Thyroid CancerPhase 2NCT01709292
Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer
A Phase 2 study testing Vemurafenib (All Groups), Vemurafenib (Post Surgery) - Group A + C and Post Surgery - Group B in people with Thyroid Cancer, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thyroid Cancer
- What is being tested?
- Vemurafenib (All Groups), Vemurafenib (Post Surgery) - Group A + C and Post Surgery - Group B
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Nov 2012to 30 Sep 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Primary papillary thyroid cancer (PTC), which appears to be stage T3 or T4 on imaging or with macroscopic lymph node involvement AND requires surgical resection.
- Persistent or locally recurrent PTC with macroscopic lymph node involvement which requires surgical resection.
- Patients deemed inoperable (no scheduled surgery) are eligible for this trial, as they could be surgical candidates after treatment with vemurafenib. Inoperable patients must be naïve to therapies targeting the MAPK pathway.
- BRAF V600E mutation detected in the primary tumor or the recurrent/persistent tumor.
- Total bilirubin </= 1.5 x upper limit of normal (ULN). Patients with Gilbert's syndrome are excluded from this requirement. Aspartate transaminase (serum glutamic oxaloacetic transaminase) / alanine transaminase (serum glutamic pyruvic transaminase) (AST[SGOT]/ALT[SGPT]) </= 2.5 x ULN, (5 x ULN for patients with concurrent liver metastases). Serum creatinine </= within 1.5 x ULN. Absolute neutrophil count (ANC) >/= 1.0 x 10^9/L; platelets >/= 100 x 10^9/L, HgB>9 mg/dL
- Antiangiogenic therapy, specifically vascular endothelial growth factor (VEGF) and VEGF receptor (VEGFR) inhibitors, can interfere with wound healing and therefore will only be allowed if the agent has been discontinued for at least 14 days prior to day 1. Group C patients must be naïve to therapies which target mitogen-activated protein kinase (MAPK).
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- Ability to swallow pills.
- Eastern Cooperative Oncology Group (ECOG) performance status </= 2
- Age >/= 18
- Ability to provide consent.
Who cannot join
- Histological diagnosis other than PTC. Patients with anaplastic tumors are not eligible. However, patients whose tumors contain areas of un-differentiated or dedifferentiated histology may enroll provided the original diagnosis was clearly PTC, and the tumor histology remains predominantly papillary at enrollment.
- Refractory nausea and vomiting, malabsorption, or significant bowel resection that would preclude adequate absorption.
- Known hepatitis B or C virus (HBV or HCV) infection, unless the patient has been cleared for chemotherapy from experts on viral hepatitis (infectious disease specialists or hepatologists).
- Pregnant or lactating women. All pre-menopausal women being screened must have a negative serum pregnancy test within 14 days prior to commencement of dosing. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for >/= 1 year
- Untreated brain metastases.
- Chemotherapy or targeted therapy within 14 days or 5 half-lives (whichever is longer) prior to the start of study treatment.
- Patients with history of long QT syndrome, uncorrectable electrolyte abnormalities, or QTc>500msec.
- History of significant cardiac disease or uncontrolled arrhythmias.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Pharmacodynamic Study of Vemurafenib in the Neoadjuvant Setting in Patients With Locally Advanced Thyroid Cancer
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.
Read the detailed description
Study Drug Administration and Groups:
If you are found to be eligible to take part in this study, you will take 4 tablets of vemurafenib by mouth 2 times each day for 56 days. You should take each dose about 12 hours apart. If you miss taking the drug at a scheduled time by more than 4 hours, you should skip that dose and wait until the next dose.
After 56 days, the treatment you receive will depend on what group you are assigned to:
- If the disease has spread outside the neck, you will be in Group A.
- If the disease is able to be entirely removed through surgery, you will be in Group B.
- If the disease is unable to be operated on, you will be in Group C.
Study Visits:
At all visits, you will be asked about any side effects you may be having and about any other drugs you may be taking.
Before you take the study drug (Baseline):
- You will have a procedure called a laryngoscopy, in which a tube with a small camera in inserted into the nose to inspect your sinus, throat, and voice box.
- You will have a colonoscopy if you have a history of abnormal growth in the bowel (polyps or colon cancer). During this procedure a tube with a small camera is inserted in the anus to inspect your large bowel.
At Day 28:
- You will have a physical exam, including measurement of your vital signs.
- Your performance status will be recorded.
- You will have an EKG.
- Blood (about 1 tablespoon) will be drawn for routine tests.
Within 1 week before Day 56, you will have CT scans of the neck, chest, and abdomen to check the status of the disease.
At Day 56, if you are in Group A or B, you will have surgery to remove the tumor. You will sign a separate consent form that describes this surgery and its risks. If you are in Group C, you will have a core biopsy. During the surgery/biopsy, blood (about 1 teaspoon) will also be collected for biomarker testing.
Follow-Up:
Your follow-up after the surgery/biopsy will depend on which study group you are in.
Groups A and C:
If you are in Group A, you will stop taking the study drug for about 2-4 weeks after you have surgery. If you are in Group C, you will not stop taking the study drug. Every 4 weeks (after you resume taking the study drug if you are in Group A, and after the biopsy if you are in Group C):
- You will have a physical exam, including measurement of your vital signs and a skin exam.
- Your performance status will be recorded.
- Blood (about 1 tablespoon) will be drawn for routine tests.
- You will be asked about any other drugs you may be taking and about any side effects you may be having.
- If you are in Group A, these tests will also be performed the day you start taking the study drug again. You will also have an EKG.
Additionally, every 8 weeks (if you are in Group A or C), you will have CT scans of the neck, chest, and abdomen to check the status of the disease. Blood (about 1 teaspoon) will also be drawn for biomarker testing at the first of these visits.
About 1 year after your laryngoscopy and colonoscopy, these procedures will be performed again to check for side effects related to the study drug. If you did not receive a baseline colonoscopy, this procedure will be performed about 1 year after starting vemurafenib. If you continue on study drug for more than a year, you will have a laryngoscopy every 12 months.
At any time the study doctor thinks it is needed, you will have a colonoscopy and laryngoscopy.
About 1 year after the surgery/biopsy, you will return to clinic to check if the disease has come back. At this visit, you will have a CT scan or ultrasound of the neck, and blood (about 1 tablespoon) will be drawn for tests of your thyroid function.
If after 4 months the study doctor thinks it is in your best interest, you will only need to have study visits every 8 weeks. You will still have blood (about 1 tablespoon) drawn for routine tests at a local lab and you will be called by the study staff to check on you. Each call will last about 5-10 minutes.
If you are in Group C, and if during follow-up the study doctor decides that the disease can be operated on, you will be crossed over to Group A or B. You will have surgery and have the follow-up visits described for that study group.
Group B:
If you are in Group B, you will have a follow-up visit about 2 weeks after surgery. The following tests and procedures will be performed:
- You will have a physical exam, including measurement of your vital signs.
- Your performance status will be recorded.
- Blood (about 1 tablespoon) will be drawn for routine tests.
- You will be asked about any other drugs you may be taking and about any side effects you may be having.
You will have a second follow-up visit about 8 weeks after surgery. You will have a CT scan of the neck to check the status of the disease. You will have the option of continuing to receive vemurafenib if your doctor thinks it is in your best interest. If you do continue on vemurafenib, you will follow the Group A follow-up schedule (above).
About 1 year after your laryngoscopy and colonoscopy, these procedures will be performed again to check for side effects related to the study drug. If you did not receive a baseline colonoscopy, this procedure will be performed about 1 year after starting vemurafenib.
At any time the study doctor thinks it is needed, you will have a colonoscopy and laryngoscopy.
About 1 year after the surgery/biopsy, you will return to clinic to check if the disease has come back. At this visit, you will have a CT scan or ultrasound of the neck, and blood (about 1 tablespoon) will be drawn for tests of your thyroid function.
Length of Study:
If you are in Group A or C, you will continue to take the study drug for as long as the study doctor thinks it is in your best interest. If you are in Group B, you will take the study drug for 56 days and will no longer take it after surgery. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over when you have completed the follow-up visits.
End-of-Study Visit/Call:
After you stop taking the study drug, you will have an end-of-study visit or phone call. You will be asked about any other drugs you may be taking and about any side effects you may have had. If you are called, the call will last about 5-10 minutes.
This is an investigational study. Vemurafenib is FDA approved and commercially available for the treatment of late-stage melanoma. It is investigational to use this drug to treat thyroid cancer.
Up to 22 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Thyroid Cancer
- Being tested
- Vemurafenib (All Groups), Vemurafenib (Post Surgery) - Group A + C, Post Surgery - Group B
- Keywords
- Thyroid Cancer, ERK, Extracellular-signal-regulated kinase, Biomarkers, Papillary thyroid cancer, PTC, BRAF mutated, Vemurafenib, PLX4032, RO5185426, Zelboraf™
- Medical subject headings
- Thyroid Neoplasms, Thyroid Cancer, Papillary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vemurafenib (All Groups)Drug
Also called PLX4032, RO5185426, Zelboraf™
960 mg by mouth 2 times a day 56 days prior to surgery (patients not planned for surgical resection will have a core biopsy at day 56.
Vemurafenib (Post Surgery) - Group A + CDrug
Also called PLX4032, RO5185426, Zelboraf™
Group A: 960 mg by mouth twice a day 2 weeks post-surgery. Restaged 8 weeks after resuming drug. If patients demonstrate either stable or regressing disease, they will continue on vemurafenib with restaging occurring every 8 weeks until they are no longer benefitting from the drug.
Group C: Patients not scheduled for surgical resection undergo a CT scan and core biopsy at day 56. Vemurafenib 960 mg by mouth twice a day, continued unless there is evidence of progressive disease on day 56 CT scan. Patients restaged every 8 weeks and continue vemurafenib until no longer benefitting from the drug. Patients evaluated for resectability after each CT scan is performed. If patient is scheduled for resection, then patient will continue vemurafenib until surgery and will follow the same treatment schema as patients in Groups A and B.
Post Surgery - Group BOther
Patients who have had a complete surgical resection, and who do not have evidence of metastatic disease will discontinue vemurafenib after surgery. Patients restaged with CT neck 8 weeks after surgery.
| Study group | Role | What they receive |
|---|---|---|
| Vemurafenib - (Presurgery) | Experimental | 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). Receives: Drug: Vemurafenib (All Groups); Drug: Vemurafenib (Post Surgery) - Group A + C; Other: Post Surgery - Group B |
| Vemurafenib (Post Surgery) - Group A | Experimental | 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. Receives: Drug: Vemurafenib (Post Surgery) - Group A + C |
| Post Surgery - Group B | No intervention | Post Surgery - Group B: Patients discontinue vemurafenib after surgery but will be restaged with CT neck 8 weeks after surgery. |
| Vemurafenib - Group C | Experimental | 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. Receives: Drug: Vemurafenib (Post Surgery) - Group A + C |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in ERK (Extracellular-Signal-Regulated Kinase) Phosphorylation and Tumor Size
ERK phosphorylation and tumor size measured at baseline before first dose of Vemurafenib and again after 56 days of Vemurafenib. We will test whether correlation between percent change in ERK phosphorylation and percent change in tumor size is different from 0 with a t-test.
Measured: 56 days
Secondary outcomes (1)
Objective Response Rate
Objective response rate defined as proportion of patients who have had a partial response (PR) or complete response (CR) after initiation of therapy with Vemurafenib, using RECIST 1.1. All patients who complete baseline and day 56 scans evaluable for this endpoint.
Measured: 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2012 (estimated)
- Study start7 Nov 2012
- Primary completion30 Sep 2028 (estimated)
- Study completion30 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thyroid CancerStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Maria E. Cabanillas, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01709292
- Sponsor's protocol ID
- 2012-0471
- Other IDs
- NCI-2012-02171
- Status last verified
- Sep 2026
- First submitted
- 25 Jul 2012
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacodynamic Study of Vemurafenib in the Neoadjuvant Setting in Patients With Locally Advanced Thyroid Cancer
- ClinicalTrials.gov ID
- NCT01709292
- Acronym / sponsor ID
- 2012-0471
- Conditions
- Thyroid Cancer
- Keywords
- Thyroid Cancer, ERK, Extracellular-signal-regulated kinase, Biomarkers, Papillary thyroid cancer, PTC, BRAF mutated, Vemurafenib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 7 Nov 2012 (actual)
- Primary completion
- 30 Sep 2028 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 18 Oct 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study Drug Administration and Groups:
If you are found to be eligible to take part in this study, you will take 4 tablets of vemurafenib by mouth 2 times each day for 56 days. You should take each dose about 12 hours apart. If you miss taking the drug at a scheduled time by more than 4 hours, you should skip that dose and wait until the next dose.
After 56 days, the treatment you receive will depend on what group you are assigned to:
- If the disease has spread outside the neck, you will be in Group A.
- If the disease is able to be entirely removed through surgery, you will be in Group B.
- If the disease is unable to be operated on, you will be in Group C.
Study Visits:
At all visits, you will be asked about any side effects you may be having and about any other drugs you may be taking.
Before you take the study drug (Baseline):
- You will have a procedure called a laryngoscopy, in which a tube with a small camera in inserted into the nose to inspect your sinus, throat, and voice box.
- You will have a colonoscopy if you have a history of abnormal growth in the bowel (polyps or colon cancer). During this procedure a tube with a small camera is inserted in the anus to inspect your large bowel.
At Day 28:
- You will have a physical exam, including measurement of your vital signs.
- Your performance status will be recorded.
- You will have an EKG.
- Blood (about 1 tablespoon) will be drawn for routine tests.
Within 1 week before Day 56, you will have CT scans of the neck, chest, and abdomen to check the status of the disease.
At Day 56, if you are in Group A or B, you will have surgery to remove the tumor. You will sign a separate consent form that describes this surgery and its risks. If you are in Group C, you will have a core biopsy. During the surgery/biopsy, blood (about 1 teaspoon) will also be collected for biomarker testing.
Follow-Up:
Your follow-up after the surgery/biopsy will depend on which study group you are in.
Groups A and C:
If you are in Group A, you will stop taking the study drug for about 2-4 weeks after you have surgery. If you are in Group C, you will not stop taking the study drug. Every 4 weeks (after you resume taking the study drug if you are in Group A, and after the biopsy if you are in Group C):
- You will have a physical exam, including measurement of your vital signs and a skin exam.
- Your performance status will be recorded.
- Blood (about 1 tablespoon) will be drawn for routine tests.
- You will be asked about any other drugs you may be taking and about any side effects you may be having.
- If you are in Group A, these tests will also be performed the day you start taking the study drug again. You will also have an EKG.
Additionally, every 8 weeks (if you are in Group A or C), you will have CT scans of the neck, chest, and abdomen to check the status of the disease. Blood (about 1 teaspoon) will also be drawn for biomarker testing at the first of these visits.
About 1 year after your laryngoscopy and colonoscopy, these procedures will be performed again to check for side effects related to the study drug. If you did not receive a baseline colonoscopy, this procedure will be performed about 1 year after starting vemurafenib. If you continue on study drug for more than a year, you will have a laryngoscopy every 12 months.
At any time the study doctor thinks it is needed, you will have a colonoscopy and laryngoscopy.
