Clinical Study Protocol RTA 402-C-0501: A Phase I Dose-finding and Pharmacokinetic Study of RTA 402 (CDDO-Me) Administered Orally for 21 Days of a 28-day Cycle in Patients With Advanced Solid Tumors or Lymphoid Malignancies
RTA 402 is a new man-made drug that is designed to inhibit (turn off) genes that may
activate your cancer cells and cause them to grow. This drug has not been tested in humans.
If you are found to be eligible to take part in this study, you will take RTA 402 by mouth
(several capsules a day) in the morning with a glass of water (before eating) on the dose
level that will be assigned to you. The dose level you receive will depend on when you begin
on this study. You will receive the study drug for 21 days in a row. You will not take the
study drug for the next 7 days. This is considered a rest period. These 28 days equal 1
cycle. You should take your study drug as instructed. If a dose is missed or vomited, it
will not be replaced either on that day or at the end of the 21-day treatment period.
At the beginning of the study, one to three patients may be enrolled at each dose level, and
dose will be doubled between groups until the first occurrence of intolerable side effects
during Cycle 1. At the first occurrence of less severe side effects, groups will be
expanded to 3-4 patients. Dose escalation (increasing) will continue in this way until
researchers learn the highest dose that is best tolerated. You will remain at the same dose
level that you started on for at least 2 cycles. After at least 2 cycles, your dose may be
increased to another dose that has been determined to be safe. If your disease gets worse
or you experience any intolerable side effects, you will be taken off this study.
On Cycle 1 on Days 1 and 21, you will be asked to go to the Clinical and Translational
Research Center for your dose of the study drug. You will be asked about how you are
feeling, and about any medications you may be taking including any over the counter
medication, herbs, and alcohol. You will be asked to report any bad side effects you may
experience from the study drug. You will have blood draws (about 1 1/2 teaspoons each time)
for PK studies. You will have these PK blood draws before you take RTA 402 at 15 minutes, 30
minutes, and 1, 2, 4, 6, 8, and 24 hours after you take the study drug. You will have your
vital signs measured at 2, 4, and 8 hours after taking RTA 402. You will have your urine
collected over a 24-hour period. The study staff will provide the urine sample container and
explain how to collect your urine.
During Cycle 1, you will return to the clinic every week to have routine blood drawn (about
2 teaspoons) to check the status of your disease. You will be asked how you are feeling and
about any medications you may be taking including any over the counter medication, herbs,
Each week during Cycle 1 and on Day 28 of each additional cycle, you will complete 2
questionnaires that will take about 15 minutes to complete. You will also have your body
weight measured and be asked about your normal food intake. Your body composition will be
measured using measurements that include body weight and height, skin fold thickness, and
bioelectrical impedance using the Tanita body composition monitoring scale. This is a
non-invasive method to measure total body water, total body fat, and total body lean mass.
Researchers will measure how much energy your body uses while at rest. You will also have
timed test that will involve your participation where at your fastest speed you will be
asked to walk 25 feet and walk back.
After Cycle 1, you will return to the clinic every other week for up to 18 cycles (about 18
months) as long as your health does not get worse and you do not experience any intolerable
During every other cycle starting with Cycle 2, you will be evaluated to check the status of
your cancer. You will have routine imaging studies, such as x-rays, CT scans, or MRIs to
measure your tumor. You will have a bone marrow biopsy (if you have lymphoid disease) to
measure your disease. On Day 1 of the next cycle, you will return to your doctor's office
for your next physical exam and to begin the next cycle of the study drug.
While on this study, you will not be allowed to take any other investigational agent,
chemotherapy, biologic therapy, immunotherapy, or anti-cancer treatments. You will not be
allowed to take medicine that can help increase your white blood cell count.
Once you have completed all cycles of the study drug, you will have an end-of-study visit.
During this visit, you will have a physical exam, including measurement of your vital signs.
You will have blood drawn (about 1 1/2 teaspoons) for routine tests, and a urine sample will
be collected. You will have a bone marrow biopsy (if you have lymphoid disease) to measure
your disease. You will have imaging studies, such as x-rays, CT scans, or MRIs to measure
your tumor. You will be asked to report any side effects or new medications that you may be
taking including any over the counter medication, herbs, and alcohol.
You will be followed (30 days after you are off study) and asked to report any side effects
you may be experiencing, or new medications that you may be taking. You will also have a
physical exam, including measurement of your vital signs. If your doctor thinks that you
should continue taking RTA 402 (after 18 cycles or 18 months), you will be asked to
participate in an extended study.
This is an investigational study. RTA 402 has been authorized by the FDA for use in
research only. Up to 45 patients will take part in this multicenter study. Up to 42 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
Maximum Tolerated Dose of RTA 402
28 day cycles
David S. Hong, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
|UT MD Anderson Cancer Center||Houston, Texas 77030|