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 Human Fetal Liver Cell Transplantation in Chronic Liver Failure (NCT01013194), a non-randomized phase 1, phase 2 study. The primary outcome is Patient Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01013194 |
| Phase | Phase 1, Phase 2 |
| Condition | Liver Cirrhosis |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | The Mediterranean Institute for Transplantation and Advanced Specialized Therapies |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with liver cirrhosis and chronic liver failure. The registry lists a total enrollment of 25, but it does not describe the patient characteristics or eligibility criteria in the record. The number enrolled should not be assumed to be the number included in every outcome analysis.
Intervention
The intervention was human fetal liver cell transplantation. The registry describes transplantation of liver progenitor cells isolated from human fetal liver tissue, with the stated aim of improving conventional liver therapy and broadening options other than liver transplantation. Details of the procedure and its delivery are not reported in the registry record.
Comparator
The comparison group consisted of cirrhotic patients on standard therapy. Allocation was non-randomized, so treated patients and control patients were not assigned by chance. The registry flags that the arms are not a like-for-like control comparison; differences between them may therefore reflect factors other than the intervention.
Primary question
The primary endpoint was patient survival at 1 year. The registry describes this as an assessment of survival in treated and control patients. The survival results themselves are not reported in the registry record, so this page cannot establish the size or direction of any difference between the groups.
3. Trial Design
This completed study is registered as a phase 1 and phase 2 trial of human fetal liver cell transplantation for chronic liver failure. It used a parallel-group, non-randomized design with no masking. The registry describes one group of cirrhotic patients treated with the cell transplantation and a control group of cirrhotic patients receiving standard therapy. The enrollment was 25. The sponsor was The Mediterranean Institute for Transplantation and Advanced Specialized Therapies.
The registry lists a start date of 2007-02 and a primary completion date of 2011-04. Its status is completed. These dates describe the record’s study timeline; they do not, by themselves, show when each participant entered or completed follow-up. The primary endpoint was patient survival at 1 year. The secondary outcomes were changes in Child-Pugh Score and MELD Score from baseline to 1 year follow-up.
Non-randomized allocation is central to interpreting this design. In a randomized comparison, chance assignment is intended to make groups comparable, on average, with respect to measured and unmeasured characteristics. Here, assignment was not random. The registry explicitly cautions that the arms are not a like-for-like control comparison. Thus, a difference between groups, if one were reported, could not automatically be attributed to transplantation: baseline health, treatment selection, or other differences could contribute. The registry does not report the allocation process or details needed to assess how patients entered each group.
The parallel structure means the record describes separate treated and control groups rather than a crossover design. No masking was used. The record does not report a protocol rationale for the design choices, a sample-size justification, or details of follow-up beyond the endpoint timeframes stated above. calculated tables and plots elsewhere on the page should be read alongside these design features, not as a substitute for 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 | Treated Patients | Control Group |
|---|---|---|
| STARTED | 9 | 16 |
| COMPLETED | 9 | 16 |
| NOT COMPLETED | 0 | 0 |
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 | Treated Patients | Control Patients | Total |
|---|---|---|---|---|
| Participants analysed | 9 | 16 | 25 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 9 | 16 | 25 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 3 | 3 | 6 |
| Sex: Female, Male: Male | Participants | 6 | 13 | 19 |
| Region of Enrollment: Italy | participants | 9 | 16 | 25 |
6. Primary Endpoint
The registry lists the primary outcome as Patient Survival, measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is patient survival at 1 year. The registry describes it as an assessment of survival among treated and control patients at the 1 year follow-up point. In general, survival is a clinically meaningful outcome because it captures whether participants are alive at a specified follow-up time. It can provide a direct summary of an important outcome, although interpretation depends on how the endpoint is defined and how complete follow-up is.
The registry lists the outcome unit as participants and its parameter as a number. However, the operational definition of survival, the number of participants alive in each group, and the analysis used to compare groups are not reported in the registry record. The record also does not state how participants lost to follow-up or with unknown status were handled. These details matter: a count alone does not show the precision of an estimate, and incomplete status information can affect how an endpoint is interpreted.
The endpoint timeframe is 1 year. It should not be read as a time-to-event analysis unless the registry reports such an analysis; that information is not reported in the registry record. The secondary outcomes, Child-Pugh Score and MELD Score from baseline to 1 year follow-up, offer different measures of patient status, but their results are not described. The posted results available in this record do not provide survival estimates or a treatment-effect estimate to interpret.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies patient survival at 1 year as the primary endpoint, but the survival counts or estimates are not reported in the registry record. As a result, there is no posted estimate of survival in either group, no stated difference between groups, and no confidence interval or test result to assess. It is not possible to describe the direction, size, or statistical precision of a survival contrast from this record.
The design further limits what a between-group comparison could establish. Allocation was non-randomized, and the registry itself warns that the arms are not a like-for-like control comparison. In general, non-randomized groups can differ before treatment begins. Such differences can be related to both treatment selection and outcome, creating confounding. A measured difference in survival would therefore need to be interpreted as an association in this study unless the analysis and its assumptions justified stronger causal language. The adjustment strategy is not reported in the registry record.
A confidence interval, when available, describes a range of values compatible with the data and the method used to calculate it. Its width conveys precision: a wider interval indicates greater uncertainty. A p-value, when available, addresses how compatible the data are with a specified null model under the analysis assumptions; it does not measure the probability that the intervention is effective. Neither type of inferential result is reported for the primary endpoint in the record. No conclusion about statistical significance or precision can therefore be drawn.
The registry lists secondary outcomes involving Child-Pugh Score and MELD Score from baseline to 1 year follow-up. Their result estimates and analysis details are not reported in the registry record. The existence of outcome names alone does not show whether the measures changed, whether groups differed, or how uncertain any estimate might be.
The safety entries require equally careful reading. The registry lists zero serious adverse events and zero other adverse events in the stated at-risk counts for both groups. Death counts are not reported. These safety entries do not fill the gap in the primary survival result: the adverse-event fields and survival endpoint are distinct records, and the available adverse-event entries do not state survival by group.
Overall, the posted record describes a study question and design, but it does not provide the outcome estimates needed to quantify what happened. The tables and plots on the page should be read as displays of posted information and should not be treated as a substitute for missing endpoint results, uncertainty estimates, or analysis details.
9. Statistical Methods Explained
The registry describes a parallel, non-randomized comparison. In general, parallel-group studies follow separate groups receiving different care approaches. Random allocation can help balance prognostic factors across groups; non-randomized allocation does not provide that protection. The registry specifically notes that these arms are not a like-for-like control comparison. Therefore, any group contrast needs to be interpreted with attention to possible baseline differences and confounding. The method used to allocate participants, and any adjustment for group differences, are not reported in the registry record.
What does the primary endpoint represent?
The primary endpoint is patient survival at 1 year, with the registry describing the measure in terms of participants. A binary endpoint generally classifies each participant into one of two outcome categories, such as meeting or not meeting a defined condition. That is a general description, not confirmation of the exact coding used. The registry does not state how unknown survival status was categorized, how follow-up was handled, or whether all enrolled participants contributed to this endpoint. Those operational details are not reported in the registry record.
For a binary outcome, analysts may report counts and proportions, a difference between proportions, or a relative measure. These are different summaries and answer different questions. Counts show how many participants fall into each category; an effect estimate describes the contrast between groups; and an interval can communicate uncertainty around that estimate. The posted primary outcome results and their inferential statistics are not reported in the registry record. No such estimate should be inferred from enrollment or safety counts.
Why does uncertainty matter?
In general, an estimate based on a study sample is subject to sampling variability. A confidence interval communicates the range of values that remain reasonably compatible with the observed data under a specified method and assumptions. A narrow interval indicates more precision than a wide one, but an interval does not resolve bias from non-randomized allocation, incomplete follow-up, or measurement problems. The registry record does not report a confidence interval for the primary endpoint, so its precision cannot be assessed.
A p-value is also a method-dependent summary, not a measure of the magnitude or importance of an effect. It does not give the probability that a treatment works, and it cannot correct for confounding or missing data. No primary-endpoint p-value is reported in the registry record. It is therefore not possible to describe the result as statistically significant or not statistically significant based on this record.
What can the planning illustration show?
The sample-size section on the page is a planning illustration computed from the observed effect, rather than a report of the study’s original design calculation. Its stated assumptions include a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation; the effect to detect is expressed as a stated fraction of the observed effect. The resulting sample sizes are planning quantities, not observed power and not evidence that the study had a particular probability of detecting an effect. The trial’s own sample-size justification is not reported in the registry record. The illustration also cannot repair the limitations of non-randomized allocation or supply missing endpoint estimates.
In general, planning calculations depend on assumptions about the effect to detect, variability or event frequency, significance level, allocation, and statistical method. Changing assumptions changes the calculated planning requirement. A calculation based on an observed effect is especially dependent on that effect estimate, which may itself be uncertain. The illustration should therefore be read as a conditional planning exercise, not as a definitive evaluation of adequacy for this trial.
How should multiple outcomes be considered?
The registry lists the primary survival endpoint and secondary outcomes for Child-Pugh Score and MELD Score. It also lists three outcome measures and three analyses. When a study examines multiple outcomes or performs multiple tests, the chance of at least one apparently unusual result can increase if each test is interpreted separately. In general, multiplicity methods can control or describe that issue, but their use depends on the analysis plan and the inferential goal. A multiplicity procedure is not reported in the registry record. There is also no reported analysis plan from which to determine whether the listed outcomes were tested in a prespecified order.
More broadly, key analytic details are absent from the record: the statistical model, any covariate adjustment, the handling of missing outcome data, the analysis population, and the method for uncertainty estimation are not reported in the registry record. These omissions mean that the statistical results cannot be independently characterized from the information. The design and endpoint descriptions identify what the study set out to compare, but they do not reveal how the comparison was analyzed or what its results were.
10. Multiplicity, Missing Data and Analysis Populations
The registry identifies a primary endpoint of patient survival at 1 year and secondary outcomes involving Child-Pugh Score and MELD Score from baseline to 1 year follow-up. It lists three outcome measures and three analyses. This tells readers that the record includes several measures, but it does not establish how those outcomes were prioritized in the analysis or whether any formal adjustment was made for examining multiple outcomes.
In general, testing multiple outcomes can increase the chance of observing at least one result that appears noteworthy by chance. Researchers may address this through a prespecified hierarchy, adjustment procedures, or cautious interpretation of secondary analyses. Which approach, if any, was used is not reported in the registry record. The analysis plan and multiplicity procedure are not reported in the registry record, so the secondary outcomes cannot be interpreted as confirmatory evidence based on this information alone.
The registry record does not report how missing observations were handled, whether all enrolled participants were included in each analysis, or how the analysis populations were defined. It also does not report the completeness of one-year follow-up. These details are important because exclusion or unknown status can affect both the estimate and its uncertainty. The denominator used for each endpoint should not be assumed to equal total enrollment.
Similarly, the record does not describe any imputation, sensitivity analysis, or approach to participants who discontinued follow-up. Those methods are not reported in the registry record. Readers should distinguish the registry’s total enrollment from endpoint-specific analysis counts, which are not available for the primary outcome.
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 |
|---|---|---|---|
| Treated Patients | 0 | 9 | 0.0% |
| Control Group | 0 | 16 | 0.0% |
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 |
|---|---|
| Treated Patients | 0 of 9 |
| Control Group | 0 of 16 |
The registry’s safety entries list 9 participants at risk in the treated group and 16 in the control group. For both groups, the recorded number affected is 0 for serious adverse events and 0 for other adverse events. These are the figures displayed in the record; they describe the listed safety categories and denominators, not the primary survival endpoint.
The death entries are not reported in the registry record. A missing death count must not be read as zero deaths. The registry also does not list serious adverse-event terms in the record. The absence of listed terms does not provide detail about how events were defined, collected, reviewed, or attributed.
In general, a zero recorded count means no events were entered for that category in the stated record, not that the risk of such an event is known to be absent. Interpretation depends on complete ascertainment, consistent definitions, follow-up, and the number of participants observed. Those details are not reported in the registry record. These safety figures should be reported as recorded, without treating them as proof of safety or using them to infer survival outcomes.
12. What the Result Does, and Does Not, Mean
This record establishes that a completed phase 1 and phase 2 study examined human fetal liver cell transplantation in chronic liver failure, with standard therapy as the comparison. It identifies patient survival at 1 year as the primary endpoint and lists Child-Pugh Score and MELD Score as secondary outcomes. It also documents the non-randomized, unmasked, parallel design and gives the registry’s safety counts.
It does not, in the record, provide the survival results, group-specific endpoint estimates, or measures of uncertainty. The record therefore does not establish whether survival differed between the groups, how large any difference might have been, or how precisely it was estimated. The secondary outcome results are also not reported in the registry record. It would be inappropriate to claim benefit, lack of benefit, or a particular degree of risk from outcome estimates that are not present.
