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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02158936




Registration number
NCT02158936
Ethics application status
Date submitted
5/06/2014
Date registered
9/06/2014
Date last updated
12/12/2017

Titles & IDs
Public title
A Study of Eltrombopag or Placebo in Combination With Azacitidine in Subjects With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-risk Myelodysplastic Syndromes (MDS)
Scientific title
A Randomized, Double-blind, Placebo-controlled, Phase III, Multi-centre Study of Eltrombopag or Placebo in Combination With Azacitidine in Subjects With IPSS Intermediate-1, Intermediate 2 and High-risk Myelodysplastic Syndromes (MDS) SUPPORT: A StUdy of eltromboPag in myelodysPlastic SyndrOmes Receiving azaciTidine
Secondary ID [1] 0 0
112121
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Thrombocytopaenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Eltrombopag
Treatment: Drugs - Azacitidine
Treatment: Drugs - Placebo
Treatment: Drugs - Placebo

Experimental: Eltrombopag - Eligible subject will receive a starting dose of eltrombopag of 200 milligrams (mg) (100 mg for subjects of East Asian heritage). Dose modifications of eltrombopag will be permitted by 100 mg increments (50 mg increments for East Asians) to a lowest dose of 100 mg (50 mg for East Asian heritage) or a maximum dose of 300 mg (150 mg for East Asian heritage) in order to maintain platelet counts at a safe and effective level (i.e. a level sufficient to avoid platelet transfusions and bleeding events). Subjects will receive azacitidine 75 mg/meter^2 subcutaneously once daily for 7 days (+/- 3 day treatment window permitted) every 28 days, for at least 6 cycles if tolerated and until they are no longer receiving benefit (defined as at least stable disease per the investigator's assessment) or until disease progression, death, or unacceptable toxicity/adverse event. The subject may receive eltrombopag daily for the full 28 days each cycle for as long as the subject is receiving azacitidine

Placebo Comparator: Placebo - Eligible subject will receive eltrombopag matching placebo. Subjects will receive azacitidine 75 mg/meter^2 subcutaneously once daily for 7 days (+/- 3 day treatment window permitted) every 28 days, for at least 6 cycles if tolerated and until they are no longer receiving benefit (defined as at least stable disease per the investigator's assessment) or until disease progression, death, or unacceptable toxicity/adverse event. The subject may receive eltrombopag daily for the full 28 days each cycle for as long as the subject is receiving azacitidine


Treatment: Drugs: Eltrombopag
Eltrombopag will be round film-coated tablets containing eltrombopag equivalent to 50 mg (white to off-white), 200 mg and 300 mg (green) of eltrombopag free acid

Treatment: Drugs: Azacitidine
Subcutaneous Injection (IV if local standard)

Treatment: Drugs: Placebo
Eltrombopag matching placebo tablets

Treatment: Drugs: Placebo
Eltrombopag matching placebo tablets will be supplied

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Who Were Platelet Transfusion Independent During Cycles 1-4 of Azacitidine Therapy - A subject is defined as being platelet transfusion independent if they received no platelet transfusions within the first 4 cycles of treatment with azacitidine. Subjects who died or withdrew from investigational product within the first four cycles were treated as failures (i.e. not transfusion independent) in the analysis
Timepoint [1] 0 0
4 cycles (Cycle = 28 days)
Secondary outcome [1] 0 0
Overall Survival (OS) - Overall survival is defined as the time from randomization until death due to any cause and deaths have been presented. Subjects still alive at the time of the analysis and subjects who have withdrawn from the study will be censored at the time of last contact
Timepoint [1] 0 0
Randomization until death or end of study, approximately 2 years
Secondary outcome [2] 0 0
Summary of Progression Free Survival From Investigator Assessment (ITT) - Progression-free survival, defined as the time from randomization until either disease progression or death. The modified 2006 IWG criteria for MDS used for progression assessment For patients with: Less than 5% BM blasts: = 50% increase in blasts to > 5% blasts; 5% - <10% BM blasts: = 50% increase to > 10% blasts; 10% - <20% BM blasts: = 50% increase to > 20% blasts; 20% - 30% BM blasts: = 50% increase to > 30% blasts
Timepoint [2] 0 0
First day of each cycle (Cycles 3+), at the end of therapy visit and every 3 months in follow-up for approximately 2 years
Secondary outcome [3] 0 0
Summary of Progression Free Survival From Central Review (ITT) - Progression-free survival, defined as the time from randomization until either disease progression or death. The modified 2006 IWG criteria for MDS used for progression assessment For patients with:
Less than 5% BM blasts: = 50% increase in blasts to > 5% blasts; 5% - <10% BM blasts: = 50% increase to > 10% blasts; 10% - <20% BM blasts: = 50% increase to > 20% blasts; 20% - 30% BM blasts: = 50% increase to > 30% blasts
Timepoint [3] 0 0
First day of each cycle (Cycles 3+), at the end of therapy visit and every 3 months in follow-up for approximately 2 years
Secondary outcome [4] 0 0
Summary of AML Progression From Investigator Assessment and Central Review (ITT) - Progression to AML in MDS patients with baseline bone marrow blast < 20% was defined as meeting definition of disease progression according to the modified 2006 IWG response criteria for MDS with the additional requirement that bone marrow blast or peripheral blast increases from < 20% at baseline to = 20% postbaseline. Progression assessment For patients with:
Less than 5% BM blasts: = 50% increase in blasts to > 5% blasts; 5% - <10% BM blasts: = 50% increase to > 10% blasts; 10% - <20% BM blasts: = 50% increase to > 20% blasts; 20% - 30% BM blasts: = 50% increase to > 30% blasts
Timepoint [4] 0 0
First day of each cycle (Cycles 3+), at the end of therapy visit and every 3 months in follow-up for approximately 2 years
Secondary outcome [5] 0 0
Best Disease Response From Investigator Assessment (ITT) - Best disease response is categorized as complete remission (CR), partial remission (PR), or marrow CR, stable disease, disease progression, or as non-evaluable; according to modified 2006 International Working Group (IWG) criteria for MDS
Timepoint [5] 0 0
At end of Cycle 6 (cycle=28 days) or end of therapy, whichever came first
Secondary outcome [6] 0 0
Best Disease Response From Central Review (ITT) - Best disease response is categorized as complete remission (CR), partial remission (PR), or marrow CR, stable disease, disease progression, or as non-evaluable; according to modified 2006 International Working Group (IWG) criteria for MDS
Timepoint [6] 0 0
At end of Cycle 6 (cycle=28 days) or end of therapy, whichever came first
Secondary outcome [7] 0 0
Hematologic Improvement (HI) in Platelets, Neutrophils, and Hemoglobin Based on the Modified IWG Criteria for MDS (ITT) - HI based on the modified IWG criteria for MDS. HI - Platelets (BL <100Gi/L), response criteria= BL <20: increase to>20 and 100% at least for 56 days or BL >=20: absolute increase of >=30. HI - Neutrophils (BL <1.0 Gi/L), response criteria=100% increase and an absolute increase >0.5 Gi/L over BL for at least 56 days. HI-Hemoglobin (BL <g/dL), response criteria=Hgb increase by >=1.5 g/dL over BL, RBC transfusions(given for Hgb<=9.0) reduced by >=4 per 8w from BL
Timepoint [7] 0 0
From Day 1 to 4-week follow-up (samples collected weekly in Cycle 1, Days 1 and 15 in Cycles 2-6 and Day 1 of Cycles >=7)
Secondary outcome [8] 0 0
Number of Participants Who Were Platelet Transfusion Independent (ITT Set) - Platelet transfusion independence is defined for each cycle as the number of participants who continue to the end of a cycle without requiring a platelet transfusion
Timepoint [8] 0 0
From Day 1 to end of study treatment up to approximately 2 years
Secondary outcome [9] 0 0
Bleeding Adverse Events (AEs) >= Grade 3 - Bleeding will be assessed by recording AEs or serious adverse events (SAEs) as graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.0
Timepoint [9] 0 0
