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.
The purpose of this research study is to learn more about the safety and effectiveness of a drug called sargramostim for improving cognitive function and memory in people with Alzheimer’s disease.
Main Procedures Involved: These visits may include some of the following procedures: • Cognitive testing • Twice weekly blood draws • Heart Tracing (ECG) • Health Questionnaires • Study Partner Questionnaires • Lumbar Puncture (spinal tap) • MRI • PET scan • Treatment/placebo injection 5x a week during treatment phase If determined to be eligible during screening, you will be assigned to one of two arms of the study: 1) receives study medication; 2) receives placebo (a pill or a liquid that looks like medicine but is not real)
Duration of Participation: After initial screenings at the CU Anschutz campus in Aurora, CO, participants will be able to have weekly home nursing visits. Occasional visits to campus during the study will be required for procedures that cannot be done at home. Including screening and follow-up, study participation last up to 9-1/2 months with up to 55 visits. • Screening period will last up to 8 weeks • Treatment period will last 24 weeks • Follow-up visit occurs 45 days after end of treatment Compensation: study related care and medication at no cost.
Peter Pressman, MD
Protocol Number: 19-2727
More information available at ClinicalTrials.gov: NCT04902703
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