Trial Outcomes & Findings for Efficacy and Safety of Panobinostat (LBH589) in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML) (NCT NCT00880269)
NCT ID: NCT00880269
Last Updated: 2017-02-23
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
59 participants
Primary outcome timeframe
6 cycles of treatment with a 28-day treatment cycle (Day 168)
Results posted on
2017-02-23
Participant Flow
Participant flow is based on the full analysis set (FAS) which is the same as the safety set and includes one dose of the study drug.
Although this study did not complete stage 2, it is considered as a completed study because it follows Simon's optimal two-stage design in each stratum (predefined in the protocol), the study can stop due to futility at the end of Stage 1 and not be considered as a terminated study.
Participant milestones
| Measure |
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.
|
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.
|
|---|---|---|
|
Overall Study
STARTED
|
32
|
27
|
|
Overall Study
Discontinued Treatment
|
32
|
27
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
32
|
27
|
Reasons for withdrawal
| Measure |
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.
|
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.
|
|---|---|---|
|
Overall Study
Protocol Violation
|
1
|
0
|
|
Overall Study
Disease Progression
|
13
|
11
|
|
Overall Study
New Cancer Therapy
|
1
|
0
|
|
Overall Study
Death
|
4
|
2
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
3
|
4
|
|
Overall Study
Adverse Event
|
10
|
9
|
Baseline Characteristics
Efficacy and Safety of Panobinostat (LBH589) in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)
Baseline characteristics by cohort
| Measure |
Stratum A
n=32 Participants
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.
|
Stratum B
n=27 Participants
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.
|
Total
n=59 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
63 years
STANDARD_DEVIATION 12.13 • n=5 Participants
|
68.5 years
STANDARD_DEVIATION 7.75 • n=7 Participants
|
65.5 years
STANDARD_DEVIATION 10.65 • n=5 Participants
|
|
Gender
Female
|
20 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Gender
Male
|
12 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
31 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 cycles of treatment with a 28-day treatment cycle (Day 168)Population: Full analysis set (FAS) is the same as the Safety set and includes all patients who received at least one dose of study drug. The FAS was used for final efficacy analyses.
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.
Outcome measures
| Measure |
Stratum A
n=32 Participants
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.
|
Stratum B
n=27 Participants
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.
|
|---|---|---|
|
Best Response as Per Investigator Assessment by Stratum (FAS)
Complete remission rate (CR/CRi)
|
3.1 Percentage of Participants
|
7.4 Percentage of Participants
|
|
Best Response as Per Investigator Assessment by Stratum (FAS)
Complete remission (CR)
|
0 Percentage of Participants
|
3.7 Percentage of Participants
|
|
Best Response as Per Investigator Assessment by Stratum (FAS)
CR with incomplete blood count recovery (CRi)
|
3.1 Percentage of Participants
|
3.7 Percentage of Participants
|
|
Best Response as Per Investigator Assessment by Stratum (FAS)
Partial remission (PR)
|
0 Percentage of Participants
|
0 Percentage of Participants
|
|
Best Response as Per Investigator Assessment by Stratum (FAS)
Treatment failure
|
40.6 Percentage of Participants
|
40.7 Percentage of Participants
|
|
Best Response as Per Investigator Assessment by Stratum (FAS)
Unknown
|
56.3 Percentage of Participants
|
51.9 Percentage of Participants
|
SECONDARY outcome
Timeframe: 6 treatment cycles (28-day/treatment cycle)Population: No secondary analyses were performed since study enrollment was stopped early at stage 1 for lack of evidence of activity.
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)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 treatment cycles (28-day/treatment cycle)Population: No secondary analyses were performed since study enrollment was stopped early at stage 1 for lack of evidence of activity.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 treatment cycles (28-day/treatment cycle)Population: No secondary analyses were performed since study enrollment was stopped early at stage 1 for lack of evidence of activity.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 treatment cycles (28-day/treatment cycle)Population: No secondary analyses were performed since study enrollment was stopped early at stage 1 for lack of evidence of activity.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 treatment cycles (28-day/treatment cycle)Population: No secondary analyses were performed since study enrollment was stopped early at stage 1 for lack of evidence of activity.
