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 Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP) (NCT02201290), a registered phase 3 study. The primary outcome is Number of Participants With Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02201290 |
| Phase | Phase 3 |
| Condition | Purpura, Thrombocytopenic, Idiopathic |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned pediatric patients with chronic immune (idiopathic) thrombocytopenia purpura who had previously participated in study TRA115450. The primary outcome population is listed as all treated subjects. The registry lists 9 participants enrolled, and the safety counts list 9 participants at risk. Further eligibility criteria, including how chronic ITP was defined for this study, are not reported in the registry record.
Intervention
Participants received eltrombopag in an open-label extension. The registered intervention types are eltrombopag tablets and eltrombopag PfOS. Dosing was individualized for each subject based on platelet count; the starting dose was based on the subject’s dose at the end of TRA115450, and the maximum dose was 75 mg daily. A detailed dose-adjustment schedule is not reported in the registry record.
Comparator
No control treatment or comparator group is described. The record describes age-defined cohorts, rather than a randomized comparison: participants were to be allocated to one of three cohorts by age. Allocation details are not stated, and the record does not establish that the cohorts were intended to serve as comparisons with one another. Results therefore describe the treated group, not a relative treatment effect.
Primary question
The primary registered outcome asks how many participants experienced adverse events, including ophthalmic events, with events categorized using CTCAE toxicity grades and clinical laboratory testing also described as part of the safety and tolerability assessment. The stated timeframe is up to the week 4 follow-up period. The record says no statistical analysis was planned for this primary outcome. The central question is consequently descriptive: what safety events were recorded in this extension study?
3. Trial Design
This was an open-label Phase III extension study of eltrombopag in pediatric patients with chronic ITP who had previously participated in study TRA115450. The stated purpose was to evaluate long-term safety. The study is marked completed, and the registry lists an enrollment of 9. The recorded start date is 2013-06-18, and the primary completion date is 2017-07-04.
Eltrombopag dosing was individualized according to platelet count. The starting dose was based on the subject’s dose at the end of the preceding study, and the maximum dose was 75 mg daily. The registered intervention names are eltrombopag tablets and eltrombopag PfOS. The description says participants were to be allocated to one of three age-defined cohorts: 12 to 17 years, 6 to 11 years, and 1 to 5 years. The arm description indicates tablets for the first two cohorts and says participants in the second cohort may use eltrombopag pow; the remaining description is incomplete in the registry record. Further formulation and dosing details are not reported in the registry record.
There is no comparator arm described, and the study is explicitly open-label. Allocation is not stated as randomized. The age cohorts are a design feature, not a basis for assuming like-for-like comparisons. The primary outcome is the number of participants with adverse events, with the timeframe given as up to the week 4 follow-up period. The outcome description also refers to clinical laboratory assessments and grading of adverse events using CTCAE toxicity grades. No statistical analysis was planned for the primary outcome. Details of the follow-up schedule beyond the stated timeframe, the analysis plan for other outcomes, and any sample-size justification 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.
Overall Study
| Stage | Eltrombopag |
|---|---|
| STARTED | 9 |
| COMPLETED | 4 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | Eltrombopag |
|---|---|
| Withdrawal by Subject | 2 |
| Lack of Efficacy | 2 |
| Adverse Event | 1 |
Analysis population for the primary outcome, as defined in the registry: All treated subjects
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 | Eltrombopag |
|---|---|---|
| Participants analysed | 9 | |
| Age, Continuous | years | 9.7 (SD 3.81) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 5 |
| Race/Ethnicity, Customized: White - Caucasian | Participants | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events, measured over Up to week 4 follow up period.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary registered endpoint is the number of participants with adverse events. It is a count of people experiencing an event, not a count of all event episodes. The outcome description includes ophthalmic events and says adverse events are categorized using Common Terminology Criteria for Adverse Events (CTCAE) toxicity grades. It also names clinical laboratory assessments as part of the safety and tolerability endpoints. The registered population is all treated subjects, and the stated timeframe is up to the week 4 follow-up period.
As a general statistical point, a participant-level count provides a direct descriptive measure of how many people in the observed group experienced an event during the defined period. It does not, by itself, describe how often events recurred in a participant, how long they lasted, or whether an event was caused by treatment. Grading can add information about the recorded severity category, while laboratory assessments can identify safety findings that may not be captured by symptom reports alone. The registry record does not give a statistical analysis for this primary outcome; it says none was planned. Detailed laboratory results and a breakdown of all adverse events are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry’s primary outcome is descriptive: the number of participants with adverse events. The record explicitly states that no statistical analysis was planned for this outcome. Accordingly, the posted figures should be read as event counts in the study group, rather than as an estimate of a comparative treatment effect. There is no control group described, and the study was open-label. The age-defined cohorts are not identified as comparison groups.
For serious adverse events, 3 participants were affected among 9 at risk. For other adverse events, 6 participants were affected among 9 at risk. The record lists 0 deaths. These are participant counts; they do not state how many total event episodes occurred. It is also not reported whether participants who experienced serious adverse events were among those counted for other adverse events. The two categories should therefore not be added together to infer a total number of unique participants.
The three listed serious adverse-event terms are scleral haemorrhage, autoimmune hepatitis, and epistaxis, each reported at 11.1%. Those percentages are presented as term-specific registry values. The record does not provide further details about the individual events, their timing, duration, grade, outcome, or relationship to treatment. The three listed terms should not be assumed to account for every serious adverse event unless the registry record explicitly establishes that.
Precision is limited by the small number of participants at risk. In general, estimates from small groups can be sensitive to the experience of individual participants, and their uncertainty can be substantial. No confidence intervals or inferential tests for the primary outcome are reported in the registry record. No treatment-effect estimate can be derived from a single treated group without a comparator and an analysis designed for that purpose.
The endpoint timeframe is stated as up to the week 4 follow-up period, while the study is described as a long-term safety extension. The registry record does not clarify the relationship between that endpoint window and the full duration of each participant’s exposure or follow-up. These results therefore describe the recorded safety counts in the specified outcome population and timeframe; they do not, on their own, characterize all possible longer-term risks or establish comparative safety.
9. Statistical Methods Explained
The registered primary outcome is a count of participants with adverse events, and the record says no statistical analysis was planned for it. The posted safety results are therefore best understood as descriptive summaries. They report how many participants were at risk and how many were affected, without a model-based estimate or a hypothesis test for the primary outcome.
What does a participant count represent?
In general, a participant-level count answers whether each person experienced at least one qualifying event during the relevant observation period. A person with several events may still count once for an outcome defined as the number of participants with events. This differs from an event count, which can include repeated events in the same person. The registered endpoint title and unit refer to participants, so the reported number should not be read as the total number of event episodes.
The safety record lists 9 participants at risk, with 3 affected by serious adverse events and 6 affected by other adverse events. It also lists 0 deaths. These values are descriptive and do not indicate how events were distributed over time or how many events each affected participant experienced. The record does not report participant-level data, so overlap between the serious and other adverse-event counts cannot be determined from the summary.
Why does the study design matter?
In general, a randomized controlled comparison can support an estimate of the difference between assigned groups when the design and analysis are appropriate. This study is described as open-label and as an extension for participants from TRA115450. No control group is described, and the allocation method is not stated. The age-defined cohorts are not presented as treatment comparison arms. Consequently, the adverse-event counts describe the observed treated group; they do not estimate how event frequency would differ under another treatment or under no treatment.
Open-label means treatment assignment is not masked, according to the study description. As a general consideration, knowledge of treatment can affect reporting, assessment, or decisions to investigate symptoms. Clinical laboratory assessments and CTCAE grading can provide structured safety information, but the registry record does not provide details sufficient to assess how assessments were conducted or whether assessors were independent.
What do statistical uncertainty and significance mean?
As a general statistical explanation, a confidence interval gives a range of values compatible with the observed data under a specified method and assumptions. It communicates precision as well as the central estimate. A significance test evaluates data against a specified null hypothesis under a statistical model; a p-value is not the probability that a treatment works or that a finding is true. Neither confidence intervals nor a p-value for the primary outcome are reported in the registry record. The record states that no statistical analysis was planned for that endpoint, so inferential conclusions should not be retrofitted to the event counts.
Small groups generally yield less precise estimates than larger groups, because each participant represents a substantial part of the observed experience. This is a general property of statistical estimation, not an additional result from this study. The registry’s counts should be interpreted with that limitation in mind. The record does not provide a precision estimate for the event counts.
What does CTCAE grading add?
In general, a toxicity grading system classifies adverse events by severity according to standardized criteria. Such categories can make safety reporting more consistent and help distinguish events of different clinical intensity. The registry description says adverse events were categorized using CTCAE toxicity grades, but it does not provide the distribution of events across grades. A grade distribution, if available, would address a different question from the number of participants with any event.
