Hope for teens with down syndrome regression disorder: old drugs, new purpose

NCT ID NCT05662228

Completed Disease control Sponsor: University of Colorado, Denver Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 14, 2026 · Updated 2 times

Summary

This Phase 2 trial tests three commonly used drugs—lorazepam, IVIG, and tofacitinib—in 66 people aged 8 to 30 with Down Syndrome Regression Disorder (DSRD). DSRD causes sudden severe symptoms like loss of speech, movement, and daily living skills. The study aims to see if these drugs are safe and can improve symptoms over 12 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: lorazepam, intravenous immunoglobulin (IVIG), and tofacitinib
What this could lead to: If successful, this could point toward effective treatments for Down Syndrome Regression Disorder, a rare condition causing severe behavioral and cognitive decline.
What could go wrong: This is a small Phase 2 trial with only 66 participants, so results may not apply broadly. The drugs have known side effects, and the study is not designed to prove long-term safety or efficacy.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for DOWN SYNDROME are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

autoimmune disease autoimmune disorder of the nervous system catatonia Down syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DOWN SYNDROME REGRESSION

Contacts and locations

Locations

Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.