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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-002184-17 | EudraCT Number |
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The purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study. In part 1, patients will be randomized to receive either eltrombopag or placebo for 13 weeks. All patients who complete part 1 will enter part 2. In part 2, all patients will receive 24 weeks of eltrombopag.
This is a two part, double-blind, randomized, placebo-controlled and open-label Phase III study to investigate the efficacy, safety and tolerability of eltrombopag in pediatric patients with previously treated chronic ITP. In Part 1, patients will be randomized to receive eltrombopag or placebo in a 13-week double-blind, placebo-controlled treatment period. After completing Part 1, patients will begin Part 2, in which they will receive eltrombopag in an open-label manner during a 24-week treatment period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eltrombopag plus standard of care | Experimental | Part 1, double-blind treatment group |
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| Placebo plus standard of care | Placebo Comparator | Part 1, double-blind treatment group |
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| Eltrombopag plus standard of care (Part 2 open-label) | Experimental | Part 2, open-label |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eltrombopag | Drug | Thrombopoietin receptor agonist |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Achieving a Platelet Count >=50 Giga Cells Per Liter (Gi/L) for at Least 6 Out of 8 Weeks, Between Weeks 5 and 12 of Part 1 | Participants who achieved a platelet count >=50 Gi/L for at least 6 out of 8 weeks, between Weeks 5 and 12 of Part 1, were reported. | From Week 5 up to Week 12 of Part 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Responders | Percentage of participants who responded (defined as platelet count >= 50 Gi/L in absence of rescue) at least once up to week 12 of Part 1 (Odds of achieving a platelet count >=50 Gi/L during the first 12 weeks of Part 1) | From Week 1 up to Week 12 of Part 1 |
| Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 12 Weeks of Part 1 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| GSK Clinical Trials | GlaxoSmithKline | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| GSK Investigational Site | Los Angeles | California | 90027 | United States | ||
| GSK Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29536526 | Derived | Wire MB, Li X, Zhang J, Sallas W, Aslanis V, Ouatas T. Modeling and Simulation Support Eltrombopag Dosing in Pediatric Patients With Immune Thrombocytopenia. Clin Pharmacol Ther. 2018 Dec;104(6):1199-1207. doi: 10.1002/cpt.1066. Epub 2018 Apr 17. | |
| 26231455 | Derived | Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. doi: 10.1016/S0140-6736(15)61107-2. Epub 2015 Jul 28. |
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This study was comprised of a 13-week Double-Blind (DB), randomized Treatment Period (Part 1), followed by a 24-week Open-Label (OL) eltrombopag-only period (Part 2). After completion of Part 2, participants completed a 24- to 28-week Follow-up period, including an ophthalmic examination 24 weeks after the last dose of study treatment.
Pediatric participants meeting eligibility criteria were enrolled into 3 cohorts depending upon age. Cohort 1 enrolled participants who were between 12 and 17 years old, Cohort 2 enrolled participants who were between 6 and 11 years old, and Cohort 3 enrolled participants who were between 1 and 5 years old.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part 1 (Randomized Period)-Placebo | In Part 1, participants aged between 6 and 17 years with a body weight <27 kilograms (kg) received placebo 37.5 milligrams (mg) once daily (QD), and those with a body weight >=27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 milligrams per kilogram (mg/kg) QD; participants of East Asian ancestry received a starting dose of placebo 0.8 milligrams per kilograms per day (mg/kg/day). Standard of care treatments were allowed during the study, and were prescribed based on the investigator's discretion. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1 (Randomized Period) |
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| Placebo | Drug | Placebo with no active pharmaceutical ingredient |
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Participants who achieved a platelet count >=50 Gi/L at any time during the first 12 weeks of Part 1 were reported. |
| From Baseline up to Week 12 of Part 1 |
| Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 6 Weeks of Part 1 | Participants who achieved a platelet count >=50 Gi/L at any time during the first 6 weeks of Part 1 were reported. | From Baseline up to Week 6 of Part 1 |
| Weighted Mean Platelet Count | The weighted mean platelet count is defined as "the area under the platelet-time curve divided by the duration of the study (12 weeks)". Weighted mean platelet counts from baseline to week 12 of the randomized period was compared between placebo and eltrombopag using an analysis of covariance model (ANCOVA) adjusting for baseline platelet count and age cohort. For each subject the area between two adjacent visits with platelet counts was calculated. The area was calculated for all pairs of adjacent visits starting at Day 1 of randomized period and then the total sum of all the areas was divided by the total duration of time during the randomized period. For each subject, this method calculates an 'average' platelet count and it allows the possibility that subjects may have had different number of assessments during different times relative to baseline. | Baseline and Week 12 of Part 1 |
| Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During the First 12 Weeks of Part 1 | The maximum duration for which a participant continuously maintained a platelet count >=50 Gi/L was calculated and summarized for the first 12 weeks of Part 1. Participants with non-weekly assessments were assumed to have maintained a positive response for each week between two assessments that had positive responses. If a participant achieved a positive response at an assessment and then achieved a negative response at the next assessment, then it was assumed that the participant had achieved a positive response for one day. | From Baseline up to Week 12 of Part 1 |
| Number of Participants Who Required a Protocol-defined Rescue Treatment During Part 1 | Rescue treatment is defined as either a new immune (idiopathic) thrombocytopenic purpura (ITP) medication, an increase in the dose of a concomitant ITP medication from Baseline, a platelet transfusion, or a splenectomy. | From Baseline up to Week 12 of Part 1 |
| Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the World Health Organization (WHO) Bleeding Scale During Part 1 | The WHO Bleeding Scale is a measure of bleeding severity with the following grades: Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross bleeding; Grade 4=debilitating blood loss. The WHO grades were dichotomized into the following categories: no bleeding=Grade 0; any bleeding=Grades 1 to 4; no clinically significant bleeding=Grades 0 to 1; clinically significant bleeding=Grades 2 to 4. Baseline was defined as the Day 1 assessment or the latest possible screening assessment. | From Baseline through Follow-up of Part 1 |
