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 Repeated Exposure to Eltrombopag in Adults With Idiopathic Thrombocytopenic Purpura (REPEAT) (NCT00424177), a non-randomized phase 2 study. The primary outcome is Number of Participants Who Responded (Platelet Count >=50 Gi/L and >=2x Baseline) to Eltrombopag Treatment in Cycle 2 or Cycle 3 Given Participants Responded in Cycle 1.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00424177 |
| Phase | Phase 2 |
| Condition | Purpura, Thrombocytopaenic, Idiopathic |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 66 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GlaxoSmithKline |
| Results posted | Yes |
2. Clinical Question
Population
Adults with chronic idiopathic thrombocytopenic purpura (ITP) were the intended study population. The primary analysis population was narrower: participants who entered the study, received at least one dose of eltrombopag, and responded in Cycle 1. The registry record reports enrollment of 66 participants, but it does not report the number who met all of these primary-analysis criteria.
Intervention
Participants received eltrombopag in an open-label, repeat, cyclic dosing study. The study description says it was intended to evaluate efficacy, safety, and tolerability with repeated intermittent dosing, including the durability of platelet-count responses over three cycles.
Comparator
The study used a single-group, non-randomized design. No separate comparator group is reported. As a result, its outcomes describe participants receiving eltrombopag in this study; they do not provide a randomized contrast with another treatment or with no treatment.
Primary question
Among participants who responded in Cycle 1, how many also responded to eltrombopag in Cycle 2 or Cycle 3? A response was defined as a platelet count of at least 50 Gi/L and at least twice baseline. The endpoint was assessed on Day 42 of each cycle. The registry record describes this question but does not report the observed primary endpoint count or the number of Cycle 1 responders included in that analysis.
3. Trial Design
REPEAT was a Phase 2, open-label study of repeat eltrombopag dosing in adults with chronic idiopathic thrombocytopenic purpura. It was non-randomized and used a single-group model, with 66 participants enrolled. The registry record lists the study as completed. It gives a study start of 2007-03 and a primary completion of 2008-08.
The study description emphasizes repeat, intermittent dosing across three cycles and assessment of platelet counts, response durability, safety, and tolerability. The primary endpoint focuses specifically on responses in Cycle 2 or Cycle 3 among participants who had responded in Cycle 1. This conditional population is important: the primary question is not whether all enrolled participants responded, but whether an initial response was seen again in a later cycle.
Because the study had no randomized comparator, differences over time cannot be separated from other influences using a between-group comparison. Its design is suited to describing repeated-cycle outcomes and observed safety events in participants taking eltrombopag, but it cannot by itself establish how those outcomes compare with an alternative treatment or a control condition.
The registry record does not report detailed dosing instructions, eligibility criteria beyond the adult chronic ITP description, allocation procedures beyond the non-randomized designation, or a fuller protocol. A rationale for the enrollment target and a detailed statistical analysis plan are not reported in the registry record. The record also does not report the number of participants who started each cycle or completed each assessment, apart from the safety-group counts.
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.
