Trial Outcomes & Findings for Utility of Novel BRAF Test for Melanoma (NCT NCT01840527)

Last Updated: 2024-09-04

Results Overview

The specificity and sensitivity of the blood-based assay will be determined vis-à-vis tissue-based BRAF analysis in patients with advanced and high-risk melanoma in order to provide BRAF-directed therapy when indicated. The sensitivity of an assay is expressed as the probability (as a percentage) that a sample tests positive for BRAF mutation given that the participant has BRAF mutation; it is used to assess the risk of false negative results. A percentage greater than 50% indicates that the test is a better predictor of sample diagnosis than random chance.

Recruitment status

COMPLETED

Target enrollment

220 participants

Primary outcome timeframe

2 years

Results posted on

2024-09-04

Participant Flow

Participant milestones

Participant milestones
Measure	Advanced Melanoma For patients in the advanced melanoma cohort, a one-time blood sample will be obtained prior to the commencement of systemic therapy at a time when standard-of-care blood will be drawn. While it is ideal to obtain samples prior to any systemic therapy, participants who have previously received therapy, have progressed, and are in need of additional therapy will be considered suitable for participation. Participants with known BRAF mutation (determined by standard of care tissue testing) will undergo blood draws every 4 weeks (+/- 1 week) and at the time of disease progression.	Stage II/III Melanoma For patients in the high-risk stage II/III cohort, a blood draw will be performed 4-8 weeks after the completion of surgical management at the time of standard of care blood work. In participants who sign informed consent prior to definite surgical management, a pre-operative blood draw may be obtained at the time that standard of care pre-op blood work is performed. Blood samples will then be obtained every three months following the initiation of either adjuvant therapy or routine, close follow-up for up to two years.
Overall Study STARTED	110	110
Overall Study COMPLETED	110	110
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Utility of Novel BRAF Test for Melanoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Advanced Melanoma n=110 Participants For patients in the advanced melanoma cohort, a one-time blood sample will be obtained prior to the commencement of systemic therapy at a time when standard-of-care blood will be drawn. While it is ideal to obtain samples prior to any systemic therapy, participants who have previously received therapy, have progressed, and are in need of additional therapy will be considered suitable for participation. Participants with known BRAF mutation (determined by standard of care tissue testing) will undergo blood draws every 4 weeks (+/- 1 week) and at the time of disease progression.	Stage II/III Melanoma n=110 Participants For patients in the high-risk stage II/III cohort, a blood draw will be performed 4-8 weeks after the completion of surgical management at the time of standard of care blood work. In participants who sign informed consent prior to definite surgical management, a pre-operative blood draw may be obtained at the time that standard of care pre-op blood work is performed. Blood samples will then be obtained every three months following the initiation of either adjuvant therapy or routine, close follow-up for up to two years.	Total n=220 Participants Total of all reporting groups
Age, Continuous	59.8 years n=5 Participants	59.7 years n=7 Participants	59.7 years n=5 Participants
Sex: Female, Male Female	33 Participants n=5 Participants	32 Participants n=7 Participants	65 Participants n=5 Participants
Sex: Female, Male Male	77 Participants n=5 Participants	78 Participants n=7 Participants	155 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	101 Participants n=5 Participants	101 Participants n=7 Participants	202 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	9 Participants n=5 Participants	8 Participants n=7 Participants	17 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	105 Participants n=5 Participants	105 Participants n=7 Participants	210 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Region of Enrollment United States	110 participants n=5 Participants	110 participants n=7 Participants	220 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: The study's primary outcome was not reached because the assay has not been successfully developed so no collected samples could be analyzed. More sensitive ctDNA approaches have made their way into commercial use including bespoke assays during the course of this study. The value of a highly-sensitive BRAF specific assay seemed less useful, and given the past troubles developing the current assay approach, the study team decided to apply the philanthropic funds to other projects.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 2 years

The specificity and sensitivity of the blood-based assay will be determined vis-à-vis tissue-based BRAF analysis in patients with advanced and high-risk melanoma in order to provide BRAF-directed therapy when indicated. The specificity of an assay is expressed as the probability (as a percentage) that a test returns a negative result for BRAF mutation given that the that participant does not BRAF mutation; it is used to assess the risk of false positive results. A percentage greater than 50% indicates that the test is a better predictor of sample diagnosis than random chance.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

To explore the pharmacodynamic effects of MAPK pathway inhibitors (including selective BRAF inhibitors, MEK inhibitors, and ERK inhibitors) utilizing pre-and on-treatment peripheral blood BRAFV600E mutational testing in participants with advanced melanoma.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

To define the prognostic value of peripheral blood BRAFV600 mutational testing in participants with Stage II/III melanoma

Outcome measures

Outcome data not reported

Adverse Events

Advanced Melanoma

Serious events: 0 serious events

Other events: 0 other events

Deaths: 68 deaths

Stage II/III Melanoma

Serious events: 0 serious events

Other events: 0 other events

Deaths: 40 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Ryan Sullivan

Massachusetts General Hospital Cancer Center

Phone: 6177244000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place