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 Phase II Study Of Oral PHA-848125AC In Patients With Thymic Carcinoma (NCT01011439), a na phase 2 study. The primary outcome is Progression-free Survival Rate at 3 Months.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01011439 |
| Phase | Phase 2 |
| Condition | Thymic Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 72 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Tiziana Life Sciences LTD |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent or metastatic, unresectable thymic carcinoma previously treated with chemotherapy. The primary endpoint population is described as evaluable patients meeting specified pathology, treatment-exposure and assessment requirements.
Intervention
Oral PHA-848125AC, also called milciclib maleate, described as 150 mg/day once daily for 7 consecutive days, followed by 7 days of rest in a 2-week cycle.
Comparator
There is no concurrent comparator arm. The registry classifies the study as single-group, with allocation not applicable and no masking.
Primary question
What proportion of evaluable patients were alive and progression free at 3 months since treatment start? The registry record gives the endpoint definition but does not report the observed proportion.
The study focuses on antitumor activity in a population whose disease had already been treated with chemotherapy. Its stated primary question is a short-term status measure: whether an evaluable patient was both alive and progression free at the specified time. It is not the same as asking how long every patient remained progression free, and it does not by itself describe tumor response or overall survival.
Because the design has one treatment group and no concurrent control, the endpoint can describe outcomes among patients treated in the study, but it cannot directly estimate a contrast against another treatment or against no treatment. The registry also lists confirmed objective response rate, disease control rate, progression-free survival, duration of response, overall survival and safety as secondary outcomes. Their posted results are not reported in the registry record.
3. Trial Design
This is a phase II, single-group study of oral PHA-848125AC in patients with thymic carcinoma previously treated with chemotherapy. The registry lists enrollment as 72. That registry field does not establish how many participants received treatment, met the evaluable-population requirements, or contributed to the primary endpoint denominator; those counts are not reported in the registry record.
The intervention is also named milciclib maleate. The arm description specifies 100 and 50 mg capsules and a dose of 150 mg/day once daily for 7 consecutive days, days 1 to 7, followed by 7 days of rest, days 8 to 14, in a 2-week cycle. The registry lists no masking and no allocation to multiple groups.
The brief study summary describes the intended population as patients with recurrent or metastatic, unresectable thymic carcinoma previously treated with chemotherapy, and the treatment as second-line. The primary endpoint population is narrower: the record describes evaluable patients as requiring histological confirmation by an Independent Review Committee, receipt of at least 80% of the drug in the first two cycles overall, and a baseline and at least one on-treatment tumor assessment.
The listed study start date is 2010-02-22, and the primary completion date is 2017-05-31. The status is terminated. The reason for termination and any implications for recruitment or follow-up are not reported in the registry record. There is no randomized comparison, so outcomes should be read as results from a treatment group rather than as a controlled estimate of the effect of treatment.
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 | Milciclib Maleate (PHA-848125AC) |
|---|---|
| STARTED | 72 |
| COMPLETED | 15 |
| NOT COMPLETED | 57 |
Reasons for not completing this period:
| Reason | Milciclib Maleate (PHA-848125AC) |
|---|---|
| Death | 37 |
| Lost to Follow-up | 4 |
| Withdrawal by Subject | 1 |
| Sponsor's decision | 15 |
Analysis population for the primary outcome, as defined in the registry: Evaluable patients: population consisting of all treated patients who fulfill the following conditions: * histological confirmation of thymic carcinoma by an Independent Review Committee * received at least 80% of drug in the first two cycles overall * baseline and \>/= 1 on treatment tumor assessment or die before tumor re-assessment
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 | Milciclib Maleate (PHA-848125AC) |
|---|---|---|
| Participants analysed | 72 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 57 |
| Age, Categorical: >=65 years | Participants | 15 |
| Age, Continuous | years | 53.2 (SD 13.3) |
| Sex: Female, Male: Female | Participants | 37 |
| Sex: Female, Male: Male | Participants | 35 |
| Race/Ethnicity, Customized: White | Participants | 50 |
| Race/Ethnicity, Customized: Black | Participants | 2 |
| Race/Ethnicity, Customized: Asian | Participants | 3 |
| Race/Ethnicity, Customized: Not Listed | Participants | 10 |
| Race/Ethnicity, Customized: Not allowed to ask per local regulation | Participants | 6 |
| Race/Ethnicity, Customized: Missing | Participants | 1 |
| Region of Enrollment: United States | participants | 13 |
| Region of Enrollment: Italy | participants | 36 |
| Region of Enrollment: France | participants | 23 |
| WHO - Classification: B3 - Well differantiated thymic carcinoma | Participants | 20 |
| WHO - Classification: C - Thymic Carcinoma | Participants | 52 |
| Tumor extent at study entry: Locally advanced | Participants | 1 |
| Tumor extent at study entry: Metastatic | Participants | 71 |
| Masaoka clinical staging at study entry: Stage I: grossly and microscopically encapsulated | Participants | 0 |
| Masaoka clinical staging at study entry: Stage II: thymoma invades beyond the capsule | Participants | 0 |
| Masaoka clinical staging at study entry: Stage III: macroscopic invasion neighboring organs | Participants | 1 |
| Masaoka clinical staging at study entry: Stage IV A: pleural or pericardial dissemination | Participants | 13 |
| Masaoka clinical staging at study entry: Stage IVB:hematogeneous or lymphatic dissemination | Participants | 51 |
| Masaoka clinical staging at study entry: Not Listed | Participants | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival Rate at 3 Months, measured over 3 months since treatment start.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is progression-free survival rate at 3 months since treatment start. It is defined as the proportion of successes—patients alive and progression free at that time—among the total number of evaluable patients. It is a binary status endpoint: each evaluable patient contributes a status at the specified time, rather than a duration measured across the full follow-up period.
This kind of endpoint can provide a fixed-time description of disease control and survival in the defined population. It is different from median progression-free survival, which summarizes time until progression or death, and from objective response rate, which concerns tumor shrinkage under defined response criteria. The registry lists those outcomes separately as secondary outcomes.
Use of the evaluable population means the endpoint is not necessarily based on everyone enrolled or everyone treated. Its definition requires pathology confirmation, a specified level of drug exposure during the first two cycles, and tumor assessment at baseline and on treatment. The number meeting those requirements, the number alive and progression free at 3 months, and the resulting proportion are not reported in the registry record. No confidence interval or other uncertainty estimate for the endpoint is reported there either.
In general, a fixed-time proportion is most informative when its numerator, denominator, timepoint and handling of unavailable assessments are clear. Those details determine which patients contribute to the estimate and how incomplete follow-up affects interpretation. Here, the endpoint definition states the timepoint and general evaluable-population criteria, but does not give the observed estimate or explain how missing status information was handled.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically interpretable primary outcome: the proportion of evaluable patients alive and progression free at 3 months since treatment start. However, it does not report the number of patients meeting that status, the denominator used in the analysis, or the estimated proportion. Without those data, the endpoint’s observed level and precision cannot be assessed from the registry record.
The single-group design is central to interpretation. An outcome rate in the treated group can describe what was observed in that group, but there is no concurrent control group against which to estimate a treatment contrast. Differences between this group and patients in another study could reflect differences in eligibility, disease characteristics, follow-up, assessment procedures or other factors, as well as treatment. The registry classifies allocation as not applicable and lists a single-group model; it does not present a like-for-like control comparison.
The planned evaluable population is also important. Patients must meet pathology confirmation, minimum drug exposure in the first two cycles and tumor-assessment requirements. Restricting analysis to patients meeting such criteria can make the endpoint more specific to patients with adequate assessment and exposure, but it also means that the result, if available, would not automatically describe every enrolled patient. The number excluded from the endpoint population and the reasons for exclusion are not reported in the registry record.
Precision cannot be judged without the estimate and its uncertainty interval. In general, a confidence interval describes a range of values compatible with the observed data under the analysis assumptions; a narrow interval indicates greater precision than a wide one. No primary endpoint confidence interval, standard error or other precision measure is reported in the registry record. Therefore, the posted record does not permit a conclusion about how precisely the 3-month rate was estimated.
The study status is terminated, but that status alone does not explain why the study ended or whether termination affected the analysis. The reason for termination, recruitment history, follow-up completeness and any planned or conducted interim analyses are not reported in the registry record. The listing of secondary outcomes indicates other questions of interest, but their numerical findings are not reported in the registry record. These omissions prevent a fuller assessment of the endpoint alongside response, longer-term outcomes and safety.
Safety figures are available as counts and reported percentages, but they should be interpreted separately from efficacy. They describe adverse-event reporting in the single treatment group and do not establish that an event was caused by the drug. The registry does not provide enough detail to relate the safety counts to individual treatment exposure, event timing or the circumstances of deaths.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study estimates outcomes within the group that receives the study intervention. For a binary endpoint such as alive and progression free at a prespecified time, the basic descriptive quantity is the number meeting the endpoint divided by the number included in that endpoint analysis. That proportion answers a within-group question. Without a concurrent comparator, it does not estimate a difference between treatment strategies.
The registry identifies the study as single-group, with allocation not applicable and no masking. Those design features mean the primary endpoint should not be read as a randomized treatment comparison. The registry does not report an adjustment strategy or a model for estimating a treatment effect against an external or historical group.
How should a fixed-time rate be read?
In general, a fixed-time rate classifies each person in the analysis population according to status at a specific timepoint. The registry defines success as being alive and progression free at 3 months since treatment start. This differs from a time-to-event analysis, which uses the timing of progression or death and can account for follow-up that ends at different times through methods such as censoring, subject to their assumptions.
A fixed-time rate needs a clear denominator and a rule for patients whose status cannot be determined. If missing status is treated differently across analyses, the resulting estimates can differ. Here, the endpoint description identifies an evaluable population and its key eligibility requirements, but the number analyzed and the missing-status rule are not reported in the registry record. It is therefore not possible to check from the record how the endpoint denominator was formed.
What does a confidence interval add?
General statistical explanation: a confidence interval communicates uncertainty around an estimate. The point estimate alone describes the observed value in the analysis data; the interval indicates how much uncertainty remains under the method’s assumptions. It does not make a single-group result comparative, and it does not remove possible bias from patient selection, incomplete assessment or outcome measurement.
The registry record does not report the primary estimate or a confidence interval. Consequently, neither the endpoint level nor its precision can be evaluated from the record. No unadjusted recalculation is possible without the observed numerator and denominator, and no sponsor analysis is reported for comparison.
Why does the analysis population matter?
The endpoint description defines evaluable patients using pathology confirmation by an Independent Review Committee, receipt of at least 80% of the drug in the first two cycles overall, and baseline plus at least one on-treatment tumor assessment. These conditions select patients with specified diagnostic confirmation, exposure and assessment. In general, such a population may differ from all enrolled or treated patients. The resulting endpoint, if reported, would apply directly to the defined evaluable population, not necessarily to all patients who began treatment.
The record does not report the counts for the enrolled, treated and evaluable populations, nor does it give reasons for not meeting the evaluability criteria. Those details would help readers assess selection and understand how broadly the endpoint describes the study population.
What do the planning assumptions mean?
General statistical explanation: a sample-size calculation is a planning exercise that depends on assumptions about the effect to detect, the significance level, allocation and statistical method. The registry record’s stated planning assumptions specify a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation and an effect defined as a stated fraction of the observed effect. Those assumptions describe an illustration, not the design of this single-group study. The record does not report the illustration’s numerical sample-size results in the registry record for this analysis.
Such planning values are not observed power. In general, power is a design property calculated under assumed conditions; using an observed effect after a study does not show the probability that the study correctly detected an effect. The registry’s assumptions also describe a two-arm allocation, which does not match the listed single-group design. The planning illustration therefore should not be treated as a description of how this trial was designed or as evidence of a controlled comparison.
How should multiplicity and safety be considered?
General statistical explanation: when many outcomes or analyses are examined, the chance of at least one apparently noteworthy result can increase unless the analysis plan accounts for multiple testing. The registry lists a primary endpoint and secondary outcomes that include response, disease control, progression-free survival, duration of response, overall survival and safety. A multiplicity strategy, hierarchy for testing, or distinction between confirmatory and exploratory analyses is not reported in the registry record.
Safety counts are descriptive information, not a substitute for a comparison of risk between groups. In general, event counts need their definitions, denominators, observation periods and attribution rules to support a careful interpretation. The registry reports counts for participants affected by serious and other adverse events, deaths, and percentages for selected serious-event terms. Detailed event timing, attribution and denominator definitions for those term percentages are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists progression-free survival rate at 3 months as the primary endpoint and several secondary outcomes: confirmed objective response rate, disease control rate, progression-free survival, duration of response, overall survival and overall safety profile. Considering multiple outcomes raises a general multiplicity issue: the more results examined, the greater the chance of finding an apparently notable result by chance. A testing hierarchy, adjustment for multiplicity, or statement that secondary outcomes are descriptive is not reported in the registry record. The record also does not report an interim-analysis plan.
The primary endpoint is defined for evaluable patients, with requirements for histological confirmation, receipt of at least 80% of the drug in the first two cycles overall, and baseline and at least one on-treatment tumor assessment. The numbers enrolled, treated, evaluable and analyzed for the primary endpoint are not reported in the registry record. Nor are the number of patients missing endpoint status, reasons for missingness, or the approach used to handle missing assessments.