| Number of Participants Who Achieved a Platelet Count >=50 Gi/L at Any Time During Part 2 | Participants who achieved a platelet count >=50 Gi/L at any time during Part 2 (up to Week 24) were reported. | From Baseline up to Week 24 of Part 2 |
| Number of Weeks in Which Participants Achieved a Platelet Count >=50 Gi/L, Between Weeks 4 and 24 of Part 2 | Platelet response was analyzed after Week 4 for the eltrombopag-only period to allow participants who had been randomized to placebo in the Randomized Period time to escalate to their optimal dose of eltrombopag. Participants with non-weekly assessments were assumed to have maintained a positive response for each week between two assessments that had positive responses. If the participant achieved a positive response at an assessment and then achieved a negative response at the next assessment, then it was assumed that the participant had achieved a positive response for one day. | From Week 4 up to Week 24 of Part 2 |
| Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During Part 2 | The maximum duration for which a participant continuously maintained a platelet count of >=50 Gi/L was calculated and summarized for the 24 weeks of eltrombopag dosing (Part 2). Participants with non-weekly assessments were assumed to have maintained a positive response for each week between two assessments that had positive responses. If the participant achieved a positive response at an assessment and then achieved a negative response at the next assessment, then it was assumed that the participant had achieved a positive response for one day. | From Baseline up to Week 24 of Part 2 |
| Number of Participants Who Reduced or Discontinued Baseline Concomitant ITP Medications During Part 2 Without Requiring Subsequent Rescue Therapy | Participants who discontinued or had a sustained reduction of a baseline immune (idiopathic) thrombocytopenic purpura (ITP) medication during the 24 weeks of Part 2 (Open-Label Period) and without requiring subsequent rescue therapy. For participants randomized to placebo in Part 1, Baseline is defined as Week 13 of Part 1. For participants randomized to eltrombopag in Part 1, Baseline is defined as Day 1 of Part 1. A sustained reduction was defined as a reduction for 4 weeks or more. | From Baseline up to Week 24 of Part 2 |
| Number of Participants Who Required a Protocol-defined Rescue Treatment During Part 2 | Rescue treatment was defined as either a new immune (idiopathic) thrombocytopenic purpura (ITP) medication, an increase in the dose of a concomitant ITP medication from Baseline, a platelet transfusion, or a splenectomy. | From Baseline up to Week 24 of Part 2 |
| Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the WHO Bleeding Scale During Part 2 | The WHO Bleeding Scale is a measure of bleeding severity with the following grades: Grade 0 = no bleeding, Grade 1 = petechiae, Grade 2 = mild blood loss, Grade 3 = gross bleeding and Grade 4 = debilitating blood loss. The WHO Grades were dichotomized into the following categories: no bleeding = Grade 0; any bleeding = Grade 1 to 4; no clinically significant bleeding = Grade 0 to 1; clinically significant bleeding = Grade 2 to 4. | From Baseline of Part 2 through Follow-up |
| Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) During Part 1 | An adverse event (AE) is defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability or incapacity, or is a congenital anomaly or birth defect. Medical or scientific judgment should be exercised in other situations. | From Day 1 of Treatment up to Week 13 of Part 1+ 1 day |
| Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) During Part 2 | An adverse event (AE) is defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: results in death, is life threatening; requires hospitalization or prolongation of existing hospitalization, results in disability or incapacity, or is a congenital anomaly or birth defect. Medical or scientific judgment should be exercised in other situations. | From Day 1 of Part 2 up to Week 24 of Part 2 + 1 day |
| Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During Part 1 | Clinical chemistry parameters were summarized according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.0: Grade 0 (G0), none; Grade 1 (G1), mild; Grade 2 (G2), moderate; Grade 3 (G3), severe; Grade 4 (G4), life-threatening or disabling. Clinical chemistry parameters included: aspartate amino transferase (AST), alkaline phosphatase (ALP), total bilirubin, albumin, alanine amino transferase (ALT), prothrombin international normalized ratio (PT INR), activated partial thromboplastin time (APTT), and creatinine. The Baseline value is defined as the value taken at Day 1 or, if missing, the latest non-missing Screening value. For serum creatinine, due to the variations in creatinine, the average of the Screening and the Day 1 values will be used as Baseline. The maximum post-Baseline toxicity grade includes any scheduled or unscheduled post-Baseline assessment during Part 1. | From Baseline up to Week 13 of Part 1 |
| Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During Part 2 | Clinical chemistry parameters were summarized according to the NCI CTCAE, version 4.0: G0, none; G1, mild; G2, moderate; G3, severe; G4, life-threatening or disabling. Clinical chemistry parameters included: AST, ALP, total bilirubin, albumin, ALT, and creatinine. For participants randomized to Placebo in Part 1, the BL value for Part 2 is defined as the value taken at Week 13 of Part 1. For serum creatinine, the value taken at Week 13 of Part 1 will be used as BL. For participants randomized to Eltrombopag in Part 1, the BL value is defined as the value taken on Day 1 or, if missing, the latest non-missing Screening value. For serum creatinine, due to the variations in creatinine, the average of the Screening and the Day 1 values will be used as BL. For participants who do not have both a Screening and Day 1 value, the Screening or Day 1 value will be used as BL. The maximum post-BL toxicity grade includes any scheduled or unscheduled post-BL assessment. | From Baseline (BL) of Part 2 through Follow-up |
| Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During Part 1 | Hematology parameters were summarized according to the NCI CTCAE, version 4.0: G0, none; G1, mild; G2, moderate; G3, severe; G4, life-threatening or disabling. Hematology parameters included: leukocytes, neutrophils, hemoglobin (increased), hemoglobin (anemia), lymphocytes (increased), and lymphocytes (decreased). The Baseline value is defined as the value taken at Day 1 or, if missing, the latest non-missing Screening value. The maximum post-Baseline toxicity grade includes any scheduled or unscheduled post-Baseline assessment during Part 1. | From Baseline up to Week 13 of Part 1 |
| Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During Part 2 | Hematology parameters were summarized according to the NCI CTCAE, version 4.0: G0, none, G1, mild; G2, moderate; G3, severe; G4, life-threatening or disabling. Hematology parameters included: leukocytes, neutrophils, hemoglobin (increased), hemoglobin (anemia), lymphocytes (increased), and lymphocytes (decreased). For participants randomized to Placebo in Part 1, the BL value for Part 2 is defined as the value taken at Week 13 of Part 1. For participants randomized to Eltrombopag in Part 1, the BL value is defined as the value taken on Day 1 or, if missing, the latest non-missing Screening value. For participants who do not have both a Screening and Day 1 value, the Screening or Day 1 value will be used as BL. The maximum post-BL toxicity grade includes any scheduled or unscheduled post-BL assessment. | From Baseline up to Week 24 of Part 2 and Follow-up Weeks 1 to 4 (up to Study Week 41) |
| Number of Participants With Vital Sign Data Falling Outside the Reference Ranges (RR) at the Indicated Visit During Part 1 | Vital sign measurements were taken before any blood draws and included systolic blood pressure(SBP), diastolic blood pressure(DBP), and heart rate(HR). The number of participants are reported with vital sign data falling outside the standard reference ranges RR as reference range high(RRH) and reference range low(RRL). The Baseline(BL) value is defined as the value taken at Day 1 or if missing, the latest non-missing SCR value. RR for Blood Pressure (mmHg) are read as: Lower Limit of Normal, Normal Range, Upper Limit of Normal. For Ages 1 to 5 years (yrs) ranges are SBP <85, 85 to 115, >115; DBP <45, 45 to70, >70. Ages 6 to 11 yrs: SBP <85, 85 to 120, >120;, DBP <50, 50 to 75, >75. Ages 12 to 17 yrs: SBP <95, 95 to 135, >135; DBP <55, 55 to 85, >85. RR for HR(bpm) are ages 1 to < 3 yrs: <90, 90 to 140, >140; ages 3 to < 5 yrs: <75, 75 to 130, >130, ages 5 to < 8yrs: <65, 65 to 115, >115; ages 8 to < 12yrs: <55, 55 to 110, >110; and ages 12 to 18 yrs: <55, 55 to 110, >110. | From Screening (SCR) up to Week 13 of Part 1 |
| Number of Participants With Vital Sign Data Falling Outside the Reference Ranges (RR) at the Indicated Visit During Part 2 | Vital sign measurements were taken before any blood draw and included systolic blood pressure (SBP), diastolic blood pressure (DBP), and heart rate(HR). The number of participants are reported with vital sign data falling outside the standard RR as reference range high(RRH) and reference range low(RRL) from SCR up to Week 24 of Part 2 and from Follow-up Week 1 to Week 4. RR for Blood Pressure(mmHg) are read as: Lower Limit of Normal, Normal Range, Upper Limit of Normal. For Ages 1 to 5 years (yrs) ranges are SBP <85, 85 to 115, >115; DBP <45, 45 to70, >70. Ages 6 to 11 yrs: SBP <85, 85 to 120, >120; DBP <50, 50 to 75, >75. Ages 12 to 17 yrs: SBP <95, 95 to 135, >135; DBP <55, 55 to 85, >85. RR for HR (bpm) are ages 1 to < 3 yrs: <90, 90 to 140, >140; ages 3 to < 5 yrs: <75, 75 to 130, >130, ages 5 to < 8yrs: <65, 65 to 115, >115; ages 8 to < 12yrs: <55, 55 to 110, >110; and ages 12 to 18 yrs: <55, 55 to 110, >110. | From Week 1 up to Week 24 of Part 2 and Follow-up Week 1 to Week 4 (up to Week 41) |
| Number of Participants With a Change in Visual Acuity Since Baseline at Week 12 of Part 1 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. Change in visual acuity results are presented as No (no change from Baseline), Not Clinically Significant (NCS), Improvement, and Worsening since Baseline. The Baseline value was obtained at the Screening Visit. | Baseline and Week 12 of Part 1 |
| Number of Participants With a Change in Visual Acuity Since Baseline at Week 24 of Part 2 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. Change in visual acuity results are presented as No Change, NCS, Improvement, and Worsening since Baseline. The Baseline value was obtained at the Screening Visit. | Baseline and Week 24 of Part 2 |
| Number of Participants With a Change in Visual Acuity Since Baseline at Follow-Up Week 24 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. Change in visual acuity results are presented as No Change, NCS, Improvement, and Worsening since Baseline. The Baseline value was obtained at the Screening Visit. | Baseline and Follow-Up Week 24 (Study Week 61) |
| Number of Participants With Worsening Visual Acuity Due to Cataracts at Week 12 of Part 1 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. The number of participants with worsening visual acuity due to cataracts at Week 12 of Part 1 are presented. Change due to cataracts is categorized as "Yes" or "No." | Baseline and Week 12 of Part 1 |
| Number of Participants With Worsening Visual Acuity Due to Cataracts at Week 24 of Part 2 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. The number of participants with worsening visual acuity due to cataracts at Week 24 of Part 2 are presented. Change due to cataracts is categorized as "Yes" or "No. | Baseline and Week 24 of Part 2 |
| Number of Participants With Worsening Visual Acuity Due to Cataracts at Follow-Up Week 24 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. The number of participants with worsening visual acuity due to cataracts at Follow-up Week 24 are presented. Change due to cataracts is categorized as "Yes" or "No." | Baseline and Follow-Up Week 24 (Week 61) |
| Pharmacokinetic (PK) Assessments for Eltrombopag for AUC (0-t) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. AUC(0-t) is defined as the area under the concentration-time curve over the dosing interval. The AUC(0-t) for a 50mg dose was estimated for each cohort. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort for a 50mg dose. | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
| Pharmacokinetic (PK) Assessments for Eltrombopag for Cmax | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. Cmax is defined as the maximum observed concentration. The Cmax for a 50mg dose was estimated for each cohort. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort for a 50mg dose. | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
| Pharmacokinetic (PK) Assessments for Eltrombopag for Apparent Oral Clearance (CL/F) and Apparent Intercompartmental Clearance (Q/F) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. CL/F is defined as the apparent oral clearance from plasma and Q/F is defined as apparent intercompartmental clearance. These parameters are dose independent. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort. | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
| PK Assessments for Eltrombopag for Apparent Central Volume (Vc/F) and Apparent Peripheral Volume (Vp/F) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. Vc/F is defined as the volume of the central (e.g. plasma) compartment and Vp/F is defined as the volume of the peripheral compartment. These parameters are dose independent. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort. | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
| Population PK Model Point Estimate for Eltrombopag for Absorption Rate-constant (Ka) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. Ka is defined as the absorption rate constant. This parameter is dose independent, and the population estimate Ka is reported. | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
| St. Petersburg |
| Florida |
| 33701 |
| United States |
| GSK Investigational Site | Brooklyn | New York | 11219 | United States |
| GSK Investigational Site | Salt Lake City | Utah | 84113 | United States |
| GSK Investigational Site | Capital Federal | Buenos Aires | 1425 | Argentina |
