Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia (UPCC04407)
Primary Purpose
Acute Myeloid Leukemia
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Bexarotene
Sponsored by
About this trial
This is an interventional treatment trial for Acute Myeloid Leukemia focused on measuring Acute Myeloid Leukemia, AML, Bexarotene
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
Sites / Locations
- Abramson Cancer Center of University of Pennsylvania
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
A
Arm Description
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT00615784
First Posted
February 1, 2008
Last Updated
November 23, 2020
Sponsor
Abramson Cancer Center at Penn Medicine
1. Study Identification
Unique Protocol Identification Number
NCT00615784
Brief Title
Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
Acronym
UPCC04407
Official Title
A Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
Study Type
Interventional
2. Study Status
Record Verification Date
November 2020
Overall Recruitment Status
Terminated
Why Stopped
Study terminated. Eisai's Targretin acquired by another pharmaceutical company.
Study Start Date
May 25, 2010 (Actual)
Primary Completion Date
October 1, 2013 (Actual)
Study Completion Date
November 8, 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Abramson Cancer Center at Penn Medicine
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia
Keywords
Acute Myeloid Leukemia, AML, Bexarotene
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
24 (Actual)
8. Arms, Groups, and Interventions
Arm Title
A
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Bexarotene
Intervention Description
Bexarotene given orally at a dose of 300mg/m2 until disease progression or unacceptable toxicities experienced by patient
Primary Outcome Measure Information:
Title
Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy.
Description
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
Time Frame
Two months after 17th patient has started treatment with Bexarotene, for up to 1 year
Secondary Outcome Measure Information:
Title
Bone Marrow Response Rate of Bexarotene in Subjects With AML Unable/Unwilling to Receive Systemic Chemotherapy
Description
A clinically significant result will be recorded if the patient's bone marrow blasts percentage decreased by 50% or more over pretreatment blast percentage.
Time Frame
Two months after 17th patient has started treatment with Bexarotene, up to 1 year.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Donald E. Tsai, MD, PhD
Organizational Affiliation
University of Pennsylvania
Official's Role
Principal Investigator
Facility Information:
Facility Name
Abramson Cancer Center of University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
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