NCT00880269

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.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
59

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Aug 2009

Geographic Reach
12 countries

40 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 30, 2009

Completed
14 days until next milestone

First Posted

Study publicly available on registry

April 13, 2009

Completed
4 months until next milestone

Study Start

First participant enrolled

August 1, 2009

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2012

Completed
4.6 years until next milestone

Results Posted

Study results publicly available

September 9, 2016

Completed
Last Updated

February 23, 2017

Status Verified

January 1, 2017

Enrollment Period

2.5 years

First QC Date

March 30, 2009

Results QC Date

July 21, 2016

Last Update Submit

January 8, 2017

Conditions

Refractory LeukemiaAcute Myelogenous Leukemia

Keywords

Acute myeloid leukemiaAMLrelapsed acute myeloid leukemiarefractory AMLrefractory de novo AMLrefractory secondary AMLsecondary AMLAML following myelodysplastic syndrome (MDS)AML following antecedent hematological disorder (AHD)AML resistant to therapy

Outcome Measures

Primary Outcomes (1)

  • Best Response as Per Investigator Assessment by Stratum (FAS)

    Response to treatment was defined as complete remission rate (CRR). CRR is complete remission (CR) and morphologic CR with incomplete blood count recovery (residual neutropenia or thrombocytopenia) (CRi). To stop or to proceed with Stage 2 of a given stratum of the Simon's optimal 2-stage design was based on the number of patients with CR/CRi and a safety evaluation of the patients from Stage 1 in that stratum. If early results clearly indicated that the drug was not active or worthy of further investigation, enrollment of that particular stratum would be terminated. CR and CRi were assessed by the Investigator according to IWG response Criteria for AML (Cheson et al 2003). As per protocol we would continue to stage II if ≥ 4 patients out of 26 patients enrolled to stage I had a CR or a CRi. As per response observed there was only 1 patient with CR/CRi in stratum A and 2 patients with CR/CRi in Stratum B.

    6 cycles of treatment with a 28-day treatment cycle (Day 168)

Secondary Outcomes (5)

  • Partial Response Measured in Stratum A and B

    6 treatment cycles (28-day/treatment cycle)

  • Time to Remission Measured in Stratum A and B

    6 treatment cycles (28-day/treatment cycle)

  • Duration of Remission Measured in Stratum A and B

    6 treatment cycles (28-day/treatment cycle)

  • Event-free Survival Measured in Stratum A and B

    6 treatment cycles (28-day/treatment cycle)

  • Overall Survival Measured in Stratum A and B

    6 treatment cycles (28-day/treatment cycle)

Study Arms (2)

Stratum A

EXPERIMENTAL

patients with refractory acute myelogenous leukemia (AML) initially diagnosed as de novo AML received 60 mg of panobinostat per day on three discontinuous days per week.

Drug: Panobinostat/LBH589

Stratum B

EXPERIMENTAL

patients with refractory AML initially diagnosed as AML secondary to myelodysplastic syndrome (MDS)/antecedent hematologic disorder (AHD) received 60 mg of panobinostat per day on three discontinuous days per week.

Drug: Panobinostat/LBH589

Interventions

Panobinostat/LBH589DRUG
Stratum AStratum B

Eligibility Criteria

Age18 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (40)

University of Chicago Medical Center Dept. of U. of Chicago Hosp(3)

Chicago, Illinois, 60637, United States

Location

Nebraska Methodist Hospital Nebraska Methodist Hospital(2)

Omaha, Nebraska, 68114, United States

Location

North Shore University Hospital North Shore Univ

Manhasset, New York, 10030, United States

Location

Memorial Sloan Kettering Cancer Center Dept. of MSKCC (2)

New York, New York, 10065, United States

Location

Oregon Health Sciences University Dept. of OHSU (2)

Portland, Oregon, 97239, United States

Location

University of Texas Southwestern Medical Center Dept of Simmons Cancer Center

Dallas, Texas, 75390, United States

Location

University of Texas/MD Anderson Cancer Center Dept of MD Anderson (14)

Houston, Texas, 77030-4009, United States

Location

Novartis Investigative Site

Gosford, New South Wales, 2250, Australia

Location

Novartis Investigative Site

Herston, Queensland, 4029, Australia

Location

Novartis Investigative Site

Adelaide, South Australia, 5000, Australia

Location

Novartis Investigative Site

Fitzroy, Victoria, 3065, Australia

Location

Novartis Investigative Site

Parkville, Victoria, 3002, Australia

Location

Novartis Investigative Site

Prahran, Victoria, 3181, Australia

Location

Novartis Investigative Site

Bruges, 8000, Belgium

Location

Novartis Investigative Site

Ghent, 9000, Belgium

Location

Novartis Investigative Site

Liège, 4000, Belgium

Location

Novartis Investigative Site

Bobigny, 93009, France

Location

Novartis Investigative Site

Montpellier, 34295, France

Location

Novartis Investigative Site

Nantes, 44035, France

Location

Novartis Investigative Site

Paris, 75181, France

Location

Novartis Investigative Site

Toulouse, 31059, France

Location

Novartis Investigative Site

Cologne, North Rhine-Westphalia, 50937, Germany

Location

Novartis Investigative Site

Frankfurt, 60590, Germany

Location

Novartis Investigative Site

Leipzig, 04103, Germany

Location

Novartis Investigative Site

Roma, RM, 00133, Italy

Location

Novartis Investigative Site

Roma, RM, 00161, Italy

Location

Novartis Investigative Site

Udine, UD, 33100, Italy

Location

Novartis Investigative Site

Surquillo, Lima region, 34, Peru

Location

Novartis Investigative Site

Seoul, Korea, 06591, South Korea

Location

Novartis Investigative Site

Barcelona, Barcelona, 08041, Spain

Location

Novartis Investigative Site

L'Hospitalet de Llobregat, Catalonia, 08907, Spain

Location

Novartis Investigative Site

Zurich, Switzerland, 8091, Switzerland

Location

Novartis Investigative Site

Bern, 3010, Switzerland

Location

Novartis Investigative Site

Ankara, Turkey, 06100, Turkey (Türkiye)

Location

Novartis Investigative Site

Balcova / Izmir, 35340, Turkey (Türkiye)

Location

Novartis Investigative Site

Aberdeen, Scotland, AB25 2ZN, United Kingdom

Location

Novartis Investigative Site

Leeds, West Yorkshire, LS9 7TF, United Kingdom

Location

Novartis Investigative Site

Liverpool, L7 8XP, United Kingdom

Location

Novartis Investigative Site

London, SE5 9RS, United Kingdom

Location

Novartis Investigative Site

London, W12 0HS, United Kingdom

Location

Related Links

MeSH Terms

Conditions

LeukemiaLeukemia, Myeloid, Acute

Interventions

Panobinostat

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, Myeloid

Intervention Hierarchy (Ancestors)

Hydroxamic AcidsHydroxylaminesAminesOrganic ChemicalsHydroxy AcidsCarboxylic AcidsIndolesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals
862-778-8300

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 30, 2009

First Posted

April 13, 2009

Study Start

August 1, 2009

Primary Completion

February 1, 2012

Study Completion

February 1, 2012

Last Updated

February 23, 2017

Results First Posted

September 9, 2016

Record last verified: 2017-01

Locations

KR(1)AU(6)US(7)IT(3)FR(5)BE(3)PE(1)ES(2)CH(2)TU(2)DE(3)GB(5)