Trial Outcomes & Findings for Study of LBH589 for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) (NCT NCT00939159)

Last Updated: 2018-08-23

Results Overview

Overall response rate defined by International Working Group (IWG) response criteria in myelodysplasia. Hematologic Improvement (HI) responses, at least 9 weeks: Erythroid response (pretreatment, \<11 g/dL): Hgb increase by 1.5 g/dL; Relevant reduction units of Red blood cell (RBC) transfusions by absolute number at least 4 RBC transfusions/8 week compared with pretreatment transfusion number in previous 8 weeks; Only RBC transfusions for Hgb of 9.0 g/dL pretreatment count in RBC transfusion response evaluation; Platelet response (pretreatment,\<100x10\^9/L): If starting with \>20x10\^9/L platelets: absolute increase 30x10\^9/L, Increase from baseline \<20 x10\^9/L to \>20x10\^9/L and by =/\> 100%; Neutrophil response (pretreatment, \<1.0x10\^9/L): =/\> 100% increase \& absolute increase \>0.5x10\^9/L; Progression or relapse after HI: At least 1 of the following: =/\>50% decrement from max response levels in granulocytes or platelets; Reduction in Hgb by 1.5 g/dL; or Transfusion dependence .

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

Assessment with 28-day cycle until response, then every 3 cycles as needed, for up to 24 months

Results posted on

2018-08-23

Participant Flow

Recruitment Period: August 05, 2009 to March 10, 2011. All recruitment done at The University of Texas (UT) MD Anderson Cancer Center.

Of the seventeen (17) participants enrolled, four were excluded prior to receiving treatment on the study.

Participant milestones

Participant milestones
Measure	LBH589 LBH589 20 mg capsules by mouth 3 times a week for 3 weeks in a 28-day cycle.
Overall Study STARTED	13
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of LBH589 for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	LBH589 n=13 Participants LBH589 20 mg capsules by mouth 3 times a week for 3 weeks in a 28-day cycle.
Age, Continuous	70 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Region of Enrollment United States	13 participants n=5 Participants

PRIMARY outcome

Timeframe: Assessment with 28-day cycle until response, then every 3 cycles as needed, for up to 24 months

Population: Of the seventeen participants registered, four were not treated making thirteen evaluable for response.

Outcome measures

Outcome measures
Measure	LBH589 n=13 Participants LBH589 20 mg capsules by mouth 3 times a week for 3 weeks in a 28-day cycle.
Overall Response Rate Based on the Hematologic Improvement	8 Percentage of Participants

Adverse Events

LBH589

Serious events: 4 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	LBH589 n=13 participants at risk LBH589 20 mg capsules by mouth 3 times a week for 3 weeks in a 28-day cycle.
Musculoskeletal and connective tissue disorders Disc Protrusion	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Cardiac disorders QTc Prolongation	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Infections and infestations Cholecystitis	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
General disorders Death/Disease Progression	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Nervous system disorders Confusion	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.

Other adverse events

Other adverse events
Measure	LBH589 n=13 participants at risk LBH589 20 mg capsules by mouth 3 times a week for 3 weeks in a 28-day cycle.
General disorders Fatigue	15.4% 2/13 • Number of events 2 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Gastrointestinal disorders Decreased appetite	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Skin and subcutaneous tissue disorders Rash	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Nervous system disorders Dizziness	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Respiratory, thoracic and mediastinal disorders Dyspnea	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Gastrointestinal disorders Diarrhea	7.7% 1/13 • Number of events 1 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Gastrointestinal disorders Nausea	15.4% 2/13 • Number of events 2 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.
Metabolism and nutrition disorders Hyperbilirubinemia	15.4% 2/13 • Adverse event collected for eligible participants each three week treatment cycle. Overall study period: August 06, 2009 to March 17, 2011.

Additional Information

Guillermo Garcia-Manero, MD / Associate Professor

UT MD Anderson Cancer Center

Phone: 713-792-7305

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place