These omissions matter because the observed rate depends on both who is counted and how unavailable outcomes are treated. In general, excluding patients without adequate assessments can yield an estimate for a selected subset, while other missing-data approaches rely on additional assumptions. The registry record does not describe which approach was used, so the robustness of the endpoint to missing data cannot be assessed.
The statistical analysis plan, any prespecified handling of protocol deviations, and any sensitivity analyses are not reported in the registry record. It is therefore not possible to determine whether the posted endpoint analysis followed a particular prespecified strategy or how alternative population definitions would affect it.
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 |
|---|---|---|---|
| Milciclib Maleate (PHA-848125AC) | 22 | 72 | 30.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Milciclib Maleate (PHA-848125AC) | 37 of 72 |
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 |
|---|---|
| Milciclib Maleate (PHA-848125AC) | 72 of 72 |
Most frequent serious adverse event terms
| Event term | Organ system | Milciclib Maleate (PHA-848125AC) |
|---|---|---|
| Syncope | Nervous system disorders | 2 of 72 |
| Pyrexia | General disorders | 2 of 72 |
| Sudden death | General disorders | 2 of 72 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 2 of 72 |
| Diarrhoea | Gastrointestinal disorders | 2 of 72 |
| Vomiting | Gastrointestinal disorders | 2 of 72 |
| Cerebral ischaemia | Nervous system disorders | 1 of 72 |
| Epilepsy NOS | Nervous system disorders | 1 of 72 |
| Muscle contractions involuntary | Nervous system disorders | 1 of 72 |
| Myasthenia gravis | Nervous system disorders | 1 of 72 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Milciclib Maleate (PHA-848125AC) |
|---|---|---|
| Nausea | Gastrointestinal disorders | 65 of 72 |
| Diarrhoea NOS | Gastrointestinal disorders | 56 of 72 |
| Asthenia | General disorders | 48 of 72 |
| Vomiting NOS | Gastrointestinal disorders | 47 of 72 |
| Tremor | Nervous system disorders | 25 of 72 |
| Cough | Respiratory, thoracic and mediastinal disorders | 24 of 72 |
| Anorexia | Metabolism and nutrition disorders | 23 of 72 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 20 of 72 |
| Hypophosphataemia | Metabolism and nutrition disorders | 17 of 72 |
| Headache | Nervous system disorders | 15 of 72 |
Serious events with the highest arm-level rate: syncope (2.8%), pyrexia (2.8%), sudden death (2.8%), pleural effusion (2.8%), diarrhoea (2.8%).
The registry reports 22 participants affected by serious adverse events in the milciclib maleate group, with 72 at risk. It reports 72 participants affected by other adverse events, also with 72 at risk, and 37 deaths. These are safety counts from a single treatment group, not a comparison with a control group. The record does not report whether the deaths were considered related to treatment, their causes or timing, or how they relate to the adverse-event counts.
The listed serious adverse-event terms are syncope, pyrexia, sudden death, pleural effusion, diarrhoea and vomiting, each reported as 2.8%. The record does not specify in the information available for these figures whether the percentages refer to participants or events, or give the denominator used for each term. The terms should not be added together: a participant may have more than one event, and the record does not describe overlap.
In general, an adverse event is not automatically caused by the intervention. Seriousness is a classification of the event’s consequences, not a measure of its likelihood of being drug-related. A fuller assessment would require definitions, exposure and follow-up context, attribution, event-level details and a comparator. Those details are not reported in the registry record, so the counts describe reported safety experience without establishing causation or comparative risk.
12. What the Result Does, and Does Not, Mean
The result this study set out to measure is the proportion of evaluable patients alive and progression free at 3 months since treatment start. The registry record defines that endpoint and describes the evaluable population, but does not report the observed proportion, its numerator or denominator, or an uncertainty interval. As a result, the posted record does not establish the observed level or precision of the primary outcome.
Even if a rate were available, a single-group result would describe the outcome in the study population rather than estimate how outcomes differ under another treatment. Without a concurrent control group, the design cannot separate treatment-associated outcomes from the course of disease or other influences through a randomized comparison. No claim that treatment works or does not work follows from the available record.
The endpoint also has a limited scope. It concerns status at one specified timepoint, not the full duration of progression-free survival, the likelihood of tumor response, duration of response or overall survival. Those outcomes appear among the listed secondary outcomes, but their results are not reported in the registry record. The evaluable-population definition further means that a result would apply directly to patients meeting its assessment and exposure requirements, not automatically to every patient enrolled or treated.
The registry’s safety counts provide a separate descriptive view. They do not identify whether events were caused by the drug, and they are not comparative risk estimates. In particular, the death count cannot be interpreted as treatment-related mortality without information about causes, timing and attribution, which is not reported in the registry record.
The appropriate reading is therefore bounded: the registry documents a terminated, single-group phase II study, its treatment schedule, its primary endpoint definition and selected adverse-event figures. It does not report the primary endpoint outcome or sufficient comparative and analytic detail to support a treatment-effect conclusion. The absence of a reported result in the registry record should not be mistaken for a result showing either benefit or lack of benefit.
13. Why This Trial Matters Statistically
This study illustrates why the distinction between a treatment-group outcome and a treatment effect matters. The primary endpoint is a fixed-time proportion in a single group. Such an endpoint can be clinically understandable, but its interpretation depends on a clearly defined population, complete outcome ascertainment and a measure of statistical uncertainty. The registry describes the endpoint and selected eligibility conditions, yet does not report its estimate or precision.
The evaluable-population criteria also highlight a common issue in clinical-trial reporting: the population used for an endpoint may be narrower than the population enrolled. Pathology confirmation, a minimum level of early treatment exposure and tumor assessments determine eligibility for this endpoint. Without counts and exclusions, readers cannot see how the analysis population relates to all enrolled or treated patients.
The study’s single-group structure makes the absence of a comparator especially important. A descriptive rate cannot answer the same question as a randomized contrast. This remains true even if the endpoint is measured precisely: precision around a within-group rate does not remove the uncertainty involved in attributing outcomes to the intervention.
The record also distinguishes the trial’s actual design from a planning illustration with assumptions involving two arms and equal allocation. Such illustrations can help explain hypothetical sample-size requirements under stated assumptions, but they should not be confused with the trial’s design, observed power or evidence from a concurrent control. The registry does not report numerical results for that illustration.
Finally, safety counts require context. Serious adverse-event counts, other adverse-event counts and deaths can inform descriptive review, but counts alone do not establish causality or comparative risk. This record is a useful example of why outcome estimates, denominators, uncertainty, missing-data handling and event attribution are all needed for a complete 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 most important limitation for interpreting efficacy is that the registry record does not report the primary endpoint result. The number of evaluable patients, the number alive and progression free at 3 months, and an estimate with its uncertainty are not reported in the registry record. The endpoint’s magnitude and precision therefore cannot be assessed.
The study is single-group, with no concurrent comparator and no masking. It cannot provide a randomized estimate of the difference between the intervention and another strategy. The registry gives no account of the reason for termination, recruitment progress, follow-up completeness or any effect of termination on endpoint assessment. These details are not reported in the registry record.
The endpoint is based on an evaluable population defined by pathology confirmation, early drug exposure and tumor assessments. The total counts meeting and failing those conditions, reasons for exclusion, and the relationship between enrolled, treated and analyzed populations are not reported in the registry record. Missing endpoint data and their handling are also not described. These gaps limit assessment of selection and potential missing-data bias.
The record lists secondary endpoints but does not report their results or a multiplicity strategy. The statistical analysis plan, any interim analyses, sensitivity analyses and prespecified approaches to protocol deviations are not reported in the registry record. The planning assumptions describe a hypothetical two-arm illustration and do not match the listed single-group design; numerical illustration results are not reported in the registry record.
For safety, the registry reports counts and selected serious-event percentages but does not report the detailed denominators for the terms, event timing, attribution, causes of death or overlap among events. The available safety figures cannot establish causation or comparative risk. These limitations arise from the information in the registry record and should be kept distinct from conclusions about outcomes that are not reported.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Study Of Oral PHA-848125AC In Patients With Thymic Carcinoma — NCT01011439.
- Registry record last updated 2019-02-06; status last verified 2019-01.
- Results first posted 2018-10-16.
- 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
NCT01011439 is a phase II, single-group study of oral PHA-848125AC, also named milciclib maleate, in patients with thymic carcinoma previously treated with chemotherapy. The registry lists enrollment as 72, no masking, and a terminated status. The primary endpoint is progression-free survival rate at 3 months among evaluable patients. The treatment schedule is described as 150 mg/day once daily for 7 consecutive days followed by 7 days of rest in a 2-week cycle.
The registry lists confirmed objective response rate, disease control rate, progression-free survival, duration of response, overall survival and safety as secondary outcomes. It reports selected safety counts and serious-event terms, but the primary endpoint estimate and the secondary outcome results are not reported in the registry record. The reason for termination, analysis details, missing-data handling and a concurrent comparison are also not reported. The record therefore describes the study’s design and outcome definitions without supplying the primary efficacy result needed to judge its level or precision.