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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00880269




Registration number
NCT00880269
Ethics application status
Date submitted
30/03/2009
Date registered
13/04/2009
Date last updated
23/02/2017

Titles & IDs
Public title
Efficacy and Safety of Panobinostat (LBH589) in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
Scientific title
A Phase II Study of Oral Single Agent Panobinostat in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
Secondary ID [1] 0 0
2008-002983-32
Secondary ID [2] 0 0
CLBH589B2213
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Refractory Leukemia 0 0
Acute Myelogenous Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Panobinostat/LBH589

Experimental: Stratum A - 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.

Experimental: Stratum B - 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.


Treatment: Drugs: Panobinostat/LBH589


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
6 cycles of treatment with a 28-day treatment cycle (Day 168)
Secondary outcome [1] 0 0
Partial Response Measured in Stratum A and B - As predefined in the study's protocol, stage II was not pursued due to lack of activity (at end of stage I less than 4 patients in each stratum with CR/CRi)
Timepoint [1] 0 0
6 treatment cycles (28-day/treatment cycle)
Secondary outcome [2] 0 0
Time to Remission Measured in Stratum A and B
Timepoint [2] 0 0
6 treatment cycles (28-day/treatment cycle)
Secondary outcome [3] 0 0
Duration of Remission Measured in Stratum A and B
Timepoint [3] 0 0
6 treatment cycles (28-day/treatment cycle)
Secondary outcome [4] 0 0
Event-free Survival Measured in Stratum A and B
Timepoint [4] 0 0
6 treatment cycles (28-day/treatment cycle)
Secondary outcome [5] 0 0
Overall Survival Measured in Stratum A and B
Timepoint [5] 0 0
6 treatment cycles (28-day/treatment cycle)

Eligibility
Key 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
Minimum age
18 Years
Maximum age
85 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Gosford
Recruitment hospital [2] 0 0
Novartis Investigative Site - Herston
Recruitment hospital [3] 0 0
Novartis Investigative Site - Adelaide
Recruitment hospital [4] 0 0
Novartis Investigative Site - Fitzroy
Recruitment hospital [5] 0 0
Novartis Investigative Site - Parkville
Recruitment hospital [6] 0 0
Novartis Investigative Site - Prahran
Recruitment postcode(s) [1] 0 0
2250 - Gosford
Recruitment postcode(s) [2] 0 0
4029 - Herston
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment postcode(s) [4] 0 0
3065 - Fitzroy
Recruitment postcode(s) [5] 0 0
3002 - Parkville
Recruitment postcode(s) [6] 0 0
3181 - Prahran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Nebraska
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Oregon
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
Belgium
State/province [6] 0 0
Brugge
Country [7] 0 0
Belgium
State/province [7] 0 0
Gent
Country [8] 0 0
Belgium
State/province [8] 0 0
Liege
Country [9] 0 0
France
State/province [9] 0 0
Bobigny Cedex
Country [10] 0 0
France
State/province [10] 0 0
Montpellier cedex 5
Country [11] 0 0
France
State/province [11] 0 0
Nantes
Country [12] 0 0
France
State/province [12] 0 0
Paris Cedex 4
Country [13] 0 0
France
State/province [13] 0 0
Toulouse cedex 9
Country [14] 0 0
Germany
State/province [14] 0 0
Nordrhein-Westfalen
Country [15] 0 0
Germany
State/province [15] 0 0
Frankfurt
Country [16] 0 0
Germany
State/province [16] 0 0
Leipzig
Country [17] 0 0
Italy
State/province [17] 0 0
RM
Country [18] 0 0
Italy
State/province [18] 0 0
UD
Country [19] 0 0
Korea, Republic of
State/province [19] 0 0
Korea
Country [20] 0 0
Peru
State/province [20] 0 0
Lima
Country [21] 0 0
Spain
State/province [21] 0 0
Catalunya
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Switzerland
State/province [23] 0 0
Bern
Country [24] 0 0
Switzerland
State/province [24] 0 0
Zürich
Country [25] 0 0
Turkey
State/province [25] 0 0
Ankara
Country [26] 0 0
Turkey
State/province [26] 0 0
Balcova / Izmir
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Scotland
Country [28] 0 0
United Kingdom
State/province [28] 0 0
West Yorkshire
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Liverpool
Country [30] 0 0
United Kingdom
State/province [30] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/show/NCT00880269
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications