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Slow Accrual
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Azacytidine + GM-CSF | Experimental | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Azacytidine | Drug | Starting dose: 40 mg/m^2 intravenously (IV) or subcutaneously (SQ) daily for 4 days. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) | ORR is percentage total participants with overall response (Complete Response (CR) or Partial Response (PR)) within two treatment cycles. Response based on modified International Working Group (IWG) criteria: Complete response - Bone marrow: 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia noted, Peripheral blood Hgb 11 g/dL, Platelets 100x109/L, Neutrophils 1.0x109/L, Blasts 0%. Partial response: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by 50% over pretreatment but still > 5% , Cellularity and morphology not relevant; Stable disease - Failure to achieve at least PR, but no evidence of progression for > 8 weeks; No Response or Failure - Death during treatment or disease progression characterized by worsening of cytopenias, increase in percentage of bone marrow blasts, or progression to a more advanced MDS French-American-British (FAB) classification subtype than pretreatme | Baseline up to 2 treatment cycles (8 weeks) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Zeev Estrov, MD | UT MD Anderson Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UT MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
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| Label | URL |
|---|---|
| UT MD Anderson Cancer Center Website | View source |
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Recruitment Period: 3/15/2012 through 1/14/2013. All participants recruited at The University of Texas MD Anderson Cancer Center.
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| ID | Title | Description |
|---|---|---|
| FG000 | Azacytidine + GM-CSF | Azacytidine administered intravenously (IV) or subcutaneously (SQ) at starting dose of 40 mg/m^2, daily for 4 days. Granulocyte-macrophage colony-stimulating factor (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 lasts at least 4 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 lasts at least 4 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Overall Response Rate (ORR) | ORR is percentage total participants with overall response (Complete Response (CR) or Partial Response (PR)) within two treatment cycles. Response based on modified International Working Group (IWG) criteria: Complete response - Bone marrow: 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia noted, Peripheral blood Hgb 11 g/dL, Platelets 100x109/L, Neutrophils 1.0x109/L, Blasts 0%. Partial response: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by 50% over pretreatment but still > 5% , Cellularity and morphology not relevant; Stable disease - Failure to achieve at least PR, but no evidence of progression for > 8 weeks; No Response or Failure - Death during treatment or disease progression characterized by worsening of cytopenias, increase in percentage of bone marrow blasts, or progression to a more advanced MDS French-American-British (FAB) classification subtype than pretreatme | Posted | Number | percentage of participants | Baseline up to 2 treatment cycles (8 weeks) |
|
Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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 lasts at least 4 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Zeev Estrov, MD/Professor | The University of Texas MD Anderson Cancer Center | 713-794-1675 | eharriso@mdanderson.org |
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| ID | Term |
|---|---|
| D007938 | Leukemia |
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D001374 | Azacitidine |
| D016178 | Granulocyte-Macrophage Colony-Stimulating Factor |
| C081222 | sargramostim |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
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| GM-CSF | Drug | 250 mcg/m^2 IV or SQ one day (the next day) after completion of azacytidine treatment, for 3 consecutive days. |
|
|
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| 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 Azacytidine: Starting dose: 40 mg/m^2 intravenously (IV) or subcutaneously (SQ) daily for 4 days. GM-CSF: 250 mcg/m^2 IV or SQ one day (the next day) after completion of azacytidine treatment, for 3 consecutive days. |
|
|
| 3 |
| 8 |
| 8 |
| 8 |
| Injection Site Reaction | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Urinary Tract Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Bone Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Nausea/Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Chest Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Consipation | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Fatigue | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Shortness of Breath | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Memory Change | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Blurred Vision | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Muscle Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Upper Respiratory Infecion | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Mouth Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Bone Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Vertigo | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
|
| Eye Dryness | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Injection Site Reaction | General disorders | CTCAE (4.0) | Systematic Assessment |
|
| Verebral Fracture | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Systematic Assessment |
|
| Hypotension | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
|
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| D001855 | Bone Marrow Diseases |
| D011743 |
| Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |