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 A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (NCT05543174), a na phase 3 study. The primary outcome is Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05543174 |
| Phase | Phase 3 |
| Condition | Alagille Syndrome (ALGS) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Takeda |
| Results posted | Yes |
2. Clinical Question
Population
Participants with Alagille Syndrome (ALGS) who received at least one dose of study drug. The registry record reports enrollment of 7 participants.
Intervention
TAK-625 was given orally once daily at 200 mcg per kilogram for 1 week, followed by 400 mcg per kilogram once daily after Week 1.
Comparator
There was no separate comparator group. The study is recorded as a single-group study, with allocation listed as not applicable.
Primary question
What was the mean change in fasting serum bile acid levels from Week 18 to Week 22 among participants treated with TAK-625?
The primary question concerns change over time within the treated cohort, not a difference between TAK-625 and a control treatment. The endpoint is fasting serum bile acid (sBA) level, measured in micromoles per liter (mcmol/L), with change defined as the Week 22 observed value minus the Week 18 observed value. A mean change describes the cohort’s average change, while the confidence interval is intended to convey uncertainty around that estimate.
The registry also describes outcomes involving fasting sBA, itch severity reported by observers and participants, and liver enzyme levels. Those outcomes include assessments over different time intervals, so they address distinct aspects of the study’s measurements rather than replacing the primary question. Numerical results for the primary endpoint are not reported in the registry record. Consequently, the size and direction of the average change, and its statistical precision, cannot be assessed from the endpoint description alone.
3. Trial Design
This was a Phase 3, open-label study of TAK-625 in people with Alagille Syndrome. The registry lists the study as completed, with 7 participants enrolled. It records a single-group model, no masking, and allocation as not applicable. There was no randomized control group described in the registry record, so the design does not support a direct randomized comparison between TAK-625 and another treatment or a control condition.
The treatment regimen began with TAK-625 200 mcg per kilogram orally once daily for 1 week. After Week 1, the recorded regimen was 400 mcg per kilogram orally once daily. The brief study description says participants were treated up to the end of the study, about 34 months. It also describes 9 clinic visits from the start of the study, followed by clinic visits every 12 weeks until the end of the study.
The study start date is 2023-01-16, and the primary completion date is 2023-10-25. These dates describe the registry record’s study timeline; they do not supply the duration of follow-up for each participant or the timing of each participant’s final assessment. Those details are not reported in the registry record.
The primary outcome was change in fasting sBA levels from Week 18 to Week 22. The registry says the outcome data were collected, analyzed, and reported as a single cohort, and defines the analysis population as all participants who received at least one dose of study drug. Because the numerical endpoint result and its confidence interval are not reported, the design description alone cannot show the cohort’s measured average change.
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 diagnosed with Alagille Syndrome (ALGS) were enrolled to receive TAK-625 once daily (QD). The study consisted of the screening period, 2-week dose escalation period, 46-week stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
Overall Study
| Stage | TAK-625 |
|---|---|
| STARTED | 7 |
| Dosed TAK-625 200 mcg/kg | 7 |
| Dosed TAK-625 400 mcg/kg | 7 |
| COMPLETED | 6 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | TAK-625 |
|---|---|
| Adverse Event | 1 |
Analysis population for the primary outcome, as defined in the registry: ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort.
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: Intention-to-treat (ITT) included all participants who received at least one dose of study drug. As per-planned analysis, the data for baseline measures were collected, analyzed and reported as a single cohort.
| Characteristic | Unit | TAK-625 |
|---|---|---|
| Participants analysed | 7 | |
| Age, Continuous | years | 6.143 (SD 3.8675) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 5 |
| Race/Ethnicity, Customized: Asian | Participants | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22, measured over From Week 18 to Week 22.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | micromoles per liter (mcmol/L) |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change in fasting serum bile acid (sBA) levels from Week 18 to Week 22, measured in micromoles per liter (mcmol/L). The registry defines change as the observed value at Week 22 minus the observed value at Week 18. This is a continuous outcome: it can take a range of measured values rather than representing a simple event count. The planned summary is the mean change, accompanied by a 95% confidence interval.
In general, a change endpoint describes how a measurement differs between two specified time points. It focuses the analysis on within-participant change across that interval, although the reported cohort mean alone does not show how much individual participants varied. The mean is useful for describing the average change in the measured group; it does not imply that every participant changed by that amount.
Fasting sBA is the biomarker selected for the primary endpoint in this study. The registry also lists a fasting sBA outcome from baseline to Week 18, as well as itch and liver enzyme outcomes. Those are separate measures and time windows. The rationale for selecting this specific endpoint and interval is not reported in the registry record.
The registry describes the endpoint and its intended summary but does not report the numerical mean change or confidence interval. Without those results, the observed direction, magnitude, and precision of the primary endpoint cannot be described.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a single treated cohort of 7 participants and specifies the primary measurement: mean change in fasting sBA levels from Week 18 to Week 22. It does not report the numerical mean change or its 95% confidence interval. Therefore, the posted record does not establish whether the cohort’s average measured level increased or decreased over that interval, how large any average change was, or how precisely it was estimated.
A confidence interval is a statistical expression of uncertainty around an estimate. In general, a narrower interval indicates greater precision and a wider interval indicates less precision, given the analysis and its assumptions. Because no interval limits are reported in the registry record, precision cannot be judged for this endpoint. The participant count alone cannot substitute for the actual estimate and interval, and it does not reveal the observed distribution of individual changes.
The study has no separate comparator group. The outcome is consequently a within-cohort change over time, not an estimated contrast against an untreated group, placebo, or another therapy. In general, changes observed over time in a single group can reflect multiple influences, including the treatment, the course of the condition, measurement variation, or other changes during follow-up. This design does not isolate the contribution of TAK-625 from those possible influences.
The registry lists 14 outcome measures and 0 analyses. It describes secondary outcomes involving fasting sBA, observer- and participant-reported itch measures, and alanine aminotransferase and alkaline phosphatase levels. Numerical results for these measures are not reported in the registry record. Thus, the listed outcome names indicate what was assessed or intended for assessment, not what the measured results were.
The safety record lists serious adverse events among the TAK-625 group, other adverse events, and deaths. These figures describe what was recorded for the study cohort; without a comparator, they do not quantify how event rates differ from another group. The small enrolled cohort also means that the record’s event counts are specific to a limited number of participants and should not be treated as a precise estimate of event frequency in a wider population.
Overall, the registry documents the study design, dosing schedule, endpoint definition, and some adverse event counts. It does not provide the numerical primary efficacy result. Interpretation should therefore remain limited to the study’s stated question and the information actually recorded, rather than inferring a treatment effect from the endpoint description or study completion status.
9. Statistical Methods Explained
The registry identifies the primary endpoint as a continuous measure and specifies the mean as its summary statistic. It also specifies a 95% confidence interval. The numerical estimate and interval are not reported in the registry record, so the method can be described but its result cannot be interpreted numerically.
What does a mean change represent?
In general, a mean is the arithmetic average of the observed values included in an analysis. For a change endpoint, each value represents the difference between measurements at the specified time points. Here, the registry defines each change as the Week 22 observed fasting sBA value minus the Week 18 observed value. A positive change under that definition would correspond to a higher observed value at Week 22 than at Week 18; a negative change would correspond to a lower observed value. These are general interpretations of the subtraction rule, not claims about the unreported result.
An average summarizes the cohort, but it does not describe the full spread of participant-level changes. Participants can have different changes even when the mean is near zero, and a mean can be influenced by unusually large or small observations. The registry record does not report participant-level data or a measure of dispersion for the endpoint beyond specifying a confidence interval for the mean.
How should a confidence interval be read?
In general, a confidence interval presents a range of values compatible with an estimate under the statistical method and assumptions used. It conveys sampling uncertainty, not the range containing every participant’s result and not a guarantee that the true value falls within the displayed limits. Its width is often used as an indication of precision: a wider interval reflects more uncertainty than a narrower interval, all else being equal.
The registry specifies a 95% confidence interval for the primary mean change but does not report its limits. The interval’s width and position relative to zero therefore cannot be assessed. Without the interval and point estimate, it is not possible to describe the estimate’s direction, magnitude, or precision. No statistical significance conclusion can be drawn from the endpoint description alone.
What does the single-group design allow?
The study is recorded as a single-group study with allocation listed as not applicable and masking listed as none. The primary endpoint compares measurements at two time points in the same treated cohort. This is different from a randomized comparison, which estimates a difference between groups assigned to different conditions. In general, a before-and-after change can describe the observed trajectory in a cohort, but without a concurrent comparator it cannot separate a treatment-associated change from changes that might have occurred over time for other reasons.
Open-label means that the study was not masked according to the registry. In general, awareness of treatment can be relevant to how outcomes are reported, assessed, or managed, especially for outcomes involving subjective assessments. The primary endpoint is a laboratory measure, while several listed secondary outcomes concern itch reports by participants or observers. The registry does not report additional procedures intended to limit assessment or reporting effects.
What do planning calculations mean?
In general, a sample-size calculation is a planning exercise based on assumptions about the effect to detect, variability, significance level, allocation, and statistical method. It does not describe what was observed in a trial and is not observed power. The registry’s stated illustration assumptions refer to a two-sided significance level of 0.05, equal allocation between two hypothetical arms, a normal approximation, and effects set as fractions of an observed effect. It distinguishes total event counts for time-to-event illustrations from participants per arm for other illustrations.
Those planning assumptions do not change the actual design recorded for this study: the trial is a single-group study, not a two-arm randomized comparison. The registry record does not provide the primary endpoint estimate needed to interpret an illustration based on the observed effect, nor does it report numerical sample-size illustration values in the registry record for this analysis. Any such illustration would be a hypothetical planning scenario, not evidence about the trial’s observed efficacy or power.
What information is missing for a fuller statistical reading?
The registry describes the analysis population as all participants who received at least one dose of study drug and says outcome data were collected, analyzed, and reported as a single cohort. It does not report the numerical primary result, the confidence interval limits, participant-level observations, or additional details of the statistical model. A model description, treatment of incomplete measurements, and any adjustments beyond the stated summary are not reported in the registry record. As a result, the endpoint’s statistical behavior and the exact analysis implementation cannot be independently evaluated from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 14 outcome measures, including the primary change in fasting sBA from Week 18 to Week 22 and secondary measures involving fasting sBA, itch severity, and liver enzyme levels. When a study evaluates multiple outcomes or time points, there are multiple opportunities to observe apparently notable results. In general, multiplicity procedures can be used to control the chance of false-positive conclusions across a defined family of tests. Whether such procedures were planned or used is not reported in the registry record.
The record reports 0 analyses. It gives outcome descriptions but does not provide numerical efficacy results or a detailed statistical analysis plan. The approach to missing outcome measurements, including whether incomplete data were excluded, imputed, or handled by another method, is not reported in the registry record. The number of participants contributing data to each endpoint and time point is also not reported in the registry record. These details matter because the analyzed group for a particular measurement can differ from the enrolled cohort.
The primary analysis population is described as all participants who received at least one dose of study drug, with results collected, analyzed, and reported as a single cohort. This defines the intended cohort in the registry description, but does not establish how missing Week 18 or Week 22 measurements were handled or how many participants contributed to the primary endpoint. Details of interim analyses, multiplicity control, and the full statistical plan are not reported in the registry record. The lack of those details limits assessment of how the reported outcomes, if available, would relate to the study’s prespecified inferential framework.
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 |
|---|---|---|---|
| TAK-625 | 1 | 7 | 14.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| TAK-625 | 0 of 7 |
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 |
|---|---|
| TAK-625 | 7 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | TAK-625 |
|---|---|---|
| Liver transplant | Surgical and medical procedures | 1 of 7 |
| Malnutrition | Metabolism and nutrition disorders | 1 of 7 |
| Weight gain poor | Metabolism and nutrition disorders | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | TAK-625 |
|---|---|---|
| Influenza | Infections and infestations | 4 of 7 |
| Upper respiratory tract infection | Infections and infestations | 3 of 7 |
| Alanine aminotransferase increased | Investigations | 2 of 7 |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | 2 of 7 |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | 2 of 7 |
| Hand-foot-and-mouth disease | Infections and infestations | 2 of 7 |
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | 2 of 7 |
| Administration site extravasation | General disorders | 1 of 7 |
| Anaemia | Blood and lymphatic system disorders | 1 of 7 |
| Anal rash | Gastrointestinal disorders | 1 of 7 |
Serious events with the highest arm-level rate: liver transplant (14.3%), malnutrition (14.3%), weight gain poor (14.3%).
The registry lists 7 participants at risk for serious adverse events in the TAK-625 group and 1 participant affected. It lists 7 participants at risk for other adverse events and 7 affected. Deaths are recorded as 0. The listed serious adverse event terms are liver transplant, malnutrition, and weight gain poor, each reported as 14.3%.
These figures are counts and percentages recorded for this study cohort. They do not by themselves establish that an event was caused by TAK-625. The registry record does not provide further case-level descriptions, timing, duration, severity beyond the serious-event classification, or an assessment of relatedness in the registry record for this analysis. The three listed terms should be read as event categories recorded in the registry, not as a complete clinical account of each affected participant.
Because the study has no separate comparator group, the adverse event figures cannot be used to calculate a comparative risk against another treatment or an untreated group. The enrolled cohort was 7 participants, so the recorded counts describe a small study cohort and do not provide a precise estimate of how often these events would occur in a broader population. The registry’s completed status and the death count do not change those interpretive limits.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase 3, open-label, single-group study of TAK-625 in Alagille Syndrome was completed and enrolled 7 participants. It specifies the oral dosing schedule and identifies mean change in fasting sBA from Week 18 to Week 22 as the primary endpoint. It also provides a definition of the endpoint and identifies the analysis population as participants who received at least one dose of study drug.
