Acute Myeloid Leukemia
Conditions
Keywords
Acute Myeloid Leukemia, AML, Bexarotene
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.
Interventions
DRUGBexarotene
Bexarotene given orally at a dose of 300mg/m2 until disease progression or unacceptable toxicities experienced by patient
Sponsors
Abramson Cancer Center at Penn Medicine
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy. | Two months after 17th patient has started treatment with Bexarotene, for up to 1 year | 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
| Measure | Time frame | Description |
|---|---|---|
| Bone Marrow Response Rate of Bexarotene in Subjects With AML Unable/Unwilling to Receive Systemic Chemotherapy | Two months after 17th patient has started treatment with Bexarotene, up to 1 year. | A clinically significant result will be recorded if the patient's bone marrow blasts percentage decreased by 50% or more over pretreatment blast percentage. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Bexarotene 300mg/m2 Bexarotene: Bexarotene given orally at a dose of 300mg/m2 until disease progression or unacceptable toxicities experienced by patient | 14 |
| Total | 14 |
Baseline characteristics
| Characteristic | Bexarotene 300mg/m2 |
|---|---|
| Age, Continuous | 74 years |
| Region of Enrollment United States | 14 participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 2 / 14 |
| serious Total, serious adverse events | 0 / 14 |
Outcome results
Primary
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
Time frame: Two months after 17th patient has started treatment with Bexarotene, for up to 1 year
Population: This study was completed several years ago, and the principal investigator has left the institution. Limited records are available, and despite our best efforts data are only available for 14 of the 24 enrolled participants.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Bexarotene 300mg/m2 | Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy. | 1 Participants |
Secondary
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.
Time frame: Two months after 17th patient has started treatment with Bexarotene, up to 1 year.
Population: This study was completed several years ago, and the principal investigator has left the institution. Limited records are available, and despite our best efforts data are only available for 14 of the 24 enrolled participants. Additionally only 5 patients had a bone marrow biopsy at 2 months.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Bexarotene 300mg/m2 | Bone Marrow Response Rate of Bexarotene in Subjects With AML Unable/Unwilling to Receive Systemic Chemotherapy | 1 Participants |