Trial Outcomes & Findings for Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS) (NCT NCT01542684)
NCT ID: NCT01542684
Last Updated: 2014-04-30
Results Overview
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
TERMINATED
PHASE2
8 participants
Baseline up to 2 treatment cycles (8 weeks)
2014-04-30
Participant Flow
Recruitment Period: 3/15/2012 through 1/14/2013. All participants recruited at The University of Texas MD Anderson Cancer Center.
Participant milestones
| Measure |
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.
|
|---|---|
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Overall Study
STARTED
|
8
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)
Baseline characteristics by cohort
| Measure |
Azacytidine + GM-CSF
n=8 Participants
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.
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|---|---|
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Age, Continuous
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69 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 2 treatment cycles (8 weeks)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
Outcome measures
| Measure |
Azacytidine + GM-CSF
n=8 Participants
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.
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|---|---|
|
Overall Response Rate (ORR)
Complete Response (CR)
|
0 percentage of participants
|
|
Overall Response Rate (ORR)
Partial Response (PR)
|
0 percentage of participants
|
|
Overall Response Rate (ORR)
No Response
|
100 percentage of participants
|
Adverse Events
Azacytidine + GM-CSF
Serious adverse events
| Measure |
Azacytidine + GM-CSF
n=8 participants at risk
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.
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|---|---|
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General disorders
Fever
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Infections and infestations
Injection Site Reaction
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Infections and infestations
Urinary Tract Infection
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Bone Pain
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
Other adverse events
| Measure |
Azacytidine + GM-CSF
n=8 participants at risk
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.
|
|---|---|
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General disorders
Fever
|
50.0%
4/8 • Number of events 6 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Gastrointestinal disorders
Nausea/Vomiting
|
50.0%
4/8 • Number of events 6 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
2/8 • Number of events 3 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Chest Pain
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Gastrointestinal disorders
Consipation
|
37.5%
3/8 • Number of events 3 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Fatigue
|
50.0%
4/8 • Number of events 4 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath
|
25.0%
2/8 • Number of events 2 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Nervous system disorders
Dizziness
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Nervous system disorders
Memory Change
|
12.5%
1/8 • Number of events 3 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Nervous system disorders
Blurred Vision
|
12.5%
1/8 • Number of events 2 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Muscle Pain
|
37.5%
3/8 • Number of events 4 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Infections and infestations
Upper Respiratory Infecion
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Mouth Pain
|
25.0%
2/8 • Number of events 2 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Bone Pain
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Nervous system disorders
Vertigo
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Eye Dryness
|
12.5%
1/8 • Number of events 2 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
General disorders
Injection Site Reaction
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Musculoskeletal and connective tissue disorders
Verebral Fracture
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
|
Vascular disorders
Hypotension
|
12.5%
1/8 • Number of events 1 • Adverse events captured from the time of participant consent until 30 days after the last dose of drug.
|
Additional Information
Zeev Estrov, MD/Professor
The University of Texas MD Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place