Neoplasms

Blood and Lymph Conditions

All Conditions

Immune System Diseases

Rare Diseases

Leukemia

Leukemia, Hairy Cell

Neoplasms by Histologic Type

Hematologic Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hairy Cell Leukemia

Antineoplastic Agents

All Drugs and Chemicals

Vemurafenib

Protein Kinase Inhibitors

Enzyme Inhibitors

Eligible patients will receive vemurafenib at a dose of 960mg orally twice daily (b.i.d.) continuously in cycles of 4 weeks (28 days).

Patients will receive vemurafenib at a dose of 960mg orally b.i.d. continuously in cycles of 4 weeks (28 days) as outpatient. A bone marrow aspirate and/or biopsy will be performed after the first cycle for research purposes only. After the completion of the third cycle, a repeat bone marrow aspirate and/or biopsy will be performed for assessment of response and evaluation of MRD. Following the third cycle assessments, patients who achieve complete response (CR) with detectable MRD or partial response (PR) may continue with vemurafenib for up to 3 additional cycles at the treating physician's discretion (Cycles 4-6). Patients who achieve CR without MRD will be observed as part of post-treatment followup, and may be re-treated with vemurafenib after relapse (as per below). Patients who achieve no response (NR) after the initial 3 cycles of vemurafenib will be removed from the study. They will be followed every 3 months as part of posttreatment followup for a total of 12 months.

Jae H. Park, MD

The purpose of this study is to find out what effects, good and/or bad, treatment with vemurafenib (also known as Zelboraf™) has on the patient and on leukemia. Specifically, the researchers want to know how well vemurafenib eliminates leukemia from the blood.

BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia

A Phase II Study of the BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia

as assessed by overall response rates after three months of treatment in patients with relapsed or refractory HCL.

Toxicity will be graded and recorded using the NCI Common Toxicology Criteria version 4.0.

Peripheral blood and/or bone marrow aspirate samples from pretreatment and post-treatment at specified time points will be assessed by Western Blot or by phospho-flow for the downstream targets of BRAF (MEK, pMEK, ERK, pERK) to assess the ontarget effect of the Vemurafenib.

Reactivation of MAPK pathways: Increased expression of the other RAF isoforms CRAF and ARAF), and MAPK (MAPK8 or COT) will be analyzed by Western Blot and/or real-time PCR39,40. Secondary BRAF mutations (all 18 BRAF exons) and RAS mutations40 will be analyzed by bidirectional Sanger sequencing and by Raindance multiplex PCR and Illumina next generation sequencing, respectively. Activation of RTKs (i.e. PDGFRβ and IGF-IR) will be assessed by Western Blot. Cell Biosciences NanoPro 1000 technology will be used to examine quantitative signaling on the entire MAPK, PI3K and JAK-STAT pathways41.

National Cancer Institute (NCI)

Northwestern University

Ohio State University

Dana-Farber Cancer Institute

Scripps Clinic

Northwell Health

Memorial Sloan Kettering Cancer Center

Gland and Hormone Related Diseases

Thyroid Neoplasms

Thyroid Diseases

Thyroid Cancer, Papillary

Endocrine System Diseases

Endocrine Gland Neoplasms

Head and Neck Neoplasms

Papillary Thyroid Carcinoma

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).

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.

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

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.

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 (All Groups)

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.

Vemurafenib (Post Surgery) - Group A + C

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.

Post Surgery - Group B

Maria E. Cabanillas, MD

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.

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.

Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer

Pharmacodynamic Study of Vemurafenib in the Neoadjuvant Setting in Patients With Locally Advanced Thyroid Cancer

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.

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.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia	NCT01711632	Hairy Cell Leuk...	Vemurafenib	18 Years -	Memorial Sloan Kettering Cancer Center
Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer	NCT01709292	Thyroid Cancer	Vemurafenib (Al... Vemurafenib (Po... Post Surgery - ...	18 Years -	M.D. Anderson Cancer Center
Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer	NCT01709292	Thyroid Cancer	Vemurafenib (Al... Vemurafenib (Po... Post Surgery - ...	18 Years -	M.D. Anderson Cancer Center

Spots Global Cancer Trial Database for zelboraf™

Every month we try and update this database with for zelboraf™ cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for zelboraf™

Every month we try and update this database with for zelboraf™ cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for zelboraf™ cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.