LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS) (GEPARD)
Myelodysplastic Syndrome (MDS)
About this trial
This is an interventional treatment trial for Myelodysplastic Syndrome (MDS) focused on measuring MDS, bone marrow, anemia, cytopenia, transfusion dependance, EPO, ESA, erythropoietin, LBH589, Myelodysplastic Syndromes, hematopoietic improvement, IPSS Low, IPSS Int-1, HI-E, HDAC Inhibitor, HDAC-I, DAC-I, Deacetylase-Inhibitor, Histone Deacetylase-Inhibitor, red blood cell transfusions
Eligibility Criteria
Key Inclusion Criteria:
- Patients with a lower risk MDS (LOW or INT-1 according to IPSS)
- Red blood cell transfusion dependency of at least 4 Units/8 weeks.
- Not responding to Erythropoietin stimulating agents (ESA) or having a low chance to do so
- Age-adjusted normal cardiac, kidney, liver function
Key Exclusion Criteria:
- Concomitant use of ESA
- Concomitant use of any other investigational drug
- Other malignancy that is not in remission for at least 1 year
- Platelet Count < 75 x 109/L
- Impaired cardiac function or clinically significant cardiac diseases
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
LBH589
LBH589 + Epoetin Alfa
During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.
During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.