Trial Outcomes & Findings for Binimetinib and Encorafenib for the Treatment of Pancreatic Cancer in Patients With a Somatic BRAF V600E Mutation (NCT NCT04390243)

Last Updated: 2024-05-06

Results Overview

An objective response is defined as a complete or partial response with a confirmation scan not less than 4 weeks after the initial scan. Disease status will be assessed using Response Evaluation Criteria in Solid Tumors (RECIST) version (v) 1.1 criteria. The final ORR point estimate and corresponding 95% confidence interval will be reported.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

24 weeks

Results posted on

2024-05-06

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Encorafenib, Binimetinib) Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity.\> \> Binimetinib: Given PO\> \> Encorafenib: Given PO
Overall Study STARTED	6
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Encorafenib, Binimetinib) Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity.\> \> Binimetinib: Given PO\> \> Encorafenib: Given PO
Overall Study Death	1
Overall Study Disease Progression	4
Overall Study Patient was on treatment when study was terminated	1

Baseline Characteristics

Binimetinib and Encorafenib for the Treatment of Pancreatic Cancer in Patients With a Somatic BRAF V600E Mutation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Encorafenib, Binimetinib) n=6 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity.\> \> Binimetinib: Given PO\> \> Encorafenib: Given PO
Age, Continuous	64.8 years STANDARD_DEVIATION 10.94 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 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	5 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	6 participants n=5 Participants
Line of Treatment 2nd line	4 Participants n=5 Participants
Line of Treatment 3rd line	2 Participants n=5 Participants
Histologic Grade of Malignancy G1	1 Participants n=5 Participants
Histologic Grade of Malignancy G2	3 Participants n=5 Participants
Histologic Grade of Malignancy GX	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Outcome measures

Outcome measures
Measure	Treatment (Encorafenib, Binimetinib) n=6 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. \> \> Binimetinib: Given PO \> \> Encorafenib: Given PO
Objective Response Rate (ORR) at 24 Weeks	33.33 percentage of patients Interval 4.33 to 77.72

SECONDARY outcome

Timeframe: 25 months

Progression-free survival is defined as the time from registration to clinical or radiographic disease progression or death, whichever occurs first, as defined by RECIST 1.1 criteria. PFS will be estimated using the Kaplan-Meier method. The median PFS and corresponding 95% confidence interval will be reported.

Outcome measures

Outcome measures
Measure	Treatment (Encorafenib, Binimetinib) n=6 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. \> \> Binimetinib: Given PO \> \> Encorafenib: Given PO
Progression-free Survival (PFS)	7.1 months Interval 1.7 to The upper limit of the confidence interval was not estimatable due to an insufficient number of participants with events

SECONDARY outcome

Timeframe: 25 months

Overall survival (OS) is defined as the time from registration to death from any cause. OS will be estimated using the Kaplan-Meier method. The median overall survival and corresponding 95% confidence interval will be reported.

Outcome measures

Outcome measures
Measure	Treatment (Encorafenib, Binimetinib) n=6 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. \> \> Binimetinib: Given PO \> \> Encorafenib: Given PO
Overall Survival	15.0 months Interval 3.1 to The upper limit of the confidence interval was not estimatable due to an insufficient number of participants with events

SECONDARY outcome

Timeframe: 12 months

Population: Only patients that achieved an objective response within 12 months were included in this analysis

Duration of response is defined as the duration of time from first documentation of an objective response to the earliest date disease progression is documented or death from any cause. Duration of response will be estimated using the Kaplan-Meier method. The median duration of response and corresponding 95% confidence interval will be reported.

Outcome measures

Outcome measures
Measure	Treatment (Encorafenib, Binimetinib) n=3 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. \> \> Binimetinib: Given PO \> \> Encorafenib: Given PO
Duration of Response	9.9 months Interval 8.5 to The upper limit of the confidence interval was not estimatable due to an insufficient number of participants with events

SECONDARY outcome

Timeframe: 25 months

Time to response is defined as the duration of time from registration to the first documentation of an objective response. Time to response will be estimated using the Kaplan-Meier method. The median time to response and corresponding 95% confidence interval will be reported.

