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Phase 0 Trial Evaluating the Effect of Temsirolimus on Known Pharmacodynamic Targets

Phase 0
18 Years
Open (Enrolling)
Advanced Cancer

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Trial Information

Phase 0 Trial Evaluating the Effect of Temsirolimus on Known Pharmacodynamic Targets

Study Drug:

Temsirolimus is designed to block the growth of cancer cells, which may cause the cancer
cells to die.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will receive temsirolimus by
vein over about 60 minutes on Day 1.

You will be assigned to a dose level of temsirolimus based on when you join this study. Up
to 5 dose levels of temsirolimus will be tested. Up to 3 participants will be enrolled at
each dose level. The first group of participants will receive the lowest dose level. Each
new group will receive a higher dose than the group before it, until the drug is found to
affect the enzyme that was tested for at screening.

Study Visits:

At each study visit, you will be asked about any drugs you may be taking and about any side
effects you may be having.

On Day 1:

-Blood (about 2 teaspoons each time) will be drawn before you receive the study drug and 5
times over the 24 hours after you receive the study drug for pharmacokinetic (PK) testing.
PK testing measures the amount of study drug in the body at different time points.

Blood (about 4 teaspoons each time) will be drawn for pharmacodynamic (PD) testing. PD
testing measures how the level of study drug in your body may affect the disease. This
blood will be drawn at 1 or more of the following times, but if the doctor thinks it is
needed, blood will be drawn at 2 or all 3 of the following times:

- At 4 hours (+/- 2 hours) after the dose

- At 24 hours (+/- 3 hours) after the dose

- At 72 hours (+/- 24 hours) after the dose

After the blood for PD testing has been tested and the tests show that the study drug may be
causing changes to the tumor cells in at least 2 out of 3 participants, future participants
will have tumor tissue collected before and after dosing for testing. Leftover tissue from
an earlier biopsy can be used instead of a fresh biopsy before dosing, if it is available.

Length of Study:

You will be on study for up to 4 days. You will be taken off study early if you have
intolerable side effects.

Because it takes 4 days for temsirolimus to be completely processed by the body, you will
not be able to begin receiving drugs in any other study until 4 days after the dose.

This is an investigational study. Temsirolimus is FDA approved and commercially available
for the treatment of advanced renal cancer. Its use in other types of cancer is

Up to 30 patients will take part in this study. All will be enrolled at MD Anderson.

Inclusion Criteria:

1. Patients with advanced or metastatic cancer, preferably with tumor easily accessible
for biopsy.

2. Patients should be at least four weeks or 5 half lives from the last day of
chemotherapy, antibody or other biological therapy, whichever is shorter.

3. Patients should preferably be undergoing screening for 2007-0668, 2008-0384,
2008-0425, and 2008-0827 (currently active Phase I trials involving temsirolimus).
However, patients may also be allowed on protocol if they are undergoing screening
for any study.

Exclusion Criteria:

1. Pregnant or lactating women.

2. Patients with a known hypersensitivity to any of the components or metabolites of the
drug products.

3. Patients with a known bleeding diathesis which would prevent safely obtaining a
biopsy if a biopsy is indicated.

4. Patients who are less than 18 years of age.

Type of Study:


Study Design:

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

Outcome Measure:

Participants' Pharmacodynamic (PD) Responses

Outcome Description:

PD response, significant S6 Kinase 1 (S6K1) inhibition, is defined at both the patient level and the dose level, 1) as compared with baseline, at least 50% reductions of S6K1 after treatment (biological criterion); 2) differences in log transformed S6K1 activity between post-treatment and baseline should be greater than threshold of 1.8 times standard deviation (SD) of baseline, which yields a 90% statistical confidence that it is not due to chance variation (statistical criterion).

Outcome Time Frame:

Blood drawn at 4 hours (+/- 2 hours) after study drug

Safety Issue:


Principal Investigator

Daniel Karp, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

August 2010

Completion Date:

Related Keywords:

  • Advanced Cancer
  • Cancer
  • mTOR inhibitor Temsirolimus
  • Torisel
  • CCI-779
  • pharmacodynamic targets
  • Neoplasms



UT MD Anderson Cancer Center Houston, Texas  77030