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 Treatment of Chronic Hepatitis C During Pregnancy With Sofosbuvir/Velpatasvir (NCT04382404), a na phase 1 study. The primary outcome is Maximum Concentration of Velpatasvir in Maternal Plasma.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04382404 |
| Phase | Phase 1 |
| Condition | Hepatitis C, Chronic |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 11 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Catherine Anne Chappell |
| Results posted | Yes |
2. Clinical Question
Population
Pregnant women with chronic hepatitis C virus infection. The registry reports enrollment of 11 participants; for the primary endpoint, 10 maternal participants had results from at least two visits, while one participant discontinued study drug after one dose.
Intervention
A 12-week course of the sofosbuvir/velpatasvir drug combination. The study is described as open label, meaning treatment masking was absent.
Comparator
The trial itself is single group, not a randomized comparison. The brief summary describes a planned comparison of pharmacokinetic parameters with a historical cohort of nonpregnant women, but details and results for that cohort are not reported in the registry record.
Primary question
What is the maximum concentration of velpatasvir in maternal plasma during treatment in pregnancy? This is a pharmacokinetic question about drug concentration, not a direct test of clinical benefit. The primary endpoint estimate is not reported in the registry record.
The study focuses on plasma exposure during pregnancy, with scheduled sampling designed to characterize concentrations over time after a dose. It also lists secondary outcomes concerning intracellular concentrations of a sofosbuvir metabolite in maternal samples, maternal plasma hepatitis C virus quantity after treatment, and adverse events in maternal and infant participants. Those outcome descriptions establish what the registry intended to assess; they do not, by themselves, establish the magnitude or direction of any result.
The clinical question is therefore narrower than whether treatment improves health outcomes. The primary measure is a concentration statistic, and the record does not report a viral response estimate, a comparison with untreated pregnant participants, or a randomized treatment contrast. The historical nonpregnant comparison mentioned in the summary is not a concurrent control group. No conclusions about relative treatment effects can be drawn from the design information alone.
3. Trial Design
This is a Phase 1, single-group, single-center, open-label study of sofosbuvir/velpatasvir in pregnant women with chronic hepatitis C. The registry lists enrollment of 11 and status as completed. The listed study start date is 2020-10-22, and the primary completion date is 2023-10-16. The brief summary describes a 12-week course and says the study would evaluate plasma pharmacokinetic parameters during pregnancy.
Because there is one treatment group and no random allocation, the study does not provide a randomized comparison between pregnant participants assigned to different treatments. The brief summary refers to comparison with a historical cohort of nonpregnant women. A historical comparison uses data collected outside the current trial rather than participants randomized alongside the study group. The cohort’s size, selection, measurement details, and comparative results are not reported in the registry record.
The primary endpoint is maximum maternal plasma concentration of velpatasvir, expressed in ng/mL and summarized using a geometric mean with geometric coefficient of variation. The stated observation period is up to 9 weeks from treatment initiation. Plasma sampling is described at the 3- and 9-week visits before dosing and at several post-dose times; at the 6-week visit, a single convenience blood sample was collected. The record says that 10 maternal participants contributed results from two or more visits and that one participant discontinued study drug after one dose.
These design features describe a pharmacokinetic investigation rather than a trial structured around a reported clinical outcome comparison. The specific protocol rationale, sample-size justification, and statistical analysis plan are not reported in the registry record. The enrollment figure and the number contributing repeated visits are distinct descriptions: the former is the registered enrollment, while the latter describes the primary endpoint population.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: 11 mother-infant dyads were enrolled
Overall Study
| Stage | Sofosbuvir-Velpatasvir |
|---|---|
| STARTED | 11 |
| COMPLETED | 8 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Sofosbuvir-Velpatasvir |
|---|---|
| Adverse Event | 1 |
| Lost to Follow-up | 2 |
Analysis population for the primary outcome, as defined in the registry: 10 maternal participants had results from 2 or more visits; 1 maternal participant discontinued study drug after one dose.
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.
Population: Maternal baseline characteristics are reported unless otherwise stated
| Characteristic | Unit | Sofosbuvir-Velpatasvir |
|---|---|---|
| Participants analysed | 22 | |
| Age, Continuous: Maternal Age, years | years | 30 (25 to 39) |
| Age, Continuous: Infant Age, weeks | weeks | 39.00 (35.57 to 39.43) |
| Sex: Female, Male: Mothers – Female | Participants | 11 |
| Sex: Female, Male: Mothers – Male | Participants | 0 |
| Sex: Female, Male: Infants – Female | Participants | 6 |
| Sex: Female, Male: Infants – Male | Participants | 5 |
| Ethnicity (NIH/OMB): Mothers – Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Mothers – Not Hispanic or Latino | Participants | 11 |
| Ethnicity (NIH/OMB): Mothers – Unknown or Not Reported | Participants | 0 |
| Ethnicity (NIH/OMB): Infants – Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Infants – Not Hispanic or Latino | Participants | 10 |
| Ethnicity (NIH/OMB): Infants – Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): Mothers – American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Mothers – Asian | Participants | 0 |
| Race (NIH/OMB): Mothers – Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Mothers – Black or African American | Participants | 1 |
| Race (NIH/OMB): Mothers – White | Participants | 10 |
| Race (NIH/OMB): Mothers – More than one race | Participants | 0 |
| Race (NIH/OMB): Mothers – Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): Infants – American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Infants – Asian | Participants | 0 |
| Race (NIH/OMB): Infants – Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Infants – Black or African American | Participants | 1 |
| Race (NIH/OMB): Infants – White | Participants | 9 |
| Race (NIH/OMB): Infants – More than one race | Participants | 1 |
| Race (NIH/OMB): Infants – Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 22 |
| Hepatitis C Virus Genotype: Genotype 1 | Participants | 8 |
| Hepatitis C Virus Genotype: Genotype 3 | Participants | 3 |
| Gestational Age at Treatment Start | weeks | 23.43 (23.00 to 25.86) |
| Weight: Mothers | kg | 73.5 (54.4 to 101.1) |
| Weight: Infants | kg | 2.9 (2.5 to 3.8) |
| Body Mass Index | kg/m2 | 27.0 (22.7 to 34.9) |
| Serum Creatinine | mg/dL | 0.5 (0.4 to 0.6) |
| Hematocrit | percent | 36 (33 to 38) |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Concentration of Velpatasvir in Maternal Plasma, measured over Up to 9 weeks from initiation of treatment.
| Element | Registry entry |
|---|---|
| Result type | Geometric Mean |
| Measure of spread | Geometric Coefficient of Variation |
| Unit | ng/mL |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the maximum concentration of velpatasvir measured in maternal plasma. Concentration is reported in ng/mL. The stated timeframe is up to 9 weeks from initiation of treatment. Samples were collected before dosing and at 0.5-, 1-, 2-, 3-, 4-, 5-, 8-, 12-, and 24-hour post-dose times at the 3- and 9-week visits. At the 6-week visit, a single convenience blood sample was collected rather than the described series of timed samples.
In general, maximum concentration is a pharmacokinetic measure of peak drug exposure over a sampling interval. Characterizing peak concentration can help describe how drug exposure behaves during treatment. It is not, on its own, a measure of whether a treatment changes a clinical outcome or benefits a participant. The registry does not state a separate rationale for selecting this endpoint.
The endpoint is specified as a geometric mean, with geometric coefficient of variation as its measure of dispersion. In general, a geometric mean summarizes positive measurements on a multiplicative scale, while the geometric coefficient of variation describes relative spread on that scale. These summaries differ from an arithmetic mean and standard deviation, so they should be interpreted using their stated scale.
The registry record does not report the primary endpoint estimate or its geometric coefficient of variation. The endpoint definition and sampling schedule therefore identify what was intended to be measured, but do not reveal the observed concentration level or its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes the study as completed and reports enrollment of 11. For the primary pharmacokinetic outcome, 10 maternal participants had results from two or more visits, and one participant discontinued study drug after one dose. Those details indicate the size of the enrolled group and the endpoint population as described in the record. They do not provide the observed maximum concentration of velpatasvir.
The primary outcome is a geometric mean concentration, with geometric coefficient of variation. Neither the estimate nor the dispersion value is reported in the registry record. As a result, the posted information does not permit a quantitative interpretation of maternal plasma exposure, its variation among participants, or changes over the scheduled visits. No confidence interval or other uncertainty estimate for the primary endpoint is reported in the registry record.
The brief summary says the investigators intended to compare pharmacokinetic parameters with a historical cohort of nonpregnant women. The record does not report the historical cohort’s corresponding estimate, the comparison result, or details sufficient to assess how comparable the groups or measurements were. The study’s single-group, nonrandomized design also means the treatment group is not a like-for-like randomized control comparison.
The registry lists secondary outcomes covering intracellular concentrations of GS-461203 in maternal peripheral blood mononuclear cells and dried maternal blood spots at 3, 6, and 9 weeks; maternal plasma hepatitis C virus quantity after treatment; and adverse events in maternal and infant participants. The outcome descriptions alone do not establish the results for these measures. The number of outcome measures and analyses is recorded, but outcome-specific estimates are not included in the registry record described for this analysis.
For safety, the record lists 3 of 11 maternal participants and 5 of 11 infant participants as having serious adverse events, with no deaths in either group. It also lists other adverse events for 11 of 11 participants in each group. These are registry counts, not estimates of the excess risk attributable to treatment. The record’s serious-event terms and percentages describe events recorded during the study; they do not establish that the drug caused them.
Overall, the posted information supports a description of the trial design, endpoint, participant accounting, and recorded event counts. It does not support an estimate of the primary pharmacokinetic result or a comparative treatment effect. The absence of those estimates in the registry record is a limit on interpretation, not evidence that an effect was absent.
9. Statistical Methods Explained
The trial is identified as Phase 1 and single group. Its primary outcome is a pharmacokinetic measure, rather than a randomized contrast in a clinical outcome. The registry describes a geometric mean and geometric coefficient of variation for maximum maternal plasma concentration of velpatasvir. It does not report the statistical analysis plan or the numerical primary outcome result.
What does a maximum concentration measure?
In general, a maximum concentration is the highest measured drug concentration within a defined sampling profile. Its interpretation depends on when samples were taken relative to dosing and on how the endpoint was calculated from those measurements. The registry specifies timed samples before and after dosing at the 3- and 9-week visits, as well as a single convenience sample at the 6-week visit. The exact analysis procedure used to derive the maximum is not reported in the registry record.
Sampling at multiple post-dose times can describe the concentration profile around a dose more fully than a single measurement. A single convenience sample does not provide the same timed profile as a series of post-dose samples. The record gives the sampling schedule but does not explain how measurements from different visits were combined, how irregular or missing samples were handled, or whether any model was used to estimate concentrations between observed time points. Those analytic details are not reported in the registry record.
Why use a geometric mean?
In general, concentration measurements are positive and may vary multiplicatively. A geometric mean is calculated on a logarithmic scale and then expressed back on the original scale. It describes a central value in a way that can be appropriate for multiplicative variation. It is not interchangeable with an arithmetic mean. The registry identifies the geometric mean as the summary statistic but does not report the value.
The geometric coefficient of variation is a measure of relative variability associated with a geometric-scale summary. In general, it indicates how dispersed the observations are relative to the central level, rather than expressing spread in the original concentration units as a standard deviation would. The record does not report its value, a confidence interval, or other uncertainty measure for the primary outcome. Without those results, neither the concentration level nor its precision can be assessed from the registry record.
What can a single-group design establish?
A single-group study can describe measurements observed among participants who received the study regimen. It does not create a concurrent untreated or alternative-treatment group through random assignment. Consequently, comparisons with another group require additional assumptions and attention to differences in participant selection, measurement, and study conditions. The brief summary mentions a historical cohort of nonpregnant women, but the cohort’s details and results are not reported in the registry record. A historical comparison is not equivalent to randomization within the current study.
In general, randomization helps balance measured and unmeasured characteristics between groups on average. Since this study is single group and allocation is listed as not applicable, a randomized between-group treatment effect cannot be estimated from its design. This does not prevent descriptive pharmacokinetic reporting; it limits causal interpretation of comparisons with people outside the trial.
What does the sample size tell the reader?
The registry lists enrollment of 11, while the primary endpoint population description states that 10 maternal participants had results from two or more visits and one discontinued after one dose. The registry record does not report a sample-size justification or the assumptions used to choose enrollment. The difference between enrollment and the described endpoint population should not be resolved by assuming a particular analysis population or imputation method.
In general, a small pharmacokinetic study can provide descriptive information, but the precision of estimates depends on the observed data and the analysis. Participant count alone does not establish precision. A confidence interval, if reported, would express uncertainty around an estimate under the analysis assumptions; no such interval for the primary outcome is reported in the registry record. Planning calculations based on an observed effect would not constitute observed power and would not replace the trial’s own design rationale, which is not reported.
What statistical details remain unknown?
The statistical plan, including any transformations, model specification, handling of repeated measures, criteria for excluding observations, and approach to missing measurements, is not reported in the registry record. It is also not reported whether there were interim analyses. These details matter because pharmacokinetic data may be analyzed using different summaries or models, and the choice can affect the interpretation of estimates and uncertainty.
The registry lists 34 outcome measures and 12 analyses. In general, evaluating multiple outcomes can raise a multiplicity question: if many hypotheses are tested, the chance of at least one apparently unusual result can increase unless the analysis plan addresses this issue. The record does not report a multiplicity adjustment or a prespecified hierarchy for interpreting the analyses. That absence should be described as missing information, not as proof that no such procedure was used outside the registry entry.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 34 outcome measures and 12 analyses. It names a primary pharmacokinetic endpoint and several secondary outcomes, including maternal intracellular metabolite concentrations at 3, 6, and 9 weeks, maternal plasma hepatitis C virus quantity after treatment, and adverse events among maternal and infant participants. The full statistical analysis plan and the relationship between listed outcomes and analyses are not reported in the registry record.
In general, assessing multiple endpoints or conducting multiple analyses can create a multiplicity issue: the more statistical questions examined, the greater the chance that an apparently notable finding arises by chance. A prespecified hierarchy, adjustment procedure, or other interpretation strategy may address this concern. Whether any such approach was used is not reported in the registry record, so the listed analysis count alone does not establish how multiplicity was handled.
The primary endpoint population description states that 10 maternal participants had results from two or more visits and that one participant discontinued study drug after one dose. This indicates that the available repeated-visit data did not cover every enrolled maternal participant in the same way. The record does not report the amount or pattern of missing measurements, the analysis population definition, or the method used to handle incomplete observations. It is therefore not possible to determine from the registry record whether missingness could have influenced the primary estimate.
The record also does not report whether an intention-to-treat, per-protocol, or other named analysis population was used. It does not specify whether the participant who stopped after one dose contributed to any particular analysis. These are unresolved reporting details; they should not be filled in by assumption.
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 |
|---|---|---|---|
| Sofosbuvir-Velpatasvir - Maternal Participants | 3 | 11 | 27.3% |
| Sofosbuvir-Velpatasvir - Infant Participants | 5 | 11 | 45.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Sofosbuvir-Velpatasvir - Maternal Participants | 0 of 11 |
| Sofosbuvir-Velpatasvir - Infant Participants | 0 of 11 |
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 |
|---|---|
| Sofosbuvir-Velpatasvir - Maternal Participants | 11 of 11 |
| Sofosbuvir-Velpatasvir - Infant Participants | 11 of 11 |
Most frequent serious adverse event terms
| Event term | Organ system | Sofosbuvir-Velpatasvir - Maternal Participants | Sofosbuvir-Velpatasvir - Infant Participants |
|---|---|---|---|
| Cholelithiasis | Gastrointestinal disorders | 1 of 11 | 0 of 11 |
| Acute Pyelonephritis | Infections and infestations | 1 of 11 | 0 of 11 |
| Renal calculi | Renal and urinary disorders | 1 of 11 | 0 of 11 |
| Clubfoot | Congenital, familial and genetic disorders | 0 of 11 | 1 of 11 |
| Cryptorchidism | Reproductive system and breast disorders | 0 of 11 | 1 of 11 |
| Hyperbilirubinemia | Hepatobiliary disorders | 0 of 11 | 1 of 11 |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | 0 of 11 | 1 of 11 |
| Transient tachypnea | Respiratory, thoracic and mediastinal disorders | 0 of 11 | 1 of 11 |
| Positive toxicology screen | Injury, poisoning and procedural complications | 0 of 11 | 1 of 11 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sofosbuvir-Velpatasvir - Maternal Participants | Sofosbuvir-Velpatasvir - Infant Participants |
|---|---|---|---|
| Upper respiratory tract infection | Respiratory, thoracic and mediastinal disorders | 0 of 11 | 6 of 11 |
| Anemia | Blood and lymphatic system disorders | 4 of 11 | 1 of 11 |
| Headache | Nervous system disorders | 4 of 11 | 0 of 11 |
| COVID-19 | Respiratory, thoracic and mediastinal disorders | 3 of 11 | 1 of 11 |
| Hemorrhage, postpartum | Pregnancy, puerperium and perinatal conditions | 3 of 11 | 0 of 11 |
| Vomiting | Gastrointestinal disorders | 3 of 11 | 0 of 11 |
| Otitis media | Ear and labyrinth disorders | 0 of 11 | 3 of 11 |
| Nausea | Gastrointestinal disorders | 2 of 11 | 0 of 11 |
| Fatigue | General disorders | 2 of 11 | 0 of 11 |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 2 of 11 | 0 of 11 |
Serious events with the highest arm-level rate: cholelithiasis (9.1%), acute pyelonephritis (9.1%), renal calculi (9.1%), clubfoot (9.1%), cryptorchidism (9.1%).
The registry reports serious adverse events separately for maternal and infant participants. In the maternal group, 3 of 11 participants are listed as having serious adverse events; in the infant group, 5 of 11 are listed. The record lists no deaths in either group. It also reports other adverse events for 11 of 11 participants in each group.
The serious-event terms listed in the registry include Cholelithiasis, Acute Pyelonephritis, Renal calculi, Clubfoot, Cryptorchidism, and Hyperbilirubinemia, each shown with 9.1%. The record does not provide further event-level detail in the registry record, such as timing, duration, recurrence, or the number of events per affected participant. These terms and percentages should be read as registry summaries, not as a complete clinical account of each event.
In general, an adverse event recorded during a study is not necessarily caused by the study treatment. The counts describe events among participants in the trial; they do not establish a causal relationship or quantify excess risk relative to a control group. Because this study has a single treatment group, the listed counts do not provide a randomized safety comparison. The registry record does not report additional information needed to assess event attribution or comparative safety.
12. What the Result Does, and Does Not, Mean
The study addresses a specific pharmacokinetic question: maximum velpatasvir concentration in maternal plasma during treatment in pregnancy. Its Phase 1, single-group design and sampling schedule describe how that question was approached. The registry identifies the summary measure and the endpoint population, but does not report the primary concentration estimate or its variability. The central pharmacokinetic result therefore cannot be interpreted quantitatively from the registry record.
The record also describes a planned comparison with a historical cohort of nonpregnant women. No comparative estimate or supporting cohort details are reported. Even if results from such a cohort were available, a historical comparison would require careful consideration of differences in participants and measurement conditions; it would not be a randomized comparison within this trial.
The adverse-event counts document serious events and other events recorded among maternal and infant participants. They do not show whether the treatment caused those events, and the lack of a concurrent randomized control group prevents a like-for-like estimate of comparative risk. The absence of deaths in the recorded groups is a descriptive fact, not a complete measure of safety.
These registry entries do not establish an effect on hepatitis C virus quantity, a clinical benefit, or a treatment effect relative to another regimen. The secondary outcome names indicate what was intended to be measured, but the outcome values are not reported in the information available for this analysis. Likewise, the registry’s completed status does not substitute for numerical outcome reporting.
