Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)

NCT ID: NCT01542684

Last Updated: 2014-04-30

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-03-31

Study Completion Date

2013-04-30

Brief Summary

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The goal of this clinical research study is to learn if the combination of azacitidine and GM-CSF can help to control MDS. The safety of these drugs will also be studied.

Azacitidine is designed to block certain proteins that stop the function of tumor-fighting genes. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better.

Granulocyte-macrophage colony-stimulating factor (GM-CSF) is designed to help produce white blood cells. This may help to fight infections.

Detailed Description

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Study Drug Administration:

If you are found to be eligible to take part in this study, on Days 1-4 of every cycle, you will receive azacitidine by vein over 15-30 minutes.

You may receive drugs to help prevent nausea and vomiting before you receive your dose of azacitidine.

On Days 5-7 of every cycle, you will receive GM-CSF by vein over about 15 minutes or by injection.

Each study cycle will be about 4-6 weeks, depending on the study doctor's decision.

Study Visits:

One (1) time each week during every cycle, blood (about 2-3 teaspoons) will be drawn for routine tests.

At any time, if your doctor thinks it is needed, you will have a bone marrow aspirate to check the status of the disease.

Length of Study:

You may continue taking the study drugs for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

Your follow-up visits will be per standard of care for the disease.

This is an investigational study. Both azacitidine and GM-CSF are FDA approved and commercially available for the treatment of MDS. The study drug combination to treat MDS is considered investigational.

Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.

Conditions

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Leukemia

Keywords

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Leukemia Myelodysplastic Syndrome MDS Azacytidine 5-Azacytidine 5-AZA Vidaza 5-AZC Aza-CR Ladakamycin NSC-102816 GM-CSF Sargramostim Leukine

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Azacytidine + GM-CSF

Azacytidine administered intravenously (IV) or subcutaneously (SQ) at starting dose of 40 mg/m\^2, daily for 4 days.

GM-CSF administered IV or subcutaneously at 250 mcg/m\^2 one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.

Each treatment cycle will last at least 4 weeks

Group Type EXPERIMENTAL

Azacytidine

Intervention Type DRUG

Starting dose: 40 mg/m\^2 intravenously (IV) or subcutaneously (SQ) daily for 4 days.

GM-CSF

Intervention Type DRUG

250 mcg/m\^2 IV or SQ one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.

Interventions

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Azacytidine

Starting dose: 40 mg/m\^2 intravenously (IV) or subcutaneously (SQ) daily for 4 days.

Intervention Type DRUG

GM-CSF

250 mcg/m\^2 IV or SQ one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.

Intervention Type DRUG

Other Intervention Names

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5-Azacytidine 5-AZA Vidaza 5-AZC AZA-CR Ladakamycin NSC-102816 Saragramostim Leukine

Eligibility Criteria

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Inclusion Criteria

1. Patients with low- or intermediate-1-risk MDS according to the International Prognostic Scoring System (IPSS) classification
2. Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UT MD Anderson Cancer Center.
3. Age \>/= 18 years old.
4. Prior therapy with growth factor support, lenalidomide, or other investigational agents is allowed.
5. Previously untreated patients are eligible for this study.

Exclusion Criteria

1. Any previous adverse reaction (\>/= Common Terminology Criteria for Adverse Events (CTCAE) grade 2) to GM-CSF.
2. Prior treatment with azacytidine or decitabine.
3. Unresolved diarrhea \>/= CTCAE grade 2.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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M.D. Anderson Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Zeev Estrov, MD

Role: PRINCIPAL_INVESTIGATOR

UT MD Anderson Cancer Center

Locations

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UT MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Countries

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United States

Related Links

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http://www.mdanderson.org

UT MD Anderson Cancer Center Website

Other Identifiers

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2011-1123

Identifier Type: -

Identifier Source: org_study_id