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

Novartis

Status and phase: Terminated; Phase 2

Conditions: Myelodysplastic Syndrome (MDS)

Treatments: Drug: Epoetin Alfa; Drug: LBH589

Study type: Interventional

Funder types: Industry

Identifiers

NCT01034657

EudraCT 2009-010403-84 (Registry Identifier)

CLBH589BDE04

2009-010403-84

Details and patient eligibility

About

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.

Enrollment: 34 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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34 participants in 2 patient groups

LBH589

Experimental group

Description:

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.

Treatment:

Drug: LBH589

LBH589 + Epoetin Alfa

Experimental group

Description:

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.

Treatment:

Drug: LBH589

Drug: Epoetin Alfa