From Day 1 to 4-week follow-up up to approximately 2 years
Secondary outcome [10] 0 0
Number of of Subjects With Azacitidine Dose Delays, Dose Reductions, Interruptions - The proportion of subjects with any delay, reduction or interruption in dosage of Azacitidine excluding those for non-medical reasons will be analyzed
Timepoint [10] 0 0
From Day 1 to 4-week follow-up up to approximately 2 years
Secondary outcome [11] 0 0
Response Levels in All Domains of Euroquol-5 Dimensions of Health, 3 Response Levels (EQ-5D-3L™) - The EQ-5D is a general health status and health utility measure which captures 5 dimensions of health state: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. (EQ-5D is a trademark of the Stichting EuroQol Group) . C=cycle, D=Day
Timepoint [11] 0 0
From Day 1 to 4-week follow-up up to approximately 2 years
Secondary outcome [12] 0 0
Functional Assessment of Chronic Disease Therapy-fatigue Subscale (FACIT-Fatigue) (ITT) - The FACIT-Fatigue subscale measures severity and impact of fatigue on functioning and Health Related QoL experienced in the past 7 days. Scale is a 13 item measure of fatigure. Items are scored on a 0-4 response scale ranging from "not at all" to "very much so". All items are summed to create a single fatigure score with a range from 0 to 52. Items are reverse scored when appropriate to provide a scale in which higher scores represent better functioning or less fatiigue (The FACIT Fatigue Scale is owned by David Cella, Ph.D.)
Timepoint [12] 0 0
From Day 1 to 4-week follow-up (Approximate median 9 Cycles+4 weeks follow-up) up to approximately 2 years
Secondary outcome [13] 0 0
Medical Resource Utilization (MRU): Event -Hospitalizations Inpatient and Outpatient - MRU data will be collected for each subject. Events corresponding to unscheduled (not scheduled per protocol) hospitalizations
Timepoint [13] 0 0
From Day 1 to 4-week follow-up (Approximate median 9 Cycles+4 weeks follow-up) up to approximately 2 years
Secondary outcome [14] 0 0
Medical Resource Utilization (MRU): Event and Use of Site Specific Medical Resources - Non-study Laboratory Tests - MRU data will be collected for each subject. Events corresponding to unscheduled (not scheduled per protocol) hospitalizations, office visits including consultations, laboratory and diagnostic tests (lab results, imaging etc.), and procedures prior to therapy initiation and during therapy will be collected
Timepoint [14] 0 0
From Day 1 to 4-week follow-up (Approximate median 9 Cycles+4 weeks follow-up) up to approximately 2 years
Secondary outcome [15] 0 0
Medical Resource Utilization (MRU): Use of Site Specific Medical Resources - MRU data will be collected for each subject. Events corresponding to unscheduled (not scheduled per protocol) hospitalizations, office visits including consultations, laboratory and diagnostic tests (lab results, imaging etc.), and procedures prior to therapy initiation and during therapy will be collected
Timepoint [15] 0 0
From Day 1 to 4-week follow-up (Approximate median 9 Cycles+4 weeks follow-up) up to approximately 2 years
Secondary outcome [16] 0 0
Summary of Post-Hoc Estimates of Steady-state Eltrombopag Cmax and Cmin Pharmacokinetic Parameters for a 50 mg Dose - Eltrombopag concentrations were analyzed using a population PK model along with data from other studies in healthy volunteers and in patients with MDS and/or AML. Post-hoc PK parameters were derived. Only patients from this study were included (163)
Timepoint [16] 0 0
Cycle 1, Week 2: Pre-dose, 1.5 and 3 hour post dose; Cycle 1, Week 3: 4, 5.5, and 7 hours post dose
Secondary outcome [17] 0 0
Summary of Post-Hoc Estimates of Steady-state Eltrombopag AUC0 Infinity Pharmacokinetic Parameters for a 50 mg Dose - Eltrombopag concentrations were analyzed using a population PK model along with data from other studies in healthy volunteers and in patients with MDS and/or AML. Post-hoc PK parameters were derived. Only patients from this study were included (163)
Timepoint [17] 0 0
Cycle 1, Week 2: Pre-dose, 1.5 and 3 hour post dose; Cycle 1, Week 3: 4, 5.5, and 7 hours post dose
Secondary outcome [18] 0 0
AUC0-infinity -Pharmacokinetic(s) Parameter of Azacitidine - An analysis of variance (ANOVA) on AUC0-infinity. The PK parameters were log transformed prior to analysis. The model included treatment as a fixed effect. Point estimates and their associated 90% CI were constructed for the differences in PK parameter values. The point estimates and their associated 90% CI were then back transformed to provide point estimates and 90% CI for the azacitidine + eltrombopag:azacitidine + placebo PK parameter ratios.
Timepoint [18] 0 0
Cycle 2 Day 1: Pre-dose, 15 min, 0.5, 1, 2 and 4 hr post dose
Secondary outcome [19] 0 0
Cmax -Pharmacokinetic Parameter of Azacitidine - An analysis of variance (ANOVA) on Cmax . The PK parameters were log transformed prior to analysis. The model included treatment as a fixed effect. Point estimates and their associated 90% CI were constructed for the differences in PK parameter values. The point estimates and their associated 90% CI were then back transformed to provide point estimates and 90% CI for the azacitidine + ltrombopag:azacitidine + placebo PK parameter ratios.
Timepoint [19] 0 0
Cycle 2 Day 1: Pre-dose, 15 min, 0.5, 1, 2 and 4 hr post dose

Eligibility
Key inclusion criteria
- Age >=18 years (For subjects in Taiwan, Age >= 20 years)

- MDS by World Health Organization (WHO) or French-American-British (FAB) classification

- Intermediate 1, intermediate 2 or high risk MDS by IPSS

- At least one platelet count < 75 Gi/L

- Eastern Cooperative Oncology Group (ECOG) Status 0-2

- Adequate baseline organ function defined by the criteria below: total bilirubin =<
1.5x the upper limit of normal (ULN) except for Gilbert's syndrome or cases clearly
not indicative of inadequate liver function (i.e. elevation of indirect [haemolytic]
bilirubin in the absence of alanine aminotransferase [ALT] abnormality); ALT =<
2.5xULN; creatinine =< 2.5xULN

- Subjects with a corrected QT interval (QTc) <450 milliseconds (msec) or <480msec for
subjects with bundle branch block. The QTc is the QT interval corrected for heart rate
according to Fridericia's formula (QTcF), machine or manual overread. For subject
eligibility and withdrawal, QTcF will be used. For purposes of data analysis, QTcF
will be used. The QTc should be based on single or averaged QTc values of triplicate
electrocardiograms (ECGs) obtained over a brief recording period

- Subject is able to understand and comply with protocol requirements and instructions

- Subject has signed and dated informed consent

- Women must be either of non-child bearing potential, or women with child-bearing
potential and men with reproductive potential must be willing to practice acceptable
methods of birth control during the study

- Women of childbearing potential must have a negative serum or urine pregnancy test
within 7 days of first dose of study treatment and agree to use effective
contraception during the study and for 3 months following the last dose of study
treatment

- Men with a female partner of childbearing potential must have either had a prior
vasectomy or agree to use effective contraception from time of randomization until 16
weeks after the last dose of study treatment

- French subjects: In France, a subject will be eligible for inclusion in this study
only if either affiliated to or a beneficiary of a social security category
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Previous treatment with hypomethylating agent or induction chemotherapy for MDS

- Proliferative type chronic myelomonocytic leukemia with white blood cell count >12
Gi/L at any time during the 28 days before Day 1

- History of treatment with eltrombopag, romiplostim or other thrombopoietin receptor
(TPO-R) agonists

- Previous allogeneic stem-cell transplantation

- Known thrombophilic risk factors. Exception: Subjects for whom the potential benefits
of participating in the study outweigh the potential risks of thromboembolic events,
as determined by the investigator

- Treatment with an investigational drug within 30 days or 5 half-lives (whichever is
longer) preceding the first dose of investigational product (eltrombopag/placebo)

- Active and uncontrolled infections, including hepatitis B or C

- Human Immunodeficiency Virus (HIV) infection

- Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs
chemically related to eltrombopag or its excipient, or azacitidine, that
contraindicates the subjects' participation

- Pregnant or lactating female

- Any serious and/or unstable pre-existing medical condition (including any advanced
malignancy other than the disease under study), psychiatric disorder, or other
conditions that could interfere with subject's safety, obtaining informed consent or
compliance with the study procedures

- French subjects: the French subject has participated in any study using an
investigational drug during the previous 30 days

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Kogarah
Recruitment hospital [2] 0 0
Novartis Investigative Site - Adelaide
Recruitment hospital [3] 0 0
Novartis Investigative Site - Clayton
Recruitment hospital [4] 0 0
Novartis Investigative Site - East Melbourne
Recruitment hospital [5] 0 0
Novartis Investigative Site - Melbourne
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
3002 - East Melbourne
Recruitment postcode(s) [5] 0 0
3000 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Connecticut
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
United States of America
State/province [9] 0 0
Wisconsin
Country [10] 0 0
Argentina
State/province [10] 0 0
Ciudad Autonoma de Buenos Aires
Country [11] 0 0
Argentina
State/province [11] 0 0
Santa Fe
Country [12] 0 0
Austria
State/province [12] 0 0
Graz
Country [13] 0 0
Austria
State/province [13] 0 0
Innsbruck
Country [14] 0 0
Austria
State/province [14] 0 0
Linz
Country [15] 0 0
Austria
State/province [15] 0 0
Rankweil
Country [16] 0 0
Austria
State/province [16] 0 0
Salzburg
Country [17] 0 0
Austria
State/province [17] 0 0
Steyr
Country [18] 0 0
Austria
State/province [18] 0 0
Vienna
Country [19] 0 0
Belgium
State/province [19] 0 0
Brasschaat
Country [20] 0 0
Belgium
State/province [20] 0 0
Brugge
Country [21] 0 0
Belgium
State/province [21] 0 0
Leuven
Country [22] 0 0
Belgium
State/province [22] 0 0
Lodelinsart
Country [23] 0 0
Belgium
State/province [23] 0 0
Turnhout
Country [24] 0 0
Brazil
State/province [24] 0 0
Minas Gerais
Country [25] 0 0
Brazil
State/province [25] 0 0
Paraná
Country [26] 0 0
Brazil
State/province [26] 0 0
Rio Grande Do Sul
Country [27] 0 0
Brazil
State/province [27] 0 0
Santa Catarina
Country [28] 0 0
Brazil
State/province [28] 0 0
São Paulo
Country [29] 0 0
Brazil
State/province [29] 0 0
Rio de Janeiro
Country [30] 0 0
Brazil
State/province [30] 0 0
Sao Paulo - SP
Country [31] 0 0
Canada
State/province [31] 0 0
Ontario
Country [32] 0 0
Canada
State/province [32] 0 0
Quebec
Country [33] 0 0
Canada
State/province [33] 0 0
Québec
Country [34] 0 0
Czechia
State/province [34] 0 0
Brno
Country [35] 0 0
Czechia
State/province [35] 0 0
Ostrava
Country [36] 0 0
Czechia
State/province [36] 0 0
Praha 10
Country [37] 0 0
Czechia
State/province [37] 0 0
Praha 2
Country [38] 0 0
Czechia
State/province [38] 0 0
Praha
Country [39] 0 0
Denmark
State/province [39] 0 0
Aarhus
Country [40] 0 0
Denmark
State/province [40] 0 0
Koebenhavn Oe
Country [41] 0 0
France
State/province [41] 0 0
Angers Cedex 9
Country [42] 0 0
France
State/province [42] 0 0
Caen Cedex 9
Country [43] 0 0
France
State/province [43] 0 0
Le Mans
Country [44] 0 0
France
State/province [44] 0 0
Paris Cedex 12
Country [45] 0 0
France
State/province [45] 0 0
Paris
Country [46] 0 0
France
State/province [46] 0 0
Pringy Cedex
Country [47] 0 0
Germany
State/province [47] 0 0
Baden-Wuerttemberg
Country [48] 0 0
Germany
State/province [48] 0 0
Bayern
Country [49] 0 0
Germany
State/province [49] 0 0
Niedersachsen
Country [50] 0 0
Germany
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Nordrhein-Westfalen
Country [51] 0 0
Germany
State/province [51] 0 0
Sachsen
Country [52] 0 0
Greece
State/province [52] 0 0
Athens,
Country [53] 0 0
Greece
State/province [53] 0 0
Larisa
Country [54] 0 0
Greece
State/province [54] 0 0
Patra
Country [55] 0 0
Greece
State/province [55] 0 0
Thessaloniki
Country [56] 0 0
Hong Kong
State/province [56] 0 0
Chai Wan
Country [57] 0 0
Hong Kong
State/province [57] 0 0
Hong Kong
Country [58] 0 0
Hong Kong
State/province [58] 0 0
Shatin, New Territories
Country [59] 0 0
Hong Kong
State/province [59] 0 0
Tuen Mun
Country [60] 0 0
Hungary
State/province [60] 0 0
Debrecen
Country [61] 0 0
Hungary
State/province [61] 0 0
Szeged
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Ireland
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Dublin
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Ireland
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Galway
Country [64] 0 0
Ireland
State/province [64] 0 0
James Street
Country [65] 0 0
Ireland
State/province [65] 0 0
Limerick
Country [66] 0 0
Ireland
State/province [66] 0 0
Tallaght, Dublin
Country [67] 0 0
Israel
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Haifa
Country [68] 0 0
Israel
State/province [68] 0 0
Holon
Country [69] 0 0
Israel
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Jerusalem
Country [70] 0 0
Israel
State/province [70] 0 0
Kfar Saba
Country [71] 0 0
Israel
State/province [71] 0 0
Petach-Tikva
Country [72] 0 0
Israel
State/province [72] 0 0
Tel Aviv
Country [73] 0 0
Italy
State/province [73] 0 0
Calabria
Country [74] 0 0
Italy
State/province [74] 0 0
Emilia-Romagna
Country [75] 0 0
Italy
State/province [75] 0 0
Liguria
Country [76] 0 0
Italy
State/province [76] 0 0
Piemonte
Country [77] 0 0
Italy
State/province [77] 0 0
Toscana
Country [78] 0 0
Korea, Republic of
State/province [78] 0 0
Seoul
Country [79] 0 0
Mexico
State/province [79] 0 0
Nuevo León
Country [80] 0 0
Mexico
State/province [80] 0 0
Oaxaca
Country [81] 0 0
Norway
State/province [81] 0 0
Bergen
Country [82] 0 0
Norway
State/province [82] 0 0
Oslo
Country [83] 0 0
Peru
State/province [83] 0 0
Lima
Country [84] 0 0
Poland
State/province [84] 0 0
Krakow
Country [85] 0 0
Poland
State/province [85] 0 0
Legnica
Country [86] 0 0
Poland
State/province [86] 0 0
Lublin
Country [87] 0 0
Poland
State/province [87] 0 0
Opole
Country [88] 0 0
Poland
State/province [88] 0 0
Slupsk
Country [89] 0 0
Poland
State/province [89] 0 0
Torun
Country [90] 0 0
Puerto Rico
State/province [90] 0 0
San Juan
Country [91] 0 0
Russian Federation
State/province [91] 0 0
Kaluga
Country [92] 0 0
Russian Federation
State/province [92] 0 0
Moscow
Country [93] 0 0
Russian Federation
State/province [93] 0 0
Penza
Country [94] 0 0
Russian Federation
State/province [94] 0 0
St Petersburg
Country [95] 0 0
Russian Federation
State/province [95] 0 0
St'Petersburg
Country [96] 0 0
Spain
State/province [96] 0 0
Asturias
Country [97] 0 0
Spain
State/province [97] 0 0
Barcelona
Country [98] 0 0
Spain
State/province [98] 0 0
Gerona
Country [99] 0 0
Spain
State/province [99] 0 0
La Laguna (Santa Cruz De Tenerife)
Country [100] 0 0
Spain
State/province [100] 0 0
Leon
Country [101] 0 0
Spain
State/province [101] 0 0
León
Country [102] 0 0
Spain
State/province [102] 0 0
Madrid
Country [103] 0 0
Spain
State/province [103] 0 0
Majadahonda (Madrid)
Country [104] 0 0
Spain
State/province [104] 0 0
Malaga
Country [105] 0 0
Spain
State/province [105] 0 0
Málaga
Country [106] 0 0
Spain
State/province [106] 0 0
Oviedo
Country [107] 0 0
Spain
State/province [107] 0 0
Palma de Mallorca
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Spain
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Pozuelo De Alarcon/Madrid
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Spain
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Pozuelo De Alarcón/Madrid
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Spain
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Salamanca
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Spain
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Valencia
Country [112] 0 0
Sweden
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Goteborg
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Sweden
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Göteborg
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Sweden
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Stockholm
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Sweden
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Uppsala
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Switzerland
State/province [116] 0 0
Aarau
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Switzerland
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Bellinzona
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Switzerland
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Bern
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Zuerich
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Taiwan
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Changhua
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Taiwan
State/province [121] 0 0
Kaohsiung
Country [122] 0 0
Taiwan
State/province [122] 0 0
Taipei
Country [123] 0 0
Thailand
State/province [123] 0 0
Bangkok
Country [124] 0 0
Thailand
State/province [124] 0 0
ChiangMai
Country [125] 0 0
Turkey
State/province [125] 0 0
Ankara
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Izmir
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Samsun

