Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes
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|ClinicalTrials.gov Identifier: NCT02912208|
Recruitment Status : Recruiting
First Posted : September 23, 2016
Last Update Posted : January 9, 2018
Myelodysplastic syndromes (MDS) prevail in older age and are characterized by ineffective erythropoiesis and peripheral cytopenias. Supportive therapy is the main therapeutic option for most patients. Quality of Life (QoL) is mainly deteriorated by anemia and by the limitations associated with thrombocytopenia, neutropenia and transfusion dependence. The only available treatment for severe thrombocytopenia, in the presence of bleeding, is platelet transfusion.
Eltrombopag is an orally bioavailable agonist of the thrombopoietin receptor. In adult patients with chronic immune thrombocytopenia (ITP), Eltrombopag rapidly increases platelet counts and significantly reduces bleeding episodes during treatment. Eltrombopag is well tolerated. In 2007, Eltrombopag has received the Orphan Drug Designation for the treatment of ITP (EMEA/OD/031/07), and in 2008 the Food and Drug Association approved Eltrombopag for the treatment of ITP refractory or resistant. It has been shown that in patients affected by MDS and by acute myeloid leukemia, Eltrombopag neither increases the proliferation, nor the clonogenic growth capacity of bone marrow blasts. Furthermore, Eltrombopag induces an increase in the megakaryocytic differentiation and in the formation of normal megakaryocytic colonies. These results provide the rationale for pursuing further research on Eltrombopag for the treatment of thrombocytopenia in case of MDS.
The study is open to adult patients with myelodysplastic syndrome (MDS) with thrombocytopenia and low- or intermediate-1 IPSS risk (Index Prognostic Score System).
Severe thrombocytopenia associated with MDS may lead to death from hemorrhage, even in low prognostic risk patients. The benefit of platelet transfusion is short-termed. Patients become refractory in the long term. The availability of a treatment that induces the increase of platelet count is extremely important, either in terms of quality of life, and in overall survival.
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndromes Thrombocytopenia||Drug: Eltrombopag/Revolade Other: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||174 participants|
|Intervention Model:||Parallel Assignment|
|Official Title:||Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes (EQol-MDS)|
|Study Start Date :||June 2011|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||June 2019|
Experimental: Arm 1 (Eltrombopag)
Arm 1 is the active treatment arm
Eltrombopag 50 mg once daily has been selected as the starting dose for this study. Thereafter, dependent on platelet response the dose of study medication can be increased by 50 mg every 2 weeks, up to a maximum dose of 300 mg once daily (150 mg in subjects of East Asian ethnicity).
Placebo Comparator: Arm 2 (Placebo)
Arm 2 is the control arm
The administration is the same of eltrombopag
- Response rate [ Time Frame: Six months ]Proportion of patients achieving a complete response (CR) or response (R) during the treatment period
- Safety and Tolerability (number of adverse events) [ Time Frame: Six month ]Safety and tolerability in terms of frequency of adverse events (AE) and serious adverse events (SAE)
- Duration of platelet response [ Time Frame: five years ]
- long-term safety and tolerability (number adverse events in the long term) [ Time Frame: five years ]Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 and number of adverse events reporting in accordance with CTCAE v4.0
- Quality of life (QoL) score [ Time Frame: six months ]to evaluate the changes of the quality of life in the two arms
- number of monthly platelet transfusions [ Time Frame: six months ]
- duration of transfusion independence [ Time Frame: six months ]
- time to response [ Time Frame: six months ]time to response (time from starting treatment to time of achievement of CR or PR)
- incidence and severity of bleeding [ Time Frame: six months ]incidence and severity of bleeding using the WHO (World Health Organization)Bleeding Scale
- overall survival [ Time Frame: 2 and 5 years ]overall survival (OS) at 2 and at 5 years
- leukemia-free survival (LFS) [ Time Frame: 2 and 5 years ]leukemia-free survival (LFS) at 2 and at 5 years (events for LFS are defined as death and progression to AML);
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02912208
|Contact: Esther Natalie Oliva||+39 email@example.com|
|Contact: Giuseppe Iannì||+39 firstname.lastname@example.org|
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|Study Chair:||Esther Natalie Oliva||QOL-ONE Associazione Culturale e di Ricerca|