Sargramostim for Down Syndrome

Phase-Based Progress Estimates

Effectiveness

Safety

University of Colorado Anschutz Medical Campus, Aurora, CO

Down Syndrome+1 More

Sargramostim for Injection - Drug

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Eligibility

18 - 65

All Sexes

What conditions do you have?

Select

Eligibility

18 - 65

All Sexes

What conditions do you have?

Select

Study 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.

Eligible Conditions

Down Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Down Syndrome

Idursulfase-IT

Sargramostim

Inspire Upper Airway Stimulation (UAS) System

Study Objectives

1 Primary · 1 Secondary · Reporting Duration: Informed consent to Follow-up Visit (20 weeks)

Week 20

CANTAB - Cambridge Neuropsychological Test Automated Battery) (selected subtests)

Down syndrome Mental Status Exam (DSMSE)

KBIT II - Kaufman Brief Intelligence Test II

Leiter 3 International Performance Scale (selected subtests)

NEPSY II (selected subtest)

PPVT 5 - Peabody Picture Vocabulary Test, 5th edition

Patient-Reported Outcomes Measurement Information System (PROMIS) survey of quality of life

SB 5 - Stanford Binet Intelligence Scales 5th edition (selected subtest)

Vineland Adaptive Behavior Scales

Week 20

Safety as measured by number of Adverse Events (AEs) by body system

Trial Safety

Safety Progress

2 of 3

This is further along than 68% of similar trials

Other trials for Down Syndrome

Idursulfase-IT

Sargramostim

Inspire Upper Airway Stimulation (UAS) System

Trial Design

2 Treatment Groups

Sargramostim

1 of 2

Placebo Control - Saline

1 of 2

Experimental Treatment

Non-Treatment Group

42 Total Participants · 2 Treatment Groups

Primary Treatment: Sargramostim · Has Placebo Group · Phase 2

Sargramostim

Drug

Experimental Group · 1 Intervention: Sargramostim for Injection · Intervention Types: Drug

Placebo Control - Saline

Drug

PlaceboComparator Group · 1 Intervention: Saline Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline

Screening: ~3 weeks

Treatment: Varies

Reporting: informed consent to follow-up visit (20 weeks)

Closest Location: University of Colorado Anschutz Medical Campus · Aurora, CO

2015First Recorded Clinical Trial

1 TrialsResearching Down Syndrome

86 CompletedClinical Trials

Eligibility Criteria

Age 18 - 65 · All Participants · 9 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

You have a male or female with Down syndrome between 18-35 years of age.

You have a cytogenetic diagnosis of full trisomy 21 or complete unbalanced translocation of chromosome 21.

You are physically able to participate in the study.

You must reside within a proximity of the study site that will not preclude their regularly scheduled participation in the trial.

You are willing to be a surrogate mother for the duration of the study.

You are stable on all other medications for at least 30 days prior to initial screening visit.

About The Reviewer

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

References