Journal
CONTEMPORARY CLINICAL TRIALS
Volume 91, Issue -, Pages -Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.cct.2020.105975
Keywords
Peripheral artery disease; GM-CSF; Angiogenesis; Claudication
Funding
- National Heart, Lung, and Blood Institute of the National Institutes of Health [1R61HL138657-01, 4R33HL138657-02, 5R33HL138657-03]
- Abraham J. & Phyllis Katz Foundation (Atlanta, GA)
- American Heart Association [15SFCRN23910003, 19POST34400057]
- NIH [1P20HL113451-01, 1P30DK111024-03S1, 5R01HL095479-08, 3RF1AG051633-01S2, 5R01AG042127-06, 2P01HL086773-08, U54AG062334-01, 1R01HL141205-01, 5P01HL101398-02, 5P01HL086773-09 1RF1AG051633-01, R01 NS064162-01, R01 HL89650-01, HL095479-01, 1DP3DK094346-01, 2P01HL086773]
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Background: Lower extremity peripheral arterial disease (PAD) is a public health problem and many patients with PAD experience claudication despite adequate medical and/or surgical management. Mobilization of endogenous progenitor cells using Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) is a novel therapeutic option that has shown promising results in experimental models and phase I/IIA clinical trials. The GPAD-3 trial will study the effect of two successive administrations of GM-CSF at 3-month interval for improving claudication among patients with lower extremity PAD. Methods: We plan to recruit 176 patients in this ongoing randomized, double-blind, placebo-controlled Phase IIB trial. After screening for inclusion and exclusion criteria, eligible subjects undergo a 4-week screening phase where they perform subcutaneous placebo injections thrice weekly and walk at least three times a day until they develop claudication. After the screening phase, eligible subjects undergo baseline testing and are randomized 2:1 to receive 500 mu g/day of GM-CSF subcutaneously thrice weekly for three weeks or placebo injections. After 3 months, follow-up endpoint testing is performed and subjects in the GM-CSF group receive the second administration of the drug for three weeks while subjects in placebo group receive matching placebo injections. All participants undergo endpoint testing at six-month and nine-month follow-up. The primary endpoint is change in 6-min walk distance between baseline and 6-month follow-up. Conclusion: GPAD-3 explores a novel approach to address the need for alternative therapies that can alleviate symptoms among patients with lower extremity PAD. If successful, this study will pave the way for a pivotal Phase III trial.
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