Vemurafenib for Thyroid Cancer

Recruiting in Palo Alto (17 mi)

Overseen by Maria E. Cabanillas, M.D.

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: M.D. Anderson Cancer Center

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

The goal of this clinical research study is to learn about how vemurafenib may affect certain biomarkers in patients with PTC. Biomarkers are in the blood/tissue and may be related to your reaction to the study drug. The safety of this drug will also be studied. Vemurafenib is designed to block the BRAF gene mutation. This mutation causes cancer and cancer growth. By blocking this mutation, the drug may kill the cancer cells with the mutation and/or stop the tumor from growing.

Research Team

Maria E. Cabanillas, M.D.

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

Adults with advanced papillary thyroid cancer (PTC) that's inoperable may join this trial. They should have the BRAF V600E mutation, be new to MAPK pathway therapies, and have their major organs functioning well. Pregnant women, those with significant heart disease or untreated brain metastases, or recent chemotherapy/targeted therapy users are excluded.

Inclusion Criteria

Your blood creatinine level is within a certain range.

Your absolute neutrophil count is at least 1.0 x 10^9 per liter.

Your hemoglobin level is higher than 9 mg/dL.

Exclusion Criteria

I have severe nausea, vomiting, or issues absorbing food due to bowel surgery.

I have brain metastases that have not been treated.

I haven't had chemotherapy or targeted therapy within the last 14 days or 5 half-lives, whichever is longer.

Treatment Details

Interventions

• Vemurafenib (Kinase Inhibitor)

Trial Overview The study tests vemurafenib's effects on PTC biomarkers before surgery (neoadjuvant). All participants receive vemurafenib; Group A and C continue post-surgery. The drug aims to block a gene mutation responsible for cancer growth.

Participant Groups

Group I: Vemurafenib - Group C Experimental Treatment 1 Intervention

Patients not scheduled for surgical resection undergo a CT scan and core biopsy at day 56, Vemurafenib 960 mg by mouth twice a day unless there is evidence of progressive disease on day 56 CT scan. Patients evaluated for resectability after each CT scan is performed. If scheduled for resection, patient continues vemurafenib until surgery and follows same treatment schema as patients in Groups A and B.

Group II: Vemurafenib - (Presurgery) Experimental Treatment 3 Interventions

All Groups: Vemurafenib 960 mg by mouth 2 times a day for 56 days prior to surgery (patients not planned for surgical resection will have a core biopsy at day 56 +/- 7 days).

Group III: Vemurafenib (Post Surgery) - Group A Experimental Treatment 1 Intervention

Vemurafenib 960 mg by mouth two times a day 2 weeks post-surgery, or if patients have not sufficiently recovered at that point, as soon as their condition permits. Patients in Group A restaged 8 weeks after resuming drug. If patients in Group A demonstrate either stable or regressing disease, they will continue on vemurafenib with restaging occurring every 8 weeks until no longer benefitting from the drug.

Group IV: Post Surgery - Group B Active Control 1 Intervention

Post Surgery - Group B: Patients discontinue vemurafenib after surgery but will be restaged with CT neck 8 weeks after surgery.

Vemurafenib is already approved in Canada for the following indications:

Approved in Canada as Zelboraf for:

• Unresectable or metastatic melanoma with BRAF V600E mutation

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Dr. Peter WT Pisters

M.D. Anderson Cancer Center

Chief Executive Officer since 2017

MD from University of Western Ontario

Dr. Jeffrey E. Lee

M.D. Anderson Cancer Center

Chief Medical Officer

MD from Stanford University School of Medicine

Genentech, Inc.

Industry Sponsor

Trials

1,578

Recruited

569,000+

Ashley Magargee

Genentech, Inc.

Chief Executive Officer since 2024

MBA from Harvard University, BA from Princeton University

Levi Garraway

Genentech, Inc.

Chief Medical Officer since 2021

MD, PhD