Trial Outcomes & Findings for Study of Panobinostat Monotherapy in Women With v-ERB-B2 Avian Erythroblastic Leukemia Viral Oncogene Homolog 2 (HER2) Positive Locally Recurrent or Metastatic Breast Cancer (NCT NCT00777335)

Last Updated: 2015-11-24

Results Overview

The assessment of OR is based on the response of target lesion, of non-target lesion and on presence of new lesions (RECIST Criteria (V1.0)-assessed by CT scan spiral and bone scan) * CR:Disappearance of all target lesions * PR:\>=30% increase in the sum of the longest diameter (SLD),taking as reference the nadir SLD * Progressive Disease (PD):\>=20% increase in the SLD, taking as reference the nadir SLD, or the appearance of one or more new lesions * Stable Disease(SD):Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the nadir SLD

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

At screening, every 2 cycles (i.e. 6 weeks) during the first 6 cycles, every 3 cycles (i.e. 9 weeks) during the subsequent cycles and at the End of Treatment (EOT) visit. After the EOT, the tumor assessments should be performed every 9 weeks.

Results posted on

2015-11-24

Participant Flow

Subjects were screened and enrolled at 28 sites in 4 countries (USA, Canada, France, Belgium).

Participant milestones

Participant milestones
Measure	Panobinostat Intra-venous (i.v.)	Panobinostat Oral
Overall Study STARTED	2	2
Overall Study COMPLETED	2	2
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Panobinostat Monotherapy in Women With v-ERB-B2 Avian Erythroblastic Leukemia Viral Oncogene Homolog 2 (HER2) Positive Locally Recurrent or Metastatic Breast Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Panobinostat Intra-venous (i.v.) n=2 Participants	Panobinostat Oral n=2 Participants	Total n=4 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Age, Continuous	48.1 years STANDARD_DEVIATION 20.8 • n=5 Participants	59.0 years STANDARD_DEVIATION 11.5 • n=7 Participants	53.5 years STANDARD_DEVIATION 15.1 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants
Eastern Cooperative Oncology Group Performance Status (ECOG PS) PS 0 (Fully active)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Eastern Cooperative Oncology Group Performance Status (ECOG PS) PS 1 (Restricted in physically strenuous activity)	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Eastern Cooperative Oncology Group Performance Status (ECOG PS) PS 2 (Ambulatory and capable of all selfcare)	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Eastern Cooperative Oncology Group Performance Status (ECOG PS) PS 3 (Capable of only limited selfcare)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Eastern Cooperative Oncology Group Performance Status (ECOG PS) PS 4 (Completely disabled)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Eastern Cooperative Oncology Group Performance Status (ECOG PS) PS 5 (Dead)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Number of lines of prior chemotherapy in metastatic setting 1 line of prior chemotherapy	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Number of lines of prior chemotherapy in metastatic setting 2 lines of prior chemotherapy	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Number of lines of prior chemotherapy in metastatic setting 3 lines of prior chemotherapy	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: At screening, every 2 cycles (i.e. 6 weeks) during the first 6 cycles, every 3 cycles (i.e. 9 weeks) during the subsequent cycles and at the End of Treatment (EOT) visit. After the EOT, the tumor assessments should be performed every 9 weeks.

Outcome measures

Outcome measures
Measure	Panobinostat Intra-venous (i.v.) n=2 Participants	Panobinostat Oral n=2 Participants
Overall Response (OR) Rate (as Determined by the Investigator): the Number of Patients Assigned to a Treatment Arm With a Confirmed Best Response of Complete Response(CR) or Partial Response (PR). Progressive Disease (PD)	2 participants	1 participants
Overall Response (OR) Rate (as Determined by the Investigator): the Number of Patients Assigned to a Treatment Arm With a Confirmed Best Response of Complete Response(CR) or Partial Response (PR). Stable Disease (SD)	0 participants	1 participants
Overall Response (OR) Rate (as Determined by the Investigator): the Number of Patients Assigned to a Treatment Arm With a Confirmed Best Response of Complete Response(CR) or Partial Response (PR). Complete Response (CR)	0 participants	0 participants
Overall Response (OR) Rate (as Determined by the Investigator): the Number of Patients Assigned to a Treatment Arm With a Confirmed Best Response of Complete Response(CR) or Partial Response (PR). Partial Reponse (PR)	0 participants	0 participants

SECONDARY outcome

Timeframe: Panobinostat intra-venous (i.v.): All cycles pre-dose measurements. For cycles 1 and 2, post-dose measurements as well. / Panobinostat oral: Pre-dose and post-dose measurements for all cycles. Note: each cycle = 3 weeks

Prolonged QTcF: QTcF \>450 msec and increase of baseline on greater than or equal to 60 msec.

Outcome measures

Outcome measures
Measure	Panobinostat Intra-venous (i.v.) n=2 Participants	Panobinostat Oral n=2 Participants
Corrected QT Interval Fridericia's Formula (QTcF) Electrocardiogram QT normal	1 participants	2 participants
Corrected QT Interval Fridericia's Formula (QTcF) Electrocardiogram QT prolonged	1 participants	0 participants

Adverse Events

Panobinostat Intra-venous (i.v.)

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Panobinostat Oral

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Panobinostat Intra-venous (i.v.) n=2 participants at risk	Panobinostat Oral n=2 participants at risk
Investigations Electrocardiogram T wave inversion	50.0% 1/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Respiratory, thoracic and mediastinal disorders Dyspnea	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Respiratory, thoracic and mediastinal disorders Pleural Effusion	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.

Other adverse events

Additional Information

Dr. Richard Finn

University of California Los Angeles (UCLA) / Cancer International Research Group (CIRG) (Translational Research in Oncology TRIO) Inc.

Phone: + 1 310 586 2091

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Panobinostat Intra-venous (i.v.) n=2 participants at risk	Panobinostat Oral n=2 participants at risk
Investigations Weight decreased	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Metabolism and nutrition disorders Decreased appetite	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Nervous system disorders Dizziness	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Nervous system disorders Dysgueusia	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Nervous system disorders Headache	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Nervous system disorders Peripheral Motor Neuropathy	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Respiratory, thoracic and mediastinal disorders Dyspnoea	50.0% 1/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Skin and subcutaneous tissue disorders Alopecia	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Skin and subcutaneous tissue disorders Petechiae	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	100.0% 2/2 • Number of events 4 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Cardiac disorders Tachycardia	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Gastrointestinal disorders Abdominal Pain	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Gastrointestinal disorders Constipation	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	50.0% 1/2 • Number of events 1 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Gastrointestinal disorders Diarrhoea	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Gastrointestinal disorders Nausea	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
General disorders Fatigue	100.0% 2/2 • Number of events 3 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	100.0% 2/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.
Investigations Electrocardiogram QT prolonged	50.0% 1/2 • Number of events 2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.	0.00% 0/2 • The period for collection and reporting of adverse events extends from the time the patient signs the informed consent form (even if the event is not considered to be related to the study drug) until 28 days after the last receipt of the study treatment.