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 Efficacy Study Looking at a Single-dose of One of Three Dose Levels of AIN457 in Patients With Chronic Plaque-type Psoriasis (NCT00770965), a randomized phase 2 study. The primary outcome is Change From Baseline in Psoriasis Area and Severity Index (PASI) Scores.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00770965 |
| Phase | Phase 2 |
| Condition | Chronic Plaque Psoriasis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 80 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned people with moderate to severe chronic plaque psoriasis. The registry record describes a diagnosis of this condition as the basis for enrollment. It does not report further eligibility details or describe the characteristics of the participants who entered each treatment group.
Intervention
Participants were assigned to one of three single-dose AIN457 groups. The doses were 0.3 mg/kg, 1.0 mg/kg, and 3.0 mg/kg, each given intravenously on Day 1. This design focused on comparing separate dose levels rather than a repeated-dose schedule.
Comparator
The comparator was placebo to AIN457A, administered intravenously on day 1. The study was described as placebo-controlled, randomized, double-blind, and parallel-group. The registry record does not describe the procedures used to maintain masking or the allocation process in further detail.
Primary question
The primary outcome was change from baseline in Psoriasis Area and Severity Index (PASI) scores, assessed from baseline to week 4. The intended efficacy question was whether PASI changed after each single dose compared with placebo. The registry record says efficacy could not be analyzed because participants enrolled at one site were excluded from planned efficacy analyses following audit findings, leaving an insufficient number of participants.
3. Trial Design
This was a Phase II, randomized, double-blind, parallel-group, placebo-controlled study of a single dose of AIN457. Its stated purpose was to assess the efficacy of three dose levels in people with chronic plaque psoriasis. Participants were assigned to AIN457 0.3 mg/kg, AIN457 1.0 mg/kg, AIN457 3.0 mg/kg, or placebo. Each treatment was administered intravenously on Day 1.
The registry record lists planned enrollment of 80. The listed intervention groups each had 20 participants at risk in the adverse-event results. These safety counts do not establish how many participants contributed to any efficacy analysis. In fact, the registry record says efficacy could not be analyzed because participants at one site were excluded from the planned efficacy analyses after audit findings, leaving an insufficient number of participants.
The record identifies the trial as completed and lists its start as 2008-09-10 and primary completion as 2009-09-17. It describes the study as exploratory and states that it was not powered for efficacy. Details of the allocation sequence, concealment, masking procedures, and the intended statistical plan are not reported in the registry record.
The primary outcome was change from baseline in PASI scores, with the timeframe listed as baseline and week 4. Secondary outcomes included change from baseline in PASI and Investigator Global Assessment scores. The registry record lists three outcome measures, but it does not provide efficacy estimates or results for these outcomes. It also lists no analysis entries. The study therefore has a reported design and outcome framework, but no analyzable efficacy findings 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.
Safety Set
| Stage | AIN457 0.3 mg/kg | AIN457 1.0 mg/kg | AIN457 3.0 mg/kg | Placebo |
|---|---|---|---|---|
| STARTED | 2 | 4 | 4 | 5 |
| COMPLETED | 2 | 4 | 3 | 4 |
| NOT COMPLETED | 0 | 0 | 1 | 1 |
Reasons for not completing this period:
| Reason | AIN457 0.3 mg/kg | AIN457 1.0 mg/kg | AIN457 3.0 mg/kg | Placebo |
|---|---|---|---|---|
| Withdrawal by Subject | 0 | 0 | 0 | 1 |
| Protocol deviation | 0 | 0 | 1 | 0 |
Excluded Set
| Stage | AIN457 0.3 mg/kg | AIN457 1.0 mg/kg | AIN457 3.0 mg/kg | Placebo |
|---|---|---|---|---|
| STARTED | 18 | 16 | 16 | 15 |
| COMPLETED | 3 | 3 | 2 | 2 |
| NOT COMPLETED | 15 | 13 | 14 | 13 |
Reasons for not completing this period:
| Reason | AIN457 0.3 mg/kg | AIN457 1.0 mg/kg | AIN457 3.0 mg/kg | Placebo |
|---|---|---|---|---|
| Administrative problems | 14 | 12 | 12 | 10 |
| Withdrawal by Subject | 1 | 1 | 2 | 3 |
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 | AIN457 0.3 mg/kg | AIN457 1.0 mg/kg | AIN457 3.0 mg/kg | Placebo | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 2 | 4 | 4 | 5 | 15 | |
| Age, Continuous | Years | 53.5 (SD 9.19) | 53.3 (SD 11.79) | 51.5 (SD 8.35) | 52.4 (SD 14.05) | 52.5 (SD 10.38) |
| Sex: Female, Male: Female | Participants | 1 | 2 | 1 | 3 | 7 |
| Sex: Female, Male: Male | Participants | 1 | 2 | 3 | 2 | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Psoriasis Area and Severity Index (PASI) Scores, measured over baseline, week 4.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was change from baseline in Psoriasis Area and Severity Index (PASI) scores, assessed at baseline and week 4. PASI is a clinical measure used to characterize psoriasis severity across body regions, taking account of affected area and features of disease severity. In general, a change-from-baseline outcome describes how a participant's score at follow-up differs from the starting measurement. The registry record does not specify a unit, parameter, or measure of dispersion for this endpoint.
Change from baseline is used in trials to describe the direction and magnitude of change over a defined follow-up period. A comparison with a concurrent control group can help distinguish changes observed under treatment from changes that may also occur without the active intervention. That comparison depends on having usable outcome measurements and a prespecified analysis; the statistical plan is not reported in the registry record.
For this study, the listed assessment timeframe was baseline and week 4. The registry record states that the study was not powered for efficacy and that efficacy could not be analyzed after participants enrolled at one site were excluded from the planned efficacy analyses because of audit findings. It therefore supplies no primary endpoint estimate or comparison to interpret. The rationale for selecting this particular endpoint is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a randomized comparison of three single AIN457 dose levels with placebo, but it does not report an efficacy result. Its primary outcome was change from baseline in PASI scores at baseline and week 4. The record explicitly states that the study was not powered for efficacy and that efficacy could not be analyzed because participants from one site were excluded from planned efficacy analyses following audit findings, leaving an insufficient number of participants.
This means the registry record does not support an estimate of the difference in PASI change between any AIN457 group and placebo. It also does not report confidence intervals, p-values, or other inferential results for the primary endpoint. There is consequently no reported efficacy estimate whose size or precision can be assessed. The absence of an analyzable estimate is not evidence that treatment groups had the same outcome, nor does it establish that one dose differed from another.
The record lists change from baseline in PASI and Investigator Global Assessment scores among secondary outcomes, but it does not provide results for these outcomes. It therefore cannot answer whether the secondary measures changed, how they compared across groups, or how uncertain those comparisons might be. The number of analysis entries listed in the registry record is 0.
The exclusion described in the record is important to interpretation. Participants at the site were excluded from planned efficacy analyses because of audit findings, and the record says this left too few participants for efficacy analysis. It does not describe the audit findings in detail, the reason the site was audited, or whether safety summaries also excluded these participants. The listed adverse-event groups should therefore be read as reported safety results, not as a substitute for the missing efficacy analysis.
In general, randomized allocation can support a fair comparison when outcomes are collected and analyzed as planned. Here, the record's stated inability to analyze efficacy prevents that comparison from being evaluated. The safety counts describe adverse events recorded in treatment groups, but they do not establish an efficacy effect or resolve the missing efficacy information. Any stronger claim about benefit, lack of benefit, dose response, or the cause of the site exclusion would go beyond what the registry record reports.
9. Statistical Methods Explained
The available statistical information is limited. The registry record identifies the study as randomized, double-blind, parallel-group, and placebo-controlled, and it names the primary outcome and its assessment timeframe. It also states that the trial was not powered for efficacy. A detailed statistical analysis plan, including the methods intended for the primary comparison, is not reported in the registry record. No efficacy analysis entries are listed.
What does randomization contribute?
In general, randomization assigns participants to treatment groups using a chance-based process. When implemented and retained as planned, it helps reduce systematic differences between groups at the start of a trial. This supports interpretation of between-group differences as potentially related to assigned treatment rather than to measured or unmeasured baseline factors. Randomization does not guarantee that groups are identical, and it cannot compensate for outcomes that are unavailable or an analysis that cannot be conducted.
The registry record identifies randomization but does not describe how the allocation sequence was generated, how assignments were concealed, or whether any stratification was used. Those details are not reported in the registry record, so the specific protections against allocation bias cannot be assessed from this entry.
What does double-blind mean?
In general, double-blinding means that two relevant parties, often participants and investigators or assessors, do not know the assigned treatment. Masking can reduce the risk that expectations influence care, outcome assessment, or participant reporting. The registry record identifies the design as double-blind but does not specify which parties were masked, how masking was maintained, or whether the placebo was matched to the active treatment. These operational details are not reported in the registry record.
What does the parallel-group design compare?
In a parallel-group study, participants remain in their assigned group while outcomes are assessed. This trial had three AIN457 dose groups and a placebo group, with one intravenous administration on Day 1. A comparison among these groups could have addressed the primary PASI outcome and the listed secondary outcomes if analyzable measurements and an appropriate analysis were available. The registry record says the efficacy analysis could not be performed because of the site exclusion and insufficient participant numbers.
How should change from baseline be understood?
In general, a change-from-baseline analysis uses the starting score and a later score to describe how the outcome shifted during follow-up. A treatment comparison may analyze the change within each group, compare changes between groups, or adjust the follow-up score for the baseline value. These approaches have different assumptions and can yield different estimates. The registry record does not state which approach was planned for PASI, whether baseline adjustment was intended, or how repeated or incomplete measurements would be handled.
What does a confidence interval add?
In general, a confidence interval expresses the range of values reasonably compatible with an estimate under the statistical model and its assumptions. A narrower interval indicates greater precision, while a wider interval indicates greater uncertainty. No primary efficacy estimate or confidence interval is reported in the registry record, so precision for the intended PASI comparison cannot be evaluated.
Why does over-dispersion matter?
In general, over-dispersion occurs when data vary more than a statistical model assumes. If the model understates variability, standard errors and confidence intervals can be too small, making evidence appear more precise than it is. This issue is most relevant when an outcome's distribution and the model's assumptions are known. The registry record does not provide PASI results, model details, or a statistical analysis plan, so it is not possible to determine whether over-dispersion was relevant to the planned efficacy analysis or how it would have been addressed.
What can the sample-size illustrations tell readers?
Any sample-size illustrations associated with the posted results are planning calculations, not observed power and not evidence that the study was adequately powered. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. The record states that this study was not powered for efficacy. It does not report the original sample-size justification or the assumptions used to design the trial, and the absence of an analyzable efficacy estimate limits the use of observed-effect-based planning illustrations.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary PASI change-from-baseline outcome and secondary outcomes of change from baseline in PASI and Investigator Global Assessment scores. It does not report a statistical plan describing how these outcomes would be tested, how the three AIN457 dose groups would be compared with placebo, or how multiplicity would be controlled. Multiplicity arises in general when several hypotheses or outcomes are tested: without an appropriate strategy, the chance of at least one apparently positive finding can increase. No adjustment method or testing hierarchy is reported in the registry record.
Missing-data methods are also not reported in the registry record. The entry does not describe how absent week 4 measurements, withdrawals, or other incomplete outcome data would have been handled. It does state that participants enrolled at one site, which included 65 participants, were excluded from planned efficacy analyses because of audit findings. The record attributes the inability to analyze efficacy to this exclusion leaving an insufficient number of participants. It does not describe the audit findings further or explain how the exclusion affected safety analyses.
The analysis populations are not described in the registry record. It is therefore not possible to determine whether the planned efficacy analysis would have followed an intention-to-treat principle, used a per-protocol population, or applied another definition. The record's lack of an efficacy analysis means that neither the impact of the site exclusion nor the handling of missing observations can be evaluated through reported efficacy results.
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 |
|---|---|---|---|
| AIN457 0.3 mg/kg | 1 | 20 | 5.0% |
| AIN457 1 mg/kg | 1 | 20 | 5.0% |
| AIN457 3 mg/kg | 0 | 20 | 0.0% |
| Placebo | 0 | 20 | 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 |
|---|---|
| AIN457 0.3 mg/kg | 3 of 20 |
| AIN457 1 mg/kg | 4 of 20 |
| AIN457 3 mg/kg | 5 of 20 |
| Placebo | 3 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | AIN457 0.3 mg/kg | AIN457 1 mg/kg | AIN457 3 mg/kg | Placebo |
|---|---|---|---|---|---|
| FEMORAL HERNIA, OBSTRUCTIVE | Gastrointestinal disorders | 1 of 20 | 0 of 20 | 0 of 20 | 0 of 20 |
| HIP FRACTURE | Injury, poisoning and procedural complications | 0 of 20 | 1 of 20 | 0 of 20 | 0 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | AIN457 0.3 mg/kg | AIN457 1 mg/kg | AIN457 3 mg/kg | Placebo |
|---|---|---|---|---|---|
| HYPERTENSION | Vascular disorders | 2 of 20 | 1 of 20 | 1 of 20 | 0 of 20 |
| NASOPHARYNGITIS | Infections and infestations | 0 of 20 | 2 of 20 | 0 of 20 | 1 of 20 |
| SINUS CONGESTION | Respiratory, thoracic and mediastinal disorders | 1 of 20 | 2 of 20 | 0 of 20 | 0 of 20 |
| HEADACHE | Nervous system disorders | 0 of 20 | 1 of 20 | 0 of 20 | 1 of 20 |
| ABDOMINAL PAIN | Gastrointestinal disorders | 1 of 20 | 0 of 20 | 0 of 20 | 0 of 20 |
| NAUSEA | Gastrointestinal disorders | 1 of 20 | 0 of 20 | 0 of 20 | 0 of 20 |
| NECROTISING COLITIS | Gastrointestinal disorders | 1 of 20 | 0 of 20 | 0 of 20 | 0 of 20 |
| VOMITING | Gastrointestinal disorders | 1 of 20 | 0 of 20 | 0 of 20 | 0 of 20 |
| CYST | General disorders | 0 of 20 | 0 of 20 | 0 of 20 | 1 of 20 |
| INFLUENZA LIKE ILLNESS | General disorders | 0 of 20 | 0 of 20 | 1 of 20 | 0 of 20 |
Serious events with the highest arm-level rate: femoral hernia, obstructive (5.0%), hip fracture (5.0%).
The adverse-event results list serious adverse events and other adverse events separately for the three AIN457 groups and placebo. Each listed group has 20 participants at risk for these summaries. The number recorded as affected by a serious adverse event was 1 in the AIN457 0.3 mg/kg group, 1 in the AIN457 1 mg/kg group, 0 in the AIN457 3 mg/kg group, and 0 in the placebo group. These are group-level counts as reported; they do not establish a causal relationship to treatment.
The counts for participants affected by other adverse events were 3, 4, 5, and 3, respectively, across the listed groups. The registry record also lists femoral hernia, obstructive, and hip fracture as top serious-event terms, each at 5.0%. These terms are not linked to a specific treatment group in the listed information, so they should not be assigned to one.
Event counts are descriptive, and the record does not report a statistical comparison of safety outcomes. Deaths are not reported in the registry record. These safety summaries do not supply the missing efficacy analysis and should be interpreted separately from the PASI outcome.
12. What the Result Does, and Does Not, Mean
This registry record documents a completed Phase II study designed to compare three single intravenous doses of AIN457 with placebo in people with chronic plaque psoriasis. It identifies the intended primary outcome as change from baseline in PASI scores between baseline and week 4, and lists further outcomes involving PASI and Investigator Global Assessment scores.
What the record does not provide is an efficacy estimate. It states that the trial was not powered for efficacy and that efficacy could not be analyzed after participants at one site were excluded from planned efficacy analyses following audit findings. The exclusion left an insufficient number of participants. As a result, the posted results do not show how PASI changed in any group or whether the groups differed. They also do not report estimates of uncertainty for an efficacy comparison.