Even if a group contrast were available, non-randomized allocation would require cautious interpretation. General differences between patients selected for transplantation and those receiving standard therapy could contribute to an observed association. Without details of baseline comparability and adjustment, the extent of that concern cannot be assessed. The registry itself warns that the groups are not a like-for-like control comparison.
The adverse-event entries are limited to the stated categories and at-risk counts; the death counts are not reported in the registry record. Those figures should not be substituted for the primary survival endpoint. Nor does a planning illustration establish observed power or show that the trial could reliably detect a clinically relevant difference. The illustration is conditional on its assumptions.
The most defensible reading is therefore descriptive: the registry records the research question, design, endpoints, timeline, and some safety information, while key efficacy estimates and statistical details are not reported in the registry record. The tables and plots on the page can help organize the available information, but they cannot resolve these gaps.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its central comparison is non-randomized. Clinical studies often need to compare groups formed through treatment decisions rather than chance assignment. In such settings, the comparison can still describe outcomes, but causal interpretation depends on how groups differ and how those differences are addressed. Here the registry explicitly cautions that the treated and control arms are not a like-for-like comparison, making that concern central rather than incidental.
The study also illustrates the difference between knowing an endpoint and having an interpretable result. The primary question is clearly identified as patient survival at 1 year, but the associated survival estimates are not reported in the registry record. Without event counts, effect estimates, and uncertainty measures, readers cannot judge the size or precision of a group contrast. Outcome labels alone are not evidence of an outcome.
Another statistical lesson is the distinction between safety counts and the primary efficacy endpoint. The registry lists zero affected participants in the serious and other adverse-event categories for the at-risk groups, while death counts are not reported. These entries do not provide the one-year survival comparison. Keeping endpoint definitions separate prevents an apparently simple count from being used to answer a different question.
Finally, the planning illustration shows why assumptions matter. A planning calculation based on an observed effect can help explore sample-size requirements under stated assumptions, but it is not observed power and does not replace a prespecified design justification. The trial’s own sample-size justification is not reported in the registry record. Together, these points make the study useful for learning how design, outcome reporting, uncertainty, and planning assumptions shape statistical 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 main trial-specific limitation is non-randomized allocation. The registry explicitly states that the arms are not a like-for-like control comparison. Participants receiving transplantation may have differed from those receiving standard therapy in ways related to outcome, and the information needed to assess such differences is not reported in the registry record. The analysis approach for addressing potential confounding is also not reported in the registry record.
The primary endpoint is described, but its results are not reported in the registry record. There are no survival counts or effect estimates to assess, and no confidence interval or p-value to indicate precision or compatibility with a specified statistical model. The secondary outcome results for Child-Pugh Score and MELD Score are likewise not reported in the registry record.
The record does not report the eligibility criteria, baseline characteristics, endpoint definitions in operational detail, or participant flow by outcome. It also does not describe completeness of one-year follow-up, missing-data handling, or the analysis population. These omissions prevent assessment of whether the reported groups and denominators correspond to all enrolled participants for each endpoint.
Details of the statistical model, any covariate adjustment, multiplicity handling, and the trial’s own sample-size justification are not reported in the registry record. The safety entries list event counts but do not provide detailed event definitions or ascertainment procedures; death counts are not reported in the registry record. These limitations are specific to the information available for this trial and restrict both causal interpretation and assessment of the precision and completeness of its outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Human Fetal Liver Cell Transplantation in Chronic Liver Failure — NCT01013194.
- Registry record last updated 2015-11-03; status last verified 2015-10.
- Results first posted 2015-11-03.
- 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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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This completed phase 1 and phase 2 study examined human fetal liver cell transplantation for chronic liver failure in patients with liver cirrhosis. The registry lists enrollment of 25, a start date of 2007-02, and primary completion on 2011-04. Its parallel-group design was non-randomized and unmasked. The comparison was between treated patients and control patients receiving standard therapy.
The primary endpoint was patient survival at 1 year. Secondary outcomes were Child-Pugh Score and MELD Score from baseline to 1 year follow-up. The registry lists safety denominators of 9 treated participants and 16 control participants, with zero recorded as affected for serious and other adverse events in both groups; death counts are not reported in the registry record.
The survival and secondary outcome results, uncertainty estimates, detailed analysis methods, missing-data handling, and analysis populations are not reported in the registry record. The non-randomized comparison also limits causal interpretation.