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Phase I Study of Gimatecan in Patients With Myelodysplastic Syndromes

Phase 1
18 Years
Open (Enrolling)
Myelodysplastic Syndromes, MDS

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Trial Information

Phase I Study of Gimatecan in Patients With Myelodysplastic Syndromes

The Study Drug:

Gimatecan is designed to block the function of an important protein (topoisomerase I) in
cancer cells that helps repair damage to DNA (the genetic material of cells). When this
protein is blocked, cancer cells may die.

Screening Tests:

Before you can start receiving the study drug, you will have what are called "screening
tests." These tests will help the doctor decide if you are eligible to take part in this

- You will have a physical exam.

- Your complete medical history will be recorded.

- Your vital signs (blood pressure, pulse, breathing rate, and temperature), height, and
weight will be measured.

- You will be asked how well you are able to perform the normal activities of daily
living (a performance status evaluation).

- You will be asked about any medications you may be taking.

- Blood (about 1 tablespoon) will be drawn for routine tests.

- Women who are able to have children must have a negative blood (about 2 teaspoons) or
urine pregnancy test.

- You will have a bone marrow aspirate performed. To collect a bone marrow aspirate, an
area of the hip is numbed with anesthetic, and a small amount of bone marrow is
withdrawn through a large needle.

- If your doctor thinks it is necessary, you will have a bone marrow biopsy when you have
the bone marrow aspirate done. To collect a bone marrow biopsy, an area of the hip is
numbed with anesthetic, and a small amount of bone marrow and bone is withdrawn through
a large needle.

- You will complete a questionnaire to see how the disease may be affecting your ability
to do your daily activities. The questionnaire will take about 15 minutes to complete.

Study Drug Dose Level:

If you are found to be eligible to take part in this study, the amount of study drug you
receive will be based on when you join the study. Three (3) to 6 participants will be
enrolled in this study and given a certain dose. If they do not experience intolerable side
effects, the next 3-6 participants enrolled will receive a higher dose. This process will
continue until researchers find the highest dose of the drug that can be given without
causing serious or intolerable side effects. The study doctor will tell you what dose you
will be receiving and how this compares to the doses other participants have received.

Study Drug Administration:

You will take gimatecan on Days 1-5. On Days 6-28, you will take no study drug. Each 28-day
period is called a study "cycle." If your doctor thinks it is necessary, your study cycles
may be shortened so that you will receive the study drug more often.

You will take the medication on an empty stomach, swallowing the capsules whole. You should
not eat for 1 hour after taking the medication. If you take other medications while you are
on this study, they must be taken 4 hours before or after you take gimatecan.

Study Visits:

Blood (about 2 tablespoons each time) will be drawn for routine tests every 1-4 weeks for
the length of this study. Most of these blood tests will be done once a week, but some will
only be done once every 4 weeks.

You will have a physical exam every 4 weeks for the first 3 months, then every 3 months.

At the end of Cycle 1, and then every 9-12 weeks after that, you will have a bone marrow
aspirate and/or biopsy. Bone marrow biopsies and aspirates may be collected more often if
your study doctor thinks it is necessary.

Once per week for the first 3 cycles, you will complete a symptom questionnaire. This will
be done either at a study visit, or you will be called by a member of study staff. The
questionnaire will take about 15 minutes to complete.

Length of Study:

You can remain on this study for up to 12 months. If, after this time, it is considered
that gimatecan is helping you, the study doctor will discuss the possibility of continuing
the study drug therapy outside of this study. You may be taken off this study early if the
disease gets worse or intolerable side effects occur.

End-of-Study Visit:

Once you are off this study, you will have an end-of-study visit, and the following tests
will be performed.

- You will have a physical exam.

- You will be asked about any side effects you may be experiencing.

- Blood (about 4 teaspoons) will be drawn for routine tests.

- You will repeat the symptom questionnaire.

This is an investigational study. Gimatecan is not FDA approved or commercially available.
It has been authorized for use in research only. Up to 30 patients will take part in this
study. All will be enrolled at M. D. Anderson.

Inclusion Criteria:

1. Patients with MDS with >/= 5% blasts or IPSS risk group intermediate (1 or 2) or high
(i.e., IPSS score 0.5 or higher).

2. Patients must have failed prior therapy with either chemotherapy (e.g., ara-C-based
chemotherapy, etc) or biologic agents (e.g., hypomethylating agents, arsenic,
thalidomide, CC5013, farnesyl transferase inhibitors, ATG, cyclosporine, etc).

3. Age >/= 18 years. Because no dosing or adverse event data are currently available on
the use of Gimatecan in patients <18 years of age, children are excluded from this
study but will be eligible for future pediatric single-agent trials, if applicable.

4. ECOG performance status 0-2.

5. Patients must have normal organ function as defined below: 1) Total bilirubin: 1.5 x institutional upper limit of normal; 2) ALT(SGPT): upper limit of normal; 3) Creatinine:
6. The effects of Gimatecan on the developing human fetus at the recommended therapeutic
dose are unknown. For this reason women of child-bearing potential (ie, not
post-menopausal for at least 12 months and not surgically sterile) and men must agree
to use double-barrier contraception prior to study entry, for the duration of study
participation, and for 3 months following discontinuation of study treatment. Should
a woman become pregnant or suspect she is pregnant while participating in this study,
she should inform her treating physician immediately.

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

Exclusion Criteria:

1. Patients who have received only supportive care (transfusions and/or hematopoietic
growth factors) for MDS.

2. Patients who have had chemotherapy or radiotherapy within 4 weeks or 5 half-lives of
the agent in question (6 weeks for nitrosoureas or mitomycin C), whichever is
greater, prior to entering the study or those who have not recovered to at least
grade 1 from adverse events due to agents administered more than 4 weeks earlier. The
use of hydroxyurea is allowed up to 48 hours prior to the start of therapy with

3. Uncontrolled intercurrent illness including, but not limited to, active uncontrolled
infection, symptomatic congestive heart failure, unstable angina pectoris,
symptomatic cardiac arrhythmia requiring and not responding to medical intervention,
or psychiatric illness/social situations that would limit compliance with study

4. Women who are pregnant or breast-feeding.

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 Time Frame:

Evaulate with each 28 Day Cycle

Safety Issue:


Principal Investigator

Jorge E. Cortes, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

August 2007

Completion Date:

Related Keywords:

  • Myelodysplastic Syndromes
  • MDS
  • Myelodysplastic Syndromes
  • MDS
  • Gimatecan
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia



The University of Texas M.D. Anderson Cancer Center Houston, Texas