Clinical trial NCT00880269: Efficacy and Safety of Panobinostat (LBH589) in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
Official title: A Phase II Study of Oral Single Agent Panobinostat in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Refractory Leukemia
Acute Myelogenous Leukemia
Keywords: Acute myeloid leukemia
AML
relapsed acute myeloid leukemia
refractory AML
refractory de novo AML
refractory secondary AML
secondary AML
AML following myelodysplastic syndrome (MDS)
AML following antecedent hematological disorder (AHD)
AML resistant to therapy
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2009-04-13
Enrollment: 59
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study was to evaluate the efficacy and safety of single agent oral panobinostat in patients who have refractory de novo or refractory secondary AML.


Eligibility criteria: Inclusion Criteria:

* Written informed consent prior to study-specific screening procedures
* Life expectancy of ≥ 60 days
* Eastern Cooperative Group (ECOG) performance status ≤ 2
* Refractory AML with confirmed initial diagnosis of de novo AML (excluding APL) - OR- Refractory AML with confirmed initial diagnosis of AML (excluding APL) secondary to AHD or MDS with either condition precedent to AML (MDS/AHD)
* Negative serum pregnancy test (within 7 days of first dose)
* Negative urine pregnancy test immediately prior to first dose

Exclusion Criteria:

* Known HIV
* Psychiatric disorder that interfered with ability to understand the study and give informed consent, and/or would impact study participation or follow-up
* Concurrent use of medications that might prolong the QT interval or of inducing Torsade de Pointes
* Female patients who were pregnant or breast-feeding or patients of childbearing potential who were not willing to use a double barrier method of contraception during the study and for 3 months following the last dose of study drug.
* Male patients whose sexual partner(s) were women of childbearing potential who were not willing to use a double method of contraception, one of which included a condom, during the study and for 3 months after the end of treatment
* Patient unable to swallow capsules
* Patients with impaired gastrointestinal systems which might cause interference with digesting and absorbing panobinostat

Other Protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Best Response as Per Investigator Assessment by Stratum (FAS)
timeFrame: 6 cycles of treatment with a 28-day treatment cycle (Day 168)
Secondary outcomes:
measure: Partial Response Measured in Stratum A and B
timeFrame: 6 treatment cycles (28-day/treatment cycle)
measure: Time to Remission Measured in Stratum A and B
timeFrame: 6 treatment cycles (28-day/treatment cycle)
measure: Duration of Remission Measured in Stratum A and B
timeFrame: 6 treatment cycles (28-day/treatment cycle)
measure: Event-free Survival Measured in Stratum A and B
timeFrame: 6 treatment cycles (28-day/treatment cycle)
measure: Overall Survival Measured in Stratum A and B
timeFrame: 6 treatment cycles (28-day/treatment cycle)
Other outcomes:
Locations:
city: Chicago
country: United States
facility: University of Chicago Medical Center Dept. of U. of Chicago Hosp(3)
state: Illinois
city: Omaha
country: United States
facility: Nebraska Methodist Hospital Nebraska Methodist Hospital(2)
state: Nebraska
city: Manhasset
country: United States
facility: North Shore University Hospital North Shore Univ
state: New York
city: NY
country: United States
facility: Memorial Sloan Kettering Cancer Center Dept. of MSKCC (2)
state: New York
city: Portland
country: United States
facility: Oregon Health Sciences University Dept. of OHSU (2)
state: Oregon
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center Dept of Simmons Cancer Center
state: Texas
city: Houston
country: United States
facility: University of Texas/MD Anderson Cancer Center Dept of MD Anderson (14)
state: Texas
city: Gosford
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Herston
country: Australia
facility: Novartis Investigative Site
state: Queensland
city: Adelaide
country: Australia
facility: Novartis Investigative Site
state: South Australia
city: Fitzroy
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Parkville
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Prahran
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Brugge
country: Belgium
facility: Novartis Investigative Site
city: Gent
country: Belgium
facility: Novartis Investigative Site
city: Liege
country: Belgium
facility: Novartis Investigative Site
city: Bobigny Cedex
country: France
facility: Novartis Investigative Site
city: Montpellier cedex 5
country: France
facility: Novartis Investigative Site
city: Nantes
country: France
facility: Novartis Investigative Site
city: Paris Cedex 4
country: France
facility: Novartis Investigative Site
city: Toulouse cedex 9
country: France
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
state: Nordrhein-Westfalen
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Leipzig
country: Germany
facility: Novartis Investigative Site
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Udine
country: Italy
facility: Novartis Investigative Site
state: UD
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
state: Korea
city: Surquillo
country: Peru
facility: Novartis Investigative Site
state: Lima
city: Hospitalet de LLobregat
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Barcelona
country: Spain
facility: Novartis Investigative Site
city: Bern
country: Switzerland
facility: Novartis Investigative Site
city: Zürich
country: Switzerland
facility: Novartis Investigative Site
city: Ankara
country: Turkey
facility: Novartis Investigative Site
city: Balcova / Izmir
country: Turkey
facility: Novartis Investigative Site
city: Aberdeen
country: United Kingdom
facility: Novartis Investigative Site
state: Scotland
city: Leeds
country: United Kingdom
facility: Novartis Investigative Site
state: West Yorkshire
city: Liverpool
country: United Kingdom
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: