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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-000114-19 | EudraCT Number |
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This was a worldwide, three-part (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension), multi-center study to evaluate the effect of eltrombopag in subjects with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have thrombocytopenia due to bone marrow insufficiency from their underlying disease or prior chemotherapy. This objective was assessed by a composite primary endpoint that consists of the following: the proportion of ≥Grade 3 hemorrhagic adverse events, or platelet counts <10 Gi/L, or platelet transfusions. Patients with MDS or AML and Grade 4 thrombocytopenia due to bone marrow insufficiency from their underlying disease or prior chemotherapy were enrolled in the study. No low or intermediate-1 risk MDS subjects were enrolled in the study. Subjects must have had at least one of the following during the 4 weeks prior to enrolment: platelet count <10 Gi/L, platelet transfusion, or symptomatic hemorrhagic event. Supportive standard of care (SOC), including hydroxyurea, was allowed as indicated by local practice throughout the study. The study had 3 sequential parts. Subjects who were enrolled in Part 1 (open-label) cannot be enrolled in Part 2 of the study (randomized, double-blind); however, subjects who completed the treatment period for Part 1 or Part 2 (8 and 12 weeks, respectively) continued in Part 3 (extension) if the investigator determined that the subject was receiving clinical benefit on treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1, Open Label | Experimental | 100 mg daily (50 mg for subjects of East Asian heritage), intrasubject dose escalations to a maximum dose 300 mg (150 mg for subjects of East Asian heritage) are allowed. |
|
| Part 2, eltrombopag arm | Experimental | 100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage) |
|
| Part 2, placebo arm | Experimental | 100 mg matching placebo daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg matching placebo (150 mg for subjects of East Asian heritage) |
|
| part 3 extension | Experimental | 100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eltrombopag | Drug | 100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Platelet Response up to Week 8 During Part 1 | A participant was considered as a responder if he/she met the following response criteria: a Baseline platelet count <20 Giga cells per liter (Gi/L) and a post-Baseline increased to >20 Gi/L and at least 2 times the Baseline value; or a Baseline platelet count >=20 Gi/L and a post-Baseline absolute platelet count increased to >=50 Gi/L and at least 2 times the Baseline value. The response criteria was evaluated at each visit. Increase in platelet count observed up to 3 days after a platelet transfusion was not considered as a platelet response. The Part 1 Population was comprised of all participants enrolled into Part 1. | From Baseline up to Week 8 during Part 1 |
| Clinically Relevant Thrombocytopenic Events (CRTE) From Week 5 up to Week 12 During Part 2 | A participant was considered to have a CRTE at a given assessment if he/she had platelet counts <10 Gi/L, or platelet transfusions, or >=Grade 3 hemorrhagic adverse events. CRTEs during Weeks 5 to 12 were compared between treatments using a generalized linear mixed model. Average of weekly proportion of subjects with CRTE during Week 5 to 12 was estimated for each treatment. Intent to Treat (ITT) Population was comprised of all randomized participants during Part 2. | From Week 5 up to Week 12 during Part 2 |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Eltrombopag Pharmacokinetic Concentration-Time Data - by Visit (Part 1 Subjects) | Day 1 to week 8 | |
| Plasma Eltrombopag Pharmacokinetic Concentration-Time Data - by Visit (Part 2 Subjects) | Day 1 to week 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Phoenix | Arizona | 85016 | United States | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37231838 | Derived | Mittelman M, Platzbecker U, Grosicki S, Lawniczek T, Zhu Z, Selleslag D. Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study. Acta Haematol. 2023;146(5):373-378. doi: 10.1159/000531146. Epub 2023 May 19. | |
| 29241762 |
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Participants with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) and Grade 4 thrombocytopenia due to bone marrow insufficiency and had at least one of the following: platelet count <10 Giga cells per liter, platelet transfusion or symptomatic hemorrhagic event during the 4 weeks prior to enrollment, were enrolled in the study.
Part 1, 17 subjects received open-label eltrombopag. Part 2, 145 subjects were randomized to receive eltrombopag plus SOC (N=98) or placebo plus SOC (N=47).
