Phase I/II Study of RAD001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Chronic Myeloid Leukemia in Blastic-Phase, Agnogenic Myeloid Metaplasia, Chronic Lymphocytic Leukemia, T-Cell Leukemia, or Mantle Cell Lymphoma
RAD001 is a new drug that was designed to block proteins that are important in the
development and growth of cancer.
If you are eligible to take part in this study, you will receive treatment with RAD001 by
mouth every day for as long as you stay on study. Four weeks (28 days) of treatment is
considered one course of therapy. The first 3 to 6 participants on study will receive the
lowest dose of RAD001. If that dose is safe, the next group of 3 to 6 patients will receive
double the dose as the first 3-6 participants. If that dose is safe, all further
participants will start treatment at that dose.
While on study, you will have weekly blood tests (about 2 teaspoons). Bone marrow aspirates
and/or biopsies, x-rays and/or scans will be performed every 4-12 weeks and as often as the
physician feels it is necessary. A physical exam will be done at Weeks 5, 7, 9, 11 and
anytime the physician sees fit. Vital signs will be taken every week.
You may be removed from this study if you don't respond after 4 courses of therapy,
intolerable side effects occur, or if the disease worsens. Your dose may be temporarily
held or decreased if certain side effects occur. If you are benefitting from the therapy,
you may continue on it indefinitely as long as continue to benefit.
Once you come off study, a physical exam, measurement of vital signs, a blood test (about 2
teaspoons), a bone marrow aspirate and/or biopsy, x-rays, and/or scans will be done.
This is an investigational study. The FDA has authorized RAD001 for use in research only.
A total of 70 -125 patients will take part in this study. All will be enrolled at M. D.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum Tolerated Dose (MTD)
If either 0 of 3 or 1 of 6 patients assigned to receive dose of 10 mg experiences grade 3 or 4 drug related toxicities, then this dose is the MTD. If greater than 2 patients experience grade 3 or 4 toxicities, either in the first 3 patients or in the first 6 patients, then the MTD is 5 mg.
Francis J. Giles, MD
UT MD Anderson Cancer Center
United States: Food and Drug Administration
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