The study can be understood as a small, early-phase investigation of drug concentrations during pregnancy, with maternal and infant adverse-event recording. Its design and the information reported define the scope of any interpretation. Missing endpoint estimates and analysis details leave the magnitude, precision, and comparative meaning of the pharmacokinetic findings unresolved in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for the type of question it asks. Rather than estimating a randomized treatment effect on a clinical endpoint, it focuses on maternal plasma pharmacokinetics during pregnancy. The primary measure, maximum velpatasvir concentration, is a description of drug exposure. Keeping that distinction clear prevents a concentration endpoint from being interpreted as a direct clinical outcome.
The sampling schedule is part of the statistical meaning of the endpoint. Timed samples at the 3- and 9-week visits are described alongside a single convenience sample at the 6-week visit. The record does not state how these different sampling patterns were combined or analyzed. That omission matters because the result’s meaning depends not only on the named endpoint, but also on how observed concentrations were summarized.
Participant accounting also matters. Enrollment is 11, while 10 maternal participants had results from two or more visits and one participant discontinued after one dose. A reader needs those details to understand which participants are described as contributing repeated-visit results. The registry does not report the analysis population definition or approach to incomplete data, so the exact relationship between enrollment and each outcome analysis remains uncertain.
Finally, the record illustrates the limits of a single-group design when a historical comparison is mentioned. Such a comparison can provide context, but it does not have the protections of random assignment within a concurrent trial. Here, the historical cohort details and comparison results are not reported. The trial’s statistical contribution therefore cannot be judged from design labels alone; numerical estimates, uncertainty measures, and analysis details are needed for a fuller interpretation.
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 registry describes a single-center, single-group, open-label Phase 1 study with enrollment of 11. This design is suited to describing measurements within the treated group, but it does not provide a randomized comparator. The brief summary mentions a historical cohort of nonpregnant women, yet details about that cohort and the comparison results are not reported in the registry record. Differences between historical and trial populations or measurement procedures therefore cannot be assessed.
The primary endpoint is defined, but its numerical estimate and geometric coefficient of variation are not reported. No confidence interval or other uncertainty measure is reported for that outcome. Without these quantities, the primary result’s magnitude and precision cannot be evaluated. The record also does not report the full analysis method used to derive the maximum concentration.
Participant information indicates that 10 maternal participants had results from two or more visits and that one participant discontinued after one dose. The registry does not explain how incomplete visits or measurements were handled, define the analysis population, or report whether the participant who discontinued contributed to particular analyses. The potential effect of incomplete observations cannot be assessed from the record.
The sampling schedule includes repeated timed samples at some visits and a single convenience sample at another. The record does not report how that difference was reflected in the analysis. It also does not report a sample-size justification, the statistical analysis plan, any interim analyses, or how multiplicity across the listed outcomes and analyses was addressed.
Although the registry reports adverse-event counts, it does not provide enough event-level or comparative information in the registry record to determine attribution or relative risk. These limitations concern what the registry record reports; they do not establish that additional analyses or details were absent from other study documents.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treatment of Chronic Hepatitis C During Pregnancy With Sofosbuvir/Velpatasvir — NCT04382404.
- Registry record last updated 2025-01-01; status last verified 2024-12.
- Results first posted 2025-01-01.
- 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 1 study examined sofosbuvir/velpatasvir in pregnant women with chronic hepatitis C. It is described as a single-center, single-group, open-label trial of a 12-week course, with enrollment of 11. The primary endpoint was maximum maternal plasma velpatasvir concentration, summarized as a geometric mean with geometric coefficient of variation.
The registry states that 10 maternal participants had results from two or more visits and one discontinued after one dose. It does not report the primary endpoint estimate, its variability, or a comparison result for the historical nonpregnant cohort mentioned in the brief summary. Maternal and infant serious adverse-event counts are reported as 3 of 11 and 5 of 11, respectively, with no deaths in either group.
The record does not report the statistical analysis plan, sample-size justification, missing-data method, or multiplicity approach. The posted information supports a description of the study design and reported event counts, but does not permit a quantitative interpretation of the primary pharmacokinetic result.