Cycle 1: Eltrombopag 50 mg Starting Dose
| Stage | Treatment Period |
|---|---|
| STARTED | 66 |
| COMPLETED | 55 |
| NOT COMPLETED | 11 |
Reasons for not completing this period:
| Reason | Treatment Period |
|---|---|
| Lack of Efficacy | 8 |
| Adverse Event | 1 |
| Prolonged Response | 1 |
| Relocation | 1 |
Cycle 2: Eltrombopag 50 or 75 mg
| Stage | Treatment Period |
|---|---|
| STARTED | 55 |
| COMPLETED | 51 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Treatment Period |
|---|---|
| Lack of Efficacy | 2 |
| Prolonged Response | 1 |
| Withdrawal by Subject | 1 |
Cycle 3: Eltrombopag 50 or 75 mg
| Stage | Treatment Period |
|---|---|
| STARTED | 51 |
| COMPLETED | 48 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Treatment Period |
|---|---|
| Lack of Efficacy | 1 |
| Withdrawal by Subject | 1 |
| Physician Decision | 1 |
Analysis population for the primary outcome, as defined in the registry: Primary Analysis Population: All participants who entered the study, received at least 1 dose of eltrombopag, and responded in Cycle 1
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 | Overall Study Population |
|---|---|---|
| Participants analysed | 66 | |
| Age Continuous | years | 49.6 (SD 14.61) |
| Sex: Female, Male: Female | Participants | 45 |
| Sex: Female, Male: Male | Participants | 21 |
| Race/Ethnicity, Customized: White - White/Caucasian/European | participants | 47 |
| Race/Ethnicity, Customized: Asian - East Asian | participants | 10 |
| Race/Ethnicity, Customized: White - Arabic/North African | participants | 6 |
| Race/Ethnicity, Customized: African American/African | participants | 2 |
| Race/Ethnicity, Customized: American Indian or Alaska Native | participants | 1 |
| Baseline Platelet Count: Less than 20 giga per liter (Gi/L) | participants | 4 |
| Baseline Platelet Count: 20 Gi/L to 30 Gi/L | participants | 29 |
| Baseline Platelet Count: Greater than 30 Gi/L to 50 Gi/L | participants | 31 |
| Baseline Platelet Count: Greater than 50 Gi/L | participants | 2 |
| Baseline Splenectomy Status: Yes | participants | 20 |
| Baseline Splenectomy Status: No | participants | 46 |
| Use of Idiopathic Thrombocytopenic Purpura Medication at Baseline: Yes | participants | 22 |
| Use of Idiopathic Thrombocytopenic Purpura Medication at Baseline: No | participants | 44 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants Who Responded (Platelet Count >=50 Gi/L and >=2x Baseline) to Eltrombopag Treatment in Cycle 2 or Cycle 3 Given Participants Responded in Cycle 1, measured over Day 42 of each cycle.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is a count of participants who responded in Cycle 2 or Cycle 3, among those who responded in Cycle 1. Response required both a platelet count of at least 50 Gi/L and a count at least twice the participant’s baseline. The assessment was scheduled for Day 42 of each cycle, with platelet count measured from a blood draw.
This endpoint combines an absolute platelet-count threshold with a comparison to baseline. In general, using both conditions defines a response more specifically than relying on either criterion alone. Restricting the primary analysis to initial responders makes the question one of repeat response or durability after an earlier response, rather than the chance of response among everyone enrolled.
The conditional definition also affects interpretation. The result, if reported, would apply to the Cycle 1 responder population meeting the stated treatment and entry criteria. It would not directly describe the response experience of all 66 enrolled participants. The registry record does not report the primary endpoint count, the number of Cycle 1 responders in the primary analysis population, or an uncertainty interval for the result. Therefore the magnitude and precision of the observed repeat-response outcome cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and design but does not report the primary endpoint result. It defines response by platelet count and specifies assessment on Day 42 of each cycle, yet gives no count of participants responding in Cycle 2 or Cycle 3 among Cycle 1 responders. The number of participants who entered that primary analysis is also not reported in the registry record. Without those results, the frequency of repeat response and its precision cannot be described.
The study’s single-group, non-randomized design places a limit on what any reported response count could establish. Such a count can describe what happened among participants treated in the study, but it does not estimate a randomized treatment contrast. Changes across cycles could be described, but without a comparator they cannot be attributed to eltrombopag relative to another treatment or an untreated course.
The endpoint is conditional on response during Cycle 1. That makes it relevant to the question of whether an initial response was seen again during later treatment, while also limiting its scope. It would not represent response among all enrolled participants, and interpretation would require knowing how many participants were eligible for the primary analysis and how many had outcome assessments. Those counts and the primary outcome result are not reported in the registry record.
The safety data include counts of participants affected by serious and other adverse events across treatment-cycle and post-dose periods. Across all cycles, the record lists 4 participants with serious adverse events and 55 with other adverse events, with 66 participants at risk in each category. By cycle, the serious-event counts are 0 in Cycle 1, 1 in Cycle 2, and 0 in Cycle 3; the corresponding counts for other adverse events are 39, 30, and 38. These are descriptive counts, not comparisons against a control group. The cycle-specific groups have different numbers at risk, and the registry record does not give event-level timing or an analysis comparing cycles.
In general, statistical precision concerns how much uncertainty remains around an estimate. A confidence interval can communicate that uncertainty, but the registry record does not report an interval for the primary endpoint. Nor does it report a primary endpoint estimate or a test result. Consequently, no conclusion about the estimated frequency or statistical evidence for repeat response can be drawn from the posted results described in the registry record.
Safety counts should likewise be read as observed reports within the recorded periods, not as proof that an event was caused by treatment. The registry record does not report deaths in the adverse-event fields, so the death count is not reported in the registry record; an unreported count should not be interpreted as zero.
9. Statistical Methods Explained
What does the endpoint count?
The primary endpoint is a binary response summarized as a number of participants. Each person either met the stated response definition at the relevant assessment or did not. The definition requires a platelet count of at least 50 Gi/L and at least twice baseline. The primary analysis is limited to people who entered the study, received at least one dose, and responded in Cycle 1. It then counts responses in Cycle 2 or Cycle 3, assessed on Day 42 of each cycle.
In general, a binary endpoint makes the outcome definition explicit, but a count alone is difficult to interpret without its denominator. For this endpoint, the relevant denominator would be the number of participants in the primary analysis population, not automatically the total enrollment. That analysis-population count is not reported in the registry record, nor is the endpoint count.
What does a single-group design allow?
In general, a single-group study describes outcomes in a group receiving the study intervention. It does not provide the randomized, concurrent comparison needed to estimate a treatment difference against a comparator group. This study is designated non-randomized and single-group, and no comparator arm is reported. Its repeated-cycle assessments can describe whether participants who responded initially were recorded as responding later, but they do not isolate the effect of treatment from other possible explanations for observed changes.
Open-label means the study is not described as masked. In general, lack of masking can affect outcomes that involve judgment or reporting. The primary response measure is based on a platelet count from a blood draw, but the registry record does not report further details about measurement procedures, laboratory masking, or quality-control methods. Those methods are therefore not reported in the registry record.
How should uncertainty be assessed?
In general, a point estimate gives a single summary of an outcome, while a confidence interval gives a range of values compatible with the data under the analysis assumptions. A narrower interval generally indicates greater precision; a wider interval indicates more uncertainty. The registry record does not report a primary endpoint estimate or confidence interval, so neither the estimated response frequency nor its precision can be evaluated.
For a count outcome, interpretation depends on the underlying denominator and the way uncertainty was calculated. The registry record does not report those details for the primary result. No p-value or other primary test result is reported in the registry record. It would be inappropriate to infer statistical significance from the endpoint definition or study design alone.
How do repeated cycles affect interpretation?
The endpoint concerns later responses among people who already responded in Cycle 1. This is a conditional analysis: eligibility for the primary analysis depends on an earlier outcome. In general, conditioning on an earlier response can make the analyzed group differ from all enrolled participants. The finding, if available, would answer a question about repeat response among initial responders, not the broader question of how often all participants responded.
Repeated measurements can also be related within a participant. In general, observations from the same person are not necessarily independent, and analyses of repeated outcomes may need to account for that dependence. The registry record does not report the statistical method used to handle repeated measurements, nor does it describe an adjustment for within-participant dependence.
What do the safety counts represent?
The adverse-event record gives counts of participants at risk and counts affected, separated into serious and other events for treatment cycles and periods after the last dose. These are descriptive summaries. In general, serious and non-serious events differ in their classification, and neither count alone establishes causation. The all-cycles entry is a summary category; it should not be combined arithmetically with cycle-specific entries, because the same participant may contribute across cycles.
The registry record lists serious-event terms with percentages, including Pneumonia at 1.8%, and Abdominal pain upper, Pancreatic carcinoma, Ear hemorrhage, Mouth Hemorrhage, and Epistaxis at 1.5% each. The record does not explain the denominator or provide event-level context alongside these terms, so those percentages should be read as registry-listed values rather than independently interpreted estimates.
