NCT05482334

Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome

Phase II Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: University of Colorado, Denver · Academic / Other
• Sex: All
• Age: 18 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This trial protocol is designed to evaluate primarily whether the use of sargramostim (recombinant human GM-CSF), administered five days per week for four consecutive weeks (20 treatment days), will be well tolerated by and safe for use in young adult participants with Down syndrome.

Detailed description

This trial protocol is designed to evaluate primarily whether the use of sargramostim (recombinant human GM-CSF), administered five days per week (250 μg/m2/day subcutaneously) for four consecutive weeks (20 treatment days), will be well tolerated by and safe for use in young adult participants (age 18-35) with Down Syndrome; Secondarily whether sargramostim will have an impact on cognition, and exploratory whether sargramostim has an impact upon activities of daily and quality of life, and impact upon several biomarkers associated with DS, as evaluated by multimodal neuroimaging techniques and blood analyses.

Conditions

• Down Syndrome

Interventions

Type	Name	Description
DRUG	Sargramostim for Injection	Recombinant human GM-CSF
DRUG	Saline Placebo	Bacteriostatic Saline

Timeline

• Start date: 2023-10-23
• Primary completion: 2026-09-01
• Completion: 2026-09-01
• First posted: 2022-08-01
• Last updated: 2025-04-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study