Part 3, 59 subjects from Part 2 entered Part 3. SOC was allowed as needed throughout the study. Subjects could receive disease-modifying therapy as needed.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part 1: Eltrombopag | The eltrombopag starting dose the participants received was 100 milligrams (mg) daily (50 mg for participants of East Asian heritage). The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. After all participants finished the 8 week treatment period, platelet response and safety were analyzed before initiating Part 2 of the study. Supportive standard of care was allowed as needed. The duration of Part 1 was 8 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1: Open-label Phase |
|
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|
| placebo | Drug | 100 mg matching placebo daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg matching placebo (150 mg for subjects of East Asian heritage) |
|
| Mean Number of Platelet Transfusions | Weeks 5 to 12 |
| Hematologic Improvement | Definitions of hematologic improvement for platelets, neutrophils, and hemoglobin were based on modified International Working Group (IWG) consensus criteria. | Weeks 5 to 12 |
| Change in Mean Platelet Count | Baseline to Week 12 |
| Maximum Duration of Platelet Transfusion Independence | Weeks 5 to 12 |
| Number of Participants With Maximum Bleeding Grade According to World Health Organization on Bleeding Scale | Occurrence and severity of bleeding, measured using the WHO Bleeding Scale Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross blood loss; Grade 4=debilitating blood loss. | Weeks 5 to 12 |
| Independent Reviewer-Assessed Best Response | Participants were evaluated in accordance with the modified International Working Group (Cheson, 2006). CR: Bone marrow blasts <5%, Hgb ≥11g/dL, Hematologic Improvement - Platelets (Baseline <20Gi/L: >20 Gi/L and 2x baseline; Baseline ≥20 Gi/L: ≥50 Gi/L and 2x baseline), Neutrophils ≥1.0 Gi/L, Peripheral blasts 0%. PR: Bone marrow blasts decreased by ≥50% but >5%, Peripheral blood as in CR. Marrow CR: Bone marrow blasts <5% and decrease by ≥50%, Note any Hematologic Improvements, Stable disease: Failure to achieve PR, but no evidence of progression for >8w, Cytogenetic response: Complete: disappearance of chromosomal abnormality; no new abnormalities Partial: ≥50% reduction of chromosomal abnormality. | up to week 12 |
| Independent Reviewer Assessed Disease Progression | Up to week 12 |
| Median Overall Survival | Up to 13 months |
| Summary of Health Outcomes | The number of subject with medical resource utilization (MRU) data are reported in this table. MRU included number of emergency room visits, number of home healthcare visits, number of hospitalization days, number of medication or surgery specialist visits, number of procedures inpatient, number of procedures outpatient, number of non-study radiology visits, number of non-study laboratory visits, number of nurse practitioner/physician assistance/nurse visits, number of primary care physician visits, number of telephone consultations. | week 12 |
| Functional Assessment of Cancer Therapy (FACT) | The FACT-Th-18 is the most widely used and accepted tool evaluating health-related quality-of-life outcomes in cancer patients with chronically low platelets (where Th designates thrombocytopenia). The entire FACT-Th-18 was used in this trial, which includes the 18-item thrombocytopenia subscale used to assess the impact of symptoms, signs, and functional consequences of thrombocytopenia in MDS and AML subjects. The FACT-Th-18 is a validated and reliable instrument with known psychometric properties. FACT-ThS is an 18 item questionnaire specific to assessing the impact of symptoms, signs, and functional consequences of Thrombocytopenia. ThS score ranges from 0 to 72 with higher the score, the better the QoL. FACT G total score moves from 0 to 108 where higher the score, the better the HRQL. FACT-Th Total Score is calculated by adding the FACT-ThS and FACT-G score. Total score ranges from 0 to 180 and again, higher the score, the better the HRQL. | Change from baseline, up to week 12 |
