A Phase I Study of Doxercalciferol in Recurrent Pediatric Solid Tumors
The Study Drug:
Doxercalciferol is designed to "copy the actions" of Vitamin D without causing blood levels
of calcium to be too high.
Study Drug Dose Level:
If you are found to be eligible to take part in this study, you will begin receiving
doxercalciferol. The dose you will receive will be based on how many participants have been
enrolled before you, and on the safety data that is available. You will remain on the same
dose level throughout this study. There will be up to 9 participants enrolled in each group
of the same dose. The first group of participants enrolled on this study will be given small
doses of doxercalciferol. If no intolerable side effects are experienced, the next group of
participants will be enrolled at a higher dose level. This process will continue until
researchers find the highest tolerable dose of doxercalciferol, among the 5 dose levels
being tested, that can be given without intolerable side effects occurring.
Study Drug Administration:
If you are found to be eligible to take part in this study, you will begin the first "cycle"
of the study drug. Each cycle of chemotherapy will last 28 days.
Doxercalciferol will be taken by mouth every day for 28 days.
Every week during Cycle 1, you will have a physical exam, and urine will be collected for
Two (2) times every week while you are on study, blood (about 2 teaspoons) will be drawn for
During Cycles 2 and beyond, you will have the following tests and procedures performed:
- Urine will be collected for routine tests.
- ECG and ECHO will be repeated again only if your doctor feels it is necessary.
- You may also have a computed tomography (CT) scan and/or magnetic resonance imaging
(MRI) to check the status of the disease.
Length of Study:
You may remain on this study for up to 12 cycles.You will be taken off this study if the
disease gets worse or intolerable side effects occur. The study doctor may stop you from
taking part in this study at any time if he/she believes it is in your best interest or if
you do not follow the study rules.
You can stop participating in this study at any time. Tell the study doctor if you are
thinking about leaving the study so that any risks can be reviewed by your study doctor and
so that follow-up care and testing can be discussed. The study doctor will tell you how to
Once you are off-study, you will have an end-of-study visit. At this visit, the following
tests and procedures will be performed.
- You will have a physical exam.
- Blood (around 2 teaspoons) and urine will be collected for routine tests.
- You may also have a CT scan and/or MRI to check the status of the disease.
After the end-of-study visit, you will have a study visit every month for the first year and
then once a year for 10 years. At these visits, you will have a physical exam.
You may also have CT scans and/or MRIs every 8 weeks for the 1st 6 months, every 3 months
for the next year, then every 6 months for 1 1/2 years, and then at least 1 time a year
after that. This will be done to check the status of your disease.
Researchers would like to keep track of your medical condition and some general health
information about you through yearly follow-up visits, letters, and/or phone contact, for up
to 10 years. More information (such as other treatments and/or disease response) will be
collected if follow-up studies show changes in the disease or in your overall health.
If the disease has gotten worse, researchers will keep track of which parts of your body are
involved. Researchers will also collect information about any other cancers or major organ
problems you might have developed. Keeping in touch with you and checking on your condition
will help researchers look at the long-term effects of the study.
This is an investigational study. Doxercalciferol is FDA approved and commercially
available for reducing high levels of a hormone in patients undergoing chronic renal
dialysis. Its use in this study, for this disease, is investigational.
Up to 24 patients will take part in the 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
Maximum Tolerated Dose (MTD)
Continual reassessment method during 28 day cycles, weekly visits for first cycle and monthly for each additional cycle
Peter E. Zage, MD, PhD
M.D. Anderson Cancer Center
United States: Institutional Review Board
|UT MD Anderson Cancer Center||Houston, Texas 77030|