or adjusting statistical tests to account for multiple comparisons. Because no primary outcome result or inferential tests are reported, the consequences of multiplicity for statistical conclusions cannot be assessed.
The primary outcome description says the descriptive analysis was performed for all participants (15). That identifies the stated population for this summary, but does not explain how the analysis handled unavailable or unusable concentration measurements. Missing-data counts, reasons for missingness, imputation methods, and sensitivity analyses are not reported in the registry record. It is therefore not possible to judge whether missing measurements could affect the descriptive result.
The record does not provide a fuller definition of analysis populations, such as rules for inclusion based on treatment received or protocol adherence. The registry entries available do not specify whether measurements were repeated, how timing deviations were handled, or whether exclusions were made. These details are not reported in the registry record and should not be assumed.
The stated planning illustration concerns assumed two-arm designs and fractions of an observed effect. It does not describe multiplicity control or missing-data handling for this single-group study. The registry record does not report a statistical analysis plan that connects these planning assumptions to the primary outcome analysis.
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 |
|---|---|---|---|
| Doravirine+TAF/FTC Arm (Single Arm) | 0 | 15 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Doravirine+TAF/FTC Arm (Single Arm) | 0 of 15 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Doravirine+TAF/FTC Arm (Single Arm) | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Doravirine+TAF/FTC Arm (Single Arm) |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 1 of 15 |
| Thoracic pain | Musculoskeletal and connective tissue disorders | 1 of 15 |
The registry lists 0 serious adverse events among 15 at risk and 1 other adverse event among 15 at risk for the doravirine plus TAF/FTC single arm. These are counts, not comparative estimates. There is no separate control group in this study, so the figures cannot quantify a difference in safety between regimens.
The record lists no serious adverse-event terms among the top serious terms. The registry entries available do not give a description of the other event, its severity, timing, outcome, or assessment of its relationship to treatment. Those details are not reported in the registry record. The figures also do not establish that no other types of symptoms or outcomes occurred; they describe only the adverse-event categories and counts included in the record.
Safety counts should be interpreted with the size and design of the study in mind, without converting the counts into percentages or treating a lack of reported serious events as proof of absence of risk. The registry record does not provide a comparative safety analysis or enough detail to assess event patterns over time.
12. What the Result Does, and Does Not, Mean
The record describes a study intended to characterize total doravirine concentration in cerebrospinal fluid after 4 weeks of doravirine with FTC/TAF. It specifies a median and interquartile range for a descriptive analysis of all participants (15). The result itself—the numerical concentration summary—is not reported in the registry record. As a result, the record does not allow readers to judge the observed concentration level or how variable the measurements were.
The study description situates cerebrospinal-fluid measurement within interest in drug exposure and HIV suppression in the central nervous system. But drug concentration and viral suppression are different outcomes. The primary measure is concentration; the record does not provide a numerical primary endpoint result or a reported result for suppression in cerebrospinal fluid. The background rationale should not be mistaken for an observed finding.
Because the study has a single group and no listed comparator, it cannot establish a relative effect versus another treatment. The combined regimen also means that an outcome under treatment would not isolate doravirine’s contribution. These limitations do not negate the descriptive purpose; they define the scope of what the design could address.
The record lists 0 serious adverse events and 1 other adverse event in the arm, but does not provide further event detail in the registry record. Those counts alone do not establish comparative safety or rule out risks. Overall, the registry entry documents the intended question, treatment regimen, outcome summary method, and limited safety counts, while leaving the numerical primary results and important analytic details unreported.
13. Why This Trial Matters Statistically
Statistically, the study illustrates the difference between defining an outcome and reporting its result. The registry identifies cerebrospinal-fluid doravirine concentration as the primary measure and specifies median and interquartile range summaries. Yet without those numerical summaries, readers cannot assess the observed exposure or its variability. A clear endpoint definition is useful, but it does not substitute for outcome estimates.
The single-group design is also important for interpreting pharmacokinetic evidence. It can provide a descriptive account of concentrations during the stated doravirine plus FTC/TAF regimen, but it does not create a randomized comparison with another regimen. The combined treatment further limits attribution to an individual drug. These design features determine which statistical questions can be addressed, regardless of the clinical interest of the study topic.
The record’s stated interest in both drug concentration and antiviral activity highlights the need to distinguish pharmacokinetic outcomes from virologic outcomes. Concentration may be relevant to questions about drug exposure, but it is not interchangeable with a measure of viral suppression. In this record, the primary outcome is concentration, and numerical values for it are not reported.
Finally, the registry’s planning assumptions should be kept separate from observed results. A sample-size illustration can describe hypothetical design choices, but it is not evidence of observed power and cannot fill in missing outcome estimates. This record is therefore most informative about the study’s question and design, and less informative about the magnitude or precision of its primary result.
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 central limitation for interpreting the primary outcome is that the registry record does not report its numerical median or interquartile range. Without these values, the size and spread of cerebrospinal-fluid concentrations cannot be evaluated. No confidence interval or other uncertainty estimate for the primary outcome is reported either.
The study is single group, with no like-for-like control comparison. Therefore, it cannot estimate a relative difference between regimens. Doravirine is administered with FTC/TAF, so an outcome under the combined regimen cannot be attributed to doravirine alone. Masking is listed as none, and the record does not describe laboratory measurement procedures or quality-control methods.
Although the study description refers to antiviral activity and maintaining suppression in cerebrospinal fluid, the primary outcome listed is drug concentration. A numerical suppression result or detailed suppression endpoint is not included in the registry record. The number and definitions of the other outcome measures are also not reported beyond the record’s listing of 8 outcome measures.
Further limitations arise from missing analytic detail. The registry record does not report a detailed statistical analysis plan, missing-data methods, a multiplicity approach, interim analyses, or a sample-size justification. It does not provide individual concentration data or information about their distribution. These omissions prevent a fuller assessment of precision, robustness, and the handling of incomplete measurements. The adverse-event record likewise lacks descriptive detail beyond the reported counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Doravirine Concentrations and Antiviral Activity in Cerebrospinal Fluid in HIV-1 Infected Individuals — NCT04079452.
- Registry record last updated 2022-04-28; status last verified 2022-04.
- Results first posted 2022-04-28.
- 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
This completed phase 3 study enrolled 15 people with HIV-1 infection and used a single-group design without masking. The regimen was doravirine 100 mg once daily with Descovy® TAF/FTC 25/200 mg once daily during 4 weeks. The primary outcome was total doravirine concentration in cerebrospinal fluid at 4 weeks, to be summarized by median and interquartile range for all participants (15).
The registry record does not report the numerical primary outcome result. It lists 0 serious adverse events and 1 other adverse event among 15 at risk. The study began on 2020-02-18, reached primary completion on 2020-08-31, and is listed as completed. Its single-group design supports description of the regimen but not a comparative treatment estimate.