Clinical trial · Interventional
Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML
NCT00425477CI-TRIAL-00035163completedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic/Myeloproliferative Diseases | Myelodysplastic/Myeloproliferative Neoplasm | ALIAS | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
Interventions
Interventions (7)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| bexarotene | Drug | Bexarotene | ALIAS |
| biopsy | Procedure | — | UNRESOLVED |
| cytogenetic analysis | Genetic | — | UNRESOLVED |
| flow cytometry | Other | — | UNRESOLVED |
| fluorescence in situ hybridization | Genetic | — | UNRESOLVED |
| laboratory biomarker analysis | Other | — | UNRESOLVED |
| sargramostim | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Bexarotene + GM-CSF
- description
- 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.
- interventionNames
- Biological: sargramostim
- Drug: bexarotene
- Genetic: cytogenetic analysis
- Genetic: fluorescence in situ hybridization
- Other: flow cytometry
- Other: laboratory biomarker analysis
- Procedure: biopsy
Primary outcomes (1)
- measure
- Clinical Response (Complete and Partial)
- timeFrame
- assessed after 2 cycles, up to 2 years
- description
- Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.
Secondary outcomes (2)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 120 Years
Show eligibility criteria text
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 gemfibrozilReferences
Publications (0)
Data not yet available
No reference posted for this study.