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 Study of Various Treatments in Non-alcoholic Fatty Liver Disease (NAFLD) Patients Who Have Aspects of Non-alcoholic Steatohepatitis (NASH) (NCT04147195), a randomized phase 2 study. The primary outcome is Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04147195 |
| Phase | Phase 2 |
| Condition | Non-alcoholic Fatty Liver Disease, Nonalcoholic Steatohepatitis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 41 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Adults with non-alcoholic fatty liver disease (NAFLD) who manifest a non-alcoholic steatohepatitis (NASH)-like biomarker phenotype. The posted record describes 41 participants enrolled in the study; the primary safety population is participants who received at least one dose of study treatment.
Intervention
LYS006 20 mg, administered orally twice per day for 12 weeks. The registry also describes a combination arm in which LYS006 20 mg was administered orally twice per day with LJN452 200ug orally once daily for 12 weeks.
Comparator
The active comparison is LYS006 alone versus LYS006 with LJN452. The record identifies the study as randomized, but does not report the allocation procedure or details of how participants were assigned to the arms.
Primary question
Among participants who received at least one dose, how many experienced adverse events (AEs) or serious adverse events (SAEs) during treatment and follow-up? The primary outcome is a safety count, not a direct measure of liver disease improvement. The study description also includes safety, tolerability, pharmacokinetic and efficacy objectives, but efficacy results are not reported in the registry record.
The study is a phase 2 investigation in a biomarker-defined clinical population, rather than a broad assessment of every person with NAFLD. Its primary question is descriptive: it records the number of participants meeting adverse-event categories. The registry does not report a formal statistical comparison between arms for this primary outcome, so the counts alone should not be interpreted as a test of whether the treatment groups differ.
3. Trial Design
The registry describes NCT04147195 as a randomized, open-label, parallel-group phase 2 study sponsored by Novartis Pharmaceuticals. Enrollment was 41 participants. The study began on 2020-06-04, and primary completion was recorded as 2022-01-06. Its status is terminated. The reason for termination is not reported in the registry record.
The two described treatment arms were LYS006 alone and LYS006 with LJN452. Participants in the LYS006 arm received 20 mg orally twice per day for 12 weeks. In the combination arm, LYS006 20 mg was given orally twice per day together with LJN452 200ug orally once daily for 12 weeks. The record lists Tropifexor as an intervention, while the combination-arm description names LJN452; how these names relate is not reported in the registry record. It is therefore safest to describe the arms using the treatment names and regimens given in their respective registry entries rather than infer an equivalence.
Randomized allocation means treatment assignment is intended to be determined by chance, but the specific sequence-generation and allocation-concealment procedures are not reported in the registry record. Open-label means the record identifies no masking. The record does not state which groups, if any, knew the assigned treatment during outcome assessment. In general, lack of masking can affect reporting or assessment of outcomes that require judgment, while some laboratory or instrument-based measurements may be less directly influenced; the extent of any such effect in this trial is not reported.
The primary safety outcome was assessed from the start of treatment to 28 days after the end of treatment, with assessment up to a maximum duration of 113 Days. The primary safety population comprised participants who received at least one dose. The registry lists additional outcomes including liver fibrosis markers, fasting lipid profile, percent liver fat, body weight, insulin resistance, fasting glucose, fasting insulin and hemoglobin A1c. Outcome results for these measures are not reported in the registry record.
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: Participants underwent a Screening period of up to 33 days followed by a Baseline assessment period of up to 27 days.
Overall Study
| Stage | LYS006 | LYS006 + LJN452 |
|---|---|---|
| STARTED | 20 | 21 |
| PD Analysis Set | 20 | 17 |
| COMPLETED | 16 | 15 |
| NOT COMPLETED | 4 | 6 |
Reasons for not completing this period:
| Reason | LYS006 | LYS006 + LJN452 |
|---|---|---|
| Adverse Event | 0 | 3 |
| Study Terminated by Sponsor | 4 | 2 |
| Withdrawal by Subject | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: All participants who received at least one dose of study treatment
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 | LYS006 | LYS006 + LJN452 | Total |
|---|---|---|---|---|
| Participants analysed | 20 | 21 | 41 | |
| Age, Continuous | Year | 52.0 (SD 9.23) | 54.9 (SD 8.36) | 53.5 (SD 8.81) |
| Sex: Female, Male: Female | Participants | 11 | 11 | 22 |
| Sex: Female, Male: Male | Participants | 9 | 10 | 19 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 1 | 2 |
| Race (NIH/OMB): White | Participants | 19 | 17 | 36 |
| Race (NIH/OMB): More than one race | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs), measured over From the start of treatment to 28 days after end of treatment, assessed up to maximum duration of 113 Days.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events (AEs) and serious adverse events (SAEs). Its definition also includes significant changes from baseline in vital signs, electrocardiograms and laboratory parameters when those changes qualify and are reported as AEs. Results are expressed as numbers of participants in each category, rather than as a continuous measurement or a change in a disease marker.
A safety count is useful for describing how many treated participants had recorded events during a defined observation period. It can help characterize the types of safety outcomes monitored in an early-phase trial and provide context for continued investigation. The population for this outcome includes all participants who received at least one dose, and the observation window runs from treatment start through 28 days after treatment ends, assessed up to a maximum duration of 113 Days.
Counts do not by themselves describe event severity beyond the distinction between the reported SAE and other-event categories. Nor do they indicate how long an event lasted, whether it resolved, whether participants had multiple events, or whether investigators considered an event related to treatment; those details are not reported in the registry record. A participant count also differs from an event count: a person with more than one event may still be counted once in a participant-based category. The endpoint is therefore a summary of participants affected, not a complete account of every event or its clinical course.
Because the primary measure is safety-focused, it does not answer whether liver-related outcomes changed. Although the record lists several efficacy-related secondary outcomes, their results are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results report 0 participants with SAEs in the LYS006 arm, among 20 at risk, and 0 in the LYS006 + LJN452 arm, among 21 at risk. The total is 0 among 41 participants at risk. The record also reports 9 participants with other AEs in the LYS006 arm and 15 in the combination arm, for a total of 24. There were 0 deaths in either arm and 0 overall.
These are descriptive counts in the treated safety population. They establish what the registry reports for these categories during the stated observation window; they do not establish that the underlying risks are identical between arms. With no reported SAE counts in either arm, the record supplies no observed between-arm contrast for that category. The other-AE counts differ numerically, but a numerical difference is not itself evidence of a statistically reliable treatment effect. No inferential analysis, confidence interval or p-value for these safety counts is reported in the registry record.
The primary outcome description includes significant baseline changes in vital signs, electrocardiograms and laboratory parameters when qualifying and reported as AEs. The counts should therefore be read according to that registry definition, not as a count limited to symptoms or diagnoses. The record does not provide event-level detail, severity breakdowns for the other-AE category, event duration, treatment attribution or recurrence. It also does not report the number of events per participant.
The study was randomized, but randomization does not make small groups interchangeable in every observed characteristic. General statistical interpretation: chance imbalances can remain, and a safety count from a small study can be uncertain even when no event is observed in a category. The record does not give confidence intervals for these counts, so their precision cannot be quantified from the posted results alone.
Termination is recorded, but the reason is not reported in the registry record. It should not be inferred from the event counts. The enrollment and safety population counts are recorded, but no details of withdrawals, missing safety assessments or exposure by participant are reported. The efficacy-related outcome values are also not reported in the registry record. Accordingly, the posted results support a narrow description of the recorded safety counts, not a conclusion about efficacy, comparative safety, or the reasons the study ended.
9. Statistical Methods Explained
The primary outcome is a participant count. In general, a count endpoint records how many people meet a defined event criterion during an observation period. It is different from a rate, which uses person-time, and from a continuous endpoint, such as a laboratory value. Here, the registry reports the number of participants in each adverse-event category and the number at risk in each arm. It does not report person-time or an event rate.
What does randomization contribute?
In general, randomization assigns treatment by chance and is intended to reduce systematic differences in prognostic characteristics between groups. It supports a causal comparison when assignment is implemented appropriately, follow-up is adequate, and analyses preserve the randomized comparison. The registry identifies the study as randomized, but the method of sequence generation, allocation concealment, stratification, and analysis according to original assignment is not reported in the registry record. The designation alone does not show how those procedures were carried out.
