Trial Outcomes & Findings for Dabrafenib and Trametinib in Treating Patients With BRAF Mutated Ameloblastoma (NCT NCT02367859)

Last Updated: 2024-01-30

Results Overview

Tumor response was assessed per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Overall tumor response was assessed as the number of participants achieving either a complete response (CR) or a partial response (PR). The criteria are: * CR = Disappearance of all target lesions * PR = ≥ 30% decrease in the sum of the longest diameter of target lesions * Overall Response (OR) = CR + PR * Progressive disease (PD) = 20% increase in the sum of the longest diameter of target lesions, and/or the appearance of one or more new lesion(s) * Stable disease (SD) = Small changes that do not meet any of the above criteria The outcome is reported as the number of participants achieving the different levels of tumor response per RECIST, a number without dispersion.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

6 weeks

Results posted on

2024-01-30

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Dabrafenib) Patients receive dabrafenib orally twice daily every 12 hours plus trametinib daily orally for 6 weeks in the absence of disease progression or unacceptable toxicity. Patients whose disease is judged to be not amenable to resection will continue dabrafenib and trametinib indefinitely as long as there has not been tumor progression.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Dabrafenib and Trametinib in Treating Patients With BRAF Mutated Ameloblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Dabrafenib) n=1 Participants Patients receive dabrafenib orally twice daily every 12 hours plus trametinib daily orally for 6 weeks in the absence of disease progression or unacceptable toxicity. Patients whose disease is judged to be not amenable to resection will continue dabrafenib and trametinib indefinitely as long as there has not been tumor progression.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 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	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 weeks

Outcome measures

Outcome measures
Measure	Treatment (Dabrafenib) n=1 Participants Patients receive dabrafenib orally twice daily every 12 hours plus trametinib daily orally for 6 weeks in the absence of disease progression or unacceptable toxicity. Patients whose disease is judged to be not amenable to resection will continue dabrafenib and trametinib indefinitely as long as there has not been tumor progression.
Tumor Response Complete Response (CR)	0 Participants
Tumor Response Partial Response (PR)	0 Participants
Tumor Response Overall Response (OR)	0 Participants
Tumor Response Progressive disease (PD)	0 Participants
Tumor Response Stable disease (SD)	1 Participants

SECONDARY outcome

Timeframe: 6 weeks

Population: On the basis of futility due to study closure with inadequate accrual, post-surgical research specimens were not analyzed.

Percent of tumor necrosis at the time of endpoint biopsy or surgical resection will be assessed. The tumor specimen from resection will be examined and the volume of the necrosis compared to the volume of the total tumor determined by central pathology. Percent tumor necrosis, in increments of 10%, will be determined. The outcome will be reported as the mean percent tumor necrosis, with standard deviation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 weeks

Population: On the basis of futility due to study closure with inadequate accrual, post-surgical research specimens were not analyzed.

An immunohistochemical laboratory analysis to assess proliferation will be performed on the initial pre-treatment biopsy (baseline) vs either the endpoint tumor biopsy or the tumor specimen from the resection. Ki-67 immunohistochemistry will be scored by at least 2 pathologists using percentage positive cells as the primary metric. Proliferation will be assessed as the difference from baseline in Ki-67 labeling to the end of treatment (tumor biopsy or the tumor specimen). The outcome will be expressed as the mean, with standard deviation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 weeks

Population: On the basis of futility due to study closure with inadequate accrual, post-surgical research specimens were not analyzed.

An immunohistochemical laboratory analysis to assess phosphorylation of the tumor markers MEK and ERK on the initial pre-treatment biopsy (baseline) vs either the endpoint tumor biopsy or the tumor specimen from the resection. Immunohistochemistry will be scored by at least 2 pathologists using percentage positive cells and intensity of staining as the primary metrics. Phosphorylation of MEK and ERK will be assessed as the observed difference in phosphorylated MEK and ERK labeling from baseline to the end of treatment (tumor biopsy or the tumor specimen). The outcome will be expressed as the mean, with standard deviation.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Dabrafenib)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment (Dabrafenib) n=1 participants at risk Patients receive dabrafenib orally twice daily every 12 hours plus trametinib daily orally for 6 weeks in the absence of disease progression or unacceptable toxicity. Patients whose disease is judged to be not amenable to resection will continue dabrafenib and trametinib indefinitely as long as there has not been tumor progression.
General disorders Flu like symptoms	100.0% 1/1 • Number of events 1 • 6 weeks
Nervous system disorders Headache	100.0% 1/1 • Number of events 1 • 6 weeks
General disorders Fatigue	100.0% 1/1 • Number of events 1 • 6 weeks
Musculoskeletal and connective tissue disorders Myalgia	100.0% 1/1 • Number of events 1 • 6 weeks
Skin and subcutaneous tissue disorders Rash acneform	100.0% 1/1 • Number of events 1 • 6 weeks
Investigations Weight loss	100.0% 1/1 • Number of events 1 • 6 weeks
Metabolism and nutrition disorders Anorexia	100.0% 1/1 • Number of events 1 • 6 weeks
Skin and subcutaneous tissue disorders Dermatologic syndrome	100.0% 1/1 • Number of events 1 • 6 weeks

Additional Information

Dr. A. Dimitrios Colevas, Professor of Medicine (Oncology)

Stanford University

Phone: 650-724-9707

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place