What can planning calculations tell a reader?
In general, a sample-size calculation is a planning exercise based on assumptions about the outcome, the effect to detect, the significance level, allocation, and analysis method. It is not an observed result, and it is not observed power. Any planning illustration should not be confused with evidence from enrolled participants. The registry record does not report the study’s own sample-size justification or detailed planning rationale. It also does not report an interim-analysis plan, a prespecified multiplicity procedure, or enough primary outcome results to assess whether a planning calculation matches the study’s observed data.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary endpoint and secondary outcomes concerning response during treatment, platelet-count changes over three cycles, rescue medication, anti-platelet antibody levels, bleeding signs and symptoms, and additional outcome measures. Assessing several outcomes can raise a multiplicity question: in general, the more statistical tests performed, the greater the chance that at least one result may appear notable by chance. Whether and how to adjust for multiple testing depends on the analysis plan and the intended claims.
A prespecified multiplicity strategy, the number of formal hypothesis tests, and any hierarchy among outcomes are not reported in the registry record. The record does not provide enough analysis detail to determine which outcomes were treated as confirmatory or exploratory. No interim-analysis plan is reported in the registry record.
Missing-data handling is also not reported in the registry record. The primary endpoint depends on response in Cycle 1 and assessment in a later cycle; the record does not describe how participants with missing platelet counts, discontinuation, or incomplete cycle participation were handled. The number included in the primary analysis population is not reported, even though its eligibility criteria are stated. As a result, the extent and possible effect of missing outcome information cannot be assessed.
The primary analysis population is defined as all participants who entered the study, received at least one dose, and responded in Cycle 1. This definition is not the same as an analysis of all enrolled participants. The registry record does not report additional analysis populations, sensitivity analyses, or the number of participants meeting the primary-population criteria. These omissions limit assessment of how robust the primary result would be to alternative analysis choices.
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 |
|---|---|---|---|
| Cycle 1 | 0 | 66 | 0.0% |
| Cycle 2 | 1 | 55 | 1.8% |
| Cycle 3 | 0 | 51 | 0.0% |
| More Than 1 to 30 Days After Last Dose | 1 | 66 | 1.5% |
| More Than 30 Days After Last Dose | 1 | 66 | 1.5% |
| All Cycles | 4 | 66 | 6.1% |
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 |
|---|---|
| Cycle 1 | 39 of 66 |
| Cycle 2 | 30 of 55 |
| Cycle 3 | 38 of 51 |
| More Than 1 to 30 Days After Last Dose | 17 of 66 |
| More Than 30 Days After Last Dose | 7 of 66 |
| All Cycles | 55 of 66 |
Most frequent serious adverse event terms
| Event term | Organ system | Cycle 1 | Cycle 2 | Cycle 3 | More Than 1 to 30 Days After Last Dose | More Than 30 Days After Last Dose | All Cycles |
|---|---|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | 0 of 66 | 1 of 55 | 0 of 51 | 0 of 66 | 0 of 66 | 1 of 66 |
| Abdominal pain upper | Gastrointestinal disorders | 0 of 66 | 0 of 55 | 0 of 51 | 1 of 66 | 0 of 66 | 1 of 66 |
| Pancreatic carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 66 | 0 of 55 | 0 of 51 | 1 of 66 | 0 of 66 | 1 of 66 |
| Ear hemorrhage | Ear and labyrinth disorders | 0 of 66 | 0 of 55 | 0 of 51 | 0 of 66 | 1 of 66 | 1 of 66 |
| Mouth Hemorrhage | Gastrointestinal disorders | 0 of 66 | 0 of 55 | 0 of 51 | 0 of 66 | 1 of 66 | 1 of 66 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 0 of 66 | 0 of 55 | 0 of 51 | 0 of 66 | 1 of 66 | 1 of 66 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cycle 1 | Cycle 2 | Cycle 3 | More Than 1 to 30 Days After Last Dose | More Than 30 Days After Last Dose | All Cycles |
|---|---|---|---|---|---|---|---|
| Headache | Nervous system disorders | 9 of 66 | 10 of 55 | 7 of 51 | 5 of 66 | 0 of 66 | 16 of 66 |
| Nasopharyngitis | Infections and infestations | 5 of 66 | 2 of 55 | 5 of 51 | 3 of 66 | 1 of 66 | 10 of 66 |
| Fatigue | General disorders | 1 of 66 | 5 of 55 | 7 of 51 | 3 of 66 | 0 of 66 | 10 of 66 |
| Diarrhoea | Gastrointestinal disorders | 6 of 66 | 1 of 55 | 3 of 51 | 1 of 66 | 1 of 66 | 8 of 66 |
| Back pain | Musculoskeletal and connective tissue disorders | 3 of 66 | 4 of 55 | 1 of 51 | 0 of 66 | 0 of 66 | 7 of 66 |
| Upper respiratory tract infection | Infections and infestations | 2 of 66 | 1 of 55 | 3 of 51 | 3 of 66 | 0 of 66 | 5 of 66 |
| Nausea | Gastrointestinal disorders | 3 of 66 | 3 of 55 | 0 of 51 | 0 of 66 | 0 of 66 | 5 of 66 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 3 of 66 | 1 of 55 | 1 of 51 | 1 of 66 | 0 of 66 | 5 of 66 |
| Helicobacter pylori | Investigations | 0 of 66 | 0 of 55 | 3 of 51 | 2 of 66 | 3 of 66 | 3 of 66 |
| Vomiting | Gastrointestinal disorders | 2 of 66 | 2 of 55 | 1 of 51 | 0 of 66 | 0 of 66 | 5 of 66 |
Serious events with the highest arm-level rate: pneumonia (1.8%), abdominal pain upper (1.5%), pancreatic carcinoma (1.5%), ear hemorrhage (1.5%), mouth hemorrhage (1.5%).
For all cycles, the registry record lists 66 participants at risk for serious adverse events and 4 affected; for other adverse events, it lists 66 at risk and 55 affected. The cycle-specific entries report 66 at risk in Cycle 1, with 0 affected by serious events and 39 by other events; 55 at risk in Cycle 2, with 1 affected by serious events and 30 by other events; and 51 at risk in Cycle 3, with 0 affected by serious events and 38 by other events.
After the last dose, the record lists 1 participant with a serious adverse event in the period more than 1 to 30 days after the last dose, and 1 in the period more than 30 days after the last dose. It lists 17 and 7 participants, respectively, with other adverse events in those periods. Each post-dose entry lists 66 participants at risk.
These counts describe participants recorded in each period. They should not be added across periods: participants may appear in more than one period, and the all-cycles summary is not an additional treatment cycle. The registry record does not provide a death count; deaths are not reported in the registry record. The serious-event terms include registry-listed percentages, but their denominator and event-level context are not reported. These descriptive data do not establish that an event was caused by treatment or allow comparison with an unreported control group.
12. What the Result Does, and Does Not, Mean
The study addresses a specific statistical question: among participants who responded in Cycle 1, how many responded again in Cycle 2 or Cycle 3 under repeat eltrombopag dosing? It defines response using both a platelet-count threshold and a comparison with baseline, and it identifies Day 42 of each cycle as the assessment time. These features clarify what the primary endpoint was intended to measure.
However, the registry record does not report the primary response count, the number of Cycle 1 responders in the primary analysis, or an uncertainty interval. The observed magnitude and precision of the primary outcome therefore cannot be described. The endpoint definition alone does not show how often repeat response occurred.
The design also shapes the scope of any interpretation. A single-group, non-randomized study can describe observed outcomes in its participants, but it does not offer a randomized comparison with another treatment or a control condition. If platelet counts or responses changed across cycles, those observations would not on their own establish why the changes occurred. The primary analysis is additionally restricted to initial responders, so it would not directly describe outcomes for all enrolled participants.