This lack of an analyzed result should not be mistaken for evidence that the treatments had identical effects, that an effect was absent, or that a particular dose was superior. Those conclusions require usable comparative outcome data and an analysis, neither of which is reported for efficacy in the registry record. The record also does not explain the audit findings, so their nature and implications cannot be inferred.
The adverse-event summaries provide descriptive counts by group, including serious-event counts and other adverse-event counts. They are separate from the efficacy question and do not establish treatment effects on PASI. The registry record does not report a statistical comparison of these safety outcomes.
Overall, the entry is informative about the trial's design, treatment groups, intended endpoints, the stated reason efficacy could not be analyzed, and selected adverse-event counts. It does not provide evidence that can quantify the efficacy comparison or its precision. Any interpretation beyond these reported details would require information not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates an important distinction between a trial's design and the evidence ultimately available for analysis. Randomization, masking, a placebo group, and a prespecified clinical outcome can provide a structure for a comparative study. But those design features do not yield an efficacy estimate when the planned analysis cannot be performed. The registry record states that participants at one site were excluded from planned efficacy analyses after audit findings and that the remaining number was insufficient for efficacy analysis.
That account makes analysis populations and data integrity relevant to statistical interpretation. Excluding a site can affect which participants contribute to an outcome analysis and can reduce the information available. The registry record does not provide details that would allow readers to evaluate the audit findings, the decision process for excluding the site, or the consequences for the composition of the analysis population. It also does not provide an alternative efficacy analysis.
The record further distinguishes an exploratory study from a study designed to establish efficacy: it describes the study as exploratory and says it was not powered for efficacy. A planning calculation based on an observed effect, if considered, is not a measure of observed power and cannot replace the study's own design justification. The original sample-size rationale is not reported in the registry record.
Statistically, the case highlights why reporting the outcome definitions, analysis populations, missing-data handling, and analytic methods matters alongside treatment-group counts. Without an efficacy estimate, interval, or analysis description, readers cannot assess the size or precision of the intended PASI comparison. The safety summaries remain descriptive and do not answer that efficacy question.
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 explicit in the registry record: efficacy could not be analyzed after all participants enrolled at one site were excluded from planned efficacy analyses because of audit findings. The record states that the site included 65 participants and that the resulting number of participants was insufficient for efficacy analysis. It does not describe the findings, their scope, or the rationale for excluding the site's participants in greater detail.
The record does not provide a primary efficacy estimate, a treatment-group comparison, a confidence interval, or a p-value. It also does not report the analysis population, statistical model, missing-data approach, multiplicity strategy, or detailed statistical analysis plan. Consequently, readers cannot assess how the intended comparisons would have been carried out or how uncertainty and incomplete observations would have been addressed.
The trial is described as exploratory and not powered for efficacy, but the original sample-size justification is not reported in the registry record. The listed secondary outcomes have no reported results. The entry also does not provide detailed participant characteristics, baseline PASI values, or the distribution of follow-up data by group.
Safety results are limited to the listed adverse-event counts and serious-event terms. The record does not report statistical comparisons for these outcomes, and deaths are not reported in the registry record. The reason for the site audit and any broader implications for the safety summaries are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Efficacy Study Looking at a Single-dose of One of Three Dose Levels of AIN457 in Patients With Chronic Plaque-type Psoriasis — NCT00770965.
- Registry record last updated 2018-03-12; status last verified 2018-02.
- Results first posted 2015-02-18.
- 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 record describes a completed Phase II randomized, double-blind, parallel-group, placebo-controlled study of one intravenous dose of AIN457 at 0.3 mg/kg, 1.0 mg/kg, or 3.0 mg/kg, compared with placebo, in people with chronic plaque psoriasis. Planned enrollment was 80. The primary outcome was change from baseline in PASI scores, with assessments listed at baseline and week 4.
The registry record says the study was not powered for efficacy. It reports that participants enrolled at one site were excluded from planned efficacy analyses after audit findings, leaving an insufficient number of participants; efficacy therefore could not be analyzed. No efficacy estimates are reported. Secondary outcomes included change from baseline in PASI and Investigator Global Assessment scores.
Adverse-event summaries list serious and other events by group. The record does not report the statistical analysis plan, analysis populations, missing-data methods, or multiplicity procedures.