The description also includes clinical laboratory assessments. Laboratory measures can be summarized as continuous values, categorical abnormalities, or changes over time, depending on the data and analysis. The registry record does not report the specific laboratory results, the rules for abnormality, or an analysis of change. No conclusions about those measures can be drawn from the participant counts alone.
Why are event categories and follow-up definitions important?
In general, a clearly defined observation window helps readers understand which events can contribute to an endpoint and how exposure time relates to the count. Here, the primary outcome timeframe is described as up to the week 4 follow-up period, while the overall study is described as a long-term safety extension. The record does not explain the relationship between these descriptions or report person-time. Without that information, the event counts cannot be interpreted as rates per unit of exposure, and comparisons with studies using different observation periods would require caution.
The record lists serious-event terms and term-specific percentages, but does not provide enough detail to reconstruct event histories or determine causality. In general, adverse-event reporting identifies events that occurred during a study; it does not automatically mean the study treatment caused them. Assessing causation requires context, including timing, alternative explanations, clinical assessment, and an appropriate comparative design. Those details are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record does not report a planned statistical analysis for the primary outcome; it explicitly says no statistical analysis was planned. It lists one outcome measure and no analysis entries. A multiplicity adjustment is therefore not described. In general, multiplicity concerns arise when several hypotheses or outcomes are tested, because repeated testing can increase the chance of at least one apparently notable result. That general issue should not be mistaken for evidence that a particular adjustment was required or omitted in this study.
Missing-data handling is not reported in the registry record. The record does not state whether any participants withdrew, whether outcome status was available for every treated subject, how incomplete follow-up was handled, or whether any imputation method was used. The safety group lists 9 participants at risk, but that count alone does not establish completeness of all assessments throughout the stated timeframe.
The primary outcome population is described as all treated subjects. More detailed analysis-population definitions are not reported in the registry record. It is not stated how participants were included if they had incomplete dosing, incomplete laboratory assessments, or incomplete event follow-up. No sensitivity analysis is reported.
These omissions limit assessment of how robust a formal statistical conclusion might be, but no formal primary-outcome analysis is reported. The available result should therefore be read as a descriptive safety account, with missing-data and multiplicity procedures not reported in the registry record.
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 |
|---|---|---|---|
| Eltrombopag | 3 | 9 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Eltrombopag | 0 of 9 |
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 |
|---|---|
| Eltrombopag | 6 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | Eltrombopag |
|---|---|---|
| Scleral haemorrhage | Eye disorders | 1 of 9 |
| Autoimmune hepatitis | Hepatobiliary disorders | 1 of 9 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 1 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Eltrombopag |
|---|---|---|
| Nasopharyngitis | Infections and infestations | 3 of 9 |
| Headache | Nervous system disorders | 2 of 9 |
| Pharyngitis | Infections and infestations | 1 of 9 |
| Tonsillitis | Infections and infestations | 1 of 9 |
| Blood bilirubin increased | Investigations | 1 of 9 |
| Iron deficiency | Metabolism and nutrition disorders | 1 of 9 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 1 of 9 |
Serious events with the highest arm-level rate: scleral haemorrhage (11.1%), autoimmune hepatitis (11.1%), epistaxis (11.1%).
The serious adverse-event summary lists 9 participants at risk and 3 affected. The record also lists 0 deaths. For other adverse events, 9 participants were at risk and 6 were affected. These are counts of participants, not necessarily counts of individual event episodes. The record does not say whether people could appear in both the serious and other adverse-event categories, so the counts should not be combined to infer the number of unique participants with any adverse event.
The registry lists scleral haemorrhage, autoimmune hepatitis, and epistaxis among the top serious adverse-event terms, each at 11.1%. Those percentages are term-specific values as reported. The registry record does not provide the event-level details needed to establish whether terms refer to separate participants, whether a participant had more than one term, or how each event was assessed.
As a general point, an adverse event is an event recorded during a study; its presence alone does not establish that treatment caused it. Seriousness, severity grade, and causality are distinct concepts. The outcome description refers to CTCAE toxicity grades, but the grade distribution and causal assessments are not reported in the registry record. The counts describe what was recorded in this small treated group, not a comparison with another treatment.
12. What the Result Does, and Does Not, Mean
The results describe safety events recorded in an open-label extension study of eltrombopag among pediatric patients with chronic ITP who had previously participated in TRA115450. The registry lists 9 participants enrolled and 9 participants at risk in the adverse-event summary. It reports 3 participants affected by serious adverse events, 6 affected by other adverse events, and 0 deaths. Three serious-event terms are listed, each at 11.1%.
