Hope for teens with down syndrome regression disorder: old drugs, new purpose
NCT ID NCT05662228
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States