Clinical trial NCT01034657: LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS)
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Myelodysplastic Syndrome (MDS)
Keywords: 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
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2009-12-17
Enrollment: 34
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study assessed 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. The study had a non-randomized core phase followed by a randomized phase.


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

Primary outcomes:
measure: Percentage of Participants With Hematological Response of the Erythropoetic System (HI-E) - Core Phase
timeFrame: 16 weeks
Secondary outcomes:
measure: Percentage of Participants With HI-E - Randomized Phase
timeFrame: 32 weeks, 52 weeks
measure: Percentage of Participants With Objective Response During Core Phase
timeFrame: 16 weeks
measure: Percentage of Participants With Objective Response During the Randomized Phase
timeFrame: 32 weeks, 48 weeks
measure: Frequency Distribution of IPSS Score Status - Core Phase
timeFrame: baseline
measure: Frequency Distribution of IPSS Score Status - Randomized Phase
timeFrame: 52 weeks
measure: Mean Single Scoring Values of the IPSS - Core Phase
timeFrame: baseline
measure: Mean Single Scoring Values of the IPSS - Randomized Phase
timeFrame: 52 weeks
measure: Overall Survival (OS) - Overall Period
timeFrame: 48 weeks
measure: Time to Response - Overall Period
timeFrame: 52 weeks
measure: Event-free Survival (EFS) - Overall Period
timeFrame: 52 weeks
measure: Progression-free Survival (PFS) - Overall Period
timeFrame: 52 weeks
measure: Disease-free Survival (DFS) - Overall Period
timeFrame: 52 weeks
measure: Time to Cause-specific Death - Overall Period
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Mannheim
country: Germany
facility: Novartis Investigative Site
state: Baden-Württemberg
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Bonn
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Duisburg
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Goettingen
country: Germany
facility: Novartis Investigative Site
city: Hannover
country: Germany
facility: Novartis Investigative Site
city: Leipzig
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Ulm
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: