Trial Outcomes & Findings for Lapatinib in Stage IV Melanoma With ERBB4 Mutations (NCT NCT01264081)

NCT ID: NCT01264081

Last Updated: 2019-11-13

Results Overview

The count of participants with a partial response and complete response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria. Partial response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. Progressive disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). The appearance of one or more new lesions is also considered progressions.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 34 participants
• Primary outcome timeframe: 3 years
• Results posted on: 2019-11-13

Participant Flow

Thirty participants were registered, tested for the ERBB4 mutation, and taken off study as screening failures as they did not have the ERBB4 mutation to be treated with Lapatinib.

Participant milestones

Measure	Lapatinib Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Participants Screened STARTED	34
Participants Screened COMPLETED	4
Participants Screened NOT COMPLETED	30
Started Treatment STARTED	4
Started Treatment COMPLETED	2
Started Treatment NOT COMPLETED	2
Analysis STARTED	2
Analysis COMPLETED	2
Analysis NOT COMPLETED	0

Reasons for withdrawal

Measure	Lapatinib Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Participants Screened Screening failures	30
Started Treatment Withdrawal by Subject	1
Started Treatment Not complete cycle 1 due to death	1

Baseline Characteristics

Lapatinib in Stage IV Melanoma With ERBB4 Mutations

Baseline characteristics by cohort

Measure	Lapatinib n=34 Participants Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	34 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	49 years STANDARD_DEVIATION 0 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	29 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	34 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 years

Population: Only two participants could be evaluated for response and they had SD (none with a PR or CR). Other participants did not reach the evaluation point (i.e. discontinued Lapatinib)as per study protocol, therefore not evaluable.

The count of participants with a partial response and complete response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria. Partial response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Complete response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. Progressive disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). The appearance of one or more new lesions is also considered progressions.

Outcome measures

Measure	Lapatinib n=2 Participants Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Count of Participants With a Partial Response (PR) and Complete Response (CR) to Lapatinib Who Have Metastatic Melanoma Harboring ERBB4 Mutations. Partial Response	0 Participants
Count of Participants With a Partial Response (PR) and Complete Response (CR) to Lapatinib Who Have Metastatic Melanoma Harboring ERBB4 Mutations. Complete Response	0 Participants
Count of Participants With a Partial Response (PR) and Complete Response (CR) to Lapatinib Who Have Metastatic Melanoma Harboring ERBB4 Mutations. Stable Disease	2 Participants
Count of Participants With a Partial Response (PR) and Complete Response (CR) to Lapatinib Who Have Metastatic Melanoma Harboring ERBB4 Mutations. Progressive Disease	0 Participants

SECONDARY outcome

Timeframe: Date treatment consent signed to date off study, approximately, 3 years

Population: Three study participants were affected by the adverse events: 2 - who completed the study and 1 who did not complete cycle 1 due to death of progressive disease. The participant who withdrew from study didn't take Lapatinib and experience any adverse events.

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Measure	Lapatinib n=3 Participants Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Number of Participants With Serious and Non-Serious Adverse Events	3 Participants

Adverse Events

Lapatinib

Serious events: 3 serious events

Other events: 3 other events

Deaths: 2 deaths

Serious adverse events

Measure	Lapatinib n=3 participants at risk Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Vascular disorders Thromboembolic event	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately, 3 years. Three study participants were affected by the adverse events: 2 - who completed the study and 1 who did not complete cycle 1 due to death of progressive disease. The participant who withdrew from study didn't take Lapatinib and experience any adverse events.
General disorders Death NOS	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately, 3 years. Three study participants were affected by the adverse events: 2 - who completed the study and 1 who did not complete cycle 1 due to death of progressive disease. The participant who withdrew from study didn't take Lapatinib and experience any adverse events.

Other adverse events

Measure	Lapatinib n=3 participants at risk Lapatinib: 500 mg PO (orally) twice a day for 28 days (1 cycle). Up to 27 cycles allowed if response seen.
Skin and subcutaneous tissue disorders Rash maculo-paular	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately, 3 years. Three study participants were affected by the adverse events: 2 - who completed the study and 1 who did not complete cycle 1 due to death of progressive disease. The participant who withdrew from study didn't take Lapatinib and experience any adverse events.
Psychiatric disorders Agitation	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately, 3 years. Three study participants were affected by the adverse events: 2 - who completed the study and 1 who did not complete cycle 1 due to death of progressive disease. The participant who withdrew from study didn't take Lapatinib and experience any adverse events.

Additional Information

Dr. Udo Rudloff'

National Cancer Institute

Phone: 301-496-3098

Email: rudloff@nih.gov

Results disclosure agreements

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