Outcome measures
Outcome data not reported
Adverse Events
Stratum A
Serious events: 29 serious events
Other events: 32 other events
Deaths: 0 deaths
Stratum B
Serious events: 23 serious events
Other events: 26 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Stratum A
n=32 participants at risk
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.
|
Stratum B
n=27 participants at risk
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.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
15.6%
5/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
18.5%
5/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Leukocytosis
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Neutropenia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
25.0%
8/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
29.6%
8/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Cardiac disorders
Angina pectoris
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Cardiac disorders
Atrial fibrillation
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Congenital, familial and genetic disorders
Aplasia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Abdominal pain
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Diarrhoea
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Impaired gastric emptying
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Mouth ulceration
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Nausea
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Vomiting
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Asthenia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Fatigue
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
General physical health deterioration
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Pyrexia
|
15.6%
5/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Abdominal infection
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Abscess limb
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Bacterial infection
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Bacterial sepsis
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Diverticulitis
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Escherichia infection
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Escherichia urinary tract infection
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Gastroenteritis viral
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Influenza
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Lower respiratory tract infection fungal
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Neutropenic sepsis
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Pneumonia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Pneumonia fungal
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Pseudomonal sepsis
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Pseudomonas infection
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Pyelonephritis
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Sepsis
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Septic shock
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Soft tissue infection
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Staphylococcal sepsis
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Streptococcal sepsis
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Urinary tract infection
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Injury, poisoning and procedural complications
Fall
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Blood creatinine increased
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Electrocardiogram ST segment depression
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Electrocardiogram T wave abnormal
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
General physical condition abnormal
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Platelet count decreased
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Fluid overload
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour flare
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Convulsion
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Headache
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Lethargy
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Loss of consciousness
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Somnolence
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Syncope
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Psychiatric disorders
Completed suicide
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Psychiatric disorders
Mental status changes
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Vascular disorders
Circulatory collapse
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Vascular disorders
Hypotension
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Vascular disorders
Systolic hypertension
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
Other adverse events
| Measure |
Stratum A
n=32 participants at risk
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.
|
Stratum B
n=27 participants at risk
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.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
28.1%
9/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
18.8%
6/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Neutropenia
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
14.8%
4/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
37.5%
12/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
25.9%
7/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Eye disorders
Dry eye
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Abdominal pain
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Aphthous stomatitis
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Constipation
|
21.9%
7/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
14.8%
4/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Diarrhoea
|
78.1%
25/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
55.6%
15/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Dry mouth
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Dyspepsia
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Dysphagia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Flatulence
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Gingival bleeding
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Mouth ulceration
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Nausea
|
56.2%
18/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
51.9%
14/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Odynophagia
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Stomatitis
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Gastrointestinal disorders
Vomiting
|
37.5%
12/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
29.6%
8/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Asthenia
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
18.5%
5/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Chills
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Fatigue
|
31.2%
10/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
33.3%
9/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Non-cardiac chest pain
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Oedema peripheral
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Pain
|
15.6%
5/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
General disorders
Pyrexia
|
50.0%
16/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
25.9%
7/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Skin infection
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Infections and infestations
Urinary tract infection
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Blood potassium decreased
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Electrocardiogram QT prolonged
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Neutrophil count decreased
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Platelet count decreased
|
15.6%
5/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
Weight decreased
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Investigations
White blood cell count decreased
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Decreased appetite
|
31.2%
10/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
33.3%
9/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Dehydration
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
14.8%
4/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
28.1%
9/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
3.1%
1/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Dizziness
|
0.00%
0/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Dysgeusia
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Nervous system disorders
Headache
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Psychiatric disorders
Agitation
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Psychiatric disorders
Anxiety
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Psychiatric disorders
Insomnia
|
15.6%
5/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Psychiatric disorders
Mental status changes
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
7.4%
2/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
18.8%
6/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
12.5%
4/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
3.7%
1/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Vascular disorders
Hypotension
|
9.4%
3/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
11.1%
3/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
|
Vascular disorders
Thrombophlebitis
|
6.2%
2/32 • Adverse events were followed for 28 days after last dose (Day 467).
|
0.00%
0/27 • Adverse events were followed for 28 days after last dose (Day 467).
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER