Trial Outcomes & Findings for LEE001 and Chemoembolization In Patients With Advanced Hepatocellular Carcinoma (NCT NCT02524119)

Results Overview

CT Chest scans must be performed at baseline. CT-Chest/abdomen/pelvis must be performed every 8 weeks during the treatment phase (12 months). Once the patient has been discontinued from the study and enters the efficacy phase, radiological assessment (CT or MRI) will continue every 8 weeks until progression or for the first 12 months, whichever comes first. After a year, radiological (CT or MRI) assessments will be performed every 12 weeks for up to 1 year.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

Every 8 weeks for up to 3 years.

Results posted on

2021-02-01

Participant Flow

Participant milestones

Participant milestones
Measure	LEE001 With Chemoembolization A total of 40 patients will be enrolled and undergo chemoembolization. Patients will receive LEE011 (600 mg PO once daily, 3 weeks on/1 week off) on Day 1 with chemoembolization. Patients can receive a total of 4 chemoembolization treatments within 6 month following first treatment as needed to treat initial HCC lesion. LEE011: 600 mg PO once daily will be given orally on days 1-21 of a 28 day cycle (3 weeks on / 1 week off) :until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment due to any other reason Chemoembolization: Patients will be treated with chemoembolization once every 4 weeks with up to 4 total chemoembolizations within the first 6 months from the initial chemoembolization.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

LEE001 and Chemoembolization In Patients With Advanced Hepatocellular Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	LEE001 With Chemoembolization n=5 Participants A total of 40 patients will be enrolled and undergo chemoembolization. Patients will receive LEE011 (600 mg PO once daily, 3 weeks on/1 week off) on Day 1 with chemoembolization. Patients can receive a total of 4 chemoembolization treatments within 6 month following first treatment as needed to treat initial HCC lesion. LEE011: 600 mg PO once daily will be given orally on days 1-21 of a 28 day cycle (3 weeks on / 1 week off) :until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment due to any other reason Chemoembolization: Patients will be treated with chemoembolization once every 4 weeks with up to 4 total chemoembolizations within the first 6 months from the initial chemoembolization.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race/Ethnicity, Customized White Non-Hispanic	3 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	1 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants
Race/Ethnicity, Customized Other	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Every 8 weeks for up to 3 years.

Population: Data were not collected for this outcome due to early study termination

CT Chest scans must be performed at baseline. CT-Chest/abdomen/pelvis must be performed every 8 weeks during the treatment phase (12 months). Once the patient has been discontinued from the study and enters the efficacy phase, radiological assessment (CT or MRI) will continue every 8 weeks until progression or for the first 12 months, whichever comes first. After a year, radiological (CT or MRI) assessments will be performed every 12 weeks for up to 1 year.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Every 12 weeks for up to 3 years.

Population: Data were not collected for this outcome due to early study termination

CT Chest scans must be performed at baseline. CT-Chest/abdomen/pelvis must be performed every 8 weeks during the treatment phase (12 months). Once the patient has been discontinued from the study and enters the efficacy phase, radiological assessment (CT or MRI) will continue every 8 weeks until progression or for 3 years, whichever comes first. After a year, radiological (CT or MRI) assessments will be performed every 12 weeks for up to 3year.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Each visit for up to 3 years

Adverse events will be determined according to Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0.

Outcome measures

Outcome measures
Measure	LEE001 With Chemoembolization n=5 Participants A total of 40 patients will be enrolled and undergo chemoembolization. Patients will receive LEE011 (600 mg PO once daily, 3 weeks on/1 week off) on Day 1 with chemoembolization. Patients can receive a total of 4 chemoembolization treatments within 6 month following first treatment as needed to treat initial HCC lesion. LEE011: 600 mg PO once daily will be given orally on days 1-21 of a 28 day cycle (3 weeks on / 1 week off) :until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment due to any other reason Chemoembolization: Patients will be treated with chemoembolization once every 4 weeks with up to 4 total chemoembolizations within the first 6 months from the initial chemoembolization.
Number of Participants With Adverse Events	1 Participants

SECONDARY outcome

Timeframe: At each patient visit while on LEE001 for up to 3 years

The tolerability of LEE in combination with chemoembolization will be measured by number of adverse events.

Outcome measures

Outcome measures
Measure	LEE001 With Chemoembolization n=5 Participants A total of 40 patients will be enrolled and undergo chemoembolization. Patients will receive LEE011 (600 mg PO once daily, 3 weeks on/1 week off) on Day 1 with chemoembolization. Patients can receive a total of 4 chemoembolization treatments within 6 month following first treatment as needed to treat initial HCC lesion. LEE011: 600 mg PO once daily will be given orally on days 1-21 of a 28 day cycle (3 weeks on / 1 week off) :until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment due to any other reason Chemoembolization: Patients will be treated with chemoembolization once every 4 weeks with up to 4 total chemoembolizations within the first 6 months from the initial chemoembolization.
Tolerability, as Measured by Number of Adverse Events	1 Adverse Events

SECONDARY outcome

Timeframe: Every 8 weeks for 3 years

ORR was defined as number of participants with confirmed complete response (CR) or confirmed partial response (PR) according to the RECIST 1.1.

Outcome measures

Outcome measures
Measure	LEE001 With Chemoembolization n=5 Participants A total of 40 patients will be enrolled and undergo chemoembolization. Patients will receive LEE011 (600 mg PO once daily, 3 weeks on/1 week off) on Day 1 with chemoembolization. Patients can receive a total of 4 chemoembolization treatments within 6 month following first treatment as needed to treat initial HCC lesion. LEE011: 600 mg PO once daily will be given orally on days 1-21 of a 28 day cycle (3 weeks on / 1 week off) :until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment due to any other reason Chemoembolization: Patients will be treated with chemoembolization once every 4 weeks with up to 4 total chemoembolizations within the first 6 months from the initial chemoembolization.
Objective Response Rate (ORR) Based on mRECIST and RECIST 1.1	2 Participants

Adverse Events

LEE001 With Chemoembolization

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	LEE001 With Chemoembolization n=5 participants at risk A total of 40 patients will be enrolled and undergo chemoembolization. Patients will receive LEE011 (600 mg PO once daily, 3 weeks on/1 week off) on Day 1 with chemoembolization. Patients can receive a total of 4 chemoembolization treatments within 6 month following first treatment as needed to treat initial HCC lesion. LEE011: 600 mg PO once daily will be given orally on days 1-21 of a 28 day cycle (3 weeks on / 1 week off) :until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment due to any other reason Chemoembolization: Patients will be treated with chemoembolization once every 4 weeks with up to 4 total chemoembolizations within the first 6 months from the initial chemoembolization.
Infections and infestations Hepatic infection	20.0% 1/5 • Number of events 1 • 2 years

Other adverse events

Adverse event data not reported

Additional Information

Dr. Muhammad Beg

UT Southwestern Medical Center

Phone: 2146484180

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place