Alternative Dosing Regimens of Subcutaneous Azacitidine for Myelodysplastic Syndromes
Sponsor
Celgene (Industry)
Overall Status
Completed
CT.gov ID
NCT00102687
Collaborator
(none)
151
31
5
43
4.9
0.1
Study Details
Study Description
Brief Summary
The purpose of this study is to determine if azacitidine, combined with Best Supportive Care (BSC), is effective in treating myelodysplastic syndromes (MDS) when given according to a different doses and dosing schedules.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Detailed Description
Comparison/Control Interventions: The comparison is azacitidine at different doses and schedules.
Duration of Intervention: Treatment lasted for a maximum of 18 cycles, which is up to 24 months.
Study Design
Study Type:
Interventional
Actual Enrollment
:
151 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Multicenter, Randomized, Open-Label Study Comparing Three Alternative Dosing Regimens of Subcutaneous Azacitidine Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes
Actual Study Start Date
:
Jan 1, 2005
Actual Primary Completion Date
:
Aug 1, 2008
Actual Study Completion Date
:
Aug 1, 2008
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Aza-5 Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. |
Drug: azacitidine
Azacitidine is administered subcutaneously
Total of 18 cycles on treatment or early discontinuation.
|
| Experimental: Aza-5-2-2 Azacitidine administered subcutaneously at 75mg/m^2 for 5days with 2 days off, then for an additional 2 days, on a 28 day cycle. |
Drug: azacitidine
Azacitidine is administered subcutaneously
Total of 18 cycles on treatment or early discontinuation.
|
| Experimental: Aza-5-2-5 Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
Drug: azacitidine
Azacitidine is administered subcutaneously
Total of 18 cycles on treatment or early discontinuation.
|
| Experimental: Maintenance Aza 5 days q 4 weeks Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks. |
Drug: azacitidine
Azacitidine is administered subcutaneously
Total of 18 cycles on treatment or early discontinuation.
|
| Experimental: Maintenance Aza 5 days q 6 weeks Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks. |
Drug: azacitidine
Azacitidine is administered subcutaneously
Total of 18 cycles on treatment or early discontinuation.
|
Outcome Measures
Primary Outcome Measures
- Number of Participants In Best Hematological Response Categories as Determined by the Investigator Using International Working Group 2000 (IWG 2000) Criteria For Myelodysplastic Syndromes (MDS) During the Initial Study Period. [Day 1 (randomization) to 6 months]
Participant counts by best hematological response; complete remission(CR) is better than a partial remission(PR) which is better than stable disease(SD). Investigator determined responses followed IWG 2000 criteria for MDS CR: repeat bone marrow show <5% myeloblasts, and peripheral blood evaluations lasting >=2 months of hemoglobin(>110 g/L), neutrophils(>=1.5x10^9/L), platelets(>=100x10^9/L), blasts (0%) and no dysplasia PR is the same as CR for peripheral blood: bone marrow shows blasts decrease by >=50% or a less advanced FAB classification from pretreatment (see Population Descrip)
- Number of Participants With Best Hematological Improvement Derived Using International Working Group 2000 (IWG 2000) Criteria for MDS During the Initial Study Period. [Day 1 (randomization) to 6 months]
IWG 2000 Criteria: Pretreatment=hemoglobin <110g/L or RBC transfusion-dependence, platelet count <100x10^9/L or platelet transfusion dependence, absolute neutrophil count <1.5x10^9/L. Erythroid response: Major->20g/L increase in hemoglobin or transfusion independence. Minor- 10-20g/L increase in hemoglobin or >=50% decrease in transfusion requirements. Platelet response: Major-absolute increase of platelet count by >=30x10^9/L or platelet transfusion independence. Minor->=50% increase in platelet count with net increase >10x10^9/L but <30x10^9/L. (continued in Population Description)
- Number of Participants With Overall Best Hematologic Response and Hematologic Improvement Based on IWG 2000 Criteria For MDS During the Initial Study Period [Day 1 (randomization) to 6 months]
Number of participants whose best hematological outcome was either complete remission (CR), partial remission (PR) (as determined by the investigator), or any hematologic improvement (based on the IWG 2000 criteria for MDS). See previous outcomes for detailed definitions.
- Number of Participants Who Improved or Maintained The Hematologic Response From the Initial Study Period (Based on IWG 2000 Criteria For MDS) During the Maintenance Period [24 months]
Hematologic response during the maintenance period are compared to the response in the initial study period. Initial response could have been a complete remission, a partial remission, stable disease or a hematologic improvement. Maintenance period best response is after randomization to a maintenance arm for those randomized, and is after the start of cycle 7 for those remaining on initial period treatment throughout the study.
Secondary Outcome Measures
- Baseline Hemoglobin Values [Day 1 (randomization)]
The median values for hemoglobin based on blood tests performed on study day 1 (prior to study treatment) constitute a baseline measure for hemoglobin. Baseline values are used to compare to values following treatment.
- Change From Baseline in Hemoglobin at End of Initial Study Period (6 Months) [6 months]
The difference between hemoglobin values at the end of the initial study period minus the hemoglobin values at baseline.
- Change From Baseline in Hemoglobin at the End of the Maintenance Study Period [24 months]
The difference between hemoglobin values at the end of the maintenance study period minus the hemoglobin values at baseline.
- Baseline Platelet Values [Day 1 (randomization)]
The median values for platelets based on blood tests performed on study day 1 (prior to study treatment) constitute a baseline measure for platelets. Baseline values are used to compare to values following treatment.
- Change From Baseline in Platelets at the End of Initial Study Period (6 Months) [6 months]
The difference between platelet values at the end of the initial study period minus the platelet values at baseline.
- Change From Baseline in Platelets at the End of the Maintenance Study Period (Month 24) [24 months]
The difference between platelet values at the end of the maintenance study period minus the platelet values at baseline.
- Baseline Absolute Neutrophil Count (ANC) Values [Day 1 (randomization)]
The median values for ANC based on blood tests performed on study day 1 (prior to study treatment) constitute a baseline measure for ANC. Baseline values are used to compare to values following treatment.
- Change From Baseline in Absolute Neutrophil Count (ANC) at the End of Initial Study Period (6 Months) [6 months]
The difference between ANC values at the end of the initial study period minus the ANC values at baseline.
- Change From Baseline in Absolute Neutrophil Count (ANC) at the End of the Maintenance Study Period (Month 24) [24 months]
The difference between ANC values at the end of the maintenance study period minus the ANC values at baseline.
- Red Blood Cell (RBC) Transfusion Status at Baseline and End of Initial Study Period (6 Months) [6 months]
Shift table comparing the RBC transfusion status of patients at the end of the initial study period to the transfusion status at baseline.
- Platelet Transfusion Status at Baseline and End of Initial Study Period (6 Months) [6 months]
Shift table comparing the platelet transfusion status of patients at the end of the initial study period to the transfusion status at baseline.
- Red Blood Cell (RBC) Transfusion Status at Baseline and End of Maintenance Study Period (24 Months) [24 months]
Shift table comparing the RBC transfusion status of patients at the end of the maintenance study period to the transfusion status at baseline.
- Platelet Transfusion Status at Baseline and End of Maintenance Study Period (24 Months) [24 months]
Shift table comparing the platelet transfusion status of patients at the end of the maintenance study period to the transfusion status at baseline.
- Change From Baseline in the Number of Infections Requiring Treatment With IV Antibiotics Per Treatment Cycle (28 Days) for the Initial Study Period [6 months]
Baseline uses the average number of infections requiring IV antibiotic treatment from the 28 days prior to and including the day of first dose to an initial treatment arm. Initial study period values total the number of infections requiring IV antibiotic treatment divided by the number of treatment cycles (each cycle is approximately one month).
- Change From Baseline in the Number of Infections Requiring Treatment With IV Antibiotics Per Treatment Cycle (28 Days) for the Maintenance Study Period [24 months]
Baseline uses the average number of infections requiring IV antibiotic treatment from the 28 days prior to and including the day of first dose to an initial treatment arm. Maintenance study period values total the number of infections requiring IV antibiotic treatment divided by the number of treatment cycles (each cycle is approximately one month).
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Diagnosis of refractory anemia, refractory anemia with ringed sideroblasts and at least one of the following: a)Anemia with hemoglobin <110g/L and requires at least 1 unit packed red blood cell transfusions every 28 days; b)Thrombocytopenia with platelet counts <100 x 109/L; or c)Neutropenia with absolute neutrophil count <1.5 x 109/L.
-
OR, Refractory anemia with excess blasts or refractory anemia with excess blast in transformation, according to the French-American-British classification system for MDS.
-
At least 18 years of age.
-
Have a life expectancy of >7 months.
-
Unlikely to proceed to bone marrow or stem cell transplantation therapy following remission.
-
Have serum bilirubin levels less than or equal to 1.5 times the upper limit of the normal (ULN) range for the laboratory.
-
Have serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum glutamic-pyruvic transaminase (alanine aminotransferase) levels less than or equal to 2 x ULN.
-
Have serum creatinine levels less than or equal to 1.5 x ULN.
Exclusion Criteria:
-
Secondary MDS.
-
Prior treatment with azacitidine.
-
Any prior history of Acute Myeloid Leukemia (AML).
-
Malignant or metastatic disease within the previous 12 months.
-
Uncorrected red cell folate deficiency or vitamin B12 deficiency.
-
Hepatic tumors.
-
Radiation, chemotherapy, or cytotoxic therapy for non-MDS conditions in the previous 12 months.
-
Known or suspected hypersensitivity to azacitidine or mannitol.
-
Prior transplantation or cytotoxic therapy to treat MDS. Prior use of Revlimid and Thalomid allowed after 30 day washout.
-
Serious medical illness likely to limit survival to less than or equal to 7 months.
-
Treatment with androgenic hormones during the previous 14 days
-
Active viral infection with known human immunodeficiency virus or vial hepatitis Type B or C.
-
Treatment with other investigational drugs with the previous 30 days.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Comprehensive Blood and Cancer Center, Research Department | Bakersfield | California | United States | 93309 |
| 2 | Tower Cancer Research Foundation | Beverly Hills | California | United States | 90211 |
| 3 | Cancer Center of Colorado Springs, The Oncology Clinic, PC | Colorado Springs | Colorado | United States | 80907 |
| 4 | Rocky Mountain Cancer Centers, LLP | Denver | Colorado | United States | 80218 |
| 5 | Washington Cancer Institute | Washington | District of Columbia | United States | 20010 |
| 6 | Florida Cancer Institute | New Port Richey | Florida | United States | 34652 |
| 7 | Cancer Centers of Florida, P.A. | Ocoee | Florida | United States | 34761 |
| 8 | Joliet Oncology-Hematology Associates, Ltd. | Joliet | Illinois | United States | 60435 |
| 9 | Oncology/Hematology Associates of Central Illinois, PC | Peoria | Illinois | United States | 61615-7828 |
| 10 | Central Indiana Cancer Centers | Indianapolis | Indiana | United States | 46227 |
| 11 | Hematology & Oncology Specialists LLC | Metairie | Louisiana | United States | 70115 |
| 12 | Great Lakes Cancer Institute Breslin Cancer Center | Lansing | Michigan | United States | 48910 |
| 13 | The Center for Cancer Care and Research | Saint Louis | Missouri | United States | 63141 |
| 14 | Hackensack University Medical Center | Hackensack | New Jersey | United States | 07601 |
| 15 | Greater Dayton Cancer Center | Kettering | Ohio | United States | 45409 |
| 16 | Western Pennsylvania Cancer Institute | Pittsburgh | Pennsylvania | United States | 15224 |
| 17 | Oncology Services of Aberdeen | Aberdeen | South Dakota | United States | 57401 |
| 18 | Avera Cancer Institute Leukemia-Bone Marrow Transplant Center | Sioux Falls | South Dakota | United States | 57105 |
| 19 | McLeod Cancer and Blood Center | Johnson City | Tennessee | United States | 37604 |
| 20 | The Sarah Cannon Research Institute | Nashville | Tennessee | United States | 37203 |
| 21 | Texas Oncology, P.A. | Bedford | Texas | United States | 76022 |
| 22 | Texas Cancer Center at Medical City | Dallas | Texas | United States | 75230 |
| 23 | Texas Oncology, PA | Fort Worth | Texas | United States | 76104 |
| 24 | San Antonio Tumor & Blood Clinic | Fredericksburg | Texas | United States | 78624 |
| 25 | Cancer Care Centers of South Texas - HOAST | San Antonio | Texas | United States | 78229 |
| 26 | Virginia Oncology Associates - Lake Wright Cancer Center | Norfolk | Virginia | United States | 23502 |
| 27 | Highline Medical Oncology | Burien | Washington | United States | 98166 |
| 28 | Puget Sound Cancer Center | Edmonds | Washington | United States | 98026 |
| 29 | Puget Sound Cancer Center | Seattle | Washington | United States | 98133 |
| 30 | Cancer Care Northwest | Spokane | Washington | United States | 99218 |
| 31 | Northwest Cancer Specialists, P.C. | Vancouver | Washington | United States | 98684 |
Sponsors and Collaborators
- Celgene
Investigators
- Study Director: CL Beach, Celgene Corporation
Study Documents (Full-Text)
None provided.More Information
Publications
- Komrokji R, Swern AS, Grinblatt D, Lyons RM, Tobiasson M, Silverman LR, Sayar H, Vij R, Fliss A, Tu N, Sugrue MM. Azacitidine in Lower-Risk Myelodysplastic Syndromes: A Meta-Analysis of Data from Prospective Studies. Oncologist. 2018 Feb;23(2):159-170. doi: 10.1634/theoncologist.2017-0215. Epub 2017 Nov 8.
- Lyons R, et al. Tolerability and hematologic improvement assessed using three alternative dosing schedules of azacitidine in patients with myelodysplastic syndromes. Presented at the 2007 ASCO Annual Meeting, June 1-5, 2007, Chicago, IL. Abstract No. 7083
- R. Lyons, et al. Rapid onset of effectiveness with three alternative azacitidine (aza) dosing regimens in patients (pts) with myelodysplastic syndromes (MDS). Haematologica 2008;93(suppl 1):Abs.0232.
Responsible Party:
Celgene
ClinicalTrials.gov Identifier:
NCT00102687
Other Study ID Numbers:
- AZA PH US 2004 CL003
First Posted:
Feb 1, 2005
Last Update Posted:
Nov 22, 2019
Last Verified:
Nov 1, 2019
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail | One hundred and eighty-four patients were screened. |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks |
|---|---|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23 | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23 |
| Period Title: Initial Period | |||||
| STARTED | 50 | 50 | 51 | 0 | 0 |
| COMPLETED | 32 | 22 | 25 | 0 | 0 |
| NOT COMPLETED | 18 | 28 | 26 | 0 | 0 |
| Period Title: Initial Period | |||||
| STARTED | 13 | 6 | 8 | 22 | 21 |
| COMPLETED | 6 | 2 | 2 | 12 | 8 |
| NOT COMPLETED | 7 | 4 | 6 | 10 | 13 |
Baseline Characteristics
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 | Total |
|---|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. | Total of all reporting groups |
| Overall Participants | 50 | 50 | 51 | 151 |
| Age (years) [Median (Full Range) ] | ||||
| Median (Full Range) [years] |
76.0
|
73.0
|
76.0
|
75.0
|
| Sex: Female, Male (Count of Participants) | ||||
| Female |
17
34%
|
22
44%
|
14
27.5%
|
53
35.1%
|
| Male |
33
66%
|
28
56%
|
37
72.5%
|
98
64.9%
|
| Race/Ethnicity, Customized (Count of Participants) | ||||
| Caucasian |
47
94%
|
41
82%
|
48
94.1%
|
136
90.1%
|
| Black or African American |
0
0%
|
4
8%
|
2
3.9%
|
6
4%
|
| Asian/Oriental |
1
2%
|
0
0%
|
0
0%
|
1
0.7%
|
| Hispanic |
2
4%
|
5
10%
|
1
2%
|
8
5.3%
|
| Other |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| ECOG Performance Status (Count of Participants) | ||||
| 0=Fully active |
12
24%
|
19
38%
|
14
27.5%
|
45
29.8%
|
| 1=Restricted |
32
64%
|
23
46%
|
29
56.9%
|
84
55.6%
|
| 2=Ambulatory/capable of self care |
3
6%
|
5
10%
|
7
13.7%
|
15
9.9%
|
| 3=Capable/limited care |
3
6%
|
3
6%
|
1
2%
|
7
4.6%
|
| FAB Classification (Count of Participants) | ||||
| RA |
22
44%
|
22
44%
|
21
41.2%
|
65
43%
|
| RARS |
7
14%
|
7
14%
|
7
13.7%
|
21
13.9%
|
| RAEB |
14
28%
|
14
28%
|
17
33.3%
|
45
29.8%
|
| RAEB-T |
2
4%
|
1
2%
|
1
2%
|
4
2.6%
|
| CMMoL with <5% blasts |
3
6%
|
3
6%
|
1
2%
|
7
4.6%
|
| CMMoL with >=5% blasts |
2
4%
|
1
2%
|
1
2%
|
4
2.6%
|
| CMMoL with missing blasts |
0
0%
|
2
4%
|
3
5.9%
|
5
3.3%
|
| AML |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Other |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Transfusion Dependence - Platelets (Count of Participants) | ||||
| Platelet transfusion dependent |
4
8%
|
2
4%
|
1
2%
|
7
4.6%
|
| Not platelet transfusion dependent |
46
92%
|
48
96%
|
50
98%
|
144
95.4%
|
| Transfusion Dependence Status - RBCs (Count of Participants) | ||||
| RBC transfusion dependent |
25
50%
|
24
48%
|
22
43.1%
|
71
47%
|
| Not RBC transfusion dependent |
25
50%
|
26
52%
|
29
56.9%
|
80
53%
|
Outcome Measures
| Title | Number of Participants In Best Hematological Response Categories as Determined by the Investigator Using International Working Group 2000 (IWG 2000) Criteria For Myelodysplastic Syndromes (MDS) During the Initial Study Period. |
|---|---|
| Description | Participant counts by best hematological response; complete remission(CR) is better than a partial remission(PR) which is better than stable disease(SD). Investigator determined responses followed IWG 2000 criteria for MDS CR: repeat bone marrow show <5% myeloblasts, and peripheral blood evaluations lasting >=2 months of hemoglobin(>110 g/L), neutrophils(>=1.5x10^9/L), platelets(>=100x10^9/L), blasts (0%) and no dysplasia PR is the same as CR for peripheral blood: bone marrow shows blasts decrease by >=50% or a less advanced FAB classification from pretreatment (see Population Descrip) |
| Time Frame | Day 1 (randomization) to 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population. Patients without a second bone marrow assessment could not be evaluated for hematologic response. (Outcome Description continued)SD is a failure to achieve at least a PR, but with no evidence of progression for at least 2 months. |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 50 | 51 |
| Overall Response (CR+PR) |
4
8%
|
3
6%
|
4
7.8%
|
| Complete remission (CR) |
2
4%
|
2
4%
|
2
3.9%
|
| Partial remission (PR) |
2
4%
|
1
2%
|
2
3.9%
|
| Stable disease |
22
44%
|
19
38%
|
20
39.2%
|
| Title | Number of Participants With Best Hematological Improvement Derived Using International Working Group 2000 (IWG 2000) Criteria for MDS During the Initial Study Period. |
|---|---|
| Description | IWG 2000 Criteria: Pretreatment=hemoglobin <110g/L or RBC transfusion-dependence, platelet count <100x10^9/L or platelet transfusion dependence, absolute neutrophil count <1.5x10^9/L. Erythroid response: Major->20g/L increase in hemoglobin or transfusion independence. Minor- 10-20g/L increase in hemoglobin or >=50% decrease in transfusion requirements. Platelet response: Major-absolute increase of platelet count by >=30x10^9/L or platelet transfusion independence. Minor->=50% increase in platelet count with net increase >10x10^9/L but <30x10^9/L. (continued in Population Description) |
| Time Frame | Day 1 (randomization) to 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population. Patients count only once for best response within an improvement category. (Outcome Description continued) Neutrophil response: Major->=100% increase in neutrophil count or an absolute increase of >0.5x10^9/L. Minor->=100% increase but an absolute increase of <0.5x10^9/L. |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 50 | 51 |
| Any Improvement n=50,49,47 |
29
58%
|
20
40%
|
26
51%
|
| Erythroid response - Major n=44,43,41 |
18
36%
|
15
30%
|
18
35.3%
|
| Erythroid response - Minor n=44,43,41 |
4
8%
|
1
2%
|
2
3.9%
|
| Platelet response - Major n=24,28,28 |
10
20%
|
11
22%
|
10
19.6%
|
| Platelet response - Minor n=24,28,28 |
2
4%
|
0
0%
|
0
0%
|
| Neutrophil response - Major n=24,22,16 |
4
8%
|
3
6%
|
3
5.9%
|
| Neutrophil response - Minor n=24,22,16 |
2
4%
|
0
0%
|
0
0%
|
| Title | Number of Participants With Overall Best Hematologic Response and Hematologic Improvement Based on IWG 2000 Criteria For MDS During the Initial Study Period |
|---|---|
| Description | Number of participants whose best hematological outcome was either complete remission (CR), partial remission (PR) (as determined by the investigator), or any hematologic improvement (based on the IWG 2000 criteria for MDS). See previous outcomes for detailed definitions. |
| Time Frame | Day 1 (randomization) to 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 50 | 51 |
| Number [participants] |
30
60%
|
20
40%
|
27
52.9%
|
| Title | Baseline Hemoglobin Values |
|---|---|
| Description | The median values for hemoglobin based on blood tests performed on study day 1 (prior to study treatment) constitute a baseline measure for hemoglobin. Baseline values are used to compare to values following treatment. |
| Time Frame | Day 1 (randomization) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 49 | 50 |
| Median (Full Range) [g/L] |
94.0
|
98.0
|
95.5
|
| Title | Change From Baseline in Hemoglobin at End of Initial Study Period (6 Months) |
|---|---|
| Description | The difference between hemoglobin values at the end of the initial study period minus the hemoglobin values at baseline. |
| Time Frame | 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 49 | 46 |
| Median (Full Range) [g/L] |
2.3
|
2.9
|
5.3
|
| Title | Number of Participants Who Improved or Maintained The Hematologic Response From the Initial Study Period (Based on IWG 2000 Criteria For MDS) During the Maintenance Period |
|---|---|
| Description | Hematologic response during the maintenance period are compared to the response in the initial study period. Initial response could have been a complete remission, a partial remission, stable disease or a hematologic improvement. Maintenance period best response is after randomization to a maintenance arm for those randomized, and is after the start of cycle 7 for those remaining on initial period treatment throughout the study. |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population. |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23 | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23 | Combines participants who continued their original treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 27 |
| Improved response from initial study period |
7
14%
|
10
20%
|
12
23.5%
|
| Maintained response from initial study period |
6
12%
|
5
10%
|
10
19.6%
|
| Title | Change From Baseline in Hemoglobin at the End of the Maintenance Study Period |
|---|---|
| Description | The difference between hemoglobin values at the end of the maintenance study period minus the hemoglobin values at baseline. |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23 | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23 | Combines participants who continued their original treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 26 |
| Median (Full Range) [g/L] |
5.9
|
7.3
|
14.5
|
| Title | Baseline Platelet Values |
|---|---|
| Description | The median values for platelets based on blood tests performed on study day 1 (prior to study treatment) constitute a baseline measure for platelets. Baseline values are used to compare to values following treatment. |
| Time Frame | Day 1 (randomization) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 49 | 49 | 49 |
| Median (Full Range) [x10(9)/L] |
110.0
|
90.0
|
86.0
|
| Title | Change From Baseline in Platelets at the End of Initial Study Period (6 Months) |
|---|---|
| Description | The difference between platelet values at the end of the initial study period minus the platelet values at baseline. |
| Time Frame | 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 49 | 49 | 45 |
| Median (Full Range) [x10^9/L] |
4.8
|
5.7
|
12.6
|
| Title | Change From Baseline in Platelets at the End of the Maintenance Study Period (Month 24) |
|---|---|
| Description | The difference between platelet values at the end of the maintenance study period minus the platelet values at baseline. |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23. | Combines participants who continued their original treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 26 |
| Median (Full Range) [x10^9/L] |
19.8
|
7.7
|
17.3
|
| Title | Baseline Absolute Neutrophil Count (ANC) Values |
|---|---|
| Description | The median values for ANC based on blood tests performed on study day 1 (prior to study treatment) constitute a baseline measure for ANC. Baseline values are used to compare to values following treatment. |
| Time Frame | Day 1 (randomization) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 49 | 50 |
| Median (Full Range) [x10^9/L] |
1.6
|
1.9
|
2.3
|
| Title | Change From Baseline in Absolute Neutrophil Count (ANC) at the End of Initial Study Period (6 Months) |
|---|---|
| Description | The difference between ANC values at the end of the initial study period minus the ANC values at baseline. |
| Time Frame | 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 49 | 46 |
| Median (Full Range) [x10^9/L] |
-0.3
|
-0.3
|
-0.4
|
| Title | Change From Baseline in Absolute Neutrophil Count (ANC) at the End of the Maintenance Study Period (Month 24) |
|---|---|
| Description | The difference between ANC values at the end of the maintenance study period minus the ANC values at baseline. |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23. | Combines participants who continued their treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 26 |
| Median (Full Range) [x10^9/L] |
-0.1
|
-0.2
|
-0.4
|
| Title | Red Blood Cell (RBC) Transfusion Status at Baseline and End of Initial Study Period (6 Months) |
|---|---|
| Description | Shift table comparing the RBC transfusion status of patients at the end of the initial study period to the transfusion status at baseline. |
| Time Frame | 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 50 | 51 |
| Baseline Dependent(n=25,24,22) - 6 mo Independent |
16
32%
|
14
28%
|
13
25.5%
|
| Baseline Dependent(n=25,24,22) - 6 mo Dependent |
9
18%
|
10
20%
|
9
17.6%
|
| Baseline Independent(n=25,26,29)- 6 mo Independent |
19
38%
|
16
32%
|
19
37.3%
|
| Baseline Independent(n=25,26,29) - 6 mo Dependent |
6
12%
|
10
20%
|
10
19.6%
|
| Title | Platelet Transfusion Status at Baseline and End of Initial Study Period (6 Months) |
|---|---|
| Description | Shift table comparing the platelet transfusion status of patients at the end of the initial study period to the transfusion status at baseline. |
| Time Frame | 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 50 | 51 |
| Baseline Dependent (n=4,2,1) - 6 mo Independent |
3
6%
|
2
4%
|
0
0%
|
| Baseline Dependent (n=4,2,1) - 6 mo Dependent |
1
2%
|
0
0%
|
1
2%
|
| Baseline Independent(n=46,48,50)- 6 mo Independent |
43
86%
|
42
84%
|
41
80.4%
|
| Baseline Independent (n=46,48,50) - 6 mo Dependent |
3
6%
|
6
12%
|
9
17.6%
|
| Title | Red Blood Cell (RBC) Transfusion Status at Baseline and End of Maintenance Study Period (24 Months) |
|---|---|
| Description | Shift table comparing the RBC transfusion status of patients at the end of the maintenance study period to the transfusion status at baseline. |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23. | Combines participants who continued their original treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 27 |
| Baseline Dependent (n=6,12,12) - 24 mo Independent |
5
10%
|
12
24%
|
10
19.6%
|
| Baseline Dependent (n=6,12,12) - 24 mo Dependent |
1
2%
|
0
0%
|
2
3.9%
|
| Baseline Independent(n=16,9,15)- 24 mo Independent |
15
30%
|
9
18%
|
15
29.4%
|
| Baseline Independent (n=16,9,15) - 24 mo Dependent |
1
2%
|
0
0%
|
0
0%
|
| Title | Platelet Transfusion Status at Baseline and End of Maintenance Study Period (24 Months) |
|---|---|
| Description | Shift table comparing the platelet transfusion status of patients at the end of the maintenance study period to the transfusion status at baseline. |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23 | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23 | Combines participants who continued their original treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 27 |
| Baseline Dependent (n=1,1,2) - 24 mo Independent |
1
2%
|
1
2%
|
2
3.9%
|
| Baseline Dependent (n=1,1,2) - 24 mo Dependent |
0
0%
|
0
0%
|
0
0%
|
| Baseline Independent(n=21,20,25)-24 mo Independent |
21
42%
|
20
40%
|
25
49%
|
| Baseline Independent(n=21,20,25) - 24 mo Dependent |
0
0%
|
0
0%
|
0
0%
|
| Title | Change From Baseline in the Number of Infections Requiring Treatment With IV Antibiotics Per Treatment Cycle (28 Days) for the Initial Study Period |
|---|---|
| Description | Baseline uses the average number of infections requiring IV antibiotic treatment from the 28 days prior to and including the day of first dose to an initial treatment arm. Initial study period values total the number of infections requiring IV antibiotic treatment divided by the number of treatment cycles (each cycle is approximately one month). |
| Time Frame | 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. |
| Measure Participants | 50 | 50 | 48 |
| Median (Full Range) [infections per cycle] |
0.00
|
0.00
|
0.00
|
| Title | Change From Baseline in the Number of Infections Requiring Treatment With IV Antibiotics Per Treatment Cycle (28 Days) for the Maintenance Study Period |
|---|---|
| Description | Baseline uses the average number of infections requiring IV antibiotic treatment from the 28 days prior to and including the day of first dose to an initial treatment arm. Maintenance study period values total the number of infections requiring IV antibiotic treatment divided by the number of treatment cycles (each cycle is approximately one month). |
| Time Frame | 24 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent to treat population |
| Arm/Group Title | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | Initial Period Treatment Continued Into Maintenance |
|---|---|---|---|
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23. | Combines participants who continued their original treatment assigned during the initial study period through month 7 to month 23. |
| Measure Participants | 22 | 21 | 27 |
| Median (Full Range) [infections per cycle] |
0.00
|
0.00
|
0.00
|
Adverse Events
| Time Frame | The 3 groups in the Initial Period cover Day 1-6 months, and up to 24 months for participants that continued that treatment. The 2 Maintenance Period groups cover months 7-24 for subjects who were randomized to 1 of the 2 Maintenance treatment arms. | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | Safety population includes participants who received study treatment. Treatment emergent AEs include events within 42 days of the date of last dose. | |||||||||
| Arm/Group Title | Aza-5 | Aza-5-2-2 | Aza-5-2-5 | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | |||||
| Arm/Group Description | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days with 2 days off, then for an additional 2 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 50mg/m^2 for 5 days with 2 days off, then for an additional 5 days, on a 28 day cycle. | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 4 weeks from month 7 to month 23 | Azacitidine administered subcutaneously at 75mg/m^2 for 5 days every 6 weeks from month 7 to month 23 | |||||
All Cause Mortality |
||||||||||
| Aza-5 | Aza-5-2-2 | Aza-5-2-5 | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | / (NaN) | / (NaN) | / (NaN) | / (NaN) | / (NaN) | |||||
Serious Adverse Events |
||||||||||
| Aza-5 | Aza-5-2-2 | Aza-5-2-5 | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 18/50 (36%) | 27/50 (54%) | 22/48 (45.8%) | 7/22 (31.8%) | 13/21 (61.9%) | |||||
| Blood and lymphatic system disorders | ||||||||||
| Anaemia | 2/50 (4%) | 5/50 (10%) | 1/48 (2.1%) | 0/22 (0%) | 3/21 (14.3%) | |||||
| Febrile neutropenia | 4/50 (8%) | 2/50 (4%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Neutropenia | 0/50 (0%) | 1/50 (2%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pancytopenia | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Thrombocytopenia | 1/50 (2%) | 2/50 (4%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Cardiac disorders | ||||||||||
| Angina pectoris | 0/50 (0%) | 1/50 (2%) | 1/48 (2.1%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Atrial fibrillation | 1/50 (2%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Bradycardia | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Cardiac failure congestive | 0/50 (0%) | 2/50 (4%) | 2/48 (4.2%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Cardiac valve vegetation | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Myocardial infarction | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 1/22 (4.5%) | 1/21 (4.8%) | |||||
| Myocardial ischaemia | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Gastrointestinal disorders | ||||||||||
| Abdominal pain | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Colitis ischaemic | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Colonic polyp | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Constipation | 1/50 (2%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Dysphagia | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Enteritis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Gastritis | 0/50 (0%) | 1/50 (2%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Gastritis erosive | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Gastrointestinal haemorrhage | 1/50 (2%) | 1/50 (2%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Inguinal hernia, obstructive | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Intestinal obstruction | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Intussusception | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Mallory-Weiss syndrome | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Oesophageal ulcer | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Oesophagitis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pancreatitis | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Rectal haemorrhage | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Small intestinal obstruction | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| General disorders | ||||||||||
| Chest pain | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Fatigue | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Gait disturbance | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Pyrexia | 2/50 (4%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hepatobiliary disorders | ||||||||||
| Bile duct stone | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Cholangitis | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Infections and infestations | ||||||||||
| Bronchitis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Bronchopulmonary aspergillosis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Candida sepsis | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Catheter related infection | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Catheter site cellulitis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Cellulitis | 1/50 (2%) | 1/50 (2%) | 1/48 (2.1%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Diverticulitis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Enterobacter bacteraemia | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Enterococcal sepsis | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Escherichia bacteraemia | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Fungal oesophagitis | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Herpes zoster disseminated | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Incision site cellulitis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Lobar pneumonia | 0/50 (0%) | 2/50 (4%) | 2/48 (4.2%) | 1/22 (4.5%) | 1/21 (4.8%) | |||||
| Lung infection pseudomonal | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Oesophageal candidiasis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Oral herpes | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pneumonia | 1/50 (2%) | 1/50 (2%) | 5/48 (10.4%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Pseudomonal sepsis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Rectal abscess | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Sepsis | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Septic shock | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Staphylococcal infection | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Staphylococcal sepsis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Staphylococcal bacteraemia | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Urinary tract infection | 0/50 (0%) | 0/50 (0%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Urinary tract infection enterococcal | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Ankle fracture | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Burns second degree | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Contusion | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Fall | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hip fracture | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Subdural haematoma | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Traumatic haematoma | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Metabolism and nutrition disorders | ||||||||||
| Dehydration | 1/50 (2%) | 1/50 (2%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Fluid overload | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hypokalaemia | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hyponatraemia | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hypovolaemia | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Arthralgia | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Intervertebral disc protrusion | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||||||||
| Acute myeloid leukaemia | 1/50 (2%) | 4/50 (8%) | 0/48 (0%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| B-cell lymphoma | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Carcinoid tumour | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Chloroma | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Colon cancer | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Myelodysplastic syndrome | 3/50 (6%) | 0/50 (0%) | 1/48 (2.1%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Non-small cell lung cancer metastatic | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 1/22 (4.5%) | 0/21 (0%) | |||||
