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 MMF for HIV Reservoir Reduction (NCT03262441), a na phase 2 study. The primary outcome is Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 T Cells Over 12 Months.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03262441 |
| Phase | Phase 2 |
| Condition | Human Immunodeficiency Virus I Infection |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fred Hutchinson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Participants with Human Immunodeficiency Virus I Infection were enrolled. The registry record lists an enrollment of 5. Detailed eligibility criteria and participant characteristics are not reported in the registry record.
Intervention
The intervention was mycophenolate mofetil (MMF). The regimen began with 500 mg once per day for one week, then 500 mg twice daily for 22 months if the lower dose was tolerated. The study description frames treatment in the context of antiretroviral therapy (ART).
Comparator
No control group or comparator is listed in the registry design fields. The record identifies a single-group model and allocation as not applicable. Therefore, a between-group treatment comparison cannot be read from the registry record.
Primary question
The primary question was whether cell-associated HIV DNA (ca-DNA) levels changed over 12 months among participants on MMF. The listed endpoint is a regression slope, calculated from measurements at four time points between 0 and 12 months.
The study’s brief summary describes an open-label, randomized Phase II study intended to assess whether MMF given over 22 months meaningfully decreases the size of the HIV reservoir. However, the registry design fields classify the study as single-group, with allocation recorded as NA, and do not list a comparator. Those details do not establish a randomized treatment contrast. The record therefore supports describing the planned within-group question, but not interpreting the study as a like-for-like comparison of MMF with another treatment or with no treatment.
The primary endpoint focuses on ca-DNA over 12 months, while the treatment description extends to 22 months. The study summary also names safety, CD4+ T-cell measures, opportunistic infections, reservoir composition, other reservoir measures, and influenza-vaccine response as questions of interest. These stated aims provide context, but the endpoint estimate and results for those aims are not reported in the registry record.
3. Trial Design
The registry identifies this as a Phase II study with a single-group model, no masking, and allocation recorded as NA. Enrollment is listed as 5, and the study status is completed. The registry lists a start date of 2018-02-12 and a primary completion date of 2019-08-31. The sponsor is Fred Hutchinson Cancer Center.
There is a difference between the brief study description and the registry’s design fields: the description calls the study randomized, while the structured design information lists a single group and no applicable allocation. No comparator arm is listed. In light of that record, treatment effects relative to a control group cannot be inferred from the design details available. The record does not explain the difference between these descriptions.
The intervention was mycophenolate mofetil 500 mg tablets. The regimen specifies 500 mg once per day for one week as a lead-in intended to limit drug-related side effects. If participants tolerated the lower dose, the regimen then increased to 500 mg twice daily by mouth for 22 months. The summary refers to MMF alongside ART, and describes hypotheses concerning ART adherence and antiviral efficacy. The registry record does not report further details about ART regimens.
The primary outcome is a mean continuous measure: the regression slope of change in cell-associated HIV DNA, measured by multiplexed digital droplet PCR. The specified timeframe is 12 months, using four measurement time points between 0 and 12 months. The registry also lists blood CD4+ T cells per mm³ of blood and incidence of opportunistic infection as secondary outcomes. It reports 5 outcome measures and 3 analyses, but the detailed analyses and results are not reported in the registry record.
The record does not report a detailed statistical analysis plan, the method for handling missing observations, the analysis population, or a sample-size justification. It also does not report details of any interim analyses. These omissions limit what can be concluded about how the study’s stated aims were operationalized and analyzed.
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 | Mycophenolate Mofetil |
|---|---|
| STARTED | 5 |
| COMPLETED | 4 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Mycophenolate Mofetil |
|---|---|
| Withdrawal by Subject | 1 |
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 | Mycophenolate Mofetil |
|---|---|---|
| Participants analysed | 5 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 5 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 4 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| 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 | 4 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 T Cells Over 12 Months, measured over 12 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | log10 caDNA copies per 10^6 T-cells/week |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the regression slope of change in cell-associated HIV DNA (ca-DNA), measured by multiplexed digital droplet PCR. It is expressed as log10 caDNA copies per 10^6 T-cells/week, and the specified timeframe is 12 months. The slope is described as being calculated from four time points between 0 and 12 months among participants on MMF.
A slope represents the direction and rate of change over the measurement period, rather than a single measurement at one visit. In general, using measurements over time can describe a pattern of change while retaining information from more than one observation. The log10 scale expresses the outcome on a transformed scale; it can be useful when measurements cover a wide range. These are general statistical explanations, not additional findings about this study.
The endpoint targets cell-associated HIV DNA as one measure of the HIV reservoir. It is distinct from the other reservoir-related measures named in the study summary, including cell-associated RNA and quantitative viral outgrowth. The endpoint’s relevance is that it provides a defined quantitative outcome for assessing change in one biological measure over the stated follow-up period. The record’s definition does not itself establish the size or direction of any observed change.
The registry record does not report the estimated slope, its 95% confidence interval, or a statistical test result. It also does not report the underlying ca-DNA observations. Consequently, the precision and direction of the primary endpoint result cannot be assessed from the registry record. The endpoint definition explains what was intended to be measured, but without an estimate it cannot show how the measurements changed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a specific primary outcome: the regression slope of ca-DNA change over 12 months, with the result intended to be expressed as a mean and accompanied by a 95% confidence interval. However, the primary endpoint estimate and confidence interval are not reported in the registry record. There is therefore no posted numerical result from which to describe the direction, magnitude, or precision of ca-DNA change.
A confidence interval is, in general, a way to express uncertainty around an estimated quantity. Its width can indicate how precisely an estimate is determined under the analysis used. Without the estimate and interval, it is not possible to judge whether the primary result is precise or highly uncertain. The registry record also does not report a p-value for the primary endpoint, so statistical evidence against a null hypothesis cannot be described from the record.
The study description states that MMF was intended to be given over 22 months, whereas the primary endpoint’s timeframe is 12 months. These are distinct durations in the record: the longer treatment period does not substitute for a reported endpoint estimate at the specified primary timeframe. The registry also lists hypotheses about tolerability, ART adherence and antiviral efficacy, CD4+ T-cell measures, opportunistic infections, reservoir composition, other reservoir measures, and influenza-vaccine response. Results for those questions are not reported in the registry record.
The design information also constrains interpretation. Although the brief summary calls the study randomized, the structured fields identify a single group, allocation as NA, and no comparator. No like-for-like control comparison is listed. Any within-group change, if reported elsewhere, would not by itself separate treatment-associated change from other influences over time. In this record, even that within-group primary estimate is not reported.
The adverse-event entries list 5 at risk, 1 affected by a serious adverse event, 0 affected by other adverse events, and 0 deaths for the MMF group. Finger cellulitis is listed among the top serious terms at 20.0%. These figures describe the recorded safety information; they do not establish whether an event was caused by MMF. The small enrollment and absence of reported outcome estimates also limit the conclusions that can be drawn from this registry record.
Planning calculations, when presented, should be distinguished from observed results. The stated sample-size assumptions describe illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Such illustrations are not observed power, and the registry design fields do not list two comparison arms. Without the observed primary estimate and the values of any planning illustrations, no numerical planning conclusion can be assessed from the registry record.
9. Statistical Methods Explained
The primary outcome is a continuous measurement summarized as a mean. The registry describes the primary measure as a regression slope: change in cell-associated HIV DNA (ca-DNA) over the 12-month timeframe, measured by multiplexed digital droplet PCR at four time points between 0 and 12 months. The unit is log10 caDNA copies per 10^6 T-cells/week. The record specifies a 95% confidence interval as the dispersion measure, but the estimate and interval are not reported in the registry record.
What does a regression slope represent?
In general, a regression slope describes how an outcome changes in relation to time or another explanatory variable. For a repeated-measure outcome, a slope can summarize the direction and rate of change across the observation period, rather than treating each measurement as an unrelated result. A slope is an estimate, so its interpretation depends on its scale, sign, and uncertainty. Here, the registry identifies the measure and timeframe but does not report the estimated slope. It is therefore not possible to characterize the observed trajectory.
The outcome is reported on a log10 scale. In general, logarithmic scales are useful for measures that may span a broad range, and they change how distances between values are interpreted. The registry gives the unit as log10 caDNA copies per 10^6 T-cells/week. No transformation details beyond that unit, or details about how the slope was fitted, are reported in the registry record.
How should the confidence interval be understood?
In general, a confidence interval conveys uncertainty around an estimate under the specified statistical model and procedure. The estimate is the central quantity of interest, and the interval gives a range of values compatible with the data and method at the stated confidence level. A narrower interval usually indicates greater precision than a wider one, although precision does not by itself establish clinical or biological importance. The listed 95% confidence interval cannot be interpreted because its bounds and the point estimate are not reported in the registry record.
The registry classifies the endpoint as a mean of a continuous measure. That label describes the type of summary, but it does not reveal whether the statistical model accounted for within-participant correlation among repeated measurements, how departures from model assumptions were assessed, or whether adjustments were used. Those analysis details are not reported in the registry record. These are important distinctions in general: repeated observations from the same participant are often related, and an analysis may need to account for that dependence to quantify uncertainty appropriately.
Why does over-dispersion matter in general?
Over-dispersion is a general issue in statistical models for count data when observed variability exceeds what the model assumes. If it is not addressed, standard errors and confidence intervals can be too small, making results appear more precise than warranted. The primary endpoint in this record is described as a continuous slope, not as a count outcome. The registry record does not report whether over-dispersion was relevant to any secondary or other analyses, or how such analyses were modeled. This explanation is general and does not assert that over-dispersion occurred in this study.
