Trial Outcomes & Findings for Testing Trametinib as a Potential Targeted Treatment in Cancers With GNAQ or GNA11 Genetic Changes (MATCH-Subprotocol S2) (NCT NCT04439357)

Last Updated: 2025-09-16

Results Overview

Overall response rate was defined as the proportion of patients with best overall response of complete response (CR) or partial response (PR) among all eligible and treated patients. Best overall response was evaluated using the Response Evaluation Criteria in Solid Tumors version 1.1, the Cheson (2014) criteria for lymphoma patients, and the Response Assessment in Neuro-Oncology criteria for glioblastoma patients. Please refer to the protocol for the detailed definitions of response criteria. The 90% two-sided binomial exact confidence interval was calculated for ORR.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Tumor assessments occurred at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Results posted on

2025-09-16

Participant Flow

Seven patients were assigned to Subprotocol S2, 6 from screening cohort and 1 from outside laboratory. Of the 7 patients, 4 patients were enrolled in arm S2 between July 2016 and January 2019.

To be assigned to a specific MATCH subprotocol, patients needed to submit a tumor biopsy for molecular characterization and those with molecular variants addressed by treatments included in the trial entered corresponding MATCH subprotocol. For the subprotocol S2, patients had to have a qualifying GNAQ or GNA11 actionable mutation.

Participant milestones

Participant milestones
Measure	Treatment (Trametinib) Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
Overall Study STARTED	4
Overall Study Eligible and Started Protocol Therapy	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Trametinib) Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
Overall Study Disease Progression	4

Baseline Characteristics

Testing Trametinib as a Potential Targeted Treatment in Cancers With GNAQ or GNA11 Genetic Changes (MATCH-Subprotocol S2)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Trametinib) n=4 Participants Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
Age, Continuous	62 years n=5 Participants
Sex: Female, Male Female	3 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	4 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	0 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	4 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

PRIMARY outcome

Timeframe: Tumor assessments occurred at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Population: Patients who were eligible and received protocol treatment

Outcome measures

Outcome measures
Measure	Treatment (Trametinib) n=4 Participants Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
Objective Response Rate (ORR)	25 percentage of participants Interval 1.3 to 75.1

SECONDARY outcome

Timeframe: Assessed at baseline, then every 2 cycles for the first 26 cycles, and every 3 cycles thereafter until disease progression, up to 3 years post registration, from which 6-month PFS rate is determined

Population: Patients who were eligible and received protocol treatment

Progression free survival is defined as time from treatment start date to date of progression or death from any cause, whichever occurs first. Disease progression was evaluated using the Response Evaluation Criteria in Solid Tumors version 1.1, the Cheson (2014) criteria for lymphoma patients, and the Response Assessment in Neuro-Oncology criteria for glioblastoma patients. Please refer to the protocol for detailed definitions of disease progression. 6 month PFS rate was estimated using the Kaplan-Meier method, which can provide a point estimate for any specific time point.

Outcome measures

Outcome measures
Measure	Treatment (Trametinib) n=4 Participants Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
6-month Progression-free Survival (PFS) Rate	50 percentage of participants Interval 22.0 to 100.0

SECONDARY outcome

Timeframe: Assessed at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Population: Patients who were eligible and received protocol treatment

PFS was defined as time from treatment start date to date of disease progression or death from any causes, whichever occurred first. Median PFS was estimated using the Kaplan-Meier method. Disease progression was evaluated using the Response Evaluation Criteria in Solid Tumors version 1.1, the Cheson (2014) criteria for lymphoma patients, and the Response Assessment in Neuro-Oncology criteria for glioblastoma patients. Please refer to the protocol for detailed definitions of disease progression.

Outcome measures

Outcome measures
Measure	Treatment (Trametinib) n=4 Participants Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
Progression Free Survival (PFS)	6.5 months Interval 1.8 to Median PFS 90% CI upper bound could not be determined due to insufficient number of participants with events

Adverse Events

Treatment (Trametinib)

Serious events: 2 serious events

Other events: 4 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Trametinib) n=4 participants at risk Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
Vascular disorders Hypertension	50.0% 2/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis

Other adverse events

Other adverse events
Measure	Treatment (Trametinib) n=4 participants at risk Patients receive trametinib dimethyl sulfoxide 2mg PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Trametinib Dimethyl Sulfoxide: Given PO
General disorders Edema limbs	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
General disorders Fatigue	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Alopecia	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Dry skin	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Hirsutism	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Pruritus	50.0% 2/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Rash acneiform	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Rash maculo-papular	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, specify	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Gastrointestinal disorders Cheilitis	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Gastrointestinal disorders Diarrhea	75.0% 3/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Gastrointestinal disorders Dry mouth	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Gastrointestinal disorders Mucositis oral	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Gastrointestinal disorders Nausea	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Immune system disorders Allergic reaction	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Infections and infestations Skin infection	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Investigations Aspartate aminotransferase increased	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Investigations Neutrophil count decreased	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Investigations Investigations - Other, specify	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Metabolism and nutrition disorders Hypoalbuminemia	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Metabolism and nutrition disorders Hypomagnesemia	50.0% 2/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Musculoskeletal and connective tissue disorders Generalized muscle weakness	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Nervous system disorders Dizziness	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Nervous system disorders Dysgeusia	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Renal and urinary disorders Urine discoloration	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Reproductive system and breast disorders Reproductive system and breast disorders - Other, specify	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Vascular disorders Hypertension	50.0% 2/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis
Vascular disorders Lymphedema	25.0% 1/4 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis and all patients received protocol treatment were included in the toxicity analysis

Additional Information

Study Statistician

ECOG-ACRIN Statistical Office

Phone: 617-632-3012

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60