Outcome measures

Outcome measures
Measure	Treatment (Encorafenib, Binimetinib) n=6 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. \> \> Binimetinib: Given PO \> \> Encorafenib: Given PO
Time to Response	3.9 months Interval 3.6 to The upper limit of the confidence interval was not estimatable due to an insufficient number of participants with events

SECONDARY outcome

Timeframe: 25 months

Adverse Events as defined by National Institute of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) version (v)5.0. All patients who have initiated treatment will be considered evaluable for adverse event analyses. The rate of patients experiencing a grade 3+ adverse event will be reported.

Outcome measures

Outcome measures
Measure	Treatment (Encorafenib, Binimetinib) n=6 Participants Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. \> \> Binimetinib: Given PO \> \> Encorafenib: Given PO
Number of Patients With Grade 3+ Adverse Events	3 Participants

Adverse Events

Treatment (Encorafenib, Binimetinib)

Serious events: 1 serious events

Other events: 6 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Encorafenib, Binimetinib) n=6 participants at risk Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. * Binimetinib: Given PO * Encorafenib: Given PO
General disorders Disease progression	16.7% 1/6 • Number of events 1 • 25 months
Renal and urinary disorders Acute kidney injury	16.7% 1/6 • Number of events 1 • 25 months

Other adverse events

Other adverse events
Measure	Treatment (Encorafenib, Binimetinib) n=6 participants at risk Patients receive encorafenib PO QD and binimetinib PO BID on days 1-25. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity. * Binimetinib: Given PO * Encorafenib: Given PO
Blood and lymphatic system disorders Anemia	33.3% 2/6 • Number of events 4 • 25 months
Blood and lymphatic system disorders Blood and lymph sys disorders - Oth Spec	33.3% 2/6 • Number of events 2 • 25 months
Eye disorders Eye disorders - Other, specify	16.7% 1/6 • Number of events 1 • 25 months
Eye disorders Floaters	33.3% 2/6 • Number of events 2 • 25 months
Gastrointestinal disorders Bloating	16.7% 1/6 • Number of events 4 • 25 months
Gastrointestinal disorders Diarrhea	33.3% 2/6 • Number of events 14 • 25 months
Gastrointestinal disorders Nausea	33.3% 2/6 • Number of events 2 • 25 months
Gastrointestinal disorders Vomiting	16.7% 1/6 • Number of events 1 • 25 months
General disorders Fatigue	83.3% 5/6 • Number of events 44 • 25 months
Infections and infestations Infections and infestations - Oth spec	16.7% 1/6 • Number of events 1 • 25 months
Investigations Alanine aminotransferase increased	16.7% 1/6 • Number of events 3 • 25 months
Investigations Aspartate aminotransferase increased	16.7% 1/6 • Number of events 1 • 25 months
Investigations CPK increased	16.7% 1/6 • Number of events 1 • 25 months
Investigations Neutrophil count decreased	33.3% 2/6 • Number of events 8 • 25 months
Investigations Weight gain	16.7% 1/6 • Number of events 3 • 25 months
Investigations Weight loss	16.7% 1/6 • Number of events 1 • 25 months
Investigations White blood cell decreased	16.7% 1/6 • Number of events 2 • 25 months
Metabolism and nutrition disorders Hypocalcemia	16.7% 1/6 • Number of events 1 • 25 months
Musculoskeletal and connective tissue disorders Arthralgia	16.7% 1/6 • Number of events 3 • 25 months
Musculoskeletal and connective tissue disorders Back pain	16.7% 1/6 • Number of events 1 • 25 months
Musculoskeletal and connective tissue disorders Myalgia	33.3% 2/6 • Number of events 8 • 25 months
Musculoskeletal and connective tissue disorders Pain in extremity	16.7% 1/6 • Number of events 13 • 25 months
Nervous system disorders Headache	16.7% 1/6 • Number of events 1 • 25 months
Respiratory, thoracic and mediastinal disorders Sinus disorder	16.7% 1/6 • Number of events 1 • 25 months
Skin and subcutaneous tissue disorders Rash acneiform	16.7% 1/6 • Number of events 2 • 25 months
Skin and subcutaneous tissue disorders Skin and subcut tissue disord - Oth spec	16.7% 1/6 • Number of events 3 • 25 months
Vascular disorders Thromboembolic event	33.3% 2/6 • Number of events 2 • 25 months

Additional Information

Andrew Hendifar, M.D.

Cedar Sinai Medical Center

Phone: 310-423-2217

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place