Ignite Creation Date:
2025-12-25 @ 2:20 AM
Ignite Modification Date:
2025-12-27 @ 11:23 PM
Study NCT ID:
NCT05482334
Status:
WITHDRAWN
Last Update Posted:
2025-04-02
First Post:
2022-07-28
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome
Sponsor:
University of Colorado, Denver
Organization:
Study Overview
Official Title:
Phase II Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome
Status:
WITHDRAWN
Status Verified Date:
2025-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Difficulties enrolling participants due to study design
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 1R61AG074859-01 | NIH | None | https://reporter.nih.gov/quic… | View |