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Study Summary
No. 2011-0448:.......Thyroid......Maria E. Cabanillas......Endocrinology
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Study Summary Title
Study Summary
Number:
2011-0448
Study Title:An Open-Label, Multi-Center Phase II Study of the BRAF Inhibitor Vemurafenib in Patients with Metastatic or Unresectable Papillary Thyroid Cancer (PTC) positive for the BRAF V600 Mutation and Resistant to Radioactive Iodine
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Physician New Patient Referral
Name:Maria E. CabanillasPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:EndocrinologyReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-563-0764
Contact us about clinical trials
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General Information
Disease Group:ThyroidSupported By:F. Hoffman-LaRoche
Phase of Study:Phase IIReturn
Visit:
Treatment
Agents:
RO5185426Home Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
The goal of this clinical research study is to learn if vemurafenib can help to
control papillary thyroid cancer. Researchers will also be studying the drug's
effect on biomarkers (proteins in the blood/tissue that may be related to your
reaction to the study drug). The safety and possible side effects 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.
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Study Objectives / Outcomes
Primary:
  • To evaluate best overall response rate (BORR; defined as complete response [CR] + partial response [PR]) in TKI-naïve patients using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
Secondary:
  • To evaluate clinical benefit (defined as overall response rate [ORR] + stable disease [SD]) in TKI -naïve patients
  • To further assess efficacy of Vemurafenib using the following secondary variables:
    duration of response, progression-free survival (PFS), and overall survival (OS) in TKI-naïve patients
  • To evaluate efficacy (BORR, clinical benefit rate (CBR), duration of response, PFS, and OS) in TKI exposed patients
  • To evaluate the tolerability and safety profile of Vemurafenib using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE - version 4.0)
    (version 4.0) in both TKI naïve and TKI treated patients
  • To characterize the pharmacokinetic (PK) profile of Vemurafenib in patients with thyroid cancer

Exploratory:
  • To evaluate potential exploratory biomarkers that may be relevant:
    - To predict responsiveness and acquired resistance to Vemurafenib
    - To investigate mechanism of action and pharmacodynamic effects of Vemurafenib
  • To evaluate the molecular characterization of SCCs (cutaneous and non-cutaneous) or other new primary neoplasms that may be observed in patients treated with Vemurafenib
  • To evaluate the Roche Companion Diagnostic (CoDx) cobas® 4800 BRAFV600 Test for the detection of BRAFV600 from formalin-fixed paraffin-embedded (FFPE) thyroid tumor samples
  • To correlate prevalence of BRAFV600 mutations in tumor and blood
  • To correlate serum thyroglobulin levels and clinical response
  • To correlate early positron emission tomography (PET) scan response and clinical response
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Study Status Information
Study Activation / Registration Date:10/24/2011
IRB Review and Approval Date:10/24/2011
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Closed
Projected Accrual:40-50
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Male or female patients >/= 18 years of age

2) Histologically confirmed papillary thyroid cancer that is metastatic or unresectable and for which standard curative or palliative measures do not exist or are no longer effective. NOTE: Patients whose tumors exhibit areas of "other histology" may be enrolled, provided the tumor histology remains predominantly papillary. Patients whose tumors exhibit "mixed" histology may be discussed with the Medical Monitor if there are questions about eligibility.

3) BRAF V600-positive thyroid cancer tissue, as determined by the Roche-designated Central Reference Laboratory using the cobas® 4800 BRAF V600 Mutation Test. Testing requires a formalin-fixed paraffin-embedded tumor tissue block or unstained sections from such a block. Samples may be either archival or new. Fine needle and core needle biopsies will not be accepted.

4) Must have radioactive iodine (RAI) resistant disease, defined by any one of the following: (1) lack of RAI uptake on either a low-dose diagnostic or a post-therapy RAI scan in the measurable lesion (or lesions) demonstrated previously (without time limitation), or (2) radiographic progression of disease within 18 months of last course of RAI therapy despite the presence of RAI uptake on the scans performed with that prior therapy, or (3) patient that has exceeded a cumulative activity of at least 600 mCi of radioiodine therapy

5) Thyroid carcinoma tissue, either archival or recent biopsy, must be available for submission and review by a central pathology laboratory.

6) Allowed Prior therapy: Cohort 1 may have received surgery, RAI, and/or standard of care conventional chemotherapy (e.g., doxorubicin). Cohort 2: may have received surgery, RAI and standard of care chemotherapy (e.g., doxorubicin), and must also have received prior treatment with investigational or commercial tyrosine kinase inhibitor with activity against VEGFR2 provided the drug is not a specific/selective BRAF or MEK pathway inhibitor. [Note: Acceptable prior chemotherapy can be discussed with the sponsor].

7) Must have fully recovered from the effects of any previous therapies.

8) Radiologic (computed tomography [CT] or magnetic resonance imaging [MRI]) evidence of clinically relevant disease progression (as per RECIST 1.1) within the preceding 14 months prior to planned first treatment.

9) Measurable disease (by RECIST Version 1.1 criteria)

10) Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1

11) Life expectancy > 3 months

12) Ability to swallow pills

13) Must have a head CT/MRI to evaluate for central nervous system (CNS) metastasis within 28 days prior to study drug treatment (Cycle 1 Day 1). Patients with radiographically stable, asymptomatic previously treated lesions are eligible provided: (1) Patient has received prior treatment [including radiation therapy, stereotactic radiosurgery, surgical resection] to the site(s) of CNS metastatic disease >/= 28 days prior to starting study treatment, (2) Patient has no requirement for glucocorticoids, and discontinued >/= 21 days prior to starting study treatment), (3) Patient is not taking anticonvulsants (discontinuedat least 3 weeks prior to treatment), and (4) Patient has no overt evidence of neurological deficit

14) Prior Surgery (excluding tumor biopsy at baseline for biomarker analysis) must have occurred at least 14 days prior to first dose of study treatment, and patients must have recovered from any effects from surgery and have adequate wound healing prior to first dose of study treatment.

15) External beam radiotherapy for the treatment of a symptomatic (e.g. bone) metastasis as clinically indicated must be at least 14 days prior to first dose of study treatment

16) Thyroid stimulating hormone (TSH) level <0.5 mIU/L

17) Completed baseline skin exam by a dermatologist or other qualified physician for cutaneous squamous cell carcinoma. Exam must be negative or if, suspected cutaneous squamous-cell carcinoma (SCC), basal-cell carcinoma (BCC), or other suspicious lesions are identified they must be excised, and there must be adequate wound healing prior to study treatment.

18) Adequate hematologic, renal and liver function: (1) Absolute neutrophil count (ANC) >1.5 x 10^9/L, (2) Platelet count >100 x 10^9/L, (3) Hemoglobin >9 g/dL , (4) Serum creatinine </= 1.5 X ULN or CrCl>40 ml/hr by Cockcroft-Gault formula , (5) AST and ALT </= 2.5 times upper limit of normal (ULN) (</= 5 times ULN for patients with concurrent liver metastases) , (6) Bilirubin </= 1.5 times ULN, (7) Alkaline phosphatase </= 2.5 times ULN (</= 5 times ULN for patients with concurrent liver metastases)

19) Negative serum pregnancy test within 14 days prior to commencement of dosing in pre-menopausal women. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for >/= 1 year

20) Fertile men and women must use an effective method of contraception during treatment and for at least 6 months after completion of treatment as directed by their physician (in accordance with local requirements)

21) Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before trial entry

22) Before study entry, signed informed consent must be obtained from patient prior to performing any studyrelated procedures

Exclusion Criteria:1) Histological diagnosis other than papillary thyroid carcinoma (PTC), including squamous cell variants of PTC or PTC with areas of squamous metaplasia. NOTE: 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. Patients whose tumors exhibit mixed histology may be discussed with the Medical Monitor.

2) Active or untreated CNS metastases.

3) History of, or known, carcinomatous meningitis

4) Anticipated or ongoing administration of any anti-cancer therapies other than those administered in this study

5) Active squamous cell skin cancer that has not been excised or has not yet adequately healed post excision

6) Pregnant or lactating women

7) Previous treatment with any commercial or investigational targeted agents that specifically and selectively targets the MEK or BRAF pathway

8) Received any investigational treatment within 28 days prior to start of study treatment

9) Prior radioactive iodine therapy within 28 days prior to start of study treatment

10) Chemotherapy or targeted therapy (in case of cohort 2) within 28 days prior to start of study treatment

11) Prior radiotherapy to the only measurable lesion.

12) Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant bowel resection that would preclude adequate absorption.

13) QTc> 450 msec on screening ECG or history of congenital long QT syndrome or uncorrectable electrolytes abnormalities. Note: At least one QTc measurement using Fridericia's correction (QTcF) must be 450 msec. Additionally, cases of patients with stable asymptomatic conduction delays (e.g., right bundle branch block) with QTc >450 msec may be discussed with the Medical Monitor for potential inclusion.

14) NCI CTCAE Version 4.0 grade 3 hemorrhage within 28 days of starting the study treatment

15) Any of the following within the 6 months prior to study drug administration: myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, or active pulmonary embolism

16) Known clinically significant active infection at the time of study treatment start

17) History of allogeneic bone marrow transplantation or organ transplantation

18) Other severe, acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results, which in the judgment of the investigator would make the patient inappropriate for entry into this study

19) Patients with a previous malignancy are excluded except for patients with adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer and/or curatively treated cancer, from which the patient is currently disease-free, or any malignancy from which the patient has been continuously disease-free for at least 5 years. Also, isolated elevation in prostate-specific antigen (PSA) in the absence of prostate cancer is allowed

20) Known human immunodeficiency virus (HIV) positivity or acquirred immune deficiency syndrome (AIDS)-related illness, hepatitis B virus (HBV), or active hepatitis C virus (HCV)

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Links
Registration Number: NCT01286753
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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