| Non-small cell lung cancer stage IV | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Small cell lung cancer metastatic | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Small cell lung cancer stage unspecified | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Nervous system disorders | ||||||||||
| Cerebrovascular accident | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 1/22 (4.5%) | 0/21 (0%) | |||||
| Dysarthria | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Headache | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 1/21 (4.8%) | |||||
| Normal pressure hydrocephalus | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Syncope | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Transient ischaemic attack | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 1/22 (4.5%) | 0/21 (0%) | |||||
| Psychiatric disorders | ||||||||||
| Confusional state | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Mental status changes | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Renal and urinary disorders | ||||||||||
| Calculus urinary | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Nephrolithiasis | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Reproductive system and breast disorders | ||||||||||
| Vaginal polyp | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Chronic obstructive pulmonary disease | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Dyspnoea | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pneumonia aspiration | 1/50 (2%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pneumonitis | 0/50 (0%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pulmonary embolism | 0/50 (0%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
Other (Not Including Serious) Adverse Events |
||||||||||
| Aza-5 | Aza-5-2-2 | Aza-5-2-5 | Maintenance Aza 5 Days q 4 Weeks | Maintenance Aza 5 Days q 6 Weeks | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 49/50 (98%) | 50/50 (100%) | 46/48 (95.8%) | 18/22 (81.8%) | 18/21 (85.7%) | |||||
| Blood and lymphatic system disorders | ||||||||||
| Anaemia | 11/50 (22%) | 17/50 (34%) | 12/48 (25%) | 3/22 (13.6%) | 5/21 (23.8%) | |||||
| Febrile neutropenia | 1/50 (2%) | 3/50 (6%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Leukopenia | 8/50 (16%) | 10/50 (20%) | 9/48 (18.8%) | 0/22 (0%) | 0/21 (0%) | |||||
| Lymphopenia | 5/50 (10%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Neutropenia | 15/50 (30%) | 22/50 (44%) | 17/48 (35.4%) | 2/22 (9.1%) | 2/21 (9.5%) | |||||
| Thrombocytopenia | 12/50 (24%) | 14/50 (28%) | 12/48 (25%) | 2/22 (9.1%) | 5/21 (23.8%) | |||||
| Cardiac disorders | ||||||||||
| Atrial fibrillation | 2/50 (4%) | 0/50 (0%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Gastrointestinal disorders | ||||||||||
| Abdominal pain | 7/50 (14%) | 7/50 (14%) | 6/48 (12.5%) | 0/22 (0%) | 0/21 (0%) | |||||
| Constipation | 28/50 (56%) | 25/50 (50%) | 21/48 (43.8%) | 2/22 (9.1%) | 2/21 (9.5%) | |||||
| Diarrhoea | 13/50 (26%) | 15/50 (30%) | 14/48 (29.2%) | 3/22 (13.6%) | 5/21 (23.8%) | |||||
| Dyspepsia | 1/50 (2%) | 6/50 (12%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Haemorrhoids | 2/50 (4%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Nausea | 31/50 (62%) | 31/50 (62%) | 23/48 (47.9%) | 2/22 (9.1%) | 6/21 (28.6%) | |||||
| Rectal haemorrhage | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Stomatitis | 1/50 (2%) | 4/50 (8%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Vomiting | 18/50 (36%) | 12/50 (24%) | 13/48 (27.1%) | 1/22 (4.5%) | 2/21 (9.5%) | |||||
| General disorders | ||||||||||
| Asthenia | 10/50 (20%) | 8/50 (16%) | 8/48 (16.7%) | 1/22 (4.5%) | 4/21 (19%) | |||||
| Chest pain | 1/50 (2%) | 5/50 (10%) | 1/48 (2.1%) | 1/22 (4.5%) | 2/21 (9.5%) | |||||
| Chills | 2/50 (4%) | 3/50 (6%) | 2/48 (4.2%) | 2/22 (9.1%) | 2/21 (9.5%) | |||||
| Fatigue | 25/50 (50%) | 30/50 (60%) | 29/48 (60.4%) | 5/22 (22.7%) | 3/21 (14.3%) | |||||
| Gait disturbance | 0/50 (0%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site bruising | 11/50 (22%) | 9/50 (18%) | 3/48 (6.3%) | 1/22 (4.5%) | 2/21 (9.5%) | |||||
| Injection site desquamation | 7/50 (14%) | 2/50 (4%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site erythema | 28/50 (56%) | 28/50 (56%) | 26/48 (54.2%) | 1/22 (4.5%) | 3/21 (14.3%) | |||||
| Injection site haemorrhage | 0/50 (0%) | 1/50 (2%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site inflammation | 3/50 (6%) | 1/50 (2%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site nodule | 2/50 (4%) | 1/50 (2%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site pain | 15/50 (30%) | 18/50 (36%) | 18/48 (37.5%) | 1/22 (4.5%) | 3/21 (14.3%) | |||||
| Injection site pruritus | 12/50 (24%) | 6/50 (12%) | 4/48 (8.3%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Injection site rash | 5/50 (10%) | 6/50 (12%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site urticaria | 3/50 (6%) | 1/50 (2%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Injection site warmth | 4/50 (8%) | 2/50 (4%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Oedema peripheral | 4/50 (8%) | 14/50 (28%) | 16/48 (33.3%) | 3/22 (13.6%) | 3/21 (14.3%) | |||||
| Pain | 4/50 (8%) | 1/50 (2%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pyrexia | 5/50 (10%) | 12/50 (24%) | 10/48 (20.8%) | 3/22 (13.6%) | 5/21 (23.8%) | |||||
| Infections and infestations | ||||||||||
| Cellulitis | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 2/21 (9.5%) | |||||
| Nasopharyngitis | 1/50 (2%) | 3/50 (6%) | 1/48 (2.1%) | 3/22 (13.6%) | 0/21 (0%) | |||||
| Oral candidiasis | 0/50 (0%) | 3/50 (6%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Oral herpes | 3/50 (6%) | 3/50 (6%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Upper respiratory tract infection | 1/50 (2%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Urinary tract infection | 9/50 (18%) | 4/50 (8%) | 6/48 (12.5%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Contusion | 7/50 (14%) | 7/50 (14%) | 7/48 (14.6%) | 1/22 (4.5%) | 3/21 (14.3%) | |||||
