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A Phase I Study of Bexarotene in Patients With Acute Myeloid Leukemia

Phase 1
18 Years
Not Enrolling
AML, Acute Myeloid Leukemia

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Trial Information

A Phase I Study of Bexarotene in Patients With Acute Myeloid Leukemia

Despite recent advances in cancer treatment, the prognosis is still poor for patients with
relapsed or chemotherapy resistant AML. Further aggressive chemotherapy can be attempted,
but generally yields poor results. This clinical study is the first use of bexarotene in
the treatment of patient with relapsed or chemotherapy resistant AML. The main purpose of
the study is to identify the maximum safe dose of bexarotene in patient with AML. Another
objective of the study is to explore the side effect profile of bexarotene in AML patients.
The study is organized so that the initial patients will get a low dose of bexarotene to be
taken daily. If these patients tolerate the drug, then later patients will get higher daily
doses. Further groups of patients will continue to increase their dose of bexarotene until
a maximum tolerated dose is identified. The stu dy will end at that point. Patients will
take the drug daily by mouth until such a time that their AML is worsening or they are
experiencing unacceptable side effects. Their participating will end at that point.

Inclusion Criteria:

- Age >18 years.

- Must have a histologically confirmed diagnosis of non-M3 AML as proven by bone
marrow biopsy. Patients with CML in myeloid blast crisis are eligible.

- Willing and able to give informed consent.

- Must have received prior induction therapy with conventional chemotherapy and/or
Mylotarg or otherwise not be eligible for conventional chemotherapy

- ECOG performance status of 0-2

- Must have recovered from the toxicities of prior chemotherapy.

- Women of childbearing potential must use effective contraception after enrollment in
this study and have a negative pregnancy test within 1 week of study enrollment.
They must continue to use effective contraception for 3 months after stopping

- Men must agree to use effective methods of contraception while taking bexarotene and
for 3 months after stopping therapy.

Exclusion Criteria:

- History of pancreatitis.

- Active alcohol abuse

- Taken bexarotene in the past.

- WBC >10,000/uL at the time of enrollment. Patients may be taking hydrea for WBC
control at the time of enrollment.

- Cytotoxic chemotherapy or Mylotarg within the past 7 days other than hydrea.

- Significant organ dysfunction: total bilirubin>3x ULN, AST or ALT>3x ULN,
creatinine>4mg/dL, on blood pressure supporting medications or mechanical

- Serious medical or psychiatric conditions that may compromise the safety of the
patient while participating in this study.

- Women of childbearing potential who are pregnant or actively breast feeding.

- Active participant in any other investigational treatment study for their AML.

- Unable/unwilling to perform required follow-up.

- Life expectancy of less than 1 month.

- Use of blood growth factors (G-CSF, GM-CSF, Aranesp, erythropoietin, or Neumega)
within 1 week prior to treatment initiation.

- Uncontrolled hyperlipidemia (triglycerides>1000 while on treatment with triglyceride
lowering medications).

- History of myeloablative allogeneic stem cell transplant.

- Known history of HIV.

- Uncontrolled active infection

- Known active CNS involvement with AML

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To identify the maximum tolerated daily dose of bexarotene in patients with Acute Myeloid Leukemia

Principal Investigator

Donald E Tsai, M.D., Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Pennsylvania


United States: Institutional Review Board

Study ID:

UPCC 12403



Start Date:

January 2004

Completion Date:

Related Keywords:

  • AML
  • Acute Myeloid Leukemia
  • AML
  • Acute Myeloid Leukemia
  • Bexarotene
  • Leukemia
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid



Abramson Cancer Center Of University of Pennsylvania Philadelphia, Pennsylvania  19104