A Phase II Study to Evaluate the Efficacy and Toxicity of Oxaliplatin in Combination With Gemcitabine as First and Second Line Therapy in Unknown Primary Cancer
Oxaliplatin is a chemotherapy drug that causes the death of cancer cells and other actively
dividing cells by interfering with Deoxyribonucleic acid (DNA) function. Gemcitabine
prevents cells from making DNA and Ribonucleic acid (RNA) that are necessary for cell
growth, thus disrupting the growth of the cancer cells, which causes the cancer cells to
start to die.
After the screening portion of the study, if you are eligible to begin study treatment, you
will come to M. D. Anderson at least every two weeks (14 days) for treatment. Each 14-day
period of treatment is called a "cycle" of therapy. You will receive at least 3 cycles of
therapy (6 weeks) unless side effects become intolerable or the disease progresses.
When you begin treatment, you will have a small tube (central venous line) inserted into a
large vein under the skin of the chest or through a vein in the arm for administration of
oxaliplatin and gemcitabine. The central venous line will remain in place the entire time
you are taking part in this study. Both drugs must be given at M. D. Anderson. On Day 1 of
each cycle, you will receive gemcitabine injected into a vein over 2 hours. On Day 2 of each
cycle you will receive oxaliplatin injected into a vein over 2 hours. This drug schedule
will be repeated every 2 weeks.
You will first be asked to fill out several questionnaires that deal with your quality of
life. These will help the researchers understand how the study drugs affect your quality of
life including your pain level, fatigue (tiredness) level, appetite, and ability to perform
everyday tasks, in spite of whether there is any shrinkage of the tumor. It will take about
30 minutes to an hour to complete the forms. You will also be asked to complete a
questionnaire (one time only) that asks questions about why you decided to take part in this
During the first cycle of therapy, blood (about 2 1/2 teaspoons) will be collected once a
week for routine tests. Before each new cycle of therapy, you will have a complete physical
exam, urine will be collected, and blood will be drawn (about 2 ½ teaspoons) for routine
tests. You will be asked to tell the study doctor about all medications you have taken since
you started taking the study drugs and any health problems that you may have experienced.
You will also have either computed tomography (CT) scans or an Magnetic resonance imaging
(MRI) of the tumor(s) every 6 weeks and at the end of the study.
All tests before each new cycle of treatment and when treatment stops must be done at M. D.
Anderson. Extra tests may be done during the study if your doctor feels it is necessary for
your care. Every 6 weeks for as long as you are on study, you will also be asked to fill out
the questionnaires that help study doctors understand your quality of life. It will take
about 30 minutes to an hour to complete the forms.
If you experience intolerable side effects, treatment may be delayed, stopped, or you may
receive smaller doses of the treatment. You may continue to receive treatment on this
study, unless the disease gets worse or you experience any intolerable side effects. If
this happens, you will be taken off the study and your doctor will discuss other treatment
options with you.
When you stop taking part in the study for any reason, you will have blood (about 3
teaspoons) collected for final routine tests. You will have one more physical exam and
either a CT scan or a MRI to check on the status of the disease. You will also be asked
once more to fill out the questionnaires that help assess your quality of life. It will
take about 30 minutes to an hour to complete the forms.
Once you stop receiving study treatment, you will be contacted by phone every three months
for the rest of your life to check on how you are doing and any symptoms you may be
This is an investigational study. Oxaliplatin is FDA approved for treatment of advanced
cancer of the colon or rectum, and gemcitabine is FDA approved for the treatment of advanced
cancer of the pancreas. However, the combination of the two drugs for treatment of cancers
with unknown primaries is still considered investigational. Up to 81 people 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
Complete Response (CR): Disappearance of all target lesions; Partial Response (PR): At least 30% decrease in sum of longest diameter (LD) of target lesions, reference baseline sum LD; Progressive Disease (PD): At least 20% increase in sum of LD of target lesions, reference smallest sum LD recorded since treatment started or appearance of 1 or more new lesions; Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor increase to qualify for PD, reference smallest sum LD since treatment started.
Response to treatment measured using Response Evaluation Criteria in Solid Tumors (RECIST) criteria with radiological evaluation at 6 weeks and reevaluated every 6 weeks.
Gauri R. Varadhachary, MD
M.D. Anderson Cancer Center
United States: Institutional Review Board
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