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

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ClinicalTrials.gov Identifier: NCT01542684
Recruitment Status : Terminated (Slow Accrual)
First Posted : March 2, 2012
Results First Posted : April 30, 2014
Last Update Posted : April 30, 2014
Sponsor:
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Leukemia
Interventions Drug: Azacytidine
Drug: GM-CSF
Enrollment 8
Recruitment Details Recruitment Period: 3/15/2012 through 1/14/2013. All participants recruited at The University of Texas MD Anderson Cancer Center.
Pre-assignment Details  
Arm/Group Title Azacytidine + GM-CSF
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 8
Completed 8
Not Completed 0
Arm/Group Title Azacytidine + GM-CSF
Hide Arm/Group Description

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.

Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 8 participants
69
(54 to 82)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
1
  12.5%
Male
7
  87.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants
8
1.Primary Outcome
Title Overall Response Rate (ORR)
Hide Description 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
Time Frame Baseline up to 2 treatment cycles (8 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Azacytidine + GM-CSF
Hide Arm/Group Description:

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.

Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: percentage of participants
Complete Response (CR) 0
Partial Response (PR) 0
No Response 100
Time Frame Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Azacytidine + GM-CSF
Hide Arm/Group Description

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.

All-Cause Mortality
Azacytidine + GM-CSF
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Azacytidine + GM-CSF
Affected / at Risk (%) # Events
Total   3/8 (37.50%)    
General disorders   
Fever  1  1/8 (12.50%)  1
Bone Pain  1  1/8 (12.50%)  1
Infections and infestations   
Injection Site Reaction  1  1/8 (12.50%)  1
Urinary Tract Infection  1  1/8 (12.50%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Azacytidine + GM-CSF
Affected / at Risk (%) # Events
Total   8/8 (100.00%)    
Gastrointestinal disorders   
Nausea/Vomiting  1  4/8 (50.00%)  6
Diarrhea  1  2/8 (25.00%)  3
Consipation  1  3/8 (37.50%)  3
General disorders   
Fever  1  4/8 (50.00%)  6
Chest Pain  1  1/8 (12.50%)  1
Fatigue  1  4/8 (50.00%)  4
Muscle Pain  1  3/8 (37.50%)  4
Mouth Pain  1  2/8 (25.00%)  2
Bone Pain  1  1/8 (12.50%)  1
Eye Dryness  1  1/8 (12.50%)  2
Injection Site Reaction  1  1/8 (12.50%)  1
Infections and infestations   
Upper Respiratory Infecion  1  1/8 (12.50%)  1
Musculoskeletal and connective tissue disorders   
Verebral Fracture  1  1/8 (12.50%)  1
Nervous system disorders   
Dizziness  1  1/8 (12.50%)  1
Memory Change  1  1/8 (12.50%)  3
Blurred Vision  1  1/8 (12.50%)  2
Vertigo  1  1/8 (12.50%)  1
Respiratory, thoracic and mediastinal disorders   
Shortness of Breath  1  2/8 (25.00%)  2
Cough  1  1/8 (12.50%)  1
Vascular disorders   
Hypotension  1  1/8 (12.50%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Zeev Estrov, MD/Professor
Organization: The University of Texas MD Anderson Cancer Center
Phone: 713-794-1675
EMail: eharriso@mdanderson.org
Layout table for additonal information
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01542684     History of Changes
Other Study ID Numbers: 2011-1123
First Submitted: February 27, 2012
First Posted: March 2, 2012
Results First Submitted: March 28, 2014
Results First Posted: April 30, 2014
Last Update Posted: April 30, 2014