Patients with lower and intermediate-1 risk myelodysplastic syndromes.
Conditions
Interventions
Trade Name: Revolade
Product Name: Eltrombopag
Product Code: SB-497115
Pharmaceutical Form: Coated tablet
INN or Proposed INN: ELTROMBOPAG
CAS Number: 496775-61-2
Current Sponsor code: SB-497115
Conce
Sponsors
University of Crete
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Adult subjects (= 18 years old) with low and intermediate-1 MDS type according to the WHO classification and IPSS. 2. Mean baseline platelet count =65 years) yes F.1.3.1 Number of subjects for this age range 25
Exclusion criteria
Exclusion criteria: 1. Subjects with intermediate-2 and high-risk MDS type according to the WHO classification and IPSS. 2. Patients not eligible for platelet transfusions. 3. Treatment with epigenetic or immunosuppressive therapy within 6 weeks of screening. 4. History of treatment for cancer with systemic chemotherapy and/or radiotherapy within the last 12 months. 5. History of treatment with romiplostim or other TPO-R agonists. 6. Pre-existing cardiovascular disease (including heart failure) or arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation) or subjects with a QTc> 450 msec. 7. Bone marrow fibrosis that leads to an inability to aspirate marrow for assessment. 8. Subjects with Spleen size >16 cm. 9. Leukocytosis (>=25000 uL) prior to day 1 of treatment. 10. Subjects with 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. 11. Previous treatment or known or suspected hypersensitivity to eltrombopag or its components. 12. Pregnant or lactating women. Women of childbearing potential must have a negative pregnancy test which is to be performed within 7 days of dosing. 13. Women of childbearing potential, including women whose last menstrual period was less than one year prior to screening, unable or unwilling to use adequate contraception from study start to one year after the last dose of protocol therapy. Adequate contraception is defined as abstinence, oral hormonal birth control, hormonal birth control injections, implants of etonogestrel or levonorgestrel, estrogenic vaginal ring, percutaneous contraceptive patches, intrauterine device, male partner sterilization if male partner is sole partner for that patient and male condom combined with a female diaphragm, either with or without a vaginal spermicide. 14. Male subjects unable or unwilling to use adequate contraception methods from study start to one year after the last dose of protocol therapy. 15. Current alcohol or drug abuse. 16. Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the 1st dose of study medication. 17. Active and uncontrolled infections. 18. Subjects infected with HepB, HepC or HIV. 19. Subjects with liver cirrhosis.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluation of the proportion of patients with an increase of the platelet count (eltrombopag effect on platelet count), according to the international criteria. Measurement of survival, differentiation and maturation characteristics of BMMCs & megakaryotic progenitor cells throughout the study’s duration.;Primary end point(s): Evaluation of the platelet count throughout the duration of the study and evaluation of the type of response according to international criteria. Measurement of platelet response parameters before treatment and at end of month 3, month 12 and month 24.;Timepoint(s) of evaluation of this end point: Throughout the duration of the study.;Secondary Objective: 1. Safety and tolerability of eltrombopag. 2. Change in bone marrow blast counts from baseline and as well as differences in % of bone marrow blasts between the two groups. 3. Progression of disease. 4. Overall survival. 5. Number of platelet transfusions. 6. Duration of platelet transfusion-independence. 7. Incidence and severity of bleeding. 8. Changes in hemoglobin levels and neutrophil counts. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Clinical examination findings, clinical follow-up, vital signs, laboratory tests and reported adverse events (including bleeding and transfusion-related AEs). • Percentage of blasts in blood or/and bone marrow. • Percentage of neutrophils, platelets, haemoglobin, appearance of chloroma, appearance of B symptoms. • Disease response, disease progression, final outcome. • Number of platelet transfusions. • Duration of platelet transfusion-independence. • Incidence and severity of bleeding (according WHO Bleeding Scale). • Improvement in haemoglobin levels, platelets and neutrophil counts.;Timepoint(s) of evaluation of this end point: Throughout the duration of the study. | — |
Countries
Greece
Contacts
Public ContactHelen Papadaki
University of Crete
Outcome results
None listed