Research Study Summary
Phase I Study of the RAF Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib in Children with Refractory Solid Tumors or Refractory Leukemias
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
- To determine if genetic differences in individuals affect how
they respond to Sorafenib (pharmacogenetics)
- To determine whether Sorafenib is a beneficial treatment for
- To assess the effect of Sorafenib on blood flow in the tumor by
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
- X-rays, CT scans, or other tests are needed to check the
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
- 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
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
What are the risks?
A detailed list of side effects will be provided to those patients
interested in knowing more about the study.
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