LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk MDS (GEPARD)

This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: December 16, 2009
Last updated: July 25, 2013
Last verified: July 2013
This study will assess the efficacy and safety of LBH589 as single agent and in combination with ESA in red blood cell transfusion-dependent Low and Int-1 MDS patients being either refractory to ESA or with a low probability of response.

Condition Intervention Phase
Myelodysplastic Syndrome (MDS)
Drug: oral LBH589/panobinostat
Drug: oral LBH589/panobinostat + Epoetin Alfa HEXAL®
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A One Year, Open Label, Multicenter Trial of LBH589 Alone or in Combination With ESA in Red Blood Cell Transfusion-dependent LOW and INT-1 MDS Patients Being Either Refractory to ESA or With a Low Probability of Response - the GErman PAnobinostat Low Risk MDS Trial - GEPARD Study

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Evaluation of the hematological improvement of the erythropoietic system (HI-E) using modified IWG criteria (Cheson 2006) in patients treated with LBH589 single agent [ Time Frame: 16 weeks ]

Secondary Outcome Measures:
  • Comparison of the hematological improvement of the erythropoietic system (HI-E) using modified IWG criteria (Cheson 2006) in patients treated with either LBH589 single agent or with LBH589 and ESA combination treatment. [ Time Frame: up to 48 weeks ]
  • Evaluation of the objective response rate (CR + PR and HI-P and HI-N) according to modified IWG criteria (Cheson et al.). [ Time Frame: up to 48 weeks ]
  • Determination of the IPSS status as well as the single scoring values of the IPSS for patients at baseline and EOS (end of study). [ Time Frame: up to 48 weeks ]
  • Determination of the time to response, event-free survival, progression-free survival (PFS), disease-free survival (DFS), time to cause-specific death, and overall survival (OS) in this patient population. [ Time Frame: up to 48 weeks ]
  • Evaluation of the safety and tolerability profile of LBH589 and LBH589 + ESA in low and INT-1 risk MDS patients [ Time Frame: up to 48 weeks ]

Enrollment: 33
Study Start Date: November 2009
Study Completion Date: August 2012
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LBH589 Drug: oral LBH589/panobinostat
Experimental: LBH589 + Epoetin Alfa Drug: oral LBH589/panobinostat + Epoetin Alfa HEXAL®


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01034657

Novartis Investigative Site
Mannheim, Baden-Württemberg, Germany, 68305
Novartis Investigative Site
Berlin, Germany, 12200
Novartis Investigative Site
Bonn, Germany, 53105
Novartis Investigative Site
Dresden, Germany, 01307
Novartis Investigative Site
Duesseldorf, Germany, 40225
Novartis Investigative Site
Duisburg, Germany, 47166
Novartis Investigative Site
Frankfurt, Germany, 60590
Novartis Investigative Site
Göttingen, Germany, D-37075
Novartis Investigative Site
Hannover, Germany, 30625
Novartis Investigative Site
Leipzig, Germany, 04103
Novartis Investigative Site
Muenchen, Germany, 81675
Novartis Investigative Site
Ulm, Germany, 89081
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals Identifier: NCT01034657     History of Changes
Other Study ID Numbers: CLBH589BDE04  EudraCT 2009-010403-84  2009-010403-84 
Study First Received: December 16, 2009
Last Updated: July 25, 2013

Keywords provided by Novartis:
bone marrow
transfusion dependance
Myelodysplastic Syndromes
hematopoietic improvement
IPSS Int-1
HDAC Inhibitor
Histone Deacetylase-Inhibitor
red blood cell transfusions

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Histone Deacetylase Inhibitors
Epoetin Alfa
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Hematinics processed this record on January 19, 2017