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

This study has been terminated.
(The study was terminated due to lack of efficacy of single agent LBH589 in the 4 month open label core phase and due to enrollment difficulties.)
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01034657
First received: December 16, 2009
Last updated: November 30, 2016
Last verified: November 2016
Results First Received: August 25, 2016  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Myelodysplastic Syndrome (MDS)
Interventions: Drug: LBH589
Drug: Epoetin Alfa

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was divided into a core phase and a randomized phase. The core phase had an open-label single arm study design. The randomized phase was open-label with two parallel treatment arms for participants with stable disease. Participants with Hematological response of the erythropoietin system remained on single agent oral LBH589.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants with stable disease were eligible for randomization to single agent LBH589 or LBH589 + epoetin alfa. Participants with progressive disease were discontinued. Seven participants who completed the core phase withdrew before the randomization phase.

Reporting Groups
  Description
LBH589 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.
LBH589 + Epoetin Alfa 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.
Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.

Participant Flow for 2 periods

Period 1:   Core Phase
    LBH589   LBH589 + Epoetin Alfa   Not Randomized
STARTED   34   0   0 
COMPLETED   20   0   0 
NOT COMPLETED   14   0   0 
Withdrawal by Subject                1                0                0 
Lack of Efficacy                2                0                0 
Abnormal laboratory value                1                0                0 
Adverse Event                8                0                0 
Protocol Violation                2                0                0 

Period 2:   Randomized Phase
    LBH589   LBH589 + Epoetin Alfa   Not Randomized
STARTED   6   6   1 
COMPLETED   1   0   0 
NOT COMPLETED   5   6   1 
Withdrawal by Subject                0                2                0 
Lack of Efficacy                5                1                0 
Abnormal laboratory value                0                1                0 
Adverse Event                0                2                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Core Phase (all participants)

Reporting Groups
  Description
LBH589 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.

Baseline Measures
   LBH589 
Overall Participants Analyzed 
[Units: Participants]
 34 
Age 
[Units: Years]
Mean (Standard Deviation)
 65.4  (7.70) 
Gender 
[Units: Participants]
Count of Participants
 
Female      10  29.4% 
Male      24  70.6% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Percentage of Participants With Hematological Response of the Erythropoetic System (HI-E) - Core Phase   [ Time Frame: 16 weeks ]

2.  Secondary:   Percentage of Participants With HI-E - Randomized Phase   [ Time Frame: 32 weeks, 52 weeks ]

3.  Secondary:   Percentage of Participants With Objective Response During Core Phase   [ Time Frame: 16 weeks ]

4.  Secondary:   Percentage of Participants With Objective Response During the Randomized Phase   [ Time Frame: 32 weeks, 48 weeks ]

5.  Secondary:   Frequency Distribution of IPSS Score Status - Core Phase   [ Time Frame: baseline ]

6.  Secondary:   Frequency Distribution of IPSS Score Status - Randomized Phase   [ Time Frame: 52 weeks ]

7.  Secondary:   Mean Single Scoring Values of the IPSS - Core Phase   [ Time Frame: baseline ]

8.  Secondary:   Mean Single Scoring Values of the IPSS - Randomized Phase   [ Time Frame: 52 weeks ]

9.  Secondary:   Overall Survival (OS) - Overall Period   [ Time Frame: 48 weeks ]

10.  Secondary:   Time to Response - Overall Period   [ Time Frame: 52 weeks ]

11.  Secondary:   Event-free Survival (EFS) - Overall Period   [ Time Frame: 52 weeks ]

12.  Secondary:   Progression-free Survival (PFS) - Overall Period   [ Time Frame: 52 weeks ]

13.  Secondary:   Disease-free Survival (DFS) - Overall Period   [ Time Frame: 52 weeks ]

14.  Secondary:   Time to Cause-specific Death - Overall Period   [ Time Frame: 52 weeks ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis
phone: 862-778-8300



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01034657     History of Changes
Other Study ID Numbers: CLBH589BDE04
EudraCT 2009-010403-84 ( Registry Identifier: EudraCT )
2009-010403-84
Study First Received: December 16, 2009
Results First Received: August 25, 2016
Last Updated: November 30, 2016