Refractory LeukemiaPhase 2NCT00880269
Efficacy and Safety of Panobinostat (LBH589) in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
A Phase 2 study testing Panobinostat/LBH589 in people with Refractory Leukemia and Acute Myelogenous Leukemia, run by Novartis Pharmaceuticals at 40 sites in 12 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Refractory Leukemia, Acute Myelogenous Leukemia
- What is being tested?
- Panobinostat/LBH589
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 59 participants
- Where
- 40 sitesin 12 countries
- Study dates
- Aug 2009to Feb 2012
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
40 sites in 12 countries.
40 sites in 12 countries
United States7 sites
University of Chicago Medical Center Dept. of U. of Chicago Hosp(3)
Chicago, Illinois, 60637
University of Texas Southwestern Medical Center Dept of Simmons Cancer Center
Dallas, Texas, 75390
University of Texas/MD Anderson Cancer Center Dept of MD Anderson (14)
Houston, Texas, 77030-4009
North Shore University Hospital North Shore Univ
Manhasset, New York, 10030
Memorial Sloan Kettering Cancer Center Dept. of MSKCC (2)
New York, New York, 10065
Nebraska Methodist Hospital Nebraska Methodist Hospital(2)
Omaha, Nebraska, 68114
Oregon Health Sciences University Dept. of OHSU (2)
Portland, Oregon, 97239
Australia6 sites
Novartis Investigative Site
Adelaide, South Australia, 5000
Novartis Investigative Site
Fitzroy, Victoria, 3065
Novartis Investigative Site
Gosford, New South Wales, 2250
Novartis Investigative Site
Herston, Queensland, 4029
Novartis Investigative Site
Parkville, Victoria, 3002
Novartis Investigative Site
Prahran, Victoria, 3181
France5 sites
Novartis Investigative Site
Bobigny, 93009
Novartis Investigative Site
Montpellier, 34295
Novartis Investigative Site
Nantes, 44035
Novartis Investigative Site
Paris, 75181
Novartis Investigative Site
Toulouse, 31059
United Kingdom5 sites
Novartis Investigative Site
Aberdeen, Scotland, AB25 2ZN
Novartis Investigative Site
Leeds, West Yorkshire, LS9 7TF
Novartis Investigative Site
Liverpool, L7 8XP
Novartis Investigative Site
London, SE5 9RS
Novartis Investigative Site
London, W12 0HS
Belgium3 sites
Novartis Investigative Site
Bruges, 8000
Novartis Investigative Site
Ghent, 9000
Novartis Investigative Site
Liège, 4000
Germany3 sites
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Frankfurt, 60590
Novartis Investigative Site
Leipzig, 04103
Italy3 sites
Novartis Investigative Site
Roma, RM, 00133
Novartis Investigative Site
Roma, RM, 00161
Novartis Investigative Site
Udine, UD, 33100
Spain2 sites
Novartis Investigative Site
Barcelona, Barcelona, 08041
Novartis Investigative Site
L'Hospitalet de Llobregat, Catalonia, 08907
Switzerland2 sites
Novartis Investigative Site
Bern, 3010
Novartis Investigative Site
Zurich, Switzerland, 8091
Turkey (Türkiye)2 sites
Novartis Investigative Site
Ankara, Turkey, 06100
Novartis Investigative Site
Balcova / Izmir, 35340
About this study
Official title: A Phase II Study of Oral Single Agent Panobinostat in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
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.
- Conditions
- Refractory Leukemia, Acute Myelogenous Leukemia
- Being tested
- Panobinostat/LBH589
- 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
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Panobinostat/LBH589Drug
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Panobinostat/LBH589 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 cycles of treatment with a 28-day treatment cycle (Day 168)
Secondary outcomes (5)
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)
Measured: 6 treatment cycles (28-day/treatment cycle)
Time to Remission Measured in Stratum A and B
Measured: 6 treatment cycles (28-day/treatment cycle)
Duration of Remission Measured in Stratum A and B
Measured: 6 treatment cycles (28-day/treatment cycle)
Event-free Survival Measured in Stratum A and B
Measured: 6 treatment cycles (28-day/treatment cycle)
Overall Survival Measured in Stratum A and B
Measured: 6 treatment cycles (28-day/treatment cycle)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2009 (estimated)
- Study startAug 2009
- Primary completionFeb 2012
- Study completionFeb 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Refractory LeukemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00880269
- Sponsor's protocol ID
- CLBH589B2213
- Other IDs
- 2008-002983-32, 2008
- Status last verified
- Jan 2017
- First submitted
- 30 Mar 2009
- Last updated
- 23 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Oral Single Agent Panobinostat in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
- ClinicalTrials.gov ID
- NCT00880269
- Acronym / sponsor ID
- CLBH589B2213
- 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)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Aug 2009
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 13 Apr 2009 (estimated)
- Results
- Posted 9 Sep 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stratum A | Experimental | Drug: Panobinostat/LBH589patients 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. |
| Stratum B | Experimental | Drug: Panobinostat/LBH589patients 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. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Panobinostat/LBH589 | Drug | Stratum A, Stratum B |
Primary outcome
Best Response as Per Investigator Assessment by Stratum (FAS)
Time frame: 6 cycles of treatment with a 28-day treatment cycle (Day 168)
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.
5 secondary outcomes
Partial Response Measured in Stratum A and B
Time frame: 6 treatment cycles (28-day/treatment cycle)
Time to Remission Measured in Stratum A and B
Time frame: 6 treatment cycles (28-day/treatment cycle)
Duration of Remission Measured in Stratum A and B
Time frame: 6 treatment cycles (28-day/treatment cycle)
Event-free Survival Measured in Stratum A and B
Time frame: 6 treatment cycles (28-day/treatment cycle)
Overall Survival Measured in Stratum A and B
Time frame: 6 treatment cycles (28-day/treatment cycle)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
40 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Panobinostat/LBH589 in people with Refractory Leukemia and Acute Myelogenous Leukemia, run by Novartis Pharmaceuticals at 40 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, Australia, France, United Kingdom, Belgium and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Sep 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Best Response as Per Investigator Assessment by Stratum (FAS) (time frame: 6 cycles of treatment with a 28-day treatment cycle (Day 168)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2017.