These figures can describe the observed experience in this study group, but they do not establish how frequently events would occur in a different group or under a different treatment. No comparator is described, allocation is not stated as randomized, and the study is open-label. Age-defined cohorts do not function as a control group on the information reported. The registry also says that no statistical analysis was planned for the primary outcome, and it reports no confidence intervals or hypothesis tests for these counts.
As a general statistical principle, a small study can identify events that occurred among its participants, but it may be unable to characterize uncommon events precisely. Absence of a reported event in this group does not establish that the event cannot occur. Likewise, an event recorded during treatment does not on its own establish that treatment caused it. The registry record does not provide event-level timing, causality assessments, or enough detail to make those determinations.
The study is described as a long-term safety extension, while the primary outcome timeframe is stated as up to the week 4 follow-up period. The record does not clarify how that endpoint window relates to each participant’s overall exposure or follow-up. The reported counts should therefore be understood within the stated outcome population and timeframe, rather than as a complete estimate of long-term risk.
Overall, the posted results provide descriptive safety information from a small, treated group. They do not quantify a comparative treatment effect, establish causation, or support a conclusion about efficacy. Further details on event grades, laboratory findings, follow-up completeness, and analysis methods are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable for the distinction between a safety extension and a comparative treatment trial. Its stated purpose was long-term safety evaluation, and the registered primary outcome counts participants with adverse events. That is a meaningful descriptive question, but it is different from testing whether event risk differs between treatments. No control group is described, so a relative effect cannot be estimated from the posted counts.
The study also illustrates why endpoint definitions and timeframes matter. The outcome is a participant count, the event categories include serious and other adverse events, and the timeframe is described as up to the week 4 follow-up period. The overall study is called a long-term extension. Since the registry does not clarify how these time descriptions relate, readers should avoid treating the primary outcome as a complete account of every event throughout all exposure.
The group size is small: the adverse-event summary lists 9 participants at risk. In general, small groups can provide useful descriptions of observed events while leaving substantial uncertainty about the frequency of less common events. The registry does not report inferential estimates or a statistical analysis for the primary outcome, so the counts should not be presented as precise population-level estimates.
The record also highlights the value of distinguishing an event’s occurrence from its seriousness, grade, and causal relationship to treatment. CTCAE grading and laboratory assessments are named in the outcome description, but detailed grade and laboratory results are not reported. The available information therefore supports a limited, transparent description of recorded safety experience, with important questions about comparison, precision, and detail remaining unanswered.
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 small safety group is an important limitation: 9 participants were listed at risk. Counts from a group of this size provide limited information about less common events and cannot precisely characterize a broad range of possible safety outcomes. No confidence intervals or other estimates of precision are reported in the registry record.
The study is open-label and has no comparator group described. Allocation is not stated, and the age-defined cohorts should not be treated as like-for-like control groups. As a result, the event counts cannot separate treatment-associated effects from the underlying condition, other exposures, or other explanations. The registry record does not report causal assessments for the listed events.
The primary outcome timeframe is stated as up to the week 4 follow-up period, whereas the study is described as a long-term safety extension. The registry record does not explain how the outcome window relates to participants’ full treatment exposure or follow-up, or provide person-time. It is therefore not possible to interpret these counts as event rates over a clearly specified longer-term exposure period.
Further limitations arise from missing detail. The registry record does not report the event-grade distribution, complete clinical laboratory results, event timing and duration, participant-level overlap between categories, missing-data handling, or a detailed dose-adjustment schedule. The arm description is incomplete. The primary outcome has no planned statistical analysis, and other analysis details are not reported. These constraints call for a descriptive, rather than comparative or causal, reading of the posted safety counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP) — NCT02201290.
- Registry record last updated 2019-07-05; status last verified 2019-04.
- Results first posted 2019-07-05.
- 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
NCT02201290 is a completed, open-label Phase III extension study of eltrombopag in pediatric patients with chronic ITP who previously participated in TRA115450. The registry lists an enrollment of 9. Treatment was individualized according to platelet count, with the starting dose based on the dose at the end of the prior study and a maximum dose of 75 mg daily. Participants were described in age-defined cohorts.
The primary outcome was the number of participants with adverse events, including ophthalmic events, with CTCAE grading and clinical laboratory assessments described. The timeframe was up to the week 4 follow-up period, and the record says no statistical analysis was planned for this outcome. The safety summary lists 9 participants at risk, 3 with serious adverse events, 6 with other adverse events, and 0 deaths. Three serious-event terms are listed, each at 11.1%. The record provides descriptive counts, not a controlled estimate of comparative safety.