The safety record reports participants affected by serious and other adverse events over treatment and post-dose periods. These counts can be read as descriptive observations in the recorded groups. They do not show comparative risk, establish causation, or supply the event-level context needed to evaluate individual reports. A death count is not reported in the registry record, which is distinct from a report of zero deaths.
Overall, the registry record supports a description of the trial’s intended question, design, and recorded safety counts. It does not provide the primary efficacy result or enough statistical detail to assess its precision, missing-data sensitivity, or multiplicity. It therefore cannot support a quantitative conclusion about the frequency of repeat response or a comparative claim about treatment effects.
13. Why This Trial Matters Statistically
This study is statistically notable for asking about repeat response rather than only initial response. Its primary endpoint conditions on a response in Cycle 1 and then considers response in Cycle 2 or Cycle 3. That distinction matters because the analyzed population is defined by an earlier outcome. The result, if reported, would describe repeat response among initial responders rather than response among all participants enrolled.
The trial also illustrates why a clear endpoint definition does not substitute for outcome reporting. The registry record specifies the platelet-count criteria, assessment day, and primary-analysis population, but it does not report the primary count or the denominator for that analysis. Without both, a reader cannot establish the observed frequency or judge its precision.
The non-randomized, single-group structure is another important statistical feature. Repeated observations can describe patterns within participants, but they do not create a concurrent control comparison. This distinction helps prevent descriptive changes from being mistaken for a comparative treatment effect. It also makes the absence of a reported comparator central to the scope of conclusions.
Finally, the adverse-event record separates serious and other events across cycles and periods after dosing. Those categories and periods support descriptive reading, but overlapping participants mean the entries are not independent totals to combine. The study’s value for statistical interpretation therefore lies in the questions it frames and the limitations made visible by its design and reporting, as well as in the safety counts that are recorded.
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 consequential limitation in the registry record is that the primary endpoint result is not reported. The response count, the number of Cycle 1 responders eligible for the primary analysis, and any uncertainty interval are absent. This prevents assessment of the endpoint’s observed frequency and precision.
The primary analysis population is conditional on having responded in Cycle 1. The record does not report how many participants met that condition or how many had outcome data at the later assessments. It also does not explain how missing platelet counts, discontinuation, or incomplete cycle participation were handled. The effect of missing outcome information therefore cannot be evaluated.
The single-group, non-randomized design has no reported comparator arm. Consequently, it cannot supply a randomized estimate of the difference between eltrombopag and another treatment or control condition. The open-label designation and the lack of further detail about measurement procedures also leave some aspects of outcome assessment unclear; the registry record does not report whether laboratory assessment was masked.
The registry record does not report a detailed statistical analysis plan, multiplicity procedure, interim-analysis plan, sample-size justification, or sensitivity analyses. It also does not provide detailed dosing information, event-level safety context, or a death count. The safety summaries are descriptive and include entries across multiple periods, so they should not be treated as independent groups or causal estimates. These gaps limit both efficacy interpretation and detailed assessment of safety reporting.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Repeated Exposure to Eltrombopag in Adults With Idiopathic Thrombocytopenic Purpura (REPEAT) — NCT00424177.
- Registry record last updated 2013-07-30; status last verified 2012-05.
- Results first posted 2009-08-11.
- 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
REPEAT (NCT00424177) was a completed Phase 2, open-label, non-randomized, single-group study of repeated eltrombopag dosing in adults with chronic idiopathic thrombocytopenic purpura. The record lists 66 enrolled participants and describes three cycles of intermittent treatment.
The primary endpoint counted participants responding in Cycle 2 or Cycle 3 among those who responded in Cycle 1. Response required a platelet count of at least 50 Gi/L and at least twice baseline, assessed on Day 42 of each cycle. The primary endpoint result and analysis-population count are not reported in the registry record.
Safety summaries list serious and other adverse events by cycle and after the last dose. These are descriptive counts, with no reported randomized comparator. The registry record does not report the primary result, detailed missing-data methods, a multiplicity plan, or a sample-size justification.