| GSK Investigational Site | Brno | 613 00 | Czechia |
| GSK Investigational Site | Olomouc | 775 20 | Czechia |
| GSK Investigational Site | Ostrava | 708 52 | Czechia |
| GSK Investigational Site | Prague | 154 00 | Czechia |
| GSK Investigational Site | Freiburg im Breisgau | Baden-Wurttemberg | 79106 | Germany |
| GSK Investigational Site | Mainz | Rhineland-Palatinate | 55131 | Germany |
| GSK Investigational Site | Berlin | State of Berlin | 13353 | Germany |
| GSK Investigational Site | Pokfulam | Hong Kong |
| GSK Investigational Site | Shatin | Hong Kong |
| GSK Investigational Site | Beersheba | 84101 | Israel |
| GSK Investigational Site | Haifa | 31048 | Israel |
| GSK Investigational Site | Petah Tikva | 49202 | Israel |
| GSK Investigational Site | Ramat Gan | 52621 | Israel |
| GSK Investigational Site | Rehovot | 76100 | Israel |
| GSK Investigational Site | Tel Aviv | 64239 | Israel |
| GSK Investigational Site | Bologna | Emilia-Romagna | 40138 | Italy |
| GSK Investigational Site | Rome | Lazio | 00165 | Italy |
| GSK Investigational Site | Monza | Lombardy | 20900 | Italy |
| GSK Investigational Site | Turin | Piedmont | 10126 | Italy |
| GSK Investigational Site | Bydgoszcz | Poland |
| GSK Investigational Site | Gdansk | 80-952 | Poland |
| GSK Investigational Site | Lodz | 91-738 | Poland |
| GSK Investigational Site | Lublin | 20-093 | Poland |
| GSK Investigational Site | Krasnodar | 350007 | Russia |
| GSK Investigational Site | Moscow | 105077 | Russia |
| GSK Investigational Site | Moscow | 117198 | Russia |
| GSK Investigational Site | Saint Petersburg | 198205 | Russia |
| GSK Investigational Site | Barcelona | 08035 | Spain |
| GSK Investigational Site | Madrid | 28007 | Spain |
| GSK Investigational Site | Madrid | Spain |
| GSK Investigational Site | Málaga | 29011 | Spain |
| GSK Investigational Site | Murcia (El Palmar) | 30120 | Spain |
| GSK Investigational Site | Pamplona | 31008 | Spain |
| GSK Investigational Site | Valencia | 46026 | Spain |
| GSK Investigational Site | Tainan | 704 | Taiwan |
| GSK Investigational Site | Taoyuan | 333 | Taiwan |
| GSK Investigational Site | Bangkok | 10330 | Thailand |
| GSK Investigational Site | Bangkok | Thailand |
| GSK Investigational Site | Khon Kaen | 40002 | Thailand |
| GSK Investigational Site | Songkhla | 90110 | Thailand |
| GSK Investigational Site | Birmingham | B4 6NH | United Kingdom |
| GSK Investigational Site | Cardiff | CF14 4XW | United Kingdom |
| GSK Investigational Site | London | SW17 0QT | United Kingdom |
| GSK Investigational Site | Manchester | M13 9WL | United Kingdom |
| GSK Investigational Site | Romford | RM7 0AG | United Kingdom |
| GSK Investigational Site | Sheffield | S10 2TH | United Kingdom |
| FG001 | Part 1 (Randomized Period)-Eltrombopag | In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. Participants continued on the same dose of eltrombopag in Part 2 unless adjustments were warranted according to the dosing guidelines. Standard of care treatments were allowed during the study, and were prescribed based on the investigator's discretion. |
| FG002 | Part 2 (Open-Label Period) Eltrombopag | All participants receiving placebo in Part 1 received eltrombopag in Part 2 following starting dose guidelines for Part 1. Participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. Participants receiving eltrombopag in Part 1 continued on the same dose of eltrombopag in Part 2 unless adjustments were warranted according to the dosing guidelines. Standard of care treatments were allowed during the study, and were prescribed based on the investigator's discretion. |
| COMPLETED |
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| NOT COMPLETED |
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| Part 2 (Open-Label Period)-Eltrombopag |
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Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received placebo 37.5 mg QD, and those with a body weight >=27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of placebo 0.8 mg/kg/day. In Part 2, participants received eltrombopag. Participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
| BG001 | Eltrombopag | In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. Participants continued on the same dose of eltrombopag in Part 2 unless adjustments were warranted according to the dosing guidelines. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Number of Participants Achieving a Platelet Count >=50 Giga Cells Per Liter (Gi/L) for at Least 6 Out of 8 Weeks, Between Weeks 5 and 12 of Part 1 | Participants who achieved a platelet count >=50 Gi/L for at least 6 out of 8 weeks, between Weeks 5 and 12 of Part 1, were reported. | Intent-to-Treat (ITT) Population: all randomized participants. The ITT Population was the primary population used for assessing efficacy. | Posted | Number | Participants | From Week 5 up to Week 12 of Part 1 |
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| Secondary | Percentage of Responders | Percentage of participants who responded (defined as platelet count >= 50 Gi/L in absence of rescue) at least once up to week 12 of Part 1 (Odds of achieving a platelet count >=50 Gi/L during the first 12 weeks of Part 1) | ITT Population | Posted | Number | Percentage of participants | From Week 1 up to Week 12 of Part 1 |
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| Secondary | Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 12 Weeks of Part 1 | Participants who achieved a platelet count >=50 Gi/L at any time during the first 12 weeks of Part 1 were reported. | ITT Population | Posted | Number | Participants | From Baseline up to Week 12 of Part 1 |
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| Secondary | Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 6 Weeks of Part 1 | Participants who achieved a platelet count >=50 Gi/L at any time during the first 6 weeks of Part 1 were reported. | ITT Population | Posted | Number | Participants | From Baseline up to Week 6 of Part 1 |
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| Secondary | Weighted Mean Platelet Count | The weighted mean platelet count is defined as "the area under the platelet-time curve divided by the duration of the study (12 weeks)". Weighted mean platelet counts from baseline to week 12 of the randomized period was compared between placebo and eltrombopag using an analysis of covariance model (ANCOVA) adjusting for baseline platelet count and age cohort. For each subject the area between two adjacent visits with platelet counts was calculated. The area was calculated for all pairs of adjacent visits starting at Day 1 of randomized period and then the total sum of all the areas was divided by the total duration of time during the randomized period. For each subject, this method calculates an 'average' platelet count and it allows the possibility that subjects may have had different number of assessments during different times relative to baseline. | ITT Population. Only those participants with a value at Baseline and post-Baseline were analyzed. | Posted | Mean | Standard Deviation | Gi/L | Baseline and Week 12 of Part 1 |
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| Secondary | Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During the First 12 Weeks of Part 1 | The maximum duration for which a participant continuously maintained a platelet count >=50 Gi/L was calculated and summarized for the first 12 weeks of Part 1. Participants with non-weekly assessments were assumed to have maintained a positive response for each week between two assessments that had positive responses. If a participant achieved a positive response at an assessment and then achieved a negative response at the next assessment, then it was assumed that the participant had achieved a positive response for one day. | ITT Population | Posted | Mean | Standard Deviation | Weeks | From Baseline up to Week 12 of Part 1 |
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| Secondary | Number of Participants Who Required a Protocol-defined Rescue Treatment During Part 1 | Rescue treatment is defined as either a new immune (idiopathic) thrombocytopenic purpura (ITP) medication, an increase in the dose of a concomitant ITP medication from Baseline, a platelet transfusion, or a splenectomy. | ITT Population | Posted | Number | Participants | From Baseline up to Week 12 of Part 1 |
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| Secondary | Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the World Health Organization (WHO) Bleeding Scale During Part 1 | The WHO Bleeding Scale is a measure of bleeding severity with the following grades: Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross bleeding; Grade 4=debilitating blood loss. The WHO grades were dichotomized into the following categories: no bleeding=Grade 0; any bleeding=Grades 1 to 4; no clinically significant bleeding=Grades 0 to 1; clinically significant bleeding=Grades 2 to 4. Baseline was defined as the Day 1 assessment or the latest possible screening assessment. | ITT Population. Only those participants that did not enroll in Part 2 were analyzed during the follow-up visits. Only those participants available at the specified time points were analyzed (represented by n=X,X in the category titles). | Posted | Number | Participants | From Baseline through Follow-up of Part 1 |
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| Secondary | Number of Participants Who Achieved a Platelet Count >=50 Gi/L at Any Time During Part 2 | Participants who achieved a platelet count >=50 Gi/L at any time during Part 2 (up to Week 24) were reported. | ITT Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Number | Participants | From Baseline up to Week 24 of Part 2 |
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| Secondary | Number of Weeks in Which Participants Achieved a Platelet Count >=50 Gi/L, Between Weeks 4 and 24 of Part 2 | Platelet response was analyzed after Week 4 for the eltrombopag-only period to allow participants who had been randomized to placebo in the Randomized Period time to escalate to their optimal dose of eltrombopag. Participants with non-weekly assessments were assumed to have maintained a positive response for each week between two assessments that had positive responses. If the participant achieved a positive response at an assessment and then achieved a negative response at the next assessment, then it was assumed that the participant had achieved a positive response for one day. | ITT Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Mean | Standard Deviation | Weeks | From Week 4 up to Week 24 of Part 2 |
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| Secondary | Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During Part 2 | The maximum duration for which a participant continuously maintained a platelet count of >=50 Gi/L was calculated and summarized for the 24 weeks of eltrombopag dosing (Part 2). Participants with non-weekly assessments were assumed to have maintained a positive response for each week between two assessments that had positive responses. If the participant achieved a positive response at an assessment and then achieved a negative response at the next assessment, then it was assumed that the participant had achieved a positive response for one day. | ITT Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Mean | Standard Deviation | Weeks | From Baseline up to Week 24 of Part 2 |
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| Secondary | Number of Participants Who Reduced or Discontinued Baseline Concomitant ITP Medications During Part 2 Without Requiring Subsequent Rescue Therapy | Participants who discontinued or had a sustained reduction of a baseline immune (idiopathic) thrombocytopenic purpura (ITP) medication during the 24 weeks of Part 2 (Open-Label Period) and without requiring subsequent rescue therapy. For participants randomized to placebo in Part 1, Baseline is defined as Week 13 of Part 1. For participants randomized to eltrombopag in Part 1, Baseline is defined as Day 1 of Part 1. A sustained reduction was defined as a reduction for 4 weeks or more. | ITT Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) and taking an ITP medication at Baseline were analyzed. | Posted | Number | Participants | From Baseline up to Week 24 of Part 2 |
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| Secondary | Number of Participants Who Required a Protocol-defined Rescue Treatment During Part 2 | Rescue treatment was defined as either a new immune (idiopathic) thrombocytopenic purpura (ITP) medication, an increase in the dose of a concomitant ITP medication from Baseline, a platelet transfusion, or a splenectomy. | ITT Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Number | Participants | From Baseline up to Week 24 of Part 2 |
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| Secondary | Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the WHO Bleeding Scale During Part 2 | The WHO Bleeding Scale is a measure of bleeding severity with the following grades: Grade 0 = no bleeding, Grade 1 = petechiae, Grade 2 = mild blood loss, Grade 3 = gross bleeding and Grade 4 = debilitating blood loss. The WHO Grades were dichotomized into the following categories: no bleeding = Grade 0; any bleeding = Grade 1 to 4; no clinically significant bleeding = Grade 0 to 1; clinically significant bleeding = Grade 2 to 4. | ITT Population. Only those participants who entered into Part 2 open-label Eltrombopag only phase were analyzed. Only those participants available at the specified time points were analyzed (represented by n=X in the category titles). | Posted | Number | Participants | From Baseline of Part 2 through Follow-up |
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| Secondary | Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) During Part 1 | An adverse event (AE) is defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability or incapacity, or is a congenital anomaly or birth defect. Medical or scientific judgment should be exercised in other situations. | Safety Population: all participants who received at least one dose of the investigational product | Posted | Number | Participants | From Day 1 of Treatment up to Week 13 of Part 1+ 1 day |
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| Secondary | Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) During Part 2 | An adverse event (AE) is defined as any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: results in death, is life threatening; requires hospitalization or prolongation of existing hospitalization, results in disability or incapacity, or is a congenital anomaly or birth defect. Medical or scientific judgment should be exercised in other situations. | Safety Population | Posted | Number | Participants | From Day 1 of Part 2 up to Week 24 of Part 2 + 1 day |
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| Secondary | Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During Part 1 | Clinical chemistry parameters were summarized according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.0: Grade 0 (G0), none; Grade 1 (G1), mild; Grade 2 (G2), moderate; Grade 3 (G3), severe; Grade 4 (G4), life-threatening or disabling. Clinical chemistry parameters included: aspartate amino transferase (AST), alkaline phosphatase (ALP), total bilirubin, albumin, alanine amino transferase (ALT), prothrombin international normalized ratio (PT INR), activated partial thromboplastin time (APTT), and creatinine. The Baseline value is defined as the value taken at Day 1 or, if missing, the latest non-missing Screening value. For serum creatinine, due to the variations in creatinine, the average of the Screening and the Day 1 values will be used as Baseline. The maximum post-Baseline toxicity grade includes any scheduled or unscheduled post-Baseline assessment during Part 1. | Safety Population. Only those participants available at the specified time points were analyzed (represented by n=X,X in the category titles). | Posted | Number | Participants | From Baseline up to Week 13 of Part 1 |
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| Secondary | Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During Part 2 | Clinical chemistry parameters were summarized according to the NCI CTCAE, version 4.0: G0, none; G1, mild; G2, moderate; G3, severe; G4, life-threatening or disabling. Clinical chemistry parameters included: AST, ALP, total bilirubin, albumin, ALT, and creatinine. For participants randomized to Placebo in Part 1, the BL value for Part 2 is defined as the value taken at Week 13 of Part 1. For serum creatinine, the value taken at Week 13 of Part 1 will be used as BL. For participants randomized to Eltrombopag in Part 1, the BL value is defined as the value taken on Day 1 or, if missing, the latest non-missing Screening value. For serum creatinine, due to the variations in creatinine, the average of the Screening and the Day 1 values will be used as BL. For participants who do not have both a Screening and Day 1 value, the Screening or Day 1 value will be used as BL. The maximum post-BL toxicity grade includes any scheduled or unscheduled post-BL assessment. | Safety Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Number | Participants | From Baseline (BL) of Part 2 through Follow-up |
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| Secondary | Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During Part 1 | Hematology parameters were summarized according to the NCI CTCAE, version 4.0: G0, none; G1, mild; G2, moderate; G3, severe; G4, life-threatening or disabling. Hematology parameters included: leukocytes, neutrophils, hemoglobin (increased), hemoglobin (anemia), lymphocytes (increased), and lymphocytes (decreased). The Baseline value is defined as the value taken at Day 1 or, if missing, the latest non-missing Screening value. The maximum post-Baseline toxicity grade includes any scheduled or unscheduled post-Baseline assessment during Part 1. | Safety Population. Only those participants available at the specified time points were analyzed (represented by n=X,X in the category titles). | Posted | Number | Participants | From Baseline up to Week 13 of Part 1 |
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| Secondary | Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During Part 2 | Hematology parameters were summarized according to the NCI CTCAE, version 4.0: G0, none, G1, mild; G2, moderate; G3, severe; G4, life-threatening or disabling. Hematology parameters included: leukocytes, neutrophils, hemoglobin (increased), hemoglobin (anemia), lymphocytes (increased), and lymphocytes (decreased). For participants randomized to Placebo in Part 1, the BL value for Part 2 is defined as the value taken at Week 13 of Part 1. For participants randomized to Eltrombopag in Part 1, the BL value is defined as the value taken on Day 1 or, if missing, the latest non-missing Screening value. For participants who do not have both a Screening and Day 1 value, the Screening or Day 1 value will be used as BL. The maximum post-BL toxicity grade includes any scheduled or unscheduled post-BL assessment. | Safety Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Number | Participants | From Baseline up to Week 24 of Part 2 and Follow-up Weeks 1 to 4 (up to Study Week 41) |
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| Secondary | Number of Participants With Vital Sign Data Falling Outside the Reference Ranges (RR) at the Indicated Visit During Part 1 | Vital sign measurements were taken before any blood draws and included systolic blood pressure(SBP), diastolic blood pressure(DBP), and heart rate(HR). The number of participants are reported with vital sign data falling outside the standard reference ranges RR as reference range high(RRH) and reference range low(RRL). The Baseline(BL) value is defined as the value taken at Day 1 or if missing, the latest non-missing SCR value. RR for Blood Pressure (mmHg) are read as: Lower Limit of Normal, Normal Range, Upper Limit of Normal. For Ages 1 to 5 years (yrs) ranges are SBP <85, 85 to 115, >115; DBP <45, 45 to70, >70. Ages 6 to 11 yrs: SBP <85, 85 to 120, >120;, DBP <50, 50 to 75, >75. Ages 12 to 17 yrs: SBP <95, 95 to 135, >135; DBP <55, 55 to 85, >85. RR for HR(bpm) are ages 1 to < 3 yrs: <90, 90 to 140, >140; ages 3 to < 5 yrs: <75, 75 to 130, >130, ages 5 to < 8yrs: <65, 65 to 115, >115; ages 8 to < 12yrs: <55, 55 to 110, >110; and ages 12 to 18 yrs: <55, 55 to 110, >110. | Safety Population. Only those participants available at the specified time points were analyzed (represented by n=X,X in the category titles). | Posted | Number | Participants | From Screening (SCR) up to Week 13 of Part 1 |
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| Secondary | Number of Participants With Vital Sign Data Falling Outside the Reference Ranges (RR) at the Indicated Visit During Part 2 | Vital sign measurements were taken before any blood draw and included systolic blood pressure (SBP), diastolic blood pressure (DBP), and heart rate(HR). The number of participants are reported with vital sign data falling outside the standard RR as reference range high(RRH) and reference range low(RRL) from SCR up to Week 24 of Part 2 and from Follow-up Week 1 to Week 4. RR for Blood Pressure(mmHg) are read as: Lower Limit of Normal, Normal Range, Upper Limit of Normal. For Ages 1 to 5 years (yrs) ranges are SBP <85, 85 to 115, >115; DBP <45, 45 to70, >70. Ages 6 to 11 yrs: SBP <85, 85 to 120, >120; DBP <50, 50 to 75, >75. Ages 12 to 17 yrs: SBP <95, 95 to 135, >135; DBP <55, 55 to 85, >85. RR for HR (bpm) are ages 1 to < 3 yrs: <90, 90 to 140, >140; ages 3 to < 5 yrs: <75, 75 to 130, >130, ages 5 to < 8yrs: <65, 65 to 115, >115; ages 8 to < 12yrs: <55, 55 to 110, >110; and ages 12 to 18 yrs: <55, 55 to 110, >110. | Safety Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. Only those participants available at the specified time points were analyzed (represented by n=X in the category titles). | Posted | Number | Participants | From Week 1 up to Week 24 of Part 2 and Follow-up Week 1 to Week 4 (up to Week 41) |
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| Secondary | Number of Participants With a Change in Visual Acuity Since Baseline at Week 12 of Part 1 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. Change in visual acuity results are presented as No (no change from Baseline), Not Clinically Significant (NCS), Improvement, and Worsening since Baseline. The Baseline value was obtained at the Screening Visit. | Safety Population | Posted | Number | Participants | Baseline and Week 12 of Part 1 |
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| Secondary | Number of Participants With a Change in Visual Acuity Since Baseline at Week 24 of Part 2 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. Change in visual acuity results are presented as No Change, NCS, Improvement, and Worsening since Baseline. The Baseline value was obtained at the Screening Visit. | Safety Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Number | Participants | Baseline and Week 24 of Part 2 |
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| Secondary | Number of Participants With a Change in Visual Acuity Since Baseline at Follow-Up Week 24 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. Change in visual acuity results are presented as No Change, NCS, Improvement, and Worsening since Baseline. The Baseline value was obtained at the Screening Visit. | Safety Population. Only those participants who entered into Part 2 (Open-label eltrombopag-only phase) were analyzed. | Posted | Number | Participants | Baseline and Follow-Up Week 24 (Study Week 61) |
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| Secondary | Number of Participants With Worsening Visual Acuity Due to Cataracts at Week 12 of Part 1 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. The number of participants with worsening visual acuity due to cataracts at Week 12 of Part 1 are presented. Change due to cataracts is categorized as "Yes" or "No." | Safety Population. Only those participants who had a result of 'worsening' in assessment of change of visual acuity at this timepoint were analyzed. | Posted | Number | Participants | Baseline and Week 12 of Part 1 |
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| Secondary | Number of Participants With Worsening Visual Acuity Due to Cataracts at Week 24 of Part 2 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. The number of participants with worsening visual acuity due to cataracts at Week 24 of Part 2 are presented. Change due to cataracts is categorized as "Yes" or "No. | Safety Population. Only those participants who had worsening visual acuity at Week 24 were analyzed. | Posted | Number | Participants | Baseline and Week 24 of Part 2 |
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| Secondary | Number of Participants With Worsening Visual Acuity Due to Cataracts at Follow-Up Week 24 | The visual acuity assessment was performed by an ophthalmologist or an optometrist under the guidance of an ophthalmologist. Visual acuity is defined as acuteness or clearness of vision. The number of participants with worsening visual acuity due to cataracts at Follow-up Week 24 are presented. Change due to cataracts is categorized as "Yes" or "No." | Safety Population. Only those participants who had worsening visual acuity at Week 61 were analyzed. | Posted | Number | Participants | Baseline and Follow-Up Week 24 (Week 61) |
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| Secondary | Pharmacokinetic (PK) Assessments for Eltrombopag for AUC (0-t) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. AUC(0-t) is defined as the area under the concentration-time curve over the dosing interval. The AUC(0-t) for a 50mg dose was estimated for each cohort. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort for a 50mg dose. | PK Population. Only those participants who provided pharmacokinetic samples were analyzed | Posted | Geometric Mean | 95% Confidence Interval | micrograms*hour per milliliter (ug.h/mL) | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
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| Secondary | Pharmacokinetic (PK) Assessments for Eltrombopag for Cmax | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. Cmax is defined as the maximum observed concentration. The Cmax for a 50mg dose was estimated for each cohort. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort for a 50mg dose. | PK Population. Only those participants who provided pharmacokinetic samples were analyzed | Posted | Geometric Mean | 95% Confidence Interval | micrograms per milliliter (ug/mL) | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
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| Secondary | Pharmacokinetic (PK) Assessments for Eltrombopag for Apparent Oral Clearance (CL/F) and Apparent Intercompartmental Clearance (Q/F) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. CL/F is defined as the apparent oral clearance from plasma and Q/F is defined as apparent intercompartmental clearance. These parameters are dose independent. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort. | PK Population. Only those participants who provided pharmacokinetic samples were analyzed | Posted | Geometric Mean | 95% Confidence Interval | Liters per hour (L/hr) | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
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| Secondary | PK Assessments for Eltrombopag for Apparent Central Volume (Vc/F) and Apparent Peripheral Volume (Vp/F) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. Vc/F is defined as the volume of the central (e.g. plasma) compartment and Vp/F is defined as the volume of the peripheral compartment. These parameters are dose independent. From the final model, a single value of each PK parameter was estimated for each subject, and geometric mean (95% CI) values are presented for each cohort. | PK Population. Only those participants who provided pharmacokinetic samples were analyzed | Posted | Geometric Mean | 95% Confidence Interval | Liters (L) | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
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| Secondary | Population PK Model Point Estimate for Eltrombopag for Absorption Rate-constant (Ka) | Single PK samples were collected at each visit during Part 1 Weeks 2, 4, 6, 8, 10, 12 and at each weekly or monthly visit during Part 2 Weeks 1-12 (Study Weeks 13-37). The concentration data were pooled across visits to identify population PK and variability parameter estimates and covariate effects. Ka is defined as the absorption rate constant. This parameter is dose independent, and the population estimate Ka is reported. | PK Population. Only those participants who provided pharmacokinetic samples were analyzed | Posted | Number | 1/h | Part 1 Weeks 2, 4, 6, 8, 10, 12, and Part 2 Weeks 1-12 (Study Weeks 13 - 37) |
|
On-treatment adverse events (AEs) and serious adverse events (SAEs) are defined as events occuring from the start of investigational product until follow up (up to 61 weeks).
