Eltrombopag for the treatment of thrombocytopenia due to low- and intermediate risk myelodysplastic syndromes.

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 174

Inclusion Criteria

1. Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and stable disease.
2. Subjects must have a platelet count taken within the 4 weeks prior to randomization that is <30 Gi/L.
3. Subjects must be ineligible or relapsed or refractory to receive other treatment options (such as azacitidine or lenalidomide) and must be ineligible to receive intensive chemotherapy or autologous/allogeneic stem cell transplantation.
4. Subjects must have platelet count and platelet transfusion data available over a
period of 8 weeks prior to randomization.
5. During the 2 months prior to randomization, subjects must have a baseline BM
examination which includes cytomorphology and cytogenetics. Histopathology
should be performed.
6. Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte colonystimulating
factor (G-CSF) in subjects with severe neutropenia and recurrent
infections are allowed during the study as per accepted standards [23]. Subjects
who enter the study on ESAs or G-CSF should continue at the same dose schedule
until the optimal dose of study medication has been established.
7. ECOG Performance Status 0-3 (Appendix 4).
8. Subject is able to understand and comply with protocol requirements and instructions.
9. Subject has signed and dated informed consent.
10. Adequate baseline organ function defined by the criteria below:
total bilirubin (except for Gilbert’s Syndrome) = 1.5xULN
ALT and AST = 3xULN
creatinine = 2xULN
albumin must not be below the lower limit of normal by more than 20%.
11. Subject is practicing an acceptable method of contraception. Female subjects (or female partners of male subjects) must either be of non-childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal >1 year), or of childbearing potential and use of an highly effective method of contraception from 2 weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 87
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 87

Exclusion Criteria

1. MDS with intermediate-2 or high IPSS risk
2. History of treatment for cancer other than MDS with systemic chemotherapy and/or radiotherapy within the last 2 years.
3. History of treatment with romiplostim or other TPO-R agonists.
4. Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association [NYHA] Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. persistent atrial fibrillation), or subjects with a QTc >450 msec (QTc >480 msec for subjects with Bundle Branch Block).
5. BM fibrosis that leads to an inability to aspirate marrow for assessment.
6. Peripheral monocytosis > 1000/uL prior to Day 1 of study medication.
7. Leukocytosis >=25,000/uL prior to Day 1 of study medication.
8. Female subjects who are nursing or pregnant (positive serum or urine Beta-human chorionic gonadotropin [B-hCG] pregnancy test) at screening or pre-dose on Day 1.
9. Current alcohol or drug abuse.
10. Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
11. Active and uncontrolled infections.
12. Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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