Trial Outcomes & Findings for Testing GDC-0449 (Vismodegib) as Potentially Targeted Treatment in Cancers With Smoothened or Patched 1 Mutant Tumors (MATCH - Subprotocol T) (NCT NCT06357988)

Last Updated: 2026-05-06

Results Overview

ORR is defined as the percentage of patients whose tumors have a complete or partial response to treatment among analyzable patients. Objective response is defined consistent with 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. Details about how to define complete response and partial response can be found in the master protocol. 90% two-sided binomial exact confidence interval is calculated for ORR.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

34 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

2026-05-06

Participant Flow

Subprotocol T was activated on February 25, 2016. Thirty-four patients were enrolled on EAY131-T between June 20, 2016, and September 22, 2020. Fifteen patients were enrolled on the basis of the results from the NCI-MATCH assay and 19 on the basis of the outside assay results.

Patients with Patched-1 (PTCH1) and Smoothened (SMO) alterations (excluding basal cell carcinoma) were assigned to sub-protocol T. The mutation status was determined by an NCI-MATCH approved laboratory for 19 patients in this arm, these cases had to be confirmed to be used in primary analysis.

Participant milestones

Participant milestones
Measure	Treatment (Vismodegib) Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	34
Overall Study Started Protocol Therapy	33
Overall Study Eligible, Treated and Mutation Status Confirmed	25
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	34

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Vismodegib) Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Overall Study Never Start Protocol Therapy	1
Overall Study Ineligible	2
Overall Study Mutation Status Not Confirmed	6
Overall Study Adverse Event	1
Overall Study Death	4
Overall Study Disease Progression	15
Overall Study Other Complicating Disease	1
Overall Study Withdrawal by Subject	4

Baseline Characteristics

Testing GDC-0449 (Vismodegib) as Potentially Targeted Treatment in Cancers With Smoothened or Patched 1 Mutant Tumors (MATCH - Subprotocol T)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Vismodegib) n=25 Participants Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Age, Continuous	65 years n=54 Participants
Sex: Female, Male Female	12 Participants n=54 Participants
Sex: Female, Male Male	13 Participants n=54 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=54 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	23 Participants n=54 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=54 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=54 Participants
Race (NIH/OMB) Asian	1 Participants n=54 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=54 Participants
Race (NIH/OMB) Black or African American	1 Participants n=54 Participants
Race (NIH/OMB) White	23 Participants n=54 Participants
Race (NIH/OMB) More than one race	0 Participants n=54 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=54 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: Eligible, treated and mutation status confirmed

Outcome measures

Outcome measures
Measure	Treatment (Vismodegib) n=25 Participants Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Objective Response Rate (ORR)	8 percentage of participants Interval 1.4 to 23.1

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: Eligible, treated and mutation status confirmed

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 (Vismodegib) n=25 Participants Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
6-month Progression-free Survival (PFS) Rate	24 percentage of participants Interval 8.8 to 39.2

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: Eligible, treated and mutation status confirmed

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 (Vismodegib) n=25 Participants Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Progression Free Survival	1.8 months Interval 1.7 to 3.5

Adverse Events

Treatment (Vismodegib)

Serious events: 2 serious events

Other events: 19 other events

Deaths: 27 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Vismodegib) n=33 participants at risk Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Cardiac disorders Supraventricular tachycardia	3.0% 1/33 • Number of events 2 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Renal and urinary disorders Acute kidney injury	3.0% 1/33 • Number of events 2 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).

Other adverse events

Other adverse events
Measure	Treatment (Vismodegib) n=33 participants at risk Patients receive vismodegib 150 mg orally (PO) daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Blood and lymphatic system disorders Anemia	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
General disorders Fatigue	33.3% 11/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Skin and subcutaneous tissue disorders Alopecia	21.2% 7/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Gastrointestinal disorders Constipation	12.1% 4/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Gastrointestinal disorders Diarrhea	12.1% 4/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Gastrointestinal disorders Nausea	15.2% 5/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Gastrointestinal disorders Stomach pain	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Gastrointestinal disorders Vomiting	12.1% 4/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Investigations Alkaline phosphatase increased	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Investigations Weight loss	21.2% 7/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Metabolism and nutrition disorders Anorexia	24.2% 8/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Metabolism and nutrition disorders Hypercalcemia	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Metabolism and nutrition disorders Hyponatremia	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Musculoskeletal and connective tissue disorders Generalized muscle weakness	9.1% 3/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Musculoskeletal and connective tissue disorders Myalgia	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, specify	15.2% 5/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Nervous system disorders Dizziness	9.1% 3/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Nervous system disorders Dysgeusia	18.2% 6/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).
Nervous system disorders Headache	6.1% 2/33 • Number of events 19 • 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. Thirty-three patients were included in the toxicity analysis (excluding one who did not receive treatment).

Additional Information

Study Statistician

ECOG-ACRIN Cancer Research Group

Phone: 617-632-3012

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60