| EQ-5D Utility Score Analysis | EuroQoL Five Dimensions Questionnaire (EQ-5D) is a standardized generic preference based health related quality of life instrument. It records how one's health is "today" and consists of a descriptive system. The Descriptive system is Comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension on the EQ-5D involves a 3-point response scale which indicates the level of impairment (level 1 = no problem; level 2 = some or moderate problem(s) and level 3 = unable, or extreme problems). Level of problem reported in each EQ-5D dimension determines a unique health state which is converted into a weighted health state index by applying scores from EQ-5D preference weights elicited from general population samples. This generates a unique description of the subjects' health status, which is valued between 0 (representing death) and 1 (representing perfect health). Higher the score, the better the quality of life. EQ-ED is a score. | Change from baseline, up to week 12 |
| Hot Springs |
| Arkansas |
| 71913 |
| United States |
| Novartis Investigative Site | Jonesboro | Arkansas | 72401 | United States |
| Novartis Investigative Site | Los Angeles | California | 90095 | United States |
| Novartis Investigative Site | Stanford | California | 94305 | United States |
| Novartis Investigative Site | Jacksonville | Florida | 32256 | United States |
| Novartis Investigative Site | Orlando | Florida | 32806 | United States |
| Novartis Investigative Site | West Palm Beach | Florida | 33401 | United States |
| Novartis Investigative Site | Augusta | Georgia | 30912 | United States |
| Novartis Investigative Site | Baltimore | Maryland | 21201 | United States |
| Novartis Investigative Site | Boston | Massachusetts | 02115 | United States |
| Novartis Investigative Site | Kansas City | Missouri | 64128 | United States |
| Novartis Investigative Site | Hackensack | New Jersey | 07601 | United States |
| Novartis Investigative Site | Voorhees Township | New Jersey | 08043 | United States |
| Novartis Investigative Site | The Bronx | New York | 10467 | United States |
| Novartis Investigative Site | Philadelphia | Pennsylvania | 19140 | United States |
| Novartis Investigative Site | Seattle | Washington | 98108 | United States |
| Novartis Investigative Site | Milwaukee | Wisconsin | 53226 | United States |
| Novartis Investigative Site | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1431FWO | Argentina |
| Novartis Investigative Site | La Plata | Buenos Aires | B1900AXI | Argentina |
| Novartis Investigative Site | Buenos Aires | 1405 | Argentina |
| Novartis Investigative Site | Buenos Aires | C1025ABH | Argentina |
| Novartis Investigative Site | Ciudad Autónoma de Buenos Aires | 1405 | Argentina |
| Novartis Investigative Site | Antwerp | 2060 | Belgium |
| Novartis Investigative Site | Brasschaat | 2930 | Belgium |
| Novartis Investigative Site | Bruges | 8000 | Belgium |
| Novartis Investigative Site | Brussels | 1000 | Belgium |
| Novartis Investigative Site | Ghent | 9000 | Belgium |
| Novartis Investigative Site | Leuven | 3000 | Belgium |
| Novartis Investigative Site | Yvoir | 5530 | Belgium |
| Novartis Investigative Site | Goiânia - GO | Goiás | 74605-020 | Brazil |
| Novartis Investigative Site | Goiânia - GO | Goiás | 74605-020 | Brazil |
| Novartis Investigative Site | Curitiba | Paraná | 81520-060 | Brazil |
| Novartis Investigative Site | Curitiba | Paraná | 81520-060 | Brazil |
| Novartis Investigative Site | Porto Alegre | Rio Grande do Sul | 90035-903 | Brazil |
| Novartis Investigative Site | Barretos | São Paulo | 14784-400 | Brazil |
| Novartis Investigative Site | Barretos | São Paulo | 14784-400 | Brazil |
| Novartis Investigative Site | São Paulo | São Paulo | 01236030 | Brazil |
| Novartis Investigative Site | Rio de Janeiro | 20211-030 | Brazil |
| Novartis Investigative Site | São Paulo | 01236030 | Brazil |
| Novartis Investigative Site | Halifax | Nova Scotia | B3H 2Y9 | Canada |
| Novartis Investigative Site | Toronto | Ontario | M5G 2M9 | Canada |
| Novartis Investigative Site | Montreal | Quebec | H3T 1E2 | Canada |
| Novartis Investigative Site | Brno | 625 00 | Czechia |
| Novartis Investigative Site | Prague | 100 34 | Czechia |
| Novartis Investigative Site | Prague | 128 08 | Czechia |
| Novartis Investigative Site | Prague | 128 20 | Czechia |
| Novartis Investigative Site | Mannheim | Baden-Wurttemberg | 68167 | Germany |
| Novartis Investigative Site | Stuttgart | Baden-Wurttemberg | 70199 | Germany |
| Novartis Investigative Site | Göttingen | Lower Saxony | 37075 | Germany |
| Novartis Investigative Site | Cologne | North Rhine-Westphalia | 50937 | Germany |
| Novartis Investigative Site | Düsseldorf | North Rhine-Westphalia | 40225 | Germany |
| Novartis Investigative Site | Dresden | Saxony | 01307 | Germany |
| Novartis Investigative Site | Athens | 11527 | Greece |
| Novartis Investigative Site | Heraklion, Crete | 71201 | Greece |
| Novartis Investigative Site | Ioannina | 45 500 | Greece |
| Novartis Investigative Site | Thessaloniki | 57010 | Greece |
| Novartis Investigative Site | Chai Wan | Hong Kong |
| Novartis Investigative Site | Shatin, New Territories | Hong Kong |
| Novartis Investigative Site | Budapest | 1088 | Hungary |
| Novartis Investigative Site | Debrecen | 4012 | Hungary |
| Novartis Investigative Site | Kaposvár | 7400 | Hungary |
| Novartis Investigative Site | Kaposvár | 7400 | Hungary |
| Novartis Investigative Site | Szeged | 6720 | Hungary |
| Novartis Investigative Site | Cork | Ireland |
| Novartis Investigative Site | Dublin | 7 | Ireland |
| Novartis Investigative Site | James Street | 8 | Ireland |
| Novartis Investigative Site | Limerick | Ireland |
| Novartis Investigative Site | Tallaght, Dublin | 24 | Ireland |
| Novartis Investigative Site | Tullamore | Ireland |
| Novartis Investigative Site | Beersheba | 84101 | Israel |
| Novartis Investigative Site | Haifa | 31048 | Israel |
| Novartis Investigative Site | Haifa | 31096 | Israel |
| Novartis Investigative Site | Holon | 58100 | Israel |
| Novartis Investigative Site | Jerusalem | 91120 | Israel |
| Novartis Investigative Site | Petah Tikva | 49100 | Israel |
| Novartis Investigative Site | Ramat Gan | 52621 | Israel |
| Novartis Investigative Site | Rehovot | 76100 | Israel |
| Novartis Investigative Site | Tel Aviv | 64239 | Israel |
| Novartis Investigative Site | Bologna | Emilia-Romagna | 40138 | Italy |
| Novartis Investigative Site | Brescia | Lombardy | 25123 | Italy |
| Novartis Investigative Site | Milan | Lombardy | 20122 | Italy |
| Novartis Investigative Site | Milan | Lombardy | 20162 | Italy |
| Novartis Investigative Site | Alessandria | Piedmont | 15100 | Italy |
| Novartis Investigative Site | Florence | Tuscany | 50134 | Italy |
| Novartis Investigative Site | Monterrey | Nuevo León | 64460 | Mexico |
| Novartis Investigative Site | Monterrey | Nuevo León | 64460 | Mexico |
| Novartis Investigative Site | Chihuahua City | 31203 | Mexico |
| Novartis Investigative Site | Mexico City | CP 14080 | Mexico |
| Novartis Investigative Site | Oaxaca City | 68000 | Mexico |
| Novartis Investigative Site | Amsterdam | 1081 HV | Netherlands |
| Novartis Investigative Site | Chorzów | 41-500 | Poland |
| Novartis Investigative Site | Torun | 87-100 | Poland |
| Novartis Investigative Site | Warsaw | 02-097 | Poland |
| Novartis Investigative Site | San Juan | 00927 | Puerto Rico |
| Novartis Investigative Site | Nizhny Novgorod | 603126 | Russia |
| Novartis Investigative Site | Petrozavodsk | 185019 | Russia |
| Novartis Investigative Site | Saint Petersburg | 197 089 | Russia |
| Novartis Investigative Site | St'Petersburg | 191024 | Russia |
| Novartis Investigative Site | St'Petersburg | 197341 | Russia |
| Novartis Investigative Site | Hwasun | 519-809 | South Korea |
| Novartis Investigative Site | Seoul | 110-744 | South Korea |
| Novartis Investigative Site | Seoul | 120-752 | South Korea |
| Novartis Investigative Site | Barcelona | 08036 | Spain |
| Novartis Investigative Site | Granada | Spain |
| Novartis Investigative Site | Madrid | 28034 | Spain |
| Novartis Investigative Site | Málaga | 29010 | Spain |
| Novartis Investigative Site | Málaga | 29010 | Spain |
| Novartis Investigative Site | Pozuelo de Alarcón/Madrid | 28223 | Spain |
| Novartis Investigative Site | Pozuelo de Alarcón/Madrid | 28223 | Spain |
| Novartis Investigative Site | Salamanca | 37007 | Spain |
| Novartis Investigative Site | Santander | 39008 | Spain |
| Novartis Investigative Site | Kaohsiung City | 83301 | Taiwan |
| Novartis Investigative Site | Tainan County | 736 | Taiwan |
| Novartis Investigative Site | Taoyuan County | 333 | Taiwan |
| Novartis Investigative Site | Bangkok | 10330 | Thailand |
| Novartis Investigative Site | Bangkok | 10400 | Thailand |
| Novartis Investigative Site | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Khon Kaen | 40002 | Thailand |
| Novartis Investigative Site | Songkhla | 90110 | Thailand |
| Mittelman M, Platzbecker U, Afanasyev B, Grosicki S, Wong RSM, Anagnostopoulos A, Brenner B, Denzlinger C, Rossi G, Nagler A, Garcia-Delgado R, Portella MSO, Zhu Z, Selleslag D. Eltrombopag for advanced myelodysplastic syndromes or acute myeloid leukaemia and severe thrombocytopenia (ASPIRE): a randomised, placebo-controlled, phase 2 trial. Lancet Haematol. 2018 Jan;5(1):e34-e43. doi: 10.1016/S2352-3026(17)30228-4. Epub 2017 Dec 11. |
