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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

Phase 1/Phase 2
Not Enrolling
Leukemia, Mantle Cell Lymphoma, Myelofibrosis

Thank you

Trial Information

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.

Inclusion Criteria:

1. Patients with advanced, relapsed or refractory: acute leukemias (AML, ALL), MDS, CMML
in transformation with greater than or equal to 10% peripheral blood/bone marrow
blasts, CML in blastic phase, agnogenic myeloid metaplasia (AMM), CLL, T-cell
leukemia, or mantle cell lymphoma.

2. Serum bilirubin <= 2 mg/dL, SGOT or SGPT < 3 upper limit of normal, serum creatinine
<= 2 mg/dL, unless considered due to organ leukemic involvement or Gilbert's syndrome

3. The effects of RAD001 on the developing human fetus are unknown. For this reason and
because inhibitors of mRNA translation are known to be teratogenic, women of
child-bearing potential and men must agree to use adequate contraception (hormonal or
barrier method of birth control; abstinence) for the duration of study participation.
Should a woman become pregnant or suspect she is pregnant while participating in this
study, she should inform her treating physician immediately.

4. Ability to understand and the willingness to sign a written informed consent

Exclusion Criteria:

1. Patients who have had cytotoxic chemotherapy (other than hydroxyurea or
corticosteroids) or radiotherapy within 7 days prior to entering the study.

2. Patients may not be receiving any other cytotoxic investigational agents.

3. History of allergic reactions attributed to compounds of similar chemical or biologic
composition to RAD001. RAD001 must not be administered to patients with known
hypersensitivity to everolimus, sirolimus or to any of its excipients. Excipients
include butylated hydroxytoluene, magnesium stearate, hydroxypropylmethyl-cellulose,
crospovidone, and lactose.

4. Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that would limit compliance with
study requirements.

5. Pregnant women are excluded from this study because RAD001 has a potential for
teratogenic or abortifacient effects. Because there is an unknown but potential risk
for adverse events in nursing infants secondary to treatment of the mother with
RAD001, breastfeeding should be discontinued if the mother is treated with RAD001.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Maximum Tolerated Dose (MTD)

Outcome Description:

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.

Outcome Time Frame:

28 days

Safety Issue:


Principal Investigator

Francis J. Giles, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

April 2004

Completion Date:

September 2006

Related Keywords:

  • Leukemia
  • Mantle Cell Lymphoma
  • Myelofibrosis
  • Leukemia
  • Mantle cell lymphoma
  • Myelofibrosis
  • Relapsed or Refractory Acute Myeloid Leukemia
  • Acute Lymphocytic Leukemia
  • Chronic Myeloid Leukemia in Blastic-Phase
  • Agnogenic Myeloid Metaplasia
  • Chronic Lymphocytic Leukemia
  • T-Cell Leukemia
  • RAD001
  • Everolimus
  • Afinitor
  • Primary Myelofibrosis
  • Leukemia
  • Leukemia, Lymphocytic, Chronic, B-Cell
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Leukemia, T-Cell
  • Lymphoma
  • Metaplasia
  • Lymphoma, Mantle-Cell



M.D. Anderson Cancer Center Houston, Texas  77030