Even with randomization, modest group sizes can leave imbalances by chance. This matters when interpreting participant counts, because the observed number of events can be affected by baseline characteristics and by the amount of exposure and follow-up. The posted record gives 20 participants at risk in the LYS006 arm and 21 in the combination arm for the safety categories. It does not report individual follow-up time or exposure duration for each participant.
How should counts be interpreted?
A participant count is readily understood, but it contains limited information about uncertainty. General statistical explanation: an estimate based on a count can vary substantially from the underlying event experience, especially when the number of participants is limited or an event is uncommon. A confidence interval, when reported, communicates a range of values compatible with the data under a specified statistical model. No confidence interval for the safety categories is reported in the registry record.
A zero count does not prove that the event cannot occur. It means no participant in the recorded group was reported as having that category during the specified assessment period. The width of any uncertainty around a zero count depends on the design and analysis assumptions; because no interval or model is reported, a numerical precision statement cannot be made from these results. Likewise, the difference between the other-AE counts is descriptive unless accompanied by an appropriate comparative analysis and uncertainty estimate.
What do the posted results estimate?
The primary safety population is all participants who received at least one dose. This is a treated population definition. It is not necessarily the same as an intention-to-treat population, which in general includes all randomized participants in their assigned groups regardless of treatment received. The registry does not report the number randomized, the number who began treatment beyond the described safety population, or the analysis population for secondary outcomes. The primary outcome therefore describes the recorded treated population rather than establishing what an all-randomized analysis would show.
The observation period is defined from treatment start through 28 days after treatment ends, assessed up to a maximum duration of 113 Days. A defined period improves interpretability by specifying when events count toward the endpoint. Different follow-up periods can produce different opportunities to observe events, so comparisons are clearest when follow-up is comparable. Arm-specific follow-up completeness and exposure durations are not reported in the registry record.
What is the role of planning illustrations?
General statistical explanation: sample-size planning translates a chosen target effect and assumptions about variability or event frequency into a proposed number of participants. A planning calculation is conditional on those assumptions; it is not an estimate of what the study achieved. Any sample-size illustrations associated with this record are described as planning based on the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. They are illustrations, not observed power and not evidence that the trial was designed around those assumptions. The trial's own sample-size justification is not reported in the registry record.
In general, power is a design property calculated for a specified effect and model. It is not a measure of the probability that a particular observed result is true. Calculating power after observing results does not repair imprecision or substitute for confidence intervals. The registry does not report a trial-specific power calculation or a sample-size rationale. Any planning outputs should therefore be interpreted only as conditional illustrations, not as evidence about the certainty of the safety findings.
What analyses are not described?
The registry does not report a model for comparing adverse-event counts, adjustment for baseline characteristics, a hypothesis test, or a multiplicity procedure. It also does not report a plan for handling missing values, censoring, treatment discontinuation, or participants with incomplete follow-up. General statistical explanation: these choices can affect estimates and uncertainty, so a descriptive result should not be mistaken for a fully specified comparative analysis. The record reports outcome counts, but not enough analytic detail to reconstruct a formal treatment-effect estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple secondary outcomes, including measures of hepatic fibrosis, fasting lipids, liver fat, body weight, insulin resistance, glucose, insulin and hemoglobin A1c. Testing multiple outcomes can increase the chance of obtaining at least one apparently unusual result by chance. General statistical explanation: multiplicity procedures control or describe that risk, depending on the method used. A multiplicity plan, hierarchy among outcomes, or adjustment procedure is not reported in the registry record. The record lists 13 outcome measures, but the number of analyses recorded is 0; outcome-specific analysis results are not reported.
Missing-data handling is also not described. The registry does not report the number of participants with missing outcome measurements, reasons for missingness, imputation methods, or sensitivity analyses. General statistical explanation: missingness can affect interpretation when the probability of a measurement being absent is related to participants’ health or treatment experience. Without details of completeness and handling, the potential effect cannot be assessed.
The primary safety population is defined as all participants who received at least one dose. That definition is not the same as a reported intention-to-treat analysis. The number randomized and the number excluded from the treated safety population are not reported in the registry record. The analysis populations used for secondary outcomes are also not reported. No conclusions can be drawn about how the randomized groups were analyzed beyond the stated safety-population definition.
The primary endpoint contains several categories and the registry record does not reduce it to one comparison. A prespecified primary contrast or statistical test is not reported. It is therefore not possible to identify a single inferential result or to assess whether any analysis accounted for multiple categories or outcomes.
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 |
|---|---|---|---|
| LYS006 | 0 | 20 | 0.0% |
| LYS006 + LJN452 | 0 | 21 | 0.0% |
| Total | 0 | 41 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| LYS006 | 0 of 20 |
| LYS006 + LJN452 | 0 of 21 |
| Total | 0 of 41 |
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 |
|---|---|
| LYS006 | 9 of 20 |
| LYS006 + LJN452 | 15 of 21 |
| Total | 24 of 41 |
Most frequent non-serious adverse event terms
| Event term | Organ system | LYS006 | LYS006 + LJN452 | Total |
|---|---|---|---|---|
| Pruritus | Skin and subcutaneous tissue disorders | 0 of 20 | 13 of 21 | 13 of 41 |
| Headache | Nervous system disorders | 4 of 20 | 4 of 21 | 8 of 41 |
| Nausea | Gastrointestinal disorders | 3 of 20 | 2 of 21 | 5 of 41 |
| Hyperglycaemia | Metabolism and nutrition disorders | 1 of 20 | 3 of 21 | 4 of 41 |
| Abdominal pain upper | Gastrointestinal disorders | 1 of 20 | 2 of 21 | 3 of 41 |
| Diarrhoea | Gastrointestinal disorders | 3 of 20 | 0 of 21 | 3 of 41 |
| Blood alkaline phosphatase increased | Investigations | 0 of 20 | 3 of 21 | 3 of 41 |
| Alanine aminotransferase increased | Investigations | 0 of 20 | 2 of 21 | 2 of 41 |
| Aspartate aminotransferase increased | Investigations | 0 of 20 | 2 of 21 | 2 of 41 |
| Dizziness | Nervous system disorders | 2 of 20 | 0 of 21 | 2 of 41 |
The SAE count is 0 of 20 participants at risk in the LYS006 arm and 0 of 21 in the LYS006 + LJN452 arm. Overall, 0 of 41 participants at risk were reported with an SAE. The death count is 0 in each arm and 0 overall. These counts describe the categories recorded during the primary safety observation window; they do not establish that the possibility of such events is absent.
The registry separately reports other AEs in 9 of 20 participants receiving LYS006 and 15 of 21 receiving the combination, with 24 of 41 overall. These are participant counts, not counts of every event. A participant may have experienced more than one event, but event totals are not reported. The meaning of “other” should not be expanded beyond the registry’s category; detailed severity, attribution and event descriptions are not reported in the registry record.
In general, safety counts should be considered alongside exposure duration, follow-up completeness and event definitions. The registry specifies assessment from treatment start to 28 days after treatment ended, up to a maximum duration of 113 Days. It does not provide arm-specific exposure time, individual event narratives or a comparative statistical analysis. The counts therefore describe what was recorded in this study, without quantifying a precise difference in safety between regimens.
12. What the Result Does, and Does Not, Mean
The result establishes that the registry reports no SAEs and no deaths among the participants at risk in either arm. It also reports other AEs in both treatment groups. Those observations are useful descriptive information about the recorded safety experience over the stated follow-up period. They do not demonstrate that the regimens have the same safety profile, nor do they establish that one regimen has more or fewer events in the broader population.
The study enrolled 41 participants, and the primary outcome population includes people who received at least one dose. With this limited study population, the counts provide only a bounded snapshot of recorded events. General statistical explanation: absence of an observed event is not proof that the event is impossible, and observed counts can be imprecise. The registry does not report uncertainty intervals or a formal treatment comparison to quantify the precision of the safety findings.
The primary outcome is not an efficacy endpoint. Although the study was designed to evaluate efficacy as well as safety, the registry lists secondary measures of liver fat, fibrosis-related markers, lipid profile, weight and metabolic measures without reporting their results. The posted safety findings therefore do not establish whether any of those measures changed, or whether any treatment affected them.
