A open-label, multi-center 2-year safety study to ascertain the baseline levels of bone
marrow fibers in previously treated adults with chronic immune (idiopathic) thrombocytopenic
purpura (ITP) and to evaluate the long-term effect of eltrombopag on bone marrow fibers. The
study will also describe the long-term safety and tolerability of oral eltrombopag treatment
in subjects with chronic ITP.
The duration of the screening period is up to 8 weeks. Eltrombopag will be administered for
at least 2-years followed by a 4-week follow-up period. Bone marrow biopsies will be
performed at screening, after 1-year and 2-years of eltrombopag treatment, and at early
withdrawal of treatment. The screening bone marrow biopsy should be performed within 8 weeks
of planned start of study medication and the bone marrow biopsy block must be available for
central laboratory processing.
Inclusion Criteria:
- Subjects must have signed and dated a written informed consent and be able to
understand and comply with protocol requirements and instructions.
- Adults (≥18 years) diagnosed with chronic ITP according to the American Society for
Hematology/British Committee for Standards in Hematology (ASH/BCSH) guidelines
[George, 1996; BCSH, 2003; Provan, 2009]. In addition, a peripheral blood smear
should support the diagnosis of ITP with no evidence of other disease causative of
thrombocytopenia (e. g., pseudo thrombocytopenia, myelofibrosis). The physical
examination should not suggest any disease, which may cause thrombocytopenia other
than ITP.
- Subjects must be physically eligible for serial bone marrow biopsies and must have a
bone marrow biopsy performed during screening, and be willing to remain on the study
for at least 2 years with annual bone marrow biopsies.
- Subjects, who previously received eltrombopag or romiplostim, must have completed
treatment with these therapies at least 6 months prior to the screening bone marrow
biopsy.
- Subjects must have the following clinical chemistry values:
- ALT and AST < 2xULN;
- Bilirubin <1. 5xULN (except for Gilbert's Syndrome);
- Subjects are practicing an acceptable method of contraception as specified in the
protocol.
- In France, subjects will be eligible for inclusion in this study, only if either
affiliated to or a beneficiary of a social security category.
Exclusion Criteria:
- Subjects with any clinically relevant abnormality, other than ITP, or any other
medical condition or circumstance, which in the opinion of the investigator makes the
subject unsuitable for participation in the study or suggests another primary
diagnosis (e. g., thrombocytopenia is secondary to another disease).
- Subjects with any concurrent malignant disease and/or a recent history of cancer
treatment with systemic chemotherapy and/or radiotherapy.
Exception: Subjects with a history of completely resected non-melanoma skin cancer or
successfully treated in situ carcinoma are eligible.
- Subjects with any prior history of arterial or venous thrombosis (stroke, transient
ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism),
AND ≥ two of the following risk factors: hormone replacement therapy, systemic
contraception (containing estrogen), smoking, diabetes, hypercholesterolemia,
hypertension, cancer, hereditary thrombophilic disorders (e. g., Factor V Leiden,
ATIII deficiency, etc), or any family history of arterial or venous thrombosis.
- Subjects with screening bone marrow fibers of either MF Grade 3 using European
Consensus scale or Grade 4 using Bauermeister scale.
- Subjects with a QTc >450 msec or > 480 msec for subjects with Bundle Branch Block.
- Female subjects who are nursing or pregnant (positive serum or urine β-human
chorionic gonadotrophin (β-hCG) pregnancy test) at screening.
- Subjects treated with an investigational drug (other than a thrombopopoetin-receptor
(TPO-R) agonist) within 30 days or five half-lives (whichever is longer) preceding
the first dose of eltrombopag in the study. (For romiplostim or eltrombopag, see
inclusion criterion #4).
- Subjects treated with any TPO-R agonist other than romiplostim or eltrombopag.
- Subjects with recent history of alcohol/drug abuse as determined by the investigator.
US GSK Clinical Trials Call Center, Phone: 877-379-3718, Email: GSKClinicalSupportHD@gsk.com
GSK Investigational Site, Hradec Kralove, Czech Republic; Recruiting
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GSK Investigational Site, Olomouc 775 20, Czech Republic; Recruiting
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GSK Investigational Site, Praha 2 128 08, Czech Republic; Recruiting
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GSK Investigational Site, Caen cedex 9 14033, France; Not yet recruiting
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GSK Investigational Site, Créteil 94010, France; Active, not recruiting
GSK Investigational Site, Pessac Cedex 33604, France; Recruiting
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GSK Investigational Site, Berlin 13353, Germany; Recruiting
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GSK Investigational Site, Shatin, New Territories, Hong Kong; Recruiting
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GSK Investigational Site, Debrecen 4012, Hungary; Recruiting
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GSK Investigational Site, Győr 9023, Hungary; Recruiting
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GSK Investigational Site, Szeged 6720, Hungary; Recruiting
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GSK Investigational Site, Ludhiana 141008, India; Recruiting
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GSK Investigational Site, Pune 411004, India; Recruiting
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GSK Investigational Site, Surat 395002, India; Recruiting
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GSK Investigational Site, Vellore 632004, India; Recruiting
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GSK Investigational Site, Seongnam-si Gyeonggi-do 463-712, Korea, Republic of; Active, not recruiting
GSK Investigational Site, Seoul 137-701, Korea, Republic of; Active, not recruiting
GSK Investigational Site, Seoul 135-710, Korea, Republic of; Active, not recruiting
GSK Investigational Site, Karachi 75300, Pakistan; Recruiting
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GSK Investigational Site, Lahore 54600, Pakistan; Recruiting
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GSK Investigational Site, Moscow 125167, Russian Federation; Recruiting
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GSK Investigational Site, Novosibirsk 630087, Russian Federation; Recruiting
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GSK Investigational Site, St Petersburg 193024, Russian Federation; Recruiting
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GSK Investigational Site, Muenchen, Bayern 81377, Germany; Not yet recruiting
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GSK Investigational Site, Muenchen, Bayern 81241, Germany; Recruiting
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GSK Investigational Site, Bologna, Emilia-Romagna 40138, Italy; Recruiting
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GSK Investigational Site, Milano, Lombardia 20162, Italy; Not yet recruiting
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GSK Investigational Site, Milano, Lombardia 20132, Italy; Completed
GSK Investigational Site, Duisburg, Nordrhein-Westfalen 47166, Germany; Recruiting
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GSK Investigational Site, Padova, Veneto 35128, Italy; Recruiting
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GSK Investigational Site, Vicenza, Veneto 36100, Italy; Recruiting
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