← Back to Search

Cytokine

Sargramostim for Down Syndrome

Phase 2
Recruiting
Led By Peter Pressman, MD
Research Sponsored by University of Colorado, Denver
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Males or females with Down syndrome between 18-35 years of age
A cytogenetic diagnosis of full trisomy 21 or complete unbalanced translocation of chromosome 21
Must not have
Known renal dysfunction or serum creatinine greater than 150 micromoles/L, or Glomerular Filtration Rate (GFR) less than 55 ml/min
History of asplenia, hyposplenia, or splenectomy (for any indication)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up informed consent to follow-up visit (20 weeks)

Summary

This trial will test if a drug is safe and tolerated by young adults with Down Syndrome.

Who is the study for?
Adults with Down syndrome aged 18-35, who have a caregiver available for support, can participate in this trial. They must be healthy enough to join, not pregnant or planning pregnancy, and live close to the study site. Participants need recent thyroid checks and stable medication use.
What is being tested?
The trial is testing Sargramostim (GM-CSF), given five days a week for four weeks, against a saline placebo to see if it's safe and tolerable for young adults with Down syndrome.
What are the potential side effects?
Potential side effects of Sargramostim may include allergic reactions due to yeast-derived products or other components in the drug, fluid retention issues like swelling or weight gain, respiratory symptoms such as difficulty breathing, and blood pressure changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am between 18-35 years old and have Down syndrome.
Select...
I have been diagnosed with Down syndrome.
Select...
I have a caregiver who can be with me for at least 12 hours a week and come to my appointments.
Select...
I've been tested for hypothyroidism in the last 6 months and am stable on my medication.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My kidney function is impaired with high creatinine or low GFR.
Select...
I have had my spleen removed or it does not work properly.
Select...
My liver isn't working well, or my ALT levels are three times higher than normal.
Select...
I have a history of blood clots in my veins or lungs.
Select...
I regularly take an anti-cholinergic drug more than twice a week.
Select...
I have had seizures, not including fever-related ones as a baby.
Select...
I regularly take NSAIDs, but not more than 81 mg of aspirin daily.
Select...
I have moderate to severe lung problems like COPD.
Select...
I have heart problems that need treatment.
Select...
I have difficult veins for drawing blood.
Select...
I am currently taking medications that suppress my immune system.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~informed consent to follow-up visit (20 weeks)
This trial's timeline: 3 weeks for screening, Varies for treatment, and informed consent to follow-up visit (20 weeks) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Safety as measured by number of Adverse Events (AEs) by body system
Secondary study objectives
Down Syndrome
Other study objectives
CANTAB - Cambridge Neuropsychological Test Automated Battery) (selected subtests)
KBIT II - Kaufman Brief Intelligence Test II
Leiter 3 International Performance Scale (selected subtests)
+5 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: SargramostimExperimental Treatment1 Intervention
Sargramostim 250 μg/m2/day subcutaneously (5 days per week)
Group II: Placebo Control - SalinePlacebo Group1 Intervention
Placebo equivalent volume subcutaneously (5 days per week)

Find a Location

Who is running the clinical trial?

University of Colorado, DenverLead Sponsor
1,810 Previous Clinical Trials
2,920,058 Total Patients Enrolled
8 Trials studying Down Syndrome
3,576 Patients Enrolled for Down Syndrome
National Institute on Aging (NIA)NIH
1,802 Previous Clinical Trials
28,193,939 Total Patients Enrolled
10 Trials studying Down Syndrome
1,881 Patients Enrolled for Down Syndrome
Peter Pressman, MDPrincipal InvestigatorCU Alzheimer's and Cognition Center
1 Previous Clinical Trials
42 Total Patients Enrolled

Media Library

Sargramostim (Cytokine) Clinical Trial Eligibility Overview. Trial Name: NCT05482334 — Phase 2
Down Syndrome Research Study Groups: Placebo Control - Saline, Sargramostim
Down Syndrome Clinical Trial 2023: Sargramostim Highlights & Side Effects. Trial Name: NCT05482334 — Phase 2
Sargramostim (Cytokine) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05482334 — Phase 2
~25 spots leftby Sep 2026