Myelodysplastic syndromes
Conditions
Brief summary
Phase 1. Proportion of patients obtaining CR or R during the six month treatment period., Phase 1. Safety and tolerability parameters including non-hematological laboratory Grade 3/Grade 4 toxicities, change in bone marrow blast counts from baseline and adverse events., Phase 2. Duration of platelet response., Phase 1. Long-term safety and tolerability.
Detailed description
Changes in quality of life (QoL) scores., Frequency of platelet transfusions during the treatment and follow-up periods., Duration of platelet transfusion independence., Difference in time to response (time from starting treatment to time of achievement of CR or R)., Duration of response during the treatment and follow-up periods., Incidence and severity of bleeding using the WHO Bleeding Scale., OS and LFS., Eltrombopag population pharmacokinetic parameters and plasma concentration data. The relationship between eltrombopag pharmacokinetics and relevant safety and efficacy endpoints will be explored, as data permit., Phase 1 primary endpoints have been reached and have been publish (Lancet Haematol. 2017 Mar;4(3):e127-e136)
Interventions
DRUGRevolade 25 mg film-coated tablets
Sponsors
Associazione Qol-One
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase 1. Proportion of patients obtaining CR or R during the six month treatment period., Phase 1. Safety and tolerability parameters including non-hematological laboratory Grade 3/Grade 4 toxicities, change in bone marrow blast counts from baseline and adverse events., Phase 2. Duration of platelet response., Phase 1. Long-term safety and tolerability. | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in quality of life (QoL) scores., Frequency of platelet transfusions during the treatment and follow-up periods., Duration of platelet transfusion independence., Difference in time to response (time from starting treatment to time of achievement of CR or R)., Duration of response during the treatment and follow-up periods., Incidence and severity of bleeding using the WHO Bleeding Scale., OS and LFS., Eltrombopag population pharmacokinetic parameters and plasma concentration data. The relationship between eltrombopag pharmacokinetics and relevant safety and efficacy endpoints will be explored, as data permit., Phase 1 primary endpoints have been reached and have been publish (Lancet Haematol. 2017 Mar;4(3):e127-e136) | — |
Countries
Italy
Outcome results
None listed