Phase 2 N=159 Randomized Triple-blind Treatment

Granulocyte-Macrophage Stimulating Factor in the Treatment of Peripheral Arterial Disease

Peripheral Arterial Disease

Bottom Line

View on ClinicalTrials.gov: NCT01041417 ↗

Enrolled (actual)

159

Serious AEs

11.3%

Results posted

Dec 2014

Primary outcome: Primary: Change in Peak Walking Time During Treadmill Exercise Tolerance Test From Baseline to 3 Months — 109; 56 seconds

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Granulocyte-Macrophage Stimulating Factor (GM-CSF) (Drug); Placebo (Drug)
Age: Adult, Older Adult · 21+ yrs
Sex: All
Sponsor: Emory University
Primary completion: Mar 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Peak Walking Time During Treadmill Exercise Tolerance Test From Baseline to 3 Months	109; 56	—
SECONDARY Change in Peak Walking Time During Treadmill Exercise Tolerance Test From Baseline to 6 Months	112; 77	—
SECONDARY Change in Claudication Onset Time (COT) From Baseline to 3 Months	81; 61	—
SECONDARY Change in Claudication Onset Time (COT) From Baseline to 6 Months	93; 61	—
SECONDARY Change in Walking Distance Scores on Walking Impairment Questionnaire (WIQ) From Baseline to 3 Months	12.5; 4.8	—
SECONDARY Change in Walking Distance Scores on Walking Impairment Questionnaire (WIQ) From Baseline to 6 Months	8.0; 3.7	—
SECONDARY Change in Walking Speed Score on Walking Impairment Questionnaire (WIQ) From Baseline to 3 Months	9.1; 7.5	—
SECONDARY Change in Walking Speed Score on Walking Impairment Questionnaire (WIQ) From Baseline to 6 Months	9.9; 8.5	—
SECONDARY Change in Stair Climbing Score on Walking Impairment Questionnaire (WIQ) From Baseline to 3 Months	8.5; 4.2	—
SECONDARY Change in Stair Climbing Score on Walking Impairment Questionnaire (WIQ) From Baseline to 6 Months	10.4; 6.6	—
SECONDARY Change in Score on Physical Composite Score (PCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 3 Months	4.1; 2.1	—
SECONDARY Change in Score on Physical Composite Score (PCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 6 Months	5.2; 3.1	—
SECONDARY Change in Score on Mental Composite Score (MCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 3 Months	0.7; 2.4	—
SECONDARY Change in Score on Mental Composite Score (MCS) Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 6 Months	-0.2; 1.1	—
SECONDARY Change in Score on Physical Functioning Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 3 Months	11.4; 4.8	—
SECONDARY Change in Score on Physical Functioning Subscale of the Short Form 36 Health Survey (SF-36) From Baseline to 6 Months	14.3; 8.1	—

Summary

Peripheral arterial disease is a common condition in older adults involving poor arterial circulation in the legs leading to leg pain and debility. The body's own circulating blood vessel stem cells may help to improve circulation. This study will test whether treatment with the drug granulocyte macrophage colony stimulating factor (GM-CSF) will improve symptoms and signs of peripheral arterial disease over placebo after four weeks of therapy. As well this study will examine whether improvements in blood vessel function can be observed. Finally, we will measure blood vessel function and stem cell levels in order to determine whether they can help to predict whether patients wither peripheral arterial disease will suffer further cardiovascular complications.

Eligibility Criteria

Inclusion Criteria

160 males or post-menopausal females between 21 and 80 years of age. Female subjects must be (a) post-menopausal, (b) surgically sterile or (c) use adequate birth control and have a negative pregnancy test within 3 days prior to administration of study drug and should not be breastfeeding.
Documented PAD (By Ankle-Brachial Indices or Angiographically)
Clinically stable (at least 2 months) history of intermittent claudication with no change in symptom severity in the 2 months prior to screening.
On stable statin therapy for previous 3 months.
Peak Walking Time (PWT) between 1 and 12 minutes on a standardized Gardner treadmill protocol.
A Doppler-derived ankle-brachial index (ABI) of 1.3 (non-compressible arteries) a Toe-Brachial Index (TBI) of 0.85 to 1.0 , and a reduction of 20% in ABI measured within 1 minute of treadmill testing.
On appropriate and stable medical therapy for atherosclerosis for at least 2 months.
Able to give informed consent.
Diabetics with a dilated eye exam excluding proliferative retinopathy in the previous 12 months.

Exclusion Criteria

Recent or current active infections (treated with antibiotics).
Recent (3 months) change in statin therapy
Critical limb ischemia either chronic (category 3 and 4 of SVS classification) or acute ischemia manifested by rest pain, ulceration, or gangrene.
Lower extremity vascular surgery, angioplasty or lumbar sympathectomy within 3 months of enrollment.
Participation in a structured exercise treatment protocol within 3 months of enrollment.
Prior myeloid cancer.
Unstable angina, myocardial infarction, TIA, stroke or revascularization in the preceding 4 months.
Severe heart failure (Class III or IV), heart muscle disease or atrial fibrillation.
Limitation on exercise for symptoms other than intermittent claudication such as arthritis or dyspnea.
Uncontrolled diabetes mellitus (defined as HbA1c > 10.0).
Chronic renal disease (creatinine of >2.5 mg/dl) or hepatic disease (> 3 X elevations in AST and ALT).
Ophthalmologic conditions associated with a neo-vascular response.
Alcohol or drug abuse, or any other disease process that, in the opinion of the PI, will interfere with the ability of the patient to participate in the study.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01041417). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.