Sorafenib Tosylate in Treating Patients With Progressive Metastatic Neuroendocrine Tumors

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
National Cancer Institute (NCI) Identifier:
First received: August 16, 2005
Last updated: June 11, 2013
Last verified: April 2013

This phase II trial is studying how well sorafenib tosylate works in treating patients with progressive metastatic neuroendocrine tumors. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.

Condition Intervention Phase
Metastatic Gastrointestinal Carcinoid Tumor
Neuroendocrine Tumor
Pancreatic Polypeptide Tumor
Recurrent Gastrointestinal Carcinoid Tumor
Recurrent Islet Cell Carcinoma
WDHA Syndrome
Drug: sorafenib tosylate
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Trial of Bay 43-9006 in Progressive Metastatic Neuroendocrine Tumors

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Confirmed response rate (CR or PR) estimated by the number of successes divided by the total number of evaluable patients [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
    Kaplan-Meier methodology will be used to estimate the final success proportion (i.e., confirmed response rate with a 95% confidence interval).

Secondary Outcome Measures:
  • Toxicity defined as adverse events that are classified as either possibly, probably, or definitely related to study treatment, graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]
    The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns.

  • Biochemical levels and response defined as reduction in elevated hormone level by at least 50% or normalization of an elevated value [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
    Changes in hormone levels will be assessed via summary statistics, graphical techniques, and nonparametric methods, as well as correlated with radiographic response.

  • Survival time [ Time Frame: From registration to death due to any cause, assessed up to 2 years ] [ Designated as safety issue: No ]
    Estimated using the method of Kaplan-Meier.

  • Time to disease progression [ Time Frame: From randomization to documentation of disease progression, assessed up to 2 years ] [ Designated as safety issue: No ]
    Estimated using the method of Kaplan-Meier.

  • Duration of response [ Time Frame: The date from which the patients objective status is first noted to be either a CR or PR to the date progression is documented, assessed up to 2 years ] [ Designated as safety issue: No ]
    Descriptively summarized and graphically evaluated.

  • Time to treatment failure [ Time Frame: From the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, refusal, or death, assessed up to 2 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 90
Study Start Date: June 2005
Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment (sorafenib tosylate)
Patients receive oral sorafenib twice daily on days 1-28.
Drug: sorafenib tosylate
Given orally
Other Names:
  • BAY 43-9006
  • BAY 43-9006 Tosylate Salt
  • BAY 54-9085
  • Nexavar
  • SFN

Detailed Description:


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.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • 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 phenytoin)
  • 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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00131911

United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Principal Investigator: Timothy Hobday Mayo Clinic
  More Information

No publications provided

Responsible Party: National Cancer Institute (NCI) Identifier: NCT00131911     History of Changes
Other Study ID Numbers: NCI-2009-00121, MC044H, 7046, CDR0000437792, N01CM62205
Study First Received: August 16, 2005
Last Updated: June 11, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Carcinoid Tumor
Carcinoma, Islet Cell
Gastrointestinal Neoplasms
Malignant Carcinoid Syndrome
Neuroendocrine Tumors
Adenoma, Islet Cell
Carcinoma, Neuroendocrine
Digestive System Diseases
Digestive System Neoplasms
Endocrine Gland Neoplasms
Endocrine System Diseases
Gastrointestinal Diseases
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neuroectodermal Tumors
Pancreatic Diseases
Pancreatic Neoplasms
Sorafenib processed this record on October 23, 2014