Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Mobilization of Progenitor Cells in Peripheral Arterial Disease (GPAD-2)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Arshed A. Quyyumi, Emory University
ClinicalTrials.gov Identifier:
NCT01041417
First received: December 29, 2009
Last updated: November 15, 2013
Last verified: November 2013
  Purpose

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.


Condition Intervention Phase
Peripheral Arterial Disease
Drug: Granulocyte-Macrophage Colony Stimulating Factor
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: Granulocyte-Macrophage Stimulating Factor (GM-CSF) and Mobilization of Progenitor Cells in Peripheral Arterial Disease: A Phase II Randomized Study

Resource links provided by NLM:


Further study details as provided by Emory University:

Primary Outcome Measures:
  • To investigate whether GM-CSF will improve symptoms of claudication in patients with PAD, measured objectively as improvement in treadmill exercise tolerance. [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in exercise tolerance will be due to improvement in collateral blood flow measured by ankle-brachial index, transcutaneous oxygen concentration, and calf blood flow measured by MRI, and/or improvement in endothelial dysfunction. [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 180
Study Start Date: September 2009
Estimated Study Completion Date: December 2013
Primary Completion Date: January 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GM-CSF Drug: Granulocyte-Macrophage Colony Stimulating Factor
80 subjects will be randomized to receive GM-CSF Monday, Wednesday and Friday for 4 weeks of therapy.
Other Name: GM-CSF, Leukine
Placebo Comparator: Placebo
80 patients will receive placebo on Monday, Wednesday, and Friday for 4 weeks.
Drug: Placebo
80 subjects will be randomized to receive placebo on Monday, Wednesday and Friday for 4 weeks.

  Eligibility

Ages Eligible for Study:   21 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 < 0.85 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 > 0.85 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01041417

Locations
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
Sponsors and Collaborators
Emory University
Investigators
Principal Investigator: Arshed Quyyumi, MD Emory University
  More Information

No publications provided by Emory University

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Arshed A. Quyyumi, Professor, Emory University
ClinicalTrials.gov Identifier: NCT01041417     History of Changes
Other Study ID Numbers: IRB00021976, NIH 1RC2HL101515-01
Study First Received: December 29, 2009
Last Updated: November 15, 2013
Health Authority: United States: Emory University Institutional Review Board

Keywords provided by Emory University:
claudication

Additional relevant MeSH terms:
Peripheral Arterial Disease
Peripheral Vascular Diseases
Atherosclerosis
Arteriosclerosis
Arterial Occlusive Diseases
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on September 29, 2014