SAEs and non-serious AEs were collected in members of the Safety Population, comprised of all participants who had received at least one dose of the investigational product.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part 1: Eltrombopag | In Part 1, participants aged between 6 and 17 years with a body weight less than 27 kg received eltrombopag 37.5 mg QD, and those with a body weight greater than or equal to 27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. | 5 | 63 | 51 | 63 | ||
| EG001 | Part 1: Placebo | In Part 1, participants aged between 6 and 17 years with a body weight less than 27 kg received placebo 37.5 mg QD, and those with a body weight greater than or equal to 27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of placebo 0.8 mg/kg/day. | 4 | 29 | 19 | 29 | ||
| EG002 | Part 2: Eltrombopag | In Part 2, participants continued on the same dose of eltrombopag received in Part 1 unless adjustments were warranted according to the dosing guidelines. | 9 | 87 | 68 | 87 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gingivitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Meningitis aseptic | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Alanine aminotransferase abnormal | Investigations | MedDRA | Systematic Assessment |
| |
| Aspartate aminotransferase abnormal | Investigations | MedDRA | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Dengue fever | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Haematoma infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Aspartate Aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Alanine Aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA | Systematic Assessment |
| |
| Blood alkaline Phosphatase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA | Systematic Assessment |
| |
| Bronchitis | Investigations | MedDRA | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Allergy to chemicals | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Bulimia nervosa | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Furuncle | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Lice infestation | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Lip haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Motion sickness | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain | General disorders | MedDRA | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Platelet count increased | Investigations | MedDRA | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Retinal vascular disorder | Eye disorders | MedDRA | Systematic Assessment |
| |
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Subcutaneous abscess | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tongue haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Viral pharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Blood albumin increased | Investigations | MedDRA | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Helminthic infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Otosalpingitis | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Pericoronitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tongue biting | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Urethral haemorrhage | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Hyperaemia | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Acute tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Adenoiditis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Aphthous stomatitis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA | Systematic Assessment |
| |
| Breast mass | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Bronchostenosis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Campylobacter gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Essential hypertension | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Eyelid infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| Glucose-6-phosphate dehydrogenase deficiency | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA | Systematic Assessment |
| |
| Herpes virus infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Infection parasitic | Infections and infestations | MedDRA | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Menarche | Social circumstances | MedDRA | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Migraine with aura | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Odynophagia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Oral mucosal blistering | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Pharyngeal erythema | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Pupils unequal | Eye disorders | MedDRA | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Seborrhoeic dermatitis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Tic | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Visual impairment | Eye disorders | MedDRA | Systematic Assessment |
|
GSK agreements may vary with individual investigators, but will not prohibit any investigator from publishing. GSK supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| GSK Response Center | GlaxoSmithKline | 866-435-7343 |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| D001791 | Blood Platelet Disorders |
| D013921 | Thrombocytopenia |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C520809 | eltrombopag |
Not provided
Not provided
Not provided
| Withdrawal by parent or guardian |
|
| Male |
|
| Central/South Asian Heritage |
|
| Japanese/East Asian/South East Asian Heritage |
|
| Arabic/North African Heritage |
|
| White/Caucasian/European Heritage |
|
| Mixed Race |
|
| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|
| Participants |
|
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| Units | Counts |
|---|
| Participants |
|
|
In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
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|
|
| Units | Counts |
|---|---|
| Participants |
|
|
In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day.
|
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| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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| Units |
|---|
| Counts |
|---|
| Participants |
|
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| OG001 | Part 1 (Randomized Period) - Eltrombopag | In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
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|
|
|
|
|
| OG001 | Part 1 (Randomized Period)-Eltrombopag | In Part 1, participants aged between 6 and 17 years with a body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
|
|
|
|
|
|
|
| Participants |
|
|
|
|
|
|
|
|
Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag as per age criteria as follows: body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. |
| OG002 | Eltrombopag Cohort 3 (1-5 Years) | Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag 1.2 mg/kg QD and; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
|
Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag as per age criteria as follows: body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD.
| OG002 | Eltrombopag Cohort 3 (1-5 Years) | Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag 1.2 mg/kg QD and; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
|
Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag as per age criteria as follows: body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. |
| OG002 | Eltrombopag Cohort 3 (1-5 Years) | Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag 1.2 mg/kg QD and; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
|
Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag as per age criteria as follows: body weight <27 kg received eltrombopag 37.5 mg QD, and those with a body weight >=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. |
| OG002 | Eltrombopag Cohort 3 (1-5 Years) | Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag 1.2 mg/kg QD and; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
|
| OG002 | Eltrombopag Cohort 3 (1-5 Years) | Participants who received eltrombopag in Part 1 continued on the same dose in Part 2 unless adjustments were warranted according to the dosing guidelines. Participants who received placebo in Part 1 received Eltrombopag 1.2 mg/kg QD and; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day. |
|
|