Trial Outcomes & Findings for Trial of LBH589 in Metastatic Thyroid Cancer (NCT NCT01013597)

NCT ID: NCT01013597

Last Updated: 2019-11-29

Results Overview

per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v.1.0) for target lesions and assessed by CT/MRI: "Response" includes Complete Response (CR, disappearance of all target lesions), or Partial Response (PR, \>=30% decrease in the sum of the longest diameter of target lesions). "No Response" includes Stable Disease (SD, neither sufficient shrinkage to qualify for partial response nor sufficient increase to qualify for progressive disease), and Progressive Disease (PD, at least a 20% increase in the sum of the longest diameters of target lesions, taking as reference the smallest sum longest diameter recorded since the baseline measurements, or the appearance of one or more new lesion(s).)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

Every 8 weeks.

Results posted on

2019-11-29

Participant Flow

Participant milestones

Participant milestones
Measure
LBH589
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Overall Study
STARTED
13
Overall Study
COMPLETED
12
Overall Study
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
LBH589
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Overall Study
Withdrawal by Subject
1

Baseline Characteristics

Trial of LBH589 in Metastatic Thyroid Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
LBH589
n=13 Participants
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Age, Customized
30-39 years
1 participants
n=93 Participants
Age, Customized
40-49 years
2 participants
n=93 Participants
Age, Customized
50-59 years
2 participants
n=93 Participants
Age, Customized
60-69 years
6 participants
n=93 Participants
Age, Customized
70-79 years
2 participants
n=93 Participants
Sex: Female, Male
Female
7 Participants
n=93 Participants
Sex: Female, Male
Male
6 Participants
n=93 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=93 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
13 Participants
n=93 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants
Race (NIH/OMB)
Asian
0 Participants
n=93 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=93 Participants
Race (NIH/OMB)
White
12 Participants
n=93 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=93 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
Region of Enrollment
United States
13 participants
n=93 Participants

PRIMARY outcome

Timeframe: Every 8 weeks.

per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v.1.0) for target lesions and assessed by CT/MRI: "Response" includes Complete Response (CR, disappearance of all target lesions), or Partial Response (PR, \>=30% decrease in the sum of the longest diameter of target lesions). "No Response" includes Stable Disease (SD, neither sufficient shrinkage to qualify for partial response nor sufficient increase to qualify for progressive disease), and Progressive Disease (PD, at least a 20% increase in the sum of the longest diameters of target lesions, taking as reference the smallest sum longest diameter recorded since the baseline measurements, or the appearance of one or more new lesion(s).)

Outcome measures

Outcome measures
Measure
LBH589
n=13 Participants
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Tumor Response Rate to LBH589.
No response
13 participants
Tumor Response Rate to LBH589.
Response
0 participants

SECONDARY outcome

Timeframe: End of study

Population: Notch1 protein expression was not measured due to lack of efficiency of study intervention

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Every 3 months until progression up to 5 years

Progression is defined using the Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Time to progression is defined as the number of days from the day of first LBH589 administration to the day the patient experienced an event of disease progression or death, whichever came first. Progression was assessed every 3 months until death or up to 5 years, whichever occurred first.

Outcome measures

Outcome measures
Measure
LBH589
n=13 Participants
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Time to Progression of Thyroid Cancer
3.6 months
Interval 1.8 to 5.8

SECONDARY outcome

Timeframe: Every 3 months up to 5 years

For a given patient, overall survival (OS) is defined as the number of days from the day of first LBH589 administration until the patient's death. If a patient was alive at the time of analysis, then the patient's data is censored at the date of the last available evaluation.Survival was assessed every three months until death or final data analysis, whichever occurred first.

Outcome measures

Outcome measures
Measure
LBH589
n=13 Participants
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Overall Survival
18.4 months
Interval 5.8 to
upper limit not reached

SECONDARY outcome

Timeframe: Baseline and end of treatment, up to 1 year

Change in serum Thyroglobulin level from baseline to end of treatment. Treatment continued until either extraordinary medical circumstances, disease progression, toxicity, subject withdrawal, or death. At the time subjects came off of study treatment for one of the reasons already listed, a sample was collected for tumor markers.

Outcome measures

Outcome measures
Measure
LBH589
n=11 Participants
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Impact of LBH589 on Tumor Markers for Thyroid Cancer
4.58 ng/mL
Standard Deviation 716.65

SECONDARY outcome

Timeframe: Every 4 weeks, up to 5 years

Most frequent toxicities at least possibly related to panobinostat, grades 2-4 (grading based on NCI common terminology criteria for adverse events CTCAE version 3). Toxicities were collected from the time the patient provided informed consent until 4 weeks after the patient stopped LBH589.

Outcome measures

Outcome measures
Measure
LBH589
n=13 Participants
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Toxicity of LBH589
Asthenia
1 participants
Toxicity of LBH589
Rash
1 participants
Toxicity of LBH589
Anemia
3 participants
Toxicity of LBH589
Leukopenia
7 participants
Toxicity of LBH589
Neutropenia
7 participants
Toxicity of LBH589
Lymphopenia
8 participants
Toxicity of LBH589
Thrombocytopenia
8 participants
Toxicity of LBH589
Hypoalbuminemia
3 participants
Toxicity of LBH589
Fatigue
8 participants
Toxicity of LBH589
Diarrhea
4 participants
Toxicity of LBH589
Headache
1 participants
Toxicity of LBH589
Bone Pain
1 participants
Toxicity of LBH589
Hyperglycemia
1 participants
Toxicity of LBH589
Hypocalcemia
2 participants
Toxicity of LBH589
Anorexia
4 participants
Toxicity of LBH589
Anxiety
1 participants
Toxicity of LBH589
Elevated GGT
1 participants
Toxicity of LBH589
Pulmonary embolism
1 participants

SECONDARY outcome

Timeframe: Every 4 weeks, up to 5 years

Tolerability and toxicity were not assessed separately, therefore tolerability is reported as toxicity.

Outcome measures

Outcome data not reported

Adverse Events

LBH589

Serious events: 6 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
LBH589
n=13 participants at risk
LBH589: LBH589 20mg by mouth three times weekly (Monday/Wednesday/Friday) for 28-day cycles.
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC)
7.7%
1/13
Blood and lymphatic system disorders
Platelets
7.7%
1/13
General disorders
Death, NOS
23.1%
3/13
General disorders
Hemorrhage/bleeding
7.7%
1/13
Infections and infestations
Colitis, infectious
7.7%
1/13
Nervous system disorders
CNS cerebrovascular ischemia
7.7%
1/13
Nervous system disorders
Other
7.7%
1/13
Respiratory, thoracic and mediastinal disorders
Pleural effusion, non-malignant
7.7%
1/13
Vascular disorders
Thrombosis/thrombus/embolism
7.7%
1/13

Other adverse events

Adverse event data not reported

Additional Information

Abigail Mapes, Thoracic Oncology Research Program Manager

University of Wisconsin Carbone Cancer Center

Phone: 608-262-8158

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place