Phase 2
N=26
Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
Leukemia · Myelodysplastic Syndromes · Myelodysplastic/Myeloproliferative Diseases
Bottom Line
View on ClinicalTrials.gov: NCT00425477 ↗Enrolled (actual)
26
Serious AEs
26.9%
Results posted
Sep 2018
Primary outcome: Primary: Clinical Response (Complete and Partial) — 0; 4; 4; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- sargramostim (Biological); bexarotene (Drug); cytogenetic analysis (Genetic); fluorescence in situ hybridization (Genetic); flow cytometry (Other); laboratory biomarker analysis (Other); biopsy (Procedure)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Primary completion
- Sep 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Response (Complete and Partial) |
0; 4; 4; 5 | — |
| SECONDARY Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements |
524; 931 | — |
| SECONDARY Biological Activity as Measured by in Vivo Induction of Terminal Differentiation of Myeloid Progenitors and in Vivo Changes in Detectable Chromosomal Abnormalities |
— | — |
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00425477). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.