Phase II Study of RAD001 Plus Octreotide Depot in Patients With Metastatic or Unresectable Low Grade Neuroendocrine Carcinoma (Carcinoid, Islet Cell)
RAD001 is a new drug that is designed to block a protein that is important in the growth of
cancer cells. Octreotide Depot is FDA approved for the treatment of carcinoid syndrome and
hormonal symptoms from certain islet cell carcinomas. Octreotide Depot may also help to
block certain proteins that are important in tumor growth.
Before you can start treatment on the study, you will have what are called "screening
tests". These tests will help the doctor decide if you are eligible to take part in the
study. You will be asked questions about your medical history and about any medications you
are currently taking or have taken in the past. You will have a complete physical exam and
your heart rate, temperature, breathing rate, blood pressure, height, and weight will be
measured. You will be asked about your ability to perform every day activities. Blood
(about 2 teaspoons) will be collected for routine tests. You will have an electrocardiogram
(ECG - a test that measures the electrical activity of the heart) and scans (either Computed
Tomography/CT or Magnetic Resonance Imaging/MRI) to evaluate the cancer. Women who are able
to have children must have a negative blood pregnancy test.
If the screening evaluations show you are eligible to take part in the study, you may begin
treatment. You will take RAD001 by mouth once a day, every day while on study. You should
take it in a fasting state or after no more than a light, fat-free meal. You should take
RAD001 about the same time each day. Octreotide Depot will be given as an injection into
the muscle of either buttock once every 4 weeks while on study. This will be done at M. D.
Anderson. Four weeks (28 days) is called one course of treatment.
Clinic visits will occur every 2 weeks during the first 4 weeks and every 4 weeks from then
on. At each clinic visit, you will be asked questions about your medical history and about
any medications you are currently taking or have taken in the past. You will have a
complete physical exam and your heart rate, temperature, breathing rate, blood pressure,
height, and weight will be measured. You will be asked about your ability to perform every
day activities. Blood samples (about 1 teaspoons) for routine tests will be collected every
2 weeks for the first 8 weeks. After that, blood samples (about 2 teaspoons) will be
collected every 4 weeks. CT or MRI scan(s) will be performed every 12 weeks.
If a sample of your tumor tissue that was removed previously is available, it will be
analyzed for expression of proteins that may effect tumor growth. However, if a sample is
not available, you will not be asked to undergo a biopsy to collect this tissue. This sample
may analyzed at any time during the study.
If you experience severe side effects, treatment may be delayed, stopped, or you may receive
smaller doses of RAD001 and/or Octreotide Depot. You may continue to receive up to at least
12 courses of study treatment unless the disease gets worse, you decide not to take part any
longer, or your doctor decides it is in your best interest to stop treatment. It may be
possible to continue treatment beyond 12 courses if you are benefitting from this treatment.
When you stop study treatment, you will be asked to have some tests and evaluations done.
About 4 teaspoons of blood will be taken for routine lab tests, You will also have a
physical exam and CT scan or MRI scan will be done to check the size and location of your
This is an investigational study. RAD001 is investigational and is not commercially
available. The drug combination in this study is also investigational. RAD001 is
manufactured by Novartis Pharmaceuticals Corporation. About 60 patients will take part in
this study. All will be enrolled at M. D. Anderson.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Progression Free Survival (PFS)
PFS is measured from date of trial entry until documented progression of disease or death from any cause. PFS is measured by computed tomography (CT) scans or magnetic resonance imaging (MRI) performed at baseline and after every three cycles.
PFS assessed every 12 weeks (at the end of every 3 cycles) or more frequently if clinically indicated
James Yao, M.D.
M.D. Anderson Cancer Center
United States: Food and Drug Administration
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