LEE001 and Chemoembolization In Patients With Advanced Hepatocellular Carcinoma (LEE001)

• ClinicalTrials.gov Identifier: NCT02524119
• Recruitment Status: Terminated
• First Posted: August 14, 2015
• Results First Posted: February 1, 2021
• Last Update Posted: February 1, 2021
• Sponsor: University of Texas Southwestern Medical Center
• Collaborator: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Muhammad Beg, University of Texas Southwestern Medical Center

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Hepatocellular Carcinoma
• Interventions: Drug: LEE011; Procedure: Chemoembolization
• Enrollment: 5

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description	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.
Period Title: Overall Study
Started	5
Completed	5
Not Completed	0

Baseline Characteristics

Arm/Group Title		LEE001 With Chemoembolization
Arm/Group Description		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 Number of Baseline Participants		5
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	5 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	2 40.0%
	>=65 years	3 60.0%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	5 participants
	Female	1 20.0%
	Male	4 80.0%
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	5 participants
White Non-Hispanic		3 60.0%
Black or African American		1 20.0%
Hispanic or Latino		1 20.0%
Asian		0 0.0%
Other		0 0.0%

Outcome Measures

1. Primary Outcome

Title	Progression Free Survival
Description	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.
Time Frame	Every 8 weeks for up to 3 years.

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description:	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 Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

2. Secondary Outcome

Title	Overall Survival
Description	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.
Time Frame	Every 12 weeks for up to 3 years.

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description:	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 Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

3. Secondary Outcome

Title	Number of Participants With Adverse Events
Description	Adverse events will be determined according to Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0.
Time Frame	Each visit for up to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description:	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 Number of Participants Analyzed	5
Measure Type: Count of Participants; Unit of Measure: Participants
	1 20.0%

4. Secondary Outcome

Title	Tolerability, as Measured by Number of Adverse Events
Description	The tolerability of LEE in combination with chemoembolization will be measured by number of adverse events.
Time Frame	At each patient visit while on LEE001 for up to 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description:	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 Number of Participants Analyzed	5
Measure Type: Number; Unit of Measure: Adverse Events
	1

5. Secondary Outcome

Title	Objective Response Rate (ORR) Based on mRECIST and RECIST 1.1
Description	ORR was defined as number of participants with confirmed complete response (CR) or confirmed partial response (PR) according to the RECIST 1.1.
Time Frame	Every 8 weeks for 3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description:	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 Number of Participants Analyzed	5
Measure Type: Count of Participants; Unit of Measure: Participants
	2 40.0%

Adverse Events

Time Frame	2 years
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	LEE001 With Chemoembolization
Arm/Group Description	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.
All-Cause Mortality
	LEE001 With Chemoembolization
	Affected / at Risk (%)
Total	0/5 (0.00%)
Serious Adverse Events
	LEE001 With Chemoembolization
	Affected / at Risk (%)	# Events
Total	1/5 (20.00%)
Infections and infestations
Hepatic infection *	1/5 (20.00%)	1
* Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	LEE001 With Chemoembolization
	Affected / at Risk (%)	# Events
Total	0/5 (0.00%)

Limitations and Caveats

Data were not collected for some outcome due to early study termination.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Dr. Muhammad Beg
• Organization: UT Southwestern Medical Center
• Phone: 2146484180
• EMail: Muhammad.Beg@UTSouthwestern.edu

• Responsible Party: Muhammad Beg, University of Texas Southwestern Medical Center
• ClinicalTrials.gov Identifier: NCT02524119
• Other Study ID Numbers: STU 052015-073
• First Submitted: July 30, 2015
• First Posted: August 14, 2015
• Results First Submitted: January 8, 2021
• Results First Posted: February 1, 2021
• Last Update Posted: February 1, 2021