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NCT04381897 · ClinicalTrials.gov registry record · Phase 2
Use of N-Acetylcysteine in the Treatment of Repetitive and Self-Injurious Behaviors in Cornelia de Lange Syndrome
A Phase 2 study, sponsored by Johns Hopkins University.
- Not yet
- Registry status
- Phase 2
- Development phase
- 10
- Enrollment target
NCT04381897: Clinical Trial Phase 2 study, sponsored by Johns Hopkins University.
NCT04381897 is a Phase 2 study that has not yet begun recruiting, run by Johns Hopkins University. The registered enrollment target is 10 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04381897, a Phase 2 study, has not yet begun recruiting, sponsored by Johns Hopkins University.
- NOT YET RECRUITING
- Registry status
- Phase 2
- Development phase
- 10 participants
- Enrollment target
Study Summary
This research project is a randomized cross-over pilot trial which aims to test the efficacy of N-acetylcysteine (NAC) for the treatment of Repetitive Behaviors (RB) and self-injurious behavior (SIB) in patients with Cornelia de Lange Syndrome (CdLs). NAC is a known anti-oxidative stress and neuroprotective agent, which has been shown to decrease the occurrence of SIB such as skin picking. NAC has also shown partial response in trials for compulsive behaviors in Obsessive Compulsive Disorder (OCD) and related disorders in autism. Cornelia de Lange syndrome (CdLS) is a genetic disorder with autistic features, including RBs and SIB. In this randomized clinical trial, participants with CdLS will be blindly assigned one of two possible treatment arms: 1) placebo (8 weeks) and NAC (8 weeks); or 2) NAC (8 weeks) and placebo (8 weeks), with an intermediate 2-week washout period.
Primary Outcome
CYBOCS-PDD scores range from a minimum of 0 to a maximum of 20, higher scores indicate greater symptom severity. Efficacy of the intervention would be assessed by ≥35% reduction in CYBOCS-PDD repetitive behaviors measure score.
Interventions
- OTHER Placebo
- DRUG N-acetyl cysteine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2026-08-01 |
| Est. Completion | 2027-05-01 |
| Phase | Phase 2 |
What is known before NCT04381897 opens enrollment
NCT04381897 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04381897 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04381897 about?
NCT04381897 is a clinical study titled "Use of N-Acetylcysteine in the Treatment of Repetitive and Self-Injurious Behaviors in Cornelia de Lange Syndrome". This research project is a randomized cross-over pilot trial which aims to test the efficacy of N-acetylcysteine (NAC) for the treatment of Repetitive Behaviors (RB) and self-injurious behavior (SIB) in patients with Cornelia de Lange Syndrome (CdLs). NAC is a known anti-oxidative stress and neurop...
What is the current status of trial NCT04381897?
This trial is currently not yet recruiting. It is a Phase 2 study. The enrollment target is 10 participants. The study started on 2026-08-01. Estimated completion is 2027-05-01.
What interventions are being tested in trial NCT04381897?
The interventions under investigation include: Placebo (OTHER), N-acetyl cysteine (DRUG).
Who is sponsoring clinical trial NCT04381897?
This trial is sponsored by Johns Hopkins University, which has 1,448 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04381897's enrollment target sits among peer trials
10 1968th of 2000 higher than 12 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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