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A Phase II Trial of Bay 43-9006 in Progressive Metastatic Neuroendocrine Tumors

Phase 2
18 Years
Open (Enrolling)
Gastrinoma, Glucagonoma, Insulinoma, Metastatic Gastrointestinal Carcinoid Tumor, Neuroendocrine Tumor, Pancreatic Polypeptide Tumor, Recurrent Gastrointestinal Carcinoid Tumor, Recurrent Islet Cell Carcinoma, Somatostatinoma, WDHA Syndrome

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Trial Information

A Phase II Trial of Bay 43-9006 in Progressive Metastatic Neuroendocrine Tumors


I. To determine the objective tumor response rate of BAY 43-9006 (sorafenib tosylate) in
patients with advanced neuroendocrine tumors.


I. Adverse event rate(s). II. Progression free survival and time to progression. III.
Improvement in circulating hormone levels. IV. Overall survival.

OUTLINE: This is a multicenter study. Patients are stratified according to tumor type
(carcinoid vs islet cell/other well-differentiated tumor).

Patients receive oral sorafenib tosylate twice daily on days 1-28. Courses repeat every 28
days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed every 3 months until disease
progression and then every 6 months for up to 2 years from study entry.

Inclusion Criteria


- Histologically confirmed neuroendocrine tumor:

- Carcinoid tumor OR islet cell carcinoma/other well-differentiated tumor

- No anaplastic or high-grade histology

- Metastatic disease

- Measurable disease

- No thyroid carcinoma of any histology, thymoma, or pheochromocytoma/paraganglioma

- No known brain metastases

- Performance status:

- ECOG 0-2

- Life expectancy:

- At least 24 weeks

- Hematopoietic:

- Absolute neutrophil count >= 1,500/mm3

- Platelet count >= 100,000/mm3

- No bleeding diathesis

- Hepatic:

- Bilirubin =< 2 times upper limit of normal (ULN)

- AST =< 3 times ULN (5 times ULN if liver metastases are present)

- INR normal

- PTT normal

- Renal:

- Creatinine =< 1.5 times ULN

- Cardiovascular:

No poorly controlled hypertension; No symptoms of congestive heart failure; No unstable
angina pectoris; No cardiac arrhythmia

- Gastrointestinal:

- Able to swallow capsules intact

- No gastrointestinal tract disease resulting in an inability to take oral
medication (e.g., dysphagia)

- No requirement for IV alimentation

- No active peptic ulcer disease

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No ongoing or active infection

- No psychiatric illness or social situation that would preclude study compliance

- No other invasive malignancy within the past 3 years except adequately treated basal
cell or squamous cell skin cancer or carcinoma in situ of the cervix

- No other uncontrolled illness

- At least 4 weeks since prior interferon

- No more than 1 prior systemic chemotherapy regimen:

Chemoembolization is not considered systemic chemotherapy

- At least 4 weeks since prior chemoembolization

- At least 3 weeks since prior radiotherapy

- No prior procedures adversely affecting intestinal absorption

- At least 4 weeks since prior hepatic artery embolization

- No other prior systemic therapy

- No other concurrent investigational treatment

- No concurrent combination antiretroviral therapy for HIV-positive patients

- No concurrent enzyme-inducing anticonvulsants (e.g., carbamazepine, phenobarbital, or

- No concurrent rifampin

- No concurrent Hypericum perforatum (St. John's wort)

- Prior or concurrent octreotide for symptomatic treatment allowed

- No concurrent therapeutic anticoagulation:

Concurrent prophylactic anticoagulation (i.e., low dose warfarin) of venous or arterial
access devices allowed provided requirements for INR or PTT are met

- At least 4 weeks since prior major surgery

- Recovered from all prior therapy

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Confirmed response rate (CR or PR) estimated by the number of successes divided by the total number of evaluable patients

Outcome Description:

Kaplan-Meier methodology will be used to estimate the final success proportion (i.e., confirmed response rate with a 95% confidence interval).

Outcome Time Frame:

Up to 2 years

Safety Issue:


Principal Investigator

Timothy Hobday

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic


United States: Food and Drug Administration

Study ID:




Start Date:

June 2005

Completion Date:

Related Keywords:

  • Gastrinoma
  • Glucagonoma
  • Insulinoma
  • Metastatic Gastrointestinal Carcinoid Tumor
  • Neuroendocrine Tumor
  • Pancreatic Polypeptide Tumor
  • Recurrent Gastrointestinal Carcinoid Tumor
  • Recurrent Islet Cell Carcinoma
  • Somatostatinoma
  • WDHA Syndrome
  • Carcinoid Tumor
  • Carcinoma
  • Gastrinoma
  • Zollinger-Ellison Syndrome
  • Glucagonoma
  • Insulinoma
  • Somatostatinoma
  • Neuroendocrine Tumors
  • Malignant Carcinoid Syndrome
  • Gastrointestinal Neoplasms
  • Carcinoma, Islet Cell



Mayo Clinic Rochester, Minnesota  55905