Phase I Study of the RAF Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib in Children with Refractory Solid Tumors or Refractory Leukemias

Description

What is the purpose of this study?
The goals of this study are:

• To find the highest safe dose of Sorafenib that can be given to children with solid tumors without causing severe side effects
• To find out if the highest safe dose of Sorafenib in children with solid tumors is also safe for children with leukemia
• To learn what kind of side effects Sorafenib can cause
• To learn more about the pharmacology (how the body handles the drug) of Sorafenib
• To learn about the effects of Sorafenib on cells and proteins in the blood
• To learn if the tumor (leukemia) shows changes in the expression of genes called RAS, RAF, or flt-3, which are targets for Sorafenib
• To determine if genetic differences in individuals affect how they respond to Sorafenib (pharmacogenetics)
• To determine whether Sorafenib is a beneficial treatment for the tumor
• To assess the effect of Sorafenib on blood flow in the tumor by using MRI

Who will be included in this study?
Patients who are = 24 months and = 21 years old at study entry who have a recurrent or progressive tumor or leukemia that can not be cured by any know standard of treatment may be eligible to participate in this clinical trial.

What is involved?
Patients may be in the study for up to 24 cycles of therapy as long as they are responding to therapy and have no serious side effects from Sorafenib. Each cycle of therapy is 28 days long. After patients are finished taking Sorafenib, the study doctor will ask patients to visit the office for follow-up exams.

Standard Medical Tests
Before the study begins:
Patients will need to have the following exams, tests, or procedures to find out if they can be in the study. These exams, tests, or procedures are part of regular cancer care and may be done even if patients do not join the study. If some tests have been performed recently, they may not need to be repeated. This will be up to the study doctor.

• A medical history
• Physical exam
• Vital signs (blood pressure, pulse, temperature)
• Blood tests
• Urine tests
• Pregnancy test (for woman of child bearing age)
• If the patient is less than 18 years old, plain X-ray of lower legs
• X-rays, CT scans, or other tests are needed to check the tumor

During the study
Patients will need the following tests and procedures during the study. They are part of regular cancer care, although they may be done more often because patients are in this study.

• Physical exam: Once a week while on study
• Blood pressure: Once a week during the first treatment cycle and every other week after that while on study
• Blood tests: Twice a week during the first treatment cycle and weekly during subsequent treatment cycles while on study
• Urine tests: Prior to every treatment cycle
• Pregnancy test: Prior to the first treatment cycle and prior to every radiology scan
• CT or MRI (magnetic resonance imaging) of the tumor to analyze the response of the tumor to Sorafenib prior to each odd numbered treatment cycle starting with cycle 3
• Leukemia patients: Bone marrow aspirate prior to every treatment cycle to analyze the response of the tumor cells to Sorafenib
• Optional research studies

What are the benefits?
There may be no direct benefit for participants who agree to take part in this research study. The potential benefit of the treatment with Sorafenib is that it may cause the cancer to stop growing or to shrink for a period of time. It may lessen the symptoms, such as pain, that are caused by the cancer. Because there is not much information about the Sorafenib effect on cancers in humans, we do not know if patients will benefit from taking part in this study. Information learned from this study may help future patients with cancer.

Will I get all the facts about the study?
Parents interested in having their child participate, as well as adult patients/subjects, will be scheduled with a physician who will thoroughly explain all of the details of the study. The physician will review the consent form with the parent/guardian/adult subject and will be sure that all questions are answered. The consent form goes over all of the procedures, the risks, the benefits, the compensation, who to contact with questions or concerns and more. Study procedures will not begin until a parent/guardian or adult subject has signed this form and, if of age, the child has given at minimum his/her verbal agreement.

What are the risks?
A detailed list of side effects will be provided to those patients interested in knowing more about the study.

Details