| FG001 | Part 2: Placebo | Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study. |
| FG002 | Part 2: Eltrombopag | The eltrombopag starting dose the participants received was 100 mg daily (50 mg for participants of East Asian heritage). The duration of treatment with the initial dose level prior to first dose escalation was determined based on the platelet response and toxicity observed in Part 1. The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. Supportive standard of care was allowed as needed throughout the study. |
| FG003 | Part 3: Eltrombopag | 23 subjects of the 47 randomized to the placebo group in Part 2 entered part 3. 36 subjects of the 98 randomized to the Etrombopag group in Part 2 entered part 3. All subjects received eltrombopag. Part 3 subjects could also have received treatment for their disease per local SOC, including azacitidine, decitabine, lenalidomide, and chemotherapy. The duration of Part 3 was to be 10 months for subjects from Part 1and 9 months for subjects from Part 2. |
| COMPLETED |
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| NOT COMPLETED |
|
|
| Part 2; Double-blind Phase |
|
|
| Part 3: Extension |
|
|
Part 3, 59 subjects from Part 2 entered Part 3 (23 of 47 subjects in the placebo group in Part 2 and 36 of 98 subjects in the eltrombopag group in Part 2). All subjects who entered Part 3 received treatment with eltrombopag. SOC was allowed as needed throughout the study. Subjects could receive disease-modifying therapy as needed.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Part 1: Eltrombopag | The eltrombopag starting dose the participants received was 100 mg daily (50 mg for participants of East Asian heritage). The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. After all participants finished the 8 week treatment period, platelet response and safety were analyzed before initiating Part 2 of the study. Supportive standard of care was allowed as needed. The duration of Part 1 was 8 weeks. |
| BG001 | Part 2: Placebo | Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study. |
| BG002 | Part 2: Eltrombopag | The eltrombopag starting dose the participants received was 100 mg daily (50 mg for participants of East Asian heritage). The duration of treatment with the initial dose level prior to first dose escalation was determined based on the platelet response and toxicity observed in Part 1. The dose of eltrombopag was escalated from 100 mg to 200 mg (50 mg to 100 mg for East Asians) and further from 200 mg to 300 mg once daily (100 mg to 150 mg for East Asians) based on the platelet response and safety data after two weeks of current dose level. Supportive standard of care was allowed as needed throughout the study. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| 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 |
|
| 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 | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Platelet Response up to Week 8 During Part 1 | A participant was considered as a responder if he/she met the following response criteria: a Baseline platelet count <20 Giga cells per liter (Gi/L) and a post-Baseline increased to >20 Gi/L and at least 2 times the Baseline value; or a Baseline platelet count >=20 Gi/L and a post-Baseline absolute platelet count increased to >=50 Gi/L and at least 2 times the Baseline value. The response criteria was evaluated at each visit. Increase in platelet count observed up to 3 days after a platelet transfusion was not considered as a platelet response. The Part 1 Population was comprised of all participants enrolled into Part 1. | Part 1 Population: all subjects who enrolled in Part 1. | Posted | Number | Participants | From Baseline up to Week 8 during Part 1 |
|
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| Primary | Clinically Relevant Thrombocytopenic Events (CRTE) From Week 5 up to Week 12 During Part 2 | A participant was considered to have a CRTE at a given assessment if he/she had platelet counts <10 Gi/L, or platelet transfusions, or >=Grade 3 hemorrhagic adverse events. CRTEs during Weeks 5 to 12 were compared between treatments using a generalized linear mixed model. Average of weekly proportion of subjects with CRTE during Week 5 to 12 was estimated for each treatment. Intent to Treat (ITT) Population was comprised of all randomized participants during Part 2. | Part 2 Population (Intent-To-Treat (ITT) Population: all randomized subjects in part 2) | Posted | Mean | 95% Confidence Interval | percentages of participants | From Week 5 up to Week 12 during Part 2 |
| |||||||||||||||||||||||||||
| Secondary | Plasma Eltrombopag Pharmacokinetic Concentration-Time Data - by Visit (Part 1 Subjects) | Pharmacokinetic population: all subjects in All Subjects Population who had a PK blood sample obtained/analyzed. | Posted | Mean | Standard Deviation | ug/mL | Day 1 to week 8 |
|
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| Secondary | Plasma Eltrombopag Pharmacokinetic Concentration-Time Data - by Visit (Part 2 Subjects) | Pharmacokinetic population | Posted | Mean | Standard Deviation | ug/mL | Day 1 to week 12 |
|
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| Secondary | Mean Number of Platelet Transfusions | Part 2 Population (Intent-to-Treat population) | Posted | Mean | Standard Deviation | Number of platelet transfusions | Weeks 5 to 12 |
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| Secondary | Hematologic Improvement | Definitions of hematologic improvement for platelets, neutrophils, and hemoglobin were based on modified International Working Group (IWG) consensus criteria. | Part 2 Population (Intent-to-Treat population) | Posted | Number | Number of subjects | Weeks 5 to 12 |
|
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| Secondary | Change in Mean Platelet Count | Part 2 Population (Intent-to-Treat population) | Posted | Mean | Standard Deviation | Gi/L | Baseline to Week 12 |
|
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| Secondary | Maximum Duration of Platelet Transfusion Independence | Part 2 Population (Intent-to-Treat population) | Posted | Mean | Standard Deviation | maximum duration of platelet transfusion | Weeks 5 to 12 |
|
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| Secondary | Number of Participants With Maximum Bleeding Grade According to World Health Organization on Bleeding Scale | Occurrence and severity of bleeding, measured using the WHO Bleeding Scale Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross blood loss; Grade 4=debilitating blood loss. | Part 2 Population (Intent-to-Treat population) | Posted | Number | Number of subjects | Weeks 5 to 12 |
| ||||||||||||||||||||||||||||
| Secondary | Independent Reviewer-Assessed Best Response | Participants were evaluated in accordance with the modified International Working Group (Cheson, 2006). CR: Bone marrow blasts <5%, Hgb ≥11g/dL, Hematologic Improvement - Platelets (Baseline <20Gi/L: >20 Gi/L and 2x baseline; Baseline ≥20 Gi/L: ≥50 Gi/L and 2x baseline), Neutrophils ≥1.0 Gi/L, Peripheral blasts 0%. PR: Bone marrow blasts decreased by ≥50% but >5%, Peripheral blood as in CR. Marrow CR: Bone marrow blasts <5% and decrease by ≥50%, Note any Hematologic Improvements, Stable disease: Failure to achieve PR, but no evidence of progression for >8w, Cytogenetic response: Complete: disappearance of chromosomal abnormality; no new abnormalities Partial: ≥50% reduction of chromosomal abnormality. | Part 2 Population (Intent-to-Treat population) | Posted | Number | Number of subjects | up to week 12 |
| ||||||||||||||||||||||||||||
| Secondary | Independent Reviewer Assessed Disease Progression | Part 2 Population (Intent-to-Treat population) | Posted | Number | Number of subjects | Up to week 12 |
|
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| Secondary | Median Overall Survival | Part 2 Population (Intent-to-Treat population) | Posted | Median | 95% Confidence Interval | Months | Up to 13 months |
|
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| Secondary | Summary of Health Outcomes | The number of subject with medical resource utilization (MRU) data are reported in this table. MRU included number of emergency room visits, number of home healthcare visits, number of hospitalization days, number of medication or surgery specialist visits, number of procedures inpatient, number of procedures outpatient, number of non-study radiology visits, number of non-study laboratory visits, number of nurse practitioner/physician assistance/nurse visits, number of primary care physician visits, number of telephone consultations. | Part 2 Population (Intent-to-Treat population) | Posted | Number | Subject with Events | week 12 |
| ||||||||||||||||||||||||||||
| Secondary | Functional Assessment of Cancer Therapy (FACT) | The FACT-Th-18 is the most widely used and accepted tool evaluating health-related quality-of-life outcomes in cancer patients with chronically low platelets (where Th designates thrombocytopenia). The entire FACT-Th-18 was used in this trial, which includes the 18-item thrombocytopenia subscale used to assess the impact of symptoms, signs, and functional consequences of thrombocytopenia in MDS and AML subjects. The FACT-Th-18 is a validated and reliable instrument with known psychometric properties. FACT-ThS is an 18 item questionnaire specific to assessing the impact of symptoms, signs, and functional consequences of Thrombocytopenia. ThS score ranges from 0 to 72 with higher the score, the better the QoL. FACT G total score moves from 0 to 108 where higher the score, the better the HRQL. FACT-Th Total Score is calculated by adding the FACT-ThS and FACT-G score. Total score ranges from 0 to 180 and again, higher the score, the better the HRQL. | Part 2 Population (Intent-to-Treat population) | Posted | Mean | Standard Error | Adjusted mean change from baseline | Change from baseline, up to week 12 |
| |||||||||||||||||||||||||||
| Secondary | EQ-5D Utility Score Analysis | EuroQoL Five Dimensions Questionnaire (EQ-5D) is a standardized generic preference based health related quality of life instrument. It records how one's health is "today" and consists of a descriptive system. The Descriptive system is Comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension on the EQ-5D involves a 3-point response scale which indicates the level of impairment (level 1 = no problem; level 2 = some or moderate problem(s) and level 3 = unable, or extreme problems). Level of problem reported in each EQ-5D dimension determines a unique health state which is converted into a weighted health state index by applying scores from EQ-5D preference weights elicited from general population samples. This generates a unique description of the subjects' health status, which is valued between 0 (representing death) and 1 (representing perfect health). Higher the score, the better the quality of life. EQ-ED is a score. | Part 2 Population (Intent-to-Treat population) | Posted | Mean | Standard Error | Adjusted mean change from baseline | Change from baseline, up to week 12 |
|
Not provided
Not provided
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 | Eltrombopag, Part 1 Subjects | Eltrombopag, Part 1 subjects | 9 | 17 | 17 | 17 | ||
| EG001 | Eltrombopag, Part 2 Plus 1 Day From Part 2 Subjects | Eltrombopag, Part 2 plus 1 day from Part 2 subjects | 56 | 97 | 89 | 97 | ||
| EG002 | Placebo, Part 2 Plus 1 Day From Part 2 Subjects | Placebo, Part 2 plus 1 day from Part 2 subjects | 32 | 47 | 44 | 47 | ||
| EG003 | Eltrombopag, Part 3 Plus 30 Days From Part 2 Subjects | Eltrombopag, Part 3 plus 30 Days from Part 2 subjects | 39 | 59 | 54 | 59 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Splenic artery thrombosis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Splenic infarction | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Adrenal disorder | Endocrine disorders | MedDRA | Systematic Assessment |
| |
| Retinal vein occlusion | Eye disorders | MedDRA | Systematic Assessment |
| |
| Vitreous haemorrhage | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gastric haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gastrointestinal hypomotility | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Intestinal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Intestinal ischaemia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Oral mucosal blistering | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Retroperitoneal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA | Systematic Assessment |
| |
| Chills | General disorders | MedDRA | Systematic Assessment |
| |
| Death | General disorders | MedDRA | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA | Systematic Assessment |
| |