The registry records the trial as terminated, but does not report the reason. Neither the termination status nor the safety counts alone explain why the study ended. The record also does not report a sample-size justification, detailed statistical plan, missing-data approach, or the results of a comparative analysis. Interpretation should remain close to what is recorded: counts of participants with safety events in two randomized, open-label treatment arms, not a definitive claim about comparative benefit, harm or effectiveness.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a descriptive safety result and a comparative treatment-effect estimate. The primary endpoint records participant counts for AEs and SAEs. Counts can communicate the observed experience directly, but without an inferential analysis or uncertainty interval they do not quantify how precisely the underlying event experience is known or establish a difference between arms.
The design includes randomization, which is intended in general to support fair comparisons by assigning treatment by chance. At the same time, the record identifies an open-label study and a treated safety population. Those features matter when interpreting what is measured and who is included. The registry does not report the randomization procedures, the handling of participants who did not receive treatment, or the statistical analysis plan, so the strength and implementation of the comparative framework cannot be fully evaluated from the record.
The study also illustrates why outcome definition and follow-up matter. Its safety endpoint includes specified clinical and measurement changes when qualifying and reported as AEs, and it uses a defined treatment and follow-up window. The registry lists a broad set of additional outcomes, but their results are not reported. A long list of outcomes is not itself evidence that each was measured successfully or analyzed in a way that supports a clear conclusion.
Finally, the study status is recorded as terminated, with no reason stated. That missing context limits interpretation of study completion and the available results. Statistical reading requires separating what is observed from what remains unknown: the safety counts are reported, while comparative uncertainty, efficacy findings, and the explanation for termination are not reported in the registry record.
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 participant count is limited: enrollment is 41, with 20 participants at risk in the LYS006 arm and 21 in the combination arm for the primary safety categories. The record does not report confidence intervals or another measure of uncertainty for those counts. This limits the precision with which the safety experience can be characterized, particularly for categories with no recorded participants.
The study is open-label. In general, knowledge of treatment assignment can influence reporting, clinical assessment or decisions about whether a change qualifies as an adverse event. The registry does not report blinding of outcome assessors or procedures intended to limit such influence. The extent of any impact on the recorded outcomes cannot be determined from the registry record.
Termination is recorded, but its reason is not reported. The record does not state whether termination affected planned enrollment, follow-up, or outcome reporting. The actual enrollment is reported, but the study's sample-size justification is not reported. The record does not provide a detailed analysis plan, inferential comparison for the safety endpoint, or multiplicity procedure.
Safety findings are limited to participant counts in the reported categories. Event-level descriptions, severity for other AEs, relatedness to treatment, repeated events, time to event, and individual follow-up duration are not reported. Missing outcome data and the approach to incomplete follow-up are also not reported in the registry record.
There is a naming inconsistency to keep in view: Tropifexor appears in the intervention list, while the combination regimen is described using LJN452. The record does not explain the relationship between these names. In addition, although efficacy outcomes are listed, their results are not reported in the registry record, so the available result information does not support interpretation of those outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Various Treatments in Non-alcoholic Fatty Liver Disease (NAFLD) Patients Who Have Aspects of Non-alcoholic Steatohepatitis (NASH) — NCT04147195.
- Registry record last updated 2023-08-21; status last verified 2023-08.
- Results first posted 2023-01-26.
- 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
NCT04147195 is a randomized, open-label, parallel-group phase 2 study of treatments for adults with NAFLD and a NASH-like biomarker phenotype. Enrollment was 41, and the registry status is terminated. The described regimens were LYS006 alone and LYS006 with LJN452, each administered for 12 weeks as specified in the arm descriptions.
The primary outcome counted participants with AEs and SAEs among those who received at least one dose. The registry reports 0 SAEs in either arm, 9 participants with other AEs in the LYS006 arm, 15 in the combination arm, and 0 deaths overall. The safety observation period extended from treatment start to 28 days after treatment ended, up to a maximum duration of 113 Days.
The registry lists secondary efficacy and safety outcomes, but their results, the reason for termination, a formal comparative analysis, and the trial's sample-size justification are not reported in the registry record. The safety counts should therefore be read as descriptive findings rather than as a conclusive comparison of the regimens.