| Excoriation | 1/50 (2%) | 2/50 (4%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Rib fracture | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 2/21 (9.5%) | |||||
| Skin laceration | 3/50 (6%) | 2/50 (4%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Investigations | ||||||||||
| Blood bilirubin increased | 0/50 (0%) | 0/50 (0%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Blood urea increased | 1/50 (2%) | 3/50 (6%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Weight decreased | 1/50 (2%) | 0/50 (0%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Metabolism and nutrition disorders | ||||||||||
| Anorexia | 5/50 (10%) | 13/50 (26%) | 6/48 (12.5%) | 0/22 (0%) | 0/21 (0%) | |||||
| Decreased appetite | 5/50 (10%) | 2/50 (4%) | 5/48 (10.4%) | 0/22 (0%) | 0/21 (0%) | |||||
| Dehydration | 1/50 (2%) | 4/50 (8%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hyperglycaemia | 4/50 (8%) | 8/50 (16%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hypokalaemia | 4/50 (8%) | 5/50 (10%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hyponatraemia | 0/50 (0%) | 4/50 (8%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Arthralgia | 6/50 (12%) | 8/50 (16%) | 4/48 (8.3%) | 1/22 (4.5%) | 4/21 (19%) | |||||
| Back pain | 5/50 (10%) | 3/50 (6%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Bone pain | 3/50 (6%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Joint swelling | 0/50 (0%) | 3/50 (6%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Muscle spasms | 2/50 (4%) | 3/50 (6%) | 3/48 (6.3%) | 2/22 (9.1%) | 0/21 (0%) | |||||
| Muscular weakness | 3/50 (6%) | 3/50 (6%) | 2/48 (4.2%) | 3/22 (13.6%) | 0/21 (0%) | |||||
| Musculoskeletal pain | 4/50 (8%) | 1/50 (2%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Musculoskeletal stiffness | 3/50 (6%) | 2/50 (4%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Myalgia | 5/50 (10%) | 6/50 (12%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pain in extremity | 3/50 (6%) | 4/50 (8%) | 2/48 (4.2%) | 0/22 (0%) | 2/21 (9.5%) | |||||
| Pain in jaw | 3/50 (6%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Nervous system disorders | ||||||||||
| Dizziness | 11/50 (22%) | 12/50 (24%) | 11/48 (22.9%) | 3/22 (13.6%) | 1/21 (4.8%) | |||||
| Headache | 13/50 (26%) | 4/50 (8%) | 7/48 (14.6%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hypoaesthesia | 3/50 (6%) | 0/50 (0%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Neuropathy peripheral | 2/50 (4%) | 3/50 (6%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Paraesthesia | 3/50 (6%) | 2/50 (4%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Syncope | 1/50 (2%) | 1/50 (2%) | 3/48 (6.3%) | 2/22 (9.1%) | 0/21 (0%) | |||||
| Psychiatric disorders | ||||||||||
| Anxiety | 3/50 (6%) | 5/50 (10%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Depression | 3/50 (6%) | 8/50 (16%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Insomnia | 8/50 (16%) | 9/50 (18%) | 5/48 (10.4%) | 0/22 (0%) | 0/21 (0%) | |||||
| Renal and urinary disorders | ||||||||||
| Dysuria | 0/50 (0%) | 2/50 (4%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pollakiuria | 1/50 (2%) | 3/50 (6%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Urinary hesitation | 0/50 (0%) | 0/50 (0%) | 4/48 (8.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Cough | 8/50 (16%) | 7/50 (14%) | 5/48 (10.4%) | 3/22 (13.6%) | 2/21 (9.5%) | |||||
| Dyspnoea | 17/50 (34%) | 11/50 (22%) | 9/48 (18.8%) | 0/22 (0%) | 2/21 (9.5%) | |||||
| Dyspnoea exertional | 7/50 (14%) | 2/50 (4%) | 6/48 (12.5%) | 0/22 (0%) | 0/21 (0%) | |||||
| Epistaxis | 7/50 (14%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Nasal congestion | 2/50 (4%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pharyngolaryngeal pain | 3/50 (6%) | 6/50 (12%) | 5/48 (10.4%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Pleural effusion | 0/50 (0%) | 4/50 (8%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Postnasal drip | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Productive cough | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 2/21 (9.5%) | |||||
| Sinus congestion | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 2/22 (9.1%) | 1/21 (4.8%) | |||||
| Skin and subcutaneous tissue disorders | ||||||||||
| Dry skin | 1/50 (2%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Erythema | 2/50 (4%) | 4/50 (8%) | 2/48 (4.2%) | 0/22 (0%) | 0/21 (0%) | |||||
| Hyperhidrosis | 1/50 (2%) | 3/50 (6%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Night sweats | 2/50 (4%) | 3/50 (6%) | 0/48 (0%) | 0/22 (0%) | 0/21 (0%) | |||||
| Petechiae | 3/50 (6%) | 5/50 (10%) | 3/48 (6.3%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pruritus | 3/50 (6%) | 8/50 (16%) | 6/48 (12.5%) | 0/22 (0%) | 0/21 (0%) | |||||
| Pruritus generalised | 7/50 (14%) | 2/50 (4%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
| Rash | 12/50 (24%) | 8/50 (16%) | 4/48 (8.3%) | 0/22 (0%) | 3/21 (14.3%) | |||||
| Skin disorder | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 0/22 (0%) | 2/21 (9.5%) | |||||
| Vascular disorders | ||||||||||
| Hypertension | 0/50 (0%) | 0/50 (0%) | 0/48 (0%) | 2/22 (9.1%) | 0/21 (0%) | |||||
| Hypotension | 4/50 (8%) | 6/50 (12%) | 2/48 (4.2%) | 0/22 (0%) | 3/21 (14.3%) | |||||
| Pallor | 3/50 (6%) | 2/50 (4%) | 1/48 (2.1%) | 0/22 (0%) | 0/21 (0%) | |||||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
The investigator shall have the right to publish and/or present study data provided that the investigator shall (i)furnish the sponsor a copy of any proposed publication or presentation generally thirty (30) days in advance of the submission, (ii) delete any confidential information of the sponsor, and (iii) delay submission for generally up to ninety (90) days to permit the preparation and filing of intellectual property applications or until sponsor gives its consent in a timely manner.
Results Point of Contact
| Name/Title | CL Beach |
|---|---|
| Organization | Celgene Corporation |
| Phone | |
| CLBeach@celgene.com |
Responsible Party:
Celgene
ClinicalTrials.gov Identifier:
NCT00102687
Other Study ID Numbers:
- AZA PH US 2004 CL003
First Posted:
Feb 1, 2005
Last Update Posted:
Nov 22, 2019
Last Verified:
Nov 1, 2019