| Multi-organ failure | General disorders | MedDRA | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Hepatitis | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Abscess neck | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Catheter site cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cholecystitis infective | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Endocarditis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Enteritis infectious | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Escherichia bacteraemia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Osteomyelitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Splenic abscess | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Streptococcal sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Viral oesophagitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Subdural haemorrhage | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Wound haemorrhage | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| Lung neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Diabetic neuropathy | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Subarachnoid haemorrhage | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Laryngeal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Lung consolidation | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pulmonary alveolar haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Arterial thrombosis | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Thrombophlebitis | Vascular disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Blood disorder | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA | Systematic Assessment |
| |
| Eye haemorrhage | Eye disorders | MedDRA | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA | Systematic Assessment |
| |
| Ocular icterus | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gingival swelling | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Catheter site related reaction | General disorders | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA | Systematic Assessment |
| |
| Pain | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Xerosis | General disorders | MedDRA | Systematic Assessment |
| |
| Hepatotoxicity | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Arthritis infective | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Bacterial infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Escherichia infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Klebsiella sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Post procedural haematoma | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood albumin decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood phosphorus decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood sodium increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA | Systematic Assessment |
| |
| Body temperature fluctuation | Investigations | MedDRA | Systematic Assessment |
| |
| Liver function test abnormal | Investigations | MedDRA | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Serum ferritin increased | Investigations | MedDRA | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Aphonia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Mental impairment | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Poor quality sleep | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Initial insomnia | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Azotaemia | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Blood blister | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Purpura | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Skin haemorrhage | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 |
| ID | Term |
|---|---|
| D013921 | Thrombocytopenia |
| D015470 | Leukemia, Myeloid, Acute |
| D009196 | Myeloproliferative Disorders |
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001791 | Blood Platelet Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000095542 | Cytopenia |
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C520809 | eltrombopag |
Not provided
Not provided
Not provided
| Withdrawal by Subject |
|
| Protocol Violation |
|
| Lack of Efficacy |
|
| Adverse Event |
|
| Not treated |
|
| Adverse Event |
|
| Physician Decision |
|
| Male |
|
| Asian - East Asian Heritage |
|
| Asian - South East Asian Heritage |
|
| White - Arabic/North African Heritage |
|
| White - White/Caucasian/European Heritage |
|
|
|
|
|
|
|
| Participants |
|
|
|
|
| Units | Counts |
|---|
| Participants |
|
|
Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study. |
|
|
|
|
|
|
| OG001 | Part 2: Placebo | Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study. |
|
|
| OG001 | Part 2: Placebo | Participants received eltrombopag matching placebo once daily (3 tablets). Supportive standard of care was allowed as needed throughout the study. |
|
|