Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to evaluate the activity of bexarotene, a retinoic acid class drug, in patients with Acute Myeloid Leukemia (AML) that has returned after or is resistant to standard chemotherapy or are otherwise not eligible for conventional chemotherapy. Retinoic acids are a class of drugs related to Vitamin A, and have a wide range of effects within normal and malignant cells that affect cell growth and cell death.

Official Title
-----------------
A Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia

Conditions
-----------------
Acute Myeloid Leukemia

Intervention / Treatment
-----------------
* Drug: Bexarotene


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Age >= 18 years Confirmed diagnosis of AML as proven by bone marrow biopsy Must have received prior induction therapy with conventional chemotherapy and/or Mylotarg or otherwise not eligible for conventional chemotherapy ECOG performance status of 0-2 Recovered from toxicities of prior chemotherapy Exclusion Criteria: History of pancreatitis Active alcohol abuse Taken bexarotene in the past WBC > 10,000/uL at time of enrollment Cytotoxic therapy within the past 14 days other than hydrea, low dose cytarabine or low dose Mylotarg Significant organ disfunction: total bilirubin > 3x ULN, AST or ALT >3 x ULN, creatinine > 3 mg/dL, on blood pressure supporting medications or mechanical ventilation Active participant in any other investigational treatment study for AML 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 Known history of HIV Known active CNS involvement with AML Women of childbearing potential or active breast feeding

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br> | Drug: Bexarotene<br>* Bexarotene given orally at a dose of 300mg/m2 until disease progression or unacceptable toxicities experienced by patient<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy. | Hematologic response will be assessed according to modified criteria of an international working group defined by Cheson et al, Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood, 1 December 2000, Vol. 96, No. 12, pp. 3671-3674 | Two months after 17th patient has started treatment with Bexarotene, for up to 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Bone Marrow Response Rate of Bexarotene in Subjects With AML Unable/Unwilling to Receive Systemic Chemotherapy | A clinically significant result will be recorded if the patient's bone marrow blasts percentage decreased by 50% or more over pretreatment blast percentage. | Two months after 17th patient has started treatment with Bexarotene, up to 1 year. | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Acute Myeloid Leukemia, AML, Bexarotene