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Eltrombopag olamine (SB-497115-GR) is an orally bioavailable, small molecule thrombopoietin
receptor agonist that may be beneficial in medical disorders associated with
thrombocytopenia. Eltrombopag has been shown to increase platelet counts in patients with
thrombocytopenia from various etiologies (Idiopathic thrombocytopenic purpura [ITP], liver
disease, aplastic anemia and chemotherapy induced thrombocytopenia). Approximately 350
subjects will be randomized in a 1:1 ratio (175 into the eltrombopag arm and 175 into the
placebo arm). Approximately 55 subjects will be enrolled into the azacitidine. Subjects with
intermediate-1, intermediate-2 or high risk MDS by IPSS, and baseline platelet count of <75
Giga (10^9) per liter (Gi/L) will only be enrolled. This is a randomized, double-blind,
parallel group, placebo-controlled study designed to explore the platelet supportive care
effects of eltrombopag versus placebo in combination with the standard of care
hypomethylating agent, azacitidine. The primary objective of this study is to determine the
effect of eltrombopag versus placebo on the proportion of subjects who are platelet
transfusion free during the first 4 cycles of azacitidine therapy. Key secondary endpoints
include overall survival, disease response, and disease progression.
Trial website
https://clinicaltrials.gov/show/NCT02158936
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02158936