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.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01034657
First received: December 16, 2009
Last updated: July 25, 2013
Last verified: July 2013
  Purpose

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 ] [ Designated as safety issue: No ]

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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]

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®

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01034657

Locations
Germany
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
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov 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
Health Authority: United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by Novartis:
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

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Epoetin Alfa
Histone Deacetylase Inhibitors
Hematinics
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on April 20, 2014