A Phase I Study of Doxercalciferol in Recurrent Pediatric Solid Tumors
2 Years
26 Years
Both
Solid Tumors
Trial Information
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.
Study Visits:
Every week during Cycle 1, you will have a physical exam, and urine will be collected for
routine tests.
Two (2) times every week while you are on study, blood (about 2 teaspoons) will be drawn for
routine tests.
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
stop safely.
End-of-Study Visit:
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.
Long-Term Follow-Up:
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.
Inclusion Criteria:
1. Written and voluntary consent obtained from the patient or their legal guardians.
2. Patient must be greater than 2 and less than 26 years old at time of study entry or
at time of initial diagnosis
3. Patient must have had histologic verification of a solid malignancy at initial
diagnosis (excluding brain stem tumors and visual pathway gliomas)
4. Patient must have recurrent measurable or evaluable disease after therapy or
refractory to conventional therapy with presence of disease confirmed by standard
imaging or biopsy
5. Must start protocol therapy within two weeks of disease evaluation and determination
of eligibility.
6. Performance status of >20% on Lansky play scale for subjects <10 years of age, >20%
on Karnofsky score for subjects >= 10 years of age
7. Patient must have fully recovered to less than or equal to grade 1 from the acute
toxic effects of prior therapy, to meet eligibility criteria
8. Adequate bone marrow function defined as peripheral absolute neutrophil count (ANC)
equal or more than 500/mm^3, Hemoglobin equal or more than 8g/dl, Platelets equal or
more than 20,000/mm^3
9. Adequate renal function defined as serum creatinine <1.5 X upper limit of normal
(ULN) or age adjusted creatinine clearance of >70ml/min/1.73m^2
10. Adequate liver function defined as total bilirubin <1.5 X upper limit of normal (ULN)
and AST <2 X upper limit of normal (ULN)
11. Serum calcium, phosphorus, or PTH levels must be adjusted values per institutional guidelines.
12. Urine calcium level must be guidelines
13. Women of childbearing potential must have a negative serum or urine pregnancy test
and not be breastfeeding prior to study therapy. Women of childbearing potential must
be using an adequate form of contraception to avoid pregnancy during therapy and for
at least 4 weeks after the end of study therapy in such a manner that the risk of
pregnancy is minimized (Women of childbearing potential defined as not
post-menopausal for 12 months or no previous surgical sterilization)
Exclusion Criteria:
1. Any past history of urine calcium stones, idiopathic hypercalcemia, hereditary bone
disease, or hypercalciuria in the subject
2. Concurrent Medications: Patients currently taking digitalis or thiazides are
ineligible
3. Concurrent Medications: Oral calcium and vitamin D supplements and all homeopathic
medications must be discontinued 2 weeks prior to study entry and while on study
therapy.
4. Use of other anti-tumor therapy, including but not limited to chemotherapy, radiation
therapy, immunotherapy, other investigational agents or other biologic therapy,
during study therapy, with the exception of palliative radiation therapy to non-index
lesions.
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Maximum Tolerated Dose (MTD)
Outcome Time Frame:
Continual reassessment method during 28 day cycles, weekly visits for first cycle and monthly for each additional cycle
Safety Issue:
Yes
Principal Investigator
Peter E. Zage, MD, PhD
Investigator Role:
Principal Investigator
Investigator Affiliation:
M.D. Anderson Cancer Center
Authority:
United States: Institutional Review Board
Study ID:
2006-0423
NCT ID:
NCT00511017
Start Date:
August 2007
Completion Date:
September 2010
Related Keywords:
- Solid Tumors
- Solid Tumors
- Doxercalciferol
- Pediatric Cancer
- Recurrent Pediatric Solid Tumors
- Calcium
- Vitamin D
- Neoplasms
Name | Location |
|---|---|
| UT MD Anderson Cancer Center | Houston, Texas 77030 |