Other considerations for repeated-measure analyses include the number and timing of available observations, missing measurements, and whether the modeled pattern is appropriate for the data. The record states that four time points between 0 and 12 months were used to calculate the endpoint, but does not list the individual measurement times, the fitting method, or procedures for observations that were missing or unusable. These details are not reported in the registry record, so the slope’s modeling assumptions cannot be evaluated from the record.
What do the planning assumptions mean?
The stated sample-size assumptions describe a planning illustration, not a result from the trial. They specify a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect defined as a stated fraction of the observed effect. In general, a sample-size calculation asks how many participants per arm would be needed under those assumptions for a chosen power. Such calculations depend on the assumed effect and variability, among other design choices. The registry record does not report the numerical planning estimates or the observed effect used to generate them.
These planning illustrations should not be described as observed power. Observed power is not a substitute for reporting an estimate and its uncertainty; the information needed to interpret the primary result is the effect estimate and its interval. The equal-allocation, two-arm assumption also does not match the structured design information, which lists a single group and allocation as NA. The planning assumptions therefore cannot be read as evidence that this completed study had a two-arm comparison.
The record reports 5 outcome measures and 3 analyses. Those counts do not specify which outcomes were included in which analyses, whether the primary outcome had a prespecified model, or whether any analysis was confirmatory. The brief summary names several hypotheses, but a hypothesis list is not a full statistical analysis plan. The plan for multiplicity, missing observations, covariate adjustment, and analysis populations is not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The study summary lists multiple hypotheses, covering safety and tolerability, ART adherence and antiviral efficacy, CD4+ T-cell measures, opportunistic infections, reservoir size and composition, detectable reservoir measures, and influenza-vaccine response. The registry lists 5 outcome measures and 3 analyses. These details indicate that the study addressed more than one question, but they do not establish how many formal statistical tests were conducted or how outcome measures were mapped to analyses.
When several outcomes or hypotheses are examined, the chance of at least one apparently noteworthy result can increase. In general, multiplicity procedures can be used to control that risk, or investigators may designate one outcome as primary and treat others as exploratory. The registry identifies a primary endpoint, but a multiplicity adjustment or a hierarchy for secondary outcomes is not reported in the registry record. The statistical analysis plan is not reported in the registry record.
The registry record does not report how missing measurements were handled, whether all enrolled participants were included in the primary analysis, or what analysis population was used. It does not describe exclusion criteria after enrollment, adherence to assigned treatment, or the handling of measurements that could not be quantified. These omissions matter because analyses based on different available observations or populations can lead to different estimates and uncertainty.
The record also does not report whether an interim analysis was planned or conducted, how protocol deviations were handled, or whether any sensitivity analyses were performed. No assumptions about these procedures can be made from the listed endpoint definition or study status. In a study with a single-group design, the analysis would describe change within that group unless a comparator were otherwise documented; the registry design fields do not list one.
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 |
|---|---|---|---|
| Mycophenolate Mofetil | 1 | 5 | 20.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Mycophenolate Mofetil | 0 of 5 |
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 |
|---|---|
| Mycophenolate Mofetil | 0 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Mycophenolate Mofetil |
|---|---|---|
| Finger cellulitis | Skin and subcutaneous tissue disorders | 1 of 5 |
Serious events with the highest arm-level rate: finger cellulitis (20.0%).
The registry lists the MMF group as having 5 participants at risk for serious adverse events and 5 at risk for other adverse events. It records 1 participant affected by a serious adverse event, 0 affected by other adverse events, and 0 deaths. Finger cellulitis appears among the top serious adverse event terms, at 20.0%.
These entries describe the safety counts and term reported in the registry; they do not by themselves establish a causal relationship between MMF and an event. A recorded serious adverse event is not automatically an adverse reaction caused by treatment. The record does not report further details about the event, its timing, severity, outcome, or the assessment of relatedness. It also does not provide a comparator group against which to contextualize the safety figures.
With an enrollment of 5, the safety record is based on a small study group. In general, safety counts from a small group cannot precisely characterize uncommon risks, and zero recorded events does not establish that an event cannot occur. The registry entries should therefore be read as a description of recorded events in this study, not as a general estimate of treatment safety.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed Phase II study of MMF for HIV reservoir reduction and defines a primary endpoint based on the slope of ca-DNA change over 12 months. It also records the intervention regimen and a small set of safety figures. These details establish what the study set out to examine and what was entered in the registry. They do not provide the primary endpoint estimate or its confidence interval.
