Trial Outcomes & Findings for A Screening Study to Detect BRAF V600 Mutation-Positive Patients For Enrollment Into Clinical Research Studies of Zelboraf (Vemurafenib) (NCT NCT01804140)

Last Updated: 2016-11-02

Results Overview

Formalin-fixed paraffin-embedded (FFPEs) tumor samples (at least 5 serially-cut, unstained, 5 micrometer \[μm\] sections) were collected from eligible participants who consented to participate in the study. FFPE tumor samples were either from archived sections (from the initial diagnosis of cancer) or from fresh biopsies that were performed according to local standards. Tumor samples were then sent to a central laboratory to identify activating BRAF V600 mutations. Identification of mutations was done using bidirectional direct Sanger sequencing procedure.

Recruitment status

COMPLETED

Target enrollment

662 participants

Primary outcome timeframe

Up to 1 year

Results posted on

2016-11-02

Participant Flow

Participant milestones

Participant milestones
Measure	Participants With Confirmed Solid Tumors or Multiple Myeloma Participants with solid tumors (other than metastatic melanoma or papillary thyroid cancer) or multiple myeloma who met the inclusion criteria and did not meet exclusion criteria of the study were enrolled and were evaluated for the presence of BRAF V600 mutations by collecting tumor samples or performing fresh biopsies according to local standards.
Overall Study STARTED	662
Overall Study COMPLETED	548
Overall Study NOT COMPLETED	114

Reasons for withdrawal

Reasons for withdrawal
Measure	Participants With Confirmed Solid Tumors or Multiple Myeloma Participants with solid tumors (other than metastatic melanoma or papillary thyroid cancer) or multiple myeloma who met the inclusion criteria and did not meet exclusion criteria of the study were enrolled and were evaluated for the presence of BRAF V600 mutations by collecting tumor samples or performing fresh biopsies according to local standards.
Overall Study No sampling or biopsy procedures done	114

Baseline Characteristics

A Screening Study to Detect BRAF V600 Mutation-Positive Patients For Enrollment Into Clinical Research Studies of Zelboraf (Vemurafenib)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Participants With Confirmed Solid Tumors or Multiple Myeloma n=548 Participants Participants with solid tumors (other than metastatic melanoma or papillary thyroid cancer) or multiple myeloma who met the inclusion criteria and did not meet exclusion criteria of the study were enrolled and were evaluated for the presence of BRAF V600 mutations by collecting tumor samples or performing fresh biopsies according to local standards.
Age, Continuous	63.3 years STANDARD_DEVIATION 12.38 • n=5 Participants
Gender Female	326 Participants n=5 Participants
Gender Male	222 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 1 year

Population: All the participants who were enrolled and were evaluable for the BRAF V600 mutations were included in the analysis. n is the number of participants with that type of cancer in the total population.

Outcome measures

Outcome measures
Measure	Participants With Confirmed Solid Tumors or Multiple Myeloma n=548 Participants Participants with solid tumors (other than metastatic melanoma or papillary thyroid cancer) or multiple myeloma who met the inclusion criteria and did not meet exclusion criteria of the study were enrolled and were evaluated for the presence of BRAF V600 mutations by collecting tumor samples or performing fresh biopsies according to local standards.
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Breast cancer (n=85)	0 percentage of participants Interval 0.0 to 0.0
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Non-small cell lung carcinoma (n=65)	3 percentage of participants Interval 0.4 to 10.7
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Colorectal cancer (n=74)	11 percentage of participants Interval 4.8 to 20.2
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Ovarian cancer (n=66)	0 percentage of participants Interval 0.0 to 0.0
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Prostate cancer (n=21)	0 percentage of participants Interval 0.0 to 0.0
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Multiple myeloma (n=31)	3 percentage of participants Interval 0.1 to 16.7
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Biliary tract cancer (n=16)	6 percentage of participants Interval 0.2 to 30.2
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Other cancer (n=190)	2 percentage of participants Interval 0.6 to 5.3
Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type Overall (n=540)	3 percentage of participants Interval 1.7 to 4.7

PRIMARY outcome

Timeframe: Up to 1 year

Population: All the participants who were enrolled and were evaluable for the BRAF V600 mutations were included in the analysis.

FFPEs tumor samples (at least 5 serially-cut, unstained, 5 μm sections) were collected from eligible participants who consented to participate in the study. FFPE tumor samples were either from archived sections (from the initial diagnosis of cancer) or from fresh biopsies that were performed according to local standards. Tumor samples were then sent to a central laboratory to identify activating BRAF V600 mutations. Identification of mutations was done using bidirectional direct Sanger sequencing procedure. V600E, V600K, V600D, and V600R are the different types of BRAF V600 mutations.

Outcome measures

Outcome measures
Measure	Participants With Confirmed Solid Tumors or Multiple Myeloma n=548 Participants Participants with solid tumors (other than metastatic melanoma or papillary thyroid cancer) or multiple myeloma who met the inclusion criteria and did not meet exclusion criteria of the study were enrolled and were evaluated for the presence of BRAF V600 mutations by collecting tumor samples or performing fresh biopsies according to local standards.
Number of Participants Classified Based on Different Types of BRAF V600 Mutation Patterns in Tumor Samples BRAF V600E	16 participants
Number of Participants Classified Based on Different Types of BRAF V600 Mutation Patterns in Tumor Samples BRAF V600K	0 participants
Number of Participants Classified Based on Different Types of BRAF V600 Mutation Patterns in Tumor Samples BRAF V600D	0 participants
Number of Participants Classified Based on Different Types of BRAF V600 Mutation Patterns in Tumor Samples BRAF V600R	0 participants
Number of Participants Classified Based on Different Types of BRAF V600 Mutation Patterns in Tumor Samples Other V600	0 participants

Adverse Events

Participants With Confirmed Solid Tumors or Multiple Myeloma

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Medical Communications

Hoffmann-LaRoche

Phone: 800-821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Publication restrictions are in place

Restriction type: OTHER