Could a common supplement ease severe behaviors in rare genetic disorder?
NCT ID NCT04381897
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This pilot study tests whether N-acetylcysteine (NAC), an antioxidant supplement, can reduce repetitive and self-injurious behaviors in people with Cornelia de Lange syndrome (CdLS). Ten participants aged 13–35 will receive both NAC and a placebo in random order over 18 weeks. The goal is to see if NAC leads to a meaningful drop in symptom severity.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
N-acetylcysteine (NAC)
What this could lead to
If it works, this could point toward a safe, accessible treatment to reduce distressing repetitive and self-injurious behaviors in people with Cornelia de Lange syndrome.
What could go wrong
This is a very small pilot study with only 10 participants, so results may not apply broadly. NAC has shown mixed results in similar conditions, and the trial is not yet recruiting.
Disclaimer
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This is a summary of
the original study
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••