Without the primary result, the record does not show whether ca-DNA increased, decreased, or remained relatively stable during the measurement period. It does not establish the magnitude or precision of any change. The study summary’s hypotheses are research questions, not reported findings. Results for reservoir composition, cell-associated DNA or RNA beyond the stated endpoint, quantitative viral outgrowth, CD4+ T-cell measures, opportunistic infections, ART adherence, antiviral efficacy, and influenza-vaccine response are not reported in the registry record.
The record’s design fields describe a single-group study with allocation as NA, and no comparator is listed. Although the brief summary uses the word randomized, those design fields do not support a like-for-like controlled comparison. Without a documented comparator and reported results, the registry cannot establish a causal effect attributable to MMF. Even a within-group change would need interpretation in light of the study’s design and analysis methods.
The safety entries are similarly limited: they describe recorded adverse-event figures for the study group, but do not establish causation or characterize risk in a broader population. The record does not report detailed event narratives or a formal analysis of safety outcomes.
Accordingly, the registry record can be used to understand the study’s stated aims, intervention, design classification, endpoint definition, and recorded safety counts. It cannot support conclusions about whether the intended reservoir reduction occurred, how precisely it was measured, or whether observed changes were due to MMF. Those conclusions require outcome estimates and analysis details not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable because it poses a longitudinal question about a biological measure: how ca-DNA changes over time during MMF treatment. A regression slope can summarize the trajectory across repeated measurements, while a confidence interval can communicate uncertainty around that summary. The registry provides the endpoint definition and measurement schedule, but not the estimate or interval. That contrast illustrates why a clearly specified outcome is not a substitute for a reported result.
The design fields also matter for interpretation. A within-group trajectory and a controlled treatment contrast answer different questions. The registry describes a single-group model and lists no comparator, while the brief summary calls the study randomized. Recognizing that distinction is essential: without a comparator, a change over time cannot automatically be attributed to treatment. The record does not reconcile the difference in descriptions.
The enrollment of 5 is relevant to how cautiously the reported safety entries should be read and to the precision that might be expected from estimates. No primary estimate or confidence interval is reported, so the actual precision cannot be evaluated. The safety counts are informative as a registry description but do not characterize uncommon events reliably.
Finally, the summary names several biological and safety hypotheses, while the registry lists multiple outcome measures and analyses without reporting a complete analysis plan. This makes transparency about multiplicity, missing data, and analysis populations important. Those details are not reported in the registry record. The study therefore highlights a general statistical principle: the endpoint, design, analysis, and uncertainty all need to be read together before drawing conclusions from a clinical study.
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 main limitation for interpreting the registry record is that the primary endpoint estimate and its 95% confidence interval are not reported in the registry record. No primary p-value or underlying ca-DNA measurements are reported either. As a result, the direction, magnitude, and precision of the primary outcome cannot be assessed.
The design information is not fully aligned across the record. The brief summary describes an open-label, randomized study, but the structured fields state single-group, allocation NA, and list no comparator. The record does not explain this discrepancy. It therefore does not support a randomized between-group interpretation or a like-for-like control comparison.
Enrollment is listed as 5, limiting the amount of information available for estimating outcome patterns and characterizing safety. The registry does not report detailed participant characteristics, eligibility criteria, or information about treatment adherence and follow-up completion. It also does not report how missing observations were handled or which participants were included in each analysis.
The primary endpoint is defined as a slope calculated from four time points between 0 and 12 months, but the model-fitting details and individual measurement times are not reported. The statistical analysis plan, multiplicity approach, covariate adjustments, and sensitivity analyses are also not reported in the registry record. These gaps prevent assessment of key analytical choices.
The safety record lists counts and a serious adverse event term, but does not report event timing, outcomes, severity details, or relatedness assessments. The registry’s summary of several efficacy and safety hypotheses does not include results for those questions. These limitations are specific to what is documented in the registry record and should not be mistaken for evidence that unreported analyses or results did not exist elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: MMF for HIV Reservoir Reduction — NCT03262441.
- Registry record last updated 2020-12-03; status last verified 2020-11.
- Results first posted 2020-12-03.
- 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 II study of mycophenolate mofetil for HIV reservoir reduction, sponsored by Fred Hutchinson Cancer Center. It lists enrollment of 5, a single-group design, no masking, and allocation as NA. The brief summary calls the study randomized, but no comparator is listed in the structured design fields.
The intervention began with 500 mg once per day for one week, followed by 500 mg twice daily for 22 months if tolerated. The primary endpoint is the regression slope of ca-DNA change over 12 months, measured by multiplexed digital droplet PCR at four time points. Its estimate and 95% confidence interval are not reported in the registry record. Safety entries list 1 serious adverse event-affected participant among 5 at risk, 0 other adverse event-affected participants, and 0 deaths; finger cellulitis is listed at 20.0% among top serious terms.
The record defines the study question and records selected safety information, but does not report the primary result, detailed analysis plan, missing-data methods, or results for the other stated hypotheses.