Phase II Trial to Evaluate Safety and Efficacy of GM-CSF/Sargramostim in Alzheimer's Disease (SESAD)
This trial protocol is designed to evaluate primarily whether the long-term use of sargramostim (recombinant human GM-CSF), administered five days per week for six consecutive months (24 weeks), will be tolerated by and safe for use in participants with mild-to-moderate AD, secondarily whether sargramostim can slow, halt, or reverse cognitive decline, and exploratory whether sargramostim can slow, halt, or reverse decline in activities of daily living, reverse or improve several biomarkers associated with AD, as evaluated by multimodal neuroimaging techniques and blood and cerebrospinal fluid analyses. This trial extends the safety results from recently completed Phase 2 double-blind, placebo-controlled clinical trial in mild to moderate AD participants (NCT01409915, COMIRB#12-1273), using sargramostim that was administered five days per week for three consecutive weeks and in which there were no incidence of drug-related serious adverse events (SAEs). This concluded in December 2019, and although the trial only treated a small number of participants for a very short treatment phase and was not powered to adequately evaluate efficacy measures, an interim analysis has hinted at potential improvements in some secondary and exploratory efficacy measures. Thus, this 6-month trial protocol aims to evaluate sargramostim treatment in participants with mild-to-moderate AD over an extended period of time and determine whether sargramostim can indeed safely ameliorate cognitive decline and reverse pathological biomarkers of AD, in a similar fashion to our preclinical studies showing GM-CSF?s effects in transgenic animal models of AD.
Brain Imaging Center (BIC)
University of Colorado Hospital
Peter Pressman, MD
Protocol Number: 19-2727
More information available at ClinicalTrials.gov: NCT04902703
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