Could a common supplement ease severe behaviors in rare genetic disorder?
NCT ID NCT04381897
First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 1 time
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.
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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
Locations
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Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205, United States
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