Using GM-CSF to treat Peripheral Artery Disease

Granulocyte-Macrophage Stimulating Factor (GM-CSF) in Peripheral Arterial Disease: The GPAD-3 Study

PHASE2 · Emory University · NCT03304821

This study is testing if a new treatment called GM-CSF can help people with peripheral artery disease feel better and improve their blood flow.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment176 (estimated)
Ages21 Years to 85 Years
SexAll
SponsorEmory University (other)
Locations1 site (Atlanta, Georgia)
Trial IDNCT03304821 on ClinicalTrials.gov

What this trial studies

This study investigates the effects of granulocyte-macrophage colony stimulating factor (GM-CSF) on patients with peripheral artery disease (PAD) and intermittent claudication. Participants will be randomly assigned to receive either GM-CSF or a placebo through subcutaneous injections three times a week for three weeks. The study aims to determine if GM-CSF can improve symptoms and blood flow in individuals suffering from PAD. After a four-week screening phase, participants will undergo baseline testing and follow-up assessments to evaluate the treatment's effectiveness.

Who should consider this trial

Good fit: Ideal candidates include post-menopausal women or surgically sterile females with documented symptomatic PAD and a history of intermittent claudication.

Not a fit: Patients with non-compressible arteries or those who are not clinically stable may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve symptoms and quality of life for patients with peripheral artery disease.

How similar studies have performed: Previous studies have shown improvements in claudication symptoms with GM-CSF, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* 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 symptomatic PAD
* Clinically stable (at least 2 months prior to enrollment) history of intermittent claudication or walking impairment (Rutherford Class II) with no change in symptom severity in the 2 months prior to screening.
* On statin therapy for previous 3 months prior to enrollment, unless statin intolerant.
* Peak Walking Time (PWT) between 1 and 12 minutes on a standardized Gardner treadmill protocol or modified Gardner protocol or less than 12 minutes on a modified Bruce protocol in case PWT on Gardner protocol is more than 12 minutes.
* A Doppler-derived ankle-brachial index (ABI) of \< 0.90 in the symptomatic limb after 10 minutes of rest at screening. For subjects with an ABI of \>1.3 (non-compressible arteries) a Toe-Brachial Index (TBI) of \< 0.70 must be obtained for subject qualification, or if ABI is \> 0.9 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 prior to enrollment.
* Able to give informed consent.
* Diabetics with a dilated eye exam excluding proliferative retinopathy in the previous 12 months prior to enrollment.

Exclusion Criteria:

* Recent or current active infections (treated with antibiotics)
* Recent (6 months prior to randomization) or current active cancer undergoing treatment
* Recent (3 months prior to randomization) change in statin or cilostazol therapy
* Critical limb ischemia either chronic (Rutherford Class \>II) or acute ischemia manifested by rest pain, ulceration, or gangrene
* Recent (3 months prior to randomization) lower extremity vascular surgery, angioplasty or lumbar sympathectomy
* Planned participation in a structured exercise treatment protocol in the future or within period of study
* Prior myeloid malignancy
* Recent (3 months prior to randomization) Unstable angina, myocardial infarction, transient ischemic attack (TIA), stroke or revascularization
* Severe heart failure (Class III or IV) or heart muscle disease
* Limitation on exercise for symptoms other than intermittent claudication such as arthritis or dyspnea
* Below- or above-knee amputation; wheelchair confinement
* Use of a walking aid other than a cane
* Walking impairment for reasons other than PAD e.g. Parkinson's disease
* Uncontrolled diabetes mellitus (defined as HbA1c \> 10.0)
* Chronic renal disease (creatinine of \>2.5 mg/dl) or hepatic disease (\> 3 X elevations in aspartate aminotransferase (AST) and alanine aminotransferase (ALT))
* White blood cell count \< 3k/cmm
* Hemoglobin (HGB) \< 10g/dL
* Blood Pressure Systolic \>180 and/or Diastolic \>100
* Taking Immunosuppressant drugs
* 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
* Inability to attend study visits

Where this trial is running

Atlanta, Georgia

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Peripheral Artery Disease, Atherosclerotic peripheral artery disease, Granulocyte-macrophage colony stimulating factor, Atherosclerosis, Cardiology, Vascular disease

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.