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Trial record 32 of 70 for:    stem cell peripheral arterial disease AND cell therapy

Granulocyte-Macrophage Stimulating Factor in the Treatment of Peripheral Arterial Disease (GPAD-2)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01041417
Recruitment Status : Completed
First Posted : December 31, 2009
Results First Posted : December 23, 2014
Last Update Posted : December 23, 2014
Sponsor:
Collaborators:
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Arshed A. Quyyumi, Emory University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Peripheral Arterial Disease
Interventions Drug: Granulocyte-Macrophage Stimulating Factor (GM-CSF)
Drug: Placebo
Enrollment 159
Recruitment Details Between January 2010 and July 2012, 322 individuals with peripheral artery disease were assessed for eligibility. 159 subjects were enrolled into the study from Emory Healthcare, Veterans Affairs hospitals in Atlanta, and Medical College of Georgia.
Pre-assignment Details  
Arm/Group Title GM-CSF Placebo
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Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Period Title: Overall Study
Started 80 79
Completed 73 76
Not Completed 7 3
Reason Not Completed
Adverse Event             5             2
Lost to Follow-up             1             1
Death             1             0
Arm/Group Title GM-CSF Placebo Total
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Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks Total of all reporting groups
Overall Number of Baseline Participants 80 79 159
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 80 participants 79 participants 159 participants
64.3  (7.9) 63.6  (7.9) 63.9  (7.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 80 participants 79 participants 159 participants
Female
10
  12.5%
10
  12.7%
20
  12.6%
Male
70
  87.5%
69
  87.3%
139
  87.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 80 participants 79 participants 159 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
45
  56.3%
34
  43.0%
79
  49.7%
White
33
  41.3%
45
  57.0%
78
  49.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
2
   2.5%
0
   0.0%
2
   1.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 80 participants 79 participants 159 participants
80 79 159
1.Primary Outcome
Title Change in Peak Walking Time During Treadmill Exercise Tolerance Test From Baseline to 3 Months
Hide Description Exercise Tolerance Test (ETT) was conducted using the Gardner protocol. Participants exercised on a treadmill, starting at 2.0 mph. The intensity of exercise (speed) was increased in grade of 2% every 2 minutes. Participants were asked to exercise until symptom limitation and the time measured in seconds from the ETT was used for data analysis.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: seconds
109
(67 to 151)
56
(14 to 98)
2.Secondary Outcome
Title Change in Peak Walking Time During Treadmill Exercise Tolerance Test From Baseline to 6 Months
Hide Description Exercise Tolerance Test (ETT) was conducted using the Gardner protocol. Participants exercised on a treadmill, starting at 2.0 mph. The intensity of exercise (speed) was increased in grade of 2% every 2 minutes. Participants were asked to exercise until symptom limitation and the time measured in seconds from the ETT was used for data analysis.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: seconds
112
(71 to 153)
77
(36 to 117)
3.Secondary Outcome
Title Change in Claudication Onset Time (COT) From Baseline to 3 Months
Hide Description Claudication is pain, tired or weak feeling that occurs in the legs, usually during activity such as walking. The COT was measured as the time to onset of the participant's typical claudication as the maximum distance the patient could walk on the treadmill.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: seconds
81
(52 to 111)
61
(32 to 91)
4.Secondary Outcome
Title Change in Claudication Onset Time (COT) From Baseline to 6 Months
Hide Description Claudication is pain, tired or weak feeling that occurs in the legs, usually during activity such as walking. The COT was measured as the time to onset of the participant's typical claudication as the maximum distance the patient could walk on the treadmill.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: seconds
93
(64 to 122)
61
(33 to 89)
5.Secondary Outcome
Title Change in Walking Distance Scores on Walking Impairment Questionnaire (WIQ) From Baseline to 3 Months
Hide Description The WIQ quantifies walking difficulty on a 100-point scale, in which 0 indicates extreme difficulty and 100 indicates no difficulty with walking distance.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
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Granulocyte-Macrophage Colony Stimulating Factor: 80 subjects were randomized to receive GM-CSF Monday, Wednesday and Friday for 4 weeks of therapy.
Placebo: 79 subjects were randomized to receive placebo on Monday, Wednesday and Friday for 4 weeks.
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
12.5
(6.4 to 18.7)
4.8
(-0.2 to 9.8)
6.Secondary Outcome
Title Change in Walking Distance Scores on Walking Impairment Questionnaire (WIQ) From Baseline to 6 Months
Hide Description The WIQ quantifies walking difficulty on a 100-point scale, in which 0 indicates extreme difficulty and 100 indicates no difficulty with walking distance.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
8.0
(2.1 to 14.0)
3.7
(-0.9 to 8.3)
7.Secondary Outcome
Title Change in Walking Speed Score on Walking Impairment Questionnaire (WIQ) From Baseline to 3 Months
Hide Description The WIQ quantifies walking difficulty on a 100-point scale, in which 0 indicates extreme difficulty and 100 indicates no difficulty with walking speed.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:
Granulocyte-Macrophage Colony Stimulating Factor: 80 subjects were randomized to receive GM-CSF Monday, Wednesday and Friday for 4 weeks of therapy.
Placebo: 79 subjects were randomized to receive placebo on Monday, Wednesday and Friday for 4 weeks.
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
9.1
(4.2 to 14.0)
7.5
(2.1 to 12.8)
8.Secondary Outcome
Title Change in Walking Speed Score on Walking Impairment Questionnaire (WIQ) From Baseline to 6 Months
Hide Description The WIQ quantifies walking difficulty on a 100-point scale, in which 0 indicates extreme difficulty and 100 indicates no difficulty with walking speed.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:
Granulocyte-Macrophage Colony Stimulating Factor: 80 subjects were randomized to receive GM-CSF Monday, Wednesday and Friday for 4 weeks of therapy.
Placebo: 79 subjects were randomized to receive placebo on Monday, Wednesday and Friday for 4 weeks.
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
9.9
(4.6 to 15.3)
8.5
(3.8 to 13.2)
9.Secondary Outcome
Title Change in Stair Climbing Score on Walking Impairment Questionnaire (WIQ) From Baseline to 3 Months
Hide Description The WIQ quantifies walking difficulty on a 100-point scale, in which 0 indicates extreme difficulty and 100 indicates no difficulty with stair climbing elements.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:
Granulocyte-Macrophage Colony Stimulating Factor: 80 subjects were randomized to receive GM-CSF Monday, Wednesday and Friday for 4 weeks of therapy.
Placebo: 79 subjects were randomized to receive placebo on Monday, Wednesday and Friday for 4 weeks.
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
8.5
(3.0 to 13.9)
4.2
(-2.2 to 10.6)
10.Secondary Outcome
Title Change in Stair Climbing Score on Walking Impairment Questionnaire (WIQ) From Baseline to 6 Months
Hide Description The WIQ quantifies walking difficulty on a 100-point scale, in which 0 indicates extreme difficulty and 100 indicates no difficulty with stair climbing elements.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:
Granulocyte-Macrophage Colony Stimulating Factor: 80 subjects were randomized to receive GM-CSF Monday, Wednesday and Friday for 4 weeks of therapy.
Placebo: 79 subjects were randomized to receive placebo on Monday, Wednesday and Friday for 4 weeks.
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
10.4
(4.9 to 15.9)
6.6
(1.2 to 11.9)
11.Secondary Outcome
Title Change in Score on Physical Composite Score (PCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 3 Months
Hide Description The SF-36 is a standard quality of life instrument. The PCS represents the the physical burden on quality of life and is a summary of questions related to physical impact of a disease or condition (physical function, role physical, bodily pain, and general health). PCS is a summary measure derived from 8 scale score and the score ranges from 0-100; higher scores indicate better performance.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
4.1
(2.4 to 5.8)
2.1
(0.5 to 3.7)
12.Secondary Outcome
Title Change in Score on Physical Composite Score (PCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 6 Months
Hide Description The SF-36 is a standard quality of life instrument. The PCS represents the the physical burden on quality of life and is a summary of questions related to physical impact of a disease or condition (physical function, role physical, bodily pain, and general health). PCS is a summary measure derived from 8 scale score and the score ranges from 0-100; higher scores indicate better performance.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
5.2
(3.3 to 7.0)
3.1
(1.5 to 4.7)
13.Secondary Outcome
Title Change in Score on Mental Composite Score (MCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 3 Months
Hide Description The SF-36 is a standard quality of life instrument. The MCS represents the the mental burden on quality of life and is a summary of questions related to mental impact of a disease or condition (mental function, role emotional, vitality, and mental health). MCS is a summary measure derived from 8 scale score and the score ranges from 0-100; higher scores indicate better performance.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
0.7
(-1.5 to 2.9)
2.4
(-0.2 to 5.0)
14.Secondary Outcome
Title Change in Score on Mental Composite Score (MCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 6 Months
Hide Description The SF-36 is a standard quality of life instrument. The MCS represents the the mental burden on quality of life and is a summary of questions related to mental impact of a disease or condition (mental function, role emotional, vitality, and mental health). MCS is a summary measure derived from 8 scale score and the score ranges from 0-100; higher scores indicate better performance.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-0.2
(-2.4 to 2.0)
1.1
(-1.0 to 3.2)
15.Secondary Outcome
Title Change in Score on Physical Functioning Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 3 Months
Hide Description The SF-36 is a standard quality of life instrument. The physical functioning represents limitations in physical activities because of health problems. Physical functioning is a summary measure derived from 8 scale scores and the score ranges from 0-100; higher scores indicate better performance.
Time Frame Baseline, 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
11.4
(6.7 to 16.1)
4.8
(-0.1 to 9.6)
16.Secondary Outcome
Title Change in Score on Physical Functioning Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 6 Months
Hide Description The SF-36 is a standard quality of life instrument. The physical functioning represents limitations in physical activities because of health problems. Physical functioning is a summary measure derived from 8 scale scores and the score ranges from 0-100; higher scores indicate better performance.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description:

Granulocyte-Macrophage Colony Stimulating Factor injection - three times for four weeks

500 microgram dose of GM-CSF

Saline injection - three times for four weeks
Overall Number of Participants Analyzed 73 76
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
14.3
(9.7 to 18.9)
8.1
(3.7 to 12.4)
Time Frame Adverse events were collected at weeks 1, 2, 3, 4, 8 and 3 month follow-up and 6 month follow-up
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title GM-CSF Placebo
Hide Arm/Group Description

Granulocyte-Macrophage Colony Stimulating Factor injection - Monday, Wednesday and Friday for 4 weeks of therapy.

500 microgram dose of GM-CSF

Saline injection - Monday, Wednesday and Friday for 4 weeks.
All-Cause Mortality
GM-CSF Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
GM-CSF Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/80 (11.25%)      9/79 (11.39%)    
Cardiac disorders     
Heart Failure  0/80 (0.00%)  0 1/79 (1.27%)  1
Gastrointestinal disorders     
Abdominal Pain  1/80 (1.25%)  1 1/79 (1.27%)  1
Small Bowel Obstruction  0/80 (0.00%)  0 1/79 (1.27%)  1
Musculoskeletal and connective tissue disorders     
Chest pain, non-cardiac  1/80 (1.25%)  1 3/79 (3.80%)  3
Trauma [1]  0/80 (0.00%)  0 1/79 (1.27%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Prostate Cancer  1/80 (1.25%)  1 1/79 (1.27%)  1
Esophageal Cancer  1/80 (1.25%)  1 0/79 (0.00%)  0
Nervous system disorders     
Stroke  1/80 (1.25%)  1 0/79 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
COPD Exacerbation  1/80 (1.25%)  1 0/79 (0.00%)  0
Social circumstances     
Death  1/73 (1.37%)  1 0/76 (0.00%)  0
Surgical and medical procedures     
Elective Procedure [2]  1/80 (1.25%)  1 1/79 (1.27%)  1
Vascular disorders     
Critical Limb Ischemia  1/80 (1.25%)  1 0/79 (0.00%)  0
[1]
Admission for trauma
[2]
Admission for elective procedure
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GM-CSF Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   79/80 (98.75%)      69/79 (87.34%)    
Cardiac disorders     
Difficulty breathing  5/80 (6.25%)  5 8/79 (10.13%)  8
Chest pain  6/80 (7.50%)  6 7/79 (8.86%)  7
Gastrointestinal disorders     
Gastrointestinal side effects  21/80 (26.25%)  21 11/79 (13.92%)  11
General disorders     
Night sweats  13/80 (16.25%)  13 6/79 (7.59%)  6
Fatigue  17/80 (21.25%)  17 9/79 (11.39%)  9
Musculoskeletal and connective tissue disorders     
Joint/back/muscle pain  18/80 (22.50%)  18 14/79 (17.72%)  14
Swelling  9/80 (11.25%)  9 4/79 (5.06%)  4
Nervous system disorders     
Headaches  18/80 (22.50%)  18 9/79 (11.39%)  9
Skin and subcutaneous tissue disorders     
Rash  41/80 (51.25%)  41 1/79 (1.27%)  1
Study population were subjects with claudication who underwent angiography. Study design could not show therapeutic benefit of GM-CSF therapy with dose and duration changes. And study design encouraged all subjects to walk to claudication daily.
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: Dr. Arshed Quyyumi
Organization: Emory University School of Medicine
Phone: 4047273655
EMail: aquyyum@emory.edu
Layout table for additonal information
Responsible Party: Arshed A. Quyyumi, Emory University
ClinicalTrials.gov Identifier: NCT01041417     History of Changes
Other Study ID Numbers: IRB00030362
1RC2HL101515-01 ( U.S. NIH Grant/Contract )
First Submitted: December 29, 2009
First Posted: December 31, 2009
Results First Submitted: May 15, 2014
Results First Posted: December 23, 2014
Last Update Posted: December 23, 2014