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Randomized Phase II Study of Clofarabine Alone Versus Clofarabine in Combination With Low-Dose Cytarabine in Previously Untreated Patients >= 60 Years With AML and High-Risk MDS

Phase 2
60 Years
Not Enrolling
Acute Myeloid Leukemia, Myelodysplastic Syndrome

Thank you

Trial Information

Randomized Phase II Study of Clofarabine Alone Versus Clofarabine in Combination With Low-Dose Cytarabine in Previously Untreated Patients >= 60 Years With AML and High-Risk MDS

Clofarabine is a chemotherapy drug that is designed to interfere with the growth and
development of cancer cells. Ara-C is a chemotherapy drug which is approved for the
treatment of AML and MDS. Although there is experience with the combination of both drugs,
there have not been trials that explored the particular doses and schedule of clofarabine
plus ara-C that you may receive.

Before you can start treatment on the study, you will have what are called "screening
tests". These tests will help the doctor decide if you are eligible to take part in the
study. You will have a complete medical history and physical exam. You will also be asked
about what medications you are taking currently and about the level of your daily
activities. About 2 tablespoons of blood will be collected for routine blood tests and to
make sure you are not at increased risk for developing side effects.

Before your first treatment (usually within 14 days), you may have bone marrow samples
collected. To collect a bone marrow sample, an area of the hip or chest bone is numbed with
anesthetic and a small amount of bone marrow is withdrawn through a large needle. The
procedure will be explained to you by your doctor and will require you to sign a separate
consent document.

Early study results showed that there is clearly a better response with the combination
treatment compared to the clofarabine alone treatment. Because of this, all participants in
this study will now be assigned to the clofarabine plus ara-C group. You will receive
clofarabine through a vein daily for 5 days in a row. In addition, you will receive
injections of ara-C under the skin once a day for 14 days in a row. On those days when both
clofarabine and ara-C are taken, the clofarabine will be given approximately 4 hours before
the ara-C injections. You can be taught to give the ara-C injections to yourself. Each cycle
may be repeated every 3 to 6 weeks. You will be required to record the injections in a
medication diary.

Up to 2 of these cycles (for both groups) can be given at this dose schedule. If you show a
response to treatment, you can continue with up to 12 cycles of therapy, during which
clofarabine will be given for 3 days instead of 5 and ara-C for 7 days instead of 14.
Maintenance courses may be given on average every 4 to 7 weeks.

Before every treatment course, you will have a physical exam including measurement of your
weight and vital signs. You will also be asked how you are feeling and how you are able to
go about your daily routine. At least once a week (more often if your doctor feels it is
necessary), you will have blood samples (about 1-2 teaspoons) collected for routine lab
tests. Around 3 weeks after your first treatment, you may have samples of bone marrow
collected. After that, the bone marrow collections will be performed every 2 weeks (or more
often if your doctor feels it is necessary). The bone marrow sample will be tested to
evaluate the response of the disease to therapy.

You will need to stay in Houston for the first 4 weeks of treatment. After that, you have to
return to Houston to receive the clofarabine treatment, but you can have check-up visits and
blood tests with your local doctor. If the disease gets worse or you experience any
intolerable side effects, you will be taken off the study and your doctor will discuss other
treatment options with you.

After you finished your treatment, and as long you are participating on this study you will
be scheduled every 3-6 months to check on the status of the disease and your overall health
as long as you stay on the study. Once you are taken off the study, your doctor will decide
how often you will have follow-up as part of your standard care.

This is an investigational study. Clofarabine is authorized by the Food and Drug
Administration (FDA) for use in research only. Up to 108 participants will take part in this
study. All will be enrolled at M. D. Anderson.

Inclusion Criteria:

- Previously untreated AML and high-risk MDS ( > 10% blasts, or International
Prognostic Scoring System (IPSS) intermediate-2). Prior therapy with hydroxyurea,
single agent chemotherapy (e.g. decitabine), hematopoietic growth factors, biological
or "targeted" therapies are allowed.

- Age > 60 years.

- Eastern Cooperative Oncology Group (ECOG) performance status
- Sign a written informed consent form.

- Adequate liver function (total bilirubin < 2mg/dL,serum glutamic pyruvic transaminase
(SGPT) or Serum glutamic oxaloacetic transaminase (SGOT) < x 4 upper limit of normal
(ULN)) and renal function (serum creatinine < 2mg/dL).

Exclusion Criteria:

- Patients with >= New York Heart Association (NYHA) grade 3 heart disease as assessed
by history and/or physical examination.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Diagnostic

Outcome Measure:

Number of Participants With Response

Outcome Description:

Participant responses are categorized as 'Complete Remission,' Complete Remission, No Platelet Recovery,' 'No Response.' Complete Remission: Disappearance of all clinical and/or radiologic evidence of disease. Neutrophil count > 1.0 x 109/L and platelet count > 100 x 109/L, and normal bone marrow differential (< 5% blasts); Complete Remission, No Platelet Recovery: Peripheral blood and bone marrow results as for CR, but with platelet counts of < 100 x 109/L. Blood draws once a week until remission then every 2 to 8 weeks during therapy.

Outcome Time Frame:

Every 2 to 8 weeks

Safety Issue:


Principal Investigator

Stefan Faderl, MD

Investigator Role:

Study Chair

Investigator Affiliation:

The University of Texas MD Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

July 2004

Completion Date:

February 2008

Related Keywords:

  • Acute Myeloid Leukemia
  • Myelodysplastic Syndrome
  • Acute Myeloid Leukemia
  • AML
  • High-Risk Myelodysplastic Syndrome
  • MDS
  • Clofarabine
  • Cytarabine
  • ara-C
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia



M.D. Anderson Cancer Center Houston, Texas  77030