Clinical trial NCT00425477
Conditions: Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases
Keywords: refractory anemia with excess blasts recurrent adult acute myeloid leukemia untreated adult acute myeloid leukemia secondary acute myeloid leukemia de novo myelodysplastic syndromes previously treated myelodysplastic syndromes secondary myelodysplastic syndromes myelodysplastic/myeloproliferative disease, unclassifiable refractory anemia with ringed sideroblasts refractory cytopenia with multilineage dysplasia chronic myelomonocytic leukemia adult acute myeloid leukemia with 11q23 (MLL) abnormalities adult acute minimally differentiated myeloid leukemia (M0) adult acute myeloblastic leukemia without maturation (M1) adult acute myeloblastic leukemia with maturation (M2) adult acute myelomonocytic leukemia (M4) adult acute monoblastic leukemia (M5a) adult acute monocytic leukemia (M5b) adult pure erythroid leukemia (M6b) adult erythroleukemia (M6a) adult acute megakaryoblastic leukemia (M7) adult acute myeloid leukemia with t(16;16)(p13;q22) adult acute myeloid leukemia with t(8;21)(q22;q22) adult acute myeloid leukemia with inv(16)(p13;q22)
Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Start date: 2006-11-01; Completion date: 2016-09-30
Enrollment: 26 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
RATIONALE: Bexarotene may help cancer or abnormal cells become more like normal cells, and to grow and spread more slowly. Colony-stimulating factors, such as GM-CSF, may increase the number of immune cells found in bone marrow or peripheral blood. Giving bexarotene together with GM-CSF may be an effective treatment for myelodysplastic syndrome (MDS) or acute myeloid leukemia.

PURPOSE: This phase II trial is studying how well giving bexarotene together with GM-CSF works in treating patients with MDS or acute myeloid leukemia.

Detailed description:
OBJECTIVES:

Primary

* Assess the clinical response in patients with myelodysplastic syndromes or acute myeloid leukemia treated with bexarotene and sargramostim (GM-CSF).

Secondary

* Determine the clinical activity of this regimen, in terms of transfusion requirements, in these patients.
* Determine the biological activity of this regimen, in terms of biological markers and cytogenetic abnormalities, in these patients.
* Assess the toxicity profile of this regimen in these patients.

OUTLINE: Patients receive oral bexarotene and sargramostim (GM-CSF) subcutaneously on days 1-28. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.

Blood and bone marrow samples are collected at baseline and after 1 or 2 courses of study therapy. Samples are examined by flow cytometry for laboratory studies, including biological markers, and by fluorescent in situ hybridization (FISH) for cytogenetic changes.

After completion of study treatment, patients are followed periodically for 6 months.

PROJECTED ACCRUAL: A total of 18 patients will be accrued for this study.

Arms: arm 1: BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Interventions: intervention 1: sargramostim intervention 2: bexarotene intervention 3: cytogenetic analysis intervention 4: fluorescence in situ hybridization intervention 5: flow cytometry intervention 6: laboratory biomarker analysis intervention 7: biopsy
Intervention descriptions: intervention 1: None intervention 2: None intervention 3: None intervention 4: None intervention 5: None intervention 6: None intervention 7: None

Protocol documents: