NCT00425477 · REGISTRY CONDITION SEARCH

Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2018-10-05

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Clinical Response (Complete and Partial)

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outcome · POSTED

Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements

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outcome · POSTED

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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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

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
healthy Volunteers: false
maximum Age: 120 Years
minimum Age: 18 Years
sex: ALL
std Ages: ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

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.
intervention Names
Biological: sargramostim
Drug: bexarotene
Genetic: cytogenetic analysis
Genetic: fluorescence in situ hybridization
Other: flow cytometry
Other: laboratory biomarker analysis
Procedure: biopsy
label
Bexarotene + GM-CSF
type
EXPERIMENTAL

interventions

arm Group Labels
Bexarotene + GM-CSF
name
sargramostim
type
BIOLOGICAL
arm Group Labels
Bexarotene + GM-CSF
name
bexarotene
type
DRUG
arm Group Labels
Bexarotene + GM-CSF
name
cytogenetic analysis
type
GENETIC
arm Group Labels
Bexarotene + GM-CSF
name
fluorescence in situ hybridization
type
GENETIC
arm Group Labels
Bexarotene + GM-CSF
name
flow cytometry
type
OTHER
arm Group Labels
Bexarotene + GM-CSF
name
laboratory biomarker analysis
type
OTHER
arm Group Labels
Bexarotene + GM-CSF
name
biopsy
type
PROCEDURE

Study design

allocation

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 26
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.
measure
Clinical Response (Complete and Partial)
time Frame
assessed after 2 cycles, up to 2 years

secondary Outcomes

description
ANC count at baseline and after two cycles were measured and compared. Due to the limited number of clinical responders, the changes in transfusion requirements were not measured.
measure
Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements
time Frame
Baseline and after two cycles
measure
Biological Activity as Measured by in Vivo Induction of Terminal Differentiation of Myeloid Progenitors and in Vivo Changes in Detectable Chromosomal Abnormalities
time Frame
Baseline and 6, 12, 24, and 36 weeks

Full study description

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.

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice