Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
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ClinicalTrials.gov Identifier: NCT00425477 |
Recruitment Status
: Unknown
Verified October 2015 by Sidney Kimmel Comprehensive Cancer Center.
Recruitment status was: Active, not recruiting
First Posted
: January 23, 2007
Last Update Posted
: November 2, 2015
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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.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases | Biological: sargramostim Drug: bexarotene Genetic: cytogenetic analysis Genetic: fluorescence in situ hybridization Other: flow cytometry Other: laboratory biomarker analysis Procedure: biopsy | Phase 2 |
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.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 26 participants |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML |
Study Start Date : | November 2006 |
Estimated Primary Completion Date : | July 2016 |

- Clinical response (complete and partial)
- Clinical activity as measured by improved peripheral blood counts and changes in transfusion requirements
- Biological activity as measured by in vivo induction of terminal differentiation of myeloid progenitors and in vivo changes in detectable chromosomal abnormalities

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Ages Eligible for Study: | 18 Years to 120 Years (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
-
Diagnosis (confirmed by bone marrow aspirate and/or biopsy) of 1 of the following:
-
Myelodysplastic syndromes of 1 of the following cell types:
- Refractory anemia (RA) with ringed sideroblasts
- Refractory cytopenia with multilineage dysplasia (RCMD)
- RCMD and ringed sideroblasts
- RA with excess blasts-1
- RA with excess blasts-2
- Myelodysplastic syndromes, unclassified
- Chronic myelomonocytic leukemia
-
Relapsed or refractory acute myeloid leukemia (AML), meeting 1 of the following criteria:
- Recurrent genetic abnormalities (11q23 [MLL] abnormalities)
- Multilineage dysplasia
- Therapy-related AML
-
Not otherwise categorized, including any of the following:
- M0 minimally differentiated
- M1 without maturation
- M2 with maturation
- M4 myelomonocytic leukemia
- M5 monoblastic/monocytic leukemia
- M6 erythroid leukemia
- M7 megakaryoblastic leukemia
-
- Newly diagnosed untreated AML allowed provided patient does not qualify for or refused potentially curative intensive chemotherapeutic regimens
- No RA with 5q-syndrome
- No peripheral leukemia with blast count > 30,000/mm³ (uncontrolled with hydroxyurea)
- Relatively stable bone marrow function for > 7 days (i.e., no WBC doubling to > 10,000/mm^3)
- No acute promyelocytic leukemia
- No clinical symptoms of active CNS disease (if CNS disease is suspected, patient must have lumbar puncture with negative cytology)
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Creatinine ≤ 2.0 mg/dL
- Bilirubin ≤ 1.6 mg/dL (unless secondary to hemolysis)
- AST and ALT ≤ 4 times upper limit of normal (unless disease related)
- Hemoglobin ≥ 8 g/dL (transfusions allowed)
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective barrier contraception
- No untreated positive blood cultures or progressive infection as assessed by radiographic studies
- No history of intolerance to sargramostim (GM-CSF)
PRIOR CONCURRENT THERAPY:
- Recovered from prior therapy
-
At least 2 weeks since prior treatment for myeloid disorder, including any of the following:
- Chemotherapy
- Hematopoietic growth factors
- Biologic therapy (e.g., monoclonal antibodies)
- Hydroxyurea for patients with WBC > 10,000/mm^3 allowed
- No concurrent vitamin A supplementation
- No concurrent gemfibrozil

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00425477
United States, Maryland | |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | |
Baltimore, Maryland, United States, 21231-2410 |
Study Chair: | B. Douglas Smith, MD | Sidney Kimmel Comprehensive Cancer Center |
Responsible Party: | Sidney Kimmel Comprehensive Cancer Center |
ClinicalTrials.gov Identifier: | NCT00425477 History of Changes |
Other Study ID Numbers: |
J0675 CDR0000525989 P30CA006973 ( U.S. NIH Grant/Contract ) JHOC-J0675 JHOC-NA_00003076 |
First Posted: | January 23, 2007 Key Record Dates |
Last Update Posted: | November 2, 2015 |
Last Verified: | October 2015 |
Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
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) |
Additional relevant MeSH terms:
Syndrome Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Myelodysplastic Syndromes Preleukemia Myeloproliferative Disorders Myelodysplastic-Myeloproliferative Diseases Disease Pathologic Processes |
Neoplasms by Histologic Type Neoplasms Bone Marrow Diseases Hematologic Diseases Precancerous Conditions Bexarotene Anticarcinogenic Agents Protective Agents Physiological Effects of Drugs Antineoplastic Agents |