About 1 year after the surgery/biopsy, you will return to clinic to check if the disease has come back. At this visit, you will have a CT scan or ultrasound of the neck, and blood (about 1 tablespoon) will be drawn for tests of your thyroid function.
If after 4 months the study doctor thinks it is in your best interest, you will only need to have study visits every 8 weeks. You will still have blood (about 1 tablespoon) drawn for routine tests at a local lab and you will be called by the study staff to check on you. Each call will last about 5-10 minutes.
If you are in Group C, and if during follow-up the study doctor decides that the disease can be operated on, you will be crossed over to Group A or B. You will have surgery and have the follow-up visits described for that study group.
Group B:
If you are in Group B, you will have a follow-up visit about 2 weeks after surgery. The following tests and procedures will be performed:
- You will have a physical exam, including measurement of your vital signs.
- Your performance status will be recorded.
- Blood (about 1 tablespoon) will be drawn for routine tests.
- You will be asked about any other drugs you may be taking and about any side effects you may be having.
You will have a second follow-up visit about 8 weeks after surgery. You will have a CT scan of the neck to check the status of the disease. You will have the option of continuing to receive vemurafenib if your doctor thinks it is in your best interest. If you do continue on vemurafenib, you will follow the Group A follow-up schedule (above).
About 1 year after your laryngoscopy and colonoscopy, these procedures will be performed again to check for side effects related to the study drug. If you did not receive a baseline colonoscopy, this procedure will be performed about 1 year after starting vemurafenib.
At any time the study doctor thinks it is needed, you will have a colonoscopy and laryngoscopy.
About 1 year after the surgery/biopsy, you will return to clinic to check if the disease has come back. At this visit, you will have a CT scan or ultrasound of the neck, and blood (about 1 tablespoon) will be drawn for tests of your thyroid function.
Length of Study:
If you are in Group A or C, you will continue to take the study drug for as long as the study doctor thinks it is in your best interest. If you are in Group B, you will take the study drug for 56 days and will no longer take it after surgery. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over when you have completed the follow-up visits.
End-of-Study Visit/Call:
After you stop taking the study drug, you will have an end-of-study visit or phone call. You will be asked about any other drugs you may be taking and about any side effects you may have had. If you are called, the call will last about 5-10 minutes.
This is an investigational study. Vemurafenib is FDA approved and commercially available for the treatment of late-stage melanoma. It is investigational to use this drug to treat thyroid cancer.
Up to 22 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Vemurafenib - (Presurgery) | Experimental | Drug: Vemurafenib (All Groups), Drug: Vemurafenib (Post Surgery) - Group A + C, Other: Post Surgery - Group BAll 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). |
| Vemurafenib (Post Surgery) - Group A | Experimental | Drug: Vemurafenib (Post Surgery) - Group A + CVemurafenib 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. |
| Post Surgery - Group B | No intervention | –Post Surgery - Group B: Patients discontinue vemurafenib after surgery but will be restaged with CT neck 8 weeks after surgery. |
| Vemurafenib - Group C | Experimental | Drug: Vemurafenib (Post Surgery) - Group A + CPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Primary papillary thyroid cancer (PTC), which appears to be stage T3 or T4 on imaging or with macroscopic lymph node involvement AND requires surgical resection.
- Persistent or locally recurrent PTC with macroscopic lymph node involvement which requires surgical resection.
- Patients deemed inoperable (no scheduled surgery) are eligible for this trial, as they could be surgical candidates after treatment with vemurafenib. Inoperable patients must be naïve to therapies targeting the MAPK pathway.
- BRAF V600E mutation detected in the primary tumor or the recurrent/persistent tumor.
- Total bilirubin </= 1.5 x upper limit of normal (ULN). Patients with Gilbert's syndrome are excluded from this requirement. Aspartate transaminase (serum glutamic oxaloacetic transaminase) / alanine transaminase (serum glutamic pyruvic transaminase) (AST[SGOT]/ALT[SGPT]) </= 2.5 x ULN, (5 x ULN for patients with concurrent liver metastases). Serum creatinine </= within 1.5 x ULN. Absolute neutrophil count (ANC) >/= 1.0 x 10^9/L; platelets >/= 100 x 10^9/L, HgB>9 mg/dL
Key exclusion criteria (8 listed)
- Histological diagnosis other than PTC. Patients with anaplastic tumors are not eligible. However, patients whose tumors contain areas of un-differentiated or dedifferentiated histology may enroll provided the original diagnosis was clearly PTC, and the tumor histology remains predominantly papillary at enrollment.
- Refractory nausea and vomiting, malabsorption, or significant bowel resection that would preclude adequate absorption.
- Known hepatitis B or C virus (HBV or HCV) infection, unless the patient has been cleared for chemotherapy from experts on viral hepatitis (infectious disease specialists or hepatologists).
- Pregnant or lactating women. All pre-menopausal women being screened must have a negative serum pregnancy test within 14 days prior to commencement of dosing. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for >/= 1 year
- Untreated brain metastases.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vemurafenib (All Groups)Also: PLX4032, RO5185426, Zelboraf™960 mg by mouth 2 times a day 56 days prior to surgery (patients not planned for surgical resection will have a core biopsy at day 56. | Drug | Vemurafenib - (Presurgery) |
| Vemurafenib (Post Surgery) - Group A + CAlso: PLX4032, RO5185426, Zelboraf™Group A: 960 mg by mouth twice a day 2 weeks post-surgery. Restaged 8 weeks after resuming drug. If patients demonstrate either stable or regressing disease, they will continue on vemurafenib with restaging occurring every 8 weeks until they are no longer benefitting from the drug. Group C: Patients not scheduled for surgical resection undergo a CT scan and core biopsy at day 56. Vemurafenib 960 mg by mouth twice a day, continued unless there is evidence of progressive disease on day 56 CT scan. Patients restaged every 8 weeks and continue vemurafenib until no longer benefitting from the drug. Patients evaluated for resectability after each CT scan is performed. If patient is scheduled for resection, then patient will continue vemurafenib until surgery and will follow the same treatment schema as patients in Groups A and B. | Drug | Vemurafenib (Post Surgery) - Group A, Vemurafenib - (Presurgery), Vemurafenib - Group C |
| Post Surgery - Group BPatients who have had a complete surgical resection, and who do not have evidence of metastatic disease will discontinue vemurafenib after surgery. Patients restaged with CT neck 8 weeks after surgery. | Other | Vemurafenib - (Presurgery) |
Primary outcome
Percent Change in ERK (Extracellular-Signal-Regulated Kinase) Phosphorylation and Tumor Size
Time frame: 56 days
ERK phosphorylation and tumor size measured at baseline before first dose of Vemurafenib and again after 56 days of Vemurafenib. We will test whether correlation between percent change in ERK phosphorylation and percent change in tumor size is different from 0 with a t-test.
1 secondary outcome
Objective Response Rate
Time frame: 56 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2026.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Bladder Carcinoma, Cervical Carcinoma
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177Lu-DOTA-EB-TATE in Adult Patients With Metastatic, Radioactive Iodine Non-Responsive Oncocytic (Hurthle-Cell) Thyroid Cancer
Thyroid Cancer
RecruitingObservational study
DICER1-related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study
Pleuropulmonary Blastoma, Cystic Nephroma
Recruiting
Evaluation of DNA Methylation Signatures for the Diagnosis and Management of Thyroid Nodules
Thyroid Gland Carcinoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maria E. Cabanillas, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vemurafenib (All Groups), Vemurafenib (Post Surgery) - Group A + C and Post Surgery - Group B in people with Thyroid Cancer, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change in ERK (Extracellular-Signal-Regulated Kinase) Phosphorylation and Tumor Size (time frame: 56 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026.