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LeukemiaPhase 2NCT00425477

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

A Phase 2 study testing sargramostim, bexarotene and cytogenetic analysis in people with Leukemia and Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 120 years
What is being tested?
sargramostim, bexarotene and cytogenetic analysis
Could I receive a placebo?
No, everyone receives an active treatment
How many people
26 participants
Where
1 siteUnited States
Study dates
Nov 2006to 30 Sep 2016
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 120 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231-2410

Other trials in United States

About this study

Official title: A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML

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.

Read the 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.

Being tested
sargramostim, bexarotene, cytogenetic analysis, fluorescence in situ hybridization, flow cytometry
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)
Medical subject headings
Leukemia, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Anemia, Refractory, with Excess of Blasts, Leukemia, Myeloid, Acute, Myeloproliferative Disorders, Leukemia, Myelomonocytic, Chronic, Congenital Abnormalities

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • sargramostimBiological

  • bexaroteneDrug

  • cytogenetic analysisGenetic

  • fluorescence in situ hybridizationGenetic

  • flow cytometryOther

  • laboratory biomarker analysisOther

  • biopsyProcedure or surgery

Study groups in NCT00425477
Study groupRoleWhat they receive
Bexarotene + GM-CSFExperimentalBEX 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.
Receives: Biological: sargramostim; Drug: bexarotene; Genetic: cytogenetic analysis; Genetic: fluorescence in situ hybridization; Other: flow cytometry; Other: laboratory biomarker analysis; Procedure: biopsy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinical Response (Complete and Partial)

    Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.

    Measured: assessed after 2 cycles, up to 2 years

Secondary outcomes (2)

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

    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.

    Measured: Baseline and after two cycles

  2. Biological Activity as Measured by in Vivo Induction of Terminal Differentiation of Myeloid Progenitors and in Vivo Changes in Detectable Chromosomal Abnormalities

    Measured: Baseline and 6, 12, 24, and 36 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Jan 2007 (estimated)
  2. Study startNov 2006
  3. Primary completion30 Sep 2016
  4. Study completion30 Sep 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00425477
Sponsor's protocol ID
J0675 CDR0000525989
Other IDs
P30CA006973, JHOC-J0675, JHOC-NA_00003076
Status last verified
Sep 2018
First submitted
19 Jan 2007
Last updated
5 Oct 2018

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing sargramostim, bexarotene and cytogenetic analysis in people with Leukemia and Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Sep 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Clinical Response (Complete and Partial) (time frame: assessed after 2 cycles, up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00425477.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2018.

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