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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 belowSelect...
I am between 18-35 years old and have Down syndrome.
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I have been diagnosed with Down syndrome.
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I have a caregiver who can be with me for at least 12 hours a week and come to my appointments.
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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.
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I have had my spleen removed or it does not work properly.
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My liver isn't working well, or my ALT levels are three times higher than normal.
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I have a history of blood clots in my veins or lungs.
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I regularly take an anti-cholinergic drug more than twice a week.
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I have had seizures, not including fever-related ones as a baby.
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I regularly take NSAIDs, but not more than 81 mg of aspirin daily.
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I have moderate to severe lung problems like COPD.
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I have heart problems that need treatment.
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I have difficult veins for drawing blood.
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I am currently taking medications that suppress my immune system.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ informed consent to follow-up visit (20 weeks)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~informed consent to follow-up visit (20 weeks)
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 moreTrial 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
1,806 Previous Clinical Trials
2,822,835 Total Patients Enrolled
8 Trials studying Down Syndrome
3,576 Patients Enrolled for Down Syndrome
National Institute on Aging (NIA)NIH
1,789 Previous Clinical Trials
28,188,290 Total Patients Enrolled
9 Trials studying Down Syndrome
1,505 Patients Enrolled for Down Syndrome
Peter Pressman, MDPrincipal InvestigatorCU Alzheimer's and Cognition Center
1 Previous Clinical Trials
42 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had seizures, not including fever-related ones as a baby.My liver isn't working well, or my ALT levels are three times higher than normal.I am between 18-35 years old and have Down syndrome.Your blood pressure is higher than 160 over 95 when checked before the study.My kidney function is impaired with high creatinine or low GFR.My medications have been the same for the last 30 days.I have had my spleen removed or it does not work properly.You have tested positive for hepatitis B, hepatitis C, HIV, or syphilis.I have been diagnosed with Down syndrome.I have a history of blood clots in my veins or lungs.I don't have any health issues that would affect the study or need treatments that could alter my immune system.You have signs of fluid buildup in your body before joining the study.I regularly take an anti-cholinergic drug more than twice a week.I haven't taken any experimental drugs recently.I can give my consent or have someone legally authorized to do so on my behalf.I can see and hear well enough to participate in the trial.I've been tested for hypothyroidism in the last 6 months and am stable on my medication.I regularly take NSAIDs, but not more than 81 mg of aspirin daily.I have a caregiver who can be with me for at least 12 hours a week and come to my appointments.I have moderate to severe lung problems like COPD.Your heart rate is less than 50 beats per minute when checked by the study doctor.Your heart's electrical activity stays elevated for too long, especially if you are a female with a QTc interval greater than 470 ms, or a male with a QTc interval greater than 450 ms.I have heart problems that need treatment.I have difficult veins for drawing blood.I haven't had cancer, except for non-dangerous skin cancer, in the last 5 years.You have had severe allergic reactions to drugs or ingredients in the study medication.I am not pregnant, breastfeeding, and use effective birth control or am surgically sterilized.I have not received a live vaccine in the last six weeks and do not plan to during the study.You have had an allergic reaction to latex or yeast in the past.I am currently taking medications that suppress my immune system.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo Control - Saline
- Group 2: Sargramostim
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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