Sorafenib Phase II Study for Japanese Anaplastic or Medullary Thyroid Carcinoma Patients

Study Overview
=================
Brief Summary
-----------------
The objectives of this study are to evaluate safety, efficacy and pharmacokinetics of sorafenib for the treatment of Japanese patients with anaplastic thyroid carcinoma (ATC) or locally advanced or metastatic medullary thyroid carcinoma (MTC).

Official Title
-----------------
A Multi-center, Single-arm, Open-label Phase II Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Sorafenib in Japanese Patients With Anaplastic Thyroid Carcinoma or Locally Advanced or Metastatic Medullary Thyroid Carcinoma

Conditions
-----------------
Thyroid Carcinoma

Intervention / Treatment
-----------------
* Drug: Sorafenib (Nexavar,BAY43-9006)


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Japanese patients with ATC (Anaplastic thyroid carcinoma) or locally advanced or metastatic MTC (medullary thyroid carcinoma) Not a candidate for surgery or radiotherapy with curative intent Histologically or cytologically confirmed ATC or MTC Measurable or non-measurable disease (but clinically evaluable) according to RECIST 1.1. Age >= 18 years Adequate bone marrow, liver and renal function to be conducted within 14 days prior to treatment Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 or 1 Life expectancy of at least 12 weeks Exclusion Criteria: Histologic subtypes of thyroid cancer other than anaplastic or medullary carcinoma Prior anti-cancer treatment with tyrosine kinase inhibitors, monoclonal antibodies (licensed or investigational) that target VEGF or VEGF (vascular endothelial growth factor) receptors or other targeted agents Prior chemotherapy for thyroid cancer (only one regimen is allowed) Major surgery, open biopsy, or significant traumatic injury within 30 days prior to enrollment in the study. Subjects with tracheal, bronchial or esophageal infiltration with significant risk of bleeding but without having received local treatment prior to enrollment in the study

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br>Sorafenib 400 mg bid continuous dose | Drug: Sorafenib (Nexavar,BAY43-9006)<br>* Sorafenib 400 mg will be administered orally,twice daily in a 28 day cycle<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of subjects with adverse events and serious adverse events as a measure of safety and tolerability |  | 6 months | 
| Change in red blood cell count |  | Baseline and 6 months | 
| Change in white blood cell count |  | Baseline and 6 months | 
| Change in alanine aminotransaminase level (ALT) |  | Baseline and 6 months | 
| Change in aspartate aminotransferase level (AST) |  | Baseline and 6 months | 
| Change in blood pressure |  | Baseline and 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best overall response based on RECIST 1.1 criteria |  | Baseline and every 56 days up to progressive disease,an expected average of 8 months | 
| Plasma concentration of sorafenib |  | Cycle 2 Day 1 | 
| Progression-free survival (PFS) |  | Baseline to progression or death by any reason | 
| Overall survival (OS) |  | Baseline to death by any reason | 
| Response rate (RR) | RR based on RECIST 1.1 criteria | Baseline and every 56 days up to progressive disease | 
| Disease control rate (DCR) | DCR based on RECIST 1.1 criteria | Baseline and every 56 days up to progressive disease | 
| Maximum reduction from baseline in the target lesion size |  | Baseline and every 56 days up to progressive disease | 
| Maximum percentage change of calcitonin and Carcinoembryonic antigen (CEA) values from baseline | MTC subjects only | Baseline and every 56 days up to progressive disease | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Anaplastic and medullary thyroid carcinoma, Thyroid Neoplasms