However, the numerical mean change and its 95% confidence interval are not reported in the registry record. The posted information therefore cannot establish the direction or size of the observed average change, or how precisely it was estimated. Outcome descriptions alone are not outcome results. Study completion also does not show whether an endpoint changed or whether the treatment caused any change.
Even if a within-cohort change were reported, the single-group design would describe change over time among treated participants rather than a treatment effect estimated against a control group. In general, a controlled comparison helps distinguish differences associated with assigned treatment from other temporal influences. The design recorded does not make that contrast.
The adverse event information documents events in the study cohort, including serious events and other events, but does not provide a comparator-based safety estimate. The listed event figures should not be read as proof of causation or as a prediction of event frequency in a larger group. The registry does not report further detail in the registry record for this analysis.
Accordingly, the strongest conclusions are about what the study was designed to measure and what the registry records about its design and safety counts. The primary numerical efficacy result, analysis details needed to evaluate it, and a controlled treatment comparison are not reported in the registry record. No conclusion about treatment benefit or lack of benefit follows from the absence of those results.
13. Why This Trial Matters Statistically
This study is statistically notable because it asks a defined biomarker question in a single cohort and specifies a change interval from Week 18 to Week 22. The endpoint definition makes the direction of a numerical change interpretable: the later observed value is compared with the earlier observed value. Reporting a mean with a confidence interval would ordinarily allow readers to consider both the cohort’s average change and the precision of that estimate.
The registry also illustrates the difference between a clinical study design and a hypothetical sample-size planning exercise. The actual study is recorded as single-group, with allocation not applicable. The stated planning illustration assumptions involve equal allocation between two arms and effects expressed as fractions of an observed effect. Those assumptions describe a hypothetical planning framework and do not turn this study into a two-arm comparison or establish observed power.
There is an important distinction between an endpoint being named and its result being available. The registry lists 14 outcome measures and 0 analyses, while the numerical primary endpoint estimate and confidence interval are not reported in the registry record. Without those results, readers cannot assess magnitude, direction, or precision. This makes clear why complete outcome reporting is necessary for a statistical interpretation, beyond a design description and an outcome definition.
The single-group design also illustrates a core principle of inference: change over time within a treated cohort is not equivalent to a comparative treatment effect. A comparator can help account for changes that might occur without the study treatment, while the record contains no separate control group. The distinction is relevant both for efficacy interpretation and for reading adverse event counts, which are descriptive rather than comparative in this 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 primary limitation for interpreting the study’s efficacy is that the numerical primary endpoint result and its 95% confidence interval are not reported in the registry record. The endpoint is defined, but without the estimate and interval the observed direction, magnitude, and precision cannot be evaluated. Numerical results for the listed secondary outcomes are also not reported in the registry record.
The study enrolled 7 participants and used a single-group, open-label design with no separate comparator. The observed within-cohort change, if reported, could not by itself distinguish treatment-associated change from other changes over time. The small cohort also means the recorded adverse event counts describe only this study group; they cannot establish comparative safety or provide a precise estimate for a broader population.
The analysis population is described as participants who received at least one dose, but the number contributing data to the primary endpoint is not reported in the registry record. Handling of missing measurements, details of the statistical analysis, and any multiplicity approach are not reported in the registry record. These omissions prevent assessment of how incomplete outcome data or multiple outcomes were addressed.
The registry lists serious adverse event terms and overall affected counts but does not provide further case-level detail in the registry record for this analysis. Relatedness assessments, timing, and other contextual details are not reported in the registry record. The stated planning assumptions are hypothetical and are not evidence of observed power or proof that the actual study was designed around those calculations.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) — NCT05543174.
- Registry record last updated 2026-01-29; status last verified 2026-01.
- Results first posted 2024-10-15.
- 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 3, open-label, single-group study of TAK-625 for Alagille Syndrome. Enrollment was 7. Participants received 200 mcg per kilogram orally once daily for 1 week, followed by 400 mcg per kilogram once daily after Week 1.
The primary endpoint was mean change in fasting sBA from Week 18 to Week 22, defined as the Week 22 value minus the Week 18 value and intended to be reported with a 95% confidence interval. The numerical result and interval are not reported in the registry record. The record lists 14 outcome measures and 0 analyses.
For safety, the registry records 1 participant affected by a serious adverse event, 7 affected by other adverse events, and 0 deaths. No separate comparator group is described, so the efficacy and safety information is descriptive of the study cohort rather than a randomized comparison.